Cassava Sciences Phase 3 Study of Simufilam Fails to Meet Co-Primary Endpoints

Cassava Sciences Phase 3 Study of Simufilam Fails to Meet Co-Primary Endpoints

By, Admin 28 November 2024

Cassava Sciences’ ReThink-ALZ phase 3 study of simufilam in mild-to-moderate AD fails to meet co-primary endpoints

Overview

Cassava Sciences, Inc., a clinical-stage biotechnology company focused on developing a novel, investigational treatment for Alzheimer’s disease (AD) dementia, announced that the topline results from the phase 3 ReThink-ALZ study of simufilam in mild-to-moderate AD did not meet each of the pre-specified co-primary, secondary and exploratory biomarker endpoints. The co-primary endpoints were the change in cognition and function from baseline to the end of the double-blind treatment period at week 52, assessed by the ADAS-COG12 and ADCS-ADL scales, comparing simufilam to placebo. Simufilam continued to demonstrate an overall favourable safety profile.

Statement from the CEO

  • The results are disappointing for patients and their families who are living with this disease and physicians who have been looking for novel treatment options. We took careful measures to enroll patients with mild-to-moderate AD. Despite that, the loss of cognition in the placebo group was less pronounced than was previously reported in other placebo-controlled studies in AD. We are working to understand this better,” said Rick Barry, president and chief executive officer. 
  • A result like this has implications on our second phase 3 trial, ReFocus-ALZ. We have made the difficult decision to discontinue ReFocus-ALZ, given the nature of today’s reported results. The complete 52-week dataset will be available from the study along with a large portion of 76-week data. We intend to report detailed analyses of both studies in the future. We will also be discontinuing the Open Label Extension study.”
  • Barry continued, “We have a special gratitude for the patients and their families and caregivers who participated in our clinical program for AD. We are also immensely grateful to our employees, study investigators and site coordinators, as well as our other partners, for their commitment to this program. We hope the information we have gathered can ultimately be used to benefit ongoing research in AD.”

1ry data

  • The table below provides a high-level summary of the co-primary endpoints data. 
  • Topline analysis of the mild and moderate sub-groups, likewise, did not demonstrate statistical significance at week 52.

The ReThink-ALZ Trial

  • ReThink-ALZ (NCT04994483) is a phase 3 trial designed to evaluate the safety and efficacy of simufilam compared to a placebo in a multi-centre, double-blinded, placebo-controlled, randomized parallel group study involving over 75 clinical trial sites in the US, Canada and Australia. 
  • The trial randomized 804 people with confirmed mild or moderate AD, defined by several well validated parameters including a mini-mental state examination (MMSE) of >16 and <27, stratified as mild or moderate. 
  • Subjects were randomized 1:1 to receive simufilam 100 mg (n=403) or a matched placebo (n=401), dosed orally twice daily (BID) for 52 weeks.

The Co-Primary & Other Endpoints

  • The co-primary endpoints were the change in cognition and function from baseline to the end of the double-blind treatment period at week 52, assessed by the ADAS-COG12 and ADCS-ADL scales, comparing simufilam to placebo. 
  • Secondary endpoints also included several well validated measures of neuropsychiatric symptoms and caregiver burden. 
  • Safety was evaluated through multiple measures, including adverse event monitoring. 
  • The study also included a pharmacokinetic and plasma biomarker sub-study comprised of approximately 100 subjects, evaluated at three timepoints. 
  • ReThink-ALZ was conducted under a Special Protocol Assessment (SPA) with the US Food and Drug Administration (FDA).

Simufilam is a proprietary, investigational oral small molecule that targets the filamin A protein.

About Cassava Sciences

  • Cassava Sciences is a clinical-stage biotechnology company based in Austin, Texas. 
  • Its mission is to detect and treat neurodegenerative diseases, such as Alzheimer’s disease.

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