Good point that the number of patients is no longer about ten, as was originally communicated as being sufficient to evaluate the proof of concept, but 62. Phase 1 may be those 10, and for further progression, it seems that even one response, as stated by Bono, was enough.
This concerns patients who previously received an anti-PD-1 response, meaning patients for whom nivolumab or similar drugs were previously effective but who have developed resistance, likely due to the TME, or tumor microenvironment, becoming immunosuppressive.
Since this is a non-randomized study, one should not expect marketing authorizations yet, but if Phase 2 involves approx. 25 melanoma and 25 NSCLC (non-small cell lung cancer) patients, one might draw some conclusions from the ratio of responses compared to historical results obtained from re-administering anti-PD-1 to similar patients. Or from the results of competing combinations.
And biomarkers are of particular interest. What does the administration of Bex alone at the beginning of treatment achieve? It is certain that those changes are not caused by nivo. Are these potential changes related to the responses seen later? These provide indirect information about Bex. And yes, going forward, it would still need to be verified through randomization.
If one wants to compare BLAZE’s preliminary or final results with BEXMAB’s results, one can consider the size of the market. There is a 20-fold difference between the MDS and anti-PD-(L)1 markets. Patents are expiring and prices are falling, but new indications are constantly emerging.
If the results related to MDS caused/cause—if and when they do—a 1 kg hammer blow, those related to BLAZE will cause a 20 kg sledgehammer pounding. In terms of potential economic impact, that is. And that is a metaphor, just to be clear. Juho clarified that “biggest pharma” might not necessarily be interested in the MDS market, but they are interested in solid tumors.
The recurring laments that everything will die along with MDS are not based on anything. It is clear that an investor’s knees will buckle and their backside will get soaked in seawater if BEXERA fails, but the biggest market expectations are in that solid tumor area.
Furthermore, the “knowledge” that nothing can happen before we have indisputable evidence from randomized trials is not based on anything either. Molecules, platforms, and companies that have no proper clinical evidence are constantly being bought.