I also wonder if there’s a reason to smile here? Vino pino and Clark Kent could now explain their opinion in simple terms to laypeople and translate these results into an easily understandable format. Investment decisions are, of course, everyone’s own, but this case is difficult to understand without training in the field.
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If/when Bexist becomes an approved medicine in the future, it would apparently be interpreted as a prescription drug in the United States 
https://grok.com/share/c2hhcmQtMg%3D%3D_59238161-41ba-49f5-805d-610f192e9d57
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Faron Pharmaceuticals presents promising Phase 1/2 data from BEXMAB Study at MDS 2025 plenary session
BEXMAB data highlights meaningful clinical benefit, with beneficial immunological and hematological impact in r/r MDS
Encouraging survival; Median overall survival 13.4 months in 20 high and very high-risk r/r MDS patients treated with bexmarilimab + azacitidine; 4 patients successfully bridged to hematopoietic stem cell transplant
55% patients showed ≥50% reduction in bone marrow; 21% of transfusion-dependent patients became transfusion-independent
Faron will be hosting a virtual webinar to discuss the full analysis of r/r MDS as well as new frontline HR MDS patient data on Monday, 2 June 2025. To register for the event visit: BEXMAB Phase II study results
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“Full analysis” is promised in the webinar on June 2nd.
Hopefully, we will see the baseline data of the study patients, such as the number of previous treatments, age, and gender distribution.
Blast reduction has been highlighted; this is the same thing as bone marrow response mCR, which probably has no significance for the prognosis..
The progression to transplant has been brought up. It should be remembered that according to Bexmab’s inclusion criteria, patients who would be “unfit for HSCT” were not specifically selected for the study. It’s difficult to draw any conclusions from this. There could be many reasons why transplantation was only reached now.
Clever-1 high patients respond better, but again, without a control group, we don’t actually know what causes it. Nor have I seen data on Clever-1 concentration as a prognostic factor in MDS.
Zeidan’s comments are politely optimistic, as is customary in academic etiquette. The phrase “if these trends continue to hold..” leaves a lingering thought; does that mean more data is needed before planning phase 3 is worthwhile?
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So, Zeidan’s comment and the entire press release concern this now-analyzed group of the first 20 patients. More data, i.e., a significant increase in the patient cohort, is coming in 3 weeks.
The press release contained, as additional information not directly related to last weekend’s congress presentation, this webcast after ASCO on 2.6.
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That was probably the only new piece of information in Zeidan’s entire presentation and the press release drafted from it.
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Will there be any further additions to the r/r patients, I understood not? Their results are ”especially” good according to Zeidan.
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So, weren’t there 35 r/r patients coming (including the 3 who were on a 1 mg/kg dose), or am I remembering wrong?
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Indeed it was, my bad. It’s difficult to find information when many announcements only talk about percentages and don’t include quantities. We’ll see if that full analysis is really all 35…
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That presentation at ASCO is precisely the responses from all phase 1/2 patients, i.e., 35 r/r and 20 first-line high-risk, and a webcast will be held on Monday immediately after the nio results are published at ASCO.
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There’s a lot of information and knowledgeable people on this forum; thank you very much for all the input. I still have a couple of clarifying questions for a medical layman regarding this last presentation (engineers will drop out when formulas are missing): So, was this Rotterdam merely a criterion exercise, or was there any genuine added value in this information regarding risk reduction? The second question relates to references, i.e., ASCO will provide more comprehensive results with new (and probably old) criteria. What are these compared to when the results are based on new criteria? Are the old ones also translated to this new ‘language’? Thanks in advance.
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I’d like to clarify further that the question is mainly about what these are compared to on the regulatory side, i.e., in marketing authorization processes, etc.
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In the Bexmab study, there is no control group, meaning comparisons can only be made to historical survival times and the types of responses when using aza alone. From now on, Faron will apparently present responses using both new and old response criteria. Previous studies are not based on the new criteria, so direct comparisons cannot be made from these to older studies.
In Phase 3, a true control group will be included, where responses can also be compared regardless of the criteria used. Survival, of course, remains survival; so far, it looks good compared to historical studies, and it’s good news that mOS of 13.4 months has been maintained.
It was also good that Zeidan, in light of the information gathered so far, saw that proceeding to Phase 3 seems justified. For me, that was a positive addition to the previous announcement.
Today’s stock price drop might not be related to Faron’s announcement at all, but rather to Trump’s threats to lower drug prices, and perhaps to the fact that the stock price jumped correspondingly on Friday evening?
Generally speaking, pharmaceutical company stock prices are down today.
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This is the core issue. Regardless of what those responses are (HI, CR, mCR…), the final marketing authorization will only be granted if life expectancy increases and additional worthwhile life is gained. But that’s a time-consuming task, so now we are trying to predict based on those responses.
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The quality of life also looks good here, if life expectancy increases, meaning only mild and very few side effects have been observed (usually, cancer treatments have many side effects). And at least some have been able to stop red blood cell transfusions or doses of white blood cell growth factors, which also reduces costs and is, for the patient, a quality-of-life-enhancing and hospital-care-reducing matter.
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OK, thanks. And in a potential phase 3 study, would the AZA+placebo group be compared to AZA+Bex, but still also to historical data of AZA monotherapy alone (with a larger sample size)?
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In Phase 3, practically only aza+bex and aza alone are compared. This is done with a sufficiently large patient population to have the statistical power to prove a difference in efficacy.
In the case of Bexmab, it is actually the case that the FDA has instructed to conduct Phase 3 specifically in MDS high risk first-line. It is possible to apply for accelerated approval in r/r first, based on responses, WITHOUT conducting a separate Phase 3 in r/r, provided that the first-line Phase 3 has been initiated and preliminary evidence of efficacy based on responses has been obtained. So, the r/r Phase 2 data is intended to lead first to preliminary marketing authorization and ultimately to final marketing authorization, if the Phase 2 data also shows prolonged survival.
But it is true that in r/r, historical data must be compared as part of the evaluation. The FDA does not recommend a control group in r/r because there are no other treatment options against which a control group study could ethically be conducted, and therefore approval could be based on demonstrating efficacy in first-line disease and showing meaningful responses and prolonged life expectancy in r/r compared to historical data. This is how I understand the matter.
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That is probably the median. What would the average be? I’m thinking that even if we get about 20% to bone marrow transplant and recover, it wouldn’t affect the median even if someone gained 20 extra years.
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It has been discussed on the forum how those who have undergone a bone marrow transplant are handled in research/by the FDA, but apparently no understanding has been reached on that matter…
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If we look at the VERONA trial, i.e., aza+venetoclax in first-line MDS, the inclusion criteria regarding transplant eligibility are either:
- Transplant-eligible, but a decision on transplantation has not been made
- Transplant-ineligible
In group 1), the life expectancy is naturally significantly better than in group 2).
It is likely ensured through statistical methods before the study begins that the groups are balanced in this regard (stratified randomization). The primary endpoint compares the median overall survival, and those who received a transplant are not excluded from the analyses.
In the current BEXMAB, it doesn’t really matter how they are handled, as the matter has no relevance to FDA approval processes.
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