Ultragenyx

Ultragenyx is a biopharmaceutical company involved in the R&D of novel products used to treat rare and ultra-rare life-threatening diseases such as angelman syndrome and X-linked hypophosphatemia. The company has survived a number of drug failures in its past [2] and has learned to lean more on partnerships with traditional rivals Genetex, Kyowa Hakko Kirin, Mereo Biopharma and Daiichi Sankyo.[3] Burosumab, Triheptanoin, Vestronidase alfa have received FDA approval.

Ultragenyx
TypePublic
NASDAQ: RARE
IndustryBiotechnology
Life Sciences
Founded2010
Headquarters
Novato, CA
,
United States
Areas served
International
Key people
Emil Kakkis
president & founder
ProductsResearch Antibodies, Biotech
Revenue $104Million[1]
Number of employees
740 (2019)
Websitewww.ultragenyx.com

History

Ultragenyx was founded in 2010 by current CEO Emil Kakkis. Emil's past research into ultra-rare diseases steered the company's focus to "meeting the need for treatments for ultra-rare diseases"—understood as affecting fewer than one patient per 50,000 people.[4][5] Four years later on February 4, 2014 it went public with an IPO that raised $126 million.[6] At the time it had four drugs in clinical trials.

Notable products
  • Burosumab (KRN-23; brandname Crysvita) has been approved as a therapy for X-linked hypophosphatemia.[7][8] It works by raising the levels of phosphate in the blood thereby making it possible for faster healing of bone lesions in osteomalacia.[9]
  • Triheptanoin, a purified medium-chain triglyceride, has been approved for the treatment of long-chain fatty acid oxidation disorders, in which the body is unable to produce energy from fat.[10] Due to its odd-chain properties, Triheptanoin is broken down into metabolites that replace deficient intermediates in the Citric Acid Cycle.[11]
  • Vestronidase alfa It was approved in the United States in November 2017, to treat children and adults with an inherited metabolic condition called mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome.[12]

Investigational products

Collaboration with Arcturus

Since 2015 Ultragenix has been getting help from Arcturus Therapeutics to develop mRNA products. Arcturus' clinical programs also include a potential covid-19 vaccine candidate.[17]

Gene therapy technology

Research into new ways of using biotech appears to have caught the company's attention. Ultragenyx's adeno-associated virus (AAV)-based gene therapy manufacturing technology is being used to mass produce investigational drugs for AAV gene therapy.[19] This technology was acquired via a 2017 acquisition of Dimension Therapeutics.[20]

Product failures

The company's recent string of collaborations with competitors on product research and development has come after a number of its own product failures. Investment banks Evercore and Jefferies have said that a number of items in the product stream have "meaningful and underappreciated, clinical risk."[21] [22] The company has been accused of rushing through market application submissions irrespective of their completeness.[23]

  • Ace-ER's - Possible treatment for GNE Myopahy was terminated after failing to meet three key secondary endpoints that evaluated patients' physical functions, leg muscle strength and knee extension force.[21] It was previously shown to be effective in mice. [24]
  • UX007 - Despite failing a mid stage study, Ultragenyx continued forward with stage 3.[21]

References
  1. https://www.macrotrends.net/stocks/charts/RARE/ultragenyx-pharmaceutical/revenue
  2. "Ultragenyx looks ahead after triheptanoin failure". March 23, 2017.
  3. Denworth, Lydia (2020). "Is gene therapy ready to treat some forms of autism?". Science. doi:10.1126/science.abf2497.
  4. "US National Institute of Health Emil Kakkis Interview". 2018.
  5. "UCLA Medicine Fall 2001 Neufeld Steps Down as Department Chair with Big Plans for Her Future" (PDF). 2001.
  6. "Ultragenyx raises $126 million in IPO". February 5, 2014.
  7. Lamb, Y. N. (2018). "Burosumab: First Global Approval". Drugs. 78 (6): 707–714. doi:10.1007/s40265-018-0905-7. PMID 29679282. S2CID 5022649.
  8. "Specialty Pipeline Monthly Update" (PDF). September 2018.
  9. "FDA.gov". June 18, 2020.
  10. "2020: Truly the Year Of the Specialty Drug". December 15, 2020.
  11. "Baylor College of Medicine and Texas Children's Hospital". Retrieved December 27, 2020.
  12. "Drug Trial Snapshot: Mepsevii". U.S. Food and Drug Administration (FDA). 4 December 2017. Archived from the original on 10 December 2019. Retrieved 9 December 2019. This article incorporates text from this source, which is in the public domain.
  13. "A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome (KIK-AS)". 2020.
  14. "GeneTx and Ultragenyx Announce Investigational New Drug (IND)". January 15, 2020.
  15. Carpenter, T. O.; Whyte, M. P.; Imel, E. A.; Boot, A. M.; Högler, W.; Linglart, A.; Padidela, R.; Van't Hoff, W.; Mao, M.; Chen, C. Y.; Skrinar, A.; Kakkis, E.; San Martin, J.; Portale, A. A. (2018). "Burosumab Therapy in Children with X-Linked Hypophosphatemia". The New England Journal of Medicine. 378 (21): 1987–1998. doi:10.1056/NEJMoa1714641. hdl:1805/18603. PMID 29791829. S2CID 44135503.
  16. "Mereo BioPharma Group Pact With Ultragenyx Pharmaceutical to Develop Setrusumab to Strengthen Bones". December 21, 2020.
  17. "Global Legal Chronicle". June 1, 2020.
  18. "mRNA therapeutics - Arcturus Therapeutics/Ultragenyx Pharmaceutical". Adis Insights, Springer. 21 Jun 2019.
  19. "Daiichi Sankyo Enters Partnership with Ultragenyx for a Total of $225 Million". March 31, 2020.
  20. "Bruised Ultragenyx attempts swoop for in-demand Dimension". September 18, 2017.
  21. "Ultragenyx shelves genetic disease drug after trial failure". August 23, 2017.
  22. Dayen, David Monopolized: Life in the Age of Corporate Power, p. 120, at Google Books
  23. Geigert, John The Challenge of CMC Regulatory Compliance for Biopharmaceuticals, p. 420, at Google Books
  24. Takeda. Translational Research in Muscular Dystrophy, p. 72, at Google Books
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