Phase 1 clinical program in healthy volunteers is ongoing in Australia; IND clearance will facilitate program expansion to assess JNT-517 in individuals with PKU.

BOSTON, MA, USA I January 06, 2023 I Jnana Therapeutics, a biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for challenging-to-drug targets, today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) of its Investigational New Drug (IND) application for the clinical study of JNT-517 for the treatment of phenylketonuria (PKU).

Jnana’s ongoing Phase 1 program of JNT-517 was initiated in Australia in the fourth quarter of 2022 in healthy volunteers (the Phase 1a sub-study). Following Phase 1a, the program will expand to include clinical sites in the U.S., and the Phase 1b sub-study will evaluate JNT-517 in individuals with PKU, with the potential to demonstrate clinical proof of concept supporting a subsequent registrational program.

“Clearance of our IND for JNT-517 by the FDA, Jnana’s first IND filing, marks an exciting next step for Jnana and we believe for individuals with PKU who have limited treatment options,” said Joanne Kotz, Ph.D., co-founder and CEO of Jnana Therapeutics. “We are very excited to advance our Phase 1 program and look forward to working with investigators, advocacy groups, and individuals living with PKU.”

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.

About JNT-517

JNT-517 is a 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 on LinkedIn.

SOURCE: Jnana Therapeutics