Spesolimab meets primary and key secondary endpoint for prevention of generalized pustular psoriasis flares

EFFISAYIL™ 2 met its primary and key secondary endpoint, demonstrating that spesolimab, an anti-interleukin-36 receptor antibody, can prevent flares in adolescents and adults with generalized pustular psoriasis (GPP) up to 48 weeks.^1,2 Safety data were in line with previously conducted clinical trials with spesolimab. 

“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,” said Carinne Brouillon, Member of the Board of Managing Directors, responsible for Human Pharma, Boehringer Ingelheim. “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. We look forward to presenting the data later this year and sharing the findings with regulatory authorities.” 

GPP is a rare skin disease, which is distinct from plaque psoriasis.⁴ 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.⁴ 

Spesolimab (marketed as SPEVIGO^®) is currently indicated 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 the pathogenesis of several autoinflammatory diseases, including GPP.^4,6,7 Spesolimab has been approved by regulatory authorities in several countries including the US, Japan, Mainland China and the European Union to treat GPP flares in adults.^8,9 

It is the first approved 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. Further spesolimab data is expected later this year, including the final EFFISAYIL™ 2 results investigating prevention of GPP flares. Spesolimab is also under investigation for the treatment of other IL-36 mediated skin diseases.¹⁰ 

About the EFFISAYIL^TM clinical trial program 

The EFFISAYIL^TM clinical trial program includes: 

• EFFISAYIL^TM 1: Treatment with spesolimab demonstrated rapid pustular and skin clearance in patients with GPP flares, sustained over 12 weeks.³ These results supported the approval of spesolimab (SPEVIGO^®) as the first specific treatment for GPP flares in adults in major markets including the US, Japan, Mainland China and the European Union.^8,9 

• EFFISAYIL^TM 2: A multicenter, randomized, double-blind, placebo-controlled Phase IIb study evaluating the efficacy and safety of maintenance treatment with subcutaneous (SC) spesolimab for the prevention of GPP flares and sustained control of GPP symptoms in adolescents and adults.^1,2 

• EFFISAYIL^TM ON: To evaluate the long-term safety and efficacy of Spesolimab in patients with GPP, who have completed previous Spesolimab trials.⁵ 

Results from the EFFISAYIL^TM 2 and EFFISAYIL^TM ON trials will be presented later this year. 

About generalized pustular psoriasis (GPP) 

GPP is a rare, heterogenous and potentially life-threatening neutrophilic skin disease, which is clinically distinct from plaque psoriasis.^4,11 GPP is caused by neutrophils (a type of white blood cell) accumulating in the skin, resulting in painful, sterile pustules all over the body.^4,11 The clinical course varies, with some patients having a relapsing disease with recurrent flares, and others having a persistent disease with intermittent flares.¹¹ 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.⁴ This chronic, systemic disease has a substantial quality of life impact for patients and places an increased burden on healthcare systems.¹² GPP has a varied prevalence across different geographical regions and more women are affected than men.^4,12-15 There is a high unmet need for treatments with an acceptable safety profile that can prevent the occurrence of GPP flares.^3,16 

GPP flares can lead to hospitalization with serious complications, including heart failure, renal failure and sepsis, with the unpredictability and severity of these flares greatly affecting a person’s quality of life.^4,16 

About Boehringer Ingelheim  

Boehringer Ingelheim is working on breakthrough therapies that transform lives, today and for generations to come. 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. More than 52,000 employees serve over 130 markets in the three business areas, Human Pharma, Animal Health, and Biopharmaceutical Contract Manufacturing. Learn more at www.boehringer-ingelheim.com. 

Intended audiences 

This press release is issued from our Corporate Headquarters in Ingelheim, Germany and is intended to provide information about our global business. Please be aware that information relating to the approval status and labels of approved products may vary from country to country, and a country-specific press release on this topic may have been issued in the countries where we do business.

References

1. Morita A, Choon SE, Bachelez H et al. Design of Effisayil^TM 2: A randomized, double-blind, placebo-controlled study of spesolimab in preventing flares in patients with generalized pustular psoriasis. Dermatol Ther (Heidelb) (2023) 13:347–359.
2. A study to test whether BI 655130 (spesolimab) prevents flare-ups in patients with generalized pustular psoriasis. www.clinicaltrials.gov/ct2/show/study/NCT04399837?term=Boehringer. Last accessed January 2023.
3. Bachelez H et al. Trial of spesolimab for generalized pustular psoriasis. NEJM. 2021;385:2431-40.
4. Crowley JJ, et al. A brief guide to pustular psoriasis for primary care providers, Postgraduate Medicine. 2021;133(3):330-344.
5. Effisayil ON: An open-label, long term extension study to assess the safety and efficacy of spesolimab treatment in patients with generalized pustular psoriasis (GPP). https://clinicaltrials.gov/ct2/show/NCT03886246?term=Effisayil&draw=2&rank=1. Last accessed January 2023.
6. Furue K, et al. Highlighting Interleukin-36 Signalling in plaque psoriasis and pustular psoriasis. Acta Derm Venereol. 2018;98:5–13.
7. 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.
8. Federal Drug Administration. New Drug Approvals for 2022. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/761244s000lbl.pdf. Accessed January 2023.
9. EC approval statement (data on file).
10. A study to test whether spesolimab helps people with a skin disease called hidradenitis suppurativa. Available at: https://clinicaltrials.gov/ct2/show/NCT04762277.January 2023.
11. Navarini AA, et al. European consensus statement on phenotypes of pustular psoriasis. JEADV. 2017;31:1792-1799.
12. 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.
13. Augey F, et al. Generalized pustular psoriasis (Zumbusch): a French epidemiological survey. Eur J Derm. 2006; 16(6):669-673.
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. Jin H, et al. Clinical features and course of generalized pustular psoriasis in Korea. J Dermatol. 2015; 42(7):674-678.
16. Puig L, Choon SE, Gottlieb AB, et al. Generalized pustular psoriasis: a global Delphi consensus on clinical course, diagnosis, treatment goals, and disease management. J Eur Acad Dermatol Venereol. 2023 Jan 6. doi: 10.1111/jdv.18851.