'Topical Therapy for Treating the Wounds and Lesions on the Entire Skin Surface'

MT PLEASANT, S.C., Aug. 22, 2023 /PRNewswire/ -- Paradigm Therapeutics Inc., a biopharmaceutical company, today announced the completion of the acquisition of the worldwide rights of SD-101, a topical whole-body treatment for all subtypes of Epidermolysis Bullosa (EB), from Amicus Therapeutics. Terms of the deal have not been disclosed.

SD-101 received Rare Pediatric Disease designation from the U.S. Food and Drug Administration (FDA) covering the broad treatment of EB. In addition to the Rare Pediatric Disease designation, SD-101 was granted Orphan Drug designation from the FDA and the European Commission (EC) and was the first EB treatment to receive the FDA's Breakthrough Therapy designation.

"We are excited about the opportunity to continue the development of SD-101 and to discuss the current data with the FDA to expedite a path forward towards registration of this therapy for patients with all EB subtypes," said Robert Ryan., Ph.D., Chief Executive Officer of Paradigm Therapeutics. "There is a tremendous need for novel treatment options for treating the debilitating skin effects in patients with Epidermolysis Bullosa. The Paradigm Therapeutics team will be working passionately to provide patients with a new therapeutic alternative, in this disease with no effective whole-body treatments."

Dr. Ryan added, "Epidermolysis Bullosa is a devastating disease. We believe the clinical data show beneficial effects of the use of SD-101 across the range of EB patients, and strongly support progression towards registration. Results from the completed Phase II and Phase III trials indicate that SD-101 is a product candidate for whole body treatment of the lesions and wounds on skin in pediatric and adult patients with EB, who have no other whole body treatment alternatives. Results also demonstrated that SD-101 has a favorable safety profile."

About Epidermolysis Bullosa (EB)

Epidermolysis bullosa is a rare, devastating genetic disorder causing very fragile skin that leads to severe skin blistering and open wounds often beginning at birth. There are no approved therapies to treat the whole body of patients with EB, which affects many children and adults throughout their lifetimes. The primary standard of care consists of pain management, and cleaning and bandaging of open wounds to prevent infection.  EB is a chronic disease, potentially disfiguring, and in some cases fatal. There are many genetic and symptomatic variations of EB, but all forms share the common symptom of fragile skin that blisters and tears, sometimes from the slightest friction or trauma.

About SD-101

SD-101 is a topical cream that has demonstrated potential to provide improvement in treating the severe skin disruptions seen in patients across all EB subtypes. Phase II and Phase III studies were conducted in children and adults with either Simplex, Recessive Dystrophic (RDEB), or Junctional EB. A total of 217 EB patients were included in these studies. SD-101 was applied topically over the entire body daily in adults and children as young as 21 days old for a period of three months. The primary outcome measurement was assessment of target wound closure, in addition to secondary measurements including reduction in body surface area (BSA) coverage of blisters and wounds. 

About Paradigm Therapeutics

Paradigm Therapeutics is a privately held, clinical-stage pharmaceutical company focused on developing innovative therapies to address critical medical needs in the treatment of rare diseases. Paradigm's main focus is the treatment of Epidermolysis Bullosa (EB).  SD-101, its leading therapy for the treatment of skin effects in patients with Epidermolysis Bullosa, has completed Phase II and Phase III clinical trials. SD-101 is a topical cream that has demonstrated potential to provide improvement in treating the severe skin effects seen in patients across all EB subtypes. 

Company Contact
Dr. Robert Ryan
Chief  Executive Officer
rryan@paradigmtherapeutics.com
919-274-0703
www.paradigmtherapeutics.com

Source: Paradigm Therapeutics