RARE Ultragenyx Pharmaceutical Inc.
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
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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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DTX301
Ornithine Transcarbamylase (OTC) Deficiency
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Phase 1/2
Phase 1/2
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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 |
|---|---|---|
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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.
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Latest News
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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
-
View Full Article Hide Full Article
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 - Citi's 15th Annual BioPharma Virtual Conference as part of a panel discussion on Wednesday, September 9, 2020 at 4:15 PM ET.
-
View Full Article Hide Full Article
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 -
View Full Article Hide Full Article
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 -
View Full Article Hide Full Article
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 - 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.