VY-AADC for advanced Parkinson’s disease on track
for start of planned global, pivotal Phase 2-3 program during the
middle of this year
Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene
therapy company focused on developing life-changing treatments for
severe neurological diseases today reported its fourth quarter and
full year 2017 financial results, provided corporate highlights,
goals and financial guidance and will host a conference call and
webcast today at 8:00 a.m. ET to discuss these results.
“Our strong performance in 2017 was underscored
by advancing our lead program VY-AADC for advanced Parkinson’s
disease including successfully manufacturing our gene therapy
product candidate using our baculovirus/Sf9 cell process and
commercial-scale GMP runs, completing our dose-ranging Phase
1b clinical trial as well as our posterior trajectory trial to
select both an optimal dose and delivery protocol for the planned
pivotal trial, and reporting longer-term Phase 1b data with
clinically meaningful and durable responses with this one-time
treatment,” said Steven Paul, M.D., president and chief executive
officer of Voyager Therapeutics. “This performance sets us up to
initiate a planned pivotal program in the middle of this year,
moving this potential treatment closer to the hundreds of thousands
of advanced Parkinson’s disease patients who require better control
of their motor function in order to improve their quality of life.
Our preclinical pipeline targeting other severe neurological
diseases continues to advance towards the clinic and with our
recently announced collaboration with AbbVie, we continue to
successfully leverage our gene therapy platform and expertise
towards an exciting new area of vectorized immunotherapy initially
to deliver monoclonal antibodies directed against tau for the
treatment of Alzheimer’s disease and related neurodegenerative
diseases.”
2017 and Recent Key Pipeline and
Corporate Highlights
- Announced updated results to the ongoing, dose-ranging Phase 1b
program of VY-AADC for advanced Parkinson’s disease. At our
mid-dose studied in Cohort 2 at 18 months, patients had a mean
increase of approximately five hours a day of on-time without any
dyskinesia and experienced 65% less off-time, making this the
likely dose for the pivotal Phase 2-3 program.
- Investigational New Drug (IND) application cleared by the Food
and Drug Administration (FDA) for VY-AADC in January 2018, allowing
the Company to formally initiate clinical trial sites and screen
and begin dosing patients for its pivotal Phase 2-3 program for
advanced Parkinson’s disease. As part of this IND, the chemistry,
manufacturing, and controls (CMC) section included data
demonstrating comparability between VY-AADC produced under good
manufacturing process (GMP) using Voyager’s baculovirus/Sf9
manufacturing process and VY-AADC produced using a mammalian cell
system consisting of triple-transfection of human embryonic kidney
(HEK293) cells similar to that used in Voyager’s Phase 1b clinical
trial.
- Initiated a separate Phase 1 trial and successfully dosed seven
patients with advanced Parkinson’s disease with a posterior (i.e.,
back of the head) infusion trajectory of VY-AADC. A posterior
trajectory better aligns the infusion of VY-AADC with the
anatomical structure of the putamen and resulted in higher total
volume of coverage of the putamen and shorter total procedure time
compared to Cohorts 1 through 3 from the ongoing Phase 1b trial
that employed a transfrontal, or top of the head, delivery approach
into the putamen.
- In February 2018, FDA granted VY-AADC Fast Track designation
for advanced Parkinson’s disease. Fast Track designation is granted
for programs that demonstrate the potential to address an unmet
medical need for a serious or life-threatening disease and is
intended to facilitate development and expedited regulatory review,
including the ability to submit completed sections of a Biologics
License Application for a “rolling” review or
submission.
- Gained worldwide development and commercial rights to VY-AADC
for the treatment of advanced Parkinson’s disease. Voyager is on
track to dose the first patient in its planned global, pivotal
Phase 2-3 program during mid-2018.
- Entered into an exclusive strategic collaboration and option
agreement with AbbVie to develop and commercialize vectorized
antibodies directed against tau for the treatment of Alzheimer’s
disease and other neurodegenerative diseases, combining Voyager’s
gene therapy platform with AbbVie’s monoclonal antibody expertise,
global clinical development and commercial capabilities. The
research period is underway for each company to identify up to five
antibodies for inclusion in the collaboration. Voyager received $69
million upfront cash payment and is eligible to receive potentially
up to $155 million in preclinical and Phase 1 option payments as
well as development and regulatory milestone payments and
royalties.
- Further strengthened the balance sheet with approximately $127
million of additional capital raised from the upfront cash payment
from the AbbVie collaboration and a follow-on public equity
offering in November, extending Voyager’s expected ability to fund
its operating expenses and capital expenditure requirements into
early 2020.
- Announced plans during 2018 for Steven Paul, M.D., to
transition from president and chief executive officer to executive
science advisor where he will focus on preclinical discovery
research and portfolio development including progressing the AbbVie
collaboration. In addition to the executive science advisor
position, Dr. Paul will continue to serve on Voyager’s Board of
Directors, and as a member of Voyager’s Science & Technology
Committee.
- Strengthened the management team with the additions of Matthew
P. Ottmer as Chief Operating Officer and Luis Maranga, Ph.D. as
Chief Technical Operations Officer. Mr. Ottmer brings to Voyager
more than 18 years of biotechnology industry experience, including
executive leadership of business operations, product development,
and commercialization across multiple therapeutic areas and all
stages of development. Dr. Maranga joins Voyager with over 20 years
of biotechnology industry experience, focused on bioprocess
development, CMC, GMP, process validation and regulatory
submissions, and facilities management including extensive work
with the baculovirus/Sf9 expression system.
Corporate Goals and 2018 Financial
Guidance
Voyager is committed to becoming the leading
gene therapy company focused on severe neurological diseases with
expertise in discovery, development, manufacturing and
commercialization of gene therapy products for people living with
these devastating diseases. During 2018, Voyager plans to achieve
the following corporate goals towards fulfilling this
commitment:
- During the second quarter of this year, complete a Type C
meeting with the Office of Tissues and Advanced Therapies division
of the FDA’s Center for Biologics Evaluation and Research and
incorporate feedback from this meeting into the Phase 2-3 pivotal
program for VY-AADC for advanced Parkinson’s disease.
- During the second quarter of this year, provide six-month
safety and motor function data from the Phase 1 trial of VY-AADC
using the posterior infusion trajectory. Seven patients have been
dosed using this trajectory as the likely preferred surgical
approach for the Phase 2-3 pivotal program.
- During mid-2018, dose the first patient in the planned Phase
2-3 pivotal program for advanced Parkinson’s disease.
Neurosurgical and neurology clinical trial sites have been
identified and are being activated through institutional review
board processes, after which, patient referral, screening and
dosing can proceed.
- During the second half of 2018, provide longer-term safety,
biomarker, motor function and quality of life data from Cohorts 1-3
and from patients in the posterior trajectory trial of VY-AADC for
advanced Parkinson’s disease.
- Advance multiple preclinical programs towards clinical trials
through further vector optimization and exploration of additional
routes of administration, to support filing two IND applications
from the ALS SOD1, Huntington’s disease, and Friedreich’s ataxia
programs during 2019.
- Continue to identify, evaluate and progress collaborative
business development opportunities for certain Voyager programs,
technology platform capabilities, or both.
- Based on the Company’s current operating plan, Voyager expects
to end 2018 with cash, cash equivalents and marketable debt
securities of approximately $125 million to $135 million, which
includes the $69 million upfront payment from AbbVie, and projects
that its existing cash, cash equivalents and marketable debt
securities will be sufficient to fund operating expenses and
capital expenditure requirements into early 2020.
Fourth Quarter and Full Year 2017
Financial Results
For the fourth quarter and full year of 2017,
Voyager reported:
A GAAP net loss of $11.8 million, or $0.40 per
share, for the fourth quarter ended December 31, 2017, compared to
a GAAP net loss of $14.7 million, or $0.57 per share, for the same
period in 2016. The Company reported a GAAP net loss of $70.7
million, or $2.64 per share, for the full year ended December 31,
2017, compared to a net loss of $40.2 million, or $1.59 per share,
for the same period in 2016.
Collaboration revenues of $6.3 million for the
fourth quarter of 2017 compared to collaboration revenues of $2.4
million for the fourth quarter of 2016. Collaboration
revenues of $10.1 million for the full year ended December 31, 2017
compared to collaboration revenues of $14.2 million for the full
year ended December 31, 2016. Collaboration revenues reflect
recognition of payment for research and development services
provided by Voyager for various programs under the Sanofi Genzyme
collaboration agreement. Collaboration revenues can vary
based on quarterly assessments of expected or anticipated efforts
under the collaboration. The increase in collaboration
revenues for the fourth quarter 2017 compared to the same period in
2016 reflect revenue recognition as a result of Sanofi Genzyme’s
decision not to exercise its option to the ex-U.S. rights to the
Parkinson’s disease program and Voyager’s recognition of the
portion of the agreement allocated to that license option. The
decrease in collaboration revenues for the full year 2017 compared
to the same period in 2016 reflects the change in the estimated
period for reaching development milestones for certain preclinical
programs under the collaboration agreement.
Research and development (R&D) expenses of
$13.3 million for the fourth quarter ended December 31, 2017
compared to $12.7 million for the same period in 2016.
R&D expenses of $62.3 million for the year ended December 31,
2017 compared to $42.2 million for the same period in 2016. The
increase in R&D expenses related primarily to expenditures
associated with the development of Voyager’s pipeline including the
ongoing Phase 1 trial for VY-AADC, and increased personnel and
facility costs to support the advancement of the pipeline
programs.
General and administrative (G&A) expenses of
$5.4 million for the fourth quarter ended December 31, 2017
compared to $3.5 million for the same period in 2016. G&A
expenses of $19.7 million for the year ended December 31, 2017
compared to $13.3 million for the same period in 2016. The
increase in G&A expenses was primarily due to personnel and
facility costs to support Voyager’s pipeline programs.
Cash, cash equivalents, and marketable debt
securities as of December 31, 2017 were $169.1 million.
Conference Call Information
Voyager will host a conference call and webcast
today at 8:00 a.m. ET. 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
3688788. 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 Voyager Therapeutics
Voyager Therapeutics is a clinical-stage gene
therapy company focused on 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. Voyager’s pipeline focuses on severe
neurological diseases in need of effective new therapies, including
advanced Parkinson’s disease, a monogenic form of ALS called SOD1,
Huntington’s disease, Friedreich’s ataxia, and neurodegenerative
diseases related to defective or excess aggregation of tau protein
in the brain including Alzheimer’s disease and severe, chronic
pain. Voyager has broad strategic collaborations with Sanofi
Genzyme, the specialty care global business unit of Sanofi, AbbVie,
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.
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,”
“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 its CEO transition plans,
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 initiate, enroll and complete,
clinical trials, the potential clinical utility of its product
candidates, its ability to continue to develop its product engine,
its ability to develop manufacturing capability for its products
and successfully transition its manufacturing process, its ability
to perform under existing collaborations with, among others, Sanofi
Genzyme and AbbVie and to add new programs to its pipeline, its
ability to enter into new partnerships or collaborations, its
expected cash, cash equivalents and marketable debt securities at
the end of a fiscal period 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. Such risks and
uncertainties include, among others, those related to the search
for a new CEO; the initiation and conduct of preclinical studies
and clinical trials; the availability of data from clinical trials;
the expectations for regulatory submissions and approvals; the
continued development of the product engine; Voyager’s scientific
approach and general development progress; the availability or
commercial potential of Voyager’s product candidates; the
sufficiency of cash resources; need for additional financing; and
the possibility and timing of Voyager’s partner’s exercise of their
options to the collaboration programs. 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.
Investor Relations: Matt
OsborneVice President of Investor Relations & Corporate
Communications857-259-5353mosborne@vygr.com
Media:
Katie EnglemanPure Communications,
Inc.910-509-3977Katie@purecommunicationsinc.com
|
|
Selected Financial
Information($-amounts in thousands, except per share
data)(Unaudited) |
|
|
|
|
|
Three Months Ended |
|
Year Ended |
|
|
|
December 31, |
|
December 31, |
|
Statement of Operations Items: |
|
2017 |
|
|
2016 |
|
|
2017 |
|
|
2016 |
|
|
Collaboration
revenue |
|
$ |
6,345 |
|
|
$ |
2,362 |
|
|
$ |
10,135 |
|
|
|
14,220 |
|
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
13,327 |
|
|
|
12,723 |
|
|
|
62,260 |
|
|
|
42,249 |
|
|
General
and administrative |
|
|
5,366 |
|
|
|
3,481 |
|
|
|
19,738 |
|
|
|
13,270 |
|
|
Total operating
expenses |
|
|
18,693 |
|
|
|
16,204 |
|
|
|
81,998 |
|
|
|
55,519 |
|
|
Operating loss |
|
|
(12,348 |
) |
|
|
(13,842 |
) |
|
|
(71,863 |
) |
|
|
(41,299 |
) |
|
Total other income
(loss) |
|
|
550 |
|
|
|
(477 |
) |
|
|
1,165 |
|
|
|
1,158 |
|
|
Loss before income
taxes |
|
|
(11,798 |
) |
|
|
(14,319 |
) |
|
|
(70,698 |
) |
|
|
(40,141 |
) |
|
Income tax (benefit)
provision |
|
|
(31 |
) |
|
|
355 |
|
|
|
— |
|
|
|
52 |
|
|
Net loss |
|
$ |
(11,767 |
) |
|
$ |
(14,674 |
) |
|
$ |
(70,698 |
) |
|
$ |
(40,193 |
) |
|
Net loss per share,
basic and diluted |
|
$ |
(0.40 |
) |
|
$ |
(0.57 |
) |
|
$ |
(2.64 |
) |
|
$ |
(1.59 |
) |
|
Weighted-average common
shares outstanding, basic and diluted |
|
|
29,281,071 |
|
|
|
25,526,843 |
|
|
|
26,803,711 |
|
|
|
25,302,414 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
December 31, |
|
Selected
Balance Sheet Items |
|
2017 |
|
2016 |
|
Cash, cash equivalents,
and marketable debt securities |
|
$ |
169,052 |
|
$ |
174,418 |
|
Total assets |
|
$ |
184,477 |
|
$ |
189,566 |
|
Accounts payable and
accrued expenses |
|
$ |
12,517 |
|
$ |
7,038 |
|
Deferred revenue |
|
$ |
31,560 |
|
$ |
41,582 |
|
Total stockholders’
equity |
|
$ |
134,051 |
|
$ |
135,922 |
|
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