SONATA-HCM Study Design Presented at Heart Failure 2025, the Annual Congress of the Heart Failure Association of the European Society of Cardiology
Lexicon Pharmaceuticals (NASDAQ: LXRX) presented the design of its SONATA-HCM Phase 3 clinical trial at Heart Failure 2025, evaluating sotagliflozin for both obstructive and non-obstructive hypertrophic cardiomyopathy (HCM). The study is actively enrolling 500 patients across 20 countries, with all sites expected to be operational by September 2025.
The trial aims to assess sotagliflozin's efficacy on symptoms, function, and patient-reported outcomes in HCM patients. The primary endpoint measures symptom improvement using the Kansas City Cardiomyopathy Questionnaire at week 26. Recent evidence published in The Lancet (February 2025) suggests sotagliflozin's dual SGLT1 and SGLT2 inhibition reduces major adverse cardiovascular events.
If successful, the study could support a supplemental new drug application (sNDA) for sotagliflozin in both HCM types, addressing a significant unmet need, particularly for non-obstructive HCM where no approved therapies exist.Lexicon Pharmaceuticals (NASDAQ: LXRX) ha presentato il disegno del suo studio clinico di fase 3 SONATA-HCM al congresso Heart Failure 2025, valutando sotagliflozin sia per la cardiomiopatia ipertrofica ostruttiva sia non ostruttiva (HCM). Lo studio sta reclutando attivamente 500 pazienti in 20 paesi, con l'obiettivo che tutti i centri siano operativi entro settembre 2025.
Il trial mira a valutare l'efficacia di sotagliflozin sui sintomi, la funzionalità e gli esiti riportati dai pazienti affetti da HCM. L'endpoint primario misura il miglioramento dei sintomi tramite il Kansas City Cardiomyopathy Questionnaire alla settimana 26. Evidenze recenti pubblicate su The Lancet (febbraio 2025) indicano che l'inibizione duale di SGLT1 e SGLT2 da parte di sotagliflozin riduce gli eventi cardiovascolari avversi maggiori.
Se i risultati saranno positivi, lo studio potrebbe supportare una domanda supplementare di approvazione del farmaco (sNDA) per sotagliflozin in entrambi i tipi di HCM, rispondendo a un bisogno clinico significativo, soprattutto per la HCM non ostruttiva, per la quale non esistono terapie approvate.
Lexicon Pharmaceuticals (NASDAQ: LXRX) presentó el diseño de su ensayo clínico de fase 3 SONATA-HCM en Heart Failure 2025, evaluando sotagliflozina tanto para la miocardiopatía hipertrófica obstructiva como no obstructiva (HCM). El estudio está reclutando activamente a 500 pacientes en 20 países, con la expectativa de que todos los centros estén operativos para septiembre de 2025.
El ensayo tiene como objetivo evaluar la eficacia de sotagliflozina en los síntomas, la función y los resultados reportados por los pacientes con HCM. El objetivo principal mide la mejoría de los síntomas mediante el Kansas City Cardiomyopathy Questionnaire en la semana 26. Evidencias recientes publicadas en The Lancet (febrero de 2025) sugieren que la inhibición dual de SGLT1 y SGLT2 por sotagliflozina reduce eventos cardiovasculares adversos mayores.
Si tiene éxito, el estudio podría respaldar una solicitud suplementaria de nuevo medicamento (sNDA) para sotagliflozina en ambos tipos de HCM, abordando una necesidad clínica importante, especialmente para la HCM no obstructiva, donde no existen terapias aprobadas.
렉시콘 파마슈티컬스(NASDAQ: LXRX)는 Heart Failure 2025에서 폐쇄성 및 비폐쇄성 비대성 심근병증(HCM)에 대한 소타글리플로진의 임상 3상 시험인 SONATA-HCM의 설계안을 발표했습니다. 이 연구는 20개국에서 500명의 환자를 적극적으로 모집 중이며, 모든 연구 기관은 2025년 9월까지 가동될 예정입니다.
본 임상시험은 HCM 환자의 증상, 기능 및 환자 보고 결과에 대한 소타글리플로진의 효능을 평가하는 것을 목표로 합니다. 주요 평가 변수는 26주차에 Kansas City Cardiomyopathy Questionnaire를 통해 증상 개선을 측정합니다. 2025년 2월 The Lancet에 발표된 최신 연구 결과에 따르면, 소타글리플로진의 SGLT1 및 SGLT2 이중 억제 작용이 주요 심혈관계 부작용을 감소시키는 것으로 나타났습니다.
성공할 경우, 이 연구는 폐쇄성 및 비폐쇄성 HCM 모두에 대해 소타글리플로진의 보충 신약 신청(sNDA)을 지원할 수 있으며, 특히 승인된 치료제가 없는 비폐쇄성 HCM 환자들에게 중요한 미충족 수요를 해결할 수 있을 것입니다.
Lexicon Pharmaceuticals (NASDAQ : LXRX) a présenté la conception de son essai clinique de phase 3 SONATA-HCM lors du congrès Heart Failure 2025, évaluant le sotagliflozine pour la cardiomyopathie hypertrophique obstructive et non obstructive (HCM). L'étude recrute activement 500 patients dans 20 pays, avec l'objectif que tous les sites soient opérationnels d'ici septembre 2025.
L'essai vise à évaluer l'efficacité du sotagliflozine sur les symptômes, la fonction et les résultats rapportés par les patients atteints de HCM. Le critère principal mesure l'amélioration des symptômes via le Kansas City Cardiomyopathy Questionnaire à la semaine 26. Des preuves récentes publiées dans The Lancet (février 2025) suggèrent que l'inhibition double de SGLT1 et SGLT2 par le sotagliflozine réduit les événements cardiovasculaires majeurs indésirables.
En cas de succès, l'étude pourrait soutenir une demande d'autorisation de mise sur le marché complémentaire (sNDA) pour le sotagliflozine dans les deux types de HCM, répondant à un besoin médical important, notamment pour la HCM non obstructive où aucune thérapie approuvée n'existe actuellement.
Lexicon Pharmaceuticals (NASDAQ: LXRX) stellte auf der Heart Failure 2025 das Design seiner Phase-3-Studie SONATA-HCM vor, in der sotagliflozin sowohl bei obstruktiver als auch nicht-obstruktiver hypertropher Kardiomyopathie (HCM) untersucht wird. Die Studie rekrutiert aktiv 500 Patienten in 20 Ländern, wobei alle Studienzentren bis September 2025 einsatzbereit sein sollen.
Ziel der Studie ist es, die Wirksamkeit von sotagliflozin auf Symptome, Funktion und patientenberichtete Ergebnisse bei HCM-Patienten zu bewerten. Der primäre Endpunkt misst die Verbesserung der Symptome anhand des Kansas City Cardiomyopathy Questionnaire in Woche 26. Jüngste Studien, veröffentlicht im Februar 2025 in The Lancet, deuten darauf hin, dass die duale Hemmung von SGLT1 und SGLT2 durch sotagliflozin schwere kardiovaskuläre Ereignisse reduziert.
Bei Erfolg könnte die Studie eine ergänzende Zulassungsanfrage (sNDA) für sotagliflozin bei beiden HCM-Typen unterstützen und damit einen bedeutenden ungedeckten medizinischen Bedarf abdecken, insbesondere bei der nicht-obstruktiven HCM, für die derzeit keine zugelassenen Therapien existieren.
- Only ongoing Phase 3 study targeting both obstructive and non-obstructive HCM
- Potential to address unmet medical need with no approved therapies for non-obstructive HCM
- Study could support broad label expansion for sotagliflozin
- Recent evidence shows reduction in major adverse cardiovascular events
- Results and efficacy not yet proven for HCM indication
- Complete site operations not expected until Q3 2025, indicating lengthy timeline to potential approval
Insights
Lexicon's Phase 3 SONATA-HCM trial for sotagliflozin presents a significant opportunity to address unmet needs in hypertrophic cardiomyopathy treatment.
Lexicon's SONATA-HCM trial represents a potentially significant advancement in the treatment landscape for hypertrophic cardiomyopathy (HCM). As the only ongoing Phase 3 study that includes both obstructive and non-obstructive HCM patients, this positions Lexicon uniquely in the field. The trial design is particularly notable for its inclusivity of non-obstructive HCM patients – a population with no currently approved therapies, highlighting a substantial unmet medical need.
The study's robust design includes 500 patients across 20 countries, evenly split between obstructive and non-obstructive HCM subtypes. The primary endpoint focuses on symptomatic improvement measured by the validated Kansas City Cardiomyopathy Questionnaire, an appropriate patient-centered outcome for this condition where quality of life impairment is significant.
Sotagliflozin's dual inhibition of both SGLT1 and SGLT2 differentiates it from other SGLT2-only inhibitors. The press release references recent evidence published in The Lancet suggesting this dual mechanism reduces major adverse cardiovascular events. This pharmacological profile could potentially address the complex pathophysiology of HCM, though this remains to be proven in the current trial.
If successful, this trial could support a supplemental new drug application that would expand sotagliflozin's approved indications. The drug already has an established safety record in heart failure, which could streamline regulatory consideration. For HCM patients, especially those with the non-obstructive variant who currently lack treatment options, sotagliflozin could represent a significant therapeutic advance.
Lexicon is evaluating the safety and efficacy of sotagliflozin in patients with both obstructive and non-obstructive hypertrophic cardiomyopathy (HCM)
Pivotal Phase 3 clinical trial is actively enrolling patients in 20 countries, with all sites expected to be operational by Q3 2025
THE WOODLANDS, Texas, May 19, 2025 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced details of its “SOtaglifloziN in Patients with SymptomATic obstructive And non-obstructive Hypertrophic CardioMyopathy (SONATA-HCM)” study were presented on Saturday, May 17. The study design was disclosed in Belgrade, Serbia at one of the premier international heart failure meetings: Heart Failure 2025, the Annual Congress of the Heart Failure Association of the European Society of Cardiology.
Enrollment is underway in SONATA-HCM, with all study sites in 20 countries expected to be operational by September 2025. Lexicon believes there is potential for the study results to support a supplemental new drug application (sNDA) for sotagliflozin for both obstructive and non-obstructive HCM.
SONATA-HCM is the only ongoing Phase 3 study in both obstructive and non-obstructive HCM. It is a randomized, double-blind, placebo-controlled multicenter trial that will evaluate the efficacy of sotagliflozin, a dual SGLT1 and SGLT2 inhibitor, on symptoms, function, and other patient-reported outcomes, as well as safety in patients with symptomatic HCM. The study is designed to enroll 500 patients worldwide, 250 with obstructive HCM and 250 with non-obstructive HCM. The primary efficacy endpoint is improvement in symptoms, as measured by change from baseline to week 26, in the Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ) CSS survey.
"There is a huge unmet need for people suffering from HCM, with no approved or effective therapies for non-obstructive HCM," said Sharlene M. Day, M.D., co-principal investigator for SONATA-HCM and Presidential Professor and Director of Translational Research in the Division of Cardiovascular Medicine and the Cardiovascular Institute at the University of Pennsylvania Perelman School of Medicine. "I'm very excited to be testing the potential for sotagliflozin to improve symptoms in a broad number of patients across the spectrum of HCM, as has previously been demonstrated in patients with heart failure with reduced and preserved ejection fraction. Sotagliflozin’s tolerability profile and immediate benefits observed in patients with heart failure could potentially make it a very appealing option for patients with HCM if the benefits do indeed extend to this population."
As a chronic, progressive disease, HCM can worsen over time and lead to other complications, including heart failure and stroke. Sotagliflozin is a proven drug for the treatment of heart failure, with an established safety record and significant use in clinical practice. A growing body of evidence suggests that sotagliflozin’s dual inhibition of SGLT1 and SGLT2 reduces the risk of major adverse cardiovascular events (MACE), including heart attack and stroke, as most recently published in The Lancet in February 2025.
“SONATA-HCM is an important study with the potential to support a broad label to enhance the care of patients with both obstructive and non-obstructive HCM,” said Craig Granowitz, M.D., Ph.D., Lexicon’s senior vice president and chief medical officer.
For more information on the study, visit the SONATA-HCM page on ClinicalTrials.gov.
About Sotagliflozin
Discovered using Lexicon’s unique approach to gene science, sotagliflozin is an oral inhibitor of two proteins responsible for glucose regulation known as sodium-glucose cotransporter types 2 and 1 (SGLT2 and SGLT1). SGLT2 is responsible for glucose and sodium reabsorption by the kidney and SGLT1 is responsible for glucose and sodium absorption in the gastrointestinal tract. Sotagliflozin has been studied in multiple patient populations encompassing heart failure, diabetes, and chronic kidney disease in clinical studies involving approximately 20,000 patients.
About Lexicon Pharmaceuticals
Lexicon is a biopharmaceutical company with a mission of pioneering medicines that transform patients’ lives. Through the Genome5000™ program, Lexicon’s unique genomics target discovery platform, Lexicon scientists studied the role and function of nearly 5,000 genes and identified more than 100 protein targets with significant therapeutic potential in a range of diseases. Through the precise targeting of these proteins, Lexicon is pioneering the discovery and development of innovative medicines to treat disease safely and effectively. Lexicon has a pipeline of promising drug candidates in discovery and clinical and preclinical development in neuropathic pain, hypertrophic cardiomyopathy (HCM), obesity, metabolism and other indications. For additional information, please visit www.lexpharma.com.
Safe Harbor Statement
This press release contains “forward-looking statements,” including statements relating to Lexicon’s financial position and long-term outlook on its business, including the commercialization of its approved products and the clinical development of, regulatory filings for, and potential therapeutic and commercial potential of its other drug candidates. In addition, this press release also contains forward looking statements relating to Lexicon’s growth and future operating results, discovery, development and commercialization of products, strategic alliances and intellectual property, as well as other matters that are not historical facts or information. All forward-looking statements are based on management’s current assumptions and expectations and involve risks, uncertainties and other important factors, specifically including Lexicon’s ability to meet its capital requirements, successfully commercialize its approved products, successfully conduct preclinical and clinical development and obtain necessary regulatory approvals of its other drug candidates on its anticipated timelines, achieve its operational objectives, obtain patent protection for its discoveries and establish strategic alliances, as well as additional factors relating to manufacturing, intellectual property rights, and the therapeutic or commercial value of its approved products and other drug candidates. Any of these risks, uncertainties and other factors may cause Lexicon’s actual results to be materially different from any future results expressed or implied by such forward-looking statements. Information identifying such important factors is contained under “Risk Factors” in Lexicon’s annual report on Form 10-K for the year ended December 31, 2024, as filed with the Securities and Exchange Commission. Lexicon undertakes no obligation to update or revise any such forward-looking statements, whether as a result of new information, future events or otherwise.
For Investor and Media Inquiries:
Lisa DeFrancesco
Lexicon Pharmaceuticals, Inc.
lexinvest@lexpharma.com
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