Press release
Jun 5, 2018

MiNA Therapeutics Presents Initial Results at ASCO from First-in-Human MTL-CEBPA Study in Advanced Liver Cancer Patients

  • Well tolerated in patients with healthy and impaired liver function
  • Blood samples demonstrate proof of RNA-activating mechanism
  • Evidence of target engagement
Tokyo, Japan and London, UK, 5 June 2018 – Sosei Group Corporation (“Sosei”; TSE Mothers Index: 4565) announces that MiNA Therapeutics (“MiNA”), a UK company in which Sosei has a strategic minority holding, today announced preliminary results from its ongoing Phase I study of small activating RNA (saRNA) candidate MTL-CEBPA in advanced liver cancer. Below is an excerpt of today’s press release from MiNA. For full details, please see the media section of MiNA’s website here (English only).
In the study, MTL-CEBPA was found to mediate RNA-activating activity in white blood cells. The data are being presented at the 2018 American Society of Clinical Oncology (ASCO) Annual Meeting in a poster titled "Preliminary results of a first-in-human, first-in-class phase I study of MTL-CEBPA, a small activating RNA (saRNA) targeting the transcription factor C/EBP-a in patients with advanced liver cancer" in the Developmental Therapeutics – Clinical Pharmacology and Experimental Therapeutics poster discussion session being held on Monday 4 June 2018 from 3:00pm to 4:15pm CDT.
MTL-CEBPA was evaluated in the dose escalation part of a Phase I clinical trial in patients with advanced liver cancer. As of the data cut-off date of 31 March 2018, 23 patients had been treated once weekly at six dose levels (ranging from 28 mg/m2 to 160 mg/m2) and five patients had been treated twice weekly at 70 mg/m2.
MTL-CEBPA was well tolerated in patients at all doses and no Maximum Tolerated Dose has yet been identified. The large majority of adverse events (AEs) reported by investigators were mild to moderate in severity. Twelve (43%) patients experienced AEs no higher than Grade 2. AEs of Grade 3 or higher included hyperbilirubinaemia (11%), elevated gamma glutamyl transferase (GGT) (11%), hypophosphataemia (11%), anaemia (7%) and hypertension (7%). Only three (11%) patients discontinued treatment with MTL-CEBPA due to possible drug-related toxicities including acute coronary syndrome, hyperbilirubinaemia, and elevated GGT.
Pharmacokinetic data from this study showed that Cmax (peak plasma concentration of drug) and AUC (area under the curve) were dose proportional with no evidence of drug accumulation.
CEBPA gene expression was analysed in white blood cells of ten patients across multiple dose levels and timepoints. The level of CEBPA gene expression was significantly higher on treatment than at baseline, supporting target engagement of MTL-CEBPA. Consistent with up-regulation of CEBPA, which has a role in myeloid differentiation, significant and repeated increases in neutrophils were observed after dosing MTL-CEBPA.

Enrollment in the dose escalation part of the Phase I clinical trial has been completed. Enrollment is starting for the dose expansion part of the Phase I clinical trial in multiple sites in the United Kingdom and Asia. For more information, please contact MiNA at

MiNA Therapeutics is an associate company of Sosei. In May 2017, Sosei acquired a 25.6% equity share and an exclusive option to potentially acquire MiNA Therapeutics.