Neurocrine Commits $175M to Voyager in New Neuro Gene Therapy Alliance - MedCity News

Two years after Neurocrine Biosciences pulled out of a Parkinson’s disease gene therapy partnership with Voyager Therapeutics, the neuroscience company is back to launch a new pact for multitherapy covering Parkinson’s disease and other central nervous system (CNS) diseases.

Neurocrine kicks off the alliance with a $175 million payment, an amount that gives the San Diego-based biotech global rights to a preclinical gene therapy that targets a specific genetic signature. The deal also includes three additional Voyager therapeutic programs for rare CNS targets. Neurocrine has agreed to fund all costs of the collaboration.

The genetic target for the flagship program is GBA1. Mutations in this gene are a known risk factor for neurodegenerative disorders caused by misfolded forms of a protein called alpha synuclein. Cambridge, Massachusetts-based Voyager aims to treat such diseases by replacing the mutated gene.

Approximately 10% of Parkinson’s patients have GBA1 mutations, according to Voyager. These mutations reduce the expression of an enzyme important for regulating lipid metabolism in cells. Insufficient levels of this enzyme lead to the accumulation of alpha synuclein, which is thought to be toxic to neurons. The treatment of Parkinson’s disease patients with GBA1 mutations is the lead indication for the Voyager gene therapy. Other disorders caused by a GBA1 mutation that can affect the CNS include dementia with Lewy bodies and Gaucher disease.

Voyager’s gene therapies come from the company’s proprietary TRACER technology. This platform discovers novel capsids, protein shells encapsulating the genetic cargo of gene therapy. These shells are used to deliver therapy to certain types of tissue. The alliance with Neurocrine is focused on bringing gene therapies into the brain. Pharmaceutical industry partners using Voyager technology to deliver gene therapy to the brain include Pfizer and Novartis.

Voyager and Neurocrine began working together in 2019, an alliance that spans four programs. The most advanced of these, a Parkinson’s gene therapy called NBlb-1817, has reached Phase 2 testing. But safety concerns have prompted the FDA to place a clinical hold on this gene therapy. in in early 2021, Neurocrine announced that it would terminate its alliance under this program.

The upfront payment to Voyager for the new alliance comes down to $136 million in cash and the purchase of about $39 million worth of the biotech’s stock. Voyager’s $8.88 purchase price represents a 50% premium to Voyager’s average stock price over the last 30 days. The milestone payments could bring Voyager up to $1.5 billion in additional payments, plus sales royalties on any commercialized products.

The new deal with Neurocrine allows Voyager to share the costs and potential profits of the GBA1 program. Voyager can exercise that option after the Phase 1 data is read. Expect the company to provide more details about the deal later this week. Voyager has a the presentation is scheduled for Thursday during the annual JP Morgan Healthcare conference in San Francisco.

Voyager and Neurocrine aren’t the only companies pursuing therapies targeting GBA1. Eli Lilly subsidiary Prevail Therapeutics advances its GBA1 gene therapy to split mid-stage tests in Parkinson’s and Gaucher. Startup Vanqua Bio is developing a small-molecule drug that treats Parkinson’s patients who have GBA1 mutations.

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