Tonix Pharmaceuticals stock halted ahead of FDA approval news
CARLSBAD, Calif. - Biogen reported positive interim results from a Phase 1 study of salanersen (ION306/BIIB115), an investigational treatment for spinal muscular atrophy (SMA), according to a press release statement.
The drug, developed using Ionis Pharmaceuticals’ (NASDAQ:IONS) antisense technology, was administered as a once-yearly dose to children with SMA who had previously received gene therapy but showed suboptimal clinical responses. Ionis, with a market capitalization of $6.3 billion and strong liquidity position (current ratio of 9.66), has generated revenues of $717 million in the last twelve months. InvestingPro analysis indicates the company maintains moderate debt levels while showing robust short-term financial strength.
In the open-label portion of the trial involving 24 participants, those who received either 40 mg or 80 mg of salanersen experienced substantial reductions in neurofilament light chain (NfL), a marker of neurodegeneration. Patients with elevated baseline NfL levels showed mean reductions of 70% at six months, which were maintained throughout the one-year dosing interval.
Among eight participants aged 2-12 who received the 40 mg dose and completed at least one year of follow-up, half achieved new World Health Organization motor milestones they previously could not accomplish independently, such as walking, crawling, standing or sitting.
These participants also showed improvements in motor function measurements, with mean increases of 3.3 points on the Hammersmith Functional Motor Scale-Expanded and 5.3 points on the Revised Upper Limb Module.
The drug was generally well-tolerated, with most adverse events being mild to moderate. The most common side effects reported were fever and upper respiratory tract infection.
Salanersen uses the same mechanism of action as Biogen’s approved SMA treatment SPINRAZA (nusinersen) but is designed for greater potency and less frequent dosing.
Biogen is currently engaging with global health authorities regarding the design of Phase 3 studies. Ionis Pharmaceuticals (NASDAQ:IONS) discovered salanersen and licensed global rights to Biogen. The market has responded positively to recent developments, with Ionis shares showing a 7% gain over the past week. According to InvestingPro, which offers comprehensive analysis of 1,400+ stocks including IONS, seven analysts have recently revised their earnings expectations upward for the upcoming period. Subscribers can access additional insights, including 10 more ProTips and detailed financial metrics, through the platform’s Pro Research Report.
In other recent news, Ionis Pharmaceuticals reported positive topline results from its Phase 3 study of olezarsen, a treatment for moderate hypertriglyceridemia. The study demonstrated a significant reduction in triglyceride levels, achieving a 61% reduction at an 80 mg dose and a 58% reduction at a 50 mg dose, both with strong statistical significance. Additionally, Ionis has initiated a Phase 3 trial for ION582, an investigational treatment for Angelman syndrome, with the first participant already dosed. The trial aims to evaluate improvements in expressive communication among participants. Meanwhile, Stifel analysts have maintained a Hold rating on Ionis, with a price target of $38, following discussions about the company’s pipeline and strategic initiatives. Ionis is also progressing with its cardiovascular outcomes trial for eplontersen, with results anticipated in 2026. In leadership news, Ionis announced that Richard Geary, Executive Vice President and Chief Development Officer, will retire in January 2026, with Holly Kordasiewicz set to succeed him. At its recent annual stockholders meeting, Ionis elected four directors and approved amendments to its 2011 Equity Incentive Plan.
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