Otsuka Pharmaceutical Co., Ltd. (Otsuka) announces that it has completed the acquisition of Jnana Therapeutics Inc. (Jnana), based in Boston, Massachusetts. Jnana has become a direct subsidiary of Otsuka America, Inc. (OAI), which is a fully owned subsidiary of Otsuka.
Based on the terms of the agreement signed in August of this year, Otsuka paid (U.S.) $800 million to the shareholders of Jnana as consideration for the acquisition of all outstanding shares of the company. In addition, Otsuka may pay up to (U.S.) $325 million to Jnana shareholders in development and regulatory milestone payments based on the progress of future development products.
Jnana utilizes an innovative drug discovery approach, the Reactive Affinity Probe Interaction Discovery (RAPID) platform, for a variety of drug targets on which it has been difficult to obtain small-molecule drugs through traditional screening systems. With its focus on identifying first-in-class compounds for autoimmune and rare diseases, Jnana is in a unique competitive position. In the autoimmune disease field, the company is pursuing small molecule drug discovery for highly validated targets such as interferon regulatory factor 3 (IRF3), a master transcription factor for the production of interferon.
In addition, using this drug discovery technology, Jnana developed JNT-517, an oral small-molecule inhibitor of a protein that regulates phenylalanine reabsorption in the kidneys, and is conducting a Phase 1b/2 study in patients with phenylketonuria (PKU), a rare, inherited metabolic disorder in which symptoms usually begin to appear in infancy. Earlier this month, Jnana presented the results of the Phase 1b/2 study at the Society for the Study of Inborn Errors of Metabolism (SSIEM) 2024 Annual Symposium . In addition to data for the 75-mg dose, new data for the 150-mg dose (both twice daily) were disclosed at this conference presentation.
Summary of P1b/2 study interim data
- A strong reduction in blood phenylalanine levels was observed within one week of the start of dosing, regardless of the severity of the disease or non-responsiveness to existing treatments
- Twice-daily oral treatment with JNT-517 at 150-mg dose delivered statistically significant mean blood phenylalanine reduction of 60% versus baseline in individuals with PKU
- JNT-517 was safe and well tolerated with no serious adverse events observed
JNT-517 has received orphan drug designation and rare pediatric disease designation from the U.S. FDA and orphan designation from the European Medicines Agency (EMA) for the treatment of PKU. JNT-517 has also been granted eligibility for the EMA PRIME (PRIority MEdicines) scheme for the treatment of hyperphenylalaninemia. Based on the data from this Phase 1b/2 study, the company is currently engaging in discussions with U.S. and European regulatory authorities and plans to initiate a Phase 3 study in early 2025.
