Merck doubles down on Skyhawk’s tech to drug RNA, promising $600M per autoimmune, metabolic target

Early collaborations with big-name partners lent Skyhawk Therapeutics both the endorsements and the capital to pursue a small molecule platform for drugging RNA. And they soon wanted more. When Merck signed on to discover candidates against neurodegeneration and cancer last July, Biogen was just expanding the list of neuro targets they want to go after.

Now Merck is coming back for its own round two, adding autoimmune and metabolic diseases to the menu.

Kathleen McCarthy

As in the original deal, the pharma giant isn’t breaking down the upfront, sticking to the $600 million per program figure to illustrate their interest here. Licenses to any IP arising from the collaboration goes to Merck, which would take over development once it exercises an option.

At the core of Skyhawk’s platform is RNA splicing modification — a way to coax dysfunctional genes into producing functional proteins without altering them permanently. Kathleen McCarthy, its co-founder and CSO, was part of a team that hit on their phenomenon by reverse engineering how risdiplam, an SMA drug now shepherded by Roche and PTC Therapeutics, worked.

The approach offers a handle on undruggable targets, said Dean Li, SVP of discovery sciences and translational medicine at Merck Research Laboratories.

Bill Haney

Bill Haney, the angel investor and CEO, bet that it could apply broadly beyond rare diseases. Multiple drug developers have since chimed in over $300 million upfront to match the $8 million seed round he initially put together with some unconventional backers.

“Merck has been a wonderful partner in discovering novel drug candidates for neurological diseases and cancer,” Haney said in a statement.

Other biotechs have sprung up in the RNA modulation space, including Ribometrix, which counts Merck as an investor, as well as Arrakis, where Michael Gilman has stuck to a more measured approach on the BD front.