Net income was
Current Updates on Phase 3 Clinical Program
Background - Our Phase 3 program consists of two global, double-blind, randomized, placebo-controlled studies of simufilam in patients with mild-to-moderate Alzheimer s disease dementia. The goal is to evaluate overall risk/benefit for simufilam 100 mg twice-daily versus placebo in a large population of people with Alzheimer s disease over 12 and 18 months.
The target study population is people with mild-to-moderate Alzheimer s (MMSE score of 16-27) who are biomarker-positive for Alzheimer s disease pathology, and who meet other inclusion/exclusion eligibility criteria of the study protocols.
Phase 3 Trials - Our first Phase 3 study, called RETHINK-ALZ, is designed to evaluate the safety and efficacy of simufilam 100 mg tablets twice-daily versus matching placebo over 52 weeks (NCT04994483). Our second Phase 3 study, called REFOCUS-ALZ, is designed to evaluate the safety and efficacy of oral simufilam 100 mg and 50 mg tablets twice-daily versus matching placebo over 76 weeks (NCT05026177). Clinical sites are in
Patient Enrollment - Both Phase 3 studies are fully enrolled. Approximately 1,900 patients are randomized in these studies, with approximately 800 patients randomized in the 52-week study (RETHINK-ALZ) and approximately 1,100 patients randomized in the 76-week study (REFOCUS-ALZ). Approximately 90% of patients are recruited from clinical sites in the
Patient Completion - Over 435 patients have completed the 52-week RETHINK-ALZ study. Over 300 patients have completed the 76-week REFOCUS-ALZ study, for a total of over 735 completers.
Data Safety and Monitoring Board (DSMB) - The DSMB is composed of independent clinical research experts who periodically review interim patient safety data. Routine, scheduled DSMB meetings were held
Co-primary Efficacy Outcomes - The pre-specified efficacy endpoints are ADAS-Cog12, a cognitive scale, and ADCS-ADL, a functional scale. iADRS is a combination of scores from ADAS-Cog and ADCS-ADL. Because the distribution of study participants is numerically skewed towards mild patients, we expect to rely predominantly on mild patients to evaluate drug safety and efficacy.
Phase 3 Efficacy Results - All efficacy data from our Phase 3 program remain blinded. No interim analyses on efficacy outcomes are planned. We anticipate top-line data readout for our 52-week study (RETHINK-ALZ) approximately year-end 2024. We anticipate top-line data readout for our 76-week study (REFOCUS-ALZ) approximately mid-year 2025.
Open-label Extension Study - This study is designed to provide no-cost access to oral simufilam for up to one year to Alzheimer s patients who have successfully completed a Phase 3 study of simufilam and who meet other entry criteria. Approximately 90% of patients who ve completed treatment in a Phase 3 study have opted to enter the open-label extension study. To date, over 655 patients have entered the open-label extension study.
Financial Results for First Quarter 2024
At
The total gross proceeds received from the cash-exercise of Warrants were
Net income was
Net cash used in operations was
Net cash use in operations for first half 2024 is expected to be
Research and development (R&D) expenses were
General and administrative (G&A) expenses were
In
About Simufilam
Simufilam is
About
Contact:
Tel: (512) 501-2450
Email: ESchoen@CassavaSciences.com
Cautionary Note Regarding Forward-Looking Statements
This news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, that may include but are not limited to statements regarding: the design, scope, conduct, continuation, completion, intended purpose, or future results of our on-going Phase 3 program of simufilam in patients with Alzheimer s disease; the timing of anticipated milestones; interim safety data for the Phase 3 program sourced from prior DSMB meetings or other sources; the treatment of people with Alzheimer s disease dementia; the safety or efficacy of simufilam in people with Alzheimer s disease dementia; expected cash use in future periods; comments made by our employees regarding simufilam, drug effects, and the treatment of Alzheimer s disease and potential benefits, if any, of our product candidates. These statements may be identified by words such as anticipate, believe, could, expect, forecast, intend, may, plan, possible, potential, will, and other words and terms of similar meaning.
Such statements are based largely on our current expectations and projections about future events. Such statements speak only as of the date of this news release and are subject to a number of risks, uncertainties and assumptions, including, but not limited to, those risks relating to the ability to conduct or complete clinical studies on expected timelines, the ability to demonstrate the specificity, safety, efficacy or potential health benefits of our product candidates, the apparent ability of simufilam to favor patients with mild Alzheimer s disease; the apparent safety or tolerance of simufilam in our open-label clinical trials; our current expectations regarding timing of clinical data for our Phase 3 studies; any expected clinical results of Phase 3 studies; the treatment of people with Alzheimer s disease dementia and comments made by our employees regarding simufilam, drug effects, and the treatment of Alzheimer s disease; potential benefits, if any, of our product candidates and including those described in the section entitled Risk Factors in our Annual Report on Form 10-K for the year ended
All our pharmaceutical assets under development are investigational product candidates. These have not been approved for use in any medical indication by any regulatory authority in any jurisdiction and their safety, efficacy or other desirable attributes, if any, have not been established in any patient population. Consequently, none of our product candidates are approved or available for sale anywhere in the world.
Our clinical results from earlier-stage clinical trials may not be indicative of future results from later-stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements or any scientific data we present or publish.
We are in the business of new drug discovery and development. Our research and development activities are long, complex, costly and involve a high degree of risk. Holders of our common stock should carefully read our Annual Report on Form 10-K in its entirety, including the risk factors therein. Because risk is fundamental to the process of drug discovery and development, you are cautioned to not invest in our publicly traded securities unless you are prepared to sustain a total loss of the money you have invested.
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