Deel 2
4. Cx611 - severe sepsis
Patient recruitment is expected to start in autumn this year (during the flu season ... saw the referred patients is an activity peak). Bravo confirms that the decline in the beginning of the study in the second half (instead of early 2016) is related to the need to ensure solid funding before the launch, which was released among others by European funds obtained (which finance half of the test). The opportunity is also very important given the huge mortality rate and the lack of effective therapeutic alternative at present. However, it will take a little patience: given the goal to have strong clinical results and unequivocal Phase 2 is a large trial (180 patients) and rigorous recruitment will take time, probably a two years. Being optimistic, the results should come out at the earliest at the end of 2017.
5. Japan and legislation favorable to cell therapies
I asked Eduardo Bravo the opportunity that could represent Japan for TiGenix, given the very favorable legislative direction of the country towards cell therapies. After all, Mesoblast went there (with JCR Pharmaceuticals local partner) and recently obtained approval of its allogeneic therapy against graft rejection (graft-versus-host disease, GvHD) including reimbursement at a very high price (on average $ 150k per patient).
Specifically, regarding the Cx601, the very low prevalence of Crohn's disease in Japan is an element that hinders potential partnerships for this product ... especially if very, very few patients, the costs required to the placing on the market in this country are prohibitive for the return expected.
So if a development is to consider that side, it will probably rather for AlloCSC or Cx611, which target indications of high prevalence worldwide.
6. Conclusion
After this event, my overall impression of TiGenix and its management is: it is a highly motivated and experienced team, with the clear objective of making biotech a world leader in its field. The seriousness of the scientific project and will conduct irreproachable clinical trials is evident. Clinical results and milestones achieved over the last two years are undeniably great progress, while biotech is clearly past two fingers to put the key under the mat there not long ago (summer 2013 catastrophic). Eduardo Bravo explicitly mentioned these very difficult times painful concessions had to be made to save the biotech (investments with huge discounts to avoid bankruptcy) and it has obviously left its mark - there is no triumphalism, but a some relief to have arrived so far, and also some bitterness at the market reaction and the current valuation of the biotech, considered well below the real potential.
Some suggest a lack of long term vision or direction: it could not be further from reality as I could observe first source. However, what is also recognized by the management, it is a necessity to provide more adequate and more broadly on this vision (at least when the restrictions on the IPO will be more strictly ...). This is one of the main commitments of the coming year, which has also started with the recruitment of Ray Campos, responsible for relations with investors and specialist-oriented communication market, which it will be the main task.
Finally, the shareholders present may, I think, demonstrate more great accessibility of the whole team and a real attention to our questions (the session of Q & A lasted over an hour and no subjects' was taboo). The biggest challenge now is to show through this vision and attention to the greatest number of investors.