Aevitas Therapeutics, a Fortress Biotech subsidiary company, and 4D Molecular Therapeutics (4DMT), a clinical-stage biotherapeutics company harnessing the power of directed evolution for genetic medicines targeting large-market diseases, have announced the execution of an asset purchase agreement for 4DMT to acquire Aevitas’ proprietary rights to its short-form human complement factor H (sCFH) asset for the treatment of complement-mediated diseases.
Under the terms of the agreement, 4DMT will make cash payments to Aevitas totaling up to ~$140 million in potential late-stage development, regulatory and sales milestones. A range of single-digit royalties on net sales are also payable. The aforementioned payments are payable solely to Aevitas, and 4DMT will be responsible for license payment obligations to University of Pennsylvania, where the sCFH technology was co-invented and co-developed by Dr. Wenchao Song, a professor of Pharmacology at the Perelman School of Medicine.
Lindsay A. Rosenwald, M.D., Fortress’s chairman, president and Chief Executive Officer and Aevitas’ executive chairman, said: “This agreement with 4DMT allows Fortress to focus on acquiring and developing clinical-stage treatments, while potentially expediting the development and commercialization of this preclinical sCFH technology.
“Partnering with 4DMT further validates the Fortress business model of identifying and developing promising treatments for patients, while pursuing opportunities that potentially maximize shareholder value. We look forward to 4DMT using their vector platform to continue the development of the sCFH asset to potentially treat Geographic Atrophy (GA) and other diseases.”
Aevitas’ transgene encoding sCFH, a shortened and optimized form of a natural inhibitor of the inflammatory complement pathway, will be combined with 4DMT’s proprietary retinotropic R100 vector to form product candidate 4D-175 for treatment of GA secondary to age-related macular degeneration (AMD).
sCFH is an engineered and optimized version of Complement Factor H (CFH) that can fit into AAV vectors with robust expression and functionality confirmed in cultured human cells in vitro, and in multiple preclinical animal models in vivo. Restoring CFH function using the sCFH protein has the potential to restore normal complement regulation and reduce retinal injury that manifests as progressive GA.
“We are pleased to execute this asset purchase agreement with Aevitas to combine an innovative and differentiated preclinical GA product candidate into our large market ophthalmology portfolio which leverages our clinically validated R100 retinotropic vector,” said David Kirn, M.D., co-founder and Chief Executive Officer of 4DMT. “This represents continued value generation from our robust product design and development engine to take advantage of the vector modularity of our platform in the ophthalmology therapeutic area.”