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CARLSBAD, Calif. - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), a $5 billion market cap biotechnology company currently trading near its 52-week low of $30.23, has shared additional data from its clinical studies on donidalorsen, an investigational RNA-targeted medicine for hereditary angioedema (HAE), at the 2025 AAAAI/WAO Joint Congress in San Diego. According to InvestingPro data, while the company maintains strong liquidity with a current ratio of 8.51, it faces near-term challenges with revenue declining 10.49% over the last twelve months. The company reported findings from the Phase 3 OASIS and OASISplus studies, as well as three-year data from a Phase 2 study, highlighting the drug’s potential to reduce HAE attack rates and improve patient quality of life.
The new analyses suggest that donidalorsen may offer benefits to patients with HAE, a genetic condition that causes severe swelling attacks. The drug is currently under review by the U.S. Food and Drug Administration (FDA), with a Prescription Drug User Fee Act (PDUFA) target action date set for August 21, 2025. With analyst price targets ranging from $37 to $78, as revealed by InvestingPro’s comprehensive analysis, the market appears to be closely watching this regulatory milestone.
Kenneth Newman, M.D., senior vice president and head of clinical development at Ionis, commented on the significance of the findings. He emphasized the need for an effective and well-tolerated prophylactic treatment for HAE, noting that donidalorsen could be administered monthly or every two months using an autoinjector.
The presentations at the congress included a subanalysis of adolescent patients, long-term prophylaxis results, and patient-reported outcomes such as injection-site pain and treatment satisfaction. Ionis highlighted the drug’s sustained efficacy and safety profile, which could make it the prophylactic treatment of choice for those living with HAE.
Donidalorsen targets prekallikrein (PKK), a key player in activating inflammatory mediators associated with acute HAE attacks. By reducing PKK production, the drug aims to prevent these attacks. However, it is important to note that donidalorsen remains an investigational medicine and has not yet been approved for any disease by regulatory authorities.
Ionis has been at the forefront of RNA-targeted medicine for three decades, with a pipeline focused on neurology, cardiology, and other areas of high patient need. The company’s commitment to innovation and patient care continues to drive its research and development efforts. Despite reporting a net loss of $454 million in the last twelve months, InvestingPro analysis reveals 8+ additional key insights about the company’s financial health and future prospects, available through their detailed Pro Research Report, which provides comprehensive analysis of over 1,400 US stocks.
While the press release contains forward-looking statements about the potential of donidalorsen and Ionis’ business, these are subject to risks and uncertainties inherent in the development of pharmaceutical products. The information provided is based on a press release statement from Ionis Pharmaceuticals, Inc.
In other recent news, Ionis Pharmaceuticals reported its fourth-quarter 2024 earnings, surpassing analyst expectations with an earnings per share (EPS) of -0.66, significantly better than the forecasted -1.11. The company’s revenue reached $227 million, exceeding the anticipated $135.58 million, driven by strong product sales, including SPINRAZA royalties and WAYNEUWA product revenue. Raymond (NSE:RYMD) James maintained a Strong Buy rating on Ionis Pharmaceuticals, though it slightly reduced the price target from $61 to $60. Meanwhile, Stifel lowered its price target to $38, maintaining a Hold rating, citing increased spending assumptions.
Ionis has successfully launched Wainua for the treatment of ATTR-PN in the U.S., with $10 million in royalties for the last quarter, contributing to the full year’s $20 million. The company is preparing for several key developments, including the Phase 3 trial for ION582 in Angelman syndrome scheduled for the first half of 2025 and the anticipated European Union approval for Wainua following a positive opinion from the CHMP. The launch of Tryngolza for familial chylomicronemia syndrome is underway, with no payer resistance reported.
Additionally, Ionis is advancing its pipeline, with the Phase 3 trial for ATTR-CM expected to yield data in the second half of 2026. The cardiovascular disease outcomes data for Pelacarsen, partnered with Novartis (SIX:NOVN), has been delayed to the first half of 2026. The company anticipates a New Drug Application filing in 2026, conditional on positive data, which could result in a $50 million payment upon acceptance. Ionis is also progressing with donidalorsen for hereditary angioedema, with an FDA PDUFA date set for August 21, 2025.
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