Ultragenyx Reports Fourth Quarter and Full Year 2019 Financial Results and Corporate Update
2019 total revenue is
2020 Crysvita revenue in Ultragenyx territories guidance of
“In 2019 we created a substantial foundation based on the strong launch of Crysvita and the validation of our gene therapy platform across multiple clinical programs,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “In 2020, we anticipate building on this momentum as we look to the potential approvals of UX007 for LC-FAOD and Crysvita for TIO. We also expect to advance our gene therapy programs into pivotal studies and bring two new programs into the clinic while continuing to exercise financial discipline.”
Fourth Quarter and Full Year 2019 Financial Results
For the fourth quarter of 2019, Ultragenyx reported $35.6 million in total revenue. Ultragenyx recognized $29.9 million in total Crysvita revenue. This includes $26.1 million in collaboration revenue in the North American1 profit share territory and $2.2 million in royalty revenue in the European territory from the collaboration and license agreement with Kyowa Kirin Co., Ltd (KKC). Net product sales for Crysvita in other regions were $1.6 million. Mepsevii® (vestronidase alfa) product revenue for the fourth quarter of 2019 was $4.4 million, and UX007 named patient revenue was $1.2 million. Ultragenyx recognized $0.1 million in revenue from its research agreement with Bayer.
Net revenue for the year ended December 31, 2019 totaled $103.7 million. In 2019, Ultragenyx recognized $87.3 million in total Crysvita revenue. This includes $74.9 million in collaboration revenue in the North American1 profit share territory and $8.1 million in royalty revenue in the European territory from the collaboration and license agreement with KKC. Net product sales for Crysvita in other regions totaled $4.3 million. Mepsevii product revenue for the year ended December 31, 2019 was $12.6 million, and UX007 named patient revenue was $3.3 million. Ultragenyx recognized $0.5 million in revenue from its research agreement with Bayer in the year ended December 31, 2019.
Total operating expenses for the fourth quarter of 2019 were
For the fourth quarter of 2019, Ultragenyx reported a net loss of
Cash, Cash Equivalents and Investments
Cash, cash equivalents, and investments were
Crysvita Guidance in Ultragenyx Territories
In 2020, the company continues to expect Crysvita revenue in the Ultragenyx territories to be between
2020 Expected Net Cash Burn Rate
The company also expects a more than 20 percent reduction in net cash burn (net cash used in operations plus capital expenditures) in 2020 compared to 2019 due to a combination of financial discipline in spending with flattening operating expense growth, combined with the significantly larger growth in revenue.
Recent Updates and Upcoming Milestones
Crysvita for X-linked Hypophosphatemia: Non-dilutive
December 2019, 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. The review process is on track for a decision by the PDUFA date.
DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Positive data from higher dose 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. 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 (AS): Partnered program with GeneTx; IND active and enrollment of first patient expected in first half 2020
- An investigational new drug (IND) application is now active and GeneTx has received
Institutional Review Board(IRB) approval from the first study site. Enrollment in the Phase 1/2 study of GTX-102 is expected to begin in the first half of 2020.
February 2020, Ultragenyx paid $25 millionafter the acceptance of the IND to maintain its option to acquire GeneTx until the earlier of 30 months from the first dosing of a patient in the Phase 1/2 study (subject to extensions) or 90 days after results are available from that study.
UX701 for Wilson Disease: IND expected by the end of the year
- An IND application is expected by the end 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.
DTX201 / BAY 2599023 for Hemophilia A: Partnered with Bayer; Cohort 2 data presented at
- Three cohorts with two patients each, six patients in total, have been dosed with AAVhu37 (DTX201 / BAY 2599023), using material from Ultragenyx’s proprietary HeLa manufacturing platform. Longer term dose cohort 1 and new dose cohort 2 data presented at EAHAD showed three of four patients have achieved clinically meaningful FVIII levels. One patient in Cohort 1 has achieved clinically meaningful FVIII levels and has experienced four bleeds since receiving treatment down from 99 bleeds the prior year. Both patients in dose cohort 2 achieved clinically meaningful FVIII levels out to 24 and 30 weeks. Patient 4 has been off treatment and bleed free for seven months as of the data cut-off date. The second patient in dose cohort 2 had mild ALT/AST elevations that was managed with a short tapering course of corticosteroids.
Conference Call and Webcast Information
Ultragenyx will host a conference call today,
1: Corrected to North American from U.S.
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,
|Ultragenyx Pharmaceutical Inc.|
|Selected Statement of Operations Financial Data|
|(in thousands, except share and per share amounts)|
|Three Months Ended December 31,||Year Ended December 31,|
|Statement of Operations Data:|
|Collaboration and license||$||28,423||$||12,797||$||83,493||$||41,693|
|Cost of sales||5,107||507||9,008||1,146|
|Research and development||83,061||71,618||357,355||293,998|
|Selling, general and administrative||41,877||34,476||161,524||127,724|
|Total operating expenses||130,045||106,601||527,887||422,868|
|Loss from operations||(94,452||)||(90,340||)||(424,173||)||(371,373||)|
|Gain from sale of priority review vouchers||—||—||—||170,322|
|Change in fair value of investment in Arcturus equity securities||1,419||—||13,413||—|
|Non-cash interest expense on liability related to the sale of future royalties||(1,135||)||—||(1,135||)||—|
|Other income (expense), net||2,924||2,640||12,451||3,954|
|Loss before income taxes||(91,244||)||(87,700||)||(399,444||)||(197,097||)|
|Provision for income taxes||(2,561||)||(126||)||(3,283||)||(514||)|
|Net loss per share, basic and diluted||$||(1.62||)||$||(1.73||)||$||(7.12||)||$||(3.97||)|
|Shares used in computing net loss per share, basic and diluted||57,808,025||50,694,007||56,576,885||49,775,223|
|Ultragenyx Pharmaceutical Inc.|
|Selected Balance Sheet Financial Data|
|December 31,||December 31,|
|Balance Sheet Data:|
|Cash, cash equivalents, and investments||$||760,404||$||459,706|
|Total stockholders' equity||653,764||608,908|
Investors & Media
Source: Ultragenyx Pharmaceutical Inc.