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

BARCELONA, July 19, 2011 /PRNewswire/ --

• 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