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


- New clinical trial data published today in The New England Journal of Medicine showed that spesolimab, a novel IL-36 receptor antagonist, demonstrated rapid clearance of pustules in adult patients with generalized pustular psoriasis (GPP) experiencing a flare
- The study met the primary endpoint: 54% of patients had no visible pustules after a single dose of spesolimab, compared to 6% receiving placebo at week one
- FDA has already granted Priority Review, Breakthrough Therapy Designation, and Orphan Drug Designation for spesolimab


RIDGEFIELD, Conn., Dec. 23, 2021 /PRNewswire/ -- Boehringer Ingelheim announced today the publication in The New England Journal of Medicine of new data from the pivotal Phase II Effisayil™ 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.

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). There is a high unmet need for treatments that can rapidly and completely resolve the signs and symptoms of GPP flares. Flares greatly affect a person's quality of life and can lead to hospitalization with serious complications, including heart failure, renal failure, sepsis, and death.

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

After one week of treatment, spesolimab was associated with a higher number of infections and systemic drug reactions compared to placebo.

For some patients, pustular and skin clearance were maintained 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. 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. Serious adverse events were reported in 6% of patients treated with spesolimab. Two patients receiving spesolimab were reported to have drug reactions with eosinophilia and systemic symptoms.

"With no approved treatments in the U.S. for GPP flares, there is a significant unmet need for people with this distressing and painful skin condition, which often requires emergency care," said Mark Lebwohl, M.D., 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 clear the skin of the signs and symptoms of a GPP flare after only one week, with effects observed through the end of the study."

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.

"At Boehringer Ingelheim, we are committed to finding transformative therapies to help advance treatment for people who urgently need them," said Matt Frankel, M.D., Vice President, Clinical Development and Medical Affairs, Specialty Care, Boehringer Ingelheim. "The findings indicate that spesolimab may have a significant and positive impact on the wellbeing of patients experiencing a GPP flare."

The clinical program for spesolimab includes two other trials that are currently underway. The Effisayil™ 2 study will assess the safety and efficacy of spesolimab for the treatment of GPP, including flare prevention and symptom control over 48 weeks. Effisayil™ ON is a five-year, open-label extension study that will provide additional long-term efficacy and safety data for spesolimab in the treatment of GPP.

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 pathogeneses of several autoimmune diseases, including GPP. 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 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.

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).

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 healthcare burden. GPP has a varied prevalence across different geographical regions and more women are affected than men.

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