Lead program VY-AADC01 for advanced Parkinson’s
disease on track to report 6-month data from Cohort 3 and
longer-term data from Cohorts 1 and 2 in mid-year 2017
Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene
therapy company developing life-changing treatments for severe
diseases of the central nervous system (CNS), today reported its
fourth quarter and full year 2016 financial results, and provided
corporate highlights, goals and financial guidance.
“Voyager’s exceptional performance and
accomplishments during 2016 created strong momentum for the company
for 2017 and beyond as we continue to focus our efforts on
developing gene therapies for devastating diseases of the CNS,”
said Steven Paul, M.D., president and chief executive officer of
Voyager Therapeutics. “The positive interim Phase 1b data we
provided late last year for VY-AADC01 for advanced Parkinson’s
disease provided proof-of-concept that a one-time, targeted
delivery of a gene therapy was well tolerated and could enhance
patients’ sensitivity to levodopa while at the same time generate
durable, dose-related, and clinically meaningful improvements in
patients’ motor function. Additional data from this trial
mid-year from more patients for longer duration will inform the
design of a double-blind, placebo-controlled trial expected to
start later this year. Our pipeline programs are rapidly
progressing with three INDs planned within the next 24 months for
our monogenic ALS, Huntington’s disease, and Friedreich’s ataxia
programs. During the year, we allocated capital wisely
towards our manufacturing capabilities and vector engineering
platform providing us with a strong foundation for the
transformative years ahead.”
2016 and Recent Key Pipeline and
Corporate Highlights
Lead program highlights:
- In early December, reported positive interim results at six
months from the Phase 1b trial of VY-AADC01 for advanced
Parkinson’s disease demonstrating that real-time, intra-operative
accurate MRI-guided delivery of escalating doses of VY-AADC01 were
well tolerated, increased coverage of the putamen, increased
aromatic L-amino acid decarboxylase (AADC) enzyme activity,
enhanced response to levodopa, and generated durable, dose-related,
clinically meaningful improvements in various measures of patients’
motor function. This was especially evident at the higher
dose in Cohort 2 with improved motor symptoms as measured by part
three of the United Parkinson’s Disease Rating Scale (UPDRS-III)
off medication and on medication scores, and patient-reported diary
hours, suggesting higher peak effects and a longer duration of
action of levodopa. These effects were maintained and in some
patients improved at 12 months of follow-up.
- Announced acceptance of the interim Phase 1b data for VY-AADC01
as an emerging (late-breaking) oral presentation at the upcoming
AAN Annual Meeting being held April 22-28, 2017, in Boston,
MA. The interim Phase 1b data will be presented during the
Emerging Science Session taking place Tuesday, April 25, from 5:45
p.m. to 6:30 p.m. EDT.
- Reported the relationship between surgical coverage of the
region of the brain (the putamen) targeted with VY-AADC01 and AADC
enzyme activity that is responsible for converting levodopa to
dopamine in the putamen using [18F] fluorodopa (or F-Dopa) positron
emission tomography (PET). The results demonstrated that
surgical coverage of the putamen with VY-AADC01 was highly
correlated with change in AADC enzyme activity measured by F-Dopa
PET (r=0.81, p<0.05).
- Completed enrollment of five patients in Cohort 3 and reported
greater average coverage of the putamen in Cohort 3 (42%) compared
to Cohort 2 (34%) with similar infusion volumes and Cohort 1 (21%)
with a lower infusion volume. All five patients from Cohort 3
were discharged from the hospital within two days after completing
surgery.
Pipeline program highlights:
- Selected VY-SOD101 as a clinical candidate for the treatment of
ALS due to mutations in the superoxide dismutase 1 gene (SOD1) and
progressed potential lead candidates for VY-HTT01 for Huntington’s
disease, and VY-FXN01 for Friedreich’s ataxia.
- At the company’s R&D day in April, announced two new
preclinical programs; VY-TAU01 and VY-NAV01, which are focused on
the molecular targets tau and Nav1.7, respectively, and for which
Voyager owns worldwide rights to both programs. VY-TAU01 is
an adeno-associated virus (AAV) vectorized version of an anti-tau
monoclonal antibody for direct one-time delivery to the CNS.
VY-TAU01 could be a potential treatment for severe neurological
disorders, such as frontotemporal dementia and Alzheimer’s
disease. Based on preclinical data, Voyager believes that
this approach could achieve significantly higher levels of the
therapeutic anti-tau antibody in the CNS when compared to the
systemic administration of an antibody. VY-NAV01 targets the
knockdown, or silencing, of Nav1.7 in sensory neurons of the dorsal
root ganglia as a potential one-time treatment of certain forms of
severe, chronic pain. Such an approach may avoid the
dose-limiting side effects associated with the non-selective
profile of many current treatments used to treat severe, chronic
pain, and achieve a durable clinical benefit following a single
administration of the therapy.
Recent corporate highlights:
- 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. In September,
Voyager expanded its portfolio of AAV capsids through a
co-exclusive worldwide license agreement with the California
Institute of Technology (Caltech) related to novel AAV capsids with
enhanced blood-brain barrier penetration and CNS targeting. In
partnership with Dr. Ben Deverman and Professor Viviana Gradinaru
at Caltech, Voyager continues to advance this technology which, in
preclinical studies, generated AAV variants that broadly transduced
the CNS with enhanced efficiency after intravenous
injection.
- In September, announced a research collaboration with CHDI
Foundation, Inc. (CHDI) to advance Voyager’s VY-HTT01 program, an
AAV-mediated gene-silencing therapy for Huntington’s disease.
The collaboration builds upon a previous collaboration between CHDI
and Sanofi Genzyme and includes funding from CHDI to help support
preparation for and filing of an investigational new drug
application, as well as completion of a Phase 1 clinical
trial.
- Promoted Dinah Sah, Ph.D., to chief scientific officer.
As Voyager’s research and preclinical program leader since early
2014, Dr. Sah has overseen and led the research pipeline, driven
strong collaboration efforts with external industry and academic
groups, and built teams that include research and
development. Dr. Sah has planned, organized and successfully
led the research leading to clinical candidate nomination for
VY-SOD101, as well as the progression of the Huntington’s disease
and Friedreich’s ataxia programs towards lead selection. With
her 24-year industry track record that includes in-depth expertise
in RNA interference and leadership of multiple programs from
research to Phase 1, Dr. Sah will oversee the pipeline programs as
they advance from research to early development, as well as
platform discovery and translational work.
- Promoted Bernard Ravina, M.D., M.S., to chief medical
officer. As Voyager’s clinical development leader since early
2014, Dr. Ravina planned and successfully implemented Voyager’s
Phase 1b clinical trial for VY-AADC01 for advanced Parkinson’s
disease, identified, opened and expanded clinical trial sites,
drove strong patient and physician participation, and generated
support and key input from key opinion leaders. With his
track record and experience with other neurological diseases and a
team that includes clinical operations, medical, patient advocacy
and preclinical safety, Dr. Ravina will oversee the pipeline
programs as they advance through clinical development.
- Appointed Jane Pritchett Henderson as chief financial officer,
and strengthened the Board of Directors with the appointments of
Wendy L. Dixon, Ph.D., and Glenn F. Pierce, M.D., Ph.D. Ms.
Henderson brings more than 28 years of life sciences industry and
banking experience and leadership to Voyager, most recently serving
as chief financial and business officer of Kolltan Pharmaceuticals,
Inc. Dr. Dixon brings over 35 years of global
biopharmaceutical leadership experience where, as a senior
executive, she combined her technical and commercial background to
direct the development, launch and growth of over 20 new
pharmaceutical products, including many highly successful
multi-billion dollar global brands across multiple therapeutic
areas including oncology, virology, immunology and neurology.
Dr. Pierce serves as entrepreneur-in-residence at Third Rock
Ventures, having joined the company in 2016 after more than 30
years of research and development experience working with
biotechnology companies including Biogen, where he most recently
served as chief medical officer leading the hematology, cell and
gene therapies division.
Corporate Goals and 2017 Financial
Guidance
Voyager remains committed to becoming the
leading gene therapy company focused on severe diseases of the CNS
with expertise in discovery, development, manufacturing and
commercialization of gene therapy products for people living with
these devastating diseases. The significant accomplishments
achieved during 2016 provide a solid foundation for continued
progress during 2017 and beyond, as measured by the planned
achievements of the following corporate goals and 2017 financial
guidance:
- For VY-AADC01, provide six-month safety, biomarker and motor
function data from Cohort 3, as well as longer-term safety and
motor function data from Cohorts 1 and 2, from the ongoing Phase 1b
trial for advanced Parkinson’s disease in mid-2017.
- For VY-AADC01, initiate a posterior (i.e., back of the head)
infusion trajectory. A posterior trajectory aligns the
infusion of VY-AADC01 with the anatomical structure of the putamen
and could result in a higher total volume of coverage of the
putamen. Data from this trial will also help inform the
design of the double-blind, placebo-controlled trial planned to
begin during the fourth quarter of 2017.
- Initiate a double-blind, placebo-controlled trial for VY-AADC01
for advanced Parkinson’s disease during the fourth quarter of
2017.
- Advance multiple preclinical programs towards clinical trials,
with the goal of filing three IND applications within the next
24-months for the VY-SOD101, VY-HTT01, and VY-FXN01 programs,
including an IND for VY-SOD101 during the fourth quarter of 2017.
- Identify, evaluate and progress collaborative opportunities for
certain unpartnered Voyager programs or our technology platform
capabilities.
- Based on the company’s current operating plan, Voyager expects
to end 2017 with cash, cash equivalents and marketable debt
securities of approximately $90 million to $100 million and
projects that its existing cash, cash equivalents and marketable
debt securities will be sufficient to fund operating expenses and
capital expenditure requirements into 2019.
Fourth Quarter and Full Year 2016
Financial Results
Voyager reported a GAAP net loss of $14.7
million, or $0.57 per share, for the fourth quarter ended December
31, 2016, compared to a GAAP net loss of $8.8 million, or $0.67 per
share, for the same period in 2015. The company reported a
net loss of $40.2 million, or $1.59 per share, for the full year
ended December 31, 2016, compared to a net loss of $38.3 million,
or $9.14 per share, for the same period in 2015.
Collaboration revenues of $2.4 million for the
fourth quarter of 2016 compared to collaboration revenues of $4.9
million for the fourth quarter of 2015. Collaboration
revenues of $14.2 million for the full year ended December 31, 2016
compared to collaboration revenues of $17.3 million for the full
year ended December 31, 2015. Collaboration revenues reflect
recognition of payment for research and development services
provided by Voyager for various programs under the Sanofi Genzyme
collaboration agreement. The decrease in collaboration
revenues for the fourth quarter and full year 2016 compared to the
same periods in 2015 reflect deprioritized development of VY-SMN101
for spinal muscular atrophy and a reduction of certain services
provided by Sanofi Genzyme.
Research and development (R&D) expenses of
$12.7 million for the fourth quarter ended December 31, 2016
compared to $9.2 million for the same period in 2015. R&D
expenses of $42.2 million for the year ended December 31, 2016
compared to $27.7 million for the same period in 2015. The increase
in R&D expenses was largely due to expenditures associated with
the development of Voyager’s pipeline and product engine, increased
facility expenses and personnel costs to support the advancement of
the pipeline programs.
General and administrative (G&A) expenses of
$3.5 million for the fourth quarter ended December 31, 2016
compared to $3.2 million for the same period in 2015. G&A
expenses of $13.3 million for the year ended December 31, 2016
compared to $9.9 million for the same period in 2015. The
increase in G&A expenses was primarily due to personnel costs
to support Voyager’s growth and facility costs.
Cash, cash equivalents, and marketable debt
securities as of December 31, 2016 were $174.4 million.
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
83618016. 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-AADC01
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 worldwide[2]. 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 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 ago[3]. 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 dopamine[4].
The intrinsic neurons in the putamen, however, do not degenerate in
Parkinson’s disease[5],[6]. VY-AADC01, 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-AADC01,
therefore, has the potential to durably enhance the conversion of
levodopa to dopamine and provide clinically meaningful improvements
in motor symptoms following a single administration.
About Voyager Therapeutics
Voyager Therapeutics is a clinical-stage gene
therapy company developing life-changing treatments for severe
diseases of the CNS. 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
CNS diseases in need of effective new therapies, including advanced
Parkinson’s disease, a monogenic form of ALS, Friedreich’s ataxia,
Huntington’s disease, 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 law. 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 Quarterly
Report on Form 10-Q filed with the Securities and Exchange
Commission, as updated by its future 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
estimates) |
(Unaudited) |
|
|
|
|
|
|
|
Three Months Ended |
|
Year Ended |
|
|
December 31, |
|
December 31, |
Statement of
Operations Items: |
|
2016 |
|
2015 |
|
2016 |
|
2015 |
Collaboration
revenue |
|
$ |
2,362 |
|
|
$ |
4,937 |
|
|
$ |
14,220 |
|
|
|
17,334 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
12,723 |
|
|
|
9,220 |
|
|
|
42,249 |
|
|
|
27,679 |
|
General
and administrative |
|
|
3,481 |
|
|
|
3,157 |
|
|
|
13,270 |
|
|
|
9,909 |
|
Total operating
expenses |
|
|
16,204 |
|
|
|
12,377 |
|
|
|
55,519 |
|
|
|
37,588 |
|
Operating loss |
|
|
(13,842 |
) |
|
|
(7,440 |
) |
|
|
(41,299 |
) |
|
|
(20,254 |
) |
Total other income
(expense) |
|
|
(476 |
) |
|
|
157 |
|
|
|
1,158 |
|
|
|
(9,418 |
) |
Loss before income
taxes |
|
|
(14,318 |
) |
|
|
(7,283 |
) |
|
|
(40,141 |
) |
|
|
(29,672 |
) |
Income tax expense |
|
|
355 |
|
|
|
— |
|
|
|
52 |
|
|
|
— |
|
Net loss |
|
|
(14,673 |
) |
|
|
(7,283 |
) |
|
|
(40,193 |
) |
|
|
(29,672 |
) |
GAAP charges related to
pre-IPO preferred stock |
|
|
— |
|
|
|
(1,534 |
) |
|
|
— |
|
|
|
(8,618 |
) |
Net loss attributable
to common stockholders |
|
$ |
(14,673 |
) |
|
$ |
(8,817 |
) |
|
$ |
(40,193 |
) |
|
|
(38,290 |
) |
Net loss per share
attributable to common stockholders, basic and diluted |
|
$ |
(0.57 |
) |
|
$ |
(0.67 |
) |
|
$ |
(1.59 |
) |
|
|
(9.14 |
) |
Weighted-average common
shares outstanding, basic and diluted |
|
|
25,526,843 |
|
|
|
13,178,922 |
|
|
|
25,302,414 |
|
|
|
4,191,210 |
|
|
|
December 31, |
Selected
Balance Sheet Items |
|
2016 |
|
2015 |
Cash, cash equivalents,
and marketable debt securities |
|
$ |
174,418 |
|
$ |
224,345 |
Total assets |
|
$ |
189,566 |
|
$ |
229,457 |
Accounts payable and
accrued expenses |
|
$ |
7,038 |
|
$ |
4,042 |
Deferred revenue |
|
$ |
41,582 |
|
$ |
54,982 |
Total stockholders’
equity |
|
$ |
135,922 |
|
$ |
169,074 |
|
|
|
|
|
|
|
[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
Investor Relations:
Matt Osborne
Head of Investor Relations & Corporate Communications
857-259-5353
mosborne@vygr.com
Media:
Josephine Butler
Pure Communications, Inc.
910-337-0707
jbutler@purecommunications.com
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