Checking on the systemic mastocytosis landscape with a focus on the potential of Ayvakit (avapritinib), BLU-263, and Bezuclastinib (CGT9486/ PLX9486)Ticker(s): BPMC, COGT
Institution: MD Anderson
- Associate Professor of Medicine, Department of Leukemia at MD Anderson.
- Manages 40 patients with systemic mastocytosis.
- Research focus is on myeloproliferative neoplasms (MPNs). Heavily involved in clinical trial design for patients with MPN, and also in other areas of MPN research; leads number of important therapeutic clinical trials in myelofibrosis at MDACC, as well as in other MPNs such as essential thrombocythemia and systemic mastocytosis (PI on APEX, SUMMIT, HARBOR).
Please describe your clinical practice; roughly how many patients with systemic mastocytosis do you currently manage?Added By: joe_mccann
What % of your patients do you believe are a good candidate for Ayvakit?Added By: joe_mccann
What is the treatment paradigm of aggressive SM now with Ayvakit approved? What is the treatment paradigm of indolent SM?Added By: joe_mccann
What % of patients do you plan to prescribe Ayvakit to? Any difference in usage for aSM vs. SM with hematologic neoplasm vs. mast cell leukemia? Are there any patients you are using Midostaurin ahead of Ayvakit?
What are your thoughts on the label? Any concerns with the Intracranial Hemorrhage warning?
Has the cost of the drug posed any issues for reimbursement yet? $400K annual list price
- What is your best estimate for the total annual drug cost for your typical iSM patient today? Do you think Avapritinib’s current price of $400K/year will be a barrier to use in iSM?
How do you currently treat iSM patients? What % of patients are you able to control symptoms with currently available treatment options?Added By: joe_mccann
What is the remaining unmet need in iSM?
What are your thoughts on the initial avapritinib data in iSM patients from PIONEER?
What % of your iSM patients could benefit from avapritinib? If approved and the profile is consistent with reported data, what % of patients would you prescribe it to?
Are you familiar with the recent Celldex data for its anti-KIT antibody in a couple other mast cell diseases? Do you have any views on whether this drug could be a viable treatment in iSM given the strong reductions in skin mast cells and serum tryptase?
Are any other development candidates in iSM interesting to you?
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