MTB-1 - MTB-1 mediated gene regulation

Frequently Asked Questions (FAQs) about this research:


What stage is this research?

  • We are expecting to file and IND in May and First-in Human Phase 1 study in normal healthy adult volunteers is anticipated for summer 2017.


Where is this research being done and who is funding this research?

  • The pre-clinical research was performed and funded by Mitobridge and Astellas Pharma will conduct and fund the clinical development.


What is the goal or purpose of this research?

  • The goal of the Phase 1 study is to demonstrate safety and tolerability in normal healthy adult volunteers. The study will also characterize concentration and biological effects of varied doses of MTB-1 in order to pave the way for future studies in DMD patients.


What is the current state of this research?

  • Our pre-clinical data demonstrate that MTB-1 can improve mitochondrial function and have relevent, beneficial effects in pre-clinical models of Duchenne, including mdx mice and muscle cells from patients with Duchenne.


What steps need to be completed before moving into a clinical trial?

  • The IND needs to be filed with the FDA. The first DMD patient trial is anticipated for second half of 2018.


Where would a clinical trial take place?

  • The first clinical trial will be conducted at a single study site in the US. The sites for the DMD patient studies have not yet been determined.


Who would be eligible to participate in a clinical trial?

  • Normal healthy adult volunteers will participate in the First-in-Human PI study. The mechanism of action of MTB-1 is not dependent on a particular dystrophin Glossary Link mutation and therefore DMD patients with all mutation types could be included in subsequent clinical trials.


Where can I learn more about this research?