RARE Ultragenyx Pharmaceutical Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
|---|---|---|
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GTX-102
Angelman Syndrome
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Phase 1/2
Phase 1/2
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
|
DTX301
Ornithine Transcarbamylase (OTC) Deficiency
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Phase 1/2
Phase 1/2
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Phase 1/2 ongoing. Phase 3 trial to commence 2H 2021.
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UX143 (setrusumab)
Osteogenesis Imperfecta
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Phase 2/3
Phase 2/3
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Phase 2/3 trial to commence 2H 2021.
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UX053
GSDIII debrancher deficiency
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Phase 1/2
Phase 1/2
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Phase 1/2 trial to commence 2H 2021.
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UX701
Wilson Disease
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Phase 1/2
Phase 1/2
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Phase 1/2/3 trial to commence 1H 2021.
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DTX401
Glycogen Storage Disease Type Ia (GSDIa)
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Phase 1/2
Phase 1/2
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Phase 1/2 trial ongoing. Phase 3 trial to commence 1H 2021.
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UX007 (triheptanoin)
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
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Approved
Approved
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FDA Approval announced June 30, 2020.
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Burosumab
Tumor-induced osteomalacia (TIO)
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Approved
Approved
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FDA Approval announced June 18, 2020.
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UX007 (triheptanoin)
Glut1 DS patients with the movement disorder phenotype
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Phase 3
Phase 3
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Phase 3 data released October 26, 2018 - endpoints not met.
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Crysvita (Burosumab)
X-Linked Hypophosphatemia (XLH)
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Approved
Approved
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Approval announced April 17, 2018.
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MEPSEVII (vestronidase alfa)
Mucopolysaccharidosis 7 (MPS 7)
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Approved
Approved
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Approved under priority review November 15, 2017.
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|
Aceneuramic acid extended release (Ace-ER)
GNE Myopathy
|
Phase 3
Phase 3
|
Phase 3 data released August 22, 2017 - endpoints not met.
|
Latest News
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Foundation's collaboration with premier rare disease company strengthens efforts to reach more patients in need
n-Lorem Foundation, a nonprofit organization that provides free, lifetime supplies of individualized RNA-targeted medicines to patients living with ultra-rare diseases, today announced a new partnership with Ultragenyx Pharmaceutical (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases.
n-Lorem Foundation is the first and only foundation with a mission to provide potentially life-saving treatments to patients who have diseases caused by extremely rare mutations (1 to 30 patients worldwide) for free, for life, by coupling advanced genomic diagnostics with antisense oligonucleotides (ASOs) medicines. The new collaboration with Ultragenyx, one of the leading rare disease companies in the world, and its CEO and founder, Dr. Emil Kakkis, bolsters n-Lorem's mission to bring immediate hope and rapid treatment to ultra-rare disease patients in need.
"On behalf of the patients we serve, I thank Dr. Emil Kakkis and Ultragenyx for their generous support, and I look forward to working with Emil and his team at Ultragenyx to bring therapies to patients with ultra-rare diseases for free, for life," said Dr. Stanley T. Crooke, Founder, CEO and Chairman of n-Lorem Foundation. "Ultragenyx joins Ionis Pharmaceuticals, Biogen, Charles River Labs, Covance by Labcorp, and Korea Institute of Toxicology (KIT) as industry partners supporting our efforts at n-Lorem. We look forward to working with other health care companies in the future."
Ultragenyx focuses on developing products for serious rare and ultra-rare genetic diseases, and Dr. Kakkis brings a wealth of valuable expertise through his work on policy and industry issues affecting rare disease treatment development. The additional resources, financial contribution and experience from both the company and Dr. Kakkis will be critical as n-Lorem continues to scale-up its unique charitable treatment model to help patients with ultra-rare mutations.
"Today, we have the science to treat some of the rarest diseases in the world, and these patients deserve treatments," said Emil D. Kakkis, M.D., Ph.D., CEO and President of Ultragenyx Pharmaceutical. "We are proud to partner with n-Lorem, a first-of-its-kind organization that is using a proven technology to develop treatments for patients with these ultra-rare diseases for the first time."
To learn how your organization can partner with n-Lorem and help answer the challenge of treating patients with ultra-rare diseases, contact Tracy Johnson, Executive Director ([email protected]). Learn more about n-Lorem's mission at www.nlorem.org, and please consider giving to n-Lorem to bring hope, possibility and treatment options to these desperate patients and families.
- Watch: n-Lorem Foundation: Creating a Better Future for Ultra-Rare, One Patient at a Time
- Watch: n-Lorem Foundation's 1st year of Progress
About n-Lorem Foundation
n-Lorem Foundation is a non-profit organization established to apply the efficiency, versatility and specificity of antisense technology to charitably provide experimental antisense oligonucleotide (ASO) medicines to treat patients with ultra-rare diseases (1 to 30 patients) that are the result of a single genetic defect unique to only one or very few individuals. ASOs are short strands of modified DNA that can specifically target the transcripts of a defective gene to correct the abnormality. The advantage of experimental ASO medicines is that they can be developed rapidly, inexpensively and are highly specific. n-Lorem was founded by Stanley T. Crooke, M.D., Ph.D., former chairman and CEO, and current executive chairman of the board of Ionis Pharmaceuticals, who founded Ionis Pharmaceuticals in 1989 and, through his vision and leadership, established the company as the leader in RNA-targeted therapeutics. To learn more about the n-Lorem Foundation, visit www.nlorem.org, and follow us on Twitter, Facebook, LinkedIn and YouTube.
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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210405005044/en/
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NOVATO, Calif., March 08, 2021 (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 the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for UX053, an investigational mRNA therapy being evaluated for the treatment of Glycogen Storage Disease Type III (GSDIII). Enrollment in a Phase 1/2 study is expected to begin in the second half of 2021.
"FDA IND clearance paves the way for UX053 to enter the clinic as the first possible pharmacologic treatment option for patients with GSDIII," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "UX053 is the most advanced of our investigational mRNA therapies, with multiple others in earlier preclinical development, and will be one of the first mRNA programs to enter clinical development for rare genetic diseases."
Ultragenyx is developing UX053 and a number of other mRNA therapies in the preclinical stage for undisclosed indications. These mRNA therapies come out of a long-term collaboration with Arcturus Therapeutics, a clinical-stage messenger RNA medicines company.
Study Design
The two-part Phase 1/2 clinical trial will evaluate the safety, tolerability, and efficacy of UX053 in adults with GSDIII. Part 1 is open label and will enroll up to 12 patients who receive a single ascending dose of UX053. Part 2 is a randomized, double-blind, placebo-controlled multi-ascending study of 5 doses. It will enroll up to 16 patients across four cohorts randomized 3:1 to UX053 or placebo. In addition to safety, tolerability, and pharmacokinetics, study endpoints include clinician- and patient-reported outcomes, muscle strength, and biomarkers of liver, cardiac, and muscle health.
About Glycogen Storage Disease Type III
Our preclinical candidate UX053 is being developed for the treatment of GSDIII, a disease caused by a glycogen debranching enzyme (AGL) deficiency that results in glycogen accumulation in the liver and muscle. GSDIII can cause hepatomegaly, hypoglycemia, hyperlipidemia, some progressive liver cirrhosis, and skeletal and cardiac muscle disease. There are no approved treatment options for GSDIII and the current standard of care is a strict diet, including frequent doses of cornstarch, to reduce the risk of hypoglycemia. GSDIII affects more than 10,000 patients in the developed world.
About UX053
UX053 is an investigational mRNA-based biologic therapy encoding full-length, glycogen debranching enzyme encapsulated in a lipid nanoparticle (LNP) designed to provide the deficient protein in GSDIII.
About Ultragenyx Pharmaceutical Inc.
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 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, risks related to reliance on third party partners to conduct certain activities on the company's behalf, 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 Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 12, 2021, and its subsequent periodic reports filed with the Securities and Exchange Commission.
Contact Ultragenyx Pharmaceutical Inc.
Investors & Media
Joshua Higa
415-660-0951
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NOVATO, Calif., March 03, 2021 (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 Barclays Global Healthcare Conference on Wednesday, March 10, 2021 at 4:45 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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NOVATO, Calif., Feb. 17, 2021 (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:
- SVB Leerink 10th Annual Global Healthcare Conference on Wednesday, February 24, 2021 at 2:20 PM ET.
- Cowen 41st Annual Health Care Conference on Monday, March 1, 2021 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 - SVB Leerink 10th Annual Global Healthcare Conference on Wednesday, February 24, 2021 at 2:20 PM ET.
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NOVATO, Calif., Feb. 17, 2021 (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 Health Canada has approved Dojolvi™ (triheptanoin) as a source of calories and fatty acids for the treatment of adult and pediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD). Long-chain fatty acid oxidation disorders (LC-FAOD) are a group of rare, genetic, life-threatening disorders caused by defects in the enzymes needed to produce energy from fatty acids. 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.
"Living with a serious disease like LC-FAOD comes with unique challenges such as management of caloric intake and around-the-clock monitoring of symptom deterioration and/or fatigue. We are pleased to bring Canadian patients with LC-FAOD an approved therapy to help them manage their disease," said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx. "This would not have been possible without the LC-FAOD community, including all the patients, families and physicians who participated in the clinical trials. Similar to the approach we took in the United States to ensure access following FDA approval of Dojolvi, our goal is to ensure that patients in Canada 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 marked 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 Canada due to the risk of 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 8,000 to 14,000 children and adults in the developed world, including Canada.
"The number of options available to treat patients with rare long chain fatty oxidation defects have been quite limited for many years," said Dr. Pranesh Chakraborty Associate Professor, Department of Pediatrics, University of Ottawa, Executive Director of Newborn Screening Ontario, and Co-Lead of the Canadian Inherited Metabolic Diseases Research Network. "Clinicians and patients, who have been working with researchers over many years to study the safety and efficacy of Dojolvi, are very excited to have this new therapy available to help patients in Canada."
Ultragenyx expects Dojolvi to be available to patients in Canada in April and will be working closely with private insurers and through the public drug plan process to pursue coverage for Dojolvi as quickly as possible. To support access, Ultragenyx's UltraCare® program helps patients and caregivers understand insurance coverage. The UltraCare program may also assist patients in finding support for access to Ultragenyx medicines, including Dojolvi. Dedicated UltraCare Nurse Case Managers are available Monday through Friday from 8 a.m. to 8 p.m. Eastern Time at 1 -833-388-5872 (U-LTRA).
About Dojolvi™ (triheptanoin)
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.Dojolvi is indicated as a source of calories and fatty acids for the treatment of adult and pediatric patients with long-chain fatty acid oxidation disorders (LC-FAOD).
For important safety information, please consult the Dojolvi Product Monograph at www.ultragenyx.com/canada/medicines/dojolvi-product-monograph-CANADA/
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.
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, risks related to reliance on third party partners to conduct certain activities on the company's behalf, 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 Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 12, 2021, and its subsequent periodic reports filed with the Securities and Exchange Commission.Contact Ultragenyx Pharmaceutical Inc.
Investors & Media
Joshua Higa
+1-415-660-0951