Ionis (IONS) Posts New Upbeat Data From Amyloidosis Drug Study

·3-min read

Ionis Pharmaceuticals IONS announced positive top-line data from the 66-week analysis of the phase III NEURO-TTRansform study on its investigational anti-sense drug eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The candidate is being developed in partnership with AstraZeneca AZN.

Analysis of the data from the NEURO-TTRansform study showed that treatment with eplontersen achieved its co-primary endpoints of a sustained reduction in transthyretin (“TTR”) concentration, benefits in neuropathy and quality of life through 66 weeks. These results were consistent with the positive 35-week analysis of the study reported last year in June.

The study met its first two co-primary endpoints of change in the modified Neuropathy Impairment Score +7 (mNIS+7) and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), with statistical significance. The mNIS+7 measures neuropathic disease progression, while the Norfolk QoL-DN is an instrument to assess the quality of life in diabetic polyneuropathy.

The study met its third co-primary endpoint of eplontersen, exhibiting a statistically significant and clinically meaningful improvement in percent change in serum TTR concentration.

A new drug application (NDA) was filed by Ionis and AstraZeneca with the FDA last December, seeking approval for eplontersen in ATTRv-PN. A final decision on this NDA is expected by this year’s end.

In the year so far, shares of Ionis have declined 10.1% compared to the industry’s 1.4% fall.

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AstraZeneca and Ionis entered a strategic collaboration last December to develop and commercialize eplontersen for treating TTR amyloidosis. AstraZeneca and Ionis are jointly responsible for developing and marketing the drug in the United States. However, AstraZeneca will have exclusive global rights to develop and commercialize the drug outside the country (excluding Latin America).

Apart from ATTRv-PN, Ionis and AstraZeneca also evaluating eplontersen as a potential treatment for amyloid transthyretin cardiomyopathy (ATTR-CM) in the phase III CARDIO-TTRansform study.

Other than AstraZeneca, Ionis has partnerships with many pharma giants like Biogen BIIB and Novartis NVS. These partnerships enable Ionis to enjoy diverse revenue streams, including commercial products and royalties and numerous sources of collaborative and R&D revenues.

Biogen and Ionis are collaborating on developing advanced treatments for neurological disorders. Ionis has licensed Spinraza to Biogen, which is approved for treating spinal muscular atrophy in pediatric and adult patients. While Biogen is responsible for commercializing Spinraza worldwide, Ionis receives royalties from Biogen on Spinraza’s sales.

The company is developing another pipeline candidate, pelacarsen, in collaboration with Novartis. Ionis and Novartis are evaluating pelacarsen in the ongoing phase III cardiovascular outcome study, HORIZON, in patients with established cardiovascular disease and elevated lipoprotein(a), or Lp(a). Novartis is responsible for leading the global development and commercialization activities for the candidate.

Ionis Pharmaceuticals, Inc. Price


Ionis Pharmaceuticals, Inc. Price
Ionis Pharmaceuticals, Inc. Price

Ionis Pharmaceuticals, Inc. price | Ionis Pharmaceuticals, Inc. Quote


Zacks Rank

Ionis currently carries a Zacks Rank #3 (Hold).You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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