AlgoTx Completes Phase II Trial of ATX01 for Chemotherapy-Induced Peripheral Neuropathy

Key Insights

• AlgoTx has announced the completion of the Last Patient Last Visit (LPLV) in its Phase II clinical trial, ACT, evaluating ATX01 for chemotherapy-induced peripheral neuropathy (CIPN).
• The international, double-blind, placebo-controlled trial included 276 patients across more than 40 sites in multiple countries.
• ATX01, a topical formulation of amitriptyline, targets nociceptive sodium channels to alleviate pain associated with CIPN, which currently lacks approved treatments.

AlgoTherapeutix (AlgoTx) has reached a significant milestone in its clinical development program for ATX01, a novel therapeutic for chemotherapy-induced peripheral neuropathy (CIPN). The company announced the completion of the Last Patient Last Visit (LPLV) in its international Phase II clinical trial, known as 'ACT (ATX01 in ChemoTherapy-induced peripheral neuropathy)'. This double-blind, placebo-controlled study, conducted across more than 40 sites in the US, Belgium, Czechia, France, Italy, Poland, and Spain, enrolled 276 patients suffering from CIPN.

CIPN is a common and debilitating side effect of neurotoxic cancer treatments, affecting up to two-thirds of chemotherapy patients. It results from nerve degeneration and causes severe pain in the extremities. Currently, there are no approved treatments for CIPN, representing a significant unmet medical need.

ATX01 is a high-concentration, non-opioid topical formulation of amitriptyline designed to selectively target specific nociceptive sodium channels in the peripheral nervous system involved in pain signaling. The twice-daily local application aims to deliver sufficient drug concentrations to alleviate pain while minimizing systemic exposure and potential toxicity.

"We’re delighted with the solid progress of our ACT clinical trial and commend the patients, investigators and AlgoTx clinical team for their dedication in achieving this major milestone," said Stéphane Thiroloix, AlgoTx’s Founder, President & CEO. Philippe Picaut, AlgoTx’s Chief Development Officer, added, "CIPN affects two thirds of chemotherapy patients, representing a high unmet need without any approved treatments. Any progress in this field is therefore very gratifying. We look forward to sharing the ACT data and progressing to the next stage."

The ACT trial is evaluating the efficacy and safety of two concentrations of ATX01 (10% and 15%) compared to placebo over a 12-week treatment period in adult cancer survivors with CIPN. The company anticipates releasing the final data from the Phase II trial in early 2025 and is already planning to expedite a Phase 3 trial.