Arrowhead Pharmaceuticals (ARWR) Study Result summary

FDA approval and clinical highlights

• Redemplo (plozasiran) received FDA approval as the first RNAi/siRNA-based medicine for adults with FCS, showing a median triglyceride reduction of 80% and reduced acute pancreatitis incidence in both genetically confirmed and clinically defined FCS patients.

• Approval was based on the pivotal, randomized, double-blind, placebo-controlled Phase III PALISADE trial, which met all primary and key secondary endpoints, with over 50% of patients reaching triglyceride levels below 500 mg/dL and around 75% below 880 mg/dL.

• Redemplo is administered as a subcutaneous injection every three months, is self-administered, well-tolerated, and has no contraindications, warnings, or precautions in its label.

• The most common adverse reactions were hyperglycemia, headache, nausea, and injection site reactions, with discontinuations due to adverse events in 6% of patients and no deaths reported.

• Both genetic and clinical FCS patients experienced similar efficacy, and the FDA label includes both populations, expanding the eligible patient base.

Commercial strategy and pricing

• Redemplo launches with a single annual WAC price of $60,000, designed to reflect its value for both current FCS and future high-risk SHTG populations.

• The pricing strategy was developed in consultation with payers and consultants, focusing on high-risk SHTG (triglycerides >880 mg/dL or prior pancreatitis), and is considered compelling given the high cost and severity of pancreatitis.

• The commercial launch targets approximately 5,000 healthcare professionals directly and a broader audience through non-personal promotion, supported by a comprehensive patient support program, Rely On REDEMPLO, which includes financial assistance.

• The company is preparing for global expansion, including a European launch, and is open to partnerships but not dependent on them.

• Manufacturing scale-up is on track to meet anticipated demand for both FCS and future SHTG indications.

Pipeline and future development

• Ongoing phase III studies (SHASTA-3, SHASTA-4, MUIR-3) in SHTG are expected to read out in Q3 2026, with an SNDA filing planned for Q4 2026 to expand Redemplo's indication.

• Additional pipeline assets include Zodasiran for HoFH (phase III completion expected in 2027) and Arrow Dimer PA for mixed hyperlipidemia, with first clinical data anticipated in 2026.

• The company aims to regularly advance two to four new drug candidates into the clinic annually, leveraging its TRIM RNAi platform to address a broad spectrum of lipid and cardiometabolic disorders.

• Early clinical data from obesity and CNS programs are also expected in 2026, supporting a diversified growth strategy.

• The company expects to have 20 drug candidates in clinical studies by year-end, with a balanced mix of wholly owned and partnered programs.