Ultragenyx Reports Fourth Quarter and Full Year 2013 Financial Results

Ultragenyx Reports Fourth Quarter and Full Year 2013 Financial Results

NOVATO, Calif., March 24, 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 business highlights for the fourth quarter and year ended December
31, 2013.

"Ultragenyx completed a landmark year in 2013, during which we achieved
multiple milestones in our vision to build a next generation rare disease
company," said Emil D. Kakkis, Ph.D., M.D., Chief Executive Officer and
President of Ultragenyx. "We have expanded our pipeline to five clinical
programs in Phase 1/2 or Phase 2 studies, and with the completion of our
initial public offering in early 2014, we believe we are well-positioned to
advance multiple programs in parallel to address the significant unmet medical
needs of these patients."

Fourth Quarter and Full Year 2013 Financial Results

For the fourth quarter of 2013, Ultragenyx reported a net loss attributable to
common stockholders of $18.7 million, or $4.98 per share, basic and diluted,
compared with a net loss attributable to common stockholders for the fourth
quarter of 2012 of $6.8 million, or $2.83 per share, basic and diluted.

For the year ended December 31, 2013, Ultragenyx reported a net loss
attributable to common stockholders of $50.3 million, or $14.87 per share,
basic and diluted, compared with a net loss attributable to common
stockholders for the year ended December 31, 2012 of $19.6 million, or $14.20
per share, basic and diluted.

Total operating expenses for 2013 were $32.3 million compared with $16.0
million for 2012. Total operating expenses for the fourth quarter of 2013 were
$9.5 million compared with $4.7 million for the same period in 2012. The
increase in total operating expenses is due to the addition of triheptanoin
and KRN23 to Ultragenyx's clinical development pipeline, advancements in the
development of sialic acid extended release (SA-ER) and recombinant human
beta-glucuronidase (rhGUS), and increased headcount to support the company's

Cash, cash equivalents, and short-term investments were $53.4 million as of
December 31, 2013. Based on current operating levels, the company expects that
existing cash, cash equivalents and short-term investments, including
approximately $121.7 million in net proceeds received from the initial public
offering in February 2014, will be sufficient to fund operations into 2016.

Recent Highlights

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

  *Results from the Phase 1 single dose study in 38 adult XLH patients were
    presented at the American Society for Bone and Mineral Research Annual
    Meeting in October 2013 and published in the Journal of Clinical
    Investigation in February 2014^1. The data demonstrated that KRN23 was
    well tolerated and increased serum phosphate and vitamin D levels compared
    to placebo at higher doses.

rhGUS in Mucopolysaccharidosis 7 (MPS 7)

  *In December 2013, we initiated a Phase 1/2 study to evaluate the safety,
    tolerability, efficacy, and dose of rhGUS in up to five MPS 7 patients
    between five and 30 years of age.
  *In February 2014, results from the treatment of a single patient under an
    emergency investigational new drug (eIND) application were presented at
    the Lysosomal Disease Network's 10^th Annual World Symposium. The
    preliminary data showed a reduction in lysosomal storage based on reduced
    excretion of urinary glycosaminoglycans and a reduction in the size of the
    enlarged liver and spleen. The patient showed an improvement of pulmonary
    function and no infusion-associated reactions during the first 14 weeks of

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

  *In February 2014, we initiated a Phase 2 study in approximately 30
    severely affected LC-FAOD patients. A principal goal of the study is to
    determine the appropriate clinical endpoints and patient population for
    testing in potential later-stage pivotal studies.
  *Multiple investigator-sponsored and compassionate use trials testing
    triheptanoin in LC-FAOD patients are also ongoing.

Triheptanoin in Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)

  *In March 2014, we initiated a randomized, double-blind,
    placebo-controlled, parallel-group Phase 2 study in up to 50 Glut1 DS
    patients between three and 17 years of age inclusive who are currently not
    fully compliant with ketogenic diet and continue to have seizures. The
    primary efficacy objective is the reduction in frequency of seizures
    compared to placebo.
  *Multiple investigator-sponsored and compassionate use trials in Glut1 DS
    as well as in other indications are also ongoing.

SA-ER in Hereditary Inclusion Body Myopathy (HIBM)

  *In December 2013, we released top-line results after 48 weeks of treatment
    from the Phase 2 randomized study of SA-ER in 47 HIBM patients. The data
    showed that a modest increase in upper extremity muscle strength composite
    at the higher dose compared to a decline in the lower dose group was
    statistically significant. A positive trend was seen in a patient-reported
    outcome of functional activity. The results were consistent with the
    24-week analysis. SA-ER appeared to be well tolerated with no serious
    adverse events observed to date in either dose group.
  *We continue to treat patients in an extension study evaluating an
    increased daily dosage of sialic acid based on the dose dependence
    observed at weeks 24 and 48 of the Phase 2 study.

Corporate Highlights

  *In February 2014, we closed our initial public offering of 6,624,423
    shares of common stock at an initial public offering price of $21.00 per
    share, which includes the exercise in full by the underwriters of their
    option to purchase up to an additional 864,054 additional shares of common
    stock. Net proceeds from the offering were approximately $121.7 million,
    after deducting underwriting discounts and commissions, estimated offering
    expenses, and a dividend paid to preferred stockholders.
  *In January 2014, concurrent with the pricing of our initial public
    offering, we appointed Clay B. Siegall, Ph.D. and Matthew K. Fust to our
    board of directors. Dr. Siegall is President, Chief Executive Officer, and
    Chairman of the Board of Seattle Genetics, Inc. Mr. Fust is former
    Executive Vice President and Chief Financial Officer of Onyx
    Pharmaceuticals, Inc.

Anticipated 2014 Program Milestones

KRN23 in XLH

  *Release data from the Phase 1/2 adult repeat-dose studies completed by our
    collaborative partner Kyowa Hakko Kirin Co., Ltd. (KHK).
  *Initiate a Phase 2 study in pediatric XLH patients following discussions
    with multiple regulatory agencies on our pediatric study design.

rhGUS in MPS 7

  *Release early urinary glycosaminoglycans data on the first three MPS 7
    patients dosed with rhGUS in our Phase 1/2 study in March 2014 at the
    Annual Clinical Genetics Meeting (ACMG).
  *Release interim 12-week data from our Phase 1/2 study of rhGUS in patients
    with MPS 7 in the second half of 2014.
  *Initiate a pivotal Phase 3 study of at least 12 patients in the second
    half of 2014 if the 12-week Phase 1/2 data are positive.

Triheptanoin in LC-FAOD

  *Continue to enroll and treat patients in the ongoing Phase 2 study of
    triheptanoin in approximately 30 severely affected LC-FAOD patients. We
    expect that data from this study should be available in 2015.

Triheptanoin in Glut1 DS

  *Continue to enroll and treat patients in the ongoing Phase 2 study of
    triheptanoin in up to 50 Glut1 DS patients. We expect to release data from
    this trial in 2015.


  *Present detailed data from the 48-week randomized, controlled Phase 2
    study of SA-ER in 47 HIBM patients at the American Academy of Neurology
    (AAN) Annual Meeting in April. The abstract was one of ten chosen for
    presentation in a late-breaking news session called the Emerging Sciences
    session. A brief topline presentation of the results will take place on
    April 30, 2014 at approximately 6:30pm ET and will be followed by a poster
  *Release data from the ongoing extension study of SA-ER at a higher daily
    dosage in late 2014.

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 metabolic 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

^1Carpenter TO, et al. Randomized trial of the anti-FGF23 antibody KRN23 in
X-linked hypophosphatemia. J Clin Invest. 2014. doi:10.1172/JCI72829.

Forward-Looking Statements

Except for the historical information contained herein, the matters set forth
in this press release, including statements regarding Ultragenyx's plans,
potential opportunities, expectations, projections, goals, objectives,
milestones, strategies, product pipeline, clinical studies, product
development, release of data and the 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
prospectus filed with the Securities and Exchange Commission on January 31,
2014, and its future periodic reports to be 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     Year Ended
                                December 31,           December 31,
                               2012       2013        2012        2013
Statements of Operations Data:                                  
Operating expenses:                                             
Research and development        $3,775   $8,204    $12,641   $27,829
General and administrative      903       1,321      3,344      4,451
Total operating expenses        4,678     9,525      15,985     32,280
Loss from operations            (4,678)   (9,525)    (15,985)   (32,280)
Other expense, net              (253)     (1,792)    (349)      (2,790)
Net loss                        $(4,931) $(11,317) $(16,334) $(35,070)
Net loss attributable to common $(6,813) $(18,665) $(19,561) $(50,289)
Net loss per share attributable
to common stockholders, basic   $(2.83)  $(4.98)   $(14.20)  $(14.87)
and diluted
Shares used to compute net loss
per share attributable to       2,404,852 3,744,525  1,377,207  3,382,489
common stockholders, basic and
Ultragenyx Pharmaceutical Inc.
Selected Balance Sheets Financial Data
(in thousands)
                                         As of December 31,      
                                         2012        2013        
Balance Sheet Data:                                             
Cash, cash equivalents and                $86,190   $53,377   
short-term investments
Working capital                           83,257     49,304     
Total assets                              88,316     59,649     
Convertible preferred stock               518        3,419      
warrant liability
Convertible preferred stock               111,387    124,930    
Deficit accumulated during the            (27,058)   (74,836)   
development stage
Total stockholders' deficit               (27,047)   (74,821)   

CONTACT: Ultragenyx Pharmaceutical Inc.
         For Media, Bee Nguyen
         For Investors, Robert Anstey
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