NOVATO, Calif., May 07, 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 ended March 31, 2018.
“With the recent approvals and launches of Crysvita in the United States and Europe, we have transformed into a commercial stage company with two medicines now available for patients,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We continue to advance our clinical and preclinical programs and expect significant progress across our two clinical-stage gene therapy programs as well as our small molecule and biologics programs this year.”
First Quarter 2018 Financial Results
For the first quarter of 2018, Ultragenyx reported net income of $30.3 million, or $0.63 per basic share and $0.62 per diluted share, compared with a net loss for the first quarter of 2017 of $68.3 million, or $1.63 per share, basic and diluted. The income for the first quarter of 2018 includes the $130 million gain from the sale of the priority review voucher (PRV). The net income for the first quarter of 2018 reflected cash used in operations of $89.5 million compared to $61.2 million reflected in the net loss for the same period in 2017.
For the first quarter of 2018, Ultragenyx reported $10.7 million in total revenue, which includes $1.3 million in product revenue from Mepsevii™ (vestronidase alfa) and UX007, and $9.4 million in collaboration and license revenue, primarily from our research agreement with Bayer. Total operating expenses for the first quarter of 2018 were $107.2 million compared with $70.0 million for the same period in 2017, including non-cash stock-based compensation of $18.8 million and $14.5 million in the first quarter of 2018 and 2017, 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 $571.3 million as of March 31, 2018.
Recent Highlights
Crysvita® (burosumab) in X-Linked Hypophosphatemia (XLH)
DTX301 Gene Therapy in ornithine transcarbamylase (OTC) Deficiency
DTX401 Gene Therapy in glycogen storage disease type Ia (GSDIa)
Corporate
Upcoming Key Milestones
Crysvita (burosumab) in XLH
Crysvita (burosumab) in tumor-induced osteomalacia (TIO)
Mepsevii (vestronidase alfa) in mucopolysaccharidosis VII (MPS VII)
UX007 in long-chain fatty acid oxidation disorders (FAOD) and glucose transporter type-1 deficiency syndrome (Glut1 DS)
Conference Call & Webcast Information
Ultragenyx will host a conference call today, Monday, May 7, 2018 at 5pm ET to discuss first quarter 2018 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 3748439. 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.
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 by the U.S. FDA for the treatment of children and adults with MPS VII.
Ultragenyx is conducting Phase 2 and Phase 3 studies of burosumab in patients with XLH and TIO, both rare diseases that impair bone mineralization; a Phase 2 clinical study of UX007 in patients severely affected by 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 Glut1 DS, a brain energy deficiency, who are experiencing movement disorders; a Phase 1/2 study of DTX301 gene therapy in OTC deficiency, a urea cycle disorder that causes ammonia to accumulate in the blood; and a Phase 1/2 study of DTX401 gene therapy in GSDIa, a disease that arises from a defect in G6Pase, an essential enzyme in glycogen and glucose metabolism.
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 Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 21, 2018, 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 March 31, | ||||||||
2018 | 2017 | |||||||
Statement of Operations Data: | ||||||||
Revenues: | ||||||||
Collaboration and license | $ | 9,362 | $ | — | ||||
Product sales | 1,315 | — | ||||||
Total revenues | 10,677 | — | ||||||
Operating expenses: | ||||||||
Cost of goods sold | 225 | — | ||||||
Research and development | 75,504 | 51,269 | ||||||
Selling, general and administrative | 31,435 | 18,685 | ||||||
Total operating expenses | 107,164 | 69,954 | ||||||
Loss from operations | (96,487 | ) | (69,954 | ) | ||||
Gain from sale of priority review voucher | 130,000 | — | ||||||
Other income (expense), net | (3,221 | ) | 1,664 | |||||
Income (loss) before income taxes | 30,292 | (68,290 | ) | |||||
Income tax provision | (39 | ) | — | |||||
Net income (loss) | $ | 30,253 | $ | (68,290 | ) | |||
Net income (loss) per share: | ||||||||
Basic | $ | 0.63 | $ | (1.63 | ) | |||
Diluted | $ | 0.62 | $ | (1.63 | ) | |||
Shares used in computing net income (loss) per share: | ||||||||
Basic | 48,190,511 | 41,841,612 | ||||||
Diluted | 49,077,742 | 41,841,612 | ||||||
Ultragenyx Pharmaceutical Inc. | ||||||
Selected Balance Sheets Financial Data | ||||||
(in thousands) | ||||||
(unaudited) | ||||||
March 31, | December 31, | |||||
2018 | 2017 | |||||
Balance Sheet Data: | ||||||
Cash, cash equivalents and investments | $ | 571,253 | $ | 244,468 | ||
Working capital | 523,997 | 198,569 | ||||
Total assets | 817,350 | 490,753 | ||||
Total stockholders' equity | 723,657 | 383,454 | ||||
Contact Ultragenyx Pharmaceutical Inc.
Investors & Media
Danielle Keatley
415-475-6876