VY-FXN01 / Voyager, Sanofi - LARVOL DELTA

Voyager Therapeutics : Management's Discussion And Analysis of Financial Condition and Results of operations (Form 10-Q) (Market Screener) - "We and Neurocrine are in the process of identifying a lead candidate which will comprise an optimal capsid, promoter, and FXN transgene....To evaluate the therapeutic potential of our vectors, we have conducted testing in a new genetic mouse model of Friedreich's ataxia. In this preclinical model of Friedreich's ataxia, our gene therapy candidates durably improved sensory function, and rescued the Friedreich's ataxia phenotype....Once we and Neurocrine identify a lead candidate for this program, we plan to complete IND enabling studies to evaluate its safety and efficacy."

Preclinical • Friedreich ataxia

Neurocrine Biosciences reports third quarter 2019 financial results (PRNewswire) - "During the first quarter of 2019, Neurocrine Biosciences formed a strategic collaboration with Voyager Therapeutics focused on the development and commercialization of Voyager's gene therapy programs...VY-FXN01 for Friedreich's ataxia, as well as rights to two programs to be determined."

Licensing / partnership

Voyager Therapeutics is a speculative bet on gene therapies (SeekingAlpha) - "Neurocrine has agreed to fund the development of VY-FXN01 through phase 1. After data readout, Voyager has the option to either: co-commercialize VY-FXN01 in the US under a 60/40 cost and profit sharing arrangement, or grant Neurocrine full US rights in exchange for milestone payments and royalties based on US sales."

Financing

Voyager Therapeutics Announces Restructured Gene Therapy Relationship with Sanofi Genzyme and Portfolio Update (GlobeNewswire, Voyager Therapeutics, Inc.) - Gains worldwide rights to VY-HTT01 for Huntington’s disease. Resources to be reallocated to VY-HTT01 and other programs.. rights to the VY-FXN01 Friedreich’s ataxia program. rights to VY-FXN01 are, in turn, transferred from Voyager to Neurocrine Biosciences under the terms of the collaboration agreement between Voyager and Neurocrine Biosciences announced in January 2019.

Commercial • Preclinical • Regulatory

Voyager Therapeutics Reports Fourth Quarter and Full Year 2018 Financial Results and Corporate Highlights (GlobeNewswire, Voyager Therapeutics, Inc.) - "Initiated RESTORE-1 Phase 2 trial for VY-AADC for Parkinson’s disease...and a planned Phase 3 trial (RESTORE-2) of similar size and design to RESTORE-1 incorporate guidance from the FDA from the Type B meeting to conduct two adequate and well-controlled clinical trials for a large patient population such as Parkinson’s disease...Advance Friedreich’s ataxia program VY-FXN01 towards identifying a lead candidate and IND-enabling preclinical pharmacology and toxicology studies."

New P2 trial • New P3 trial • Preclinical