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

/EIN News/ -- PARIS, Feb. 18, 2025 (GLOBE NEWSWIRE) -- 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

Media Contacts

AlgoTx
CEO Stéphane Thiroloix
stephane@algotx.com

Scius Communications
Katja Stout
Katja@sciuscommunications.com
+44 778 943 5990

Daniel Gooch
daniel@sciuscommunications.com
+44 774 787 5479

About AlgoTherapeutix (AlgoTx)

AlgoTherapeutix (AlgoTx) is a clinical stage biotechnology company developing a targeted, first in class therapeutic for peripheral neuropathic pain. Its lead asset, ATX01, is a proprietary topical formulation of amitriptyline for use in chemotherapy-induced peripheral neuropathy (CIPN), an area of high unmet need associated with unbearable pain in patients’ extremities and for which there is currently no viable treatment options.

ATX01 targets the signaling pathways involved in the regulation of pain by inhibiting specific nociceptive sodium channel receptors. Its topical administration has been designed to provide targeted relief from the excruciating pain in patients’ extremities from CIPN, which is caused by nerve degeneration and is a common side effect of cancer treatment, occurring in 68% of patients. ATX01 was granted FDA fast track development status in CIPN.

The company has completed recruitment and supporting analysis of data of its international Phase 2 clinical trial of 276 patients with CIPN (the ‘ACT’ study).

The company has seasoned leadership, with experience of bringing global products successfully to market, and is backed by an international board. AlgoTx raised a Series B round of EUR 20 million in 2023 and has raised EUR 35 million to date. AlgoTx has a consortium of investors including Relyens Innovation Santé / Turenne Capital, who led the Series B fund raise, along with UI Investissement, and angel investors. Historical investors include Bpifrance through its Innobio 2 fund and Omnes Capital.

For more information, see the company’s website and follow AlgoTx on LinkedIn.