Neurocrine Biosciences and Voyager Therapeutics have joined efforts in a new strategic collaboration to further develop and market Voyager’s gene therapies VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia.
With this partnership, Neurocrine Biosciences will apply its expertise in neuroscience, drug development, and commercialization to help expedite the development of Voyager’s gene therapy programs.
“We are excited to collaborate with Voyager to advance our shared mission to discover and develop medicines that can benefit the lives of people with serious neurological disorders,” Kevin Gorman, PhD, CEO of Neurocrine, said in a press release.
Neurocrine will have the opportunity to expand its “clinical development pipeline addressing neurological disorders, leverage Voyager’s expertise in [central nervous system]-focused gene therapy, and develop potential treatments for diseases,” he said.
Based on the terms of the agreement, Neurocrine is going to finance the Phase 2-3 pivotal program for VY-AADC for Parkinson’s disease. After the completion of the Phase 2 RESTORE-1 trial (NCT03562494), in which the therapy is currently being evaluated, Voyager will have the option to decide whether it will share commercialization responsibilities or give Neurocrine full rights to the therapy in the U.S. in return for milestone payments and royalties.
A similar plan was established for the future of VY-FXN01 for Friedreich’s ataxia. In addition, Neurocrine also agreed to fund two additional gene therapies programs yet to be determined.
“This is a transformational collaboration for Voyager that greatly enhances our efforts towards becoming the leading, fully-integrated gene therapy company focused on severe neurological diseases while allowing us to continue to invest in our additional pipeline programs and platform,” said Andre Turenne, president and CEO of Voyager.
VY-AADC is an investigational gene therapy that uses a modified and harmless adeno-associated virus to deliver the ADDC gene directly into the putamen brain region, which is involved in movement control.
This gene provides instructions for making an enzyme, called 1-amino acid decarboxylase (AADC), that converts levodopa (the gold standard treatment for Parkinson’s) to dopamine — a signaling molecule that acts as a messenger between brain cells and is present at lower levels in Parkinson’s patients.
Researchers believe that with a single administration of VY-AADC, it may be possible to achieve the sustainable conversion of levodopa to dopamine, enhancing its clinical effects and significantly restoring the motor function of Parkinson’s patients.
The ongoing RESTORE-1 trial is currently recruiting individuals who have been diagnosed with Parkinson’s for four years or more and who are not responding well to oral medications. Eligible patients also need to have at least three hours of daily “off” periods — characterized by the return of motor and non-motor symptoms when levodopa’s effects wear off.
The study will evaluate the impact of VY-AADC on patients’ motor symptom fluctuations, measured by a self-reported patient diary and response to levopoda treatment. Researchers will also evaluate changes in quality of life and global function, as well as changes in non-motor symptoms, upon treatment with VY-AADC compared to placebo.
VY-AADC received the regenerative medicine advanced therapy designation from the U.S. Food and Drug Administration in June 2018 for the treatment of therapy-resistant motor fluctuations in Parkinson’s patients.
The FDA’s decision was supported by positive results of a Phase 1b trial (NCT01973543) in 15 Parkinson’s patients, in which a single administration of the therapy induced robust and durable improvements in patients’ motor function up to three years after treatment.
VY-AADC treatment also effectively increased AADC enzyme activity, allowing patients to reduce their doses of oral levodopa and improving their quality of life.
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