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)
- KRN23 Conditional Marketing Authorization Application
(MAA) for XLH filed and accepted by the European Medicines Agency
(EMA) in December 2016. An opinion from the Committee for
Medicinal Products for Human Use is expected in the second half of
2017.
UX007 in Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
- Positive 78-week data from the Phase 2 study in LC-FAOD
showed a reduction in frequency and total duration of major medical
events. We continue to further develop the Phase 3 study
design and endpoints before meeting with regulators and initiating
the study in 2017.
Upcoming Key Milestones
KRN23 in XLH
- Data from the Phase 3 study in adult
XLH patients expected in the first half of 2017. The
fully-enrolled Phase 3 study of monthly KRN23 compared with placebo
over 24 weeks in 134 adult XLH patients will evaluate change in
serum phosphorus levels as the primary endpoint, and pain,
stiffness and physical function as key secondary endpoints.
- Ultragenyx plans to submit a biologics license
application (BLA) to the U.S. FDA for KRN23 in the second half of
2017. The company continues to discuss the details of the
planned submission with FDA, and expects to submit both pediatric
Phase 2 data and adult Phase 3 data, if positive.
Based on discussions with the FDA, the
pediatric Phase 3 study is currently not expected to be required
for a U.S. filing. In June 2016, the FDA granted
breakthrough therapy designation to KRN23 for the treatment of
X-linked hypophosphatemia (XLH) in pediatric patients one year of
age and older.
rhGUS in MPS 7
- Ultragenyx has met with FDA and EMA and plans to submit
regulatory filings in the first half of 2017, based on Phase 3
study results. In Europe, the primary endpoint is the
percent reduction in urinary glycosaminoglycans (GAG) excretion
after 24 weeks of treatment. The EMA has indicated that some
evidence or trend in improvement in clinical endpoints would also
be necessary for approval. In the US, there is no primary endpoint
declared; the FDA will consider the totality of data on a
per-patient basis.
UX007 in Glut1 Deficiency Syndrome (Glut1 DS)
- Phase 3 movement disorder study in Glut1 DS patients
expected to initiate imminently. The study is expected to
enroll approximately 40 patients and be a randomized, double-blind,
placebo-controlled, double cross-over study. The study is designed
to assess the impact of UX007 on disabling movement disorder events
as recorded by a patient diary.
- Phase 2 seizure study data in Glut1 DS patients
expected in the first quarter of 2017. The ongoing
placebo-controlled study is evaluating frequency of generalized and
partial tonic-clonic seizures by patient diary, absence seizures by
EEG, and cognitive function. The last patient visit has taken place
and the data are being prepared for analysis.
Aceneuramic Acid Extended Release (Ace-ER) in GNE Myopathy
- Data from the pivotal Phase 3 study in GNE myopathy
expected in the second half of 2017. The fully-enrolled
randomized, double-blind, placebo-controlled international study in
89 patients is evaluating the efficacy and safety of Ace-ER
compared with placebo over 48 weeks. We plan to submit an NDA and
MAA based on the Phase 3 data, if positive.
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) |
|
|
|
|
|
|
|
|
|
|
|
(unaudited) |
|
|
|
|
|
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
|
2016 |
|
2015 |
|
2016 |
|
2015 |
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) |
(unaudited) |
|
|
|
|
|
|
|
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.
Investors & Media
Ryan Martins
844-758-7273
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