Neurotrope Bioscience announced that dosing of patients has started in its Phase 2 clinical trial to further evaluate Bryostatin-1, the company’s investigational candidate for the treatment of moderate to severe Alzheimer’s disease.
“Completion of enrollment in our confirmatory Phase 2 trial is an important step toward understanding the transformative potential of Bryostatin in moderate to severe Alzheimer’s disease, a neglected area of AD [Alzheimer’s disease] research with no effective treatment options,” Daniel Alkon, president and chief scientific officer of Neurotrope, said in a press release.
The randomized, double-blind, and placebo-controlled Phase 2 study (NCT03560245) is comparing bryostatin to placebo for the treatment of moderately severe to severe Alzheimer’s disease in individuals not receiving Namenda (memantine), an approved medicine marketed for these disease stages. Patients who went through a wash-out period of 30 days without talking Namenda were allowed to enroll.
A total of 108 individuals are being randomized to 20 micrograms (µg) Bryostatin-1 delivered into the blood (intravenously) or a placebo for 12 weeks, followed by a 30-day post-treatment evaluation. The first two doses were a loading dose 20% higher than the assigned dose and administered one week apart. In total, patients will receive seven doses of either Bryostatin-1 or placebo.
The trial’s primary efficacy endpoint, or goal, is the safety and efficacy of Bryostatin-1 as a treatment for cognitive deficits. Efficacy will be measured as change in the Severe Impairment Battery (SIB) score from the beginning of the study up to week 15. The SIB assesses cognition in Alzheimer’s disease. Test questions measure domains such as attention, language, orientation, memory, or social skills. Lower scores indicate greater cognitive impairment.
Secondary efficacy endpoints include changes in SIB between baseline, or study start, and weeks five, nine, 13 and 15.
“We believe that Bryostatin may have transformative potential as a treatment for moderate to severe AD,” said Charles Ryan, chief executive officer of Neurotrope.
“This confirmatory Phase 2 study could be a critical point of validation for our Bryostatin platform. We look forward to reporting top-line data from this study in the second half of 2019,” Ryan added.
Bryostatin is a small molecule designed to penetrate the blood-brain-barrier and specifically activate the protein kinase C (PKCϵ). Its activity enhances nerve cells’ capacity to communicate with each other, while supporting the repair of damaged communication nodes (synapses).
Preclinical studies have shown that treatment with Bryostatin not only restored synapses, but also triggered the formation of new ones while preventing cell death.
Results from the company’s previous exploratory Phase 2 clinical trial (NCT02431468) showed that Bryostatin-1 led to a sustained improvement in cognition compared to placebo in patients with moderate-to-severe Alzheimer’s disease.
“Bryostatin uses a novel, multi-modal mechanism of action which has demonstrated the ability to generate new, mature synaptic connections and prevent neuronal death in AD models. The promising data from our previous exploratory Phase 2 trial showed greater than baseline improvements in Severe Impairment Battery (SIB) scores for patients in the 20 µg Bryostatin-1 dose group, suggesting the potential to translate Bryostatin’s neurorestorative properties in the clinic,” Alkon said.
The current trial is due to finish in July.
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