Australian life sciences company, QBiotics Group, has announced the publication of positive results from a first-in-human phase 1 clinical trial of its anticancer pharmaceutical, tigilanol tiglate (EBC-46) in EBioMedicine, a peer-reviewed translational biomedical research journal by The Lancet.
The company said the trial was an open-label, single-arm, non-randomised, dose-escalation study.
Its key objectives were to determine the safety profile, tolerability, pharmacokinetics (PK) and preliminary anti-tumour efficacy of tigilanol tiglate when administered once by intratumoural injection.
It said tigilanol tiglate was generally well tolerated and doses escalated from 0.06 to 3.60 mg/m2, without reaching a maximum tolerated dose.
The study was conducted in 22 patients at four hospital sites in Australia. Patients were recruited with a range of tumour types including squamous cell carcinoma, basal cell carcinoma, melanoma, breast adenocarcinoma, atypical fibroxanthoma, atypical myxoid fibrosarcoma, metastatic colorectal adenocarcinoma, adenoid cystic carcinoma and angiosarcoma. Signs of clinical activity were observed in all nine tumour types, even at the lowest doses, it said.
“As this was a first-in-human, single dose safety study, the expectation of a strong anticancer response was low. However, the results revealed a 27% treatment response (in 6 patients), including an 18% complete response (in 4 patients) with full tumour destruction across a wide variety of solid tumour types," said QBiotics Group CEO and managing director, Dr Victoria Gordon.
“Solid tumours account for up to 80% of all tumour types,3 so the results from this Phase I study indicate potentially broad applications for tigilanol tiglate in a range of tumours, and an important advancement for our pharmaceutical,
“Additionally, two patients with melanoma demonstrated an anenestic (or abscopal) response, where non-injected tumours at different locations in the body also reduced in size. These results were achieved despite many patients not receiving an optimal dose."
Dr Gordon continued, “Given the very good safety profile, and positive antitumour responses observed, this study supports further development of tigilanol tiglate as a potential treatment of solid tumours.
“Results from this study also underpins selection of our initial lead indication and our recently announced Phase I/II trial of tigilanol tiglate in patients with Head and Neck Squamous Cell Carcinoma (HNSCC), in which the first patient was successfully dosed last week.”
The phase 1/2 open label 'QBC46-H03' study is a dose-escalation study in patients with HNSCC. It is aimed at determining the maximum tolerated dose (MTD) and recommended dose level for further studies. The study will also investigate safety, tolerability and tumour response following single or multiple (two to three) doses of tigilanol tiglate. It will enrol up to 40 patients from the Tata Medical Centre in Kolkata, the Tata Memorial Hospital in Mumbai, and other clinical sites in Australia.