MONTEFIORE MEDICAL CENTER

Approval of ursodeoxycholic acid (UDCA) in 1997 significantly reduced liver transplant and death risks for primary biliary cholangitis (PBC), but up to 40% of patients do not sufficiently respond. Off-label use of fibrates and the 2016 approval of obeticholic acid (Ocaliva) as a second-line treatment provided additional options, though Ocaliva has downsides like increased pruritus. New PPAR agonists, seladelpar and elafibranor, have been approved for PBC, showing potential benefits but with unknown long-term risks.

The ENCORE trial showed AXS-12, an investigational narcolepsy treatment, significantly reduced cataplexy attacks and improved daytime sleepiness, cognition, and productivity. Axsome plans to file an NDA for AXS-12 and request a pre-NDA meeting with the FDA.

Axsome Therapeutics' reboxetine (AXS-12) met primary endpoint in phase 3 ENCORE trial, significantly reducing cataplexy attacks in narcolepsy patients compared to placebo. Long-term dosing showed sustained improvements in cataplexy, excessive daytime sleepiness, cognition, and overall function, with a favorable safety profile.

Axsome Therapeutics' AXS-12 met primary endpoint in Phase III ENCORE trial, reducing cataplexy attacks by 72% at one month and 82% at six months. The company plans to submit an NDA to the FDA.

AXS-12 significantly reduced cataplexy attacks, improved cognition, and overall narcolepsy symptoms compared to placebo in the ENCORE Phase 3 trial. Patients experienced substantial reductions in cataplexy and excessive daytime sleepiness, with sustained improvements in cognition and overall function. AXS-12 was well-tolerated with a safety profile consistent with previous trials.

Axsome Therapeutics announces AXS-12 (reboxetine) achieved primary endpoint in ENCORE Phase 3 trial for narcolepsy, showing significant reduction in cataplexy attacks and improved safety profile.

Nerivio REN wearable, a non-drug therapy for acute migraine treatment, has received FDA clearance for use in children aged 8 years and older, becoming the first such therapy for this age group. The approval is based on a study showing safety and efficacy in children aged 6 to 11 years, with 72.2% reporting pain relief and 83.3% functional disability relief.

Medtronic receives FDA IDE approval for Prevail™ coronary paclitaxel drug-coated balloon (DCB) pivotal trial for in-stent restenosis (ISR) and de novo small vessel disease. The Prevail Global Study will enroll up to 1,205 patients across 65 global centers to assess safety and efficacy, including the first head-to-head randomized evaluation of two DCBs in the U.S. Data will support regulatory submissions in the U.S. and Japan.

HIVMA recognizes Daniel R. Kuritzkes, Oni Blackstock, and Onyema Ogbuagu at IDWeek 2024 for their contributions to HIV medicine, including research on drug-resistant HIV, health equity, and innovative clinical trials.

Kintara Therapeutics provides corporate and REM-001 clinical study updates, including a merger agreement with TuHURA Biosciences and a special stockholder meeting on September 20, 2024. The REM-001 study for cutaneous metastatic breast cancer has enrolled four of ten needed patients, with no safety issues identified.