Amylyx Pharmaceuticals

Amylyx Pharmaceuticals has discontinued the ORION program of AMX0035 for progressive supranuclear palsy after the drug failed to show differences compared to placebo on primary or secondary outcomes at Week 24.

The HDAC inhibitors market across seven major markets is expected to surge significantly by 2034, driven by innovation in cancer therapies and expanding applications beyond traditional hematologic malignancies.

The progressive supranuclear palsy (PSP) market is experiencing significant growth driven by emerging therapies including AZP2006 and AMX0035, which are advancing through clinical trials with promising results.

DelveInsight's analysis reveals 4+ key companies are developing 6+ therapies for congenital hyperinsulinism, with emerging treatments including CRN-04777, HM 15136, RZ358, and dasiglucagon expected to significantly impact the market.

Biogen's Qalsody (tofersen) receives FDA accelerated approval as the first therapy for SOD1-mutated ALS, marking a significant breakthrough in genetic-specific ALS treatment.

The FDA has lifted the clinical hold on Amylyx Pharmaceuticals' Phase 1 trial of AMX0114, an antisense oligonucleotide for ALS treatment.

Amylyx Pharmaceuticals has appointed Dan Monahan as Chief Commercial Officer to lead the commercialization of their product portfolio.

• GLP-1 receptor agonists like semaglutide and tirzepatide showed potential beyond diabetes and weight loss, including cardiovascular and mental health benefits. • CRISPR gene editing entered the market with Casgevy for sickle cell disease and β-thalassemia, though uptake has been slow due to complex treatment requirements. • Protein-folding algorithms developed by Nobel laureates revolutionized biochemical research and found applications in pharmaceutical drug discovery. • Antibody-drug conjugates (ADCs) saw major investment, with firms focusing on novel linker chemistries to enhance their efficacy and target specificity.

Amylyx Pharmaceuticals initiates Phase 3 LUCIDITY trial for avexitide, a GLP-1 receptor antagonist, targeting post-bariatric hypoglycemia (PBH).

Amylyx reported positive topline data from its Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome, showing improvement or stabilization across key disease measures.