Neurocrine agrees to pay up to $1.7bn in gene therapy collaboration

By Maggie Lynch

- Last updated on GMT

(Image: Getty/Sudok1)
(Image: Getty/Sudok1)

Related tags Gene therapy Gene therapy candidate Neurology Parkinson's disease freidriech's ataxia Clinical development

Neurocrine and Voyager enter an agreement to fund and develop gene therapy candidates for the treatment of severe neurological diseases.

Per the agreement, Neurocrine Biosciences will pay Voyager Therapeutics $165m (€143.7m) in cash, which includes a $115m upfront payment and a $50m equity investment for the development of two projects for the treatment of Parkinson’s disease and Freidriech’s ataxia (FA).

Voyager will also receive funding from Neurocrine for the collaboration programs, as Voyager could receive up to $1.7bn in development, regulatory, and commercial milestone payments across both programs.


The collaboration will include two projects, the first of which is the clinical development of the Phase II-III pivotal program for VY-AADC, an investigational gene therapy product.

VY-AADC is designed to deliver the AADC gene directly to neurons where dopamine receptors are located. The drug has the potential to improve and restore motor function in patients, and in early-stage trials, has seen patients’ symptoms improve following a single administration of the product.

The US Food and Drug Administration (FDA) granted a Regenerative Medicine Advanced Therapy designation to this product, for the treatment of Parkinson’s disease patients with motor fluctuations, based upon clinical data from a Phase Ib trial.

Neurocrine has agreed to fund this program, while Voyager has the option to co-commercialize VY-AADC or grant its partner the full global commercial rights in exchange for milestone payments and royalties after the Phase II data readout.


The second project is the development of the Phase I clinical trial of VY-FXN01 for the treatment of FA – a rare and severe inherited neurological disease caused by mutations in the frataxin (FXN) gene.

VY-FXN01 was designed to restore FXN protein levels to at least 50% of relevant neurons and cardiac myocytes to slow the progression of the disease. In a pre-clinical model of FA, the gene therapy vector product improved ataxia and sensory function.

After the completion of Phase I studies for this product, Voyager’s options will include co-commercializing the program under a 60/40 cost and profit sharing agreement, or grant Neurocrine full commercial rights in exchange for milestone payments and royalties based on US sales.

“The partnership with Voyager allows us to expand our clinical development pipeline addressing neurological disorders, leverage Voyager’s expertise in CNS-focused gene therapy, and develop potential treatments for diseases, such as Parkinson’s disease and Friedreich’s ataxia, which have significant unmet clinical needs,”​ Kevin Gorman, CEO of Neurocrine Biosciences said in a statement.

Sanofi Genzyme retains the right to exercise an option for the rights to VY-FXN01 outside of the US following the Phase I trial. 

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