Acute Respiratory Distress Syndrome (ARDS)

In May 2019 we presented positive results for an exploratory clinical study evaluating MultiStem cell therapy for treatment of Acute Respiratory Distress Syndrome, or ARDS at the American Thoracic Society Meeting in Dallas. ARDS is a serious immunological and inflammatory condition characterized by widespread inflammation in the lungs. ARDS can be triggered by pneumonia, sepsis, or trauma and represents a major cause of morbidity and mortality in the critical care setting. It has significant implications, as it prolongs intensive care unit (ICU) and hospital stays and requires convalescence in the hospital and rehabilitation.

Patients in the exploratory study were evaluated through 28 days for the primary clinical assessment and will be further assessed through a one-year follow-up period. Data highlights from the initial evaluation include the following results from the double-blind, randomized, placebo-controlled portion of the study:

  • Lower mortality of 25% in the MultiStem treatment group vs. 40% in the placebo group;
  • 40.2% higher ventilator-free (VF) days, (12.9 VF days in the MultiStem treatment group vs. 9.2 VF days for the placebo group);
  • 27.2% higher ICU-free days, (10.3 days in MultiStem subjects vs. 8.1 days for subjects receiving placebo);
  • In more severe ARDS patients (as evident in a prospectively defined analysis), the difference between MultiStem treatment and placebo was greater – 25% mortality in MultiStem group vs. 50% in placebo group, 14.6 VF days in MultiStem group vs. 8.0 VF days in placebo group, and 11.4 ICU-free days in MultiStem group versus 5.9 ICU-free days in placebo group; and
  • MultiStem treatment was well tolerated in this very sick ARDS patient population, with no serious adverse events related to administration.

The study was designed to evaluate the impact of MultiStem treatment in subjects with acute onset of moderate to severe ARDS and was conducted at sites in the United States and United Kingdom. The study included two parts – a small initial dose confirmation phase, followed by the larger double blinded, placebo-controlled and randomized phase (Phase 2a portion). Treatment was required to begin within four days of ARDS diagnosis with an average treatment time of approximately two days from the diagnosis. Six subjects were treated with MultiStem in the initial portion of the study, and in the Phase 2a portion of the study, 20 subjects were treated with an intravenous (IV) administration of 900 million MultiStem cells and 10 subjects received IV placebo. As disclosed previously, the study was not powered for the efficacy outcomes. Athersys will continue to evaluate the data as the one-year follow-up period is completed for all patients in the trial.

In May 2019 we announced that our planned Phase 3 study for ARDS has received Fast Track Designation from the FDA.  In addition, our partner HEALIOS K.K. is currently running the ONE-BRIDGE study using MultiStem cell therapy. On November 14, 2019, HEALIOS K.K. received notification of Orphan Designation for their ARDS program, which is meant to expedite the regulatory process for development and provides other meaningful benefits.

There are limited interventions and no effective drug treatments for ARDS, making it an area of high unmet clinical need with high treatment costs. Given ARDS high treatment costs, a successful cell therapy could be expected to generate significant savings for the healthcare system by reducing days on a ventilator, days in the ICU and total days in the hospital, and importantly, could reduce mortality and improve quality of life for those suffering from the condition. The medical need for a safe and effective treatment of ARDS is significant due to its high mortality rate, and it annually affects approximately 400,000 - 500,000 patients in Europe, the United States and Japan, alone.