Drug intelligence / Profile preview

META-001-PH

Development stage
Preclinical
Lead developer
META Pharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

META-001-PH is an investigational small molecule drug developed by META Pharmaceuticals in collaboration with XtalPi for the treatment of primary hyperoxaluria (PH). PH is a rare autosomal recessive metabolic disorder characterized by the overproduction of oxalate, which leads to recurrent kidney stones, nephrocalcinosis, and potentially end-stage renal disease. Developed using AI-driven discovery platforms, META-001-PH is an orally administered, once-daily therapy that has shown the potential to reduce urinary oxalate excretion by up to 80% in preclinical models. The compound received FDA Rare Pediatric Disease Designation in August 2024 and is progressing through IND-enabling studies, with Phase 1 clinical trials expected to commence in Australia in the first half of 2025.

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