Skyhawk Therapeutics Partners with Merck to Discover and Develop RNA-Binding Molecules

Partnership handshake

Privately-held Skyhawk Therapeutics struck an agreement with Merck to discover and develop small molecules that modulate RNA splicing as a new modality for the potential treatment of certain neurological diseases and cancer.

Waltham, Mass.-based Skyhawk, which launched last year, said it will use its proprietary SkySTAR technology platform will be employed to discover and develop innovative RNA-binding small molecules. Under terms of the agreement, Skyhawk is eligible to receive approximately $600 million per program target. Bill Hanley, co-founder and chief executive officer of Skyhawk, said the company looks forward to the collaboration as it will demonstrate the ability of SkySTAR to “deliver novel drug candidates for the disease targets Merck has selected and advancing those compounds to address the unmet medical needs of patients.”

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Skyhawk is targeting diseases driven by a type of RNA missplicing called exon skipping, which occurs when key regions on the RNA are left out during the RNA splicing process. Skyhawk's proprietary technology enables the rational design of small molecules that target specific binding pocket regions on RNA, using both sequence and structural specificity, at particular moments in the RNA splicing process. By doing so, they reverse the missplicing and treat the disease, the company said. The SkySTAR (Skyhawk Small molecule Therapeutics for Alternative splicing in RNA) platform integrates information from computational, kinetic and structural models of RNA in order to generate “unique and selective families of chemistry for each target,” the company said.

Under the agreement struck with Merck, Skyhawk will grant the pharma giant the option to exclusively license worldwide intellectual property rights to candidates discovered and developed under the collaboration that are directed to program targets. If Merck exercises that option, the larger company will be responsible for further development and commercialization. Skyhawk will receive an upfront cash payment and, to the extent Merck exercises its option, potential milestone payments and royalties on sales of approved products resulting from the collaboration.

Dean Y. Li, senior vice president, discovery and translational medicine, Merck Research Laboratories, said RNA splicing modification provides a new approach to modulating targets previously considered undruggable. Li said Merck looks forward to its collaboration to “explore the potential of this new modality.”

Merck isn’t the only dance partner for Skyhawk. This morning, the company announced an expansion of its agreement with Biogen. The initial agreement, which include a $74 million upfront payment, was forged in January with the companies using the SkySTAR platform to target neurological diseases. The research is paying off as Skyhawk said the new agreement extends Biogen's exclusive license to worldwide intellectual property rights beyond the original collaboration's research-stage therapeutic candidates for the treatment of conditions including multiple sclerosis, spinal muscular atrophy and additional neurological disorders. As part of the expansion, Skyhawk receives an upfront payment from Biogen and may receive potential future milestone payments and royalties.

In May, Skyhawk and Takeda Pharmaceutical also forged an agreement to use the SkySTAR platform to target neurological diseases.

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