- First in-human Clinical Phase I study of intratumoural tigilanol tiglate 
(EBC-46) has been published in The Lancet's EBioMedicine journal; 
- An efficacious dose was achieved and a Maximum Tolerated Dose (MTD) was not 
reached, indicating tigilanol tiglate tolerability in humans; 
- Promising outcomes attained in patients with a variety of solid tumour types, 
with a 27% treatment response, including 18% complete response (full tumour 
destruction);[1] 
- Following these positive results, a Phase I/II trial of tigilanol tiglate in 
patients with Head and Neck Squamous Cell Carcinoma (HNSCC) is underway, with 
the first patient successfully dosed. 

Australian life sciences company, QBiotics Group Limited (QBiotics) is today 
announcing the publication[1] of positive results from a first in-human Phase I 
clinical trial of its anticancer pharmaceutical, tigilanol tiglate (EBC-46) in 
EBioMedicine, a peer-reviewed translational biomedical research journal by The 
Lancet. 

The key objectives of this Phase I, open-label, single-arm, non-randomised, 
dose-escalation study were to determine the safety profile, tolerability, 
pharmacokinetics (PK), and preliminary antitumour efficacy of tigilanol tiglate 
when administered once by intratumoural injection. Tigilanol tiglate was 
generally well tolerated and doses escalated from 0.06 to 3.60 mg/m[2], without 
reaching a maximum tolerated dose. 

The study was conducted in 22 patients at four hospital sites in Australia[1].  
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. 

"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. 

"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," said QBiotics Group CEO and Managing Director, Dr Victoria 
Gordon. 

"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,[1]" added Dr Gordon. 

The vast majority (96%) of adverse events (AEs) were mild to moderate, with the 
most commonly reported AE being injection site reaction related to the mode of 
action of tigilanol tiglate. AEs were generally managed with symptomatic 
therapy.[1] 

"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," said Dr Gordon

"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," Dr Gordon said.

The Phase I/II open label "QBC46-H03" study, is a dose escalation study in 
patients with HNSCC 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. 

ISSUED BY                           
 QBIOTICS GROUP LIMITED - www.QBiotics.com   
 

 
 
FOR FURTHER
INFORMATION
 DR VICTORIA GORDON, CEO & MANAGING DIRECTOR, QBIOTICS GROUP 
victoria.gordon@qbiotics.com or + 61 418 453 737
 

ABOUT QBIOTICS


QBiotics is a public unlisted Australian life sciences company which discovers, 
develops and commercialises novel anticancer and wound healing small molecules 
for human and veterinary markets. Its lead product, tigilanol tiglate, is an 
anticancer pharmaceutical targeting a range of solid tumours across multiple 
species. QBiotics' business model is to develop products that have application 
in both human and veterinary markets. Success in the veterinary programs 
validates QBiotics' technology and de-risks human development, while generating 
early, non-diluting revenues. 

More information: https://qbiotics.com

ABOUT TIGILANOL TIGLATE 

Tigilanol tiglate is a novel, small molecule undergoing development as an 
intratumoural injection treatment for solid tumours. Its effect on tumours is 
multimodal and involves direct local effects on the injected tumour as well as 
effects on distal, non-injected tumours. Complete destruction of the injected 
tumour is mediated via tumour vascular disruption as well as death of tumour 
cells by oncosis. Following tumour destruction, rapid wound healing has been 
shown to ensue.

A single injection of tigilanol tiglate has been shown in canine patients to 
ablate (completely destroy) 75% of treated tumours.[4] Veterinary use of 
tigilanol tiglate (branded STELFONTA(R) has recently received a majority vote 
by the European Medicines Authority (EMA) for marketing authorisation in Europe 
as a treatment for canine mast cell tumours, and is also under review by the US 
Food and Drug Administration - Center for Veterinary Medicine (FDA-CVM) for 
marketing in the USA. STELFONTA(R) is partnered with Virbac, a global animal 
health company for marketing and distribution in the EU and USA pending 
regulatory approval. Launch of the product is expected early 2020 in both 
regions. 

References
 
[1]Panizza, B.J., et al., Phase I dose-escalation study to determine the 
safety, tolerability, preliminary efficacy and pharmacokinetics of an 
intratumoral injection of tigilanol tiglate (EBC-46). EBioMedicine, 2019.
 
[2]Squamous cell carcinoma, melanoma, basal cell carcinoma, breast 
adenocarcinoma, atypical fibroxanthoma, atypical myxoid fibrosarcoma, 
metastatic colorectal adenocarcinoma, adenoid cystic carcinoma and angiosarcoma
 
[3]Dunn, B., Cancer: Solving an age-old problem. Nature, 2012. 483(7387): p. 
S2-6.
 
[4]QBiotics Group Ltd., Data on file. 2019.
 

SOURCE  QBiotics Group Ltd.