Ultragenyx announces 2020 milestones
Recent Updates and Upcoming Milestones: Crysvita for X-linked Hypophosphatemia: Non-dilutive $320 million royalty sale of future European royalties. In December, Ultragenyx sold to Royalty Pharma for $320 million its royalty interest in Crysvita in the European territory, where it is being commercialized by Kyowa Kirin Co., Ltd. Crysvita for Tumor-Induced Osteomalacia: Supplemental Biologics License Application submitted. Ultragenyx submitted the sBLA to the U.S. FDA on December 18, 2019 and expects to hear back from FDA on submission acceptance and review designation in February 2020. UX007 for Long-Chain Fatty Acid Oxidation Disorders: NDA under review by U.S. FDA. The U.S. FDA accepted for review the New Drug Application and has set a Prescription Drug User Fee Act date of July 31, 2020. The FDA has indicated that it is not currently planning to hold an advisory committee meeting to discuss the application. DTX301 for Ornithine Transcarbamylase Deficiency: Positive data from higher cohort of Phase 1/2 study; data from prophylactic steroid cohort in second half 2020. Recent positive data from Cohort 3 indicated two confirmed responders and a potential third responder out of three total patients, as well as a new responder in Cohort 2. There are currently up to six responders of the nine dosed to date with a more consistent response at higher doses. Ultragenyx is initiating a fourth cohort using prophylactic steroids at the same dose as Cohort 3. The first patient is expected to be enrolled in the first half of 2020, and data from the prophylactic steroid cohort are expected in the second half of 2020. DTX401 for Glycogen Storage Disease Type Ia: Positive data from Phase 1/2 study; enrollment complete in confirmatory cohort and data expected in first half 2020. Enrollment is complete in the confirmatory cohort of three patients at the second dose cohort, with data expected in the first half of 2020. Following the results of the confirmatory cohort, a Phase 3 study could begin in the second half of 2020. GTX-102 for Angelman Syndrome: Partnered program with GeneTx; IND expected in first half 2020. An investigational new drug application is expected in the first half of 2020. In August 2019, Ultragenyx and GeneTx announced a partnership to develop GTX-102 with Ultragenyx receiving an exclusive option to acquire GeneTx. UX701 for Wilson Disease: IND expected in second half 2020: An IND application is expected in the second half of 2020 for a new gene therapy for Wilson disease, a larger rare metabolic disease. UX701 will be the company's second clinical program to utilize its HeLa manufacturing system. The Hemophilia A program partnered with Bayer uses the HeLa system and has released its first clinical data.