What Comes Next For Arrowhead Pharmaceuticals After The 78% Run

The MHRA approved plozasiran (Redemplo) to treat familial chylomicronaemia syndrome (FCS), a rare genetic condition causing high blood fat levels. The drug, marketed by Arrowhead Pharmaceuticals, reduced triglycerides by 80% in clinical trials. Common side effects include hyperglycaemia and nausea. The treatment is administered as a 25 mg injection every three months.

The MHRA authorized Arrowhead Pharmaceuticals' plozasiran (Redemplo) to reduce triglycerides in adults with familial chylomicronaemia syndrome (FCS). A phase 3 trial showed an 80% median reduction in triglycerides with plozasiran vs. 17% with placebo. The drug is administered as a 25-mg subcutaneous injection every 3 months. Common side effects include hyperglycemia, headache, and nausea.

The FDA launched a Rare Disease Framework Initiative to boost drug development for rare diseases. Arrowhead Pharmaceuticals reported a 73% decrease in tau protein in a Phase 1/2a study of ARO-MAPT. First Tracks Biotherapeutics announced positive Phase 1b results for ANB033 in celiac disease. BioNTech's BNT113 missed its first primary endpoint in a head and neck cancer trial. GSK plans to cut over 1,000 jobs at a Belgium site.

Arrowhead Pharmaceuticals' ARO-MAPT showed significant tau protein reduction in a Phase 1/2a study, with up to 85% reduction at high doses. Analysts view the data favorably, suggesting a potential competitive edge over Biogen's diranersen. The trial is ongoing, with safety data showing the drug was well tolerated. Arrowhead plans further studies and potential expansion into other diseases.

Cantor Fitzgerald reiterated an Overweight rating on Arrowhead Pharmaceuticals (ARWR) after positive Phase 1 data for ARO-MAPT showed a 70-75% reduction in cerebrospinal fluid tau. The company's market cap is $8.25B, with a 52.8% return over the past year. ARWR reported Q3 2026 revenue of $75.2M, beating estimates, but with a net loss of $1.36 per share. Analysts have price targets up to $126, with a consensus Strong Buy rating.

Chardan Capital reaffirmed its Buy rating on Arrowhead Pharma with a $115 price target, suggesting a 97% upside from the last close. The target is based on strong clinical data and growth potential from its RNAi therapies.