SanBio: SB623, an Investigational Product, Granted Advanced Therapy Medicinal Product Classification by European Medicines Agency

MOUNTAIN VIEW, Calif.–(BUSINESS WIRE)–The SanBio Group (SanBio Co., Ltd. and SanBio, Inc.)(TOKYO:4592), a
scientific leader in regenerative medicine for neurological disorders,
today announced that Advanced Therapy Medicinal Product (ATMP)
classification has been granted for SB623 for the treatment of patients
with motor deficits arising from Acquired Brain Injury, including
traumatic brain injury, ischemic stroke and haemorrhagic stroke.

Advanced therapy medicinal products (ATMPs) are medicines for human use
that are based on genes, tissues or cells. Determined by the Committee
for Advanced Therapies (CAT) at the European Medicines Agency (EMA),
treatments that receive ATMP classification may offer groundbreaking new
opportunities for the treatment of disease and injury.¹

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.

“Traumatic brain injury is among the most common health conditions faced
worldwide, with more than 57,000 TBI-related deaths and 1.5 million
TBI-related hospital discharges occurring each year in Europe,²”
said Keita Mori, chief executive officer at SanBio. “We hope this ATMP
classification allows us the opportunity to work closely with European
regulators around the development and regulatory pathway for SB623.”

On April 16, SanBio presented positive Phase 2 results from the STEMTRA
trial at the American Association of Neurological Surgeons (AANS) annual
scientific meeting. 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

In this study, 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 (p=0.044). Of
patients treated with SB623, 18 (39.1%) reached this threshold compared
to one control patient (6.7%; p=0.04). No new safety signals were
identified. The most commonly reported adverse event were headaches.
There were no significant differences in the rate of adverse events
between patients treated with SB623 and placebo.

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.

¹ Advanced therapy medicinal products: overview. European
Medicines Agency. https://www.ema.europa.eu/en/human-regulatory/overview/advanced-therapy-medicinal-products-overview
Last accessed April 17, 2019.
² Majdan M, Plancikova D,
et al. Years of life lost due to traumatic brain injury in Europe: A
cross-sectional analysis of 16 countries. PLOS Medicine.
2017;14(7).
³ Feys HM et al., 1998; van der Lee JH, et
al., 2001

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.

Contacts

For more information, contact:
SanBio Co., Ltd.
Yoshihiro
Kakutani, Corporate Officer of Management Administration
[email protected]