Clinical Trial News

The FDA has granted accelerated approval to adagrasib combined with cetuximab for treating adults with KRAS G12C-mutated locally advanced or metastatic colorectal cancer, following prior chemotherapy treatments. This decision is based on the efficacy shown in the KRYSTAL-1 study, with a 34% overall response rate among patients.

Kyverna Therapeutics' KYV-101, a new CAR-T cell therapy for stiff person syndrome, received FDA's RMAT designation, promising accelerated development and approval. The therapy aims to reset patients' immune systems, offering hope for durable remission. Clinical trials show positive results, with ongoing studies for multiple sclerosis, myasthenia gravis, systemic sclerosis, and lupus.

Eli Lilly's Alzheimer's drug, Kisunla, received FDA approval, expected to generate billions. However, the stock didn't surge as the approval was already priced in. The company's focus and investor excitement are more on its GLP-1 treatments for obesity and diabetes, indicating future growth potential beyond Kisunla.

Phase 3 trial data showed the pozelimab and cemdisiran (poze-cemdi) combination achieved greater control of intravascular hemolysis in PNH patients compared to ravulizumab, with 96% achieving adequate LDH control versus 80% with ravulizumab. Poze-cemdi also demonstrated complete, uninterrupted inhibition of terminal complement, offering potential for less frequent, subcutaneous administration.

Ibrahim Aldoss presented phase 2 results of the WU-CART-007 trial at EHA 2024, showing a 91% overall response rate and manageable safety profile for W-T7, a CD7-targeted CAR T-cell therapy in relapsed/refractory T-ALL/LBL patients. A registration study is planned to further evaluate efficacy.

The AMPLIFY trial introduced AVO as a new standard for untreated CLL patients with wild-type TP53, excluding high-risk TP53 aberration cases. A phase II study evaluated AVO in high-risk CLL patients, showing 42% CR with BM-uMRD rates and 71% BM-uMRD rates in TP53 aberration patients. AVO was well-tolerated, with low-grade toxicities, supporting its use as a standard treatment for high-risk CLL.

Eli Lilly's tirzepatide, marketed as Mounjaro for diabetes and Zepbound for obesity, shows promise in treating sleep apnea, with recent studies indicating significant reductions in sleep apnea events. Lilly seeks FDA approval for this expanded use, potentially boosting sales. The company's strong pipeline, including Alzheimer's drug donanemab, and robust financial performance, driven by high demand for its drugs, position it well for future growth.

Uncommon Cures, a U.S. company, launched a boutique clinical trial program for rare diseases, underestimating the demand. With eight contracts and talks with over 50 clients, CEO Marshall Summar highlights the need for a new model blending regulatory tasks with a concierge-based facility. The model, expanding internationally with sites in D.C., Utah, Chile, Switzerland, and UAE, aims to streamline trials, reduce costs by over 40%, and improve patient access globally.

The global mRNA cancer vaccines clinical trials and market future outlook for 2024 highlights over 60 vaccines in trials, with the US and China leading in research. mRNA vaccines represent a novel immunotherapy method for cancer, leveraging the body's immune system to combat the disease. Despite no current approvals, the sector shows significant growth potential, with the first commercial mRNA vaccine expected by 2029. The technology's flexibility, rapid production, and personalized approach offer advantages over traditional cancer vaccines.

Spectral Medical Inc. announced MNP LLP as its new auditor following PricewaterhouseCoopers LLP's resignation, approved by the Finance & Audit Committee and board. PwC's resignation was not due to disagreements. Spectral, advancing sepsis treatment, looks forward to MNP's expertise. The company is in Phase 3 for FDA approval of its septic shock treatment, Toraymyxin™.