Scioderm’s Investigational New Drug (IND) Application for SD-101
for Treatment of Epidermolysis Bullosa (EB) Allowed by the FDA
Raleigh, N.C. (February 1, 2013) - Scioderm announced that the US Food and Drug
Administration (FDA) has reviewed and allowed the investigational new drug (IND) application
for SD-101 to proceed. SD-101 is a topical treatment being developed for the treatment of
Epidermolysis Bullosa (EB). The Company plans to initiate a Phase 1 study in the coming
months, with initiation of the Phase 2B/3 study in EB patients to occur in the second half of
2013.
EB is a rare genetic condition that in all of its forms, share the prominent manifestation of
extremely fragile skin that blisters or tears with the slightest friction or trauma. The disease
affects not only the skin but also many internal organs and bodily systems. This particular
manifestation has led to EB patients being known as Butterfly children due to the analogous
nature of the fragility of the skin to the wings of a butterfly. As of today there is no cure or
effective treatment. Wound care, pain management and preventative bandaging are the only
options available for caregivers, usually the parents or other family members. The more severe
forms of the disease lead to scarring, disfigurement, disability and early death, usually before the
age of 30.
"We are excited to reach this important corporate milestone and to advance this novel therapy in
the clinic," stated Robert Ryan, Ph.D., Chairman of the Board and Co-Founder of
Scioderm. "We look forward to continuing our rapid development of this program to treat a
disease where there are no current effective therapies. In addition, we look forward to working
closely with the FDA to expedite the continuing clinical testing and regulatory review of SD101.”
“I am enthused by the results SD-101 has demonstrated and with the rapid progress Scioderm
has achieved in bringing this potential treatment to the clinic,” said Brett Kopelan, Executive
Director of the Dystrophic EB Research Association of America (DebRA) and father to a 5-yearold girl with recessive dystrophic EB. “I look forward to working with Scioderm as they work
towards improving the quality of life of those with EB by targeting the chronic wounds that are
the hallmark of this disease.”
About SD-101
SD-101 is a topical cream that has previously demonstrated potential to provide improvement in
treating the severe skin effects seen in patients across all EB subtypes. An open- label study was
conducted previously in children with either Simplex, Recessive Dystrophic (RDEB), or
Junctional EB. The primary outcome measurements were assessment of target wound reduction
and closure, and reduction in body surface area (BSA) coverage of lesions and erosions. In the
clinical trial, SD-101 application resulted in complete closure of 88% of target chronic lesions
within one month, in addition to a 57% reduction in BSA coverage of lesions and erosions after 3
months of daily treatment. SD-101 was well tolerated by the children, with daily administration
up to 3 months.
About Scioderm
Scioderm is a privately held, clinical-stage pharmaceutical company focused on developing
topical products to address critical medical needs in the treatment of chronic skin
diseases. Additional information about Scioderm can be found at www.sderm.com.
Company Contact:
Robert Coull
Chief Operating Officer
[email protected]
(917) 345-2504
SOURCE Scioderm
Forward-Looking Statements
Except for the historical information contained herein, the matters discussed in this press release
are forward-looking statements that involve risks and uncertainties, including: our dependence
on third parties for the development, regulatory approval and successful commercialization of
our products, the inherent risk of failure in developing product candidates based on new
technologies, risks associated with the costs of clinical development efforts, as well as other
risks. Actual results may differ materially from those projected. These forward-looking
statements represent our judgment as of the date of the release. Scioderm disclaims any intent or
obligation to update these forward-looking statements.