Results from the phase 2 and long-term trials will be presented later this year, following further data collection.
Spesolimab (Spevigo; Boehringer Ingelheim), an anti-interleukin-36 receptor antibody that targets a pathogenic immune system signaling pathway, can prevent flares in adolescents and adults with rare generalized pustular psoriasis (GPP), according to data published from the phase 2 EFFISAYIL 2 trial (NCT04399837).
The EFFISAYIL 2 trial met its primary and secondary endpoints. It showed that spesolimab, which is a novel, humanized, selective antibody, can be a safe and effective maintenance treatment that prevents GPP flare-ups and controls its associated symptoms.
“The EFFISAYIL 2 results reinforce the potential of spesolimab to prevent GPP flares, giving patients the power to plan their lives, regardless of their disease,” said Carinne Brouillon, member of the board of managing directors, Boehringer Ingelheim, in a press release.
GPP is a skin disease that is clinically different than plaque psoriasis. GPP is caused by white blood cells (neutrophils) that accumulate on the skin, which develops into painful, sterile pustules. GPP can cause recurrent or intermittent flares that are highly unpredictable and range in severity—if left untreated, a patient can experience life-threatening sepsis, renal failure, or multisystem organ failure.
“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 in the press release.
The EFFISAYIL clinical trial program was created to evaluate spesolimab as a preventative treatment for a range of patients with GPP. The first EFFISAYIL trial looked at spesolimab treatment over 12 weeks and showed that spesolimab significantly increased skin and pustular clearance, becoming the first GPP flare-specific treatment to become available in the United States, Japan, Mainland China, and European Union markets.
Spesolimab blocks the interleukin-36 receptor (IL-36R) pathway, a known pathway that is involved in GPP and several other autoinflammatory diseases. It is the first treatment approved for GPP flare-ups that specifically targets the IL-36 pathway. It is also the first to have been studied in a statistically powered, randomized, and placebo-controlled trial.
EFFISAYIL 2 was a multicenter, randomized, double-blind, placebo-controlled phase 2 trial which evaluated the efficacy and safety of subcutaneous (SC) spesolimab as a preventative and maintenance treatment for GPP. The effects of spesolimab lasted for 11 months after treatment in both adolescents and adults.
Investigators will study the same patient population in the EFFISAYIL ON trial, looking at long-term safety and efficacy. Additionally, spesolimab is being investigated as a treatment for different IL-36 mediated skin diseases, and investigators await more data about the results of the EFFISAYIL 2 trial.
“We look forward to presenting the data later this year and sharing the findings with regulatory authorities,” Brouillon said in the press release.
Spesolimab meets primary and key secondary endpoint for prevention of generalized pustular psoriasis flares. News Release. January 30, 2023. Accessed January 31, 2023. https://www.boehringer-ingelheim.com/human-health/skin-diseases/gpp/spesolimab-meets-key-endpoint-for-gpp-flare-prevention