Pleco Therapeutics has announced a positive outcome from its Pre-Investigational New Drug (Pre-IND) meeting with the U.S. Food and Drug Administration (FDA) regarding PTX-252, an intravenous formulation being developed for patients with Acute Myeloid Leukemia (AML).
The Netherlands-based clinical-stage biopharmaceutical company received constructive feedback and support from the FDA's Division of Hematologic Malignancies for its proposed development strategy. This guidance provides Pleco with a clear pathway to advance PTX-252 into clinical trials.
Novel Mechanism of Action Targets Tumor Microenvironment
PTX-252 represents an innovative approach to cancer treatment. The small molecule is designed to remove toxic heavy metals from the tumor microenvironment, which subsequently improves the functioning of the tumor suppressor protein p53. This mechanism enhances the sensitivity of cancer cells to chemotherapy, potentially improving treatment outcomes.
The FDA has already granted PTX-252 Orphan Drug Designation for AML treatment, highlighting the compound's potential significance in addressing unmet needs in this difficult-to-treat hematologic malignancy.
Executive Leadership Emphasizes Regulatory Milestone
Ivo Timmermans, Founder and co-CEO of Pleco Therapeutics, emphasized the importance of this regulatory milestone: "The FDA's constructive feedback supports our mission to bring PTX-252 to cancer patients with limited treatment options. The guidance provides a clear path forward to opening the IND to advance PTX-252 into the clinic as swiftly as possible."
Michael Stalhamer, President of Pleco Therapeutics USA, added: "Collaborating with the US FDA and other Regulatory Bodies early in the development of a medicinal product is a key step. Pleco Therapeutics is grateful for the feedback which helps to define and streamline our clinical development program, while ensuring a safe and effective regulatory pathway to patients."
AML Treatment Landscape and Unmet Needs
AML is an aggressive blood cancer characterized by the rapid growth of abnormal white blood cells that accumulate in the bone marrow and interfere with the production of normal blood cells. Despite advances in treatment options, AML continues to have a poor prognosis for many patients, particularly older adults and those with certain genetic profiles.
Current standard treatments include intensive chemotherapy, targeted therapies, and stem cell transplantation. However, treatment resistance and relapse remain significant challenges, highlighting the need for novel therapeutic approaches like PTX-252.
Collaborative Development Efforts
Pleco is co-developing PTX-252 with Hyloris Pharmaceuticals, a Belgian-based specialty pharmaceutical company. This collaboration combines Pleco's expertise in developing novel cancer therapies with Hyloris' experience in pharmaceutical development and commercialization.
Next Steps in Development
Following this positive Pre-IND meeting, Pleco Therapeutics is expected to proceed with the necessary nonclinical and clinical studies required for a formal IND submission. If approved, the IND would allow the company to initiate clinical trials to evaluate the safety and efficacy of PTX-252 in patients with AML.
The company's focus on improving the tumor microenvironment represents a distinctive approach in oncology drug development, potentially complementing existing treatment modalities and addressing mechanisms of treatment resistance.
