5 May 2020 - - The first patients have been enrolled in US-based biotechnology company Athersys, Inc's (NASDAQ: ATHX) pivotal Phase 2/3 study entitled, MultiStem Administration for COVID-19 Induced Acute Respiratory Distress Syndrome (MACOVIA), the company said.

The patients were enrolled at University Hospital's Cleveland Medical Center (UH Cleveland), a leading pulmonary critical care center and a nationally ranked hospital. Medpace, a leading contract research organization based in Cincinnati, Ohio, is serving as the CRO for this study.

With the continued spread of COVID-19 and the resulting cases of severe respiratory distress among those patients that become seriously ill, there is an immediate need for therapies.

The company's program evaluating administration of MultiStem for the treatment of ARDS was recently granted Fast Track designation by the FDA based on the promising Phase 1/2 data from its previously completed MUST-ARDS trial. Currently there are no FDA-approved medicines for the treatment of ARDS.

Other MACOVIA clinical investigators at UH Cleveland include Dr. Rana Hejal, MD, Pulmonary and Critical Care Medicine Specialist and Medical Intensive Care Unit director, Dr. Catalina V. Teba, MD and Dr. Olivia Giddings, MD, PhD, both Pulmonary and Critical Care Medicine Specialists.

This study is designed to enroll approximately 400 subjects. The first patients were enrolled into the first cohort of the study, which is an open-label, single active treatment arm to evaluate the safety of MultiStem administered at two dose levels to study subjects with moderate to severe ARDS associated with COVID-19.

If the treatment is well tolerated in this first cohort, the study is designed to further evaluate MultiStem efficacy, safety and tolerability in this patient population using a robustly powered, double-blind, randomized and placebo-controlled trial protocol.

The primary efficacy endpoint for the MACOVIA study will compare the number of ventilator-free days through day 28 among MultiStem and placebo treatment groups.

Secondary objectives of the study are to evaluate 60-day all-cause mortality, time in the intensive care unit, pulmonary function, tolerability and quality of life among survivors through one-year of follow-up.

MultiStem cell 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, it may have 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.

ARDS is a serious respiratory condition characterised by widespread inflammation in the lungs. ARDS can be triggered by pneumonia, sepsis, trauma, or other events and represents a major cause of morbidity and mortality in the critical care setting.

ARDS is associated with a high mortality rate and significant long-term complications and disability among survivors. Among survivors, the condition prolongs ICU and hospital stays and often requires extended convalescence in the hospital and rehabilitation care settings. There are limited interventions and no effective drug treatments for ARDS.

There is a large unmet need for a safe treatment that can reduced mortality and improve Quality of Life for those surviving ARDS. Additionally, given the high healthcare resource burden associated with treatment of ARDS patients, a successful therapy could be expected to generate significant savings for the healthcare system by reducing days on a ventilator and in the ICU, or in the setting of a widespread high pathogenicity respiratory virus pandemic, make those resources more rapidly available to other patients.

COVID-19 is the infectious disease caused by the most recently discovered human coronavirus, SARS-CoV-2. This new disease was unknown before the outbreak was first discovered in Wuhan, China, in December 2019.

Older people and those with underlying medical problems such as high blood pressure, heart problems or diabetes, are more likely to develop serious illness, but even young, previously healthy people can suffer severe disease and complications such as ARDS.

Data are still emerging, but recently published case series suggest mortality rates among COVID-19 patients who develop ARDS may be 50% to 70%, or perhaps even higher in some environments.

MultiStem cell therapy is a patented regenerative medicine product candidate 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 may distinguish it from traditional biopharmaceutical therapies focused on a single mechanism of benefit.

MultiStem represents a unique "off-the-shelf" stem cell product candidate 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 favorable efficacy data, its novel mechanisms of action, and favorable and consistent tolerability data 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.

Medpace is a scientifically driven, global, full-service clinical contract research organization providing Phase I-IV clinical development services to the biotechnology, pharmaceutical and medical device industries.

Medpace's mission is to accelerate the global development of safe and effective medical therapeutics through its high-science and disciplined operating approach that leverages regulatory and therapeutic expertise across all major areas including oncology, cardiology, metabolic disease, endocrinology, central nervous system and anti-viral and anti-infective.

Headquartered in Cincinnati, Ohio, Medpace employs approximately 3,600 people across 37 countries.

Athersys 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, cardiovascular 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 the MultiStem cell therapy toward commercialization.