Vincere lands MJFF grant to support mitochondrial therapy through IND-enabling studies ahead of first-in-human trials in 2026.
Mitochondrial therapeutics biotech Vincere Biosciences has been awarded a $5 million grant from The Michael J Fox Foundation for Parkinson’s Research to move its lead Parkinson’s program into the clinic. The funding will advance the company’s USP30 inhibitor and biomarker development strategy through IND-enabling studies in preparation for a planned Phase 1 trial in 2026.
The award comes amid growing momentum in the mitochondrial medicine field. Earlier this year, Stealth BioTherapeutics secured first ever FDA approval for a therapy targeting mitochondrial biology. The clearance of Forzinity for Barth syndrome was the first formal recognition that modulating mitochondrial function can yield therapeutic benefit in human disease.
The milestone had implications for programs pursuing similar biological pathways, including Vincere, whose CEO Spring Behrouz told us that the approval felt like “watching a decades-long mission finally land on its target planet.”
“This is fantastic news for those of us heading toward the FDA with other approaches to improve mitochondrial health,” she said. “Mitochondrial dysfunction is a core driver of both disease and aging which itself is the biggest risk factor for most diseases.”
Vincere is developing small-molecule therapeutics aimed at restoring mitophagy, the process of mitochondrial quality control that is disrupted in Parkinson’s disease and other age-related conditions. Its lead candidate inhibits USP30, a mitochondrial deubiquitinating enzyme that counteracts the ubiquitin tagging required for mitophagy. In healthy cells, ubiquitin chains on the outer mitochondrial membrane recruit autophagic machinery to remove damaged mitochondria. USP30 removes these chains, slowing the clearance and repair of defective organelles. Backed by the MJFF, Vincere’s strategy is to block this activity, accelerate the removal of dysfunctional mitochondria and help restore cellular energy balance.
“Parkinson’s disease is the visible tip of a much bigger iceberg: age-related failure of cellular clean-up,” Behrouz told us. “What we’re doing at Vincere is restoring the clean-up of mitochondria, the cell’s powerhouses. We know this is a process that declines with age and specifically in brains of patients with Parkinson’s disease – so the double-hit means Parkinson’s disease manifests later in life.”
Behrouz said that Vincere’s preclinical work suggests the company’s molecules remove only damaged mitochondria, without affecting healthy ones, which is key for safety.
“We have also shown that we can enhance this mitochondrial repair, mitophagy, in the brains of mice with the effect being much more robust in aged-animals known to be deficient in the clean-up process,” she said. “But anyone who’s walked the journey of drug discovery understands the weight of that long twenty-year march from hypothesis to healing. And Parkinson’s patients cannot wait for another slow chapter of incremental progress. That urgency is why I feel an indescribable impatience to bring forward something that can change health in a profound way.”
Co-founder Andy Lee told us that Parkinson’s disease provides a “unique platform” to investigate age-related mechanisms that may translate to longevity.
“Aging is the biggest risk factor for Parkinson’s, and there are about a dozen genetic causes that give us clues into the mechanism,” he said. “Some of those directly oppose the action of our target, USP30, and I think that’s a great clue that correcting this deficit may also impact aging. For the past few years we have been laser focused on engineering a great molecule that can go all the way in human trials. It’s great to have a clear line of sight on that next stage where we can start to explore efficacy in people who need it most.”
The new MJFF grant extends a long-running relationship between the two organizations, following earlier support for Vincere’s preclinical work in 2019 and 2022.
“Our collaboration with Vincere Biosciences over the years has supported the advancement of research targeting mitochondrial dysfunction, a key driver of Parkinson’s pathology,” said MJFF’s lead scientific program manager Dr Jessica Tome Garcia. “This next phase of work builds on that foundation and represents important progress toward disease-modifying therapies that could meaningfully improve patients’ lives.”
The company is now preparing the pharmacology, toxicology and regulatory studies needed for an IND submission. The foundation’s support will also support biomarker development to measure target engagement and support translation into clinical settings.
Having pursued USP30 since its algorithms prioritized the enzyme in 2018, Vincere claims that interest in the target has grown in the years since, supported by evidence that mitochondrial dysfunction is a shared feature across neurodegenerative and systemic diseases. While Parkinson’s remains the company’s primary focus, the biological role of mitophagy extends to organs with high metabolic demand, including the kidney, heart and muscle. Research across these systems has linked reduced mitophagy to acute kidney injury, progression to chronic kidney disease, cardiac aging and poor recovery after myocardial infarction, suggesting potential future applications for the company’s mitophagy-enhancing therapeutics.
