FDA Approves PureTech's KarXT for Adult Schizophrenia Treatment

PureTech Health plc, a clinical-stage biotherapeutics company, has announced the U.S. Food and Drug Administration (FDA) approval of KarXT (xanomeline and trospium chloride) for treating schizophrenia in adults. Initially developed by PureTech, this approval activates two milestone payments totaling $29 million to the company under agreements with Royalty Pharma and Karuna Therapeutics, a company that PureTech founded and which was acquired by Bristol Myers Squibb (BMS) in March 2024. KarXT will be marketed under the name Cobenfy by BMS.

Cobenfy was developed by PureTech by combining two active molecules, xanomeline and trospium chloride, to address prior tolerability issues with xanomeline, a promising drug for treating neuropsychiatric conditions like schizophrenia. PureTech advanced the development by establishing Karuna Therapeutics, which later became a publicly traded company.

Eric Elenko, PhD, Co-founder and President of PureTech, emphasized the importance of the FDA approval, highlighting it as a significant step in their mission to transform patient lives. Elenko remarked on the challenge of overcoming tolerability issues that had previously hindered xanomeline, expressing pride in the program’s success and the company’s unique research and development model.

The approval of Cobenfy underscores the effectiveness of PureTech’s model, which generates both clinical and financial value. PureTech has monetized its equity in Karuna, obtaining approximately $1.1 billion, including proceeds from the BMS acquisition. This business model has enabled PureTech to fund its own programs, such as LYT-100 (deupirfenidone), LYT-200 (anti-galectin-9 mAb), and the GlyphTM platform for Seaport Therapeutics.

Bharatt Chowrira, PhD, JD, CEO of PureTech, congratulated the Karuna and BMS teams, noting that the FDA approval exemplifies PureTech’s successful R&D approach, which has now resulted in three FDA-approved therapeutics. Chowrira highlighted PureTech’s ongoing commitment to leveraging this model for future innovations.

Looking ahead, PureTech aims to continue focusing on validated biologics and small molecules with clinical data for diseases with significant unmet needs. LYT-100 (deupirfenidone) is in development for idiopathic pulmonary fibrosis (IPF), a rare and progressive lung disease. PureTech expects to release topline data from the Phase 2b trial of LYT-100 in IPF patients by the end of the year, alongside additional data from its oncology program, LYT-200 (anti-galectin-9 mAb).

Cobenfy, which combines xanomeline and trospium chloride, has specific safety information and contraindications. It is not recommended for patients with urinary retention, moderate to severe hepatic impairment, and certain other conditions. The drug may cause urinary retention, increased heart rate, and other adverse reactions, particularly in patients with renal impairment or narrow-angle glaucoma. Patients are advised to monitor for symptoms and consult healthcare providers as needed.

PureTech Health continues to drive the development of new therapeutics, with its extensive pipeline of 29 programs, three of which have received FDA approval. PureTech’s team, along with its network of scientists and industry leaders, remains focused on advancing therapeutic candidates through various clinical stages.

PureTech Health is dedicated to creating new classes of medicines to improve the lives of patients with severe diseases, leveraging its robust R&D engine and strategic partnerships to achieve clinical and financial success.