Neurocrine Biosciences Acquires Rights to Voyager’s Parkinson’s Gene Therapy VY-AADC in $165 Million Deal

VCBeat (WeChat Official Account: vcbeat) learned from foreign media that U.S. biopharmaceutical company Neurocrine Biosciences recently announced an agreement to pay Voyager Therapeutics (“Voyager”) $115 million in cash to acquire $50 million worth of Voyager’s stock at a price of $11.96 per share. Neurocrine will obtain the rights to VY-AADC, an experimental gene therapy for Parkinson’s disease developed by Voyager, but will bear the costs of late-stage clinical development.

VY-AADC is currently in mid-stage testing. Once data from the ongoing RESTORE-1 trial become available, Voyager may choose to separate ownership of VY-AADC from development costs, or transfer ownership to Neurocrine in exchange for downstream payments and royalties.

Voyager is a gene therapy company dedicated to developing treatments for debilitating diseases of the central nervous system (CNS). Founded in 2013 and headquartered in Massachusetts, USA, Voyager advances adeno-associated virus (AAV) gene therapy through innovation and investment in vector optimization engineering, delivery technologies, and process development and manufacturing. The company’s initial R&D focus is on novel therapies for CNS disorders, including Parkinson’s disease and Friedreich’s ataxia.

Voyager’s purchase agreement with Neurocrine also includes an alliance concerning FY-FXN01. FY-FXN01 is a gene therapy for Friedreich’s ataxia that has not yet entered human clinical trials. Similar to VY-AADC, Voyager may choose to share costs and profits with Neurocrine, or opt to divest the development costs in exchange for licensing rights.

In a November report, Voyager Therapeutics stated that it is conducting a placebo-controlled Phase II study. However, because the FDA considers this trial an “early-stage” exploratory study, it is not yet eligible for approval via the gene therapy application pathway. Following meetings with the FDA, Voyager announced that during the study’s resumption phase, it will enroll an additional 1 to 100 patients and conduct a “staggered parallel” trial. If the data provided by both Voyager and Neurocrine Biosciences are positive, the FDA may approve their applications.

Neurocrine Biosciences is a biopharmaceutical company headquartered in San Diego, dedicated to developing treatments for neurological and endocrine-related disorders. The Company discovers, develops, and markets INGREZZA capsules, the first FDA-approved product for the treatment of tardive dyskinesia (a movement disorder characterized by involuntary movements) in adults.

Currently, Neurocrine’s research team is advancing novel small-molecule compounds into clinical studies to develop small-molecule antagonists targeting G protein-coupled receptors.

Andre Turenne, President and Chief Executive Officer of Voyager, stated: “Neurocrine Biosciences is an ideal partner, with established expertise in the development and commercialization of therapies for movement disorders and other neurological diseases. This collaboration is transformative for Voyager, significantly enhancing our potential to become a leading, fully integrated gene therapy company, while allowing us to continue investing in our other programs and platforms.”

Parkinson's Disease (PD), also known as paralysis agitans, is one of the most common neurodegenerative disorders. The etiology and pathogenesis of PD remain unclear but may be associated with social factors, medication-related factors, and patient-specific factors. The pathological changes in PD include depigmentation of neurons in the substantia nigra pars compacta and the locus coeruleus, pallor of the substantia nigra, and the presence of Lewy bodies.

Friedreich's Ataxia is a group of hereditary degenerative diseases characterized by chronic progressive cerebellar ataxia. A family history of the disease, clinical manifestations of ataxia, and pathological changes predominantly involving cerebellar damage constitute the three hallmark features of Friedreich's Ataxia. In addition to affecting the cerebellum and its conducting fibers, the disease frequently involves the posterior columns of the spinal cord, pyramidal tracts, pontine nuclei, basal ganglia, cranial nerve nuclei, spinal ganglia, and the autonomic nervous system.

(Compiled by Liu Ting)