Cassava Sciences Announces Expansion of Open-Label Extension Trials

Rhea-AI Summary

Cassava Sciences (Nasdaq: SAVA) has announced an expansion of its open-label extension trials for simufilam in Alzheimer's disease. The trials will be extended by up to 36 months, allowing patients who participated in randomized trials to continue treatment if desired. This extension bridges the gap between trial completion and Phase 3 results reporting.

Key points:

• 89% of Phase 3 patients have chosen to continue open-label treatment
• Cognition and plasma biomarker monitoring will be added to gather long-term data
• Patients who completed the extension trial can now re-enroll
• Two ongoing Phase 3 studies: RETHINK-ALZ (804 patients) and REFOCUS-ALZ (1,125 patients)
• Top-line results expected by end of 2024 for RETHINK-ALZ and mid-2025 for REFOCUS-ALZ

Positive

• High patient retention rate (89%) in open-label extension trials indicates positive reception of simufilam treatment
• Extension of trials by up to 36 months allows for collection of valuable long-term data on simufilam's efficacy
• Addition of cognition and plasma biomarker monitoring to gather more comprehensive long-term data
• Large-scale Phase 3 trials with 804 and 1,125 patients respectively, indicating robust study design

Negative

• Extended trials may increase research and development costs for the company
• Delayed reporting of final results due to extended trial duration

Medical Research Analyst

The expansion of Cassava Sciences' open-label extension trials for simufilam in Alzheimer's disease is a significant development. This move demonstrates the company's commitment to long-term patient care and data collection, which could prove important for the drug's potential approval and market success.

Key points to consider:

• The extension allows for up to 36 additional months of treatment, providing a bridge between trial completion and potential FDA approval.
• The high retention rate of 89% in the Phase 3 program indicates strong patient interest and potentially positive experiences with simufilam.
• The addition of cognition and plasma biomarker monitoring in the extension trials could provide valuable long-term efficacy and safety data.

However, investors should note that open-label trials are subject to placebo effects and bias. The true efficacy of simufilam will be determined by the ongoing double-blind, placebo-controlled Phase 3 trials, with results expected in late 2024 and mid-2025.

This extension strategy could give Cassava a competitive edge in the Alzheimer's market by potentially accumulating a larger, long-term safety database. It may also help maintain patient engagement, which could be beneficial if rapid market penetration is needed post-approval.

Financial Analyst

From a financial perspective, Cassava Sciences' decision to extend the open-label trials has both positive and negative implications:

• Positive: This move could enhance the company's data package for FDA submission, potentially increasing the chances of approval and subsequent market success.
• Negative: Extending trials means increased costs, which could strain the company's financial resources, especially considering Cassava's size.

Investors should consider the following:

• The extension may delay the need for a new capital raise, as patients continue to receive treatment through the trial rather than commercially.
• If successful, this strategy could lead to faster market adoption post-approval, potentially accelerating revenue generation.
• The high patient retention rate (89%) in the Phase 3 program could be seen as a positive signal, potentially boosting investor confidence.

However, it's important to remember that the success of simufilam is not guaranteed. The Alzheimer's drug market is notoriously challenging, with many high-profile failures. Investors should closely monitor the upcoming Phase 3 results, as these will be the primary drivers of Cassava's stock performance in the near term.

Market Research Analyst

Cassava Sciences' extension of open-label trials for simufilam could have significant implications for the Alzheimer's disease market:

• It demonstrates a patient-centric approach, which could enhance the company's reputation among patients, caregivers and healthcare providers.
• The move could provide Cassava with a competitive advantage in terms of long-term safety and efficacy data, important in the Alzheimer's market where long-term treatment is often necessary.
• The high retention rate (89%) suggests strong patient satisfaction, which could translate to high demand if the drug is approved.

However, challenges remain:

• The Alzheimer's market is highly competitive, with several large pharmaceutical companies developing treatments.
• Recent controversies in Alzheimer's drug approvals (e.g., Aduhelm) have increased scrutiny on new treatments, potentially raising the bar for approval.

If simufilam proves successful in Phase 3 trials and gains approval, Cassava's strategy of maintaining patient engagement through extended open-label trials could facilitate rapid market penetration. This could be particularly valuable in a market where early mover advantage is crucial.

Investors should watch for the Phase 3 results and any signals from regulatory bodies regarding their stance on Alzheimer's treatments. These factors will be critical in determining simufilam's and Cassava's potential market position.

• Ongoing open-label extension trials of simufilam in Alzheimer’s disease to be extended by up to an additional 36 months.

• The extension provides participants the opportunity to continue on treatment, if they desire, pending results of the ongoing pivotal Phase 3 trials.

• Company plans to add cognition and plasma biomarker monitoring to the open-label extension trials to gather additional long-term data on simufilam treatment.

AUSTIN, Texas, July 30, 2024 (GLOBE NEWSWIRE) -- Cassava Sciences, Inc. (Nasdaq: SAVA) today announced that the Company will extend by up to an additional 36 months each of the open-label extension trials in its ongoing Phase 2 and Phase 3 clinical programs. These amendments to the protocols will allow patients who have previously participated in a randomized trial of simufilam in Alzheimer’s disease, if they desire, to continue open-label treatment with simufilam. This expansion of the open-label extension trials offers a bridge for any gap between patients ending treatment in a clinical trial and the Company reporting to regulatory authorities the results of the ongoing, randomized, placebo-controlled Phase 3 trials. The open-label extension can continue for up to 36 months or until a new drug application for simufilam has been reviewed by FDA.

Cassava also plans to add cognition and plasma biomarker monitoring to its open-label extension trial for patients who have completed the Phase 3 trials in order to gather additional long-term data on the potential impact of simufilam treatment.

“For a company of our size, it is not a trivial commitment to expand our open-label extension studies in this manner. But we believe that this is the best answer for our patients and, ultimately, that is who we serve,” said Cassava Executive Chairman Rick Barry.

Approximately 89% of patients in Cassava’s ongoing Phase 3 program have elected to continue with open-label treatment with simufilam after completion of the blinded trials. Prior to this announced change, approximately 100 patients completed the open-label Phase 3 extension trial and had no option but to discontinue treatment. These patients will now be given the opportunity to re-enroll in an open-label extension trial, if they choose to do so.

James Kupiec, MD, Cassava’s Chief Medical Officer stated, “This decision by the Company is consistent with our commitment to patients. We have expanded the open-label trials in direct response to requests from our clinical research sites.”

Ongoing Phase 3 Studies of Simufilam in Alzheimer’s Disease
Cassava is currently running two double-blind, randomized, placebo-controlled studies of simufilam in patients with mild-to-moderate Alzheimer’s disease dementia. RETHINK-ALZ is a 52-week trial in which 804 patients were randomized 1:1 between simufilam 100 mg and placebo. REFOCUS-ALZ is a 76-week trial in which 1,125 patients were randomized 1:1:1 between simufilam 100 mg, simufilam 50 mg, and placebo. The trials are being conducted at 172 clinical sites in the United States, Canada, Puerto Rico, Australia, and South Korea.

The Company expects to announce top-line results of RETHINK-ALZ by the end of 2024. Top-line results for REFOCUS-ALZ are anticipated approximately mid-2025.

Open-label Extension Study Design
These studies are designed to provide no-cost access to oral simufilam to Alzheimer’s patients who have successfully completed a Phase 2 or Phase 3 study of simufilam and who meet other entry criteria. Each clinical investigational site must choose whether to participate in the open-label extension studies.

For more information about the Phase 3 open-label-extension study, please visit ClinicalTrials.gov: https://www.clinicaltrials.gov/ct2/show/NCT05575076?term=simufilam&draw=2&rank=1

Cautionary Note Regarding Forward-Looking Statements:

This letter 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: our ability to extend our existing open-label extension trials, as contemplated or at all; clinical trials of Cassava’s product candidates; and the potential benefits, if any, of Cassava’s product candidates. These statements may be identified by words such as “anticipate,” “believe,” “could,” “expect,” “forecast,” “intend,” “may,” “opportunities,” “plan,” “possible,” “potential,” “will,” and other words and terms of similar meaning. Such statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to, those described in the section entitled “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and future reports filed with the SEC. In light of these risks, uncertainties and assumptions, the forward-looking statements and events discussed in this letter are inherently uncertain and may not occur, and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. Accordingly, you should not rely upon forward-looking statements as predictions of future events. Except as required by law, the Company disclaims any intention or responsibility for updating or revising any forward-looking statements.

Cassava’s 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 the Company presents or publishes or has presented or published previously.

For more information:
Sitrick And Company
1-800-550-7521
Mike_Sitrick@Sitrick.com
Seth Lubove: slubove@sitrick.com
NY:
Rich Wilner: rwilner@sitrick.com 800-699-1481

FAQ

What is the purpose of Cassava Sciences extending its open-label extension trials for simufilam (SAVA)?

The extension allows patients who participated in randomized trials of simufilam for Alzheimer's disease to continue treatment for up to 36 additional months, bridging the gap between trial completion and Phase 3 results reporting.

When are the top-line results expected for Cassava Sciences' (SAVA) Phase 3 trials of simufilam?

Top-line results for the RETHINK-ALZ trial are expected by the end of 2024, while results for the REFOCUS-ALZ trial are anticipated around mid-2025.

How many patients are involved in Cassava Sciences' (SAVA) Phase 3 trials for simufilam?

The RETHINK-ALZ trial involves 804 patients, while the REFOCUS-ALZ trial includes 1,125 patients, totaling 1,929 participants across both Phase 3 studies.

What percentage of patients chose to continue with open-label treatment in Cassava Sciences' (SAVA) Phase 3 program?

Approximately 89% of patients in Cassava's ongoing Phase 3 program have elected to continue with open-label treatment with simufilam after completion of the blinded trials.