Neurotrope announced it has dosed the first patient in its long-term Phase II study of Bryostatin-1 as a potential treatment for Alzheimer’s disease. The study is conducted in collaboration with the National Institutes of Health, which awarded the company a $2.7m grant for the work.
Expected to enroll an estimated 100 patients, the new study is designed to evaluate Bryostatin-1 without Namenda (mematine) over the course of a six-month period, including two 11-week dosing cycles. The research will focus on patients with moderately severe cases of the disease.
The patient population at the center of the study reportedly demonstrated the most evidence of benefit in prior research. The study will center on assessing sustained cognitive benefit as measured by the widely accepted Severe Impairment Battery score, and researchers will analyze data in consultation with Richard Thompson, senior scientist from the Bloomberg School of Public Health at Johns Hopkins University.
Daniel Alkon, Neurotrope’s president and chief scientific officer, said Neurotrope and NIH representatives reviewed data from previous Bryostatin-1 trials, determining a study to evaluate its long therapeutic effects sans Namenda should be a priority.
“We have made rapid progress in commencing this study and are very pleased to announce today that the first patient has been dosed after significant progress in patient enrollment. Among its potential effects, bryostatin has been shown, pre-clinically, to reverse damage to the synaptic connections between neurons that occurs in AD patients," Alkon explained.
Alkon added, “AD remains among the most significant public health challenges of our modern era and, with support from the NIH, we hope to deliver progress toward advancing a new and innovative, disease modifying treatment."
The study reportedly is supported by Phase II clinical data from a completed pilot trial that evaluated Bryostatin-1 in the absence of Namenda in a short-term, 11-week treatment protocol. Results from that study reportedly showed the drug was well tolerated and showed signs of cognitive benefit; a second pilot trial using the same treatment protocol showed a similar SIB improvement compared to baseline for the Moderate Stratum cohort, researchers reported.
Researchers believe the therapeutic strategy likely is a first-in-class for the potential regeneration of synapses lost in neurodegenerative diseases such as Alzheimer’s disease, multiple sclerosis and Fragile X mental retardation. The signals of cognitive benefit observed during the Neurotrope Bryostatin-1 pilot trials were sustained over several weeks and, researchers claim, may indicate the generation of new synapses in pre-clinical models caused by the activation of the PKC epsilon – BDNF (brain derived nerve factor) pathways by bryostatin and related compounds in the Neurotrope platform.
"We believe that Neurotrope's proprietary platform holds potential for developing treatments for Alzheimer's and other major neurological diseases," stated Josh Silverman, chairman of the board of Neurotrope. "Dosing the first patient in our Phase 2 study of Bryostatin-1 in AD is an important milestone for Neurotrope, and we remain grateful for the ongoing support and validation from the National Institute on Aging at the NIH."
Silverman added that the research also has benefited from ongoing support of the National Cancer Institute, which has provided bryostatin as the natural drug substance.