Aceragen Launches with Acquisition of Enzyvant‘s Investigational Therapy for Farber Disease and $35 Million Product Financing with NovaQuest
Retrieved on:
Monday, May 3, 2021
Biotechnology, Pharmaceutical, Health, Other Health, Drug discovery, Pharmaceutical industry, Preclinical development, Farber, Farber disease and Acid Ceramidase Deficiency, Aceragen, NovaQuest Capital Management, Enzyvant, FARBER DISEASE AND ACID CERAMIDASE DEFICIENCY, ACERAGEN, NOVAQUEST CAPITAL MANAGEMENT, ENZYVANT
Enzyvant will receive an upfront payment and development and sales-based milestones up to $226 million, as well as tiered royalties on net sales.
Key Points:
- Enzyvant will receive an upfront payment and development and sales-based milestones up to $226 million, as well as tiered royalties on net sales.
- Preclinical development for rhAC has been completed, including in vivo proof of concept in a mouse model of Farber disease, and IND-enabling GLP toxicology studies.
- Patients with the most rapidly progressive form of Farber disease usually do not live beyond the first few years of life.
- Enzyvant\xe2\x80\x99s lead asset is the investigational tissue-based regenerative therapy, RVT-802, for congenital athymia, an ultra-rare and life-threatening pediatric immunodeficiency.