Voyager Therapeutics Transforms Pipeline and Increases Investment in Next-Generation TRACER™ AAV Capsid and Vectorized Antibody Platform Technologies
August 09 2021 - 7:00AM
Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company
developing life-changing treatments and next-generation platform
technologies, today introduced new programs in Huntington’s
disease, a monogenic form of ALS (SOD1), spinal muscular atrophy,
and diseases linked to GBA1 mutations, all powered by its
proprietary AAV capsids that have demonstrated superior transgene
expression in the brain compared to AAV9 delivery in non-human
primates. The Company also announced increased investment in its
RNA-driven TRACER (Tropism Redirection of AAV by Cell-type-specific
Expression of RNA) AAV screening technology to expand discovery of
novel capsids with broad tissue tropism in CNS, cardiac, and
skeletal tissues.
“Given recent breakthroughs with our platform technologies,
Voyager has transformed its pipeline to focus on advancing
innovative gene therapies that leverage our proprietary
next-generation AAV capsids and vectorized antibodies. In parallel,
we are bolstering investment in our TRACER platform to produce
additional targeted capsids with enhanced tissue- and
cellular-specificity and to expand the potential of AAV-mediated
genetic delivery of antibody therapies,” said Michael Higgins,
Interim CEO of Voyager. “Voyager’s capsids have achieved superior
transduction in targeted tissues over AAV9 in non-human primates,
and we strongly believe that our platform has opened up a wide
array of opportunities in CNS, as well as other tissues. Because
our novel capsids have significant potential to be more reliably
on-target with less risk of dose-limiting toxicities, we believe
they could enable a new generation of gene therapies developed
internally, as well as by collaborators or licensees.”
“After a rigorous and thoughtful evaluation of existing
programs, we have refocused the Company on delivery technologies to
power new and second-generation programs which we believe have
robust target validation, an efficient path to preclinical and
clinical proof of concept, and may provide meaningful therapeutic
benefit,” said Glenn Pierce, M.D., Ph.D., Interim CSO of Voyager.
“As part of the evaluation, we have discontinued our
first-generation Huntington’s disease program and initiated a
second-generation program using a novel, proprietary AAV capsid
that may enable intravenous administration and achieve widespread
distribution to affected tissue. We firmly believe that HD remains
an ideal target and that applying our clinical development
experience to introduce a new approach creates the opportunity to
confer greater patient benefit with a more favorable safety
profile. We are grateful to the HD community for supporting our
research and remain fully committed to advancing our work to serve
patients and those who care for them.”
Pipeline and Platform Updates
Pipeline Re-evaluation Yields High-Potential
Programs
- Voyager believes its proprietary AAV capsids may enable new,
best-in-class programs for spinal muscular atrophy and diseases
linked to GBA1 mutations given initial data demonstrating its
capsids are more reliably on-target with less risk of dose-limiting
toxicities than existing AAV capsids. Second-generation and new
pre-clinical programs have been initiated in the following areas:
- Huntington’s disease (second generation)
- Monogenic ALS (SOD1) (second generation)
- Spinal muscular atrophy
- Diseases linked to GBA1 mutations
- Internal development of first-generation, surgically-based gene
therapy programs for the treatment of Huntington’s disease and
monogenic ALS (SOD1) have been discontinued with efforts being
redirected to second-generation programs.
- The Company is exploring various indications across the
spectrum of GBA1 mutations, including Parkinson’s disease, Lewy
body dementia, and Gaucher disease, as well as vectorized
antibodies to explore undisclosed indications in
neuro-oncology.
- Voyager’s current program in Friedreich’s ataxia remains
partnered with Neurocrine Biosciences, Inc., and the Company’s
wholly-owned vectorized antibody program focused on various
tauopathies continues as planned.
TRACER AAV Capsid Discovery Platform Demonstrates
Superior CNS Targeting
- The TRACER platform is designed to identify novel capsids that
overcome the limitations of existing AAV capsids. The platform may
be flexibly applied to discover capsids with enhanced tropism to
target tissues, such as the CNS, and cardiac and skeletal muscle
that may lead to treatments for a broad range of diseases.
- Initial TRACER capsid data have demonstrated robust delivery
across the blood-brain barrier and widespread CNS transduction in
non-human primates compared to AAV9 delivery, following intravenous
(IV) administration. Capsid 9P801 displayed more than 1,000-fold
higher transgene expression in the brain compared to AAV9 delivery
in non-human primates.
- Capsid 9P804 displayed strong cardiac transduction and
significant dorsal root ganglia detargeting, which may avoid
toxicities associated with AAV9 delivery.
- Voyager is proceeding with screens of nine additional AAV
capsid campaigns to further expand its capsid library and identify
capsids optimized for specific applications.
Vectorized Anti-Tau Antibody Platform Demonstrates
Durable CNS Expression
- Initial preclinical data have demonstrated a reduction of
pathological tau with Voyager’s vectorized anti-tau antibody and
durable expression in CNS.
- The Company’s vectorized antibodies may represent a new
single-dose therapeutic strategy for treating various tauopathies,
including progressive supranuclear palsy and frontotemporal
dementia.
- Voyager has developed modular antibody vectorization cassettes,
which consist of an AAV vector and a transgene encoding anti-tau
monoclonal full-length antibodies. Following IV administration and
transduction of target cells in the brain, the expressed antibodies
are designed to be functionally reconstituted and subsequently
secreted into the parenchyma, thereby enabling potential
therapeutic benefit for multiple CNS diseases.
Corporate Updates and Anticipated
Milestones
Leadership Transitions
- In May, the Company announced the appointment of Michael
Higgins as interim chief executive officer and Glenn Pierce, M.D.,
Ph.D., as interim chief scientific officer. Mr. Higgins has served
on Voyager’s board of directors since July 2015 and has been chair
of the board since June 2019. He is also the chair of the board of
Pulmatrix and a board member for Genocea Biosciences, Nocion
Therapeutics, Camp4 Therapeutics, and Sea Pharmaceuticals. Dr.
Pierce has been a member of the board of directors since January
2017. He serves as entrepreneur-in-residence at Third Rock
Ventures, where he co-founded Ambys Medicines. Dr. Pierce
previously served in a number of roles at Biogen, most recently as
chief medical officer leading the hematology, cell, and gene
therapies division. He is the co-author of more than 150 scientific
papers and has received more than 15 patents.
Upcoming Events and Presentations
- Voyager plans to participate in the following investor events:
- Wedbush Pacgrow Healthcare Conference, August 11, 2021
- Canaccord Genuity Growth Conference, August 12, 2021
- Wells Fargo Virtual Healthcare Conference, September 9,
2021
- Morgan Stanley Global Healthcare Conference, September 14,
2021
- Baird Healthcare Conference, September 15, 2021
Second Quarter 2021 Financial Results
- Collaboration Revenues: Collaboration revenue
was $1.4 million for the second quarter of 2021, compared to
collaboration revenue of $28.7 million for the same period of 2020.
The decrease in collaboration revenue was largely due to a
reduction of revenue related to research services and cost
reimbursements from the collaborations with Neurocrine and AbbVie.
In February 2021, Neurocrine provided notice that, effective August
2, 2021, it was terminating its participation in the VY-AADC
program for Parkinson’s disease under the collaboration agreement
between the Company and Neurocrine, and that wind-down activities,
including the termination by Neurocrine of its support for on-going
development activities for the VY-AADC program would commence
immediately. Additionally, the collaborations with AbbVie were
terminated in August 2020.
- Net Loss: Net loss was $30.1 million for the
second quarter of 2021, compared to a net loss of $8.7 million for
the same period of 2020.
- R&D Expenses: Research and development
expenses were $19.5 million for the second quarter of 2021,
compared to $29.4 million for the same period in 2020. The decrease
in R&D expenses was primarily related to lower manufacturing
and clinical expenses for the VY-AADC program for Parkinson’s
disease.
- G&A Expenses: General and administrative
expenses were $10.4 million for the second quarter of 2021,
compared to $8.2 million for the same period in 2020. The increase
in G&A expenses was primarily related to the one-time
recognition of severance and stock-based compensation expense
related to a former key executive, and increased facility
costs.
- Cash Position: Cash, cash equivalents and
marketable debt securities as of June 30, 2021 were $143.0
million.
Financial Guidance
- Based on the Company’s current operating plan and excluding any
potential financing or business development activities in 2021,
Voyager anticipates cash, cash equivalents, and marketable debt
securities will be between $85 million and $90 million at the end
of 2021.
- Due in part to cost savings realized as a result of the
pipeline re-evaluation, Voyager expects that its cash, cash
equivalents and marketable debt securities will be sufficient to
meet the Company’s planned operating expenses and capital
expenditure requirements into early 2023.
About the TRACER™ AAV Capsid Discovery
PlatformVoyager’s TRACER™ system is a broadly-applicable,
RNA-based functional screening platform that allows for rapid in
vivo evolution of AAV capsids delivered intravenously with
cell-specific transduction properties in non-human primates (NHP).
Initial data from the first of many libraries screened in NHPs
demonstrated the proprietary capsid variants effectively penetrated
the blood-brain barrier and achieved widespread biodistribution and
transduction of multiple regions of the brain. Voyager is
proceeding with screens of additional NHP campaigns from AAV9 and
other capsid serotypes to enable the identification of novel AAV
vectors optimized for specific therapeutic applications. Initial
results have demonstrated the ability of certain capsids to
transduce cardiac tissue and to detarget the dorsal root
ganglia.
About Voyager TherapeuticsVoyager Therapeutics
(Nasdaq: VYGR) is leading the next generation of AAV gene therapy
to unlock the potential of the technology to treat devastating
diseases. Proprietary capsids born from the Company’s TRACERTM
screening platform are powering a rich early-stage pipeline of new
and second-generation programs and may elevate the field to
overcome the limitations of conventional gene therapy vectors
across neurological disorders and other therapeutic
areas. voyagertherapeutics.com
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Forward-Looking StatementsThis 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,” “would,” “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, activities, goals and reporting of
results of its research activities, preclinical and other
development programs and clinical trials; the timing of planned
presentations at medical, scientific or other conferences; its
ability to identify and attract parties to participate in research
and development collaborations; its ability to advance its
AAV-based gene therapies into, and successfully initiate, enroll
and complete, clinical trials; the potential clinical utility of
its product candidates; the continuing progress in Voyager’s
development of novel AAV capsids; its ability to continue to
develop its TRACER AAV capsid discovery platform; its ability to
perform under existing collaborations including the potential
benefits, timing and future operation of its collaboration with
Neurocrine Biosciences; its ability to add new programs to its
pipeline; the regulatory pathway of, and the timing or likelihood
of its regulatory filings and approvals for, any of the Company’s
product candidates; its ability to operate its research and
development activities efficiently and effectively; the utility and
value of Voyager’s patent portfolio; Voyager’s anticipated
financial results, including Voyager’s available cash, cash
equivalents and marketable debt securities and the receipt by
Voyager of revenues or reimbursement payments from collaboration
partners, Voyager’s operating expenses; and Voyager’s ability to
fund its operating expenses with its current cash, cash equivalents
and marketable debt securities though a stated time period are
forward looking.
All forward-looking statements are based on estimates and
assumptions by Voyager’s management that, although Voyager believes
such forward-looking statements 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. Such risks and uncertainties
include, among others, the severity and length of the COVID-19
health crisis, the imposition of governmental controls and guidance
addressing the COVID health crisis, and the financial and human
resources available to Voyager to manage the COVID-19 health
crisis; the initiation and conduct of research activities,
preclinical studies and clinical trials; the availability of data
from research activities, preclinical studies and clinical trials,
and the ability to effectively present such data; the expectations
for regulatory communications, submissions and approvals; the
continued development of various technology platforms, including
Voyager’s TRACER platform; Voyager’s scientific approach and
general development progress; the ability to attract and retain
talented contractors and employees; the ability to create and
protect intellectual property; the sufficiency of cash resources;
the possibility or the timing of the exercise of development,
commercialization, license and other options under collaborations;
the ability of Voyager to negotiate and complete licensing or
collaboration agreements on terms acceptable to Voyager and third
parties; and the availability or commercial potential of Voyager’s
product candidates. 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. All information in the
press release is as of the date of this press release, and any
forward-looking statement speaks only as of the date on which it
was made. Voyager undertakes no obligation to publicly update or
revise this information or any forward-looking statement, whether
as a result of new information, future events or otherwise, except
as required by law.
Investor
Inquiries: investors@voyagertherapeutics.com
Media
Inquiries: Scott
Santiamossantiamo@voyagertherapeutics.com
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: |
2021 |
|
2020 |
|
2021 |
|
2020 |
Collaboration revenue |
$ |
1,357 |
|
|
$ |
28,681 |
|
|
$ |
7,858 |
|
|
$ |
46,748 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
19,505 |
|
|
|
29,423 |
|
|
|
41,851 |
|
|
|
61,718 |
|
General and administrative |
|
10,437 |
|
|
|
8,239 |
|
|
|
20,181 |
|
|
|
18,444 |
|
Total operating expenses |
|
29,942 |
|
|
|
37,662 |
|
|
|
62,032 |
|
|
|
80,162 |
|
Operating loss |
|
(28,585 |
) |
|
|
(8,981 |
) |
|
|
(54,174 |
) |
|
|
(33,414 |
) |
Total other (expense)
income |
|
(1,535 |
) |
|
|
300 |
|
|
|
2,405 |
|
|
|
470 |
|
Net loss |
$ |
(30,120 |
) |
|
$ |
(8,681 |
) |
|
$ |
(51,769 |
) |
|
$ |
(32,944 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share, basic and
diluted |
$ |
(0.80 |
) |
|
$ |
(0.23 |
) |
|
$ |
(1.38 |
) |
|
$ |
(0.89 |
) |
Weighted-average common shares
outstanding, basic and diluted |
|
37,581,381 |
|
|
|
37,029,524 |
|
|
|
37,543,387 |
|
|
|
36,996,390 |
|
|
|
|
|
|
|
|
|
June 30, |
|
December 31, |
|
Selected Balance Sheet
Items |
2021 |
|
2020 |
|
Cash, cash equivalents, and
marketable debt securities |
$ |
142,981 |
|
$ |
174,782 |
|
Total assets |
$ |
208,727 |
|
$ |
261,584 |
|
Accounts payable and accrued
expenses |
$ |
11,749 |
|
$ |
14,839 |
|
Deferred revenue |
$ |
39,725 |
|
$ |
43,817 |
|
Total stockholders’
equity |
$ |
110,536 |
|
$ |
154,320 |
|
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