Preliminary top-line results from the study also revealed no reports of drug-related deaths.
Milciclib is a small molecule inhibitor of several cyclin-dependent kinases involved in the progression of the cell cycle. Overexpression of these kinases is believed to be related to the development of chemotherapy resistance.
The multi-centre, single-arm, repeated-dose trial assessed the safety, tolerability and anti-tumour activity of the drug as a monotherapy in 31 sorafenib-refractory or intolerant unresectable or metastatic advanced HCC patients.
Conducted in Italy, Greece, and Israel, the study measured overall safety as the primary endpoint, while the secondary efficacy endpoints were progression-free survival (PFS) and time to progression (TTP).
Of the 28 evaluable subjects, 14 completed the six-month period of the trial. Milciclib demonstrated manageable toxicities, with the most frequent adverse events being diarrhoea, nausea, and fatigue.
Following the trial, nine out of 14 patients continued treatment under compassionate use.
Four patients were treated with the study drug for nine, 11, 13 and 16 months. One of the remaining five patients is at eight months, three are at nine, and one is at 11 months of therapy.
Tiziana Life Sciences CEO and CSO Dr Kunwar Shailubhai said: “We are very pleased with the clinical activity and tolerability of Milciclib in these advanced cases of HCC. It is an important milestone to move forward with further clinical development of Milciclib either as a single agent or in combination with other HCC drugs.”
Prior Phase I and Phase II trials also yielded positive data for the drug in advanced solid malignancies such as thymic carcinoma, thymoma, colon, and pancreatic cancer.