Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company
dedicated to advancing neurogenetic medicines, today reported first
quarter 2024 financial and operating results.
“We have obtained IND clearance for our anti-tau antibody
VY-TAU01 for Alzheimer’s disease, and we expect to dose the first
subject in our single ascending dose trial in healthy volunteers in
the coming weeks,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief
Executive Officer of Voyager. “Our gene therapy pipeline is also
advancing, with development candidates selected in the GBA1 and
Friedreich’s Ataxia programs partnered with Neurocrine, as well as
in our wholly-owned SOD1-ALS program. We expect to achieve IND
filings for all three of these gene therapy programs in 2025. We
maintain a strong cash position of approximately $400 million at
quarter-end, with runway into 2027, which we anticipate will enable
us to reach multiple data readouts in 2025 and 2026.”
First Quarter 2024 and Recent Highlights
- Obtained IND clearance for
VY-TAU01 for Alzheimer’s disease: Received clearance of
the Investigational New Drug (IND) application filed with the U.S.
Food and Drug Administration (FDA) for VY-TAU01, an anti-tau
antibody for the treatment of Alzheimer’s disease.
- Development candidate
selected in GBA1 gene therapy program: Announced that the
joint steering committee with collaborator Neurocrine Biosciences
selected a lead development candidate for the GBA1 gene therapy
program for the treatment of Parkinson’s disease and other
GBA1-mediated diseases, triggering a $3 million milestone payment
to Voyager.
- Development candidate
selected in FXN gene therapy program: Announced that the
joint steering committee with collaborator Neurocrine Biosciences
selected a lead development candidate for the FXN gene therapy
program for the treatment of Friedreich’s Ataxia, triggering a $5
million milestone payment to Voyager.
- Toby Ferguson, M.D., Ph.D.,
appointed as Chief Medical Officer: Dr. Ferguson is an
experienced biotechnology executive with a proven record in
advancing portfolios of novel therapies across diverse indications
in central nervous system (CNS), neuromuscular, and rare
diseases.
- Strategic collaboration and
capsid license agreement with Novartis: Announced a
strategic collaboration and capsid license agreement with Novartis
Pharma AG to advance potential gene therapies for Huntington’s
disease (HD) and spinal muscular atrophy (SMA). Novartis paid
Voyager $80 million of consideration up front and $20 million for
the purchase of newly issued equity in Voyager. Voyager is eligible
to receive up to $1.2 billion in preclinical, development,
regulatory and sales milestones, as well as tiered royalties on
global net sales of products incorporating Voyager’s TRACER™
capsids.
- Completion of public
offering: Completed an underwritten public offering of
shares of common stock (or pre-funded warrants to purchase common
stock in lieu thereof) for aggregate gross proceeds of
approximately $100 million.
- Presented data at AD/PD 2024
and ASGCT 2024:
- 2024 International Conference on
Alzheimer’s and Parkinson’s Diseases and Related Neurological
Disorders (AD/PD™ 2024): Presented data demonstrating robust
reductions in human tau messenger RNA (mRNA) and protein across the
brain following a single intravenous (IV) administration of a tau
silencing gene therapy candidate in mice expressing human tau. Also
presented data demonstrating that VY-TAU01 was well-tolerated
following IV administration in non-human primates (NHPs).
- American Society of Gene & Cell
Therapy’s (ASGCT) 27th annual meeting: Presented a broad set of
translational data, including new data demonstrating enhanced brain
tropism of second-generation TRACER-derived AAV capsids,
translatability as evidenced by cross-species and receptor data;
and activity against therapeutic targets in Alzheimer’s disease and
ALS.
Anticipated Upcoming Milestones
- VY-TAU01 anti-tau antibody
for Alzheimer’s disease: Voyager expects to dose the first
subject in a Phase 1a single ascending dose trial in healthy
volunteers in the coming weeks and initiate a Phase 1b multiple
ascending dose trial in patients with early Alzheimer’s disease in
2025. The multiple ascending dose trial has the potential to
generate initial data for slowing the spread of pathological tau
via tau PET imaging in 2026.
- VY9323 SOD1
silencing gene therapy program for ALS: Voyager expects to
file an IND in mid-2025 and initiate a Phase 1 clinical trial in
ALS patients. The Phase 1 trial has the potential to generate
proof-of-concept data based on biomarkers.
- Partnered programs:
Voyager and Neurocrine Biosciences expect to file INDs for the GBA1
and Friedreich’s Ataxia gene therapy programs in 2025.
First Quarter 2024 Financial Results
- Collaboration
Revenues: Voyager had collaboration revenue of $19.5
million for the first quarter of 2024, compared to $150.5 million
for the same period in 2023. The decrease was primarily due to the
first quarter 2023 recognition of $69.5 million from the 2023
Neurocrine Collaboration Agreement and $79.0 million from Novartis’
exercise of two capsid options.
- Net (Loss) Income:
Net loss was $11.3 million for the first quarter of 2024, compared
to net income of $124.0 million for the same period in 2023. The
decrease is primarily due to reduced collaboration revenue
recognized in the first quarter of 2024, as discussed above.
- R&D Expenses:
Research and development expenses were $27.1 million for the first
quarter of 2024, compared to $18.6 million for the same period in
2023. The increase in R&D expenses was primarily a result
of increased program-related spending, particularly manufacturing
and IND-enabling studies for the VY-TAU01 anti-tau antibody
program, the VY9323 SOD1-ALS gene therapy program, and the
initiation of spend on the Novartis HD program during the first
quarter of 2024, along with increased headcount costs.
- G&A Expenses:
General and administrative expenses were $8.6 million for the first
quarter of 2024, compared to $9.0 million for the same period in
2023.
- Cash Position:
Cash, cash equivalents and marketable securities as of March 31,
2024, were $400.5 million.
Financial Guidance
Voyager is committed to maintaining a strong balance sheet that
supports the advancement and growth of its platform and pipeline.
Voyager continues to assess its planned cash needs both during the
current period and in future periods. We expect our cash, cash
equivalents, and marketable securities, along with amounts expected
to be received as reimbursement for development costs under the
Neurocrine and Novartis collaborations, certain near-term
milestones, and interest income, to be sufficient to meet Voyager’s
planned operating expenses and capital expenditure requirements
into 2027.
Conference Call
Voyager will host a conference call and webcast today at 4:30
p.m. ET to discuss first quarter 2024 financial and operating
results. To participate via telephone and join the call live,
please register in advance here:
https://register.vevent.com/register/BI1f6af80e7a614ca7925cbad2f35a55c6.
Upon registration, telephone participants will receive a
confirmation email detailing how to join the conference call,
including the dial-in number and a unique passcode. A live webcast
of the call will also be available on the Investors section of the
Voyager website at ir.voyagertherapeutics.com, and a replay of the
call will be available at the same link approximately two hours
after its completion. The replay will be available for at least 30
days following the conclusion of the call.
About the TRACER™ Capsid Discovery Platform
Voyager’s TRACER™ (Tropism Redirection of AAV by
Cell-type-specific Expression of RNA) capsid discovery platform is
a broadly applicable, RNA-based screening platform that enables
rapid discovery of novel AAV capsids to enable gene therapy.
Voyager has leveraged TRACER to create multiple families of novel
capsids that, following intravenous delivery in preclinical
studies, harness the extensive vasculature of the central nervous
system (CNS) to cross the blood-brain barrier and transduce a broad
range of CNS regions and cell types. In cross-species preclinical
studies (rodents and multiple non-human primate species),
intravenous delivery of TRACER-generated capsids resulted in
widespread payload expression across the CNS at relatively low
doses, enabling selection of multiple development candidates in
Voyager’s wholly-owned and partnered gene therapy programs for
neurologic diseases.
About Voyager Therapeutics
Voyager Therapeutics, Inc. (Nasdaq: VYGR) is a biotechnology
company dedicated to leveraging the power of human genetics to
modify the course of – and ultimately cure – neurological diseases.
Our pipeline includes programs for Alzheimer’s disease, amyotrophic
lateral sclerosis (ALS), Parkinson’s disease, and multiple other
diseases of the central nervous system. Many of our programs are
derived from our TRACER™ AAV capsid discovery platform, which we
have used to generate novel capsids and identify associated
receptors to potentially enable high brain penetration with genetic
medicines following intravenous dosing. Some of our programs are
wholly owned, and some are advancing with partners including
Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; Neurocrine
Biosciences, Inc.; and Sangamo Therapeutics, Inc. For more
information, visit www.voyagertherapeutics.com.
Voyager Therapeutics® is a registered trademark, and TRACER™ is
a trademark, of Voyager Therapeutics, Inc.
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 “expect,” “will,” “believe,” “anticipate,”
“potential,” “trigger” or “continue,” and other similar expressions
are intended to identify forward-looking statements.
For example, all statements Voyager makes regarding Voyager’s
ability to advance its AAV-based gene therapy programs and tau
antibody program, including expectations for Voyager’s achievement
of preclinical and clinical development milestones for its
potential development candidates such as IND filings, the
initiation of clinical trials, and generation of clinical data and
proof-of-concept; Voyager’s ability to advance gene therapy product
candidates under the Neurocrine and Novartis collaborations;
Voyager’s anticipated financial results, including the anticipated
receipt by Voyager of revenues or reimbursement payments from
collaboration partners; and Voyager’s cash runway and ability to
generate sufficient cash resources to enable it to continue its
business and operations 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 expectations and decisions of regulatory
authorities; the timing, initiation, conduct and outcomes of
Voyager’s preclinical and clinical studies; the availability of
data from clinical trials; the availability or commercial potential
of product candidates under collaborations; the willingness and
ability of Voyager's collaboration partners to meet obligations
under collaboration agreements with Voyager; the continued
development of Voyager’s technology platforms, including Voyager’s
TRACER platform and its antibody screening technology; Voyager’s
scientific approach and program development progress, and the
restricted supply of critical research components; the development
by third parties of capsid identification platforms that may be
competitive to Voyager’s TRACER capsid discovery platform;
Voyager’s ability to create and protect intellectual property
rights associated with the TRACER capsid discovery platform, the
capsids identified by the platform, and development candidates for
Voyager’s pipeline programs; the possibility or the timing of
Voyager’s receipt of program reimbursement, development or
commercialization milestones, option exercise, and other payments
under Voyager’s existing licensing or collaboration agreements; the
ability of Voyager to negotiate and complete licensing or
collaboration agreements with other parties on terms acceptable to
Voyager and the third parties; the success of programs controlled
by third party collaboration partners in which Voyager retains a
financial interest, and the success of Voyager’s product
candidates; the ability to attract and retain talented directors,
employees, and contractors; and the sufficiency of cash resources
to fund its operations and pursue its corporate objectives.
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. 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.
Contacts
Trista Morrison, NACD.DC, tmorrison@vygr.com Investors: Adam
Bero, Ph.D., abero@kendallir.com Media: Brooke Shenkin,
brooke@scientpr.com
Selected Financial Information ($ amounts in
thousands, except per share data) (Unaudited) |
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
|
March 31, |
|
Statement of
Operations Items: |
|
2024 |
|
|
2023 |
|
Collaboration revenue |
|
$ |
19,516 |
|
|
$ |
150,480 |
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development |
|
|
27,092 |
|
|
|
18,568 |
|
General and administrative |
|
|
8,607 |
|
|
|
9,028 |
|
Total operating expenses |
|
|
35,699 |
|
|
|
27,596 |
|
Operating (loss) income |
|
|
(16,183 |
) |
|
|
122,884 |
|
Total other income |
|
|
4,867 |
|
|
|
1,864 |
|
(Loss) income before income
taxes |
|
|
(11,316 |
) |
|
|
124,748 |
|
Income tax provision |
|
|
14 |
|
|
|
704 |
|
Net (loss) income |
|
$ |
(11,330 |
) |
|
$ |
124,044 |
|
Net (loss) income per share,
basic |
|
$ |
(0.20 |
) |
|
$ |
3.05 |
|
Net (loss) income per share,
diluted |
|
$ |
(0.20 |
) |
|
$ |
2.94 |
|
Weighted-average common shares
outstanding, basic |
|
|
57,117,046 |
|
|
|
40,632,087 |
|
Weighted-average common shares
outstanding, diluted |
|
|
57,117,046 |
|
|
|
42,161,326 |
|
|
|
|
|
|
|
|
|
|
|
March 31, |
|
December 31, |
|
Selected Balance Sheet
Items |
|
2024 |
|
2023 |
|
Cash, cash equivalents, and
marketable securities |
|
$ |
400,548 |
|
$ |
230,875 |
|
Total assets |
|
$ |
469,592 |
|
$ |
351,281 |
|
Accounts payable and accrued
expenses |
|
$ |
15,174 |
|
$ |
18,427 |
|
Deferred revenue |
|
$ |
64,596 |
|
$ |
75,240 |
|
Total stockholders’
equity |
|
$ |
340,886 |
|
$ |
236,320 |
|
GAAP vs. Non-GAAP Financial MeasuresVoyager’s
financial statements are prepared in accordance with generally
accepted accounting principles in the United States, or GAAP, and
represent revenue and expenses as reported to the Securities and
Exchange Commission. Voyager has provided in this release certain
financial information that has not been prepared in accordance with
GAAP, including net collaboration revenue and net research and
development expenses, which exclude the impact of reimbursement by
Neurocrine Biosciences (Neurocrine) for expenses we incur in
conducting preclinical development activities under our
collaboration agreements. Management uses these non-GAAP measures
to evaluate the Company’s operating performance in a manner that
allows for meaningful period-to-period comparison and analysis of
trends in its business. Management believes that such non-GAAP
measures are important in comparing current results with prior
period results and are useful to investors and financial analysts
in assessing the Company’s operating performance. Non-GAAP
financial measures are not required to be uniformly applied, are
not audited and should not be considered in isolation. The non-GAAP
measures give investors and financial analysts a better
understanding of our net revenue and net research and development
expenses without the pass-through impact of Neurocrine costs. The
non-GAAP financial information presented here should be considered
in conjunction with, and not as a substitute for, the financial
information presented in accordance with GAAP. Investors are
encouraged to review the reconciliation of these non-GAAP measures
to their most directly comparable GAAP financial measures set forth
below.
|
|
|
|
|
|
|
|
Reconciliation of GAAP to Non-GAAP Measures |
(in thousands) |
|
|
Three Months Ended |
|
|
|
March 31, |
|
|
|
2024 |
|
2023 |
|
GAAP collaboration
revenue |
|
$ |
19,516 |
|
$ |
150,480 |
|
Revenue recognized for reimbursed research and development services
(Note 1) |
|
$ |
3,178 |
|
$ |
328 |
|
Net collaboration revenue |
|
$ |
16,338 |
|
$ |
150,152 |
|
|
|
|
|
|
|
|
|
GAAP total research and
development expenses |
|
$ |
27,092 |
|
$ |
18,568 |
|
Expenses incurred for reimbursed research and development services
(Note 1) |
|
$ |
3,178 |
|
$ |
328 |
|
Net research and development
expenses |
|
$ |
23,914 |
|
$ |
18,240 |
|
Note 1: Under the Company's existing collaboration agreements
with Neurocrine and Novartis, Neurocrine and Novartis have agreed
to be responsible for all costs the Company incurs in conducting
preclinical development activities for certain collaboration
programs, in accordance with joint steering committee agreed upon
workplans and budgets. Reimbursable research and development
services performed during the period are captured within
collaboration revenue and research and development expenses in the
Company's consolidated statements of operations. During the three
months ended March 31, 2024, we incurred $3.2 million of
reimbursable research and development services recorded within
collaboration revenue and research and development expenses. During
the three months ended March 31, 2023, we incurred $0.3 million of
reimbursable research and development services recorded within
collaboration revenue and research and development expenses.
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