Ultragenyx Pharmaceutical Inc.
Nov 7, 2016

Ultragenyx Reports Third Quarter 2016 Financial Results and Corporate Update

NOVATO, Calif., Nov. 07, 2016 (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 quarter ended September 30, 2016.

"Based on recent discussions with the FDA, we now plan to file for US approval of KRN23 in the second half of 2017," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We expect continued clinical and regulatory progress across all of our programs, including filings for rhGUS and the European filing for KRN23."

Third Quarter 2016 Financial Results

For the third quarter of 2016, Ultragenyx reported a net loss of $64.9 million, or $1.64 per share, basic and diluted, compared with a net loss for the third quarter of 2015 of $39.2 million, or $1.03 per share, basic and diluted. For the nine months ended September 30, 2016, net loss was $174.6 million, or $4.46 per share, basic and diluted, compared with a net loss for the same period in 2015 of $90.4 million, or $2.51 per share, basic and diluted. This reflected cash used in operations of $113.3 million for the nine months ended September 30, 2016 compared to $65.5 million for the same period in 2015.

Total operating expenses for the third quarter of 2016 were $65.9 million compared with $39.9 million for the same period in 2015, including non-cash stock-based compensation of $13.7 million and $7.9 million in the third quarter of 2016 and 2015, respectively.  Total operating expenses for the nine months ended September 30, 2016 were $177.6 million compared with $91.6 million for the same period in 2015, including non-cash stock-based compensation of $34.8 million and $15.4 million in the first nine months of 2016 and 2015, 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.

Cash, cash equivalents, and investments were $472.6 million as of September 30, 2016.

Recent Highlights

KRN23 anti-FGF23 Monoclonal Antibody in X-Linked Hypophosphatemia (XLH) and Tumor-Induced Osteomalacia (TIO)

rhGUS in MPS 7

Corporate Updates

Upcoming Milestones

KRN23 in XLH

rhGUS in 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

Ultragenyx will host a conference call today, Monday, November 7, 2016 at 5pm ET to discuss third quarter 2016 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 7847134. The replay of the call will be available for one year.

About Ultragenyx

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.

Ultragenyx has completed a Phase 3 study of recombinant human beta-glucuronidase (rhGUS) in patients with mucopolysaccharidosis 7 (MPS 7), a rare lysosomal storage disease, and is conducting a Phase 3 study of aceneuramic acid extended-release (Ace-ER) in patients with GNE myopathy, a progressive muscle-wasting disorder; a Phase 2 study for UX007 in patients with glucose transporter type-1 deficiency syndrome (Glut1 DS), a brain energy deficiency; a Phase 2 clinical study of UX007 in patients severely affected by long-chain fatty acid oxidation disorders (LC-FAOD), a genetic disorder in which the body is unable to convert long chain fatty acids into energy; and Phase 2 and Phase 3 studies of KRN23, an antibody targeting fibroblast growth factor 23 (FGF23), in pediatric and adult patients with X-linked hypophosphatemia (XLH) and tumor induced osteomalacia (TIO), both rare diseases that impair bone mineralization.

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, expectations regarding the adequacy of clinical data to support approval of product candidates, its intent to file for conditional approval and its expectations regarding timing of receiving potential approval of its product candidates, 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), whether the Phase 3 results for Ace-ER will in fact confirm or mirror the results from the prior Phase 2 study, whether the FDA will accept the planned BLA submission for KRN23, 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 9, 2016, 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,
  2016   2015   2016   2015 
Statements of Operations Data:       
Revenue$  111  $  -   $  128  $  -  
Operating expenses:       
Research and development   48,711     29,704     132,458     70,172 
General and administrative   17,183     10,232     45,128     21,408 
Total operating expenses   65,894     39,936     177,586     91,580 
Loss from operations   (65,783)    (39,936)    (177,458)    (91,580)
Other income, net   876     704     2,871     1,182 
Net loss$  (64,907) $  (39,232) $  (174,587) $  (90,398)
Net loss per share, basic and diluted$  (1.64) $  (1.03) $  (4.46) $  (2.51)
Shares used in computing net loss per share,       
basic and diluted   39,551,923     38,268,632     39,184,994     36,086,598 


 Ultragenyx Pharmaceutical Inc. 
 Selected Balance Sheets Financial Data 
 (in thousands) 
   September 30, December 31, 
    2016   2015  
 Balance Sheet Data:     
 Cash, cash equivalents and investments $  472,641  $  536,256  
 Working capital    366,354     422,289  
 Total assets    515,166     559,569  
 Total stockholders' equity    455,890     531,090  



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