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In an eight-week obese non-human primate study, lean mass made up 6% of the weight lost with iBio’s Activin E antibody IBIO-610 plus semaglutide, compared with 22% with semaglutide alone. iBio plans to begin first-in-human studies in the first half of 2027.

Clinical results reveal new opportunities and setbacks in rare disease and cancer care, shaping treatment strategies and future regulatory decisions.

Phase 3 interim data for RYZ101, the lead asset from Bristol Myers Squibb’s $4.1 billion RayzeBio deal, meet the primary endpoint.

Efzimfotase alfa missed significance on the six-minute walk test in adolescents and adults, while the FDA review of the therapy proceeds toward 2027.

Odyssey's 146-patient phase 2a trial will pair OD-001 with vedolizumab, targeting both innate and adaptive immune drivers of ulcerative colitis.

MT027, T-MAXIMUM's B7-H3 CAR-T therapy, earns a second FDA-cleared phase 2 trial, now testing lung and breast cancer brain metastases.

New clinical data highlight improved disease control, reduced eye bulging, and weight-loss benefits, potentially broadening options for patients with unmet needs.

Antisense oligonucleotides are reshaping drug design, from RNase H gapmers to inhaled delivery; this FAQ explains how they work and why trials still fail.

Bristol Myers Squibb's Nathan Pennell explains how the MRD endpoint supports accelerated approval in multiple myeloma without changing how clinicians monitor patients.

Teprotumumab N01 (Sycume) reduced eye bulging in 60.4% of patients with chronic, inactive thyroid eye disease in a phase 3 trial.

Tecentriq plus chemotherapy gains FDA approval for stage III dMMR colon cancer after a phase 3 trial showed improved disease-free survival.

New clinical milestones, manufacturing investments, and regulatory incentives could help developers overcome barriers to delivering treatments for patients with rare diseases.

Genglycos, the gene therapy that earned the voucher, will fund new Ultragenyx therapies for other rare and ultra-rare diseases.