Orbus Therapeutics is developing an oral/IV formulation of eflornithine, a repurposed ornithine decarboxylase inhibitor, as a targeted chemotherapy for recurrent or progressive grade 3 astrocytoma. The therapy has completed a Phase II trial showing safety and preliminary efficacy, and is paired with a biomarker assay to select patients with elevated enzyme activity. Orbus holds orphan‑drug designations in the US and EU and is preparing for commercial launch in North America and Europe.
Funding
Funding not disclosed
Founders
Product
Problem
Patients with rare, aggressive brain tumors such as grade 3 astrocytoma have limited therapeutic options beyond surgery, radiation, and temozolomide, which provide modest benefit and carry significant toxicity. The lack of approved, disease‑specific chemotherapies leaves a substantial unmet medical need for this underserved patient population.
Solution
Orbus Therapeutics is advancing eflornithine, a repurposed small‑molecule inhibitor of ornithine decarboxylase, as a targeted chemotherapy for recurrent or progressive grade 3 astrocytoma. The company has completed the STELLAR Phase II trial, demonstrating safety and preliminary efficacy in this indication. Leveraging orphan‑drug designations and a streamlined regulatory pathway, Orbus is preparing for commercial launch in North America and Europe. The product is formulated for systemic administration with pharmacokinetic properties that enable adequate central‑nervous‑system exposure. Orbus also offers a companion diagnostic strategy to identify patients most likely to respond based on tumor biomarker profiling.
Target Audience
The primary customers are neuro‑oncology specialists and academic cancer centers treating patients with recurrent or progressive grade 3 astrocytoma, as well as healthcare systems seeking approved therapies for rare central‑nervous‑system malignancies.
Features
- Oral/IV formulation of eflornithine optimized for blood‑brain‑barrier penetration and sustained target inhibition
- Completed STELLAR Phase II clinical trial (NCT02796261) with published efficacy and safety data for grade 3 astrocytoma
- Orphan‑drug designation in the United States and European Union, supporting accelerated review and market exclusivity
- Integrated biomarker assay to select patients with elevated ornithine decarboxylase activity, enhancing therapeutic precision
- Scalable GMP manufacturing process leveraging existing eflornithine production infrastructure, reducing time‑to‑market
- Regulatory submission package aligned with FDA’s Rare Pediatric Disease Designation and EMA’s PRIME scheme for accelerated access
- Post‑approval pharmacovigilance and real‑world evidence program to monitor long‑term outcomes in neuro‑oncology settings