Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR) today announced additional Phase 1/2 clinical data on ARO-APOC3, the company’s investigational RNA interference (RNAi) therapeutic targeting apolipoprotein C-III (APOC3) being developed as a treatment for patients with hypertriglyceridemia (HTG), severe hypertriglyceridemia (sHTG), and familial chylomicronemia syndrome (FCS), at the American Heart Association (AHA) Scientific Sessions 2021.
Four genetically confirmed FCS patients received 50 mg ARO-APOC3 and 26 multifactorial chylomicronemia (MCM or non-FCS) patients received 10, 25, 50, or 100 mg ARO-APOC3 on days 1 and 29. Since similar responses were observed among MCM pts, results were pooled across dose levels. Safety and pharmacodynamic responses were examined. Maximal mean and median changes from baseline for APOC3, triglycerides (TG), non-HDL-C, and HDL-C were reported.
Key data presented include the following:
- In patients with FCS compared with non-FCS, ARO-APOC3 achieved similar levels of reduction of APOC3 and changes in key lipid parameters
- In patients with FCS compared with non-FCS, safety parameters were similar and comparable
- In patients with sHTG, ARO-APOC3 was well tolerated, and consistently decreased APOC3, TG, and non‑HDL-C, and increased HDL-C, independent of underlying genetic cause of HTG
- ARO-APOC3 may represent a promising RNAi therapeutic for the treatment of sHTG with infrequent dosing of every 3 months or every 6 months
- APOC3 was reduced by 98% in FCS patients and 96% in MCM patients
- Both groups showed similar maximum median reductions in TG of 91% and 90%, respectively
- Non-HDL-C was reduced by 58% and 49%, respectively
- HDL-C increased by 152% and 111%, respectively
- ARO-APOC3 was generally well tolerated
- No treatment emergent adverse event (TEAE)-related study drug discontinuation, dose interruptions, or premature study withdrawals
- No clear pattern of an increased frequency or intensity of adverse events with increasing dose level
- TEAEs and the safety parameters were similar and comparable with FCS compared to non-FCS subjects
- 2 serious adverse events (chest pain and acute pancreatitis) not related to ARO-APOC3 in 2 subjects in the non-FCS group – both subjects completed the study
A copy of the presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.
About Arrowhead Pharmaceuticals
Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.
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Source: Arrowhead Pharmaceuticals, Inc.
Arrowhead Pharmaceuticals, Inc.
Vince Anzalone, CFA