Pano Therapeutics Advances PAN1195 Toward Clinical Development in Fragile X Syndrome
June 1, 2026 — Pano Therapeutics today provided an update on its lead program, PAN1195, a CNS-optimized biguanide being developed for Fragile X Syndrome. PAN1195 has been designed to redirect the clinically validated biguanide (metformin-class) pharmacology into the brain, with the goal of supporting a disciplined biomarker-led path into early clinical development.
In Fragile X Syndrome, loss of the FMRP protein leads to dysregulated synaptic translation through hyperactivation of the ERK–mTOR–eIF4E pathway. Biguanide pharmacology converges on this same translation-control axis. Published preclinical work has shown that metformin, the prototypical biguanide, can rescue core phenotypes in mouse models of Fragile X Syndrome and normalize ERK signaling, eIF4E phosphorylation, and FXS-relevant downstream targets. Published randomized clinical experience with metformin in Fragile X Syndrome has further supported the class-level proof of biology underlying Pano’s approach.
PAN1195 is being developed with the intent to improve on the metformin precedent through transporter-guided design for stronger and more consistent brain exposure, a once-daily oral pharmacokinetic profile, and reduced dependence on renal elimination. Pano pairs this differentiated profile with cross-species quantitative EEG and pharmacodynamic biomarkers, which the company intends to use to support objective target engagement, dose selection, and capital-efficient early clinical learning.
Beyond Fragile X Syndrome, Pano sees expansion potential across a biologically related cluster of syndromic neurodevelopmental disorders, beginning with Phelan-McDermid Syndrome and Tuberous Sclerosis Complex, which share translation-control dysregulation, inhibitory-network biology, and EEG biomarker tooling with Fragile X Syndrome.
“Fragile X Syndrome is the right lead indication for a CNS-optimized biguanide because the biology, the development pathway, and the clinical precedent are unusually well aligned,” said Ben Gibson, Chief Executive Officer and Co-founder of Pano Therapeutics. “PAN1195 is being advanced with an explicit focus on objective biomarker readouts and disciplined translational science, in service of patients and families for whom no approved core-symptom therapy currently exists.”
About Pano Therapeutics
Pano Therapeutics is a preclinical-stage biotechnology company developing CNS-optimized biguanides for neurodevelopmental disorders. The company’s lead candidate, PAN1195, is being advanced toward clinical development in Fragile X Syndrome, with planned expansion in Phelan-McDermid Syndrome and Tuberous Sclerosis Complex. Pano’s development strategy is anchored in cross-species quantitative EEG and pharmacodynamic biomarkers intended to support objective target engagement and disciplined clinical learning. For more information, visit www.panotherapeutics.com.
Media Contact
Ben Gibson, CEO — ben@panotherapeutics.com