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Ref Type Abstract
PMID
Authors
Title 1132P Regorafenib in Caucasian patients with pretreated advanced KIT-mutant melanoma: A dual center case series
Journal Vol Issue Date Pages Journal Short Title
Annals of Oncology 35 Supplement 2 September 2024 S743-S744 Ann Oncol
URL https://www.annalsofoncology.org/article/S0923-7534(24)03654-8/fulltext
Abstract Text Background Oncogenic KIT-mutations (KITmut) are found in +/- 3% of melanoma patients (pts), most frequently in mucosal (15-21%), acro-lentiginous (11-23%), and melanoma arising on sun-damaged skin (CSDS; 16-27%). Only a minority of pts (20-30%) responds to treatment with small molecule KIT-inhibitors (e.g, imatinib, sunitinib, nilotinib, dasatinib). The activity of regorafenib (REGO, a multi-targeted small molecule inhibitor of KIT, TIE2, VEGFR, PDGFR, RET, RAF, and CSF-1R kinases) has never been studied in a Caucasian population with KIT-mutant melanoma. Methods The outcome of a prospectively identified cohort of advanced KITmut melanoma pts treated with REGO at two medical centers in the EU was investigated (database lock (DBL): 25APR2024). Results Eight patients were included: median age 60.7 years [range 41-76]; Caucasian (100%); AJCC stage IV-M1c: 7 pts, and -M1d: 1 pt; elevated baseline LDH: 3 pts; ECOG performance status 0 and 1: resp. 6- and 2 pts); primary mucosal 2-, acro-lentiginous 3-, and CSDS 3 pts. Oncogenic KITmut were documented in exon 11 (n=5), -13 (n=2), and -17 (n=1). Three pts participated in the prospective REGOMEL phase II clinical trial and 4 pts were treated on a compassionate use basis. All pts were pretreated and had previously progressed on anti-PD-1 and -CTLA-4 based therapy, 3 pts on imatinib. REGO was administered orally, once daily (QD), continuously, at a dose of 40 to 120 mg (n=7), or on a 21/28d regimen of 120 mg QD (n=1). In 7 response evaluable pts, the best overall responses (RECIST v. 1.1) were 1 complete response and 6 partial responses (overall response rate 100%, all responses were confirmed). After a median follow-up of 43w [range 3-65w], treatment and responses were ongoing in respectively 6 and 4 pts. Median duration of response was not reached [range 6-59w]. Median progression free survival was not reached and all pts were alive at DBL. All pts had at least 1 TRAE. Grade 3 TRAE included arterial hypertension, and digestive bleeding (n=1, each). Conclusions REGO demonstrated a manageable safety profile and high anti-tumor activity in this Caucasian population of pretreated advanced KITmut melanoma pts deserving further evaluation in a prospective clinical trial. Legal entity responsible for the study The authors. Funding Bayer

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Molecular Profile Treatment Approach
Gene Name Source Synonyms Protein Domains Gene Description Gene Role
Therapy Name Drugs Efficacy Evidence Clinical Trials
Drug Name Trade Name Synonyms Drug Classes Drug Description
Gene Variant Impact Protein Effect Variant Description Associated with drug Resistance
Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
KIT act mut melanoma predicted - sensitive Regorafenib Case Reports/Case Series Actionable In a clinical case study, Stivarga (regorafenib) treatment demonstrated safety and preliminary activity in patients with melanoma harboring activating mutations in KIT, resulting in an overall response rate of 100% (7/7, 1 complete and 6 partial responses), with median duration of response and median progression-free survival not reached and response ongoing in 4 patients at a median follow-up of 43 weeks (Ann Oncol (2024) 35 (suppl_2): S743-S744). detail...