SPINOGENIX is pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide.
Unique Approaches for Conditions Involving the Loss or Dysfunction of Synapses
Patent-protected compound
Spinogenix has designed small molecules to help restore the brain connections (synapses) in neurodegenerative, neuropsychiatric and neurodevelopmental conditions including amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, schizophrenia, Fragile X syndrome and many others.
Reverse of the synaptic degeneration process
Spinogenix has demonstrated SPG302 as an innovative treatment for regenerating synapses to reverse declines in cognitive and motor function, which is fundamentally different from other therapeutics that aim to slow degeneration.
Once-a-day tablet
Spinogenix has developed SPG302 as a once-a-day tablet, which is significantly different from recently approved antibody therapies for Alzheimer’s disease that require IV infusion by a healthcare professional.
Regenerative Platform Approach
Multiple NIH and DOD grants have been awarded to support our unique treatment for Alzheimer’s and ALS patients. This approach leverages our TAGS™ technology to trigger the formation of new synaptic connections, in effect regenerating synapses that have been lost.
Current Treatments Leave a Critical Gap in Effective and Patient-Friendly Solutions
Our unique approaches aim to have a transformative impact on patient outcomes while avoiding burdens imposed by many current therapies for conditions involving synapse loss.
SPG302 Phase 2 Clinical Trials in ALS, Alzheimer’s Disease, and Schizophrenia
SPG302 is the first synaptic regenerative therapy to be tested in ALS, Alzheimer’s and schizophrenia.
Spinogenix has completed its first-in-human Phase 1 study evaluating the safety, tolerability, and pharmacokinetics of SPG302 in healthy volunteers. The Company successfully completed Phase 2A trials in ALS (NCT05882695) and in Alzheimer’s Disease (NCT06427668) with ongoing special access scheme in AUS. The FDA-authorized an expanded access program for 200 people in ALS in the US. A Phase 2 trial in Schizophrenia (NCT06442462) is currently enrolling in the US.
FDA & EMA have granted SPG302 Orphan Drug Designation in ALS.
SPG601 Completed Phase 2 Clinical Trial in Fragile X Syndrome
SPG601 is a novel BK channel positive modulator designed to correct a specific synaptic dysfunction in Fragile X Syndrome (FXS), the leading inherited form of intellectual disability and known cause of autism. There are no FDA-approved treatments for FXS.
Spinogenix completed a Phase 2a trial for SPG601 in FXS patients at Cincinnati Children’s Hospital and recently announced positive type C meeting with the FDA providing clear path forward on the overall design of the registrational directed trial of SPG601 in people with FXS.
The U.S. FDA and EMA have granted SPG601 Orphan Drug Designation in FXS and the FDA has awarded fast track designation in FXS.
To my knowledge, this is the first clinical trial in ALS focused on regenerating synapses with a small molecule. It has the potential to be used in combination with many other treatments approved and in development.
-Dr. Merit cudkowicz, director of the Sean m. healey &
amg center for ALS at massachusetts general hospital
