RIDGEFIELD, Conn., March 28, 2022 /PRNewswire/ -- Boehringer Ingelheim announced today new data from the pivotal Phase II Effisayil™ 1 trial, presented at the 2022 American Academy of Dermatology (AAD) Annual Meeting in Boston.

The Effisayil™ 1 trial, recently published in The New England Journal of Medicine, showed significant clearance of skin pustules in patients with generalized pustular psoriasis (GPP) flares within the first week after treatment with spesolimab versus placebo. This effect was sustained over 12 weeks, according to data presented at AAD, which found that 84.4% of patients had no visible pustules after the 12-week trial duration and 81.3% had clear/almost clear skin.

"GPP is an unpredictable, painful, and potentially life-threatening rare skin disease with no available FDA-approved treatment options," said Boni Elewski, M.D., trial investigator and Chair, Department of Dermatology at The University of Alabama School of Medicine. "The findings presented at this year's AAD Annual Meeting showed that the efficacy of spesolimab is sustained over 12 weeks, providing further evidence of the rapid benefit that spesolimab could bring to patients living with the burden of GPP flares."

GPP is a rare, potentially life-threatening neutrophilic skin disease, which is distinct from plaque psoriasis. It is characterized by episodes of widespread eruptions of painful, sterile pustules (blisters of non-infectious pus). GPP flares greatly affect a person's quality of life and can lead to serious and life-threatening complications, including heart failure, renal failure, and sepsis.

According to additional data presented at the AAD Annual Meeting, rapid skin clearance observed in the first week following treatment with spesolimab was generally consistent across patient subgroups, including age, gender, ethnicity, and IL-36 gene mutation status. Also, significant improvements were shown within one week in patient-reported outcomes related to pain, fatigue, quality of life, and skin symptoms after treatment with spesolimab.

In the Effisayil™ 1 trial, adverse events were reported in 66% of patients treated with spesolimab and 56% of those receiving placebo after the first week. Infections were reported by 17% and 6% of patients in the spesolimab and placebo groups, respectively (at week one). Serious adverse events were reported in 6% of patients treated with spesolimab (at week one). Two patients receiving spesolimab were reported to have drug reactions with eosinophilia and systemic symptoms.
 

"With these additional data, we are gaining a more complete picture of spesolimab as a possible first-in-class treatment approved for GPP patients," said Matt Frankel, M.D., Vice President, Clinical Development and Medical Affairs, Specialty Care, Boehringer Ingelheim. "GPP has a significant impact on a patient's life, and we remain committed to bringing spesolimab to patients as quickly as possible."

The U.S. Food and Drug Administration (FDA) accepted a Biologics License Application (BLA) and granted Priority Review for spesolimab for the treatment of GPP flares. The FDA has granted spesolimab Orphan Drug Designation for the treatment of GPP and Breakthrough Therapy Designation for spesolimab for the treatment of GPP flares in adults.

About spesolimab
Spesolimab is a novel, humanized, selective antibody that blocks the activation of the interleukin-36 receptor (IL-36R), a signaling pathway within the immune system shown to be involved in several autoimmune diseases pathogeneses, including GPP. It is the first investigational treatment to specifically target the IL-36 pathway for the treatment of GPP flares that has been evaluated in a statistically powered, randomized, placebo-controlled trial. Spesolimab is also under investigation for the prevention of GPP flares and for the treatment of other neutrophilic skin diseases, such as palmoplantar pustulosis (PPP) and hidradenitis suppurativa (HS).

About the Effisayil 1™ clinical trial
Effisayil™ 1 (NCT03782792) was a 12-week Phase II trial that investigated patients with a GPP flare (N=53), randomly assigned 2:1 to a single 900 mg intravenous dose of spesolimab or placebo. The primary endpoint was a GPPGA pustulation subscore of 0 (no visible pustules) at week one. The key secondary endpoint was a GPPGA total score of 0/1 (clear/almost clear skin) at week one.

After one week, 54% of patients (19 out of 35) treated with spesolimab showed no visible pustules (GPPGA pustulation subscore of 0), compared to 6% of patients (1 out of 18) treated with placebo. In addition, 43% of patients (15 out of 35) treated with spesolimab showed clear/almost clear skin (GPPGA total score of 0/1), compared to 11% of patients (2 out of 18) in the placebo group.

About generalized pustular psoriasis (GPP)
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, resulting in painful, sterile pustules all over the body. The clinical course varies, with some patients having a relapsing disease with recurrent flares, and others having a persistent disease with intermittent flares. While the severity of GPP flares can vary, if left untreated they can be life-threatening due to complications such as sepsis and multisystem organ failure. This chronic, systemic disease has a substantial quality of life impact for patients and increased healthcare burden. GPP has a varied prevalence across different geographical regions and more women are affected than men.

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Boehringer Ingelheim
Boehringer Ingelheim is working on breakthrough therapies that improve the lives of humans and animals. As a leading research-driven biopharmaceutical company, the company creates value through innovation in areas of high unmet medical need. Founded in 1885 and family-owned ever since, Boehringer Ingelheim takes a long-term perspective. Around 52,000 employees serve more than 130 markets in the three business areas, Human Pharma, Animal Health, and Biopharmaceutical Contract Manufacturing. Learn more at www.boehringer-ingelheim.com.