Type of information: Development agreement
Compound: VY-AADC, VY-FXN01
Company: Voyager Therapeutics (USA-MA) Neurocrine Biosciences (USA - CA)
Therapeutic area: Rare diseases - Neurological diseases - Neurodegenerative diseases
Type agreement: development, commercialization
Action mechanism: gene therapy. VY-AADC is an adeno-associated viral vector serotype 2 encoding human aromatic L-amino acid decarboxylase (AAV2-hAADC). It is 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 express the AADC enzyme to convert levodopa into dopamine. The approach with VY-AADC, therefore, has the potential to durably enhance the conversion of levodopa to dopamine and provide clinically meaningful improvements by restoring motor function in patients and improving symptoms following a single administration. VY-FXN01 is an adeno-associated viral vector that delivers a functional version of frataxin (FXN) gene to the sensory pathways through intrathecal or intravenous injection.
Disease: Parkinson’s Disease - Friedreich’s Ataxia
Details: • On January 29, 2019, Neurocrine Biosciences and Voyager Therapeutics announced the formation of a strategic collaboration 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. This collaboration combines Neurocrine Biosciences' expertise in neuroscience, drug development and commercialization with Voyager's innovative gene therapy programs targeting severe neurological diseases.
Financial terms: Under the terms of 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 described below. In addition, Voyager may be entitled to earn up to $1.7 billion in development, regulatory and commercial milestone payments across the four programs. Under terms of the agreement for VY-AADC for Parkinson's disease: Neurocrine Biosciences has agreed to fund the clinical development of the Phase 2-3 pivotal program for VY-AADC. After the data readout of the Phase 2 RESTORE-1 trial, Voyager has the option to either: (1) co-commercialize VY-AADC with Neurocrine Biosciences in the U.S. under a 50/50 cost- and profit-sharing arrangement and receive milestones and royalties based on ex-U.S. sales, or (2) grant Neurocrine Biosciences full global commercial rights in exchange for milestone payments and royalties based on global sales. Under terms of the agreement for VY-FXN01 for Friedreich's ataxia: Neurocrine Biosciences has agreed to fund the development through the Phase 1 clinical trial of VY-FXN01. After the data readout of the Phase 1 trial, Voyager has the option to either: (1) co-commercialize VY-FXN01 with Neurocrine Biosciences in the U.S. under a 60/40 cost- and profit-sharing arrangement, or (2) grant Neurocrine Biosciences full U.S. commercial rights in exchange for milestone payments and royalties based on U.S. sales. Sanofi Genzyme retains an option for ex-U.S. rights to VY-FXN01 following the data readout of the Phase 1 trial. Under terms of 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.