TARRYTOWN, N.Y. and MENLO PARK, Calif.—Regeneron Pharmaceuticals Inc. and Avalanche Biotechnologies, Inc. have struck a broad collaboration for the discovery, development and commercialization of novel gene therapy products for treating ophthalmologic diseases. The collaboration includes novel gene therapy vectors and proprietary molecules discovered jointly by the two companies and developed by the proprietary Avalanche Ocular BioFactory platform, an adeno-associated virus (AAV)-based, next-generation platform for the discovery and development of gene therapy vectors for ophthalmology. The platform utilizes the body’s own cells to continue producing therapeutic protein after one injection.
Per the terms of the agreement, Regeneron will pay Avalanche an upfront cash payment, as well as contingent payments of up to $640 million if certain development and regulatory milestones are achieved. Avalanche is also eligible to receive a royalty on worldwide net sales of products resulting from the collaboration. The deal covers up to eight therapeutic targets, and Regeneron will have exclusive worldwide rights for each product it advances in clinical development, while Avalanche has the option to share in development costs and profits for products directed toward two collaboration targets it selects.
“We look forward to the opportunity to collaborate with Avalanche, a leader in the field of next-generation gene therapy technologies,” Dr. George D. Yancopoulos, chief scientific officer of Regeneron and president of Regeneron Laboratories, said in a press release. “This collaboration highlights the commitment by Regeneron to invest in potentially breakthrough therapies that could benefit patients with sight-threatening diseases.”
In addition, this collaboration gives Regeneron a time-limited right of first negotiation for certain rights to AVA-101, Avalanche’s gene therapy product that targets vascular endothelial growth factor, upon completion of its ongoing Phase 2a trial. The compound is currently being developed as a potential treatment for wet age-related macular degeneration.
“We are excited to work with Regeneron to discover and develop novel gene therapy medicines for serious eye diseases. The collaboration will bring together Avalanche’s novel platform technology with Regeneron’s proprietary molecules and research capabilities, with the goal of creating a new class of next-generation biologics in ophthalmology. Regeneron is a terrific partner for their scientific leadership, as well as their product development capabilities and commercialization track-record,” Dr. Thomas W. Chalberg, co-founder and CEO of Avalanche, commented in a statement.
Avalanche recently announced the completion of a $55-million Series B financing, the proceeds from which will be used to advance a number of its clinical programs, including AVA-101, toward manufacturing and clinical infrastructure for AVA-101 and to accelerate the development of pipeline programs based on the BioFactory platform.
The financing was led by Venrock, with new investors consisting of Deerfield, Adage Capital Management, Redmile Group, Rock Springs Capital, Sabby Capital, an affiliate of Cowen & Company and two undisclosed blue-chip healthcare funds. Cowen & Company served as the sole placement agent for the financing.