Clinical Trial News

Pre-ordering for epinephrine nasal spray (neffy) has launched for treating allergic reactions, including anaphylaxis. The FDA approved trofinetide (Daybue) for Rett syndrome, marking a milestone in treatment. A study in JAMA Pediatrics found not all screen time before bed negatively impacts sleep in children. Another study in The American Journal of Clinical Nutrition linked fish intake during pregnancy to reduced autism risk in children.

Classic psychedelics like LSD and psilocybin are well-tolerated in clinical settings, with serious adverse events being rare, according to a study in JAMA Psychiatry. However, incomplete adverse event reporting highlights the need for improved safety monitoring. The study found no serious adverse events among healthy participants, but about 4% of those with neuropsychiatric disorders experienced serious events. The research emphasizes the need for rigorous pharmacovigilance practices to accurately quantify risks and benefits.

Ram Selvaraju, H.C. Wainwright analyst, maintains Buy rating on Bioxcel Therapeutics (BTAI) with $7.00 price target, citing SERENITY At-Home Phase 3 trial initiation for BXCL501, IGALMI's post-marketing study success, and FDA's positive review of SERENITY protocol. Canaccord Genuity also reiterates Buy rating with $7.00 target.

Qualigen Therapeutics, Inc. is offering 14,724,058 shares of common stock and 11,972,754 pre-funded warrants to purchase up to 11,972,754 shares of common stock at $0.13 per share. The offering is on a 'reasonable best efforts' basis, with no minimum required. The net proceeds will be used for operations, clinical trials, and other corporate purposes. The common stock is listed on The Nasdaq Capital Market under the symbol 'QLGN'.

The Reagan Udall Foundation hosted a meeting on advancing PTSD treatments, highlighting the unmet need and discussing current clinical trials, including those for MDMA-assisted therapy. Participants emphasized the importance of representative clinical trials and the impact of trauma on lives, with some criticizing the FDA's handling of MDMA trials. Despite challenges, there is optimism for future advancements in PTSD treatment.

A study comparing traditional psychiatric care and a collaborative care model for depression and anxiety found both models significantly improved symptoms, with stronger results in collaborative care. Published in the Journal of Clinical Psychiatry, the study tracked 1,607 depression and 570 anxiety patients in collaborative care, and 8,773 depression and 2,365 anxiety patients in specialty psychiatry over 6 months. Collaborative care patients saw greater symptom reduction, with 9-point and 5.4-point decreases for depression and anxiety, respectively, compared to 5-point and 2.8-point decreases in specialty care. The collaborative care model, which includes regular symptom monitoring and personalized treatment, showed higher remission rates and addressed the clinician shortage.

NeurologyLive's Friday 5 highlights: 1) Sharon Cohen on mivelsiran for early-stage Alzheimer disease; 2) Aliza Alter on BHV-7000 for focal epilepsy; 3) Jim Cassidy on mirdametinib for neurofibromatosis type 1; 4) Crystal Proud on SMA research and treatments; 5) Michael Levy on ravulizumab for NMOSD.

CGTLive®’s Weekly Rewind highlights 5 gene and cell therapy advances: therapy for HLA-DRB1*15:01 positive progressive MS patients, newborn screening enhancements, GT201 IND clearance in China, 85% reduction in HS D2S6 levels in CSF, and data from the DEVOTE trial.

Cartesian Therapeutics' Descartes-08, an autologous BCMA-directed mRNA CAR-T therapy, showed deep remission in MG patients up to 12 months post-treatment at the ASGCT 27th Annual Meeting. The therapy, administered without lymphodepletion chemotherapy in an outpatient setting, demonstrated a strong safety profile with no cytokine release syndrome or neurotoxicity. Challenges included integrating different medical teams and COVID-19 affecting enrollment. Future plans include trials for systemic lupus and other autoimmune diseases, with an ongoing double-blind placebo-controlled trial in MG.

One year of adjuvant trastuzumab improved disease-free survival (DFS) over 9 weeks in ERBB2-positive breast cancer patients, with no significant difference in overall survival (OS). The 9-week regimen may be an option for patients unable to tolerate or afford 1-year treatment. DFS was shorter in the 9-week group (HR 1.36; 90% CI, 1.14-1.62; P = .004), while OS rates were similar at 5 and 10 years. Factors like docetaxel dose, positive axillary nodes, and disease stage affected DFS, but not OS. Four cardiac deaths occurred, three in the 9-week group. The study included 2176 women, randomized to 9 weeks or 1 year of trastuzumab, with chemotherapy cycles. The 9-week regimen offers benefits like reduced cardiac monitoring, fewer visits, and lower cost, but has limitations such as short follow-up and limited generalizability.