Neurocrine Biosciences and Voyager Therapeutics Form Strategic Development and Commercialization Collaboration for Parkinson’s Disease and Friedreich’s Ataxia
- Neurocrine Biosciences has agreed to fund the development through the Phase 1 clinical trial of VY-FXN01. After the data readout of the Phase 1 trial, Voyager has the option to either: (1) co-commercialize VY-FXN01 with Neurocrine Biosciences in the U.S. under a 60/40 cost- and profit-sharing arrangement, or (2) grant Neurocrine Biosciences full U.S. commercial rights in exchange for milestone payments and royalties based on U.S. sales. Sanofi Genzyme retains an option for ex-U.S. rights to VY-FXN01 following the data readout of the Phase 1 trial.
Under terms of the agreement for the two programs to be determined:
- Neurocrine Biosciences has agreed to fund the development of these programs to be determined and Voyager will have the right to earn milestone payments and royalties based on global sales.
The effectiveness of the collaboration agreement and the closing of the sale and issuance of Voyager common stock described above are subject to certain conditions including the expiration or termination of the applicable waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and other customary closing conditions.
Centerview Partners served as Neurocrine Biosciences' financial advisor and Cooley LLPserved as Neurocrine Biosciences' legal counsel for the collaboration. Wilmer Cutler Pickering Hale and Dorr LLP served as legal counsel for Voyager.
Conference Call Information
Neurocrine Biosciences will host a conference call and webcast today at 8:00 a.m. EST. The live call may be accessed by dialing (866) 342-8588 (U.S.) or (203) 518-9865 (International) using the conference ID: NBIX.
Voyager will host a conference call and webcast today at 8:45 a.m. EST. The live call may be accessed by dialing (877) 851-3834 and referencing conference ID number 3181907.
A live audio webcast of the conference calls will be available online in the Investors section on Neurocrine Biosciences' website at www.neurocrine.com, and from the Investors & Media section of Voyager Therapeutics' website at www.voyagertherapeutics.com. A replay of the webcasts will be available on the website approximately one hour after the conclusion of each event and will be archived for approximately one month.
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.
The FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation for VY-AADC for the treatment of Parkinson's disease in patients with motor fluctuations who are refractory to medical management. RMAT designation is an expedited program for the advancement and approval of regenerative medicine products, including gene therapy products. RMAT designation was granted based on clinical data from the Phase 1b trial with VY-AADC in patients with Parkinson's disease. During this trial, one-time administrations of VY-AADC demonstrated robust and durable improvements in patients' motor function along with substantial reductions in use of daily oral levodopa and other Parkinson's disease medications. Infusions of VY-AADC have been well-tolerated in this trial with no vector-related serious adverse events reported to date.
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 clinicaltrials@vygr.com.
About Voyager's VY-FXN01 for Friedreich's Ataxia
Friedreich's ataxia (FA) is a rare, severe, inherited neurological disease caused by mutations in the frataxin (FXN) gene leading to decreased expression of FXN, which results in severe sensory impairment, progressive loss of the ability to walk, generalized weakness, and loss of sensation, as well as severe and potentially fatal cardiomyopathy. The typical age of onset is 10 to 12 years with reduced life expectancy between 35 to 45 years of age due to neurological and cardiac complications. The goal of VY-FXN01 is to restore FXN protein levels, with a one-time treatment, to at least 50% of normal in relevant neurons and cardiac myocytes, to slow the progression of disease.
In a preclinical model of FA disease, Voyager's frataxin gene therapy vector durably improved ataxia and sensory function, and rescued the FA phenotype based on multiple functional tests. In physiological and behavioral assays, Voyager's frataxin gene therapy vector demonstrated dose-dependent and durable responses for more than 10 months after a single administration, preventing central and peripheral disease progression. Additional preclinical studies are underway at Voyager including steps to identify a lead clinical candidate for the treatment of FA during 2019.
About Neurocrine Biosciences
Neurocrine Biosciences, a San Diego based biopharmaceutical company, is focused on developing treatments for neurological and endocrine related disorders. The company discovered, developed and markets INGREZZA® (valbenazine) capsules, the first FDA-approved product indicated for the treatment of adults with tardive dyskinesia, an involuntary movement disorder. Discovered and developed through Phase II clinical trials by Neurocrine, ORILISSA®(elagolix), the first FDA-approved oral medication for the management of endometriosis with associated moderate to severe pain in over a decade, is marketed by AbbVie as part of a collaboration to develop and commercialize elagolix for women's health. Neurocrine's clinical development programs include opicapone as an adjunctive therapy to levodopa/DOPA decarboxylase inhibitors in Parkinson's disease patients, elagolix for uterine fibroids with AbbVie, valbenazine for the treatment of Tourette syndrome, and NBI-74788 for the treatment of congenital adrenal hyperplasia (CAH). For more information and the latest updates from Neurocrine Biosciences, please visit www.neurocrine.com.
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 Parkinson's disease, a monogenic form of ALS called SOD1, Huntington's disease, Friedreich's ataxia, neurodegenerative diseases related to defective or excess aggregation of tau protein in the brain including Alzheimer's disease and severe, chronic pain. Voyager has strategic collaborations with Sanofi Genzyme, AbbVie and Neurocrine Biosciences. 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 on Voyager Therapeutics, please visit the company's website at www.voyagertherapeutics.com.
http://ir.voyagertherapeutics.com/
