The EFFISAYIL 2 trial, which evaluated maintenance treatment with subcutaneous spesolimab for the prevention of GPP flares and sustained control of GPP symptoms, met its primary and key secondary endpoint and reinforced previous positive results in the EFFISAYIL clinical programme. Spesolimab, sold under the brand name Spevigo, is already approved in major markets for GPP flares in adults. The antibody was most recently approved by the European Commission in December 2022 for this indication, a decision supported by results from the phase 2 EFFISAYIL 1 trial. In EFFISAYIL 1, after one week, 54% of patients treated with a single dose of Spevigo showed no visible pustules, compared to 6% of patients in the placebo group. After 12 weeks, more than four out of five Spevigo-treated patients had no visible pustules and clear/almost clear skin. “These results support positive results from the previous phase 2 study in the EFFISAYIL clinical trial programme, which formed the basis of spesolimab approvals as the first specific treatment for GPP flares in adults in major markets including the US, Japan, Mainland China and the EU,” said Carinne Brouillon, member of the board of managing directors, responsible for human pharma at BI. GPP is a rare, heterogenous and potentially life-threatening neutrophilic skin disease, which is clinically distinct from plaque psoriasis. GPP is caused by neutrophils – a type of white blood cell – accumulating in the skin, causing painful, sterile pustules all over the body. Despite the varying severity of GPP flares, if left untreated they can be life-threatening due to complications such as sepsis and multisystem organ failure, and the unpredictability and severity of these flares greatly affects quality of life.
“Painful GPP flares can occur suddenly, escalate quickly and may require urgent hospital care leaving people anxious and uncertain about what the future might hold,” Brouillon said.
BI reports that there is a ‘high unmet need’ for treatments with an acceptable safety profile that can rapidly resolve the symptoms of GPP flares and prevent their reoccurrence.