INM-755 (Cannabinol) Cream for Treatment of Epidermolysis Bullosa

A Randomised, Double-Blind, Vehicle-Controlled Phase 2 Study of Topically Applied INM-755 (Cannabinol) Cream in Patients With Epidermolysis Bullosa

The purpose of this study is to evaluate the safety of INM-755 (cannabinol) cream and obtain preliminary evidence of efficacy in treating symptoms and healing wounds over a 28-day period in patients with epidermolysis bullosa (EB).

This is an international, multicenter study to evaluate the safety and obtain preliminary evidence of efficacy of topically applied INM-755 (cannabinol) cream in up to 20 patients with inherited EB (Simplex, Dystrophic, Junctional, or Kindler). The study uses a within-patient, double-blind design in which matched index areas are randomized to INM-755 (cannabinol) cream or vehicle cream as a control.

Selected index areas may be wounds or non-wound areas. Randomized treatments are applied daily to non-wound areas and every 1, 2, or 3 days on wounds, according to the patient's schedule for dressing changes. Treatment is over a 28-day period. EB symptoms are evaluated based on patient-reported outcomes. Wound healing is measured by digital photography. In this study designed to obtain preliminary evidence of efficacy, there is no single primary efficacy endpoint. Efficacy endpoints vary according to the presenting symptoms in each patient, which may include the presence of open wounds, wound pain associated with dressing changes, background wound pain (not procedurally linked), wound itch, and itch in non-wound areas. Net benefit from INM-755 cream is evaluated within each patient and based on their clinical needs at baseline.

Enrollment is beginning with adults and may be expanded to include adolescents.

Key Inclusion Criteria:

• Male or female patients aged ≥18 years with documented diagnosis of any of the following types of inherited EB: Simplex, Junctional, Dystrophic, Kindler. Enrolment will be extended to patients ≥12 years old and <18 years old after positive opinion of a data monitoring committee after at least 4 adult patients have completed study.

• Presence of at least 1 pair of well-matched index areas. Up to 2 pairs of index areas (1 pair of non-wound and 1 pair of wound index areas) can be selected in each patient

• For non-wound itch index areas, both areas should:

  1. Have chronic itch with a score ≥40 mm on a 100 mm VAS
  2. Not exceed 20 percent of body surface area (BSA) or be less than 1 percent of BSA
  3. Be similar size, up to twice the area of the smaller index area

• For wound index areas, both wounds should:

  1. Have a surface area ≥5 cm^2 and ≤50 cm^2 inclusive and be aged ≥3 weeks
  2. Be well matched for size (up to twice the area of the smaller index area) and age (both either ≥3 weeks to 3 months or >3 months)

• Female patients of childbearing potential or men whose sexual partners are women of childbearing potential (WOCBP) must use highly effective birth control

• WOCBP must have a negative urine pregnancy test result at baseline

• Must provide written consent (or assent for patients aged <18 years with parental/guardian consent)

Key Exclusion Criteria:

• EB index areas have evidence of infection
• Patient has a systemic infection or used systemic antibiotics for EB-related infections within 7 days
• Use of systemic corticosteroids within 30 days or of topical corticosteroids on chosen index areas within 14 days
• Immunosuppressive therapy or cytotoxic chemotherapy within 60 days
• Use of any high potency opioid within 30 days
• Use of cannabis, cannabis extracts, or any cannabinoid products for medical or recreational use by any route of administration within 2 weeks
• Prior stem cell transplant or gene therapy for EB
• History of malignancy including basal cell and squamous cell carcinomas
• Arterial or venous disorder resulting in ulcerated wounds
• Uncontrolled diabetes mellitus
• Chronic pruritus primarily attributable to pathologies or conditions other than EB
• Blood transfusion to treat anemia within the past 3 months
• Use of any investigational drug within 30 days or 5 half-lives (whichever is longer)
• An underlying condition which places the patient at unacceptable risk
• Women who are pregnant, breastfeeding (lactating), or planning to become pregnant during the study