Amylyx Pharmaceuticals

Amylyx Pharmaceuticals announces design of pivotal Phase 3 LUCIDITY trial for avexitide, an investigational GLP-1 receptor antagonist for post-bariatric hypoglycemia, with first participant dosing expected in Q1 2025 and topline results anticipated in 2026. Avexitide has FDA Breakthrough Therapy and Orphan Drug designations.

Amylyx Pharmaceuticals announces Phase 3 LUCIDITY trial design for avexitide, a GLP-1 receptor antagonist, to treat post-bariatric hypoglycemia (PBH). The trial aims to reduce hypoglycemia events and is expected to start dosing in Q1 2025, with topline data anticipated in 2026. Avexitide has shown consistent, dose-dependent effects in previous PBH trials.

The neurodegenerative disorder therapeutics market is projected to grow from $18.53 billion in 2023 to $28.33 billion in 2028 at a CAGR of 9.0%, driven by factors like aging population, rising prevalence of neurodegenerative disorders, and strategic collaborations among pharmaceutical companies.

Amylyx reports Q3 2024 financials: $234.4M cash, positive Phase 2 HELIOS trial data for AMX0035 in Wolfram syndrome, plans Phase 3 avexitide program for PBH in Q1 2025, and expects interim data from ORION trial in mid-2025.

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EMA won't renew Translarna's permit; Novartis pays $70M for a cancer drug; Amylyx's AMX0035 shows promise in rare disease; FDA delays Ocaliva approval; Australia rejects Leqembi; Amicus settles with Teva over Galafold.

Amylyx Pharmaceuticals reports positive Phase II HELIOS trial results for AMX0035, showing sustained improvements in pancreatic function and other measures in adults with Wolfram syndrome. The trial's primary endpoint, C-peptide response, demonstrated positive change from baseline at week 24, with secondary endpoints also showing improvements. Amylyx plans to engage with the FDA for a Phase III program.

Amylyx Pharmaceuticals reports positive Phase 2 HELIOS trial results for AMX0035 in Wolfram syndrome, showing sustained improvements in pancreatic function, glycemic control, and vision.