Lexicon Pharmaceuticals Completes Patient Enrolment In Pivotal Phase 3 SONATA-HCM Trial

Lexicon Pharmaceuticals, Inc. has announced the completion of patient randomisation in its pivotal Phase 3 SONATA-HCM clinical trial evaluating sotagliflozin as a potential treatment for patients with symptomatic hypertrophic cardiomyopathy (HCM), including both non-obstructive (nHCM) and obstructive (oHCM) forms of the disease. The milestone marks the successful completion of enrolment as the company moves closer to evaluating the therapy's effectiveness in a large global patient population.

The SONATA-HCM trial exceeded its original enrolment target of 500 patients, recruiting participants from more than 130 clinical sites across 20 countries. The study includes a substantial majority of patients with non-obstructive HCM, a group for whom treatment options remain limited, while also enrolling a meaningful number of patients with obstructive HCM. Lexicon believes this broad study population will allow a comprehensive evaluation of sotagliflozin across the full spectrum of symptomatic HCM. Topline results from the trial are expected in the first quarter of 2027.

Craig Granowitz, Senior Vice President and Chief Medical Officer at Lexicon Pharmaceuticals, said completing enrolment represents an important milestone for people living with hypertrophic cardiomyopathy, a chronic and progressive heart condition. He noted that sotagliflozin's dual inhibition of SGLT1 and SGLT2 provides a unique mechanism of action compared with currently available treatments, which could make it a differentiated treatment option for patients with symptomatic HCM. He added that the company looks forward to reporting the study's topline results in early 2027.

SONATA-HCM is currently the only ongoing Phase 3 clinical trial evaluating a therapy in both non-obstructive and obstructive hypertrophic cardiomyopathy and is the largest Phase 3 study to include both patient populations. The randomised, double-blind, placebo-controlled multinational trial is designed to assess the effects of sotagliflozin on symptoms, physical function, patient-reported outcomes and safety. The primary endpoint measures changes in patients' symptoms after 26 weeks of treatment using the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS). Patients receiving stable guideline-directed HCM therapies, including cardiac myosin inhibitors, were eligible to participate if they met the study criteria.

Sharlene M. Day, co-principal investigator of the SONATA-HCM trial and Director of Translational Research at the Penn Cardiovascular Institute, said there remains a significant need for additional treatment options that can better manage persistent symptoms and improve daily functioning for people living with hypertrophic cardiomyopathy. She added that a well-tolerated medicine with a different mechanism of action that complements existing therapies could represent an important advancement for both physicians and patients.

Carolyn Y. Ho, co-principal investigator of the study, Professor of Medicine at Harvard Medical School and Medical Director of the Cardiovascular Genetics Center at Brigham and Women's Hospital, described the completion of enrolment as a major achievement for the HCM research community. She thanked the investigators, study teams and participants whose commitment made the trial possible, noting that their contribution is essential to advancing research and potentially bringing a new treatment option to patients living with hypertrophic cardiomyopathy.