Ionis Pharmaceuticals, Inc. has announced a licensing agreement for Zilganersen for Alexander disease with Recordati. The partnership will support the development and commercialization of zilganersen in countries outside the United States. Furthermore, Recordati will bring its rare disease expertise and global commercialization capabilities to expand patient access.
Under the agreement, Recordati has received exclusive rights to develop and commercialize zilganersen across all markets outside the U.S. Meanwhile, Ionis Pharmaceuticals will continue leading global development activities. Additionally, the company will manage commercial responsibilities for zilganersen in the United States, subject to FDA approval.
Recordati will oversee regulatory submissions and commercialization strategies in international markets. Moreover, the company will provide country-specific support for early access programs. These efforts will follow regional regulatory requirements and healthcare access conditions.
Ionis and Recordati Strengthen Rare Disease Treatment Partnership
“Recordati combines proven rare disease and global commercialization expertise with deep experience navigating regional and local regulatory environments and the infrastructure needed to reach patients outside the United States, making them the right partner to deliver zilganersen to patients with urgency. They recognize, as we do, the high unmet need for this rare, serious and often fatal neurodegenerative disease,” said Brett P. Monia, Ph.D., chief executive officer, Ionis. “Together, we are committed to working closely to help bring zilganersen to people living with Alexander disease outside the United States and enable broader access to this important treatment.”
Ionis plans to independently commercialize zilganersen in the U.S. following potential FDA approval. Currently, the therapy remains under FDA review, with a Prescription Drug User Fee Act (PDUFA) action date scheduled for September 22.
If approved, zilganersen could become the first and only disease-modifying treatment available for Alexander disease. In addition, the launch would represent Ionis’ first independent commercial launch in neurology. The milestone supports Ionis’ broader objective of delivering advanced medicines for serious neurological conditions.
Rob Koremans, Chief Executive Officer, Recordati, commented, “We are very pleased to partner with Ionis on the development and commercialization of zilganersen outside the United States. The addition of zilganersen further strengthens our Rare Diseases portfolio with a potentially transformative therapy for patients affected by Alexander disease, a devastating and progressive neurological disorder with significant unmet medical need. By combining Ionis’ innovation and development expertise with Recordati’s global rare disease capabilities, we believe we are well positioned to maximize the potential of zilganersen and bring a much-needed treatment option to patients with Alexander disease.”
Zilganersen Clinical Trial Data Demonstrate Favorable Treatment Effects
Ionis reported additional data from its pivotal trial of zilganersen, following positive top-line results. The findings point to possible benefits in several areas of Alexander disease symptoms.
The clinical study primary endpoint was achieved in subjects five years of age and older. Specifically, zilganersen 50 mg demonstrated statistically significant and clinically meaningful stabilization of gait speed. This improvement was assessed with the 10-Meter Walk Test (10MWT), which is a measure of gross motor function in neurological disorders.
Moreover, patient, caregiver and clinician-reported secondary and exploratory assessments consistently supported the treatment benefits of zilganersen. The therapy also showed a good safety and tolerability profile in the study.
Most treatment-emergent adverse events were of mild or moderate severity. The incidence of serious treatment-emergent adverse events was also lower in patients receiving zilganersen than in the control group. The rate was 37.5% for the zilganersen 25 mg or 50 mg groups vs 47.1% for the pooled control group.
Under the terms of the licensing agreement, Recordati will pay Ionis $30 million up front. In addition, Ionis is eligible for additional milestone payments and tiered royalties. These royalties could be in the mid 20% range based on annual net sales.
The partnership between Ionis and Recordati is intended to speed access to zilganersen for people living with Alexander disease across the globe. Therefore, the partnership represents a significant step forward in advancing treatment options for rare diseases and improving access to innovative neurological therapies.
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News Source: Businesswire.com
