The treatment aims to reduce prolonged QTc interval in patients with long QT syndrome.
An orphan drug designation (ODD) has been granted by the FDA to the novel, first-in-class serum glucocorticoid regulated kinase 1 (SGK1) inhibitor LQT-1213 for the treatment of patients with long QT syndrome (LQTS), according to a news release from Thryv Therapeutics.1
LQTS is a heart rhythm disorder that affects electrical signals that travel to the heart and cause it to beat erratically. The disorder can lead to sudden fainting and seizures with little to no warning. Young people with LQTS are saddled with a higher risk of sudden cardiac death.2
There are 2 types of LQTS: congenital or acquired. Congenital LQTS comprises an array of rare orphan diseases where patients are genetically predisposed to chronic prolongation of their QTc interval. Patients with acquired LQTS can develop the condition from the administration of treatments that block electrical pathways in the heart, leading to a similar prolongation of QT.3
“People with long QT syndrome deserve a properly studied and FDA-approved therapy to help in their battle against this potentially lethal genetic disease,” Debra Odink, PhD, president and chief development officer of Thryv Therapeutics, said in the news release. “This designation reinforces the potential of LQT-1213 to fulfill this unmet need and provides critical incentives for Thryv to accelerate our efforts to deploy prospectively designed, pivotal efficacy studies in people with congenital Long QT Syndrome.”1
This regulatory action is based on clinical data from the ongoing proof-of-concept WAVE 1 (NCT05906732) trial. Earlier this year, Thryv Therapeutics presented positive results from part 1 of this trial, which evaluated LQT-1213 for the reduction of QTcF in participants with dofetilide-induced LQTS.3,4
Following 2 days of treatment with dofetilide, which is an agent known to prolong QTc, individuals with meaningful increases in their QTc interval were treated with 6 days of increasing LQT-1213 doses, with a consistent dose of dofetilide. The investigators consistently monitored and received regular electrocardiograms to assess patient safety and treatment efficacy.3
Trial Name: Study of LQT-1213 on QTc-induced Prolongation in Healthy Adult Subjects (Part1) and on Congenital Long QT in Patients Diagnosed With Type 1, 2 or 3 Long QT Syndrome (Part 2)
ClinicalTrials.gov ID: NCT05906732
Sponsor: Thryv Therapeutics, Inc
Estimated Completion Date: February 28, 2025
In the trial, individuals who experienced larger dofetilide-induced prolongation of QTcF and were treated with LQT-1213 had more robust, clinically meaningful reductions of QT that was consistent with available scientific evidence. Importantly, the treatment was well-tolerated, with no serious adverse events or treatment-related trial discontinuations, according to Thryv.3
ODDs are granted by the FDA to drugs and biologics that target rare diseases, which are defined as those that affect fewer than 200,000 people in the United States. Benefits offered to support the development of LQT-1213 because of its ODD include tax credits for clinical trial expenses, exemption from certain FDA fees, and up to 7 years of market exclusivity after regulatory approval.1
“We remain focused on bringing innovative treatments to adults and children with rare diseases who have limited options,” Odink said. Thryv Therapeutics develops multiple SGK1 inhibitors for the treatment of cardiometabolic stress associated with arrhythmic diseases.1
