Artax Biopharma achieves clinical validation of first-in-class, oral, Nck modulator AX-158 for autoimmune disease in phase 2a study

Novel breakthrough: first molecule to modulate the fundamental mechanism of T cell activation underlying autoimmune pathology

Statistically significant improvements in T cell and psoriasis-related biomarkers in line with PASI improvements in Phase 2a in patients with psoriasis

Phase 2a confirms well-tolerated safety profile, with no infections or discontinuations

CAMBRIDGE, Mass., Jan. 22, 2025 /PRNewswire/ -- Artax Biopharma, Inc., a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases, today announces achievement of clinical validation through its Phase 2a clinical trial in psoriasis with fully proprietary oral agent AX-158. These results confirm the strong Phase 1 and preclinical data package, underscoring the potential breadth and depth of Nck modulation, a completely novel mechanism in autoimmune disease.

AX-158 was evaluated in a Phase 2a trial in people with mild to moderate plaque psoriasis in multiple centers in the UK. Participants were randomized 2:1 to receive a single daily dose of 10mg AX-158 or placebo. A total of 30 participants were treated for 28 days and followed for an additional 30 days for safety. The main objectives of the study were safety and proof of mechanism.

The Phase 2a study confirmed the well-tolerated safety profile seen in the Phase 1 studies, with no infections reported and no severe adverse events. The few adverse events reported were mild or moderate in nature and resolved without intervention, and there were no trial discontinuations.

An extensive psoriasis biomarker analysis was designed, conducted, and analyzed by Dr. James Krueger, Head of Laboratory for Investigative Dermatology at the Rockefeller University and member of the Scientific Advisory Board at Artax Biopharma. His analysis demonstrates statistically significant and consistent effects on both disease and pathway-related genes in participants treated with AX-158. Dr. Kruger commented that "For a novel mechanism of action, it is exciting to see how it can begin to impact disease processes, and I look forward to seeing how the response expands over a longer treatment period in future trials."

CEO Rob Armstrong added: "Today's results validate this truly orthogonal, novel mechanism of action in the autoimmune space. We were thoroughly delighted to see that Nck modulation was able to impact relevant biomarkers as well as clinical measurements, particularly in the moderate patient population. We were pleased to observe that PASI 75 responses also progressed in line with biomarker data in this study. We would like to thank the investigative clinicians, their teams, and all participating patients for their valuable contribution to advancing this science. There is so much work left to do in the autoimmune space. With AX-158 and our portfolio of Nck modulators, we see great potential to address the treatment gap for patients." 

The company intends to report the results at an upcoming medical conference.

Given the broad effect of Nck modulation across all T helper cell populations (Th17, Th1/0 and Th2) in pre-clinical and animal models, the team is planning further studies in additional autoimmune diseases such as atopic dermatitis. Clinical results from these studies are anticipated by the end of 2026. 

About Artax Biopharma, Nck modulation, and AX-158
Artax Biopharma is a clinical-stage biotechnology company transforming the treatment of T Cell-driven autoimmune diseases. Artax's first-in-class oral small molecules aim to deliver immune system modulation without immunosuppression, potentially unlocking new treatment options as both monotherapy and in combination with other treatments.
We believe there is significant potential for Nck modulation to revolutionize treatment of T Cell-driven diseases. Immunomodulation maintains healthy control of the immune system, while addressing the underlying source of T Cell-driven diseases. Central to a well-functioning immune system is the T Cell Receptor (TCR). When TCR signaling becomes dysregulated, it causes T Cell-driven conditions, including autoimmune diseases. We believe the immunomodulation mechanism offered by our investigational agents holds broad potential to revolutionize how these T Cell-driven autoimmune diseases are addressed, while not impairing the ability of a patient's immune system to function properly.
AX-158, our lead Nck modulator, has shown strong, broad cytokine modulation as well as modulation of mixed lymphocyte reactions. Good data on therapeutic efficacy with AX-158 were observed in murine models of self-antigen activation (EAE), with a prolonged pharmacodynamic effect in EAE, suggesting durable immune modulation. AX-158 showed no immunosuppression in models of strong antigen stimulation. Studies with AX-158 showed substantial preclinical evidence of activity in the Th₂, Th₁₇, Th₁/Th₀ pathways, suggesting that applications could be quite broad across the autoimmune space.
Artax Biopharma is based in the Boston area and raised funding from investors including Advent Life Sciences, Sound Bioventures, The Termeer Family Office, the Fuhrer Family Office and Columbus Venture Partners. For more info, see www.artaxbiopharma.com or follow us on LinkedIn. 

For future clinical study inquiries: [email protected]  

SOURCE Artax Biopharma