Ultragenyx Pharmaceutical Inc.
Feb 16, 2017

Ultragenyx Reports Fourth Quarter and Full Year 2016 Financial Results and Corporate Update

NOVATO, Calif., Feb. 16, 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 and full year ended December 31, 2016.

"In 2016 we advanced each program in our clinical pipeline and continued to build our earlier stage pipeline through two new partnerships and ongoing progress with our translational research program," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "In 2017 we expect to drive two products through the regulatory process with two more in Phase 3 studies." 

Fourth Quarter and Full Year 2016 Financial Results

For the fourth quarter of 2016, Ultragenyx reported a net loss of $71.3 million, or $1.75 per share, basic and diluted, compared with a net loss for the fourth quarter of 2015 of $55.2 million, or $1.42 per share, basic and diluted. For the year ended December 31, 2016, net loss was $245.9 million, or $6.21 per share, basic and diluted, compared with a net loss for the same period in 2015 of $145.6 million, or $3.96 per share, basic and diluted. This reflected cash used in operations of $161.0 million for the year ended December 31, 2016 compared to $106.0 million for the same period in 2015.

Total operating expenses for the fourth quarter of 2016 were $70.6 million compared with $56.2 million for the same period in 2015, including non-cash stock-based compensation of $13.5 million and $9.5 million in the fourth quarter of 2016 and 2015, respectively.  Total operating expenses for the year ended December 31, 2016 were $248.1 million compared with $147.7 million for the same period in 2015, including non-cash stock-based compensation of $48.3 million and $24.9 million in 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 $498.1 million as of December 31, 2016.

Recent Highlights

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

UX007 in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)

Upcoming Key Milestones

KRN23 in XLH

rhGUS in MPS 7

UX007 in 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, Thursday, February 16, 2017 at 5pm ET to discuss fourth quarter and full year 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 67687697. 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 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, 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 November 8, 2016, 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 December 31, Year Ended December 31,
Statement of Operations Data:        
Revenue $5  $-  $133  $- 
Operating expenses:        
Research and development  50,746   44,565   183,204   114,737 
General and administrative  19,808   11,593   64,936   33,001 
Total operating expenses  70,554   56,158   248,140   147,738 
Loss from operations  (70,549)  (56,158)  (248,007)  (147,738)
Other income (expense), net  (703)  938   2,168   2,120 
Loss before income taxes  (71,252)  (55,220)  (245,839)  (145,618)
Income tax provision  (35)  -   (35)  - 
Net loss $(71,287) $(55,220) $(245,874) $(145,618)
Net loss per share, basic and diluted $(1.75) $(1.42) $(6.21) $(3.96)
Shares used in computing net loss per share,        
basic and diluted  40,783,829   38,847,922   39,586,908   36,782,603 

Ultragenyx Pharmaceutical Inc.
Selected Balance Sheets Financial Data
(in thousands)
  December 31, December 31,
  2016 2015
Balance Sheet Data:    
Cash, cash equivalents and investments $498,111 $536,256
Working capital  341,436  422,289
Total assets  540,626  559,569
Total stockholders' equity  473,974  531,090
Contact Ultragenyx Pharmaceutical Inc.
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Ryan Martins