Full Press Release Details
Contact Ultragenyx Pharmaceutical Inc.
Ultragenyx Reports Third Quarter
2015 Financial Results and Corporate Update
NOVATO, CA November 9, 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 September 30, 2015.
We have continued to make progress across our product portfolio, including filing for conditional approval in Europe with Ace-ER, reporting interim data
for UX007 in long chain fatty acid oxidation disorders (LC-FAOD) and in five infants with cardiomyopathy due to LC-FAOD, announcing plans to initiate a Phase 3 study with UX007 in Glut1 DS patients with the movement disorder phenotype, and
initiating a multi-target collaboration for messenger RNA (mRNA) therapeutics, said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. We look forward to closing out the year with the 36-patient data from
the Phase 2 study of KRN23 in pediatric XLH patients and Ultragenyx s first R&D day in New York on December 3.
For the third quarter of 2015, Ultragenyx reported a net loss attributable to common stockholders of $39.2 million, or $1.03 per
share, basic and diluted, compared with a net loss attributable to common stockholders for the third quarter of 2014 of $15.8 million, or $0.50 per share, basic and diluted. For the nine months ended September 30, 2015, net loss attributable to
common stockholders was $90.4 million, or $2.51 per share, basic and diluted, compared with a net loss attributable to common stockholders for the same period in 2014 of $47.9 million, or $1.73 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 third quarter of 2015 were $39.9 million compared with $15.8 million for the same period in 2014. Non-cash stock-based
compensation accounted for $7.9 million and $1.7 million of total operating expenses in the third quarter of 2015 and 2014, respectively. Total operating expenses for the nine months ended September 30, 2015 were $91.6 million compared with
$39.8 million for the same period in 2014. Non-cash stock-based compensation accounted for $15.4 million and $3.4 million of total operating expenses in the first nine months of 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, as well as related increases in stock compensation expenses.
Cash, cash equivalents, and investments were $581.9 million as of September 30, 2015.
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)
in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) and Glut1 Deficiency Syndrome (Glut1 DS)
Four of the 29 patients discontinued prior to 24 weeks, including only one for diarrhea attributed to UX007. The three other patients withdrew
consent for reasons unrelated to UX007 treatment. All the remaining 25 patients opted to stay on drug after 24 weeks. One serious related adverse event for moderate gastroenteritis with vomiting was considered treatment-related. Overall,
18 patients (62%) had treatment-related adverse events, most of which were mild-to-moderate in nature. The most common treatment-related adverse events were diarrhea, abdominal/gastrointestinal pain, and vomiting.
Extended Release (Ace-ER) in GNE Myopathy
Conference Call & Webcast Information
Ultragenyx will host a conference call today, Monday, November 9, 2015 at 5pm ET to discuss third 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 71521135. 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 August 14, 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 September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2015 | 2014 | 2015 | 2014 | |||||||||||||
| Statements of Operations Data: | ||||||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 29,704 | $ | 12,854 | $ | 70,172 | $ | 32,446 | ||||||||
| General and administrative | 10,232 | 2,981 | 21,408 | 7,389 | ||||||||||||
| Total operating expenses | 39,936 | 15,835 | 91,580 | 39,835 | ||||||||||||
| Loss from operations | (39,936 | ) | (15,835 | ) | (91,580 | ) | (39,835 | ) | ||||||||
| Other income (expense), net | 704 | (14 | ) | 1,182 | (3,229 | ) | ||||||||||
| Net loss | $ | (39,232 | ) | $ | (15,849 | ) | $ | (90,398 | ) | $ | (43,064 | ) | ||||
| Net loss attributable to common stockholders | $ | (39,232 | ) | $ | (15,849 | ) | $ | (90,398 | ) | $ | (47,872 | ) | ||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | (1.03 | ) | $ | (0.50 | ) | $ | (2.51 | ) | $ | (1.73 | ) | ||||
| Shares used to compute net loss per share attributable to common stockholders, basic and diluted | 38,268,632 | 31,631,385 | 36,086,598 | 27,697,137 |
Ultragenyx Pharmaceutical Inc.
Selected Balance Sheets Financial Data
| September 30, | December 31, | |||||||
| 2015 | 2014 | |||||||
| Balance Sheet Data: | ||||||||
| Cash, cash equivalents and investments | $ | 581,875 | $ | 187,487 | ||||
| Working capital | 435,974 | 180,899 | ||||||
| Total assets | 602,130 | 197,967 | ||||||
| Total stockholders equity | 576,763 | 184,945 |