Treatment to protect mitochondria may enter clinical trials this year

Asha preparing to test ASHA-091, targeting health of a cell's energy source

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by Andrea Lobo |

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An experimental therapy called ASHA-091, which was seen to restore normal mitochondria function and possibly be disease modifying in a mouse model of Parkinson’s disease, may soon be moving into clinical trials, its developer, Asha Therapeutics, reports.

Problems with the workings of mitochondria, a cell’s energy source, are known in neurodegenerative disorders like Parkinson’s and Alzheimer’s disease. Asha anticipates beginning clinical testing of ASHA-091 in both these disorders before the end of this year, it announced in a company press release.

ASHA-091 is designed to specifically inhibit DRP1 activation, a regulatory protein with a role in the fragmentation of mitochondria.

“A key feature of most neurodegenerative disease … is the hyper-fragmentation of mitochondria. This promotes neuroinflammation, neuronal dysfunction, and ultimately neurodegeneration,” the company reports on a therapy webpage. ASHA-091 is a “first-in-class highly specific inhibitor of mitochondrial fragmentation that restores normal cellular function.”

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In preparation for those planned trials, Asha announced the appointment of two neurologists to its scientific advisory board: Michael Gold, MD, and Allan Levey, MD. Both have expertise in neurodegenerative diseases and behavioral neurology.

“Their expertise comes at a pivotal moment for Asha as we transition our first-in-class lead programs designed using our PRISM technology to clinical trials … [that] will facilitate our transition to a clinical-stage company and most importantly bring Asha’s disease-modifying medicines to patients with neurological and oncological diseases in need of effective therapies,” said Bradlee Heckmann, PhD, Asha’s chief scientific officer and scientific co-founder.

Heckmann presented preclinical efficacy and safety data on ASHA-091 at the AD/PD 2024 International Conference on Alzheimer’s and Parkinson’s Diseases, held March 5-9 in Lisbon, Portugal, and virtually. The presentation was titled “ASHA-091: Novel Pharmacological Targeting of Mitochondrial Dysfunction to Ameliorate Parkinson’s & Alzheimer’s Disease Pathology.”

Parkinson’s is caused by the dysfunction and death of nerve cells responsible for producing dopamine, a chemical that neurons use to communicate with each other. The loss of these dopaminergic neurons affects dopamine signaling in the brain, important for the control of voluntary movement.

Although what causes such neuronal loss is not fully known, it has been associated with the dysfunction of mitochondria, cellular structures commonly referred to as the cell’s powerhouses. Restoring mitochondrial function could help to restore health to dopaminergic neurons and ease Parkinson’s symptoms.

ASHA-091, designed using the company’s PRISM molecular technology platform, was reported to be safe and to effectively reverse motor impairment in a mouse model of Parkinson’s disease, and in animal models of similar disorders.

The PRISM platform speeds the drug discovery process — potentially turning years into months of work — by not needing to screen millions of compounds to assess their potential efficacy, as is currently done.

PRISM technology, according to Asha, is a novel physics-based therapeutic design platform, based on proprietary software and advanced simulation programs, that allows the custom designing of new compounds for specific disease targets. According to the company, these compounds also come optimized for use in clinical trials.