QurAlis has announced topline results from its Phase I proof-of-mechanism clinical trial of QRL-101 in patients with amyotrophic lateral sclerosis (ALS).
The data provide the first evidence of target engagement with a selective Kv7.2/7.3 ion channel opener in these patients.
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Kv7.2/7.3 channels are voltage-gated potassium channels involved in regulating membrane potential and neuronal excitability.
QRL-101 is a selective activator of Kv7.2/7.3 channels in development for addressing disease progression in ALS associated with neuronal hyperexcitability.
This condition affects both genetic and sporadic ALS cases and is often linked to mis-splicing of the KCNQ2 gene in pre-messenger RNA (mRNA). QurAlis is also studying QRL-101 in developmental and epileptic encephalopathies (DEE).
The therapy’s tolerability, safety, and pharmacokinetic (PK) profile matched earlier studies. No serious adverse events or discontinuations due to adverse events occurred.
QRL-101-04 was a Phase I single-dose, placebo-controlled trial assessing tolerability and safety in ALS patients.
The trial enrolled 12 ALS patients and assessed three dose levels, randomised 3:1 between QRL-101 and placebo.
Its primary endpoint was exploration of the PK and pharmacodynamic relationship, with the trial not powered for statistically significant efficacy.
QurAlis CEO and co-founder Kasper Roet said: “This is the first time we are seeing target engagement of QRL-101 in ALS patients with a biomarker which predicts survival in ALS.
“This is extremely encouraging as new research published in Nature Neuroscience provides additional evidence that loss of TDP-43 function drives mis-splicing of the KCNQ2 potassium channel, producing a dysfunctional isoform that disrupts neuronal excitability in ALS and frontotemporal dementia, further validating this disease mechanism.”
In June 2023, QurAlis received clinical trial authorisation (CTA) in the EU to initiate a first-in-human, Phase I ANQUR clinical trial of QRL-201 for the treatment of ALS.
