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Ionis (IONS) Partner Wraps Up Cardiovascular Study Enrollment

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Ionis Pharmaceuticals (IONS - Free Report) announced that its partner, Novartis (NVS - Free Report) has completed the enrollment of participants in a pivotal phase III Lp(a) HORIZON study, which is evaluating its investigational antisense drug, pelacarsen, to reduce cardiovascular events in patients by lowering elevated lipoprotein(a) or Lp(a).

The Lp(a) HORIZON study is evaluating pelacarsen to reduce cardiovascular risks in patients with a confirmed cardiovascular disease and elevated levels of Lp(a). The study participants will be subcutaneously administered 80mg of pelacarsen, monthly. Top-line data from this study is expected in 2025.

This late-stage study was initiated following the success of the phase II study, wherein treatment with pelacarsen reduced Lp(a) levels below the recommended level in 98% of study participants with the same dosage levels as the Lp(a) HORIZON study.

In the year so far, shares of Ionis have risen 28.2% against the industry’s 20% decline.

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Per management estimates, more than 8 million patients worldwide are living with elevated Lp(a) levels. Presently, there are no approved therapies or treatments available to lower Lp(a) levels.

Pelacarsen has been designed to inhibit production of apolipoprotein(a) in the liver, which in turn will reduce elevated Lp(a) levels. An elevation of Lp(a) levels is responsible for inducing cardiovascular risks, which cannot be controlled with changes in diet or lifestyle.

Pelacarsen was licensed by Ionis to Novartis in 2019. Per the terms of the agreement between the two companies, Novartis is responsible for leading the global development and commercialization activities for the candidate.

Other than Novartis, Ionis has partnerships with many drug giants like AstraZeneca (AZN - Free Report) and Biogen (BIIB - Free Report) . These partnerships enable IONS to enjoy diverse revenue streams, which include commercial products and royalties, as well as, numerous sources of collaborative and R&D revenues.

The company is developing another antisense drug, eplontersen, in collaboration with AstraZeneca, to treat patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). Last month, Ionis and AstraZeneca reported data from the phase III NEURO-TTRansform study, which evaluated eplontersen in ATTRv-PN. The study met its co-primary and secondary endpoints. Based on these results, Ionis and AstraZeneca plan to file a new drug application, seeking marketing approval for eplontersen in ATTRv-PN later this year.

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 (SMA) in pediatric and adult patients. While BIIB is responsible for commercializing Spinraza worldwide, Ionis receives royalties from Biogen on Spinraza’s sales.

Biogen has also expanded its collaboration with Ionis to identify new gene therapies for the treatment of SMA as well as a broad range of neurological diseases. Under these collaborations, the companies together are currently developing numerous investigational medicines to treat neurodegenerative diseases like amyotrophic lateral sclerosis (ALS), SMA, Alzheimer’s disease and Parkinson’s disease. Some candidates that Ionis is developing in partnership with Biogen are tofersen for SOD1-ALS, ION541 for ALS and ION859 for Parkinson’s disease.

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Ionis currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.