AlgoTx’s ATX01 Shows Promise Despite Missing Primary Endpoint in Phase 2 CIPN Trial

AlgoTx completed a 276-patient Phase 2 trial (ACT) for ATX01, a topical amitriptyline formulation targeting chemotherapy-induced peripheral neuropathy (CIPN)29.

The trial failed to meet its primary endpoint due to a high placebo effect in several study sites26.

ATX01 showed encouraging efficacy signals, particularly at the higher 15% concentration, with a 1.6-point improvement on the Numerical Pain Rating Scale29.

In a post-hoc analysis of low-placebo sites, ATX01 15% demonstrated statistically significant superiority to placebo on most primary and secondary endpoints29.

ATX01 was well-tolerated, with a favorable safety profile29.

AlgoTx plans to explore further development of ATX01 15%, considering alternative trial designs to mitigate the placebo effect29.

CIPN affects approximately two-thirds of chemotherapy patients and currently has no approved treatments12.

The trial was conducted across over 40 sites in the US and Europe, under the guidance of Professor Guido Cavaletti from the University of Milano-Bicocca12.

AlgoTx's approach involves targeting specific nociceptive sodium channels in the peripheral nervous system to alleviate pain while minimizing systemic exposure29.

The company views these results as a significant step forward in addressing the unmet need for pain relief in cancer survivors experiencing CIPN29.

Sources:

1. https://pharmatimes.com/news/algotx-completes-pivotal-phase-2-trial-for-ground-breaking-cipn-treatment/

2. https://www.biospace.com/press-releases/algotx-announces-results-of-its-phase-2-act-trial-of-atx01-in-chemotherapy-induced-peripheral-neuropathy

6. https://www.biospace.com/drug-development/algotx-stumbles-in-non-opioid-pain-trial-following-vertexs-journavx-approval

9. https://www.globenewswire.com/news-release/2025/02/18/3027514/0/en/AlgoTx-Announces-Results-of-its-Phase-2-ACT-Trial-of-ATX01-in-Chemotherapy-Induced-Peripheral-Neuropathy.html