Commons, Marty

DelveInsight's Alpha Thalassemia Market Insights report forecasts significant market growth (2020-2034) driven by rising prevalence, genetic research advancements, and emerging therapies from companies like Agios Pharmaceuticals, Forma Therapeutics, and Novartis Pharmaceuticals.

The phase II GEOMETRY mono-1 trial results, published in The Lancet Oncology, show capmatinib's efficacy in NSCLC patients with MET exon 14 mutations. Among 60 treatment-naive patients, 68% had objective responses, with a median duration of response of 16.6 months. Among 100 previously treated patients, 44% responded, with a median duration of response of 9.7 months. Adverse events were common but manageable.

Asciminib, a BCR::ABL1 inhibitor targeting the ABL myristoyl pocket, shows efficacy against CML-CP, including T315I mutation, with 40 mg b.i.d. and 200 mg b.i.d. dosing regimens. An 80 mg q.d. regimen is proposed for improved patient compliance, supported by model-informed drug development, demonstrating similar efficacy and safety profiles to 40 mg b.i.d.