Jnana Therapeutics to Present Preclinical Data for its PKU Program at the 2022 National PKU Alliance (NPKUA) Conference

BOSTON, July 8, 2022 /PRNewswire/ -- Jnana Therapeutics, a biotechnology company leveraging its next-generation chemoproteomics platform to discover medicines for highly validated, previously undruggable targets to treat diseases with high unmet need, will present preclinical data for its lead program, a potential first-in-class oral approach for the treatment of phenylketonuria (PKU). Data will be presented at the 2022 National PKU Alliance Conference in Vancouver, Wash. July 9 from 3-4:30 pm, as a poster titled "Inhibition of SLC6A19 as a Therapeutic Strategy for the Treatment of PKU."

Jnana's development candidate, JNT-517, is a small molecule, allosteric inhibitor of SLC6A19, a metabolite transporter responsible for reabsorption of phenylalanine (Phe) in the kidney. In people with PKU, a rare inherited metabolic disease, Phe accumulates to abnormally high levels in the blood which, if left untreated, results in progressive and severe neurological impairment and neuropsychological complications. Preclinical data to be presented at the National PKU Alliance Conference demonstrate that inhibition of SLC6A19 increases urinary excretion of Phe and, in turn, significantly reduces blood Phe levels in a mouse model of PKU.

"There are currently limited treatment options for people living with PKU," said John Throup, Ph.D., Senior Vice President & Head of Development at Jnana Therapeutics. "We are highly encouraged by these preclinical data confirming that SLC6A19 inhibition significantly reduces plasma Phe concentrations, the underlying driver of PKU disease, in a PKU mouse model. As a highly potent and selective inhibitor of SLC6A19, JNT-517 has the potential to be a first-in-class targeted oral treatment for all individuals with PKU, regardless of genetic background or age. We look forward to partnering with the PKU community to explore the therapeutic potential of JNT-517 in PKU."

JNT-517 is the first in a pipeline of potential therapies discovered by 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.

"JNT-517 is a prime example of the power of Jnana's RAPID platform to identify binding sites on the most challenging of therapeutic targets," said Joel Barrish, Ph.D., Co-founder, President and Chief Scientific Officer of Jnana. "We are now actively applying RAPID against other well-validated but previously undruggable targets, particularly in immune-mediated diseases and cancer, to deliver transformative treatment options to patients in need."

Based on JNT-517's promising preclinical profile, Jnana plans to initiate a Phase 1 clinical study later this year to assess safety, pharmacokinetics, and pharmacodynamics of JNT-517 in healthy volunteers, followed by a PKU patient cohort to obtain key proof-of-concept data intended to support a subsequent registrational program.

Phenylketonuria (PKU) is an inherited metabolic disease 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. Inhibition of the SLC transporter SLC6A19, responsible for kidney reabsorption of Phe back into the bloodstream, may offer a novel approach for the treatment of PKU.

Jnana Therapeutics is a biotechnology company leveraging its next-generation chemoproteomics RAPID platform to discover medicines for highly validated, previously undruggable 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 lead program is a potential first-in-class oral approach, targeting an allosteric site on the phenylalanine transporter SLC6A19, 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. For more information, please visit www.jnanatx.com and follow us on Twitter and on LinkedIn.

SOURCE Jnana Therapeutics