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).

Shares of MBX Biosciences Inc. hit a 52-week high on preliminary phase I data for its MBX-4291, a GLP-1/GIP co-agonist prodrug for obesity. Though early and still blinded, findings from the trial showed promising efficacy, along with good tolerability and the potential for once-monthly dosing, factors that will likely be critical for MBX to compete in the crowded obesity space.

Climb Bio Inc. outlined May 5 an enticing data spill ahead this year with Fc-enhanced monoclonal antibody budoprutug (budo) in autoimmune diseases. Mizuho analyst Joseph Catanzaro appreciated in his report Wellesley Hills, Mass.-based Climb’s “conviction that CD19 represents a best-in-class pan-B-cell depletion target across antibody-mediated autoimmune diseases – a relative white space for conventional mAbs.”

Hopes piqued last November for detalimogene voraplasmid in bladder cancer took a hit from the latest word from the study, as did shares of Engene Therapeutics Inc. (NASDAQ:ENGN), which closed May 7 at $1.72, down 80.6%, or $7.13.

A lower-than-expected increase in dystrophin over baseline in the first and lowest-dose cohort of a phase I/II study of ENTR-601-44 in Duchenne muscular dystrophy (DMD) caused shares of Entrada Therapeutics Inc. to plunge more than 57%, despite the cohort meeting the safety and tolerability primary objective.

Avalo Therapeutics Inc. may have designed in lead asset abdakibart, a drug for hidradenitis suppurativa (HS) that offers a novel mechanism of action, wide dosing interval, and more-than-satisfying outcomes on the Hidradenitis Suppurativa Clinical Response 75 endpoint, just what the doctor – and Wall Street – ordered.

Medical Microinstruments Inc.’s (MMI) Symani surgical system was used to treat a patient with Alzheimer’s disease as part of a study evaluating robotic-enabled microsurgery for the condition. The procedure investigated whether restoring lymphatic drainage pathways in the deep cervical lymph nodes could improve the clearance of harmful neurotoxins believed to contribute to the progression of Alzheimer’s.

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.