The US FDA has granted the Rare Pediatric Disease Designation (RPDD) for META-001-PH, a small-molecule drug co-developed by XtalPi's AI drug discovery team and biopharmaceutical company META Pharmaceuticals, to treat primary hyperoxaluria (PH). This rare genetic disorder can cause dangerous kidney stone formation and lead to renal failure.

META-001-PH is designed to reduce urinary oxalate excretion—a main contributor to the disease—by as much as 80%. This orally administered treatment aims to control kidney stone formation in PH patients long-term. The companies expect to push the drug into clinical trials in early 2025, with a clinical Phase I safety assessment on healthy subjects planned to be carried out in Australia.

Analyst QuickTake: The drug discovery collaboration between XtalPi and META Pharmaceuticals was established in June 2023 after XtalPi’s contribution to META’s two consecutive seed and pre-A funding rounds which raised USD 15 million. The purpose of the collaboration was for XtalPi to back META’s program of converting its novel drug targets into first-in-class therapeutics for autoimmune diseases, with the help of XtalPi’s AI-powered drug discovery platform.