ESSENCE Study: Efficacy and Safety of SD-101 Cream in Participants With Epidermolysis Bullosa

This was a Phase 3, multi-center, randomized, double-blind, placebo-controlled study to
assess the efficacy and safety of SD-101-6.0 cream versus placebo (SD-101-0.0) cream on skin
lesions in participants with Simplex, Recessive Dystrophic, or Junctional non-Herlitz
Epidermolysis Bullosa (EB). Epidermolysis Bullosa is a rare group of inherited disorders that
typically manifest at birth as blistering and lesion formation on the skin in response to
little or no apparent trauma. In this study, SD-101-6.0 cream or placebo (SD-101-0.0) cream
was to be applied topically, once a day to the entire body for a period of 90 days.
Participants had 1 target wound selected at baseline by the investigator. The selected target
wound was required to have been present for at least 21 days. Photographic confirmation of
the target wound location was collected at baseline, and the picture saved from the first
visit was used to confirm location of the target wound at subsequent visits. The participant
returned to the study site for Visit 2 (approximately 14 days from baseline), Visit 3
(approximately 30 days from baseline), Visit 4 (approximately 60 days from baseline), and
Visit 5 (approximately 90 days from baseline) to have the target wound assessed for the level
of healing. In addition, itching, pain, body surface area, target wound closure, and scarring
of healed target wound were assessed at each visit. The ARANZ SilhouetteStar™ was used to
measure the target wound at all visits.

Inclusion Criteria:

- Informed Consent form signed by the participant or participant's legal representative;
if the participant was under the age of 18 but capable of providing assent, signed
assent from the participant.

- Participant (or caretaker) must have been willing to comply with all protocol
requirements.

- Diagnosis of Simplex, Recessive Dystrophic, or Junctional non-Herlitz EB.

- Participant must have had 1 target wound (size 10 to 50 cm^2) at study entry.

- Participants 1 month and older.

- Target wound must have been present for at least 21 days.

Exclusion Criteria:

- Participants who did not meet the entry criteria outlined above.

- Selected target wound did not have clinical evidence of local infection.

- Use of any investigational drug within the 30 days before enrollment.

- Use of immunotherapy or cytotoxic chemotherapy within the 60 days before enrollment.

- Use of systemic or topical steroidal therapy within the 30 days before enrollment.
(Inhaled steroids and ophthalmic drops containing steroids were allowed).

- Use of systemic antibiotics within the 7 days before enrollment.

- Current or former malignancy.

- Arterial or venous disorder resulting in ulcerated lesions.

- Pregnancy or breastfeeding during the study. (A urine pregnancy test was performed at
screening and every 30 days until the final visit for female participants of
childbearing potential).

- Females of childbearing potential who were not abstinent and not practicing a
medically acceptable method of contraception.