Arrowhead Pharmaceuticals Spotlights Dual-Target RNAi Data, Plozasiran Launch Progress

Arrowhead reported early promise for ARO-DIMER-PA , with a single RNAi molecule reducing two lipid-related genes and producing an approximately 50% reduction in ApoB. The company plans to add multidose testing and potentially advance to Phase III, with additional data expected later. Plozasiran’s FCS launch is progressing faster than anticipated , supported by stronger-than-expected patient identification and broad payer coverage. Arrowhead is pursuing approval in severe hypertriglyceridemia after studies showed approximately 80% triglyceride reductions and fewer acute pancreatitis events. The pipeline remains focused on neurological and metabolic diseases, including ARO-MAPT for tau-related disorders and ARO-INHBE/ARO-ALK7 for MASH, obesity and related conditions. Initial human data for ARO-MAPT are expected by late September or early October, with further metabolic-program updates anticipated toward year-end.

Arrowhead Pharmaceuticals NASDAQ: ARWR executives outlined early clinical data for its RNA interference dimer program, progress in the launch of plozasiran and plans for central nervous system and metabolic disease candidates during the Morgan Stanley Global Healthcare Conference. Get ARWR alerts: Sign Up Early Dimer Data Points to Dual-Target Approach Chief Executive Officer Chris Anzalone said the company’s ARO-DIMER-PA candidate represented the first demonstration of knockdown of two genes with a single RNAi molecule. The program is designed for patients with mixed hyperlipidemia, a population Arrowhead estimated at about 20 million people in the U.S. with elevated LDL cholesterol and triglycerides. Anzalone said the goal is to reduce LDL at levels comparable to PCSK9 inhibitors while also lowering triglycerides at levels seen with Arrowhead’s plozasiran program. Chief Medical Officer James Hamilton highlighted an approximately 50% reduction in ApoB in the early data, which he said was modestly greater than results reported for some PCSK9 inhibitors in similar populations. The disclosed results were from single-dose cohorts, Hamilton said. The study is fully enrolled, and the company expects to present multidose data at a future medical conference, potentially in the fourth quarter. Arrowhead is evaluating quarterly dosing and will assess whether dosing can be extended to every six months. The company plans to amend the current study to add a multidose portion in mixed-hyperlipidemia patients before proceeding to a Phase III study using LDL reduction as the primary endpoint. Anzalone said Arrowhead hopes to pursue a regulatory path based on LDL reduction while conducting a longer-term cardiovascular outcomes trial. Arrowhead is also developing additional dimer programs in cardiovascular disease, obesity and metabolic dysfunction-associated steatohepatitis, or MASH. Anzalone said the company expects to provide further details on certain additional dimer candidates in 2027.

Plozasiran Launch and SHTG Expansion Plans Anzalone said the commercial launch of plozasiran in familial chylomicronemia syndrome, or FCS, has been smooth and somewhat faster than anticipated. He said Arrowhead has found more clinical FCS patients than initially expected, has expanded its sales force, and has secured coverage policies from most payers in FCS. The company is now focused on seeking approval in severe hypertriglyceridemia, or SHTG. Arrowhead acquired a priority review voucher to accelerate the review process, according to Anzalone. Hamilton summarized results from the SHASTA-3 and SHASTA-4 studies, reporting triglyceride reductions of 80% from baseline. He said ...