Expect These Alzheimer Phase 3 Trial Results in 2016
While you’re waiting for other stuff to happen to Martin Shkreli, anticipate these results from interventional phase 3 trials in Alzheimer disease in 2016.*
- Safety and Efficacy Study Evaluating TRx0237 in Subjects With Mild to Moderate Alzheimer’s Disease: Tau Therapeutics will complete its international, 15-month, phase 3 study of 2 doses of the anti-tau TRx0237 in 800+ people with mild-moderate AD. Primary efficacy measures are the ADAS-cog11 and the ADCS-CGIC. Secondary efficacy measures include the ADCS-ADL23 and the MMSE. Biomarkers to support any disease-modifying effects are temporal-lobe glucose uptake via FDG-PET and whole-brain volume via MRI. In this phase 3 study and another (see below), TRx0237 (75 or 125 mg bid) will be delivered in a new “stable reduced form” (LMTX®), to address any delivery issues in the phase 2 trial.
- The Study of Nasal Insulin in the Fight Against Forgetfulness (SNIFF**): Sponsored by the University of Southern California with collaborators Wake Forest and the National Institute on Aging, this placebo-controlled, 18-month, phase 2/3 study is assessing the potential cognitive benefits of intranasal insulin (Humulin® R U-100) in 240 people with amnestic MCI or mild AD. Primary outcome measures include the ADAS-cog12 and daily-functional outcomes. Maybe there is a market for Afrezza® after all?
- Safety and Efficacy Study Evaluating TRx0237 in Subjects With Mild Alzheimer’s Disease: This is Tau’s companion phase 3 of TRx0237 (aka LMTX®). However, an alternative dosage of 100 mg bid is being given. Expected enrollment is 700 people with mild AD, and trial duration is 18 months. As in the 2-dose study, primary efficacy measures include the ADAS-cog11 and the ADCS-CGIC, along with the biomarker assessment of FDG-PET.
- Study of Lu AE58054 in Patients With Mild-Moderate Alzheimer’s Disease Treated With Donepezil (STARSHINE): Lundbeck and Otsuka will complete an international, 24-week, phase 3 trial of their 5-HT6-receptor antagonist (Lu AE58054, idalopirdine) in 930 people with mild-moderate AD. All enrollees are taking 10 mg of donepezil. The primary outcome measure is the ADAS-cog. Lu AE58054 is generally considered a symptomatic therapy (like donepezil), but anticipate Lundbeck/Otsuka to make claims (outright or veiled) about disease modification–providing that cognition data are positive.
- A Study to Evaluate Albumin and Immunoglobulin in Alzheimer’s Disease (AMBAR): Despite Baxter’s failure with IV Ig in AD, Grifols Biologicals, Inc., plowed ahead with an international, phase 2/3 trial of “short-term” plasma exchange with 20% albumin ± IV Ig in 350 people with mild-moderate AD. Sham plasma exchange has been incorporated into the trial design. The primary outcome measure at 14 months is the ADAS-cog. Secondary outcome measures include CSF biomarkers and structural changes via MRI.