Apellis, Beam ink base-editing pact to tackle complement-driven diseases

Apellis Pharmaceuticals and Beam Therapeutics have forged a five-year research partnership that will apply the latter's base-editing platform toward discovering new treatments for complement-driven diseases, the companies announced Wednesday. The pair said they will collaborate on six programmes focused on C3 and other complement targets. The news sent Beam's stock climbing as much as 18%.

Through the deal, Beam will receive a total of $75 million in upfront and near-term milestones from Apellis. Beam will apply its base-editing technology and conduct preclinical work on programmes that target specific genes within the complement system in various organs, including the eye, liver and brain.

Targeted tweaks 

Base editing represents a potential new class of precision genetic medicine that allows single base changes at targeted genomic sequences without making double-stranded breaks in the DNA. Editing key elements of the complement pathway in target organs may be able to "alter the complement cascade and durably address diseases driven by abnormal complement activity," the companies said.

For its part, Apellis will have exclusive rights to license each of the six programmes, after which it would take over their subsequent development. If it chooses to opt-in, Beam stands to pocket additional development, regulatory and sales milestones, as well as royalty payments on future sales. Beam may elect to enter a 50-50 joint development and commercialisation arrangement with Apellis on one of the licensed programmes. The five-year term of the collaboration may also be extended up to two years on a per year and programme-by-programme basis.

In May, the FDA approved Apellis' Empaveli (pegcetacoplan), which binds to complement protein C3, to treat adults with paroxysmal nocturnal haemoglobinuria. A filing is also under review by EU regulators.