Athersys Granted Clinical Type B Meeting with FDA for MASTERS-2 Clinical Trial Protocol Discussion

Rhea-AI Summary

Athersys, Inc. (NASDAQ: ATHX) announced the scheduling of a clinical Type B meeting with the U.S. Food & Drug Administration (FDA) to discuss modifications to its pivotal Phase 3 MASTERS-2 clinical trial for ischemic stroke. This follows the company receiving Regenerative Medicine Advanced Therapy (RMAT) and fast track designations for its MultiStem therapy (invimestrocel). The meeting is set for late March 2023, during which Athersys aims to establish more relevant primary and secondary outcomes for the trial based on earlier data. The company will provide further updates during a business call on February 14, 2023.

Positive

• Granting of Type B meeting with FDA is a significant step toward advancing clinical trials.
• MultiStem therapy has received RMAT and fast track designations, aiding in expedited program advancement.
• Upcoming meeting aims to refine trial outcomes for better assessment of therapy's benefits.

Negative

• Dependency on FDA's acceptance of proposed protocol changes may pose risks to trial advancement.
• Potential for unfavorable results from ongoing clinical trials could impact future development.

CLEVELAND--(BUSINESS WIRE)-- Athersys, Inc. (NASDAQ: ATHX), a cell therapy and regenerative medicine company developing MultiStem^® (invimestrocel) for critical care indications, announced today that the Company has been granted a clinical Type B meeting with the U.S. Food & Drug Administration (FDA). The Company was previously granted Regenerative Medicine Advanced Therapy (RMAT) and fast track designations for the use of MultiStem in the treatment of ischemic stroke. These designations enable sponsors to work closely with the FDA and receive its guidance on expediting advancement of designated programs. In the Type B meeting with the FDA, scheduled for late March, the Company intends to discuss proposed modifications to the ongoing pivotal Phase 3 MASTERS-2 clinical trial, which is under special protocol assessment (SPA) agreement.

“This an important step forward as we continue to advance MASTERS-2,” commented Manal Morsy, M.D., Ph.D., MBA, Executive Vice President and Head of Global Regulatory Affairs. “We are grateful to the FDA for its continued guidance and support for the development of high unmet medical need programs. We look forward to a collaborative exchange regarding the proposed modifications for the pivotal ischemic stroke study.”

During the upcoming Type B meeting, the Company will bring forward proposed modifications to the MASTERS-2 trial, with the intent of establishing primary and secondary outcomes that best reflect the potential benefit of MultiStem treatment with ischemic stroke patients. The decision to request this meeting was made following data analysis from the Phase 2 MASTERS-1 trial and the more recent Phase 2/3 TREASURE ischemic stroke trial completed by Healios in Japan. Athersys also considered guidance from clinical key opinion leaders on the evolving practices in standard of care for acute ischemic stroke treatment and engaged expert statisticians to confirm the potential of the proposed protocol amendments.

The Company will be hosting a business update call later this morning, February 14^th at 11:00 a.m. ET, during which more details will be provided. Additional information and registration for the business update call can be found here.

About MultiStem^®

MultiStem^® (invimestrocel) cell therapy is a patented regenerative medicine product in clinical development that has shown the ability to promote tissue repair and healing in a variety of ways, such as through the production of therapeutic factors in response to signals of inflammation and tissue damage. MultiStem therapy’s potential for multidimensional therapeutic impact distinguishes it from traditional biopharmaceutical therapies focused on a single mechanism of benefit. The therapy represents a unique "off-the-shelf" stem cell product that can be manufactured in a scalable manner, may be stored for years in frozen form, and is administered without tissue matching or the need for immune suppression. Based upon its efficacy profile, its novel mechanisms of action, and a favorable and consistent tolerability demonstrated in clinical studies, we believe that MultiStem therapy could provide a meaningful benefit to patients, including those suffering from serious diseases and conditions with unmet medical need.

About Athersys

Athersys is a biotechnology company engaged in the discovery and development of therapeutic product candidates designed to extend and enhance the quality of human life. The Company is developing its MultiStem^® cell therapy product, a patented, adult-derived “off-the-shelf” stem cell product, initially for disease indications in the neurological, inflammatory and immune, and other critical care indications and has several ongoing clinical trials evaluating this potential regenerative medicine product. Athersys has forged strategic partnerships and a broad network of collaborations to further advance MultiStem cell therapy toward commercialization. Investors and others should note that we may post information about the Company on our website at www.athersys.com and/or on our accounts on Twitter, Facebook, LinkedIn or other social media platforms. It is possible that the postings could include information deemed to be material information. Therefore, we encourage investors, the media and others interested in the Company to review the information we post on our website at www.athersys.com and on our social media accounts. Follow Athersys on Twitter at www.twitter.com/athersys. Information that we may post about the Company on our website and/or on our accounts on Twitter, Facebook, LinkedIn or other social media platforms may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks and uncertainties. You should not place undue reliance on forward-looking statements contained on our website and/or on our accounts on Twitter, Facebook, LinkedIn or other social media platforms, and we undertake no obligation to publicly update forward-looking statements, whether as a result of new information, future events or otherwise.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that involve risks and uncertainties. These forward-looking statements relate to, among other things, the expected timetable for development of our product candidates, our growth strategy, and our future financial performance, including our operations, economic performance, financial condition, prospects, and other future events. We have attempted to identify forward-looking statements by using such words as “anticipates,” “believes,” “can,” “continue,” “could,” “estimates,” “expects,” “intends,” “may,” “plans,” “potential,” “should,” “suggest,” “will,” or other similar expressions. These forward-looking statements are only predictions and are largely based on our current expectations. In addition, a number of known and unknown risks, uncertainties, and other factors could affect the accuracy of these statements. Some of the more significant known risks that we face are the risk that we will be unable to raise capital to fund our operations in the near term and long term, including our ability to obtain funding through public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources, on terms acceptable to us or at all, and to continue as a going concern and our ability to successfully resolve the payment issues with our primary contract manufacturer and gain access to our clinical product. The following risks and uncertainties may cause our actual results, levels of activity, performance, or achievements to differ materially from any future results, levels of activity, performance, or achievements expressed or implied by these forward-looking statements: our collaborators’ ability and willingness to continue to fulfill their obligations under the terms of our collaboration agreements and generate sales related to our technologies. In addition, a number of known and unknown risks, uncertainties, and other factors could affect the accuracy of these statements. Some of the more significant known risks that we face are the risk that we will be unable to raise capital to fund our operations in the near term and long term, including our ability to obtain funding through public or private equity offerings, debt financings, collaborations and licensing arrangements or other sources, on terms acceptable to us or at all, and to continue as a going concern and our ability to successfully resolve the payment issues with our primary contract manufacturer and gain access to our clinical product. The following risks and uncertainties may cause our actual results, levels of activity, performance, or achievements to differ materially from any future results, levels of activity, performance, or achievements expressed or implied by these forward-looking statements: the outcome of the Type B meeting with the FDA, including whether the FDA accepts any protocol changes to our MASTERS-2 trial design and the timing of such acceptance, if at all; our collaborators’ ability and willingness to continue to fulfill their obligations under the terms of our collaboration agreements and generate sales related to our technologies; the possibility of unfavorable results from ongoing and additional clinical trials involving MultiStem; the risk that positive results in a clinical trial may not be replicated in subsequent or confirmatory trials or success in an early stage clinical trial may not be predictive of results in later stage or large scale clinical trials; our ability to regain compliance with the Nasdaq continued listing requirement; the timing and nature of results from MultiStem clinical trials, including the MASTERS-2 Phase 3 clinical trial evaluating the administration of MultiStem for the treatment of ischemic stroke; our ability to meet milestones and earn royalties under our collaboration agreements, including the success of our collaboration with Healios; the success of our MACOVIA clinical trial evaluating the administration of MultiStem for the treatment of ARDS induced by COVID-19 and other pathogens, and the MATRICS-1 clinical trial being conducted with The University of Texas Health Science Center at Houston evaluating the treatment of patients with serious traumatic injuries; the availability of product sufficient to meet our clinical needs and potential commercial demand following any approval; the possibility of delays in, adverse results of, and excessive costs of the development process; our ability to successfully initiate and complete clinical trials of our product candidates; the possibility of delays, work stoppages or interruptions in manufacturing by third parties or us, such as due to material supply constraints, contamination, operational restrictions due to COVID-19 or other public health emergencies, labor constraints, regulatory issues or other factors that could negatively impact our trials and the trials of our collaborators; uncertainty regarding market acceptance of our product candidates and our ability to generate revenues, including MultiStem cell therapy for neurological, inflammatory and immune, cardiovascular and other critical care indications; changes in external market factors; changes in our industry’s overall performance; changes in our business strategy; our ability to protect and defend our intellectual property and related business operations, including the successful prosecution of our patent applications and enforcement of our patent rights, and operate our business in an environment of rapid technology and intellectual property development; our possible inability to realize commercially valuable discoveries in our collaborations with pharmaceutical and other biotechnology companies; the success of our efforts to enter into new strategic partnerships and advance our programs; our possible inability to execute our strategy due to changes in our industry or the economy generally; changes in productivity and reliability of suppliers; the success of our competitors and the emergence of new competitors; and the risks mentioned elsewhere in our Annual Report on Form 10-K for the year ended December 31, 2021 under Item 1A, “Risk Factors” and our other lings with the SEC. You should not place undue reliance on forward-looking statements, and we undertake no obligation to publicly update forward-looking statements, whether as a result of new information, future events or otherwise.

View source version on businesswire.com: https://www.businesswire.com/news/home/20230214005062/en/

Athersys

Ellen Gurley

Manager of Corporate Communications and Investor Relations

ir@athersys.com

LHA Investor Relations

Tirth T. Patel

212-201-6614

Source: Athersys, Inc.

FAQ

What is the purpose of Athersys' upcoming Type B meeting with the FDA?

The meeting will discuss proposed modifications to the ongoing pivotal Phase 3 MASTERS-2 clinical trial for ischemic stroke.

When is Athersys' Type B meeting with the FDA scheduled?

The meeting is scheduled for late March 2023.

What designations has MultiStem therapy received?

MultiStem has received Regenerative Medicine Advanced Therapy (RMAT) and fast track designations from the FDA.

What are the potential implications of the MASTERS-2 trial modifications?

The modifications aim to establish more relevant primary and secondary outcomes, which could better reflect MultiStem's benefits for ischemic stroke patients.

What date will Athersys provide further updates about the trial?

Athersys will provide further updates during a business call on February 14, 2023.