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Rona Therapeutics Secures Investigational New Drug Clearance from FDA for RN0361

Rona Therapeutics Inc., renowned for its pioneering work in RNA-targeted therapeutics, has recently received clearance for an Investigational New Drug (IND) by the U.S. Food and Drug Administration (FDA) to advance its innovative therapy, RN0361. This targeted small interfering RNA (siRNA) drug aims to combat severe hypertriglyceridemia (SHTG), mixed dyslipidemia, and familial chylomicronemia syndrome (FCS), conditions that can lead to serious cardiovascular issues.

With the favorable results from a 6-month single-dose Phase 1 trial showing promising safety profiles and stable triglyceride reduction, Rona is set to move forward with a crucial Phase 2 study. This upcoming study will be a randomized, double-blind, placebo-controlled, multicenter trial. It will assess the efficacy, safety, and duration of RN0361's action within a hypertriglyceridemia patient population over a nine-month follow-up period.

The scientific foundation of RN0361 rests on its ability to effectively silence APOC3, a protein that plays a critical role in lipid metabolism. By utilizing Rona's proprietary siRNA conjugate and oligo chemistry platform known as GAIA, RN0361 achieves potent and durable hepatocyte-specific silencing through subcutaneous administration. This approach optimizes chemical modifications that result in significant silencing of the APOC3 mRNA, thereby substantially reducing the expression of the protein itself.

Stella Shi, the CEO and Founder of Rona Therapeutics, expressed her enthusiasm about the IND submission. She highlighted the company's commitment to advancing transformative RNA interference (RNAi) therapies for individuals suffering from severe lipid disorders. "RN0361's Phase 1 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 agencies to accelerate its development," she stated.

Providing a medical perspective, Dr. Alex M. DePaoli, Chief Medical Officer and Executive Vice President of translational medicine at Rona, shared his excitement regarding the Phase 2 study. He believes that RN0361 may provide a substantial therapeutic solution for dysmetabolic patients facing hypertriglyceridemia and its several pathological consequences. The data derived from the First In Human single-dose study already indicates remarkable potency and durability in triglyceride reduction—offering patients an invaluable tool for managing a significant unmet clinical need.

Beyond RN0361, Rona Therapeutics has established itself as a leader in the field of nucleic acid therapeutics, specializing in treatments for metabolic and neurological diseases. The company is dedicated to developing best-in-class siRNA drugs characterized by unique differentiation and innovative approaches, aiming to address unmet medical needs and significantly improve outcomes in cardiovascular diseases, obesity, and metabolic syndromes.

Furthermore, Rona Therapeutics is exploring the potential of extra-hepatic delivery methods that could benefit patients with neurological disorders and various metabolic syndromes, highlighting their commitment to broadening the accessibility and efficacy of their treatment modalities in diverse patient populations.

In conclusion, the FDA's clearance for RN0361 signifies not just a milestone for Rona Therapeutics, but also a glimmer of hope for patients battling severe lipid disorders. As further research and trials commence, the biotech community and the patients it serves will be eager to witness the outcomes and advancements that may emerge from this promising novel therapy.