1. Third quarter 2020 total revenue of $81.5 million and Crysvita1revenue of $37.3 million

    Increased lower end of Crysvita1guidance range; updated full year range is $130 million to $140 million

    Dojolvi launch off to a strong start

    NOVATO, Calif., Oct. 27, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended September 30, 2020 and updated its full year 2020 guidance.

    "Crysvita continues to deliver meaningful revenue growth and with the launches of Dojolvi and Crysvita for TIO, we are now generating sales from three products…

    Third quarter 2020 total revenue of $81.5 million and Crysvita1 revenue of $37.3 million

    Increased lower end of Crysvita1 guidance range; updated full year range is $130 million to $140 million

    Dojolvi launch off to a strong start

    NOVATO, Calif., Oct. 27, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended September 30, 2020 and updated its full year 2020 guidance.

    "Crysvita continues to deliver meaningful revenue growth and with the launches of Dojolvi and Crysvita for TIO, we are now generating sales from three products across four indications," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "The Angelman program with GeneTx recently read out very promising, early data for this neurological disorder demonstrating the potential to substantially improve clinical symptoms in that disease. The strategic collaboration with Solid Biosciences we recently announced adds an important potential Duchenne therapy to our preclinical pipeline and a sixth program to our strong gene therapy franchise."

    Third Quarter 2020 Financial Results

    Net Revenues

    For the third quarter of 2020, Ultragenyx reported $81.5 million in total revenue. Ultragenyx recognized $37.3 million in total Crysvita revenue in the Ultragenyx territories, which includes $34.1 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $3.3 million. Total royalty revenue related to European Crysvita sales were $3.3 million. Mepsevii® (vestronidase alfa) product sales for the third quarter of 2020 was $4.1 million. Dojolvi (triheptanoin) product sales in the initial quarter of our commercial launch were $3.9 million. Total revenue for the third quarter also includes $32.9 million of revenue related to the collaboration and license agreement with Daiichi Sankyo that was executed in March 2020.

    Revenue for the nine months ended September 30, 2020 was $179.5 million, including $98.5 million in total Crysvita revenue in the Ultragenyx territories. Crysvita collaboration revenue in the North American profit share territory was $91.1 million and net Crysvita product sales in other regions were $7.4 million. Total royalty revenue related to European Crysvita royalties was $10.9 million, which includes $1.5 million recognized on sales that occurred prior to January 1, 2020. Mepsevii product revenue for the nine months ended September 30, 2020 was $11.7 million. Dojolvi product revenue was $6.6 million. The technology transfer services with Daiichi Sankyo were initiated during the second quarter of 2020. For the nine months ended September 30, 2020, revenue related to this agreement was $51.7 million.

    Operating Expenses

    Total operating expenses for the third quarter of 2020 were $131.8 million, including non-cash stock-based compensation of $20.3 million. This compares to total operating expenses for the third quarter of 2019, which were $143.8 million and total non-cash stock-based compensation of $19.9 million.

    Total operating expenses for the nine months ended September 30, 2020 were $413.5 million, which includes $25.0 million to maintain the option to acquire GeneTx, $7.0 million to license certain vectors from REGENXBIO, and non-cash stock-based compensation of $62.9 million. This is compared with $397.8 million for the same period in 2019, which includes a $15.6 million research and development expense from the Arcturus collaboration amendment and non-cash stock-based compensation of $62.3 million. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows, and advances its portfolio.

    For the third quarter of 2020, Ultragenyx reported net loss of $68.8 million, or $1.13 per share basic and diluted, compared with a net loss for the third quarter of 2019 of $113.0 million, or $1.96 per share, basic and diluted. For the nine months ended September 30, 2020, net loss was $162.6 million, or $2.73 per share, basic and diluted, compared with a net loss for the same period in 2019 of $308.9 million, or $5.50 per share, basic and diluted. The net loss for the third quarter of 2020 and the net loss for the nine months ended September 30, 2020 includes an $11.5 million unrealized loss and a $91.3 million unrealized gain for the three and nine months ended September 30, 2020, respectively, from the fair value adjustment on the investment in Arcturus equity. Net cash used in operations for the first nine months of 2020 was $69.8 million, compared to net cash used of $273.3 million for the same period in 2019.

    Cash, Cash Equivalents and Investments

    Cash, cash equivalents, and investments were $765.5 million as of September 30, 2020.

    2020 Financial Guidance

    Crysvita Guidance in Ultragenyx Territories1

    The lower end of the range for 2020 Crysvita revenue in Ultragenyx territories1 has been increased to $130.0 million from $125.0 million. The updated guidance range is $130.0 million to $140.0 million.

    Program Updates and Upcoming Milestones

    Dojolvi for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD): Launched on July 22, 2020

    • The U.S. FDA approved Dojolvi for the treatment of pediatric and adult patients for all forms of LC-FAOD with a molecularly-confirmed diagnosis. Dojolvi is the first FDA-approved therapy for these lifelong and life-threatening genetic disorders and is now available to patients in the U.S.
    • Dojolvi has been submitted for approval with ANVISA in Brazil and has been submitted to Health Canada after being granted priority review. Discussions with EU regulators are ongoing. Requests for named patient access will continue to be supported during the ongoing discussions and review in these countries.

    GTX-102 for Angelman Syndrome (AS) Conducted by Partner, GeneTx: Positive interim Phase 1/2 data demonstrating substantial improvements in five out of five patients treated

    • Preliminary results from the first five patients treated indicate substantial improvements in all patients in at least two disease domains including communication, behavior, sleep, gross motor function, and fine motor function as measured by the Clinical Global Impression of Improvement Scale for Angelman Syndrome.
    • After reaching substantially higher doses as planned per protocol, all five patients experienced a serious adverse event (SAE) of lower extremity weakness believed to be related to local inflammation due to GTX-102. Following these events, the companies paused enrollment and dosing. These serious events were assessed as mild or moderate in severity. Four out of five patients have fully recovered with the fifth nearly fully recovered.
    • The study protocol will be amended to reduce the dose-level range and modify the administration process, which is expected to reduce further drug-related SAEs. The companies will obtain agreement on these modifications with the US FDA prior to resuming enrollment and dosing.

    DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Prophylactic steroid cohort is ongoing

    • A fourth cohort of three patients at the same cohort 3 dose of 1.0 × 10^13 GC/kg is ongoing using prophylactic steroids. Data from this cohort are expected by the end of 2020. Phase 3 initiation is expected in 2021.

    DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Prophylactic steroid cohort has been added, no impact to timing of Phase 3 study

    • A fourth cohort of three patients is ongoing at 6.0 x 10^12 GC/kg, the same dose as cohorts two and three, with a prophylactic steroid regimen. Data from this cohort are not expected to impact the timing of a potential Phase 3 study. Phase 3 initiation is expected in the first half of 2021.

    Crysvita for X-Linked Hypophosphatemia (XLH): Approved by European Commission (EC) for treatment of XLH in older adolescents and adults

    • The European Commission (EC) approved Crysvita for the treatment of XLH in older adolescents and adults. This expanded approval adds to the prior indication, which included children and adolescents with growing skeletons, to now include adolescents with radiographic evidence of bone disease, regardless of growth status, as well as adults with XLH are now also eligible for treatment with Crysvita.

    Crysvita for Tumor-Induced Osteomalacia (TIO): Launched in the United States

    • The US FDA approved a second indication for Crysvita in June 2020. Crysvita is now available for the treatment of fibroblast growth factor 23 (FGF23)-related hypophosphatemia in TIO associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older.

    Business Development Update

    Pre-clinical Gene Therapy: Strategic Collaboration with Solid Biosciences to Develop and Commercialize New Gene Therapies for Duchenne Muscular Dystrophy

    • Strategic collaboration combines Solid's differentiated microdystrophin construct and Ultragenyx's HeLa PCL manufacturing platform for use with AAV8 and variants. Under the terms of the collaboration, Ultragenyx was granted an exclusive license for the use of Solid's proprietary microdystrophin construct with AAV8 and variants. Ultragenyx has made a $40 million investment in Solid and has agreed to pay up to $255 million in cumulative milestone payments per product upon achievement of specified milestone events, and tiered royalties on worldwide net sales at low double digit to mid-teens percentages.

    Corporate Update

    Executive Team Hires

    • Ernie Meyer was hired as Chief Human Resources Officer. In this newly created role, Mr. Meyer leads all aspects of the Global Human Resources and Facilities operations and serves on the Executive Leadership Team.
    • Mardi Dier was hired as Chief Financial Officer, following the planned transition of Shalini Sharp. In this role, Ms. Dier is responsible for leading the corporate finance, strategy, investor relations, corporate communications, and information technology functions, and serves on the Executive Leadership Team.

    1: Ultragenyx territories include the collaboration revenue from the North American profit share territory (U.S. and Canada) and other regions where revenue from product sales are recognized by Ultragenyx (Latin America, Turkey). This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.

    Conference Call and Webcast Information

    Ultragenyx will host a conference call today, Tuesday, October 27, 2020, at 2 p.m. PT/ 5 p.m. ET to discuss the third quarter 2020 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 4067577. The replay of the call will be available for one year.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the effects from the COVID-19 pandemic on the company's commercialization activities, business and operating results, smaller than anticipated market opportunities for the company's products and product candidates, manufacturing risks, competition from other therapies or products, uncertainties related to insurance coverage and reimbursement status of the company's newly approved products, the company's evolving integrated commercial organization, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on July 30, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Joshua Higa

    (415) 475-6370



    Ultragenyx Pharmaceutical Inc.
    Selected Statement of Operations Financial Data
    (in thousands, except share and per share amounts)
    (unaudited)
            
     Three Months Ended September 30,  Nine Months Ended September 30,
     2020 2019 2020 2019
    Statement of Operations Data:       
    Revenues:       
    Collaboration and license$66,924  $21,585  $144,300  $55,070 
    Product sales 11,215   4,215   25,760   13,051 
    Non-cash collaboration royalty revenue 3,331      9,428    
    Total revenues 81,470   25,800   179,488   68,121 
    Operating expenses:       
    Cost of sales 2,348   2,683   648   3,901 
    Research and development 87,314   100,144   280,984   274,294 
    Selling, general and administrative 42,123   41,006   131,891   119,647 
    Total operating expenses 131,785   143,833   413,523   397,842 
    Loss from operations (50,315)  (118,033)  (234,035)  (329,721)
    Change in fair value of investments in equity securities (11,520)  2,166   91,348   11,994 
    Non-cash interest expense on liability related to the sale of future royalties (8,582)     (25,093)   
    Other income, net 1,885   3,166   6,362   9,527 
    Loss before income taxes (68,532)  (112,701)  (161,418)  (308,200)
    Provision for income taxes (313)  (293)  (1,137)  (722)
    Net loss$(68,845) $(112,994) $(162,555) $(308,922)
    Net loss per share, basic and diluted$(1.13) $(1.96) $(2.73) $(5.50)
    Weighted-average shares used in computing net loss per share, basic and diluted 60,687,177   57,707,694   59,564,163   56,161,996 
            



    Ultragenyx Pharmaceutical Inc.
    Selected Balance Sheet Financial Data
    (in thousands)
    (unaudited)
      September 30, December 31,
      2020 2019
    Balance Sheet Data:    
    Cash, cash equivalents, and available-for-sale investments$765,520 $760,404
    Working capital  670,817  747,717
    Total assets  1,268,385  1,135,496
    Total stockholders' equity  666,191  653,764

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  2. Efficacy in multiple functional measures observed in all five patients treated

    Highest doses associated with a significant but reversible safety issue

    Study amendment planned to focus on lower end of dose range

    Conference call to discuss results planned for 5pm Eastern Time

    SARASOTA, Fla. and NOVATO, Calif., Oct. 26, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced positive interim data from the Phase 1/2 study of GTX-102. Preliminary results from the first five patients treated indicate substantial improvements in all patients in at least…

    Efficacy in multiple functional measures observed in all five patients treated

    Highest doses associated with a significant but reversible safety issue

    Study amendment planned to focus on lower end of dose range

    Conference call to discuss results planned for 5pm Eastern Time

    SARASOTA, Fla. and NOVATO, Calif., Oct. 26, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), companies partnered in the development of intrathecally administered GTX-102, an investigational treatment for Angelman syndrome, today announced positive interim data from the Phase 1/2 study of GTX-102. Preliminary results from the first five patients treated indicate substantial improvements in all patients in at least two disease domains including communication, behavior, sleep, gross motor function, and fine motor function as measured by the Clinical Global Impression of Improvement Scale for Angelman Syndrome (CGI-I-AS) at day 128. At the highest doses, all five patients experienced a serious adverse event (SAE) of lower extremity weakness believed to be related to local inflammation due to GTX-102. Following these events, the companies paused enrollment and dosing. These SAEs were assessed as mild or moderate in severity and have generally improved over a period of a few weeks while disease domain improvements have been sustained for three months. The study protocol will be amended to reduce the dose-level range and modify the administration process, which is expected to reduce further drug-related SAEs.

    "The work that the GeneTx team and Dr. Scott Dindot and his lab have conducted over these last years provided the opportunity to impact Angelman syndrome in a fundamental manner with a potent antisense oligonucleotide," noted Emil D. Kakkis, M.D., Ph.D., CEO and President of Ultragenyx. "These initial GTX-102 findings raise the possibility of improving some of the significant symptoms of Angelman syndrome."

    "The UBE3A antisense transcript targeted by GTX-102 is a viable target for treatment," stated Scott Stromatt, M.D., Chief Medical Officer of GeneTx. "The results observed to date are encouraging and we look forward to resuming dosing at lower doses to help avoid side effects."

    "So far we are seeing rapid improvements in multiple areas, including some kids doing things they've never done before, and I don't believe this rate of progress in development skills has been seen before in Angelman syndrome," commented Elizabeth M. Berry-Kravis, M.D., Ph.D., Professor of Pediatrics, Neurological Sciences and Biochemistry, Rush University Medical Center, and investigator in the GTX-102 clinical study. "It is especially amazing that families are asking me repeatedly when they can start treatment again despite the side effects that their child experienced. That speaks to the value of what they were seeing in their child."

    Interim Efficacy Results

    The study design includes five dosing cohorts in which patients were to receive four monthly doses of GTX-102 on an intra-patient dose escalation scheme. Five patients between the ages of 5 and 15 with deletions in the maternal UBE3A gene region were enrolled in the first three cohorts and are included in the interim data analysis.

    Initial indications of benefit have been observed in all five treated patients across the key domains of communication, fine and gross motor skills, behavior, and sleep as measured by the CGI-I-AS. In some patients these initial indications of clinical improvement were observed by the investigator early in the study at the two lowest dose levels and began within weeks of the first dose.

    At day 128, all patients had a meaningful improvement in their individual global CGI-I-AS score, which evaluated overall improvement across five domains specific to the symptoms of Angelman syndrome. All five patients were assessed as ‘much improved' or ‘very much improved' on the 7-point global scale of -3 to +3 with a mean change of +2.4. All patients had at least two symptom domains that were assessed as ‘very much improved' or ‘much improved' and at least three domains that were ‘minimally improved' or better (Score of 3, 2, or 1).

    Patient-by-patient CGI-I-AS results by domain and overall are as follows:

    PatientOverall Global

    Rating
    SleepBehaviorCommunicationGross MotorFine Motor
    001+2

    Much Improved
    0+1+2+20
    002+3

    Very Much Improved
    0+1+3-2*+3
    003+2

    Much Improved
    +2+2+1-1*0
    004+2

    Much Improved
    0+3+2-1*+2
    005+3

    Very Much Improved
    +3+1+3+2+2

    *Patients 002, 003, and 004 had gross motor impairment at time of assessment due to the ongoing SAE

    Note: CGI-I-AS scale ratings: +3: very much improved, +2: much improved, +1: minimally improved, 0: no change,

    -1: minimally worse, -2: much worse, -3: very much worse.

    Data as of day 128, except day 86 for patient 005.

    Supporting the CGI-I-AS improvements were changes in other domain-specific measurable endpoints. All patients experienced numerical increases in the sub-scale growth scores of expressive and/or receptive communication of the Bayley Scales of Infant and Toddler Development (Bayley-4) domains, and three patients showed improvements in the Observed Reported Communication Ability (ORCA) measure of expressive, receptive, and pragmatic communication.

    The interim analysis did not include data from other exploratory outcome measures such as seizure frequency, sleep diaries, EEG patterns, UBE3A protein levels in the CSF, ambulation by wearable device, and adaptive behaviors.

    Caregiver reports, via functional domain questionnaire, of improvement in the patients also support the changes seen in CGI-I-AS and the other endpoints. Notable caregiver-reported changes include:

    • Acquisition of spoken words, signs and gestures, and augmentative and alternative communication abilities; two previously nonverbal patients began using words, one reaching nine words
    • Ability to respond to their name, follow commands, and focus on tasks
    • Acquisition of independent capabilities, such as self-feeding with a fork
    • Increased abilities in physical activities, such as patients swimming on own and catching/throwing a ball
    • Dramatically improved sleep
    • Decreased maladaptive behaviors
    • Increased social engagement
    • Improved gait and posture

    Interim Safety Results

    Significant but reversible lower extremity weakness has been observed as a grade 1 or 2 SAE in four patients after administration of the highest dose, which was approximately 10 times higher than the initial low dose of cohort 1, and in one patient after administration of a single dose at the second highest dose level assessed, which was approximately 6 times higher than the initial low dose of cohort 1. The SAE was not observed after the two lower starting doses in the first four patients.

    The onset began between approximately one to four weeks after the last dose. In two patients the lower extremity weakness progressed to an inability to walk or bear weight. No patients experienced upper extremity weakness. The companies paused all dosing beginning at the time of the first SAE, and patients were treated with intravenous immunoglobulin (IVIg) and corticosteroids. The most severe aspects of the neurologic findings steadily and substantially resolved within a few weeks of the dosing pause and implementation of treatment. The adverse event has completely resolved in four patients (occurring between 19 and 70 days from onset), while the findings in the one remaining patient substantially improved by three to four weeks and is now almost fully resolved.

    The lower extremity weakness was associated with an elevation of cerebrospinal fluid (CSF) protein, which has been reported in studies of other intrathecally administered antisense oligonucleotides. Magnetic resonance imaging (MRI) shows findings consistent with local inflammation in the meninges and nerve roots in the region of intrathecal administration in the lower back (lumbosacral region) at the higher doses of GTX-102. The lower extremity SAE was not observed at lower doses and the delayed events of lower extremity weakness were not observed in preclinical GLP toxicity studies at similar and higher doses.

    There have been no other SAEs reported to date with GTX-102. Other adverse events have included transient ataxia, headache, and fatigue. The acute, transient ataxia was reported in all five patients, occurred two to six hours after the intrathecal injection, lasted for 24 to 72 hours, and was dose dependent. No patients have withdrawn from the study.

    Study Next Steps

    The companies paused dosing and enrollment in the study after first observing the lower extremity SAE to evaluate the event, assess efficacy, and monitor recovery. After review of all findings and nonclinical evaluations, the study protocol will be amended to try to reduce exposure to GTX-102 at the point of local contact during intrathecal administration. The dose will be lowered to the observed safe range at which clinical improvement was first observed and a slower titration regimen will be implemented that is dependent on individual patient response and age. The administration method will be modified to help reduce local drug contact time. The companies will obtain agreement on these modifications with the U.S. Food and Drug Administration (FDA) prior to resuming enrollment and dosing. Further detail from the first five patients will be presented at the Foundation for Angelman Syndrome Therapeutics (FAST) Global Summit in December 2020. Additional safety and efficacy data from the study are expected in 2021.

    Investor Conference Call and Webcast Information

    Ultragenyx will host an investor conference call today, Monday, October 26, 2020, at 2 p.m. PT / 5 p.m. ET to discuss the results. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events.cfm. To participate on the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 5684773. The replay of the call will be available for one year.

    About Angelman Syndrome

    Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the intended target of GTX-102. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is typically not inherited but instead occurs spontaneously. It is estimated to affect 1 in 12,000 to 1 in 20,000 people globally.

    Individuals with Angelman syndrome have developmental delay, balance issues, motor impairment, and debilitating seizures. Some individuals with Angelman syndrome are unable to walk and most do not speak. Anxiety and disturbed sleep can be serious challenges in individuals with Angelman syndrome. While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome suggesting that improvement of symptoms can potentially be achieved at any age.

    About GTX-102

    GTX-102 is an investigational antisense oligonucleotide designed to target and inhibit expression of UBE3A-AS. Nonclinical studies show that GTX-102 reduces the levels of UBE3A-AS and reactivates expression of the paternal UBE3A allele in neurons of the CNS. Reactivation of paternal UBE3A expression in animal models of Angelman syndrome has been associated with improvements in some of the neurological symptoms associated with the condition. GTX-102 has been granted Orphan Drug Designation, Rare Pediatric Disease Designation, and Fast Track Designation from the U.S. Food and Drug Administration (FDA). In August 2019, GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx.

    About GeneTx Biotherapeutics

    GeneTx Biotherapeutics LLC is a startup biotechnology company singularly focused on developing and commercializing a safe and effective antisense therapeutic for the treatment of Angelman syndrome. GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research. GeneTx licensed the rights to antisense technology intellectual property from the Texas A&M University System in December 2017.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its business plans and objectives for GTX-102, the therapeutic potential and clinical benefits of GTX-102, expectations regarding the safety and tolerability of GTX-102, and future clinical developments for GTX-102 are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the Company's ability to successfully develop GTX-102 at lower doses, including the resolution of adverse events that were seen at higher doses, whether lower doses of GTX-102 are sufficiently effective to support the continued development of the program, the effects from the COVID-19 pandemic on the company's commercialization activities, business and operating results, smaller than anticipated market opportunities for the company's products and product candidates, manufacturing risks, competition from other therapies or products, uncertainties related to insurance coverage and reimbursement status of the company's newly approved products, the company's evolving integrated commercial organization, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance, the timing of clinical trial activities  and reporting results from same, and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on July 30, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts

    Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Joshua Higa

    415-660-0951

    GeneTx

    Paula Evans

    630-639-7271

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  3. -Collaboration combines Solid’s differentiated microdystrophin construct and Ultragenyx’s HeLa PCL manufacturing platform for use with AAV8 and variants-

    -Solid receives $40 million upfront via equity investment at a premium; up to $255 million in milestones plus royalty payments-

    -Solid retains exclusive rights to all other uses of its microdystrophins, including its existing SGT-001 program-

    NOVATO, Calif. and CAMBRIDGE, Mass., Oct. 23, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, and Solid Biosciences Inc. (NASDAQ:SLDB), a life sciences company focused on advancing meaningful…

    -Collaboration combines Solid’s differentiated microdystrophin construct and Ultragenyx’s HeLa PCL manufacturing platform for use with AAV8 and variants-

    -Solid receives $40 million upfront via equity investment at a premium; up to $255 million in milestones plus royalty payments-

    -Solid retains exclusive rights to all other uses of its microdystrophins, including its existing SGT-001 program-

    NOVATO, Calif. and CAMBRIDGE, Mass., Oct. 23, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, and Solid Biosciences Inc. (NASDAQ:SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced a strategic collaboration and license agreement to focus on the development and commercialization of new gene therapies for Duchenne. The parties will collaborate to develop products that combine Solid’s differentiated microdystrophin construct, Ultragenyx’s HeLa producer cell line (PCL) manufacturing platform, and AAV8 variants. The collaboration also brings together Solid’s expertise in muscle biology and Ultragenyx’s expertise in bringing novel therapies to patients with rare diseases.

    Under the terms of the collaboration, Solid granted Ultragenyx an exclusive license for any pharmaceutical product that expresses Solid’s proprietary microdystrophin construct from AAV8 and variants thereof in clade E for use in the treatment of Duchenne and other diseases resulting from lack of functional dystrophin, including Becker muscular dystrophy. Ultragenyx has made a $40 million investment in Solid and has agreed to pay up to $255 million in cumulative milestone payments per product upon achievement of specified milestone events, and tiered royalties on worldwide net sales at low double digit to mid-teens percentages. Upon achievement of proof-of-concept, Solid has the right to opt-in to co-fund collaboration programs in return for participation in a profit share or increased royalty payments.

    "We believe that Solid’s microdystrophin is best-in-class with its unique neuronal nitric oxide synthase binding domain," said Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx. "By using an AAV8 variant validated in prior human and other studies combined with our scalable, efficient HeLa producer cell line platform, we believe we can leverage our mutual strengths to develop a high-quality AAV-based treatment alternative for Duchenne."

    "Ultragenyx has a demonstrated track record of success in developing and commercializing innovative therapies for rare diseases," said Ilan Ganot, Co-Founder, President and Chief Executive Officer at Solid Biosciences. "We believe it is the partner of choice for exploring new gene therapy opportunities for patients with Duchenne."

    Solid’s proprietary microdystrophin construct has exhibited functional benefit in preclinical models. In preclinical studies, animals expressing a microdystrophin capable of restoring neuronal nitric oxide synthase (nNOS) resisted fatigue better than those expressing a microdystrophin that does not. Patients dosed with Solid’s proprietary microdystrophin construct at the 2E14 vg/kg dose in Solid’s ongoing IGNITE DMD clinical trial have also preliminarily demonstrated nNOS activity and function, further validating these preclinical results. Solid expects to dose the next patient in the IGNITE DMD clinical trial, using SGT-001 produced using its improved HSV manufacturing process, in the first quarter of 2021.

    Ultragenyx intends to use its AAV-based HeLa PCL platform including HeLa 3.0 improvements for the development of product candidates. The platform enables large 2,000 liter commercial-scale AAV-based gene therapy product manufacturing. The PCL platform yields high-quality product from a highly reproducible, highly scalable, and less expensive process â€" a distinct vantage in higher dose indications like Duchenne. The capsid planned is an AAV8 variant with a favorable immunological profile that has been used successfully in the large scale 2,000 liter production process.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency and ensuring majority access to its therapies for patients who can benefit.

    Ultragenyx currently has three AAV gene therapies in clinical development, including DTX201 that uses an AAV8 variant in the HeLa PCL platform and that is partnered with Bayer, who has released positive Phase 1/2 data in Hemophilia A. The company’s other clinical AAV8 gene therapies, DTX301 and DTX401, are in Phase 1/2 studies for ornithine transcarbamylase (OTC) deficiency and glycogen storage disease type Ia (GSDIa), respectively. An investigational new drug (IND) application is expected by the end of 2020 for a fourth AAV gene therapy for Wilson disease, which will also use the HeLa PCL AAV manufacturing platform.

    For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com.

    About Solid Biosciences

    Solid Biosciences is a life sciences company focused on advancing transformative treatments to improve the lives of patients living with Duchenne. Disease-focused and founded by a family directly impacted by Duchenne, our mandate is simple yet comprehensive â€" work to address the disease at its core by correcting the underlying mutation that causes Duchenne with our lead gene therapy candidate, SGT-001.

    Solid’s SGT-001 is a novel adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne. Duchenne is caused by mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is a systemically administered candidate that delivers a synthetic dystrophin gene, called microdystrophin, to the body. This microdystrophin encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including nNOS. Data from Solid’s preclinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.

    SGT-001 is based on pioneering research in dystrophin biology by Dr. Jeffrey Chamberlain of the University of Washington and Dr. Dongsheng Duan of the University of Missouri. SGT-001 has been granted Rare Pediatric Disease Designation, or RPDD, and Fast Track Designation in the United States and Orphan Drug Designations in both the United States and European Union.

    For more information, please visit www.solidbio.com.

    Ultragenyx Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the effects from the COVID-19 pandemic on the company’s clinical activities, business and operating results, uncertainty and potential delays related to clinical drug development, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on July 30, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Solid Biosciences Forward-Looking Statements

    This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding whether the collaboration will yield any viable product candidates, potential milestone payments or royalty payments in connection with the collaboration, the potential benefits of the collaboration, the safety or potential efficacy of SGT-001 and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," "working" and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with each party’s ability to perform its obligations under the collaboration, the Company’s ability to resume and/or continue IGNITE DMD on the timeline expected or at all; obtain and maintain necessary approvals from the FDA and other regulatory authorities; obtain and maintain the necessary approval from investigational review boards at IGNITE DMD clinical trial sites and the IGNITE DMD independent data safety monitoring board; enroll patients in IGNITE DMD; continue to advance SGT-001 in clinical trials; replicate in clinical trials positive results found in preclinical studies and earlier stages of clinical development; advance the development of its product candidates under the timelines it anticipates in current and future clinical trials; successfully optimize and scale its manufacturing process; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop DMD/Duchenne treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-001, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section, as well as discussions of potential risks, uncertainties and other important factors, in the Company’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date hereof and should not be relied upon as representing the Company’s views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

    Contacts:

    Ultragenyx

    Joshua Higa

    (415) 475-6370

    Solid Biosciences

    Investor Contact:

    David Carey

    FINN Partners

    212-867-1768

    Media Contact:

    Erich Sandoval

    FINN Partners

    917-497-2867

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  4. NOVATO, Calif. , Oct. 21, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Tuesday, October 27, 2020 at 5pm ET to discuss third quarter 2020 financial results and provide a corporate update.

    The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 4067577. The replay of the call will be available for one year.

    About Ultragenyx

    NOVATO, Calif. , Oct. 21, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Tuesday, October 27, 2020 at 5pm ET to discuss third quarter 2020 financial results and provide a corporate update.

    The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 4067577. The replay of the call will be available for one year.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com

    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Joshua Higa

    415-475-6370

     

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  5. NOVATO, Calif., Sept. 25, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will hold a virtual presentation at the Jefferies Virtual Gene Therapy/Editing Summit on Friday, October 2, 2020 at 1:00 PM ET.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed…

    NOVATO, Calif., Sept. 25, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will hold a virtual presentation at the Jefferies Virtual Gene Therapy/Editing Summit on Friday, October 2, 2020 at 1:00 PM ET.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Joshua Higa

    415-660-0951

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  6. NOVATO, Calif., Sept. 03, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the following upcoming investor conferences:

    • Citi's 15th Annual BioPharma Virtual Conference as part of a panel discussion on Wednesday, September 9, 2020 at 4:15 PM ET.
       
    • Morgan Stanley Virtual 18th Annual Global Healthcare Conference on Tuesday, September 15, 2020 at 12:30 PM ET.
       
    • BofA Securities Virtual Global Healthcare Conference on Wednesday, September 16, 2020 at 11:45 AM ET.

    The…

    NOVATO, Calif., Sept. 03, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the following upcoming investor conferences:

    • Citi's 15th Annual BioPharma Virtual Conference as part of a panel discussion on Wednesday, September 9, 2020 at 4:15 PM ET.

       
    • Morgan Stanley Virtual 18th Annual Global Healthcare Conference on Tuesday, September 15, 2020 at 12:30 PM ET.

       
    • BofA Securities Virtual Global Healthcare Conference on Wednesday, September 16, 2020 at 11:45 AM ET.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at:  www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Joshua Higa

    415-660-0951

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  7. NOVATO, Calif., Sept. 02, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it has appointed Mardi C. Dier as Executive Vice President and Chief Financial Officer (CFO), effective November 2, 2020. Ms. Dier will be responsible for leading the corporate finance, strategy, investor relations, corporate communications, and information technology functions. She will report to Emil D. Kakkis, M.D., Ph.D., Ultragenyx's Chief Executive Officer, and will serve on the executive leadership team. Ultragenyx had previously announced the planned transition of CFO Shalini…

    NOVATO, Calif., Sept. 02, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it has appointed Mardi C. Dier as Executive Vice President and Chief Financial Officer (CFO), effective November 2, 2020. Ms. Dier will be responsible for leading the corporate finance, strategy, investor relations, corporate communications, and information technology functions. She will report to Emil D. Kakkis, M.D., Ph.D., Ultragenyx's Chief Executive Officer, and will serve on the executive leadership team. Ultragenyx had previously announced the planned transition of CFO Shalini Sharp. Ms. Sharp will remain as CFO until Ms. Dier starts in November and will consult for the company for six months after.

    "We welcome Mardi to Ultragenyx and its leadership team at an important time as we build upon our solid foundation and recent successes in treating patients with serious rare diseases and strive to grow the company to the next level. Mardi is a proven industry leader with financial and strategic expertise which will serve to fortify Ultragenyx's efforts in advancing our global commercial opportunities and our promising pipeline addressing important diseases with innovative therapies," said Dr. Kakkis. "We also want to thank Shalini for her incredible contribution and dedication to the success thus far of Ultragenyx. We will miss her and sincerely wish her well with her next endeavors."

    Ms. Dier joins Ultragenyx from Portola Pharmaceuticals, where she served as Chief Financial Officer from 2006 to 2018 when her role was expanded to include Chief Business Officer from 2018 through its acquisition by Alexion Pharmaceuticals in 2020. During her tenure at Portola, she successfully led a series of private, public, and alternative financings and led the company through its pivotal transition into a commercial organization. Prior to Portola, she served as Vice President of Investor Relations at Chiron Corporation from 2003 to 2006. Earlier in her career she worked as an investment banker at Prudential Securities, and prior to that was in the audit department of KPMG Peat Marwick. She holds a B.S. in biology from Stanford University and an M.B.A. from the Anderson School at the University of California, Los Angeles. Ms. Dier serves on the Board of Directors of Adamas Pharmaceuticals, ORIC Pharmaceuticals, and Prelude Therapeutics.

    "Ultragenyx is a company truly dedicated to developing medicines for patients with serious rare diseases and I am thrilled to join a team so impassioned by its mission," said Ms. Dier. "As a global commercial company with an exciting and deep pipeline targeting a number of diseases across multiple modalities, I look forward to being part of this leadership team and helping further establish Ultragenyx as a leading, global, and diversified rare disease company."

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Joshua Higa

    (415) 475-6370

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  8. NOVATO, Calif., Sept. 01, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it has appointed Ernie Meyer as Executive Vice President and Chief Human Resources Officer, effective September 1, 2020. In this newly created role, Mr. Meyer will lead all aspects of the Global Human Resources and Facilities operations. He will report to Emil D. Kakkis, M.D., Ph.D., Ultragenyx's Chief Executive Officer, and will serve on the Executive Leadership Team.  

    "Ernie has tremendous experience building multi-national biopharmaceutical organizations, and his leadership will…

    NOVATO, Calif., Sept. 01, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it has appointed Ernie Meyer as Executive Vice President and Chief Human Resources Officer, effective September 1, 2020. In this newly created role, Mr. Meyer will lead all aspects of the Global Human Resources and Facilities operations. He will report to Emil D. Kakkis, M.D., Ph.D., Ultragenyx's Chief Executive Officer, and will serve on the Executive Leadership Team.  

    "Ernie has tremendous experience building multi-national biopharmaceutical organizations, and his leadership will be key to the continued growth and development of our excellent team as we enter the next chapter of our future," said Dr. Kakkis. "We welcome him to Ultragenyx and look forward to his contributions to help us achieve our goals as we begin our second decade as a diversified, global rare disease company."

    Mr. Meyer joins Ultragenyx from Portola Pharmaceuticals, where he was Executive Vice President, Chief Human Resources Officer since 2018. Before Portola, he spent more than 13 years at Celgene, most recently as Executive Vice President of Human Resources and Corporate Services. While at Celgene, he played a significant role in the company's growth to more than 7,000 employees in 37 countries. Prior to Celgene, Mr. Meyer spent 10 years at Motorola in a variety of human resource roles of increasing scope and responsibility. Mr. Meyer holds a B.S. in Business Management with a concentration in Management Information Systems from Widener University.

    "Ultragenyx is making great strides in the rare disease space and places tremendous focus on people – from patients and the rare disease community it serves, to the employees who make Ultragenyx the special company that it is," said Mr. Meyer. "I am delighted to have the opportunity to join the leadership team to foster and develop these strengths, and to contribute to the meaningful work the organization is doing on behalf of patients."

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Joshua Higa

    (415) 475-6370



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  9. NOVATO, Calif., Aug. 04, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will hold virtual presentations at the following upcoming investor conferences:

    • Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will hold a virtual presentation at the Wedbush PacGrow Healthcare Conference on Tuesday, August 11, 2020 at 2:55 PM ET.
       
    • Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the Canaccord Genuity 40th Annual Growth Conference on Thursday, August 13, 2020 at 2:00 PM ET.

    The live and archived…

    NOVATO, Calif., Aug. 04, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will hold virtual presentations at the following upcoming investor conferences:

    • Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will hold a virtual presentation at the Wedbush PacGrow Healthcare Conference on Tuesday, August 11, 2020 at 2:55 PM ET.

       
    • Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the Canaccord Genuity 40th Annual Growth Conference on Thursday, August 13, 2020 at 2:00 PM ET.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at:  www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Joshua Higa

    415-660-0951

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  10. Two U.S. FDA approvals in second quarter 2020

    Second quarter 2020 total revenue of $61.7 million and Crysvita revenue in Ultragenyx territories of $32.4 million

    Maintains full year 2020 guidance based on global COVID-19 impact to date

    NOVATO, Calif., July 30, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended June 30, 2020 and maintained its full year 2020 financial guidance for Crysvita in Ultragenyx territories.

    "In the second quarter we made significant progress expanding the commercial…

    Two U.S. FDA approvals in second quarter 2020

    Second quarter 2020 total revenue of $61.7 million and Crysvita revenue in Ultragenyx territories of $32.4 million

    Maintains full year 2020 guidance based on global COVID-19 impact to date

    NOVATO, Calif., July 30, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended June 30, 2020 and maintained its full year 2020 financial guidance for Crysvita in Ultragenyx territories.

    "In the second quarter we made significant progress expanding the commercial reach of Crysvita and Mepsevii despite the COVID environment. We also expanded our commercial portfolio with two FDA approvals in a two-week period as Dojolvi for LC-FAOD and Crysvita for TIO became the third and fourth approvals for Ultragenyx," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We also presented positive updates at ASGCT on both of our clinical gene therapy programs, DTX401 for GSDIa and DTX301 for OTC deficiency, and we are heading towards Phase 3 studies for each of them."

    Second Quarter 2020 Financial Results

    Net Revenues

    For the second quarter of 2020, Ultragenyx reported $61.7 million in total revenue. Ultragenyx recognized $32.4 million in total Crysvita revenue in the Ultragenyx territories, which includes $29.8 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $2.5 million. Total royalty revenue related to European Crysvita sales were $5.0 million, which includes $1.5 million recognized on sales that occurred prior to January 1, 2020. Mepsevii® (vestronidase alfa) product revenue for the second quarter of 2020 was $4.2 million, and UX007 named patient revenue was $1.3 million. Revenue for the second quarter also includes $18.9 million of revenue related to the collaboration and license agreement with Daiichi Sankyo that was executed in March 2020.

    Revenue for the six months ended June 30, 2020 was $98.0 million, including $61.2 million in total Crysvita revenue in the Ultragenyx territories. Crysvita collaboration revenue in the North American profit share territory was $57.0 million and net Crysvita product sales in other regions were $4.2 million. Total royalty revenue related to European Crysvita royalties was $7.6 million in the first half of 2020, which includes $1.5 million recognized on sales that occurred prior to January 1, 2020. Mepsevii product revenue for the six months ended June 30, 2020 was $7.6 million and UX007 named patient revenue was $2.8 million. The technology transfer services with Daiichi Sankyo were initiated during the second quarter of 2020 and Ultragenyx began recognizing revenue during the period related to the collaboration and license agreement. For the three and six months ended June 30, 2020, revenue related to this agreement was $18.9 million.

    Operating Expenses

    Total operating expenses for the second quarter of 2020 were $124.8 million, including non-cash stock-based compensation of $22.4 million. Total operating expenses for the second quarter of 2019 were $136.6 million, including a $15.6 million research and development expense from the Arcturus collaboration amendment, and non-cash stock-based compensation of $22.2 million.

    Total operating expenses for the six months ended June 30, 2020 were $281.7 million, which includes $25.0 million to maintain the option to acquire GeneTx, $7.0 million to license certain vectors from REGENXBIO, and non-cash stock-based compensation of $42.6 million. This is compared with $254.0 million for the same period in 2019, which includes a $15.6 million research and development expense from the Arcturus collaboration amendment and non-cash stock-based compensation $42.4 million. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows, and advances its portfolio.

    For the second quarter of 2020, Ultragenyx reported net income of $25.3 million, or $0.42 per basic share and $0.41 per diluted share, compared with a net loss for the second quarter of 2019 of $99.2 million, or $1.72 per share, basic and diluted. For the six months ended June 30, 2020, net loss was $93.7 million, or $1.59 per share, basic and diluted, compared with a net loss for the same period in 2019 of $195.9 million, or $3.54 per share, basic and diluted. The net income for the second quarter of 2020 and the net loss for the six months ended June 30, 2020 includes a $95.2 million unrealized gain and a $102.9 million unrealized gain for the three and six months ended June 30, 2020, respectively, from the fair value adjustment on the investment in Arcturus equity. Net cash used in operations for the first six months of 2020 was $7.8 million, which includes $134.9 million of operating cash received from Daiichi Sankyo related to the collaboration and license agreement, compared to net cash used of $184.8 million for the same period in 2019.

    Cash, Cash Equivalents and Investments

    Cash, cash equivalents, and investments were $817.5 million as of June 30, 2020.

    2020 Guidance

    Crysvita Guidance in Ultragenyx Territories

    The company continues to maintain its guidance range for 2020 Crysvita revenue in the Ultragenyx territories between $125.0 million and $140.0 million, but will continue to monitor the COVID-19 pandemic situation. Ultragenyx territories include the collaboration revenue from the North American profit share territory (U.S. and Canada) and other regions where revenue from product sales are recognized by Ultragenyx (Latin America, Turkey). This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.

    Corporate Update

    Arcturus Collaboration

    • In May 2020, Ultragenyx exercised its option to purchase 600,000 shares of Arcturus common stock at $16.00 per share. Upon completion of the new equity purchase, Ultragenyx owned 3,000,000 shares of Arcturus common stock and continued to be its largest shareholder.

    COVID-19 Update

    • Despite the ongoing pandemic, the company has been able to maintain an uninterrupted supply of medicine to patients around the world as no significant disruptions to manufacturing or distribution activities have been experienced. There has been some impact to preclinical manufacturing, clinical study site conduct, and regulatory interactions. The company is adapting clinical and commercial strategies to ensure continuity of clinical and commercial programs during this unprecedented time.

    Program Updates and Upcoming Milestones

    Dojolvi for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD): Approved by FDA on June 30, 2020

    • The U.S. FDA approved Dojolvi for the treatment of pediatric and adult patients for all forms of LC-FAOD with a molecularly-confirmed diagnosis. Dojolvi is the first FDA-approved therapy for these lifelong and life-threatening genetic disorders and is now available to patients in the U.S.



    • Dojolvi has been submitted for approval with ANVISA in Brazil and has been submitted to Health Canada after being granted priority review.

    Crysvita for Tumor-Induced Osteomalacia (TIO): Approved by FDA on June 18, 2020

    • The U.S. FDA approved Crysvita for the treatment of fibroblast growth factor 23 (FGF23)-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older. This is the second FDA approval for Crysvita, which was first approved in April 2018 for the treatment of X-linked hypophosphatemia (XLH).

    DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Positive data from confirmatory cohort in Phase 1/2 study presented at American Society of Gene & Cell Therapy (ASGCT) Annual Meeting

    • As presented at ASGCT, all three patients in the third, confirmatory dose cohort (6.0 x 10^12 GC/kg) demonstrated increased time to hypoglycemia and substantial reductions in cornstarch usage. Prolonged periods of hyperglycemia were observed in Cohort 3 with the implementation of continuous glucose monitoring (CGM), which is indicative of early transgene expression and glucose release from the liver. The early transgene expression resulted in faster cornstarch reductions in Cohort 3, with a mean reduction of 57% at week 12 compared with 38% and 14% in the first and second cohorts, respectively. Across all three cohorts in the study, 100 percent of patients have demonstrated meaningful and sustained cornstarch regimens over time and significant increases in time to hypoglycemia. After longer-term follow-up, four of six patients in the first two cohorts have discontinued daytime cornstarch.



    • Barring delays related to COVID-19, the company expects to release additional data on Cohort 3 in the second half of 2020, intends to hold an end-of-Phase 2 meeting with the U.S. FDA, and potentially initiate a Phase 3 study in early 2021.

    DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Updated positive data from Phase 1/2 study presented at ASGCT

    • Updated data presented at ASGCT confirmed that all three patients in the third dose cohort (1.0 x 10^13 GC/kg) are responders to DTX301 as shown by sustained meaningful increases in the rate of ureagenesis or reductions in ammonia levels. Six of nine patients across all three cohorts have now responded to the gene therapy, including three females and three males. All three complete responders, those who have discontinued all ammonia scavengers and liberalized their diets, remain clinically and metabolically stable after longer-term follow-up.



    • A fourth cohort of three patients at the same Cohort 3 dose is planned using prophylactic steroids. Dosing is still on hold due to COVID-19 but data are expected by the end of 2020. Ultragenyx intends to hold an end-of-phase 2 meeting with the FDA based on the first three cohorts, with Phase 3 study initiation currently expected in the first half of 2021.

    DTX201 / BAY 2599023 Partnered with Bayer for Hemophilia A: Cohort 3 data from Phase 1/2 study presented at International Society on Thrombosis and Haemostasis (ISTH) 2020 Congress

    • Three cohorts with two patients each have been dosed with AAVhu37 (DTX201 / BAY 2599023), using material from Ultragenyx's proprietary HeLa manufacturing platform. Initial data from the third dose cohort (2.0 x 10^13 GC/kg) and longer-term data from the lower dose cohorts were presented at ISTH. The data demonstrated a dose response with FVIII expression of 72.1% and 12.9% at weeks 29 and 26 in the two Cohort 3 patients. No spontaneous bleeds were reported after Cohort 3 patients reached peak expression despite prophylaxis discontinuation. One traumatic bleed occurred with no need for Factor VIII treatment. No adverse events related to DTX201 were observed, and ALT elevations in both patients were successfully treated with corticosteroids. Longer-term data from the two earlier cohorts showed sustained FVIII expression up to 16 months with no loss of expression.

    GTX-102 for Angelman Syndrome Conducted by Partner, GeneTx: First two cohorts have been fully enrolled

    • The first two cohorts of two patients each have been fully enrolled with all patients receiving multiples doses in the Phase 1/2 study of GTX-102 in Angelman Syndrome. Safety and efficacy data from the first two dose escalating cohorts are currently being evaluated and enrollment and dosing at the next dose levels are expected to resume shortly. Preliminary data from the study are expected in the first half of 2021.

    Crysvita for X-Linked Hypophosphatemia (XLH): Positive opinion from Committee for Medicinal Products for Human Use (CHMP)

    • In Europe, Crysvita received a positive opinion from the CHMP, for the use of Crysvita for the treatment of XLH in adolescents and adults. This could lead to an expansion of the current market authorization beyond the current approval for children 1 year of age and older and adolescents with growing skeletons. A formal decision from the European Commission (EC) is expected in the second half of 2020.

    Mepsevii for Mucopolysaccharidosis Type VII (MPS VII): Positive opinion from CHMP to expand the Summary of Product Characteristics in Europe

    • The CHMP provided a positive opinion on a type II variation that would expand the EMA approval to include long-term effects of Mepsevii on the reduction of urinary glycosaminoglycans (uGAGs) and improvements in the multi-domain clinical responder index (MDRI) and 6-minute walk test (6MWT). A formal decision from the EC is expected in the second half of 2020.

    Other Gene Therapy Platform Updates

    • An IND application for UX701 in Wilson disease, a larger rare metabolic disease, is expected by the end of 2020 barring any delays in nonclinical or manufacturing activities.



    • Ultragenyx and Daiichi Sankyo initiated technology transfer activities related to the non-exclusive license agreement executed in the first quarter of 2020 with respect to Ultragenyx's HeLa PCL and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products. Daiichi Sankyo will pay $25.0 million upon completion of transfer of both platforms, as well as single-digit royalties on net sales of products manufactured in either system.

    Conference Call and Webcast Information

    Ultragenyx will host a conference call today, Thursday, July 30, 2020, at 2 p.m. PT/ 5 p.m. ET to discuss the second quarter 2020 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 1808389. The replay of the call will be available for one year.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the effects from the COVID-19 pandemic on the company's commercialization activities, business and operating results, smaller than anticipated market opportunities for the company's products and product candidates, manufacturing risks, competition from other therapies or products, uncertainties related to insurance coverage and reimbursement status of the company's newly approved products, the company's evolving integrated commercial organization, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 7, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.



    Ultragenyx Pharmaceutical Inc.
    Selected Statement of Operations Financial Data
    (in thousands, except share and per share amounts)
    (unaudited)
            
     Three Months Ended June 30,  Six Months Ended June 30,
      2020   2019   2020   2019 
    Statement of Operations Data:       
    Revenues:       
    Collaboration and license$50,161  $19,247  $77,376  $33,485 
    Product sales 8,066   4,902   14,545   8,836 
    Non-cash collaboration royalty revenue 3,482      6,097    
    Total revenues 61,709   24,149   98,018   42,321 
    Operating expenses:       
    Cost of sales 1,803   766   (1,700)  1,218 
    Research and development 80,709   96,045   193,670   174,150 
    Selling, general and administrative 42,252   39,812   89,768   78,641 
    Total operating expenses 124,764   136,623   281,738   254,009 
    Loss from operations (63,055)  (112,474)  (183,720)  (211,688)
    Change in fair value of investment in Arcturus equity securities 95,200   9,828   102,868   9,828 
    Non-cash interest expense on liability related to the sale of future royalties (8,429)     (16,511)   
    Other income (expense), net 2,014   3,687   4,477   6,361 
    Income (loss) before income taxes 25,730   (98,959)  (92,886)  (195,499)
    Provision for income taxes (415)  (213)  (824)  (429)
    Net income (loss)$25,315  $(99,172) $(93,710) $(195,928)
    Net income (loss) per share:       
    Basic$0.42  $(1.72) $(1.59) $(3.54)
    Diluted$0.41  $(1.72) $(1.59) $(3.54)
    Weighted average shares used in computing net income (loss) per share:       
    Basic 59,995,617   57,519,308   58,996,278   55,376,336 
    Diluted 61,146,231   57,519,308   58,996,278   55,376,336 
            



    Ultragenyx Pharmaceutical Inc.
    Selected Balance Sheet Financial Data
    (in thousands)
    (unaudited)
      June 30,

     December 31,

       2020  2019
    Balance Sheet Data:    
    Cash, cash equivalents, and available-for-sale investments$817,484 $760,404
    Working capital  686,310  747,717
    Total assets  1,314,041  1,135,496
    Total stockholders' equity  695,587  653,764



    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Joshua Higa

    (415) 475-6370

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  11. NOVATO, Calif., July 24, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Thursday, July 30, 2020 at 5pm ET to discuss second quarter 2020 financial results and provide a corporate update.

    The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 1808389. The replay of the call will be available for one year.

    About Ultragenyx

    NOVATO, Calif., July 24, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Thursday, July 30, 2020 at 5pm ET to discuss second quarter 2020 financial results and provide a corporate update.

    The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 1808389. The replay of the call will be available for one year.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at: 

    www.ultragenyx.com

    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Joshua Higa

    415-475-6370

    Primary Logo

    View Full Article Hide Full Article
  12. NOVATO, Calif., July 22, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that Dojolvi™ (triheptanoin) is now commercially available. The U.S. Food and Drug Administration (FDA) approved Dojolvi on June 30, 2020 as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare, lifelong, and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy.

    "Dojolvi, the only FDA-approved therapy for LC-FAOD, is now…

    NOVATO, Calif., July 22, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that Dojolvi™ (triheptanoin) is now commercially available. The U.S. Food and Drug Administration (FDA) approved Dojolvi on June 30, 2020 as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD), a group of rare, lifelong, and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy.

    "Dojolvi, the only FDA-approved therapy for LC-FAOD, is now available for patients living with this serious, unpredictable, and often catastrophic disease," said Erik Harris, Chief Commercial Officer of Ultragenyx. "We are now focused on ensuring that all patients in the U.S. with LC-FAOD who might benefit from Dojolvi will have access to it."

    To support access, Ultragenyx's UltraCare® program helps patients and caregivers understand insurance coverage and assists them in finding financial support for Ultragenyx medicines, including Dojolvi, and for the administration of them. Dedicated in-house UltraCare Guides are available Monday through Friday from 9 a.m. to 8 p.m. Eastern Time at 888-756-8657.

    About Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)

    LC-FAOD are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to convert long-chain fatty acids into energy. The inability to produce energy from fat can lead to severe depletion of glucose in the body and serious complications, which can lead to hospitalizations or early death. LC-FAOD are included in newborn screening panels across the U.S. and in certain European countries due to the risk for serious outcomes including death early in life. Other current treatment options for LC-FAOD include avoidance of fasting, low-fat/high-carbohydrate diets, carnitine and even-carbon medium-chain triglyceride (MCT) oil, a medical food product. LC-FAOD affect an estimated 2,000 to 3,500 children and adults in the United States.

    About Dojolvi

    Dojolvi is a highly purified, pharmaceutical-grade, odd-carbon medium-chain triglyceride consisting of three 7-carbon fatty acids on a glycerol backbone created via a multi-step chemical process. It is designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD.

    INDICATION

    Dojolvi is indicated as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).

    IMPORTANT SAFETY INFORMATION

    WARNINGS AND PRECAUTIONS

    Feeding Tube Dysfunction

    • Feeding tube dysfunction was reported in patients receiving triheptanoin. The contribution of Dojolvi cannot be ruled out. Dojolvi should not be administered in feeding tubes manufactured of polyvinyl chloride (PVC). The feeding tube should be monitored regularly to ensure proper functioning and integrity.

    Intestinal Malabsorption in Patients with Pancreatic Insufficiency

    • Low or absent pancreatic enzymes may result in reduced absorption of heptanoate subsequently leading to insufficient supplementation of medium-chain fatty acids. Administration of Dojolvi should be avoided in patients with pancreatic insufficiency.

    ADVERSE REACTIONS

    Gastrointestinal (GI) 

    • The most common GI-related adverse reactions reported in the pooled safety population of Studies 1 and 2 were abdominal pain (abdominal discomfort, abdominal pain, abdominal distension, abdominal pain upper, GI pain) [60%], diarrhea [44%], vomiting [44%], and nausea [14%].

    DRUG INTERACTIONS

    Pancreatic Lipase Inhibitors

    • Co-administration should be avoided due to potential for reduced clinical effect of Dojolvi.

    USE IN SPECIFIC POPULATIONS

    Pregnancy and Lactation

    • There are no available data on triheptanoin use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Women are advised to report pregnancies to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657. 
    • There are no data on the presence of triheptanoin or its metabolites in human or animal milk, the effects on the breastfed infant, or the effects on milk production.

    PATIENT COUNSELING INFORMATION

    The patient or caregiver should: 

    • Read the FDA-approved patient labeling, which includes information on the appropriate oral or feeding tube preparation, administration, and storage.
    • Regularly inspect the feeding tube for proper functioning and integrity and report to the healthcare provider if any issues are identified.
    • Be informed that pancreatic insufficiency may reduce the clinical effect of Dojolvi and to report any known pancreatic insufficiency to the healthcare provider.

    You may report side effects to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 

    Please see full Prescribing Information, including the Patient Information leaflet, for a complete discussion of the risks associated with Dojolvi.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the effects from the COVID-19 pandemic on the company's commercialization activities, business and operating results, smaller than anticipated market opportunities for the company's products and product candidates, manufacturing risks, competition from other therapies or products, uncertainties related to insurance coverage and reimbursement status of the company's newly approved products, the company's evolving integrated commercial organization, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 7, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Joshua Higa

    +1-415-475-6370

      

    Primary Logo

    View Full Article Hide Full Article
  13. Fourth U.S. FDA Approval for Ultragenyx in the Past Three Years All for Rare Diseases which Previously Had No Approved Therapies

    Ultragenyx to Host Conference Call Today at 4:00 pm Eastern Time

    NOVATO, Calif., June 30, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Dojolvi™ (triheptanoin) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD are a group of rare, lifelong and life-threatening…

    Fourth U.S. FDA Approval for Ultragenyx in the Past Three Years All for Rare Diseases which Previously Had No Approved Therapies

    Ultragenyx to Host Conference Call Today at 4:00 pm Eastern Time

    NOVATO, Calif., June 30, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Dojolvi™ (triheptanoin) as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD are a group of rare, lifelong and life-threatening genetic disorders in which the body is unable to convert long-chain fatty acids into energy. Dojolvi is a highly purified, synthetic, 7-carbon fatty acid triglyceride specifically designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD.

    "With today's FDA approval, patients living with this serious, unpredictable, and often catastrophic disease now have an approved therapy. Many patients with long-chain fatty acid oxidation disorders have difficult lives with frequent hospitalizations and major medical events despite the best current care. Now these patients have an approved treatment as an option to help manage their disease," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "We are grateful to the patients, caregivers, families, dietitians and doctors involved for helping to make this community milestone possible, and we are committed to ensuring that all patients in the U.S. with LC-FAOD who might benefit from Dojolvi will have access to it."

    LC-FAOD are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to convert long-chain fatty acids into energy. The inability to produce energy from fat can lead to severe depletion of glucose in the body and serious complications, which can lead to hospitalizations or early death. LC-FAOD are included in newborn screening panels across the U.S. and in certain European countries due to the risk for serious outcomes including death early in life.  Other current treatment options for LC-FAOD include avoidance of fasting, low-fat/high-carbohydrate diets, carnitine and even-carbon medium-chain triglyceride (MCT) oil, a medical food product. LC-FAOD affect an estimated 2,000 to 3,500 children and adults in the United States.

    "FDA approval of Dojolvi for the treatment of long-chain fatty acid oxidation disorders is a huge milestone for patients and their families, as it is the first such medication," said Dr. Jerry Vockley, Professor of Human Genetics and Chief of Medical Genetics at Children's Hospital of Pittsburgh of UPMC. "While we have been able to identify these disorders at birth for many years, treatment options have been limited. Dojolvi brings hope as a new tool now available to clinicians taking care of these patients. Rare disease research, especially development of new therapies, is an arduous and time consuming process, and today's announcement represents the culmination of nearly 20 years of work on the part of numerous researchers, clinicians, and patients. I'm tremendously excited to have been a part of this process."

    Ultragenyx expects Dojolvi to be available to patients in the next 30 days. To support access, Ultragenyx's UltraCare® program helps patients and caregivers understand insurance coverage and assists them in finding financial support for Ultragenyx medicines, including Dojolvi, and for the administration of them. Dedicated in-house UltraCare Guides are available Monday through Friday from 9 a.m. to 8 p.m. Eastern Time at 888-756-8657.

    Investor Conference Call

    Ultragenyx will host a conference call today at 4:00 pm Eastern Time/ 1:00 pm Pacific Time to discuss the Dojolvi approval. The live and replayed webcast of the call will be available through the company's website at www.ultragenyx.com. To participate in the live call by phone, dial (855) 797-6910 (U.S.) or (262) 912-6260 (international) and enter the passcode 1597363. The replay of the call will be available for one year.

    About Dojolvi

    Dojolvi is a highly purified, pharmaceutical-grade, odd-carbon medium-chain triglyceride consisting of three 7-carbon fatty acids on a glycerol backbone created via a multi-step chemical process. It is designed to provide medium-chain, odd-carbon fatty acids as an energy source and metabolite replacement for people with LC-FAOD.

    Ultragenyx licensed exclusive worldwide rights to triheptanoin from Baylor Research Institute in 2013.

    INDICATION

    Dojolvi is indicated as a source of calories and fatty acids for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD).

    IMPORTANT SAFETY INFORMATION

    WARNINGS AND PRECAUTIONS

    Feeding Tube Dysfunction

    • Feeding tube dysfunction was reported in patients receiving triheptanoin. The contribution of Dojolvi cannot be ruled out. Dojolvi should not be administered in feeding tubes manufactured of polyvinyl chloride (PVC). The feeding tube should be monitored regularly to ensure proper functioning and integrity.

    Intestinal Malabsorption in Patients with Pancreatic Insufficiency

    • Low or absent pancreatic enzymes may result in reduced absorption of heptanoate subsequently leading to insufficient supplementation of medium-chain fatty acids. Administration of Dojolvi should be avoided in patients with pancreatic insufficiency.

    ADVERSE REACTIONS

    Gastrointestinal (GI)

    • The most common GI-related adverse reactions reported in the pooled safety population of Studies 1 and 2 were abdominal pain (abdominal discomfort, abdominal pain, abdominal distension, abdominal pain upper, GI pain) [60%], diarrhea [44%], vomiting [44%], and nausea [14%].

    DRUG INTERACTIONS

    Pancreatic Lipase Inhibitors

    • Co-administration should be avoided due to potential for reduced clinical effect of Dojolvi.

    USE IN SPECIFIC POPULATIONS

    Pregnancy and Lactation

    • There are no available data on triheptanoin use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Women are advised to report pregnancies to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657. 
    • There are no data on the presence of triheptanoin or its metabolites in human or animal milk, the effects on the breastfed infant, or the effects on milk production.

    PATIENT COUNSELING INFORMATION

    The patient or caregiver should: 

    • Read the FDA-approved patient labeling, which includes information on the appropriate oral or feeding tube preparation, administration, and storage.
    • Regularly inspect the feeding tube for proper functioning and integrity and report to the healthcare provider if any issues are identified.
    • Be informed that pancreatic insufficiency may reduce the clinical effect of Dojolvi and to report any known pancreatic insufficiency to the healthcare provider.

    You may report side effects to Ultragenyx Pharmaceutical Inc. at 1-888-756-8657 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 

    Please see full Prescribing Information, including the Patient Information leaflet, for a complete discussion of the risks associated with Dojolvi.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the effects from the COVID-19 pandemic on the company's commercialization activities, business and operating results, smaller than anticipated market opportunities for the company's products and product candidates, manufacturing risks, competition from other therapies or products, uncertainties related to insurance coverage and reimbursement status of the company's newly approved products, the company's evolving integrated commercial organization, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance, the timing of clinical trial activities  and reporting results from same, and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 7, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media

    Danielle Keatley

    +1-415-475-6876

                          

     

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  14. First Approved Therapy in the United States for Patients with TIO Who Cannot Undergo Surgical Removal of Tumors

    TIO is Second FDA-Approved Indication for Crysvita, Which is Also Approved for the Treatment of X-linked Hypophosphatemia (XLH)

    NOVATO, Calif. and TOKYO, June 18, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions using the latest biotechnology, today announced that the U.S. Food and Drug Administration (FDA) has approved Crysvita® (burosumab) for the…

    First Approved Therapy in the United States for Patients with TIO Who Cannot Undergo Surgical Removal of Tumors

    TIO is Second FDA-Approved Indication for Crysvita, Which is Also Approved for the Treatment of X-linked Hypophosphatemia (XLH)

    NOVATO, Calif. and TOKYO, June 18, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions using the latest biotechnology, today announced that the U.S. Food and Drug Administration (FDA) has approved Crysvita® (burosumab) for the treatment of fibroblast growth factor 23 (FGF23)-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older. Crysvita is a human antibody that blocks excess activity of FGF23, a hormone that causes phosphate urinary excretion and suppresses active vitamin D production by the kidney.

    "For approximately half of all individuals with TIO, surgical removal of the tumors is not possible, leaving these patients with no other treatment options. The FDA approval of Crysvita marks the first treatment option that addresses the cause of the severe hypophosphatemia and osteomalacia resulting from these rare tumors," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "We plan to leverage our experience and existing infrastructure with Crysvita in X-linked hypophosphatemia to bring this important medicine to patients living with the rare, painful and debilitating disorder of TIO."

    "Since its approval, Crysvita has meant a great deal to patients and families that previously had no other treatment options.  We are proud of the work that has been done to advance this discovery from our labs, through a robust clinical research program, and through the FDA's priority review process, to make this treatment available to patients with TIO," said Gary Zieziula, President, North America for Kyowa Kirin. "Our commitment to meeting the needs of patients with rare and serious diseases remains steadfast and we will continue to partner with the Ultragenyx team to address these needs with urgency."

    TIO is a rare disease caused by typically benign, slow-growing tumors that produce excess levels of FGF23, which is involved in phosphate reabsorption. Patients with TIO can experience symptoms including severe hypophosphatemia (low levels of phosphate in the blood), osteomalacia (softening of the bones), muscle weakness, fatigue, bone pain and fractures. There are an estimated 500 to 1,000 people in the United States with TIO, and approximately half of all cases are believed to be inoperable. In patients for whom the tumor or lesion is inoperable, the current treatment consists of oral phosphate and/or active vitamin D replacement. Efficacy of this management is often limited, and its benefits must be balanced with monitoring for potential risks.

    This is the second FDA-approved indication for Crysvita, which was first approved in April 2018 for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older. The XLH indication was expanded in September 2019 to include infants as young as six months of age.

    The FDA approval of Crysvita for TIO was based on data from two single-arm Phase 2 studies, a 144-week study in 14 adult patients conducted by Ultragenyx in the United States and an 88-week study in 13 adult patients conducted by Kyowa Kirin in Japan and South Korea. In both studies, Crysvita was associated with increases in serum phosphorus and serum 1,25-dihydroxyvitamin D levels. Increased phosphate levels led to improvements in osteomalacia. Additionally, whole body bone scans demonstrated reduced tracer uptake with long-term treatment suggesting healing of bone lesions. Most common adverse reactions (>10%) in TIO patients are: tooth abscess, muscle spasms, dizziness, constipation, injection site reaction, rash, and headache.

    The FDA granted Priority Review designation for the supplemental BLA for TIO, which is reserved for drugs that treat a serious condition and, if approved, would provide a significant improvement in safety or effectiveness.

    To support access, Ultragenyx has a program called UltraCare™, which helps patients and caregivers understand insurance coverage and assists them in finding financial support for Ultragenyx medicines including Crysvita, and for the administration of them. Dedicated in-house UltraCare Guides are available Monday through Friday from 9 a.m. to 8 p.m. Eastern Time at 888-756-8657.

    About Crysvita

    Crysvita (burosumab-twza) is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, against the phosphaturic hormone FGF23. FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate wasting in TIO and other hypophosphatemic conditions, including XLH, is caused by excessive levels and activity of FGF23. Crysvita is designed to bind to and thereby inhibit the biological activity of FGF23. By blocking excess FGF23 in patients with TIO and XLH, Crysvita is intended to increase phosphate reabsorption from the kidney and increase the production of active vitamin D, which enhances intestinal absorption of phosphate and calcium.

    Crysvita is approved by the U.S. FDA for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients six months of age and older and FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adults and pediatric patients 2 years of age and older, and by Health Canada and Brazil's National Health Surveillance Agency (ANVISA) for the treatment of XLH in adult and pediatric patients one year of age and older. In Japan, it is approved by the Ministry of Health, Labor and Welfare (MHLW) for the treatment of FGF23-related hypophosphatemic rickets and osteomalacia. In Europe, Crysvita has received European conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons, and an application for the expanded use in adults with XLH is currently under review by the European Medicines Agency. 

    Kyowa Kirin and Ultragenyx have been collaborating in the development and commercialization of Crysvita globally based on the collaboration and license agreement between the parties.

    U.S. INDICATION

    Crysvita® (burosumab-twza) is a fibroblast growth factor 23 (FGF23)-blocking antibody indicated for the treatment of:

    • X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older.
    • FGF23-related hypophosphatemia in tumor-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumors that cannot be curatively resected or localized in adult and pediatric patients 2 years of age and older.

    IMPORTANT SAFETY INFORMATION

    CONTRAINDICATIONS

    • With oral phosphate and/or active vitamin D analogs (e.g., calcitriol, paricalcitol, doxercalciferol, calcifediol).
    • When serum phosphorus is within or above the normal range for age.
    • In patients with severe renal impairment or end stage renal disease.         

    WARNINGS AND PRECAUTIONS

    Hypersensitivity

    • Discontinue Crysvita if serious hypersensitivity reactions occur and initiate appropriate medical treatment.           

    Hyperphosphatemia and Risk of Nephrocalcinosis

    • For patients already taking Crysvita, dose interruption and/or dose reduction may be required based on a patient's serum phosphorus levels.
    • Patients with TIO who undergo treatment of the underlying tumor should have dosing interrupted and adjusted to prevent hyperphosphatemia.

    Injection Site Reactions

    • Discontinue Crysvita if severe injection site reactions occur and administer appropriate medical treatment.

    ADVERSE REACTIONS

    Pediatric XLH Patients

    • Adverse reactions reported in 10% or more of Crysvita-treated pediatric XLH patients across all studies are: pyrexia, injection site reaction, cough, vomiting, pain in extremity, headache, tooth abscess, dental caries, diarrhea, vitamin D decreased, toothache, constipation, myalgia, rash, dizziness, and nausea.
    • Post-marketing experience reported in pediatric XLH patients receiving Crysvita – blood phosphorus increased.

                 

    Adult XLH Patients

    • Adverse reactions reported in more than 5% of Crysvita-treated adult XLH patients and in at least 2 patients more than placebo in one study are: back pain, headache, tooth infection, restless legs syndrome, vitamin D decreased, dizziness, constipation, muscle spasms, and blood phosphorus increased.
    • Spinal stenosis is prevalent in adults with XLH, and spinal cord compression has been reported. It is unknown if Crysvita therapy exacerbates spinal stenosis or spinal cord compression.

    Adult TIO Patients

    • Adverse reactions reported in more than 10% of Crysvita-treated adult TIO patients in two studies are: tooth abscess, muscle spasms, dizziness, constipation, injection site reaction, rash, and headache.

    USE IN SPECIFIC POPULATIONS

    • There are no available data on Crysvita use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at

      1-888-756-8657.
    • There is no information regarding the presence of Crysvita in human milk or the effects of Crysvita on milk production or the breastfed infant.

    PATIENT COUNSELING INFORMATION

    • Advise patients not to use any oral phosphate and/or active vitamin D analog products.
    • Instruct patients to contact their physician if hypersensitivity reactions, injection site reactions, and restless leg syndrome induction or worsening of symptoms occur.

    Side effects may be reported to the FDA at (800) FDA-1088 or www.fda.gov/medwatch.  Side effects may also be reported to Kyowa Kirin, Inc. at 1-888-756-8657.

    Please see full Prescribing Information for a complete discussion of the risks associated with CRYSVITA.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    About Kyowa Kirin

    Kyowa Kirin commits to innovative drug discovery driven by state-of-the-art technologies. The company focuses on creating new values in the four therapeutic areas: nephrology, oncology, immunology/allergy and neurology. Under the Kyowa Kirin brand, the employees from 40 group companies across North America, EMEA and Asia/Oceania unite to champion the interests of patients and their caregivers in discovering solutions to address unmet medical needs. You can learn more about the business of Kyowa Kirin at www.kyowakirin.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, Ultragenyx's reliance on its third party partner, Kyowa Kirin Co., Ltd., for the supply of Crysvita, the effects from the COVID-19 pandemic on the company's business and operating results, smaller than anticipated market opportunities for the company's products and product candidates, the company's evolving commercial infrastructure, uncertainties related to insurance coverage and reimbursement approval for the company's products, manufacturing risks, the uncertainties inherent in the clinical drug development process, including the potential for substantial delays and the risk that earlier study results may not be predictive of future study results, the lack of predictability in the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 7, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:

    Ultragenyx

    Investors & Media

    Danielle Keatley

    +1-415-475-6876

    Kyowa Kirin Co. Ltd.

    Media

    Hiroki Nakamura (Global)

    +81-3-5205-7205

    Email: 

    Lauren Walrath (US)

    +1-646-526-4454

    Email:            

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  15. NOVATO, Calif., May 28, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will hold virtual presentations at the following upcoming investor conferences:

    • Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the Jefferies Healthcare Conference on Wednesday, June 3, 2020 at 2 PM ET.
    • Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will hold a virtual presentation at the Goldman Sachs 41st Annual Global Healthcare Conference on Tuesday, June 9, 2020 at 3:50 PM ET.

    The live and archived webcast…

    NOVATO, Calif., May 28, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will hold virtual presentations at the following upcoming investor conferences:

    • Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the Jefferies Healthcare Conference on Wednesday, June 3, 2020 at 2 PM ET.
    • Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will hold a virtual presentation at the Goldman Sachs 41st Annual Global Healthcare Conference on Tuesday, June 9, 2020 at 3:50 PM ET.

    The live and archived webcast of these presentations will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. Replays of the webcasts will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at:  www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley              
    415-475-6876

    Primary Logo

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  16. NOVATO, Calif. and SAN DIEGO, May 21, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases and Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT), a leading messenger RNA medicines company, today announced that Ultragenyx has exercised its option to purchase 600,000 shares of Arcturus common stock at $16.00 per share.

    "We are encouraged by the advancement of Arcturus's broad nucleic acid platform across multiple therapeutic areas, including their self-replicating mRNA-based COVID-19 vaccine candidate," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx…

    NOVATO, Calif. and SAN DIEGO, May 21, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical, Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases and Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT), a leading messenger RNA medicines company, today announced that Ultragenyx has exercised its option to purchase 600,000 shares of Arcturus common stock at $16.00 per share.

    "We are encouraged by the advancement of Arcturus's broad nucleic acid platform across multiple therapeutic areas, including their self-replicating mRNA-based COVID-19 vaccine candidate," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We are also pleased with the progress of our preclinical UX053 mRNA candidate for Glycogen Storage Disease Type III and other earlier-stage opportunities we are exploring under the collaboration."

    "The new investment from Ultragenyx is a testament to the strength of our long-term collaboration in developing nucleic acid therapies," said Andrew Sassine, Chief Financial Officer of Arcturus. "The additional funding will support our clinical programs, including our efforts to move our COVID-19 vaccine candidate into clinical testing this summer."

    After completion of the new equity purchase, Ultragenyx will own 3,000,000 shares, or 14.6%, of Arcturus outstanding common stock and will continue to be its largest shareholder. The purchase was made pursuant to the equity purchase agreement between the parties entered into in June 2019 in connection with the amendment to the research collaboration and license agreement between the two companies focused on nucleic acid therapies for rare diseases that was initiated in 2015. The first disclosed indication under the collaboration is Glycogen Storage Disease Type III, and an Investigational New Drug (IND) application for this mRNA therapeutic program, UX053, is expected to be filed in 2021.

    About Ultragenyx Pharmaceutical, Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    About Arcturus Therapeutics Holdings Inc.
    Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (NASDAQ:ARCT) is a clinical-stage mRNA medicines and vaccines company with enabling technologies: (i) LUNAR® lipid-mediated delivery, (ii) STARR™ mRNA Technology and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus' diverse pipeline of RNA therapeutic candidates includes programs to potentially treat Ornithine Transcarbamylase (OTC) Deficiency, Cystic Fibrosis, Glycogen Storage Disease Type 3, Hepatitis B, non-alcoholic steatohepatitis (NASH) and a self-replicating mRNA vaccine for SARS-CoV-2. Arcturus' versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, replicon RNA, antisense RNA, microRNA, DNA, and gene editing therapeutics. Arcturus' technologies are covered by its extensive patent portfolio (187 patents and patent applications, issued in the U.S., Europe, Japan, China and other countries). Arcturus' commitment to the development of novel RNA therapeutics has led to collaborations with Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, Ultragenyx Pharmaceutical, Inc., Takeda Pharmaceutical Company Limited, CureVac AG, Synthetic Genomics Inc., Duke-NUS, Catalent Inc., and the Cystic Fibrosis Foundation. For more information visit www.ArcturusRx.com.

    Ultragenyx Forward-Looking Statements
    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with, and investment, in Arcturus, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), the effects from the COVID-19 pandemic on our clinical trial activities, business and operations and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report filed on Form 10-Q with the Securities and Exchange Commission on May 7, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Arcturus Forward-Looking Statements 
    This press release contains forward-looking statements that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact included in this press release, including those regarding Arcturus' expected performance, Arcturus' efforts to develop a vaccine against COVID-19, the forecasted safety, timing, efficacy, reliability or availability of a vaccine against COVID-19 were one to be successfully developed by Arcturus, the timing and the potential initiation of clinical trials of a vaccine against COVID-19 by Arcturus, are forward-looking statements. Arcturus may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in any forward-looking statements such as the foregoing and you should not place undue reliance on such forward-looking statements. Such statements are based on management's current expectations and involve risks and uncertainties, including those discussed under the heading "Risk Factors" in Arcturus' Annual Report on Form 10-K for the fiscal year ended December 31, 2019, filed with the SEC on March 16, 2020 and in subsequent filings with, or submissions to, the SEC. No assurances can be given that any results reported in pre-clinical studies can be replicated in further studies or in human beings, or that a vaccine can or will ever be developed or approved using Arcturus' technology. Except as otherwise required by law, Arcturus disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.

    Investor Relations & Media Contacts

    Arcturus Therapeutics
    Neda Safarzadeh
    858-900-2682

    Kendall Investor Relations
    Carlo Tanzi, Ph.D.
    617-914-0008

    Ultragenyx Pharmaceutical, Inc.
    Danielle Keatley
    415-475-6876

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  17. Hemostatic solutions innovator GATT Technologies closes €10.5M series B financing led by NGI LLC, a US based healthcare investor, along with the participation of Johnson & Johnson Innovation – JJDC, Inc., and Oost NL, and appoints Geert van Gansewinkel as its new Chief Executive Officer (CEO) and Vlad Hogenhuis as a new board member. Founder and current CEO Johan Bender will become responsible for R&D in his new role as CTO (Chief Technology Officer).

    NIJMEGEN, Netherlands, May 18, 2020 /PRNewswire/ -- GATT Technologies (the Company), a medical device company founded in 2011, developed a breakthrough patented synthetic technology intended to address severe and problematic surgical bleedings and organ leakages. The solution it has developed has…

    Hemostatic solutions innovator GATT Technologies closes €10.5M series B financing led by NGI LLC, a US based healthcare investor, along with the participation of Johnson & Johnson Innovation – JJDC, Inc., and Oost NL, and appoints Geert van Gansewinkel as its new Chief Executive Officer (CEO) and Vlad Hogenhuis as a new board member. Founder and current CEO Johan Bender will become responsible for R&D in his new role as CTO (Chief Technology Officer).

    NIJMEGEN, Netherlands, May 18, 2020 /PRNewswire/ -- GATT Technologies (the Company), a medical device company founded in 2011, developed a breakthrough patented synthetic technology intended to address severe and problematic surgical bleedings and organ leakages. The solution it has developed has been tested on animals and based on current tests shows superior results as compared against other products currently in the market in terms of speed, efficacy, and costs. Proceeds of the investment will be used to support the next clinical trial phase and further development of applications of the Company's breakthrough hemostatic technology platform. The Company expects to start clinical trials in Q2 2020.

    "The addition of Geert van Gansewinkel and Vlad Hogenhuis to the team and the further strengthening of our financial position marks a significant milestone for our Company. We are now extremely well positioned to move our first product, GATT-Patch, into clinical trials and further develop our other pipeline products and innovations," said Johan Bender.

    "I am excited to join the GATT Technologies team," said Geert van Gansewinkel. "I look forward to working with our management team, our board and our investors to build our Company and to develop a range of innovative products based on GATT's platform technology that will support saving patient lives in surgery. I was convinced that this is a revolutionary new product after reviewing the surgeon write ups who used the GATT products in a lab," said van Gansewinkel.

    About GATT Technologies

    GATT Technologies' hemostats and sealants focus on controlling severe surgical bleedings as well as preventing and treating organ leakages. Leveraging its patented technology, the Company has developed a synthetic surgical and sealant platform with the potential to become a superior alternative to the current fibrin and PEG-polymer based solutions in terms of speed, efficacy and cost. Currently, the Company is focusing on moving the GATT-Patch, a fast hemostatic sealing patch to be used in general surgery, into clinical trials in Europe and the United States. Additionally, it is further developing its other pipeline products including GATT-Powder (hemostat, laparoscopic use) and GATT-Tape (intestinal anastomotic leakage) and is assessing other potential applications based on its activated polyoxazoline technology such as bone regeneration, anti-adhesion, vascular, and ophthalmology.

    About Geert van Gansewinkel

    Geert is a senior executive with more than 20 years of experience in strategy consulting, entrepreneurship and general management. He worked as a technology and strategy consultant for Accenture and The Boston Consulting Group. In 2013, he became entrepreneur and partner at life sciences compliance software company Polaris, building and managing the European and Asian businesses. He played a key role in the sale of the business to IQVIA in 2017, where he continued working in various roles, initially global commercial head for compliance solutions, owning global sales, marketing and customer success, and most recently general manager for the Dutch business. Geert holds a Master of Science degree in International Business Administration from the University of Maastricht, and an MBA degree from IESE in Barcelona. Geert has assumed his role as CEO with GATT Technologies effective March 1st, 2020.

    About Vlad Hogenhuis

    Vlad Hogenhuis, MD, was most recently the Chief Operating Officer of Ultragenyx, Pharmaceutical Inc. (NASDAQ:RARE), a rare disease company, where he was responsible for global commercial operations, business development and manufacturing. Before Ultragenyx, he held leadership positions at GSK and Merck & Co, where he worked in a variety of operating and strategic leadership roles for 25 years. He also holds an MBA from Wharton School at the University of Philadelphia and a medical degree for the University of Leiden, The Netherlands. Dr. Vlad Hogenhuis has assumed his role as board member with GATT Technologies effective May 1st, 2020.

    About Oost NL

    Oost NL (East Netherlands Development Agency) is an agency that focuses its activities and projects on strengthening and stimulating the economy of the provinces of Gelderland and Overijssel, the Netherlands. Oost NL works with businesses in the provinces of Gelderland and Overijssel and is commissioned by the Ministry of Economic Affairs and Climate Policy and the respective Provinces.

    For more information, please visit the web site on www.gatt-tech.com.

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  18. All Patients in Cohort 3 Demonstrate Increased Time to Hypoglycemia and a Substantial Reduction in Cornstarch Use

    Introduction of Continuous Glucose Monitoring Reveals Early Transgene Expression and Enables More Rapid Reduction in Cornstarch

    Ultragenyx to Host ASGCT Recap Investor Conference Call today at 8:30 a.m. Eastern Time

    NOVATO, Calif., May 15, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced positive initial data from the confirmatory third cohort and longer-term data from the first two cohorts of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus (AAV…

    All Patients in Cohort 3 Demonstrate Increased Time to Hypoglycemia and a Substantial Reduction in Cornstarch Use

    Introduction of Continuous Glucose Monitoring Reveals Early Transgene Expression and Enables More Rapid Reduction in Cornstarch

    Ultragenyx to Host ASGCT Recap Investor Conference Call today at 8:30 a.m. Eastern Time

    NOVATO, Calif., May 15, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced positive initial data from the confirmatory third cohort and longer-term data from the first two cohorts of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus (AAV) based gene therapy for the treatment of glycogen storage disease type Ia (GSDIa). All three patients in Cohort 3 responded to therapy and demonstrated more rapid reductions in cornstarch requirements compared to the first two cohorts. Across all cohorts, 100 percent of patients (n=9) demonstrated meaningful and sustained cornstarch reductions over time and significant increases in time to hypoglycemia. Data from the Phase 1/2 study are being presented today at the American Society of Gene & Cell Therapy (ASGCT) virtual meeting.

    "In the confirmatory third cohort, continuous glucose monitoring showed early transgene expression and enabled faster and more accurate reductions in cornstarch compared with prior cohorts," said Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit. "The use of continuous glucose monitoring offers the ability to better understand the improvement in glucose metabolism after treatment with DTX401 over the course of the entire day and on an ongoing basis."

    "We are extremely excited by the results being obtained in this first in human gene therapy trial for glycogen storage disease type Ia," said David Weinstein, M.D., M.M.Sc., Professor and Director, Glycogen Storage Disease Program at Connecticut Children's Medical Center and UConn Health. "The third cohort of patients is experiencing more rapid improvement with fasting and tolerating quicker reduction in their cornstarch therapy when compared with the prior cohorts.  Most importantly, the benefits seen for patients are being maintained, and the majority of treated patients are now off of cornstarch therapy during the day after one year of treatment. In 26 years of caring for people with GSD, I have never seen this before, and the community is excited for the Phase 3 trial to commence."

    Confirmatory Cohort 3 Amendments
    Based on results and lessons learned from Cohorts 1 and 2, three modifications were made to the protocol for the confirmatory Cohort 3. First, the cornstarch dose at the start of each controlled fasting challenge was reduced from 35 grams to 5 grams to reduce an acute rise in glucose followed by an insulin surge, resulting in early termination of the fasting challenge. Second, continuous glucose monitoring (CGM) was implemented to allow more accurate cornstarch management. Third, the reactive use of steroids was optimized to begin steroids earlier, when alanine aminotransferase (ALT) levels increase from baseline, even if still within the normal range.

    Efficacy Data Summary
    Early Transgene Expression Demonstrated in Confirmatory Cohort 3
    In Cohort 3, CGM data showed early transgene expression within days of dosing with DTX401. This was demonstrated by prolonged periods of hyperglycemia, which is consistent with glucose being released from the liver and transgene expression.

    Rapid Cornstarch Reduction in Cohort 3 and Further Reduction in Earlier Cohorts
    In the confirmatory cohort, CGM enabled more rapid reduction of cornstarch intake following observations of hyperglycemia at home, which minimized the insulin response and provided more metabolic stability for patients. At Week 12, patients in Cohort 3 had reduced mean daily cornstarch intake by 57 percent, compared with 38 percent in the first cohort and 14 percent in the second cohort.

    Patients in Cohort 1 and 2 continue to decrease cornstarch with a mean 73 percent reduction as of the data cutoff of April 23, 2020, demonstrating durable responses over time. Four of the six patients in the first two cohorts have now discontinued daytime cornstarch, and one patient has completely discontinued cornstarch.

    Optimized Reactive Steroids More Effectively Mitigated Elevations in Liver Transaminases
    Patients in Cohort 3 received an optimized reactive steroid regimen as soon as alanine aminotransferase (ALT) levels began to rise above baseline, at approximately Week 4 for all three patients in the cohort. This earlier steroid use was more effective at mitigating elevations in ALT, and could further enhance the level and consistency of expression that has been demonstrated.

    Early Improvements in Time to Hypoglycemia Observed for All Patients
    By decreasing the cornstarch at the beginning of the controlled fasting challenge, the mean baseline time to hypoglycemia decreased from 4.5 hours in Cohorts 1 and 2, to 2.3 hours in Cohort 3, with two patients in Cohort 3 under 2 hours. At week 12, all patients in Cohort 3 demonstrated meaningful increases in time to hypoglycemia. Patient 7 demonstrated a 167 percent increase (1.4 hours at baseline to 3.7 hours at Week 12). Patient 8 demonstrated a 23 percent increase (3.6 hours at baseline to 4.4 hours at Week 12). Patient 9 demonstrated a 60 percent increase (1.9 hours at baseline to 3.1 hours at Week 12).

    Due to the COVID-19 pandemic, only Week 12 controlled fasting challenges have been conducted to date for Cohort 3, as patients were not able to complete the Week 24 in-hospital fasting challenges.  It will be important to monitor these patients as they adapt to their lower cornstarch regimen to continue to assess their glucose metabolism.

    Safety Summary
    As of the cutoff date, there have been no infusion-related adverse events and no treatment-related serious adverse events reported. All adverse events have been Grade 1 or 2. Seven of the nine patients in the study had mild, asymptomatic ALT elevations, including all three patients in Cohort 3.  These elevations were similar to what has been observed in other programs using AAV-based gene therapy, and were successfully treated with reactive steroids. The optimized reactive steroid regimen used in Cohort 3 was more effective at rapidly mitigating elevations in ALT compared with earlier cohorts.  

    DTX401 Phase 3 Study
    Ultragenyx is continuing to collect longer-term data from the Phase 1/2 study, which is expected to be available in the second half of 2020, barring further significant delays related to COVID-19. The company also intends to hold an end-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) in the second half of 2020. The Phase 3 study could begin by the end of this year, depending on whether there are any COVID-19 related delays.

    Conference Call and Webcast Information
    Ultragenyx will host a conference call today, Friday May 15, 2020, at 8:30 a.m. ET/ 5:30 a.m. PT during which Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will discuss the new data from the DTX301 and DTX401 studies presented at ASGCT. The live and replayed webcast of the call and slides will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 2366186. The replay of the call will be available for one year.

    DTX401 Phase 1/2 Study Design
    The open-label, multicenter Phase 1/2 study is evaluating the safety, tolerability and therapeutic response of DTX401 in adults with GSDIa. DTX401 is an AAV8 gene therapy expressing the glucose-6-phosphatase gene (G6Pase-α) under control of the native promoter. In the first cohort, three patients received a single 2.0 × 10^12 GC/kg dose of DTX401. In the second cohort, three patients each received a single 6.0 × 10^12 GC/kg dose of DTX401. In the confirmatory cohort, three patients received the same dose of DTX401 as in Cohort 2. Modifications were made to the protocol for the confirmatory cohort, including: reduction of cornstarch dose at the start of the controlled fasting challenge (decreased from 35 g to 5 g); use of CGM; implementation of an optimized reactive steroid regimen. Key efficacy assessments include time to hypoglycemia (defined as glucose <60 mg/dL or onset of clinical symptoms) during a controlled in-hospital fasting challenge, cornstarch reduction, impact on biomarkers such as lactic acid, and measurement of glycogen storage in liver by MRI.

    About GSDIa
    GSDIa is the most severe genetically inherited glycogen storage disease. It is caused by a defective gene for the enzyme G6Pase-α, resulting in the inability to regulate blood sugar (glucose). Hypoglycemia in patients with GSDIa can be life-threatening, while the accumulation of the complex sugar glycogen in certain organs and tissues can impair the ability of these tissues to function normally. If chronically untreated, patients can develop severe lactic acidosis, progress to renal failure, and potentially die in infancy or childhood. There are no approved pharmacologic therapies. An estimated 6,000 patients worldwide are affected by GSDIa.

    About DTX401

    DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-α under control of the native promoter. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase-α activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. DTX401 has been granted Orphan Drug Designation in both the United States and Europe, and Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation in the United States.

    About Ultragenyx
    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Forward-Looking Statements
    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the effects from the COVID-19 pandemic on the company's clinical trial activities, business and operating results, smaller than anticipated market opportunities for the company's products and product candidates, manufacturing risks, the uncertainties inherent in the clinical drug development process, including the potential for substantial delays and the risk that earlier study results may not be predictive of future study results, the lack of predictability in the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 7, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

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  19. All three patients in Cohort 3 now confirmed responders

    All three complete responders in the study remain clinically and metabolically stable after longer-term follow-up

    Ultragenyx to host ASGCT recap investor conference call Friday, May 15 at 8:30 a.m. Eastern Time

    NOVATO, Calif., May 13, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced positive longer-term safety and efficacy data from the first three cohorts of the ongoing Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency…

    All three patients in Cohort 3 now confirmed responders

    All three complete responders in the study remain clinically and metabolically stable after longer-term follow-up

    Ultragenyx to host ASGCT recap investor conference call Friday, May 15 at 8:30 a.m. Eastern Time

    NOVATO, Calif., May 13, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced positive longer-term safety and efficacy data from the first three cohorts of the ongoing Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. Six of nine patients in the study have responded to the gene therapy (three female, three male), including all three patients in Cohort 3 who are now confirmed responders. The three previously disclosed complete responders, who have discontinued all ammonia scavengers and liberalized their diet, remain clinically and metabolically stable after longer-term follow-up. Data from the Phase 1/2 study were presented today at the American Society of Gene & Cell Therapy (ASGCT) virtual 2020 Annual Meeting.

    "We are seeing durable and clinically meaningful responses to DTX301. We are extremely encouraged that the patients who have stopped alternate pathway medications and liberalized dietary restrictions continue to do very well over these longer periods of time," said Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit. "The recent data also reinforces the higher response rate seen with the Cohort 3 dose of 1x10^13 GC/kg. All three patients in Cohort 3 have responded to DTX301 and the treating physician of one of these patients reported that this is the best she has been health wise. This dose has been selected for the Phase 3 study, which is currently expected to initiate in the first half of 2021."

    Cohort 3 Updated Data: All three patients at 1x1013 GC/kg dose now confirmed responders

    Patient 9 (newly confirmed responder, male):
    Patient 9 showed a 188 percent increase in rate of ureagenesis, from 25 percent of normal at baseline to 73 percent of normal at Week 24. He now has shown two significantly increased ureagenesis measures and is confirmed as a responder. His ammonia levels have remained in the normal range since treatment. He has not yet discontinued alternate pathway medications or liberalized his diet.

    Patient 8 (previously disclosed responder, female):
    As previously disclosed, Patient 8 has experienced a significant and sustained reduction in her elevated baseline ammonia levels. This patient has now increased protein intake and has discontinued one of her two ammonia scavenger medications. She continues to do well clinically and the plan is to begin to taper her second alternate pathway medication once she is able to return to clinic post COVID-19 restrictions.

    Patient 7 (previously disclosed complete responder, female):
    As previously disclosed, Patient 7 demonstrated a meaningful change in rate of ureagenesis and has maintained normal ammonia levels since treatment. She continues to be clinically and metabolically stable after liberalizing diet and discontinuing ammonia scavenger medications.

    Cohorts 1 and 2 Long-term Data Show Sustained Efficacy
    The two complete responders in Cohorts 1 and 2 (Patients 1 and 4) have shown ongoing durable responses for 2 years and 1.5 years, respectively. Their ureagenesis rates remain above 100% of normal. Both patients have discontinued alternate pathway medications and liberalized restricted protein diets for more than one year, and remain stable with ammonia levels maintained in the normal range. They remain in excellent clinical condition with no significant adverse events, hospitalizations, or other events related to OTC deficiency. The third responder from the earlier cohorts (Patient 6) continues to do well, and is currently tapering her medications and liberalizing her diet. Her increase in ureagenesis and normalization of ammonia have been maintained.

    Safety Summary
    As of the data cutoff date, there have been no infusion-related adverse events and no treatment-related serious adverse events reported in the study. All adverse events have been Grade 1 or 2. As previously reported, six patients experienced mild, clinically asymptomatic elevations in ALT levels, similar to what has been observed in other programs using AAV-based gene therapy.  All six of these patients have responded to reactive tapering courses of steroids, and all patients remain clinically stable.

    Prophylactic Steroid Cohort Planned
    A fourth cohort of three patients at the Cohort 3 dose (1.0 × 10^13 GC/kg) is planned, using prophylactic steroids. Dosing in this cohort is currently on hold due to the COVID-19 pandemic, but data are still expected in the second half of 2020, barring further delays related to clinical site closures due to COVID-19.

    Phase 3 Study Planning Underway
    Ultragenyx is currently planning the Phase 3 study of DTX301 in parallel to conducting the prophylactic steroid cohort. The Company intends to hold an end of Phase 2 meeting with the U.S. Food and Drug Administration (FDA) in the second half of 2020, based on data from the first three cohorts of the Phase 1/2 study. The use of prophylactic steroids in the Phase 3 study is anticipated, and will be confirmed by the Phase 1/2 fourth cohort data once available. For the Phase 3 study, ammonia is expected to be a primary endpoint based on direct FDA feedback to date. The Phase 3 study is currently expected to begin enrollment in the first half of 2021, barring any significant delays due to COVID-19.

    Conference Call and Webcast Information
    Ultragenyx will host a conference call on Friday, May 15, 2020, at 8:30 a.m. ET/ 5:30 a.m. PT during which Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will discuss the new data from the DTX301 and DTX401 studies presented at ASGCT. The live and replayed webcast of the call and slides will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 2366186. The replay of the call will be available for one year.

    About the OTC Phase 1/2 Study (DTX301)
    The Phase 1/2 dose-escalation study evaluates the change in the rate of ureagenesis, ammonia levels, neurocognitive assessment, biomarkers, and safety of DTX301 in adult patients with OTC deficiency. Three patients have been dosed in each of three dose cohorts of 2.0 × 10^12 GC/kg (Cohort 1), 6.0 × 10^12 GC/kg (Cohort 2), and 1.0 × 10^13 GC/kg (Cohort 3). Patients in the first three cohorts received steroids to reactively manage ALT elevations. In the fourth cohort, three patients will receive a 1.0 × 10^13 GC/kg dose and will all receive a prophylactic tapering course of steroids.

    About OTC Deficiency
    OTC deficiency, the most common urea cycle disorder, is caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. Individuals with OTC deficiency can build up excessive levels of ammonia in their blood, potentially resulting in acute and chronic neurological deficits and other toxicities. It is estimated that more than 10,000 people are affected by OTC deficiency worldwide, of whom approximately 80 percent are classified as late-onset and represent a clinical spectrum of disease severity. In the late-onset form of the disease, elevated ammonia can lead to significant medical issues for patients. Neonatal onset disease occurs only in males, presents as severe disease, and can be fatal at an early age. Approved therapies, which must be taken multiple times a day for the patient's entire life, do not eliminate the risk of future metabolic crises. Currently, the only curative approach is liver transplantation.

    About DTX301
    DTX301 is an investigational AAV type 8 gene therapy designed to deliver stable expression and activity of OTC following a single intravenous infusion. It has been shown in preclinical studies to normalize levels of urinary orotic acid, a marker of ammonia metabolism. DTX301 was granted Orphan Drug Designation in both the United States and Europe.

    About Ultragenyx
    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com

    Forward-Looking Statements
    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the effects from the COVID-19 pandemic on the company's clinical trial activities, business and operating results, smaller than anticipated market opportunities for the company's products and product candidates, manufacturing risks, the uncertainties inherent in the clinical drug development process, including the potential for substantial delays and the risk that earlier study results may not be predictive of future study results, the lack of predictability in the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance, the timing of clinical trial activities  and reporting results from same, and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 7, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

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  20. NOVATO, Calif., May 11, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the BofA Securities Global Health Care Conference on Thursday, May 14, 2020 at 10:20 AM ET.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment…

    NOVATO, Calif., May 11, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the BofA Securities Global Health Care Conference on Thursday, May 14, 2020 at 10:20 AM ET.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at:  www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

    Primary Logo

    View Full Article Hide Full Article
  21. First quarter 2020 total revenue is $36.3 million

    First quarter 2020 Crysvita revenue in Ultragenyx territories is $28.8 million

    Maintains full year 2020 guidance based on COVID-19 impact to date

    NOVATO, Calif., May 06, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended March 31, 2020 and maintained its full year 2020 financial guidance.

    "The global coronavirus pandemic has created unprecedented challenges for the world at large, and I am proud of the company's continued efforts to assure an uninterrupted supply of our therapies" said Emil D…

    First quarter 2020 total revenue is $36.3 million

    First quarter 2020 Crysvita revenue in Ultragenyx territories is $28.8 million

    Maintains full year 2020 guidance based on COVID-19 impact to date

    NOVATO, Calif., May 06, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended March 31, 2020 and maintained its full year 2020 financial guidance.

    "The global coronavirus pandemic has created unprecedented challenges for the world at large, and I am proud of the company's continued efforts to assure an uninterrupted supply of our therapies" said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We delivered strong financial results in the first quarter, and our commercial business so far has been manageably impacted and resilient, though the global crisis continues. The FDA reviews for Crysvita for TIO and UX007 for LC-FAOD are on track to be completed in the coming months. Our gene therapy franchise has recently been bolstered by our recent manufacturing collaboration with Daiichi Sankyo, and our two clinical-stage gene therapy programs continue to proceed."

    COVID-19 Update
    As the COVID-19 pandemic continues to evolve, the top priority for Ultragenyx is the health and safety of its extended community, including patients, caregivers, healthcare providers, partners, and employees. To date, the company has been able to maintain an uninterrupted supply of medicine to patients around the world as no significant disruptions to manufacturing or distribution activities have been experienced. Even if there were an interruption in the supply chain, the company does not anticipate a shortage of medicine in the near term due to ample levels of inventory currently available.

    First Quarter 2020 Financial Results

    Net Revenues
    For the first quarter of 2020, Ultragenyx reported $36.3 million in total revenue. Ultragenyx recognized $28.8 million in total Crysvita revenue for Ultragenyx territories, which includes $27.2 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $1.6 million. Non-cash royalty revenue related to European Crysvita royalties were $2.6 million. Mepsevii® (vestronidase alfa) product revenue for the first quarter of 2020 was $3.4 million, and UX007 named patient revenue was $1.4 million.

    Operating Expenses
    Total operating expenses for the first quarter of 2020 were $157.0 million, which includes $25.0 million to maintain the option to acquire GeneTx after the acceptance of the investigational new drug application (IND) for GTX-102, $7.0 million for an exclusive, sublicensable, worldwide license to NAV® AAV8 and AAV9 Vectors from REGENXBIO, Inc and non-cash stock-based compensation of $20.2 million. The cost of sales for the three months ended March 31, 2020 was negative $3.5 million due to a credit with an estimated value of $4.6 million that was agreed to during the quarter with a contract manufacturer in exchange for certain Mepsevii inventory batches that did not meet specified quality standards in prior quarters. Total operating expenses in the first quarter of 2019 were $117.4 million, which includes non-cash stock-based compensation of $20.2 million. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows, and advances its portfolio.

    For the first quarter of 2020, Ultragenyx reported a net loss of $119.0 million, or $2.05 per share, basic and diluted, compared with a net loss for the first quarter of 2019 of $96.8 million, or $1.82 per share, basic and diluted. The loss for the first quarter of 2020 includes unrealized gains of $7.7 million from the fair value adjustments on the investment in Arcturus equity securities and non-cash interest expense on the liability related to the sale of future royalties of $8.1 million. The net loss for the first quarter of 2020 reflected cash used in operations of $95.2 million compared to $95.8 million for the same period in 2019.

    Cash, Cash Equivalents and Investments
    Cash, cash equivalents, and investments were $705.0 million as of March 31, 2020. This includes $75.0 million from the Daiichi Sankyo common stock purchase which closed in March 2020, but excludes the $125.0 million upfront license payment from the Daiichi Sankyo strategic manufacturing partnership which was received in April 2020.

    2020 Guidance

    Crysvita Guidance in Ultragenyx Territories
    The company currently maintains the guidance range for 2020 Crysvita revenue in the Ultragenyx territories to be between $125.0 million and $140.0 million, although it continues to monitor the COVID-19 pandemic situation. Ultragenyx territories include the collaboration revenue from the North American profit share territory (U.S. and Canada) and other regions where revenue from product sales are recognized by Ultragenyx (Latin America, Turkey). This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.

    2020 Expected Net Cash Burn Rate
    The company also expects a more than 20 percent reduction in net cash burn (net cash used in operations plus capital expenditures) in 2020 compared to 2019 due to a combination of financial discipline in spending with flattening operating expense growth, combined with a significant anticipated growth in revenue.

    Corporate Update

    Gene Therapy Manufacturing: Strategic partnership and non-exclusive license and technology access agreement with Daiichi Sankyo

    • Ultragenyx granted Daiichi Sankyo a non-exclusive license on March 31, 2020 to intellectual property, including know-how and patent applications, with respect to its HeLa PCL and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products. Daiichi Sankyo made an upfront payment in April 2020 of $125.0 million pursuant to the license agreement and in March 2020 closed on the purchase of 1,243,913 shares of Ultragenyx common stock in exchange for $75.0 million, or an effective purchase price of approximately $60 per share. Daiichi Sankyo will pay an additional $25.0 million upon completion of the technology transfer of the HeLa PCL and HEK293 platforms, as well as single-digit royalties on net sales of products manufactured in either system.

    Program Updates and Upcoming Milestones

    UX007 for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD): New Drug Application (NDA) under review by U.S. FDA

    • The U.S. FDA is currently reviewing the NDA and has set a Prescription Drug User Fee Act (PDUFA) date of July 31, 2020. The review process is currently on track for a decision by the PDUFA date. The anticipated clinical and manufacturing inspections for UX007 have been successfully completed and, at this time, the FDA has not indicated that any additional inspections would be required for completion of their review.

    Crysvita for Tumor-Induced Osteomalacia (TIO): Supplemental Biologics License Application (sBLA) under review by U.S. FDA

    • The U.S. FDA accepted for review the sBLA and has set a PDUFA date of June 18, 2020. The review process is currently on track for a decision by the PDUFA date and the FDA has not indicated, at this time, that any inspections would be required for completion of their review.

    DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Positive data from higher dose cohort of Phase 1/2 study; prophylactic steroid cohort planned and Phase 3 study planning underway

    • Positive data from this study indicated two confirmed responders and a potential third responder out of three total patients in Cohort 3, as well as a new responder in Cohort 2. There are currently up to six responders of the nine dosed to date with a more consistent response at higher doses. An update with currently available data from the first three cohorts of the study will be presented at the virtual American Society of Gene & Cell Therapy (ASGCT) on May 13.
    • A fourth cohort of three patients at the same 1.0 × 10^13 GC/kg dose is planned, using prophylactic steroids. Dosing in this cohort is currently on hold, but data are still expected in the second half of 2020, barring significant further delays related to COVID-19. Ultragenyx is simultaneously planning the Phase 3 study and is currently planning to hold an end-of-phase 2 meeting with the FDA based on the first three cohorts. The Phase 3 study is currently expected to begin enrollment in the first half of 2021.

    DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Positive data from Phase 1/2 study; enrollment complete in confirmatory cohort and data expected in second quarter 2020

    • Enrollment is complete in the confirmatory cohort of three patients at the second dose level (6.0 x 10^12 GC/kg). An update with currently available data from the confirmatory cohort will be presented at the ASGCT meeting on May 15. Certain data may not be available for later patient site visits due to COVID-19 related delays. Depending on the results of the confirmatory cohort, a Phase 3 study could begin in late 2020.

    GTX-102 for Angelman Syndrome (AS): Partnered program with GeneTx; First two patients enrolled

    • The two patients in the first cohort of the Phase 1/2 study of GTX-102 have been enrolled and continue through dose escalation. Preliminary data are expected in first half of 2021.
    • In February 2020, Ultragenyx paid $25.0 million after the acceptance of the IND to maintain its option to acquire GeneTx until the earlier of 30 months from the first dosing of a patient in the Phase 1/2 study (subject to extensions) or 90 days after results are available from that study.
    • In May 2020, the U.S. FDA granted Fast Track designation to GTX-102 for the treatment of Angelman syndrome.

    UX701 for Wilson Disease: IND anticipated by the end of the year

    • An IND application for UX701 gene therapy for Wilson disease, a larger rare metabolic disease, is expected by the end of 2020 barring any delays in nonclinical or manufacturing activities. UX701 will be the second clinical program to utilize the company's HeLa manufacturing system, in addition to the hemophilia A program being developed by our collaborative partner, Bayer.

    Conference Call and Webcast Information

    Ultragenyx will host a conference call today, Wednesday, May 6, 2020, at 2 p.m. PT/ 5 p.m. ET to discuss first quarter 2020 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 9455159. The replay of the call will be available for one year.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, Ultragenyx's reliance on its third party partner, Kyowa Kirin Co. ,Ltd., for the supply of Crysvita, the effects from the COVID-19 pandemic on the company's business and operating results, smaller than anticipated market opportunities for the company's products and product candidates, the company's evolving commercial infrastructure, uncertainties related to insurance coverage and reimbursement approval for the company's products, manufacturing risks, the uncertainties inherent in the clinical drug development process, including the potential for substantial delays and the risk that earlier study results may not be predictive of future study results, the lack of predictability in the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 14, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Ultragenyx Pharmaceutical Inc.
    Selected Statement of Operations Financial Data
    (in thousands, except share and per share amounts)
    (unaudited)
             
      Three Months Ended March 31,  
        2020       2019    
    Statement of Operations Data:        
    Revenues:        
    Collaboration and license $ 27,215     $ 14,238    
    Product sales   6,479       3,934    
    Non-cash collaboration royalty revenue   2,615          
    Total revenues   36,309       18,172    
    Operating expenses:        
    Cost of sales   (3,503 )     452    
    Research and development   112,961       78,105    
    Selling, general and administrative   47,516       38,829    
    Total operating expenses   156,974       117,386    
    Loss from operations   (120,665 )     (99,214 )  
    Change in fair value of investment in Arcturus equity
      securities
      7,668          
    Non-cash interest expense on liability related to the sale
      of future royalties
      (8,082 )        
    Other income (expense), net   2,463       2,674    
    Loss before income taxes   (118,616 )     (96,540 )  
    Provision for income taxes   (409 )     (216 )  
    Net loss $ (119,025 )   $ (96,756 )  
    Net loss per share, basic and diluted $ (2.05 )   $ (1.82 )  
    Shares used in computing net loss per share, basic and
      diluted
      57,995,999       53,209,215    
             


    Ultragenyx Pharmaceutical Inc.
    Selected Balance Sheet Financial Data
    (in thousands)
    (unaudited)
        March 31,   December 31,
          2020       2019  
    Balance Sheet Data:        
    Cash, cash equivalents, and available-for-sale investments   $ 704,989     $ 760,404  
    Working capital     697,460       747,717  
    Total assets     1,233,140       1,135,496  
    Total stockholders' equity     609,146       653,764  
             

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

    Primary Logo

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  22. SARASOTA, Fla. and NOVATO, Calif., May 04, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to GTX-102 for the treatment of Angelman syndrome (AS). GTX-102 is an investigational antisense oligonucleotide currently being evaluated in a Phase 1/2 study for the treatment of AS.

    "GeneTx is committed to improving the quality of life of individuals living with Angelman syndrome," said Paula Evans, Chief Executive Officer, GeneTx.  "Receiving Fast Track designation…

    SARASOTA, Fla. and NOVATO, Calif., May 04, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to GTX-102 for the treatment of Angelman syndrome (AS). GTX-102 is an investigational antisense oligonucleotide currently being evaluated in a Phase 1/2 study for the treatment of AS.

    "GeneTx is committed to improving the quality of life of individuals living with Angelman syndrome," said Paula Evans, Chief Executive Officer, GeneTx.  "Receiving Fast Track designation for GTX-102 is an important recognition of our antisense oligonucleotide program and the promise it might offer to this patient population."

    About Fast Track Designation

    The FDA's Fast Track designation is a program designed to facilitate the development and expedite the review of drugs intended to treat serious conditions that demonstrate the potential to address an unmet medical need. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process, and allows for a rolling review of a company's New Drug Application (NDA). Products with Fast Track designation may also be eligible for Priority Review, if relevant criteria are met at the time of NDA filing.

    About Angelman Syndrome

    Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the intended target of GTX-102. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is typically not inherited but instead occurs spontaneously.

    Individuals with Angelman syndrome have developmental delay, balance issues, motor impairment, and debilitating seizures. Some individuals with Angelman syndrome are unable to walk and most do not speak. Anxiety and disturbed sleep can be serious challenges in individuals with Angelman syndrome. While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome suggesting that improvement of symptoms can potentially be achieved at any age.

    About GTX-102 and the Ongoing Phase 1/2 Study

    GTX-102 is an investigational antisense oligonucleotide designed to target and inhibit expression of UBE3A-AS. Nonclinical studies show that GTX-102 reduces the levels of UBE3A-AS and reactivates expression of the paternal UBE3A allele in neurons of the CNS. Reactivation of paternal UBE3A expression in animal models of Angelman syndrome has been associated with improvements in some of the neurological symptoms associated with the condition. GTX-102 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA). In August 2019, GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx.

    The Phase 1/2 KIK-AS (Knockdown of UBE3A-antisense in Kids with Angelman Syndrome) study is an ongoing open-label, multiple-dose, dose-escalating study will enroll 20 patients to evaluate the safety, tolerability, and potential efficacy of GTX-102 in pediatric patients with Angelman syndrome.  Preliminary data from the first cohorts of the study are expected in the first half of 2021. This is the first investigational study testing an antisense oligonucleotide as a potential therapy to treat AS.  Further details can be referenced at: https://clinicaltrials.gov/ct2/show/NCT04259281.

    About GeneTx Biotherapeutics

    GeneTx Biotherapeutics LLC is a startup biotechnology company singularly focused on developing and commercializing a safe and effective antisense therapeutic for the treatment of Angelman syndrome. GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research. GeneTx licensed the rights to antisense technology intellectual property from The Texas A&M University System in December 2017.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    Ultragenyx Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our partnership with GeneTx, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), the effects from the COVID-19 pandemic on clinical trial activities and other business operations, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report filed on Form 10-K with the Securities and Exchange Commission on February 14, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:

    GeneTx
    Paula Evans
    630-639-7271

    Ultragenyx
    Investors & Media
    Danielle Keatley
    415-475-6876

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  23. NOVATO, Calif., April 30, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Wednesday, May 6, 2020 at 5pm ET to discuss first quarter 2020 financial results and provide a corporate update.

    The live and replayed webcast of the call will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 9455159. The replay of the call will be available for one year.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx…

    NOVATO, Calif., April 30, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Wednesday, May 6, 2020 at 5pm ET to discuss first quarter 2020 financial results and provide a corporate update.

    The live and replayed webcast of the call will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 9455159. The replay of the call will be available for one year.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

    Primary Logo

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  24. NOVATO, Calif., April 28, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that clinical, preclinical and manufacturing data from its investigational gene therapy programs will be presented at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting, which will be held virtually May 12-15, 2020. Information will be available at www.asgct.org.

    Nine Ultragenyx abstracts have been accepted for virtual presentation, including:

    • An oral presentation of available data from the confirmatory cohort of the  Phase 1/2 study of DTX401, an AAV-based gene therapy for the treatment…

    NOVATO, Calif., April 28, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that clinical, preclinical and manufacturing data from its investigational gene therapy programs will be presented at the American Society of Gene & Cell Therapy (ASGCT) 23rd Annual Meeting, which will be held virtually May 12-15, 2020. Information will be available at www.asgct.org.

    Nine Ultragenyx abstracts have been accepted for virtual presentation, including:

    • An oral presentation of available data from the confirmatory cohort of the  Phase 1/2 study of DTX401, an AAV-based gene therapy for the treatment of glycogen storage disease Type Ia (GSDIa) (Abstract #1306)
    • An oral presentation of new data from the first three cohorts of the Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency (Abstract #505)
    • Multiple presentations, including two orals, highlighting the HeLa producer cell line platform and improvements in the HEK293 triple transfection system (Abstracts #539 and #543)

    "We have made meaningful progress across all of our gene therapy programs, including continued improvements to both the HeLa PCL and HEK293 transient transfection manufacturing technology platforms which we believe will enable highly reproducible, more consistent and scalable gene therapy manufacturing," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In addition to the presentations that support our gene therapy manufacturing approach, we look forward to sharing first-time results from the confirmatory cohort of our GSDIa study and updated clinical data from the OTC study."

    Details of the nine presentations are as follows:

    Tuesday, May 12
    Oral Presentation
    Abstract #98: AAV9/hCDKL5 delivery to cerebrospinal fluid of juvenile CDKL5-deficient mice improves learning and memory and motor function in adult mice

    • Time: 5:15-5:30 PM ET

    Poster Presentation
    Abstract #172: Characterization and correction of an in vitro model of Wilson Disease by recombinant adeno-associated virus (rAAV) delivered ATP7B transgene 

    • Time: 5:30-6:30 PM ET

    Poster Presentation
    Abstract #451: Rapid CMC development and pre-commercial considerations for rAAV gene therapy products for rare diseases 

    • Time: 5:30-6:30 PM ET

    Wednesday, May 13
    Oral Presentation
    Abstract #539: HeLa 3.0: CRISPR knockout of genes modulating titer in established rAAV-producing cell lines 

    • Time: 4:00-4:15 PM ET

    Oral Presentation
    Abstract #505: AAV8 gene therapy as a potential treatment in adults with late-onset OTC deficiency: results from a Phase 1/2 clinical trial

    • Time: 4:15-4:30 PM ET

    Oral Presentation
    Abstract #543: Characterization of phenotypic and genotypic stability of rAAV producing HeLa cell lines

    • Time: 4:45-5:00 PM ET

    Poster Presentation
    Abstract #586: Gene therapy for Wilson Disease using rAAV to restore ATP7B gene function 

    • Time: 5:30-6:30 PM ET

    Thursday, May 14
    Poster Presentation
    Abstract #1008: Elongation of the Rep-Cap cassette with a cellular intron reduces reverse-packaged Rep-Cap trans plasmid sequences and increases therapeutic vector genome packaging in a HEK293 triple transfection rAAV vector production system

    • Time: 5:30-6:30 PM ET

    Friday, May 15
    Oral Presentation
    Abstract #1306: AAV8-mediated liver-directed gene therapy as a potential therapeutic option in adults with glycogen storage disease type Ia (GSDIa): results from a Phase 1/2 clinical trial

    • Time: 10:30-10:45 PM ET

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    Forward-Looking Statements
    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), the effects from the COVID-19 pandemic on clinical trial activities and other business operations, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report filed on Form 10-K with the Securities and Exchange Commission on February 14, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:

    Ultragenyx
    Investors & Media
    Danielle Keatley
    415-475-6876

     

    Primary Logo

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  25. Daiichi Sankyo granted non-exclusive license to Ultragenyx HeLa manufacturing platform

    Ultragenyx to receive $200 million upfront, including $125 million in cash and $75 million via equity investment

    NOVATO, Calif., March 31, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced a strategic partnership and non-exclusive license and technology access agreement with Daiichi Sankyo Company, Limited for Ultragenyx's proprietary AAV-based gene therapy manufacturing technologies. Ultragenyx's HeLa producer cell line (PCL) platform enables large commercial-scale AAV-based gene therapy…

    Daiichi Sankyo granted non-exclusive license to Ultragenyx HeLa manufacturing platform

    Ultragenyx to receive $200 million upfront, including $125 million in cash and $75 million via equity investment

    NOVATO, Calif., March 31, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced a strategic partnership and non-exclusive license and technology access agreement with Daiichi Sankyo Company, Limited for Ultragenyx's proprietary AAV-based gene therapy manufacturing technologies. Ultragenyx's HeLa producer cell line (PCL) platform enables large commercial-scale AAV-based gene therapy product manufacturing that is intended to be highly reproducible, more consistent, and less expensive than other gene therapy manufacturing platforms. In addition, Ultragenyx has developed a proprietary HEK293 transient transfection system for AAV manufacture which is also a subject of the collaboration.

    Under the license and technology access agreement, Ultragenyx granted Daiichi Sankyo a non-exclusive license to intellectual property, including know-how and patent applications, with respect to its HeLa PCL and HEK293 transient transfection manufacturing technology platforms for AAV-based gene therapy products. The parties will collaborate closely as part of a technology transfer plan to enable Daiichi Sankyo to use the technologies for its internal gene therapy programs. Ultragenyx retains the exclusive right to use its manufacturing technology for its current target indications and additional indications identified now and in the future.

    "This new partnership with Daiichi Sankyo provides further validation of the value of Ultragenyx's gene therapy-related technologies, especially our HeLa producer cell line platform that we believe is the most scalable mammalian cell AAV manufacturing system," said Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx. "We are encouraged that our proprietary technologies continue to enable the development of numerous programs, both internally and for our partners, to treat patients with serious unmet medical needs."

    "We are currently doing discovery research for gene therapy drugs using AAV vectors as one of our focused modalities toward sustained growth beyond achievement of our 2025 vision," said Masayuki Yabuta, PhD, Executive Officer, Head of Biologics Division, Daiichi Sankyo. "In order to provide these drugs to patients in the future, manufacturing technology must be established early. Ultragenyx's proprietary technology is particularly excellent in terms of stable quality, high production efficiency, and ability to accommodate mass production."

    Daiichi Sankyo will be responsible for the manufacture, development, and commercialization of products manufactured with the Ultragenyx technology, except that Ultragenyx receives an option to co-develop and co-commercialize rare disease products at the IND stage. Ultragenyx will also provide strategic consultation to Daiichi Sankyo on the development of both AAV-based gene therapy products and other products for rare diseases.

    Under the terms of the agreements, Daiichi Sankyo will make an upfront payment of $125 million and will purchase $75 million of Ultragenyx common stock at a price of approximately $60 per share. Daiichi Sankyo will pay an additional $25 million upon completion of the technology transfer of the HeLa PCL and HEK293 platforms as well as single-digit royalties on net sales of products manufactured in either system. Daiichi Sankyo will reimburse Ultragenyx for all costs associated with the transfer of the manufacturing technology.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    About Daiichi Sankyo

    Daiichi Sankyo Group is dedicated to the creation and supply of innovative pharmaceutical therapies to improve standards of care and address diversified, unmet medical needs of people globally by leveraging our world-class science and technology. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 15,000 employees around the world draw upon a rich legacy of innovation and a robust pipeline of promising new medicines to help people. In addition to a strong portfolio of medicines for cardiovascular diseases, under the Group's 2025 Vision to become a "Global Pharma Innovator with Competitive Advantage in Oncology," Daiichi Sankyo is primarily focused on providing novel therapies in oncology, as well as other research areas centered around rare diseases and immune disorders. For more information, please visit: www.daiichisankyo.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report filed on Form 10-K with the Securities and Exchange Commission on February 14, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:

    Ultragenyx
    Investors & Media
    Danielle Keatley
    415-475-6876

    Primary Logo

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  26. - REGENXBIO grants Ultragenyx exclusive, worldwide rights to NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder
    - Agreement for use of NAV Vectors expands ongoing relationship between Ultragenyx and REGENXBIO

    ROCKVILLE, Md. and NOVATO, Calif., March 31, 2020 (GLOBE NEWSWIRE) -- REGENXBIO Inc. (NASDAQ:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced…

    - REGENXBIO grants Ultragenyx exclusive, worldwide rights to NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder
    - Agreement for use of NAV Vectors expands ongoing relationship between Ultragenyx and REGENXBIO

    ROCKVILLE, Md. and NOVATO, Calif., March 31, 2020 (GLOBE NEWSWIRE) -- REGENXBIO Inc. (NASDAQ:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced a new exclusive, worldwide license agreement, extending the companies' existing gene therapy partnership.

    Under the terms of the agreement, REGENXBIO has granted Ultragenyx an exclusive, worldwide license, with rights to sublicense, to REGENXBIO's NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder. In return for these rights, REGENXBIO will receive an upfront payment of $7 million, ongoing fees, milestone payments, and royalties on net sales of products incorporating the licensed intellectual property.

    "We are pleased to expand our long-standing relationship with Ultragenyx through this new license agreement, which will enable Ultragenyx to apply our proprietary NAV Vectors to the development of a new gene therapy for rare diseases," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "Today's announcement provides further validation of the breadth and depth of our intellectual property portfolio, and we remain committed to working closely with leading companies to enable the development of important gene therapy programs."

    "This latest license agreement reinforces Ultragenyx's commitment to developing gene therapies for multiple rare diseases and adds a new indication to our relationship with REGENXBIO," said Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx.

    Ultragenyx is a longstanding licensee partner of REGENXBIO. In addition to today's agreement, Ultragenyx has in-licensed NAV Vectors for the development of gene therapies to treat Ornithine Transcarbamylase (OTC) Deficiency, Glycogen Storage Disease Type Ia (GSDIa), CDKL5 Deficiency, Hemophilia A, and Wilson Disease.

    About REGENXBIO Inc.              

    REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    REGENXBIO Forward-Looking Statements

    This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

    Ultragenyx Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report filed on Form 10-K with the Securities and Exchange Commission on February 14, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    REGENXBIO Contacts:

    Tricia Truehart
    Investor Relations and Corporate Communications
    347-926-7709

    Investors:
    Heather Savelle, 212-600-1902

    Media:
    David Rosen, 212-600-1902

    Ultragenyx Contact:

    Investors & Media
    Danielle Keatley
    415-475-6876

     

    Primary Logo

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  27. ROCKVILLE, Md. and NOVATO, Calif., March 31, 2020 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced a new exclusive, worldwide license agreement, extending the companies' existing gene therapy partnership.

    Under the terms of the agreement, REGENXBIO has granted Ultragenyx an exclusive, worldwide license, with rights to sublicense, to REGENXBIO's NAV AAV8 and AAV9 Vectors for the development…

    ROCKVILLE, Md. and NOVATO, Calif., March 31, 2020 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV Technology Platform, and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced a new exclusive, worldwide license agreement, extending the companies' existing gene therapy partnership.

    Under the terms of the agreement, REGENXBIO has granted Ultragenyx an exclusive, worldwide license, with rights to sublicense, to REGENXBIO's NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder. In return for these rights, REGENXBIO will receive an upfront payment of $7 million, ongoing fees, milestone payments, and royalties on net sales of products incorporating the licensed intellectual property.

    "We are pleased to expand our long-standing relationship with Ultragenyx through this new license agreement, which will enable Ultragenyx to apply our proprietary NAV Vectors to the development of a new gene therapy for rare diseases," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "Today's announcement provides further validation of the breadth and depth of our intellectual property portfolio, and we remain committed to working closely with leading companies to enable the development of important gene therapy programs."

    "This latest license agreement reinforces Ultragenyx's commitment to developing gene therapies for multiple rare diseases and adds a new indication to our relationship with REGENXBIO," said Emil D. Kakkis, MD, PhD, Chief Executive Officer and President of Ultragenyx. "

    Ultragenyx is a longstanding licensee partner of REGENXBIO. In addition to today's agreement, Ultragenyx has in-licensed NAV Vectors for the development of gene therapies to treat Ornithine Transcarbamylase (OTC) Deficiency, Glycogen Storage Disease Type Ia (GSDIa), CDKL5 Deficiency, Hemophilia A, and Wilson Disease.

    About REGENXBIO Inc.              

    REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    REGENXBIO Forward-Looking Statements

    This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations and clinical trials. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2019, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at www.sec.gov. All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

    Ultragenyx Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report filed on Form 10-K with the Securities and Exchange Commission on February 14, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    REGENXBIO Contacts:

    Tricia Truehart
    Investor Relations and Corporate Communications
    347-926-7709

    Investors:
    Heather Savelle, 212-600-1902

    Media:
    David Rosen, 212-600-1902

    Ultragenyx Contacts:

    Investors & Media
    Danielle Keatley
    415-475-6876

    (PRNewsfoto/REGENXBIO Inc.)

     

    (PRNewsfoto/REGENXBIO Inc.)

     

    Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/regenxbio-and-ultragenyx-announce-new-license-agreement-for-use-of-nav-technology-platform-for-the-treatment-of-rare-metabolic-disorder-301032846.html

    SOURCE REGENXBIO Inc.

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  28. SARASOTA, Fla. and NOVATO, Calif., March 16, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that it has dosed the first patient in its KIK-AS (Knockdown of UBE3A-antisense in Kids with Angelman Syndrome) study of GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS).

    The Phase 1/2 open-label, multiple-dose, dose-escalating study will enroll 20 patients to evaluate the safety, tolerability, and potential efficacy of GTX-102 in pediatric patients with Angelman syndrome.  This is the…

    SARASOTA, Fla. and NOVATO, Calif., March 16, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that it has dosed the first patient in its KIK-AS (Knockdown of UBE3A-antisense in Kids with Angelman Syndrome) study of GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS).

    The Phase 1/2 open-label, multiple-dose, dose-escalating study will enroll 20 patients to evaluate the safety, tolerability, and potential efficacy of GTX-102 in pediatric patients with Angelman syndrome.  This is the first investigational study testing an antisense oligonucleotide as a potential therapy to treat AS.  Further details can be referenced at: https://clinicaltrials.gov/ct2/show/NCT04259281.

    "Today is an important milestone with the dosing of the first patient in the KIK-AS study," stated Dr. Scott Stromatt, GeneTx's Chief Medical Officer   "GTX-102 has the potential to address the underlying deficiency that causes Angelman syndrome and we are excited, grateful and humbled to be leading this scientific quest.  We look forward to the results of this study and sharing them with the Angelman community."

    "The GeneTx team has achieved a tremendous accomplishment rapidly advancing this program into the clinic, and GTX-102 may one day provide patients with Angelman syndrome with the first targeted therapy and a potentially transformative option for this devastating disease," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx.

    Chicago's Rush University Medical Center is the first clinical site to begin enrolling patients in the KIK-AS study, with additional sites being planned in Boston, Cincinnati, Denver, Los Angeles, New York and Ottawa, Canada. 

    "When I held the syringe with this investigational treatment in my hand to inject it for the first time, I thought about the scientific advances in genomic and molecular medicine to produce potential treatments that bring hope of changing the disease course and ameliorating severity of symptoms in those with Angelman syndrome," said Elizabeth Berry-Kravis, site principal investigator at Rush. "This is an exciting time for the field of neurodevelopmental disorders as we embark on a path to understanding the outcomes of treatments directed at correcting the underlying molecular causes of disease." 

    Pending additional site activation, GeneTx Biotherapeutics expects to report preliminary data from the first cohorts in the study in early 2021.

    About Angelman Syndrome

    Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the intended target of GTX-102. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is typically not inherited but instead occurs spontaneously.

    Individuals with Angelman syndrome have developmental delay, balance issues, motor impairment, and debilitating seizures. Some individuals with Angelman syndrome are unable to walk and most do not speak. Anxiety and disturbed sleep can be serious challenges in individuals with Angelman syndrome. While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome suggesting that improvement of symptoms can potentially be achieved at any age.

    About GTX-102

    GTX-102 is an investigational antisense oligonucleotide designed to target and inhibit expression of UBE3A-AS. Nonclinical studies show that GTX-102 reduces the levels of UBE3A-AS and reactivates expression of the paternal UBE3A allele in neurons of the CNS. Reactivation of paternal UBE3A expression in animal models of Angelman syndrome has been associated with improvements in some of the neurological symptoms associated with the condition. GTX-102 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA). In August 2019, GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx.

    About GeneTx Biotherapeutics

    GeneTx Biotherapeutics LLC is a startup biotechnology company singularly focused on developing and commercializing a safe and effective antisense therapeutic for the treatment of Angelman syndrome. GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research. GeneTx licensed the rights to antisense technology intellectual property from The Texas A&M University System in December 2017.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    Ultragenyx Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our partnership with GeneTx, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report filed on Form 10-K with the Securities and Exchange Commission on February 14, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:

    GeneTx
    Paula Evans
    630-639-7271

    Ultragenyx
    Investors & Media
    Danielle Keatley
    415-475-6876

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  29. NOVATO, Calif., March 09, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the Barclays Global Healthcare Conference on Wednesday, March 11, 2020 at 1:35 PM ET.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment…

    NOVATO, Calif., March 09, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, will hold a virtual presentation at the Barclays Global Healthcare Conference on Wednesday, March 11, 2020 at 1:35 PM ET.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at:  www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

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  30. NOVATO, Calif., March 03, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that Shalini Sharp will step down from her position as Chief Financial Officer and Executive Vice President by September 2, 2020. At such time, Ms. Sharp plans to focus her time on her family and her role as an advisor and board member for various biotechnology companies. Ultragenyx will initiate a search for a successor, and Ms. Sharp will continue to serve as CFO until a replacement is found or until September 2, 2020. After that date, Ms. Sharp will stay on for six months to aid with the transition.

    "Shalini's financial…

    NOVATO, Calif., March 03, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that Shalini Sharp will step down from her position as Chief Financial Officer and Executive Vice President by September 2, 2020. At such time, Ms. Sharp plans to focus her time on her family and her role as an advisor and board member for various biotechnology companies. Ultragenyx will initiate a search for a successor, and Ms. Sharp will continue to serve as CFO until a replacement is found or until September 2, 2020. After that date, Ms. Sharp will stay on for six months to aid with the transition.

    "Shalini's financial and strategic leadership has been a pillar of our tremendous progress. She joined as our first finance employee in 2012 and has successfully managed our financing through going public and beyond, as well as represented the company to investors," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We are extremely grateful for her contributions in growing Ultragenyx from a small private company to a global, publicly-traded, biopharmaceutical company with multiple approved therapies and a robust pipeline. As she moves toward her transition, Shalini has put us in a strong financial position to continue to execute on our vision to bring therapies to patients with limited options."

    Ms. Sharp joined Ultragenyx in 2012 and is responsible for the company's finance, investor relations, strategic planning, and information technology functions. She also serves on the board of directors of Neurocrine Biosciences, Sutro Biopharma, Precision Biosciences, and the TB Alliance. 

    "It has been my profound privilege to help build this exceptional mission-driven, patient-focused rare disease company alongside a phenomenal team, including Emil, the executive team, and the finance team," said Ms. Sharp. "This decision is based on my personal desire to spend more time with my family, and I could not be more excited and optimistic about the future of Ultragenyx."

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    Ultragenyx Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report filed on Form 10-K with the Securities and Exchange Commission on February 14, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:
    Ultragenyx
    Investors & Media
    Danielle Keatley
    415-475-6876

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  31. NOVATO, Calif. and TOKYO, Feb. 27, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions using the latest biotechnology, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized…

    NOVATO, Calif. and TOKYO, Feb. 27, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. (Kyowa Kirin, TSE: 4151), a global specialty pharmaceutical company creating innovative medical solutions using the latest biotechnology, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental Biologics License Application (sBLA) for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The FDA has assigned priority review designation with a Prescription Drug User Fee Act (PDUFA) target date of June 18, 2020.

    "We appreciate the FDA's collaboration in evaluating the data, and as a result, we are another step closer to bringing the first treatment to patients with this devastating disease in the setting of an unresectable tumor," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "We look forward to continuing to work closely with the FDA with the goal of bringing Crysvita to patients with TIO as quickly as possible."

    "The discovery and submission of Crysvita has meant a great deal to patients and families that previously had no other treatment options," said Tomohiro Sudo, Head of Global Product Management Office of Kyowa Kirin. "If approved, we believe Crysvita may also become a meaningful treatment option for many patients with TIO in the U.S."

    The sBLA package includes data from two single-arm Phase 2 studies, a 144-week study in 14 adult patients conducted by Ultragenyx in the U.S. and an 88-week study in 13 adult patients conducted by Kyowa Kirin in Japan and South Korea.

    Crysvita is approved by the U.S. FDA for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients six months of age and older, and by Health Canada and Brazil's National Health Surveillance Agency (ANVISA) for the treatment of XLH in adult and pediatric patients one year of age and older. In Japan, it is approved by the Ministry of Health, Labor and Welfare (MHLW) for the treatment of FGF23-related hypophosphatemic rickets and osteomalacia. In Europe, Crysvita has received European conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons, and an application for the expanded use in adults with XLH is currently under review by the European Medicines Agency. 

    See below for Important Safety Information for Crysvita in X-linked hypophosphatemia.

    About Tumor-Induced Osteomalacia (TIO)
    TIO is caused by typically benign tumors that produce excess levels of fibroblast growth factor 23 (FGF23), causing phosphate wasting in the urine that leads to severe hypophosphatemia, osteomalacia, muscle weakness, fatigue, bone pain and fractures. The symptoms rapidly resolve if the causal tumors or lesion can be resected; however, there are cases in which resection is not feasible or recurrence of the tumor occurs after resection. In patients for whom the tumor or lesion is inoperable, the current treatment consists of oral phosphate and/or vitamin D replacement. Efficacy of this management is often limited, as it does not treat the underlying disease and its benefits must be balanced with monitoring for potential risks such as nephrocalcinosis, hypercalciuria and hyperparathyroidism. An estimated 500-1,000 people in the United States have TIO, and approximately half of all cases are inoperable.

    About Crysvita
    Crysvita (burosumab-twza) is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, against the phosphaturic hormone FGF23. FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate wasting in TIO and other hypophosphatemic conditions, including XLH, is caused by excessive levels and activity of FGF23. Crysvita is designed to bind to and thereby inhibit the biological activity of FGF23. By blocking excess FGF23 in patients with TIO and XLH, Crysvita is intended to increase phosphate reabsorption from the kidney and increase the production of vitamin D, which enhances intestinal absorption of phosphate and calcium.

    Kyowa Kirin and Ultragenyx have been collaborating in the development and commercialization of Crysvita globally based on the collaboration and license agreement between the parties.

    INDICATION (IN THE U.S.)
    Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older.

    IMPORTANT SAFETY INFORMATION

    Crysvita should not be taken if:

    • An oral phosphate supplement and/or a specific form of vitamin D supplement are taken (such as calcitriol, paricalcitol, doxercalciferol, calcifediol).
    • Phosphorus levels from a blood sample are within or above the normal range for age.
    • Kidney problems are present.

    What is the most important information to know about Crysvita?

    • Some patients developed allergic reactions (rash and hives) while taking Crysvita. Doctors will monitor for symptoms of an allergic reaction while Crysvita is taken.
    • High levels of phosphorus in the blood have been reported in some patients taking Crysvita. This may be related to a risk of high calcium levels in the kidneys. Doctors will collect samples to monitor levels.
    • Administration of Crysvita may result in reactions at the injection site, such as hives, reddening of the skin, rash, swelling, bruising, pain, severe itching of the skin, and collection of blood outside of a blood vessel (hematoma).

    What are the possible side effects of Crysvita?

    • The most common adverse reactions that were seen in children with XLH are:
      • Fever
      • Injection site reaction
      • Cough
      • Vomiting
      • Pain in arms and legs
      • Headache
      • Tooth infection
      • Dental cavities
      • Diarrhea
      • Decreased vitamin D levels
      • Toothache
      • Constipation
      • Muscle pain
      • Rash
      • Dizziness
      • Nausea
    • The most common adverse reactions that were seen in adults with XLH are:
      • Back pain
      • Headache
      • Tooth infection
      • Restless leg syndrome
      • Decreased vitamin D levels
      • Dizziness
      • Muscle spasms
      • Constipation
      • Phosphorus levels increased in the blood
    • Narrowing of the spaces within the spine is common in adults with XLH and pressure on the spinal cord has been reported in adults taking Crysvita. It is not known if taking Crysvita worsens the narrowing of the spaces within the spine or the pressure on the spinal cord.

    Before taking Crysvita, doctors should be informed about all medications (including supplements) and medical conditions, including if:

    • One is taking oral phosphate and/or active vitamin D (such as calcitriol, paricalcitol, doxercalciferol, calcifediol).
    • One is pregnant, thinks she may be pregnant, or plans to become pregnant. There is not enough experience to know if Crysvita may harm an unborn baby. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-888-756-8657.
    • One is breastfeeding or plans to breastfeed. There is not enough experience to know if Crysvita passes into breast milk. Women should talk with their doctors about the best way to feed their babies while taking Crysvita.

    While taking Crysvita, doctors should be informed if one experiences:

    • An allergic reaction such as rash or hives 
    • A rash, swelling, bruising or other reaction at the injection site
    • New or worsening restless leg syndrome

    These are not all the possible side effects of Crysvita. Doctors should be contacted for medical advice about side effects.

    Side effects may be reported to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. Side effects may also be reported to Kyowa Kirin, Inc. at 1-888-756-8657.

    Please see full Prescribing Information for additional Important Safety Information.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    About Kyowa Kirin
    Kyowa Kirin commits to innovate drug discovery driven by state-of-the-art technologies. The company focuses on creating new values in the four therapeutic areas: nephrology, oncology, immunology/allergy and neurology. Under the Kyowa Kirin brand, the employees from 36 group companies across North America, EMEA and Asia/Oceania unite to champion the interests of patients and their caregivers in discovering solutions wherever there are unmet medical needs. You can learn more about the business of Kyowa Kirin at www.kyowakirin.com.

    Forward-Looking Statements
    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and future regulatory interactions, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our collaboration with Kyowa Kirin, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 14, 2020, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:

    Ultragenyx
    Investors & Media
    Danielle Keatley
    +1-415-475-6876

    Kyowa Kirin Co. Ltd.
    Media
    Hiroki Nakamura
    +81-3-5205-7205
    Email:


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  32. NOVATO, Calif., Feb. 21, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Tom Kassberg, the company's Chief Business Officer, will present at the SVB Leerink Global Healthcare Conference on Wednesday, February 26, 2020 at 11:00 AM ET in New York, NY.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment…

    NOVATO, Calif., Feb. 21, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Tom Kassberg, the company's Chief Business Officer, will present at the SVB Leerink Global Healthcare Conference on Wednesday, February 26, 2020 at 11:00 AM ET in New York, NY.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at:  www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

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  33. 2019 total revenue is $103.7 million; 2019 Crysvita® (burosumab) revenue to Ultragenyx is $87.3 million

    2020 Crysvita revenue in Ultragenyx territories guidance of $125 million to $140 million reaffirmed

    NOVATO, Calif., Feb. 13, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2019 and reaffirmed its financial guidance for 2020.

    "In 2019 we created a substantial foundation based on the strong launch of Crysvita and the validation of our gene therapy platform across multiple clinical programs," said Emil D. Kakkis…

    2019 total revenue is $103.7 million; 2019 Crysvita® (burosumab) revenue to Ultragenyx is $87.3 million

    2020 Crysvita revenue in Ultragenyx territories guidance of $125 million to $140 million reaffirmed

    NOVATO, Calif., Feb. 13, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2019 and reaffirmed its financial guidance for 2020.

    "In 2019 we created a substantial foundation based on the strong launch of Crysvita and the validation of our gene therapy platform across multiple clinical programs," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In 2020, we anticipate building on this momentum as we look to the potential approvals of UX007 for LC-FAOD and Crysvita for TIO. We also expect to advance our gene therapy programs into pivotal studies and bring two new programs into the clinic while continuing to exercise financial discipline."

    Fourth Quarter and Full Year 2019 Financial Results

    Net Revenues
    For the fourth quarter of 2019, Ultragenyx reported $35.6 million in total revenue. Ultragenyx recognized $29.9 million in total Crysvita revenue. This includes $26.1 million in collaboration revenue in the U.S. profit share territory and $2.2 million in royalty revenue in the European territory from the collaboration and license agreement with Kyowa Kirin Co., Ltd (KKC). Net product sales for Crysvita in other regions were $1.6 million. Mepsevii® (vestronidase alfa) product revenue for the fourth quarter of 2019 was $4.4 million, and UX007 named patient revenue was $1.2 million. Ultragenyx recognized $0.1 million in revenue from its research agreement with Bayer.

    Net revenue for the year ended December 31, 2019 totaled $103.7 million. In 2019, Ultragenyx recognized $87.3 million in total Crysvita revenue. This includes $74.9 million in collaboration revenue in the U.S. profit share territory and $8.1 million in royalty revenue in the European territory from the collaboration and license agreement with KKC. Net product sales for Crysvita in other regions totaled $4.3 million. Mepsevii product revenue for the year ended December 31, 2019 was $12.6 million, and UX007 named patient revenue was $3.3 million. Ultragenyx recognized $0.5 million in revenue from its research agreement with Bayer in the year ended December 31, 2019.

    Operating Expenses
    Total operating expenses for the fourth quarter of 2019 were $130.0 million compared with $106.6 million for the same period in 2018, including non-cash stock-based compensation of $19.7 million and $21.1 million in the fourth quarter of 2019 and 2018, respectively. Total operating expenses for the year ended December 31, 2019, were $527.9 million, which includes $15.6 million in expenditures for Arcturus collaboration and license amendment and $20 million for the upfront payment on the GeneTx agreement, compared with operating expenses of $422.9 million for the same period in 2018. The operating expenses include non-cash stock-based compensation of $82.0 million and $80.1 million in the full year of 2019 and 2018, respectively. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows, and advances its pipeline.

    For the fourth quarter of 2019, Ultragenyx reported a net loss of $93.8 million, or $1.62 per share, basic and diluted, compared with a net loss for the fourth quarter of 2018 of $87.8 million, or $1.73 per share, basic and diluted. For the year ended December 31, 2019, the net loss was $402.7 million, or $7.12 per share, basic and diluted, compared with a net loss for the same period in 2018 of $197.6 million, or $3.97 per share, basic and diluted. The loss for the fourth quarter of 2019 and for the year ended December 31, 2019 includes unrealized gains of $1.4 million and $13.4 million, respectively, from the fair value adjustments on the investment in Arcturus equity securities. The loss from the full year 2018 was reduced by the gain on the sale of the Mepsevii priority review voucher (PRV) in January 2018 for net proceeds of $130.0 million and a $40.3 million gain from Ultragenyx's portion of the sales of the PRV received with the Crysvita approval. The net loss for the full year 2019 reflected cash used in operations of $345.4 million compared to $290.6 million for the same period in 2018.

    Cash, Cash Equivalents and Investments
    Cash, cash equivalents, and investments were $760.4 million as of December 31, 2019, including proceeds of $320.0 million received from the sale of the company's royalty interest in Crysvita in the European territory.

    2020 Guidance

    Crysvita Guidance in Ultragenyx Territories
    In 2020, the company continues to expect Crysvita revenue in the Ultragenyx territories to be between $125 million and $140 million, which excludes the European territory revenue. Ultragenyx territories include the collaboration revenue from the North American profit share territory (US and Canada) and other regions where revenue from product sales are recognized by Ultragenyx (Latin America, Turkey). In December 2019, the European territory royalty revenue rights were sold to Royalty Pharma. As a result, the company will no longer receive cash payments from the EU territory royalty revenue rights beginning January 1, 2020, until the respective threshold amount is met; however, the company will continue to record the royalty as "non-cash" revenue.

    2020 Expected Net Cash Burn Rate
    The company also expects a more than 20 percent reduction in net cash burn (net cash used in operations plus capital expenditures) in 2020 compared to 2019 due to a combination of financial discipline in spending with flattening operating expense growth, combined with the significantly larger growth in revenue.

    Recent Updates and Upcoming Milestones

    Crysvita for X-linked Hypophosphatemia: Non-dilutive $320 million sale of future European royalties

    • In December 2019, Ultragenyx sold to Royalty Pharma for $320 million its royalty interest in Crysvita in the European territory, where it is being commercialized by Kyowa Kirin Co., Ltd.

    Crysvita for Tumor-Induced Osteomalacia (TIO): Supplemental Biologics License Application (sBLA) submitted

    • Ultragenyx submitted the sBLA to the U.S. FDA on December 18, 2019 and expects to hear back from FDA on submission acceptance and review designation in February 2020.

    UX007 for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD): NDA under review by U.S. FDA

    • The U.S. FDA accepted for review the New Drug Application (NDA) and has set a Prescription Drug User Fee Act (PDUFA) date of July 31, 2020. The FDA has indicated that it is not currently planning to hold an advisory committee meeting to discuss the application. The review process is on track for a decision by the PDUFA date.

    DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Positive data from higher dose cohort of Phase 1/2 study; data from prophylactic steroid cohort in second half 2020

    • Recent positive data from Cohort 3 indicated two confirmed responders and a potential third responder out of three total patients, as well as a new responder in Cohort 2. There are currently up to six responders of the nine dosed to date with a more consistent response at higher doses.
    • Ultragenyx is initiating a fourth cohort (n=3) using prophylactic steroids at the same dose as Cohort 3. Data from the prophylactic steroid cohort are expected in the second half of 2020.

    DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Positive data from Phase 1/2 study; enrollment complete in confirmatory cohort and data expected in first half 2020

    • Enrollment is complete in the confirmatory cohort of three patients at the second dose cohort, with data expected in the first half of 2020. Following the results of the confirmatory cohort, a Phase 3 study could begin in the second half of 2020.

    GTX-102 for Angelman Syndrome (AS): Partnered program with GeneTx; IND active and enrollment of first patient expected in first half 2020 

    • An investigational new drug (IND) application is now active and GeneTx has received Institutional Review Board (IRB) approval from the first study site. Enrollment in the Phase 1/2 study of GTX-102 is expected to begin in the first half of 2020.
    • In February 2020, Ultragenyx paid $25 million after the acceptance of the IND to maintain its option to acquire GeneTx until the earlier of 30 months from the first dosing of a patient in the Phase 1/2 study (subject to extensions) or 90 days after results are available from that study.

    UX701 for Wilson Disease: IND expected by the end of the year

    • An IND application is expected by the end of 2020 for a new gene therapy for Wilson disease, a larger rare metabolic disease. UX701 will be the company's second clinical program to utilize its HeLa manufacturing system.

    DTX201 / BAY 2599023 for Hemophilia A: Partnered with Bayer; Cohort 2 data presented at European Association for Haemophilia and Allied Disorders (EAHAD)

    • Three cohorts with two patients each, six patients in total, have been dosed with AAVhu37 (DTX201 / BAY 2599023), using material from Ultragenyx's proprietary HeLa manufacturing platform. Longer term dose cohort 1 and new dose cohort 2 data presented at EAHAD showed three of four patients have achieved clinically meaningful FVIII levels. One patient in Cohort 1 has achieved clinically meaningful FVIII levels and has experienced four bleeds since receiving treatment down from 99 bleeds the prior year. Both patients in dose cohort 2 achieved clinically meaningful FVIII levels out to 24 and 30 weeks. Patient 4 has been off treatment and bleed free for seven months as of the data cut-off date. The second patient in dose cohort 2 had mild ALT/AST elevations that was managed with a short tapering course of corticosteroids.

    Conference Call and Webcast Information

    Ultragenyx will host a conference call today, Thursday, February 13, 2020, at 2 p.m. PT/ 5 p.m. ET to discuss fourth quarter and full year 2019 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 5068548. The replay of the call will be available for one year.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, Ultragenyx's reliance on its third party partner, Kyowa Kirin Co. ,Ltd., for the supply of Crysvita, smaller than anticipated market opportunities for the company's products and product candidates, the company's evolving commercial infrastructure, uncertainties related to insurance coverage and reimbursement approval for the company's products, manufacturing risks, the uncertainties inherent in the clinical drug development process, including the potential for substantial delays and the risk that earlier study results may not be predictive of future study results, the lack of predictability in the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 6, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

     
    Ultragenyx Pharmaceutical Inc.
    Selected Statement of Operations Financial Data
    (in thousands, except share and per share amounts)
    (unaudited)
                   
      Three Months Ended December 31,   Year Ended December 31,
      2019   2018   2019   2018
    Statement of Operations Data:              
    Revenues:              
    Collaboration and license $ 28,423     $ 12,797     $ 83,493     $ 41,693  
    Product sales   7,170       3,464       20,221       9,802  
    Total revenues   35,593       16,261       103,714       51,495  
    Operating expenses:              
    Cost of sales   5,107       507       9,008       1,146  
    Research and development   83,061       71,618       357,355       293,998  
    Selling, general and administrative   41,877       34,476       161,524       127,724  
    Total operating expenses   130,045       106,601       527,887       422,868  
    Loss from operations   (94,452 )     (90,340 )     (424,173 )     (371,373 )
    Gain from sale of priority review vouchers                     170,322  
    Change in fair value of investment in Arcturus equity securities   1,419             13,413        
    Non-cash interest expense on liability related to the sale of future royalties   (1,135 )           (1,135 )      
    Other income (expense), net   2,924       2,640       12,451       3,954  
    Loss before income taxes   (91,244 )     (87,700 )     (399,444 )     (197,097 )
    Provision for income taxes   (2,561 )     (126 )     (3,283 )     (514 )
    Net loss $ (93,805 )   $ (87,826 )   $ (402,727 )   $ (197,611 )
    Net loss per share, basic and diluted $ (1.62 )   $ (1.73 )   $ (7.12 )   $ (3.97 )
    Shares used in computing net loss per share, basic and diluted   57,808,025       50,694,007       56,576,885       49,775,223  
                   


    Ultragenyx Pharmaceutical Inc.
    Selected Balance Sheet Financial Data
    (in thousands)
    (unaudited)
      December 31,   December 31,
      2019   2018
    Balance Sheet Data:      
    Cash, cash equivalents, and investments $ 760,404   $ 459,706
    Working capital   747,717     447,644
    Total assets   1,135,496     719,558
    Total stockholders' equity   653,764     608,908
           

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

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  34. NOVATO, Calif., Feb. 06, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Thursday, February 13, 2020 at 5pm ET to discuss its financial results and corporate update for the fourth quarter and the year ended December 31, 2019.

    The live and replayed webcast of the call will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 5068548. The replay of the call will be available for one year.

    About Ultragenyx

    NOVATO, Calif., Feb. 06, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Thursday, February 13, 2020 at 5pm ET to discuss its financial results and corporate update for the fourth quarter and the year ended December 31, 2019.

    The live and replayed webcast of the call will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 5068548. The replay of the call will be available for one year.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876


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  35. SARASOTA, Fla. and NOVATO, Calif., Jan. 15, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that GeneTx's Investigational New Drug (IND) Application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS), filed with the U.S. Food and Drug Administration (FDA) is now active. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2020.

    "The FDA's clearance of the IND to evaluate GTX-102 in patients with Angelman syndrome represents a significant milestone for the Angelman community…

    SARASOTA, Fla. and NOVATO, Calif., Jan. 15, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that GeneTx's Investigational New Drug (IND) Application for GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS), filed with the U.S. Food and Drug Administration (FDA) is now active. Enrollment in the Phase 1/2 study is expected to begin in the first half of 2020.

    "The FDA's clearance of the IND to evaluate GTX-102 in patients with Angelman syndrome represents a significant milestone for the Angelman community," said Paula Evans, Chief Executive Officer at GeneTx.  "Assessing an investigational therapy that aims to address the underlying cause of this devastating disorder has the potential to meaningfully alter the clinical course of patients' lives."

    "Based on preclinical data, we believe that GTX-102 offers tremendous promise and may one day provide patients with a potentially transformative therapeutic option," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "We look forward to continuing our partnership with GeneTx to address this devastating disorder for which there are currently no approved treatments."

    "The FDA cleared the IND which allows GTX-102 to enter the clinic," said Scott Stromatt, MD, Chief Medical Officer, GeneTx.  "GeneTx's Phase 1/2 study, named KIK-AS (Knockdown of UBE3A-antisense in Kids with AS, is expected to enroll 20 patients at multiple trial sites, beginning in the first half of 2020. The goal of this multiple dose, dose escalating, open-label study is to examine the safety, tolerability, and pharmacokinetics of GTX-102 in pediatric patients with Angelman syndrome. On February 15, 2020 we will open a website with more information on the KIK-AS study and provide a toll free number for parents and caregivers to obtain information on enrolling into the clinical study."

    About Angelman Syndrome

    Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the intended target of GTX-102. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is typically not inherited but instead occurs spontaneously.

    Individuals with Angelman syndrome have developmental delay, balance issues, motor impairment, and debilitating seizures. Some individuals with Angelman syndrome are unable to walk and most do not speak. Anxiety and disturbed sleep can be serious challenges in individuals with Angelman syndrome. While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome suggesting that improvement of symptoms can potentially be achieved at any age.

    About GTX-102

    GTX-102 is an investigational antisense oligonucleotide designed to target and inhibit expression of UBE3A-AS. Nonclinical studies show that GTX-102 reduces the levels of UBE3A-AS and reactivates expression of the paternal UBE3A allele in neurons of the CNS. Reactivation of paternal UBE3A expression in animal models of Angelman syndrome has been associated with improvements in some of the neurological symptoms associated with the condition. GTX-102 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA). In August 2019, GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx.

    About GeneTx Biotherapeutics

    GeneTx Biotherapeutics LLC is a startup biotechnology company singularly focused on developing and commercializing a safe and effective antisense therapeutic for the treatment of Angelman syndrome. GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research. GeneTx licensed the rights to antisense technology intellectual property from The Texas A&M University System in December 2017.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    Ultragenyx Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our partnership with GeneTx, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report filed on Form 10-Q with the Securities and Exchange Commission on November 6, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:

    GeneTx
    Paula Evans
    630-639-7271

    Ultragenyx
    Investors & Media
    Danielle Keatley
    415-475-6876

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  36. Preliminary 2019 total revenue is approximately $102 million to $104 million

    Preliminary 2019 Crysvita revenue to Ultragenyx of approximately $86 million to $88 million

    2020 Crysvita revenue in Ultragenyx territories is expected to be in the range of $125 million to $140 million excluding EU royalty revenue

    Year-end 2019 cash balance of greater than $750 million

    NOVATO, Calif., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported preliminary unaudited 2019 revenue and cash and investments at year end 2019, and provided 2020 revenue guidance for Crysvita in Ultragenyx territories…

    Preliminary 2019 total revenue is approximately $102 million to $104 million

    Preliminary 2019 Crysvita revenue to Ultragenyx of approximately $86 million to $88 million

    2020 Crysvita revenue in Ultragenyx territories is expected to be in the range of $125 million to $140 million excluding EU royalty revenue

    Year-end 2019 cash balance of greater than $750 million

    NOVATO, Calif., Jan. 13, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported preliminary unaudited 2019 revenue and cash and investments at year end 2019, and provided 2020 revenue guidance for Crysvita in Ultragenyx territories.

    "We passed the $100 million revenue threshold in 2019 based on the strong launches of Crysvita and Mepsevii. We aim to build on this success in 2020 with the potential approvals of UX007 in LC-FAOD and Crysvita in TIO," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "This growing commercial foundation will continue to enable significant pipeline advancement as we execute on our gene therapy clinical studies and initiate multiple new clinical programs."

    2019 Preliminary Revenue, 2020 Crysvita Revenue Guidance, and 2019 Ending Cash Position

    2019 Preliminary Revenue (unaudited)

    Total revenue for the year ended December 31, 2019 is approximately $102 million to $104 million. Total Crysvita revenue to Ultragenyx is approximately $86 million to $88 million in 2019, which includes collaboration revenue in the North American profit share territory, royalty revenue in the European territory from Kyowa Kirin Co. Ltd., and product revenue for Crysvita in other regions.

    2020 Crysvita Guidance

    For 2020, Crysvita revenue in the Ultragenyx territories is estimated to be between $125 million and $140 million. Ultragenyx territories include the North American profit share territory and other regions where collaboration revenue and product sales are recognized by Ultragenyx. The 2020 Crysvita revenue guidance excludes the European territory royalty revenue, the rights to which were sold to Royalty Pharma. Beginning January 1, 2020, the company will no longer receive cash payments from the EU territory royalty until the respective threshold amount is met; however, the company will continue to record the royalty as "non-cash" revenue.

    The company is not providing total 2020 revenue guidance at this time. The preliminary revenue results are based on management's initial analysis of operations for the quarter and year ended December 31, 2019. The 2019 revenue and cash position included in this release are preliminary and prior to the completion of review and audit procedures by Ultragenyx's external auditors, and are therefore subject to adjustment. The Company expects to issue full financial results for the fourth quarter and fiscal year 2019 in February 2020.

    2019 Ending Cash Position (unaudited) and 2020 Expected Net Cash Burn Rate

    Cash, cash equivalents, and available-for-sale investments were greater than $750 million as of December 31, 2019, including proceeds of $320 million received from the sale of the company's royalty interest in Crysvita in the European territory. The company also expects a more than 20 percent reduction in net cash burn (net cash used in operations plus capital expenditures) in 2020 compared to 2019.

    Recent Updates and Upcoming Milestones

    Crysvita for X-linked Hypophosphatemia: Non-dilutive $320 million royalty sale of future European royalties 

    • In December, Ultragenyx sold to Royalty Pharma for $320 million its royalty interest in Crysvita in the European territory, where it is being commercialized by Kyowa Kirin Co., Ltd.

    Crysvita for Tumor-Induced Osteomalacia (TIO): Supplemental Biologics License Application (sBLA) submitted

    • Ultragenyx submitted the sBLA to the U.S. FDA on December 18, 2019 and expects to hear back from FDA on submission acceptance and review designation in February 2020.

    UX007 for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD): NDA under review by U.S. FDA

    • The U.S. FDA accepted for review the New Drug Application (NDA) and has set a Prescription Drug User Fee Act (PDUFA) date of July 31, 2020. The FDA has indicated that it is not currently planning to hold an advisory committee meeting to discuss the application.

    DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Positive data from higher cohort of Phase 1/2 study; data from prophylactic steroid cohort in second half 2020

    • Recent positive data from Cohort 3 indicated two confirmed responders and a potential third responder out of three total patients, as well as a new responder in Cohort 2. There are currently up to six responders of the nine dosed to date with a more consistent response at higher doses.

    • Ultragenyx is initiating a fourth cohort (n=3) using prophylactic steroids at the same dose as Cohort 3. The first patient is expected to be enrolled in the first half of 2020, and data from the prophylactic steroid cohort are expected in the second half of 2020.

    DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Positive data from Phase 1/2 study; enrollment complete in confirmatory cohort and data expected in first half 2020

    • Enrollment is complete in the confirmatory cohort of three patients at the second dose cohort, with data expected in the first half of 2020. Following the results of the confirmatory cohort, a Phase 3 study could begin in the second half of 2020.

    GTX-102 for Angelman Syndrome: Partnered program with GeneTx; IND expected in first half 2020 

    • An investigational new drug (IND) application is expected in the first half of 2020. In August 2019, Ultragenyx and GeneTx announced a partnership to develop GTX-102 with Ultragenyx receiving an exclusive option to acquire GeneTx.

    UX701 for Wilson Disease: IND expected in second half 2020

    • An IND application is expected in the second half of 2020 for a new gene therapy for Wilson disease, a larger rare metabolic disease. UX701 will be the company's second clinical program to utilize its HeLa manufacturing system. The Hemophilia A program partnered with Bayer uses the HeLa system and has released its first clinical data. 

    Ultragenyx to Present at 38ᵗʰ Annual J.P. Morgan Healthcare Conference

    • Dr. Kakkis will present on Tuesday, January 14, 2020 at 12:00 p.m. PT in San Francisco. The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, Ultragenyx's reliance on its third party partner, Kyowa Kirin Co., Ltd., for the supply of Crysvita, smaller than anticipated market opportunities for the company's products and product candidates, the company's evolving commercial infrastructure, uncertainties related to insurance coverage and reimbursement approval for the company's products, manufacturing risks, the uncertainties inherent in the clinical drug development process, including the potential for substantial delays and the risk that earlier study results may not be predictive of future study results, the lack of predictability in the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company's future operating results and financial performance and the availability or commercial potential of Ultragenyx's products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 6, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

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  37. NOVATO, Calif. and TOKYO, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., ((Kyowa Kirin, TYO:4151) today announced that they submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) on December 18, 2019, for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The companies expect to hear back from FDA on submission acceptance and review designation in February 2020.

    "Approximately…

    NOVATO, Calif. and TOKYO, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., ((Kyowa Kirin, TYO:4151) today announced that they submitted a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) on December 18, 2019, for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The companies expect to hear back from FDA on submission acceptance and review designation in February 2020.

    "Approximately half of patients with TIO have tumors that cannot be surgically removed, leaving them with no other current treatment options," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "We look forward to continuing to work closely with the FDA during the review process, with the goal of bringing Crysvita to patients with TIO in the U.S."

    The sBLA package includes data from two single-arm Phase 2 studies, a 144-week study in 14 adult patients conducted by Ultragenyx in the U.S. and an 88-week study in 13 adult patients conducted by Kyowa Kirin in Japan and South Korea. In both studies, Crysvita was associated with increases in serum phosphorus and serum 1,25-dihydroxyvitamin D levels. Increased phosphate levels led to improvements in osteomalacia, mobility and vitality. Bone scans also demonstrated an increase in healed fractures and a decrease in new fractures during Crysvita treatment. During the studies, adverse events generally reflected the patients' underlying disease, and there were no serious treatment-related adverse events.

    Crysvita is approved by the FDA for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients six months of age and older, and by Health Canada and Brazil's National Health Surveillance Agency (ANVISA) for the treatment of XLH in adult and pediatric patients one year of age and older. It is approved by Japan's Ministry of Health, Labor and Welfare (MHLW) for the treatment of FGF23-related hypophosphatemic rickets and osteomalacia. The medicine has received European conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons, and an application for the expanded use in adults with XLH is currently under review by the European Medicines Agency.

    See below for Important Safety Information for Crysvita in X-linked hypophosphatemia.

    About Tumor-Induced Osteomalacia (TIO)
    TIO is caused by typically benign tumors that produce excess levels of fibroblast growth factor 23 (FGF23), causing phosphate wasting in the urine that leads to severe hypophosphatemia, osteomalacia, muscle weakness, fatigue, bone pain and fractures. The symptoms rapidly resolve if the causal tumors or lesion can be resected; however, there are cases in which resection is not feasible or recurrence of the tumor occurs after resection. In patients for whom the tumor or lesion is inoperable, the current treatment consists of oral phosphate and/or vitamin D replacement. Efficacy of this management is often limited, as it does not treat the underlying disease and its benefits must be balanced with monitoring for potential risks such as nephrocalcinosis, hypercalciuria and hyperparathyroidism. An estimated 500-1,000 people in the United States have TIO, and approximately half of all cases are inoperable.

    About Crysvita
    Crysvita (burosumab-twza) is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, against the phosphaturic hormone FGF23. FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate wasting in TIO and other hypophosphatemic conditions, including XLH, is caused by excessive levels and activity of FGF23. Crysvita is designed to bind to and thereby inhibit the biological activity of FGF23. By blocking excess FGF23 in patients with TIO and XLH, Crysvita is intended to increase phosphate reabsorption from the kidney and increase the production of vitamin D, which enhances intestinal absorption of phosphate and calcium.

    Kyowa Kirin and Ultragenyx have been collaborating in the development and commercialization of Crysvita globally based on the collaboration and license agreement between the parties.

    INDICATION (IN THE U.S.)
    Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older.

    IMPORTANT SAFETY INFORMATION

    Crysvita should not be taken if:

    • An oral phosphate supplement and/or a specific form of vitamin D supplement are taken (such as calcitriol, paricalcitol, doxercalciferol, calcifediol).
    • Phosphorus levels from a blood sample are within or above the normal range for age.
    • Kidney problems are present.

    What is the most important information to know about Crysvita?

    • Some patients developed allergic reactions (rash and hives) while taking Crysvita. Doctors will monitor for symptoms of an allergic reaction while Crysvita is taken.
    • High levels of phosphorus in the blood have been reported in some patients taking Crysvita. This may be related to a risk of high calcium levels in the kidneys. Doctors will collect samples to monitor levels.
    • Administration of Crysvita may result in reactions at the injection site, such as hives, reddening of the skin, rash, swelling, bruising, pain, severe itching of the skin, and collection of blood outside of a blood vessel (hematoma).

    What are the possible side effects of Crysvita?

    • The most common adverse reactions that were seen in children with XLH are:
      • Fever
      • Injection site reaction
      • Cough
      • Vomiting
      • Pain in arms and legs
      • Headache
      • Tooth infection
      • Dental cavities
      • Diarrhea
      • Decreased vitamin D levels
      • Toothache
      • Constipation
      • Muscle pain
      • Rash
      • Dizziness
      • Nausea
    • The most common adverse reactions that were seen in adults with XLH are:
      • Back pain
      • Headache
      • Tooth infection
      • Restless leg syndrome
      • Decreased vitamin D levels
      • Dizziness
      • Muscle spasms
      • Constipation
      • Phosphorus levels increased in the blood
    • Narrowing of the spaces within the spine is common in adults with XLH and pressure on the spinal cord has been reported in adults taking Crysvita. It is not known if taking Crysvita worsens the narrowing of the spaces within the spine or the pressure on the spinal cord.

    Before taking Crysvita, doctors should be informed about all medications (including supplements) and medical conditions, including if:

    • One is taking oral phosphate and/or active vitamin D (such as calcitriol, paricalcitol, doxercalciferol, calcifediol).
    • One is pregnant, thinks she may be pregnant, or plans to become pregnant. There is not enough experience to know if Crysvita may harm an unborn baby. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-888-756-8657.
    • One is breastfeeding or plans to breastfeed. There is not enough experience to know if Crysvita passes into breast milk. Women should talk with their doctors about the best way to feed their babies while taking Crysvita.

    While taking Crysvita, doctors should be informed if one experiences:

    • An allergic reaction such as rash or hives 
    • A rash, swelling, bruising or other reaction at the injection site
    • New or worsening restless leg syndrome

    These are not all the possible side effects of Crysvita. Doctors should be contacted for medical advice about side effects.

    Side effects may be reported to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. Side effects may also be reported to Kyowa Kirin, Inc. at 1-888-756-8657.

    Please see full Prescribing Information for additional Important Safety Information.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    About Kyowa Kirin
    Kyowa Kirin commits to innovate drug discovery driven by state-of-the-art technologies. The company focuses on creating new values in the four therapeutic areas: nephrology, oncology, immunology/allergy and neurology. Under the Kyowa Kirin brand, the employees from 36 group companies across North America, EMEA and Asia/Oceania unite to champion the interests of patients and their caregivers in discovering solutions wherever there are unmet medical needs. You can learn more about the business of Kyowa Kirin at www.kyowakirin.com.

    Forward-Looking Statements
    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and future regulatory interactions, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our collaboration with Kyowa Kirin, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 6, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:

    Ultragenyx
    Investors & Media
    Danielle Keatley
    +1-415-475-6876

    Kyowa Kirin Co. Ltd.
    Media
    Hiroki Nakamura
    +81-3-5205-7205
    Email:

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  38. More uniform response in Cohort 3 with two confirmed responders and one potential responder

    New female responder in Cohort 2 for a total of three confirmed female responders across all cohorts

    Up to six responders across all nine patients dosed in study

    Prophylactic steroid cohort to begin in first half 2020; data expected in second half 2020

    Ultragenyx to host conference call today at 4:30 p.m. Eastern Time

    NOVATO, Calif., Jan. 09, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced topline positive safety and efficacy data from Cohort 3 and longer-term data from Cohort 2 of the ongoing Phase 1/2…

    More uniform response in Cohort 3 with two confirmed responders and one potential responder

    New female responder in Cohort 2 for a total of three confirmed female responders across all cohorts

    Up to six responders across all nine patients dosed in study

    Prophylactic steroid cohort to begin in first half 2020; data expected in second half 2020

    Ultragenyx to host conference call today at 4:30 p.m. Eastern Time

    NOVATO, Calif., Jan. 09, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today announced topline positive safety and efficacy data from Cohort 3 and longer-term data from Cohort 2 of the ongoing Phase 1/2 study of DTX301, an investigational adeno-associated virus (AAV) gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. In Cohort 3 (n=3), there were two confirmed female responders as well a third potential male responder who requires longer-term follow-up to confirm response status. In Cohort 2, one female patient has newly demonstrated a response starting at Week 52 which was confirmed at Week 78. The two previously disclosed responders in Cohort 1 and Cohort 2 also remain clinically and metabolically stable at 104 and 78 weeks, respectively. Across all nine patients dosed in the study, up to six patients have demonstrated a response.

    "We are encouraged to see a more uniform response at the higher doses including three female responders. To date, three patients in the study have discontinued alternate pathway medication and liberalized their diets while remaining clinically and metabolically stable," said Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit. "We are moving to prophylactic steroid use in the next cohort as we believe this could further enhance the level and consistency of expression that we have demonstrated so far."

    Cohort 3 Efficacy Summary (as of December 9, 2019 cutoff date): One complete responder, one responder, and one potential responder

    Patient 7 (complete responder, female):
    Patient 7 demonstrated a clinically meaningful 79 percent change in rate of ureagenesis, from a low of 24 percent of normal at baseline to the 51 to 64 percent range, and staying at 44 percent of normal at Week 52. During this period, she reported feeling significantly better and discontinued her alternate pathway medications and liberalized her protein-restricted diet. She has remained clinically and metabolically stable without a rise in ammonia. 

    Patient 8 (responder, female):
    Patient 8 demonstrated a significant and consistent 90 percent reduction in ammonia levels, time-normalized over a 24 hour period, from a high of 184 umol/L at baseline to 19 umol/L at Week 24, which is within the normal range. Potentially aberrant high baseline ureagenesis values inconsistent with her known more severe clinical status make her ureagenesis results uninterpretable. This patient was on a tapering course of steroids at the time of last assessment and has not yet discontinued alternate pathway medications or liberalized her diet. The investigator reported that her family says her health is the best it has ever been.

    Patient 9 (potential responder, male):
    Patient 9 showed a 123 percent increase in rate of ureagenesis, from 25 percent of normal at baseline to 56 percent of normal at Week 12 while still on a steroid taper. Steroids have been shown to suppress rate of ureagenesis in other study patients. This patient has not yet discontinued alternate pathway medications or liberalized his diet. His ammonia levels have remained in the normal range and response status will be confirmed after additional follow-up.

    Cohort 2 Efficacy Summary: Two responders including new responder and previously-disclosed male complete responder

    Patient 6 (new responder, female):
    Patient 6 has now shown a 218 percent improvement in rate of ureagenesis, from 20 percent of normal at baseline to 61 percent at Week 52 and maintained at 64 percent at Week 78. In addition, she has shown a significant 74 percent reduction in ammonia levels from 156 umol/L at baseline to 40 umol/L at Week 78. She has started to taper her alternate pathway medications and liberalize her diet. With this new responder, there are two confirmed responders in cohort 2 out of three total patients.

    Safety Summary
    As of the data cutoff date, there have been no infusion-related adverse events and no treatment-related serious adverse events reported in the study. All adverse events have been Grade 1 or 2. All three patients in Cohort 3 had mild, clinically asymptomatic elevations in ALT levels, similar to what has been observed in other programs using AAV-based gene therapy.  All three patients have been responding to reactive tapering courses of steroids, and all patients remain clinically stable.

    Initiating Prophylactic Steroid Cohort
    As previously disclosed, a fourth cohort will enroll three patients at the 1.0 × 10^13 GC/kg dose, using prophylactic steroids. Patients will receive an 8-week tapering regimen of prophylactic steroids, starting at least 5 days prior to dosing with DTX301 at a starting steroid dose of 60 mg/day. The first patient is expected to be enrolled in the first half of 2020, and data from the prophylactic steroid cohort are expected in the second half of 2020.

    Potential Phase 3 Study Design
    Ultragenyx is continuing discussions with the U.S. Food and Drug Administration (FDA) regarding the potential Phase 3 study design. Ammonia is expected to be a primary endpoint based on direct FDA feedback to date, with ureagenesis as a measure of biologic activity that supports the decision for patients to discontinue alternate pathway medications.

    Conference Call and Webcast Information
    Ultragenyx will host a conference call today, Thursday, January 9, 2020, at 4:30 p.m. ET/ 1:30 p.m. PT during which Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will discuss the new data from the ongoing DTX301 Phase 1/2 Study. The live and replayed webcast of the call and slides will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 5583103. The replay of the call will be available for one year.

    About the OTC Phase 1/2 Study (DTX301)
    The Phase 1/2 study evaluates the change in the rate of ureagenesis, ammonia levels, neurocognitive assessment, biomarkers, and safety of DTX301 in patients with OTC deficiency. Three patients have been dosed in each of three dose cohorts of 2.0 × 10^12 GC/kg (Cohort 1), 6.0 × 10^12 GC/kg (Cohort 2), and 1.0 × 10^13 GC/kg (Cohort 3). Patients in the first three cohorts received steroids to reactively manage ALT elevations. In the fourth cohort, three patients will receive a 1.0 × 10^13 GC/kg dose and will all receive a prophylactic tapering course of steroids.

    About OTC Deficiency
    OTC deficiency, the most common urea cycle disorder, is caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. Individuals with OTC deficiency can build up excessive levels of ammonia in their blood, potentially resulting in acute and chronic neurological deficits and other toxicities. It is estimated that more than 10,000 patients are affected by OTC deficiency worldwide, of which approximately 80 percent are classified as late-onset and represent a clinical spectrum of disease severity. In the late-onset form of the disease, elevated ammonia can lead to significant medical issues for patients. Neonatal onset disease occurs only in males, presents as severe disease, and can be fatal at an early age. Approved therapies, which must be taken multiple times a day for the patient's entire life, do not eliminate the risk of future metabolic crises. Currently, the only curative approach is liver transplantation.

    About DTX301
    DTX301 is an investigational AAV type 8 gene therapy designed to deliver stable expression and activity of OTC following a single intravenous infusion. It has been shown in preclinical studies to normalize levels of urinary orotic acid, a marker of ammonia metabolism. DTX301 was granted Orphan Drug Designation in both the United States and Europe.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    Ultragenyx Forward-Looking Statements
    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, including the potential for substantial delays and the risk that earlier study results may not be predictive of future study results, the lack of predictability in the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 6, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

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  39. NOVATO, Calif., Dec. 19, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the 38th Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2020 at 12:00 pm PT in San Francisco.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients…

    NOVATO, Calif., Dec. 19, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the 38th Annual J.P. Morgan Healthcare Conference on Tuesday, January 14, 2020 at 12:00 pm PT in San Francisco.

    The live and archived webcast of the presentation will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at:  www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley         
    415-475-6876

    Primary Logo

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  40. NOVATO, Calif. and NEW YORK, Dec. 18, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Royalty Pharma today announced that Ultragenyx has sold to Royalty Pharma for $320 million a royalty right due to Ultragenyx from Kyowa Kirin Co., Ltd for the net sales of Crysvita® (burosumab) in the European Union (EU), the United Kingdom (UK), and Switzerland. Crysvita has received conditional marketing authorization in Europe for the treatment of X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons, and an application for the…

    NOVATO, Calif. and NEW YORK, Dec. 18, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Royalty Pharma today announced that Ultragenyx has sold to Royalty Pharma for $320 million a royalty right due to Ultragenyx from Kyowa Kirin Co., Ltd for the net sales of Crysvita® (burosumab) in the European Union (EU), the United Kingdom (UK), and Switzerland. Crysvita has received conditional marketing authorization in Europe for the treatment of X-linked hypophosphatemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons, and an application for the expanded use in adults with XLH is currently under review by the European Medicines Agency.  

    The agreement will automatically expire in the event aggregate royalty payments received by Royalty Pharma are equal to or greater than 1.9 times the purchase price prior to December 31, 2030, or in the event aggregate royalty payments are equal to or greater than 2.5 times the purchase price if the prior threshold is not met by the end of 2030. Once the threshold amount has been met, the EU, UK, and Switzerland royalty payments will revert back to Ultragenyx.

    "This non-dilutive royalty financing provides Ultragenyx with capital to support our strong launches of Crysvita in North America and Latin America and Mespevii worldwide, as well as the advancement of our clinical and translational research programs," said Shalini Sharp, Chief Financial Officer of Ultragenyx. "Crysvita is the first treatment option for XLH that targets the underlying cause of the disease and we are pleased that Royalty Pharma, a recognized leader in providing innovative capital in life sciences, has invested in the future of this therapy."

    Royalty Pharma's CEO, Pablo Legorreta, stated, "We are pleased to have the opportunity to work with Ultragenyx on this win-win transaction, in which Ultragenyx was able to pull forward capital from a passive, EU royalty stream to power its current strategic plan. Royalty Pharma adds a high quality asset that is an excellent addition to our diversified portfolio of leading biopharmaceutical royalties."

    Perella Weinberg Partners LP and J. Wood Capital Advisors LLC acted as financial advisors and Gibson, Dunn & Crutcher LLP acted as legal advisor to Ultragenyx on the transaction.  Goodwin Procter LLP and Maiwald Patentanwalts GmbH acted as legal advisors to Royalty Pharma.

    About Crysvita
    Crysvita (burosumab-twza) is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, against the phosphaturic hormone FGF23. FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate wasting in TIO and other hypophosphatemic conditions, including XLH, is caused by excessive levels and activity of FGF23. Crysvita is designed to bind to and thereby inhibit the biological activity of FGF23. By blocking excess FGF23 in patients with TIO and XLH, Crysvita is intended to increase phosphate reabsorption from the kidney and increase the production of vitamin D, which enhances intestinal absorption of phosphate and calcium.

    Crysvita is approved by the U.S. Food and Drug Administration (FDA) for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients six months of age and older, and by Health Canada and Brazil's National Health Surveillance Agency (ANVISA) for the treatment of XLH in adult and pediatric patients one year of age and older. It is approved by Japan's Ministry of Health, Labor and Welfare (MHLW) for the treatment of FGF23-related hypophosphatemic rickets and osteomalacia. The medicine has received European conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons, and an application for the expanded use in adults with XLH is currently under review by the European Medicines Agency.  

    Kyowa Kirin and Ultragenyx have been collaborating in the development and commercialization of Crysvita globally based on the collaboration and license agreement between the parties.

    INDICATION (IN THE U.S.)
    Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older.

    IMPORTANT SAFETY INFORMATION

    Crysvita should not be taken if:

    • An oral phosphate supplement and/or a specific form of vitamin D supplement are taken (such as calcitriol, paricalcitol, doxercalciferol, calcifediol).
    • Phosphorus levels from a blood sample are within or above the normal range for age.
    • Kidney problems are present.

     What is the most important information to know about Crysvita?

    • Some patients developed allergic reactions (rash and hives) while taking Crysvita. Doctors will monitor for symptoms of an allergic reaction while Crysvita is taken.
    • High levels of phosphorus in the blood have been reported in some patients taking Crysvita. This may be related to a risk of high calcium levels in the kidneys. Doctors will collect samples to monitor levels.
    • Administration of Crysvita may result in reactions at the injection site, such as hives, reddening of the skin, rash, swelling, bruising, pain, severe itching of the skin, and collection of blood outside of a blood vessel (hematoma).

    What are the possible side effects of Crysvita?

    • The most common adverse reactions that were seen in children with XLH are:
      • Fever
      • Injection site reaction
      • Cough
      • Vomiting
      • Pain in arms and legs
      • Headache
      • Tooth infection
      • Dental cavities
      • Diarrhea
      • Decreased vitamin D levels
      • Toothache
      • Constipation
      • Muscle pain
      • Rash
      • Dizziness
      • Nausea
    • The most common adverse reactions that were seen in adults with XLH are:
      • Back pain
      • Headache
      • Tooth infection
      • Restless leg syndrome
      • Decreased vitamin D levels
      • Dizziness
      • Muscle spasms
      • Constipation
      • Phosphorus levels increased in the blood
    • Narrowing of the spaces within the spine is common in adults with XLH and pressure on the spinal cord has been reported in adults taking Crysvita. It is not known if taking Crysvita worsens the narrowing of the spaces within the spine or the pressure on the spinal cord.

    Before taking Crysvita, doctors should be informed about all medications (including supplements) and medical conditions, including if:  

    • One is taking oral phosphate and/or active vitamin D (such as calcitriol, paricalcitol, doxercalciferol, calcifediol).
    • One is pregnant, thinks she may be pregnant, or plans to become pregnant. There is not enough experience to know if Crysvita may harm an unborn baby. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-888-756-8657.
    • One is breastfeeding or plans to breastfeed. There is not enough experience to know if Crysvita passes into breast milk. Women should talk with their doctors about the best way to feed their babies while taking Crysvita.

    While taking Crysvita, doctors should be informed if one experiences:

    • An allergic reaction such as rash or hives 
    • A rash, swelling, bruising or other reaction at the injection site
    • New or worsening restless leg syndrome

    These are not all the possible side effects of Crysvita. Doctors should be contacted for medical advice about side effects.

    Side effects may be reported to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. Side effects may also be reported to Kyowa Kirin, Inc. at 1-888-756-8657.

    Please see full Prescribing Information for additional Important Safety Information.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    About Royalty Pharma
    Founded in 1996, Royalty Pharma is the industry leader in acquiring pharmaceutical royalties, with over $16 billion in royalty assets. Royalty Pharma funds innovation in life sciences both directly and indirectly: directly when it partners with life sciences companies to co-develop and co-fund products in late-stage clinical trials, and indirectly when it acquires existing royalty interests from the original innovators (academic institutions, research hospitals, foundations and inventors). The company's portfolio includes royalty interests in over 50 approved products including AbbVie and J&J's Imbruvica, Astellas and Pfizer's Xtandi, Biogen's Tysabri, Gilead's HIV franchise, Merck's Januvia, Novartis' Promacta, and Vertex's Kalydeco, Symdeko and Trikafta. Royalty Pharma is also a leading investor in pre-approval royalties, having since 2011 invested over $5.6 billion in royalties on pre-approval products and committed over $1.2 billion to direct R&D funding in exchange for royalties. For more information, visit www.royaltypharma.com

    Forward-Looking Statements
    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and future regulatory interactions, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our collaboration with Kyowa Kirin, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 6, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:

    Ultragenyx
    Investors & Media
    Danielle Keatley
    +1-415-475-6876

    Royalty Pharma
    Alexander v. Perfall
    +1 (212) 883-2298

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  41. NOVATO, Calif., Nov. 08, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced upcoming presentations at the following conferences:

    • Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the Credit Suisse 28th Annual Healthcare Conference on Tuesday, November 12, 2019 at 11:30 AM MT in Scottsdale, Arizona.
       
    • Tom Kassberg, the company's Chief Business Officer, will present at the Stifel Healthcare Conference on Wednesday, November 20, 2019 at 1:50 PM ET in New York, NY.

    The live and archived webcast of the company presentations will be accessible…

    NOVATO, Calif., Nov. 08, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced upcoming presentations at the following conferences:

    • Emil D. Kakkis, M.D., Ph.D., the company's Chief Executive Officer and President, will present at the Credit Suisse 28th Annual Healthcare Conference on Tuesday, November 12, 2019 at 11:30 AM MT in Scottsdale, Arizona.
       
    • Tom Kassberg, the company's Chief Business Officer, will present at the Stifel Healthcare Conference on Wednesday, November 20, 2019 at 1:50 PM ET in New York, NY.

    The live and archived webcast of the company presentations will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at:  www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley              
    415-475-6876

    Primary Logo

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  42. Strong Crysvita® (burosumab) Launch Continues with Approximately 1,130 Patients on Reimbursed Commercial Therapy in the United States as of the end of the Third Quarter

    UX007 (triheptanoin) NDA for Treatment of Long-chain Fatty Acid Oxidation Disorders Accepted for Review by FDA

    NOVATO, Calif., Nov. 05, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results and corporate update for the quarter ended September 30, 2019.

    "The U.S. launch of Crysvita continues to be strong with the number of patients on reimbursed therapy continuing to increase, which will be further supported…

    Strong Crysvita® (burosumab) Launch Continues with Approximately 1,130 Patients on Reimbursed Commercial Therapy in the United States as of the end of the Third Quarter

    UX007 (triheptanoin) NDA for Treatment of Long-chain Fatty Acid Oxidation Disorders Accepted for Review by FDA

    NOVATO, Calif., Nov. 05, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results and corporate update for the quarter ended September 30, 2019.

    "The U.S. launch of Crysvita continues to be strong with the number of patients on reimbursed therapy continuing to increase, which will be further supported by the recently expanded label for both pediatric and adult patients," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "The New Drug Application for UX007 is now being reviewed by FDA, and our second indication for Crysvita for tumor-induced osteomalacia will be submitted in the coming months, potentially giving us approved treatments for four different diseases in 2020."

    Third Quarter 2019 Financial Results

    Net Revenues

    For the third quarter of 2019, Ultragenyx reported $25.8 million in total revenue, including $22.6 million in Crysvita revenue. Crysvita revenue includes $19.5 million in collaboration revenue in the North American profit share territory, $2.0 million in royalty revenue in the European territory from Kyowa Kirin Co., and product revenue in other regions of $1.1 million. The Company recognized $2.4 million in Mepsevii® (vestronidase alfa) product revenue, $0.7 million in UX007 named patient revenue, and also $0.1 million in revenue from its research agreement with Bayer.

    Revenue for the nine months ended September 30, 2019 was $68.1 million, including $57.3 million in Crysvita revenue. Crysvita revenue includes $48.7 million in collaboration revenue in the North American profit share territory, $5.9 million in royalty revenue in the European territory from Kyowa Kirin Co., and product revenue for Crysvita in other regions of $2.7 million. The Company recognized $8.3 million in Mepsevii product revenue, $2.0 million in UX007 named patient revenue, and also $0.5 million in revenue from its research agreement with Bayer.

    Operating Expenses

    Total operating expenses for the third quarter of 2019 were $143.8 million compared with $101.4 million for the same period in 2018, including a $20.0 million research and development expense in the third quarter of 2019 from the GeneTx agreement, and non-cash stock-based compensation of $19.9 million and $20.7 million in the third quarter of 2019 and 2018, respectively. Total operating expenses for the nine months ended September 30, 2019 were $397.8 million compared with $316.3 million for the same period in 2018, including a $15.6 million research and development expense from the Arcturus collaboration amendment, a $20.0 million research and development expense from the GeneTx agreement, and non-cash stock-based compensation of $62.3 million and $59.0 million in the first nine months of 2019 and 2018, respectively. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows, and advances its pipeline.

    For the third quarter of 2019, Ultragenyx reported a net loss of $113.0 million, or $1.96 per share, basic and diluted, compared with a net loss for the third quarter of 2018 of $87.3 million, or $1.74 per share, basic and diluted. For the nine months ended September 30, 2019, net loss was $308.9 million, or $5.50 per share, basic and diluted, compared with a net loss for the same period in 2018 of $109.8 million, or $2.22 per share, basic and diluted. The loss for the third quarter of 2019 and for the nine months ended September 30, 2019 includes unrealized gains of $2.2 million and $12.0 million, respectively, from the fair value adjustment on the investment in Arcturus equity securities. The loss for the first nine months of 2018 includes a $40.3 million gain from Ultragenyx's portion of the sale of the priority review voucher (PRV) received with the Crysvita U.S. Food and Drug Administration (FDA) approval and includes a $130.0 million gain from the sale of the PRV received with the Mepsevii FDA approval. The net loss for the first nine months of 2019 reflected cash used in operations of $273.3 million compared to $234.7 million for the same period in 2018.

    Cash, Cash Equivalents and Investments

    Cash, cash equivalents and available-for-sale investments were $527.1 million as of September 30, 2019.

    Recent Updates and Upcoming Milestones

    Crysvita Commercial Progress in X-Linked Hypophosphatemia (XLH)

    • Strong U.S. launch continues, with approximately 170 new patients on reimbursed commercial therapy in the United States in the third quarter 2019.
    • U.S. Crysvita label expanded. The U.S. FDA approved a label expansion for Crysvita, which now includes clinical data demonstrating superiority of treatment with Crysvita versus conventional therapy in pediatric patients, and improvement in stiffness and maintenance of efficacy in adult patients with longer-term treatment. The indication has also been expanded to include infants as young as six months of age.  

    Crysvita Supplemental Biologics License Application (sBLA) for Tumor-Induced Osteomalacia (TIO)

    • Ultragenyx plans to submit a sBLA to the U.S. FDA for Crysvita for the treatment of TIO in the first half of 2020. 

    UX007 NDA for the Treatment of Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Accepted for Review by U.S. FDA

    • The U.S. FDA accepted for review the New Drug Application (NDA) and has set a Prescription Drug User Fee Act (PDUFA) date of July 31, 2020. The FDA has indicated that it is not currently planning to hold an advisory committee meeting to discuss the application.

    Clinical-stage Gene Therapy Programs Advance

    • DTX301 Phase 1/2 study in ornithine transcarbamylase (OTC) deficiency:  Data from Cohorts 1 and 2 demonstrated that the two responders maintained ureagenesis levels above normal and normal ammonia levels for 78 and 52 weeks, respectively. There have been no infusion-related adverse events and no treatment-related serious adverse events reported. Ultragenyx plans to provide an update from the ongoing third dose cohort around the end of the year. The company also plans to proceed to an additional cohort using prophylactic steroids.
    • DTX401 Phase 1/2 Study in glycogen storage disease type Ia (GSDIa): Data from the first two dose cohorts demonstrated that all patients showed clinical responses, including improvements in glucose control shown by time to hypoglycemia and reductions in cornstarch requirements.  In the second dose cohort, all three patients showed a more meaningful reduction in glycogen storage as measured by liver fat fraction and improvements in metabolism as measured by lactate levels. Based on these data, Ultragenyx is conducting a confirmatory expansion cohort of three patients at the second cohort dose of 6.0 × 10^12 GC/kg, with data from this cohort expected in the first half of 2020.

    Corporate Updates

    • Agreement with GeneTx Biotherapeutics to advance treatment for Angelman Syndrome. Ultragenyx and GeneTx are collaborating on the development of GeneTx's GTX-102, an antisense oligonucleotide (ASO) for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder. GTX-102 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation, and the Investigational New Drug (IND) timing is on track. Ultragenyx made an upfront payment of $20 million which included an exclusive option to acquire GeneTx. This option may be exercised any time prior to 30 days following FDA acceptance of the IND for GTX-102. The option period may be extended by paying an option extension payment of $25 million.

    Conference Call and Webcast Information

    Ultragenyx will host a conference call today, Tuesday, November 5, 2019, at 2 p.m. PT/ 5 p.m. ET to discuss third quarter 2019 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 9946526. The replay of the call will be available for one year.

    About Ultragenyx

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    Crysvita® (burosumab) is approved in the United States for the treatment of XLH in adult and pediatric patients six months of age and older, and in Canada and Brazil for the treatment of XLH in adult and pediatric patients one year of age and older. Crysvita has received conditional marketing authorization in Europe for the treatment of XLH with radiographic evidence of bone disease in children one year of age and older and adolescents with growing skeletons. Mepsevii® (vestronidase alfa) is approved in the United States and Brazil for the treatment of children and adults with MPS VII. In Europe, Mepsevii is approved under exceptional circumstances for the treatment of non-neurological manifestations of MPS VII.

    In addition to the approved treatments for XLH and MPS VII, Ultragenyx has four clinical development programs. Crysvita is being studied for the treatment of TIO, a rare disease that impairs bone mineralization. UX007 is being studied in patients with LC-FAOD, a genetic disorder in which the body is unable to convert long chain fatty acids into energy. The company has two gene therapy pipeline candidates: DTX301 is an AAV8 gene therapy product candidate in development for the treatment of patients with OTC deficiency, the most common urea cycle disorder; and DTX401 is an AAV8 gene therapy product candidate for the treatment of patients with GSDIa.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 2, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

       
      Ultragenyx Pharmaceutical Inc.
      Selected Statement of Operations Financial Data
      (in thousands, except share and per share amounts)
      (unaudited)
                     
        Three Months Ended September 30,   Nine Months Ended September 30,
          2019       2018       2019       2018  
      Statement of Operations Data:              
      Revenues:              
      Collaboration and license $ 21,585     $ 9,015     $ 55,070     $ 28,896  
      Product sales   4,215       2,748       13,051       6,338  
      Total revenues   25,800       11,763       68,121       35,234  
      Operating expenses:              
      Cost of sales   2,683       273       3,901       639  
      Research and development   100,144       70,041       274,294       222,380  
      Selling, general and administrative   41,006       31,095       119,647       93,248  
      Total operating expenses   143,833       101,409       397,842       316,267  
      Loss from operations   (118,033 )     (89,646 )     (329,721 )     (281,033 )
      Gain from sale of priority review vouchers                     170,322  
      Change in fair value of investment in Arcturus equity
      securities
      2,166             11,994        
      Other income (expense), net   3,166       2,583       9,527       1,314  
      Loss before income taxes   (112,701 )     (87,063 )     (308,200 )     (109,397 )
      Provision for income taxes   (293 )     (247 )     (722 )     (388 )
      Net loss $ (112,994 )   $ (87,310 )   $ (308,922 )   $ (109,785 )
      Net loss per share, basic and diluted $ (1.96 )   $ (1.74 )   $ (5.50 )   $ (2.22 )
      Shares used in computing net loss per share, basic and
      diluted
      57,707,694       50,319,772       56,161,996       49,447,889  
                     


      Ultragenyx Pharmaceutical Inc.
      Selected Balance Sheet Financial Data
      (in thousands)
      (unaudited)
          September 30,   December 31,
            2019     2018
      Balance Sheet Data:        
      Cash, cash equivalents, and available-for-sale investments   $ 527,058   $ 459,706
      Working capital     523,179     447,644
      Total assets     879,944     719,558
      Total stockholders' equity     727,265     608,908

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

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  43. NOVATO, Calif., Oct. 30, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Tuesday, November 5, 2019 at 5pm ET to discuss third quarter 2019 financial results and provide a corporate update.

    The live and replayed webcast of the call will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 9946526. The replay of the call will be available for one year.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx…

    NOVATO, Calif., Oct. 30, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call on Tuesday, November 5, 2019 at 5pm ET to discuss third quarter 2019 financial results and provide a corporate update.

    The live and replayed webcast of the call will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (International) and enter the passcode 9946526. The replay of the call will be available for one year.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the company's website at www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

    Primary Logo

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  44. NOVATO, Calif., Oct. 14, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company's New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy. The FDA has assigned a standard review designation with a Prescription Drug User Fee Act (PDUFA) target date of July 31, 2020.

    "We appreciate FDA's collaboration on evaluating these data for this devastating…

    NOVATO, Calif., Oct. 14, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the company's New Drug Application (NDA) for UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of genetic disorders in which the body is unable to convert long-chain fatty acids into energy. The FDA has assigned a standard review designation with a Prescription Drug User Fee Act (PDUFA) target date of July 31, 2020.

    "We appreciate FDA's collaboration on evaluating these data for this devastating and often catastrophic disease," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "We look forward to working with the agency in the coming months with the goal of bringing this potential new treatment to patients as quickly as possible."

    The FDA has indicated that it is not currently planning to hold an advisory committee meeting to discuss the application. The NDA filing is supported by a comprehensive package of data including results from a company-sponsored Phase 2 study of UX007 in 29 patients, a long-term safety and efficacy extension study in 75 patients including 20 patients who were previously naïve to UX007, a retrospective medical record review of 20 original compassionate use patients, 67 patients treated through expanded access, and a randomized controlled investigator-sponsored study of 32 patients showing an effect on cardiac function.

    About LC-FAOD 
    LC-FAOD are a group of autosomal recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to convert long-chain fatty acids into energy. The inability to produce energy from fat can lead to severe depletion of glucose in the body and serious liver, muscle and heart disease, which can lead to hospitalizations or early death. LC-FAOD are included in newborn screening panels across the U.S. and in certain European countries. Current treatment options for LC-FAOD include avoidance of fasting, low-fat/high-carbohydrate diets, carnitine and medium-chain triglyceride (MCT) oil, a medical food product. Despite current management, many patients with LC-FAOD experience significant metabolic events that require hospitalization or can result in death.

    About UX007 (triheptanoin)
    UX007 is a highly purified, pharmaceutical-grade, medium-chain triglyceride consisting of three 7-carbon fatty acids on a glycerol backbone created via a multi-step chemical process. It is an investigational therapy that directly addresses the deficiencies in LC-FAOD by providing patients with an alternative energy source that can be metabolized to increase intermediate substrates in the Krebs cycle, a key energy-generating process. Unlike typical even-chain fatty acids, one of the Krebs cycle intermediates generated specifically by UX007 also can be converted to new glucose, potentially providing an important added therapeutic effect, particularly when glucose levels are too low.

    About Ultragenyx Pharmaceutical, Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    Ultragenyx Forward-Looking Statements
    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 2, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contact:
    Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

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  45. NOVATO, Calif., Oct. 02, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil Kakkis, the company's Chief Executive Officer and President will present at the Jefferies Gene Therapy/Editing Summit on Tuesday, October 8, 2019 at 9:45 AM ET in New York, NY.

    The live and archived webcast of the company presentations will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products…

    NOVATO, Calif., Oct. 02, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Emil Kakkis, the company's Chief Executive Officer and President will present at the Jefferies Gene Therapy/Editing Summit on Tuesday, October 8, 2019 at 9:45 AM ET in New York, NY.

    The live and archived webcast of the company presentations will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

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    View Full Article Hide Full Article
  46. Expanded Label Includes Additional Clinical Data Highlighting Superiority Over Conventional Therapy for Pediatric Patients and Extends Current U.S. Indication to Patients Aged Six Months and Older

    Expanded Label Includes Data Demonstrating Improvement in Stiffness, Continued Further Healing of Fractures and Maintenance of Efficacy with Longer-Term Treatment in Adults

    NOVATO, Calif., and TOKYO, Sept. 30, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. today announced that the U.S. Food and Drug Administration (FDA) has approved a label expansion for Crysvita® (burosumab). The label…

    Expanded Label Includes Additional Clinical Data Highlighting Superiority Over Conventional Therapy for Pediatric Patients and Extends Current U.S. Indication to Patients Aged Six Months and Older

    Expanded Label Includes Data Demonstrating Improvement in Stiffness, Continued Further Healing of Fractures and Maintenance of Efficacy with Longer-Term Treatment in Adults

    NOVATO, Calif., and TOKYO, Sept. 30, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd. today announced that the U.S. Food and Drug Administration (FDA) has approved a label expansion for Crysvita® (burosumab). The label has been updated to include new clinical data demonstrating superiority of treatment with Crysvita versus oral phosphate and active vitamin D (conventional therapy) in pediatric patients with XLH, and improvement in stiffness, and maintenance of efficacy of Crysvita in adult patients with longer-term treatment. The indication has also been expanded to include infants as young as six months of age.

    Crysvita is an antibody that binds to and inhibits the biological activity of fibroblast growth factor 23 (FGF23), restoring renal phosphate reabsorption and increasing the serum concentration of 1,25 dihydroxy vitamin D. It was first approved in the U.S. in April 2018 for the treatment of XLH in adult and pediatric patients one year of age and older.

    "We are pleased that the updated Crysvita label includes compelling results from a controlled clinical trial demonstrating that Crysvita is significantly more effective than conventional therapy in normalizing phosphorus levels, reducing rickets and lower leg deformity, and improving growth in children with XLH. Furthermore, longer-term data from a study in adults are included showing important sustained Crysvita efficacy in this life-long disease," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "As part of our commitment to support the XLH community, we worked closely with the FDA to update the prescribing information for Crysvita so physicians could make more informed treatment decisions for their patients with XLH and ensure that younger patients could be treated as this disease is present at birth."

    For the pediatric XLH population, the U.S. label update is based on 64-week efficacy and safety data from the randomized active-controlled Phase 3 study of Crysvita compared with oral phosphate and active vitamin D (conventional therapy) in 61 children with XLH. The results showed that Crysvita was superior to conventional therapy for all key efficacy endpoints, showing a meaningful improvement in rickets severity, lower limb deformity and growth. The 64-week safety profile was similar to that observed at 40 weeks and in other Crysvita pediatric XLH studies.

    For the adult XLH population, the label update incorporates results from the open-label treatment period of the Phase 3 study in 134 adult patients with XLH through week 48, demonstrating that serum phosphorus levels were maintained with no evidence of loss of effect. The updated label also includes results demonstrating the continued healing of fractures and pseudofractures at week 48 and improvement in the patient-reported outcome of stiffness at week 24. The safety profile is consistent with what has been previously observed in this study, with no new adverse reactions identified during the extended treatment period.

    About X-Linked Hypophosphatemia (XLH)
    XLH is a rare, hereditary, progressive and lifelong skeletal disorder characterized by renal phosphate wasting caused by excess FGF23 production. It affects both children and adults. In children, XLH causes rickets that leads to lower-extremity deformity, delayed growth and decreased height. Adults with XLH have an increased risk of fractures.

    About Crysvita
    Crysvita (burosumab-twza) is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Kirin, against the phosphaturic hormone fibroblast growth factor 23 (FGF23). FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate wasting in XLH is caused by excessive levels and activity of FGF23. Crysvita is designed to bind to and thereby inhibit the biological activity of FGF23. By blocking excess FGF23 in patients, Crysvita is intended to increase phosphate reabsorption from the kidney and increase the production of vitamin D, which enhances intestinal absorption of phosphate and calcium.

    Kyowa Kirin and Ultragenyx have been collaborating in the development and commercialization of Crysvita globally based on the collaboration and license agreement between the parties.

    INDICATION (IN THE U.S)
    Crysvita is indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients 6 months of age and older.

    IMPORTANT SAFETY INFORMATION

    Crysvita should not be taken if:

    • An oral phosphate supplement and/or a specific form of vitamin D supplement are taken (such as calcitriol, paricalcitol, doxercalciferol, calcifediol).
    • Phosphorus levels from a blood sample are within or above the normal range for age.
    • Kidney problems are present.

    What is the most important information to know about Crysvita?

    • Some patients developed allergic reactions (rash and hives) while taking Crysvita. Doctors will monitor for symptoms of an allergic reaction while Crysvita is taken.
    • High levels of phosphorus in the blood have been reported in some patients taking Crysvita. This may be related to a risk of high calcium levels in the kidneys. Doctors will collect samples to monitor levels.
    • Administration of Crysvita may result in reactions at the injection site, such as hives, reddening of the skin, rash, swelling, bruising, pain, severe itching of the skin, and collection of blood outside of a blood vessel (hematoma).

    What are the possible side effects of Crysvita?

    • The most common adverse reactions that were seen in children with XLH are:
      º  Fever
      º  Injection site reaction
      º  Cough
      º  Vomiting
      º  Pain in arms and legs
      º  Headache
      º  Tooth infection
      º  Dental cavities
      º  Diarrhea
      º  Decreased vitamin D levels
      º  Toothache
      º  Constipation
      º  Muscle pain
      º  Rash
      º  Dizziness
      º  Nausea
    • The most common adverse reactions that were seen in adults with XLH are:
      º  Back pain
      º  Headache
      º  Tooth infection
      º  Restless leg syndrome
      º  Decreased vitamin D levels
      º  Dizziness
      º  Muscle spasms
      º  Constipation
      º  Phosphorus levels increased in the blood
    • Narrowing of the spaces within the spine is common in adults with XLH and pressure on the spinal cord has been reported in adults taking Crysvita. It is not known if taking Crysvita worsens the narrowing of the spaces within the spine or the pressure on the spinal cord.

    Before taking Crysvita, doctors should be informed about all medications (including supplements) and medical conditions, including if:

    • One is taking oral phosphate and/or active vitamin D (such as calcitriol, paricalcitol, doxercalciferol, calcifediol).
    • One is pregnant, thinks she may be pregnant, or plans to become pregnant. There is not enough experience to know if Crysvita may harm an unborn baby. Report pregnancies to the Kyowa Kirin, Inc. Adverse Event reporting line at 1-888-756-8657.
    • One is breastfeeding or plans to breastfeed. There is not enough experience to know if Crysvita passes into breast milk. Women should talk with their doctors about the best way to feed their babies while taking Crysvita.

    While taking Crysvita, doctors should be informed if one experiences:

    • An allergic reaction such as rash or hives
    • A rash, swelling, bruising or other reaction at the injection site
    • New or worsening restless leg syndrome

    These are not all the possible side effects of Crysvita. Doctors should be contacted for medical advice about side effects.

    Side effects may be reported to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. Side effects may also be reported to Kyowa Kirin, Inc. at 1-888-756-8657.

    Please see full Prescribing Information for additional Important Safety Information.

    About Ultragenyx Pharmaceutical, Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    About Kyowa Kirin
    Kyowa Kirin commits to innovate drug discovery driven by state-of-the-art technologies. The company focuses on creating new values in the four therapeutic areas: nephrology, oncology, immunology/allergy and neurology. Under the Kyowa Kirin brand, the employees from 36 group companies across North America, EMEA and Asia/Oceania unite to champion the interests of patients and their caregivers in discovering solutions wherever there are unmet medical needs. You can learn more about the business of Kyowa Kirin at www.kyowakirin.com.

    Ultragenyx Forward-Looking Statements
    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding market opportunities for our products, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our collaboration with Kyowa Kirin, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties from ongoing regulatory scrutiny related to our commercialized products, potential adverse side effects caused by our products and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 2, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:

    Ultragenyx
    Investors & Media
    Danielle Keatley
    +1-415-475-6876

    Kyowa Kirin Co. Ltd.
    Media
    Hiroki Nakamura
    +81-3-5205-7205
    Email:

    Primary Logo

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  47. NOVATO, Calif. and TOKYO, Sept. 10, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., ((Kyowa Kirin, TYO:4151) today announced plans to submit a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The decision to submit follows the completion of a pre-sBLA meeting with the FDA and agreement on the filing package. The submission of the Crysvita…

    NOVATO, Calif. and TOKYO, Sept. 10, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Kyowa Kirin Co., Ltd., ((Kyowa Kirin, TYO:4151) today announced plans to submit a supplemental Biologics License Application (sBLA) to the U.S. Food and Drug Administration (FDA) for Crysvita® (burosumab) for the treatment of FGF23-related hypophosphatemia associated with phosphaturic mesenchymal tumors (tumor-induced osteomalacia; TIO) that cannot be curatively resected or localized. The decision to submit follows the completion of a pre-sBLA meeting with the FDA and agreement on the filing package. The submission of the Crysvita sBLA is planned for the first half of 2020 and will be based on the current clinical data package.

    "Based on productive discussions with the FDA, we will be moving forward expeditiously with an sBLA filing for Crysvita in tumor-induced osteomalacia," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "We look forward to working with the agency during the review process, and we are committed to getting this therapy to patients with this serious disease with significant unmet medical need."

    The BLA package will include data from two single-arm Phase 2 studies, a 144-week Phase 2 study in 14 adult patients conducted by Ultragenyx in the U.S. and an 88-week Phase 2 study in 13 adult patients conducted by Kyowa Kirin in Japan and South Korea. In both studies, Crysvita was associated with increases in serum phosphorus and serum 1,25-dihydroxyvitamin D levels. Increased phosphate levels led to improvements in osteomalacia, mobility, and vitality. Bone scans also demonstrated an increase in healed fractures and decrease in new fractures during Crysvita treatment. Adverse events generally reflected the patients' underlying disease, and there were no serious treatment-related adverse events during the studies. 

    Crysvita is approved by the U.S. FDA, Health Canada, and Brazil's National Health Surveillance Agency (ANVISA) for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older, and has received European conditional marketing authorization for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons.

    See below for Important Safety Information for Crysvita in X-linked hypophosphatemia.

    About Tumor-Induced Osteomalacia (TIO)

    TIO is caused by typically benign tumors that produce excess levels of fibroblast growth factor 23 (FGF23), causing phosphate wasting in the urine that leads to severe hypophosphatemia, osteomalacia, muscle weakness, fatigue, bone pain, and fractures. The symptoms rapidly resolve if the causal tumors or lesion can be resected; however, there are cases in which resection is not feasible or recurrence of the tumor occurs after resection. In patients for whom the tumor or lesion is inoperable, the current treatment consists of oral phosphate and/or vitamin D replacement. Efficacy of this management is often limited, as it does not treat the underlying disease and its benefits must be balanced with monitoring for potential risks such as nephrocalcinosis, hypercalciuria, and hyperparathyroidism. There are an estimated 500-1,000 patients with TIO in the United States, and approximately half of all cases are inoperable.

    About Crysvita

    Crysvita (burosumab-twza) is a recombinant fully human monoclonal IgG1 antibody, discovered by Kyowa Hakko Kirin, against the phosphaturic hormone FGF23. FGF23 is a hormone that reduces serum levels of phosphorus and active vitamin D by regulating phosphate excretion and active vitamin D production by the kidney. Phosphate wasting in TIO and other hypophosphatemic conditions, including XLH, is caused by excessive levels and activity of FGF23. Crysvita is designed to bind to and thereby inhibit the biological activity of FGF23. By blocking excess FGF23 in patients with TIO and XLH, Crysvita is intended to increase phosphate reabsorption from the kidney and increase the production of vitamin D, which enhances intestinal absorption of phosphate and calcium.

    Kyowa Kirin and Ultragenyx have been collaborating in the development and commercialization of Crysvita globally based on the collaboration and license agreement between the parties.

    Important Safety Information in X-Linked Hypophosphatemia

    CONTRAINDICATIONS

    • Do not use CRYSVITA with oral phosphate and active vitamin D analogs.
    • Do not initiate CRYSVITA treatment if serum phosphorus is within or above the normal range for age.
    • CRYSVITA is contraindicated in patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism.

    WARNINGS AND PRECAUTIONS
    Hypersensitivity

    • Hypersensitivity reactions (e.g. rash, urticaria) have been reported in patients with CRYSVITA. Discontinue CRYSVITA if serious hypersensitivity reactions occur and initiate appropriate medical treatment.

    Hyperphosphatemia and Risk of Nephrocalcinosis

    • Increases in serum phosphorus to above the upper limit of normal may be associated with an increased risk of nephrocalcinosis. For patients already taking CRYSVITA, dose interruption and/or dose reduction may be required based on a patient's serum phosphorus levels.

    Injection Site Reactions

    • Administration of CRYSVITA may result in local injection site reactions. Discontinue CRYSVITA if severe injection site reactions occur and administer appropriate medical treatment.

    ADVERSE REACTIONS
    Pediatric Patients

    • The most common adverse reactions (more than 10%) in pediatric XLH patients are: headache, injection site reaction, vomiting, pyrexia, pain in extremity, vitamin D decreased, rash, toothache, myalgia, tooth abscess, and dizziness.

    Adult Patients

    • The most common adverse reactions (more than 5% and in at least 2 patients more than placebo) in adult XLH patients are: back pain, headache, tooth infection, restless leg syndrome, vitamin D decreased, dizziness, constipation, blood phosphorus increased.
    • Spinal stenosis is prevalent in adults with XLH and spinal cord compression has been reported. It is unknown if CRYSVITA therapy exacerbates spinal stenosis or spinal cord compression.

    USE IN SPECIFIC POPULATIONS

    • There are no available data on CRYSVITA use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. Serum phosphorus levels should be monitored throughout pregnancy. Report pregnancies to the Ultragenyx Adverse Event reporting line at 1-888-756-8657.
    • There is no information regarding the presence of CRYSVITA in human milk, or the effects of CRYSVITA on milk production or the breastfed infant. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for CRYSVITA and any potential adverse effects on the breastfed infant from CRYSVITA or from the underlying maternal condition.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    About Kyowa Kirin

    Kyowa Kirin commits to innovate drug discovery driven by state-of-the-art technologies. The company focuses on creating new values in the four therapeutic areas: nephrology, oncology, immunology/allergy and neurology. Under the Kyowa Kirin brand, the employees from 36 group companies across North America, EMEA and Asia/Oceania unite to champion the interests of patients and their caregivers in discovering solutions wherever there are unmet medical needs. You can learn more about the business of Kyowa Kirin at www.kyowakirin.com.

    Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and future regulatory interactions, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our collaboration with Kyowa Kirin, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements.  For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on August 2, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts

    Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley
    415-475-6876

    Kyowa Kirin Co. Ltd.
    Media
    Hiroki Nakamura
    +81-3-5205-7205
    Email:

    Primary Logo

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  48. Increased Time to Hypoglycemia and Reduction in Cornstarch Use in Cohorts 1 and 2; Improvement of Additional Key Metabolic Measures Observed

    Cohort 1 Patients Continue to Demonstrate Long-Term, Durable Responses

    Company to Enroll Three Patients in Expansion Cohort to Confirm 6.0 × 10^12 GC/kg Dose as Optimal Dose for Phase 3 Study

    Company to Host Conference Call Today at 8:30am ET to Discuss Results

    NOVATO, Calif., Sept. 04, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced positive data from the second dose cohort of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus…

    Increased Time to Hypoglycemia and Reduction in Cornstarch Use in Cohorts 1 and 2; Improvement of Additional Key Metabolic Measures Observed

    Cohort 1 Patients Continue to Demonstrate Long-Term, Durable Responses

    Company to Enroll Three Patients in Expansion Cohort to Confirm 6.0 × 10^12 GC/kg Dose as Optimal Dose for Phase 3 Study

    Company to Host Conference Call Today at 8:30am ET to Discuss Results

    NOVATO, Calif., Sept. 04, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced positive data from the second dose cohort of the ongoing Phase 1/2 study of DTX401, an adeno-associated virus (AAV) based gene therapy for the treatment of glycogen storage disease type Ia (GSDIa). All three patients in Cohort 2 have shown a clinical response with improvements in glucose control and other metabolic parameters compared to baseline. Patients in the first, lower dose cohort continue to show longer-term durability in response. The data were presented today at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2019 Annual Symposium in Rotterdam, the Netherlands. Based on these results, Ultragenyx will enroll three additional patients into an expansion of cohort 2 at the same 6.0 × 10^12 GC/kg dose to confirm its use in the Phase 3 study.

    "Early data from Cohort 2 are promising, with increased week 6 glucose levels and reduced lactate measurements during the controlled fasting challenge indicating a higher level of transgene expression compared to Cohort 1. This is further supported by MRI data indicating better reduction of glycogen storage in the liver," said Eric Crombez, M.D., Chief Medical Officer of the Ultragenyx Gene Therapy development unit. "Additional assessments of time to hypoglycemia after further weaning off of cornstarch use, combined with the other positive clinical and metabolic data, should support 6.0 × 10^12 GC/kg as the dose for the upcoming Phase 3 study."  

    "The response to DTX401 has been better than expected. We have seen all of the patients wean their therapy with some already discontinuing therapy. Missed cornstarch doses no longer are resulting in hypoglycemia which previously could have been life threatening," said David Weinstein, M.D., M.M.Sc., Professor and Director, Glycogen Storage Disease Program at Connecticut Children's Medical Center and UConn Health. "The GSD community is extremely excited by the results obtained after the first year of the trial."

    DTX401 Data Summary
    Time to Hypoglycemia
    All patients have demonstrated a biological and clinical response, reflected by an increase in time to hypoglycemia (defined as glucose <60 mg/dL or onset of clinical symptoms) compared to baseline during a controlled-fasting challenge, including a sustained response through week 52 for the first two patients enrolled. All patients will continue to be followed to evaluate long-term durability of treatment.

    Cohort 2 patients showed significant hyperglycemia during the 6 week controlled fasting challenge, which was not observed at baseline and is consistent with a level of transgene expression and glucose release that was more than that observed with Cohort 1. This level of hyperglycemia results in an exaggerated insulin response, which in turn has the potential to drive hypoglycemia and an early termination of the controlled fasting challenge. Nevertheless, all Cohort 2 patients still demonstrated an increase in time to hypoglycemia in the fasting challenge.

      Time to Hypoglycemia (hours)
      Cohort 1
    (2e12 GC/kg)
    Cohort 2
    (6e12 GC/kg)
    Visit Patient 1 Patient 2 Patient 3 Patient 4 Patient 5 Patient 6
    Baseline 3.8 4.1 5.4 6.1 3.6 3.8
    W12 7.7 9.0 6.5 7.2 5.4 6.0 (+58%)
    W24 6.8 13.1 6.5 (+20%) 7.0 (+15%) 4.4 (+22%) NR
    W52 7.7 (+103%) 10.6 (+159%) NR NR NR NR

    NR= Not yet reached

    Cornstarch Reduction
    All patients have demonstrated normalization of daily glucose levels, which has allowed for clinically significant reductions in the amount of cornstarch. Significant cornstarch reduction generally occurred by week 24, and in Cohort 1 where longer-term data are available, cornstarch doses have continued to be titrated down including complete discontinuation of cornstarch for patient 1. Early titration results in Cohort 2 are promising, and titration is expected to continue over time as seen in Cohort 1.

      Cornstarch consumption (grams)
      Cohort 1
    (2e12 GC/kg)
    Cohort 2
    (6e12 GC/kg)
    Visit Patient 1 Patient 2 Patient 3 Patient 4 Patient 5 Patient 6
    Baseline 405 171 269 325 268 329
    W6 355 165 255 270 268 341
    W12 160 165 138 265 270 253
    W24 94 96 76 100 (-69%) 224 (-16%) 105(-68%)*
    W52 0 (-100%) 76 (-56%) 57 (-79%) NR NR NR

    *Cornstarch level at week 18

    Additional Metabolic Measures
    Patients have shown meaningful improvements in additional metabolic and clinical assessments. Patients in Cohort 2 have all demonstrated lower lactate levels after treatment compared to baseline and compared to Cohort 1. All three Cohort 2 patients have also shown consistent early reductions in liver fat fraction (representative of glycogen storage) as measured by MRI at week 12. Cohort 2 patients had significant hyperglycemia during their 6-week controlled fasting challenges. This hyperglycemia may have been exacerbated by the effect of steroids on the steroid-responsive normal promoter of the transgene. This further supports the effect of DTX401 on glucose production and regulation, as GSDIa patients are normally expected to become hypoglycemic during steroid treatment. The cornstarch dose at the beginning of the controlled fasting challenge will be reduced in the future to limit the onset of an insulin spike that can significantly reduce blood sugar, affecting the timed hypoglycemia test.

    Safety Summary
    As of August 31, 2019, there have been no infusion-related adverse events and no treatment-related serious adverse events reported. All adverse events have been Grade 1 or 2. Four of the six patients in Cohorts 1 and 2 had mild, asymptomatic elevations in alanine aminotransferase (ALT), similar to what has been observed in other programs using AAV-based gene therapy, and were successfully treated with a reactive tapering course of steroids.

    DTX401 Expansion Cohort Enrollment Ongoing with Data Expected First Half 2020
    Based on these results, Ultragenyx is proceeding to a confirmatory expansion cohort that will enroll three patients at the second cohort dose of 6.0 × 10^12 GC/kg. Data from the expansion cohort are expected in the first half of 2020. If the expansion cohort results are consistent with those observed to date, then the Phase 3 trial expected to begin in 2020 will study this dose level.

    DTX401 Phase 1/2 Study Design
    The open-label, multicenter Phase 1/2 study is evaluating the safety, tolerability and therapeutic response of DTX401 in adults with GSDIa. DTX401 is an AAV8 expressing the glucose-6-phosphatase gene (G6Pase-α) under control of the native promoter. In the first cohort, three patients received a single 2.0 × 10^12 GC/kg dose of DTX401. In the second cohort, three patients each received a single 6.0 × 10^12 GC/kg dose of DTX401. In the expansion cohort, three patients will receive the same dose of DTX401 as in Cohort 2. Key efficacy assessments include time to hypoglycemia (defined as glucose <60 mg/dL or onset of clinical symptoms) during a controlled in-hospital fasting challenge, cornstarch reduction, impact on biomarkers such as lactic acid, and measurement of glycogen storage in liver by MRI.

    About GSDIa
    GSDIa is the most severe genetically inherited glycogen storage disease. It is caused by a defective gene for the enzyme G6Pase-α, resulting in the inability to regulate blood sugar (glucose). Hypoglycemia in patients with GSDIa can be life-threatening, while the accumulation of the complex sugar glycogen in certain organs and tissues can impair the ability of these tissues to function normally. If chronically untreated, patients can develop severe lactic acidosis, progress to renal failure, and potentially die in infancy or childhood. There are no approved pharmacologic therapies. An estimated 6,000 patients worldwide are affected by GSDIa.

    About DTX401

    DTX401 is an investigational AAV8 gene therapy designed to deliver stable expression and activity of G6Pase-α under control of the native promoter. DTX401 is administered as a single intravenous infusion and has been shown in preclinical studies to improve G6Pase-α activity and reduce hepatic glycogen levels, a well-described biomarker of disease progression. DTX401 has been granted Orphan Drug Designation in both the United States and Europe, Regenerative Medicine Advanced Therapy (RMAT) designation and Fast Track designation in the United States.

    Conference Call Details

    Ultragenyx will host a conference call today, Wednesday, September 4 at 8:30 am ET during which Dr. Kakkis and Dr. Weinstein will discuss results of the data being presented at the SSIEM Conference. The live and replayed webcast of the call will be available through the company's website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial 855-797-6910 (USA) or 262-912-6260 (international) and enter the passcode 5648479. The replay of the call will be available for one year.

    About Ultragenyx Pharmaceutical, Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    Ultragenyx Forward-Looking Statements
    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, including the potential for substantial delays and the risk that earlier study results may not be predictive of future study results, the lack of predictability in the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report filed on Form 10-Q with the Securities and Exchange Commission on August 2, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contact Ultragenyx Pharmaceutical Inc.

    Investors & Media
    Danielle Keatley
    415-475-6876

    Primary Logo

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  49. SARASOTA, Fla. and NOVATO, Calif., Sept. 03, 2019 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation and Rare Pediatric Disease Designation to GTX-102 for the treatment of Angelman Syndrome, a serious, debilitating rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide.

    "The granting of these designations from the FDA is an enormous milestone for the Angelman community," stated Paula Evans, Chief Executive Officer of GeneTx. "We are thrilled that the FDA…

    SARASOTA, Fla. and NOVATO, Calif., Sept. 03, 2019 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation and Rare Pediatric Disease Designation to GTX-102 for the treatment of Angelman Syndrome, a serious, debilitating rare neurogenetic disorder that affects approximately 1 in 15,000 people worldwide.

    "The granting of these designations from the FDA is an enormous milestone for the Angelman community," stated Paula Evans, Chief Executive Officer of GeneTx. "We are thrilled that the FDA recognizes the urgency for the development of effective therapeutics in this patient population and that GeneTx, a company launched because of the efforts of this patient community, is the first in this space to receive the Rare Pediatric Disease Designation."

    GTX-102 is currently in late preclinical development, and an investigational new drug (IND) application is expected to be filed with the FDA in the first half of 2020. In August 2019, Ultragenyx and GeneTx announced a partnership to develop GTX-102 with Ultragenyx receiving an exclusive option to acquire GeneTx.

    About Orphan Drug Designation

    The FDA Orphan Drug Designation program provides orphan status to drugs and biologics that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the U.S. Among the benefits of orphan designation in the U.S. are seven years of market exclusivity following FDA approval, waiver or partial payment of application fees, and tax credits for clinical testing expenses conducted after orphan designation is received.

    About Rare Pediatric Disease Designation

    The FDA defines a "rare pediatric disease" as a serious or life-threatening rare disease in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years. Under the FDA's Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

    About Angelman Syndrome

    Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the target of GTX-102. In almost all cases of Angelman syndrome the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is typically not inherited but instead occurs spontaneously.

    Individuals with Angelman syndrome have developmental delay, balance issues, motor impairment, and debilitating seizures. Some individuals with Angelman syndrome are unable to walk and most do not speak. Anxiety and disturbed sleep can be serious challenges in individuals with Angelman syndrome. While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome suggesting that improvement of symptoms can potentially be achieved at any age.

    About GTX-102

    GTX-102 is an investigational antisense oligonucleotide designed to target and inhibit expression UBE3A-AS. Studies show that GTX-102 reduces the levels of UBE3A-AS and reactivates expression of the paternal UBE3A allele in neurons of the CNS. Reactivation of paternal UBE3A expression with a UBE3A-AS in animal models of Angelman syndrome has been associated with improvements in some of the neurological symptoms associated with the condition. GTX-102 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA).

    About GeneTx Biotherapeutics

    GeneTx Biotherapeutics LLC is a startup biotechnology company singularly focused on developing and commercializing a safe and effective antisense therapeutic for the treatment of Angelman syndrome. GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research. GeneTx licensed the rights to antisense technology intellectual property from The Texas A&M University System in December 2017.

    About Ultragenyx Pharmaceutical Inc.

    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    Ultragenyx Forward-Looking Statements

    Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our partnership with GeneTx, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report filed on Form 10-Q with the Securities and Exchange Commission on August 2, 2019, and its subsequent periodic reports filed with the Securities and Exchange Commission.

    Contacts:
    Ultragenyx
    Investors & Media
    Danielle Keatley
    415-475-6876

    GeneTx
    Paula Evans
    630-639-7271

    Primary Logo

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  50. NOVATO, Calif., Aug. 30, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, will present at the following conferences:

    • Baird Global Healthcare Conference on Thursday, September 5, 2019 at 12:50pm ET in New York, NY
       
    • Morgan Stanley 17th Annual Global Healthcare Conference on Wednesday, September 11, 2019 at 2:10pm ET in New York, NY

    The live and archived webcast of the company presentations will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days…

    NOVATO, Calif., Aug. 30, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today announced that Shalini Sharp, the company's Chief Financial Officer, will present at the following conferences:

    • Baird Global Healthcare Conference on Thursday, September 5, 2019 at 12:50pm ET in New York, NY
       
    • Morgan Stanley 17th Annual Global Healthcare Conference on Wednesday, September 11, 2019 at 2:10pm ET in New York, NY

    The live and archived webcast of the company presentations will be accessible from the company's website at http://ir.ultragenyx.com/events.cfm. The replay of the webcast will be available for 90 days.

    About Ultragenyx Pharmaceutical Inc.
    Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

    The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx's strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

    For more information on Ultragenyx, please visit the Company's website at www.ultragenyx.com.

    Contact Ultragenyx Pharmaceutical Inc.
    Investors & Media
    Danielle Keatley              
    415-475-6876

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