ANNUAL REPORT 2012 - TiGenix

- It is estimated that approximately 20-40 %
of RA patients do not have an adequate
response to treatment with anti-TNF agents.
6.5.2.3. Cx621
The product Cx621 completed a clinical
development Phase I safety and feasibility
study in 2012. The product is intended for the
treatment of inflammatory and autoimmune
diseases.
Autoimmune diseases are a group of more
than 100 conditions that are caused by
disruptions to immune homeostasis. This
results in the targeting of autoantigens by the
immune system, i.e. the body attacks itself.
Although the causes of autoimmune diseases
are still being investigated, recognized
risk factors include genetic predisposition,
lifestyle factors, environmental factors and
gender. The characteristic immediate result
of an autoimmune condition is inflammation.
This is the result of the aggregation of cells
and molecules associated with the immune
pathways in a tissue. While inflammation
is a critical component of healing
processes, uncontrolled and inappropriate
inflammatory processes can lead to serious
complications such as tissue degeneration.
As such autoimmune diseases are often
chronic and debilitating conditions that
place a huge burden on not only individuals
but also their health care providers.
Autoimmune diseases have for many
years been treated with anti-inflammatory
drugs such as corticosteroids, NSAIDs and
cytotoxics. Although some success has
been achieved by use of these therapies, in
general these benefits are limited. In recent
years, biologics have been developed in
order to meet the need for more specific
treatments for a range of autoimmune
diseases and as such command premium
pricing. Nevertheless, there are also major
drawbacks for this relatively new therapeutic
group. First of all, a significant portion of
treated patients (>20 %) do not have an
adequate response. Secondly, biologics
have serious safety concerns regarding
long term use : non tolerability, recurrent
secondary infections, risk of cardiotoxicity,
etc. And finally, as the efficacy is limited in
time, patients will have to switch to other
biologics.
Despite the wide variety of therapeutic
options there is thus a very high medical
need for innovative therapies that are
effective and safe and have the potential
to become a new treatment paradigm.
Adipose derived stem cell therapy, which
combines anti-inflammatory and immune
modulatory mechanisms of action,
represents a promising alternative therapy.
6.5.3. Manufacturing and logistics
of eASC products
Today, the autoimmune disease market
represents a EUR 40 billion market
opportunity based on sales of currently
marketed products in the eight major
diseases : Rheumatoid Arthritis (RA), Multiple
Sclerosis (MS), Systemic Sclerosis (SS), Lupus,
Psoriasis, Juvenile Idiopathic Arthritis (JIA),
Ankylosing Spondilytis (AS), and Inflammatory
Bowel Disease (IBD)/Crohn’s Disease (CD).
TiGenix’s eASC development stage products
are considered medicinal products
(pursuant to Spanish Order SCO/3461/2003)
and therefore must be manufactured in
compliance with cGMP in an authorized
pharmaceutical establishment. This
also applies to the medicinal products
manufactured for use in clinical trials.
68 TiGenix I annual report 2012