AlgoTx Announces Results of its Phase 2 “ACT” Trial of ATX01 in Chemotherapy-Induced Peripheral Neuropathy

Paris, France, 18 February 2025: AlgoTherapeutix (AlgoTx), a clinical stage biotechnology company developing a first-in-class therapeutic for peripheral neuropathic pain, ATX01, announced today the completion of its 276 patient Phase 2 trial known as ‘ACT’ (ATX01 in ChemoTherapy-induced peripheral neuropathy).

Conducted under the guidance of Professor Guido Cavaletti (University of Milano-Bicocca, Italy), ACT is an international, double-blind, placebo-controlled evaluation of the efficacy and safety of two concentrations of ATX01 in patients suffering from CIPN, a common side effect of neurotoxic cancer treatments caused by nerve degeneration (1).

The company’s high-concentration, non-opioid ATX01 selectively targets specific nociceptive sodium channels in the peripheral nervous system involved in pain signaling. Its twice-daily local application is designed to alleviate pain whilst minimizing toxicity through limited systemic exposure. 

ACT provided encouraging signals of ATX01 efficacy, particularly at the higher concentration of 15%. However, the high level of placebo effect experienced in several study sites prevented the overall separation of the pain response curves for ATX01 and placebo. In a post-hoc analysis in low-placebo sites, ATX01 15% showed statistically significant superiority to placebo on most primary and secondary endpoints. ATX01 was well tolerated.

Equipped with this supporting guidance on future study design, AlgoTx sees a strong rationale to further explore the development of ATX01 15% given:

• With an impact on baseline pain of 1.6 points on the NPRS*, the efficacy of ATX01 15% was of relevant magnitude for clinical efficacy.
• Per protocol and post-hoc analyses provide clinical insights on patient profiling, choice of endpoints, site variability and placebo effect.
• Trial designs can be considered to better mitigate the placebo effect, such as placebo run-in, and enrichment/withdrawal strategies.

AlgoTx’s Founder, President & CEO, Stéphane Thiroloix said, “The ACT study results are a significant step forward on a complex development pathway and we thank all the investigators across US and Europe for their great work. Whilst 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. Hence there is the potential to design a new study, with additional mitigation strategies in place, to evaluate ATX01’s efficacy to alleviate pain in CIPN without ambiguity. There remains a very high unmet need for pain relief in approximately two thirds of patients treated for cancer.”

*Numerical Pain Rating Scale

Reference:

(1) “Efficacy and Safety of ATX01 in Adult Patients With CIPN (Chemotherapy-induced Peripheral Neuropathy) (ACT)”. The purpose of this clinical trial is to compare the efficacy of twice daily applications of ATX01 (10% & 15%) versus placebo during a 12-week treatment period in treating CIPN in adult cancer survivor patients. Full details here: link