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 Squibb’s Opdivo and Roche’s 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, MBX’s 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 didn’t 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 Sanofi’s 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 Sanofi’s 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 it’s 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 zilganersen’s financial impact on the company, wrote Leerink’s Mani Foroohar.
