Neurocrine Biosciences, Voyager Therapeutics in $1.7-Billion Gene-Therapy Pact

By Patricia Van Arnum - DCAT Editorial Director

February 1, 2019

Neurocrine Biosciences, a San Diego, California-based pharmaceutical company developing small molecules for treating neurological and endocrine-related disorders, and Voyager Therapeutics, a Cambridge, Massachusetts company developing gene therapies, have formed a strategic collaboration to advance gene therapies for treating neurological diseases in a deal worth up to $1.7 billion ($165 million upfront and $1.7 billion in potential milestones).

The pact is focused on the development and commercialization of Voyager’s gene-therapy programs, VY-AADC for Parkinson’s disease and VY-FXN01 for Friedreich’s ataxia, as well as rights to two programs to be determined.

Under the agreement, Neurocrine Biosciences has agreed to pay Voyager $165 million in cash, including a $115-million upfront payment and a $50-million equity investment at a Voyager per share price of $11.96. Voyager will also receive funding from Neurocrine Biosciences for all costs incurred on these collaboration programs as specified per the agreement. In addition, Voyager may be entitled to earn up to $1.7 billion in development, regulatory, and commercial milestone payments across the four programs.

Neurocrine Biosciences has agreed to fund the clinical development of the Phase II-III program for VY-AADC. After the data readout of the Phase II trial, Voyager has the option to either: (1) co-commercialize VY-AADC with Neurocrine Biosciences in the US under a 50/50 cost- and profit-sharing arrangement and receive milestones and royalties based on ex-US sales, or (2) grant Neurocrine Biosciences full global commercial rights in exchange for milestone payments and royalties based on global sales.

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, according to information from the companies. With this approach, Voyager says that VY-AADC has the potential to enhance the conversion of levodopa to dopamine and provide improvements by restoring motor function in patients and improving symptoms following a single administration.

The US Food and Drug Administration has 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.

Under the agreement for VY-FXN01 for Friedreich’s ataxia, Neurocrine Biosciences has agreed to fund the development through the Phase I clinical trial of VY-FXN01. After the data readout of the Phase I trial, Voyager has the option to either: (1) co-commercialize VY-FXN01 with Neurocrine Biosciences in the US under a 60/40 cost- and profit-sharing arrangement, or (2) grant Neurocrine Biosciences full US commercial rights in exchange for milestone payments and royalties based on US sales. Sanofi Genzyme retains an option for ex-US rights to VY-FXN01 following the data readout of the Phase I trial.

Voyager’s VY-FXN01 is for treating Friedreich’s Ataxia, 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.

Under 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 collaboration agreement and the closing of the sale and issuance of Voyager common stock 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.

Source: Neurocrine Biosciences