sponsor decision

The goal of this open-label extension study is to assess long-term safety and tolerability of simufilam 100 mg in subjects who have completed the RETHINK-ALZ or REFOCUS-ALZ Phase 3 clinical trials.

This is a multi-national, multi-center, fixed-dose, 52-week, open-label extension study. After completing participation in either RETHINK-ALZ (PTI-125-06) or REFOCUS-ALZ (PTI-125-07), subjects will have the option to participate in this study. After the subject provides consent and the Investigator confirms the subject satisfies both inclusion and exclusion criteria, the study drug will be administered at the research site on Study Day 1 and subsequent visits will be scheduled.

We anticipate up to 1600 subjects may enroll in this study. Approximately up to 150 clinical sites in the USA, Canada, South Korea, and Australia will have the option to participate in this collaborative research effort.

For subjects electing to participate, the clinical and laboratory assessments from the Week 76 (REFOCUS-ALZ) or Week 52 (RETHINK-ALZ) End-of-Treatment visit will serve as Baseline Visit assessments for the open-label study on Study Day 1. All subjects will return in 4 weeks and every 12 weeks thereafter for safety assessments. At all post-baseline visits, subjects will report any adverse events since their last visit. In addition to adverse event monitoring, safety will be evaluated at every visit by vital signs, brief examinations, clinical laboratory tests (biochemistry, hematology, and urinalysis) and the Columbia Suicide Severity Rating Scale (C-SSRS). Study drug use since the last visit will be assessed and a new bottle of study drug will be dispensed.

The emerging subject safety assessments will be monitored throughout the study by an independent Data Safety Monitoring Board (DSMB).

Inclusion Criterion:

• Completed RETHINK-ALZ (PTI-125-07) or REFOCUS-ALZ (PTI-125-06).
• Clinical presentation continues to be consistent with Alzheimer's disease.
• Availability of a study partner.

Exclusion Criteria:

• Residence in a skilled nursing facility requiring 24-hour care.
• Evidence of a neurologic condition other than AD that significantly contributes to the subject's dementia.
• Current clinically significant psychiatric diagnosis other than AD.
• Unstable, clinically significant medical condition other than AD.
• Any medical condition that in the opinion of the investigator may affect safety or ability to complete the study.