Recently published results showed that spesolimab significantly improved the signs and symptoms of flare-ups in rare and life-threatening skin diseases, generalized pustular psoriasis
RIDGEFIELD, Connecticut., 23 December 2021 / PRNewswire / – Boehringer Ingelheim today announced the publication in The New England Journal of Medicine new data from the pivotal Phase II Effisayil ™ 1 trial, which showed that spesolimab, a first-class investigational treatment, significantly improved the signs and symptoms of generalized pustular psoriasis (GPP) in patients with relapse .
PPG is a rare and life-threatening neutrophilic skin disease distinct from plaque psoriasis. It is characterized by episodes of generalized eruptions of painful and sterile pustules (blisters of non-infectious pus). There is a great unmet need for treatments that can quickly and completely resolve the signs and symptoms of PPM flare-ups. Flare-ups greatly affect a person’s quality of life and can lead to hospitalization with serious complications, including heart failure, kidney failure, sepsis, and death.
In the 12-week trial, 53 patients with a flare-up of GPP were treated with a single intravenous dose of spesolimab or placebo. Most of the patients at the start of the trial had high or very high pustule density and impaired quality of life. The results after one week showed that:
- 54% of patients treated with spesolimab had no visible pustules, compared to 6% of those treated with placebo
- 43% of patients treated with spesolimab had fair / almost fair 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.
In some patients, pustular and dermal clearance were maintained throughout the study. This clearance was accompanied by clinically significant improvements in quality of life and in 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 experienced drug reactions with eosinophilia and systemic symptoms.
“With the absence of approved treatments in the United States for flare-ups of GPP, there is a significant unmet need for people with this distressing and painful skin condition, which often requires emergency care,” said Marc Lebwohl, MD, Principal Investigator and Publication Author, and Dean of 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 signs and symptoms of a GPP flare after just a week, with effects seen until the end of the study.”
The United States Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) and granted priority review to spesolimab for the treatment of GPP flare-ups. The FDA has granted orphan drug designation to spesolimab for the treatment of GPP and breakthrough therapy designation to spesolimab for the treatment of outbreaks of GPP in adults.
“At Boehringer Ingelheim, we are committed to finding transformative therapies to help advance treatment for those in urgent need,” said Matt frankel, MD, Vice President, Clinical Development and Medical Affairs, Specialty Care, Boehringer Ingelheim. “The results indicate that spesolimab can have a significant and positive impact on the well-being of patients with a flare-up of GPP.”
The spesolimab clinical program includes two other trials currently underway. The Effisayil ™ 2 study will evaluate the safety and efficacy of spesolimab for the treatment of PPM, including prevention of relapses 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.
Spesolimab is a novel humanized selective antibody that blocks activation of the interleukin-36 receptor (IL-36R), a signaling pathway within the immune system involved in the pathogenesis of several autoimmune diseases, including GPP . Spesolimab is also being studied for the prevention of GPP flare-ups 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 in patients with an outbreak of GPP (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 sub-score of 0 (no visible pustules) at week 1. The key secondary endpoint was a GPPGA score of 0/1 (light / almost light skin) at week 1.
After one week, 54% of patients (19 of 35) treated with spesolimab had no visible pustules (GPPGA score of 0), compared with 6% of patients (1 of 18) treated with placebo (P
About generalized pustular psoriasis (PPP)
GPP is a rare, heterogeneous, and life-threatening neutrophilic skin disease that is clinically distinct from plaque psoriasis. GPP is caused by the buildup of neutrophils (a type of white blood cell) in the skin, resulting in painful, sterile pustules all over the body. The clinical course varies, with some patients having recurrent disease with recurrent flares, and others having persistent disease with intermittent flares. Although the severity of GPP flares can vary, if left untreated they can be life threatening due to complications such as sepsis and multiple organ failure. This chronic systemic disease has a substantial impact on the quality of life of patients and the burden of health care. GPP has a varied prevalence in different geographic regions and more women are affected than men.
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