Clinical Pipeline

ARDS

We have established a diverse portfolio of diseases and conditions for treatment in the area of regenerative medicine area, with an emphasis on treating critical care indications. We are applying our patented and proprietary stem cell product candidate, MultiStem®, which we believe has the potential to treat many diseases and conditions, with a focus in neurological, inflammatory and immune disease areas, cardiovascular, and some pulmonary indications. These represent major areas of clinical need, as well as substantial commercial opportunities.

Our most advanced program is exploring the treatment of ischemic stroke, which represents the leading cause of serious disability in the United States, Europe, Japan, China and many other regions around the world. The potential for MultiStem to treat ischemic stroke is currently being evaluated in a registrational trial in Japan by a collaborative partner, HEALIOS K.K., and in a pivotal Phase 3 clinical trial being conducted by us primarily in North America and Europe under an FDA Special Protocol Assessment, or SPA. In addition, this program received the Fast Track designation, as well as the Regenerative Medicine Advanced Therapy, or RMAT, designation from the FDA. These designations are designed to accelerate the development, regulatory review and subsequent commercialization of product candidates like MultiStem cell therapy for ischemic stroke, if the clinical evaluation demonstrates appropriate safety and therapeutic effectiveness.

In recent years, we have advanced several MultiStem programs to the clinical development stage and have also established many preclinical programs through our internal efforts and a broad network of collaborations, where we have worked with investigators at over 30 institutions across the U.S. and Europe.

ARDS
Preclinical
IND/CTA
Phase 1
Phase 2
Phase 3
NDA/BLA
Commercial
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Neurological
Ischemic Stroke*

Masters-2 (Athersys) - enrollment ongoing
SPA, Fast Track and RMAT Designation from FDA & Positive Scientific Advice from EMA

Treasure (Healios) - enrollment ongoing
Priority review designation from PMDA (Sakigake)

Hemorrhagic Stroke

Ability to move directly to Phase 2

Traumatic Brain Injury

Ability to move directly to Phase 2

Multiple Sclerosis

Ability to move directly to Phase 2

Spinal Cord Injury

Ability to move directly to Phase 2

Cardiovascular
Acute Myocardial Infarction
PVD/PAD/CLI

Ability to move directly to Phase 2

Congestive Heart Failure

Ability to move directly to Phase 2

Inflammatory, Immune & Related
HSC Transplant / GvHD**

SPA agreement and Fast Track designation by FDA; Orphan drug designation by FDA and EMA

Acute Respiratory Distress Syndrome

MUST-ARDS (Athersys) – Enrollment complete;
data presented at the 2019 ATS meeting; Fast Track designation for the planned Phase 3

ONE-BRIDGE (Healios) – Enrollment ongoing

Trauma

Pending 150 patient 1/2 study with funding from the DOD (MTEC), and UTHealth

Solid Organ Transplant Support
  • * MASTERS-2 Phase 3 pivotal study under Special Protocol Assessment by FDA commenced; Also, in partnership with Healios, Phase 2/3 clinical trial commenced in Japan under new accelerated regulatory framework and received priority review designation from PMDA.
  • ** Program authorized for Phase 3 pivotal study under Special Protocol Assessment by FDA. Program awarded Orphan Drug status by FDA and EMA, and Fast Track by FDA.
Pipeline-3

We believe that our pipeline can be broadened over time, driven by our technologies, capabilities and approach, and that our MultiStem cell therapy has significant therapeutic potential across a range of clinical indications.

By maintaining a lean infrastructure, and working collaboratively with others, including leading academic researchers, clinical centers, contract research organizations and contract manufacturing organizations, we are able to efficiently and cost effectively advance multiple programs in parallel.