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Arrowhead Pharma Highlights Interim Data On ARO-APOC3, ARO-ANG3

Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR) today reported promising interim clinical results from the ongoing Phase 1/2a studies of its two RNAi-based cardiometabolic candidates, ARO-APOC3 targeting apolipoprotein

Benzinga · 02/05/2020 21:00

Arrowhead Pharmaceuticals Inc. (NASDAQ:ARWR) today reported promising interim clinical results from the ongoing Phase 1/2a studies of its two RNAi-based cardiometabolic candidates, ARO-APOC3 targeting apolipoprotein C-III (APOC3) being developed as a potential treatment for patients with severe hypertriglyceridemia, and ARO-ANG3 targeting angiopoietin like protein 3 (ANGPTL3) being developed as a potential treatment for dyslipidemias and metabolic diseases.

Javier San Martin, M.D., chief medical officer at Arrowhead, said: “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. 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 4 months or, possibly, once every 6 months dosing regimen, which also has the potential to improve patient compliance over other agents and mechanisms that require more frequent dosing. From a safety and tolerability perspective, both ARO-ANG3 and ARO-APOC3 data continue to look similar to our other TRiM™ candidates. There have been no drug-related discontinuations, and the most common adverse events reported 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. 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:

  • Severe hypertriglyceridemia patients with at least 29 days of data after receiving the first dose (50 mg dose, n=3)
    • Mean maximum reductions in APOC3 of 97%
    • Mean maximum reductions in triglycerides (TG) of 95%
    • Mean maximum absolute reduction in TG of -3183 mg/dL

Key interim results from the multiple-dose portion of the AROANG1001 Phase 1 clinical study of ARO-ANG3 include the following:

  • Hypercholesterolemia patients on a stable LDL-C lowering treatment regimen including statins, with or without ezetimibe, and some receiving PCSK9 inhibitors, with at least 29 days of data after receiving the first dose (100-300 mg dose, n=22)
    • Mean maximum reductions in ANGPTL3 of 79-88%
    • Mean maximum reductions in LDL-C of 39-42%
  • Hypertriglyceridemia patients with at least 29 days of data after receiving the first dose (200 mg dose, n=5)
    • Mean maximum reductions in ANGPTL3 of 83%
    • Mean maximum reductions in TG of 79%