Ipsen signs $1.8bn RNA drug alliance with Skyhawk

Ipsen has expanded its R&D pipeline once again, agreeing a $1.8 billion alliance with Skyhawk Therapeutics focused on the development of RNA-targeting therapies for rare neurological diseases.

The deal – which covers development, regulatory, and commercial milestones and an upfront payment – centres on an option taken by Ipsen on a pair of small-molecule candidates that modulate RNA. The identity of the compounds, or their target indications, has not been disclosed, but they are in early preclinical development.

Under the terms, Ipsen will take responsibility for further development and commercialisation of the two candidates after lead compounds have been validated for further development.

It is the US-Swiss biotech’s first publicly disclosed partnering deal for some time, although, it claims to have more than a dozen confidential programmes on the go with other companies and previously revealed programmes with MSD/Merck & Co, Biogen, Celgene (now part of Bristol-Myers Squibb), Vertex Pharma, and Roche.

Skyhawk’s drug discovery and development platform generates drug candidates that can be dosed orally and cross the blood-brain barrier into the central nervous system, and draws on computational biology data from public and proprietary databases, and artificial intelligence and machine learning to design candidates.

The approach is to develop small-molecule drugs that can correct RNA mis-splicing, leading to loss of RNA expression by exon-skipping. It is another way to go after hard-to-target disease mechanisms and – according to the biotech – is an effective way to target diseases caused by the loss of function of key proteins that are hard to address with antisense drugs or gene therapies.

Skyhawk has built a library of RNA-targeting compounds and has a screening technology that simultaneously tests dozens of targets across a range of RNA motifs to identify active compounds. Its lead in-house candidate SKY-0515 is a splicing modulator targeting HTT and the expression of huntingtin protein, with potential as a treatment for Huntington’s disease, which is in early-stage clinical testing.

It is the second licensing agreement for Ipsen this month, coming after it expanded its oncology pipeline by licensing rights to a preclinical-stage antibody-drug conjugate (ADC) for solid tumours from Sutro Biopharma in a deal worth up to $900 million.

Ipsen closed a $952 million acquisition of US biotech Albireo and its rare disease drug Bylvay (odevixibat) last year, after buying cancer specialist Epizyme for $250 million and licensing two T-cell receptor (TCR) immuno-oncology therapies from Marengo in a $1.6 billion deal.

The pharma company’s head of neuroscience R&D, Steve Glyman, said the alliance with Skyhawk would allow it to “explore the potential for modifying RNA expression across rare and debilitating neurological conditions.“

Its current neuroscience pipeline is focused on expanding the indications for botulinum toxin therapy Dysport (abobotulinumtoxin A) to include migraine treatment, as well as longer-acting follow-up IPN10200.

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