Mitobridge, Inc., a pioneer in the discovery and development of products that improve mitochondrial function, today announces a key milestone with the initiation of the first-in-human Phase I trial of its PPAR-delta (PPARd) modulator, MA-0211 (also known as MTB-1).

The study will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of MA-0211 in healthy volunteers, which will provide the basis for a trial programme in Duchenne Muscular Dystrophy (DMD) patients. MA-0211 is the first clinical compound to emerge from Mitobridge’s mitochondrial enhancement platform. The PPARd modulator aims to reverse the mitochondrial deficits in DMD, which play a key role in disease progression.

Mitobridge’s research has confirmed and expanded upon previous studies showing that mitochondrial defects contribute to abnormalities in the dystrophic muscle and play a central role in the etiology of DMD.


“PPARd modulation represents a promising therapeutic approach to improving mitochondrial function and muscle health in DMD patients,” stated Mike Patane, CSO of Mitobridge. “This milestone with our lead programme further validates our mitochondrial enhancement platform and ability to generate promising drug candidates that modulate mitochondrial function…”


Mitobridge scientists have assembled extensive nonclinical data in patient samples and genetic animal models (DMD mouse model) demonstrating that MA-0211 may be therapeutically beneficial to DMD patients. Once-daily oral dosing of MA-0211 for five weeks in mdx mice produced several therapeutic benefits including increased running endurance on a treadmill, decreased muscle necrosis and inflammation and decreased diaphragm fibrosis.

In a similar study, six months of dosing in older mdx mice resulted in decreased serum creatine kinase and improved cardiac and respiratory function compared to untreated mdx mice. The strong pre-clinical data are the basis for advancing the compound into clinical development and highlight MA-0211’s potential to reverse key defects and slow disease progression.

MA-0211 is being developed with the Company’s corporate partner, Astellas Pharma, Inc.
Recently, George Mulligan, Mitobridge’s SVP of Translation Medicine, presented an update of the MA-0211 programme at the Parent Project Muscular Dystrophy Annual Connect Conference on June 30, 2017. The presentation is available here:

Mitobridge’s full press release can be found here.


Action Duchenne will be following developments closely and look forward to sharing more information with you as we get it. As always the best place in the UK to get updates on all aspects of Duchenne is the annual Action Duchenne International Conference. This year the event is 10-12 November at the Hilton Birmingham Metropole.