Clinical Trial News

Decentralised clinical trials, leveraging tech for remote participation, ease patient burden, enhance engagement, and support sustainable research. Chiesi Farmaceutici's study on asthma patients found 80-90% positive response to home-based e-Consent, spirometry, e-Diary, and telemedicine, though challenges remain, especially for less digitally literate elderly.

FDA rejected Lykos Therapeutics' NDA for MDMA treatment of PTSD, citing insufficient evidence of efficacy and safety. The rejection followed an advisory committee meeting where concerns were raised about the study's functional unblinding and the combined drug-therapy approach. Lykos plans to meet with the FDA to discuss resubmission, while the broader impact on psychedelic research remains debated.

Multiple studies report on survival outcomes and therapies for multiple myeloma, including retrospective analyses, phase trials, and real-world data, highlighting advancements in treatment strategies and overall survival improvements.

Praxis Precision Medicines Inc. (NASDAQ:PRAX) maintains positive outlook from H.C. Wainwright, with Buy rating and $120.00 price target. Recent successes include positive Phase 2 data for relutrigine and favorable peer review results for PRAX-628. Praxis is advancing its epilepsy portfolio with key milestones expected in the next 12 months, including initial pivotal readout for PRAX-628 in focal onset seizures. Analysts highlight potential of Praxis Precision's relutrigine, with interim Essential3 Part1 analysis expected in Q4 2024.

A study by Memorial Sloan Kettering Cancer Center found CAR-T therapy does not increase the risk of secondary cancers, contrary to FDA warnings. The research, published in 'Clinical Cancer Research,' analyzed data from 18 clinical trials and seven real-world studies involving over 5,500 patients, showing similar rates of secondary cancers between CAR-T and standard treatments. The study suggests warning labels may need revision.

Experimental weight-loss pill amycretin, developed by Novo Nordisk, helps users lose up to 13% of body weight in 3 months by mimicking GLP-1 and amylin hormones. Higher doses led to more weight loss but also increased side effects like nausea and vomiting. Further clinical trials needed for full assessment.

An experimental blood test analyzing 32 proteins could predict severe lung issues like COPD, identifying higher risks for COPD, respiratory death, hospitalization, and treatment-requiring symptoms. Developed from a 30-year study, the test needs clinical trials for FDA approval but shows promise in early risk detection.

enGene Holdings Inc. announced Raj Pruthi MD MHA FACS as SVP, Urologic Oncology and Clinical Development, supporting EG-70's clinical development for BCG-unresponsive NMIBC.

Bristol Myers Squibb's schizophrenia drug KarXT, with a PDUFA date of 26 September 2024, shows strong clinical data and a differentiated mechanism. Concerns over its twice-daily dosing led Terran Biosciences to develop a once-daily oral and multi-month injectable prodrug version, TerXT and TerXT LAI, aiming to improve adherence. KarXT's potential to avoid traditional antipsychotic side effects like weight gain and diabetes risks is highlighted, with GlobalData forecasting $2.99bn in sales by 2030.

Saul Faust presents 'The HARMONIE Trial: Reimagining How to Design and Deliver Contract Commercial Clinical Trials' on Sept 13, 2024. The HARMONIE trial studied nirsevimab's effect on RSV-associated lower respiratory tract infection in infants in France, Germany, and the UK.