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Ref Type Journal Article
PMID (32357963)
Authors Sarker D, Plummer R, Meyer T, Sodergren MH, Basu B, Chee CE, Huang KW, Palmer DH, Ma YT, Evans TRJ, Spalding DRC, Pai M, Sharma R, Pinato DJ, Spicer J, Hunter S, Kwatra V, Nicholls JP, Collin D, Nutbrown R, Glenny H, Fairbairn S, Reebye V, Voutila J, Dorman S, Andrikakou P, Lloyd P, Felstead S, Vasara J, Habib R, Wood C, Saetrom P, Huber HE, Blakey DC, Rossi JJ, Habib N
Title MTL-CEBPA, a Small Activating RNA Therapeutic Upregulating C/EBP-α, in Patients with Advanced Liver Cancer: A First-in-Human, Multicenter, Open-Label, Phase I Trial.
Journal Clinical cancer research : an official journal of the American Association for Cancer Research
Date 2020 May 01
Abstract Text Transcription factor C/EBP-α (CCAAT/enhancer-binding protein alpha) acts as a master regulator of hepatic and myeloid functions and multiple oncogenic processes. MTL-CEBPA is a first-in-class small activating RNA oligonucleotide drug that upregulates C/EBP-α.We conducted a phase I, open-label, dose-escalation trial of MTL-CEBPA in adults with advanced hepatocellular carcinoma (HCC) with cirrhosis, or resulting from nonalcoholic steatohepatitis or with liver metastases. Patients received intravenous MTL-CEBPA once a week for 3 weeks followed by a rest period of 1 week per treatment cycle in the dose-escalation phase (3+3 design).Thirty-eight participants have been treated across six dose levels (28-160 mg/m2) and three dosing schedules. Thirty-four patients were evaluable for safety endpoints at 28 days. MTL-CEBPA treatment-related adverse events were not associated with dose, and no maximum dose was reached across the three schedules evaluated. Grade 3 treatment-related adverse events occurred in nine (24%) patients. In 24 patients with HCC evaluable for efficacy, an objective tumor response was achieved in one patient [4%; partial response (PR) for over 2 years] and stable disease (SD) in 12 (50%). After discontinuation of MTL-CEBPA, seven patients were treated with tyrosine kinase inhibitors (TKIs); three patients had a complete response with one further PR and two with SD.MTL-CEBPA is the first saRNA in clinical trials and demonstrates an acceptable safety profile and potential synergistic efficacy with TKIs in HCC. These encouraging phase I data validate targeting of C/EBP-α and have prompted MTL-CEBPA + sorafenib combination studies in HCC.


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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
CEBPA-51 MTL-CEBPA|NOV340 SMARTICLES|MTL-501 CEBPA-51 (MTL-CEBPA) is a small activating RNA that induces expression of CEBPA mRNA and protein, which subsequently leads to upregulation of albumin, and thereby potentially inhibiting cell growth (PMID: 28882451, PMID: 32357963).
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
Unknown unknown hepatocellular carcinoma not applicable CEBPA-51 Phase I Actionable In a Phase I trial, CEBPA-51 (MTL-CEBPA) treatment was well-tolerated and demonstrated manageable safety, and resulted in an objective response rate of 4% (1/24) in hepatocellular carcinoma patients with one partial response that lasted for 2 years, and stable disease as the best response in 12 patients, and resulted in a mean progression-free survival of 4.9 months (PMID: 32357963; NCT02716012). 32357963
Unknown unknown hepatocellular carcinoma not applicable Lenvatinib Case Reports/Case Series Actionable In a Phase I trial, a hepatocellular carcinoma patient treated with Lenvima (lenvatinib) following the discontinuation of CEBPA-51 (MTL-CEBPA) treatment achieved a partial response, but demonstrated progression at 6 months following treatment (PMID: 32357963; NCT02716012). 32357963
Unknown unknown hepatocellular carcinoma not applicable Regorafenib Case Reports/Case Series Actionable In a Phase I trial, two hepatocellular carcinoma patients treated with Stivarga (regorafenib) following the discontinuation of CEBPA-51 (MTL-CEBPA) treatment achieved stable disease 3 months following treatment, but the treatment was discontinued due to toxicity (PMID: 32357963; NCT02716012). 32357963
Unknown unknown hepatocellular carcinoma not applicable Sorafenib Case Reports/Case Series Actionable In a Phase I trial, three hepatocellular carcinoma patients treated with Nexavar (sorafenib) following CEBPA-51 (MTL-CEBPA) treatment achieved complete radiologic response (PMID: 32357963; NCT02716012). 32357963