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Ultragenyx Pharmaceutical Inc.
Company type	Public
Traded as	Nasdaq: RARE; Russell 1000 component;
Industry	Biotechnology; Life Sciences;
Founded	2010; 15 years ago
Founder	Emil Kakkis
Headquarters	Novato, California, U.S.
Areas served	International
Key people	Emil Kakkis (president)
Products	Research Antibodies; Biotech;
Revenue	US$271 million (2020)
Number of employees	893 (2020)
Website	ultragenyx.com

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^[2] and has a presence in the Boston area, including a gene therapy plant under construction as of 2021.^[3] The company’s Latin American headquarters is located in Miami.^[4]

Ultragenyx collaborates on product development with other companies including GeneTX, Kyowa Hakko Kirin, Mereo Biopharma and Daiichi Sankyo.^[5] 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.^[6] The company also has therapies approved outside the U.S. in Canada, Latin America, Europe, and Japan.^[7]

In 2020 and 2021, Ultragenyx was named one of Deloitte's fasting growing technology and life sciences companies in North America^[8] and one of the best companies to work for by BioSpace.^[9] Also in 2021, the company’s CEO, Emil Kakkis, was awarded the California Life Sciences Pantheon Leadership award.^[10]

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.^[11] 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.^[12]^[13] In 2014 the USA-based company went public with an IPO that raised $126 million.^[14] In 2015 Ultragenyx began collaborating with Arcturus Therapeutics to develop mRNA products.^[15]

Ultragenyx acquired Dimension Therapeutics in 2017^[16] 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.^[17]

In 2017, the company discontinued development of their Ace-ERs treatment for GNE myopathy after the product did not meet the study's primary endpoint, which was significant improvement in arm strength. It also failed to meet three secondary endpoints that evaluated patients' physical functions: leg muscle strength and knee extension force,^[18] which previous trials in mice had shown efficacy.^[19]

In 2020 Ultragenyx announced a new gene therapy plant being built near Boston.^[20]

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.^[21] The deal was completed in August 2022.

Products

Vestronidase alfa (brand name Mepsevii) was approved in November 2017 to treat children and adults with the inherited metabolic condition mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome.^[22]
Burosumab (KRN-23; brand name Crysvita) was approved in 2018 by the FDA to treat X-linked hypophosphatemia.^[23]^[24] In 2020 the drug was approved to treat tumor-induced osteomalacia.^[25]
Triheptanoin (brand name Dojolvi) a purified medium-chain triglyceride, was approved in 2020 for the treatment of long-chain fatty acid oxidation disorders in which the body is unable to produce energy from fat.^[26] Due to its odd-chain properties, Triheptanoin is broken down into metabolites that replace deficient intermediates in the Citric Acid Cycle.^[27] Trials on this drug as treatment for a different condition, Glut1 deficiency, a seizure disorder, were halted in 2017 due to a failure of a mid stage study.^[18]
Evinacumab-dgnb (brand name Evkeeza) was approved in February 2021 for the treatment of homozygous familial hypercholesterolemia.^[28] Developed by Regeneron, Ultragenyx acquired the sales and development rights in 2022 in markets outside the United States.^[29]

Pipeline

Biologic

UX143 (Setrusumab) - under investigation for osteogenesis imperfecta, a rare genetic disease with no approved therapies.^[30]

Gene therapy

DTX401 - in clinical development for glycogen storage disease type 1s (GSD1a).^[31]
DTX301 - in clinical development for treatment of ornithine transcarbamylase (OTC) deficiency.^[31]
DTX201/BAY 2599023 - in clinical development for treatment of hemophilia A.^[32]
UX701 - in clinical development for treatment of Wilson Disease.^[33]
UX810 - in clinical development for treatment of Duchenne Muscular Dystrophy (DMD).^[34]

ASO/mRNA

GTX-102 - in clinical development as a possible treatment option for Angelman syndrome, a rare, neurogenetic disorder.^[35]^[36]^[37]
UX053 - in clinical development for glycogen storage disease type III (GSDIII).^[38]^[39]

References

^ "Ultragenyx Pharmaceutical Revenue 2012-2021 | RARE".
^ "Ultragenyx reports nearly $73M Q3 loss as R&D spending up 30%". The North Bay Business Journal. 2021-11-03. Retrieved 2022-02-23.
^ "Rare disease drug maker Ultragenyx plans to hire 100-150 at new gene therapy facility near Boston". www.bizjournals.com. Retrieved 2022-02-23.
^ Bartucci, Camila (2021-11-23). "Ultragenyx abre central en Miami, Latam". PharmaBiz.NET (in Spanish). Retrieved 2022-03-03.
^ Denworth, Lydia (2020). "Is gene therapy ready to treat some forms of autism?". Science. doi:10.1126/science.abf2497. S2CID 242454742.
^ "Novato Firm Partners With Rival To Treat Rare Illness". Novato, CA Patch. 2022-01-21. Retrieved 2022-03-15.
^ "Health Canada Approves Ultragenyx' Drug for Group of Rare Metabolic Disorders". Global Genes. 2021-02-17. Retrieved 2022-03-18.
^ "2021 Technology Fast 500 Award Winners". Deloitte United States. Retrieved 2022-03-18.
^ "BioSpace | Best Places to Work jobs | Choose from 3,724 live job openings". www.biospace.com. Retrieved 2022-03-22.
^ "Marin County pharmaceuticals CEO wins lifetime achievement award". The North Bay Business Journal. 2021-11-17. Retrieved 2022-03-25.
^ "Providence Dr Emil Kakkis". NorthBay biz. Retrieved 2022-02-25.
^ "The road to a 'miracle drug': How Ultragenyx and partners found their way to the newest rare disease treatment". www.bizjournals.com. Retrieved 2022-03-08.
^ Commissioner, Office of the (2020-10-05). "Rare Diseases at FDA". FDA. Retrieved 2022-03-08.
^ "Ultragenyx raises $126 million in IPO". February 5, 2014.
^ Writer, GEN Staff (2015-10-29). "Ultragenyx, Arcturus Launch Up-to-$1.56B+ Rare Disease Collaboration". GEN - Genetic Engineering and Biotechnology News. Retrieved 2022-02-15.
^ "Bruised Ultragenyx attempts swoop for in-demand Dimension". September 18, 2017.
^ Writer, GEN Staff (2017-10-02). "Dimension Therapeutics Spurns Regenxbio for Sweeter Ultragenyx Offer". GEN - Genetic Engineering and Biotechnology News. Retrieved 2022-02-09.
^ ^a ^b "Ultragenyx shelves genetic disease drug after trial failure". August 23, 2017.
^ Takeda. Translational Research in Muscular Dystrophy, p. 72, at Google Books
^ "Rare disease drug maker Ultragenyx plans to hire 100-150 at new gene therapy facility near Boston". www.bizjournals.com. Retrieved 2022-03-17.
^ "Ultragenyx Pharmaceutical". July 19, 2022.
^ Writer, GEN Staff (2017-11-16). "FDA Approves Ultragenyx Enzyme Replacement Therapy for MPS VII". GEN - Genetic Engineering and Biotechnology News. Retrieved 2022-02-10.
^ Lamb, Y. N. (2018). "Burosumab: First Global Approval". Drugs. 78 (6): 707–714. doi:10.1007/s40265-018-0905-7. PMID 29679282. S2CID 5022649.
^ "Specialty Pipeline Monthly Update" (PDF). September 2018.
^ "Crysvita Approved for Treatment of Tumor-Induced Osteomalacia". MPR. 2020-06-22. Retrieved 2022-02-04.
^ "FDA Greenlights Ultragenyx's Dojolvi for Long-Chain Fatty Acid Oxidation Disorders". BioSpace. Retrieved 2022-02-10.
^ Bulik, Beth Snyder (2021-01-29). "Ultragenyx earns 2 rare disease drug approvals—and a validated commercial strategy". Fierce Pharma. Retrieved 2022-03-25.
^ Sagonowsky, Eric (2021-02-11). "Regeneron's Evkeeza, carrying big price tag, wins FDA approval in ultra-rare cholesterol disease". Fierce Pharma. Retrieved 2022-03-11.
^ Adams, Ben (2022-01-07). "Regeneron hands over Evkeeza rights to Ultragenyx outside US, with an R&D asset also thrown in". Fierce Pharma. Retrieved 2022-03-11.
^ "Mereo BioPharma Group Pact With Ultragenyx Pharmaceutical to Develop Setrusumab to Strengthen Bones". December 21, 2020.
^ ^a ^b "Company Growth, Ambition Drive Ultragenyx's Continued Success". BioSpace. Retrieved 2022-03-03.
^ PhD, Marta Figueiredo. "First 2 Adults with Severe Hemophilia A Respond Well to Gene… – Hemophilia News Today". Retrieved 2022-03-17.
^ "Astra gets Wilson disease bonus". Evaluate.com. 2021-08-26. Retrieved 2022-03-22.
^ PhD, Aisha I. Abdullah. "Ultragenyx and Solid Biosciences Partner on DMD Gene Therapy – Muscular Dystrophy News". Retrieved 2022-03-25.
^ "A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome (KIK-AS)". 2020.
^ "GeneTx and Ultragenyx Announce Investigational New Drug (IND)". CNN. January 15, 2020.
^ 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.
^ "Biotech Companies Announce Big Drug Wins with FDA". BioSpace. Retrieved 2022-02-25.
^ "mRNA therapeutics - Arcturus Therapeutics/Ultragenyx Pharmaceutical". Adis Insights, Springer. 21 Jun 2019.

[1] "Ultragenyx Pharmaceutical Revenue 2012-2021 | RARE".

[2] "Ultragenyx reports nearly $73M Q3 loss as R&D spending up 30%". The North Bay Business Journal. 2021-11-03. Retrieved 2022-02-23.

[3] "Rare disease drug maker Ultragenyx plans to hire 100-150 at new gene therapy facility near Boston". www.bizjournals.com. Retrieved 2022-02-23.

[4] Bartucci, Camila (2021-11-23). "Ultragenyx abre central en Miami, Latam". PharmaBiz.NET (in Spanish). Retrieved 2022-03-03.

[5] Denworth, Lydia (2020). "Is gene therapy ready to treat some forms of autism?". Science. doi:10.1126/science.abf2497. S2CID 242454742.

[6] "Novato Firm Partners With Rival To Treat Rare Illness". Novato, CA Patch. 2022-01-21. Retrieved 2022-03-15.

[7] "Health Canada Approves Ultragenyx' Drug for Group of Rare Metabolic Disorders". Global Genes. 2021-02-17. Retrieved 2022-03-18.

[8] "2021 Technology Fast 500 Award Winners". Deloitte United States. Retrieved 2022-03-18.

[9] "BioSpace | Best Places to Work jobs | Choose from 3,724 live job openings". www.biospace.com. Retrieved 2022-03-22.

[10] "Marin County pharmaceuticals CEO wins lifetime achievement award". The North Bay Business Journal. 2021-11-17. Retrieved 2022-03-25.

[11] "Providence Dr Emil Kakkis". NorthBay biz. Retrieved 2022-02-25.

[12] "The road to a 'miracle drug': How Ultragenyx and partners found their way to the newest rare disease treatment". www.bizjournals.com. Retrieved 2022-03-08.

[13] Commissioner, Office of the (2020-10-05). "Rare Diseases at FDA". FDA. Retrieved 2022-03-08.

[14] "Ultragenyx raises $126 million in IPO". February 5, 2014.

[15] Writer, GEN Staff (2015-10-29). "Ultragenyx, Arcturus Launch Up-to-$1.56B+ Rare Disease Collaboration". GEN - Genetic Engineering and Biotechnology News. Retrieved 2022-02-15.

[16] "Bruised Ultragenyx attempts swoop for in-demand Dimension". September 18, 2017.

[17] Writer, GEN Staff (2017-10-02). "Dimension Therapeutics Spurns Regenxbio for Sweeter Ultragenyx Offer". GEN - Genetic Engineering and Biotechnology News. Retrieved 2022-02-09.

[biopharma-18] "Ultragenyx shelves genetic disease drug after trial failure". August 23, 2017.

[19] Takeda. Translational Research in Muscular Dystrophy, p. 72, at Google Books

[20] "Rare disease drug maker Ultragenyx plans to hire 100-150 at new gene therapy facility near Boston". www.bizjournals.com. Retrieved 2022-03-17.

[21] "Ultragenyx Pharmaceutical". July 19, 2022.

[22] Writer, GEN Staff (2017-11-16). "FDA Approves Ultragenyx Enzyme Replacement Therapy for MPS VII". GEN - Genetic Engineering and Biotechnology News. Retrieved 2022-02-10.

[23] Lamb, Y. N. (2018). "Burosumab: First Global Approval". Drugs. 78 (6): 707–714. doi:10.1007/s40265-018-0905-7. PMID 29679282. S2CID 5022649.

[24] "Specialty Pipeline Monthly Update" (PDF). September 2018.

[25] "Crysvita Approved for Treatment of Tumor-Induced Osteomalacia". MPR. 2020-06-22. Retrieved 2022-02-04.

[26] "FDA Greenlights Ultragenyx's Dojolvi for Long-Chain Fatty Acid Oxidation Disorders". BioSpace. Retrieved 2022-02-10.

[27] Bulik, Beth Snyder (2021-01-29). "Ultragenyx earns 2 rare disease drug approvals—and a validated commercial strategy". Fierce Pharma. Retrieved 2022-03-25.

[28] Sagonowsky, Eric (2021-02-11). "Regeneron's Evkeeza, carrying big price tag, wins FDA approval in ultra-rare cholesterol disease". Fierce Pharma. Retrieved 2022-03-11.

[29] Adams, Ben (2022-01-07). "Regeneron hands over Evkeeza rights to Ultragenyx outside US, with an R&D asset also thrown in". Fierce Pharma. Retrieved 2022-03-11.

[30] "Mereo BioPharma Group Pact With Ultragenyx Pharmaceutical to Develop Setrusumab to Strengthen Bones". December 21, 2020.

[:0-31] "Company Growth, Ambition Drive Ultragenyx's Continued Success". BioSpace. Retrieved 2022-03-03.

[32] PhD, Marta Figueiredo. "First 2 Adults with Severe Hemophilia A Respond Well to Gene… – Hemophilia News Today". Retrieved 2022-03-17.

[33] "Astra gets Wilson disease bonus". Evaluate.com. 2021-08-26. Retrieved 2022-03-22.

[34] PhD, Aisha I. Abdullah. "Ultragenyx and Solid Biosciences Partner on DMD Gene Therapy – Muscular Dystrophy News". Retrieved 2022-03-25.

[35] "A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome (KIK-AS)". 2020.

[36] "GeneTx and Ultragenyx Announce Investigational New Drug (IND)". CNN. January 15, 2020.

[37] 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.

[38] "Biotech Companies Announce Big Drug Wins with FDA". BioSpace. Retrieved 2022-02-25.

[39] "mRNA therapeutics - Arcturus Therapeutics/Ultragenyx Pharmaceutical". Adis Insights, Springer. 21 Jun 2019.

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PROFILPELAJAR.COM

Ultragenyx

History

Products

Pipeline

Biologic

Gene therapy

ASO/mRNA

References

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