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.

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.

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).

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).

Microbiome specialist Enterobiotix Ltd. has raised £19 million (US$25.7 million) to fund phase IIb development of its lead program EBX-102-02 in the treatment of irritable bowel syndrome with constipation (IBS-C).

Three decades of trial-and-error, and the resulting safety data, in the oligonucleotide-based therapeutic space have paved way for the present-day innovations and the promise of “programmable,” precision medicine for patients, speakers at Bio Korea 2026 said April 28.

China’s investigator-initiated trial (IIT) system is increasingly being used to generate early human data in cell and gene therapies, and new changes that widen the pathway are expected to drive more multinational companies to conduct IITs in China, panelists said during the Chinabio Partnering Forum in Shanghai April 28-29.

What PTC Therapeutics Inc.’s latest data with votoplam might mean in the Huntington’s disease (HD) landscape became grist for Wall Street after the firm unveiled top-line results from the phase II Pivot-HD study, sharing data from the 24-month interim analysis of the long-term extension effort.