ANNUAL REPORT 2012 - TiGenix

place a huge burden on not only individuals
but also their health care providers.
Clinical development
TiGenix conducted a Phase I clinical trial in
Spain. 10 healthy volunteers, 5 males and 5
females, have been studied in two cohorts
with two different doses. The objective
of this trial was to confirm the feasibility
and the tolerability of the intralymphatic
injection of Cx621. The study consisted of two
intralymphatic administrations one week
apart of Cx621 into the two inguinal nodes,
with a safety follow-up of 21 days after each
administration. In each of the cohorts, two
subjects received placebo (HTS) and the
others Cx621.
Results of this study confirmed that the
intralymphatic administration was possible in
every of the 10 subjects, with no incidences
during or after the administrations, and
with no technical difficulties. There were
13 adverse events in 7 subjects, all of them
mild except one moderate, and none of
them related to the study medication. All
biological parameters and all exploratory
tests were between normal ranges
throughout the study in every case. An
increase in the surface of lymph nodes was
noted by ultrasound imaging of the inguinal
area, more evident in the active group
receiving Cx621 than in the placebo one,
but with no clinical relevance. No significant
changes were observed in the distribution
of cell subsets in the blood of the volunteers,
and no evident changes occurred in the
early activation markers.
In conclusion, the feasibility of the
intralymphatic administration and the
tolerability were confirmed.
6.5.2. Indications and target markets
Based on the competitive advantages
of the eASC anti-inflammatory mode of
action and the choice for an allogeneic
approach, TiGenix aims to exploit the
immunomodulatory capacity of eASCs
pursuing the delivery of the cells via the
most appropriate route of administration
according to the indication targeted.
These different routes of administration rely
on either systemic or local administration.
TiGenix’s eASC pipeline is pictured in figure 2.
Fig. 2 : eASC product pipeline
Indication Product Preclinical Phase I Phase II Phase III
Complex perianal
fistulas (Crohn’s)
Autoimmune
disorders
Autoimmune
disorders
Cx601
Cx611 (RA)
Cx621
Orphan designation
With a product in the last stage of clinical
development (Phase III) and two further
products in Phase I and II, TiGenix’s eASC
pipeline constitutes to date the most
advanced stem cell platform in Europe.
Assuming Cx601 yields positive Phase III data
and based on a standard regulatory pathway
for ATMPs, TiGenix anticipates generating first
64 TiGenix I annual report 2012