FDA Grants Rare Pediatric Disease Status to META-001-PH for Primary Hyperoxaluria Treatment, Announces META Pharmaceuticals

HONG KONG, Aug. 5, 2024 — META Pharmaceuticals Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its investigational new drug, META-001-PH, aimed at treating primary hyperoxaluria (PH). PH is a rare genetic disorder leading to kidney stone formation, renal failure, and can be fatal in severe cases. This new designation aims to support the development of drugs for rare pediatric diseases affecting fewer than 200,000 people in the United States, primarily occurring in patients 18 years old and younger.

Primary Hyperoxaluria (PH) is characterized by excessive oxalate production and its deposition in the body due to enzyme defects in the liver and other organs. Patients often suffer from kidney stones, nephrocalcinosis, renal failure, and oxalate deposits in other organs. Severe instances can lead to end-stage renal disease (ESRD), necessitating dialysis or transplants. Symptoms typically emerge between ages 0 to 3, and without treatment, most patients will develop ESRD during their teenage years.

The incidence of PH is approximately 1 in 58,000, impacting over 10,000 individuals in the U.S. and the European Union, and more than 20,000 people in China. Currently, there is no cure for PH. Existing treatments focus on supportive care, such as increased fluid intake to dilute urine oxalate and medications like pyridoxine (vitamin B6) to lower oxalate production. However, these treatments are not effective in controlling symptoms and disease progression in the long term.

META-001-PH is an innovative small molecule drug developed by META Pharmaceuticals for treating PH. This drug is the result of collaboration between META's chemistry team and XtalPi's AI drug discovery team. XtalPi's automated robotic chemical synthesis lab was responsible for the chemical synthesis of the related molecule series, ensuring accurate and efficient production. Preclinical experiments in animal models have demonstrated that META-001-PH can reduce urinary oxalate excretion by up to 80%. Unlike existing treatments, META-001-PH, when administered orally on a daily basis, can maintain normal oxalate levels, indicating its potential for long-term management of kidney stone formation in PH patients. The drug has also shown good tolerability and safety in preclinical animal models and is currently undergoing toxicology studies to enable Investigational New Drug (IND) application. A clinical Phase I safety assessment on healthy subjects is planned for the first half of 2025 in Australia.

The RPDD is a designation aimed at rare pediatric diseases affecting fewer than 200,000 patients and posing serious life-threatening risks to children under 18 years of age. Under this program, sponsors can receive a Priority Review Voucher (PRV) upon approval of a New Drug Application (NDA) or Biologics License Application (BLA) for a rare pediatric disease. PRVs can be used for any subsequent product approval application, reducing review time by 4-6 months, or can be traded, with recent transfer prices exceeding $100 million.

META Pharmaceuticals Inc. is an innovative company committed to discovering and developing new treatments for autoimmune disorders, metabolic diseases, and cancer. As the sole company in the Asia Pacific region leveraging immuno-metabolism theory, META is pioneering safer and more effective therapies to modulate cellular metabolism and regulate immune system function. META has developed two distinct chemical series targeting META-001 for the treatment of autoimmune diseases and primary hyperoxaluria, which could become next-generation oral treatments addressing significant clinical needs. META is jointly incubated by XtalPi, Forcefield Ventures, and IMO Ventures, with investments from Tiantu Capital, Yael Capital, Fangyuan Capital, Lead Rich International, Decent Capital, and Bopu Capital.

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