Ionis reports promising results for HAE treatment By Investing.com

CARLSBAD, Calif. – Ionis Pharmaceuticals, Inc. (NASDAQ: NASDAQ:) today unveiled positive outcomes from its Phase 3 clinical trials for donidalorsen, a treatment for hereditary angioedema (HAE). The data, presented at the European Academy of Allergy and Clinical Immunology Congress, showed that donidalorsen significantly reduced monthly HAE attack rates and improved patients’ quality of life.

The OASIS-HAE study compared donidalorsen injections every four weeks or eight weeks against a placebo. Results revealed an 81% decrease in monthly attack rates for the four-week dosing and a 55% reduction for the eight-week dosing, both statistically significant. Additionally, over 25 weeks, the four-week dosing regimen demonstrated a reduction in severe to moderate attacks by 89% and attacks requiring acute therapy by 92%.

Patients transitioning from other HAE prophylactics to donidalorsen in the OASISplus study experienced a further 62% reduction in attack rates. Furthermore, 84% of these patients preferred donidalorsen over their previous treatments, citing better control and ease of administration.

Donidalorsen was generally well-tolerated across both studies, with no serious treatment-related adverse events reported. The most common side effect noted was injection site reactions.

Ionis CEO Brett Monia expressed optimism about donidalorsen’s potential to improve the treatment landscape for HAE, a genetic condition characterized by recurrent swelling attacks. The company is seeking regulatory approval for donidalorsen, aiming to add it to their commercial pipeline upon approval.

The comprehensive OASIS clinical program has positioned donidalorsen as a promising prophylactic option for HAE patients, with the potential for monthly or bimonthly self-administration via an autoinjector.

The information in this article is based on a press release.

InvestingPro Insights

As Ionis Pharmaceuticals (NASDAQ: IONS) advances its clinical trials for donidalorsen, the company’s financial health and market performance remain critical for investors. According to InvestingPro data, Ionis has a market capitalization of $5.33 billion, reflecting its significant presence in the biopharmaceutical sector. Despite a challenging revenue trajectory with a quarterly decline of 8.45% in Q1 2023, the company has shown a strong revenue growth of 34.84% over the last twelve months as of Q1 2023. This growth is a testament to Ionis’s ongoing efforts to innovate and expand its product offerings.

The company’s stock is currently trading near its 52-week low, which may indicate a potential buying opportunity for long-term investors. However, it’s worth noting that Ionis is trading at a high Price / Book multiple of 17.98, which could suggest the stock is overvalued relative to its assets. Furthermore, with a negative gross profit margin of -19.02%, Ionis faces challenges in turning its revenues into actual profits, which is consistent with the InvestingPro Tip highlighting the company’s weak gross profit margins.

Investors should also consider that Ionis does not pay a dividend, which may influence the investment strategy for those seeking regular income. Nevertheless, the company has liquid assets that exceed its short-term obligations, providing some financial stability. For investors looking for in-depth analysis, there are additional InvestingPro Tips available, including insights on earnings revisions and the company’s anticipated sales decline. To explore these further, visit https://www.investing.com/pro/IONS and use coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.

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