Tonix Pharmaceuticals stock halted ahead of FDA approval news
Investing.com - H.C. Wainwright has reiterated its buy rating and $50.00 price target on Ionis Pharmaceuticals (NASDAQ:IONS) stock following promising clinical trial results for salanersen, a treatment for spinal muscular atrophy (SMA). The stock, currently trading at $40, has shown strong momentum with an 8% gain over the past week. According to InvestingPro data, analyst targets range from $37 to $83, with seven analysts recently revising their earnings expectations upward for the upcoming period.
The decision comes after Ionis announced Wednesday that its partner Biogen (NASDAQ:BIIB) reported top-line results from the Phase 1 trial of salanersen (ION306/BIIB115), an antisense oligonucleotide developed for SMA patients who previously received Novartis AG (SIX:NOVN)’s gene therapy ZOLGENSMA. While the company maintains strong liquidity with a current ratio of 9.66, InvestingPro analysis indicates it operates with moderate debt levels and faces profitability challenges in the current fiscal year.
Interim results from the open-label portion of the study showed that pediatric SMA patients who received either 40mg or 80mg of salanersen once per year experienced mean reductions in neurofilament light chain of 70% at six months, with the effect sustained through the one-year dosing interval.
In a subgroup with at least one year of follow-up, 50% of participants achieved new WHO motor milestones such as walking, crawling, standing, or sitting. These eight participants also demonstrated clinically meaningful improvements in motor function, including a 3.3-point mean improvement on the Hammersmith Functional Motor Scale - Expanded and a 5.3-point improvement on the Revised Upper Limb Module.
The treatment appeared safe at both the 40mg and 80mg doses, with most adverse events being mild-to-moderate in nature, while Biogen is now engaging with global health authorities on the Phase 3 program design. With the next earnings report due on July 31, investors seeking deeper insights can access comprehensive analysis and 12 additional ProTips through InvestingPro’s detailed research reports, available for over 1,400 US stocks.
In other recent news, Ionis Pharmaceuticals announced the initiation of its Phase 3 REVEAL study for ION582, targeting Angelman syndrome, a rare neurodevelopmental disorder. This global study aims to enroll approximately 200 participants to evaluate the drug’s effectiveness over a 52-week period. Meanwhile, Ionis Pharmaceuticals also reported changes in its leadership, with Richard Geary set to retire as Chief Development Officer in January 2026, to be succeeded by Holly Kordasiewicz. In a separate development, Ionis held its annual stockholders meeting, where all proposals, including the election of four directors and an amendment to the 2011 Equity Incentive Plan, were approved.
Biogen, in collaboration with Ionis Pharmaceuticals, shared promising interim results from a Phase 1 trial of salanersen, an investigational treatment for spinal muscular atrophy (SMA). Participants in the trial showed significant reductions in neurofilament light chain, a marker of neurodegeneration, and improvements in motor function. Stifel analysts maintained a Hold rating on Ionis Pharmaceuticals, with a price target of $38, highlighting the company’s ongoing projects and strategic partnerships. Ionis Pharmaceuticals is also advancing its cardiovascular outcomes trial for eplontersen, with results expected in 2026. Additionally, the company’s partnership with Biogen on an Alzheimer’s treatment is progressing, with phase 2b data anticipated in the same year.
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