Hi All,
For anyone new to CML it might be good to explain that every year in December in United States there is a major conference for hematologists worldwide where any advances in treatments or results from trials are usually reported .So has anyone followed the last conference which of course was all done online given Covid was affecting USA ,world travel and so on ?
I have gleaned that as far as CML is concerned the major reportings would have been the advances made in the development of a new tki previously known as ABL 001 or now named Asciminib;a number of the presentations did refer to the results of trials and other studies related to this potentially new tki. In fact if it is approved for treatment it would be would be tki number 6.
Sandy has already posted the summary/full paper of the Phase III trials of Bosutinib versus Asciminib.In addition there were studies of the use of Asciminib in cases where there was an inadequate response or failure to Ponatinib.To put it in context Ponatinib is usually reserved as the last line defence against very rare mutations that cannot be treated by the prior 4 tkis;it has a reputation as being not easy to manage in terms of side effects and substantial and unpredictable vascular events.
Asciminib has been described as a novel agent and the first in the class of STAMP inhibitors -Specifically Targeting the Abl Myristol Pocket.I have read that it has a different binding site/mechanism to previous tki s and because of this does not or should not allow the development of hitherto previous mutations associated with the other tki s.So it could be a lifeline to those who have not responded well to Ponatinib or are at risk of vascular/thrombotic events because of a prior health condition.
In USA Asciminib has been granted Fast Track Designation and also Breakthrough Therapy Designation which basically means that it will be considered for approval for use in a rapid manner based on the results from trials and contribution to quality of life and being life saving. It seems its prospects look good .Usually in Europe the EMA will follow FDA and historically in UK we take the cue from our European neighbours.However post Brexit will it be assessed first by MHRA then NICE ? If it is finally approved for use in UK via the NHS it will no longer be in the class of being an experimental drug.Until that stage I guess even private insurers might not fund it.
If you Google Dr Jorges Cortes/Asciminib you will find a range of You Tube presentations by him and also E-cancer video on the Asciminib trials and studies.
Has anyone else been following either ASH or the development of this potentially new tki that looks likely to be approved for use?
Regards
John