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Rona Therapeutics Advances INHBE siRNA into Phase 1 Clinical Development

Rona Therapeutics, a company focused on developing innovative therapies using RNA interference (RNAi) technology, has announced a significant breakthrough in its clinical trials. Recently, they completed dosing for Cohort 1 of their Phase 1 first-in-human study for RN3161, a GalNAc-conjugated siRNA targeting INHBE, designed specifically for treating obesity.

This first cohort has demonstrated a favorable safety and tolerability profile, giving the company confidence as they mark this milestone in their INHBE program. “We are pleased to report successful completion of our first cohort in this Phase 1 trial, an important step in advancing RN3161 and validating the potential of INHBE as a new metabolic target,” said Dr. Alex M. DePaoli, CMO of Rona Therapeutics.

The rising incidences of obesity have sparked increased interest in INHBE among scientific and industry professionals, especially following the emergence of compelling human genetic evidence that supports its role in metabolic processes. RN3161 is poised to offer a unique therapeutic approach, utilizing infrequent dosing—either once or twice a year—promising to reshape the treatment landscape for obesity.

The Phase 1 Study Details

The Phase 1 trial is meticulously structured as a randomized, double-blind, placebo-controlled study, aimed at evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and the drug's efficacy on body weight in adults who are overweight or obese. This rigorous design ensures reliable data, essential for the future of RN3161 as a viable option for managing obesity. Having successfully completed Cohort 1, Rona Therapeutics plans to continue its endeavors with additional cohorts expected to be completed in 2026.

About Rona Therapeutics

Founded in 2021, Rona Therapeutics has swiftly established itself at the forefront of RNAi therapeutic development. The company's mission is to tackle cardiometabolic disorders, including obesity and degenerative diseases, by employing state-of-the-art technologies in RNA delivery, multi-valency, and oligo chemistry. So far, Rona has advanced four programs into clinical stages, having secured approximately $200 million in funding from prestigious global healthcare investors and strategic partners.

As a pioneer in metabolic disease therapies, Rona continues to invest in its proprietary platform, reflecting its commitment to bringing next-generation siRNAs to the forefront of medical treatment.

In addition to its immediate focus on obesity, Rona Therapeutics is diligently working on expanding its portfolio to address a variety of severe health challenges using its sophisticated RNA technology. As the trial progresses, Rona is poised to not only contribute to the body of research surrounding INHBE but also to potentially innovate the standard of care for individuals struggling with obesity, offering hope for more effective treatments in the near future.