Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals reported FY2024 results, submitting its first NDA for plozasiran, with a 2025 launch anticipated. Key financial moves include a $825M licensing deal with Sarepta and $500M from Sixth Street, extending cash runway to 2028. R&D efforts include Phase 3 studies for plozasiran and new drug candidates for severe hypertriglyceridemia and obesity. The company aims to drive long-term growth through strategic collaborations and advanced R&D initiatives.

Arrowhead Pharmaceuticals announces a global licensing and collaboration agreement with Sarepta Therapeutics, including an immediate $825 million payment ($500M cash + $325M equity investment at 35% premium), plus $250 million over five years. Arrowhead is eligible for $300 million in near-term clinical trial milestone payments and up to $10 billion in future milestones plus royalties. The agreement covers multiple clinical and preclinical programs in rare genetic diseases affecting muscle, CNS, and lungs, with Sarepta selecting up to six new targets using Arrowhead's TRiM™ platform. The transaction extends Arrowhead's cash runway into 2028 and is expected to close in early 2025.

Arrowhead Pharmaceuticals submitted an FDA application for plozasiran, targeting familial chylomicronemia syndrome (FCS), with no current FDA-approved treatments. The drug showed sustained reductions in apolipoprotein C-III and triglycerides, significantly lowering pancreatitis risk. The FDA granted breakthrough therapy designation in 2024.

Arrowhead Pharmaceuticals presents new data on plozasiran from PALISADE Phase 3 and MUIR/SHASTA-2 open-label extension studies at AHA24, showing significant reductions in triglycerides and favorable lipid profile changes, supporting its development for FCS, severe hypertriglyceridemia, and mixed hyperlipidemia.

Arrowhead Pharmaceuticals submitted a New Drug Application (NDA) to the FDA for plozasiran, an investigational RNAi therapeutic for familial chylomicronemia syndrome (FCS), a severe genetic disease with no FDA-approved treatments. The NDA is supported by the SUMMIT program and the Phase 3 PALISADE study, which demonstrated significant reductions in triglycerides and acute pancreatitis risk. Arrowhead plans to seek approval from additional regulatory authorities in 2025.

Plozasiran demonstrated deep and sustained triglyceride reductions, impacting lipoproteins linked to atherosclerotic cardiovascular disease, with similar responses in genetically confirmed and clinically diagnosed FCS patients. Mean reductions reached -73% in MUIR and -86% in SHASTA-2, with favorable reductions in remnant cholesterol and non-HDL-cholesterol over 15 months.

Arrowhead Pharmaceuticals submitted a New Drug Application to the FDA for plozasiran to treat familial chylomicronemia syndrome, a disease with no current FDA-approved treatments. The submission is supported by positive results from the Phase 3 PALISADE study, which demonstrated significant reductions in triglycerides and acute pancreatitis risk.

Arrowhead Pharmaceuticals submitted an NDA to the FDA for plozasiran, a treatment for familial chylomicronemia syndrome (FCS), based on the Phase 3 PALISADE study. The drug, developed using the TRiM™ platform, aims to lower apolipoprotein C-III and has received Breakthrough Therapy, Orphan Drug, and Fast Track designations. Arrowhead plans to seek approval in other markets by 2025 and has established an expanded access program for FCS patients.

Arrowhead Pharmaceuticals submits NDA to FDA for plozasiran, targeting FCS with no current FDA-approved treatments, based on Phase 3 PALISADE study results showing 80% median triglyceride reduction and 83% acute pancreatitis risk reduction.

IgA nephropathy affects 25-50 per million annually, with 20–40% progressing to end-stage kidney disease. Diagnosed cases in the US are expected to reach 135,000 by 2030. Treatments include ACE inhibitors, ARBs, corticosteroids, and immunosuppressants. FDA-approved treatments are TARPEYO, FILSPARI, and FABHALTA. The IgA nephropathy market is projected to reach ~USD 4.1 billion by 2034, driven by novel therapies like Novartis’ Atrasentan, Otsuka/Visterra’s Sibeprenlimab, Vertex/Alpine’s Povetacicept, and others.