Rona Therapeutics Receives IND Clearance from FDA to Initiate Phase 2 Study of RN0361: an APOC3 Targeted siRNA for Management of Hypertriglyceridemia

SHANGHAI, March 17, 2025 /PRNewswire/ --Rona Therapeutics Inc., a global leader in RNA-targeted therapeutics, announced the clearance of an Investigational New Drug (IND) by the U.S. Food and Drug Administration (FDA) forRN0361-an APOC3 targeted siRNA(small interfering RNA) therapeutic for severe hypertriglyceridemia (SHTG), mixed dyslipidemia, and familial chylomicronemia syndrome (FCS) treatment. The company plans to advance RN0361 development in HTG patients based on a favorable safety profile and sustained triglyceride (TG) reduction in a 6-month single dose phase 1 clinical trial. 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.

RN0361 is a potential best-in-class APOC3 targetsiRNAutilizes Rona's proprietary siRNA conjugate and oligo chemistry platformGAIA to enable robust and durable hepatocyte-specific silencing via subcutaneous administration. The therapy achieves durable gene silencing through optimized chemical modifications, significantly silencing APOC3 mRNA and reducing the expression of APOC3 protein.

"This IND submission underscores our commitment to advance transformative RNAi therapies for patients with severe lipid disorders," saidStella Shi, CEO and Founder of Rona Therapeutics. "RN0361's Phase I data validate its potential as a best-in-class siRNA with durable efficacy and patient-friendly dosing. We look forward to collaborating with global investigators and agency to accelerate its development."

Dr. Alex M. DePaoli, CMO and EVP of translational medicine, was excited to share: "This Phase 2 study of RN0361 in dysmetabolic patients with hypertriglyceridemia will built on the evidence for RN0361 as an important therapeutic for multiple populations with significant hypertriglyceridemia and its pathologic consequences." He added, "The remarkable potency and durability of triglyceride reduction already seen in the First in Human single dose study offers patients a potential powerful tool in the management of a substantial unmet need."

About Rona Therapeutics

Rona Therapeutics is a global leader in nucleic acid innovative drug platform company, specializing in the treatment of metabolic and neurological diseases. Rona Therapeutics is always committed to developing the best and first-in-class siRNA drugs with differentiation and innovation to address unmet needs and improve outcome in cardiovascular diseases, obesity, and MASH. In addition, Rona Therapeutics is unlocking the potential of extra-hepatic delivery for neurological disorders and adipose/muscle delivery or various metabolic syndromes.