Synaptogenix Gets FDA IND Approval for Bryostatin-1 MS Trial

Synaptogenix, Inc., an emerging biopharmaceutical company focused on developing treatments for neurodegenerative diseases, recently announced that the Food & Drug Administration (FDA) has approved an Investigational New Drug (IND) application for Bryostatin-1, which is being evaluated as a potential therapy for multiple sclerosis (MS). Bryostatin-1 is a small molecule that activates protein kinase C (PKC) enzymes, which play a crucial role in maintaining synaptic health and are essential for learning and memory. The therapy might also enhance anti-inflammatory efficacy, which could prevent the degradation of the insulating sheath around nerve fibers.

The upcoming clinical trial, which is fully funded, will be conducted at the Cleveland Clinic Neurological Institute's Mellen Center for Multiple Sclerosis. This open-label study will employ cutting-edge 7-tesla (7T) magnetic resonance imaging (MRI) technology to identify biomarkers that will help in evaluating Bryostatin-1's impact on the brain. Dr. Robert Fox, Vice-Chair for Research at the Neurological Institute of Cleveland Clinic and the principal investigator for the trial, highlighted the significance of using advanced MRI technology in conjunction with behavioral and cognitive measures to assess Bryostatin-1's effects. He expressed optimism that the research and development of this drug could offer potential benefits for MS patients.

The study aims to address the often overlooked issue of cognitive decline in MS patients. Dr. Alan Tuchman, Chief Executive Officer of Synaptogenix, pointed out that cognitive impairment mechanisms in MS remain poorly understood and inadequately studied in clinical trials. He emphasized the importance of this study in addressing an unmet need with minimal expense and indicated that the company looks forward to enrolling and dosing the first patient soon.

Preclinical studies have shown that Bryostatin-1, through its activation of PKC enzymes, can prevent synapse loss and neuronal death, reduce inflammation, and mitigate the onset of neurological deficits. Dr. Daniel Alkon, President and Chief Scientific Officer of Synaptogenix, noted that these preclinical data suggest promising potential for Bryostatin-1 as a treatment for MS, particularly for its progressive forms.

Synaptogenix is in a strong financial position, with approximately $26.3 million in cash as of March 31, 2024. This solid balance sheet ensures that the Phase 1 MS trial is fully funded. The company has a relatively low cash burn rate and has adequate resources to continue the development of Bryostatin-1 for MS and other indications. Additionally, the company plans to pursue research on other innovative assets and explore potential acquisitions of asset rights.

Synaptogenix focuses on developing novel therapies for neurodegenerative diseases. The company has conducted both clinical and preclinical studies on its lead therapeutic candidate, Bryostatin-1, particularly in the context of Alzheimer’s disease. Preclinical research has also demonstrated bryostatin's regenerative mechanisms for treating Fragile X syndrome and other neurodegenerative disorders, including MS, stroke, and traumatic brain injury. The U.S. Food and Drug Administration has granted Orphan Drug Designation to Bryostatin-1 for treating Fragile X syndrome. Bryostatin-1 has already been tested in over 1,500 individuals in cancer studies, providing a substantial safety database to inform future clinical trial designs.