Company to initiate clinical study of gene-silencing drug targeting hypertriglyceridemia via a reduction in APOC3 protein expression.
Chinese biotech Rona Therapeutics has received clearance from the US FDA to initiate a Phase 2 clinical trial for its small interfering RNA (siRNA) drug designed for the treatment of hypertriglyceridemia and other metabolic conditions. The Shanghai-based company plans to evaluate the efficacy, safety and durability of its RN0361 gene-silencing therapeutic in a randomized, double-blind, placebo-controlled, multicenter study involving patients with hypertriglyceridemia over a nine-month follow-up period.
Hypertriglyceridemia, a condition characterized by elevated levels of triglycerides in the blood, increases the risk of cardiovascular diseases such as heart attack and stroke. It becomes more common with aging due to metabolic changes, including reduced lipid clearance, increased insulin resistance, and hormonal shifts that affect fat metabolism.
According to Rona, RN0361 demonstrated a favorable safety profile and sustained triglyceride reduction in a Phase 1 trial. The drug targets APOC3, a key regulator of triglyceride metabolism, using the company’s platform, which combines siRNA conjugate and oligo chemistry to enable hepatocyte-specific silencing. The approach employs optimized chemical modifications to enhance potency and durability, leading to significant and prolonged silencing of APOC3 mRNA and reduction in APOC3 protein expression.
“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,” said Rona’s Chief Medical Officer Dr Alex DePaoli. “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.”
Rona raised $35 million last year to accelerate the development of its nucleic acid drug pipeline for metabolic and neurodegenerative diseases. The company is working on programs in cardiovascular disease, obesity, and metabolic-associated steatohepatitis (MASH) using siRNA-based therapies. The company is also progressing in the area of extra-hepatic nucleic acid delivery, focusing on central nervous system disorders, including amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease.
