TAIPEI, Taiwan, Oct. 20, 2018 /PRNewswire/ -- TWi Biotechnology,
Inc. (6610 TTO), a clinical stage biotechnology company focused on
developing novel inhibitors of inflammasomes, today announced that
it has enrolled the first patient in a phase 2 trial of AC-203 for
the treatment of inherited Epidermolysis Bullosa (EB). This study
is designed to be a double-blind, intra-individual comparison,
proof-of-concept clinical trial, and will enroll patients as young
as 2-year-old. The assessment of efficacy and safety outcomes
includes reduction of lesion surface area and blister number,
improvement in pruritus and pain, decreased levels of
pro-inflammatory cytokines and tolerability of AC-203 in patients
with EB.
Based on the specific cleavage site within the skin layers, EB
is typically classified into 4 types: Simplex, Dystrophic,
Junctional, and Kindler Syndrome. The genetic basis underlying
different EB subtypes is distinct and recently inflammation is
thought to play important roles in modulating the symptoms of the
diseases. TWi Biotech has partnered with Castle Creek
Pharmaceuticals, LLC. (CCP), which is leading the global clinical
development using AC-203 (CCP code name: CCP-020) for treating EB
Simplex. "We are hopeful the unique activities of AC-203 in
inhibiting the activation of NLRP3 inflammasome and decreasing the
production of pro-inflammatory cytokines including IL-1beta and
IL-18 could reduce the formation of blisters and the severity of
skin lesions of EB patients," said
Calvin Chen, Ph.D., CEO and
president of TWi Biotech. "We are grateful for the support of local
EB patient groups. They have guided us since the beginning of the
program."
There is no approved drug for any type of EB. The wound care of
EB patients has to be meticulous and can be very traumatic to
patients and caregivers. "Because EB is a devastating disease to
the patients and their caregivers, everyday life could be a
struggle, especially for the families with children suffering
from EB," said Ms. Se-Meng Cheng, founder and CEO of the patient group
Taiwan Debra Home Care Promotion Association, who also is the
mother of a EB Junctional patient. "An effective treatment in
reducing lesion would be wonderful for people affected by the
disease not only in physical but also in mental and moral. We are
extremely excited for TWi Biotech in developing treatment for EB
patients and its decision in conducting the clinical study in
Taiwan. We are looking forward to
working with TWi Biotech and doctors in finding effective
treatments for all EB patients and their caregivers in the
world."
About AC-203
AC-203 is a proprietary topical
formulation of 1% diacerein, which has been shown to inhibit the
activity of caspase-1 and production of cytokine Interleukin-1Beta
(IL-1Beta) through inhibition of theNLRP3 inflammasome activation.
Blockade of the NLRP3 inflammasome-associated signaling pathway and
subsequent IL-1Beta production has been demonstrated to be
effective in treating a variety of diseases including arthritis,
gout, and diabetes mellitus (DM). The oral formulation containing
diacerein has been approved for with the treatment of chronic
rheumatic diseases in France and
subsequently in other EU and Middle Eastern countries such as
Spain and Italy since the mid-1990s.
About Epidermolysis Bullosa (EB)
EB is caused by
mutations in genes which consist of long strands of DNA responsible
for making and expressing proteins. At least 18 genes have been
identified to cause EB. These mutations, or errors in the genetic
code, may not allow the affected gene to produce a specific protein
at all, or to produce a defective protein with deleterious
functions. Either way, the affected protein does not work correctly
and thus resulting in extremely fragile skin. Clinical
manifestations of other EB subtypes include anemia, cardiomyopathy,
syndactyly (fusion of the fingers and toes), renal insufficiency,
dysphagia (difficulty swallowing), malnourishment, cancer,
constipation, osteoporosis, muscular dystrophy and pyloric atresia.
The number of EB patients is estimated to be around 60,000 in US
and EU combined.
About TWi Biotechnology, Inc.
TWi Biotechnology, Inc. is a leading clinical stage
biopharmaceutical company based in Taipei, Taiwan, specializing in the
development of repositioned drugs for unmet medical needs,
especially in rare diseases associated with innate immunity. The
company is expanding its product pipeline through in-licensing and
internal research. TWi Biotechnology's product development pipeline
includes two drug candidates for treating epidermolysis bullosa,
gout, hemophilic arthropathy, and immunodermatology diseases.
Media Contact
Contact Person: Weishu Lu
Phone Number: +886-2-26571788 ext.300
Email: Weishu.Lu@twibiotech.com
View original
content:http://www.prnewswire.com/news-releases/twi-biotechnology-inc-announced-the-initiation-of-phase-2-proof-of-concept-clinical-trial-of-ac-203-for-treatment-of-inherited-epidermolysis-bullosa-300734730.html
SOURCE TWi Biotechnology, Inc.