Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
PTI-125 · 2 trials · 2 indications
Blood draws will be done to evaluate levels of PTI-125 in the plasma using non-compartmental methods in WinNonlin.
Levels of PTI-125 will be assessed to determine how long it takes to reach the Cmax
Levels of PTI-125 will be assessed to determine the last time point where PTI-125 can be detected.
Levels of PTI-125 will be assessed to determine the elapsed time to where PTI-125 can last be detected in the plasma.
AUC for PTI-125 plasma concentration from time zero to the last quantifiable plasma concentration.
Assessment of the half-life in plasma of PTI-125
The time to peak drug concentration will be obtained directly from the data without interpolation
The elimination rate constant (λz) will be calculated.
The terminal elimination half-life (T1/2) will be calculated.
The AUC from time zero to the time of the last quantifiable concentration (AUClast) will be calculated.
The AUC from time zero extrapolated to infinity (AUCinf) will be calculate.
The percentage of AUCinf based on extrapolation (AUCextrap\[%\]).
The apparent oral clearance will be calculated.
Vz/F, apparent volume of distribution will be calculated.
| Arm | Type | Description |
|---|---|---|
| Simufilam (PTI-125) | EXPERIMENTAL | Simufilam (PTI-125) 100 mg oral tablets administered twice daily (BID) |
| 50 mg PTI-125 | ACTIVE_COMPARATOR | Six (6) subjects will receive a single orally administered dose of 50 mg PTI-125 in this cohort. |
| 50 mg PTI-125 Placebo | PLACEBO_COMPARATOR | Two (2) subjects will receive a single orally administered dose of 50 mg Placebo PTI-125 in this cohort. |
| 100 mg PTI-125 | ACTIVE_COMPARATOR | Six (6) subjects will receive a single orally administered dose of 100 mg PTI-125 in this cohort. |
| 100 mg PTI-125 Placebo | PLACEBO_COMPARATOR | Two (2) subjects will receive a single orally administered dose of 100 mg Placebo PTI-125 in this cohort. |
| 200 mg PTI-125 | ACTIVE_COMPARATOR | Six (6) subjects will receive a single orally administered dose of 200 mg PTI-125 in this cohort. |
| 200 mg PTI-125 Placebo | PLACEBO_COMPARATOR | Two (2) subjects will receive a single orally administered dose of 200 mg Placebo PTI-125 in this cohort. |
| Name | Type | Description |
|---|---|---|
| PTI-125, 100 mg tablets | DRUG | PTI-125, 100 mg tablets taken twice a day for 28 days |
| 50 mg PTI-125 | DRUG | PTI-125 50 mg Oral Solution |
| 100 mg PTI-125 | DRUG | PTI-125 100 mg Oral Solution |
| 200 mg PTI-125 | DRUG | PTI-125 200 mg Oral Solution |
Inclusion Criteria: * Ages \>= 50 and \<= 85 years * Informed consent form (ICF) signed by the subject or legally acceptable representative. * Clinical diagnosis of dementia due to possible or probable Alzheimer's disease * Mini-Mental State Examination score \>= 16 and \<= 24 at screening * If fem...
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PTI-125 is an investigational small molecule being developed for Alzheimer disease, including early onset Alzheimer disease. It is being studied in patients with mild to moderate Alzheimer disease. The drug is in clinical development and is not approved by the FDA.
PTI-125 targets filamin A, a protein involved in Alzheimer disease pathology. By binding to filamin A, the drug aims to disrupt the interaction between amyloid beta and the alpha7 nicotinic acetylcholine receptor, potentially reducing the toxic effects of amyloid beta in the brain.
PTI-125 is being developed by Cassava Sciences, Inc., a biopharmaceutical company. The company is conducting clinical trials to evaluate the safety and efficacy of PTI-125 in patients with Alzheimer disease.
PTI-125 is in Phase 2 clinical development for Alzheimer disease. A Phase 2 study in patients with mild to moderate Alzheimer disease has been completed. The drug is still investigational and has not received FDA approval.
PTI-125 has been studied in two completed clinical trials. NCT03748706 was a Phase 2 study in 13 patients with mild to moderate Alzheimer disease. NCT03784300 was a Phase 1 safety study in 24 healthy volunteers, including those with early onset Alzheimer disease.
PTI-125 is also known as sumifilam. The drug is being developed under the name sumifilam by Cassava Sciences, Inc. for the treatment of Alzheimer disease.