INmune Bio to Submit BLA for CORDStrom to Treat Pediatric RDEB

INmune Bio, Inc, a clinical-stage inflammation and immunology company focused on developing treatments that harness the patient’s innate immune system to fight disease, has announced the following a type C meeting with the US FDA, it intents to submit a BLA in the US and marketing authorization application (MAA) in the UK and EU supported by data from the MissionEB clinical trial investigating CORDStrom as a disease-modifying therapy for treating recessive dystrophic epidermolysis bullosa (RDEB) in paediatric patients.

Recessive dystrophic epidermolysis bullosa is a rare, severely debilitating genetic disease, which has its onset in early childhood. Patients’ skin is extremely fragile and is easily damaged, resulting in painful and itchy blistering wounds and scarring that can lead to aggressive and life-threatening skin cancer in adulthood. Long-term morbidity is driven by a debilitating itch and pain that significantly exacerbates wounds and deeply affects quality of life. The currently available treatments target active lesions via topical administration and have a limited benefit.

The company estimates roughly 4,500 children with intermediate or severe RDEB in the US, UK and EU may benefit from systemic CORDStrom therapy (all RDEB incidence: 95 per million live births, at least ~37% of all RDEB are RDEB intermediate or severe), which represents a large unmet opportunity to potentially provide routine clinical care to these children via systemic treatment.

The MissionEB study, led by Dr. Anna Martinez and team at the Great Ormond Street Hospital (GOSH) in collaboration with clinicians from Birmingham’s Children’s Hospital, was a double blind, placebo-controlled, cross-over study evaluating the safety and efficacy of CORDStrom in 30 paediatric patients (age <16 years) in the UK with intermediate or severe RDEB. Subjects were randomized to CORDStrom or placebo and received two intravenous infusions two weeks apart. Half of the patients were treated with CORDStrom and then crossed over to Placebo following a washout period and the other half were treated with Placebo and then crossed over to CORDStrom. Efficacy was assessed at 3- and 6-month from the first infusion per study arm. Thus, all patients are included in the 3- and 6-month efficacy assessment of both placebo and CORDStrom.

CORDStrom was extremely well tolerated, with no serious adverse events related to CORDStrom reported at either 3-month or 6-month post-treatment across all age and RDEB-severity patient sub-types. In children with severe disease, CORDStrom reduced itch at 3-month and led to a sustained reduction of over 27% at 6-month. These results demonstrate that a clinically meaningful reduction in itch severity is sustained over time. In children with intermediate disease severity, CORDStrom provided a broader range of improvements, including reduced skin involvement and less pain, as well as a large reduction in itch. In younger children with RDEB (age <10yrs), CORDStrom provided improvements in skin scores, indicating better skin integrity and reduced disease activity. Interviews with subjects and caregivers strongly support the clinical benefits of CORDStrom; as both caregivers and patients were able to correctly identify which treatment had been CORDStrom and which had been placebo in this cross-over study. With great interest from the patients to continue therapy, the company intends to support a 12-month open label study at GOSH, including all patients enrolled in the MissionEB study, where patients will receive 3 cycles of CORDStrom therapy at time 0, 4 and 8 months.

The US FDA granted CORDStrom an orphan drug designation on January 6, 2025. Benefits of an ODD include certain tax credits and eligibility for select grants, waiver of US FDA user fees, including the BLA application fees, access to frequent meetings with the US FDA for efficient drug development, and eligibility for seven years of market exclusivity post approval.

The company intends to prepare for a pre-BLA meeting to discuss particulars of its planned BLA submission, with intent to submit a BLA in 2025 seeking approval of CORDStrom for the treatment of RDEB in paediatric patients. Concurrently, the company will also prepare to submit MAAs to the EU and UK in 2026.

“CORDStrom represents the culmination of years of dedication by members of our cell medicines R&D team to overcoming the challenges of creating a reproducible, cGMP grade MSC drug product at reasonable costs and scale to treat rare diseases like RDEB,” said Dr. Mark Lowdell, CSO of INmune Bio and inventor of CORDStrom. “The encouraging results from the blinded randomized trial in patients with intermediate and severe RDEB, combined with regulatory support and the NIHR grant, validate our approach and strengthen our resolve to deliver life-changing therapies for patients who need them most. We are heart warmed by the feedback from patients in the MissionEB study, the dedication of Dr. Anna Martinez and her group of investigators in completing this trial, all of which strengthens our resolve to seek approval for CORDStrom in paediatric RDEB and expand the indications for CORDStrom as a drug platform in the future.

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