QurAlis Reports Early Clinical Evidence of Kv7.2/7.3 Target Engagement in ALS

QurAlis has announced topline findings from its Phase I proof-of-mechanism study of QRL-101 in patients with amyotrophic lateral sclerosis (ALS), marking the first demonstration of target engagement of a selective Kv7.2/7.3 potassium channel activator in this disease. Kv7.2/7.3 channels play a critical role in controlling neuronal excitability and membrane potential, processes that are disrupted in ALS.

QRL-101 is being developed to address neuronal hyperexcitability, a pathological feature observed in both genetic and sporadic ALS and commonly associated with mis-splicing of the KCNQ2 gene. The single-dose, randomized, placebo-controlled Phase I QRL-101-04 trial enrolled 12 ALS patients across three dose levels, with participants assigned in a 3:1 ratio to QRL-101 or placebo.

The study primarily evaluated safety, tolerability, and pharmacokinetic–pharmacodynamic relationships and was not designed to assess efficacy. QRL-101 was well tolerated, with no serious adverse events reported. Biomarker data indicated target engagement linked to ALS survival prediction, supporting the therapeutic rationale.

30-12-2025