Direct Biologics Receives FDA Clearance to Initiate Phase 1b/2a Clinical Trial of ExoFlo™ in Patients with Medically Refractory Perianal Fistulizing Crohn’s Disease

ExoFlo is the first extracellular vesicle therapeutic candidate to be evaluated in this indication

Direct Biologics, a late-stage biotechnology company leveraging its regenerative medicine platform using extracellular vesicles (EVs) secreted from bone marrow-derived mesenchymal stem cells (MSCs) to address multiple disease indications, announces U.S. Food and Drug Administration (FDA) clearance of the Company’s investigational new drug (IND) application for a Phase 1b/2a clinical trial to evaluate the safety and efficacy of ExoFlo™ for the treatment of medically refractory perianal fistulizing Crohn’s disease (CD).

“The expansion of our inflammatory bowel disease clinical program into perianal fistulizing Crohn’s disease underscores our commitment to providing treatment options to a patient population that suffers from chronic inflammatory disease,” said Mark Adams, Chief Executive Officer of Direct Biologics. “We look forward to the results of our Phase 1b/2a study given that ExoFlo is the first EV product candidate to be evaluated in this patient population.”

The multicenter, single-blind, randomized, placebo-controlled trial is expected to enroll 36 patients with medically refractory perianal fistulizing CD. The trial will have three treatment arms with cohorts receiving escalating doses of ExoFlo. Patients will be monitored for safety and evaluated for efficacy using a combination of clinical examination and radiographic evaluation with pelvic MRI.

The Phase 1b/2a clinical trial is designed to be a safety study with exploratory assessments of efficacy. The primary endpoint of the study is to evaluate the number of adverse events and serious adverse events of one 15 mL or 30 mL treatment or two treatments of 30 mL ExoFlo. The secondary endpoints include clinical and radiographic improvement, and the tertiary/exploratory endpoints are patient reported disease outcomes. The secondary endpoint of combined clinical and radiographic complete healing was selected based on previous clinical trials of MSCs in this phenotype of CD. The trial will also evaluate the efficacy of ExoFlo in fistulizing subtypes, including patients with proctitis and numerous fistula tracts.

“FDA clearance to initiate this randomized trial of ExoFlo in perianal fistulizing Crohn’s disease marks a defining moment for patients suffering from this devastating phenotype that greatly diminishes quality of life. We believe ExoFlo to be a promising therapeutic candidate for fistulizing Crohn’s disease since it is designed as an off-the-shelf, cell-derived product with anti-inflammatory, immunomodulatory and tissue restoring properties,” said Amy Lightner, M.D., Chief Medical Officer of Direct Biologics. “We look forward to the prospect of providing a regenerative product used in everyday clinical practice for this critical unmet need.”


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