Verge Genomics has completed its Phase I clinical trial of VRG50635, a small molecule PIKfyve inhibitor for treating all forms of amyotrophic lateral sclerosis (ALS), in healthy adult volunteers.

The single and multiple ascending dose escalation, placebo-controlled, double-blind, randomised study has evaluated the tolerability and safety of VRG50635 in 80 healthy adult volunteers.

It also assessed the pharmacodynamics and pharmacokinetics of the therapy.

After both single and repeated administration, VRG50635 was found to be safe and well tolerated in the study.

Favourable pharmacokinetics of VRG50635 was observed with an increase in Cmax and AUC and a terminal half-life of around 37 hours for the active metabolite thereby supporting once-daily oral dosing.

No serious adverse events were reported in the trial.

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Verge Genomics chief medical officer Diego Cadavid said: “We are extremely encouraged by these initial results which highlight the favourable safety and tolerability profile of VRG50635, and further validate the novel approach that Verge has taken to identify novel targets for complex diseases with high unmet need using human disease tissues and rapidly advancing compounds into proof-of-concept trials.

“We look forward to sharing additional information at the ENCALS meeting in Barcelona next month and anticipate progressing to a proof-of-concept study in patients with sporadic and familial ALS later this year.”

Verge is planning to initiate a proof-of-concept (PoC) study in ALS patients in the fourth quarter of this year.

Verge Genomics CEO and co-founder Alice Zhang said: “Our innovative proof-of-concept ALS study is designed to overcome historical challenges in ALS clinical trials by using state-of-the-art technology such as digital at-home devices and blood-based biomarkers that can capture richer, higher-fidelity patient data and have the potential to detect efficacy with greater sensitivity.”