Jnana Therapeutics has reported positive topline data from the Phase Ia clinical trial of JNT-517, a potential first-in-class oral treatment for phenylketonuria (PKU).
JNT-517 is a small molecule phenylalanine (Phe) transporter SLC6A19 inhibitor. SLC6A19 is responsible for kidney reabsorption of Phe back into the bloodstream and facilitates the excretion of excess Phe from the urine.
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The placebo-controlled, randomised, double-blind study evaluated the tolerability, safety, pharmacodynamics, and pharmacokinetics of JNT-517 in 64 healthy adults.
In the trial, it was observed that JNT-517 resulted in dose-dependent increases in urinary Phe across single and multiple ascending dose cohorts.
JNT-517 was found to be safe and well tolerated at all dose levels, with no serious adverse events.
The study also compared suspension and tablet formulations of JNT-517.
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By GlobalDataJnana Therapeutics co-founder and CEO Joanne Kotz said: “We are encouraged by the positive results from the Phase Ia portion of our first-in-human trial of JNT-517.
“These initial data are consistent with human genetics and preclinical data and confirm that JNT-517 inhibits SLC6A19 and results in the increased excretion of Phe at doses that are safe and well tolerated.
“Jnana’s RAPID platform enabled the identification of JNT-517, a first-in-class inhibitor acting at a cryptic allosteric site, validating RAPID’s ability to successfully identify leads for difficult-to-drug targets.”
Jnana’s ongoing JNT-517 Phase I programme was initiated in Australia in the fourth quarter of last year.
Jnana Therapeutics senior vice-president and development head John Throup said: “We look forward to partnering with the PKU community as we proceed into the Phase Ib portion of the trial in individuals with PKU, bringing us a step closer to potentially providing a first-in-class oral therapy for the treatment of PKU.”
The placebo-controlled, randomised, double-blind Phase Ib study intends to enrol participants with PKU at clinical sites in Australia and the US. It anticipates to start dosing in the third quarter of this year.
