Merck KGaA and Skyhawk Therapeutics Announce $2B+ Strategic Collaboration to Develop Novel RNA-Targeting Small Molecules for Neurological Disorders

August 18,Boston,Skyhawk TherapeuticsSkyhawk Therapeutics, Inc., a leader in discovering and developing small molecules that modulate RNA expression, announced a strategic R&D collaboration with Merck KGaA. The collaboration will focus on developing novel RNA small molecules for specific neurological indications with high unmet medical needs.New drug. (Over $2 Billion! Sanofi and Skyhawk Collaborate on RNA Small Molecule Drug Development）

According to the terms of the agreement,SkyhawkWill use its proprietary SkySTAR® (for RNA selective splicingSkyhawkSmall Molecule New Drug) Platform to Identify AgainstMerck KGaASmall molecule candidates targeting specific RNA targets. The collaboration aims to leverageSkyhawkIndustry-leading capabilities in RNA splicing modulation, combined with Merck KGaA's expertise in drug development and commercialization, expand the potential of RNA modulation in diseases where traditional approaches are challenging.

SkyhawkWill lead discovery and preclinical development efforts, after which Merck KGaA will be responsible for further development and commercialization following the exercise of the option. The total deal is valued at over $2 billion, with Skyhawk Therapeutics eligible to receive milestone payments as well as tiered royalties on commercial sales.

Amy Kao, Senior Vice President of Merck KGaA and Global Head of Neuroscience and Immunology Research, stated:"We are withSkyhawkThe collaboration aligns with our strategic focus on innovative science and next-generation technologies that have the potential to deliver impactful medicines for patients with neurological disorders. We believe RNA splicing modulation represents an exciting frontier in drug discovery.Skyhawk"Their expertise makes them an ideal partner in this field. This also aligns with our mission to bring more drugs to more patients faster."

Bill Haney, CEO of Skyhawk Therapeutics, stated:"We are proud to collaborate with Merck KGaA, a company with a strong tradition of innovation and a commitment to advancing transformative science. This collaboration highlights the capability of our SkySTAR® platform to address challenging disease biology through precise RNA targeting, and we look forward to working closely with Merck KGaA to introduce potential first-in-class medicines."

Skyhawk Therapeutics, Inc. has raised over $679 million, of which more than $479 million came from upfront payments in pharmaceutical company collaborations, with the remainder coming from equity investors, including an $8 million seed round in 2018 and a $133 million latest round of financing in 2021. For a company that has not yet entered the clinical stage, this is a significant amount of funding. Skyhawk has partnered with Merck & Co., BMS ($60 Million Upfront Payment）、Biogen（$75 million upfront payment), Takeda, Genentech, and Vertex ($40 Million Upfront Payment + $2.2 Billion Milestones) to reach a cooperation. In addition, in 2024Ipsen and Skyhawk Therapeutics Reach $1.8 Billion Collaboration.

Skyhawk, Arrakis Therapeutics, and Remix Therapeutics have been betting that they can alter the levels of disease-driving proteins by targeting upstream RNA.

Neuroscience is a key focus of Skyhawk's internal pipeline, which is led by a Phase 2/3 splicing modulator aimed at treating Huntington's disease by reducing the production of mutant HTT. In addition to neurology, Skyhawk is also developing preclinical programs targeting lymphoma and fibrosis.