Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene
therapy company developing life-changing treatments for severe
neurological diseases, today reported its second quarter of 2017
financial results and corporate highlights.
“The second quarter of 2017 was a very
productive period for our company,” said Steve Paul, M.D.,
president and chief executive officer of Voyager
Therapeutics. “Our Parkinson’s disease program continues to
advance as we optimize dose and delivery before initiating our
pivotal program later this year. Our preclinical pipeline
programs also made solid progress toward entering the clinic.
In addition, work with our collaborators to further optimize AAV
capsids demonstrated exciting progress, including enhanced gene
transfer to the brain and spinal cord in preclinical models
compared to the historical standard, AAV9. During the
quarter, we also made a number of key hires to strengthen our
leadership team and solidify our position as the leading gene
therapy company focused on severe neurological diseases.”
Recent Program Highlights
VY-AADC for advanced Parkinson’s
disease:
Successfully administered VY-AADC01 to the first
patient in a Phase 1 trial with a posterior surgical delivery
approach. This posterior (i.e., back of the head) delivery
approach aims to further optimize the surgical delivery of
VY-AADC01 for advanced Parkinson’s disease. A posterior
approach into the putamen, the specific region of the brain
targeted by Voyager’s gene therapy program, could better align the
delivery of VY-AADC01 with the anatomical structure of the putamen
to potentially reduce the total procedure time and further increase
the total coverage of the putamen. The administration of
VY-AADC01 with this posterior approach was well-tolerated and no
serious adverse events were reported and, similar to many patients
in the Phase 1b trial, this patient was discharged from the
hospital one day after surgery. Additional patients completed
screening and will enroll shortly. Preliminary total
procedure time and putaminal coverage data from this posterior
trajectory trial will help inform the design of the pivotal Phase
2-3 program planned to initiate during late 2017.
Pipeline program and platform
highlights:
Progressed IND-enabling studies for
VY-SOD101. Earlier in the year, Voyager announced the
selection of VY-SOD101 as a clinical candidate for the treatment of
amyotrophic lateral sclerosis (ALS) caused by mutations in the
superoxide dismutase 1 gene (SOD1). With a single intrathecal
(IT) injection, VY-SOD101 has the potential to durably reduce the
levels of toxic mutant SOD1 protein in the central nervous system
(CNS) to slow the progression of disease. Preclinical data in
large mammals demonstrated that a single IT administration resulted
in robust knock-down of SOD1 in motor neurons. During the second
quarter, Voyager progressed pharmacology and toxicology studies to
support the filing of an investigational new drug (IND) application
for VY-SOD101 during late 2017 or early 2018.
Selected VY-HTT01 as a clinical candidate for
the treatment of Huntington’s disease. Huntington's disease
is a fatal, inherited neurodegenerative disease that results in the
progressive decline of motor and cognitive functions caused by an
expansion mutation in the huntingtin, or HTT, gene. VY-HTT01
is composed of an adeno-associated virus (AAV) capsid and
proprietary transgene that harnesses the RNA interference pathway
to selectively silence the production of HTT. Direct delivery
of VY-HTT01 to the brain with a one-time administration could
potentially slow or halt the progression of this uniformly fatal
disease. In preclinical models, a single administration of
VY-HTT01 was well-tolerated and resulted in robust and widespread
knockdown of HTT messenger RNA in disease-relevant regions of the
non-human primate central nervous system. The extent of HTT
mRNA suppression (greater than 50%) and high precision and
efficiency of primary microRNA processing in these preclinical
studies supported the selection of VY-HTT01 as our lead clinical
candidate. Announced the publication of new
preclinical data from the California Institute of Technology on a
second-generation gene therapy capsid demonstrating further
enhanced gene transfer to the brain. A core competency of
Voyager is the ability to genetically engineer and optimize capsids
to yield vectors with desirable properties such as enhanced tissue
specificity and improved delivery of genes to the brain and spinal
cord. Recently, the company announced the publication from
the California Institute of Technology (Caltech) in Nature
Neuroscience of new preclinical data from ongoing efforts of Dr.
Benjamin Deverman, Professor Viviana Gradinaru and the Gradinaru
Laboratory at Caltech to develop novel AAV capsids that efficiently
cross the blood-brain barrier and widely transduce, or transfer,
genes into the CNS after intravenous administration. From
these efforts, a new, second-generation AAV capsid provided up to a
100-fold increase in the transduction of the CNS in an adult mouse
model over the historical standard, AAV9, as compared with the
first-generation capsid reported last year by the Gradinaru group
that provided a more than 40-fold improvement over AAV9. Key
translational studies in non-human primates are underway by Voyager
to evaluate these AAV-variant capsids that have the potential to
transform the company’s ability to deliver gene therapies to the
CNS. Voyager obtained a co-exclusive license to the Caltech
novel AAV capsids, intellectual property and related technology in
September 2016. The license agreement covers all fields of
use and includes novel AAV capsids that have demonstrated enhanced
crossing of the blood-brain barrier for the potential treatment of
CNS diseases following systemic administration of an AAV gene
therapy vector.
Strengthened business operations and clinical
development teams:
Hired Allison Dorval as vice president of
finance. Ms. Dorval brings to Voyager over 20 years of
corporate finance and accounting experience, including nine years
of biopharmaceutical executive finance experience, most recently at
Juniper Pharmaceuticals, Inc., and Insulet Corporation, where she
led financial planning, reporting and accounting including
processes for Sarbanes-Oxley compliance. Allison is a
certified public accountant and began her career in public
accounting at PricewaterhouseCoopers, LLP where she held positions
of increasing responsibility and scope. Ms. Dorval obtained a
B.S. in Business Administration (Accounting concentration) from the
University of Vermont.
Hired Robert Blood as vice president of legal
affairs. Mr. Blood provides counsel across a wide range of
legal areas including business development and strategic
collaborations, commercial transactions, corporate governance,
intellectual property, compliance and matters related to the
securities and exchange commission. Rob joins Voyager from AMAG
Pharmaceuticals, Inc., where he was vice president of legal
affairs, deputy general counsel & chief compliance officer
providing legal counsel to support organizational growth while
managing risk in compliance with securities, healthcare and privacy
laws. Previously, Rob was associate general counsel for
EMD Serono and his large firm experience includes Goodwin Procter
LLP, and Montgomery, McCracken, Walker & Rhodes, LLP. Rob
obtained his J.D. from Northeastern University School of Law, and a
B.S. in Foreign Affairs from Georgetown University.
Hired Steven Hersch, M.D., Ph.D., as senior
director in clinical development. Dr. Hersch is a leading HD
researcher, was a founding member and co-chair of the Huntington
Study Group, the leading academic research consortium for HD, and
led the development of the Huntington’s Disease Society of
America’s Center of Excellence Program. He is also a
professor of neurology at Harvard Medical School and director of
the Laboratory of Neurodegeneration and Neurotherapeutics at
Massachusetts General Hospital. His work has encompassed the
anatomy and pharmacology of the cortex and striatum,
pathophysiology of HD, preclinical target identification and
therapeutic proof-of-concept studies in transgenic mouse models,
biomarker development, natural history and neuroimaging studies of
HD patients, clinical measure development, and the conduct of early
and late phase clinical trials. Dr. Hersch has over 125
publications to his name and earned Ph.D. and M.D. degrees from
Boston University School of Medicine, an M.A. in Medicine from
Harvard Medical School, and a B.S. in Interdisciplinary
Neuroscience from The American University.
Second Quarter 2017 Financial Results
and 2017 Guidance
Voyager reported a GAAP net loss of $18.9
million, or $0.73 per share, for the second quarter ended June 30,
2017, compared to a GAAP net loss of $9.3 million, or $0.37 per
share, for the same period in 2016.
Collaboration revenues of $1.2 million for the
second quarter of 2017 compared to $3.7 million for the second
quarter of 2016. Collaboration revenues reflect recognition
of payments for research and development services provided to
Voyager for various programs under the Sanofi-Genzyme collaboration
agreement. Collaboration revenues, which are subject to
variability based on quarterly assessments of expected or
anticipated efforts under the collaboration, decreased during the
second quarter of 2017 from the prior year period primarily due to
ongoing reviews of programs under the collaboration.
Research and development (R&D) expenses of
$15.3 million for the second quarter ended June 30, 2017 compared
to $10.5 million for the same period in 2016. The increase in
R&D expenses was primarily due to expenditures associated with
the development of Voyager’s pipeline, and increased personnel and
facility costs to support the advancement of the pipeline
programs.
General and administrative (G&A) expenses of
$4.5 million for the second quarter ended June 30, 2017 compared to
$2.9 million for the same period in 2016. The increase in
G&A expenses primarily related to personnel costs to support
Voyager’s growth and facility costs.
Total cash, cash equivalents, and marketable
debt securities as of June 30, 2017 were $141.3 million.
Based on the company’s current operating plan, Voyager continues to
expect to end 2017 with total cash, cash equivalents, and
marketable debt securities of approximately $90 million to $100
million and continues to project that its existing cash, cash
equivalents, and marketable debt securities will be sufficient to
fund operating expenses and capital expenditure requirements into
2019.
Conference Call
Information
Voyager will host a conference call and webcast
today at 8:30 a.m. EDT. The live call may be accessed by
dialing (877) 851-3834 for domestic callers or +1 (631) 291-4595
for international callers, and referencing conference ID number
62803624. A live audio webcast of the conference call will be
available online from the Investors & Media section of
Voyager’s website at www.voyagertherapeutics.com. The webcast
will be archived for 30 days.
About Parkinson’s Disease and
VY-AADC
Parkinson’s disease is a chronic, progressive
and debilitating neurodegenerative disease that affects
approximately 700,000 people in the U.S.1 and seven to 10 million
people worldwide2. It is estimated that up to 15% of the
prevalent population with Parkinson’s disease, or approximately
100,000 patients in the U.S., have motor fluctuations that are
refractory, or not well-controlled, with levodopa. While the
underlying cause of Parkinson's disease in most patients is
unknown, the motor symptoms of the disease arise from a loss of
neurons in the midbrain that produce the neurotransmitter
dopamine. Declining levels of dopamine in this particular
region of the brain (the putamen) leads to the motor symptoms
associated with Parkinson’s disease including tremors, slow
movement or loss of movement, rigidity, and postural
instability. Motor symptoms during the advanced stages of the
disease include falling, gait freezing, and difficulty with speech
and swallowing, with patients often requiring the daily assistance
of a caregiver.
There are currently no therapies that
effectively slow or reverse the progression of Parkinson’s
disease. Levodopa remains the standard of care treatment,
with its beneficial effects on symptom control having been
discovered over 40 years ago3. Patients are generally
well-controlled with oral levodopa in the early stages of the
disease, but become less responsive to treatment as the disease
progresses. Patients experience longer periods of reduced
mobility and stiffness termed off-time, or the time when medication
is no longer providing benefit, and shorter periods of on-time when
their medication is effective.
The progressive motor symptoms of Parkinson’s
disease are largely due to the death of dopamine neurons in the
substantia nigra, a part of the midbrain that converts levodopa to
dopamine, in a single step catalyzed by the enzyme AADC.
Neurons in the substantia nigra release dopamine into the putamen
where the receptors for dopamine reside. In advanced
Parkinson’s disease, neurons in the substantia nigra degenerate and
the enzyme AADC is markedly reduced in the putamen, which limits
the brain’s ability to convert oral levodopa to dopamine4.
The intrinsic neurons in the putamen, however, do not degenerate in
Parkinson’s disease5,6. VY-AADC, comprised of the
adeno-associated virus-2 capsid and a cytomegalovirus promoter to
drive AADC transgene expression, is designed to deliver the AADC
gene directly into neurons of the putamen where dopamine receptors
are located, bypassing the substantia nigra neurons and enabling
the neurons of the putamen to express the AADC enzyme to convert
levodopa into dopamine. The approach with VY-AADC, therefore,
has the potential to durably enhance the conversion of levodopa to
dopamine and provide clinically meaningful improvements by
restoring motor function in patients and improving symptoms
following a single administration.
About Amyotrophic Lateral
Sclerosis
Amyotropic Lateral Sclerosis is a rare, rapidly
progressive, fatal disease characterized by the degeneration of
nerve cells in the spinal cord and brain resulting in severe muscle
atrophy with loss of the ability to walk and speak, and premature
death. The median survival is approximately three years, and
90 percent of people with ALS die within five years of symptom
onset.7 ALS affects approximately 20,000 people in the U.S.,
with less than 10,000 new cases identified each year reflecting a
high rate of mortality and short survival, relative to other
diseases with similar incidences.8
Patients with ALS typically develop weakness in
one body region (upper or lower limb or bulbar) and then develop
symptoms and signs of progressive dysfunction of motor
neurons. The majority of ALS cases occur sporadically and
with unknown cause, but in approximately 10 percent of patients,
the cause is familial and can be linked to an identifiable genetic
defect. An estimated 20 percent of familial cases can
be attributed to mutations in SOD1, the first mutant gene
discovered to be causal for the development of ALS, through a toxic
gain of function mechanism leading to motor neuron pathogenesis.9
Riluzole is the only drug approved by the U.S. Food and Drug
Administration for the treatment of ALS. In controlled
trials, Riluzole delayed the time to onset of tracheostomy or death
by approximately two to three months but did not improve muscle
strength or neurological function.
About Voyager
Therapeutics
Voyager Therapeutics is a clinical-stage gene
therapy company developing life-changing treatments for severe
neurological diseases. Voyager is committed to advancing the
field of AAV gene therapy through innovation and investment in
vector engineering and optimization, manufacturing and dosing and
delivery techniques. The company’s pipeline focuses on severe
neurological diseases in need of effective new therapies, including
advanced Parkinson’s disease, a monogenic form of ALS, Huntington’s
disease, Friedreich’s ataxia, frontotemporal dementia, Alzheimer’s
disease and severe, chronic pain. Voyager has broad strategic
collaborations with Sanofi Genzyme, the specialty care global
business unit of Sanofi, and the University of Massachusetts
Medical School. Founded by scientific and clinical leaders in
the fields of AAV gene therapy, expressed RNA interference and
neuroscience, Voyager Therapeutics is headquartered in Cambridge,
Massachusetts. For more information, please visit
www.voyagertherapeutics.com. Follow Voyager on
LinkedIn.
Forward-Looking
Statements
This press release contains forward-looking
statements for the purposes of the safe harbor provisions under The
Private Securities Litigation Reform Act of 1995 and other federal
securities laws. The use of words such as “may,” “might,” “will,”
“should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,”
“undoubtedly,” “project,” “intend,” “future,” “potential,” or
“continue,” and other similar expressions are intended to identify
forward-looking statements. For example, all statements
Voyager makes regarding the initiation, timing, progress and
reporting of results of its preclinical programs and clinical
trials and its research and development programs, its ability to
advance its AAV-based gene therapies into, and successfully
complete, clinical trials, its ability to continue to develop its
product engine, its ability to add new programs to its pipeline,
ability to enter into new partnerships or collaborations, its
expected cash, cash equivalents and marketable debt securities at
the end of a fiscal year and anticipation for how long expected
cash, cash equivalents and marketable debt securities will last,
and the timing or likelihood of its regulatory filings and
approvals, are forward looking. All forward-looking
statements are based on estimates and assumptions by Voyager’s
management that, although Voyager believes to be reasonable, are
inherently uncertain. All forward-looking statements are
subject to risks and uncertainties that may cause actual results to
differ materially from those that Voyager expected. These
statements are also subject to a number of material risks and
uncertainties that are described in Voyager’s most recent Annual
Report on Form 10-K filed with the Securities and Exchange
Commission, as updated by its subsequent filings with the
Securities and Exchange Commission. Any forward-looking
statement speaks only as of the date on which it was made.
Voyager undertakes no obligation to publicly update or revise any
forward-looking statement, whether as a result of new information,
future events or otherwise, except as required by law.
Selected Financial Information |
($-amounts in thousands, except per share data) |
(Unaudited) |
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
Six Months Ended |
|
|
June 30, |
|
June 30, |
Statement of
Operations Items: |
|
2017 |
|
|
2016 |
|
|
2017 |
|
2016 |
|
Collaboration revenue |
|
$ |
1,177 |
|
|
$ |
3,720 |
|
|
$ |
2,642 |
|
|
$ |
8,550 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
15,300 |
|
|
|
10,484 |
|
|
|
29,372 |
|
|
|
19,216 |
|
General
and administrative |
|
|
4,516 |
|
|
|
2,854 |
|
|
|
9,430 |
|
|
|
6,419 |
|
Total operating
expenses |
|
|
19,816 |
|
|
|
13,338 |
|
|
|
38,802 |
|
|
|
25,635 |
|
Operating loss |
|
|
(18,639 |
) |
|
|
(9,618 |
) |
|
|
(36,160 |
) |
|
|
(17,085 |
) |
Total other income
(expense) |
|
|
(42 |
) |
|
|
283 |
|
|
|
606 |
|
|
|
562 |
|
Loss before income
taxes |
|
|
(18,681 |
) |
|
|
(9,335 |
) |
|
|
(35,554 |
) |
|
|
(16,523 |
) |
Income tax
provision |
|
|
(195 |
) |
|
|
— |
|
|
|
31 |
|
|
|
— |
|
Net loss |
|
$ |
(18,876 |
) |
|
$ |
(9,335 |
) |
|
$ |
(35,523 |
) |
|
$ |
(16,523 |
) |
Net loss per share,
basic and diluted |
|
$ |
(0.73 |
) |
|
$ |
(0.37 |
) |
|
$ |
(1.37 |
) |
|
$ |
(0.66 |
) |
Weighted-average common
shares outstanding, basic and diluted |
|
|
25,946,333 |
|
|
|
25,228,405 |
|
|
|
25,869,390 |
|
|
|
25,152,587 |
|
|
|
|
|
|
|
|
|
|
|
June 30, |
|
December 31, |
|
Selected
Balance Sheet Items: |
|
2017 |
|
2016 |
|
Cash, cash equivalents,
and marketable debt securities |
|
$ |
141,323 |
|
$ |
174,418 |
|
Total assets |
|
$ |
158,414 |
|
$ |
189,566 |
|
Accounts payable and
accrued expenses |
|
$ |
7,621 |
|
$ |
7,038 |
|
Deferred revenue |
|
$ |
39,053 |
|
$ |
41,582 |
|
Total stockholders’
equity |
|
$ |
105,291 |
|
$ |
135,922 |
|
1 Willis et al, Neuroepidemiology.2010;34:143–151
2 www.pdf.org/en/parkinson_statistics
3 Poewe W, et al, Clinical Interventions in
Aging.2010;5:229-238.
4 Lloyd, J Pharmacol Exp Ther. 1975;195:453-464, Nagatsu, J
Neural Transm Suppl.2007
5 Cold Spring Harb Perspect Med 2012;2:a009258
6 Braak et al, Cell Tissue Res.2004;318:121-134
7 Sorenson EJ, et al. (2002) Neurology 59:280-282
8 www.alsa.org
9 Rosen D, et al. (1993) Nature 362:59-62
Investor Relations:
Matt Osborne
Vice President of Investor Relations & Corporate Communications
857-259-5353
mosborne@vygr.com
Media:
Katie Engleman
Pure Communications, Inc.
910-509-3977
Katie@purecommunicationsinc.com
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