Ultragenyx Reports Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance
Preliminary 2019 total revenue is approximately
Preliminary 2019 Crysvita revenue to Ultragenyx of approximately
2020 Crysvita revenue in Ultragenyx territories is expected to be in the range of
Year-end 2019 cash balance of greater than
“We passed the
2019 Preliminary Revenue, 2020 Crysvita Revenue Guidance, and 2019 Ending Cash Position
2019 Preliminary Revenue (unaudited)
Total revenue for the year ended
2020 Crysvita Guidance
For 2020, Crysvita revenue in the Ultragenyx territories is estimated to be between
The company is not providing total 2020 revenue guidance at this time. The preliminary revenue results are based on management's initial analysis of operations for the quarter and year ended
2019 Ending Cash Position (unaudited) and 2020 Expected Net Cash Burn Rate
Cash, cash equivalents, and available-for-sale investments were greater than
Recent Updates and Upcoming Milestones
Crysvita for X-linked Hypophosphatemia: Non-dilutive
- In December, Ultragenyx sold to
Royalty Pharmafor $320 millionits royalty interest in Crysvita in the European territory, where it is being commercialized by Kyowa Kirin Co., Ltd.
Crysvita for Tumor-Induced Osteomalacia (TIO): Supplemental Biologics License Application (sBLA) submitted
- Ultragenyx submitted the sBLA to the U.S.
FDAon December 18, 2019and expects to hear back from FDAon submission acceptance and review designation in February 2020.
UX007 for Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD): NDA under review by U.S.
- The U.S.
FDAaccepted for review the New Drug Application (NDA) and has set a Prescription Drug User Fee Act (PDUFA) 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 (OTC) 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 (n=3) 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 (GSDIa): 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 (IND) 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.
Ultragenyx to Present at 38ᵗʰ Annual
- Dr. Kakkis will present on
Tuesday, January 14, 2020at 12:00 p.m. PTin San Francisco. The live and archived webcast of the presentation will be accessible from the company’s website at http://ir.ultragenyx.com/events.cfm
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com.
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, Ultragenyx’s reliance on its third party partner,
Investors & Media
Source: Ultragenyx Pharmaceutical Inc.