EXP01: Nonclinical-Stage Disease-Modifying MSC-EV Asset for Lung Injury (IPF & ARDS) | FDA Pre-IND Cleared

EXORPHIA is pioneering next-generation extracellular vesicle (EV) therapeutics to overcome the efficacy and safety limitations of existing treatments. Our lead pulmonary asset, EXP01, is a nonclinical-stage, disease-modifying therapeutic based on pre-conditioned mesenchymal stem cell-derived EVs (Prime-EV™), offering a novel multi-target approach with dual anti-inflammatory and anti-fibrotic actions for Idiopathic Pulmonary Fibrosis (IPF) and Acute Respiratory Distress Syndrome (ARDS).

Key Highlights & In Vivo PoC:

• Superior Anti-Fibrotic Efficacy in IPF: In therapeutic bleomycin-induced IPF models, EXP01 significantly reduced fibrosis (Ashcroft score), demonstrating efficacy superior to Nintedanib tested at 20× its clinical equivalent dose.
• Rapid Anti-Inflammatory Action in ARDS: In LPS-induced ARDS models, a single dose of EXP01 reduced BALF leukocytes by ~50%, matching the efficacy of high-dose Dexamethasone (30× clinical dose).
• Favorable Safety & Tolerability: Repeat-dose toxicological evaluations of EXP01 showed a favorable safety profile with no adverse signs.

Proprietary Technology & IP:
• Mechanism of Action (MoA): Driven by a published, miR-4516-mediated anti-fibrotic pathway.
• Proprietary Manufacturing & QC: Built on our Prime-EV™ cell pre-conditioning technology that enriches active biological cargo, with miR-4516 established as a functional QC biomarker for lot release.
• Strong IP Protection: Composition of matter patent pending (WO/2024/242132).

Regulatory Status & Asset Readiness:
• FDA Pre-IND Consultation Completed: FDA supported our proposed nonclinical, CMC, and preclinical validation plans for ARDS.
• Ready for Out-Licensing: The nonclinical PoC and CMC package are established, making EXP01 an attractive asset for a pharma partner to lead IND-enabling studies and clinical development.

Licensing Opportunity:
EXORPHIA is actively seeking global or regional out-licensing partners to acquire and advance EXP01.