– Independent data monitoring committee recommends pausing study enrollment due to lack of efficacy in pre-specified interim analysis –
– Company will conduct further analysis of trial data to determine next steps –
– No new safety findings were observed for ULTOMIRIS use in COVID-19 –
BOSTON, MA, USA I January 13, 2021 IAlexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the decision to pause further enrollment in the global Phase 3 study of ULTOMIRIS® (ravulizumab-cwvz) in adults with severe COVID-19 requiring mechanical ventilation. This decision is based on the recommendation of an independent data monitoring committee (IDMC), following their review of data from a pre-specified interim analysis. The IDMC recommended that additional enrollment be paused, pending further analysis of the data, due to lack of efficacy when ULTOMIRIS was added to best supportive care, compared to best supportive care alone. There were no new safety findings observed. The study will continue for patients already enrolled, including completion of all study visits and planned ULTOMIRIS dosing according to the study protocol.
“We would like to thank the patients and their families, as well as investigators and healthcare professionals, who were essential to this study. We greatly value their contributions to help investigate potential ways to address this devastating pandemic,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “While initial anecdotal reports from compassionate use cases were promising, these results demonstrate the importance of conducting controlled clinical trials to fully evaluate the potential of new treatment approaches and generate the necessary evidence to make informed decisions. We are disappointed in this initial outcome, but plan to further analyze the data to identify potential subgroups who may benefit and to determine next steps. In addition, we remain fully committed to our efforts to serve the rare disease community and to continuing to provide ULTOMIRIS to the patients who currently rely on it.”
The IDMC’s recommendation was based on a pre-planned interim analysis of the primary endpoint – survival at Day 29 – once 122 patients completed the 29-day primary evaluation period. No secondary endpoints were analyzed as part of the interim analysis.
In the UK, the TACTIC-R platform study led by Cambridge University Hospitals NHS Foundation Trust, which includes an ULTOMIRIS cohort, is evaluating the potential of earlier immune modulatory treatment (hospitalized patients not requiring mechanical ventilation) in preventing progression of the virus, including reducing the need for ICU admission and ventilation. This independent study remains ongoing.
About ULTOMIRIS® (ravulizumab-cwvz)
ULTOMIRIS® (ravulizumab–cwvz) is the first and only long-acting C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. ULTOMIRIS is administered intravenously every eight weeks or, for pediatric patients less than 20 kg, every four weeks, following a loading dose. ULTOMIRIS is approved in the United States (U.S.), European Union (EU) and Japan as a treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH). It is also approved in the U.S. and Japan for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients, as well as in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS.
INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS® (ravulizumab-cwvz)
INDICATIONS
What is ULTOMIRIS?
ULTOMIRIS is a prescription medicine used to treat:
- adults with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).
- adults and children 1 month of age and older with a disease called atypical Hemolytic Uremic Syndrome (aHUS). ULTOMIRIS is not used in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
It is not known if ULTOMIRIS is safe and effective in children with PNH.
It is not known if ULTOMIRIS is safe and effective in children younger than 1 month of age.
Please see the accompanying full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections/sepsis.
About Alexion
Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.
SOURCE: Alexion Pharmaceuticals
Post Views: 608
– Independent data monitoring committee recommends pausing study enrollment due to lack of efficacy in pre-specified interim analysis –
– Company will conduct further analysis of trial data to determine next steps –
– No new safety findings were observed for ULTOMIRIS use in COVID-19 –
BOSTON, MA, USA I January 13, 2021 IAlexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the decision to pause further enrollment in the global Phase 3 study of ULTOMIRIS® (ravulizumab-cwvz) in adults with severe COVID-19 requiring mechanical ventilation. This decision is based on the recommendation of an independent data monitoring committee (IDMC), following their review of data from a pre-specified interim analysis. The IDMC recommended that additional enrollment be paused, pending further analysis of the data, due to lack of efficacy when ULTOMIRIS was added to best supportive care, compared to best supportive care alone. There were no new safety findings observed. The study will continue for patients already enrolled, including completion of all study visits and planned ULTOMIRIS dosing according to the study protocol.
“We would like to thank the patients and their families, as well as investigators and healthcare professionals, who were essential to this study. We greatly value their contributions to help investigate potential ways to address this devastating pandemic,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “While initial anecdotal reports from compassionate use cases were promising, these results demonstrate the importance of conducting controlled clinical trials to fully evaluate the potential of new treatment approaches and generate the necessary evidence to make informed decisions. We are disappointed in this initial outcome, but plan to further analyze the data to identify potential subgroups who may benefit and to determine next steps. In addition, we remain fully committed to our efforts to serve the rare disease community and to continuing to provide ULTOMIRIS to the patients who currently rely on it.”
The IDMC’s recommendation was based on a pre-planned interim analysis of the primary endpoint – survival at Day 29 – once 122 patients completed the 29-day primary evaluation period. No secondary endpoints were analyzed as part of the interim analysis.
In the UK, the TACTIC-R platform study led by Cambridge University Hospitals NHS Foundation Trust, which includes an ULTOMIRIS cohort, is evaluating the potential of earlier immune modulatory treatment (hospitalized patients not requiring mechanical ventilation) in preventing progression of the virus, including reducing the need for ICU admission and ventilation. This independent study remains ongoing.
About ULTOMIRIS® (ravulizumab-cwvz)
ULTOMIRIS® (ravulizumab–cwvz) is the first and only long-acting C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. ULTOMIRIS is administered intravenously every eight weeks or, for pediatric patients less than 20 kg, every four weeks, following a loading dose. ULTOMIRIS is approved in the United States (U.S.), European Union (EU) and Japan as a treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH). It is also approved in the U.S. and Japan for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients, as well as in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS.
INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS® (ravulizumab-cwvz)
INDICATIONS
What is ULTOMIRIS?
ULTOMIRIS is a prescription medicine used to treat:
- adults with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).
- adults and children 1 month of age and older with a disease called atypical Hemolytic Uremic Syndrome (aHUS). ULTOMIRIS is not used in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
It is not known if ULTOMIRIS is safe and effective in children with PNH.
It is not known if ULTOMIRIS is safe and effective in children younger than 1 month of age.
Please see the accompanying full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious and life-threatening meningococcal infections/sepsis.
About Alexion
Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.
SOURCE: Alexion Pharmaceuticals
Post Views: 608
