A Platform of Novel Synaptic Regenerative Investigational Therapies Designed to Restore Neuronal Function

For conditions such as Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), schizophrenia, glaucoma, and Fragile X Syndrome (FXS), there remains a critical gap in effective and patient-friendly solutions. At Spinogenix, we want to do more than slow disease progression. Patients deserve new therapies that offer renewed hope. Our unique approach has the potential to be used alone as a “monotherapy” or in combination with approved therapies.

Spinogenix is pioneering a new way to treat conditions characterized by the loss or dysfunction of the fundamental building blocks in the brain called synapses, specifically, the connections between neurons allowing us to think, plan, remember, see, and control motor functions. Our novel small molecules have the ability to restore neuronal synapses, with the potential to reverse cognitive and motor function decline – either as standalone treatments or in combination with existing approved therapies.

What Are Brain Synapses and Why Are They Important?

Brain Synapses are connections between neurons through which signals flow from one neuron to another. They are a fundamental and indispensable physical basis for neural communication, enabling cognition, movement, perception, memory, and other faculties of mind. Deterioration of brain synapses is an early and progressive feature of a broad spectrum of neurodegenerative, neuropsychiatric, and other diseases collectively known as the “synaptopathies”. For any investigational therapy to have a chance at reversing the symptoms of these diseases in patients, it must promote the restoration of brain synapses.

Alzheimer’s Disease (AD)

Alzheimer’s disease is the most common cause of dementia, accounting for approximately 60–70% of dementia cases worldwide and leading to progressive cognitive decline and loss of independence1.

Amyotrophic Lateral Sclerosis (ALS)

ALS, also called Lou Gehrig’s disease, is a neurodegenerative disease that results in the progressive loss of motor neurons that control voluntary muscles.

Schizophrenia (SCZ)

Schizophrenia is one of the most debilitating and prevalent psychiatric conditions, affecting around 24 million people worldwide.

Fragile X syndrome (FXS)

Fragile X syndrome is a neurodevelopmental disorder caused by full mutation and methylation of the FMR1 gene on the X chromosome9,10.

Glaucoma

Glaucoma is the leading cause of permanent blindness, affecting around 80 million people globally, including over 3 million in the U.S.

*To find references 1, 9, 10, click the accompanying Read More About tab

The Benefits of a Tablet

Many emerging therapies for neurodegenerative diseases such as ALS and Alzheimer’s disease are large macromolecules called “biologics”. These biologics have difficulty penetrating the central nervous system (CNS) and require IV infusion or intrathecal injections. Such therapies, which include antibodies that bind to toxic proteins or oligonucleotides that suppress the expression of a disease-related gene product, are difficult to administer and costly to produce at scale.

At Spinogenix, we develop small molecules designed to cross the blood-brain barrier. Tazbentetol and SPG601 are designed to be taken by mouth as a once-a-day pill, which has multiple benefits: easier administration, better patient compliance, and significantly lower cost. Together, they represent a potentially simple yet powerful approach to treating synaptopathies.

Regenerating Synapses – Tazbentetol and the TAGS Technology Platform

Tazbentetol is Spinogenix’s first-in-class synaptic regenerative therapy – designed not to mask symptoms, but to regrow the brain connections lost to disease. Built on the TAGS™ (Transient Activators of Glutamatergic Synapses) platform, tazbentetol triggers neurons to produce new, functional synapses. Key features of this regenerative approach include:

Rapid Acting

Tazbentetol induces the formation of new, functional dendritic spine synapses within hours in laboratory studies, while synaptic and behavioral benefits are evident within weeks of daily investigational treatment in animal models.

Sufficient Transient Exposure

Dosing is daily and the half life in the blood is short as the investigation therapeutic is targeted to synaptic regeneration and subsequently improvements in cognitive and motor symptoms across multiple animal models, including ALS, Alzheimer’s, spinal cord injury, and traumatic brain injury.

Self-Perpetuating and Self-Limiting

Tazbentetol activates a process that carries forward on its own to regenerate physiologically relevant numbers and shapes of dendritic spine synapses. Investigational treatment with higher doses than necessary, or for longer periods, does not alter this activity profile.

Durable Effects

New synapses induced by tazbentetol may persist even after treatment is stopped.

Correcting Synapses – SPG601 and BK Channel Modulation for Fragile X Syndrome

FXS is the leading inherited form of intellectual disability and a known cause of autism, resulting from the silencing of the Fmr1 gene. This leads to widespread disruptions in synaptic function, linked in part to reduced activity of large-conductance, calcium-activated potassium (“BK”) channels.

SPG601 is a small molecule that works at the synaptic level, targeting a well-established molecular dysfunction in FXS, to improve core symptoms and challenging behaviors, which can include severe anxiety, social aversion, hyperactivity and attention deficit. SPG601 targets large-conductance, calcium-activated potassium (“BK”) channels, increasing their activation to correct specific synaptic dysfunctions that are thought to underlie many core symptoms of FXS.