BOSTON– PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), a clinical-stage biotherapeutics company dedicated to changing the lives of patients with devastating diseases, today announced that KarXT (xanomeline and trospium chloride), which was initially invented and advanced by PureTech, has received U.S. Food and Drug Administration (“FDA”) approval for the treatment of schizophrenia in adults. The FDA approval triggers two separate milestone payments to PureTech totaling $29 million under agreements with Royalty Pharma and PureTech’s Founded Entity, Karuna Therapeutics, which was acquired by Bristol Myers Squibb (NYSE: BMY) (“BMS”) in March of 2024. Under these agreements, PureTech is also entitled to potential future payments related to additional milestones as well as approximately 2% royalties on net annual sales over $2 billion. Following the acquisition of Karuna, KarXT is now under the stewardship of BMS and will be marketed as Cobenfy.
Cobenfy was invented at PureTech by combining two biologically active molecules – xanomeline and trospium chloride – to address a tolerability challenge that had held back a potential new class of medicines for the treatment of neuropsychiatric conditions, such as schizophrenia. Consistent with its unique model of drug development, PureTech advanced Cobenfy by founding Karuna Therapeutics, which later became a publicly traded company on Nasdaq.
Eric Elenko, PhD, Co-founder and President of PureTech said: “The FDA approval of Cobenfy is a significant milestone in our mission to transform the lives of patients with devastating diseases. Our initial hypothesis was that we could overcome the tolerability issues that had hindered the development of an otherwise promising drug, xanomeline, and we were able to test and validate this concept early on. We are immensely proud that our dedication to this program has led to the first major innovation in decades for those living with schizophrenia, and I am equally pleased that our unique approach to R&D has delivered yet another novel therapeutic to patients. Congratulations to the teams at Karuna and BMS on this historic accomplishment.”
The FDA approval of Cobenfy is further validation of PureTech’s model and a hallmark of how it creates value both clinically and financially. PureTech’s monetization of equity holdings in Karuna, including gross proceeds from the BMS acquisition of Karuna, and a strategic royalty agreement with Royalty Pharma have enabled PureTech to generate approximately $1.1 billion to date after directing $18.5 million toward Karuna’s founding and Cobenfy’s development. PureTech’s business model is designed to repeat and scale this type of outcome, and proceeds from the success of Cobenfy have enabled PureTech to self-fund the advancement of several programs – including LYT-100 (deupirfenidone), LYT-200 (anti-galectin-9 mAb), and the GlyphTM platform supporting the pipeline of Seaport Therapeutics.
Bharatt Chowrira, PhD, JD, Chief Executive Officer of PureTech said: “Congratulations to the Karuna and BMS teams for delivering a groundbreaking treatment to people with schizophrenia. The FDA approval of Cobenfy is a testament to our unique R&D engine, which has now produced three FDA approved therapeutics. We’ve applied this approach across our portfolio, from our late-stage Internal Program LYT-100 (deupirfenidone) to our newly launched Founded Entity, Seaport Therapeutics, and we will continue to leverage this successful drug development model as we enter our next phase of innovation.”
PureTech’s next wave of innovation continues to focus on validated biologic and small molecule modalities with human clinical data in diseases with significant unmet need. LYT-100 (deupirfenidone) is PureTech’s wholly-owned program in development for the treatment of idiopathic pulmonary fibrosis (IPF), a rare progressive lung disease with no cure. The LYT-100 program leverages extensive prior clinical data and follows the same blueprint used with Cobenfy to unlock the full therapeutic potential of an efficacious but poorly tolerated medicine. PureTech anticipates topline data from the Phase 2b clinical trial of LYT-100 in patients with IPF by the end of the year, as well as additional readouts from its oncology program, LYT-200 (anti-galectin-9 monoclonal antibody).
