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|Voyager Therapeutics Joins Michael J. Fox Foundation Initiatives and Education Campaigns to Accelerate the Development of New therapies and a Cure for Parkinson’s Disease|
“We are dedicated to finding a cure for Parkinson's disease and accelerating breakthroughs patients can feel in their everyday lives,” said
“We are thrilled to join The Michael J. Fox Foundation’s efforts to bring industry, advocacy, and academia together through its PPMI and PDEC initiatives,” said
About the Parkinson’s Progression Markers Initiative
PPMI is a landmark natural history study launched in 2010 to help identify, develop, and validate biomarkers of Parkinson’s disease progression to further the development of disease-modifying therapies. PPMI is funded by MJFF and a consortium of key industry players, non-profit organizations, and private individuals and involves active participation of stakeholders from government, the biopharmaceutical industry, and academia to collectively design, implement and fund this comprehensive program. With involvement from more than 30 clinical sites in 11 countries and more than 1,000 participants, PPMI collects and examines clinical and biologic measures to track disease progression in early-stage Parkinson’s and prodromal patients and certain genetic populations for use in clinical trials of disease-modifying therapies. Voyager representatives will join PPMI’s
About the Parkinson’s
Launched in 2016, PDEC is an alliance between MJFF and industry partners committed to patient and caregiver knowledge. PDEC supports the development of MJFF’s evergreen educational programs (e.g., Third Thursdays Webinar series) and spotlights annual campaigns focused on addressing unmet needs in Parkinson’s community education. Past campaigns have included a narrative on the Parkinson’s disease patient journey, a companion pack for clinical trial volunteers, and study teams to address research participation, and a research initiative aimed at addressing barriers to communication between patients, care-givers and healthcare practitioners.
In 2019 and 2020, MJFF, in partnership with PDEC, will lead an effort to bring education to people with Parkinson’s, supporters, and caregivers through local community activities and broad targeted educational content that speaks to all aspects of the Parkinson’s experience. MJFF aims to harness the power of peer-to-peer interaction in empowering community members to take action and live well with Parkinson’s.
About Voyager’s VY-AADC Gene Therapy for Parkinson’s Disease
VY-AADC is an investigational gene therapy product 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 produce the AADC enzyme to convert levodopa into dopamine. With this approach, VY-AADC has the potential to durably enhance the conversion of levodopa to dopamine and provide clinically meaningful improvements by restoring motor function in patients and improving symptoms following a single administration.
Voyager recently initiated the Phase 2 RESTORE-1 trial in patients who have been diagnosed with Parkinson’s disease for at least four years, are not responding adequately to oral medications, and have at least three hours of OFF time during the day as measured by a validated self-reported patient diary.
For additional information regarding Voyager’s RESTORE-1 Phase 2 clinical trial with its gene therapy VY-AADC for the treatment of Parkinson’s disease, please use the following link or email Voyager at firstname.lastname@example.org.
About Parkinson’s Disease
Parkinson’s disease is a chronic, progressive and debilitating neurodegenerative disease that affects approximately 1,000,000 people in the U.S.1 and seven to 10 million people worldwide2. 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 and the enzyme needed to convert levodopa to dopamine in this region of the brain, the putamen, leads to the motor symptoms associated with Parkinson’s disease including tremors, slow movement or loss of movement, rigidity, and postural instability. Additional motor symptoms during the advanced stages of the disease include falling 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 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 levodopa to dopamine4. The intrinsic neurons in the putamen, however, do not degenerate in Parkinson’s disease5,6.
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 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 gene therapy platform, 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
1 Willis et al, Neuroepidemiology.2010;34:143–151