It’s a study, it’s called the MANEUVER study. It’s a double-blind randomised study with three different parts. At ASCO this year we presented part 1 and now we have a longer follow-up that was presented at ESMO ’25. The study is about a randomisation for pimicotinib versus placebo in a 2:1 randomisation. This was for patients with unresectable diffuse-type tenosynovial giant cell tumours of the joints, usually of the bigger joints – of the knee, hip and ankle.
There are several drugs that are already studied in the same indication – pexidartinib and vimseltinib – so this is the third TKI in a row that is studied for this indication. The unique thing about this study is that it is being performed in both Asia and Europe and the US so it’s truly a global study. The outcomes of the study are very positive, so we are seeing after 24 weeks of treatment, which is the primary endpoint of the study, with longer follow-up that the response rate, according to RECIST is 54% at the ASCO presentation and now 76.2%. So it’s quite a high RECIST response rate. The tumour volume score also increased from 61.9% to 74.6% and a median duration of response was not reached.
What is the clinical significance of these results?
The clinical significance of these results is that we now have another TKI that will most likely be registered across the globe, at least in Asia, Europe and the US, that’s what I hope, that is highly effective but also has acceptable toxicity. The main toxicity of this drug is oedema and skin rash but it was all very much manageable. Some patients got a dose reduction from 50mg to 25mg per day but it all worked well, not only based on RECIST but the key secondary endpoints were much more important to me as a physician, and to the patients of course, and that’s the pain scale, the quality of life, the PROMIS quality of life questionnaires, and they all improved significantly. So we now have another option for these patients in the near future and in a situation where surgery is not always the first or later choice because of the high recurrence rate and the great morbidity associated with these surgeries.
Is there anything else you would like to add?
Just to be clear, after the first 25 weeks of placebo-controlled treatment, all patients were able to cross over to active treatment and this will be continued throughout the next year. So we will also get information on long-term efficacy and toxicity which is extremely important once we will use the drug in our clinical practices.
