Jnana Therapeutics to Advance JNT-517 as Development Candidate and Potential First-in-Class Oral Treatment for PKU

BOSTON--(BUSINESS WIRE)-- Jnana Therapeutics, a biotechnology company utilizing its next generation chemoproteomic platform to address well-validated but hard-to-drug targets, today announced that it has selected JNT-517 as a development candidate and a potential first-in-class oral approach for the treatment of phenylketonuria (PKU), a rare inherited metabolic disease. The company also announced plans to initiate a Phase 1 clinical study with JNT-517 later this year.

JNT-517 targets SLC6A19, a metabolite transporter responsible for kidney reabsorption of phenylalanine (Phe). Based on human genetic evidence and in vivo studies in disease relevant animal models, SLC6A19 inhibition is expected to reduce the elevated blood Phe levels that drive PKU disease pathologies and thereby offers a promising and potentially transformational approach to treating PKU.

The discovery of JNT-517 is based on Jnana’s pioneering research and was enabled by RAPID, the company’s high-throughput screening-enabled chemoproteomic platform. The company recently generated the first cryo-EM structure of SLC6A19 in complex with a small molecule ligand, revealing that Jnana’s proprietary small molecule inhibitors bind in an unprecedented cryptic allosteric site on the SLC transporter.

“The advancement of our allosteric SLC6A19 inhibitor towards the clinic provides a compelling demonstration of the ability of the RAPID platform to deliver novel approaches to unlock hard-to-drug targets. We believe that JNT-517 offers a differentiated approach to PKU with an oral small molecule that can be used to treat any PKU patient, notwithstanding age or PAH mutation,” said Joel Barrish, Ph.D., co-founder, President and Chief Scientific Officer of Jnana Therapeutics. “RAPID has also enabled exciting progress in building a pipeline of highly validated and previously undruggable targets in immune-mediated diseases and oncology.

Following completion of ongoing IND-enabling studies, Jnana will initiate a Phase 1 clinical study to assess safety, pharmacokinetics, and pharmacodynamic biomarkers of JNT-517. The Phase 1 study design will include single and multiple dose escalation in healthy volunteers followed by a PKU patient cohort to obtain key proof-of-concept data that will support a subsequent registrational program.

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, these 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 Jnana Therapeutics

Jnana Therapeutics is a biotechnology company utilizing their RAPID platform to address well-validated but hard-to-drug targets, including the solute carrier (SLC) family of metabolite transporters. Jnana is focused on developing best-in-class therapies to treat a wide range of diseases, including rare diseases, immune-mediated diseases and cancer. Headquartered in Boston, Jnana is founded by world-renowned scientists and backed by leading life science investors. For more information, please visit www.jnanatx.com and follow us on Twitter and on LinkedIn.

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