Ionis licenses zilganersen to Recordati for ex-U.S. markets

Ionis Pharmaceuticals (Nasdaq: IONS) has entered into a license agreement with Recordati, granting the Italian drugmaker exclusive rights to develop and commercialize zilganersen outside the U.S. Zilganersen is an investigational treatment for Alexander disease (AxD), a rare and progressive neurological condition.

Under the agreement, Ionis will receive an upfront payment of $30 million, with eligibility for additional milestone payments and a tiered royalty of up to the mid-20% range on annual net sales. Ionis retains sole commercial rights for zilganersen in the U.S. and will continue to lead global development. Recordati will handle regulatory filings and commercialization in all other markets.

Zilganersen is currently under FDA review, with a Prescription Drug User Fee Act (PDUFA) action date of September 22. The FDA has granted the drug Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations. The European Medicines Agency has also granted it Orphan Drug designation.

Ionis previously reported positive top-line results from a pivotal study of zilganersen. The study met its primary endpoint in individuals aged five and older, with the 50 mg dose showing statistically significant stabilization of gait speed on the 10-Meter Walk Test compared to control at Week 61. Serious treatment-emergent adverse events occurred in 37.5% of the zilganersen group versus 47.1% in the pooled control group.

AxD affects approximately one per one to three million people worldwide and currently has no approved disease-modifying treatments. The disease typically leads to death within 14 to 25 years after symptom onset.

According to a statement from the press release, Ionis CEO Brett P. Monia said Recordati has "the infrastructure needed to reach patients outside the United States," citing the company's rare disease commercialization experience.

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