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repinatrabit
- Development stage
- Phase 3
- Lead developer
- Taiho Pharmaceutical
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Repinatrabit (formerly JNT-517) is a first-in-class, oral small molecule inhibitor of the solute carrier family 6 member 19 (SLC6A19) transporter, also known as B0AT1. Developed by Jnana Therapeutics (now a subsidiary of Otsuka Pharmaceutical), it is designed to treat phenylketonuria (PKU) by blocking the reabsorption of phenylalanine in the kidney, thereby promoting its excretion in the urine and lowering blood phenylalanine levels. This mechanism is independent of the phenylalanine hydroxylase (PAH) enzyme, making it potentially suitable for all PKU patients regardless of their specific genetic mutation. The drug was discovered using Jnana's RAPID chemoproteomics platform and is currently being evaluated in Phase 3 clinical trials.
- Other names
- repinatrabit
02
Targets
SLC6A19 (Na+-dependent system B0 transporter)
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