ARDS program well-positioned for an expedited path to commercialization with RMAT and Fast Track designation
September 23, 2020; Cleveland — Athersys, 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. 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.
As stated by Dr. Manal Morsy, Senior Vice President and Head of Global Regulatory Affairs:
“We are very pleased to have received our second MultiStem program RMAT designation. 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. For more detailed information on the Company’s ARDS program, please visit the ARDS page on the Athersys website.
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising regenerative medicine therapies, which are defined as a cell therapy, a therapeutic tissue engineering product, or a human cell and tissue product. An investigational drug is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease; and preliminary clinical evidence indicates that the therapy has the potential to address unmet medical needs for that disease. Advantages of the RMAT designation include all the benefits of the fast track and breakthrough therapy designation programs, including early interactions with FDA that may be used to discuss potential surrogate or intermediate endpoints to support accelerated approval.
ARDS is a serious respiratory condition characterized 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 reduce mortality and improve Quality of Life (QoL) 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.
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.
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 commercialization.