23 September 2020 - - US-based regenerative medicine company Athersys, Inc's MultiStem cell therapy was granted Regenerative Medicine Advanced Therapy designation from the US Food and Drug Administration for the acute respiratory distress syndrome program, the company said.

The RMAT designation enables sponsors to work closely with the FDA and receive their guidance on expediting development of their products, including providing advice on generating the evidence needed to support approval in an efficient manner.

RMAT designation invites the company to schedule a Type B meeting with the FDA to discuss multidisciplinary strategic development plans, including expediting manufacturing development for commercialization to support priority review and/or accelerated approval.

The RMAT designation for ARDS is granted in addition to the previously obtained Fast Track designation awarded in May 2019.

MultiStem is the only cell therapy programme for ARDS that has both Fast Track and RMAT designation from the FDA.

Also, the company's partner in Japan, HEALIOS K.K. (Healios), is anticipating the completion of enrollment in its orphan designated ARDS clinical trial (ONE-BRIDGE) by the end of this year.

The company has conducted extensive research exploring MultiStem cell therapy for the treatment of pulmonary distress and recently completed an exploratory Phase 1/2 clinical trial for the treatment of ARDS (the MUST-ARDS study).

Participants in the randomized, double-blind, placebo-controlled MUST-ARDS study were evaluated through 28 days for the primary clinical assessment and further assessed through a one-year follow-up period.

Patients that received MultiStem experienced lower mortality, fewer days on a ventilator, fewer days in the intensive care unit, and reported a higher quality of life after one-year post-ARDS than patients that received the placebo.

Following the encouraging results of the MUST-ARDS study, the company began planning for the next stage of clinical evaluation.

In response to the COVID-19 pandemic, the company expedited the initiation of a pivotal Phase 2/3 clinical trial evaluating MultiStem cell therapy for the treatment of COVID-19 induced ARDS (the MACOVIA study), which is now enrolling patients.

The primary efficacy endpoint for the randomised, double-blind, placebo-controlled 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 QoL among survivors through one-year of follow-up.

MultiStem 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 because it is not virus- or pathogen-specific.

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.

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, 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 commercialisation.