Lexicon Pharmaceuticals (LXRX) used the Citi Biopharma Back to School Summit 2026 to provide an update on its clinical pipeline, emphasizing that data from the Phase III SONATA-HCM trial in hypertrophic cardiomyopathy is targeted for the first quarter of 2027.
CEO Mike Exton referred to the HCM readout as the company’s “North Star” event. The trial has enrolled over 500 patients, exceeding its original enrollment plan, and combines both obstructive and non-obstructive HCM populations into a single primary analysis group. More non-obstructive patients were enrolled than obstructive ones, according to management.
Sotagliflozin will also be evaluated in a small cohort of patients simultaneously taking cardiac myosin inhibitors, a dual-therapy arm that makes SONATA-HCM the sole trial with this design feature. The primary endpoint is improvement in Kansas City Cardiomyopathy Questionnaire (KCCQ) score, with the study powered for a 4-point to 5-point placebo-adjusted benefit. Management said a statistically significant 3-point gain would constitute a commercial success. Secondary endpoints include New York Heart Association functional class, while peak VO2 serves as a co-primary endpoint outside the United States.
On the type 1 diabetes front, Lexicon plans to resubmit its regulatory application for sotagliflozin in the fourth quarter of 2024. The Steno-2-1 study, conducted in Denmark, enrolled 2,000 patients in a treatment-algorithm design, with roughly 1,000 placed in an enhanced-treatment group that included sotagliflozin alongside options such as semaglutide. Lexicon met the FDA-agreed sotagliflozin exposure target by the end of August 2024, and observed diabetic ketoacidosis rates were lower than those seen in the historic inTandem registration program and comparable to the standard-of-care group in Steno-2-1. Preparation involved a more-than-nine-month collaborative process with the FDA and the Steno-2 study team.
Pilavapadin, an AAK1 inhibitor being developed for diabetic peripheral neuropathic pain, is phase III-ready with its dose already determined, though the program was temporarily deprioritized relative to the HCM readout to align resource allocation. Preclinical data on AAK1 in additional cardiometabolic indications are scheduled for presentation later in 2024.
Lexicon also expanded on its partnership with Novo Nordisk around LX9851, an ACSL5 inhibitor targeting obesity, MASH, and lipid management through early triglyceride metabolism in the gut and liver. The deal was described as “very lucrative” for a preclinical asset and has expanded Lexicon’s financial flexibility, reducing pressure for dilutive financing or asset sales. Novo Nordisk views LX9851 as mission-critical and is advancing it as a once-daily oral combination therapy.
Additionally, Lexicon disclosed plans to implement AI tools to mine its Genome5000 database, which contains knockout work across approximately 5,000 genes spanning 60 to 70 therapeutic areas, including oncology, metabolism, cardiovascular disease, and immunology, with origins dating back roughly three decades.
Shares of Lexicon fell 3.14% to $2.16 on the day of the summit, down from a previous close of $2.23. The stock trades within a 52-week range of $1.06 to $2.68 and carries a market capitalization of approximately $963 million.
