AANS 2019: SB623 Demonstrated Statistically Significant Improvement in Motor Function Among Patients with Chronic Motor Deficit from Traumatic Brain Injury (TBI)

Positive results from the STEMTRA Phase 2 trial were presented today
at the American Association of Neurological Surgeons (AANS) annual
scientific meeting

Data demonstrates the possibility of a stem cell treatment for
chronic motor deficit from TBI

MOUNTAIN VIEW, Calif.–(BUSINESS WIRE)–The SanBio Group (SanBio Co., Ltd. and SanBio, Inc.), a scientific
leader in regenerative medicine for neurological disorders, today
announced that SB623 met its primary endpoint, with patients treated
with SB623 achieving an average 8.7 point improvement from baseline in
the FMMS, versus 2.4 in the control group, at 24 weeks, in a Phase 2
trial of patients with chronic motor deficit from traumatic brain injury
(TBI). Detailed results from the STEMTRA Phase 2 trial evaluating the
efficacy and safety of SB623 were presented today at the American
Association of Neurological Surgeons (AANS) annual scientific meeting in
San Diego.

SB623 is an investigational product made from modified and cultured
adult bone marrow-derived mesenchymal stem cells that undergo temporary
genetic modification. Implantation of SB623 cells into injured nerve
tissue in the brain is expected to trigger the brain’s natural
regenerative ability to recover lost motor functions.

According to the Centers for Disease Control (CDC), approximately 5.3
million people in the United States are living with a TBI-related
disability and more than 280,000 people annually suffer permanent
disability.i The effects of TBI are often long-lasting, with
more than one-third of severe TBI patients displaying a neuromotor
abnormality two years following an injury.ii

Traumatic brain injury is among the most common health conditions faced
worldwide, yet its devastating and long-lasting effects are often
overlooked and underestimated,” said Dr. Okonkwo, M.D., Ph.D., professor
of neurological surgery and director of the Neurotrauma Clinical Trials
Center (NCTC) at the University of Pittsburgh. “The results of this
study are truly groundbreaking, demonstrating the possibility of
regenerating the brain following injury—a finding that could have
significant implications for research in traumatic brain injury and
other brain diseases.”

In this clinical study involving a total of 61 patients, 46 were treated
with SB623 and 15 underwent sham surgery as a control group. Improvement
was measured by the change from baseline in the Fugl-Meyer Motor Scale
(FMMS) score. This scale measures changes in motor impairment and a 10
or more point improvement has been considered a clinically meaningful
threshold in the context of acquired brain injury.3 Of
patients treated with SB623, 18 (39.1%) reached this threshold compared
to one control patient (6.7%). This difference was statistically
significant (p=0.044).

No new safety signals were identified. The most commonly reported
adverse event were headaches and 34.4% of patients treated with SB623
had headache up to 7 days after surgery. There were no significant
differences in the rate of adverse events between patients treated with
SB623 and placebo (p=0.25).

These data are the first in the world to demonstrate the possibility of
a stem cell treatment that may regenerate brain cells following
traumatic brain injury, which affects thousands of people each year and
has no underlying cure,” said Keita Mori, chief executive officer at
SanBio. “These results represent a significant step forward in the
development of regenerative medicines for neurological disorders, and we
are thrilled to be able to share these findings at the largest gathering
of brain surgeons in the world.”

SanBio plans to initiate a Phase 3 trial for SB623 for the treatment of
chronic motor deficit from TBI by the end of fiscal year ending January
31, 2020. The company is also aiming to submit an application for
manufacturing and marketing approval for its TBI program in Japan during
the fiscal year ending January 31, 2020 (February 2019–January 2020),
using the conditional and term-limited authorization system for
regenerative medicine products under the Revised Pharmaceutical Affairs
Act of Japan.

About the STEMTRA Trial

STEMTRA is a 12-month, Phase 2, randomized, double-blind, surgical
sham-controlled, global trial evaluating the efficacy and safety of
SB623 compared to sham surgery in patients with stable chronic motor
deficits secondary to traumatic brain injury. In this study, SB623 cells
were implanted directly around the site of brain injury.

To be eligible for this trial, patients (ages 18-75) must have been at
least 12 months post-TBI and had a Glasgow Outcome Scale extended
(GOS-E) score of 3-6 (e.g., moderate or severe disability). Patients
must also have been able to undergo all planned neurological assessments
and had no seizures in prior three months. The primary endpoint was mean
change from baseline in Fugl-Meyer Motor Scale (FMMS) score at six
months. The STEMTRA trial enrolled 61 patients from 13 surgical and 18
assessment sites in the U.S., Japan and Ukraine.

About SanBio, Inc.

SanBio is a regenerative medicine company headquartered in Tokyo and
Mountain View, California, with cell-based products in various stages of
research, development and clinical trials. Its proprietary cell-based
investigational product, SB623, is currently in a Phase 2b clinical
trial for treatment of chronic motor deficit resulting from ischemic
stroke, and in a Phase 2 clinical trial for treatment of chronic motor
deficit resulting from traumatic brain injury. More information about
SanBio, Inc. is available at http://sanbio.com.

i Centers for Disease Control and Prevention. Report to
Congress on Traumatic Brain Injury in the United States: Epidemiology
and Rehabilitation. National Center for Injury Prevention and Control;
Division of Unintentional Injury Prevention. Atlanta, GA. 2014.
Walker WC, Pickett TC. Motor impairment after severe traumatic brain
injury: a longitudinal multicenter study. Journal of Rehabilitation
Research & Development
. 2007;44(7):975-982.

1 Feys HM et al., 1998; van der Lee JH, et al., 2001


Shannon Moore, Edelman