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May 5, 2014

Regeneron to Use Avalanche Platform in $640M+ Gene Therapy Collaboration

  • Regeneron Pharmaceuticals will use Avalanche Biotechnologies’ adeno-associated virus (AAV)-based drug-delivery platform in a collaboration to commercialize its lead drug and new gene therapy products for ophthalmologic diseases, the companies said today. The collaboration could net Avalanche more than $640 million. 

    Under the collaboration, Regeneron will apply the Avalanche Ocular BioFactory™ to discover and develop gene therapy vectors for ophthalmology. BioFactory is designed to use the body's own cells to produce therapeutic protein after only a single injection. That in turn blocks VEGF signaling, treating the disease.

    Avalanche says its platform reaches peak expression after 4-6 weeks, at which point the treatment can be maintained on an ongoing basis with a continuous, steady-state level of therapeutic protein, vs. a half-dozen or so re-injections a year with current treatments. Based on preclinical studies, Avalanche says the therapeutic effect from its lead product AVA-101 can be maintained for at least 18 months and has potential to last for several years following treatment from a single injection.

    Regeneron agreed to pay Avalanche an undisclosed upfront cash payment, contingent payments of up to $640 million tied to development and regulatory milestones, plus a royalty on worldwide net sales of collaboration products. 

    The collaboration covers up to eight distinct therapeutic targets, with Regeneron holding exclusive worldwide rights for each product it moves forward in clinical development. Avalanche has the option to share in development costs and profits for products directed toward two collaboration therapeutic targets it will select.

    Regeneron has a time-limited right of first negotiation for certain rights to AVA-101 upon completion of an ongoing Phase IIa trial. AVA-101 is Avalanche's gene therapy product targeting vascular endothelial growth factor (VEGF), now under development for the treatment of wet age-related macular degeneration (AMD).

    "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,” Thomas W. Chalberg, Ph.D., Avalanche’s co-founder and CEO, said in a statement.

    Avalanche’s collaboration with Regeneron comes almost two weeks after Avalanche completed a $55 million Series B financing intended to advance AVA-101 and other company clinical programs in retinal disorders. Avalanche said it will also use proceeds from the financing to invest in manufacturing and clinical infrastructure for AVA-101, as well as accelerate the development of pipeline programs based on the BioFactory platform.


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