Ionis, Recordati Partner to Expand Zilganersen Access Globally

Ionis Partners with Recordati to Commercialize Alexander Disease Therapy Outside U.S.

Ionis Pharmaceuticals has entered into a strategic licensing agreement with Recordati to expand the global reach of its investigational therapy, zilganersen, for the treatment of Alexander disease (AxD). Under the agreement, Recordati will receive exclusive rights to develop and commercialize the RNA-targeted medicine in all countries outside the United States, while Ionis will retain full commercial rights in the U.S. and continue leading the drug’s global development program.

The partnership marks an important step in Ionis’ efforts to bring what could become the first disease-modifying treatment for Alexander disease to patients worldwide. Alexander disease is a rare, progressive, and often fatal neurological disorder with no approved disease-modifying therapies currently available.

As part of the collaboration, Recordati will oversee regulatory submissions and commercialization activities outside the U.S. The company will also manage country-specific early access programs where permitted by local regulations, leveraging its experience in rare diseases and international regulatory processes. Ionis will continue to direct the clinical development of zilganersen while preparing for its planned commercial launch in the United States.

Ionis Chief Executive Officer Brett P. Monia said Recordati’s extensive expertise in rare diseases and established commercial infrastructure make it an ideal partner to accelerate access to the treatment for patients beyond the U.S. He noted that both companies recognize the urgent unmet medical need in Alexander disease and share the goal of delivering the therapy to patients as quickly as possible.

For Ionis, the anticipated U.S. launch of zilganersen represents more than just another product introduction. If approved, the medicine would become the company’s first independently commercialized neurology therapy, supporting its long-term strategy of building a leadership position in treatments for serious neurological disorders.

The therapy is currently under review by the U.S. Food and Drug Administration, with a Prescription Drug User Fee Act (PDUFA) decision expected on September 22. Regulatory approval would position zilganersen as the first treatment specifically designed to modify the progression of Alexander disease rather than simply managing symptoms.

Recordati Chief Executive Officer Rob Koremans described the collaboration as a significant addition to the company’s growing rare disease portfolio. He said the agreement combines Ionis’ innovation in RNA-targeted therapeutics with Recordati’s global expertise in developing and commercializing medicines for rare diseases. According to Koremans, the companies are well positioned to maximize the therapy’s potential and provide a much-needed treatment option for patients living with the devastating neurological condition.

The licensing agreement follows encouraging clinical results reported by Ionis from the pivotal study evaluating zilganersen. The company recently announced additional positive data that reinforced the treatment’s potential benefits across multiple symptoms associated with Alexander disease.

The pivotal trial achieved its primary endpoint in patients aged five years and older. Participants receiving the 50 mg dose of zilganersen demonstrated statistically significant and clinically meaningful stabilization of gait speed, measured using the widely accepted 10-Meter Walk Test, after 61 weeks of treatment compared with the control group. Secondary and exploratory assessments, including evaluations reported by patients, caregivers, and clinicians, also consistently favored the investigational therapy.

Safety findings from the study further supported the treatment’s profile. Most adverse events were reported as mild to moderate, while serious treatment-emergent adverse events occurred less frequently among patients receiving zilganersen than those in the pooled control group.

Financially, the agreement provides Ionis with an upfront payment of $30 million. The biotechnology company will also be eligible for additional milestone payments tied to future development and commercial achievements, along with tiered royalties reaching up to the mid-20% range based on annual net sales outside the United States.

The partnership reflects a growing trend in the biotechnology industry, where innovative drug developers collaborate with established rare disease specialists to accelerate global regulatory approvals and expand patient access. Should zilganersen receive regulatory clearance, the collaboration between Ionis and Recordati could significantly improve treatment availability for patients affected by one of the world’s rarest neurodegenerative disorders.