Chapters Transcript Video Andrew A. Lane, MD, PhD discuss results from a phase 2 Study Tagraxofusp, azacitidine, and venetoclax (TAG-AZA-VEN) triplet therapy shows efficacy, tolerability, and transplant potential in patients with blastic plasmacytoid dendtritic cell neoplasm (BPDCN) BPDCN is a rare subtype of leukemia for which we have a center at Dana-Farber that studies the disease in the laboratory and in clinical trials for patients. And there's only one approved drug for BPDCN, and it's called tagraxapus. It targets a molecule called CD 123 on the surface of the BPDCN cells. And while tagraxapus is an efficacious drug and it helps get many patients into remission, There are some challenges with tagraxapus, being that we have to give daily infusions intravenously for 5 days, and that we also have to admit patients to the hospital during the first cycle to monitor for side effects of therapy. So with that basis and some work that we had done in the laboratory trying to understand mechanisms of response and resistance to tagraxapus in BPDCN, where we found that the resistance to tagraxapus can be overcome by treatment with a drug called azacytidine, which is a chemotherapy drug that we already use in leukemia, and that venetaclax, which is a BCL-2 inhibitor, is particularly potent in BPDCN cells. So with that information, we first performed a study in patients with AML and MDS that was published last year where we figured out the safe doses to combine the triplet regimen of tagraxapus, azacytidine, and venetoclax. And here what we're presenting is the phase 2 data of 27 patients, about half and half each with previously untreated, so newly diagnosed BPTCN or relapsed BPTCN, where we use the triplet combination. And the bottom line is that the combination was safe. We didn't see any new safety signals that were unexpected based on what we know about tagraxahos be alone or Venetoclax azacytidine alone or from the triplet that we had seen in patients with AML. And secondly, we saw a good signal of efficacy. So we saw a very high response rate, including that most patients achieved a complete response, including nearly all of the patients with previously untreated disease. And that we were able to get many patients to an allogeneic stem cell transplant after the treatment, which is usually our goal with patients with BPDCN after we get them into remission. And it looked like the capillary leak syndrome, which is the key toxicity of tagraxapus that we follow patients for in the hospital, might be the same or even a little bit better with this regimen of Tegraxapus veneneclaxinazacytidine, probably because in this regimen we give only 3 days of tagraxapus as opposed to the 5 days when it's given as a single agent in BPDCN. So we're really excited about these results, and we think that this could actually lead to a new standard of care treatment for BPDCN. A caveat is that of course this regimen is not tested head to head against any of the other regimens we use in BPDCN. It's very difficult to do that in this rare disease, but I think given that there's a lot of familiarity with venetoclax and azacytidine already in leukemia patients, it's an approved regimen for patients with AML. And because we're combining it with an already approved drug in BPTCN, we hope that this may become a new regimen that can help more patients with potentially less toxicity. Published December 18, 2025 Created by Related Presenters Andrew Lane, MD, PhD Medical Oncology View full profile
