EXORPHIA, INC. (the Company) is pleased to announce that on February 16, 2024 (U.S. time), the Company received a positive response from the U.S. Food and Drug Administration (FDA) regarding the Pre-IND application for a planned Phase 1/2 clinical trial of EXP01 in ARDS patients. Based on the favorable results from pilot manufacturing by the CDMO (contract manufacturing organization) and preclinical studies, the Company submitted a request for a pre-IND meeting to the FDA in December 2023 to advance development toward clinical trials further.
In the pre-IND, the Company inquired through a document in a question-and-answer format to confirm the appropriateness of EXP01’s manufacturing and quality safety, preclinical studies, and trial design. After reviewing the FDA’s response document, the Company confirmed that the remaining development can proceed toward the IND application without making significant changes to the Company’s development plan. Following this positive feedback, the Company plans to conduct a Phase 1/2 clinical trial of EXP01 in ARDS patients in 2025. This clinical trial will provide essential insights into the safety and therapeutic efficacy of the Company’s EV formulation.
■About EXP01:
EXP01 is an EV formulation derived from bone marrow mesenchymal stem cells (MSCs). Utilizing the Company’s proprietary cell activation technology, it is characterized by its excellent anti-inflammatory and anti-fibrotic effects while having a low risk of side effects. While lung disorders are the initial target indications, the Company also evaluates the potential for expanding the application to other organs. It is expected to apply to various diseases, focusing on inflammatory diseases and fibrosis. The Company is conducting research and development of EXP01 as a therapeutic agent for lung disorders (idiopathic pulmonary fibrosis (IPF) and acute respiratory distress syndrome (ARDS)). Non-clinical pharmacological efficacy studies, pharmacokinetics, and safety studies have been conducted, and preparations are underway for clinical trials in the United States.
■About EXORPHIA, INC.:
EXORPHIA is a biotech company that develops innovative exosome-based therapeutics to address unmet medical needs. With its laboratory and office in central Tokyo, EXORPHIA leverages its proprietary cell activation, EV modification, purification, and analysis technologies to build a robust pipeline targeting various diseases. The company’s mission is to create a healthier society by harnessing the potential of pharmaceutical-grade exosomes.
Learn about Pre-IND Meetings: https://toolkit.ncats.nih.gov/module/prepare-for-clinical-trials/participating-in-initial-meetings-with-regulators/learn-about-pre-ind-meetings/#:~:text=A%20pre%2DIND%20meeting%20is,facilitate%20faster%20therapy%20approval%20processes
Contact for inquiries regarding this matter:
EXORPHIA, INC. Public Relations
Email: pr@exorphia.com
