Ultragenyx Explained

Ultragenyx Pharmaceutical Inc.
Type:Public
Founder:Emil Kakkis
Key People:Emil Kakkis (president)
Areas Served:International
Hq Location City:Novato, California
Hq Location Country:U.S.
Num Employees:893 (2020)

Ultragenyx Pharmaceutical Inc. is an American biopharmaceutical company involved in the research and development of novel products for treatment of rare and ultra-rare genetic diseases for which there are typically no approved treatments and high unmet medical need. The company works with multiple drug modalities including biologics, small molecule, gene therapies, and ASO and mRNAs in the disease categories of bone, endocrine, metabolic, muscle and CNS diseases.

Ultragenyx is based in Novato, CA and Brisbane, CA[1] and has a presence in the Boston area, including a gene therapy plant under construction as of 2021.[2] The company’s Latin American headquarters is located in Miami.[3]

Ultragenyx collaborates on product development with other companies including GeneTX, Kyowa Hakko Kirin, Mereo Biopharma and Daiichi Sankyo.[4] Ultragenyx has three products Burosumab, Triheptanoin and Vestronidase alfa that have received FDA approval and several others currently in clinical trials. The company also holds the non-US commercial rights to Regeneron’s evinacumab-dgnb, which is approved by the FDA and EMA.[5] The company also has therapies approved outside the U.S. in Canada, Latin America, Europe, and Japan.[6]

In 2020 and 2021, Ultragenyx was named one of Deloitte's fasting growing technology and life sciences companies in North America[7] and one of the best companies to work for by BioSpace.[8] Also in 2021, the company’s CEO, Emil Kakkis, was awarded the California Life Sciences Pantheon Leadership award.[9]

History

Ultragenyx Pharmaceutical Inc. was founded in 2010 by Emil Kakkis based on his history of developing therapies for rare disease starting at Harbor-UCLA Medical Center, continuing through his role as chief medical officer of BioMarin and as founder of the EveryLife Foundation.[10] He became the chief executive officer and president, focusing the company to meet the treatment needs for rare diseases understood as affecting fewer than 200,000 people in the U.S.[11] [12] In 2014 the USA-based company went public with an IPO that raised $126 million.[13] In 2015 Ultragenyx began collaborating with Arcturus Therapeutics to develop mRNA products.[14]

Ultragenyx acquired Dimension Therapeutics in 2017[15] to obtain adeno-associated virus (AAV)-based gene therapy manufacturing technology as well as three internal candidates and one partnered candidate with Bayer for hemophilia A.[16]

In 2017, the company discontinued development of Ace-ERs treatment for GNE myopathy after the product didn't meet the study's primary endpoint which was significant improvement in arm strength. It also failed to meet three key secondary endpoints that evaluated patients' physical functions, leg muscle strength and knee extension force.[17] Previous trials in mice had shown efficacy.[18]

In 2020 Ultragenyx announced a new gene therapy plant being built near Boston.[19]

In July 2022 Ultragenyx announced that it would exercise its option to acquire GeneTx Biotherapeutics for an up-front cash payment of $75 million, plus another $115 million in potential milestone-dependent payments. Ultragenyx will add the antisense oligonucleotide therapy GTX-102 to its broad pipeline of therapies indicated for various rare diseases. GTX-102 is currently in early-stage development for Angelman syndrome.[20] The deal was completed in August 2022.

Products

Pipeline

Biologic

Gene therapy

ASO/mRNA

Notes and References

  1. Web site: 2021-11-03. Ultragenyx reports nearly $73M Q3 loss as R&D spending up 30%. 2022-02-23. The North Bay Business Journal. en-US.
  2. Web site: Rare disease drug maker Ultragenyx plans to hire 100-150 at new gene therapy facility near Boston. 2022-02-23. www.bizjournals.com.
  3. Web site: Bartucci . Camila . 2021-11-23 . Ultragenyx abre central en Miami, Latam . 2022-03-03 . PharmaBiz.NET . es.
  4. Denworth . Lydia . 2020 . Is gene therapy ready to treat some forms of autism? . Science . 10.1126/science.abf2497. 242454742 .
  5. Web site: 2022-01-21 . Novato Firm Partners With Rival To Treat Rare Illness . 2022-03-15 . Novato, CA Patch . en.
  6. Web site: 2021-02-17 . Health Canada Approves Ultragenyx' Drug for Group of Rare Metabolic Disorders . 2022-03-18 . Global Genes . en-US.
  7. Web site: 2021 Technology Fast 500 Award Winners . 2022-03-18 . Deloitte United States . en-us.
  8. Web site: BioSpace Best Places to Work jobs Choose from 3,724 live job openings . 2022-03-22 . www.biospace.com.
  9. Web site: 2021-11-17 . Marin County pharmaceuticals CEO wins lifetime achievement award . 2022-03-25 . The North Bay Business Journal . en-US.
  10. Web site: Providence Dr Emil Kakkis. 2022-02-25. NorthBay biz. en-US.
  11. Web site: The road to a 'miracle drug': How Ultragenyx and partners found their way to the newest rare disease treatment . 2022-03-08 . www.bizjournals.com.
  12. Web site: Commissioner . Office of the . 2020-10-05 . Rare Diseases at FDA . 2022-03-08 . FDA . en.
  13. Web site: Ultragenyx raises $126 million in IPO. February 5, 2014.
  14. Web site: Writer. GEN Staff. 2015-10-29. Ultragenyx, Arcturus Launch Up-to-$1.56B+ Rare Disease Collaboration. 2022-02-15. GEN - Genetic Engineering and Biotechnology News. en-US.
  15. Web site: September 18, 2017. Bruised Ultragenyx attempts swoop for in-demand Dimension.
  16. Web site: Writer. GEN Staff. 2017-10-02. Dimension Therapeutics Spurns Regenxbio for Sweeter Ultragenyx Offer. 2022-02-09. GEN - Genetic Engineering and Biotechnology News. en-US.
  17. Web site: August 23, 2017 . Ultragenyx shelves genetic disease drug after trial failure .
  18. Takeda.
  19. Web site: Rare disease drug maker Ultragenyx plans to hire 100-150 at new gene therapy facility near Boston . 2022-03-17 . www.bizjournals.com.
  20. Web site: Ultragenyx Pharmaceutical. July 19, 2022.
  21. Web site: Writer . GEN Staff . 2017-11-16 . FDA Approves Ultragenyx Enzyme Replacement Therapy for MPS VII . 2022-02-10 . GEN - Genetic Engineering and Biotechnology News . en-US.
  22. 29679282. 2018. Lamb. Y. N.. Burosumab: First Global Approval. Drugs. 78. 6. 707–714. 10.1007/s40265-018-0905-7. 5022649.
  23. Web site: Specialty Pipeline Monthly Update. September 2018.
  24. Web site: 2020-06-22. Crysvita Approved for Treatment of Tumor-Induced Osteomalacia. 2022-02-04. MPR. en-US.
  25. Web site: FDA Greenlights Ultragenyx's Dojolvi for Long-Chain Fatty Acid Oxidation Disorders. 2022-02-10. BioSpace. en-US.
  26. Web site: Bulik . Beth Snyder . 2021-01-29 . Ultragenyx earns 2 rare disease drug approvals—and a validated commercial strategy . 2022-03-25 . Fierce Pharma . en.
  27. Web site: Sagonowsky . Eric . 2021-02-11 . Regeneron's Evkeeza, carrying big price tag, wins FDA approval in ultra-rare cholesterol disease . 2022-03-11 . Fierce Pharma . en.
  28. Web site: Adams . Ben . 2022-01-07 . Regeneron hands over Evkeeza rights to Ultragenyx outside US, with an R&D asset also thrown in . 2022-03-11 . Fierce Pharma . en.
  29. Web site: December 21, 2020. Mereo BioPharma Group Pact With Ultragenyx Pharmaceutical to Develop Setrusumab to Strengthen Bones.
  30. Web site: Company Growth, Ambition Drive Ultragenyx's Continued Success . 2022-03-03 . BioSpace . en-US.
  31. Web site: PhD . Marta Figueiredo . First 2 Adults with Severe Hemophilia A Respond Well to Gene… – Hemophilia News Today . 2022-03-17 . en-US.
  32. Web site: 2021-08-26 . Astra gets Wilson disease bonus . 2022-03-22 . Evaluate.com . en.
  33. Web site: PhD . Aisha I. Abdullah . Ultragenyx and Solid Biosciences Partner on DMD Gene Therapy – Muscular Dystrophy News . 2022-03-25 . en-US.
  34. Web site: A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome (KIK-AS). 2020.
  35. Web site: GeneTx and Ultragenyx Announce Investigational New Drug (IND). . January 15, 2020.
  36. 29791829. 2018. 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.. Burosumab Therapy in Children with X-Linked Hypophosphatemia. The New England Journal of Medicine. 378. 21. 1987–1998. 10.1056/NEJMoa1714641. 1805/18603. 44135503. free.
  37. Web site: Biotech Companies Announce Big Drug Wins with FDA. 2022-02-25. BioSpace. en-US.
  38. Web site: mRNA therapeutics - Arcturus Therapeutics/Ultragenyx Pharmaceutical. 21 Jun 2019. Adis Insights, Springer.

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