Arrowhead Pharmaceuticals

Sanofi has acquired rights to develop and commercialize plozasiran, a first-in-class RNAi therapeutic for familial chylomicronemia syndrome and severe hypertriglyceridemia, in Greater China for $130 million upfront plus up to $265 million in milestones.

Arrowhead Pharmaceuticals earned a $100 million milestone payment from Sarepta Therapeutics after reaching enrollment targets for ARO-DM1, an RNAi therapeutic for type 1 myotonic dystrophy.

Licensing agreements for CNS-targeted oligonucleotide therapeutics increased 339% from 2023 to 2024, reaching $2.81 billion in total deal value according to GlobalData's analysis.

Arrowhead Pharmaceuticals has dosed the first subjects in a Phase 1/2a clinical trial of ARO-ALK7, marking the first investigational RNAi therapeutic to target a gene expressed in adipose tissue for obesity treatment.

Plozasiran, an investigational hepatocyte-targeted APOC3 small interfering RNA developed by Arrowhead Pharmaceuticals, demonstrated significant reductions in triglyceride levels and improved lipid profiles in patients with mixed hyperlipidemia.

A comprehensive pipeline analysis reveals over five companies developing RAGE inhibitors across multiple clinical stages, with promising applications in chronic inflammatory diseases and neurodegenerative disorders.

Arrowhead Pharmaceuticals' plozasiran, a novel treatment for familial chylomicronemia syndrome (FCS), has been filed for review by the European Medicines Agency and is being fast-tracked for potential EU marketing approval.

Epicrispr Biotechnologies has raised $68 million in Series B funding to develop EPI-321, a first-in-class epigenetic therapy targeting facioscapulohumeral muscular dystrophy (FSHD).

Arrowhead Pharmaceuticals' ARO-C3 demonstrated significant efficacy in IgA nephropathy patients, achieving mean C3 reductions of 89% and sustained reductions exceeding 87% through week 24 of the Phase 1/2 trial.

• Pfizer is prioritizing its oral obesity drug, danuglipron, with late-stage studies planned for the second half of 2025, aiming for a novel mechanism of action. • Regeneron anticipates Phase III data readouts for itepekimab in COPD and fianlimab in melanoma, potentially expanding its portfolio alongside Dupixent. • BioNTech is advancing its mRNA-based cancer immunotherapies, including BNT327/PM8002 for solid tumors and BNT323/DB-1303, an ADC for endometrial cancer. • Sarepta Therapeutics reported strong initial sales for Elevidys in Duchenne muscular dystrophy and is pursuing a Phase III trial for limb-girdle muscular dystrophy.