Apellis Pharmaceuticals recently won its first FDA approval for a drug that treats a rare blood disorder by hitting a specific target in the complement system, a family of proteins that helps cells of the immune system. The company wants to bring complement system drugs to other diseases in other parts of the body and it is turning to the gene-editing technology of Beam Therapeutics for help.
On Wednesday, the companies announced a research partnership that will apply Beam’s base-editing technology to undisclosed complement system targets. Beam will handle the preclinical research for the up to six programs targeting genes within the complement system. Those genes are in organs that include the eye, liver, and brain. Apellis has the right to license each of the programs and will be responsible for clinical development if it exercises those rights.
Waltham, Massachusetts-based Apellis is paying Beam $50 million up front, plus another $25 million on the one-year anniversary of the contract. The $75 million sum is described as a combination of upfront and near-term milestone payments. Beam, which is headquartered in Cambridge, Massachusetts, can choose to share equally in the development and commercialization of one of the licensed programs.
“This collaboration allows us to combine our proprietary technologies and capabilities in base editing with Apellis’s expertise in targeting the complement pathway to develop new medicines for diseases driven by complement biology,” Beam CEO John Evans said in a prepared statement. “This also represents an important strategic initiative to explore opportunities that expand the application of base editing to address more biologically complex diseases for patients in need of new treatment options.”
In May, the FDA approved Apellis drug Empaveli as a treatment for paroxysmal nocturnal hemoglobinuria (PNH), a disease in which the complement system attacks red blood cells, leading to blood clots and organ damage. Empaveli targets a complement system called C3. The new partnership could develop additional therapies that block C3. But Apellis and Beam say that base editing could also expand complement system-targeting therapies to other proteins in the complement cascade.
Beam’s base-editing technology offers a more precise way of editing the genome. Unlike the edits of CRISPR, which makes double-stranded breaks in DNA, base editing can change individual letters of the genome. The companies say that applying this precise approach to editing has the potential to durably alter the complement cascade to address diseases caused by complement system dysfunction. The Apellis/Beam alliance comes four months after Beam announced its $120 million acquisition of Guide Therapeutics, a startup whose technology identifies the lipid nanoparticles best suited for delivering a genomic medicine to particular tissue types.
Apellis and Beam did not disclose the diseases that they aim to treat. For each program Apellis licenses under the partnership, Beam will be eligible for additional milestone payments, plus royalties from sales if any of them reach the market. The value of those additional milestone payments was not disclosed. The collaboration spans five years but may be extended for up to two years on a per year and program-by program-basis.
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