AlgoTherapeutix (AlgoTx) has announced results from its completed Phase 2 'ACT' trial evaluating ATX01, a first-in-class therapeutic for chemotherapy-induced peripheral neuropathy (CIPN). The international, double-blind, placebo-controlled study involved 276 patients and tested two concentrations of the novel topical treatment.
Clinical Efficacy and Trial Outcomes
The higher concentration formulation of ATX01 (15%) showed encouraging efficacy signals in treating CIPN, achieving a 1.6-point reduction in baseline pain on the Numerical Pain Rating Scale (NPRS). However, the trial's primary endpoint was not met due to an unexpectedly high placebo response in several study sites, which masked the treatment's overall effect.
Post-hoc analysis of data from sites with lower placebo responses revealed statistically significant superiority of ATX01 15% compared to placebo across most primary and secondary endpoints. The drug demonstrated a favorable safety profile throughout the study.
Innovative Therapeutic Approach
ATX01 represents a novel approach to treating CIPN, utilizing a high-concentration, non-opioid formulation that selectively targets specific nociceptive sodium channels in the peripheral nervous system. The treatment is administered topically twice daily, designed to maximize pain relief while minimizing systemic exposure and associated toxicity.
Clinical Need and Market Potential
CIPN affects approximately 68% of cancer patients receiving treatment, representing a significant unmet medical need. The condition, caused by nerve degeneration due to neurotoxic cancer treatments, currently lacks viable treatment options for the debilitating pain it causes in patients' extremities.
Future Development Plans
Stéphane Thiroloix, AlgoTx's Founder, President & CEO, commented on the results: "The ACT study results are a significant step forward on a complex development pathway. While we have seen encouraging signs of clinical efficacy with ATX01, we did not hit our primary endpoint due to a strong placebo effect in some centers."
The company has identified several strategies to optimize future trials, including:
- Implementation of placebo run-in periods
- Adoption of enrichment and withdrawal strategies
- Refined patient profiling
- Modified endpoint selection
- Better control of site variability
Regulatory and Development Status
ATX01 has received FDA fast track development status for CIPN, highlighting the treatment's potential importance in addressing this significant medical need. The company's development program has been strengthened by insights gained from the Phase 2 trial, providing valuable guidance for future study design.
