FDA accepts Jnana Therapeutics' IND application for metabolic disorder drug

Jan. 6, 2023

Boston-based biotech Jnana Therapeutics announced this week that the FDA had given clearance to its Investigational New Drug (IND) application for a potential first-in-class oral therapy for Phenylketonuria (PKU).

Patients with PKU suffer from an inherited deficiency of phenylalanine hydroxylase, an enzyme that is required for the breakdown of phenylalanine, found in protein-containing foods. This causes a dangerous accumulation of Phe in the blood, and if left untreated can result in neurotoxic complications. 

JNT-517 is Jnana’s wholly-owned lead program, and it offers a new approach to reducing blood levels of Phe, by directly inhibiting reabsorption back into the bloodstream. The drug is a small-molecule inhibitor of the phenylalanine transporter SLC6A19, and works as a cryptic allosteric site that helps block kidney reabsorption of the enzyme. 

The biotech has had an ongoing phase 1 program of the drug in Australia in healthy individuals, and the program will now expand to include a sub-study to evaluate JNT-517 in individuals with PKU. 

Founded in 2017, Jnana has focused on developing its next-generation chemoproteomics RAPID platform to “discover medicines for highly validated, challenging-to-drug targets to treat diseases with high unmet need.” PKU for example, affects only 1 in every 10,000 to 15,000 babies born in the United States. The biotech has received funding from leading life science investors like Pfizer Ventures, and AbbVie Ventures.