Ionis Pharmaceuticals has offloaded regional rights to its near-approval rare disease asset zilganersen, pocketing $30 million upfront from Recordati for an ex-U.S. license to the drug candidate.
Zilganersen will become Ionis’ first wholly owned launch in neurology if the FDA approves the antisense oligonucleotide for the treatment of Alexander disease (AxD) in September. The biotech has identified the drug candidate as a launchpad for its ambition to commercialize neurology therapies in the U.S., but it is taking a different approach overseas.
Recordati is paying $30 million upfront and committing to undisclosed milestones, plus tiered royalties up to the mid-20% range, for rights to zilganersen outside the U.S. The agreement makes Recordati responsible for international regulatory filings and commercialization, including country-specific support for early access pathways based on local regulations and access dynamics.
Italy’s Recordati, which is the subject (PDF) of a 10.7-billion-euro ($12.2 billion) buyout offer, has built rare disease capabilities and expertise to commercialize drugs such as cold agglutinin disease treatment Enjaymo. Zilganersen, which has orphan drug designation in Europe, will slot into the multiproduct rare disease portfolio.
Ionis will continue to lead zilganersen development globally and maintain sole commercial responsibility for the molecule in the U.S. The FDA is set to rule on whether to approve the drug candidate by Sept. 22.
AxD is a niche market. Ionis believes the neurological disorder affects only around 300 people in the U.S., Kyle Jenne, the company’s chief global product strategy officer, said on an earnings call in February. The biotech has predicted peak sales will top $100 million. TD Cowen analysts have forecast sales should approach that threshold by 2032, while their peers at William Blair have forecast a peak of $295 million.
