What does the current pipeline in MDS look like?

FAQ Library published on October 4, 2019
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David A. Sallman, MD
Assistant Member
Malignant Hematology Department
Moffitt Cancer Center
Tampa, Florida
What does the current pipeline in MDS look like?

My name is Dr. David Sallman from Moffitt Cancer Center. I am an Assistant Professor in the Malignant Hematology Department. I wanted to just give briefly an exciting update as far as the potential pipeline in myelodysplastic syndrome. The plenary session at ASH of last year was of course with luspatercept, which showed an improvement as far as transfusion independence in patients that had refractory anemia with ring sideroblasts and had failed erythropoietin- stimulating agents, and so we are hoping that potentially luspatercept could be our first new approved drug as early as late 2019 if not in early 2020. This again is for a low-risk MDS patient population. I think in high-risk MDS patients, the combinations with azacitidine are quite exciting, specifically for P53 which is a group with the poorest outcomes. There has been data presented previously of azacitidine in combination with APR-246 with significantly improved response rates. There is now an ongoing phase 3 study testing this combination versus azacitidine alone. I think the other exciting things will be both for approved agents for other indications as well as some other novel therapies, again in combination with azacitidine or decitabine. The ones to highlight would be venetoclax, which again has changed the paradigm in combination with hypomethylating agents for AML, but also ivosidenib and enasidenib are showing significant activity both by themselves and in combination with HMA therapies. Lastly, some of the more novel immune therapies are azacitidine with 5F9 which inhibits a “do not eat me” signal, as well as potential other markers such as anti-TIM-3 therapies. I think hopefully we can really move the bar forward for patients above azacitidine on its own.

Last modified: November 15, 2019