[E-Daily YU JIN-HEE Reporter] Innovative drug developer #PenetriumBio has begun the formal regulatory approval process to advance its core pipeline candidate into a U.S. Phase 2 clinical trial. The company’s strategy is to leverage the extensive human safety data it has already secured in the field of infectious diseases to demonstrate the drug’s anti-cancer efficacy at an early stage.

PenetriumBio announced on the 14th that it is formally preparing to submit an Investigational New Drug (IND) application to initiate a U.S. Phase 2 clinical trial for “Penetrium,” a candidate drug for intractable solid tumors. This U.S. clinical trial is designed based on human safety data already validated for Penetrium in the same formulation.

A drug with the same formulation as Penetrium has previously undergone global clinical trials as a treatment for COVID-19. The company has reliably secured real-world human administration data from approximately 300 patients in South Korea. Currently, a Phase 2/3 clinical trial for dengue fever treatment is proceeding smoothly in Vietnam. This means that key indicators related to human safety and pharmacokinetics (PK) have been extensively accumulated.

The U.S. clinical trial used this accumulated safety data as its core basis. Rather than conducting an initial safety confirmation phase, the clinical protocol was designed to proceed directly to Phase 2, which aims to verify actual therapeutic efficacy in cancer patients.

However, safety measures are also being implemented in parallel to gradually verify the safety of combination therapy—including immunotherapy—in an initial patient cohort. The final optimal dose for the cancer indication will be determined through the Phase 1 clinical trial currently underway in South Korea. The U.S. and South Korean clinical trials follow a parallel development structure tailored to each country’s regulatory environment and clinical objectives.

The U.S. Food and Drug Administration (FDA)’s clinical trial application process does not uniformly restrict the initial phase of a trial. If the applicant company scientifically substantiates its submission with highly reliable preclinical data, human dosing experience, toxicity and safety data, and manufacturing and quality control (CMC) data, the FDA may approve immediate entry into Phase 2 following review. The company plans to submit a clinical trial plan, including the Phase 2 protocol, based on the data it has secured. The decision to initiate the clinical trial will be finalized following the FDA’s final review.

This clinical trial design underwent thorough preliminary review by a local contract research organization (CRO) specializing in FDA regulatory affairs and a clinical task force (TF) led by Kim Taek-sung, President of U.S. Operations. Professor Sandip Patel of the University of California, San Diego (UCSD), who has extensive experience in solid tumor “basket” clinical trial designs, is participating as the Principal Investigator (PI). Professor Patel is meticulously designing the target cancer types, patient selection criteria, and detailed plans for evaluating safety and efficacy.

Jo Won-dong, Chairman of PenetriumBio, explained, “Proceeding with a U.S. Phase 2 clinical trial is not an irregularity that bypasses essential safety verification procedures or arbitrarily skips clinical stages,” adding, “It is a legitimate development strategy derived through thorough planning and verification procedures by local U.S. experts, based on the large-scale human administration data we have already secured.”

He continued, “The final decision on whether to commence the clinical trial will be determined through official review by regulatory authorities,” and added, “We will transparently disclose key progress updates to the market—such as the submission of the clinical trial protocol and its results—based on verified facts.”