Start of in-Patient Clinical Trials With NT-KO-003, the First Neuroprotective Drug for Multiple Sclerosis Without Immunosuppressive Effects

BARCELONA, July 19, 2011 -

• NT-KO-003, developed by Advancell and Neurotec Pharma, is the
first MS drug with neuroprotective effects that does not cause
immunosuppression.

• This treatment is one of the first
to be administered orally, whereas the few treatments available in
Spain require parenteral administration (intravenous or
subcutaneous) and have significant side effects.

• NeuroAdvan, the Phase IIa clinical trial, will be led by Dr
Pablo Villoslada, director of Neuroimmunology at IDIBAPS, Hospital
Clínic de Barcelona, ??and conducted in 105 patients
in 14 hospitals in Europe. It will  represent an investment of
2.2 M €, part of which will be financed by the Catalan Local
Government and the Ministry of Science and Innovation.

• This project brings to clinical proof of concept the first
molecule arising from Neurotec’s research in the field of acute and
chronic central nervous system diseases; Advancell reaffirms its
business model consisting of the development of new drugs to Phase
II, and to subsequently license them to a pharmaceutical
company.

The biotechnological companies Advancell and Neurotec Pharma,
both based in Barcelona Science Park (PCB), Spain, have announced
the initiation of a clinical Phase IIa study with NT-KO-003, an
innovative oral therapy for multiple sclerosis (MS). NT-KO-003 was
codeveloped by both companies and its mechanism of action is
completely different from the drugs currently used to treat this
disease.

The clinical trial, called NeuroAdvan, will be conducted in 105
patients from 11 hospitals in Spain (4 of them in Catalonia) and 3
in Germany, and will be led by Dr Pablo Villoslada, director of
Neuroimmunology at IDIBAPS, Hospital Clínic de Barcelona, Spain.
The project is being conducted in collaboration with the MRI
Analysis and Research Center in Neuroimaging and Multiple Sclerosis
(CARM) at the Hospital Vall d’Hebron, Barcelona, led by Dr.
Alex Rovira, and also with advice from Dr. Rafael Arroyo at the San
Carlos Cinical Hospital, Madrid, Spainm and Dr. Xavier Montalban
from Hospital Vall d’Hebron, Spain. Results from the study are
expected in late 2012.

MS affects about 30,000 people in Spain according to the Spanish
Association of Multiple Sclerosis, and about 2.5 million worldwide,
according to World Health Organization (WHO). MS mainly affects
young adults (mostly women),  aged between 20 and 50. The
drugs currently used to treat MS are immunomodulators with
significant side effects, limited effectiveness and require
parenteral administration (intravenous or subcutaneous), thus
making adherence to the therapies difficult.

In animal models during the preclinical phase, NT-KO-003 has
demonstrated an anti-inflammatory and neuroprotective effect,
rather than an immunosuppressive effect. Consequently, NT-KO-003
can be combined with other drugs when necessary without increasing
toxicity and can be administered to nearly all patients affected by
the disease. It is also one of the first oral treatments that can
potentially slow the progression of MS and reduce the neurological
damage caused by the disease.

Because of its neuroprotective mechanism of action, Advancell and
Neurotec are exploring the possibility of using NT-KO-003 in the
treatment of other neuromuscular diseases, such as amyotrophic
lateral sclerosis, which mainly affects men aged between 40 and 70
and causes the death of 50% of patients it in the first three years
after the onset of symptoms.

According to the project managers at Advancell, Mercè de Frias, and
Neurotec Pharma, Javier Bustos, “the promising results of
NT-KO-003 in animals convinced us to start clinical trials in MS
patients. We managed to involve doctors and patients’ associations
in the design of the trial protocol and we believe the study meets
the current needs of MS patients”

“At present, the main concern of MS patients is to maintain a
good quality of life. They need to stop the progression of the
disease, or at least to slow down, and if possible to recover from
the serious side-effects. Since MS is a chronic disease that
affects young people who are working and have families, new
treatments should be easy to manage and should have no side
effects, so as not to interfere with patients lives. NT-KO-003
holds great great promise because of its safety and ease-of-use,
which are very good, and unlike current drugs it does not alter
patients’ quality of life, and also because it’s mechanism of
actionis new and complementary to existing treatments”,
says Dr. Villoslada. “Furthrmore, all the neurologists
participating in this study are really motivated because this
treatment has arisen from basic research done by colleagues from
our country and may be beneficial to our patients. This is an
example of real innovation”.

An investment of EUR 2.2 million will be made in this clinical
trial, and Advancell and Neurotec Pharma will receive funding
support from the Catalan Local Government, through the Nuclis of
Technological Innovation Program of ACC1Ó, and from the Ministry of
Science and Innovation, through the subprogram INNPACTO, which is
part of the National Plan for Scientific Research, Technological
Development and Innovation 2008-2011 (IPT-010000-2010-035). Both
programs are financed by the European Regional Development Fund
(FEDER). The total cost of NT-KO-003 development is about EUR 4
million, with an estimated potential market of around EUR 5.3
billion.

This project will lead  to clinical proof of concept of the
first molecule arising from Neurotec’s research and Advancell
reaffirms its business model of developing new drugs to Phase II,.
Once the trial has been successfully completed, both companies plan
to license the drug to a pharmaceutical company, which will
continue its development and bring it to market.

According to the director of the Advancell’s Therapeutics Unit,
Clara Campàs and Advancell´s CEO, Kenneth Weissmahr, “with this
project, Advancell demonstrates its know-how in drug development
and shows its commitment to develop projects with significant
social and economic return in addition to responding to a difficult
disease with significant therapeutic needs “

According to the CEO of Neurotec Pharma, Marco Pugliese,
“this clinical trial is the proof that projects born in the
universitary environment can be successfully translated to the
business world and quickly arrive to the patients thanks to a
reprofiling strategy, which is increasingly used to develop new
therapies”.

About Advancell

Advancell is focused on the development of drugs for unmet
clinical needs, especially in the areas of oncology, dermatology
and the central nervous system. The company draws on findings from
the academic environment, develops them until Phase II and then
licenses them to a pharmaceutical company. Advancell’s mission is
to build a portfolio of high value pharmaceutical products, with a
defined cost and duration (maximum EUR 3 million and three years of
development). In addition to NT-KO-003, its products at clinical
stage are focused on nanomedicine for psoriasis (with ISDIN); an
orphan drug for CLL and multiple myeloma; a topical formulation for
palmo-plantar erythrodysesthesia; and a topical treatment for
atopic dermatitis in dogs. Advancell is located in the Barcelona
Science Park, Barcelona, Spain, and has a nanomedicine research
center at the University of Santiago de Compostela, Spain.

About Neurotec Pharma

Neurotec Pharma SL
is a spin-off of the Universitat de Barcelona, located in the
Barcelona Science Park, Barcelona, Spain. Its business model is
focused on the development of new treatments for diseases of the
central nervous system based on the reprofiling of drugs with
peripheral use. This strategy is less risky, has reduced research
time and needs less investment. Neurotec Pharma performs the
screening and selection of potentially interesting molecules by
using in vitro tools and then checking their effectiveness in
accepted animal models of diseases of central nervous system
involving inflammation and neurodegeneration. Its innovative line
of research is to control the activity of microglial cells involved
in a wide range of diseases related to brain inflamation or acute
cerebral damage.

More information:
Azucena Berea        
Comunicación y Relaciones Corporativas        
       
Parc Científic Barcelona        
Tel: +93-403-4662              
             
aberea@pcb.ub.es

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