Spesolimab prevented generalized pustular psoriasis flares in Effisayil™ 2 trial

Ridgefield, Connecticut July 4, 2023 – Today, at the 25^th World Congress of Dermatology (WCD), Boehringer Ingelheim presented new late-breaking data from the Effisayil™ 2 trial showing that spesolimab significantly reduced the risk of GPP flares by 84% over 48 weeks compared to placebo. Furthermore, in the trial with 123 patients, no flares were observed after Week 4 of spesolimab treatment in the high dose group (n=30).

“Effisayil™ 2 is the first and largest multinational randomized clinical trial to evaluate a treatment for the prevention of GPP flares,” said Bruce Strober, MD, PhD, Clinical Professor, Dermatology, Yale University and Central Connecticut Dermatology. “These results provide further compelling clinical evidence for the role IL-36 signaling plays in the pathogenesis of GPP. Moving forward, our hope is that dermatologists not only have a specific treatment for GPP flares, but that we can effectively prevent them in the future.”

GPP flares, characterized by painful pustules all over the body, may require emergency care and can lead to serious, life-threatening complications such as sepsis, shock and multisystem organ failure. In addition to the acute distress of a GPP flare, living with the uncertainty of the impact of the next flare can have a significant emotional burden on patients.

“At IFPA we meet people living with GPP, who share their story of physical pain due to the symptoms of the disease, and the mental burden that comes with the flares,” said Frida Dunger Johnsson, Executive Director, International Federation of Psoriasis Associations. “Any progress made to ease the burden of the disease improves the quality of life of people living with GPP.”

"Through our comprehensive Effisayil™ clinical program we have already delivered spesolimab as a major advancement for GPP patients," said Carinne Brouillon, Head of Human Pharma, Boehringer Ingelheim. "The Effisayil™ 2 trial results build on this success bringing us closer to achieving our ultimate goal of a flare-free future for everyone living with GPP."

The Effisayil™ 2 trial showed a similar incidence of patients with adverse events across spesolimab and placebo treatment arms.

About spesolimab
Spesolimab is a novel, humanized, selective antibody that specifically 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.

It is the first approved treatment for GPP flares in adults that has been evaluated in a statistically-powered, randomized, placebo-controlled trial. To date, SPEVIGO® (spesolimab) has been approved by regulatory authorities in almost 40 countries including the US, Japan, Mainland China, and the European Union to treat GPP flares in adults.

It recently received Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) and the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) as an investigational treatment for the prevention of GPP flares. Spesolimab is also under investigation for the treatment of other IL-36 mediated skin diseases.

About the Effisayil^TM clinical trial program
The Effisayil™ clinical trial program evaluated the largest and broadest population of GPP patients in trials of a therapy specifically targeting the IL-36 pathway for GPP:

• Effisayil™ 1: A Phase II study that demonstrated treatment with a single intravenous dose of spesolimab significantly improved signs and symptoms of generalized pustular psoriasis (GPP) in patients experiencing a flare. These results supported the approval of spesolimab (SPEVIGO®) as the first specific treatment for GPP flares in adults in major markets
• Effisayil™ 2: A Phase IIb study that showed spesolimab significantly reduced the risk of GPP flares by 84% over 48 weeks compared to placebo. In the trial with 123 patients, no flares were observed after Week 4 of spesolimab treatment in the high dose group (n=30).
• Effisayil™ ON: An open-label extension study to evaluate the long-term safety and efficacy of spesolimab in patients with GPP, who have completed previous spesolimab trials

About generalized pustular psoriasis (GPP)
GPP is a rare, chronic, often unpredictable and potentially life-threatening systemic neutrophilic skin disease, which is genetically and 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 lead to hospitalization and become life-threatening due to complications such as sepsis and multisystem organ failure. This systemic disease has a substantial quality of life impact for patients and places an increased burden on healthcare systems. While epidemiological data vary between geographical regions, prevalence estimates vary between 0.18 and 46 per 100,000 with more women affected than men. There is a high unmet need for treatments with a favorable safety profile that can prevent the occurrence of GPP flares.

About 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.us.

What is SPEVIGO?

SPEVIGO is a prescription medicine used to treat generalized pustular psoriasis (GPP) flares in adults. It is not known if SPEVIGO is safe and effective in children.

Important Safety Information

Do not receive SPEVIGO if you have had a severe or life-threatening allergic reaction to spesolimab-sbzo or any of the ingredients in SPEVIGO.

What is the most important information I should know about SPEVIGO?

SPEVIGO may cause serious side effects, including:

• Infections. SPEVIGO may lower the ability of your immune system to fight infections and may increase your risk of infections. Your healthcare provider should check you for infections and tuberculosis (TB) before starting treatment with SPEVIGO and may treat you for TB before you begin treatment with SPEVIGO if you have a history of TB or have active TB. Your healthcare provider should watch you closely for signs and symptoms of TB after treatment with SPEVIGO. Tell your healthcare provider right away if you have an infection or have symptoms of an infection, including:
  • fevers, chills, or sweats
  • muscle aches
  • cough
  • shortness of breath
  • blood in your phlegm (mucus)
  • burning when you urinate
  • urinating more often than normal
• Allergic reactions and infusion-related reactions. Serious allergic reactions may happen during or after your infusion of SPEVIGO. If you have a serious allergic reaction, your healthcare provider will stop treatment with SPEVIGO. If you have an infusion-related reaction, your healthcare provider will stop your SPEVIGO infusion and treat your symptoms and may restart SPEVIGO at a slower infusion rate. Tell your healthcare provider or get emergency medical help right away if you get any of the following symptoms during or after your infusion of SPEVIGO:
  • feeling faint, dizzy, or lightheaded
  • swelling of your face, eyelids, lips, mouth, tongue, or throat
  • trouble breathing or throat tightness
  • fever
  • mouth sores
  • chest tightness
  • hives or skin rash that is different than the rash from generalized pustular psoriasis (GPP)
  • itching
  • swollen lymph nodes

Before you receive SPEVIGO, tell your healthcare provider about all of your medical conditions, including if you:

• have an infection that does not go away or that keeps coming back.
• have TB or have been in close contact with someone with TB.
• have recently received or are scheduled to receive an immunization (vaccine). You should not receive live vaccines after treatment with SPEVIGO.
• are pregnant or plan to become pregnant. It is not known if SPEVIGO can harm your unborn baby.
• are breastfeeding or plan to breastfeed. It is not known if SPEVIGO passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby during treatment with SPEVIGO.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What are the most common side effects of SPEVIGO:

• feeling tired or weak
• nausea and vomiting
• headache
• itching or itchy bumps
• a collection of blood under the skin at the infusion site or bruising
• urinary tract infection

These are not all of the possible side effects of SPEVIGO. Call your doctor for medical advice about side effects.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1‑800‑FDA‑1088.

For more information, please see Prescribing Information and Medication Guide.

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