INmune Bio (NASDAQ:INMB) used a company webcast to highlight updates from its Mission EB phase III clinical trial evaluating CORDStrom, an intravenous mesenchymal stromal cell (MSC) therapy, in children with recessive dystrophic epidermolysis bullosa (RDEB). CEO David Moss said the presentation would focus on “pivotal updates” from Mission EB and the potential of CORDStrom to address the systemic inflammation that drives RDEB complications.
RDEB described as progressive, multisystem disease with an early “window of opportunity”
Chief Investigator Dr. Anna Martinez of Great Ormond Street Hospital (GOSH) described RDEB as a multisystemic disorder that worsens over time, moving beyond skin fragility into internal complications and escalating inflammation. She outlined a disease course in which infants can initially stabilize, but from around 18 months onward patients often show increasing inflammatory burden, weight decline, anemia, and esophageal strictures. From ages 10 to 20, she said wounds increasingly become chronic and healing capacity drops, while inflammation contributes to pubertal delay, growth failure, osteoporosis, fractures, and worsening anemia. After age 20, she described an “exponential increase in inflammation” and a high risk of squamous cell carcinoma, with mean survival of 2.4 years after tumor development.
Current treatment landscape and unmet need
Martinez contrasted CORDStrom with available therapies, emphasizing the need for systemic anti-inflammatory treatment. She cited two currently available options:
- Filsuvez, a topical cream approved in the U.K. for children over six months, which she described as expensive and often discontinued by patients because they find it ineffective.
- Vyjuvek, a topical gene therapy gel approved in the U.S. and Europe (but not the U.K.), which she said is limited by the small area that can be treated at once and does not provide a systemic anti-inflammatory effect. She also highlighted the cost, estimating about GBP 25,000 per patient per week and a global lifetime cost estimate of about $15 million per patient for the drug alone.
She said patient priorities from a global survey included improvements in pain, itch, mental health, reduction in inflammation, and relief of esophageal issues—underscoring demand for anti-inflammatory therapies.
Mission EB trial design and reported results
Mission EB was conducted at GOSH and Birmingham Children’s Hospital. Martinez said the study used a double-blind, placebo-controlled crossover design: participants received two infusions (two weeks apart) of either CORDStrom or placebo, followed by a nine-month washout and then the alternate treatment. She said 37 children were recruited, six withdrew, and results were available on 30 patients; 124 infusions were administered. Martinez reported that the trial observed no toxicity events related to the drug and “no serious adverse events.”
In the cohort of 30 patients (16 intermediate and 14 severe disease; 21 under age 10), Martinez reported itch reductions of 21% at three months and 26% at six months after two infusions. She said itch reduction was maintained at six months and was largest in the most severe patients, reaching a 27.5% reduction at six months. She also noted that among intermediate patients, average pain improved by 22% and worst pain by 28%.
Martinez said that in the most severe subgroup, skin improvements were not evident at three months, but by six months the group showed additional itch improvement and “big improvement” in iScore-EB patient-reported skin scores, which she attributed in part to sustained itch relief supporting better wound healing over time.
Patient and caregiver feedback: “they simply felt better”
Martinez also presented qualitative findings from blinded interviews conducted three months after treatment with either CORDStrom or placebo. She said 10 of 13 participants reported they could tell when they received CORDStrom rather than placebo because they felt better. She shared examples of reported improvements, including more energy, appetite, weight gain, less pain with itching, and reduced pain during bathing.
Both Martinez and Dr. Mark Lowdell, inventor of CORDStrom, said the ability of families to identify treatment order despite blinding was notable in a disease area where double-blind studies are uncommon.
Mechanism, manufacturing, and regulatory timeline
Lowdell described CORDStrom as a pooled MSC product derived from four donor umbilical cords, designed to reduce donor-to-donor variability and enable potency-based product definition. He outlined a proposed mechanism in which secreted factors (including PGE2 and IDO) could shift pro-inflammatory M1 macrophages toward anti-inflammatory M2 macrophages and increase wound-healing cytokines. He also cited serum findings shown during the presentation, including reported increases in IL-13 and IL-10 after CORDStrom and reductions in TNF-alpha, alongside increases in wound-healing signals such as TGF-beta and VEGF.
On manufacturing, Lowdell said the company has established scaled production using bioreactors, estimating that a single run could produce about 200 doses, which he said would be enough to treat about 30 patients for an annual run.
The speakers also discussed next steps, including an open-label follow-on study called Mission EB Deliver, planned to treat prior trial participants and additional children with RDEB over 12 months, using two infusions every four months. Lowdell outlined regulatory milestones discussed on the webcast, including MHRA interactions (with a Pediatric Investigation Plan response expected March 9 and a pre-MAA meeting scheduled for the second week of May), a planned U.K. marketing authorization submission around July, and a BLA submission to the FDA targeted by year-end after additional CMC steps requested by the agency.
In closing remarks, Moss said the company aims to advance CORDStrom toward becoming what it described as a systemic, disease-modifying treatment for RDEB, with a focus on intervening early in childhood before severe complications develop.
About INmune Bio (NASDAQ:INMB)
INmune Bio, Inc is a clinical-stage immunology company based in San Diego, California, focused on harnessing the innate immune system to combat a range of serious diseases. The company’s research and development efforts concentrate on modulating cellular pathways to restore and enhance the body’s natural defenses, particularly in oncology and neurodegenerative conditions.
The company’s lead therapeutic candidate, XPro1595 (also referred to as INB03), is a proprietary selective inhibitor of soluble tumor necrosis factor (TNF) designed to reduce chronic inflammation without impairing membrane-bound TNF functions.
