Tigenix: Maria Pascual. ChondroCelect®, Europe's first and only approved cell-based product




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Video title: Tigenix: Maria Pascual. ChondroCelect®, Europe's first and only approved cell-based product
Released on: November 14, 2012. © PharmaTelevision Ltd
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In this episode of PharmaTelevision News Review, filmed at BioPartnering Future Europe in Brussels, Fintan Walton talks to Maria Pascual, VP of Regulatory Affairs and Corporate QA at Tigenix
TiGenix: Specialisation in the area of cell therapy
Fintan Walton:
Hello and welcome to PharmaTelevision News Review here at BioPartnering Future Europe, in Brussels. On this show I have Maria Pascual, who is Vice President Regulatory Affairs and Corporate Quality at a company called TiGenix, welcome.
Maria Pascual:
Thank you and my pleasure to be here today.
Fintan Walton:
So TiGenix is a company that has many places, is in many places, it's headquarter is in Leuven, it's got operations in Madrid, and also in Holland, in the Netherlands. So tell us what is the basis of the company? What is it's, what's the area of specialization?
Maria Pascual:
TiGenix is a European leader in cell therapy with a marketed product for cartilage repair, ChondroCelect, and has also a solid pipeline of late stage clinical programs using adipose-derived stem cells for the treatment of autoimmune and inflammatory disorders. So as you said this is based in Leuven and has operations in Madrid, and in Sittard-Geleen, the Netherlands, where it has a brand new state-of-the-art manufacturing facility for the commercial manufacturing of ChondroCelect.
Fintan Walton:
Okay, wonderful, and but how was the company formed, what's its origins to the company?
Maria Pascual:
So TiGenix as of today is the result of the combination and successful integration of the original TiGenix and Cellerix back in May 2011 [PharmaDeals ID = 39476].
Fintan Walton:
Okay, so TiGenix has merged with Cellerix and now it's a company that's publically quoted on Euronext, is that correct?
Maria Pascual:
Correct.
ChondroCelet : Product for treatment of cartilage repair
Fintan Walton:
Right, okay. So you are a public quoted company, you are in the cell therapy field, now one of the products you did describe just briefly there was ChondroCelect, could you tell us a little bit about that particular product?
Maria Pascual:
So ChondroCelect has been the first advance therapy medicinal products approved European Medicines Agency and it's used for the treatment of cartilage regeneration of the knee and also has been the first and then only so far fully reimbursed ATMP product in Europe, so that's been quite of a benchmark and then achievement here.
Challenges in taking cell based therapy through to regulation
Fintan Walton:
So Maria, obviously there is a difference between taking a cell based therapy and a drug based therapy, so what are the differences and what are the challenges of taking cell based therapy through to regulation?
Maria Pascual:
That's a good question, so these are obviously are very special products different from any other medicinal product, so in brief I could say that you have to have solid science and then to understand very well your product to be able to smartly position in it within the regulatory framework and do your risk based assessment to define what is the type of studies in full clinical development that you have to throw to file your registrations of the product.
Fintan Walton:
And I suppose the other part of that question really is is how close are the regulatory bodies themselves like the EMEA, and the FDA to understanding the issues that relate to cell based therapies? How much in tune are they in understanding the challenges for taking a product like yours through to regulation?
Maria Pascual:
So indeed we have improved a lot in this last 6 to 8years, I remember very well just nine-years ago we went for the first time for a scientific advise with the regulators and that was I mean tougher times, but we all are all parts did a good education through these years, but my piece of advice for the companies developing cell based products would be to start an early and continuous dialogue with regulators to really anticipate and to manage expectations, and I think that there another very good exercise is to contribute to their development of the guidelines and provisions because these cell legislation is still immature and we've done it and that helped the TiGenix I think a lot to establish this relationship and goodwill with regulators to move together smoothly into the next milestones of the development.
TiGenix's platform technology and programs
Fintan Walton:
Right, now obviously cell therapy is still in its infancy, and obviously getting a product to market is really very important, behind the ChondroCelect product there is a platform that's technology base, tell us about that.
Maria Pascual:
Yes, so the TiGenix has a strong pipeline which is a very unique combination of the top line revenues of the marketed product ChondroCelect and the stem cell platform that has a solid IP and currently with one program in Phase III, one program in Phase II, and the third program in Phase I.
Fintan Walton:
Okay, and those are directed to what form of therapy?
Maria Pascual:
Exactly, so the lead candidate what we call Cx601is the intravenous administration of adipose derived stem cells for the treatment of fistula in Crohn's, Phase III trial is going right now in eight European countries, 46 sites and has been designed as a pivotal trial for registration purposes has been discussed with scientific advise with the regulatory bodies of the European Medicines Agency, had the first patient implanted July this year and final clinical results would be available the second half of 2014, that's the lead candidate and we have another couple of products for autoimmune and inflammatory disorders. We have an intravenous product for rheumatoid arthritis, clinical trial has been completed already and interim safety data will be out November this year and the final clinical report April 2013 and then we come to the last product which is a very novel intra-lymphatic administration of the stem cells and a Phase I feasibility trial has been also completed and very successfully. So and just to mention that all these products are allogeneic candidates, so it's off of the-shelf ready to use products in the adult cells and use adipose tissue as a source of cells which has some nice advantages over probably the bone marrow which is the other most common source used in the cell therapy.
Funding and strategic alliances
Fintan Walton:
Okay, now obviously doing clinical trials is an expensive activity, is all of that being funded by yourselves or what sort of strategic alliances do you have?
Maria Pascual:
So currently we are seeking for partnering alternatives for lead candidate 601 and we are in late stage conversations with some partners. So we believe that is coming in the short future and also distribution agreements of the ChondroCelect for several regions as well.
Fintan Walton:
Okay, so you've got some revenue coming through obviously from ChondroCelect, you are looking for strategic partners to take your additional products which are in clinical development into some form of partnership, and presumably you'll continue to have support for your current investors supported by the fact you are in Euronext?
Maria Pascual:
Yes, as you told there, very correct.
Future vision
Fintan Walton:
Okay, so it's fascinating because the story of cell therapy and the evolution of cell therapy based companies is one that's still being watched, we still have to see how that whole area actually plays out, and just then to finally if I could ask you Maria just in terms of where you see TiGenix, how your boards and your CEO see TiGenix going forward? What's the vision for this company?
Maria Pascual:
Yes, so certainly we want to partner this 601 product and then move it to other regions, and we want to all have very soon the clinical strong data validating the autoimmune platform for indications like RA bigger markets, so that would really open it up and then will take the company to the next stage. And so I think it was a great, a very unique regulatory experience that we got with the ChondroCelect, and then really we hope that will enable the company to bring any other product effectively and in a cost effective manner to the market.
Fintan Walton:
Now just on the ChondroCelect, obviously it is registered here in Europe, is there any attempt to do that in the US?
Maria Pascual:
Well I not ourselves, but obviously again having conversations with others that have interest.
Fintan Walton:
Have a strategic interest?
Maria Pascual:
Best interest in the product.
Fintan Walton:
Okay. Maria Pascual, thank you very much indeed for coming on the show.
Maria Pascual:
Thank you. Thanks.
Fintan Walton
Dr Fintan Walton is the Founder and CEO of PharmaVentures . After completing his doctoral research on the genetics of cell proliferation at the University of Michigan(US)and Trinity College (Dublin, Ireland), Dr Walton gained broad commercial experience in biotechnology in management positions at Bass and Celltech plc (1982-1992).
Maria Pascual
Vice President
At the time of this PTV News interview Maria Pascual serves as Vice President, Regulatory Affairs and Corporate Quality at TiGenix. Dr. Mar"a Pascual is VP of Regulatory Affairs and Corporate QA at TiGenix. Graduated in Pharmacy from the University of Granada and obtained her PhD in Immunology from the Spanish National Research Council (CSIC), Master in Regulatory Affairs (Universidad Aut"noma de Barcelona). Intensive research career in the CSIC and is a winner of the international Julia Bodmer Award. Her international experience includes work in the Mayo Clinic (MN, USA) and the Leiden University Medical Center (Netherlands). Dr. Pascual's research activities have focused on the immunology field. She specialized in drug development and regulatory affairs for advanced therapies. She has 10 years experience in cell therapy companies with different positions in the area of regulatory affairs - formerly VP Regulatory and Manufacturing at Cellerix. She is an active participant in several technical working groups contributing extensively for the European industry in the advanced therapies regulation and its implementation plan. Currently, Board member of ASEBIO (Spanish trade association of Biotech companies) and Chair of Regulatory Committee at the European Alliance for Advanced Therapies. Throughout her career, Dr. Pascual has published numerous scientific papers in the immunology field and has produced manuscripts, book chapters and position papers in the regulatory aspects of Advanced Therapy Medicinal products.
PharmaVentures
PharmaVentures is a corporate finance and transactions advisory firm that has served hundreds of clients worldwide in relation to their strategic deal making in the pharmaceutical, life science and healthcare sectors. Our key offerings include: Transactions / deal negotiations; Product / technology valuations; Deal term advice; Due diligence & expert reports; Strategy formulation; Alliance management; and Expert opinion for litigation/arbitration cases. PharmaVentures provides the global expertise to ensure our clients generate the highest possible return on investment from all their deal making activities. We have experience of all therapeutic areas and can offer advice on both product and technology commercialisation.
TiGenix
TiGenix NV (NYSE Euronext: TIG) is a leading European cell therapy company with a commercial product and an advanced clinical stage pipeline of adult stem cell programs. The company's lead product, ChondroCelect, for cartilage repair in the knee, is the first and only approved cell-based product in Europe, and is currently being launched in different European countries. TiGenix's stem cell programs are based on a validated platform of allogeneic expanded adipose-derived stem cells (eASCs) targeting autoimmune and inflammatory diseases. Built on solid pre-clinical and CMC packages, they are being developed in close consultation with the European Medicines Agency. The company has initiated a Phase III clinical trial in complex perianal fistulas in patients with Crohn's disease, is conducting a Phase IIa trial in rheumatoid arthritis, and successfully concluded a Phase I trial to investigate the potential of intra-lymphatic administration of eASCs for autoimmune disorders. TiGenix is based out of Leuven (Belgium), and has operations in Madrid (Spain), and Sittard-Geleen (the Netherlands).