Positive Top-Line Phase 1 MEND Results for MND/ALS Treatment by PharmAust

PharmAust Limited, a biotech firm, has reported that its drug monepantel (MPL) has outperformed the FDA-approved Relyvrio® in terms of safety and tolerability. In a Phase 1 clinical trial known as MEND, monepantel showed promising preliminary results, with a 58% reduction in disease progression rate in patients with Motor Neurone Disease (MND) or Amyotrophic Lateral Sclerosis (ALS), as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). The drug was well-tolerated by all participants, with no dose-limiting toxicities or treatment-related deaths observed.
The study was designed to assess the safety, tolerability, and pharmacokinetics of MPL in MND/ALS patients through a single-dose and a 4-week repeated dose regimen. The trial involved 12 patients divided into two cohorts, with Cohort 2 receiving higher dosages. The higher dose demonstrated a dose-response effect, suggesting the potential for slowing disease progression.
In addition to the primary efficacy measure ALSFRS-R, the study also evaluated other clinical efficacy markers, including quality of life and cognitive assessments. The results indicated that monepantel could potentially extend the median survival of patients by 13.5 to 56.5 months, a significant improvement over current treatments that extend life expectancy by 2 to 9 months.
Pharmacokinetic findings showed that MPL and its active metabolite, MPL sulfone, were present in cerebrospinal fluid, indicating their ability to cross the blood-brain barrier. The drug also engaged the mTOR pathway, a key cellular process involved in neurodegenerative diseases.
The positive outcomes of the MEND study have led to the identification of an optimal dose for a pivotal Phase 2/3 clinical trial, which is scheduled to begin in the third quarter of 2024. PharmAust's CEO, Dr. Michael Thurn, expressed excitement about the results and the potential impact on patients with MND/ALS. The company also acknowledged the support of FightMND, a leading organization dedicated to finding a cure for the disease.
PharmAust is focused on developing therapeutics for neurodegenerative diseases and cancer in animals, with monepantel as its leading program for MND/ALS treatment. The company anticipates initiating a pivotal field trial for canine B-Cell Lymphoma and aims for accelerated approval from the US Food and Drug Administration in the future.

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