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Maggie's Pearl

Rochester, Minnesota, United Statesmaggiespearl.com ↗

Ownership: Private
Development stage: Phase 3

Overview

Maggie’s Pearl has fully enrolled a Phase III clinical trial at Mayo Clinic to test the safety and efficacy of epalrestat—a small molecule previously approved in Asia for diabetic neuropathy—in up to 40 children with PMM2-CDG. The program builds on positive results from compassionate use in a single patient and foundational research using model organisms and patient-derived cells. The company leverages decades of safety data for epalrestat from its use in adults with diabetes

Therapeutic areas: Rare Diseases
Modalities: Small molecule drug repurposing
Industry: Biotech

Drug pipeline

1 asset

epalrestat

Phase 3

Indications: PMM2-CDG

Partnerships

Perlara PBC (biotech public benefit corporation)Mayo Clinic

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