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InnoPharmax Inc. (TWSE: 4172) today announced that its orally administered orphan drug for biliary tract cancer, D07001-Softgel, has officially advanced into a global pivotal Phase III clinical trial. The Company has partnered with a leading U.S.-based contract research organization (CRO) with over 30 years of experience in rare diseases and oncology. The CRO has a strong track record of successfully supporting multiple drug approvals by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), and is recognized for its excellence in execution efficiency, quality, and regulatory compliance.
Under this collaboration, the international CRO will conduct the global clinical trial to support a U.S. FDA New Drug Application (NDA). The study is expected to enroll approximately 240 subjects. The Company aims to initiate patient enrollment in the U.S. by the end of 2025 and complete enrollment required for interim analysis by the end of 2026.
According to global market data, biliary tract cancer represents a disease with significant unmet medical need. Coupled with orphan drug regulatory pathways, D07001-Softgel has the potential to achieve accelerated approval and secure a niche market position if the Phase III trial is successful.
In December 2024, the FDA approved the Phase III clinical trial protocol for D07001-Softgel. This international study targets patients with advanced biliary tract cancer who are in third-line treatment or are unwilling to receive second-line injectable chemotherapy. The trial is designed as a multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy of D07001-Softgel in combination with capecitabine versus placebo in combination with capecitabine, with overall survival (OS) as the primary endpoint along with multiple secondary endpoints.
In the Phase I/Ib clinical trial, D07001-Softgel demonstrated excellent safety and favorable pharmacokinetic (PK) profiles. This was followed by a successful safety review by the Safety Review Committee (SRC) in the Phase IIa study. The results were published in April 2025 in the internationally recognized journal The Oncologist (Vol. 30, No. 4; Title: “Phase I study of oral metronomic gemcitabine (D07001)”):
https://academic.oup.com/oncolo/article/30/4/oyaf051/8117452
These findings further validate InnoPharmax’s leadership in oral microemulsion drug delivery platform technology.
In parallel, the Company is advancing a Phase II basket trial targeting two high-potential indications—pancreatic cancer (PAC) and non-small cell lung cancer (NSCLC)—to rapidly identify efficacy signals. Through a multi-regional development strategy and a “one drug, multiple indications” approach, InnoPharmax aims to expand the clinical value of D07001 and enhance overall investment returns.
