Zeidan ei lepää edes Jouluna. 7 minuutin video Vumedi sivustolla.
Samat kalvot kuin ASH25:ssa joten ei erityistä uutta dataa. Uskaltaa silti tässä jo kehua Bexiä laajemmin. Hyvä myös että kiinnittävät tulevassa Bex tutkimuksessa erityistä huomiota ongelmakohtiin johon aiemmat tutkimukset eivät ole riittävästi ottaneet huomioon.
Video katsottavissa vain rekisteröityneille.
Videon lopusta pätkä koneella litteroituna:
(Transcribed by TurboScribe.ai.)
… So, this is the data on the 21 patients who were treated in the frontline. 20 of those patients was efficacy-valuable, and you can see that the CR rate was around 45% by 2006 criteria and 29% by 2023 criteria. As a reminder, this expected CR with the aza monotherapy is in the range of 15%, and several of those patients had very good responses that are less than CR, what we call CR-equivalent or CR-limited, with a composite CR of 53% by the 2023 criteria.
7 of these 20 patients were able to proceed to transplant, and the median duration of CR was 12 months, and most patients had less reduction, including those with TP53. In the refractory lab setting, this is a very challenging treatment setup to treat. Two patients had CR.
However, many patients had other responses, such as partial response or hematologic improvement, with an overall response rate of 64% and a median duration of response that was 7 months when you sensor for transplant. Importantly, in the refractory lab setting, the overall survival looked actually quite good at 14.5 months, and we know that the median survival after HMA failure is in the range of 6 to 8 months in other studies, especially considering that many of those patients didn’t go to transplant. And when you look by TP53 status, the median survival for TP53 patients was 7.5 months and was not reached for the wild-type TP53.
In the front-line setting, the follow-up is shorter and the overall survival data is still maturing. When we look at the treatment-naive high-risk MDS patients, specifically on TP53, we see 70% CR rate, which again, I think is encouraging when you think about the outcomes that are typical for this patient population, especially with a median duration of response for the TP53-mutated high-risk patients of 10 months, which I think compares favorably to historical control for patients who had TP53-mutated high-risk disease. And many patients also achieved MRD negativity using local assays, especially flow cytometry, 45% of patients.
So, I think the totality of this data suggests that the efficacy and the safety profile of Bexmarilimab with azacitidine is good and promising and should be studied in a randomized phase 2, 3 study, which we are moving forward. We have worked with the sponsor to design a study that addresses many of the issues that happened in previous phase 3 studies, including the failed VERONA and other phase 3 studies. I’m referring you here to an editorial or a perspective we wrote in Blood about the issues related to drug development in high-risk MDS and the lessons we learned from these failed phase 3 trials, which we tried really to address in the design of the registrational global phase 2, 3 study that will look at aza placebo versus aza Bexmarilimab in the frontline setting, which we hope to start in 2026 and has been discussed already with the regulators, and I’m very excited about this study going forward.
So, in summary, I think the data totality suggests the safety and the efficacy of this combination, especially in the frontline setting, including patients with TP53-mutated disease and excellent survival in the relapsed refractory disease. And we look forward to seeing the data from the randomized registrational phase 2 study. And at the end, I’d like to thank the study participants, their families, and all the co-investigators on this trial, and I’ll be happy to take questions.