QBiotics Group Ltd has achieved a significant regulatory milestone as its pioneering intratumoural oncology asset, tigilanol tiglate, receives Orphan Drug Designation (ODD) from the United States Food and Drug Administration (FDA) for the treatment of soft tissue sarcoma.
The designation underscores the drug's potential as a promising treatment for rare diseases affecting fewer than 200,000 individuals annually in the US, offering a seven-year exclusive marketing period post-approval, fee exemptions, and tax credits for clinical trials.
Strategic milestone in oncology
"Soft tissue sarcomas constitute a rare group of tumours comprising more than 80 subtypes that affect both adults and children," QBiotics executive director of Strategic Alliances and Investor Relations Dr Victoria Gordon said.
"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.”
Clinical trial insights
The company's ongoing Phase 2 clinical trial (QB46C-H07) at Memorial Sloan Kettering Cancer Center, under the guidance of Principal Investigator Edmund Bartlett, MD, is evaluating the efficacy and safety of tigilanol tiglate in advanced and/or metastatic soft tissue sarcomas (STS). With an estimated 124,573 new cases of STS globally in 2021 and a growing incidence rate, the trial's outcomes are eagerly anticipated.
The global prevalence of soft tissue sarcoma
Data from GlobalData® and the American Cancer Society highlight the global and US incidence of soft tissue sarcoma, emphasising the need for innovative treatments. The Cancer Council of Victoria also notes the impact of this cancer type in Australia, with around 1,600 diagnoses annually.
QB46C-H07 aims to establish the preliminary efficacy and safety of tigilanol tiglate, focusing on tumour volume reduction, adverse event incidence, and pharmacokinetics. The trial also explores recurrence rates and tumour response in biopsy samples, providing a comprehensive evaluation of the drug's impact.
QBiotics, an Australian life sciences company, is at the forefront of developing pharmaceuticals from nature for unmet medical needs in humans and animals.
With a business model that spans veterinary and human markets, QBiotics emphasises the validation and de-risking of its technology through success in veterinary programs. Tigilanol tiglate, alongside a leading wound healing product, represents QBiotics' commitment to addressing cancer and chronic wounds through innovative treatments.
The promise of Tigilanol Tiglate
Tigilanol tiglate's novel mechanism, inducing tumour cell death, stimulating immune responses, and promoting wound healing, positions it as a groundbreaking approach to treating solid tumours.
With ongoing clinical trials for head and neck cancer and soft tissue sarcoma, alongside its veterinary success with STELFONTA®, QBiotics continues to pioneer in the field of oncology and wound care.