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Pasadena’s Arrowhead Pharmaceuticals, Still Buoyed by $3.7 Billion Deal, Announces New ‘Promising’ Clinical trial

Published on Thursday, February 13, 2020 | 6:31 am
Research at Arrowhead Pharmaceutical. Courtesy photo

Pasadena-based Arrowhead Pharmaceuticals Inc. said it has seen “promising” interim clinical results from the ongoing Phase 1/2a studies of two of its drugs based on the gene-silencing or RNAi (ribonucleic acid interference) platform.

The drugs are ARO-APOC3, being developed as a treatment for patients with severe hypertriglyceridemia, and ARO-ANG3, being developed to treat dyslipidemias and metabolic diseases.

“The interim results from the multiple-dose portion of the Phase 1 studies of Arrowhead’s cardiometabolic candidates ARO-APOC3 and ARO-ANG3 are highly encouraging and support our belief that RNAi may be the optimal mechanism to inhibit APOC3 and ANGPTL3,” Dr. Javier San Martin, Chief Medical Officer at Arrowhead, said. “We achieved high levels of APOC3 and ANGPTL3 protein knockdown, which led to impressive reductions in triglycerides and other lipid parameters. In addition, the long duration of effect of ARO-APOC3 and ARO-ANG3 enables a convenient once every four months or, possibly, once every six months dosing regimen, which also has the potential to improve patient compliance over other agents and mechanisms that require more frequent dosing.”

San Martin added there have been no drug-related discontinuations, and if there were any adverse events reported, the most common were headache, respiratory tract infections, and local injection site reactions.

“This high level of pharmacologic activity with good safety and tolerability to date is precisely what we were hoping for,” San Martin continued. “We look forward to further investigating the potential for ARO-APOC3 and ARO-ANG3 to provide clinical benefits in patients.”

Key interim results from the multiple-dose portion of the AROAPOC31001 Phase 1 clinical study of ARO-APOC3 include the following:

The tests involved severe hypertriglyceridemia patients and hypercholesterolemia patients who received varying doses of the drug candidates, and showed marked improvements such as reduced triglyceride and LDL-C (Low-density lipoprotein cholesterol) levels, among other beneficial results.

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