8-K: INmune Bio to Seek FDA Approval for CORDStrom in Rare Skin Disorder

INmune Bio plans to submit a Biologics License Application (BLA) to the FDA for CORDStrom, a treatment for recessive dystrophic epidermolysis bullosa (RDEB) in pediatric patients, following positive clinical trial results.

Summary

• INmune Bio is moving forward with plans to seek regulatory approval for CORDStrom, a treatment for recessive dystrophic epidermolysis bullosa (RDEB) in pediatric patients.
• The decision follows a Type C meeting with the FDA and is supported by data from the MissionEB clinical trial.
• The company intends to submit a Biologics License Application (BLA) to the FDA in the US and Marketing Authorization Applications (MAA) in the UK and EU.
• The MissionEB study, a double-blind, placebo-controlled, cross-over study, evaluated the safety and efficacy of CORDStrom in 30 pediatric patients with intermediate or severe RDEB.
• CORDStrom was well-tolerated, with no serious adverse events related to the treatment reported.
• The company estimates that approximately 4,500 children in the US, UK, and EU could benefit from CORDStrom therapy.
• INmune Bio has entered into an exclusive commercial license with Great Ormond Street Hospital (GOSH) for the MissionEB clinical data.
• The company will pay GOSH an initial fee of $0.3 million and a milestone payment of $7.5 million upon receipt of the first marketing authorization from the FDA, EMA, or MHRA.
• The FDA has granted CORDStrom a Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD).
• The company plans to submit a BLA in 2025 and MAAs to the EU and UK in 2026.

Sentiment

Score: 8

Explanation: The document presents a positive outlook for INmune Bio, driven by promising clinical trial results, regulatory support, and a strategic partnership. The company is moving forward with plans to seek regulatory approval for CORDStrom, which could address a significant unmet need in the treatment of RDEB.

Positives

• CORDStrom was well-tolerated in the MissionEB study, with no serious adverse events related to the treatment.
• The MissionEB study showed positive results, including a reduction in itch, skin involvement, and pain in children with RDEB.
• The FDA granted CORDStrom a Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD), providing potential benefits such as tax credits, user fee waivers, and market exclusivity.
• The company has secured an exclusive commercial license with Great Ormond Street Hospital (GOSH) for the MissionEB clinical data.
• INmune Bio plans 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.

Risks

• Clinical trials are in early stages and there is no assurance that any specific outcome will be achieved.
• There is no assurance that CORDStrom will be approved by the FDA or any regulatory body.
• The company's ability to produce more drug for clinical trials is a risk.
• The availability of substantial additional funding for the Company to continue its operations and to conduct research and development, clinical studies and future product commercialization is a risk.
• The company's business, research, product development, regulatory approval, marketing and distribution plans and strategies are subject to risks and uncertainties.

Future Outlook

INmune Bio plans to submit a BLA to the FDA in 2025 and MAAs to the EU and UK in 2026, seeking approval of CORDStrom for the treatment of RDEB in pediatric patients. The company also plans to report top-line data on cognitive function in its MINDFuL study in June of this year, and further plans to announce additional data in its CaRe PC study as it becomes available throughout 2025.

Management Comments

• 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 pediatric RDEB and expand the indications for CORDStrom as a drug platform in the future.

Industry Context

This announcement is significant in the context of the rare disease treatment landscape, where there is a high unmet need for effective therapies. CORDStrom, if approved, could become a valuable treatment option for RDEB, a severely debilitating genetic disease. The company's focus on systemic treatment represents a potential advancement over existing topical treatments.

Comparison to Industry Standards

• The current standard of care for RDEB primarily involves topical treatments aimed at managing active lesions, which offer limited benefits.
• CORDStrom's potential as a systemic therapy could represent a significant improvement over existing treatments.
• Companies like Abeona Therapeutics and Krystal Biotech are also developing gene therapies for DEB, but CORDStrom's cell-based approach offers a different mechanism of action.
• The success of CORDStrom will depend on its ability to demonstrate superior efficacy and safety compared to existing and emerging therapies.

Stakeholder Impact

• Shareholders: The positive clinical trial results and plans for regulatory submission could increase shareholder value.
• Patients: CORDStrom, if approved, could provide a new treatment option for RDEB, improving their quality of life.
• Employees: The company's progress could create new job opportunities and enhance employee morale.
• Great Ormond Street Hospital (GOSH): The partnership with INmune Bio could provide financial benefits and access to CORDStrom for their patients.

Next Steps

• Prepare for a pre-BLA meeting to discuss particulars of its planned BLA submission.
• Submit a BLA in 2025 seeking approval of CORDStrom for the treatment of RDEB in pediatric patients.
• Prepare to submit MAAs to the EU and UK in 2026.
• 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.
• Report top-line data on cognitive function in its MINDFuL study in June of this year.
• Announce additional data in its CaRe PC study as it becomes available throughout 2025.

Key Dates

Keywords

CORDStrom, RDEB, Epidermolysis Bullosa, INmune Bio, FDA, BLA, Orphan Drug Designation, Rare Pediatric Disease Designation, GOSH, Clinical Trial