Closing the gap between industry and academia to place patients directly at the center of research efforts.
LQT Therapeutics, a privately-held biopharmaceutical company advancing novel SGK1 kinase therapeutics, today announced that the project, “SILENCE-LQTS: SGK1 inhibition as a novel therapeutic approach in Long QT Syndrome” was selected for funding by the European Joint Program on Rare Diseases (EJP RD). The EJP RD is a collaboration of over 130 institutions from 35 countries, working together to create a comprehensive, sustainable ecosystem between research, care, and medical innovation.
Congenital Long QT Syndrome (LQTS) is a rare inherited disorder associated with life- threatening arrhythmias and sudden cardiac death (SCD) in relatively young and otherwise healthy individuals. LQTS has a heterogeneous genetic basis, usually impacting one of 3 key cardiac ion channels caused by mutations in distinct genes. Current symptom-directed therapies aimed at reducing arrhythmia triggering events, including lifestyle changes, beta blockade, and left cardiac sympathetic denervation, only partly prevent arrhythmic events, and syncope and SCD still occurs in a substantial number of LQTS patients.
Within SILENCE-LQTS, the EJP RD consortium will investigate a novel, mechanism- targeted therapy, comprising pharmacological inhibition of the serum and glucocorticoid regulated kinase-1 (SGK1). In contrast to current symptom-directed therapies, this novel approach is designed to correct the pro-arrhythmic alterations in ion channel homeostasis caused by the underlying genetic defect. Efficacy of a SGK1 inhibitor, developed by LQT Therapeutics, will be systematically tested to establish the anti-arrhythmic potential of SGK1 inhibition, paving the way for future clinical application aimed at preventing symptoms and SCD in LQTS.
“Because current symptom-directed therapies aimed at reducing arrhythmia triggering events, including lifestyle changes, beta blockade, and left cardiac sympathetic denervation, only partly prevent (lethal) arrhythmic events, there remains a crucial unmet need for novel, more efficacious, mechanism-driven therapies,” said Arthur Wilde, MD, PhD, Amsterdam University Medical Centers and EJP RD Consortium Member.
LQT Therapeutics has shown that SGK1 inhibition in cardiomyocytes from patients with different LQT genetic mutations leads to shortening of the action potential duration. If this behavior translates in vivo, there is the potential to change the lives of patients with Congenital Long QT Syndrome, many of whom rely on medications that are not fully effective and have unpleasant side effects or implanted cardiac defibrillators.
“This award represents a significant milestone for LQT Therapeutics as we advance our platform of SGK1 inhibitors,” said Debra Odink, PhD, Chief Development Officer of LQT Therapeutics. “We are grateful to the EJP RD for this award and the opportunity of working with the consortium. Advancing therapeutics for people with Long QT Syndrome is a focus of LQT Therapeutics and we aim to restore normal life to those that live in fear of developing a potentially life-threatening arrhythmia.”
About LQT Therapeutics, Inc.
LQT Therapeutics, Inc. is pioneering a precision medicine approach to treat patients with Long QT Syndrome and other arrhythmias based on research from Beth Israel Deacons Medical Center, Massachusetts General Hospital, and Sanofi, S.A. By combining leading- edge cardiovascular genetics and diagnostics with recent advances in the understanding of the role of SGK1, LQT Therapeutics seeks to make a meaningful difference in the lives of people suffering from Long QT Syndrome and resistant cancers. Launched in 2019 by the Fonds de Solidarité FTQ. LQT Therapeutics, Inc. was founded by world-class experts in cardiovascular disease, cardiac muscle biology and drug development. For more information, please visit www.lqttrx.com
About the EJP RD
The European Joint Program on Rare Diseases (EJP RD) is a program aiming to create an effective rare diseases research ecosystem for progress, innovation and for the benefit of everyone with a rare disease. We support rare diseases stakeholders by funding research, bringing together data resources and tools, providing dedicated training courses, and translating high quality research into effective treatments. Consortium members include Arthur Wilde (Amsterdam University Medical Center), Lia Crotti (Istituto Auxologico Italiano), Katja Odening (University of Bern) and Lior Gepstein (Technion – Israel Institute of Technology), along with the patient advocacy organization, Hart4Onderzoek (Hart4Research) of the Netherlands.
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