Ultragenyx Reports Second Quarter 2019 Financial Results and Corporate Update
Strong Crysvita® (burosumab) Launch Continues with Approximately 960 Patients on Reimbursed Commercial Therapy in
Ultragenyx Submits New Drug Application to
“The U.S. launch of Crysvita continues to be strong with the number of patients on reimbursed therapy increasing significantly this quarter and the number of unique prescribers continuing to grow steadily,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “Earlier this week we submitted the New Drug Application for UX007, which marks significant progress in bringing this important therapy to patients, and we are advancing our gene therapy platform with updates expected from the two clinical-stage programs in the third quarter.”
Second Quarter 2019 Financial Results
For the second quarter of 2019, Ultragenyx reported
Revenue for the six months ended
Total operating expenses for the second quarter of 2019 were
For the second 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 960 patients on reimbursed commercial therapy in
the United Statesat the end of the second quarter 2019.
UX007 NDA submitted to
- Ultragenyx submitted a New Drug Application (NDA) to the
FDAearlier this week. The FDApreviously granted Rare Pediatric Disease Designation and Fast Track designation, which enables eligibility for Priority Review, if relevant criteria are met. The company expects to hear back from FDAon submission acceptance and review designation within 60 days.
Clinical-stage Gene Therapy Programs Advance
- DTX301 Phase 1/2 study in ornithine transcarbamylase (OTC) deficiency: Previous data from Cohorts 1 and 2 demonstrated that the two responders maintained ureagenesis levels above normal for 78 and 52 weeks, respectively. There have been no infusion-related adverse events and no treatment-related serious adverse events reported.
- DTX401 Phase 1/2 Study in glycogen storage disease type Ia (GSDIa): Previous data from Cohort 1 demonstrated that all three patients showed improvements in glucose control reflected by prolonged time to hypoglycemia during a controlled fasting challenge. All patients have sustained their reductions in cornstarch compared to baseline, and continue to maintain normal glucose levels throughout the day and overnight. There have been no infusion-related adverse events and no treatment-related serious adverse events reported.
- Ultragenyx plans to provide updates from the third dose cohort of the DTX301 study in OTC and the second dose cohort of the DTX 401 study in GSDIa in the third quarter of 2019.
- Expanded collaboration with
Arcturus Therapeuticsto develop additional nucleic acid therapies. The research collaboration and license agreement with Arcturus Therapeuticsnow includes the discovery and development of mRNA, DNA, and siRNA therapeutics for up to 12 rare disease targets. Erik Harrisnamed Chief Commercial Officer. In June 2019, Ultragenyx promoted Erik Harristo Executive Vice President and Chief Commercial Officer. Mr. Harris joined Ultragenyx in 2017 as Senior Vice President, Head of North American Commercial Operations, leading the launches of Crysvita and Mepsevii. In his new role, Mr. Harris will be responsible for all commercial operations in North America, Europe, and Latin America.
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 June 30,||Six Months Ended June 30,|
|Statement of Operations Data:|
|Collaboration and license||$||19,247||$||10,519||$||33,485||$||19,881|
|Cost of sales||766||141||1,218||366|
|Research and development||96,045||76,835||174,150||152,339|
|Selling, general and administrative||39,812||30,718||78,641||62,153|
|Total operating expenses||136,623||107,694||254,009||214,858|
|Loss from operations||(112,474||)||(94,900||)||(211,688||)||(191,387||)|
|Gain from sale of priority review vouchers||—||40,322||—||170,322|
|Change in fair value of investment in Arcturus equity securities||9,828||—||9,828||—|
|Other income (expense), net||3,687||1,952||6,361||(1,269||)|
|Loss before income taxes||(98,959||)||(52,626||)||(195,499||)||(22,334||)|
|Provision for income taxes||(213||)||(102||)||(429||)||(141||)|
|Net loss per share, basic and diluted||$||(1.72||)||$||(1.06||)||$||(3.54||)||$||(0.46||)|
|Shares used in computing net loss per share, basic and diluted||57,519,308||49,819,528||55,376,336||49,046,838|
|Ultragenyx Pharmaceutical Inc.|
|Selected Balance Sheet Financial Data|
|June 30,||December 31,|
|Balance Sheet Data:|
|Cash, cash equivalents, and available-for-sale investments||$||618,286||$||459,706|
|Total stockholders' equity||819,787||608,908|
Investors & Media
Source: Ultragenyx Pharmaceutical Inc.