Clinical Trial News

The SOPHIA phase 3 trial showed margetuximab plus chemotherapy improved progression-free survival (PFS) but not overall survival (OS) compared to trastuzumab plus chemotherapy in HER2-positive advanced breast cancer patients. Margetuximab's Fc engineering enhances antibody-dependent cell-mediated cytotoxicity (ADCC), offering a potential treatment alternative despite no OS benefit.

The FDA's draft guidance on decentralized clinical trials (DCTs) has been released, aiming to reduce regulatory uncertainties and support decentralization in trials. Despite its embrace, challenges remain, including PI oversight, HCP use, data collection scalability, and terminology clarity. The industry seeks more clarity, with the FDA expected to finalize the guidance after considering feedback.

A prospective, multicenter study evaluates the diagnostic performance of the one-step nucleic acid amplification (OSNA) method for detecting sentinel lymph node (SLN) metastases in early-stage endometrial cancer, comparing it with standard ultrastaging. The study found that OSNA has high specificity and accuracy, offering the advantage of analyzing the entire lymph node to eliminate sampling bias.

The FDA approved Elevidys, the first gene therapy for Duchenne muscular dystrophy (DMD) in children aged 4-5 with a specific DMD gene mutation. It produces a micro-dystrophin protein to address the genetic cause, differing from symptom-focused treatments. Approval was based on increased protein expression, predicting clinical benefit, with ongoing trials to confirm efficacy.

Sarepta Therapeutics received FDA accelerated approval for Elevidys, a gene therapy for DMD in children aged 4-5, pending confirmatory trial results. Bristol Myers Squibb's Camzyos was approved in the EU for treating obstructive HCM. FibroGen's pamrevlumab failed in a phase III IPF study, leading to discontinuation of related trials. Intercept Pharmaceuticals faced FDA rejection for its NASH treatment, prompting restructuring. MoonLake Immunotherapeutics reported positive phase II results for sonelokimab in treating HS.

Nexcella, Inc., a subsidiary of Immix Biopharma, completed a Pre-IND meeting with the FDA for NXC-201, targeting AL amyloidosis and multiple myeloma. The meeting discussed US manufacturing and clinical trials, aiming for regulatory clarity to proceed with an IND application. NXC-201, a BCMA-targeted CAR-T cell therapy, has shown promising response rates in ongoing trials.

Janssen submitted a Marketing Authorisation Application to the EMA for a single tablet combination therapy of macitentan and tadalafil for PAH treatment, based on Phase 3 A DUE study results showing significant pulmonary haemodynamic improvement. This therapy aims to simplify treatment regimens, potentially improving adherence and outcomes for PAH patients.

The article provides an in-depth look at the current landscape of anti-obesity medications (AOMs) approved for long-term weight management, highlighting the importance of pharmacotherapy in treating obesity, especially for individuals with a BMI of 25 kg/m2 or higher who have not achieved weight loss through non-pharmacological treatments. It covers the updated obesity pharmacotherapy guidelines recommended by the Korean Society for the Study of Obesity (KSSO), the currently available AOMs in Korea, and upcoming and potential future AOMs undergoing clinical trials.

GLP-1 agonists, initially developed for type 2 diabetes treatment, have shown significant efficacy in promoting weight loss in both preclinical and clinical studies. This review highlights the role of GLP-1 agonists in weight management, their pleiotropic effects, and their impact on hyperglycemia, insulin sensitivity, blood pressure, and cardio-metabolic and renal protection.

Sequana Medical has revealed additional data from its North American pivotal study, POSEIDON, showcasing the alfapump's effectiveness in controlling ascites, improving patients' quality of life, and demonstrating a favorable survival rate. The study, focusing on patients with recurrent or refractory ascites due to liver cirrhosis, also highlighted the device's safety profile and its potential to significantly reduce the need for therapeutic paracentesis.