Full Press Release Details
Contact Ultragenyx Pharmaceutical Inc.
Ultragenyx Reports Second
Quarter 2015 Financial Results and Corporate Update
NOVATO, CA August 13, 2015 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 June 30, 2015.
The second quarter included important data releases on two programs, including data from an investigator study of triheptanoin in Glut1 DS patients with
movement disorder and KRN23 in pediatric XLH patients, commented Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. We expect to see continued clinical and regulatory progress this year as we report
additional data on triheptanoin and KRN23, advance our Phase 3 programs for rhGUS, Ace-ER, and KRN23, and file our first MAA for Ace-ER.
Quarter 2015 Financial Results
For the second quarter of 2015, Ultragenyx reported a net loss attributable to common stockholders of $29.8 million, or
$0.83 per share, basic and diluted, compared with a net loss attributable to common stockholders for the second quarter of 2014 of $13.6 million, or $0.45 per share, basic and diluted. For the six months ended June 30, 2015, net loss
attributable to common stockholders was $51.2 million, or $1.46 per share, basic and diluted, compared with a net loss attributable to common stockholders for the same period in 2014 of $32.0 million, or $1.25 per share, basic and diluted. Net loss
attributable to common stockholders differs from net loss due to dividends and other charges related to outstanding preferred stock, which was converted into common stock upon the company s initial public offering.
Total operating expenses for the second quarter of 2015 were $30.1 million compared with $13.7 million for the same period in 2014. Non-cash stock-based
compensation accounted for $5.1 million and $0.9 million of total operating expenses in the second quarter of 2015 and 2014, respectively. Total operating expenses for the six months ended June 30, 2015 were $51.6 million compared with $24.0
million for the same period in 2014. Non-cash stock-based compensation accounted for $7.5 million and $1.7 million of total operating expenses in the first half of 2015 and 2014, respectively. The increase in total operating expenses is due to the
increase in clinical trial, manufacturing, other development activities, and supportive general and administrative costs as the company s pipeline continues to advance to the late stages of development.
Cash, cash equivalents, and short-term investments were $326.0 million as of June 30, 2015. Subsequent
to the end of the quarter, the company completed a public offering of 2,530,000 shares of common stock at a price to the public of $120.00 per share, which includes the exercise in full by the underwriters of their option to purchase 330,000
additional shares of common stock. Net proceeds to Ultragenyx from the offering were approximately $286.6 million.
KRN23 anti-FGF23 Monoclonal Antibody in X-Linked Hypophosphatemia (XLH)
KRN23 anti-FGF23 Monoclonal Antibody in Tumor-Induced Osteomalacia (TIO)
rhGUS in Mucopolysaccharidosis 7 (MPS 7)
Triheptanoin in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) and Glut1 Deficiency Syndrome (Glut1 DS)
Aceneuramic Acid Extended Release (Ace-ER) in GNE Myopathy
Conference Call & Webcast Information
Ultragenyx will host a conference call today, Thursday, August 13, 2015 at 5pm ET to discuss second quarter 2015 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 94596921. The replay of the call will be available for one year.
Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market 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.
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 the timing of release of additional data for its product candidates, plans to initiate additional
studies for its product candidates and timing regarding these studies, plans regarding ongoing studies for existing programs and intent to file for conditional approval, 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, including 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 May 12, 2015, and its subsequent periodic reports filed
with the Securities and Exchange Commission.
Ultragenyx Pharmaceutical Inc.
Selected Statements of Operations Financial Data
(in thousands, except share and per share amounts)
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||||||
| Statements of Operations Data: | ||||||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 23,104 | $ | 11,239 | $ | 40,468 | $ | 19,592 | ||||||||
| General and administrative | 7,038 | 2,422 | 11,176 | 4,408 | ||||||||||||
| Total operating expenses | 30,142 | 13,661 | 51,644 | 24,000 | ||||||||||||
| Loss from operations | (30,142 | ) | (13,661 | ) | $ | (51,644 | ) | $ | (24,000 | ) | ||||||
| Other income (expense) | 355 | 76 | 478 | (3,215 | ) | |||||||||||
| Net loss | $ | (29,787 | ) | $ | (13,585 | ) | $ | (51,166 | ) | $ | (27,215 | ) | ||||
| Net loss attributable to common stockholders | $ | (29,787 | ) | $ | (13,585 | ) | $ | (51,166 | ) | $ | (32,023 | ) | ||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.83 | ) | $ | (0.45 | ) | $ | (1.46 | ) | $ | (1.25 | ) | ||||
| Shares used to compute net loss per share attributable to common stockholders, basic and diluted | 35,937,442 | 30,055,943 | 34,977,498 | 25,697,407 |
Ultragenyx Pharmaceutical Inc.
Selected Balance Sheets Financial Data
| June 30, | December 31, | |||||||
| 2015 | 2014 | |||||||
| Balance Sheet Data: | ||||||||
| Cash, cash equivalents and short-term investments | $ | 326,029 | $ | 187,487 | ||||
| Working capital | 313,488 | 180,899 | ||||||
| Total assets | 343,004 | 197,967 | ||||||
| Total stockholders equity | 319,278 | 184,945 |