Our Progress: Congenital Long QT Syndrome & Acquired (Drug-Induced) Long QT
Pre-Clinical Progress:
We have rapidly advanced our portfolio of SGK1 inhibitors to investigate their use as precision therapeutics in various genetic arrhythmias. Our progress to date includes:
Repeated positive in vitro effects on APD90 (a surrogate for QT interval) using our lead SGK1 inhibitor in cardiomyocytes derived from LQTS patient stem cells of all three major Long QT Syndrome Types 1, 2, and 3.
Repeated positive effects of our lead SGK1 inhibitor in models of drug-induced Long QT.
Clinical Progress:
Completion of pre-clinical toxicology studies for initiation of clinical studies in humans.
Completion of the Phase 1 healthy volunteer clinical study.
Wave Studies
Completion of the Wave I Part 1 clinical proof-of-efficacy study, demonstrating that LQT-1213 rapidly and meaningfully reduces QT interval in individuals with prolonged QT induced by dofetilide. Read More
Completion of the Wave I Part 2 clinical proof-of-efficacy study, demonstrating statistically significant and clinically meaningful reductions in QTcF in patients diagnosed with congenital Long QT Syndrome Types 2 and 3. These results validate the therapeutic potential of LQT-1213 to address the underlying cause of QT prolongation and support the initiation of pivotal studies in 2025. Read More
Initiation of the Wave I Part 3 clinical proof-of-efficacy study in patients diagnosed with Long QT Syndrome Type 1 expected in 2025.
MyQTWave is a non-interventional and observational study designed to better understand the symptoms and daily challenges faced by individuals living with Long QT Syndrome.
Initiation of the non-interventional MyQTWave study for Long QT Syndrome Types 2 and 3 expected in 2025.
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Congenital Long QT Syndrome is a rare genetic heart rhythm condition that lengthens the time for the heart to restore its electrical charge between beats. The QT interval is measured between the Q and T waves on an electrocardiogram (ECG or EKG). Prolongation of the QT interval increases an individual’s risk of developing an unexpected and life-threatening arrhythmia called “torsades de pointes”. People with Long QT Syndrome are at greatest risk to experience serious arrhythmias in response to exercise and stress.
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A prolonged QT interval can occur in response to acute or chronic use of certain prescription medications. This is known as Acquired or Drug-Induced Long QT. These medications are prescribed when their benefits outweigh the risks of QT prolongation, especially when limited treatment options are available. The risk of QT prolongation with certain medications increases with underlying risk factors, including congenital Long QT Syndrome and/or abnormalities in bodily electrolytes (such as low potassium or low magnesium). These situations must be carefully assessed and managed by a healthcare provider.
Our Progress: Heart Failure & Atrial Fibrillation
Pre-Clinical Progress:
Demonstration of the preventive effect of an SGK1 inhibitor in a model of high-fat diet-induced atrial fibrillation. Read More
Demonstration of the treatment and protective effect of SGK1 inhibition in two independent preclinical models of heart failure with preserved ejection fraction (HFpEF). The topline data from these studies were presented at the 2024 American Heart Association (AHA) Scientific Sessions.
Completion of IND/CTA-enabling preclinical studies.
Clinical Progress:
Completion of dosing in the Phase 1 healthy volunteer clinical study. Read More
Initiation of Phase 2 proof-of-concept clinical studies for THRV-1268 targeting patients with heart failure and patients with obesity-related paroxysmal atrial fibrillation, expected in 2025.
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Atrial fibrillation is a type of arrhythmia (irregular heart rhythm) which may present as a rapid, slow, or abnormal heart rate. These arrhythmias reduce the ability of the heart to efficiently to pump blood and may lead to blood clots localized in the heart which can dislodge and increase the risk of stroke, heart failure and other heart-related complications.
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Heart failure is a chronic condition in which the heart's ability to pump blood effectively is impaired, either due to reduced systolic, pumping function (HF with reduced ejection fraction; HFrEF) or with normal systolic function but impaired diastolic filling between beats (HF with preserved ejection fraction; HFpEF). This diminished cardiac performance leads to inadequate blood flow to meet the body's needs, causing symptoms such as fatigue, shortness of breath, reduced ability to exercise, and fluid retention. The most common risks associated with the development of heart failure include coronary artery disease, heart attack (myocardial infarction), high blood pressure, obesity, smoking, excessive alcohol, physical inactivity, family history and genetic disorders.