RARE — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-06

• UX111 (rebisufligene etisparvovec) for the treatment of Sanfilippo syndrome type A, or MPS IIIA

• GTX-102 (apazunersen) for the treatment of Angelman Syndrome

• GTX-102 is an antisense oligonucleotide, or ASO, administered by intrathecal injection that inhibits expression of the paternal UBE3A antisense. GTX-102 is being developed for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder caused by loss-of-function of the maternal…

• UX016 is a small-molecule prodrug composed of sialic acid (SA; also known as N‑acetylneuraminic acid [NANA]) and a C16 fatty acid tail designed to improve biodistribution to target tissues, like muscle, more effectively and efficiently than free SA. UX016 is being developed for the treatment of GNE …

• UX143 (setrusumab) for the treatment of Osteogenesis Imperfecta, or OI

Text removed vs the prior filing · source: 10-Q · 2025-11-05

• UX143 (setrusumab) for the treatment of Osteogenesis Imperfecta, or OI

• UX143 is a fully human monoclonal antibody administered by IV that inhibits sclerostin, a protein that acts on a key bone-signaling pathway by inhibiting the activity of bone-forming cells and promoting bone resorption. UX143 is being developed for the treatment of OI, or brittle bone disease, which…

• GTX-102 (apazunersen) for the treatment of Angelman Syndrome

• GTX-102 is an antisense oligonucleotide, or ASO, administered by intrathecal injection that inhibits expression of the paternal UBE3A antisense. GTX-102 is being developed for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder caused by loss-of-function of the maternal…

• UX111 (rebisufligene etisparvovec) for the treatment of Sanfilippo syndrome type A or MPS IIIA

Text added vs the prior filing · source: 10-Q · 2026-05-06

• Ultragenyx Pharmaceutical Inc. and Baylor Research Institute v. Somerset Therapeutics LLC, Somerset Pharma LLC, Somerset Therapeutics Private Limited, and Odin Pharmaceuticals LLC

• In September 2024, we filed a patent infringement suit in the United States District Court for the District of New Jersey asserting U.S. Patent No. 8,697,748, or the ’748 patent, a 2029-expiring patent listed in the FDA’s Orange Book for Dojolvi® (triheptanoin), under the Hatch-Waxman Act against Na…

• In February 2026, we were granted U.S. Patent No. 12,551,461, or the ’461 patent. The ’461 patent was listed in the FDA’s Orange Book for Dojolvi® shortly after issuance. In April 2026, we filed an additional patent infringement suit asserting the ’461 patent against Navinta, Aurobindo, and Esjay. T…

• In April 2026, we filed a patent infringement suit asserting the ’748 and ’461 patents under the Hatch-Waxman Act against Somerset Therapeutics LLC, Somerset Pharma LLC, Somerset Therapeutics Private Limited, and Odin Pharmaceuticals LLC, or collectively, Somerset, in the United States District Cour…

• In addition to the ’748 and ’461 patents for Dojolvi listed in the Orange Book, Dojolvi is also protected in the U.S. by orphan drug exclusivity for the treatment of pediatric and adult patients with molecularly confirmed long-chain fatty acid oxidation disorders (LC-FAOD) until June 30, 2027.

Text removed vs the prior filing · source: 10-Q · 2025-11-05

• On September 26, 2024, we filed a patent infringement suit under the Hatch-Waxman Act against Navinta LLC, or Navinta, Aurobindo Pharma Limited, or Aurobindo, and Esjay Pharma LLC, or Esjay, in the United States District Court for the District of New Jersey. The suit is in response to notices from N…

• Aurobindo and Navinta answered the complaint in December 2024. Esjay filed a motion to dismiss the suit in December 2024. We filed an opposition to Esjay’s motion to dismiss in January 2025. In April 2025, Navinta filed a motion for judgment on the pleadings, which we opposed in May 2025. Discovery …

• Catalent filed its response, which included a motion to dismiss the fraud claim alleged in the suit, in December2024. We filed an amended complaint in reply to Catalent’s response in February 2025. Catalent moved to dismiss the amended complaint in March 2025. We filed our opposition to Catalent’s m…

• Except as disclosed above, we are not currently a party to any other material legal proceedings. We may, however, in the ordinary course of business face various claims brought by third parties or government regulators and, from time to time, make claims or take legal actions to assert our rights, i…

Text added vs the prior filing · source: 10-Q · 2026-05-06

• Our future financial performance depends on the successful commercialization of our products and product candidates.

• We are dependent on KKC for the supply and commercialization of Crysvita in certain major markets.

• We may face competition from biosimilars or from generic versions of our small-molecule products and product candidates.

• Changing regulatory standards may make it difficult to accurately predict the likelihood of obtaining marketing approval.

• Increasing use of social media could give rise to additional liability;

Text removed vs the prior filing · source: 10-Q · 2025-11-05

• The following description of the risk factors associated with our business includes any material changes to and supersedes the description of the risk factors associated with our business previously disclosed in Part I, Item 1A of the Annual Report.

• We have limited experience in generating revenue from product sales.

• We are dependent on KKC for the commercialization of Crysvita in certain major markets, including the U.S. and Canada, and for our supply of Crysvita in our markets.

• We may face competition from biosimilars of our biologics products and product candidates or from generic versions of our small-molecule products and product candidates, which may result in a material decline in sales of affected products.

• Our employees or consultants may engage in misconduct which could cause significant liability for us.

Text added vs the prior filing · source: 10-Q · 2026-05-06

• Karah Parschauer, Executive Vice President, Chief Legal Officer and Corporate Affairs

Text removed vs the prior filing · source: 10-Q · 2025-11-05

• Howard Horn, Chief Financial Officer and EVP, Corporate Strategy

• On September 15, 2025, Dr. Suliman amended this Rule 10b5-1 trading arrangement to, among other things, amend the number of shares that could be sold under the plan.