The BioPharm Brief: Trials Test CAR-T, Immunology and Targeted Oncology

Welcome to The BioPharm Brief, your daily snapshot of the latest developments shaping biopharmaceutical research, clinical progress, and regulatory strategy. Today’s update explores experimental cell therapies for brain tumors, a combination approach for ulcerative colitis, mixed results in a rare bone disease, and a late-stage radiopharmaceutical trial success in neuroendocrine tumors.

T-MAXIMUM Pharmaceutical has received FDA clearance to begin a phase 2 study of MT027, an investigational, donor-derived chimeric antigen receptor T-cell therapy targeting B7-H3 in brain metastases from lung or breast cancer. The clearance marks the program’s second phase 2 study authorization, following its development in recurrent glioblastoma. Early results in a small group of patients with lung cancer brain metastases showed partial responses in 2 of 3 evaluable patients. Larger studies will need to establish whether these preliminary findings translate into meaningful clinical benefit.

Shifting from oncology to inflammatory disease, Odyssey Therapeutics is advancing a phase 2a trial combining OD-001 with vedolizumab for moderate-to-severe ulcerative colitis. The study plans to enroll up to 146 patients and will test whether targeting both innate and adaptive immune pathways can improve disease control. OD-001 inhibits receptor-interacting protein kinase 2-mediated innate immune activation, while vedolizumab limits immune-cell trafficking to the gut. The combination trial’s induction results are expected in the second half of 2027.

In rare disease research, Alexion’s efzimfotase alfa missed the primary endpoint in the phase 3 HICKORY trial for hypophosphatasia in adolescents and adults. Although treated patients walked farther than those receiving placebo, the difference on the 6-minute walk test was not statistically significant. Measures of fatigue and certain subgroup analyses favored treatment, but these findings remain exploratory. FDA is reviewing the therapy, with a decision expected in the first half of 2027.

By contrast, Bristol Myers Squibb reported that RYZ101 met the primary endpoint in the phase 3 ACTION-1 trial, improving progression-free survival in patients with advanced gastroenteropancreatic neuroendocrine tumors whose disease progressed after lutetium-177-based therapy. Detailed efficacy figures have not yet been released, and the company plans to discuss next steps with FDA.

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Key takeaways

• MT027 advances into phase 2 testing for brain metastases from lung and breast cancer.
• Odyssey tests a dual-pathway approach to ulcerative colitis, while efzimfotase alfa faces uncertainty after missing its primary endpoint.
• RYZ101 meets its phase 3 primary endpoint, potentially expanding options for patients with previously treated neuroendocrine tumors.