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Voyager Enters Collaboration with Neurocrine to Develop and Commercialize its Gene Therapies for Neurological Diseases

January 9, 2023

Voyager Therapeutics entered a new strategic collaboration with Neurocrine Biosciences to advance multiple gene therapies for the treatment of neurological diseases in a deal potentially valued at up to $1.7 billion.

Photo: Alfred Sandrock, CEO of Voyager

The collaboration includes Voyager’s preclinical, intravenously administered GBA1 gene therapy program for Parkinson’s disease and other GBA1-mediated diseases, which combines a GBA1 gene replacement payload with novel capsids from Voyager’s TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) platform.

In addition, Neurocrine Biosciences and Voyager have agreed to collaborate on three new gene therapy programs directed to rare CNS targets, each also leveraging Voyager’s novel TRACER capsids. The collaboration builds upon the long-standing strategic partnership between Neurocrine Biosciences and Voyager and continues to combine Voyager’s expertise in novel capsid discovery, payload design, and neuropharmacology with Neurocrine Biosciences’ expertise in neuroscience and the clinical and commercial development of therapies for patients suffering from serious neurological diseases.

GBA1 is the gene encoding the lysosomal enzyme glucocerebrosidase, and mutations in this gene have been associated with multiple diseases. Up to 10 percent of Parkinson’s disease patients have a mutation in GBA1, the most common genetic risk factor, increasing the risk of the disease approximately 20-fold. When homozygous, pathologic variants in GBA1 cause the lysosomal disorder, Gaucher disease. AAV-based gene replacement therapies using a blood-brain barrier-crossing capsid have the potential to achieve sustained correction of such disorders affecting the central nervous system. In a GBA loss-of-function preclinical model, Voyager has demonstrated CNS target engagement using its intravenous CNS-tropic capsids and delivery of therapeutically relevant levels of the enzyme GCase, which is encoded by GBA1.

“This new collaboration with Voyager encompassing GBA1-mediated diseases such as Parkinson’s disease and Gaucher’s disease complements our existing collaboration around Friedreich’s ataxia and other CNS targets, establishing the foundation for a strong franchise of next-generation gene therapies utilizing Voyager’s TRACER capsids to treat serious neurological diseases,” said Jude Onyia, chief scientific officer of Neurocrine Biosciences. “We believe GBA1 gene therapy has the potential to play a transformational role in the future treatment of Parkinson’s disease and other serious neurological diseases.”

Under the terms of the agreement, Neurocrine Biosciences has agreed to pay Voyager $175 million up front, of which Neurocrine Biosciences has agreed to pay approximately $136 million in cash and to purchase approximately $39 million of newly issued equity in Voyager at a price of $8.88 per share, which represents a 50 percent premium to the average daily volume-weighted average price of Voyager’s stock over the 30 trading days prior to the execution of the transaction. In addition, Neurocrine Biosciences has agreed to fund all costs incurred under the collaboration, subject to the cost- and profit-sharing option terms below.

Neurocrine Biosciences has agreed to fund development through the completion of a first phase 1 trial. Following the data readout from such trial, Voyager has the right, but not the obligation, to elect to co-develop and co-commercialize the GBA1 program with Neurocrine Biosciences in the U.S. under a 50/50 cost- and profit-sharing arrangement in lieu of receiving further U.S. milestone-based payments and royalties or alternatively be eligible for U.S.-based development, regulatory, and commercial milestone payments and tiered royalties, with Neurocrine Biosciences maintaining responsibility for all development and commercialization expenses.

If Voyager declines its option for cost and profit sharing on the GBA1 program, under the terms of the collaboration agreement, Voyager will be eligible for up to $985 million in total development milestone payments plus substantial potential commercial milestone payments, and tiered royalties ranging from low double-digit to twenty percent on U.S. net sales. Irrespective of Voyager’s election on its cost- and profit-sharing option, Voyager shall be eligible for potential ex-U.S.-based regulatory and commercial milestone payments, as well as royalties ranging from high-single-digits to mid-teens on ex-U.S. net sales.

Voyager is eligible to earn up to $175 million in development milestone payments plus substantial potential commercial milestone payments for each of the three new gene therapy programs in the collaboration, and tiered high single-digit to mid-teens royalties on U.S. net sales and mid-single-digit to low double-digit royalties on ex-U.S. net sales. Neurocrine Biosciences has agreed to fully fund the development of the three new programs.

Following the completion of the transaction, Jude Onyia will join Voyager’s board of directors.

“This collaboration illustrates the value-creation opportunity presented by combining Voyager’s novel TRACER capsid platform with our deep knowledge of neuropharmacology and payloads to advance next-generation gene therapies for CNS diseases,” said Alfred Sandrock, CEO of Voyager.

Author: Rare Daily Staff

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