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Turn Therapeutics Appoints Former FDA Commissioner Dr. Stephen M. Hahn to Lead Clinical and Regulatory Strategy

Turn Therapeutics Appoints Former FDA Commissioner Dr. Stephen M. Hahn to Lead Clinical and Regulatory Strategy

Turn Therapeutics Appoints Former FDA Commissioner Dr. Stephen M. Hahn to Lead Clinical and Regulatory Strategy

Amylyx Pharmaceuticals Publishes Phase 2 Trial Data for AMX0035 in Wolfram Syndrome

Amylyx Pharmaceuticals Publishes Phase 2 Trial Data for AMX0035 in Wolfram Syndrome

Amylyx Pharmaceuticals Publishes Phase 2 Trial Data for AMX0035 in Wolfram Syndrome

iECURE Reports Positive Results in OTC-HOPE Trial for Neonatal-Onset OTC Deficiency

iECURE Reports Positive Results in OTC-HOPE Trial for Neonatal-Onset OTC Deficiency

iECURE, a clinical-stage genome editing company, has announced promising results from the ongoing OTC-HOPE clinical trial, which is testing the ECUR-506 therapy in infants with neonatal-onset ornithine transcarbamylase (OTC) deficiency. The trial's preliminary analysis of the low-dose cohort showed a durable clinical response in the first treated infant, with no hyperammonemic events (HAEs) reported 18 months post-treatment. The study also observed statistically significant reductions in the annualized rates of HAEs and hyperammonemic crises (HACs) among participants. These findings were presented at the Society for Inherited Metabolic Disorders (SIMD) 2026 Annual Meeting. OTC deficiency is a rare genetic disorder that leads to toxic ammonia accumulation in the bloodstream, causing severe neurological complications. Current treatments involve strict dietary restrictions and ammonia scavenger medications, which do not fully prevent metabolic crises.

MSD's Sac-TMT Shows Promise in Phase 3 Endometrial Cancer Trial

MSD's Sac-TMT Shows Promise in Phase 3 Endometrial Cancer Trial

MSD's Sac-TMT Shows Promise in Phase 3 Endometrial Cancer Trial

Turn Therapeutics Appoints Former FDA Commissioner Dr. Stephen M. Hahn as Executive Clinical Lead

Turn Therapeutics Appoints Former FDA Commissioner Dr. Stephen M. Hahn as Executive Clinical Lead

Turn Therapeutics Appoints Former FDA Commissioner Dr. Stephen M. Hahn as Executive Clinical Lead

FDA Alignment and AI Integration Crucial for Rare Disease Treatment Advancements

FDA Alignment and AI Integration Crucial for Rare Disease Treatment Advancements

FDA Alignment and AI Integration Crucial for Rare Disease Treatment Advancements

Lantern Pharma's LP-300 Trial for Never-Smokers with NSCLC Advances with FDA Support

Lantern Pharma's LP-300 Trial for Never-Smokers with NSCLC Advances with FDA Support

Lantern Pharma's LP-300 Trial for Never-Smokers with NSCLC Advances with FDA Support

ImmunityBio Secures Exclusive U.S. Rights to Tokyo Strain of BCG to Address Bladder Cancer Treatment Shortage

ImmunityBio Secures Exclusive U.S. Rights to Tokyo Strain of BCG to Address Bladder Cancer Treatment Shortage

ImmunityBio Secures Exclusive U.S. Rights to Tokyo Strain of BCG to Address Bladder Cancer Treatment Shortage

FDA Alignment and AI Integration Critical for Rare Disease Drug Development

FDA Alignment and AI Integration Critical for Rare Disease Drug Development

FDA Alignment and AI Integration Critical for Rare Disease Drug Development

Prokarium's ZH9 Shows Promising Results in Bladder Cancer Trial

Prokarium's ZH9 Shows Promising Results in Bladder Cancer Trial

Prokarium's ZH9 Shows Promising Results in Bladder Cancer Trial

Atisama Therapeutics Progresses RB042 in Phase 1 Trial and Appoints New Board Chair

Atisama Therapeutics Progresses RB042 in Phase 1 Trial and Appoints New Board Chair

Atisama Therapeutics Progresses RB042 in Phase 1 Trial and Appoints New Board Chair

Amylyx Pharmaceuticals Publishes Phase 2 Trial Data for AMX0035 in Wolfram Syndrome

Amylyx Pharmaceuticals Publishes Phase 2 Trial Data for AMX0035 in Wolfram Syndrome

Amylyx Pharmaceuticals has announced the publication of results from its Phase 2 open-label HELIOS trial in The Journal of Clinical Investigation. The trial evaluated AMX0035, a combination of sodium phenylbutyrate and taurursodiol, in adults with Wolfram syndrome. The study showed improvements in pancreatic beta cell function and glycemic control over 48 weeks, with stabilization in visual acuity. The trial's findings suggest AMX0035's potential to alter the progression of Wolfram syndrome, a rare genetic disorder characterized by diabetes and neurodegeneration. The drug was generally well-tolerated, with no serious adverse events reported.