The trial evaluated the initial tolerability, safety and impact on clinical outcomes and neurodegeneration biomarkers of BRAVYL in amyotrophic lateral sclerosis (ALS) patients. Credit: Kateryna Kon/Shutterstock.

Woolsey Pharmaceuticals has announced encouraging results from its Phase IIa study of BRAVYL, revealing a substantial decline in neurofilament light chain (NfL) levels and potential clinical benefits for patients with ALS.

Dubbed Rho kinasE inhibition in Amyotrophic Lateral Sclerosis (REAL), the open-label, single-arm trial evaluated the initial tolerability, safety and impact on clinical outcomes and neurodegeneration biomarkers of BRAVYL in ALS patients.

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It enrolled 31 subjects in the initial patient cohort, 25 of whom were receiving the 24-week course of treatment at a dose of 180mg per day. Of these subjects, 20 were part of the study’s first extension phase.

The primary safety endpoint was met, with BRAVYL proving safe and well-tolerated without any drug-associated adverse events leading to participant withdrawal.

Furthermore, no unexpected safety findings have emerged, with some patients receiving treatment for up to 18 months.

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A significant finding of the study was BRAVYL’s ability to decrease NfL, a marker of neuronal axon damage, by 15% after 24 weeks of treatment, which is a substantial improvement considering that NfL levels typically increase by an average of 11% over six months in ALS patients.

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This data suggests that BRAVYL could potentially reduce NfL by up to 26% compared with a placebo group over the same duration.

Furthermore, larger reductions in NfL correlated with less deterioration in the ALS Functional Rating Scale-Revised (ALSFRS-R) scores, indicating that the clinical benefits of reducing NfL may be observed concurrently with the reductions.

Given the 180mg/day dose’s safety and tolerability, the REAL study is expanding to screen and potentially enrol up to 15 subjects at a 300mg/day dose.

These participants will undergo treatment for 24 weeks, with the option to continue in an extension treatment phase.