Spinogenix is a clinical-stage company that is addressing the unmet needs for therapeutics that reverse synaptic loss and dysfunction with its synaptic regenerative drug, tazbentetol, and synaptic corrective drug, SPG601.
Tazbentetol is a first-in-class oral small molecule proven to regrow brain synapses, the vital connections between brain cells. Clinical data for Alzheimer’s disease showed rapid, significant improvements in memory and thinking, and for ALS a clinically meaningful slowing of disease progression. Tazbentetol is also being evaluated in schizophrenia, with a Phase 2 trial currently underway. Tazbentetol has been granted Orphan Drug Designation by both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) for ALS, and Spinogenix has established an Expanded Access Program to provide up to 200 eligible ALS patients access to the investigational therapy who do not qualify for clinical trials.
SPG601 is a first-in-class oral small molecule proven to normalize a signature brain abnormality in Fragile X Syndrome (FXS), the leading inherited form of intellectual disability and a known cause of autism for which there are currently no approved treatments. Clinical data showed that SPG601 corrected abnormal patterns of brain activity and improved attention and focus, a key area of difficulty for people living with FXS. A positive Type C meeting with the FDA has provided a clear regulatory path forward, and SPG601 is now advancing toward late-stage development for this rare disease. SPG601 has been granted Orphan Drug Designation by both the FDA and EMA, as well as FDA Fast Track Designation, for FXS.
