Athersys (ATHX) Reports Publication of Data from Placebo-Controlled Clinical Trial Evaluating MultiStem Cell Therapy for Acute Respiratory Distress Syndrome

November 30, 2021 6:03 AM EST

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Athersys, Inc. (NASDAQ: ATHX) announced today that a manuscript reporting data from the Company’s MUST-ARDS clinical trial have been published in the peer-reviewed journal Intensive Care Medicine. MUST-ARDS was a randomized, double-blind placebo-controlled Phase 1/2 trial evaluating the safety and efficacy of MultiStem® (invimestrocel) cell therapy in patients with acute respiratory distress syndrome (ARDS). Enrolling patients at 12 intensive care units in the United Kingdom and the U.S., the study confirmed that intravenous administration of MultiStem cell therapy was well-tolerated among critically ill patients early in the course of moderate to severe ARDS. The trial also assessed multiple efficacy endpoints, including mortality, liberation from mechanical ventilation, discharge from intensive care, and quality of life (QoL) among survivors over one full year of recovery. Mechanisms of action were further explored by comparing acute changes in plasma inflammatory and lung injury biomarkers in MultiStem-treated and placebo-treated study participants. The results of this study provided the basis for the Food and Drug Administration (FDA) to grant the Company’s ARDS program Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designation. The open access publication can be found online at the following link: https://link.springer.com/content/pdf/10.1007/s00134-021-06570-4.pdf.

Highlights from the publication, including new and previously disclosed data:

  • MultiStem treatment was well-tolerated in this clinical trial, with no allergic or serious adverse reactions associated with the cell therapy in any cohort through one year of follow-up;
  • Notably higher median ICU-free and ventilator-free days (VFDs) among MultiStem cell recipients compared to the placebo group at the 28-day benchmark;
  • Lower all-cause mortality in the MultiStem-treatment group compared to the placebo group;
  • QoL outcomes, assessed by the EQ-5D-3L at days 28, 90, and 365, showed greater recovery among survivors who received MultiStem treatment compared to those who received placebo;
  • Decreases in several pro-inflammatory plasma biomarkers were observed in the cell treatment group through Day 7 compared with increases among placebo recipients; and
  • Similar acute plasma biomarker responses to MultiStem, including decreases in pro-inflammatory cytokines IL-1beta, TNFα, IL-6, IFN-gamma and IL-2, have been observed following treatment for ischemic stroke.

In addition (not reported in the published manuscript), preliminary analyses of the data pooled from the MUST-ARDS study and the recently completed ONE-BRIDGE study, which was conducted by Athersys’ partner, HEALIOS K.K. (Healios), in Japan, further supports potential clinically meaningful benefit of MultiStem as a treatment for ARDS patients. These pooled randomized data sets include 40 non-COVID ARDS patients treated with MultiStem cell therapy and 20 ARDS patients treated with placebo or receiving standard of care. Analysis of covariance, adjusting for baseline severity, age, and study participation, revealed an estimate of 5.4 greater (90% confidence interval 0.48-10.32; two-sided p=0.07) VFDs among MultiStem recipients compared to the control group. Similar analyses supported lower mortality in the MultiStem treatment group as well. The pooled data analyses can be found on the Company’s corporate presentation at this link: https://s23.q4cdn.com/674737627/files/11-02-2021-ATHX-Corp-Investor-Presentation.pdf.

“The MUST-ARDS study, completed before the emergence of COVID-19 as a human disease, was an important contribution to the field of cell therapy in the treatment of ARDS. The trial confirmed tolerability of MultiStem infusion in critically ill adults with severe hypoxemic respiratory failure, and often with other coincident organ failure syndromes, and revealed very encouraging signs of improved outcomes that matter to patients and their families,” commented Dr. Eric Jenkins, study co-author, Senior Medical Director and Head of Clinical Programs at Athersys.

“The results of this study, previously presented only as an oral abstract, provided the basis for the advancement of Healios’ ONE-BRIDGE ARDS trial and our own MACoVIA pivotal trial of MultiStem for the treatment of ARDS resulting from COVID and other severe infections. While the study was small, and therefore not powered to provide definitive conclusions about efficacy, the trial provided a strong foundation of safety and persuading support that MultiStem could be an effective immunomodulatory therapy for this previously untreatable and high morbidity inflammatory lung injury syndrome. These results encouraged Athersys, Healios, our clinical collaborators, and others in the field to advance cell therapy for the treatment of ARDS into confirmatory and late-stage clinical trials. It is important that these results are now available in the peer-reviewed literature. I would like to thank our co-authors, the MUST-ARDS clinical research teams, and, most importantly, the patients and families, who on their behalf chose, in their most desperate vulnerability, to contribute to the advancement of medical care for future patients,” concluded Dr. Jenkins.

There remains a strong need for a safe treatment option that can reduce the mortality of patients who develop ARDS, speed recovery, and improve the quality of life for survivors. The MUST-ARDS study has shown safety and tolerability of intravenous administration of MultiStem at doses up to 900 million cells in patients with ARDS. The study data, along with preclinical data, suggest that MultiStem may modulate multiple host responses to tissue injury simultaneously, including down-regulation of hyperinflammatory responses, stimulation of tissue repair, and restoration immune system balance, and support the rationale to conduct an additional larger size study to evaluate efficacy. The Company’s MACoVIA study is a multi-center, randomized, double-blind, placebo-controlled, Phase 2/3 trial to investigate the therapeutic efficacy of MultiStem for the treatment of pathogen-induced ARDS.



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