Qbiotics Pty Ltd.

Tigilanol tiglate is a chemical found in the seeds of the pink fruit of the blushwood tree. Image credit: Shutterstock/petrmalinak. Australian biotech QBiotics has won an orphan drug designation for a drug based on a chemical obtained from the blushwood tree, which is being investigated as a treatment for a rare group of cancers called soft tissue sarcoma . Qbiotics will now be in line for tax credits for US-based clinical trials and, if the therapy is approved, the potential for seven years of market exclusivity for the given indication. A veterinary formulation of the drug, tigilanol tiglate, is already approved for use in the US, UK, and Australia under the trade name Stelfonta. QBiotics started life as EcoBiotics, which was established in 2000 to discover potential drugs from Queensland’s tropical rainforest. QBiotics itself was founded in 2004 as a subsidiary of EcoBiotics, and the two groups merged in 2017. The company has signed several biodiscovery agreements covering the rainforest areas in the country in the past decade. The company has not disclosed how it scales tigilanol tiglate – a chemical found in the seeds of the pink fruit of the blushwood tree. Tigilanol tiglate is administered directly into a solid tumour mass by intratumoural injection. See Also: Regeneron and Sanofi’s Dupixent bags fifth drug label in Japan Dragonfly and Gilead enter partnership for cancer therapy The drug candidate works by disrupting blood vessels keeping the tumour alive and directly killing cancer cells by oncosis. QBiotics states that tigilanol tiglate also induces tumour site healing and can even be administered systemically to induce indirect anti-tumour responses. QBitotics’ Phase II clinical trial in soft tissue sarcoma (NCT05755113) is ongoing with ten participants estimated to be enrolled. The primary endpoint is tumour ablation. Soft tissue sarcoma is the broad term for rare cancers that grow in soft tissues anywhere in the body. There are several targeted therapies approved to treat soft tissue sarcoma – last month, Adaptimmune applied to the FDA for approval of afami-cel. If approved, it would be the first T-cell therapy for a type of soft tissue sarcoma. QBiotics is exploring its lead candidate as a monotherapy and in combination with MSD’s checkpoint inhibitor Keytruda (pembrolizumab) following a partnership with the company signed in 2020. A Phase I/II trial with the treatment paradigm is ongoing in advanced melanoma. As per the company, a dose-escalation Phase I trial in 22 patients with a range of solid tumours treated with tigilanol tiglate, demonstrated a complete response of 18%. Stable disease was seen in 64% of the treatment group, and the remaining 22% demonstrated partial response. QBiotics is conducting further clinical trials with tigilanol tiglate as a monotherapy in squamous cell carcinoma, head and neck tumours (NCT05608876), and advanced melanoma (NCT05234437). A model by GlobalData estimates that there were just over 125,000 new cases of soft tissue sarcoma globally in 2023, with incidence growing at 0.54% per year. GlobalData is the parent company of Pharmaceutical Technology.

DF1001 is being evaluated in adult patients for the treatment of advanced solid HER-2 positive tumours. Credit: Ground Picture / Shutterstock.com. Dragonfly Therapeutics has entered a clinical collaboration with Gilead Sciences to assess the potential of its investigational drug candidate DF1001 with the latter’s Trodelvy for two cancer indications. The study of the combination regimen will focus on metastatic breast cancer (mBC) and non-small cell lung cancer (NSCLC). DF1001 is designed to act on natural killer (NK) cells and T-cell activation signals, leveraging co-stimulation of NK receptor NKG2D and CD16 for NK cell activation. Trodelvy is a Trophoblast cell-surface antigen 2-directed antibody-drug conjugate. Dragonfly will maintain operational control of the trial, with the first patients receiving the combination treatment in the second quarter of 2024. See Also: Otsuka wins FDA breakthrough therapy designation for IgAN drug QBiotics wins FDA orphan status for rainforest-derived cancer drug Study sites are already operational in the US, Belgium, France, Denmark and the Netherlands. The study is set to expand, with additional sites in Europe, North America and Asia Pacific. Developed using Dragonfly’s TriNKET platform, DF1001 is being evaluated in adult patients for the treatment of advanced solid human epidermal growth factor receptor 2-positive tumours. It has shown potential to induce anti-tumour immunity in people who are ineligible for or not adequately responding to existing therapies. DF1001 represents the most advanced candidate in the TriKNETs pipeline that Dragonfly is developing to meet unmet patient needs across several disease areas. Dragonfly research and development president Joseph Eid stated: “DF1001 is the first of eight Dragonfly-developed drugs in the clinic and has shown clinical benefit in mBC, NSCLC and colorectal cancer in a heterogeneous Phase I population with 22% RECIST [a standard method to show how a cancer patient responds to a therapy] responders and 39% clinical benefit in mBC patients at active dose levels, with no dose-limiting toxicities even in a heavily-pre-treated population. “It has been demonstrated to be well-tolerated across all dose levels in the Phase I  study as a monotherapy and pharmacodynamic activity was demonstrated in 28 out of 42 (67%) paired biopsies from 0.5mg/kg-15mg/kg, where an increase in CD8 and NK cell infiltration was observed consistent with preclinical models and supporting the TriNKET immune modulating MoA [effect in the body].”

Aurlumyn injection is a new treatment option for adults with severe frostbite. Credit: movchanzemtsova / Shutterstock.com. The US Food and Drug Administration (FDA) has approved Eicos Sciences’s Aurlumyn (iloprost) injection, a new treatment for adults with severe frostbite to lower the risk of amputation of fingers or toes. Aurlumyn contains iloprost, a vasodilator that opens blood vessels and stops blood clotting, as its active ingredient. The efficacy of iloprost in treating severe frostbite was established through an open-label controlled trial in 47 adult subjects. Participants were randomised into three groups, with group one receiving iloprost intravenously for six hours daily for up to eight days along with aspirin and standard of care. Groups two and three were administered other medications, with and without iloprost, which are not approved for frostbite treatment. See Also: Otsuka wins FDA breakthrough therapy designation for IgAN drug QBiotics wins FDA orphan status for rainforest-derived cancer drug The trial’s primary efficacy measure was bone scans on day seven following the frostbite incident to predict the need for amputation. The scans showed that none of the subjects who received iloprost alone (group one) required amputation compared to 19% in group two and 60% in group three. This indicated a significant reduction in the presence of bone scan abnormalities in groups receiving iloprost. Most subjects had follow-up data available to confirm whether they underwent amputation. This aligned with the bone scan findings. Headache, flushing, heart palpitations, nausea and hypotension were common side effects linked to Aurlumyn treatment. The treatment carries a warning for potential symptomatic hypotension. Aurlumyn was granted priority review and orphan drug designations for this indication, highlighting its significance in addressing severe frostbite, a condition that can escalate from mild to severe, potentially leading to permanent tissue damage and the cessation of blood flow. Iloprost originally received approval in 2004 for pulmonary arterial hypertension. FDA Center for Drug Evaluation and Research Division of Cardiology and Nephrology director Norman Stockbridge stated: “This approval provides patients with the first-ever treatment option for severe frostbite. “Having this new option provides physicians with a tool that will help prevent the life-changing amputation of one’s frostbitten fingers or toes.”