Merck KGaA swoops into Skyhawk neurological disease pact worth up to $2B

Merck KGaA has swooped into a neurological-focused pact with RNA biotech Skyhawk Therapeutics that could top out at $2 billion.

Under the agreement, Skyhawk will use its RNA splicing platform, dubbed SkySTAR, to identify small molecule candidates directed at specific RNA targets selected by Germany-based Merck. 

While the companies didn’t drill into the exact neurological indications being considered, the collaboration is designed to “expand the potential of RNA modulation in diseases where traditional approaches have proven challenging," Skyhawk said in an Aug. 18 press release.

Skyhawk will oversee this discovery and preclinical work, with Merck having an option to pay to take the candidates into human trials and beyond. 

Payments to Skyhawk’s could eventually stretch to $2 billion—although the release didn’t offer a breakdown of the terms of the deal—on top of tiered royalties on sales should any drugs make it to market.

“Our collaboration with Skyhawk aligns with our strategic focus on innovative science and next-generation technologies that have the potential to deliver impactful medicines to patients with neurological conditions,” Amy Kao, M.D., senior vice president and global head of the neuroscience and immunology research unit at Merck KGaA, said in the release.

“We believe RNA splicing modulation represents an exciting frontier in drug discovery, and Skyhawk's expertise positions them as an ideal partner in this space,” Kao added.

Going after so-called "undruggable" disease targets, Skyhawk and peers such as Arrakis Therapeutics and Remix Therapeutics have been betting they can alter levels of disease-driving proteins by hitting RNA upstream.

Neuroscience is a focus of Skyhawk’s internal pipeline, which is led by a phase 2/3-stage splicing modulator designed to treat Huntington's disease by reducing the production of mutated HTT. Beyond neurology, Skyhawk also has preclinical programs for lymphoma and fibrosis in the works.

Meanwhile, Germany’s Merck isn’t the first pharma—or even the first Merck—to spot potential in Skyhawk’s RNA splicing tech. U.S.-based Merck & Co. penned a pair of deals with the biotech over the course of 2019 and 2020 to work across autoimmune, metabolic and neurological diseases.

Other Skyhawk collaborators have included Vertex, Ipsen and Takeda.

Today's agreement “underscores the power of our SkySTAR platform to address challenging disease biology through precise RNA targeting, and we look forward to working closely with Merck KGaA … to bring potential first-in-class medicines forward," Skyhawk CEO Bill Haney said in a statement.