RARE Ultragenyx Pharmaceutical Inc.

86.04
+1.39  (+2%)
Previous Close 84.65
Open 84.88
52 Week Low 31.99
52 Week High 86.11
Market Cap $5,140,820,222
Shares 59,749,189
Float 43,230,357
Enterprise Value $4,436,935,848
Volume 665,237
Av. Daily Volume 567,246
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Upcoming Catalysts

Drug Stage Catalyst Date
GTX-102
Angelman Syndrome
Phase 1/2
Phase 1/2
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Drug Pipeline

Drug Stage Notes
UX007 (triheptanoin)
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Approved
Approved
FDA Approval announced June 30, 2020.
Burosumab
Tumor-induced osteomalacia (TIO)
Approved
Approved
FDA Approval announced June 18, 2020.
DTX401
Glycogen Storage Disease Type Ia (GSDIa)
Phase 1/2
Phase 1/2
Phase 1/2 data noted all three patients in Cohort 3 responded and demonstrated more rapid reductions in cornstarch requirements compared to the first two cohorts.
DTX301
Ornithine Transcarbamylase (OTC) Deficiency
Phase 1/2
Phase 1/2
Phase 1/2 update at ASGCT on May 13, 2020 - All three patients in Cohort 3 now confirmed responders. Phase 3 trial to commence 1H 2021.
UX007 (triheptanoin)
Glut1 DS patients with the movement disorder phenotype
Phase 3
Phase 3
Phase 3 data released October 26, 2018 - endpoints not met.
Crysvita (Burosumab)
X-Linked Hypophosphatemia (XLH)
Approved
Approved
Approval announced April 17, 2018.
MEPSEVII (vestronidase alfa)
Mucopolysaccharidosis 7 (MPS 7)
Approved
Approved
Approved under priority review November 15, 2017.
Aceneuramic acid extended release (Ace-ER)
GNE Myopathy
Phase 3
Phase 3
Phase 3 data released August 22, 2017 - endpoints not met.

Latest News

  1. 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

                          

     

    Primary Logo

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  2. 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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  3. 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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  4. 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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  5. 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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    SOURCE GATT Technologies

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