FDA Authorizes Athersys to Initiate a Pivotal Clinical Trial Evaluating MultiStem Cell Therapy

In a Phase 1/2 study of MultiStem therapy for the treatment of ARDS, favorable tolerability data and meaningful potential benefits in mortality, ventilator-free days and ICU-free days observed

Athersys, Inc. announced that the US Food and Drug Administration (FDA) has authorized the Company to initiate a Phase 2/3 pivotal study to assess the safety and efficacy of MultiStem therapy in subjects with moderate to severe acute respiratory distress syndrome (ARDS) induced by the novel coronavirus disease (COVID-19). This program falls under the current Investigational New Drug (IND) application for the Company’s completed MUST-ARDS study and, therefore, a new IND does not need to be filed. The Company plans to open the first clinical sites for recruitment of this MACOVIA (MultiStem Administration for COVID-19 Induced ARDS) study this quarter.

FDA authorizes #Athersys to initiate a pivotal clinical trial evaluating #MultiStem Cell Therapy in patients with #COVID19 induced #ARDS.

According to the World Health Organization (WHO) and other recent clinical and epidemiological data, ARDS is the leading cause of death among COVID-19 infected patients. There are no FDA-approved medicines for the treatment of ARDS. Athersys recently completed a Phase 1/2 study evaluating administration of MultiStem to patients with ARDS and based on the promising data from the study, the program was recently granted Fast Track designation by the FDA.

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This trial will be a multicenter study featuring an open-label lead-in followed by a double-blinded, randomized, placebo-controlled Phase 2/3 portion. The primary objectives of the MACOVIA study are to evaluate the safety and efficacy of MultiStem therapy as a treatment for subjects with moderate to severe ARDS due to COVID-19. The primary efficacy endpoint will be number of ventilator-free days through day 28 as compared to placebo, a well-established endpoint for ARDS trials that evaluates an intervention’s combined impact on survival and liberation from invasive mechanical ventilation. The secondary objectives of this study are to evaluate pulmonary function, all-cause mortality, tolerability and quality of life (QoL) among survivors associated with MultiStem therapy as a treatment for subjects with moderate to severe ARDS due to COVID-19. The study is designed to enroll approximately 400 subjects and will be conducted at leading pulmonary critical care centers throughout the United States. The first cohort of the study will be open-label, with a single active treatment arm to evaluate the safety of the MultiStem product candidate at two dose levels. The second cohort will be a double-blind, randomized, placebo-controlled run-in phase to evaluate the efficacy of MultiStem. The design of the third planned cohort will be based on analysis of the results of the second cohort. The intent-to-treat population will include all randomized subjects (i.e., subjects from the second and third cohorts).

“We are grateful for the FDA’s timely review and feedback during our design of this pivotal Phase 2/3 study,” commented Dr. Eric Jenkins, MD, Senior Medical Director and Head of Clinical Operations at Athersys. “With encouraging non-clinical and clinical data, affirmed by the FDA’s Fast Track designation for ARDS, Athersys and its collaborating clinical investigators are highly motivated by the FDA’s authorization that we may proceed with enrollment of the first open-label cohort to evaluate safety. We are in communication with FDA regarding the enrollment of further cohorts to conduct a scientifically rigorous evaluation of MultiStem’s safety and efficacy in the treatment of ARDS due to COVID-19. We believe that MultiStem treatment, by modulating patients’ hyperinflammatory response to highly pathogenic respiratory viruses, including SARS-CoV-2 which causes COVID-19, represents a very promising approach to improving outcomes in patients who suffer the most severe manifestations of these illnesses.”

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With the spread of COVID-19 and the resulting ARDS cases, there is an immediate need for therapies for the treatment of ARDS. The data from the Company’s Phase 1/2 MUST-ARDS study met its primary endpoint of tolerability and participants were evaluated through 28 days on key clinical parameters as well as improvement in pulmonary function. As previously reported, study subjects receiving MultiStem experienced less mortality, more ventilator-free days, and more intensive care unit (ICU)-free days during the 28-day clinical evaluation period than the subjects who received placebo. These differences were observed to be greatest among the prospectively defined patients with more severe ARDS, defined as patients with PaO2/FiO2 above or below 150mmHg. Importantly, among all randomized subjects in the study, 45% of MultiStem treated subjects were off the ventilator within seven days or less, compared to only 20% of placebo recipients. Biomarker analysis confirmed a reduction in inflammatory biomarkers among MultiStem treated subjects. In addition, results from the one-year follow up were consistent with the positive day-28 clinical results and an evaluation of the subjects’ QoL, as measured by patient-reported EQ-5D-3L self-care responses and visual analogue scale (VAS) pain scores, more positive among patients who received MultiStem. The data from this study were presented at the American Thoracic Society Meeting in May 2019.

MultiStem therapy’s potential for multidimensional therapeutic impact may distinguish it from traditional biopharmaceutical therapies focused on a single mechanism of benefit. Since MultiStem is not virus- or pathogen-specific, we believe it has the potential to treat ARDS that develops from a variety of causes, including COVID-19, as well as other pathogen-induced or non-infectious causes of severe lung inflammation leading to ARDS. The Company is in discussions with the Biomedical Advanced Research and Development Authority (BARDA) to expedite the advancement of MultiStem to treat patients with ARDS resulting from the COVID-19 epidemic and other potential pandemic outbreaks.

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