BLA Submission Planned for CORDStrom in RDEB

An announcement by INmune Bio, Inc. points to a plan by the company to submit a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for approval of CORDStrom treatment of recessive dystrophic epidermolysis bullosa (RDEB).1

The announcement to submit a BLA for CORDStrom follows a Type C meeting with FDA officials and was supported by findings from the MissionEB clinical trial. This study had assessed CORDStrom as a disease-modifying therapy for pediatric patients with RDEB.

“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,” Mark Lowdell, CSO of INmune Bio and inventor of CORDStrom, said in a statement.1

RDEB itself is a rare, severely debilitating genetic disease with an onset taking place in early childhood. RDEB is characterized by extremely fragile and easily damaged skin, with painful and itchy blistering wounds and scarring resulting from this damaged skin. RDEB can also result in aggressive and life-threatening skin cancer among adults.

RDEB is a subtype of epidermolysis bullosa that’s long-term morbidity is driven by a debilitating pruritus and pain, deeply impacting patients’ quality of life. Currently accessible treatments target active lesions through the use of topical administration and may have limited benefits.

In INmune Bio’s announcement, they highlight estimates that approximately 4500 children who are known to have intermediate or severe RDEB within the US, the EU, and the UK may see benefits from systemic CORDStrom treatment. The medication itself was evaluated during the MissionEB study, by a team of investigators at both the Great Ormond Street Hospital (GOSH) and Birmingham’s Children’s Hospital.

The double blind, placebo-controlled, crossover clinical study was designed by investigators to assess CORDstrom’s safety and efficacy. They looked at 30 pediatric patients who were aged <16 years and had intermediate or severe RDEB. Subjects in MissionEB were randomized to either the CORDStrom or placebo arm and were given a pair of intravenous infusions that were spaced 2 weeks apart.1

The investigators required half of the patients treated with CORDStrom to cross over to the placebo arm after a washout period and the other half were treated with placebo and then switched to CORDStrom. The MissionEB team evaluated the treatment’s efficacy at the 3- and 6-month marks following the first infusion per study arm.

Overall, CORDStrom was shown by the investigators to be significantly well-tolerated among trial participants. The team further concluded that there had not been reports of serious adverse events associated with CORDStrom that subjects reported at either 3 months or 6 months in the post-treatment period across all of the age and RDEB-severity subtypes.1

CORDStrom was noted by the investigative team as having reduced itch at the 3-month mark among children with severe disease, resulting in a sustained reduction of over 27% at the 6-month mark and suggesting clinically meaningful pruritus reductions sustained over time.1 Individuals reporting intermediate disease severity were shown to have a broader range of improvements when treated with the medication, including diminished pain and skin involvement, as well as a major reduction in pruritus.

CORDStrom was also noted to have provided skin score improvements In younger children with RDEB who were also < 10 years in age.1 During interviews with both trial participants and their caregivers, the company found that treatment with CORDStrom and treatment with a placebo had both been correctly identified by those surveyed.

INmune Bio is slated to support a 12-month open label study being conducted at GOSH that would involve all of the participants enrolled in the MissionEB trial. In this upcoming analysis, subjects would be given 3 cycles of CORDStrom treatment at the 0, 4 and 8-month marks. The company also intends to submit a BLA to the FDA in 2025 with the aim of receiving approval of CORDStrom for RDEB in the pediatric patient population.

“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 pediatric RDEB and expand the indications for CORDStrom as a drug platform in the future," Lowdell said in a statement.1

References

1. INmune Bio Announces Plan to Submit FDA Biologics License Application (BLA) Seeking Approval of CORDStrom for Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB). INmune Bio, Inc. February 10, 2025. https://www.globenewswire.com/news-release/2025/02/10/3023306/0/en/INmune-Bio-Announces-Plan-to-Submit-FDA-Biologics-License-Application-BLA-Seeking-Approval-of-CORDStrom-for-Treatment-of-Recessive-Dystrophic-Epidermolysis-Bullosa-RDEB.html.