Tigenix:
Not only does Cx601 become the first ever recorded successful allogeneic "off-the-shelf" stem cell therapy to go through a large, randomized controlled trial, but also it is first and foremost a solid product which answers an unmet therapeutic need in a highly debilitating condition and brings hope to thousands of patients across the world. A single injection of Cx601 in highly refractory Crohn's disease patients with complex perianal fistulas was able to completely cure about 50% of all patients, whereas even the more advanced medical options like biologics (or anti-TNF alpha drugs) have poor efficacy rates (about 23%) and potentially dangerous side effects. In comparison with conventional treatments, Cx601's safety profile proved to be stellar with less treatment-related adverse events observed in the Cx601 treatment arm than in the placebo arm.
In brief, what this means is that about one out of two patients treated with Cx601 will be spared the otherwise unavoidable surgical procedure needed to close their fistulas, which is the only option available to about four out of five patients today - surgery is a risky and painful procedure to which many doctors are reluctant, due to the difficulty of achieving sustainable results and patients' satisfaction since the rate of relapse is still very high and the outcome variable (including risks of anal incontinence). Therefore, with a much better safety profile and a more reliable closure rate than any other non-surgical option, the company's treatment becomes a significant, life-changing option, which makes it very likely that the acceptance of the product will be strong within the medical community and capture a significant portion of the target population, which represents about 120,000 new patients each year in the U.S. and Europe alone (see my previous focus article for more details).
Cx601 is now a mostly de-risked and deeply undervalued asset...
With this in mind and according to pricing estimations - based on Remicade which is priced around $20k per year even with less-than-impressive results and a questionable safety profile - it is estimated that peak sales of Cx601 could reach over $670 million per year with reasonable market share assumptions (see Table 1 below).