Amicus’ SD-101 Cream Misses Goals in Epidermolysis Bullosa Phase 3 Trial

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by Magdalena Kegel |

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Amicus Therapeutics’ Phase 3 trial of SD-101 cream to treat wounds in patients with epidermolysis bullosa did not meet the company’s primary and secondary treatment goals, Amicus announced.

The 169-patient study, in which SD-101 was compared to a placebo, did not detect any difference in the time it took for wounds to close. Also, the percentage of patients whose wounds closed were similar for both groups at three months. This means the trial missed both of its main goals.

At three months, 49% of patients in the SD-101 group saw their target wounds heal. In the placebo group, that number was 54%. Analyses also showed that secondary outcomes were similar in the treated and control groups. Secondary goals included changes in lesional skin, itching, pain, and total body wound burden.

“We are disappointed that this Phase 3 study of SD-101 did not meet the primary endpoints in epidermolysis bullosa, an utterly devastating rare genetic disorder with no approved treatment options,” John F. Crowley, Amicus’ chairman and CEO, said in a press release.

The Phase 3 trial, called ESSENCE (NCT02384460), recruited 169 patients with all three main types of the skin disease: simplex, recessive dystrophic, and junctional non-Herlitz epidermolysis bullosa.

Participants were randomly split into two groups: 82 patients received treatment with SD-101 and 87 received a placebo cream.

Safety was similar in the two groups, with the most common treatment-emergent adverse events being common colds, itching, and fever.

Nonetheless, researchers said that they noted trends in wound closure in certain patient subgroups. Amicus now plans to work together with physicians, patient organizations, and regulators involved in the epidermolysis bullosa community to further analyze the data.

Meanwhile, patients enrolled in the company’s extension studies of the SD-101 cream (NCT02090283 and NCT02670330) will have the opportunity to remain on treatment, Amicus stated.

Nevertheless, the clinical trial failure appears to be the end of SD-101 development, with Amicus saying that it currently has no plans for additional studies and does not plan a marketing launch.

The company’s earlier Phase 2b trial showed promise for both time to wound closure and percentage of patients with closed wounds. But the trial was small and findings did not translate into significant differences in the Phase 3 trial.

“In keeping with our Amicus mission, we have a strong commitment to the EB [epidermolysis bullosa] community and will work closely with investigators and other leading experts to understand and to share these data,” Crowley said.

“We would like to sincerely thank the patients, families, clinical investigators, regulators and our Amicus team involved in this EB program. In seeking to develop novel, high-quality therapies for those living with devastating rare diseases we may sometimes fail. But we would rather be the first to fail than the last to try,” Crowley said.