Ionis Pharmaceuticals IONS announced positive results from two partnered programs, both of which are in phase III development.
IONS Secures Phase III Win in ALS
The first study, called FUSION, evaluated ulefnersen for the treatment of patients with amyotrophic lateral sclerosis (ALS) caused by mutations in the fused in sarcoma (FUS) gene. The study met its primary endpoint that assessed functional impairment and survival for 72 weeks.
Though Ionis did not report numbers supporting the results, it disclosed that they were statistically significant. The company plans to present detailed data at a future medical meeting and submit the same for publication in a peer-reviewed journal.
The FUSION results are particularly significant because FUS-ALS is a rare, genetically defined form of ALS for which there are currently no approved therapies specifically targeting its underlying genetic cause. Per Ionis, ulefnersen has the potential to be the first disease-modifying therapy for FUS-ALS patients.
Ionis licensed worldwide commercialization rights for the drug to Otsuka Pharmaceutical in 2024. The company is eligible for milestone payments, as well as tiered royalties on net sales.
Following the FUSION results, Otsuka plans to discuss the data with the FDA and other global health authorities regarding potential expedited regulatory submission pathways.
IONS Reports Positive Interim Results on Partnered Kidney Disease Drug
The second study, called IMAgINATION, was conducted by the company’s partner Roche RHHBY. This study evaluated sefaxersen in adults with primary immunoglobulin A nephropathy (IgAN), a rare, progressive kidney disease.
Unlike FUSION, where Ionis reported top-line results, IMAgINATION met its primary endpoint in a prespecified interim analysis. Although Ionis and Roche did not report the magnitude of the treatment effect, they stated that sefaxersen demonstrated statistically significant and clinically meaningful reductions in proteinuria (excess protein in the urine) after 37 weeks.
The companies plan to present detailed interim data at an upcoming medical meeting and share the results with health authorities. IMAgINATION will continue in a blinded manner to evaluate kidney function over two years, with estimated glomerular filtration rate (eGFR) at week 105 serving as the longer-term measure.
Roche licensed sefaxersen from Ionis in 2022 and is responsible for the phase III study, as well as future global development, regulatory and commercialization activities. Ionis is eligible for milestone payments, as well as tiered royalties on net sales of the drug.
IONS Stock Performance
Year to date, Ionis’ shares have lost 42% compared with the industry’s 2% decline.
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Recent Developments in IONS Stock
The two positive clinical updates come on the heels of back-to-back clinical setbacks for Ionis in two other partnered programs, both in the cardiovascular space. In July, Ionis and partner AstraZeneca AZN reported that the phase III CARDIO-TTRansform study on Wainua in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) failed to meet its primary endpoint. More recently, Ionis’ partner Novartis NVS reported that the phase III Lp(a)HORIZON study on pelacarsen also did not meet its primary endpoint.
Still, not every recent development has been negative. Earlier this month, Ionis secured FDA approval for Zanvastro for the treatment of pediatric and adult patients with Alexander disease (AxD), making it the first and only disease-modifying treatment for the rare neurological disorder. The approval marked Ionis’ first independent launch from its neurology pipeline and its second independent launch in 2026, further expanding the company’s portfolio of wholly owned marketed medicines. However, given the ultra-rare nature of AxD, the drug’s commercial opportunity is expected to be limited.
Against this mixed backdrop, the positive FUSION and IMAgINATION results provide positive late-stage clinical updates for two additional partnered Ionis programs.
Ionis Pharmaceuticals, Inc. Price
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IONS’ Zacks Rank
Ionis currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here.
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