Ultragenyx Pharmaceutical Inc.
May 12, 2014

Ultragenyx Reports First Quarter 2014 Financial Results and Corporate Update

NOVATO, Calif., May 12, 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 corporate update for the first quarter ended March 31, 2014.

"We have continued to make progress with our pipeline of clinical-stage programs for five diseases since the completion of our initial public offering in early 2014," said Emil D. Kakkis, Ph.D., M.D., Chief Executive Officer and President of Ultragenyx. "We are encouraged by the clinical results from our recombinant human beta-glucuronidase (rhGUS), sialic acid extended-release (SA-ER), and KRN23 programs, as well as the initiation of clinical studies for our triheptanoin programs."

First Quarter 2014 Financial Results

For the first quarter of 2014, Ultragenyx reported a net loss attributable to common stockholders of $18.4 million, or $0.85 per share, basic and diluted, compared with a net loss attributable to common stockholders for the first quarter of 2013 of $8.2 million, or $2.84 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 first quarter of 2014 were $10.3 million compared with $6.7 million for the same period in 2013. The increase in total operating expenses is due to the expansion and advancement of Ultragenyx's clinical development pipeline, including the initiation of clinical studies for triheptanoin and recombinant human beta-glucuronidase (rhGUS), as well as increased headcount.

Cash, cash equivalents, and short-term investments were $165.4 million as of March 31, 2014. Based on current operating levels, the company expects that this will be sufficient to fund operations into 2016.

Corporate Update

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)

Sialic Acid Extended-Release (SA-ER) in Hereditary Inclusion Body Myopathy (HIBM)

About Ultragenyx

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 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, timing of initiation of additional studies for its product candidates, plans regarding ongoing studies for existing programs and 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2013, filed with the Securities and Exchange Commission on March 24, 2014, 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,
Statements of Operations Data:    
Operating expenses:    
Research and development  $ 8,353  $ 5,664
General and administrative  1,986  1,083
Total operating expenses  10,339  6,747
Loss from operations  (10,339)  (6,747)
Other expense, net  (3,291)  12
Net loss  $ (13,630)  $ (6,735)
Net loss attributable to common stockholders  $ (18,438)  $ (8,205)
Net loss per share attributable to common stockholders, basic and diluted  $ (0.85)  $ (2.84)
Shares used to compute net loss per share attributable to common stockholders, basic and diluted  21,582,435  2,893,997
Ultragenyx Pharmaceutical Inc.
Selected Balance Sheets Financial Data
(in thousands)
 March 31,December 31,
Balance Sheet Data:    
Cash, cash equivalents and short-term investments  $ 165,397  $ 53,377
Working capital  162,825  49,304
Total assets  172,707  59,649
Convertible preferred stock warrant liability  —  3,419
Convertible preferred stock  —  124,930
Deficit accumulated during the development stage  (92,869)  (74,836)
Total stockholders' equity (deficit)  165,714  (74,821)
CONTACT: Ultragenyx Pharmaceutical Inc.


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