Startup Skyhawk Therapeutics Snags $100 Million From Financing Round and Deal With Celgene

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Massachusetts-based Skyhawk Therapeutics is closing out the month of June with some positive news that will carry over through the near future. The company closed a $40 million equity investment round and struck a five-year collaborative agreement with Celgene that includes an upfront $60 million.

Skyhawk launched earlier this year with a focus on the development of small molecule therapeutics that correct RNA mis-splicing that is typically called "exon skipping." That error occurs when key regions on the RNA are left out during the RNA splicing process. Skyhawk is developing proprietary technology that “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.” This approach has the potential to reverse the mis-splicing and treat the disease, the company said. There are more than 50 known diseases driven by exon skipping, from broad-based neurological conditions to major cancers and more. It’s the focus on neurological conditions that drew Celgene to the startup.

With the collaborative agreement in hand, Celgene Corporation will receive exclusive options to license a number of Skyhawk's product candidates focused on high-value targets in neurological indications. Celgene is looking to harness the company’s STAR technology platform to target five neurological targets, including amyotrophic lateral sclerosis (ALS) and Huntington's disease.

Richard Hargreaves, head of Neuroscience and Imaging Research and Early Development at Celgene, said targeting RNS is becoming an “increasingly important approach” in neurological research.

“This collaboration to discover and develop small molecule splicing modifiers extends our commitment to the neurodegenerative disease area consistent with our leadership and focus on protein homeostasis, and strategy to collaborate with innovators who are focused on disruptive approaches to treat disease. We are looking forward to working with Skyhawk's team of world-leading experts and hope this collaboration will assist each company to realize its mission to bring innovative therapies to patients,” Hargreaves said in a statement.

If the Celgene targets hit, Skyhawk stands to earn even more money from undisclosed milestone payments and potential royalties. Also, if the approach proves fruitful, Celgene could return to Skyhawk for additional licensing agreements.

Proceeds from the Celgene deal, as well as the $40 million raised in the equity investment round, will be used to advance the company’s STAR (Small molecule Therapies for Alternative splicing in RNA) platform and its own pipeline research into RNA mutation-associated oncology and neurology diseases. Skyhawk said it anticipates its first oncology treatment will be in the clinic by the end of 2019. On its website the company does not provide much detail into its targets, only referring to them with numbers. The oncology therapeutics is referred to as Skyhawk Oncology Target 1. Although not confirmed, Skyhawk does list pancreatic cancer as a related target.

The company also lists three neurological targets in the same numerical manner.

Participants in the round included many of the investors from the seed round including Alexandria Venture Investments and the Duke of Bedford, along with new investments by GreatPoint Ventures and Celgene Corporation. 

Skyhawk Chief Executive Officer Bill Haney said the company is “well positioned and capitalized” to move forward with its programs. 

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