NOVATO, Calif., Feb. 20, 2018 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, today reported its financial results and corporate update for the quarter and full year ended December 31, 2017.
“2017 was a transformative year for Ultragenyx with our first product approval and launch of Mepsevii™, significant clinical and regulatory progress, and the addition of gene therapy development and manufacturing technology through the acquisition of Dimension Therapeutics,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “The momentum will continue in 2018 with the potential for approval and launch of burosumab in the U.S. and Europe, and significant progress expected with our clinical, gene therapy and translational research programs.”
Fourth Quarter and Full Year 2017 Financial Results
For the fourth quarter of 2017, Ultragenyx reported a net loss of $81.7 million, or $1.89 per share, basic and diluted, compared with a net loss for the fourth quarter of 2016 of $71.3 million, or $1.75 per share, basic and diluted. For the year ended December 31, 2017, net loss was $302.1 million, or $7.12 per share, basic and diluted, compared with a net loss for the same period in 2016 of $245.9 million, or $6.21 per share, basic and diluted. The net loss for the fourth quarter and for the full year of 2017 includes a non-recurring tax benefit from the Dimension acquisition of $16.2 million as a result of the change in the U.S. corporate tax rate from 34% to 21% on the deferred tax liability. The net loss for the full year 2017 reflected cash used in operations of $253.8 million for the year ended December 31, 2017 compared to $161.0 million for the same period in 2016.
The U.S. Food and Drug Administration (FDA) approved Mepsevii for the treatment of children and adults with Mucopolysaccharidosis VII (MPS VII) on November 15, 2017. For the fourth quarter and full year of 2017, Ultragenyx reported $0.3 million and $0.5 million respectively in Mepsevii revenues. Total operating expenses for the fourth quarter of 2017 were $99.2 million compared with $70.6 million for the same period in 2016, including non-cash stock-based compensation of $19.5 million and $13.5 million in the fourth quarter of 2017 and 2016, respectively. Total operating expenses for the year ended December 31, 2017 were $331.6 million compared with $248.1 million for the same period in 2016, including non-cash stock-based compensation of $68.0 million and $48.3 million in 2017 and 2016, respectively. The increase in total operating expenses is due to the increase in development, commercial, and general and administrative costs as the company commercializes, grows and advances its pipeline.
Cash, cash equivalents, and investments were $244.5 million as of December 31, 2017. In January 2018, Ultragenyx completed the sale of its Rare Pediatric Disease Priority Review Voucher for $130.0 million and also raised approximately $271.0 million in net proceeds through an equity offering.
Recent Highlights
Mepsevii (vestronidase alfa) for MPS VII
Burosumab (KRN23) anti-FGF23 Monoclonal Antibody in X-Linked Hypophosphatemia (XLH)
DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
Corporate
Upcoming Key Milestones
Burosumab in XLH
Burosumab in tumor-induced osteomalacia (TIO)
UX007 in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency
DTX401 Gene Therapy in Glycogen storage disease type Ia (GSDIa)
DTX201 Gene Therapy in Hemophilia A
Conference Call & Webcast Information
Ultragenyx will host a conference call today, Tuesday, February 20, 2018 at 5pm ET to discuss fourth quarter and full year 2017 financial results and to 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 1987066. The replay of the call will be available for one year.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing to patients novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.
Mepsevii™ (vestronidase alfa) is approved by the U.S. FDA for the treatment of children and adults with mucopolysaccharidosis VII (MPS VII). Ultragenyx is conducting Phase 2 and Phase 3 studies of burosumab, in pediatric and adult patients with XLH and tumor induced osteomalacia (TIO), both rare diseases that impair bone mineralization; a Phase 2 clinical study of UX007 in patients severely affected by long-chain fatty acid oxidation disorders (LC-FAOD), a genetic disorder in which the body is unable to convert long chain fatty acids into energy; a Phase 3 study for UX007 in patients with glucose transporter type-1 deficiency syndrome (Glut1 DS), a brain energy deficiency, who are experiencing movement disorders; and a Phase 1/2 study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency.
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.
Forward-Looking Statements
Except for the historical information contained herein, the matters set forth in this press release, including statements regarding Ultragenyx's expectations regarding ongoing or additional studies for its product candidates and timing regarding these studies, the design of clinical studies, the demonstrated impact of clinical data and other information to support approval of product candidates, potential indications for its product candidates, discussions with regulatory authorities, the potential issuance of a priority review voucher, sufficiency for, and timing of, regulatory submissions and approvals, and the timing and locations of commercialization efforts 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 our 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 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 the company in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 3, 2017, and its subsequent periodic reports filed with the Securities and Exchange Commission.
Ultragenyx Pharmaceutical Inc. | |||||||||||||||
Selected Statement of Operations Financial Data | |||||||||||||||
(in thousands, except share and per share amounts) | |||||||||||||||
(unaudited) | |||||||||||||||
Three Months Ended December 31, | Year Ended December 31, | ||||||||||||||
2017 | 2016 | 2017 | 2016 | ||||||||||||
Statement of Operations Data: | |||||||||||||||
Revenues: | |||||||||||||||
Collaboraion and license | $ | 2,136 | $ | - | $ | 2,136 | $ | - | |||||||
Product sales | 278 | 5 | 476 | 133 | |||||||||||
Total revenues | 2,414 | 5 | 2,612 | 133 | |||||||||||
Operating expenses: | |||||||||||||||
Cost of sales | 1 | - | 1 | - | |||||||||||
Research and development | 61,527 | 50,746 | 231,644 | 183,204 | |||||||||||
Selling, general and administrative | 37,720 | 19,808 | 99,909 | 64,936 | |||||||||||
Total operating expenses | 99,248 | 70,554 | 331,554 | 248,140 | |||||||||||
Loss from operations | (96,834 | ) | (70,549 | ) | (328,942 | ) | (248,007 | ) | |||||||
Other income (expense) | (1,114 | ) | (703 | ) | 10,604 | 2,168 | |||||||||
Loss before income taxes | (97,948 | ) | (71,252 | ) | (318,338 | ) | (245,839 | ) | |||||||
Benefit from (provision for) income taxes | 16,217 | (35 | ) | 16,199 | (35 | ) | |||||||||
Net loss | $ | (81,731 | ) | $ | (71,287 | ) | $ | (302,139 | ) | $ | (245,874 | ) | |||
Net loss per share, basic and diluted | $ | (1.89 | ) | $ | (1.75 | ) | $ | (7.12 | ) | $ | (6.21 | ) | |||
Shares used in computing net loss per share, | |||||||||||||||
basic and diluted | 43,137,679 | 40,783,829 | 42,453,135 | 39,586,908 | |||||||||||
Ultragenyx Pharmaceutical Inc. | |||||
Selected Balance Sheet Financial Data | |||||
(in thousands) | |||||
(unaudited) | |||||
December 31, | December 31, | ||||
2017 | 2016 | ||||
Balance Sheet Data: | |||||
Cash, cash equivalents and investments | $ | 244,468 | $ | 498,111 | |
Working capital | 198,569 | 341,436 | |||
Total assets | 490,753 | 540,626 | |||
Total stockholders' equity | 383,454 | 473,974 | |||
Contact Ultragenyx Pharmaceutical Inc.
Investors & Media
Danielle Keatley
415-475-6876