Ionis Pharmaceuticals IONS announced that the European Commission (“EC”) has approved Wainzua (eplontersen) for treating adult patients with hereditary transthyretin-mediated amyloidosis with stage 1 or stage 2 polyneuropathy (commonly called hATTR-PN or ATTRv-PN). The drug has been developed in partnership with AstraZeneca AZN.

This approval was expected as the EMA’s Committee for Medicinal Products for Human Use (“CHMP”) issued a positive opinion recommending the approval of Wainzua in the above indication in October.

The EC and CHMP decisions are based on positive data from the phase III NEURO-TTRansform study, which evaluated the safety and efficacy of Wainzua in patients with ATTRv-PN. Data from this study showed that treatment with the drug led to consistent and sustained benefit in the co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment. Treatment with Wainzua also improved quality of life versus placebo, a key secondary endpoint of the NEURO-TTRansform study.

With this approval, Wainzua has become the only approved medication in the EU for ATTRv-PN that can be self-administered monthly via an auto-injector. The drug was approved for a similar indication by the FDA in December 2023 and is being marketed under the name Wainua — also approved on data from the NEURO-TTRansform study.

ATTRv-PN is a debilitating disease, which leads to peripheral nerve damage and affects an estimated 40,000 people worldwide. It can cause motor disability within five years of diagnosis, which can become fatal if not treated properly within a decade.

IONS Stock Performance

Year to date, shares of Ionis have underperformed the industry, as seen in the chart below.

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More Updates on IONS/AZN's Wainzua

While Ionis and AstraZeneca jointly market Wainua for ATTRv-PN in the United States, AZN has exclusive rights to commercialize it outside U.S. markets. In the United States, Ionis receives royalties from AstraZeneca on the drug’s sales.

AstraZeneca and Ionis are also developing Wainua/Wainzua for the treatment of transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in the phase III CARDIO-TTRansform study. Data from the study is expected next year.

Ionis Boasts a Diverse Revenue Stream

IONS enjoys a diverse stream of revenues, including commercial products and royalties and numerous sources of collaborative and R&D revenues. Apart from AstraZeneca, the company has collaboration deals with leading drugmakers/biotech companies, such as Biogen BIIB, GSK plc GSK and Novartis, to develop and market its medicines.

Ionis earns commercial revenues in the form of royalty payments on net sales of Spinraza, approved in the United States to treat spinal muscular atrophy (SMA) worldwide. Ionis licensed this drug to Biogen, which is responsible for commercializing it. Ionis and Biogen also market Qalsody, which was approved by the FDA in April 2023 for amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations.

Novartis and GSK are its partners for pelacarsen and bepirovirsen, respectively. While the GSK-partnered drug is being developed in two late-stage studies for chronic hepatitis B (CHB), the Novartis-partnered drug is being developed in a late-stage study for patients with cardiovascular disease due to elevated Lp(a) levels. Data from the Novartis and GSK-partnered drug studies are expected in 2025 and 2026, respectively.

Ionis’ collaboration with the above leading drugmakers/biotech companies provides it with funds to invest in its wholly-owned pipeline to drive future revenue growth. Recently, it achieved a milestone when the FDA approved Tryngolza in familial chylomicronemia syndrome (FCS) indication. Besides being Ionis’ first wholly-owned drug, the drug also marks the company’s first independent commercial launch. A regulatory filing for this FCS drug is under review in the EU.

Some of its other wholly-owned candidates include donidalorsen for hereditary angioedema (HAE), zilganersen for Alexander’s disease and ulefnersen for amyotrophic lateral sclerosis (ALS). These drugs are already being evaluated in late-stage studies. The expansion of its internal pipeline should help Ionis diversify its revenue stream and lower its dependence on collaboration partners.

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 Rank (Strong Buy) stocks here.

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This article originally published on Zacks Investment Research (zacks.com).

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