Arrowhead Pharmaceuticals Inc.’s tox blowup in 2016 set back its clinical timelines, but it also prompted the company to bring a more versatile platform online faster than originally planned. Early data from replacement candidates suggest management’s choice to scrap its initial programs and instead accelerate development of a next-generation RNAi delivery platform is paying off.
On Sept. 6 at the World Gastroenterologists Summit, the company reported the first efficacy data for a candidate from its new subcutaneous Targeted RNAi Molecule (TRiM) platform. In a Phase I/II study in patients with chronic HBV, 100 mg of ARO-HBV lowered hepatitis B surface antigen (HBsAg) by a mean of 2.0 log10, or a 99% reduction in HBsAg, and a 200 mg dose lowered HBsAg by a mean of 1.4 log10, or 96% reduction.
Arrowhead reported that ARO-HBV was “generally well-tolerated” among 40 patients with chronic HBV. The most common adverse events were injection site reactions associated with roughly 10% of injections.
The company had reported safety data in June from another program to treat alpha α-1 antitrypsin (AAT; A1AT; SERPINA1) deficiency, at the Alpha-1 Foundation National Education Conference.