Type of information: Development agreement
Compound: gene therapy programs for Parkinson’s disease, Friedreich’s ataxia, Huntington’s disease and other CNS disorders
Company: Genzyme (USA - MA), a Sanofi company (France) Voyager Therapeutics (USA - MA)
Therapeutic area: CNS diseases - Neurodegenerative diseases
Type agreement: development - commercialisation
Action mechanism: gene therapy
Disease: Parkinson’s disease, Friedreich’s ataxia and Huntington’s disease and other CNS disorders
Voyager will drive research and development activities for all programs, working with Genzyme in a highly collaborative way. Genzyme will have the option to license several programs following completion of an initial proof-of-concept human clinical trial. However, Voyager will retain all U.S. rights to its lead product programs in Parkinson’s disease (VY-AADC01) and Friedreich’s ataxia (VY-FXN01). Voyager will split U.S. profits with Genzyme for the Huntington’s disease program (VY-HTT01). In addition, Voyager’s lead amyotrophic lateral sclerosis (ALS) program (VY-SOD101) is not part of the collaboration and Voyager retains worldwide rights to this program.
- • On June 17, 2019, Voyager Therapeutics announced a restructuring of its gene therapy relationship with Sanofi Genzyme. Under the terms of the agreement, Voyager gains worldwide rights to the VY-HTT01 Huntington’s disease program and ex-U.S. rights to the VY-FXN01 Friedreich’s ataxia program. The ex-U.S. 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. Additionally, Sanofi Genzyme obtains exclusive option rights to select novel AAV capsids owned or controlled by Voyager for exclusive use for up to two non-central nervous system (non-CNS) indications.
In consideration of the rights returned, Voyager has agreed to make a $10 million upfront payment to Sanofi Genzyme. This upfront payment is partially offset by a $5 million payment from Neurocrine Biosciences to Voyager to facilitate the transfer of the ex-U.S. rights to VY-FXN01 from Voyager to Neurocrine Biosciences. An additional $10 million milestone payment is due to Sanofi Genzyme from Voyager upon filing of an investigational new drug (IND) application for VY-HTT01 or, if applicable, certain backup compounds for the treatment of Huntington’s disease. Preclinical studies are underway with VY-HTT01 which, if successful, are expected to support a potential filing of an IND application in late 2019.
In connection with the restructuring of the gene therapy relationship with Sanofi Genzyme, and to focus its resources on the now wholly owned Huntington’s disease program and additional new discovery efforts, Voyager intends to seek a partner to advance its preclinical program for SOD1 ALS. Given this portfolio decision, Voyager no longer expects to file an IND application for VY-SOD102 in 2019.
- The companies have agreed to terminate Sanofi Genzyme’s option rights and joint efforts under the original 2015 collaboration agreement and amend and restate a separate agreement focused on the discovery and development of novel AAV capsids for use in programs to be developed and commercialized by Sanofi Genzyme. The terms of the restructured relationship are summarized below:Sanofi Genzyme’s options to acquire U.S. co-commercialization rights and ex-U.S. development and commercialization rights to VY-HTT01 for the treatment of Huntington’s disease are terminated. Voyager now holds worldwide rights to VY-HTT01 for the treatment of Huntington’s disease.
Sanofi Genzyme’s option to acquire ex-U.S. development and commercialization rights to the VY-FXN01 Friedreich’s ataxia program is terminated. Ex-U.S. rights to this program are transferred from Voyager to Neurocrine Biosciences as provided under an amended collaboration agreement between Voyager and Neurocrine Biosciences.
Sanofi Genzyme’s option to acquire development and commercialization rights to a future Voyager CNS orphan program is terminated.
The collaboration between Voyager and Sanofi Genzyme concerning spinal muscular atrophy (SMA) is terminated, and intellectual property rights under the collaboration to the SMA program are returned or exclusively licensed to Sanofi Genzyme.
Voyager has agreed to make a $10 million upfront payment to Sanofi Genzyme and an additional $10 million milestone payment to Sanofi Genzyme upon the potential filing of an IND application for VY-HTT01 or, if applicable, certain backup compounds for the treatment of Huntington’s disease. Voyager has also agreed to pay low-single-digit royalties to Sanofi Genzyme on Voyager’s worldwide net sales of such product candidate for the treatment of Huntington’s disease.
Voyager grants to Sanofi Genzyme exclusive options to select Voyager-owned or controlled AAV capsids from Voyager’s novel AAV capsid development efforts for exclusive use in up to two non-CNS indications. In connection with any potential exercise by Sanofi Genzyme of such options, Voyager is entitled to receive option exercise payments, development, regulatory, and commercial milestone payments, and low- to mid-single-digit royalties on worldwide net sales of products containing licensed capsids.
• On February 11, 2015, Voyager Therapeutics, a gene therapy company developing life-changing treatments for fatal and debilitating diseases of the central nervous system (CNS), and Genzyme, a Sanofi company and gene therapy pioneer, announced a major strategic collaboration to discover, develop and commercialize novel gene therapies for severe CNS disorders. The collaboration will leverage Genzyme’s long-standing commitment and scientific leadership in the field of adeno-associated virus (AAV) gene therapy and Voyager’s industry-leading AAV product engine to develop breakthrough therapies for patients suffering from severe CNS disorders. The alliance will encompass multiple gene therapy programs, including programs for Parkinson’s disease, Friedreich’s ataxia and Huntington’s disease, as well as other CNS disorders. Each program targets a severe, debilitating disease and has the potential to deliver transformational therapeutic benefit for patients. The collaboration portfolio created will combine programs and intellectual property from both companies.
- Genzyme will make an upfront commitment of $100 million to Voyager, including $65 million in cash, a $30 million equity investment in Voyager and additional in-kind contributions. Voyager is eligible to receive future potential development and sales milestone payments of up to $745 million, as well as tiered royalties on product sales. Goodwin Procter served as legal advisor to Voyager.
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