Skyhawk Therapeutics flying high with strong end to June

The startup has closed a $40M funding round and landed a deal with biopharmaceutical giant Celgene

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CAMBRIDGE, Mass.—Skyhawk Therapeutics is making impressive strides for a startup company, going from launch to landing new investors—and a big-name partner—in just six months. Skyhawk announced on June 26 that it had closed a $40-million equity investment round and established a global strategic collaboration with a Celgene Corporation affiliate.
The startup launched this January with $8 million in financing. That seed round was led by Alexandria Venture Investments and family investors Tim Disney and the Duke of Bedford, all of whom participated in the latest funding round as well. GreatPoint Ventures, Celgene Corporation, ShangPharma Innovation, Agent Capital, the Reilly Family and other private, tech-focused family investment firms participated in the all-common stock equity round in June as well.
The proceeds from this financing will be used to advance the STAR* (Small molecule Therapeutics for Alternative splicing RNA) technology platform to discover and develop small molecules capable of fixing RNA mutations, and Skyhawk has a rather ambitious goal of having its first oncology drug in the clinic by the end of next year. Though no specifics are shared on the website, Skyhawk has four compounds in its pipeline, one of which is aimed at an oncology target while the other three have neurology targets. The oncology target is approaching filing status, while two neurology targets are at the lead selection stage and the third is in preclinical IND.
As for Skyhawk's collaboration with the Celgene affiliate, the companies will be working together on the discovery, development and commercialization of innovative small-molecule treatment options for neurological diseases. Per the terms of the agreement, Celgene gains an exclusive option to in-license worldwide intellectual property rights associated with therapeutic candidates for a maximum of five programs with possible applications in amyotrophic lateral sclerosis (ALS), Huntington's disease and other neurological disorders. In return, Skyhawk will receive $60 million up front, with the potential for future license fees, milestone payments and royalties. The companies will primarily be leveraging Skyhawk's STAR* technology platform in this effort.
"Through execution of this strategic alliance with Celgene and our new investment round, Skyhawk is well positioned and capitalized to continue advancing our mission of revolutionizing disease treatment with small molecules that correct RNA expression," Bill Haney, co-founder and CEO of Skyhawk Therapeutics, said in a press release. "Celgene is a preeminent biopharmaceutical company with a demonstrated history of recognizing disruptive science that may lead to new treatment options for patients with neurological disease. We look forward to a successful collaboration with Celgene, and to continued progress with our internal programs that target a set of exon-skipping-driven cancer indications."
RNA is responsible for translating DNA for the production of proteins, and part of the process includes splicing. As explained on Nature's Scitable website, “For most eukaryotic genes (and some prokaryotic ones), the initial RNA that is transcribed from a gene's DNA template must be processed before it becomes a mature messenger RNA (mRNA) that can direct the synthesis of protein. One of the steps in this processing, called RNA splicing, involves the removal or "splicing out" of certain sequences referred to as intervening sequences, or introns. The final mRNA thus consists of the remaining sequences, called exons, which are connected to one another through the splicing process.”
RNA can mis-splice sequences in a way known as “exon skipping,” in which significant regions of the RNA are missed during the splicing process. Skyhawk's STAR* technology makes it possible to rationally design small molecules that can target specific binding pocket regions on RNA with both sequence and structural specificity, at specific times. It is thought that this method could enable the reversal of mis-splicing to treat disease.
As Skyhawk notes on its website, “RNA mis-splicing leading to loss of RNA expression has been found to cause a growing list of diseases, from orphan and neurological conditions to major cancers. Very few treatments are available for these challenging conditions, many of which have been considered 'undruggable.'” Among those ranks are conditions such as amyotrophic lateral sclerosis, medulloblastoma, frontotemporal dementia, Parkinson's disease, pancreatic cancer and spinal muscular atrophy.
"Targeting RNA with therapeutics is becoming an increasingly important approach in neurological research," commented Dr. Richard Hargreaves, corporate vice president of Neuroscience and Imaging Research and Early Development for Celgene. "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."

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