Repinatrabit

CAS 2837993-05-0

MF C18H22F4N4O3 MW 418.4 g/mol

(3R)-3-[cyclopropyl-[[2-fluoro-4-(trifluoromethoxy)phenyl]methylcarbamoyl]amino]piperidine-1-carboxamide

(3R)-3-[cyclopropyl({[2-fluoro-4-(trifluoromethoxy)phenyl]methyl}carbamoyl)amino]piperidine-1-
carboxamide
solute carrier family 6 member 19 (SLC6A19) inhibitor(phenylketonuria), JNT-517, JNT 517, orphan drug, rare pediatric disease designations, Jnana Therapeutics, 5P44NDU6AC, JN 11804, JN-11804

Repinatrabit (JNT-517) is an investigational, oral, small-molecule drug developed by Jnana Therapeutics (now part of Otsuka Pharmaceutical) to treat Phenylketonuria (PKU). It acts as a selective inhibitor of the SLC6A19 transporter, reducing blood phenylalanine (Phe) levels by increasing its urinary excretion. 

Key Details About Repinatrabit:

• Mechanism: It targets a novel, cryptic allosteric site to block kidney reabsorption of phenylalanine, aiming to be a first-in-class oral therapy for all PKU patients, regardless of age or genotype.
• Clinical Trials: Otsuka initiated a global Phase 3 study (NCT06971731) in December 2025 to evaluate its safety and efficacy, following positive results from earlier studies.
• Status: The FDA has granted it orphan drug and rare pediatric disease designations.

• A Study to Evaluate the Safety and Efficacy of JNT-517 in Participants With Phenylketonuria (PKU)CTID: NCT06971731Phase: Phase 3Status: RecruitingDate: 2026-02-04
• A Phase 2 Study of JNT-517 in Adolescent Participants With PhenylketonuriaCTID: NCT06637514Phase: Phase 2Status: RecruitingDate: 2025-08-19
• First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With PhenylketonuriaCTID: NCT05781399Phase: Phase 1/Phase 2Status: Active, not recruitingDate: 2025-07-31
• A Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants With PhenylketonuriaCTID: NCT06628128Phase: Phase 3Status: Not yet recruitingDate: 2025-06-03

EMA Drug Information,

Type, Orphan designations

Active Substance,

(R)-3-(1-Cyclopropyl-3-(2-fluoro-4-(trifluoromethoxy)benzyl)ureido)piperidine-1-carboxamide

Intended Use, Treatment of hyperphenylalaninaemia, Status of Orphan Designation, Positive

First Published Date, 2024-08-22

PAT

https://patentscope.wipo.int/search/en/detail.jsf?docId=US463725634&_cid=P12-MMK00V-42348-1

SYN

https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2024118721&_cid=P12-MMJZU4-37251-1

PAT

https://patentscope.wipo.int/search/en/detail.jsf?docId=WO2025217521&_cid=P12-MMJZU4-37251-2

PAT

• Crystalline forms of a piperidine inhibitor of slc6a19 functionPublication Number: WO-2024118721-A1Priority Date: 2022-11-30
• Crystalline forms of a piperidine inhibitor of slc6a19 functionPublication Number: EP-4626420-A1Priority Date: 2022-11-30
• Dosing regimen for treatment of PKU with SLC6A19 functional piperidine inhibitorsPublication Number: CN-120091814-APriority Date: 2022-09-14
• Small molecule inhibitors of mammalian slc6a19 functionPublication Number: US-2024208923-A1Priority Date: 2021-03-10
• Small molecule inhibitors of mammalian slc6a19 functionPublication Number: US-2025289797-A1Priority Date: 2021-03-10

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///////////repinatrabit, ANAX, JNT-517, JNT 517, orphan drug, rare pediatric disease designations, Jnana Therapeutics, 5P44NDU6AC, JN 11804, JN-11804