MTX-101 (Mozart Therapeutics)

Study to Evaluate MTX-101 for Safety, Pharmacokinetics, and Pharmacodynamics in Healthy Adults and Patients SEATTLE, June 17, 2024 /PRNewswire/ -- Mozart Therapeutics, a clinical stage biopharmaceutical company focused on the development of CD8 Treg modulators in autoimmune diseases, today announce the dosing of the first cohort of participants in the Company's initial clinical study of MTX-101, a first-in-class CD8 Treg modulator and autoimmune checkpoint inhibitor. "Dosing the first cohort of participants in this Phase 1a/b clinical study with MTX-101 is a significant milestone for Mozart, marking our transition into the clinic and bringing us closer to reaching our goal of changing the treatment paradigm by modifying the course of autoimmune disease in every patient," said Katie Fanning, President and Chief Executive Officer of Mozart Therapeutics. "In autoimmune disease, CD8 Treg fail to recognize and eliminate pathogenic or self-reactive CD4 T cells, resulting in downstream proinflammatory events and perpetuation of tissue destruction. The aim of MTX-101 is to selectively target a fundamental cause of immune dysregulation and restore immune homeostasis in autoimmune disease" added Ms. Fanning. The Phase 1a portion of the study (NCT06324604) is a single and multiple ascending dose escalation study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of MTX-101 in healthy adults. The Phase 1b is a prospective, multi-center, randomized, double-blind, placebo-controlled trial to evaluate safety, PK, PD, and disease-specific biomarkers in patients with type 1 diabetes mellitus or celiac disease. "While the goal of this trial is to carefully characterize the profile of a novel approach to restoring immune homeostasis, we also recognize the urgent need for safer, more effective targeted approaches to preventing and treating chronic debilitating autoimmune diseases. Following completion of the Phase 1a portion, we expect to generate proof-of-mechanism data across two mechanistically and clinically different autoimmune diseases, and hopefully demonstrate the potential for CD8 Treg biology to be broadly relevant," said Dr. Jason Chien, Chief Medical Officer at Mozart. About MTX-101 MTX-101 is a bispecific antibody targeting inhibitory KIR and CD8 expressed on regulatory CD8 T cells. This autoimmune checkpoint inhibitor aims to restore the intrinsic functions of regulatory CD8 T cells, acting early in the autoimmune disease process to suppress and eliminate pathogenic cells, halt downstream inflammation, and prevent tissue destruction. About Mozart Therapeutics Mozart Therapeutics is focused on developing first-in-class disease-modifying therapies for autoimmune diseases, utilizing a novel approach to restoring immune system function by targeting the CD8 T regulatory network. The company is headquartered in Seattle, WA. For more information, visit and follow the company on LinkedIn @Mozart–tx.com. SOURCE Mozart Therapeutics

Findings highlight tolerability and pharmacology of MTX-101 in humanized IL–15 transgenic mouse model SEATTLE, March 14, 2024 /PRNewswire/ -- Mozart Therapeutics, the leading developer of CD8 Treg modulators for the treatment of autoimmune and inflammatory diseases, today announce new non-clinical data for MTX-101. The data will be presented in a late-breaking poster presentation during the Society of Toxicology 63rd Annual Meeting, March 10‒14 in Salt Lake City, Utah. MTX-101 is an antibody-based bispecific autoimmune checkpoint inhibitor that selectively targets regulatory CD8 T cells. MTX-101 has limited cross-reactivity to its targets in animal models; therefore, a humanized IL-15 transgenic mouse model was utilized for non-clinical assessment of toxicity and pharmacology. Presentation highlights include: MTX-101 was well-tolerated following single and multiple doses in the humanized mouse model MTX-101 showed sustained binding to CD8 Treg and did not demonstrate off-target cell effects or cause an increase in detectable pro-inflammatory serum cytokines Serum concentrations of MTX-101 indicated high exposure and PK parameters consistent with antibodies Data derived from the humanized mouse model aligns with previous in vitro and in vivo findings for MTX-101 "We are pleased to present early data highlighting the non-clinical safety of MTX-101. Our data underline the utility of the humanized IL-15 transgenic mouse model to assess targeting human immune system receptors with limited cross-reactivity in conventional toxicology species," said Kristine Swiderek, Ph.D., Chief Scientific Officer at Mozart Therapeutics. "This model enables the non-clinical safety assessment and an understanding of the PK/PD relationship for MTX-101 to inform our clinical development strategy." The poster "Nonclinical Assessment of Toxicity and Pharmacology of MTX-101, a Novel KIRxCD8 Targeting Bispecific CD8 Treg Modulator, in Humanized Mouse" (Abstract #5081, Late-Breaking Session 3, March 14) can be accessed from the Mozart Therapeutics website. About MTX-101 MTX-101 is a bispecific CD8 Treg modulator targeting inhibitory KIR and CD8 expressed on regulatory CD8 T cells. This autoimmune checkpoint inhibitor aims to restore the intrinsic functions of regulatory CD8 T cells, acting early in the autoimmune disease process to suppress and eliminate pathogenic autoimmune cells, halt downstream inflammation, and prevent tissue destruction. About Mozart Therapeutics Mozart Therapeutics is focused on developing first-in-class disease-modifying therapies for autoimmune and inflammatory diseases, utilizing a novel approach to restoring immune system function by targeting the CD8 T regulatory network. The company is headquartered in Seattle, WA. For more information, visit and follow the company on LinkedIn @Mozart-tx. SOURCE Mozart Therapeutics