With an upfront $800 million payment and $325 million in potential milestones, Otsuka Pharmaceutical is acquiring Jnana Therapeutics’ drug discovery platform and rare disease candidate, the companies announced Thursday.
Otsuka Pharmaceutical has struck a deal to buy Jnana Therapeutics for $800 million upfront and a potential $325 million in milestone payments for its drug discovery platform and pipeline led by a clinical-phase rare disease candidate, the companies announced Thursday.
Jnana discovers potential drug candidates by exposing protein targets to its library of chemical fragments inside living cells. The Boston-based biotech has designed the library to include a wide range of fragments that may bind and covalently label druggable pockets on the target protein. After finding a fragment that binds a target, Jnana screens a library of drug-like compounds to identify small molecules that engage the same part of the protein.
Otsuka’s statement on the reasons for the acquisition discussed Jnana’s platform before mentioning the drug candidates it has generated. The Japanese drugmaker said the platform can address hard-to-drug target classes including solute carriers, transcription factors and signaling scaffold proteins. Otsuka sees the platform as complementary to the capabilities of its existing subsidiary Astex Pharmaceuticals. Jnana, like Astex, will operate as a wholly owned subsidiary of Otsuka and will continue its R&D.
The biotech has used its platform to find small molecules that inhibit SLC6A19, a transporter responsible for intestinal absorption and renal reabsorption of phenylalanine (Phe). People with phenylketonuria (PKU) are unable to break down Phe.
Inhibiting SLC6A19 could enhance urinary excretion of Phe, helping to reduce levels of a molecule that accumulates in PKU patients and drives symptoms including brain damage. Jnana is putting that idea to the test in a Phase Ib trial. After 28 days of twice daily dosing, the biotech saw a 51% reduction in eight patients on its SLC6A19 inhibitor JNT-517. The reduction was statistically significant versus placebo.
Based on the interim results, Jnana adapted its study to include dose exploration and outlined plans to move into a Phase III trial in the first half of 2025. If the pivotal study is successful, JNT-517 could challenge BioMarin’s Kuvan (sapropterin dihydrochloride) and Palynziq (pegvaliase-pqpz) for the market. Sanofi is developing a PKU gene therapy and Moderna is testing a mRNA candidate in a Phase I/II trial.
Otsuka is acquiring a biotech that was on the radar of multiple top pharma companies. Jnana launched in 2017 with a $50 million financing round and plans to build the first drug discovery platform dedicated to targeting the solute carrier family of transporters. The venture capital wings of AbbVie and Pfizer participated in the financing, as well as in at least one of two subsequent rounds that collectively raised an additional $157 million.
Jnana partnered with Roche in 2020, receiving $40 million upfront in a multi-target deal, and expanded its collaboration with the Swiss drugmaker in 2022. Roche paid $50 million upfront to form the second collaboration.
