Clinical Trial News

ZUMA-2 study, cohort 3, showed durable responses to brexucabtagene autoleucel in BTK inhibitor–naive mantle cell lymphoma patients, with 91% objective response rate and 73% complete response at 15.5 months median follow-up. No new safety signals detected.

FDA granted accelerated approval to encorafenib (Braftovi) with cetuximab and mFOLFOX6 for metastatic colorectal cancer with BRAF V600E mutation. Efficacy was confirmed in the BREAKWATER trial, showing 61% objective response rate in the treatment arm vs. 40% in the control arm. Common adverse reactions include peripheral neuropathy and nausea. Recommended dose is 300 mg of encorafenib daily with cetuximab and mFOLFOX6.

In the JAVELIN Renal 101 trial, avelumab + axitinib showed longer PFS, higher ORR, and more durable responses vs sunitinib in aRCC patients, with manageable safety.

FDA grants Pfizer's Braftovi accelerated approval for BRAF V600E-mutant metastatic colorectal cancer, in combo with cetuximab and mFOLFOX6. BREAKWATER trial showed 61% ORR vs. 40% in control, with median DOR of 13.9 vs. 11.1 months. Common AEs include peripheral neuropathy, nausea, and fatigue.

5-year follow-up of SEQUOIA trial showed zanubrutinib maintained superior progression-free survival over bendamustine plus rituximab in untreated CLL/SLL patients, with no new safety signals.

The FDA granted priority review to taletrectinib for ROS1-positive advanced NSCLC, supported by phase 2 TRUST trials showing ORR of 88.8% in TKI-naive patients and 55.8% in previously treated patients. The FDA target action date is June 23, 2025.

Vertex Pharmaceuticals announced FDA approval of Alyftrek, a once-daily CFTR modulator for cystic fibrosis, designed to improve on Trikafta. Alyftrek, a triple-combination therapy, shares one active ingredient with Trikafta but offers benefits such as greater CFTR function control and once-daily dosing. Vertex sees potential in converting patients from Trikafta to Alyftrek and targeting those who have discontinued CFTR modulators. The list price for Alyftrek is $370,000, a 7% premium over Trikafta.

JCR Pharmaceuticals initiated Phase III trial of JR-142, a long-acting growth hormone for pediatric patients with hormone deficiency, aiming to verify non-inferiority compared to daily Growject over 52 weeks. The trial involves 54 pediatric patients and focuses on efficacy, safety, and growth outcomes.

The FDA granted Breakthrough Therapy designation to dostarlimab (Jemperli), showing a 100% clinical response rate for locally advanced dMMR/MSI-H rectal cancer. Jemperli, a PD-1-blocking monoclonal antibody, offers an immunotherapy option without chemotherapy, radiation, or surgery, with ongoing trials supporting its potential.

Zepbound, Eli Lilly's weight loss medication, became the first FDA-approved drug for obstructive sleep apnea on Dec. 20. Trials showed clinically meaningful reductions in apnea events and higher remission rates for mild obstructive sleep apnea compared to placebo. The approval is expected to influence payer coverage of GLP-1 drugs and impact the CPAP machine market.