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Ipsen, Skyhawk to develop RNA-targeting pills for rare brain diseases

Dive Brief:

  • French pharmaceutical company Ipsen will work with privately held biotechnology company Skyhawk Therapeutics to make small molecule drugs that modify RNA.
  • Through a collaboration announced Monday, Ipsen and Skyhawk plan to develop multiple RNA-targeting medicines for rare neurological diseases. Ipsen has an option to acquire two for an undisclosed sum and could pay Skyhawk as much as $1.8 billion if certain milestones are met.
  • The alliance is the latest industry collaboration focused on RNA-targeting pills, an increasingly popular area of drug research. Several startups, including Skyhawk, say the approach can help access disease targets previously thought to be “undruggable,” though much of their work remains early.

Dive Insight:

Drugs that target RNA, the messenger molecules that turn genetic blueprints into proteins, have come of age in recent years. But nearly all are administered through injections or infusions, rather than the chemical-based pills that are the bedrock of the pharmaceutical industry.

Skyhawk is among the biotechs looking to change that by developing small molecule drugs that target RNA to treat disease. Historically, RNA-targeting pills have mostly been stumbled on accidentally. Skyhawk and its peers, like Accent Therapeutics, Arrakis Therapeutics and Remix Therapeutics, aim to develop such medicines purposefully, with the goal of getting to disease targets small molecules currently can’t reach.

That pursuit has a unique set of challenges and remains in its early stages. Many of the field’s players don’t yet have prospects in clinical testing; Skyhawk’s most advanced prospect, for Huntington’s disease, entered human trials last year. Nonetheless, several have drawn the interest of large pharmaceutical companies.

Ipsen, for instance, formed a partnership with Accent Therapeutics in 2021 develop a drug for acute myeloid leukemia. Since 2020, Arrakis has signed deals with Roche and Amgen. Remix is aligned with Roche and Johnson & Johnson.

Skyhawk has several collaborations under its belt, too. Prior to joining forces with Ipsen, the biotech started alliances with Sanofi, Merck & Co. and Vertex Pharmaceuticals. The company has raised more than $180 million in private funding as well.