Zahra Mahmoudjafari:
There have been several updates in the setting of hematology. The session covering hematology updates and new drug updates in this specialty has been quite remarkable. Since September 2023, the presentation covers changes for four new drugs and 11 indication expansions. There's a lot to see here, and this presentation really is very comprehensive.
Going through a couple of the new drugs, a couple of them are for transplant, and a couple of them are for more leukemias and MDS, I should say. The first is motixafortide, which is a product that is utilized for mobilization in patients with multiple myeloma undergoing autologous transplant. This drug was approved by the FDA last year, looking at mobilization strategies, and really in the GENESIS trial showed a really improved ability for patients to mobilize. Over 90% of patients in that clinical trial did show demonstrated improvement in the ability to collect for transplant, which is fairly remarkable, because we know multiple myeloma patients often have some significant challenges with collection in autologous transplant.
There are some specific nuances though with motixafortide to be aware of, including injection site reactions and potential for some more flushing or pruritus. It's important to remember that patients should receive pre-medications, and there is a monitoring period post the dose of that infusion. Some important ramifications for advanced practice providers when you think about how to utilize this in clinical practice.
The second drug that's covered in this presentation is imetelstat, which is for the treatment of transfusion-dependent MDS. Again, some significant improvements seen here. It's an IV infusion that did show remarkable improvement for transfusion-dependent MDS patients over the course of 24 weeks.
The other drug that was approved that is reviewed in this presentation is denileukin for CTCL. This is an older drug that's come back to the market for patients with cutaneous CTCL. And I would definitely say that while it's exciting that this is back as an option for these patients, it does still have some significant side effects to be cognizant of.
The last new drug that was approved, that was presented in this presentation, is axatilimab. This is an IV agent for the treatment of graft-vs.-host disease in patients receiving allogeneic transplant after two or more lines of therapy. What's interesting about axatilimab is it presents a novel treatment option for these patients that really are running out of options. The other agents that are approved for chronic graft-vs.-host disease after two or more lines of therapy are typically oral therapies, which can be difficult to obtain and may not be the best option for certain patients that have challenges with chronic graft-vs.-host disease.
Axatilimab is an IV option. It's given 0.3 milligrams/kilo every two weeks, and this is as a result of the AGAVE trial. And what was nice about the AGAVE trial is it's a phase 2 trial that looked at three different doses of axatilimab. And what they actually found, and traditionally they don't find this, is that the lowest dose given every two weeks was the most effective dose and also the dose that led to the least amount of side effects. Ultimately that's where the approval came in with axatilimab at 0.3 milligrams/kilo every two weeks.
While this was approved by the FDA and we wanted it to be readily available, it is not yet readily available and should be here in the spring of 2025. It is available under an expanded access program for centers that have the ability to administer it, and it has been generally a pretty effective option.
The challenge that comes with axatilimab though is that it is an IV, so that does ask the patient to come in every two weeks for an IV infusion. And then there are some important monitoring parameters to keep in mind for patients, making sure that we are monitoring their liver function tests and their amylase/lipase. Most of the other toxicities that we've seen with axatilimab in the trial was related to those higher doses. Given that we have approval for the lowest of the three doses, I don't expect to see that many significant side effects, and really am excited as a potential treatment option for these patients receiving this therapy.
Rounding out the rest of the presentation included the information related to the expanded approvals, and there were 11 that were presented in this presentation. I'm not going to spend the entire time talking about every single expanded approval, but really what we saw here were expanded approvals for follicular lymphoma, CLL, multiple myeloma, and ALL. I would definitely say this is a presentation not to miss for those of you that really are looking to see what the new updates are in hematology.
