#Shanghai #USA #FDA Clearance #Rona Therapeutics #RN0361

Rona Therapeutics Advances Revolutionary siRNA for Hypertriglyceridemia

In a significant breakthrough for the management of metabolic disorders, Rona Therapeutics Inc. has announced the successful clearance of an Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA). This clearance allows the company to initiate Phase 2 clinical studies for RN0361, an innovative siRNA (small interfering RNA) therapy specifically targeting APOC3. This treatment is intended for patients suffering from severe hypertriglyceridemia, mixed dyslipidemia, and familial chylomicronemia syndrome (FCS).

Rona Therapeutics, a prominent player in RNA-targeted therapeutics, plans to leverage the positive safety profile exhibited by RN0361 during a Phase 1 clinical trial involving a six-month duration and a single dose approach. The clinical trial showcased sustained reductions in triglycerides, marking RN0361 as a potentially best-in-class therapy. Following this promising outcome, the company is set to launch a randomized, double-blind, placebo-controlled Phase 2 study that will investigate the efficacy, safety, and duration of the treatment in a broad patient population, extending over a nine-month follow-up period.

One of the standout features of RN0361 is its utilization of Rona's proprietary GAIA platform, designed to achieve robust silencing of APOC3 mRNA and subsequently reduce the expression of the APOC3 protein—crucial for managing triglyceride levels. This therapy allows for subcutaneous administration, enhancing the patient experience and compliance.

Stella Shi, CEO and Founder of Rona Therapeutics, expressed the company's dedication to advancing RNA interference (RNAi) therapies aimed at patients burdened by severe lipid disorders. In a recent statement, she emphasized the importance of the IND submission, marking a significant milestone in the development of transformative treatment options focused on critical metabolic conditions. "RN0361's Phase I data validates 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."

The excitement surrounding this Phase 2 study is echoed by Dr. Alex M. DePaoli, Chief Medical Officer and Executive Vice President of Translational Medicine at Rona. He highlighted the crucial role that RN0361 could play in treating various cohorts of patients grappling with hypertriglyceridemia and its accompanying dangers. According to Dr. DePaoli, the outstanding potency and longevity of triglyceride reductions observed in initial trials offer a remarkable opportunity for effectively managing this health crisis.

As Rona Therapeutics forges ahead with this promising research, the company aims not only to break new ground in hypertriglyceridemia management but also to pave the way for addressing unmet medical needs. Known for its commitment to pioneering nucleic acid-based therapeutics, Rona is dedicated to developing innovative solutions for metabolic and neurological diseases. With ongoing exploration into extra-hepatic delivery methods for neurological disorders and other metabolic syndromes, this latest development could serve as a pivotal moment for patients worldwide, bringing hope and potentially transforming lives.

In summary, Rona Therapeutics is on the brink of an important advancement in the fight against severe lipid disorders. The Phase 2 clinical study of the targeted siRNA RN0361 offers a promising new avenue for treatment, harnessing the power of RNA technology to tackle a significant unmet need in healthcare. As trials progress, the medical community will be keenly observing the results, which may eventually redefine therapeutic approaches for patients with hypertriglyceridemia.