Study to Evaluate MTX-101 for Safety, Pharmacokinetics, and Pharmacodynamics in Healthy Adults and Patients

SEATTLE, WA, USA I June 17, 2024 I 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–

SOURCE: Mozart Therapeutics