Drug Detail

Information about Masitinib

Generic Name
Brand Name (US)
AB Science
Drug Type
Tyrosine Kinase Inhibitor
Approval Status
Phase 3
Overall Strategy
KIT Protein Based
Block KIT
Drug Category
KIT/PDGFRA inhibitor

Masitinib is a potent KIT and PDGFRA inhibitor. It has entered into phase III trials for first and second line treatment for GIST.
We have also seen masitinib spelled as masatinib.
In early phase I/II trials, masitinib appears to be as effective or possibly more effective (the phase III trial is needed to confirm this) than imatinib given first-line.
Masitinib is dosed based on body weight.
Phase III trialls of Masitinib (AB1010) versus Imatinib in the first line and versus sunitinib in second line are now recruiting internationally. These trials are sponsored by the manufacturer AB Science. The first-line trial is for newly diagnosed GIST patients who have not received prior TKI therapy, but may have had adjuvant Imatinib. Patients must be either non-resectable or must have a recurrence after prior surgery. Patients will be randomized to receive either Masitinib 7.5 mg/kg or Imatinib 400 or 600 mg daily. Plans call for accrual of 222 patients and a primary end point of progression free survival.
Because masitinib does not target the abl kinase or the VEGF receptors, it has been speculated that it may have less heart toxicity than other KIT TKI's.
In February 2012, AB Science issued a press release about phase II trial results in imatinib-resistant GIST. Although results in this small trial suggest a longer overall survival time, some questions remain about this. For example, if treatment with TKI's was limited beyond Sutent, this would bias the trial results.
On October 30th, 2012, an application for marketing was accepted for review of masitinib for treatment of pancreatic cancer. This was based on improved overall survival (OS) of some patients in a randomized phase III trial. Improved survival was seen in patients with an unspecified blood biomarker indicative of aggressive disease (survival improved from 5 months to 11 months in this group) and in patients presenting with a certain threshold of pain intensity (45% of patients in the trial); where OS improved by 2.7 months to 8.1 months. However, in this trial, there was no difference in overall survival when the entire patient group was analyzed.
Note: Both the phase II (GIST) and phase III pancreatic cancer EMA reviews were initially turned down. Appeals may be ongoing.



AB Science announces a voluntary hold in the clinical studies of Masitinib worldwide (Jun, 2021).


AB Science website


Masitinib Phase II Trial Reports Benefit in New GISTs


Press Release - Masitinib extends overall survival in randomized phase II trial for GIST vs. Sutent for second line treatment


Masitinib, (AB1010), a potent and selective TKI of targeting KIT (PDF)


Comparative Toxicogenomics Database


Masitinib as an adjunct therapy for mild-to-moderate Alzheimer's disease: a randomised, placebo-controlled phase 2 trial


Oral tyrosine kinase inhibitor masitinib in combination with gemcitabine in patients with advanced pancreatic cancer: A multicenter phase II study.


AB Science reports phase 3 study results of masitinib in combination with Gemzar® for treatment of pancreatic cancer. Read more: AB Science reports phase 3 study results of masitinib in combination with Gemzar® for treatment of pancreatic cancer. - FiercePharma http://www.fiercepharma.com/press-releases/ab-science-reports-phase-3-study-results-masitinib-combination-gemzar-treat#ixzz2AsvsdSb7 Subscribe: http://www.fiercepharma.com/signup?sourceform=Viral-Tynt-FiercePharma-FiercePharma

Trials of this drug


Masitinib in Non Pre-treated, Inoperable Patients With Locally Advanced/Metastatic Gastro-intestinal Stromal Tumour (GIST)


A Phase 3 Study to Evaluate Efficacy and Safety of Masitinib in Comparison to Imatinib in Patients With Gastro-Intestinal Stromal Tumour in First Line Medical Treatment


A phase II study to compare efficacy and safety of masitinib at 12 mg/kg/day to sunitinib at 50 mg/day in treatment of patients with gastro-intestinal stromal tumor resistant to imatinib


Phase 1 dose-escalation study of oral tyrosine kinase inhibitor masitinib in advanced and/or metastatic solid cancers


Masitinib 2nd Line versus Sunitinib in Advanced GIST

Trial results

  Masitinib in advanced gastrointestinal stromal tumor (GIST) after failure of imatinib: A randomized controlled open-label trial - Annals of Oncology 2014 (note, this article has been peer-reviewed)
  Feb, 1st 2012 - AB Science press release - Positive clincial study data: Masitinib significantly extends overall survival as compared to Pfizer's Sutent in Gleevec-resistant GIST. Note this press release is controversial
  ASCO 2011 Poster - 4 years follow-up of the phase II trial.
  AACR Poster 2007:Abstract 5456 Final results of a Phase I trial of Masitinib (AB1010) in solid tumors. Soria, J.C.
  Eur J,. Cancer 6/21/2009 : Phase 1 dose-escalation study of oral tyrosine kinase inhibitor masitinib in advanced and/or metastatic solid cancers. Soria, J.C.
  ASCO 2007 Phase 2 Resulst Abstract 10025: Preliminary efficacy and safety results of Masitinib administered, front line in patients with advanced GIST. A phase II study.
  Eur J Cancer 2010 Phase 2 Results: Phase II study of oral masitinib mesilate in imatinib-naïve patients with locally advanced or metastatic gastro-intestinal stromal tumour (GIST).
  ASCO 2011 Phase 2 Results Abstract 85: Overall survival benefit with masitinib mesylate in imatinib-naive, locally advanced, or metastatic gastrointestinal stromal tumor (GIST): 4-years follow-up of the French Sarcoma Group phase II trial.
  ASCO 2009: Masatinib mesylate in imatinib-naive locally advanced or metastatic gastrointestinal stromal tumor (GIST): Results of the French Sarcoma Group phase II trial.

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