IONS Out-Licenses ex-U.S. Rights for Rare Neurology Drug to Recordati

Ionis Pharmaceuticals IONS signed a license agreement with Italy-based Recordati. Per the terms, Recordati will acquire exclusive rights to develop and market its wholly owned investigational drug, zilganersen, outside the United States.

A regulatory filing is currently under review with the FDA, seeking approval for the drug to treat Alexander disease (AxD), a rare, progressive and often fatal neurological disorder. A final decision is expected by Sept. 22, 2026.

If approved, zilganersen, an RNA-targeted therapy, would become Ionis’ third wholly owned approved drug, joining Tryngolza (for severe hypertriglyceridemia) and Dawnzera (for HAE). It would also mark Ionis’ commercial entry into the neurology space, diversifying its commercial portfolio of wholly owned products.

Under the terms of the agreement, Recordati will assume responsibility for regulatory filings and commercialization of zilganersen across ex-U.S. markets, while Ionis will retain exclusive commercialization rights in the United States.

In exchange, Ionis will receive $30 million as an upfront payment and will be eligible for milestone payments. The company will also earn tiered royalties of up to the mid-20% range on annual ex-U.S. net sales of zilganersen.

Zilganersen's Potential Backed by Positive Clinical Data

The FDA filing is supported by positive results from a pivotal late-stage study evaluating zilganersen in children and adults living with AxD. The study met its primary endpoint; patients who received a 50 mg dose of zilganersen achieved a statistically significant and clinically meaningful stabilization in gait speed compared with control. Treatment with the candidate also demonstrated a favorable safety and tolerability profile.

AxD is a rare neurological disease that affects astrocytes, a type of brain cell that plays multiple roles in supporting neurons and oligodendrocytes. People living with this condition experience cognitive dysfunction and progressive neurologic deterioration, including difficulty swallowing and the inability to control muscles for large movements. There are currently no disease-modifying medicines approved for AxD patients.

If approved, zilganersen would be the first disease-modifying treatment for AxD.

IONS Stock Performance

Year to date, Ionis’ shares have lost 1% compared with the industry’s nearly 6% decline.

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Recordati Deal Reinforces Ionis' U.S.-First Commercial Strategy

While the licensing agreement provides Ionis with an upfront payment and the potential for future milestone and royalty income, it underscores the company's broader commercial strategy. Rather than out-licensing zilganersen globally, Ionis has retained exclusive commercialization rights in the United States, while entrusting Recordati with regulatory and commercial responsibilities in international markets.

This structure allows Ionis to preserve the long-term value of the U.S. market, the largest commercial opportunity for the therapy, while leveraging Recordati's established infrastructure in rare diseases to expand zilganersen's reach outside the country.

The agreement aligns with Ionis' strategy of partnering with established companies to commercialize its wholly owned medicines outside the United States. Last year, the company granted Sweden-based Sobi exclusive rights to commercialize Tryngolza in markets outside the United States, Canada and China. Ionis entered into a similar licensing agreement with Japan-based Otsuka Pharmaceutical to market Dawnzera across Europe and Asia Pacific. These partnerships enable Ionis to broaden the global reach of its therapies while maintaining a focused commercial presence in its home market.

Ionis Pharmaceuticals, Inc. Price

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IONS’ Zacks Rank

Ionis currently carries a Zacks Rank #3 (Hold).

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Some better-ranked stocks from the sector are Immunocore IMCR and Indivior Pharmaceuticals INDV, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Over the past 60 days, estimates for Immunocore’s 2026 bottom line have improved from a loss per share of 88 cents to earnings of 6 cents. Over the same period, estimates for 2027 EPS have risen from 24 cents to 87 cents. IMCR’s shares have lost 12% year to date.

Immunocore’s earnings beat estimates in three of the trailing four quarters but missed the mark on one occasion, delivering an average surprise of 46.66%.

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Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 65.44%.

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