CARLSBAD, Calif. - Ionis Pharmaceuticals, Inc. (NASDAQ: NASDAQ:IONS) has announced that new three-year data from studies of its investigational medicine donidalorsen will be presented at the upcoming American College of Allergy, Asthma & Immunology Annual Scientific Meeting. The data suggest that donidalorsen may offer significant and sustained reductions in hereditary angioedema (HAE) attack rates with monthly or every two-month dosing.
The presentations will feature results from the Phase 2 open-label extension study and the pivotal Phase 3 OASIS and OASISplus studies. These findings indicate that donidalorsen has the potential to provide high levels of disease control and improve the quality of life for patients with HAE, a rare and potentially life-threatening genetic condition characterized by recurrent attacks of severe swelling.
Donidalorsen is an RNA-targeted medicine designed to reduce the production of prekallikrein (PKK), which is involved in the activation of inflammatory mediators associated with acute HAE attacks. The investigational status of donidalorsen means it has not yet been approved for the treatment of any disease by regulatory authorities.
Ionis has a history of developing RNA-targeted therapies and is actively working on a pipeline of medicines in various areas of patient need. While the company has made forward-looking statements about the potential of donidalorsen and its other developments, these are subject to the inherent risks and uncertainties of drug discovery and development.
The new data from the donidalorsen studies will be shared on October 25, 2024, at the ACAAI meeting in Boston, Massachusetts. These results build on positive outcomes previously reported from the Phase 3 OASIS-HAE and OASISplus studies, which were presented at the European Academy of Allergy and Clinical Immunology Annual Congress and published in The New England Journal of Medicine.
This information is based on a press release statement from Ionis Pharmaceuticals, Inc.
In other recent news, Ionis Pharmaceuticals has received Fast Track designation from the FDA for zilganersen, a potential treatment for the ultra-rare and fatal neurological disorder, Alexander disease. This accelerates the development and review process for the drug, which is currently the only one in clinical development for both adult and pediatric patients with this disorder. The company expects pivotal study data in the latter half of 2025. Ionis also announced a proposed public offering of its common stock, expected to amount to $500 million, managed by Morgan Stanley and Goldman Sachs & Co. LLC. This funding will be directed towards commercial launches, clinical programs, research and development, and general corporate needs. RBC Capital maintained its Outperform rating on Ionis, highlighting the company's potential in several therapeutic areas. The company also reported steady financial results, aligning with annual guidance, and launched two new treatments, WAINUA for hereditary ATTR polyneuropathy and QALSODY for ALS, in the US and Europe.
InvestingPro Insights
As Ionis Pharmaceuticals (NASDAQ: IONS) prepares to present promising data on donidalorsen, it's crucial to consider the company's financial landscape. According to InvestingPro data, Ionis has shown significant revenue growth, with a 29% increase in the last twelve months as of Q2 2024, reaching $813.46 million. This growth aligns with the company's ongoing development of innovative RNA-targeted therapies like donidalorsen.
Despite the revenue growth, InvestingPro Tips highlight that Ionis suffers from weak gross profit margins, which stood at -12.86% in the same period. This suggests that while the company is expanding its top line, it faces challenges in profitability, likely due to high research and development costs associated with its pipeline of investigational medicines.
Another relevant InvestingPro Tip indicates that analysts do not anticipate the company will be profitable this year. This aligns with the nature of pharmaceutical companies investing heavily in drug development, as seen with Ionis's focus on donidalorsen and other potential therapies.
For investors interested in a deeper analysis, InvestingPro offers additional tips and metrics that could provide valuable context to Ionis's financial health and market position. There are 7 more InvestingPro Tips available for Ionis Pharmaceuticals, offering a more comprehensive view of the company's prospects as it advances its drug candidates through clinical trials.
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