Thanks to a trial design that, according to Evercore ISI analyst Cory Kasimov, “played out exactly as drawn up,” Cytokinetics Inc. hit both of the phase III co-primary endpoints with aficamten in symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).

Top-line data from the Reveal-2 phase III trial of elegrobart to treat chronic thyroid eye disease (TED) drove Viridian Therapeutics Inc.’s stock up by 33.6%, positioning the company for a BLA filing in the first quarter of 2027. The trial met both primary endpoints at week 24: proptosis responder rate, as measured by exophthalmometry, required for a filing with the U.S. FDA; and the overall responder rate, required for an EMA filing. Doses every four weeks or every eight weeks achieved 50% and 54% PRR, respectively, compared to 15% for placebo. The doses also achieved 47% and 54% ORR vs. 15% for placebo. ORR included those with both proptosis and Clinical Activity Score response, defined as no worsening from baseline in the study eye, without deterioration in the fellow eye.

Regulatory uncertainty that dominated the life science sector in 2025 is transitioning into an execution framework for biotechnology companies, provided companies can tailor regulatory strategies early, and across the entire development cycle.

As Celcuity Inc. awaits a U.S. FDA approval decision for gedatolisib in HER-positive, HER2-negative, PIK3CA wild-type advanced breast cancer, the company already is setting its sights on a supplemental NDA filing, based on a top-line readout of the multi-target PAM inhibitor in a PIK3CA-mutant breast cancer cohort.

For the first time in 13 years, the U.S. Trade Representative (USTR) singled out one of the U.S. trading partners as the worst of the worst when it named Vietnam as a Priority Foreign Country (PFC) in its newly released Special 301 Report.

Entering what Mirum Pharmaceuticals Inc. CEO Chris Peetz called “a new phase of growth and value creation,” the company plans to submit an NDA to the U.S. FDA in the second half of this year, based on phase IIb data from the Vistas trial with oral ileal bile acid transporter (IBAT) inhibitor volixibat in primary sclerosing cholangitis (PSC).

Beone Medicines Ltd. gained an exclusive option to develop cancer immunotherapy HH-160 worldwide in a deal potentially worth more than $2 billion for Huahui Health Ltd., the developer of the trispecific antibody.

Sonire Therapeutics Inc. initiated a U.S.-based Sunrise II study of Suizenji, its novel ultrasound-guided high-intensity focused ultrasound (HIFU) system designed to ablate pancreatic tumors, after closing an $18 million series A financing round April 15.

Wall Street pundits were divided about the likely fate of ivonescimab, Summit Therapeutics Inc.’s bispecific antibody partnered with Akeso Pharmaceuticals Inc., of Hong Kong, and undergoing phase III testing in first-line squamous non-small-cell lung cancer (NSCLC).