QBiotics drug tigilanol tiglate awarded Orphan Drug Designation for patients with soft tissue sarcoma

Brisbane, 16 February 2024

QBiotics Group Limited (QBiotics) is pleased to announce that its lead intratumoural oncology asset, tigilanol tiglate, has been awarded Orphan Drug Designation for the treatment of soft tissue sarcoma by the United States Food and Drug Administration (FDA).

Orphan Drug Designation (ODD) is a special status for drugs considered promising potential treatments for patients with rare (‘orphan’) diseases, being less than 200,000 cases per annum in the United States. The designation can provide for a seven-year window of exclusive marketing rights, post-approval, as well as exemption from user fees and eligibility for tax credits for qualified clinical trials. In addition to the financial benefits, it may also potentially shorten clinical development due to closer collaboration with the FDA.

QBiotics’ Executive Director, Strategic Alliances & Investor Relations, Dr Victoria Gordon commented, “Soft tissue sarcomas constitute a rare group of tumours comprising more than 80 subtypes that affect both adults and children. The prognosis of advanced soft tissue sarcoma patients remains unfavourable and new treatments are urgently needed. The FDA Orphan Drug Designation for tigilanol tiglate signals an important milestone for QBiotics, reflecting its recognition by the FDA as a potential new treatment option for this debilitating and life-threatening disease.”

QBiotics’ Phase II clinical trial (QB46C-H07) is being conducted at Memorial Sloan Kettering Cancer Center in New York, USA with Edmund Bartlett, MD as Principal Investigator. The trial is assessing the preliminary efficacy and safety of tigilanol tiglate in patients with a range of advanced and/or metastatic STS.

GlobalData estimates that there were 124,573 new cases of STS globally in 2021, with the incidence growing at 0.54% per year.


jane.lowe@irdepartment.com.au  +61 411 117 774



GlobalData estimates that there were 124,573 new cases of soft tissue sarcoma globally in 2021, with the incidence growing at 0.54% per year.

The American Cancer Society estimates that in 2024 about 13,590 new soft tissue sarcomas will be diagnosed in the United States.

According to the Cancer Council of Victoria, around 1,600 Australians are diagnosed with a soft tissue sarcoma each year.



QB46C-H07 (Clinical trial registration number NCT05755113) is a pilot Phase II, open label clinical trial to evaluate the preliminary efficacy and safety of intratumoural tigilanol tiglate in patients with a range of advanced and/or metastatic soft tissue sarcoma.

The Primary Endpoint is ablation rate defined as the proportion of patients achieving >30% reduction in tumour volume assessed by ultrasound compared to baseline.

The Secondary Endpoints are incidence of adverse events and serious adverse events, and pharmacokinetics.

Exploratory Endpoints include local rate of recurrence at the injection site at 6 months post initial injection, and assessment of tumour response in biopsy samples. Biopsies are planned at baseline and at 14 days post-injection, and surgical specimens and blood samples will assess changes in tumour biomarkers.



QBiotics is an unlisted public Australian life sciences company that discovers and develops pharmaceuticals derived from nature to address unmet medical needs in humans and companion animals. Our current clinical focus is on novel treatments for cancer and chronic wounds.

QBiotics’ business model is to develop products that have applications in both veterinary and human markets. Success in the veterinary programme validates QBiotics technology and de-risks early stages of human development while having the potential to generate early revenue.

QBiotics’ lead oncology product, tigilanol tiglate, is a small molecule targeting a range of solid tumours and is currently in two human clinical Phase II trials treating (i) head and neck cancer, and (ii) soft tissue sarcoma. A veterinary formulation of tigilanol tiglate is registered and marketed as an oncology pharmaceutical, under the trade name STELFONTA®, in the USA, Europe, the UK and Australia.

QBiotics’ lead wound healing product is a small molecule targeting a range of wounds including chronic and infected wounds and burns. Preparation is in progress for a first-in-human Phase I/IIa clinical trial in patients with venous leg ulcers.




Tigilanol tiglate is a novel, small molecule drug discovered, and in development, by QBiotics as an intratumoural treatment for solid tumours. Tigilanol tiglate has a multifactorial mode of action that induces tumour cell death by oncosis, stimulates an immune response1 and initiates wound healing at the treatment site resulting in good functional and cosmetic outcomes.



  1. Cullen et al., 2022. Tigilanol tiglate is a naturally occurring small molecule oncolytic that effectively ablates tumors via intratumoral injection and can enhance response to immune checkpoint blockade. Journal Immunotherapy of Cancer 10 (Suppl 2): A1–A1595