Rare blood disease new drug phase 3 clinical data positive
March 16, 2018 Source: WuXi PharmaTech
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];Alexion Pharmaceuticals, Inc. today announced that the long-acting C5 complement inhibitor ALXN1210 has been shown in a phase III clinical trial in patients with paroxysmal nocturnal hemoglobinuria (PNH) compared to its company's Soliris (eculizumab). Inferiority. In addition, all six endpoints of the study supported ALXN1210. There is no significant difference in security between ALXN1210 and Soliris.
PNH is a chronic, progressive, debilitating and potentially life-threatening ultra-rare blood disease that can occur in all races, backgrounds and ages of men and women without warning, with an average age of onset of 30 years. In the early days. PNH is usually not recognized and delays range from 1 year to over 10 years. In PNH patients, chronic, uncontrolled activation of the complement cascade of the body's immune system leads to hemolysis, anemia, fatigue, dark urine and shortness of breath. The most devastating consequence of chronic hemolysis is the formation of blood clots that can damage vital organs and cause premature death. One-third of patients with PNH are expected to survive five years after diagnosis. PNH is more common in patients with aplastic anemia (AA) and myelodysplastic syndrome (MDS). PNH may be a potential cause in some patients with unexplained thrombosis.
ALXN1210 is an innovative long-acting C5 inhibitor discovered and developed by Alexion that inhibits the C5 protein in the human immune system complement cascade. When the system is activated in an uncontrolled manner, it causes severe ultra-rare blood diseases such as PNH, atypical hemolytic uremic syndrome (aHUS) and anti-acetylcholine receptor (AchR) antibody-positive myasthenia gravis (MG). ALXN1210 has obtained Orphan Drug Qualification (ODD) for the treatment of PNH patients in the United States and the European Union, as well as subcutaneous treatment in US aHUS patients.
â–² ALXN1210 mechanism of action (Source: Alexion official website)
This randomized, open-label, active-controlled, multinational and multicenter phase 3 study evaluated the efficacy and safety of ALXN1210 and Soliris in adults with PNH over 18 years of age. The study enrolled 246 adult patients diagnosed with PNH who had never received a complement inhibitor treatment with a lactate dehydrogenase (LDH) level exceeding 1.5 times the upper limit of normal (ULN) at screening and One or more PNH-related signs or symptoms such as fatigue, hemoglobinuria, abdominal pain, dyspnea, and anemia (hemoglobin <10 g / dL) occurred within 3 months of screening. Patients in the ALXN1210 group received a single dose of ALXN1210, followed by regular maintenance of the baseline body weight every 8 weeks. Patients in the Soliris group received 4 induction doses per week and then scheduled maintenance doses every 2 weeks. Both groups underwent a 26-week treatment. The study was designed to assess the non-inferiority of ALXN1210 compared to Soliris.
Studies have shown that non-inferiority is achieved at two common primary endpoints to avoid blood transfusions and maintain normal LDH levels, and is achieved at all four key secondary endpoints, which are changes in LDH levels compared to baseline. Percentage, Chronic Disease Therapy Functional Assessment (FACIT) to assess changes in patient quality of life relative to baseline, and the proportion of patients with stable hemolysis and hemoglobin levels. At the first superiority test end point breakthrough hemolysis, although ALXN1210 did not gain an advantage, a numerical trend favoring ALXN1210 was observed (Soliris: 4.0% [0.6%, 7.4%], ALXN1210: 10.7% [ 5.2%, 16.3%]), p value is 0.074. The study also confirmed that ALXN1210 can persist and completely (> 99%) inhibit complement C5 protein throughout the 8-week dosing interval. During the first to sixth months, treatment with ALXN1210 reduced the mean LDH level to near the upper limit of normal (1.0-1.1 times ULN).
â–² ALXN1210 is located in 2 middle and late clinical cases (Source: Alexion official website)
“We are very pleased with these positive data from ALXN1210, which is the first and only head-to-head comparison study with Soliris that reinforces our ambition to develop ALXN1210 as a new standard of care for PNH patients. Our hypothesis is that immediate, complete and sustained C5 inhibition is critical for patients with potentially life-threatening illnesses,†said Dr. John Orloff, executive vice president and head of research at Alexion. “Soliris sets a high standard of care. We achieved non-inferiority at the common primary endpoint and all key secondary endpoints, and seeing the numerical results supporting ALXN1210, we reached a very high level in such a rigorous study. We look forward to the United States in the second half of 2018, The EU and Japan's PNH submitted a regulatory review of the ALXN1210."
Professor Jong Wook Lee, Professor of Internal Medicine at St. Mary's Hospital, Catholic University of Korea, and Dr. Jong Wook Lee, an investigator at ALXN1210 Research, said: "This new treatment program can avoid blood transfusions and is given 6 times a year, compared to the current annual infusion of 26 times. It can quickly maintain LDH levels and is of great help to PNH patients."
We expect that the regulatory review of this new drug for rare diseases will proceed smoothly and bring this important therapy to patients as soon as possible.
Reference materials:
[1] Alexion's Prospects Rise with Positive Phase III Trial Data
[2] Alexion Announces Positive Top-Line Results Showing Successful Phase 3 Clinical Study Of ALXN1210 In Complement Inhibitor Treatment-Naïve Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
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