Ultragenyx Pharmaceutical Inc.
Jul 27, 2017

Ultragenyx Reports Second Quarter 2017 Financial Results and Corporate Update

Burosumab US BLA submission planned for August 2017

NOVATO, Calif., July 27, 2017 (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 June 30, 2017.

"We look forward to submitting our biologics license application (BLA) for burosumab and working with the FDA to bring this potential treatment to adult and pediatric patients," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We believe the first bone biopsy data from our ongoing Phase 3 bone quality study underscore burosumab's impact on the underlying bone disease in adults with XLH." 

Second Quarter 2017 Financial Results

For the second quarter of 2017, Ultragenyx reported a net loss of $72.9 million, or $1.72 per share, basic and diluted, compared with a net loss of $56.9 million, or $1.46 per share, basic and diluted in the second quarter of 2016. For the six months ended June 30, 2017, net loss was $141.2 million, or $3.35 per share, basic and diluted, compared with a net loss for the same period in 2016 of $109.7 million, or $2.81 per share, basic and diluted. This reflected cash used in operations of $110.0 million for the six months ended June 30, 2017 compared to $84.6 million for the same period in 2016.

Total operating expenses for the second quarter of 2017 were $78.4 million compared with $58.1 million for the same period in 2016, including non-cash stock-based compensation of $16.8 million and $10.9 million for the second quarter of 2017 and 2016, respectively. Total operating expenses for the six months ended June 30, 2017 were $148.4 million compared with $111.7 million for the same period in 2016, including non-cash stock-based compensation of $31.3 million and $21.1 million in the first six months of 2017 and 2016, 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 $457.5 million as of June 30, 2017.

Recent Highlights

Burosumab (KRN23) anti-FGF23 Monoclonal Antibody in X-Linked Hypophosphatemia (XLH)

Vestronidase alpha or rhGUS in Mucopolysaccharidosis 7 (MPS 7)

Upcoming Key Milestones

Burosumab in XLH

Vestronidase alpha in MPS 7

Aceneuramic Acid Extended Release (Ace-ER) in GNE Myopathy

UX007 in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD) and Glut1 Deficiency Syndrome (Glut1 DS)

Conference Call & Webcast Information

Ultragenyx will host a conference call today, Thursday, July 27, 2017 at 5pm ET to discuss second quarter 2017 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 59665992. 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.

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.

Ultragenyx Product Candidates

Ultragenyx has completed a Phase 3 study of vestronidase alpha (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; Phase 2 and Phase 3 studies of burosumab, an antibody targeting fibroblast growth factor 23 (FGF23), in pediatric and adult patients with X-linked hypophosphatemia (XLH) and a Phase 2 study in tumor induced osteomalacia (TIO), both rare diseases that impair bone mineralization; a Phase 3 study for UX007 in patients with glucose transporter type-1 deficiency syndrome (Glut1 DS), a brain energy deficiency, who are experiencing movement disorders; a Phase 2 study of UX007 in Glut1 DS patients with seizures, and 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.

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, demonstrated impact and adequacy of clinical data and other information to support approval of product candidates, its intent to make regulatory submissions and its expectations regarding timing of receiving potential approval of its product candidates, ongoing or additional studies for its product candidates and timing regarding these studies, the design of clinical studies, the extent of its translational research program, potential indications for its product candidates, discussions with regulatory authorities, and sufficiency for, and timing of, regulatory submissions, 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, 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 May 5, 2017, and its subsequent periodic reports filed with the Securities and Exchange Commission.

 Ultragenyx Pharmaceutical Inc. 
 Selected Statement of Operations Financial Data 
 (in thousands, except share and per share amounts) 
  Three Months Ended June 30,  Six Months Ended June 30,  
   2017   2016   2017   2016  
 Statement of Operations Data:        
 Revenue$-  $17  $-  $17  
 Operating expenses:        
 Research and development 58,436   43,332   109,705   83,747  
 General and administrative 20,005   14,738   38,690   27,945  
 Total operating expenses 78,441   58,070   148,395   111,692  
 Loss from operations (78,441)  (58,053)  (148,395)  (111,675) 
 Other income, net 5,564   1,130   7,228   1,995  
 Loss before income taxes (72,877)  (56,923)  (141,167)  (109,680) 
 Income tax provision (14)  -   (14)  -  
 Net loss$(72,891) $(56,923) $(141,181) $(109,680) 
 Net loss per share, basic and diluted$(1.72) $(1.46) $(3.35) $(2.81) 
 Shares used in computing net loss per share,        
 basic and diluted 42,346,830   39,028,701   42,095,750   38,999,439  

 Ultragenyx Pharmaceutical Inc. 
 Selected Balance Sheets Financial Data 
 (in thousands) 
   June 30, December 31, 
    2017  2016 
 Balance Sheet Data:     
 Cash, cash equivalents and investments $457,450 $498,111 
 Working capital  357,119  341,436 
 Total assets  497,542  540,626 
 Total stockholders' equity  429,453  473,974 


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