Ipsen, Skyhawk Ink Potential $1.8B Deal to Target RNA in Neuro Diseases

mRNA molecule_iStock, Artur Plawgo

Pictured: 3D illustration of an RNA molecule/iStock, Artur Plawgo

Ipsen and Skyhawk Therapeutics on Monday announced they have entered into an exclusive worldwide collaboration agreement to develop novel small molecule drugs against RNA targets in rare neurological conditions.

The companies did not reveal the specific financial breakdown of the deal but said that Ipsen is committing up to $1.8 billion, which includes the upfront payment as well as development, regulatory and commercial milestones. Skyhawk will also be entitled to tiered royalties on products that come out of the partnership.

Ipsen will have the option to acquire the exclusive worldwide rights to advance successful drug candidates and will take charge of all research activities for these investigational molecules, according to the announcement.

The agreement will allow Ipsen to “explore the potential for modifying RNA expression across rare and debilitating neurological conditions,” Steve Glyman, head of Ipsen’s neuroscience R&D, said in a statement, adding that the company’s “expertise in movement disorders” will be bolstered by Skyhawk’s novel technology which is “at the cutting-edge of research.”

At the center of Monday’s deal is Skyhawk’s proprietary platform, which combines four complementary data sets to discover and design small molecule drugs against RNA molecules that are upstream of disease-causing proteins. According to the biotech’s website, this approach could potentially yield treatments for “thousands” of previously undruggable targets.

The first component of Skyhawk’s platform is SKYSTAR, which combs through public and proprietary data, taking into account structural, bioinformatic and computation biology information to identify high-value RNA targets. SKYSEQ, a multiplex screening system, then tests several of these potential targets and looks through Skyhawk’s repository called SKYLIBRARY for RNA-targeting compounds likely to have therapeutic effects.

Skyhawk’s platform also uses SKYAI, its proprietary machine learning tools, to integrate information from SKYSTAR along with toxicology, animal study and chemistry data to “drive the next generation of novel RNA splicing modulators.”

The partners did not disclose what specific disease targets they will go after but Skyhawk CSO Sergey Paushkin in a statement did say noted that they will work on “rare neurological diseases for which there are no approved therapeutics.”

For Ipsen, the Skyhawk deal comes weeks after it inked a $900 million contract with Sutro Biopharma, gaining exclusive global rights to the latter’s investigational antibody-drug conjugate STRO-003, which targets the ROR1 tumor antigen and is currently in the final stages of preclinical testing.

Ipsen also recently secured the FDA’s approval for Onivyde as a first-line treatment for metastatic pancreatic cancer, when combined with three chemotherapeutic drugs.

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

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