NOVATO, Calif., March 24, 2014 (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 business highlights for the fourth quarter and year ended December 31, 2013.
"Ultragenyx completed a landmark year in 2013, during which we achieved multiple milestones in our vision to build a next generation rare disease company," said Emil D. Kakkis, Ph.D., M.D., Chief Executive Officer and President of Ultragenyx. "We have expanded our pipeline to five clinical programs in Phase 1/2 or Phase 2 studies, and with the completion of our initial public offering in early 2014, we believe we are well-positioned to advance multiple programs in parallel to address the significant unmet medical needs of these patients."
Fourth Quarter and Full Year 2013 Financial Results
For the fourth quarter of 2013, Ultragenyx reported a net loss attributable to common stockholders of $18.7 million, or $4.98 per share, basic and diluted, compared with a net loss attributable to common stockholders for the fourth quarter of 2012 of $6.8 million, or $2.83 per share, basic and diluted.
For the year ended December 31, 2013, Ultragenyx reported a net loss attributable to common stockholders of $50.3 million, or $14.87 per share, basic and diluted, compared with a net loss attributable to common stockholders for the year ended December 31, 2012 of $19.6 million, or $14.20 per share, basic and diluted.
Total operating expenses for 2013 were $32.3 million compared with $16.0 million for 2012. Total operating expenses for the fourth quarter of 2013 were $9.5 million compared with $4.7 million for the same period in 2012. The increase in total operating expenses is due to the addition of triheptanoin and KRN23 to Ultragenyx's clinical development pipeline, advancements in the development of sialic acid extended release (SA-ER) and recombinant human beta-glucuronidase (rhGUS), and increased headcount to support the company's growth.
Cash, cash equivalents, and short-term investments were $53.4 million as of December 31, 2013. Based on current operating levels, the company expects that existing cash, cash equivalents and short-term investments, including approximately $121.7 million in net proceeds received from the initial public offering in February 2014, will be sufficient to fund operations into 2016.
KRN23 anti-FGF23 Monoclonal Antibody in X-linked Hypophosphatemia (XLH)
rhGUS in Mucopolysaccharidosis 7 (MPS 7)
Triheptanoin in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Triheptanoin in Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
SA-ER in Hereditary Inclusion Body Myopathy (HIBM)
Anticipated 2014 Program Milestones
KRN23 in XLH
rhGUS in MPS 7
Triheptanoin in LC-FAOD
Triheptanoin in Glut1 DS
SA-ER in HIBM
Ultragenyx is a development-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with an initial focus on serious, debilitating metabolic 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.
1Carpenter TO, et al. Randomized trial of the anti-FGF23 antibody KRN23 in X-linked hypophosphatemia. J Clin Invest. 2014. doi:10.1172/JCI72829.
Except for the historical information contained herein, the matters set forth in this press release, including statements regarding Ultragenyx's plans, potential opportunities, expectations, projections, goals, objectives, milestones, strategies, product pipeline, clinical studies, product development, release of data and the potential benefits of its products under development 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 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 prospectus filed with the Securities and Exchange Commission on January 31, 2014, and its future periodic reports to be 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
|Statements of Operations Data:|
|Research and development||$ 3,775||$ 8,204||$ 12,641||$ 27,829|
|General and administrative||903||1,321||3,344||4,451|
|Total operating expenses||4,678||9,525||15,985||32,280|
|Loss from operations||(4,678)||(9,525)||(15,985)||(32,280)|
|Other expense, net||(253)||(1,792)||(349)||(2,790)|
|Net loss||$ (4,931)||$ (11,317)||$ (16,334)||$ (35,070)|
|Net loss attributable to common stockholders||$ (6,813)||$ (18,665)||$ (19,561)||$ (50,289)|
|Net loss per share attributable to common stockholders, basic and diluted||$ (2.83)||$ (4.98)||$ (14.20)||$ (14.87)|
|Shares used to compute net loss per share attributable to common stockholders, basic and diluted||2,404,852||3,744,525||1,377,207||3,382,489|
|Ultragenyx Pharmaceutical Inc.|
|Selected Balance Sheets Financial Data|
|As of December 31,|
|Balance Sheet Data:|
|Cash, cash equivalents and short-term investments||$ 86,190||$ 53,377|
|Convertible preferred stock warrant liability||518||3,419|
|Convertible preferred stock||111,387||124,930|
|Deficit accumulated during the development stage||(27,058)||(74,836)|
|Total stockholders' deficit||(27,047)||(74,821)|
Ultragenyx Pharmaceutical Inc. 844-758-7273 For Media, Bee Nguyen For Investors, Robert Anstey