Improved Functional and Quality of Life Data
Presented at the American Academy of Neurology (AAN) Meeting in
Boston, MA
Voyager Therapeutics, Inc. (NASDAQ:VYGR), a clinical-stage gene
therapy company developing life-changing treatments for severe
diseases of the central nervous system (CNS), today announced
dose-dependent improvements in functional and quality of life
measures from the Phase 1b trial of VY-AADC01 for advanced
Parkinson’s disease presented as part of an emerging
(late-breaking) oral presentation at the AAN Annual Meeting.
In addition, during a separate oral presentation at the AANS Annual
Scientific Meeting, investigators presented results for maximizing
coverage of the putamen during the surgical procedure for
VY-AADC01.
“We are pleased that VY-AADC01 data were
selected for oral presentation at major neurology and neurosurgery
meetings,” said Bernard Ravina, M.D., M.S., chief medical officer
of Voyager Therapeutics. “In addition to the previously
reported improvements in biomarkers and motor symptoms,
investigators at the AAN meeting presented data on patients’
improved function and quality of life. Motor fluctuations in
advanced Parkinson’s are debilitating and these function and
quality of life results are key indicators of the potential
clinical impact of a one-time treatment with VY-AADC01. Here,
we were encouraged with the 9-point improvement in PDQ-39 total
score, a patient reported outcome, in Cohort 2 at 12 months.
In addition to these data at the AAN meeting, the data at the AANS
meeting highlighted key advances in the neurosurgical techniques
used to administer VY-AADC01 and increase the coverage of the
putamen, the brain region that we are targeting with our gene
therapy approach aimed at restoring patients’ response to their
levodopa medication.”
VY-AADC01 data presented at the AAN Annual
Meeting included previously reported motor symptom data from all 10
patients treated in Cohorts 1 and 2 at six months (five patients in
each Cohort), and data from five patients in Cohort 1 and three
patients in Cohort 2 who have reached 12 months of follow-up at the
time of the interim results. In addition, investigators at
the AAN meeting presented new functional and quality of life data
as measured by the Unified Parkinson’s Disease Rating Scale (UPDRS)
Part II and Part IV, and the patient-reported 39-item Parkinson's
Disease Questionnaire (PDQ-39) demonstrating dose-dependent and
clinically meaningful improvements in these scores (Table 1).
Table 1. VY-AADC01 Phase 1b interim
results on UPDRS-II, -IV, and PDQ-39 scores
Functional/Quality of Life Measure |
Baseline score |
Results at 12-months1 |
UPDRS-II (off-medication) |
Cohort 1 (13.6) Cohort 2 (16.7) |
Cohort 1 (0.2-point worsening, SD 3.7) vs.Cohort 2 (4.0-point
improvement, SD 1.0) |
UPDRS-IV total score |
Cohort 1 (7.8)Cohort 2 (8.7) |
Cohort 1 (1.2-point improvement, SD 1.9) vs.Cohort 2 (2.7-point
improvement, SD 3.1) |
PDQ-39 total score |
Cohort 1 (18.2)Cohort 2 (12.3) |
Cohort 1 (1.9-point improvement, SD 5.6) vs.Cohort 2 (9.2-point
improvement, SD 5.5) |
1) Cohort 1 baseline and 12-month data from 5/5
patients. Cohort 2 baseline and 12-month data from 3/5
patients. 2/5 patients inCohort 2 have not reached 12-month
follow-up period at the time of the analysis |
|
- The emerging (late-breaking) oral presentation at the AAN
Annual Meeting will be presented during the Emerging Science
Session taking place this evening from 5:45 p.m. to 6:30 p.m.
EDT.
Data presented at the AANS Annual Scientific Meeting reviewed
the evolution of the real-time MRI-guided surgical procedure during
the clinical trial that resulted in increased coverage of the
putamen, a region of the brain associated with motor function in
Parkinson’s disease. In addition to increased infusion
volumes, these steps included modifications to the size of the
cannula and position of infusion sites during surgery. As
previously reported, these developments increased the average
coverage of the putamen from 21% in Cohort 1, to 34% in Cohort 2,
and 42% in Cohort 3.
- The oral presentation of these data at the AANS Meeting
occurred on April 24, during the Stereotactic and Functional
Surgery session from 2:00 p.m. to 5:30 p.m. PST.
About the Phase 1b Trial of VY-AADC01
for Advanced Parkinson’s Disease
In advanced Parkinson’s disease, the putamen is
depleted of dopamine and of the enzyme aromatic L-amino acid
decarboxylase (AADC) that is responsible for converting levodopa to
dopamine. VY-AADC01 is Voyager’s gene therapy vector that
contains the gene that encodes the AADC enzyme. The Phase 1b,
open-label trial includes 15 patients with advanced Parkinson’s
disease and disabling motor fluctuations, treated with a single
administration of VY-AADC01. The primary objective of the
trial is to assess the safety and surgical coverage of ascending
doses of VY-AADC01 in the putamen, a region of the brain associated
with motor function in Parkinson’s disease. The secondary
objectives of the trial include the assessment of AADC expression
and activity in the putamen measured by positron emission
tomography (PET) using [18F] fluorodopa (or 18F-DOPA). In
addition, changes in motor responses to levodopa are measured by a
controlled intravenous infusion of levodopa and by measuring daily
requirements for levodopa and related medications. Other
secondary objectives include assessment of motor function as
measured by the Unified Parkinson’s Disease Rating Scale (UPDRS)
and a patient-completed (Hauser) diary.
Conference Call Information
Voyager is hosting a breakfast event at the AAN
conference today at 8:00 a.m. with KOL neurologists to discuss
advanced Parkinson’s disease. The event may be accessed live
by dialing (877) 851-3834 for domestic callers or +1 (631) 291-4595
for international callers, and referencing conference ID number
5621646, or by audio webcast from the Investors & Media section
of Voyager’s website at www.voyagertherapeutics.com. The
webcast will be archived for 30 days.
About Parkinson’s Disease and VY-AADC01
Parkinson’s disease is a chronic, progressive
and debilitating neurodegenerative disease that affects
approximately 700,000 people in the U.S.1 and seven to 10
million people worldwide2. It is estimated that up to 15% of
the prevalent population with Parkinson’s disease, or approximately
100,000 patients in the U.S., have motor fluctuations that are
refractory, or not well-controlled, with levodopa. While the
underlying cause of Parkinson's disease in most patients is
unknown, the motor symptoms of the disease arise from a loss of
neurons in the midbrain that produce the neurotransmitter
dopamine. Declining levels of dopamine in this particular
region of the brain leads to the motor symptoms associated with
Parkinson’s disease including tremors, slow movement or loss of
movement, rigidity, and postural instability. Motor symptoms
during the advanced stages of the disease include falling, gait
freezing, and difficulty with speech and swallowing, with patients
often requiring the daily assistance of a caregiver.
There are currently no therapies that
effectively slow or reverse the progression of Parkinson’s disease.
Levodopa remains the standard of care treatment, with its
beneficial effects on symptom control having been discovered over
40 years ago3. Patients are generally well-controlled with
oral levodopa in the early stages of the disease, but become less
responsive to treatment as the disease progresses. Patients
experience longer periods of reduced mobility and stiffness termed
off-time, or the time when medication is no longer providing
benefit, and shorter periods of on-time when their medication is
effective.
The progressive motor symptoms of Parkinson’s
disease are largely due to the death of dopamine neurons in the
substantia nigra, a part of the midbrain that converts levodopa to
dopamine, in a single step catalyzed by the enzyme AADC.
Neurons in the substantia nigra release dopamine into the putamen
where the receptors for dopamine reside. In advanced
Parkinson’s disease, neurons in the substantia nigra degenerate and
the enzyme AADC is markedly reduced in the putamen, which limits
the brain’s ability to convert oral levodopa to dopamine4.
The intrinsic neurons in the putamen, however, do not degenerate in
Parkinson’s disease5,6. VY-AADC01, comprised of the
adeno-associated virus-2 capsid and a cytomegalovirus promoter to
drive AADC transgene expression, is designed to deliver the AADC
gene directly into neurons of the putamen where dopamine receptors
are located, bypassing the substantia nigra neurons and enabling
the neurons of the putamen to express the AADC enzyme to convert
levodopa into dopamine. The approach with VY-AADC01,
therefore, has the potential to durably enhance the conversion of
levodopa to dopamine and provide clinically meaningful improvements
in motor symptoms following a single administration.
About Voyager Therapeutics
Voyager Therapeutics is a clinical-stage gene
therapy company developing life-changing treatments for severe
diseases of the CNS. 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. The Company’s pipeline focuses on severe
CNS diseases in need of effective new therapies, including advanced
Parkinson’s disease, a monogenic form of ALS, Friedreich’s ataxia,
Huntington’s disease, frontotemporal dementia, Alzheimer’s disease
and severe, chronic pain. Voyager has broad strategic
collaborations with Sanofi Genzyme, the specialty care global
business unit of Sanofi, 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. Follow Voyager on LinkedIn.
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 law. The use of words such as “may,” “might,” “will,”
“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 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
complete, clinical trials, its ability to continue to develop its
product engine, its ability to add new programs to its pipeline,
ability to enter into new partnerships or collaborations, its
expected cash, cash equivalents and marketable debt securities at
the end of a fiscal year 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. 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 future 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.
1 Willis et al,
Neuroepidemiology.2010;34:143–1512 www.pdf.org/en/parkinson_statistics3 Poewe
W, et al, Clinical Interventions in
Aging.2010;5:229-238.4 Lloyd, J Pharmacol Exp Ther.
1975;195:453-464, Nagatsu, J Neural Transm Suppl.20075 Cold
Spring Harb Perspect Med 2012;2:a0092586 Braak et al, Cell
Tissue Res.2004;318:121-134
Investor Relations:
Matt Osborne
Head of Investor Relations & Corporate Communications
857-259-5353
mosborne@vygr.com
Media:
Katie Engleman
Pure Communications, Inc.
910-509-3977
Katie@purecommunicationsinc.com
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