QurAlis Corporation, a clinical-stage biotechnology company focused on precision medicines for neurodegenerative diseases, has completed dosing of the first participant cohort in its Phase 1 multiple-ascending dose (MAD) clinical trial of QRL-101 (QRL-101-03; NCT06532396). This trial aims to evaluate the safety, tolerability, and pharmacokinetics of QRL-101 in healthy adult volunteers. QRL-101 is a first-in-class selective Kv7.2/7.3 ion channel opener being developed for the treatment of hyperexcitability-induced disease progression in amyotrophic lateral sclerosis (ALS).
Trial Design and Objectives
The Phase 1 MAD trial is a randomized, double-blind, placebo-controlled study conducted at a single site. It is expected to enroll approximately 60 participants, randomized in a 9:3 ratio of QRL-101 to placebo across five planned cohorts. The dose range for this MAD study was determined based on the results of QurAlis' previous Phase 1 single-ascending dose (SAD) clinical trial (QRL-101-01; NCT05667779).
Prior Safety Data
The Phase 1 single-ascending dose (SAD) clinical trial enrolled 88 healthy participants. Results from the SAD trial indicated that QRL-101 was well-tolerated, with no significant safety concerns or serious adverse events reported.
Mechanism of Action and Rationale
QRL-101 targets motor system hyperexcitability, a condition present in approximately 50% of all ALS patients, which is linked to potassium channel dysfunction. Leonard H. van den Berg, M.D., Ph.D., professor of neurology and chair, TRICALS, noted that QRL-101 is a highly selective Kv7.2/7.3 ion channel opener that, in preclinical models, has demonstrated strong potential to control motor neuron hyperexcitability-induced neurodegeneration with an attractive side effect profile.
Clinical Significance
ALS is a progressive neurodegenerative disease affecting nerve cells in the brain and spinal cord, leading to muscle function loss. The average life expectancy for ALS patients is approximately three years post-diagnosis, and there is currently no cure. Doug Williamson, M.D., chief medical officer of QurAlis, stated that QRL-101 has the potential to be a first-in-class effective therapy for ALS patients suffering from hyperexcitability-induced motor neuron degeneration.
Anticipated Timeline
QurAlis anticipates reporting topline data from the Phase 1 MAD clinical trial of QRL-101 in the first half of 2025. These results are expected to support larger global studies in people living with ALS. More information about the QRL-101 Phase 1 clinical trials can be found at www.clinicaltrials.gov.
