Mozart Therapeutics, Inc.

Data highlight potential of first-in-class CD8 Treg modulator Phase 1 clinical trial underway to evaluate MTX-101 in healthy adults and patients with celiac disease or type 1 diabetes SEATTLE, June 21, 2024 /PRNewswire/ -- Mozart Therapeutics, a clinical stage biopharmaceutical company focused on the development of CD8 Treg modulators for the treatment of autoimmune diseases, today presented new preclinical data on the Company's lead program, MTX-101. The data were reviewed during an oral presentation at the 2024 American Diabetes Association 84th Scientific Sessions, held June 21–24 in Orlando, FL. Courtney Crane, Ph.D., Senior Vice President of Research at Mozart Therapeutics, presented data supporting the relevance of therapeutic modulation of the regulatory CD8 T cell network in autoimmune disease, including type 1 diabetes (T1D). Additionally, data from in vitro studies using T1D-derived PBMC demonstrate that treatment with MTX-101 increases the prevalence of CD8 Treg and that the functionality of CD8 Treg (i.e., activation and cytolytic capacity) can be restored in this autoimmune setting. "We are pleased to highlight this new preclinical data on MTX-101 in type 1 diabetes," said Dr. Crane. "These data support the ability of MTX-101 to restore CD8 Treg function in autoimmune disease. We look forward to evaluating this candidate in patients with type 1 diabetes mellitus during Part B of our ongoing Phase 1 study". 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. MTX-101 is currently being evaluated in a randomized, blinded, placebo-controlled Phase 1 clinical study (NCT06324604) in healthy adults (study Part A) and in patients with celiac disease or type 1 diabetes mellitus (study Part B). 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

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 the novel mechanism of action and potential for therapeutic activity in highly inflammatory autoimmune disease SEATTLE, May 3, 2024 /PRNewswire/ -- Mozart Therapeutics, the leading developer of CD8 Treg network modulators, today announce the release of new pre-clinical data for their second program, a KIR x ICOS bispecific antibody targeting CD8 regulatory T cells. The data will be presented during the Immunology 2024 meeting, May 3–7 in Chicago, Illinois. Presentation highlights demonstrate KIR x ICOS Modulator: Restores and potentiates CD8 Treg function through binding the inhibitory receptor KIR and the costimulatory receptor ICOS, both expressed on CD8 Treg Selectively activates CD8 Treg with dose-dependent increases in both their prevalence and capacity to eliminate pathogenic CD4 T cells Enhances ICOS signaling only in CD8 Treg and not other ICOS-expressing immune cells Reduces proinflammatory cytokines and improves survival in a highly inflammatory disease model Has acceptable tolerability in initial studies in humanized mice "Our preclinical data underscore the potential of our KIR x ICOS CD8 Treg Modulator as a selective therapeutic approach for highly inflammatory autoimmune diseases, including Crohn's disease and ulcerative colitis. Findings show that the Modulator is selective toward CD8 Treg, efficacious in models of disease, and has an encouraging safety profile," said Dr. Kristine Swiderek, Chief Scientific Officer at Mozart Therapeutics. "The data support further advancement of this promising molecule." Presentation Details: The poster can be accessed from the Mozart Therapeutics website following the May 6 presentation. About KIR x ICOS Modulator Inhibitory killer immunoglobulin-like receptors (KIR) are autoimmune checkpoints expressed on CD8 regulatory T cells, limiting their function of killing pathogenic CD4 T cells. The inducible T cell costimulator (ICOS) is upregulated on activated CD8 Treg and critical to CD8 Treg function. KIR x ICOS is a bispecific antibody targeting CD8 Treg. The molecule aims to restore CD8 Treg function through inhibition of KIR, while promoting ICOS-mediated CD8 Treg activation, in chronic and highly inflammatory autoimmune disease. 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 homeostasis by targeting the CD8 Treg network. The company is headquartered in Seattle, WA. For more information, visit and follow the company on LinkedIn @Mozart-tx. Media Contact: Julie Rathbun Rathbun Communications [email protected] 206-769-9219 SOURCE Mozart Therapeutics