AKI is a sudden decline in kidney function that can occur following ischemia, major surgery, exposure to nephrotoxic drugs, and other severe physiologic stresses that impacts over 13 million people worldwide each year. Despite its substantial clinical burden and its association with subsequent chronic kidney disease (CKD), treatment remains largely supportive. The new funding will enable Vincere to complete key IND-enabling safety studies required to advance its kidney-focused USP30 inhibitor toward an Investigational New Drug (IND) application and first-in-human clinical development.

Targeting Mitochondrial Dysfunction to Promote Kidney Recovery

Mitochondrial damage is an important feature of AKI, particularly within the highly metabolically active cells of the renal tubule. Mitophagy, the selective removal and recycling of damaged mitochondria, serves as a natural mitochondrial quality-control mechanism that can help cells recover following injury.

Vincere’s approach is designed to enhance this protective process by selectively inhibiting USP30, a mitochondrial deubiquitinating enzyme that acts as a negative regulator of mitophagy. By removing this molecular “brake,” USP30 inhibition accelerates clearance of dysfunctional mitochondria, restores mitochondrial health, and promotes recovery of injured kidney tissue.

“While we formed Vincere to seek disease modifying therapies for Parkinson’s, as a curious scientist I’m always excited to follow the data, which has led us to examine other highly energy-reliant organs such as kidneys,” said Dr. Spring Behrouz, co-founder and CEO of Vincere. “The involvement of mitochondria in kidney injury is newly emerging  and I’m grateful to the NIH for supporting this translational step that will prepare us to evaluate efficacy that could prevent over a million deaths each year from untreated AKI.”

The NIDDK award will support a comprehensive IND-enabling program, including genotoxicity and toxicology studies, 28-day Good Laboratory Practice (GLP) toxicology studies, and cardiovascular, respiratory, and central nervous system safety pharmacology. Successful completion of these studies is intended to support an IND submission and subsequent first-in-human clinical evaluation of Vincere’s kidney-focused USP30 inhibitor.

The award also highlights the broader potential of Vincere’s USP30 platform across diseases characterized by mitochondrial dysfunction. Vincere is separately advancing a CNS-penetrant USP30 inhibitor for Parkinson’s disease, supported by a $5 million award from The Michael J. Fox Foundation, demonstrating the potential to develop molecules with diverse properties from the same mechanistic platform for distinct CNS and peripheral indications.

“One of the core aspects of our computational biology platform was to aggregate data and knowledge from all tissues and therapeutic areas,” said Andy Lee, co-founder and CBO of Vincere. “Biology doesn’t share the boundaries humans like to apply, and AI has been helpful to assess connections beyond our biases. We take a similar approach to partnering, with broad exploration followed by strong commitment to follow the data as signals of alignment emerge.”

About Vincere Biosciences Inc.
https://www.vincerebio.com

Cambridge, MA-based Vincere Biosciences develops innovative, mechanism-driven therapeutics that target the intersection of aging and disease. Using proprietary computational tools and cutting-edge biology, Vincere is dedicated to discovering and developing small molecules that address the root causes of age-related conditions such as Parkinson’s and AKI, amongst others.

Contact: Anahit Campbell,
Strategic Initiatives Manager,
Vincere Biosciences
Phone: 857-245-9492
Email:campbell@vincerebio.com

View original content:https://www.prnewswire.com/news-releases/vincere-biosciences-awarded-2-7-million-nih-grant-to-advance-usp30-inhibitor-for-acute-kidney-injury-302874590.html

SOURCE Vincere Biosciences