Zilganersen(Ionis)

Today, a brief rundown of news involving Merck & Co. and MBX Biosciences, as well as updates from Roche, Sanofi and Ionis Pharmaceuticals that you may have missed.The Food and Drug Administration on Friday approved a form of Merck & Co.s Keytruda administered via an under-the-skin injection rather than through a lengthy infusion. Called Keytruda QLEX, it's been cleared for use in adults across most solid tumor indications for Keytruda, Merck said. The new version, which can be administered in one or two minutes, is one way Merck hopes to sustain Keytruda revenues following its loss of patent exclusivity later this decade. It also joins on the market similar subcutaneous forms of Bristol Myers Squibbs Opdivo and Roches Tecentriq, both of which have been approved since late 2024.Shares of MBX Biosciences more than doubled on Monday following results showing its experimental drug might have promise treating a rare endocrine condition called chronic hypoparathyroidism. According to the study findings, MBXs drug, canvuparatide, met its main goal, with 63% of treated patients maintaining normal levels of calcium in the blood over 12 weeks without needing a type of rescue therapy. The study succeeded despite a high rate of placebo responses and suggested a potential path forward as the first once-weekly treatment for the disease, wrote Jefferies analyst Roger Song. MBX went public in September 2024. Shares had been worth less than their debut price before the data announcement.Roche on Monday said a regimen involving its hormone-degrading drug giredestrant met both of its main goals in a Phase 3 trial in breast cancer, positioning the company to begin discussions with global health regulators. Roche didnt provide specifics, but said a combination of its drug and everolimus delayed disease progression in a key patient subgroup as well as the overall trial population compared to everolimus and standard care. Roche added that survival data were immature, but a clear positive trend was observed. The study, evERA, evaluated the combination in pretreated patients with metastatsic ER-positive, HER2-negative breast cancer.The FDA delayed by three months a decision on whether to approve Sanofis drug tolebrutinib for a form of multiple sclerosis. The agency had been expected to issue a verdict by Sept. 28, but revised that date to Dec. 28 because additional information submitted during the review was deemed a major amendment to Sanofis filing. Tolebrutinib could become the first therapy specifically approved for non-relapsing secondary progressive MS, a less common form of the disease in which people accumulate disabilities over time instead of experiencing periodic disease flares.Ionis Therapeutics on Monday said an RNA-based therapy its been developing met its objectives in a Phase 3 trial in a rare, deadly condition called Alexander disease. According to Ionis, treatment with zilganersen was associated with a statistically significant improvement on a type of walking test as well as consistent benefits on multiple secondary measures. Ionis will file an approval application next year. The findings add to a run of steady execution on clinical/regulatory milestones for Ionis, though the small addressable market estimated at less than 1,000 people in the U.S. could limit zilganersensfinancial impact on the company, wrote Leerinks Mani Foroohar. '

Ionis Pharmaceuticals intends to submit zilganersen for FDA approval in the first quarter of 2026, and it\'s also looking into launching an expanded access program. \n Ionis Pharmaceuticals is eyeing a new rare disease approval after its antisense oligonucleotide zilganersen successfully improved mobility in patients with the neurological condition Alexander disease.Of the 54 patients enrolled in a phase 1-3 trial, those given zilganersen could walk an average of 33.3% faster compared with baseline after 61 weeks, Ionis announced Sept. 22. The result squeaked into statistical significance with a p-value of 0.0412. Study participants received zilganersen or placebo every 12 weeks through Week 49, and their walk speed was measured 61 weeks after treatment began. At 60 weeks, eligible patients transitioned to an open-label extension period in which they continued receiving zilganersen.Most adverse events were mild or moderate, Ionis added, with more serious adverse events occurring in the placebo arm than in the treatment arm of the trial.Ionis intends to submit zilganersen for FDA approval in the first quarter of 2026, the California biotech said in the release, and is also looking into launching an expanded access program. The asset had previously snagged orphan drug and rare pediatric disease designations from the FDA as well as an orphan drug designation from the European Medicines Agency.Zilganersen treatment also led to positive changes in several secondary endpoints, Ionis said, though the company did not disclose whether these results were statistically significant. This includes improvements in patients’ self-reported most bothersome symptom and impression of their own disease severity as well as their clinicians\' impressions of their symptom improvement.This is the first time an investigational therapy has shown a disease-modifying effect in Alexander disease, according to the release.“These unprecedented results highlight the potential of zilganersen to create new possibilities for people living with Alexander disease,” Holly Kordasiewicz, Ph.D., Ionis’ senior vice president of neurology, said in the release. “These data demonstrate the promise of zilganersen to potentially transform the future treatment landscape for this condition and reinforce the power of our technology to address neurological diseases by directly targeting the underlying cause.” In Alexander disease, mutations in the gene that makes glial fibrillary acidic protein (GFAP) cause the protein to build up in the brain’s white matter, damaging the protective covering that shields nerve cells. This damage leads to physical and intellectual impairment as well as seizures and other symptoms like difficulty speaking and swallowing, depending on when the disease manifests.In some patients, the disease appears while still in the womb, with symptoms present right at birth.Zilganersen is designed to bind to the mRNA made from the GFAP gene to prevent it from being turned into protein, halting the toxic buildup that causes Alexander disease.