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

2023-04-26

临床3期临床2期临床申请

ExoFlo is the first extracellular vesicle therapeutic candidate to be evaluated in this indication AUSTIN, Texas--(BUSINESS WIRE)-- Direct Biologics LLC, 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.” About Perianal Fistulizing Crohn’s disease The prevalence of Crohn’s disease in the United States is approximately 200 per 100,000 adults and 46 per 100,000 children aged 2-17 years. One of the most severe manifestations of CD is fistulizing disease, developing in up to 50% of patients, particularly those with colonic and rectal involvement. These patients experience significant morbidity due to pain, persistent drainage, recurrent perianal sepsis and ongoing need to access medical care resulting in increased costs and impaired quality of life. Perianal fistulizing CD is notoriously difficult to cure with 37% of patients experiencing refractory disease and >90% undergoing multiple surgical interventions, putting them at risk of incontinence. The limited efficacy with available medical and surgical interventions is underscored by 20% of patients still undergoing a proctectomy with a permanent stoma. These results have spurred significant interest in identifying improved treatment options. About Direct Biologics Direct Biologics is a late-stage biotechnology company leveraging a regenerative medicine platform which uses extracellular vesicles (EVs) secreted from bone marrow-derived mesenchymal stem cells to address multiple disease indications. Direct Biologics’ mission is to be the global leader in regenerative medicine through discovery, innovation, advancement of science, and treatment of patients in a safe and effective manner. Our therapeutic product candidate, ExoFlo, is designed to be a scalable, reproducible, and effective next-generation biologic that leverages our proprietary EV platform technology designed to reduce inflammation, modulate the immune system, and restore tissue through cellular regeneration. Direct Biologics is currently conducting the global Phase 3 EXTINGUISH ARDS clinical trial of ExoFlo for the treatment of hospitalized adults with moderate-to-severe acute respiratory distress syndrome (ARDS). In addition, the Company has initiated Phase 1 clinical trials with ExoFlo for the treatment of ulcerative colitis and Crohn’s disease, and expanded access trials in solid abdominal organ transplantation and severe ARDS patients. Direct Biologics intends to pursue additional clinical applications including perianal fistulizing Crohn’s disease and necrotizing enterocolitis. Headquartered in Austin, Texas, Direct Biologics also has an R&D facility at the Center for Novel Therapeutics on the campus of University of California at San Diego, and operations and order-fulfillment center in San Antonio, Texas. For more information, please visit and follow us on Twitter @directbiologics.