Skyhawk’s RNA Splicing Tech Draws in Expanded Merck Deal

The two companies are working to develop and commercialize a particular type of therapy built on modulating RNA splicing using Skyhawk’s SkySTAR technology platform.

Waltham, Massachusetts-based biotech company Skyhawk Therapeutics is expanding its strategic collaboration deal with Merck. The two companies are working to develop and commercialize a particular type of therapy built on modulating RNA splicing using Skyhawk’s SkySTAR technology platform.

A number of diseases, from orphan and neurological diseases to cancer, is increasingly being linked to RNA mis-splicing that leads to loss of RNA expression. Skyhawk’s focus is on mediating this mis-splicing using the SkySTAR platform that leverages data from computational, kinetic and structural RNA models. The company has partnerships with a number of major biopharma and biotech companies, including Bristol Myers Squibb, Takeda, Biogen and Genentech.

The current collaboration with Merck focuses on neurodegeneration, oncology, autoimmunity and metabolic diseases. The extended collaboration will be to develop RNA-binding small molecules that modify RNA splicing. The new expansion grants Merck, through a subsidiary, the option to exclusively license global intellectual property rights to compounds discovered and developed under the collaboration that are directed to the program’s targets. Once Merck chooses to exercise its option, Merck will handle further development and commercialization.

Skyhawk will receive cash up front and various milestone payments and royalties on sales of approved products that come out of the partnership.

“Merck has been a wonderful partner in discovering novel drug candidates for neurological diseases and cancer,” said Bill Haney, co-founder and chief executive officer of Skyhawk. “Our expanded collaboration into autoimmune and metabolic diseases reflects the success to date in the SkySTAR platform’s ability to advance small molecules that can address the unmet medical needs of patients. Skyhawk’s team is delighted to be working with a partner with such a long history of commitment to challenging diseases, and relentless pursuit of developing new treatment options for patients.”

The original deal was inked in July 2019, with Skyhawk eligible for about $600 million per program target. This new expansion apparently has the same financial parameters, $600 million per program, an undisclosed upfront cash payment, and various biobucks and royalties.

Last year, Skyhawk also entered a partnership with Roche/Genentech that has an upside potential of $2 billion.

“Modulation of RNA splicing represents a novel approach for difficult-to-treat diseases,” said James Sabry, global head of Pharma Partnering for Roche, at the time. “Skyhawk has developed unique expertise in splicing biology, and we are excited to work with their team to discover potential new medicines for patients with cancer and neurodegenerative diseases.”

Skyhawk inked the Biogen deal in January 2019, with a focus on neurological diseases. Biogen paid Skyhawk $74 million up front with assorted unspecified milestones and royalties available.

Skyhawk was one of BioSpace’s NextGen Bio “Class of 2019” top 20 life science startups to watch. It has dramatically exceeded expectations. At the time, in June 2018, Skyhawk had closed on a $40 million equity round and in an unusual deal, inked a five-year collaborative agreement with Celgene that included $60 million up front.

The company has programs in medulloblastoma, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, Parkinson’s disease, pancreatic cancer and spinal muscular atrophy.

“RNA splicing modification offers a new approach to modulating targets previously considered undruggable,” said Dean Y. Li, Merck Research Laboratories’ senior vice president, Discovery Sciences and Translational Medicine. “We look forward to expanding our collaborative efforts to explore the potential of this new modality in additional disease areas.”