Skyhawk and Vertex Team Up on RNA Splicing-Based Therapies


Skyhawk Therapeutics and Vertex Pharmaceuticals inked a strategic research collaboration and licensing deal focused on developing novel small molecules that modulate RNA splicing. Skyhawk leverages its novel SkySTAR platform, building small molecules for a broad range of indications including neurodegenerative diseases and oncology.

Vertex has multiple approved drugs for cystic fibrosis (CF) and has several ongoing preclinical and clinical programs in CF, as well as a pipeline of small molecule medicines for pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney disease.

Under the terms of the deal, Vertex is paying Skyhawk $40 million upfront. Skyhawk grants Vertex options to license global intellectual property rights to compounds that come out of the collaboration directed at specific program targets. After its exercises its options, Vertex will handle development and commercialization. Skyhawk is eligible for up to $2.2 billion in potential milestone payments in addition to possible royalties on future sales.

“We believe that splice site modulation holds significant promise for the treatment of diseases which today have limited or no therapeutic options,” said Mark Bunnage, senior vice president and Site Head, Boston Research at Vertex. “This collaboration brings Skyhawk’s innovative technology together with Vertex’s research and development experience; and fits perfectly with our strategy of investing in new technologies that will help us transform multiple serious diseases.”

Skyhawk’s existing partnerships include Biogen, Bristol Myers Squibb, Merck, Roche and Takeda.

There were no disclosures about what indications Skyhawk and Vertex are interested in, other than to say “serious diseases.”

Photo by Craig F. Walker/The Boston Globe via Getty Images

Skyhawk was one of BioSpace’s NextGen Bio “Class of 2019” life sciences startups to watch. In June 2018, the company closed on a $40 million equity investment round and inked an unusual five-year collaborative agreement with Celgene that included an upfront $60 million payment. Their technology focuses on developing small molecule therapeutics that correct RNA mis-splicing, what is usually called exon skipping.

In May, Skyhawk expanded its strategic collaboration deal with Merck. A number of diseases, from orphan and neurological diseases to cancer, are increasingly being linked to RNA mis-splicing that leads to loss of RNA expression. Skyhawk works to develop molecules that mediate this mis-splicing and leverages data from computational, kinetic and structural RNA models.

The deal 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. It granted 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.

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

The 2018 partnership with Roche/Genentech deal has the chance to hit $2 billion. The 2019 Biogen deal focused on neurological diseases, with Biogen paying Skyhawk $74 million up front with various undisclosed milestones and royalties.

Skyhawk’s internal programs are in medulloblastoma, amyotrophic lateral sclerosis (ALS), frontotemporal dementia, Parkinson’s disease, pancreatic cancer and spinal muscular atrophy.


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