FDA grants orphan-drug designation for VY-HTT01
for Huntington’s disease
Voyager Therapeutics, Inc. (NASDAQ: VYGR), a clinical-stage gene
therapy company focused on developing life-changing treatments for
severe neurological diseases, today reported its first quarter 2019
financial results, program progress and corporate updates.
“The first quarter was an eventful period for
Voyager as we announced new collaborations, provided updates to our
pipeline programs, and advanced our discovery efforts focused on
novel gene therapy capsids that have the potential to cross the
blood-brain barrier and target specific cells within the brain
after a single, systemic administration,” said Andre Turenne,
president and chief executive officer of Voyager. “During the
remainder of the year, our focus will remain on advancing our
pipeline aimed at transformative therapies for patients living with
devastating neurological diseases.”
Recent Corporate and Program
Highlights
- In January 2019, Neurocrine
Biosciences, Inc. and Voyager announced a strategic collaboration
focused on the development and commercialization of two gene
therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for
Friedreich’s ataxia, as well as two other development programs to
be determined. This collaboration combines Neurocrine’s expertise
in neuroscience, drug development and commercialization with
Voyager’s innovative gene therapy programs targeting severe
neurological diseases. The collaboration became effective in March
2019.
- Announced Phase 1 trial (PD-1102)
results for VY-AADC from eight patients with Parkinson’s disease
who participated in an open-label trial to evaluate the safety and
efficacy of VY-AADC and to further assess the posterior (i.e., from
the back of the head) surgical delivery approach. These Phase 1
results were presented at the 2019 Annual Meeting of the American
Academy of Neurology. The results demonstrated that the posterior
trajectory can serve as an additional surgical delivery approach.
The results also provide further evidence to the findings from a
separate Phase 1b trial (PD-1101) indicating that increased
coverage of the putamen with VY-AADC leads to increases in AADC
enzyme activity and improvements in motor function and quality of
life in patients with Parkinson’s disease – with less need for oral
levodopa medication.
- Announced new preclinical data in
multiple presentations at the Annual Meeting of the American
Society of Gene and Cell Therapy. This preclinical data related to
Voyager’s vectorized antibody program directed against tau for the
potential treatment of Alzheimer’s disease, its TRACER™ system to
discover adeno-associated virus (AAV) capsids with blood-brain
barrier crossing and cell-specific transduction properties,
VY-SOD102 targeting a monogenic form of amyotrophic lateral
sclerosis called SOD1, and VY-HTT01 targeting Huntington’s
disease.
- VY-HTT01 granted Orphan Drug
Designation (ODD) by the U.S. Food and Drug Administration (FDA)
for the treatment of Huntington’s disease. The ODD Program provides
orphan status to drugs and biologics intended for the safe and
effective treatment, diagnosis or prevention of rare diseases that
affect fewer than 200,000 people in the U.S.
Board of Directors Leadership
Change
Voyager announced that immediately following its
2019 Annual Meeting of Stockholders, scheduled for June 13, 2019,
Michael Higgins, currently a member of the Board, will succeed Mark
Levin as Board chairman. Mr. Levin, if re-elected at the Annual
Meeting, will continue to serve as a member of the Board. Mr.
Higgins has served as a member of the Board since July 2015.
First Quarter 2019 Financial
Results
For the first quarter of 2019, Voyager
reported:
A GAAP net loss of $27.2 million, or $0.81 per
share, for the first quarter ended March 31, 2019, compared to a
GAAP net loss of $19.9 million, or $0.63 per share, for the same
period in 2018.
Collaboration revenues of $5.2 million for the
first quarter of 2019 compared to $0.9 million for the first
quarter of 2018. The increase in collaboration revenues for the
first quarter of 2019 reflects the increase in revenue related to
research services performed by Voyager under existing
collaborations with Sanofi Genzyme and AbbVie, revenue related to
research services from new collaborations with AbbVie and
Neurocrine, and the reimbursement of costs related to the
collaboration with Neurocrine.
Research and development (R&D) expenses of
$24.8 million for the first quarter ended March 31, 2019 compared
to R&D expenses of $14.9 million for the same period in 2018.
The increase in R&D expenses related primarily to an increase
associated with preclinical programs, personnel and facility costs
to support the advancement of the VY-AADC program into the
RESTORE-1 Phase 2 clinical trial, and gene therapy platform
initiatives for new capsid development.
General and administrative (G&A) expenses of
$9.7 million for the first quarter of 2019 compared to G&A
expenses of $7.2 million for the same period in 2018. The increase
in G&A expenses was primarily due to an increase in consulting
and professional fees, and personnel and facility costs to support
the advancement of Voyager’s pipeline programs, platform and
manufacturing capabilities.
Cash, cash equivalents, and marketable debt
securities as of March 31, 2019 were $358.5 million. Based on the
Company’s current operating plan, Voyager expects to end 2019 with
cash, cash equivalents and marketable debt securities of
approximately $280 million to $290 million, with full-year 2019
operating expenses expected to range from $130 million to $140
million that includes amounts reimbursable under the Neurocrine
collaboration. Voyager continues to project that its cash, cash
equivalents and marketable debt securities will be sufficient to
fund operating expenses and capital expenditure requirements to
mid-2022.
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 wholly-owned and partnered pipeline
focuses on severe neurological diseases in need of effective new
therapies, including Parkinson’s disease, a monogenic form of ALS
called SOD1, Huntington’s disease, Friedreich’s ataxia, Alzheimer’s
disease, and other neurodegenerative diseases related to defective
or excess aggregation of tau and alpha-synuclein proteins in the
brain. Voyager has strategic collaborations with Sanofi Genzyme,
AbbVie and Neurocrine. Founded by scientific and clinical leaders
in the fields of AAV gene therapy, expressed RNA interference and
neuroscience, Voyager is headquartered in Cambridge, Massachusetts.
For more information on Voyager, please visit the company’s website
at www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and
LinkedIn.
Voyager Therapeutics® is a registered trademark,
and TRACER™ is a trademark, of Voyager Therapeutics.
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 the initiation, timing,
progress, activities, goals and reporting of results of its
preclinical programs and clinical trials and its research and
development programs, the potential benefits and future operation
of the collaboration agreements with Sanofi Genzyme, AbbVie and
Neurocrine, including any potential future payments thereunder, 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 gene therapy platform and its TRACER
system, its ability to perform under existing collaborations with,
among others, Sanofi Genzyme, AbbVie and Neurocrine and to add new
programs to its pipeline, and the regulatory pathway of, and the
timing or likelihood of its regulatory filings and approvals for,
any of its product candidates, anticipated financial results,
including Voyager’s available cash and cash equivalents at the end
of 2019, the receipt by Voyager of revenues from collaboration
partners during 2019 and Voyager’s operating expenses during 2019,
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, those related to the
initiation and conduct of preclinical studies and clinical trials;
the availability of data from clinical trials; the expectations for
regulatory communications, submissions and approvals; the continued
development of the gene therapy platform and its TRACER system;
Voyager’s scientific approach and general development progress; the
sufficiency of cash resources; the possibility or the timing of the
exercise of development, commercialization and license options
under collaborations, 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 for the
year ended December 31, 2018 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.
Selected Financial
Information(amounts in thousands, except share and per
share data)(Unaudited)
Statement of Operations Items:
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
|
|
|
March 31, |
|
|
|
2019 |
|
2018 |
|
Collaboration
revenue |
|
$ |
5,197 |
|
|
$ |
942 |
|
|
Operating
expenses: |
|
|
|
|
|
|
|
Research
and development |
|
|
24,831 |
|
|
|
14,853 |
|
|
General
and administrative |
|
|
9,659 |
|
|
|
7,182 |
|
|
Total operating
expenses |
|
|
34,490 |
|
|
|
22,035 |
|
|
Operating loss |
|
|
(29,293 |
) |
|
|
(21,093 |
) |
|
Total other income |
|
|
2,123 |
|
|
|
987 |
|
|
Loss before income
taxes |
|
|
(27,170 |
) |
|
|
(20,106 |
) |
|
Income tax benefit |
|
|
— |
|
|
|
180 |
|
|
Net loss |
|
$ |
(27,170 |
) |
|
$ |
(19,926 |
) |
|
Net loss per share,
basic and diluted |
|
$ |
(0.81 |
) |
|
$ |
(0.63 |
) |
|
Weighted-average common
shares outstanding, basic and diluted |
|
|
33,353,061 |
|
|
|
31,759,870 |
|
|
|
|
|
|
|
|
|
|
Selected
Balance Sheet Items: |
|
March 31, |
|
December 31, |
|
|
|
2019 |
|
2018 |
|
Cash, cash equivalents,
and marketable debt securities |
|
$ |
358,499 |
|
$ |
155,806 |
|
Total assets |
|
$ |
411,349 |
|
$ |
177,029 |
|
Accounts payable and
accrued expenses |
|
$ |
11,537 |
|
$ |
10,826 |
|
Deferred revenue |
|
$ |
262,049 |
|
$ |
113,046 |
|
Total stockholders’
equity |
|
$ |
100,694 |
|
$ |
46,446 |
|
Investor Relations:
Matt Osborne
Vice President of Corporate Affairs, Communications and Investor Relations
857-259-5353
mosborne@vygr.com
Voyager Media:
Sheryl Seapy
W2Opure
949-903-4750
sseapy@purecommunications.com
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