Newly published trial results showed spesolimab significantly improved signs and symptoms of flare in rare, life-threatening skin disease, generalized

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Overig advies 31/12/2021 11:35
New clinical trial data published today in the New England Journal of Medicine, showed that spesolimab, a novel IL-36R antibody treatment, was effective in rapidly treating adult patients with generalized pustular psoriasis (GPP) experiencing a flare.1
The study met the primary endpoint, where 54% of patients had no visible pustules after a single dose of spesolimab, compared to 6% receiving placebo at week one.1
Spesolimab has been granted Breakthrough Therapy Designation in China and USA
Ingelheim, Germany, December 23, 2021 – Boehringer Ingelheim announced today the publication in the New England Journal of Medicine of new data from the pivotal Phase II EffisayilTM 1 trial, which showed spesolimab, a first-in-class investigational treatment, significantly improved signs and symptoms of generalized pustular psoriasis (GPP) in patients experiencing a flare.1

GPP is a rare, 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).2,3,4 There is a high unmet need for treatments that can rapidly and completely resolve the symptoms of GPP flares. Flares greatly affect a person’s quality of life5 and can lead to hospitalization with life-threatening complications, such as heart failure, renal failure and sepsis and even death.6

In the 12-week trial, 53 patients experiencing a GPP flare were treated with a single intravenous dose of spesolimab or placebo. Most patients at the outset of the trial had a high or very high density of pustules and impaired quality of life. Results after one week demonstrated that:

54% of patients treated with spesolimab showed no visible pustules compared to 6% of those treated with placebo;
43% of patients treated with spesolimab showed clear/almost clear skin compared to 11% of those in the placebo group.1
Pustular and skin clearance continued for the duration of the study. This clearance was accompanied by clinically significant improvements in quality of life and symptoms such as pain and fatigue, compared to placebo.1
Over the 12-week duration of the study, non-serious infections rates were higher in the spesolimab group compared with placebo, with no pattern regarding pathogen and affected organs. Two patients reported to have drug reactions with eosinophilia and systemic symptoms.

“With no approved treatments in the U.S. or E.U. for GPP flares, there is a significant unmet need for people with this distressing and painful skin condition, that often requires emergency care,” said Mark Lebwohl, MD, lead investigator and publication author, and Dean for Clinical Therapeutics, Icahn School of Medicine at Mount Sinai, Kimberly and Eric J. Waldman Department of Dermatology, New York. “These clinical trial results show that spesolimab has the potential to completely clear the skin of the signs and symptoms of a GPP flare after only one week, with sustained effect observed for up to 12 weeks.”

The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for the treatment of GPP, and Breakthrough Therapy Designation for spesolimab for the treatment of GPP flares. This designation is for therapies treating serious or life-threatening conditions where early clinical evidence suggests a substantial improvement compared to existing treatments. The Chinese Regulatory Authority Centre for Drug Evaluation (CDE) also recently granted Breakthrough Therapy Designation for spesolimab for the treatment of GPP flares.

“At Boehringer Ingelheim, we are committed to finding transformative therapies to help advance treatment for people who urgently need them,” said Dr Emmanuelle Clerisme-Beaty, Head of Clinical Development and Medical Affairs, Dermatology, Boehringer Ingelheim. “The findings indicate that spesolimab may have a significant and positive impact on patients experiencing a GPP flare.”

The clinical program for spesolimab includes two other trials that are currently underway. First, the Effisayil-2 trial is designed to investigate spesolimab as a maintenance treatment to prevent the occurrence of GPP flares. The Effisayil-ON trial is an open label five-year extension study to investigate the longer term efficacy and safety of spesolimab in patents with GPP.7,8

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.2,9,10 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).11,12

About the EffisayilTM 1 clinical trial
Effisayil™ 1 (NCT03782792) was a 12-week Phase II trial investigating 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 GPP Physician Global Assessment (GPPGA) pustulation subscore of 0 (no visible pustules) at week one. The key secondary endpoint was a GPPGA score of 0/1 (clear/almost clear skin) at week one.1

After one week, 54% of patients (19 out of 35) treated with spesolimab showed no visible pustules (GPPGA score of 0), compared to 6% of patients (1 out of 18) treated with placebo ( P<0.001). In addition, 43% of patients (15 out of 35) treated with spesolimab showed clear/almost clear skin (GPPGA score of 0/1), compared to 11% of patients (2 out of 18) in the placebo group ( P=0.024).1

After one week, adverse events were reported in 66% of patients treated with spesolimab and 56% of those receiving placebo. Infections were reported by 17% and 6% of patients in the spesolimab and placebo groups respectively. Serious adverse events were reported in 6% of patients treated with spesolimab.1

About generalized pustular psoriasis
GPP is a rare, heterogenous and potentially life-threatening neutrophilic skin disease, which is clinically distinct from plaque psoriasis.2,3 GPP is caused by neutrophils (a type of white blood cell) accumulating in the skin, resulting in painful, sterile pustules all over the body.3 The clinical course varies, with some patients having a relapsing disease with recurrent flares, and others having a persistent disease with intermittent flares.3 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.6 This chronic, systemic disease has a substantial quality of life impact for patients and healthcare burden.13 GPP has a varied prevalence across geographical regions and more women are affected than men.2,14,15,16

Treatments for GPP
There is a high unmet need for treatments that can rapidly resolve the symptoms of GPP flares and prevent their reoccurrence, with an acceptable safety profile. Immunomodulatory therapies, including biologics, are used in the treatment of GPP based on clinical experience in patients with plaque psoriasis. However, there is limited evidence on the efficacy and safety of these therapies in the treatment of GPP.

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1 Bachelez H et al. Trial of Spesolimab for Generalized Pustular Psoriasis. NEJM. 2021;385:2431-40.
2 Crowley JJ, et al. A brief guide to pustular psoriasis for primary care providers, Postgraduate Medicine. 2021;133(3):330-344.
3 Navarini AA, et al. European consensus statement on phenotypes of pustular psoriasis. JEADV. 2017;31:1792-1799.
4 Fujita H, et al. Japanese guidelines for the management and treatment of generalized pustular psoriasis: The new pathogenesis and treatment of GPP. J Dermatol. 2018;45(11):1235–1270.
5 Sampogna F, et al. Measuring quality of life of patients with different clinical types of psoriasis using the SF-36. Br J Dermatol. 2006;154(5):844–849.
6 Jeon C, et al. Generalized pustular psoriasis treated with apremilast in a patient with multiple medical comorbidities. JAAD. 2017;Nov;3(6):495–497.
7 A 5-year Study to Test BI 655130 in Patients With Generalized Pustular Psoriasis Who Took Part in Previous Studies With BI 655130. Available at Accessed: September 2021.
8 A Study to Test Whether BI 655130 (Spesolimab) Prevents Flare-ups in Patients With Generalized Pustular Psoriasis. Available at: Accessed: September 2021.
9 Furue K, et al. Highlighting Interleukin-36 Signalling in Plaque Psoriasis and Pustular Psoriasis. Acta Derm Venereol. 2018;98:5–13.
10 Bachelez H, et al. Inhibition of the Interleukin-36 Pathway for the Treatment of Generalized Pustular Psoriasis. N Engl J Med. 2019; 380:981-983.
11 A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Hidradenitis Suppurativa. Available at: Accessed September 2021.
12 A Study to Test Long-term Treatment With Spesolimab in People With Palmoplantar Pustulosis (PPP) Who Took Part in Previous Studies With Spesolimab. Available at Accessed: September 2021.
13 Hanna M, et al. Economic burden of generalized pustular psoriasis and palmoplantar pustulosis in the United States. Curr Med Res Opin. 2021. 37(5):735-742.
14 Ohkawara A et al. Generalized pustular psoriasis in Japan: two distinct groups formed by differences in symptoms and genetic background. Acta Derm Venereol. 1996 Jan;76(1):68–71.
15 Augey F, et al. Generalized pustular psoriasis (Zumbusch): a French epidemiological survey. European Journal of Dermatology. 2006; 16(6):669-673.
16 Jin H, et al. Clinical features and course of generalized pustular psoriasis in Korea. The Journal of Dermatology. 2015; 42(7):674-678.

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