RN0361 / Ikaria Biosci, Rona Therapeutics - LARVOL DELTA

RN0361, an siRNA Directed Against ApoC3 Demonstrates Potent and Durable Reduction of ApoC3 and Triglyceride Over 6 Months in the Clinic (AHA 2025) - "RN0361, a long acting and potent siRNA directed against ApoC3 was well tolerated with no dose limiting toxicities through the highest dose tested and demonstrated durable reduction of ApoC3, TG, VLDL-c and Remnant-c levels over at least 180 days. These data provide support for a best-in-class profile for RN0361 and support further development. A Ph2 trial in patients with significant hypertriglyceridemia is under way."

Cardiovascular • Diabetes • Dyslipidemia • Hypertriglyceridemia • Metabolic Disorders • Pancreatitis • Severe Hypertriglyceridemia • APOB • APOC3

Study of RN0361in Adult Healthy Subjects and Adult Hypertriglyceridemic Subjects (clinicaltrials.gov) - P1/2 | N=108 | Recruiting | Sponsor: Ikaria Bioscience Pty Ltd | Not yet recruiting ➔ Recruiting | Phase classification: P1 ➔ P1/2 | N=60 ➔ 108 | Trial completion date: Jun 2026 ➔ Dec 2026 | Trial primary completion date: Sep 2025 ➔ Sep 2026

Enrollment change • Enrollment open • Phase classification • Trial completion date • Trial primary completion date • Dyslipidemia • Familial Chylomicronemia Syndrome • Hypertriglyceridemia

Rona Therapeutics Receives IND Clearance from FDA to Initiate Phase 2 Study of RN0361: an APOC3 Targeted siRNA for Management of Hypertriglyceridemia (Canada Newswire) - "Rona Therapeutics Inc...announced the clearance of an Investigational New Drug (IND) by the U.S. Food and Drug Administration (FDA) for RN0361-an APOC3 targeted siRNA (small interfering RNA) therapeutic for severe hypertriglyceridemia (SHTG), mixed dyslipidemia, and familial chylomicronemia syndrome (FCS) treatment....The upcoming randomized, double-blind, placebo controlled, multicenter global Ph2 study will evaluate RN0361 efficacy, safety and duration in hypertriglyceridemia patient population for nine-month follow-up period."

IND • New P2 trial • Dyslipidemia • Familial Chylomicronemia Syndrome • Severe Hypertriglyceridemia

Study of RN0361 in Adult Subjects (clinicaltrials.gov) - P1 | N=60 | Not yet recruiting | Sponsor: Ikaria Bioscience Pty Ltd

New P1 trial • Dyslipidemia • Hypertriglyceridemia