Pharma BD Deal Intelligence
Neurocrine's $1.7B (up to) collaboration with Voyager gave it two CNS gene therapies—VY-AADC for Parkinson's and VY-FXN01 for Friedreich's ataxia—for $165M upfront, and it's a mixed bag: the lead Parkinson's asset was killed in 2021 after an FDA clinical hold, while the Friedreich's ataxia program advanced to a 2026 Phase 1 start.
Neurocrine exits $165M Parkinson pact with Voyager after FDA hold; original 2019 deal valued at up to $1.7 billion.
Second Neurocrine-Voyager pact targets underlying cause of Parkinson with $175M upfront plus $1.5B milestones.
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In January 2019 Neurocrine Biosciences formed a strategic development and commercialization collaboration with Voyager Therapeutics covering Voyager's CNS gene therapy programs: VY-AADC for Parkinson's disease and VY-FXN01 for Friedreich's ataxia, plus two additional TBD programs. Voyager received $165M upfront ($115M cash + a $50M equity investment at $11.96/share) and was eligible for up to $1.7B in development, regulatory and commercial milestones. Outcome to date is mixed: Neurocrine terminated the lead Parkinson's program (VY-AADC) in August 2021 after the FDA placed a clinical hold tied to MRI abnormalities. The Friedreich's ataxia program advanced — Neurocrine/Voyager selected NBIB-'223 (a TRACER capsid-delivered frataxin gene therapy) as the development candidate in February 2024, the program subsequently received FDA orphan drug designation and completed IND-enabling work, and Neurocrine intends to start a Phase 1 trial in the second half of 2026 pending FDA IND clearance.
90K US cases/yr · $6.6B Global PD Therapeutics (2024)
Gene therapy for Parkinson's targets the dopamine synthesis pathway. AAV-AADC gene therapy delivers the AADC enzyme gene directly to the putamen, enabling endogenous dopamine production from levodopa. Multiple programs are in clinical development.
Neurocrine/Voyager VY-AADC and Bayer/AskBio AB-1005 both target AADC gene delivery via AAV vectors. Challenges include surgical delivery, immune response to AAV, and durability of expression. Early clinical data has shown improvement in motor function and reduced levodopa requirements.
600 US cases/yr · $500M Estimated Global Market Opportunity
Friedreich's ataxia is an autosomal recessive neurodegenerative disease caused by GAA repeat expansion in the frataxin (FXN) gene. It causes progressive ataxia, cardiomyopathy, and scoliosis, typically with onset before age 25. Skyclarys (omaveloxolone) was approved in 2023 as the first treatment.
Gene therapy aims to deliver a functional frataxin gene to restore protein expression. Voyager's VY-FXN01 uses AAV to target the dorsal root ganglia and heart. The approach addresses the root cause rather than downstream effects. Challenge is achieving sufficient frataxin expression in both CNS and cardiac tissue.
Neurocrine paid $165M upfront for VY-AADC (Parkinson) and VY-FXN01 (Friedreich ataxia) programs.
FDA placed clinical hold after MRI abnormalities. Neurocrine terminated Parkinson program Aug 2021.
NBIB-'223 (frataxin gene therapy) received FDA orphan drug designation and completed IND-enabling studies; Neurocrine intends to initiate a Phase 1 trial in H2 2026 pending IND clearance.
VY-AADC terminated but Friedreich ataxia (NBIB-223) advancing to Phase 1. Partial collaboration continues.
| Deal | Year | Value | Outcome |
|---|---|---|---|
| Neurocrine Biosciences Inc. / Voyager Therapeutics Inc. (this deal) | 2019 | $1.9B | 54 |
| Neurocrine Biosciences Inc. / Takeda Pharmaceutical Company Ltd. | 2025 | — | — |
| Neurocrine Biosciences Inc. / Soleno Therapeutics, Inc. | 2026 | $2.9B | — |
| Johnson & Johnson / Legend Biotech Corporation | 2017 | $9.0B | 96 |
| Vertex Pharmaceuticals Incorporated / Semma Therapeutics Inc. | 2019 | $950M | 86 |
| Celgene Corporation / Bristol-Myers Squibb Company | 2015 | $2.0B | 85 |
| Vertex Pharmaceuticals Incorporated / CRISPR Therapeutics AG | 2015 | $2.6B | 85 |
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More: 2019 deals · Neurocrine Biosciences Inc. deals · Gene Therapy deals