Full Press Release Details
Contact Ultragenyx Pharmaceutical Inc.
Ultragenyx Reports First Quarter
2015 Financial Results and Corporate Update
NOVATO, CA May 11, 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 first quarter ended March 31, 2015.
In the first quarter we continued to execute the development plans for our broad pipeline of four clinical-stage products for six rare diseases,
commented Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. We have also made progress in our strategy of adding incremental value within our current programs with the release of positive data from an
investigator-sponsored trial in the movement disorder phenotype of Glut1 deficiency, which will lead to an expansion of that clinical program.
First Quarter 2015 Financial Results
quarter of 2015, Ultragenyx reported a net loss attributable to common stockholders of $21.4 million, or $0.63 per share, basic and diluted, compared with a net loss attributable to common stockholders for the first quarter of 2014 of $18.4 million,
or $0.85 per share, basic and diluted. First quarter of 2014 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 first quarter of 2015 were $21.5 million compared with $10.3 million for the
first quarter of 2014. 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 $342.6 million as of March 31, 2015. Based on
current operating levels, the company expects that existing cash, cash equivalents, and short-term investments will be sufficient to fund operations into 2018.
Recent Highlights & Upcoming Milestones
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 Glut1 Deficiency Syndrome (Glut1 DS) and Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Sialic Acid Extended-Release (SA-ER) in Hereditary Inclusion Body Myopathy (HIBM)
Conference Call & Webcast Information
Ultragenyx will host a conference call today, Monday, May 11, 2015 at 5pm ET, to discuss the first quarter 2015 financial results and 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 3243-3559. 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 sufficiency of existing cash, cash equivalents and short-term investments to fund operations for
projected periods of time, 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 Annual Report on Form 10-K filed with the
Securities and Exchange Commission on March 27, 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 March 31, | ||||||||
| 2015 | 2014 | |||||||
| Statements of Operations Data: | ||||||||
| Operating expenses: | ||||||||
| Research and development | $ | 17,364 | $ | 8,353 | ||||
| General and administrative | 4,138 | 1,986 | ||||||
| Total operating expenses | 21,502 | 10,339 | ||||||
| Loss from operations | (21,502 | ) | (10,339 | ) | ||||
| Other income (expense) | 123 | (3,291 | ) | |||||
| Net loss | $ | (21,379 | ) | $ | (13,630 | ) | ||
| Net loss attributable to common stockholders | $ | (21,379 | ) | $ | (18,438 | ) | ||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (0.63 | ) | $ | (0.85 | ) | ||
| Shares used to compute net loss per share attributable to common stockholders, basic and diluted | 34,008,830 | 21,582,435 | ||||||
| Ultragenyx Pharmaceutical Inc. Selected Balance Sheets Financial Data (in thousands) (unaudited) | ||||||||
| March 31, | December 31, | |||||||
| 2015 | 2014 | |||||||
| Balance Sheet Data: | ||||||||
| Cash, cash equivalents and short-term investments | $ | 342,566 | $ | 187,487 | ||||
| Working capital | 336,271 | 180,899 | ||||||
| Total assets | 355,840 | 197,967 | ||||||
| Total stockholders equity | 341,119 | 184,945 |