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Contact Ultragenyx Pharmaceutical Inc.
Ultragenyx Reports Fourth
Quarter 2015 and Full-Year 2015 Financial Results and Corporate Update
NOVATO, CA February 25, 2016 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 fourth quarter and full-year ended December 31, 2015.
2015 was a transformative year for Ultragenyx in which we advanced our entire product portfolio providing new possibilities for the treatment of
rare disease patients, said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. In 2016, we expect to have many clinical, regulatory and business development updates as we potentially begin transitioning
from a clinical development stage to a commercial company.
Fourth Quarter and Full-Year 2015 Financial Results
For the fourth quarter of 2015, Ultragenyx reported a net loss attributable to common stockholders of $55.2 million, or $1.42 per share, basic and diluted,
compared with a net loss attributable to common stockholders for the fourth quarter of 2014 of $16.7 million, or $0.52 per share, basic and diluted. This reflected cash used in operations of $40.4 million in the fourth quarter of 2015 compared to
$12.5 million in the fourth quarter of 2014. For the year ended December 31, 2015, net loss attributable to common stockholders was $145.6 million, or $3.96 per share, basic and diluted, compared with a net loss attributable to common stockholders
for the same period in 2014 of $64.6 million, or $2.25 per share, basic and diluted. This reflected cash used in operations of $106.0 million in the year ended December 31, 2015 compared to $44.6 million in the same period 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 fourth quarter of 2015 were $56.2 million compared with $16.9 million for the same period in 2014. This included a one-time
upfront payment to Arcturus of $10 million in the fourth quarter of 2015. Non-cash stock-based compensation accounted for $9.5 million and $2.0 million of total operating expenses in the fourth quarter of 2015 and 2014, respectively. Total
operating expenses for the year ended December 31, 2015 were $147.7 million compared with $56.8 million for the same period in 2014. Non-cash stock-based compensation accounted for $24.9 million and $5.4 million of total operating expenses in 2015
and 2014, respectively. The increase in total operating expenses is due to
the increase in development, commercial, and general and administrative costs as the company grows and
advances its pipeline, including related increases in stock compensation expenses.
Cash, cash equivalents, and investments were $536.3 million as of
Recent Highlights & Upcoming Milestones
KRN23 anti-FGF23 Monoclonal Antibody in X-Linked Hypophosphatemia (XLH) and Tumor-Induced Osteomalacia (TIO)
rhGUS in Mucopolysaccharidosis 7 (MPS 7)
UX007 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
will host a conference call today, Thursday, February 25, 2016 at 5pm ET to discuss fourth quarter and full-year 2015 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 45371417. The replay of the call will be available for one
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 its 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, such as
the regulatory approval process (including with respect to the MAA we filed seeking conditional approval from EMA with respect to Ace-ER), 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 10, 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)
| (unaudited) | ||||||||||||||||
| Three Months Ended December 31, | Year Ended December 31, | |||||||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||||||
| Statements of Operations Data: | ||||||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 44,565 | $ | 13,521 | $ | 114,737 | $ | 45,967 | ||||||||
| General and administrative | 11,593 | 3,422 | 33,001 | 10,811 | ||||||||||||
| Total operating expenses | 56,158 | 16,943 | 147,738 | 56,778 | ||||||||||||
| Loss from operations | (56,158 | ) | (16,943 | ) | (147,738 | ) | (56,778 | ) | ||||||||
| Other income (expense), net | 938 | 205 | 2,120 | (3,024 | ) | |||||||||||
| Net loss | $ | (55,220 | ) | $ | (16,738 | ) | $ | (145,618 | ) | $ | (59,802 | ) | ||||
| Net loss attributable to common stockholders | $ | (55,220 | ) | $ | (16,738 | ) | $ | (145,618 | ) | $ | (64,610 | ) | ||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (1.42 | ) | $ | (0.52 | ) | $ | (3.96 | ) | $ | (2.25 | ) | ||||
| Shares used to compute net loss per share attributable to common stockholders, basic and diluted | 38,847,922 | 31,897,138 | 36,782,603 | 28,755,758 |
Ultragenyx Pharmaceutical Inc.
Selected Balance Sheets Financial Data
| December 31, 2015 | December 31, 2014 | |||||||
| Balance Sheet Data: | ||||||||
| Cash, cash equivalents and investments | $ | 536,256 | $ | 187,487 | ||||
| Working capital | 422,289 | 180,899 | ||||||
| Total assets | 559,569 | 197,967 | ||||||
| Total stockholders equity | 531,090 | 184,945 |