Recordati secures ex-U.S. rights to Ionis’ Alexander disease therapy
Italian pharma Recordati has entered into a license agreement with Ionis Pharmaceuticals, Inc. for exclusive development and marketing rights to zilganersen, an investigational RNA-targeted medicine for the treatment of Alexander disease (AxD), in all countries outside the U.S.
Why it matters: Alexander disease (AxD) is a rare, progressive and often fatal neurological disorder caused by mutations in the GFAP gene. The disease affects astrocytes, critical support cells in the brain, leading to progressive loss of neurological function, including mobility, independence, swallowing and breathing. Symptoms vary by age of onset, and patients often die within 14–25 years after symptoms begin. There are currently no approved disease-modifying therapies for AxD.
By the numbers: Ionis receives $30 million up front, and is eligible to receive additional payments based on achievement of milestones and a tiered royalty of up to the mid-20% range on annual net sales.
Zoom in: Zilganersen’s pivotal study met its primary endpoint in individuals aged five or older, with the 50 mg dose demonstrating statistically significant and clinically meaningful stabilisation of gait speed as assessed by the 10-Meter Walk Test (10MWT), a commonly used measure of gross motor function in neurologic disease, compared to control at Week 61. Secondary and exploratory endpoint results from patient/caregiver- and clinician-reported outcome assessments consistently favored zilganersen.
How it works: Zilganersen is an antisense oligonucleotide medicine, designed to inhibit production of excess glial fibrillary acidic protein (GFAP) that accumulates because of disease-causing variants of the gene.
What’s next: Recordati will be responsible for regulatory filings and marketing outside the U.S., including country-specific support for early access pathways based on local regulations and access dynamics.
- Ionis plans to independently launch zilganersen in the U.S., which is currently under decision date of 22nd September.
- As the potential first and only disease modifying treatment for AxD, zilganersen would also mark Ionis’ first independent commercial launch in neurology.
The bottom line: If approved, zilganersen could be an important addition to Recordati’s portfolio of around 20 rare disease drugs. This includes Enjaymo (sutimlimab), a treatment for cold agglutinin disease that Recordati bought from Sanofi in 2024 in a deal worth up to $1.075 billion ($825 million upfront, plus $250 in sales milestone payments).
Yes, but: Recordati is one of Italy’s only publicly traded large pharmaceutical companies. At the end of March, private equity firm CVC Capital Partners and Belgian investment group Groupe Bruxelles Lambert (GBL) launched a $12.4bn (€10.7bn) takeover bid for Recordati. The outcome remains uncertain and will depend on further negotiations and regulatory approvals.
