INmune Bio to Submit BLA for FDA Approval of CORDStrom for RDEB Treatment

INmune Bio, Inc. has announced promising results from its MissionEB clinical trial, evaluating CORDStrom for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) in children. RDEB is a severe genetic disorder characterized by extremely fragile skin, leading to painful blistering and scarring, often beginning in childhood. Over time, patients may develop life-threatening complications such as aggressive skin cancer.

The MissionEB study was a double-blind, placebo-controlled, cross-over trial conducted in the UK, involving 30 pediatric patients under 16 years of age with either intermediate or severe RDEB. Participants were randomized to receive either CORDStrom or a placebo, with two infusions given two weeks apart. Following a washout period, participants were switched to the opposite treatment. The study assessed efficacy at both three and six months after the initial infusion.

Results revealed that CORDStrom was well-tolerated with no serious adverse events reported. In children with severe RDEB, the treatment significantly reduced itching, showing a 27% reduction at six months. Those with intermediate disease experienced improvement in skin condition and reduction in pain and itchiness. Younger patients under the age of ten also demonstrated better skin integrity and reduced disease activity. Feedback from patients and caregivers supported the perceived benefits of CORDStrom, as they were able to distinguish between the active treatment and placebo, highlighting CORDStrom's effectiveness.

These findings prompted INmune Bio to plan a 12-month open-label extension at Great Ormond Street Hospital (GOSH), involving all participants from the MissionEB study to receive additional CORDStrom treatments. Alongside this, INmune Bio has secured an exclusive commercial license with GOSH NHS Foundation Trust for the MissionEB data, involving an initial fee and a milestone fee upon achieving marketing authorization.

INmune Bio aims to submit a Biologics License Application (BLA) to the FDA and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) and the Medicines and Healthcare products Regulatory Agency (MHRA) using the data from the MissionEB study. The FDA has granted CORDStrom both Rare Pediatric Disease Designation and Orphan Drug Designation, which offer benefits such as tax credits, fee waivers, and market exclusivity for seven years post-approval.

CORDStrom, developed by INmune Bio, is a patent-pending mesenchymal stromal cell platform derived from human umbilical cords. It is designed to treat complex inflammatory diseases like RDEB. The platform is scalable, cost-effective, and manufactured to consistent standards, making it a promising candidate for systemic therapy in RDEB and potentially other disorders.

Dr. Mark Lowdell, Chief Scientific Officer of INmune Bio, expressed optimism about the trial results, emphasizing the commitment to developing therapies for rare diseases. The company is also preparing to report data from its other clinical programs, including trials for Alzheimer's disease and prostate cancer.

With the encouraging outcomes of the MissionEB trial combined with regulatory support, INmune Bio is moving forward with plans to seek approval for CORDStrom, aiming to provide a new treatment option for children suffering from RDEB.