MJFF grant will aid Stealth’s study of mitochondrial biomarkers

Preclinical effort will evaluate company's investigational therapy SBT-272

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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A grant from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) will support a two-year preclinical study by Stealth Biotherapeutics to evaluate its investigational therapy SBT-272, a small molecule designed to target cells’ mitochondria.

The study is intended to identify biomarkers of mitochondrial dysfunction, a phenomenon often observed in Parkinson’s disease but for which no approved treatments exist.

With the grant, the Foundation “recognizes the importance of targeting mitochondrial dysfunction in [Parkinson’s disease],” Reenie McCarthy, Stealth’s CEO, said in a press release. “We are thrilled to work with MJFF to expedite development of SBT-272 and, hopefully, expand therapeutic options for patients living with [Parkinson’s disease].”

Stealth will work with Laurie Sanders, PhD, a Parkinson’s researcher and an associate professor of neurology and pathology at Duke University School of Medicine in North Carolina.

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The workings of mitochondria in Parkinson’s disease

Mitochondria are small structures found inside cells and often referred to as powerhouses because they have inner folds where most of the energy cells need to function properly is generated. Problems with how mitochondria work may cause neurons to malfunction and die in neurological diseases such as Parkinson’s and amyotrophic lateral sclerosis (ALS).

One hallmark of Parkinson’s is the buildup of clumps of the alpha-synuclein protein inside neurons. Misshapen alpha-synuclein interacts with a fatty molecule called cardiolipin in the mitochondria’s inner folds, impairing their ability to generate energy.

SBT-272 would target cardiolipin, which is expected to help mitochondria generate more energy and to prevent oxidative stress, a type of cellular damage that drives cell death and may also contribute to Parkinson’s.

Preclinical work in a mouse model showed SBT-272 resulted in less clumping of alpha-synuclein and protected neurons from dying. It also reduced the levels of two biomarkers of inflammation.

A Phase 1 clinical study of an under-the-skin formulation is underway in healthy volunteers. Interim data showed that doses selected for further testing are expected to reach therapeutic levels in the brain and be safe and well tolerated.

The small molecule is also being investigated for treating ALS and the company received orphan drug status from the U.S. Food and Drug Administration last year based on the results of Phase 1 testing.