MedPath

Spinogenix Targets Synaptic Regeneration with Small Molecule Drugs for Neurological Diseases

• Spinogenix is developing SPG302 and SPG601, orally administered small molecules, to restore synapses in neurological diseases like ALS, Alzheimer's, schizophrenia, and Fragile X syndrome. • SPG302, a Transient Activator of Glutamatergic Synaptogenesis (TAG), stimulates new synapse formation using glutamate and is currently in Phase 2 trials across multiple indications. • Spinogenix anticipates multiple Phase 2 readouts across disease areas in 2025, with potential for SPG302 as a standalone or cocktail therapy due to its safety profile. • The company collaborates with academic institutions and utilizes grant funding to de-risk development, focusing on first-in-class treatments for high unmet needs.

Spinogenix, a Los Angeles-based company, is advancing two clinical candidates, SPG302 and SPG601, to address synaptic dysfunction in neurological diseases. These orally administered small molecules aim to restore synapses in diseases such as amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, schizophrenia, and Fragile X syndrome. The company's approach focuses on synaptic regeneration, a novel target in neurodegenerative and neurodevelopmental conditions.

SPG302: A Novel Approach to Synaptic Regeneration

SPG302, discovered internally, is part of Spinogenix’s TAG (Transient Activators of Glutamatergic Synaptogenesis) platform. This technology stimulates the formation of new synapses, utilizing glutamate as a neurotransmitter. Currently, SPG302 is being evaluated in Phase 2 clinical trials for ALS, Alzheimer’s disease, and schizophrenia. These trials aim to optimize dosing and establish objective measurements of efficacy.
Stella Sarraf, Ph.D., founder of Spinogenix, highlighted the potential of targeting synaptic regeneration in ALS, stating, "The beauty of working at the synaptic level, especially in ALS, is that no one else has worked even in that zip code. It’s completely new."

SPG601: Targeting Synaptic Dysfunction in Fragile X Syndrome

SPG601, in-licensed from academia, is a small molecule BK channel activator designed to correct synaptic dysfunction associated with Fragile X syndrome. This candidate is currently in a Phase 2 trial for Fragile X syndrome and has received orphan drug designation.

Biomarkers and Clinical Validation

Spinogenix is leveraging technologies like electroencephalography (EEG) and transcranial magnetic stimulation (TMS) to objectively measure synapses and validate drug effectiveness. These tools serve as biomarkers, providing crucial data for clinical trials and regulatory submissions.

Strategic Collaborations and Funding

Spinogenix has benefited from collaborations with academic institutions and grant funding from the NIH and Department of Defense. This support has enabled the company to de-risk its programs and conduct extensive animal studies. Sarraf noted, "All of our animal work was done by others [externally]," emphasizing the value of these collaborations.

Anticipated Milestones

Spinogenix anticipates multiple Phase 2 readouts across disease areas in 2025. The company believes that SPG302’s safety profile could position it as a standalone therapy or as part of a combination therapy. The development of a safe and effective orally available treatment would be a significant advancement for patients with these debilitating conditions.
In October 2024, Spinogenix launched an additional program in glaucoma for SPG302, further expanding its pipeline and therapeutic potential.
Subscribe Icon

Stay Updated with Our Daily Newsletter

Get the latest pharmaceutical insights, research highlights, and industry updates delivered to your inbox every day.

Related Topics

Spinogenix's SPG601 Shows Promise in Phase 2 Trial for Fragile X Syndrome

Spinogenix's SPG601 demonstrated a significant reduction in high-frequency gamma band activity, an EEG abnormality associated with Fragile X syndrome (FXS).
The Phase 2 trial met its primary endpoint, suggesting SPG601's potential to improve brain activity related to learning and memory in FXS patients.

Neurogene's Gene Therapy Shows Promise for Rett Syndrome Amid Clinical and Market Risks

Neurogene Inc., a clinical-stage biotechnology company, is making strides in the treatment of Rett syndrome with its lead product, NGN-401, currently in Phase 1/2 clinical trials. Despite facing clinical trial risks and competition, the company's innovative gene therapy approach and EXACT platform technology present significant market opportunities and potential for future growth.

MeiraGTx's AAV-GAD Gene Therapy Shows Promise in Parkinson's Disease Phase I/II Trial

MeiraGTx's AAV-GAD gene therapy met its primary endpoint, demonstrating safety and tolerability in a Phase I/II trial for Parkinson's disease.
The high-dose group showed a statistically significant 18-point improvement in the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part III score.

Spinogenix's SPG302 Enters Phase 2 Trial as Novel Schizophrenia Treatment

Spinogenix has initiated a Phase 2 clinical trial to evaluate SPG302, a novel synaptic regenerative therapy, for the treatment of schizophrenia.
SPG302 aims to restore glutamatergic synapses, addressing a key feature of schizophrenia pathogenesis linked to positive, negative, and cognitive symptoms.

SciSparc Advances Cannabinoid-Based Therapies for CNS Disorders, Including Autism

SciSparc has enrolled the first patient in its clinical trial for SCI-210, a cannabinoid-based treatment for Autism Spectrum Disorder (ASD) in children.
The company's pipeline includes SCI-110 for Tourette Syndrome and Alzheimer's agitation, and SCI-160 for pain management, all leveraging cannabinoid pharmaceuticals.

Gene Therapy Shows Promise for Rare Neurological Disorder SPG50

A Canadian family's relentless pursuit led to a breakthrough gene therapy for Spastic Paraplegia 50 (SPG50), a rare neurological disorder.
The gene therapy, developed with international collaboration, has shown significant improvements in treated children's movement, coordination, and speech.

FDA Grants Orphan Drug Status to Spinogenix's SPG601 for Fragile X Syndrome

Spinogenix, Inc. has received Orphan Drug Designation from the FDA for its drug candidate SPG601, aimed at treating Fragile X Syndrome (FXS), a condition with no current FDA-approved therapies. SPG601 targets synaptic dysfunctions by modulating BK channels, offering hope for improving the quality of life for those affected by FXS.

Spinogenix Receives FDA Orphan Drug Designation for SPG601 to Treat Fragile X Syndrome

Spinogenix, Inc. has been granted Orphan Drug Designation by the U.S. FDA for SPG601, a novel treatment for Fragile X syndrome (FXS), highlighting the urgent need for new therapies. FXS, a leading cause of inherited intellectual disability and autism, lacks FDA-approved treatments. SPG601 aims to address core symptoms by modulating BK channels to restore synaptic function.

Two Major Breakthroughs in Fragile X Syndrome Treatments

Recent advancements in Fragile X Syndrome (FXS) treatments include Spinogenix's FDA approval for a Phase II clinical trial of SPG601 and the European Commission granting orphan drug status to Shionogi’s zatolmilast. These developments aim to address the significant unmet need for effective treatments in FXS, a condition causing developmental problems and cognitive impairment.

Advancements in Neurology: From Hunter Syndrome to Alzheimer's Disease

Recent studies and trials in neurology have shown promising results across a range of conditions, including Hunter syndrome, Alzheimer's disease, and Dravet syndrome. Innovations in treatment and diagnostic tools are paving the way for more effective management of these complex conditions.

Reference News

[1]
Companies To Watch Spinogenix - Life Science Leader
lifescienceleader.com · Oct 18, 2024

Spinogenix, a small molecule drug discovery company, is testing SPG302 in ALS, Alzheimer’s, and schizophrenia, and SPG60...

© Copyright 2025. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy