ATHX stock rose in pre-market trading after the company announced an update on its product that treats acute respiratory distress syndrome.
Athersys, Inc., a leading regenerative medicine company in late-stage clinical development, announced today that MultiStem® cell therapy was granted Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the acute respiratory distress syndrome (ARDS) program. 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 program 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.
“We are very pleased to have received our second MultiStem program RMAT designation,” commented Dr. Manal Morsy, Senior Vice President and Head of Global Regulatory Affairs. “We have enjoyed close and highly efficient interactions with the FDA on our RMAT-designated ischemic stroke program and are looking forward to similar benefits and advantages of this Expedited Program for Regenerative Medicine Therapies for Serious Conditions RMAT designation, this time for ARDS.”
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 randomized, 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.