Cassava Sciences Announces Final Results of a Phase 2b Clinical Study of Sumifilam in Patients with Alzheimer’s Disease - Seite 2

“The clinical data suggest sumifilam may be slowing disease progression in Alzheimer’s patients,” said Nadav Friedmann, PhD/MD, Chief Medical Officer, Cassava Sciences. “This exciting possibility will need to be evaluated in future collaborations with patients, physicians, advisors and others.”

“Other than a few drugs to help ease the decline, there’s really nothing out there to treat people with Alzheimer’s,” said Remi Barbier, Chairman, President & CEO, Cassava Sciences. “The improvement on multiple biomarkers in this clinical study is a first and offers hope that sumifilam has potential to become a transformative treatment for people with Alzheimer’s disease.”

Phase 2b Study Design
Phase 2b was a randomized, placebo-controlled, double-blind, multi-center clinical study of sumifilam (formerly, PTI-125). Sixty-four patients with mild-to-moderate Alzheimer’s disease, age 50-85, were randomized (1:1:1) to 100 mg or 50 mg oral sumifilam or matching placebo. Treatment was administered twice daily for 28 days. Nine U.S. study sites enrolled patients. A clinical diagnosis of Alzheimer’s disease was confirmed with the Mini-Mental State Examination (MMSE) ≥16 to ≤26 and a CSF T-tau/Aβ₄₂ ratio ≥0.28. Safety was assessed by ECGs, clinical labs, adverse event monitoring and physical examinations.

Phase 2b Study Results
In this study, drug was safe and well-tolerated, with no drug-related patient discontinuations. The study used biomarkers to measure drug effects. Biomarkers are objective biological endpoints used to track the progression of Alzheimer’s disease. Molecular aberrations in the brain are reflected in biomarkers found in cerebrospinal fluid (CSF), a fluid that surrounds the brain. A key objective of this study was to measure changes in levels of CSF biomarkers in study participants before and after 28 days of treatment (i.e., percent change from baseline).

Key biomarker results include the following (all p-values versus placebo):

• Core markers of Alzheimer’s pathology are total tau (T-tau), phosphorylated tau (P-tau181), and amyloid beta42 (Aβ₄₂). In Alzheimer’s, tau and P-tau levels are elevated and Aβ₄₂ is low.
   
  -  T-tau decreased 15% (p<0.01) for patients in the 50 mg drug group.
  -  T-tau decreased 18% (p<0.01) for patients in the 100 mg drug group.
  -  P-tau decreased 8% (p<0.01) for patients in the 50 mg drug group.
  -  P-tau decreased 11% (p<0.01) for patients in the 100 mg drug group.
  -  Aβ₄₂ increased 17% (p<0.01) for patients in the 50 mg drug group.
  -  Aβ₄₂ increased 14% (p<0.01) for patients in the 100 mg drug group.