Phase 1a clinical trial of JNT-517 in healthy volunteers approaching completion

BOSTON, March 16, 2023 – Jnana Therapeutics, a clinical-stage biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, will present preclinical data for its lead program, a potential first-in-class, oral small molecule approach for the treatment of phenylketonuria (PKU). Data will be presented in a poster session at the 2023 Society for Inherited Metabolic Disorders (SIMD) Annual Meeting, which takes place March 18-21 in Salt Lake City, Utah. 

Preclinical data will be presented on Jnana’s small molecule inhibitor program targeting the neutral amino acid transporter SLC6A19. The data demonstrate that SLC6A19 inhibition results in a dose-dependent increase in urinary excretion of amino acids, including phenylalanine (Phe), and lowers plasma Phe levels in a PKU mouse model.

2023 Society for Inherited Metabolic Disorders (SIMD) Annual Meeting
March 18-21, Salt Lake City, Utah

Poster #129: A Small Molecule SLC6A19 Inhibitor Increases Urinary Phenylalanine Excretion and Reduces its Pathogenic Plasma Accumulation in a Phenylketonuria Mouse Model

“These data show that increasing urinary excretion of excess Phe by inhibiting SLC6A19 may be a novel therapeutic approach to reducing plasma Phe across a broad population of individuals living with PKU, from newborns to adults,” said John Throup, Ph.D., Senior Vice President & Head of Development at Jnana Therapeutics. “We look forward to further exploring the potential of this approach in our ongoing clinical studies with JNT-517 and sharing updates as we progress the program.” 

Jnana is approaching completion of its Phase 1a study of JNT-517, which was initiated in Australia in late 2022 in healthy volunteers. JNT-517 is the first in a pipeline of potential therapies discovered using RAPID, Jnana’s proprietary high-throughput, screening-enabled chemoproteomics platform that uncovers unique binding sites on elusive disease targets and identifies novel small molecules to modulate them.

About PKU

Phenylketonuria (PKU) is an inherited metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase (PAH). This enzyme is required for the breakdown of phenylalanine (Phe), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood. If left untreated, toxic levels of Phe in the blood can result in progressive and severe neurological impairment and neuropsychological complications. The SLC transporter SLC6A19 is responsible for kidney reabsorption of Phe back into the bloodstream, and the inhibition of SLC6A19 offers a novel, oral approach for the treatment of PKU by facilitating urinary excretion of excess Phe.

About JNT-517

JNT-517 is a selective small-molecule inhibitor of the phenylalanine transporter, SLC6A19, that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe levels. The FDA has granted JNT-517 Rare Pediatric Disease Designation.

About Jnana Therapeutics

Jnana Therapeutics is a clinical-stage biotechnology company leveraging its next-generation RAPID chemoproteomics platform to discover medicines for highly validated, challenging-to-drug targets to treat diseases with high unmet need. Jnana is focused on developing first- and best-in-class therapies to treat a wide range of diseases, including rare diseases, immune-mediated diseases and cancer. Jnana’s wholly owned lead program, JNT-517, which targets an allosteric site on the phenylalanine transporter SLC6A19, is a potential first-in-class oral approach for the treatment of phenylketonuria (PKU), a rare genetic metabolic disease. Located in Boston, Jnana brings together scientific leaders in small molecule drug discovery and development, a highly experienced management team, and the backing of leading life science investors Bain Capital Life Sciences, RA Capital Management, Polaris Partners, Versant Ventures, Avalon Ventures, Pfizer Ventures, and AbbVie Ventures. For more information, please visit and follow us on Twitter and LinkedIn.

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