Merck & Co. and Skyhawk Therapeutics have launched a collaboration that could pay the Waltham, MA, biotech approximately $600 million for every small molecule program the companies develop against neurodegenerative diseases and cancer.

The companies have agreed to discover, develop, and commercialize small molecules that modulate RNA splicing, directed to “multiple” targets, Skyhawk said, without specifying how many.

The companies plan to employ Skyhawk’s proprietary SkySTAR™ technology platform to discover and develop RNA-binding small molecules designed to selectively modify RNA splicing—a new potential treatment modality, Skyhawk and Merck reason, for at least some neurological diseases and cancer.

SkySTAR stands for “Skyhawk Small molecule Therapeutics for Alternative splicing RNA.” The platform is designed to integrate data from computational, kinetic, and structural models of RNA, in order to generate unique and selective families of chemistry for each target. Those libraries are tested in patient cells using proprietary assays, to quickly yield potent drug candidates.

Skyhawk says its approach is designed to correct the underlying genetics of disease at the mRNA level, in the nucleus of the cell, with the aim of targeting master transcriptional factors in cancer and other previously-undruggable targets in major neurological conditions, immunology, inflammation, and infectious diseases.

The company’s lead pipeline candidate, described on its website only as “Skyhawk Oncology Target 1,” has advanced past lead selection and is close to an IND filing. Three other pipeline candidates are directed against neurological targets.

Skyhawk has agreed to grant Merck, through a subsidiary, the option to exclusively license worldwide intellectual property rights to candidates discovered and developed under the collaboration. Upon exercising its option, Merck has agreed to oversee further development and commercialization.

In return, Merck agreed to pay Skyhawk an upfront cash payment, as well as potential milestone payments and royalties on sales of approved products resulting from the collaboration “to the extent Merck exercises its option,” Skyhawk said.

“A new approach”

“RNA splicing modification offers a new approach to modulating targets previously considered undruggable,” said Dean Y. Li, MD, PhD, senior vice president, discovery and translational medicine, Merck Research Laboratories. “We look forward to working with the scientists at Skyhawk to explore the potential of this new modality.”

Added Skyhawk co-founder and CEO Bill Haney: “We look forward to demonstrating the ability of our SkySTAR technology platform to deliver novel drug candidates for the disease targets Merck has selected and advancing those compounds to address the unmet medical needs of patients.”

Skyhawk announced its collaboration with Merck the same day it said it will expand a seven-month-old partnership with another biopharma giant.

Skyhawk and Biogen agreed to the expansion of their partnership to develop novel small molecule RNA splicing modifiers for neurological disease targets, launched in January. Back then, Biogen agreed to pay Skyhawk $74 million upfront, as well as undisclosed potential future milestone payments and royalties. In return, Biogen gained access to Skyhawk’s SkySTAR™ technology platform with the goal of discovering innovative small molecule treatments

Under the expanded collaboration, Biogen will pay Skyhawk additional undisclosed upfront, milestone, and royalty payments, return for an extension of its exclusive license to worldwide intellectual property rights beyond the original collaboration’s research-stage therapeutic candidates for conditions that include multiple sclerosis (MS), spinal muscular atrophy (SMA), and additional neurological disorders.

“[Biogen’s] strong scientific culture has already produced a series of leading global therapeutics and our work together to date has gone very well,” Haney added.

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