Clinical Trial News

RAPT Therapeutics' RPT193 for atopic dermatitis saw its Phase Transition Success Rate drop to 78% despite positive Phase Ib trial results. The drug showed a 36.3% improvement in EASI score over placebo. RPT193's Likelihood of Approval decreased to 3%. Rapt plans a Phase IIb trial, targeting CCR4 on Th2 cells.

Agios Pharmaceuticals has submitted a New Drug Application to the FDA for mitapivat, aiming to treat adults with pyruvate kinase deficiency, a rare genetic disease causing chronic hemolytic anemia. This submission is based on results from pivotal studies, highlighting the potential of mitapivat as a disease-modifying therapy.

NGS-MRD assay shows prognostic value in AML patients post-allo-HSCT, with persistent mutations at pre-HSCT and post-HSCT-1m linked to higher relapse and mortality risks. Conditioning intensity affects NGS-MRD's prognostic impact, suggesting serial monitoring post-HSCT for better outcomes.

Iterum Therapeutics concluded a late-cycle FDA meeting for sulopenem etzadroxil/probenecid, aimed at treating uncomplicated UTIs in patients with quinolone non-susceptible pathogens. The FDA deemed an Advisory Committee meeting unnecessary, targeting a July 25 action date. ITRM shares rose 22.8% to $1.51 premarket.

The FDA approved Johnson & Johnson's Rybrevant, a bispecific antibody for treating a specific mutation in non-small cell lung cancer. Bispecific antibodies, targeting two proteins simultaneously, are emerging as a promising treatment for various cancers, including lymphoma, leukemia, and multiple myeloma, with several drugs in advanced clinical trials.

Fast Track designation aids Alume Biosciences in expediting ALM-488's development, a fluorescent peptide-dye for intraoperative nerve visualization, highlighting its potential in surgeries where nerves are at risk. ALM-488 binds to nerves, illuminating them in real-time, with applications in various surgical fields. Alume, founded in 2017, focuses on nerve-specific targeting molecules.

AtriCure's EPi-Sense system, FDA-approved for long-standing persistent Afib, showed superior efficacy in the Converge trial. Hybrid AF therapy, a minimally invasive procedure, demonstrated significant improvements in Afib burden and freedom from arrhythmias, offering durable efficacy and enhancing electrophysiology lab efficiency.

A study found the Lyme vaccine (LYMErix) ≥3 doses 71% effective in real-world settings, the first to show such effectiveness. Despite its efficacy, the vaccine was withdrawn in 2002, highlighting a lost tool against Lyme disease's increasing public health burden.

The FDA revoked the EUA for bamlanivimab alone due to increased SARS-CoV-2 variants resistant to it, making its risks outweigh benefits. Alternative monoclonal antibody therapies remain authorized. The FDA emphasizes the importance of using therapies effective against all variants and commits to monitoring and communicating on variant impacts.

CAR T-cell therapy, specifically idecabtagene vicleucel (Abecma), was FDA-approved for multiple myeloma patients unresponsive to prior treatments. In a study, 72% of participants saw tumor reduction, with remissions lasting a median of 11 months. Despite its effectiveness, the therapy can cause severe side effects and is costly, priced at $419,500 per infusion.