Bryostatin-1, Molecule to Protect Synapses, May Move Into MS Trials

Marta Figueiredo, PhD avatar

by Marta Figueiredo, PhD |

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Synaptogenix announced plans to advance bryostatin-1, its lead candidate, into clinical trials for multiple sclerosis (MS), marking the third neurological disease the small molecule therapy is being developed to potentially treat.

“Multiple sclerosis joins Alzheimer’s disease (“AD”) and Fragile X syndrome as our third indication with potential clinical benefit from Bryostatin-1,” Daniel Alkon, MD, Synaptogenix’s president and chief scientific officer, said in a press release.

“Elimination of synapses in MS patients, like those lost in AD, has not been addressed by currently available drug strategies,” Alkon added. Synapses are the points of near contact between nerve cells where cells transmit chemical and electrical signals to one another.

“Through its [synaptic formation-promoting], restorative mechanisms of action, we believe that Bryostatin-1 is uniquely positioned to target synaptic loss and cognitive dysfunction in MS, and potentially other aspects of the disease such as inflammation and [myelin loss],” he said.

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Synaptogenix expects to file an investigational new drug application to the U.S. Food and Drug Administration in the coming months, asking for clearance to launch a first trial of bryostatin-1 in people with MS.

Should it be cleared, the study — aiming to assess the therapy’s safety and effectiveness in this patient population — will be conducted in collaboration with Cleveland Clinic through a new consulting agreement.

“We will work with the Cleveland Clinic to finalize a protocol as soon as possible with the goal of moving towards a clinical trial soon thereafter,” Alkon said.

Alan Tuchman, MD, Synaptogenix’s CEO, noted this collaboration would be similar to one the company forged with Nemours A.I. DuPont Hospital for Children to bring bryostatin-1 into trials for fragile X.

“Advancing our clinical development plans through partnership has been a strategic focus over the past year and will continue to be a key focus going forward,” Tuchman said.

Bryostatin-1 is a small molecule that works by activating protein kinase C, an enzyme required for maintaining synapse health, and involved in learning and memory. The therapy can also influence the immune system toward an anti-inflammatory environment.

Notably, bryostatin-1 can cross the blood-brain barrier, a highly selective membrane that prevents microbes and large molecules in circulating blood from reaching the central nervous system (CNS; the brain and spinal cord). This barrier is often an obstacle for CNS-targeting therapies.

As such, the therapy is expected to improve synaptic health, cognitive function, and reduce inflammation and its damaging effects in people with neurodegenerative diseases such as MS and Alzheimer’s, and neurodevelopmental conditions that include fragile X syndrome.

In preclinical studies, bryostatin-1 restored synapses and triggered the formation of new ones, while preventing nerve cell death. It also was reported to improve learning and memory in animal models of fragile X syndrome, Alzheimer’s, and to reverse neurological deficits in a mouse model of MS.

When administered directly into the bloodstream, the therapy showed a favorable safety profile in previous clinical trials in cancer and Alzheimer’s.

Bryostatin-1 is currently being evaluated in Alzheimer’s patients in a Phase 2 trial (NCT04538066) and a trial in people with fragile X syndrome is expected.

Synaptogenix was formed as a spinoff from Neurotrope — bryostatin-1’s original developer — in late 2020, as part of Neurotrope’s merger with Metuchen Pharmaceuticals, forming Petros Pharmaceuticals.

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