Merck & Co. Inc. has tapped Skyhawk Therapeutics Inc. for its expertise in the discovery and development of small molecules that modulate RNA splicing, agreeing to pay it up to $600 million per program target plus royalties on sales of any commercialized products of the collaboration. The deal, focused on potential treatments for certain neurological diseases and cancer, was accompanied by news of an expansion of Skyhawk's collaboration with Biogen Inc., which originally signed on with the Waltham, Mass-based company in January.

The deal marks Skyhawk's first external oncology collaboration following two years of work on its own internal oncology target, reflecting a deliberate move into the space that it bolstered with the recruitment of new key talents. Tyler Jacks, head of the Koch Institute for Integrative Cancer Research at MIT, joined Skyhawk's scientific advisory board as its chair, while Tai Wong, former vice president of biology at Merck-acquired Peloton Therapeutics Inc., will join Skyhawk as its vice president of oncology biology.

The new Merck and extended Biogen agreements extend a roster of collaborations that privately held Skyhawk has formed around its SkySTAR (Skyhawk Small molecule Therapeutics for Alternative splicing of RNA) platform, which already included deals with Takeda Pharmaceutical Co. Ltd. and Celgene Corp. (See BioWorld, June 27, 2018.)

"RNA splicing modification offers a new approach to modulating targets previously considered undruggable," said Dean Li, senior vice president of discovery and translational medicine at Merck Research Laboratories. Li was unavailable for further comment.

Neither Merck nor Skyhawk revealed the size of cash up-front payments accompanying each target in their collaboration, nor the agreed length of the collaboration. However, past deals featured up-front payments of $60 million from Biogen and $74 million from Celgene.

Should Merck exercise its option to exclusively license global IP rights to candidates discovered and developed in the deal, Skyhawk could earn potential milestone payments and royalties on sales of approved products resulting from the collaboration. Kenilworth, N.J.-based Merck would be responsible for further development and commercialization.

With SkySTAR, Skyhawk is taking aim at a type of RNA mis-splicing called exon skipping, in which critical regions on the RNA are essentially skipped over during the splicing process. It's a problem found to be a cause of a growing list of diseases, including orphan and neurological conditions, such as spinal muscular atrophy (SMA) and major cancers, such as pancreatic cancer, according to the company. The company's scientists seek to uncover information from computational, kinetic and structural models of RNA to find "selective families of chemistry for each target," an approach that sounds tailor-made for Merck's own approach to R&D.

"Although we're doing all of these new modalities, we are in no way moving away from chemistry," Merck's head of neuroscience, Fiona Marshall, said, during a recent investor day conference call. There are "huge technology advances," she said, but "the bottom line is that proteins that we thought were undruggable are now very much druggable."

Revenue from Merck's oncology and vaccine portfolios are central to the company's plans to weather the projected peak impact in 2023 of its loss of exclusivity on Januvia/Janumet (sitagliptin).

Biogen collaboration expanded

Alongside its Merck announcement, Skyhawk said it is expanding its collaboration with Biogen, originally announced Jan. 4. The revision extends Biogen's exclusive license to worldwide IP rights beyond the original collaboration's research-stage therapeutic candidates for the treatment of conditions including multiple sclerosis, SMA and additional neurological disorders. As part of the expansion, Skyhawk received an up-front payment from Biogen and may receive potential future milestone payments and royalties.

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