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:

AlgoTx Progresses ATX01 in Two Indications

  • First patients enrolled in ACT study in Chemotherapy-Induced Peripheral Neuropathy
  • IND opened and Fast Track Designation granted by FDA for Erythromelalgia

Suresnes, France, March 30th 2023.

European biotech AlgoTx, a clinical stage innovator in complex pain, today announces that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for its Phase 2 first-in-class candidate ATX01 in the treatment of erythromelalgia. Meanwhile, the first patients have been randomized into the “ATX01 for the pain of ChemoTherapy” (ACT) study in Chemotherapy-Induced Peripheral Neuropathy (CIPN). Both programs have been granted Fast Track Designation by the FDA.

AlgoTx is conducting the ACT study as part of its US / EU Phase 2 program in the indication of CIPN. The randomized, double-blind, placebo-controlled study aims to recruit 240 patients in more than 40 sites in the US, Belgium, Czech Republic, France, Italy, Poland and Spain.

Separately, FDA clearance of the IND for ATX01 in the indication of erythromelalgia opens the way for the Phase 2 “Efficacy of ATX01 Study in Erythromelalgia” (EASE) trial, a randomized, double-blind, placebo-controlled, cross-over study that will take place at the Mayo Clinic in the US and the Erlangen University Hospital in Germany. The study has also been approved by the German regulatory agency, BfArM.

Erythromelalgia is a rare neurovascular disease involving frequent episodes of swelling, redness, and often intense burning pain in the feet and/or hands of affected individuals. Episodes can be spontaneous or triggered by heat or exercise, and there is currently no cure for the condition nor any approved or consistently effective treatment available. AlgoTx has obtained an Orphan Disease Designation from both FDA and EMA to support development of ATX01 in this indication.

Dr Gabriel Fox, Chief Medical Officer of AlgoTx, commented: “This IND is further validation of our development work to date, and we are now eager to explore ATX01’s potential in this painful and distressing rare disease with the outstanding teams at the Mayo Clinic and Erlangen University Hospital.”

ATX01 is designed to inhibit pain messaging by directly targeting small sensory nerve fibers in the skin which generate and conduct pain messages from the peripheral nervous system to the spinal cord and brain. ATX01 acts locally to inhibit the nociceptive activity of voltage-gated sodium-channels Nav1.7, Nav1.8 and Nav1.9, in particular in peripheral sensory nerve fibers A∂ and C. Its systemic passage is minimal, enabling a favorable tolerance profile.

“With the ACT study in its active phase and the EASE study now on the launch pad, we look forward to bringing hope to those patients in extreme need of relief,  thanks to valuable support from our CRO partner Worldwide Clinical Trials, from the investigators and most importantly from the patients entering the study” said Dr Philippe Picaut, AlgoTx’s Chief Development Officer.

AlgoTx wins France Biotech’s 2023 ESG HealthTech Trophy

Paris, March 27th 2023. At France Biotech’s prestigious HealthTech Trophies Ceremony, AlgoTx was awarded the 2023 ESG HealthTech Trophy in recognition of the young biotech’s early commitment to ESG initiatives. On receiving the award on behalf of the AlgoTx team, Stéphane Thiroloix, CEO, commented that a small company can actually find it easier than a large one to define a course of action for ESG and execute consistently in that respect. He saluted the great work done by the NGO’s AlgoTx has been supporting : The Plastic Bank and Gribouilli.