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Bryostatin

Phase 2

Alzheimer Disease | Small molecule | Neurology |Petros Pharmaceuticals, Inc.|Last Updated: Jul 31, 2024

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment230

FDA Designations

No designations recorded

Clinical trial landscape

Bryostatin · 2 trials · 1 indication

Phase 2 2
NCT04538066Bryostatin Treatment of Moderately Severe Alzheimer's DiseaseAlzheimer Disease
COMPLETED122 Analytics
NCT03560245A Study of Bryostatin in Moderately Severe to Severe Alzheimer's Disease Subjects Not On MemantineAlzheimer Disease
COMPLETED108 Analytics
PHASE2COMPLETED
Bryostatin Treatment of Moderately Severe Alzheimer's Disease
Alzheimer DiseaseUnlock trial analytics
PHASE2COMPLETED
A Study of Bryostatin in Moderately Severe to Severe Alzheimer's Disease Subjects Not On Memantine
Alzheimer DiseaseUnlock trial analytics

Study Endpoints

Primary Endpoints

Safety: Treatment-emergent Adverse Events and Serious Adverse Events for All Randomized Subjects Who Received Any Study Medication
Prior to version 6 of the protocol, final assessments were performed at Week 42, 12 weeks after the last study treatment. The final study visit took place at Week 30 for subjects remaining in the study after the protocol amendment.

Treatment emergent adverse events and serious adverse events will be analyzed by treatment group.

Efficacy: Severe Impairment Battery Total Score Assessment Obtained After Completion of the Second Couse of Treatment (Week 28)
Primary efficacy analysis at Week 28

The treatment difference in the primary efficacy endpoint of Severe Impairment Battery (SIB). The SIB is used to assess cognition in subjects with moderate and severe AD. It is divided into nine subscales that include attention, language, orientation, memory, praxis, visuospatial ability, construction, social skills, orienting head to name. Non-verbal responses are allowed, thus decreasing the need for language output. Forty questions are included with a point score range of 0-100. Lower scores indicate greater cognitive impairment.

Safety: Treatment Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Baseline through 30 days post end of treatment (up to Day 107)

Incidence of adverse events (AEs), serious adverse events (SAEs), Adverse event of special interest - myalgia

Efficacy: The Primary Efficacy Endpoint is Defined as the Change From Baseline to Week 13 in the Severe Impairment Battery (SIB) Total Score in the Full Analysis Set
The change in the SIB Total Score from baseline to Week 13 (Day 91)

The Severe Impairment Battery (SIB) assesses cognition in subjects with moderate and severe Alzheimer's disease(AD). Test questions measure attention, language, orientation, memory, praxis, visuospatial ability, construction, social skills, orienting head to name. Non-verbal responses are allowed, thus decreasing the need for language output. Forty questions are included with a total point score range of 0-100. Lower scores indicate greater cognitive impairment.

Secondary Endpoints

Severe Impairment Battery (SIB) Total Score at the End of the Week 42 Follow-up Visit
Week 42 was the final follow-up for study subjects, occurring 16 weeks after the last dose of study drug. Subjects who remained in the study at the time version 6 of the protocol was implemented did not have Week 42 visit.
The SIB Total Score From Baseline at Week 13
Week 13 followed the first 12-week course of study treatment.
The Changes From Baseline in SIB Total Scores at Weeks 9, 20, 24, and 30
Weeks 9, 20 and 24 occur during the treatment phase of the study. Week 30 occurred 4 weeks after end of treatment.
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Bryostatin 1ACTIVE_COMPARATOR20ug Bryostatin will be administered over 45 minutes IV. The course of treatment will include 7 doses over the first 12 weeks, followed by a second identical treatment period beginning 30 days after completion of the first treatment period.
PlaceboPLACEBO_COMPARATORPlacebo will be administered over 45 minutes IV. The course of treatment will include 7 doses over the first 12 weeks, followed by a second identical treatment period beginning 30 days after completion of the first treatment period.
Bryostatin 20µgEXPERIMENTAL20µg Bryostatin administered IV over 45 minutes every other week after 2 initial loading doses of 24 micrograms administered weekly. A total of 7 doses administered over 12 weeks.

Interventions

NameTypeDescription
Bryostatin 1DRUGBryostatin 20 micrograms administered IV over 45 minutes every other week after 2 initial loading doses of 24 micrograms administered weekly. A total of 7 doses administered over 12 weeks, followed by a second identical course of treatment beginning 30 days after completion of the first 7-dose course.
PlaceboOTHERPlacebo administered IV over 45 minutes every other week after 2 initial loading doses of 24 micrograms administered weekly. A total of 7 doses administered over 12 weeks, followed by a second identical course of treatment beginning 30 days after completion of the first 7-dose course.
BryostatinDRUGThe investigational drug product, bryostatin, is a sterile, pyrogen-free, lyophilized powder intended for IV infusion upon reconstitution and dilution.
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Eligibility Criteria

Age Range55 Years to 85 Years
SexALL
Healthy VolunteersNo
Study Sites19

Inclusion Criteria: 1. Written informed consent from caregiver and subject (if possible) or legally acceptable representative if different from caregiver 2. Male and female subjects 55-85 years of age inclusive 3. Cognitive deficit present for at least 2 years that meet the diagnostic criteria for ...

Countries:United States
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Frequently asked questions about Bryostatin

What is Bryostatin used for?

Bryostatin is an investigational small molecule being developed for the treatment of Alzheimer's Disease, specifically moderately severe to severe forms of the condition. It is currently in Phase 2 clinical development and has not been approved by the FDA.

Who makes Bryostatin?

Bryostatin is being developed by Petros Pharmaceuticals, Inc., a company traded on the stock exchange under the ticker symbol PTPI. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Alzheimer's Disease.

What phase is Bryostatin in?

Bryostatin is in Phase 2 clinical development for the treatment of Alzheimer's Disease. It is an investigational drug, meaning it has not yet received regulatory approval and is still undergoing clinical trials to assess its safety and effectiveness.

What clinical trials is Bryostatin in?

Bryostatin has been studied in three completed Phase 2 clinical trials in the United States: NCT02431468, NCT03560245, and NCT04538066. These trials enrolled a total of 377 patients with moderately severe to severe Alzheimer's Disease, with participants aged 55 years and older.

How does Bryostatin work?

Bryostatin is a small molecule that modulates protein kinase C (PKC) activity, an enzyme involved in various cellular signaling pathways. By activating PKC, it is thought to enhance synaptic function and promote neuronal survival, which may be beneficial in treating Alzheimer's Disease.