ABL001 for Treating CML: Timothy P. Hughes, MD

ABL001 is a small molecule inhibitor of BCR-ABL, which is the same protein target of nilotinib and imatinib. Unlike the TKIs, ABL001 does not bind in the ATP domain; instead, it binds to a myristate site that causes a conformational change and switches off the kinase activity. “The advantage is that it doesn’t have effects on other kinases, unlike all of the TKIs, so it is better tolerated,” Hughes explained.

The phase 1 ABL001 study recruited over 100 patients at the time of the meeting, with more than 12-months of follow-up on some patients. “Tolerance and safety continue to be excellent and responses have been encouraging in many patients who were resistant to at least 2 TKIs,” Hughes said.

Read abstract here