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The FDA has granted an orphan drug designation to vebreltinib for the treatment of patients with non–small cell lung cancer harboring MET genomic tumor aberrations.
The FDA has granted an orphan drug designation (ODD) to vebreltinib (APL-101) for the treatment of patients with non–small cell lung cancer (NSCLC) harboring MET genomic tumor aberrations.1
Vebreltinib is a novel small molecule kinase inhibitor targeting c-MET, and it is a type 1b class highly selective c-MET inhibitor. The ongoing global phase 2 SPARTA trial (NCT03175224) is evaluating vebreltinib in patients with NSCLC and other solid tumors with MET genomic dysregulation.
“While NSCLC is the most common type of lung cancer, a subset of patients will have MET genomic dysregulations in their tumors which make them more resistant to treatment, presenting an unmet medical need,” Guo-Liang Yu, PhD, co-founder, chairman and chief executive officer of Apollomics, stated in a press release. “We are pleased to have received the ODD for vebreltinib, as patients need new and better treatment options. Through genomic testing, we can identify patients who will benefit most from a targeted treatment like vebreltinib.”
Preclinical studies have shown that vebreltinib demonstrated strong tumor inhibitory effects in c-MET dysregulated human gastric, hepatic, pancreatic and lung cancer xenograft animal models and patient-derived xenograft models.
Previously reported data from clinical trials showed that vebreltinib (PLB1001) displayed a generally well-tolerated safety profile and preliminary evidence of clinical activity in patients with NSCLC harboring a mutation that leads to MET exon 14 skipping and in patients with secondary glioblastoma multiforme harboring MET fusion and/or exon 14 skipping with evidence of brain penetration.
SPARTA is examining the efficacy of vebreltinib in patients with NSCLC with c-MET exon 14 skipping mutations, patients with cancers associated with c-MET amplifications, and patients with cancers associated with c-MET fusion.2
The trial is comprised of 7 cohorts:
Key exclusion criteria for all patients include hypersensitivity to vebreltinib, excipients of the drug product, or other components of the study treatment regimen; or known actionable mutation/gene rearrangement of EGFR (except for cohort C), ALK, ROS1, RET, NTRK, KRAS, and BRAF.
All patients in each cohort will receive 200 mg of oral vebreltinib twice daily. The primary end point of the trial is objective response rate per blinded independent review committee per RECIST v1.1 criteria. Secondary end points include duration of response, clinical benefit rate, time to progression, progression-free survival, overall survival, and safety.