Synageva BioPharma™ Reports 2012 Full Year Financial Results
LEXINGTON, Mass.--(BUSINESS WIRE)--
Synageva
BioPharma Corp. (“Synageva”) (NASDAQ:GEVA), a clinical stage
biopharmaceutical company developing therapeutic products for rare
disorders, today reported 2012 full year financial results and provided
2013 financial guidance. Synageva’s management team will host a
conference call today at 4:30 p.m. EDT to review the financial results
and provide a general business update. To participate in today’s call
via telephone, please dial (877) 312-5514 for U.S. callers, or (970)
315-0452 for international callers. In addition, the conference call
will be webcast live from the “Webcasts & Presentations” section of the
Investor Relations tab on the home page of Synageva’s website at www.synageva.com.
“2012 marked our first full year as a public company. Synageva made
significant progress in advancing our lead program, sebelipase alfa. We
announced recently that enrollment has started in the ARISE global,
Phase 3 trial. This was a key step towards providing an effective
therapy to patients suffering from LAL Deficiency,” said Sanj K. Patel,
President and Chief Executive Officer of Synageva. “We continue to
advance our other preclinical programs, and we entered 2013 with a
strong cash balance which will be thoughtfully and strategically
allocated to facilitate the achievement of our goals for 2013 and
beyond.”
Recent Company Highlights
Synageva reported progress and updates on a number of key objectives
during the past year including the following:
Presentation of three, six and nine-month data from the ongoing Phase
1/2 extension study of sebelipase alfa in adults with late onset
lysosomal acid lipase deficiency (LAL Deficiency), including
acceptance for publication of 12-week data in the journal Hepatology
Publication of recent prevalence estimates of late onset LAL
Deficiency in the online version and an upcoming print edition of the
journal Hepatology
Interactions with U.S., European and other global regulatory
authorities that allowed us to begin clinical trial site initiations
and dosing of the first patient in the Acid Lipase Replacement
Investigating Safety and Efficacy (ARISE) global, Phase 3 trial of
sebelipase alfa in children and adults with LAL Deficiency in February
2013
Continuation of treatment and enrollment in the Phase 2/3 trial of
sebelipase alfa in infants with early onset LAL Deficiency
Advancement of our SBC-103 program for Mucopolysaccharidosis Type IIIB
(MPS IIIB, also known as Sanfilippo B) by demonstrating reduced
lysosomal substrate storage in the brain, liver and kidney tissues in
a mouse model using various dosing approaches
Expansion of our global clinical, medical affairs and commercial
infrastructure to further enhance our ability to raise awareness and
help identify more patients with LAL Deficiency
Executed on plans to develop a redundant, commercial-scale
manufacturing facility to further supply protein therapeutics for our
lead programs
Strengthened balance sheet as a result of completing a follow-on
offering in January 2013, resulting in $111.0 million of net proceeds,
in addition to the Company’s $219.0 million of cash, cash equivalents
and short-term investments as of December 31, 2012
2013 Key Objectives
Corporate objectives for 2013 include the following:
Progress enrollment in the ARISE Phase 3 trial of sebelipase alfa in
children and adults with late onset LAL Deficiency
Progress enrollment in the Phase 2/3 trial of sebelipase alfa in
infants with early onset LAL Deficiency
Provide updates from the ongoing Phase 1/2 extension study of
sebelipase alfa in adults with LAL Deficiency including one year of
treatment data
Continue global disease awareness programs to support identification
of more affected infants, children and adults with LAL Deficiency
Advance SBC-103 to initiate first-in-man clinical trials for MPS IIIB
in the first half of 2014
Advance additional pipeline programs
Progress towards completion of a redundant, commercial-scale
manufacturing facility to further supply protein therapeutics for our
lead programs
2012 Full Year Financial Results
For the year ended December 31, 2012, Synageva reported a net loss of
$42.9 million compared to a net loss of $25.3 million for the
corresponding period of the prior year.
Revenue for the full year ended December 31, 2012 of $15.0 million
consists of $7.0 million of Fuzeon royalties from Roche, as well as
revenue of $7.9 million from Synageva’s collaboration with Mitsubishi
Tanabe Pharma Corporation. Total costs and expenses for the full year
2012, including R&D expenses, G&A expenses, and amortization of
developed technology totaled $58.0 million. This compares to total costs
and expenses for the full year 2011 of $27.1 million.
Synageva had cash, cash equivalents and short-term investments totaling
$219.0 million on December 31, 2012, compared with a $60.2 million cash,
cash equivalents and short-term investments balance at December 31,
2011. The cash balance at December 31, 2012 does not include proceeds
from the recent follow-on offering completed in January 2013 that
resulted in $111.0 million in net proceeds to the company.
2013 Financial Outlook
Synageva currently expects a net loss guidance of between $87.0 and
$97.0 million for 2013. The net loss is primarily due toinvestments
necessary to support the global clinical development program for
sebelipase alfa, further development of SBC-103, expansion of the global
clinical, medical affairs and commercial infrastructure, expansion of
our manufacturing capabilities, as well as advancement of other pipeline
programs. In addition, we plan to invest a portion of our capital into
additional opportunities, beyond our first-mover pipeline programs, to
leverage our proprietary production platform to develop improved
biologic therapies for unmet medical needs.
About Synageva’s Lead Program
Sebelipase
alfa (SBC-102) is a recombinant form of
the human LAL enzyme being developed as an enzyme replacement therapy
for Lysosomal Acid Lipase (LAL) Deficiency, a lysosomal storage disorder
(LSD). Sebelipase alfa is currently being evaluated in global clinical
trials and has been granted orphan designations by the U.S. Food and
Drug Administration (“FDA”), the European Medicines Agency, and the
Japanese Ministry of Health, Labour and Welfare. Additionally,
sebelipase alfa received “fast track” designation by the FDA.
About LAL Deficiency
LAL
Deficiency is a rare autosomal recessive LSD caused by a marked
decrease in LAL enzyme activity. Late onset LAL Deficiency, sometimes
called Cholesteryl Ester Storage Disease (CESD), affects both children
and adults. In these patients, the buildup of fatty material in the
liver and blood vessel walls may lead to liver cirrhosis, liver failure
and accelerated atherosclerotic events. Early onset LAL Deficiency,
sometimes called Wolman disease, affects infants and is characterized by
severe malabsorption, growth failure and liver failure, and is usually
fatal within the first six months of life. There are no approved
pharmacological therapies for LAL Deficiency. Success with stem cell and
liver transplant appears to be limited by procedure-related morbidity
and mortality.
About Synageva BioPharma Corp.
Synageva is a clinical stage biopharmaceutical company focused on the
discovery, development, and commercialization of therapeutic products
for patients with life-threatening rare diseases and unmet medical need.
Synageva has several protein therapeutics in its pipeline. The company
has a team with a proven record of bringing orphan therapies to patients.
Further information regarding Synageva BioPharma Corp. is available at www.synageva.com.
Forward-Looking Statements
This news release contains “forward-looking statements”. Such statements
generally can be identified by the use of words such as “anticipate,”
“expect,” “plan,” “could,” “intend,” “believe,” “may,” “will,”
“estimate,” “forecast,” “project,” or words of similar meaning. These
forward-looking statements address among other matters our 2013
financial guidance including our expected 2013 net loss and the factors
underlying our guidance, planned allocation of our cash, and the
potential of our programs. Many factors may cause actual results to
differ materially from forward-looking statements, including inaccurate
assumptions and a broad variety of risks and uncertainties, some of
which are known, including unanticipated costs or delays in its research
and development programs, fluctuations in royalty revenues, unplanned
costs associated with maintaining and enforcing patents and other
patent-related costs, the ability to successfully progress enrollment
and advance our programs on the timelines that we expect and those
identified under the heading “Risk Factors” in the Company’s prospectus
supplement filed with the Securities and Exchange Commission (the “SEC”)
on January 3, 2013, and other filings Synageva periodically makes with
the SEC, and others of which are not known. No forward-looking statement
is a guarantee of future results or events, and investors should avoid
placing undue reliance on such statements. Synageva undertakes no
obligation to update any forward-looking statements, whether as a result
of new information, future events or otherwise. Our business is subject
to substantial risks and uncertainties, including those referenced
above. Investors, potential investors, and others should give careful
consideration to these risks and uncertainties.
Synageva BioPharma Corp.
Consolidated Statement of Operations
(in thousands, except per share amounts)
Three Months Ended
Years Ended
December 31,
December 31,
2012
2011
2012
2011
Revenues:
Royalty revenue
$
1,995
$
1,083
$
7,023
$
1,083
Collaboration and license revenue
2,885
446
7,875
640
Other revenue
-
85
56
376
Total revenue
4,880
1,614
14,954
2,099
Costs and expenses:
Research and development
12,908
5,149
37,347
17,346
General and administrative
6,345
3,362
17,396
9,268
Amortization of developed technology
878
504
3,232
504
Total costs and expenses
20,131
9,015
57,975
27,118
Loss from operations
(15,251)
(7,401)
(43,021)
(25,019)
Other (expense) income, net
-
(76)
-
(259)
Interest income (expense), net
60
(1)
72
(28)
Net loss
$
(15,191)
$
(7,478)
$
(42,949)
$
(25,306)
Basic and diluted net income per share (1)
$
(0.62)
$
(0.65)
$
(1.90)
$
(8.58)
Weighted average shares used in basic and diluted per share
computations (1)
24,347
11,587
22,579
2,950
(1) Per share computations for fiscal 2011 are based on (i) Private
Synageva’s historic common stock balances (excluding preferred stock) up
to the Merger date (November 2, 2011) and (ii) post-Merger common stock
from the Merger date to year end.
Synageva BioPharma Corp.
Consolidated Balance Sheet Data
(in thousands)
As of December 31,
2012
2011
Cash, cash equivalents, and short-term investments
$
218,953
$
60,232
Working capital
212,028
56,393
Total assets
243,256
83,298
Accumulated deficit
(158,789)
(115,840)
Total stockholders’ equity
$
230,177
$
74,048
“Dedicated to Rare Diseases®”is a registered trademark and “Synageva
BioPharma™” is a trademark of Synageva BioPharma Corp.
Press Release $GEVA Synageva BioPharma Corp.
LEXINGTON, Mass.--(BUSINESS WIRE)-- Synageva BioPharma Corp. (“Synageva”) (NASDAQ:GEVA), a clinical stage biopharmaceutical company developing therapeutic products for rare disorders, today reported 2012 full year financial results and provided 2013 financial guidance. Synageva’s management team will host a conference call today at 4:30 p.m. EDT to review the financial results and provide a general business update. To participate in today’s call via telephone, please dial (877) 312-5514 for U.S. callers, or (970) 315-0452 for international callers. In addition, the conference call will be webcast live from the “Webcasts & Presentations” section of the Investor Relations tab on the home page of Synageva’s website at www.synageva.com.
“2012 marked our first full year as a public company. Synageva made significant progress in advancing our lead program, sebelipase alfa. We announced recently that enrollment has started in the ARISE global, Phase 3 trial. This was a key step towards providing an effective therapy to patients suffering from LAL Deficiency,” said Sanj K. Patel, President and Chief Executive Officer of Synageva. “We continue to advance our other preclinical programs, and we entered 2013 with a strong cash balance which will be thoughtfully and strategically allocated to facilitate the achievement of our goals for 2013 and beyond.”
Recent Company Highlights
Synageva reported progress and updates on a number of key objectives during the past year including the following:
2013 Key Objectives
Corporate objectives for 2013 include the following:
2012 Full Year Financial Results
For the year ended December 31, 2012, Synageva reported a net loss of $42.9 million compared to a net loss of $25.3 million for the corresponding period of the prior year.
Revenue for the full year ended December 31, 2012 of $15.0 million consists of $7.0 million of Fuzeon royalties from Roche, as well as revenue of $7.9 million from Synageva’s collaboration with Mitsubishi Tanabe Pharma Corporation. Total costs and expenses for the full year 2012, including R&D expenses, G&A expenses, and amortization of developed technology totaled $58.0 million. This compares to total costs and expenses for the full year 2011 of $27.1 million.
Synageva had cash, cash equivalents and short-term investments totaling $219.0 million on December 31, 2012, compared with a $60.2 million cash, cash equivalents and short-term investments balance at December 31, 2011. The cash balance at December 31, 2012 does not include proceeds from the recent follow-on offering completed in January 2013 that resulted in $111.0 million in net proceeds to the company.
2013 Financial Outlook
Synageva currently expects a net loss guidance of between $87.0 and $97.0 million for 2013. The net loss is primarily due to investments necessary to support the global clinical development program for sebelipase alfa, further development of SBC-103, expansion of the global clinical, medical affairs and commercial infrastructure, expansion of our manufacturing capabilities, as well as advancement of other pipeline programs. In addition, we plan to invest a portion of our capital into additional opportunities, beyond our first-mover pipeline programs, to leverage our proprietary production platform to develop improved biologic therapies for unmet medical needs.
About Synageva’s Lead Program
Sebelipase alfa (SBC-102) is a recombinant form of the human LAL enzyme being developed as an enzyme replacement therapy for Lysosomal Acid Lipase (LAL) Deficiency, a lysosomal storage disorder (LSD). Sebelipase alfa is currently being evaluated in global clinical trials and has been granted orphan designations by the U.S. Food and Drug Administration (“FDA”), the European Medicines Agency, and the Japanese Ministry of Health, Labour and Welfare. Additionally, sebelipase alfa received “fast track” designation by the FDA.
About LAL Deficiency
LAL Deficiency is a rare autosomal recessive LSD caused by a marked decrease in LAL enzyme activity. Late onset LAL Deficiency, sometimes called Cholesteryl Ester Storage Disease (CESD), affects both children and adults. In these patients, the buildup of fatty material in the liver and blood vessel walls may lead to liver cirrhosis, liver failure and accelerated atherosclerotic events. Early onset LAL Deficiency, sometimes called Wolman disease, affects infants and is characterized by severe malabsorption, growth failure and liver failure, and is usually fatal within the first six months of life. There are no approved pharmacological therapies for LAL Deficiency. Success with stem cell and liver transplant appears to be limited by procedure-related morbidity and mortality.
About Synageva BioPharma Corp.
Synageva is a clinical stage biopharmaceutical company focused on the discovery, development, and commercialization of therapeutic products for patients with life-threatening rare diseases and unmet medical need. Synageva has several protein therapeutics in its pipeline. The company has a team with a proven record of bringing orphan therapies to patients.
Further information regarding Synageva BioPharma Corp. is available at www.synageva.com.
Forward-Looking Statements
This news release contains “forward-looking statements”. Such statements generally can be identified by the use of words such as “anticipate,” “expect,” “plan,” “could,” “intend,” “believe,” “may,” “will,” “estimate,” “forecast,” “project,” or words of similar meaning. These forward-looking statements address among other matters our 2013 financial guidance including our expected 2013 net loss and the factors underlying our guidance, planned allocation of our cash, and the potential of our programs. Many factors may cause actual results to differ materially from forward-looking statements, including inaccurate assumptions and a broad variety of risks and uncertainties, some of which are known, including unanticipated costs or delays in its research and development programs, fluctuations in royalty revenues, unplanned costs associated with maintaining and enforcing patents and other patent-related costs, the ability to successfully progress enrollment and advance our programs on the timelines that we expect and those identified under the heading “Risk Factors” in the Company’s prospectus supplement filed with the Securities and Exchange Commission (the “SEC”) on January 3, 2013, and other filings Synageva periodically makes with the SEC, and others of which are not known. No forward-looking statement is a guarantee of future results or events, and investors should avoid placing undue reliance on such statements. Synageva undertakes no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise. Our business is subject to substantial risks and uncertainties, including those referenced above. Investors, potential investors, and others should give careful consideration to these risks and uncertainties.
Synageva BioPharma Corp.
Consolidated Statement of Operations
(in thousands, except per share amounts)
Weighted average shares used in basic and diluted per share computations (1)
(1) Per share computations for fiscal 2011 are based on (i) Private Synageva’s historic common stock balances (excluding preferred stock) up to the Merger date (November 2, 2011) and (ii) post-Merger common stock from the Merger date to year end.
Synageva BioPharma Corp.
Consolidated Balance Sheet Data
(in thousands)
“Dedicated to Rare Diseases®”is a registered trademark and “Synageva BioPharma™” is a trademark of Synageva BioPharma Corp.
Synageva BioPharma Corp.
Matthew Osborne, 781-357-9947
matthew.osborne@synageva.com
Source: Synageva BioPharma Corp.