Full Press Release Details
INmune Bio Announces Plan to Submit
FDA Biologics License Application (BLA) Seeking Approval of CORDStrom for Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Boca Raton, Florida, Feb. 10, 2025
(GLOBE NEWSWIRE) -- INmune Bio, Inc. (NASDAQ: INMB) (the "Company"), a clinical-stage inflammation
and immunology company focused on developing treatments that harness the patient's innate immune system to fight disease, announced
today, following a Type C meeting with the U.S. Food and Drug Administration (FDA), its intent 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 RDEB in pediatric patients.
RDEB 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 pediatric 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-months 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-months or 6-months post-treatment across all age and RDEB-severity
patient sub-types. In children with severe disease, CORDStrom reduced itch at 3-months and led to a sustained reduction of over 27% at
6-months. 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 Company and the GOSH NHS Foundation
Trust entered into an exclusive commercial license whereby the Company received exclusive commercial rights to the MissionEB clinical
data in exchange for (i) payment of a small initial fee, (ii) a single development milestone fee that becomes due on receipt of the first
to occur marketing authorization from the FDA, EMA, or MHRA, and (iii) a commitment to supply CORDStrom to MissionEB study patients that
enroll in the open label study, subject to certain limitations.
The Company participated in a Type C
meeting with the FDA, the outcome of which provided information related to CMC and other regulatory topics in anticipation of the Company's
efforts to prepare and submit a BLA.
The FDA granted CORDStrom a rare pediatric
disease designation (RPDD) for treatment of EB on December 13, 2024, ahead of the priority review voucher (PRV) sunset period, and as
such, CORDStrom remains eligible to receive a PRV if approved by the FDA on or prior to September 30, 2026, which date may be extended
by Congress. If granted, a PRV can be redeemed to receive priority review for a different product, or it may be transferred or sold.
Additionally, the FDA granted CORDStrom
an orphan drug designation (ODD) on January 6th, 2025. Benefits of an ODD include certain tax credits and eligibility for
select grants, waiver of FDA user fees, including the BLA application fees, access to frequent meetings with the FDA for efficient drug
development, and eligibility for seven (7) 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 pediatric 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 pediatric RDEB and expand the indications for CORDStrom as a drug platform in the future."
In addition to these developments involving
CORDStrom, the Company reiterates plans to report top-line data on cognitive function in its MINDFuL study, a Phase II trial investigating
XproTM for treatment of Alzheimer's disease with inflammation, in June of this year, and further plans to announce
additional data in its CaRe PC study, an open-label, phase I/IIa dose escalation and expansion study of INKmune in men with metastatic
castration-resistant prostate cancer (mCRPC) as it becomes available throughout 2025.
The company will host a webinar at 8:30
AM ET today to discuss the results of the MissionEB study investigating CORDStrom for treatment of RDEB in pediatric patients.
Date: Monday, February 10, 2025
Time: 8:30 AM Eastern Time
Webcast: Click Here or https://lifescievents.com/event/inmunebio-2/
CORDStrom is a patent-pending cell medicine
comprising aseptic, allogeneic, pooled human umbilical cord -derived mesenchymal stromal cells (hucMSCs) in suspension for injection
or infusion. The CORDStrom platform leverages, among other things, proprietary screening, pooling and expansion techniques to create
off-the-shelf, allogeneic, pooled hucMSCs as medicines to treat complex inflammatory diseases. CORDStrom products are designed to provide
high-quality, off-the-shelf, batch-to-batch consistent, scalable, cGMP manufactured, potent cellular medicines that can be produced at
low cost and with repeatable specification independent of donor characteristics. The CORDStrom product platform shares many similarities,
including reagents, equipment, and procedures, with the Company's INKmune oncology product, enabling the Company to leverage economies
of scale, experienced staff, and other resources to strategically manufacture both products in a rotational campaign with resource and
environmental efficiencies.
Initially developed at the INKmune manufacturing
facilities utilizing UK academic grant funding, CORDStrom is an MSC product platform that shows promise as a first systemic therapy for
potentially treating RDEB and many other debilitating conditions. While the first generation CORDStrom product is agnostic to disease
indication, the platform enables creation of indication-specific products, which can be tuned for optimization of anti-inflammatory,
immunomodulatory, wound healing, and other characteristics.
About RDEB, DDEB, EB
Epidermolysis Bullosa (EB) is
a group of inherited skin disorders characterized by extreme skin fragility and blistering. Among its subtypes, Dystrophic Epidermolysis
Bullosa (DEB) is notable for its division into Dominant Dystrophic EB (DDEB) and Recessive Dystrophic EB (RDEB), both caused
by mutations in the COL7A1 gene which affects type VII collagen, crucial for skin adhesion. DDEB typically presents with milder symptoms,
often limited to blistering on the hands, feet, elbows, and knees, with scarring, milia, and nail dystrophy being common. Conversely,
RDEB is much more severe, with widespread blistering that can involve both external and internal mucous membranes, leading to significant
complications like pseudosyndactyly, chronic wounds, severe scarring, and an increased risk of squamous cell carcinoma.
About INmune Bio Inc.
a publicly traded (NASDAQ: INMB), clinical-stage biotechnology company focused on developing treatments that target the innate immune
system to fight disease. INmune Bio has three product platforms: the Dominant-Negative Tumor Necrosis Factor (DN-TNF) product platform
utilizes dominant-negative technology to selectively neutralize soluble TNF, a key driver of innate immune dysfunction and a mechanistic
driver of many diseases. DN-TNF product candidates are in clinical trials to determine if they can treat Mild Alzheimer's disease,
Mild Cognitive Impairment and treatment-resistant depression (XPro ). The Natural Killer Cell Priming Platform includes INKmune developed
to prime a patient's NK cells to eliminate minimal residual disease in patients with cancer and is currently in trials in metastatic