Ionis gains EU approval for amyloidosis treatment

CARLSBAD, Calif. - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), a $5.37 billion market cap biotechnology company, has received approval from the European Union for its treatment WAINZUA, intended for adult patients with hereditary transthyretin-mediated amyloidosis with stage 1 or stage 2 polyneuropathy. According to InvestingPro analysis, the company currently appears overvalued based on its Fair Value calculations, despite facing revenue challenges with a 10.48% decline in the last twelve months. The European Commission’s decision follows a positive assessment from the Committee for Medicinal Products for Human Use (CHMP) and is based on the Phase 3 NEURO-TTRansform trial results.

The NEURO-TTRansform trial demonstrated that WAINZUA, also known as eplontersen, provided consistent and sustained improvements in neuropathy impairment and quality of life over 66 weeks compared to a placebo. It has also maintained a favorable safety and tolerability profile throughout the trial. The company’s stock has shown resilience despite broader market challenges, maintaining a low beta of 0.28, indicating lower volatility compared to the market.

Hereditary transthyretin-mediated amyloidosis is a progressive and often fatal condition that causes peripheral nerve damage and motor disability, with a potential life expectancy of just a decade without treatment. WAINZUA, which can be self-administered monthly via an auto-injector, works by reducing the production of transthyretin protein, which is associated with the disease.

Brett P. Monia, Ph.D., CEO of Ionis, highlighted the significance of the approval, noting that WAINZUA is now an accessible treatment option in North America, the UK, and across the EU. The drug is marketed in the U.S. as WAINUA™ and was first approved in December 2023.

Ionis and AstraZeneca are collaborating on the development and commercialization of WAINUA in the U.S., with AstraZeneca holding exclusive rights in other global markets. The companies are pursuing further regulatory approvals worldwide.

WAINZUA is also being evaluated in a Phase 3 study for the treatment of adults with ATTR-cardiomyopathy, a form of the disease that predominantly affects the heart.

The information reported is based on a press release statement. Ionis Pharmaceuticals has a portfolio of marketed medicines and continues to innovate in RNA-targeted therapies and gene editing. The company maintains strong liquidity with a current ratio of 8.47, though InvestingPro data reveals challenges with profitability and gross margins. The company is dedicated to bringing advances to patients with serious diseases. For deeper insights into Ionis’s financial health and growth prospects, including additional ProTips and comprehensive analysis, investors can access the detailed Pro Research Report available on InvestingPro.

In other recent news, Ionis Pharmaceuticals reported robust financial results, with total revenue reaching $705 million, surpassing expectations by 13% to 15%. Earnings per share (EPS) also outperformed projections, coming in at ($0.43), which was significantly better than anticipated. Meanwhile, Ionis presented new data on its hereditary angioedema (HAE) drug, donidalorsen, at a major medical congress, highlighting its potential to reduce HAE attack rates and improve patient quality of life. The drug is currently under review by the FDA, with a decision expected by August 2025.

Analyst firms have made adjustments to their price targets for Ionis. Bernstein lowered its target to $43 while maintaining a Market Perform rating, citing the removal of more speculative assets from its model. Similarly, Raymond James slightly reduced its target to $60 but kept a Strong Buy rating, noting successful U.S. launches and anticipated European approvals. Stifel also cut its target to $38, maintaining a Hold rating, attributing the adjustment to increased spending assumptions.

Ionis is advancing its pipeline with multiple late-stage trials, including a Phase 3 trial for Angelman syndrome set to begin in 2025. The company is also preparing for the launch of Tryngolza for familial chylomicronemia syndrome and is seeking partnerships outside the U.S. for its severe hypertriglyceridemia treatments. Additionally, Ionis is set to receive $50 million upon the acceptance of a New Drug Application for Pelacarsen, a cardiovascular drug developed in partnership with Novartis.

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