Lexicon Pharmaceuticals, Inc. (NASDAQ:LXRX), a leading cardiometabolic biopharmaceutical company, today provided an update on the baseline characteristics of the patients randomized into the ongoing pivotal Phase 3 SOtaglifloziN in Patients with SymptomATic obstructive And non-obstructive Hypertrophic CardioMyopathy (SONATA-HCM) clinical trial evaluating sotagliflozin in patients with symptomatic hypertrophic cardiomyopathy. The data will be presented in detail by Dr. Sharlene Day, M.D., Presidential Professor, Director of Translational Research of the Penn Cardiovascular Institute, University of Pennsylvania and co-chair of the SONATA-HCM steering committee, on Saturday, October 24, 2026, at 4:05 p.m. ET at the International HCM Summit in Boston, MA.
The patient enrollment profile of SONATA-HCM is consistent with the study’s inclusive design, which aimed to reflect real-world clinical practice. Patients were permitted to remain on stable background therapies, including cardiac myosin inhibitors, allowing evaluation of sotagliflozin in patients receiving currently available HCM treatments. Importantly, baseline demographic and clinical characteristics within both the non-obstructive (nHCM) and obstructive HCM (oHCM) cohorts were generally consistent with those reported in previous clinical studies of other agents in those respective patient populations, supporting the relevance of the SONATA-HCM study population to the broader HCM community.
In total, SONATA-HCM enrolled 542 patients across more than 130 sites in 20 countries, substantially exceeding the study’s target enrollment of 500 patients and a providing larger dataset that may further enhance the statistical power of the study to detect meaningful treatment effects on its primary efficacy endpoint. Among enrolled patients, 440 have nHCM and 102 have oHCM, positioning SONATA-HCM to evaluate sotagliflozin in a broad HCM population while emphasizing patients with non-obstructive disease, who currently have no FDA-approved disease-specific treatments.
The overall study population had a mean baseline Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) of 59, reflecting a considerable burden of symptoms and limitations in daily activities at study entry. KCCQ-CSS ranges from 0 to 100, with higher scores indicating better health status and fewer symptoms, demonstrating that the SONATA-HCM study population began the trial with substantial opportunity for improvement. The primary efficacy endpoint of the pivotal study is improvement in symptoms, as measured by change from baseline in KCCQ-CSS at Week 26.
"We designed SONATA-HCM to evaluate sotagliflozin in a broad and clinically relevant HCM population reflective of real-world clinical practice, and we are very pleased that the final study population met that objective," said Craig Granowitz, M.D., Ph.D., senior vice president and chief medical officer of Lexicon. "The demographic and clinical characteristics of both the obstructive and non-obstructive HCM cohorts were generally consistent with those reported in previous studies of other agents, while the baseline KCCQ score reflects a population with meaningful symptom burden. Combined with the substantial over enrollment and representation of both major HCM subtypes, we believe SONATA-HCM is well positioned to evaluate sotagliflozin across the broad spectrum of symptomatic HCM patients."
SONATA-HCM is the only ongoing Phase 3 study in both nHCM and oHCM and is the largest Phase 3 study including both nHCM and oHCM to date. Topline results are anticipated in the first quarter of 2027.