Voyager Therapeutics Reports Third Quarter 2021 Financial and Operating Results
November 02 2021 - 7:00AM
Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company
developing life-changing treatments and next-generation
adeno-associated virus (AAV) technologies, today reported third
quarter 2021 financial and operating results.
“We’ve taken notable steps in recent months to execute on our
new strategy, including our recent capsid licensing agreement with
Pfizer, continued progress with our RNA-driven AAV TRACER screening
platform, and initial data from our GBA1 gene replacement program,”
said Michael Higgins, interim CEO of Voyager. “The licensing
transaction with Pfizer showcases TRACER’s ability to produce not
only enhanced blood-brain barrier penetrant capsids, but also novel
capsids with enhanced tropism for cardiac muscle, offering promise
to unlock the fullest potential of gene therapies for a wide array
of diseases. We’ve retained all rights to our capsid library
outside of their use with two particular Pfizer transgenes, and we
believe there is significant opportunity for similar transactions
leveraging the entirety of our libraries for other targets inside
and outside of the CNS.”
“We’re pleased to also announce that in addition to our prior
results with AAV9 variants, our TRACER screening campaigns have
identified AAV5 capsid variants with enhanced brain and spinal cord
tropism, compared to conventional AAV5 and AAV9, as well as a new
class of AAV9 variants selective for glial cells, which may enable
more precise targeting of CNS diseases affecting non-neuronal
cells,” said Glenn Pierce, M.D., Ph.D., interim CSO of Voyager. “In
parallel, we’re advancing an innovative gene therapy pipeline
leveraging our novel TRACER capsids. Preclinical data from our GBA1
gene replacement program illustrates the potential of a
single-dose, IV-administered therapy to significantly raise target
protein levels in the CNS, potentially impacting Parkinson’s
disease, Lewy body dementia, and Gaucher disease.”
Pfizer License Option Agreement Supports TRACER Platform
Potential for Multiple Therapeutic Areas
- In October, Voyager and Pfizer Inc. (NYSE: PFE) entered an
agreement through which Pfizer may exercise options to license
novel capsids generated from Voyager’s RNA-driven TRACER (Tropism
Redirection of AAV by Cell-type-specific Expression of RNA)
screening technology. Under the agreement, Pfizer may evaluate
novel TRACER capsids and exercise options to license up to two
capsids for transgene-exclusive Pfizer gene therapy programs for
neurologic and cardiovascular disease associated with those
transgenes.
- The Pfizer transgenes are distinct from those planned for
Voyager’s internal pipeline, and Voyager retains global rights to
all TRACER capsids for use with other transgenes and to all other
applications of its TRACER technology.
- Voyager received a $30 million upfront payment from Pfizer and
is entitled to receive up to $20 million in exercise fees in
aggregate for two options, exercisable by Pfizer within 12 months
of signing. Voyager is also eligible to earn up to $580 million in
total development, regulatory, and commercial milestones associated
with licensed products incorporating the two undisclosed Pfizer
transgenes together with a Voyager licensed capsid. Voyager is also
eligible to receive mid- to high-single-digit tiered royalties
based on net sales of Pfizer’s products incorporating the licensed
capsids.
TRACER Identifies AAV9 and AAV5 Capsid Variants with
Broad CNS Tropism, Neuronal and Glial Cell Targeting; Screening
Campaigns Advancing
- Voyager is performing further screening with its TRACER
platform to identify additional proprietary AAV9- and AAV5-derived
capsids targeting multiple tissue and cell types for use in gene
therapies to treat a broad range of diseases.
- Numerous promising capsids have been identified from these
screens, which are undergoing rigorous testing across multiple
non-human primate and rodent species to evaluate their clinical
translatability.
- In addition to capsids with substantially enhanced CNS tropism
for neurons in non-human primates, a new array of TRACER AAV9 and
AAV5 variants show robust CNS targeting in both non-human primates
and rodents and demonstrate a strong tropism for glial cells, which
may enable more effective targeting of certain CNS diseases.
- Capsid candidates derived from AAV5 present significant
potential advantages in terms of lower prevalence of preexisting
neutralizing antibodies in the general population and
manufacturing. In addition, these variants demonstrate
cross-species translatability among primates and rodents. Initial
testing on these AAV5-derived capsids has demonstrated
approximately 20-fold improvements in brain transduction compared
with AAV9 in non-human primates.
- As previously disclosed, initial data from AAV9-derived TRACER
capsids demonstrated robust delivery across the blood-brain barrier
and widespread CNS transduction in non-human primates compared to
AAV9 delivery following intravenous (IV) administration. In
addition, a proprietary capsid displayed strong cardiac
transduction and significant dorsal root ganglia detargeting in
non-human primates, which may avoid dose-related toxicities
associated with AAV9 delivery.
Positive Preclinical Data from GBA1 Program
- Voyager continues to advance its early-stage programs including
multiple programs utilizing proprietary AAV capsids derived from
the TRACER platform. Voyager believes these capsids may enable new,
best-in-class gene therapy programs with systemic IV delivery and
lower risk of dose-limiting toxicities. Pipeline programs include
those for Huntington’s disease, monogenic ALS (SOD1), spinal
muscular atrophy, and diseases linked to GBA1 mutations, including
Parkinson’s disease, Lewy body dementia, and Gaucher disease.
- Voyager presented initial preclinical data from its GBA1 gene
replacement program at the virtual 2021 Annual Congress of the
European Society of Gene & Cell Therapy, Oct. 19-22, 2021. The
results demonstrated that an IV-administered gene replacement
therapy using a proprietary AAV capsid achieved widespread
distribution in the CNS and peripheral tissues, and raised levels
of the deficient GCase enzyme by 300% - 660% over endogenous levels
in a mouse model. By comparison, increases of 30% - 50% are
expected to be clinically impactful. These data support the
potential of a novel therapeutic approach for diseases associated
with GBA mutations, including Parkinson’s disease, Lewy body
dementia, and Gaucher disease.
Upcoming Events and Presentations
- Society for Neuro-Oncology 26th Annual Meeting, Nov. 18-21,
2021
Third Quarter 2021 Financial Results
- Collaboration Revenues: Collaboration revenue
was $1.5 million for the third quarter of 2021, compared to
collaboration revenue of $117.8 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
Voyager and Neurocrine, and that wind-down activities, including
the termination by Neurocrine of its support for ongoing
development activities for the VY-AADC program, would commence
immediately. Additionally, the collaborations with AbbVie were
terminated in August 2020 and resulted in recognition of $47.2
million of revenue related to the collaboration targeting
tauopathies and $59.4 million of revenue related to the
collaboration targeting synucleinopathies during the quarter ended
September 30, 2020, as Voyager had no further service obligations
at that time.
- Net Loss: Net loss was $25.1 million for the
third quarter of 2021, compared to net income of $85.6 million for
the same period of 2020.
- R&D Expenses: Research and development
expenses were $17.9 million for the third quarter of 2021, compared
to $25.0 million for the same period in 2020. The decrease in
R&D expenses was primarily related to lower research and
development employee-related expenses as well as manufacturing and
clinical expenses for the VY-AADC program for Parkinson’s
disease.
- G&A Expenses: General and administrative
expenses were $8.7 million for the third quarter of 2021, compared
to $8.3 million for the same period in 2020. The increase in
G&A expenses was primarily related to increased
employee-related expenses in connection with a reduction in force
and facility costs.
- Cash Position: Cash, cash equivalents, and
marketable debt securities as of September 30, 2021 were $121.5
million.
Financial Guidance
- Based on Voyager’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 greater than $130 million at the end of
2021.
About the TRACER™ AAV Capsid
Discovery Platform Voyager’s TRACER™ system is a broadly
applicable, RNA-based functional screening platform that allows for
rapid in vivo evolution of AAV capsids with enhanced tropisms and
cell- and tissue-specific transduction properties in multiple
species, including non-human primates (NHPs). 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. Separate results have demonstrated
the ability of certain capsids to transduce cardiac muscle and to
de-target the dorsal root ganglia. Voyager is proceeding with
additional capsid campaigns derived from unique capsid serotypes to
identify novel AAV vectors optimized for specific therapeutic
applications.
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
TRACER™ 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 neurologic disorders and other therapeutic areas.
voyagertherapeutics.com
LinkedIn Twitter
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 Voyager’s
ability to continue to identify and develop proprietary capsids
from its TRACER AAV screening platform; Voyager’s ability to
identify and develop proprietary capsids from its TRACER AAV
screening platform with increased transgene expression, increased
blood-brain barrier penetration and increased biodistribution
compared to conventional AAV5 and AAV9 capsids; Voyager’s ability
to utilize its novel proprietary capsids in its own product
development programs; Voyager’s ability to attract parties to
license its novel proprietary capsids or to participate with
Voyager in research and development collaborations utilizing its
novel proprietary capsid; Voyager’s ability to advance its
AAV-based gene therapy programs; Voyager’s ability to perform its
obligations under its license option agreement with Pfizer;
Voyager’s entitlement to receive upfront, milestone and royalty
based fees from Pfizer under the license option agreement;
Voyager’s ability to maintain its current partnerships and
collaborations and to enter into new partnerships or
collaborations; and Voyager’s ability to generate sufficient cash
resources to enable it to continue to identify and develop
proprietary capsids from its TRACER AAV screening platform 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 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, including key scientists and
business leaders; 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 the Pfizer license option agreement and
other 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.
Voyager
ContactsInvestorsInvestors@voyagertherapeutics.comMediaScott
Santiamo ssantiamo@vygr.com
Selected Financial
Information($-amounts in thousands, except per share
data)(Unaudited)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
Nine Months Ended |
|
|
|
September 30, |
|
September 30, |
|
Statement of
Operations Items: |
|
2021 |
|
|
2020 |
|
2021 |
|
|
2020 |
|
Collaboration revenue |
|
$ |
1,482 |
|
|
$ |
117,843 |
|
$ |
9,342 |
|
|
$ |
164,591 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
17,914 |
|
|
|
25,039 |
|
|
59,767 |
|
|
|
86,757 |
|
General and administrative |
|
|
8,714 |
|
|
|
8,277 |
|
|
28,895 |
|
|
|
26,721 |
|
Total operating expenses |
|
|
26,628 |
|
|
|
33,316 |
|
|
88,662 |
|
|
|
113,478 |
|
Operating (loss) income |
|
|
(25,146 |
) |
|
|
84,527 |
|
|
(79,320 |
) |
|
|
51,113 |
|
Total other income |
|
|
9 |
|
|
|
1,084 |
|
|
2,414 |
|
|
|
1,554 |
|
Net (loss) income |
|
$ |
(25,137 |
) |
|
$ |
85,611 |
|
$ |
(76,906 |
) |
|
$ |
52,667 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net (loss) income per share,
basic |
|
$ |
(0.67 |
) |
|
$ |
2.30 |
|
$ |
(2.04 |
) |
|
$ |
1.42 |
|
Net (loss) income per share,
diluted |
|
$ |
(0.67 |
) |
|
$ |
2.27 |
|
$ |
(2.04 |
) |
|
$ |
1.40 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Weighted-average common shares
outstanding, basic |
|
|
37,780,547 |
|
|
|
37,242,504 |
|
|
37,623,309 |
|
|
|
37,079,242 |
|
Weighted-average common shares
outstanding, diluted |
|
|
37,780,547 |
|
|
|
37,672,328 |
|
|
37,623,309 |
|
|
|
37,500,155 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30, |
|
December 31, |
|
Selected Balance Sheet
Items |
|
2021 |
|
2020 |
|
Cash, cash equivalents, and
marketable debt securities |
|
$ |
121,499 |
|
$ |
174,782 |
|
Total assets |
|
$ |
184,774 |
|
$ |
261,584 |
|
Accounts payable and accrued
expenses |
|
$ |
11,916 |
|
$ |
14,839 |
|
Deferred revenue |
|
$ |
39,176 |
|
$ |
43,817 |
|
Total stockholders’
equity |
|
$ |
87,422 |
|
$ |
154,320 |
|
Voyager Therapeutics (NASDAQ:VYGR)
Historical Stock Chart
From Aug 2024 to Sep 2024
Voyager Therapeutics (NASDAQ:VYGR)
Historical Stock Chart
From Sep 2023 to Sep 2024