Ultragenyx Reports Third Quarter 2019 Financial Results and Corporate Update
Strong Crysvita® (burosumab) Launch Continues with Approximately 1,130 Patients on Reimbursed Commercial Therapy in
UX007 (triheptanoin) NDA for Treatment of Long-chain Fatty Acid Oxidation Disorders Accepted for Review by
“The U.S. launch of Crysvita continues to be strong with the number of patients on reimbursed therapy continuing to increase, which will be further supported by the recently expanded label for both pediatric and adult patients,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “The New Drug Application for UX007 is now being reviewed by
Third Quarter 2019 Financial Results
For the third quarter of 2019, Ultragenyx reported
Revenue for the nine months ended
Total operating expenses for the third quarter of 2019 were
For the third quarter of 2019, Ultragenyx reported a net loss of
Cash, Cash Equivalents and Investments
Cash, cash equivalents and available-for-sale investments were
Recent Updates and Upcoming Milestones
Crysvita Commercial Progress in X-Linked Hypophosphatemia (XLH)
- Strong U.S. launch continues, with approximately 170 new patients on reimbursed commercial therapy in
the United Statesin the third quarter 2019.
- U.S. Crysvita label expanded. The U.S.
FDAapproved a label expansion for Crysvita, which now includes clinical data demonstrating superiority of treatment with Crysvita versus conventional therapy in pediatric patients, and improvement in stiffness and maintenance of efficacy in adult patients with longer-term treatment. The indication has also been expanded to include infants as young as six months of age.
Crysvita Supplemental Biologics License Application (sBLA) for Tumor-Induced Osteomalacia (TIO)
- Ultragenyx plans to submit a sBLA to the U.S.
FDAfor Crysvita for the treatment of TIO in the first half of 2020.
UX007 NDA for the Treatment of Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) Accepted for 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.
Clinical-stage Gene Therapy Programs Advance
- DTX301 Phase 1/2 study in ornithine transcarbamylase (OTC) deficiency: Data from Cohorts 1 and 2 demonstrated that the two responders maintained ureagenesis levels above normal and normal ammonia levels for 78 and 52 weeks, respectively. There have been no infusion-related adverse events and no treatment-related serious adverse events reported. Ultragenyx plans to provide an update from the ongoing third dose cohort around the end of the year. The company also plans to proceed to an additional cohort using prophylactic steroids.
- DTX401 Phase 1/2 Study in glycogen storage disease type Ia (GSDIa): Data from the first two dose cohorts demonstrated that all patients showed clinical responses, including improvements in glucose control shown by time to hypoglycemia and reductions in cornstarch requirements. In the second dose cohort, all three patients showed a more meaningful reduction in glycogen storage as measured by liver fat fraction and improvements in metabolism as measured by lactate levels. Based on these data, Ultragenyx is conducting a confirmatory expansion cohort of three patients at the second cohort dose of 6.0 × 10^12 GC/kg, with data from this cohort expected in the first half of 2020.
- Agreement with GeneTx Biotherapeutics to advance treatment for Angelman Syndrome. Ultragenyx and GeneTx are collaborating on the development of GeneTx’s GTX-102, an antisense oligonucleotide (ASO) for the treatment of Angelman syndrome, a debilitating and rare neurogenetic disorder. GTX-102 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation, and the Investigational New Drug (IND) timing is on track. Ultragenyx made an upfront payment of $20 million which included an exclusive option to acquire GeneTx. This option may be exercised any time prior to 30 days following
FDAacceptance of the IND for GTX-102. The option period may be extended by paying an option extension payment of $25 million.
Conference Call and Webcast Information
Ultragenyx will host a conference call today,
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.
Crysvita® (burosumab) is approved in
In addition to the approved treatments for XLH and MPS VII, Ultragenyx has four clinical development programs. Crysvita is being studied for the treatment of TIO, a rare disease that impairs bone mineralization. UX007 is being studied in patients with LC-FAOD, a genetic disorder in which the body is unable to convert long chain fatty acids into energy. The company has two gene therapy pipeline candidates: DTX301 is an AAV8 gene therapy product candidate in development for the treatment of patients with OTC deficiency, the most common urea cycle disorder; and DTX401 is an AAV8 gene therapy product candidate for the treatment of patients with GSDIa.
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 regarding 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, 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, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the
|Ultragenyx Pharmaceutical Inc.|
|Selected Statement of Operations Financial Data|
|(in thousands, except share and per share amounts)|
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Statement of Operations Data:|
|Collaboration and license||$||21,585||$||9,015||$||55,070||$||28,896|
|Cost of sales||2,683||273||3,901||639|
|Research and development||100,144||70,041||274,294||222,380|
|Selling, general and administrative||41,006||31,095||119,647||93,248|
|Total operating expenses||143,833||101,409||397,842||316,267|
|Loss from operations||(118,033||)||(89,646||)||(329,721||)||(281,033||)|
|Gain from sale of priority review vouchers||—||—||—||170,322|
|Change in fair value of investment in Arcturus equity
|Other income (expense), net||3,166||2,583||9,527||1,314|
|Loss before income taxes||(112,701||)||(87,063||)||(308,200||)||(109,397||)|
|Provision for income taxes||(293||)||(247||)||(722||)||(388||)|
|Net loss per share, basic and diluted||$||(1.96||)||$||(1.74||)||$||(5.50||)||$||(2.22||)|
|Shares used in computing net loss per share, basic and
|Ultragenyx Pharmaceutical Inc.|
|Selected Balance Sheet Financial Data|
|September 30,||December 31,|
|Balance Sheet Data:|
|Cash, cash equivalents, and available-for-sale investments||$||527,058||$||459,706|
|Total stockholders' equity||727,265||608,908|
Investors & Media
Source: Ultragenyx Pharmaceutical Inc.