AlgoTx Announces First Patients Dosed in US and European Arms of Phase II Clinical Trial of ATX01 in Rare Disease Erythromelalgia

Announces successful recruitment of half of the patients in its ongoing three-arm 240-patient phase II trial in chemotherapy-induced peripheral neuropathy

Paris, France, 12 Sept: AlgoTherapeutix (AlgoTx), a clinical stage biotechnology company developing targeted, first-in-class therapeutics for peripheral neuropathic pain, announces it has dosed the first patients of up to 14, in the US and European centers of its phase II proof of concept trial, called EASE*. Under Orphan Drug Designation from FDA and EMA, this trial investigates the efficacy of the company’s lead asset ATX01 on the pain of Erythromelalgia, a rare neurological and vascular disease characterized by painful extremities for which there is no available treatment. The trial is due to read out in H1 2024.

The company’s innovative approach is focused on targeting specific nociceptive sodium channels involved in pain signaling through using a topical administration acting locally at the root of the pain and at a dose high enough to alleviate the extreme pain.

AlgoTx’s Founder, President & CEO, Stéphane Thiroloix said, “We’re delighted with the solid progress of our clinical trials and commend the clinical teams in the US and Germany for their dedication and progress towards this major milestone. We appreciate the dire need of Erythromelalgia patients for pain relief and are focused on providing them with our targeted therapeutics.”

The Universitaetsklinikum Erlangen Hautklinik’s Dr Juergen Bauerschmitz said, “The painful « flare-ups » of erythromelalgia can significantly impact quality of life. There is a high unmet medical need for this rare condition, which we often see in our dermatology clinic, which is one of the leading dermatology centers in Germany. I am delighted to be leading the European part of the phase II clinical trial of ATX01.”

The Mayo Clinic’s Julio Sartori Valinotti MD is involved in the research in the U.S.

The company also announces that it has completed, on schedule, the recruitment of approximately half of the patients in its three-arm 240-patient phase II trial (ACT) in chemotherapy-induced peripheral neuropathy (CIPN), a common side effect of neurotoxic cancer treatments caused by nerve degeneration.

AlgoTx’s innovative approach is focused on targeting specific nociceptive sodium channels in the peripheral nervous system involved in pain signaling through the administration of ATX01 as a twice daily topical hydrogel, acting locally and at a dose high enough to alleviate the extreme pain whilst minimizing toxicity through limited systemic exposure. The patient’s pain is reduced to a bearable level over time. In patients chronically affected by CIPN after having completed their chemotherapy, ATX01 may be a drug of choice for alleviating their pain and improving their quality of life.

Erythromelalgia affects around 1-2 per 100,000 people. There are around 5,000 EM patients in the US and around 7,000 in Europe. This condition primarily affects the feet, and less commonly, the hands, arms, legs, ears, and face. The condition can have ‘flare ups’ that last from minutes to days or be a continuous chronic condition. It is characterized by intense, burning pain, increases in skin temperature, swelling and severe redness (erythema) of the affected extremities.

* EASE stands for “Efficacy of ATX01 Study in Erythromelalgia”. The trial’s official title is “A Randomized, Double-blind, Placebo-controlled, 2-period, Crossover Study to Evaluate the Efficacy and Safety of ATX01 (Topical Amitriptyline Hydrochloride 15% w/w) in Adult Patients With Pain Due to Erythromelalgia (EM)”. Full details are here: https://clinicaltrials.gov/study/NCT05917912?cond=erythomelagia&rank=1