Otsuka Pharmaceutical has picked up Boston-based Jnana Therapeutics for $800 million so the Japanese biotech can get its hands on a clinical-stage oral phenylketonuria (PKU) drug.
Under the terms of the deal, which is set to close in the third quarter of the fiscal year, Jnana’s shareholders will also be in line for up to an additional $325 million in development and regulatory milestone payments.
At the heart of the deal is JNT-517, an allosteric small-molecule inhibitor of SLC6A19, a solute carrier that regulates amino acid reabsorption in the kidney. The drug has already passed a phase 1b/2 trial to demonstrate its tolerability, and Otsuka sees potential for JNT-517 to become a first-in-class oral treatment for PKU.
PKU is a rare inherited metabolic disorder in which an amino acid called phenylalanine accumulates in the blood, leading to abnormally high levels. Most patients with the condition are not effectively served by existing therapies, according to Otsuka, meaning JNT-517 “is an approach that could address individuals of all ages across the spectrum of mild to severe disease.”
Now, the aim is to get JNT-517 into a registrational study next year.
“I am gratified that Otsuka has entered into an agreement with Jnana,” Makoto Inoue, Otsuka’s president and representative director, said in the Aug. 1 release.
“The addition of Jnana's drug discovery technology and small molecule pipeline in PKU and autoimmune diseases will strengthen our R&D in the Boston area of the U.S., one of the most important bioclusters in the world, and in a combined form will have a synergistic effect on Otsuka Pharmaceutical's global expansion,” Inoue added.
Otsuka isn’t the first biopharma to take an interest in Jnana. Roche penned two partnerships with the U.S. biotech, included a $2 billion biobucks deal to contribute discovery and preclinical work on multiple targets spanning cancer, immune-mediated diseases and neurology.
Other players are also dabbling in PKU, but it has proved to be a tricky indication. In February, Synlogic laid off 90% of its staff after its lead PKU drug appeared on track to fail a phase 3 trial.
PTC Therapeutics appeared to have more success last year in a phase 3 trial in PKU. However, the design of the study meant analysts remained unsure about the strength of PTC’s hand and whether its drug sepiapterin could rival BioMarin’s approved PKU drug Kuvan. PTC’s approval application for sepiapterin was later brushed aside by the FDA, which demanded an additional mouse study, with the drug only refiled with the regulator this week.
Days earlier, Sanofi revealed that it had removed an AAV-based gene therapy for PKU from its phase 1 pipeline.