NOVATO, Calif., Nov. 05, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results and corporate update for the quarter ended September 30, 2018.
“We’re encouraged by the growing demand for Crysvita from both children and adults with XLH in the United States, and we are making significant progress reaching doctors and helping them get patients on commercially available therapy in this early launch phase,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “Next year, we expect to file for potential approval of our third therapy, UX007 in fatty acid oxidation disorders, and we continue to advance our two gene therapy clinical programs with additional data expected around the end of this year and in 2019.”
For the third quarter of 2018, Ultragenyx reported a net loss of $87.3 million, or $1.74 per share, basic and diluted, compared with a net loss for the third quarter of 2017 of $79.2 million, or $1.87 per share, basic and diluted. For the nine months ended September 30, 2018, net loss was $109.8 million, or $2.22 per share, basic and diluted, compared with a net loss for the same period in 2017 of $220.4 million, or $5.22 per share, basic and diluted. The loss from the first nine months was reduced by the sale of the Mepsevii™ (vestronidase alfa) priority review voucher (PRV) in January 2018 for net proceeds of $130.0 million and a $40.3 million gain from Ultragenyx’s portion of the sales of the PRV received with the Crysvita® (burosumab) approval. The net loss for the first nine months of 2018 reflected cash used in operations of $234.7 million compared to $172.0 million for the same period in 2017.
For the third quarter of 2018, Ultragenyx reported $11.8 million in total revenue. For Crysvita, Ultragenyx recognized $5.4 million in profit sharing and royalty revenue from its collaboration and license agreement with Kyowa Hakko Kirin. This includes $4.4 million in collaboration revenue in the U.S. profit share territory and $1.0 million in royalty revenue in the European territory. Net product sales for Crysvita in other regions were nominal. Mepsevii product revenue for the third quarter of 2018 was $2.1 million, and UX007 named patient revenue was $0.4 million. Ultragenyx recognized $3.6 million in revenue from its research agreement with Bayer.
Total operating expenses for the third quarter of 2018 were $101.4 million compared with $83.9 million for the same period in 2017, including non-cash stock-based compensation of $20.7 million and $17.2 million in the third quarter of 2018 and 2017, respectively. Total operating expenses for the nine months ended September 30, 2018, were $316.3 million compared with $232.3 million for the same period in 2017, including non-cash stock-based compensation of $59.0 million and $48.5 million in the first nine months of 2018 and 2017, respectively. The increase in total operating expenses is due to the increase in commercial, development, and general and administrative costs as the company commercializes, grows and advances its pipeline.
Cash, Cash Equivalents and Investments
Cash, cash equivalents and investments were $503.1 million as of September 30, 2018.
Crysvita in X-Linked Hypophosphatemia (XLH)
Crysvita in Tumor-Induced Osteomalacia (TIO)
Mepsevii in Mucopolysaccharidosis VII (MPS VII)
UX007 in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) and Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
DTX401 Gene Therapy in Glycogen Storage Disease Type Ia (GSDIa)
CDKL5 Deficiency Disorder (CDD)
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Monday, November 5, 2018, at 2 p.m. PT/ 5 p.m. ET to discuss third quarter 2018 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at http://ir.ultragenyx.com/events.cfm. To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 1290627. The replay of the call will be available for one year.
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 no approved therapies.
Crysvita® (burosumab) is approved by the U.S. FDA for the treatment of XLH in adult and pediatric patients one year of age and older, and has received conditional marketing authorization in Europe for the treatment of XLH with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons. Mepsevii™ (vestronidase alfa) is approved in the United States for the treatment of children and adults with MPS VII. In Europe, Mepsevii is approved under exceptional circumstances for the treatment of non-neurological manifestations of MPS VII.
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 severely affected by 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: DTX 301 is an AAV8 gene therapy product candidate in development for the treatment of patients with OTC deficiency, the most common urea cycle disorder; and DTX 401 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 relating to Ultragenyx's plans for its clinical programs and future regulatory filings and interactions, 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 Securities and Exchange Commission on August 3, 2018, and its subsequent periodic reports filed with the Securities and Exchange Commission.
Contact Ultragenyx Pharmaceutical Inc.
Investors & Media
|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||$||9,015||$||—||$||28,896||$||—|
|Cost of sales||273||—||639||—|
|Research and development||70,041||60,412||222,380||170,117|
|Selling, general and administrative||31,095||23,499||93,248||62,189|
|Total operating expenses||101,409||83,911||316,267||232,306|
|Loss from operations||(89,646||)||(83,713||)||(281,033||)||(232,108||)|
|Gain from sale of priority review vouchers||—||—||170,322||—|
|Other income (expense), net||2,583||4,490||1,314||11,718|
|Loss before income taxes||(87,063||)||(79,223||)||(109,397||)||(220,390||)|
|Provision for income taxes||(247||)||(4||)||(388||)||(18||)|
|Net loss per share, basic and diluted||$||(1.74||)||$||(1.87||)||$||(2.22||)||$||(5.22||)|
|Shares used in computing net loss per share, basic and diluted||50,319,772||42,471,606||49,447,889||42,222,413|
|Ultragenyx Pharmaceutical Inc.|
|Selected Balance Sheet Financial Data|
|September 30,||December 31,|
|Balance Sheet Data:|
|Cash, cash equivalents and investments||$||503,098||$||244,468|
|Total stockholders' equity||662,596||383,454|