Ionis Pharmaceuticals has signed a licensing agreement with Recordati, granting the company exclusive rights to develop and commercialise the investigational medicine zilganersen for Alexander disease (AxD) in all countries outside the United States.

Under the agreement, Ionis will retain exclusive commercial rights to zilganersen in the US and will continue to lead the medicine's global development. Recordati will manage regulatory submissions and commercialisation in markets outside the US, including support for country-specific early access programmes where applicable.

Zilganersen is currently under review by the US Food and Drug Administration (FDA), with a Prescription Drug User Fee Act (PDUFA) decision expected on 22 September. If approved, it could become the first disease-modifying treatment for Alexander disease and would mark Ionis' first independent commercial launch in the neurology market.

The agreement expands Recordati's rare disease portfolio by adding a potential treatment for Alexander disease, a rare and progressive neurological disorder with limited treatment options.

The partnership follows positive results from the pivotal clinical study of zilganersen. The trial met its primary endpoint in patients aged five years and older, with the 50 mg dose showing statistically significant and clinically meaningful stabilisation of gait speed compared with the control group after 61 weeks, as measured by the 10-Meter Walk Test (10MWT). Secondary and exploratory assessments reported by patients, caregivers and clinicians also favoured zilganersen.

The investigational treatment demonstrated a favourable safety and tolerability profile, with most adverse events reported as mild or moderate. Serious treatment-emergent adverse events occurred less frequently in patients receiving zilganersen than in the control group.

Under the terms of the agreement, Ionis will receive an upfront payment of US$30 million. The company is also eligible for additional milestone payments and tiered royalties of up to the mid-20% range on annual net sales.

 

Source: ionis.com

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