Lexicon to Present Phase 3 Trial Design for Sotagliflozin in Hypertrophic Cardiomyopathy (HCM) at Upcoming Medical Congress
Lexicon Pharmaceuticals (Nasdaq: LXRX) has announced the presentation of study design details for SONATA-HCM, a Phase 3 clinical trial evaluating sotagliflozin as a potential treatment for adults with hypertrophic cardiomyopathy (HCM). The presentation will take place at the virtual Hypertrophic Cardiomyopathy Medical Society (HCMS) 2024 Scientific Sessions.
The multinational study aims to enroll adults with symptomatic obstructive or non-obstructive HCM at 130 sites across 20 countries. Patient randomization has already begun. The trial design is intended to support a supplemental new drug application (sNDA) with a broad label for both HCM types. Experts highlight the significant unmet needs in HCM treatment and express optimism about sotagliflozin's potential efficacy in this patient population.
Lexicon Pharmaceuticals (Nasdaq: LXRX) ha annunciato la presentazione dei dettagli del disegno dello studio per SONATA-HCM, una sperimentazione clinica di Fase 3 che valuta sotagliflozin come potenziale trattamento per adulti affetti da cardiomiopatia ipertrofica (HCM). La presentazione si svolgerà durante le sessioni scientifiche virtuali della Hypertrophic Cardiomyopathy Medical Society (HCMS) 2024.
Lo studio multinazionale mira a reclutare adulti con HCM sintomatica ostruttiva o non ostruttiva in 130 siti distribuiti in 20 paesi. La randomizzazione dei pazienti è già iniziata. Il disegno dello studio è concepito per supportare una domanda di nuovo farmaco supplementare (sNDA) con un'etichetta ampia per entrambi i tipi di HCM. Gli esperti evidenziano i significativi bisogni insoddisfatti nel trattamento dell'HCM e esprimono ottimismo riguardo all'efficacia potenziale di sotagliflozin in questa popolazione di pazienti.
Lexicon Pharmaceuticals (Nasdaq: LXRX) ha anunciado la presentación de los detalles del diseño del estudio para SONATA-HCM, un ensayo clínico de Fase 3 que evalúa sotagliflozin como un tratamiento potencial para adultos con cardiomiopatía hipertrófica (HCM). La presentación tendrá lugar en las Sesiones Científicas Virtuales de la Sociedad Médica de Cardiomiopatía Hipertrófica (HCMS) 2024.
El estudio multinacional tiene como objetivo inscribir adultos con HCM obstructiva o no obstructiva sintomática en 130 sitios en 20 países. La randomización de los pacientes ya ha comenzado. El diseño del ensayo está destinado a apoyar una solicitud suplementaria de nuevo fármaco (sNDA) con una etiqueta amplia para ambos tipos de HCM. Los expertos destacan las importantes necesidades no satisfechas en el tratamiento de la HCM y expresan optimismo sobre la eficacia potencial de sotagliflozin en esta población de pacientes.
Lexicon Pharmaceuticals (Nasdaq: LXRX)는 SONATA-HCM에 대한 연구 설계 세부 사항 발표를 알렸습니다. 이는 비대 심장병( HCM)을 앓고 있는 성인을 위한 잠재적인 치료법으로 sotagliflozin을 평가하는 3상 임상 시험입니다. 발표는 2024년 가상 비대심장병의학회(HCMS) 과학 세션에서 진행될 예정입니다.
이 다국적 연구는 20개국의 130개 장소에서 증상이 있는 폐색성 또는 비폐색성 HCM을 가진 성인을 등록할 계획입니다. 환자 무작위 배정이 이미 시작되었습니다. 시험 설계는 두 가지 HCM 유형에 대한 광범위한 라벨을 가진 보충 신약 신청(sNDA)을 지원하기 위해 고안되었습니다. 전문가들은 HCM 치료에서 해결되지 않은 중요한 필요성을 강조하며 sotagliflozin이 이 환자 집단에서 잠재적인 효능을 발휘할 것이라는 희망을 나타냅니다.
Lexicon Pharmaceuticals (Nasdaq: LXRX) a annoncé la présentation des détails du design de l'étude pour SONATA-HCM, un essai clinique de phase 3 évaluant sotagliflozin comme un traitement potentiel pour les adultes atteints de cardiomyopathie hypertrophique (HCM). La présentation aura lieu lors des sessions scientifiques virtuelles de la Société Médicale de Cardiomyopathie Hypertrophique (HCMS) 2024.
L'étude multinational vise à recruter des adultes souffrant d'HCM obstructive ou non obstructive symptomatique dans 130 sites à travers 20 pays. La randomisation des patients a déjà commencé. Le design de l'essai a pour but de soutenir une demande de nouveau médicament complémentaire (sNDA) avec une étiquette large pour les deux types de HCM. Les experts soulignent les besoins non satisfaits importants dans le traitement de l'HCM et expriment leur optimisme quant à l'efficacité potentielle de sotagliflozin dans cette population de patients.
Lexicon Pharmaceuticals (Nasdaq: LXRX) hat die Präsentation der Studiendesign-Details für SONATA-HCM angekündigt, eine klinische Phase-3-Studie, die sotagliflozin als potenzielle Behandlung für Erwachsene mit hypertropher Kardiomyopathie (HCM) bewertet. Die Präsentation findet im Rahmen der virtuellen Wissenschaftlichen Sitzungen der Hypertrophic Cardiomyopathy Medical Society (HCMS) 2024 statt.
Die multinationale Studie zielt darauf ab, Erwachsene mit symptomatischer obstruktiver oder nicht obstruktiver HCM an 130 Standorten in 20 Ländern zu rekrutieren. Die Randomisierung der Patienten hat bereits begonnen. Das Studiendesign soll einen supplemental new drug application (sNDA) mit einer breiten Zulassung für beide HCM-Typen unterstützen. Experten heben die erheblichen unerfüllten Bedürfnisse in der HCM-Behandlung hervor und äußern Optimismus bezüglich der potenziellen Wirksamkeit von sotagliflozin in dieser Patientengruppe.
- Phase 3 clinical trial SONATA-HCM for sotagliflozin in HCM has begun patient randomization
- Study design aims to support a broad label sNDA for both obstructive and non-obstructive HCM
- Multinational study to be conducted at 130 sites in 20 countries
- Potential for sotagliflozin to become a new treatment option for HCM patients
- None.
Insights
The SONATA-HCM Phase 3 trial for sotagliflozin in hypertrophic cardiomyopathy (HCM) represents a significant development in cardiovascular medicine. This study is noteworthy for several reasons:
- It targets both obstructive and non-obstructive HCM, potentially addressing a broader patient population.
- The trial's design aims for a supplemental new drug application (sNDA) with a comprehensive label, which could expedite market access if successful.
- With an estimated 1.1 million HCM patients in the U.S., this represents a substantial market opportunity for Lexicon.
The dual SGLT1 and SGLT2 inhibition mechanism of sotagliflozin is intriguing, as it may offer unique benefits in HCM compared to existing treatments. The involvement of prominent cardiologists and the multinational scope of the trial (130 sites in 20 countries) lend credibility to the study. However, investors should note that Phase 3 trials are costly and time-consuming, with no guarantee of success. The outcome will be important for Lexicon's pipeline and potential future revenue streams.
This Phase 3 trial announcement is a positive development for Lexicon Pharmaceuticals (LXRX). Key financial implications include:
- Potential market expansion: With a
547.66 million market cap, success in the HCM market could significantly boost Lexicon's valuation. - R&D investment: The large-scale trial (130 sites, 20 countries) suggests substantial ongoing R&D expenses, which may impact near-term financials.
- Future revenue potential: If approved, sotagliflozin for HCM could become a major revenue driver, especially given the 1.1 million potential patients in the U.S. alone.
Investors should consider the long-term potential against the near-term costs and risks. A successful outcome could lead to partnerships, licensing deals, or increased market share. However, the biotech sector is highly volatile and trial failures can severely impact stock prices. The company's cash position and burn rate will be important metrics to watch as this trial progresses.
Patient randomization is underway in SONATA-HCM clinical trial evaluating Lexicon’s novel dual SGLT1 and SGLT2 inhibitor in this underserved and growing patient population
Study design intended to enable supplemental new drug application (sNDA) with a broad label in both obstructive and non-obstructive HCM
THE WOODLANDS, Texas, Sept. 27, 2024 (GLOBE NEWSWIRE) -- Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX) today announced that it will unveil study design details of SONATA-HCM, the company’s Phase 3 clinical trial of sotagliflozin as a potential new treatment for adults with hypertrophic cardiomyopathy (HCM), at the virtual Hypertrophic Cardiomyopathy Medical Society (HCMS) 2024 Scientific Sessions. Lexicon plans to conduct the multinational study at 130 sites in 20 countries and enroll adults aged 18 years or older diagnosed with symptomatic HCM, either obstructive (oHCM) or non-obstructive (nHCM). Initial study sites have been activated and patient randomization is underway.
Details for the presentation on “A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Multicenter Study to Evaluate the Efficacy and Safety of SOtaglifloziN in Patients with SymptomATic obstructive And non-obstructive Hypertrophic CardioMyopathy (SONATA-HCM)” is as follows:
- HCMS (virtual) – a moderated poster presentation, Friday, September 27, 2:00-2:30p.m. ET, presented by Carolyn Y. Ho, M.D., Cardiology, Brigham and Women’s Hospital, Boston, MA
The estimated prevalence of HCM in the United States is about 1.1 million. The most common patient-reported symptoms include shortness of breath, lightheadedness or dizziness, fatigue, heart fluttering or palpitations, and limitations to physical activities.
“SONATA-HCM will evaluate the efficacy of sotagliflozin, a dual SGLT2 and SGLT1 inhibitor, on symptoms, function, and other patient-reported outcomes, as well as safety in patients with symptomatic HCM,” said Dr. Ho, M.D., Cardiology, Brigham and Women’s Hospital, and a co-author of the study abstract.
“Despite recent advances in the treatment of HCM, there remain significant needs among patients and clinicians for new therapeutic options,” said Sharlene M. Day, M.D., Director, Translational Research, Division of Cardiovascular Medicine and Cardiovascular Institute, Presidential Associate Professor, University of Pennsylvania Medicine, and a co-author of the study abstract. “Although HCM is a unique condition, there are overlapping features with other conditions that lead to heart failure, so I’m optimistic that sotagliflozin could work in this patient population.”
“We are excited about the potential for sotagliflozin to become a safe and effective treatment for HCM, either as standalone therapy or in conjunction with currently available medications,” said Craig Granowitz, M.D., Ph.D., Lexicon’s senior vice president and chief medical officer. “Based on our interactions with FDA, and if supported by positive study results, we plan to submit a supplemental new drug application (sNDA) with a broad proposed label to improve symptoms and physical limitations in adults with HCM.”
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 commercially launched one of these medicines, INPEFA® (sotagliflozin) in the United States, and has a pipeline of other promising drug candidates in discovery and clinical and preclinical development in neuropathic pain, diabetes and 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, 2023, 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
FAQ
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