Logribel op Seekingalpha:
TiGenix (OTC:TGXSF) just announced, a bit earlier than anticipated, the 6-month interim results from its ongoing Phase 1/2 trial of AlloCSC-01. The therapy uses cardiac stem cells sampled from donors (allogeneic therapy) and administered via intracoronary infusion to patients suffering from acute myocardial infarction (AMI). The injection occurs within 5 to 7 days following the AMI.
The trial enrolled 51 patients, split 2:1 into two groups - 34 patients receiving active therapy and 17 on placebo, in addition to best standard of care for all patients. The primary objective of the trial is to demonstrate the safety of the allogeneic therapy and to look for signs of efficacy on several parameters, with an interim analysis due after 6 months (present results) and full results delivered after 12 months of follow up.
The interim results after 6 months showed that:
- Safety is fine to this date (no death and no significant adverse event, or MACE, in the treatment arm) which was the primary objective of the trial
- Besides, the only efficacy parameter analyzed to this date (change of infarct size) did not show a significant difference between control and active arms
Some thoughts about these elements:
The good safety profile of the therapy is not a surprise in itself, but it is nevertheless a must to continue clinical developments; that's a good thing done.
Obviously, it's a shame that it was not possible to show a significant treatment effect at this preliminary stage. However, the only efficacy parameter that was computed for this interim analysis is the change of infarct size, which does not in itself reflect a direct improvement in the state of patients. For comparison, previous trials of stem cell therapies in heart disease patients such as Celyad's (NASDAQ:CYAD) (C-Cure) and Capricor's (NASDAQ:CAPR) (CAP-1002) were focused on showing improvements in left ventricular ejection fraction (LVEF), end-systolic and end-diastolic volumes (ESV, EDV). These are measurement of blood volumes ejected and remaining into the heart after contraction and those parameters are correlated with functional improvements and healthier patients - chronic heart disease is linked to the diminished efficacy of the "heart pump", so higher volumes of blood ejected (and lower volumes retained) are considered significant improvements. In TiGenix's trial, these parameters will be measured after 12 months and announced with the final results which should be released in Q1-2017.
In brief, are these results good or bad news?
Objectively, it would have been better for TiGenix (and the cardiologists responsible for conducting the trial) to be able to announce outstanding efficacy right now... this is not the case at this stage, and so, the news should not generate a big upside. However, it does not mean either that the final analysis will not bring its share of good news. The main interest of the trial will lie within the data regarding the above-mentioned parameters (LVEF, ESV, EDV) which will be compared to other similar therapies - it will give a good idea of the opportunities for further developments of the product.
One must also take into account the context of heart disease therapies: at the moment, there is no satisfying alternative to treat patients with chronic heart failure except for very costly (and risky) procedures such as heart transplant or LVADs and even modest improvements observed with a very safe cell therapy is considered worthy of further development - e.g. Celyad's C-Cure showed an absolute 7% (or 25% relative) increase in LVEF and this was judged convincing enough to conduct a large Phase 3 trial, which is poised to deliver results by the end of June.
Another fact is that stem cell therapies are still partially understood despite years of clinical development, which can lead to surprising results. For example, while Athersys' (NASDAQ:ATHX) MultiStem (allogeneic bone marrow-derived stem cells) did not show significant efficacy after 3 months in a Phase 2b stroke trial, 12-month follow up results demonstrated a significant improvement in recovery of treated patients. So, at this point, it would be premature to conclude anything about AlloCSC-01 based on partial, interim results.
Finally, it is essential to keep in mind some key elements: success in cutting edge clinical research is never certain and this is part of the game. The advantage of TiGenix over similar small-cap biotechs is that it can rely on a mostly de-risked asset, Cx601, justifying a strong fundamental value at this point. Clearly, AlloCSC-01's success would be a great upside, but even in case of failure, TiGenix's value should not be lastingly affected. At the moment, TiGenix's cash investment in AlloCSC-01 is strictly limited - $1 million in cash to acquire the asset (plus $6 million in stock) and no additional commitment in case of failure, a very reasonable bet for a fully enrolled Phase 2 clinical product. Hence, waiting for the 12-month readout should not be too worrisome given the continued progress made with other assets.