Ultragenyx Pharmaceutical Inc.
Nov 2, 2017

Ultragenyx Reports Third Quarter 2017 Financial Results and Corporate Update

NOVATO, Calif., Nov. 02, 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 September 30, 2017.

"Burosumab and vestronidase alfa regulatory submissions have been accepted and regulatory review is progressing well in both the US and European Union. We are preparing for a near-term potential launch of vestronidase alfa with a PDUFA date of November 16," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We are continuing to build a next-generation rare disease company and we expect the pending acquisition of Dimension Therapeutics to provide a unique opportunity to approach treatment of more rare diseases with gene therapy technology."

Third Quarter 2017 Financial Results

For the third quarter of 2017, Ultragenyx reported a net loss of $79.2 million, or $1.87 per share, basic and diluted, compared with a net loss of $64.9 million, or $1.64 per share, basic and diluted in the third quarter of 2016. For the nine months ended September 30, 2017, net loss was $220.4 million, or $5.22 per share, basic and diluted, compared with a net loss for the same period in 2016 of $174.6 million, or $4.46 per share, basic and diluted. This reflected cash used in operations of $172.0 million for the nine months ended September 30, 2017 compared to $113.3 million for the same period in 2016.

Total operating expenses for the third quarter of 2017 were $83.9 million compared with $65.9 million for the same period in 2016, including non-cash stock-based compensation of $17.2 million and $13.7 million for the third quarter of 2017 and 2016, respectively. Total operating expenses for the nine months ended September 30, 2017 were $232.3 million compared with $177.6 million for the same period in 2016, including non-cash stock-based compensation of $48.5 million and $34.8 million in the first nine 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 $396.0 million as of September 30, 2017.

Recent Highlights

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

Burosumab (KRN23) anti-FGF23 Monoclonal Antibody in tumor-induced osteomalacia (TIO)


Upcoming Key Milestones

Burosumab in XLH

 Vestronidase alfa or rhGUS in Mucopolysaccharidosis 7 (MPS 7)

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, November 2, 2017 at 5pm ET to discuss third 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 3976859. 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). The Company is conducting 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 tumor induced osteomalacia (TIO), both rare diseases that impair bone mineralization; 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 an ongoing 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.

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 the pending acquisition of Dimension Therapeutics, Inc. and potential benefits of the acquisition, Ultragenyx's expectations regarding ongoing or additional studies for its product candidates and timing regarding these studies, the design of clinical studies, the demonstrated impact of clinical data and other information to support approval of product candidates, , potential indications for its product candidates, discussions with regulatory authorities, the potential issuance of a priority review voucher,  and sufficiency for, and timing of, regulatory submissions and approvals, 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 July 27, 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 September 30,  Nine Months Ended September 30,  
   2017   2016   2017   2016  
 Statement of Operations Data:        
 Revenue$198  $111  $198  $128  
 Operating expenses:        
 Research and development 60,412   48,711   170,117   132,458  
 General and administrative 23,499   17,183   62,189   45,128  
 Total operating expenses 83,911   65,894   232,306   177,586  
 Loss from operations (83,713)  (65,783)  (232,108)  (177,458) 
 Other income, net 4,490   876   11,718   2,871  
 Loss before income taxes (79,223)  (64,907)  (220,390)  (174,587) 
 Income tax provision (4)  -   (18)  -  
 Net loss$(79,227) $(64,907) $(220,408) $(174,587) 
 Net loss per share, basic and diluted$(1.87) $(1.64) $(5.22) $(4.46) 
 Shares used in computing net loss per share,        
 basic and diluted 42,471,606   39,551,923   42,222,413   39,184,994  


 Ultragenyx Pharmaceutical Inc. 
 Selected Balance Sheet Financial Data 
 (in thousands) 
   September 30, December 31, 
    2017  2016 
 Balance Sheet Data:     
 Cash, cash equivalents and investments $396,030 $498,111 
 Working capital  326,642  341,436 
 Total assets  435,047  540,626 
 Total stockholders' equity  365,558  473,974 

Contact Ultragenyx Pharmaceutical Inc.
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Danielle Keatley