Sapu Nano has received regulatory approval from Australia's Human Research Ethics Committee (HREC) to initiate Phase 1 clinical trials of Sapu003, an injectable formulation of everolimus designed to address bioavailability limitations in breast cancer treatment. The company announced the milestone on September 24, 2025, marking a significant advancement for its proprietary Deciparticle™ drug delivery technology.
Addressing Critical Bioavailability Limitations
Everolimus, currently marketed as Afinitor® and FDA-approved for various cancers including advanced breast cancer, kidney cancer, and certain rare tumors, faces substantial absorption challenges in its oral formulation. According to the company, only approximately 10% of the oral drug is absorbed by the body, significantly limiting therapeutic effectiveness.
Sapu003 leverages Sapu Nano's proprietary Deciparticle™ technology to deliver everolimus intravenously, enabling 100% bioavailability. Preclinical studies suggest this approach could demonstrate superior efficacy compared to the current oral version.
"We are extremely pleased to receive approval from the HREC to proceed with human clinical trials," said Dr. Vuong Trieu, Chief Executive Officer of Sapu Nano. "Despite advances in treatment, there remains a critical unmet need for next generation mTOR inhibitors. Current therapies often extend progression-free survival for less than one year and rarely deliver long-term disease control."
Clinical Development Strategy
The Phase 1 trial represents the initial step in determining optimal dosing parameters for future clinical development, including a planned Phase 3 trial. Dr. Trieu emphasized that this dose-finding study will establish the foundation for subsequent efficacy evaluations.
Dr. Sud Agarwal, Chief Executive Officer of Ingenu, highlighted the potential clinical impact: "The approval of Sapu003 to enter human trials is a landmark moment. By enabling full drug absorption through intravenous delivery, this program has the potential to achieve meaningful tumor shrinkage where oral formulations have been limited."
Corporate Structure and Development Pipeline
Sapu Nano operates as part of the Sapu family of companies, established through GMP Biotechnology Limited, a joint venture between Oncotelic Therapeutics, Inc. (OTCQB: OTLC) and Dragon Overseas Capital Limited. Oncotelic maintains a 45% stake in GMP Bio, which advances complementary drug candidates under Dr. Trieu's leadership.
The parent company, Oncotelic Therapeutics, focuses on clinical-stage biopharmaceutical development in oncology and immunotherapy, targeting high-unmet-need cancers and rare pediatric indications. Dr. Trieu's extensive patent portfolio includes over 500 patent applications and 75 issued patents, supporting the company's intellectual property position.
Therapeutic Implications
The development addresses a fundamental challenge in mTOR inhibitor therapy, where current treatments provide limited duration of disease control. The enhanced bioavailability achieved through intravenous delivery could potentially translate to improved tumor response rates and extended progression-free survival compared to existing oral formulations.
The regulatory approval enables patient enrollment in the Australian Phase 1 study, representing a critical milestone in advancing this reformulated approach to everolimus delivery for breast cancer patients who have limited long-term treatment options with current mTOR inhibitor therapies.
