Synaptogenix, Nemours Strike Deal on Bryostatin-1 Trial

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by Marisa Wexler MS |

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Synaptogenix and Nemours A.I. DuPont Hospital for Children have signed an agreement to collaborate on a clinical trial to test the investigational medication bryostatin-1 in people with fragile X syndrome (FXS).

“We are excited to participate in a potentially breakthrough therapeutic drug strategy to address this huge unmet medical need,” Mary M. Lee, MD, said in a press release. Lee is chief scientific officer and executive vice president for Nemours Children’s Health System, and physician-in-chief for Delaware Valley operations (which includes Nemours A.I. DuPont Hospital for Children),

Bryostatin-1 is a small molecule that works by activating protein kinase C (PKC) in the brain. PKC is an enzyme that plays an important role in maintaining the health of synapses, which are the connections between neurons (nerve cells). The investigational therapy aims to improve synaptic health in fragile X and other conditions in which synapse health is impaired.

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Fragile X is caused by mutations in the gene FMR1, leading to low-to-no levels of the fragile X mental retardation protein. That protein is involved in the normal development and maturation of synapses, which is why synapse abnormalities occur in fragile X.

In a recent study done in collaboration with the FRAXA Research Foundation, bryostatin-1 treatment normalized some behavioral abnormalities, such as hyperactivity and memory impairments, in a mouse model of fragile X.

“The therapeutic strategy employed by Synaptogenix is exciting and entirely novel. The mechanism of action of bryostatin in the treatment of Fragile X is unique, and preclinical results have been very promising,” said Michael Tranfaglia, MD, medical director and chief scientific officer at the FRAXA Research Foundation.

“We believe that this compound has the potential to make a real difference in the lives of patients with Fragile X, and we look forward to supporting this important new clinical trial,” Tranfaglia added.

Under terms of the new agreement, the clinical experts at Nemours will be in charge of direct contact with participants in bryostatin-1 clinical trials —  including overseeing administration of the medication, and conducting evaluations of psychological and biological goals. Synaptogenix will provide the medication for the trial, and also will offer expertise related to dosing and designing the overall study protocol.

“We are very pleased to take this important step forward with the Nemours A.I. DuPont Hospital for Children, a leading institution for the treatment of childhood diseases,” said Alan Tuchman MD, CEO of Synaptogenix.

“We are encouraged by our collaboration with such an outstanding children’s hospital as Nemours to initiate clinical testing on a new neurorestorative clinical indication for Bryostatin,” said Daniel Alkon, MD, Synaptogenix’s president and chief scientific officer.

The U.S. Food and Drug Administration (FDA) recently granted orphan drug designation to bryostatin-1. This designation is given to medications with the potential to improve care for rare diseases; it confers certain incentives to the therapy’s developer, including seven years of market exclusivity if the medication eventually earns approval.

“Bryostatin has shown promising preclinical efficacy in animal models to reverse synaptic loss and restore cognitive functions in a broad range of pre-clinical models of neurodegeneration, including Alzheimer’s disease and those associated with both Fragile X and Autistic Spectrum Disorder. Based on those studies, the FDA has approved Bryostatin as an Orphan drug to treat Fragile X,” Alkon said.

Bryostatin-1 is being evaluated in people with Alzheimer’s disease in a Phase 2 trial (NCT04538066). The medication originally was developed by Neurotrope. Synaptogenix was formed as a spinoff from Neurotrope late last year.