Ipsen Partners with Skyhawk Therapeutics in $1.8 Billion Deal to Advance RNA-Targeted Therapies for Rare Neurological Diseases

Ipsen Makes Another Bold Move.

On April 22, Ipsen and Skyhawk Therapeutics announced a research collaboration targeting RNA for rare neurological diseases.

(Announcement Screenshot)

According to the agreement, Skyhawk will use its proprietary platform to develop RNA-targeting candidate drugs for rare neurological diseases. Ipsen will have the option to obtain global exclusive rights to two candidate products and, after validation of the development candidates, assume responsibility for further development and commercialization.

Skyhawk is eligible to receive up to $1.8 billion in development, regulatory, and commercial milestones, including upfront payments for options and research collaboration, as well as potential tiered royalties.

Ipsen Senior Vice President and Head of Neuroscience, R&D, Steve Glyman, said: "We are delighted to collaborate with Skyhawk's expert team to explore the potential of modifying RNA expression in rare and debilitating neurological diseases."

Skyhawk is a clinical-stage biopharmaceutical company focused on the discovery and development of small molecules that modulate RNA. The popularity of RNA therapies has attracted several large pharmaceutical companies, including Biogen, Takeda, Sanofi, and Merck, to establish cooperative relationships with Skyhawk.

Skyhawk's SkySTAR technology platform is a platform for discovering innovative small molecules that target RNA. It can be used to discover small molecule compounds that correct RNA splicing errors in the nucleus by binding to precursor RNA, restoring mRNA expression levels.

RNA splicing errors may lead to the loss of mRNA expression levels, resulting in a series of neurological diseases including neuroblastoma, amyotrophic lateral sclerosis (ALS), Parkinson's disease (PD), frontotemporal dementia (FTD), and spinal muscular atrophy (SMA).

The most advanced program in Skyhawk's pipeline is SKY-0515, for the treatment of Huntington's disease, which is also Skyhawk’s first candidate drug to enter the clinical stage.

SKY-0515 is a small molecule RNA splicing modifier that directly targets HTT RNA, administered orally, penetrates the brain, and distributes in peripheral tissues, offering significant benefits to patients. It is currently in Phase 1 clinical trials.

(Screenshot from the Skyhawk official website)

Ipsen focuses on three therapeutic areas: oncology, rare diseases, and neuroscience. In the field of rare diseases, it has also developed multiple drugs, including Increlex (mecasermin, for treating growth failure in children and adolescents with severe primary insulin-like growth factor-1 deficiency), Somatuline (lanreotide, for treating acromegaly and pituitary gigantism), Sohonos (palovarotene, for treating fibrodysplasia ossificans progressiva), and others.

In recent years, Ipsen has continuously expanded its presence in the rare disease field and acquired Albireo Pharma for $952 million in January last year, broadening its rare disease portfolio.

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Company Announcement

Editor: Baiji

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