NCK

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, MA, USA I January 22, 2025 I 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 Th 2 , Th 17 , Th 1 /Th 0 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: contact@artaxbiopharma.com SOURCE: Artax Biopharma

According to the National Instituye of Arthritis and Musculoskeletal and Skin Diseases, psoriasis is a chronic, long-lasting disease in which the immune system becomes overactive, causing skin cells to multiply too quickly. Patches of skin become scaly and inflamed, most often on the scalp, elbows, or knees, but other parts of the body can be affected as well. It is thought the disease currently affects 125 million people globally, with an estimated 7.5 million adults in the US impacted by the condition. “We are excited about AX-158’s potential as the first immunomodulator in the Nck blocker class. Our pre-clinical data supports the potential for AX-158 to realize effective outcomes without the immunosuppression and the side effects associated with existing autoimmune disease therapies, said Artax CEO Rob Armstrong. Immunomodulation assists the immune system in maintaining healthy control, addressing the underlying driver of autoimmune diseases. As the first in a new class of Nck blockers, AX-158 has the potential to establish a new standard of care in autoimmune disease treatment, Artax said. By selectively targeting Nck function, which plays a critical role in immune system function, AX-158 recalibrates the body’s T-cell receptor (TCR) responses. According to the company, AX-158 has ‘broad potential’ to treat many autoimmune diseases without causing immunosuppression – as the therapy allows the immune system to only activate when it recognizes a true disease threat. googletag.cmd.push(function () { googletag.display('text-ad1'); }); “The new Nck blocker class of agents, AX-158, has the potential to change how we treat autoimmune disease through targeting and tempering TCR responses so the body only, and properly, reacts against strong pathogens,” commented James Krueger, head of the laboratory for investigative dermatology at The Rockefeller University and Artax board member. “I am excited to see how this new mechanism of action translates into biomarker changes and patient responses, where to date only targeted immunosuppression has been applied.” The company said it is ‘eager’ to see positive validation for AX-158 when the trial results are available later this year. The symptoms can sometimes go through cycles, flaring for a few weeks or months followed by periods when they subside or go into remission. Mild psoriasis can often be successfully treated with creams or ointments, while moderate and severe psoriasis may currently require pills, injections, or light treatments. Managing common triggers, such as stress and skin injuries, can also help keep the symptoms under control.

Safety and Efficacy Results Are Expected in Second Half of 2024 Evaluating AX-158, a First in Class Nck Blocker, Shifting the Paradigm of Autoimmune Disease Treatment AX-158 Has Broad Potential to Treat Many Autoimmune Diseases without Causing Immunosuppression Autoimmune Diseases Remain a Significant Unmet Medical Need, Affecting Millions of Patients Globally CAMBRIDGE, Mass., Feb. 13, 2024 /PRNewswire/ -- Artax Biopharma, Inc., a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases, today announces that the first patient has been dosed in the Company's Phase 2a trial evaluating the safety and biomarker responses of AX-158 in a first proof of mechanism trial in psoriasis. The Company expects these results to be available in the second half of this year. "We are excited about AX-158's potential as the first immunomodulator in the Nck blocker class. Our pre-clinical data supports the potential for AX-158 to realize effective outcomes without the immunosuppression and the side effects associated with existing autoimmune disease therapies," stated Artax Chief Executive Officer, Rob Armstrong, Ph.D. "We are eager to see this validation when the results from this psoriasis Phase 2a trial are available later this year." Immunomodulation assists the immune system in maintaining healthy control, addressing the underlying driver of autoimmune diseases. As the first in a new class of Nck blockers, AX-158 has the potential to establish a new standard of care in autoimmune disease treatment. By selectively targeting Nck function, which plays a critical role in immune system function, AX-158 recalibrates the body's T-cell receptor (TCR) responses. This recalibration to responses allows the immune system to continue functioning properly and activate only when it recognizes true disease threats – preventing self-activation without causing immune suppression that makes the body susceptible to numerous infections. "The new Nck blocker class of agents, AX-158, has the potential to change how we treat autoimmune disease through targeting and tempering TCR responses so the body only, and properly, reacts against strong pathogens," commented James G. Krueger, M.D., Ph.D., Head of the Laboratory for Investigative Dermatology at The Rockefeller University and Artax Scientific Advisory Board member. "I am excited to see how this new mechanism of action translates into biomarker changes and patient responses, where to date only targeted immunosuppression has been applied." About Autoimmune Disease and Psoriasis Psoriasis is a common, immune-mediated disease that affects 125 million people globally. In the US 3.0% of the adult population, or more than 7.5 million adults, are affected. Psoriasis patients may exhibit visible signs of inflammation anywhere on the body as raised plaques and scales on the skin. Plaques may appear as few small patches or can affect large areas. It is possible to have psoriasis plaques and scales on more than one location on the body at a time. Treating psoriasis can help improve symptoms as well as lower the risk of developing other health conditions such as psoriatic arthritis, heart disease, obesity, diabetes, and depression. About Artax Biopharma 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. The lead program AX-158 will deliver Phase 2a Proof of Concept data in psoriasis later in 2024. We believe there is significant potential for its approach to revolutionize treatment of T Cell-driven diseases. Immunomodulation maintains healthy control of the immune system and addresses 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, and induced T Cell pathologies where medical treatments result in immune reactions (such as stem cell transplants resulting in acute graft-versus-host-disease or immuno-oncology treatments resulting in immune related-adverse events). 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. Artax Biopharma is based in the Boston area and raised Series A & B from Advent Life Sciences, Sound Bioventures, and Columbus Venture Partners. For more info, see or follow us on LinkedIn. For Phase 2a study inquiries, please contact: ax-158-1011@artaxbiopharma.com