Paradigm Therapeutics acquires rights to SD-101 cream

Experimental therapy is intended to promote wound healing, ease pain in EB

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Paradigm Therapeutics has acquired worldwide rights to SD-101, an investigational cream that’s being developed as a whole-body therapy to promote wound healing and ease pain and itching in all forms of epidermolysis bullosa (EB).

The company now plans to meet with the U.S. Food and Drug Administration (FDA) to discuss next steps in the development of SD-101.

“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,” Robert Ryan, PhD, CEO of Paradigm, said in a press release.

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“There is a tremendous need for novel treatment options for treating the debilitating skin effects in patients with Epidermolysis Bullosa,” Ryan added. “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.”

All forms of EB are characterized by fragile skin that is prone to blistering and easily forms wounds. SD-101 is a topical (skin-applied) cream that contains 6% allantoin, a compound that’s thought to help promote wound healing.

The experimental therapy was being developed previously by Amicus Therapeutics. Paradigm and Amicus did not disclose the terms of the agreement by which Paradigm now isacquiring rights to SD-101.

Amicus sponsored the Phase 3 clinical trial ESSENCE  (NCT02384460) to evaluate the efficacy of SD-101. The study included more than 150 people with simplex, recessive dystrophic, or junctional types of EB.

Participants were assigned randomly to receive SD-101, or a control cream containing all the same ingredients — except for allatoin — applied to the whole body every day for about three months. The study’s main goal was to assess whether SD-101 could promote wound healing in patients.

Results showed that ESSENCE failed to meet its primary and key secondary goals. Compared to the control cream, SD-101 did not significantly improve wound healing, nor did it ease pain or itching.

However, study investigators said this failure might not be because SD-101 is ineffective, but rather because the control cream was more effective than expected. The control group experienced improvements in wound closure, possibly because the control cream included ingredients that may promote healing, such as lanolin oil and cod liver oil.

Data from ESSENCE showed that both SD-101 and the control cream were generally well tolerated. The most common side effect from either SD-101 or the control cream was itching, reported in six patients in the SD-101 group and five controls.

‘SD-101 is a product candidate for whole body treatment’

Collectively, data from ESSENCE and earlier 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,” Ryan said. “Results also demonstrated that SD-101 has a favorable safety profile.”

“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,” Ryan added.

SD-101 has received rare pediatric disease and breakthrough therapy designations from the FDA, as well as orphan drug designation from both the FDA and the European Commission. These designations are designed to incentivize and speed the development of treatments with the potential to improve medical care for people with rare or serious diseases.