Imetelstat works:
Dr. Azra Raza: “So, I don’t think the high cost of the drug in general right now for me at least it’s a big issue. We can find those patients. And I do think that once the drug gets approved then everyone will be using this upfront. It’s not like we happily use lenalidomide or HMA in that group”.
“Now the key efficacy outcomes, of course, we are interested in transfusion independence. So, if you look at all the patients, all the treated patients, 12 achieved transfusion independence for a total response rate of 38%. Interestingly, patients who had not received these two FDA approved drugs, and had non-deletion 5q MDS, the response rate is much higher, 54%”.
“This is the waterfall plot of the longest transfusion free interval. And what it shows you basically is that these are patients, who have already gone almost to 80 weeks of being transfusion independent. Whenever we see a response lasting up for a while, that means the drug must be working at a more clonal stem cell level rather than just protecting the cells from dying”. “The median duration of transfusion independence is now heading to 2 years”.