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News on March 18, 2021 /bioon.
com" target="_blank">/ --Apellis Pharma is a leader in the development of C3 targeted therapies and is committed to developing the first and best-in-class therapies through groundbreaking targeted C3 methods for the treatment of uncontrolled or excessive activation of the complement cascade Driven a wide range of diseases, including diseases in the fields of hematology, ophthalmology and nephrology.
bioon.com" target="_blank">/ --Apellis Pharma is a leader in the development of C3 targeted therapies and is committed to developing the first and best-in-class therapies through groundbreaking targeted C3 methods for the treatment of uncontrolled or excessive activation of the complement cascade Driven a wide range of diseases, including diseases in the fields of hematology, ophthalmology and nephrology.
com" target="_blank">
Recently, Apellis and Swedish Orphan Biovitrum AB (SOBI) jointly announced the evaluation of pegcetacoplan (APL-2, a C3 inhibitor) in the treatment of paroxysmal nocturnal hemoglobinuria (PNH) head-to-head Phase 3 PEGASUS study ( NCT03500549) results have been published in the international medical journal "New England Journal of Medicine" (NEJM).
The study was conducted in adult patients who received Soliris (eculizumab, a C5 inhibitor), the standard of care for PNH, but still had persistent bioon.
com/tags/%E8%B4%AB%E8%A1%80/">anemia .
The results showed that compared with Soliris for 16 weeks, pegcetacoplan showed superiority: the hemoglobin level was statistically significantly improved, and the key clinical results also showed improvement.
These data confirm the potential of pegcetacoplan to improve the standard of care for PNH and is expected to redefine the treatment of PNH.
bioon.The study was conducted in adult patients who received Soliris (eculizumab, a C5 inhibitor), the standard of care for PNH, but still had persistent bioon.
com/tags/%E8%B4%AB%E8%A1%80/">anemia .
The results showed that compared with Soliris for 16 weeks, pegcetacoplan showed superiority: the hemoglobin level was statistically significantly improved, and the key clinical results also showed improvement.
These data confirm the potential of pegcetacoplan to improve the standard of care for PNH and is expected to redefine the treatment of PNH.
com/tags/%E8%B4%AB%E8%A1%80/">anemia
Pegcetacoplan is a C3 inhibitor.
The New Drug Application (NDA) for the treatment of PNH is undergoing priority review by the US bioon.
com/fda/" target="_blank">FDA .
The target action date is May 14, 2021.
In addition, the marketing authorization application (MAA) of pegcetacoplan for the treatment of PNH is also under review by the European Medicines Agency (EMA).
bioon.The New Drug Application (NDA) for the treatment of PNH is undergoing priority review by the US bioon.
com/fda/" target="_blank">FDA .
The target action date is May 14, 2021.
In addition, the marketing authorization application (MAA) of pegcetacoplan for the treatment of PNH is also under review by the European Medicines Agency (EMA).
com/fda/" target="_blank">FDA
Soliris is a blockbuster C5 complement inhibitor of Alexion.
It has been approved to treat a variety of rare diseases and is the standard care therapy for the treatment of PNH.
In December 2020, bioon.
com/tags/%E9%98%BF%E6%96%AF%E5%88%A9%E5%BA%B7/">AstraZeneca acquired Alexion for US$39 billion and entered the field of rare diseases.
bioon.It has been approved to treat a variety of rare diseases and is the standard care therapy for the treatment of PNH.
In December 2020, bioon.
com/tags/%E9%98%BF%E6%96%AF%E5%88%A9%E5%BA%B7/">AstraZeneca acquired Alexion for US$39 billion and entered the field of rare diseases.
com/tags/%E9%98%BF%E6%96%AF%E5%88%A9%E5%BA%B7/">AstraZeneca acquired Alexion for US$39 billion and entered the field of rare diseases.
bioon.
com/tags/%E9%98%BF%E6%96%AF%E5%88%A9%E5%BA%B7/">AstraZeneca
Currently, PNH treatment still needs new treatment methods.
Because many PNH patients treated with C5 inhibitors are still anemic, resulting in moderate to severe fatigue, and some patients continue to require blood transfusions.
Data from the Phase 3 PEGASUS study underscore the potential of pegcetacoplan as a major advancement in PNH care.
If approved, pegcetacoplan has the potential to improve PNH care standards by providing more comprehensive disease control.
Because many PNH patients treated with C5 inhibitors are still anemic, resulting in moderate to severe fatigue, and some patients continue to require blood transfusions.
Data from the Phase 3 PEGASUS study underscore the potential of pegcetacoplan as a major advancement in PNH care.
If approved, pegcetacoplan has the potential to improve PNH care standards by providing more comprehensive disease control.
png" target="_blank">
png" target="_blank">PEGASUS is a multi-center, randomized, open-label, positive drug controlled, head-to-head phase 3 study conducted in 80 PNH adult patients to evaluate the efficacy and safety of pegcetacoplan relative to Soliris.
In this study, subjects must have been receiving Soliris treatment (stable treatment for at least 3 months) and have a hemoglobin level of <10.
5g/dL at the time of the screening visit.
During the 4-week lead-in period, the patient received pegcetacoplan at a dose of 1080 mg twice a week while receiving his current dose of Soliris.
During the 16-week randomized control period, patients were randomly assigned to receive 1080 mg of pegcetacoplan (twice a week) or their current dose of Soliris.
All subjects who completed the 16-week randomized control period (n=77) entered the open-label period and received pegcetacoplan treatment from week 17 to week 48.
In this study, subjects must have been receiving Soliris treatment (stable treatment for at least 3 months) and have a hemoglobin level of <10.
5g/dL at the time of the screening visit.
During the 4-week lead-in period, the patient received pegcetacoplan at a dose of 1080 mg twice a week while receiving his current dose of Soliris.
During the 16-week randomized control period, patients were randomly assigned to receive 1080 mg of pegcetacoplan (twice a week) or their current dose of Soliris.
All subjects who completed the 16-week randomized control period (n=77) entered the open-label period and received pegcetacoplan treatment from week 17 to week 48.
The results published on NEJM confirmed that pegcetacoplan reached the primary efficacy endpoint of the study: at week 16, the superiority of pegcetacoplan over Soliris was shown.
The corrected average hemoglobin level of the two treatment groups was 3.
84g/dL, which was statistically significant.
Significantly improved.
In addition, 85% of patients in the pegcetacoplan treatment group had no blood transfusion within 16 weeks, compared with 15% in the Soliris treatment group.
Through the chronic disease treatment function assessment (FACT)-fatigue score measurement, the key markers of the disease such as absolute reticulocyte count, lactate dehydrogenase (LDH) and fatigue have been significantly improved.
The corrected average hemoglobin level of the two treatment groups was 3.
84g/dL, which was statistically significant.
Significantly improved.
In addition, 85% of patients in the pegcetacoplan treatment group had no blood transfusion within 16 weeks, compared with 15% in the Soliris treatment group.
Through the chronic disease treatment function assessment (FACT)-fatigue score measurement, the key markers of the disease such as absolute reticulocyte count, lactate dehydrogenase (LDH) and fatigue have been significantly improved.
png" target="_blank">
png" target="_blank">In this study, the safety of pegcetacoplan and Soliris are comparable.
Serious adverse reactions (SAE) occurred in 7 (17%) of 41 patients in the pegcetacoplan group, and SAE occurred in 6 (15%) of 39 patients in the Soliris group.
There were no meningitis cases and death reports in the two treatment groups.
During the 16-week randomized controlled treatment period, the most common bioon.
com/course_info/series_11.
html">adverse reactions in the pegcetacoplan group and the Soliris group were injection site reactions (37% vs 3%), diarrhea (22% vs 3%), and breakthrough hemolysis (10% vs 23 %), headache (7% vs 23%) and fatigue (5% vs 15%).
bioon. Serious adverse reactions (SAE) occurred in 7 (17%) of 41 patients in the pegcetacoplan group, and SAE occurred in 6 (15%) of 39 patients in the Soliris group.
There were no meningitis cases and death reports in the two treatment groups.
During the 16-week randomized controlled treatment period, the most common bioon.
com/course_info/series_11.
html">adverse reactions in the pegcetacoplan group and the Soliris group were injection site reactions (37% vs 3%), diarrhea (22% vs 3%), and breakthrough hemolysis (10% vs 23 %), headache (7% vs 23%) and fatigue (5% vs 15%).
com/course_info/series_11.
html">Adverse reactions
The 48-week top-line results of the PEGASUS study were recently announced: it shows that the key markers of the disease continue to improve, and the safety is consistent with the previously reported data.
It is worth mentioning that pegcetacoplan is the first research therapy to show superior hemoglobin levels than Soliris, and up to 85% of patients treated with pegcetacoplan have no blood transfusion.
Most of the PNH patients currently receiving Soliris treatment suffer from persistent anemia.
The results of the PEGASUS study show that pegcetacoplan has the potential to become a new standard of care for patients with PNH.
Most of the PNH patients currently receiving Soliris treatment suffer from persistent anemia.
The results of the PEGASUS study show that pegcetacoplan has the potential to become a new standard of care for patients with PNH.
Pegcetacoplan is an investigative and targeted C3 inhibitor designed to regulate the excessive activation of complement, which is the cause of the occurrence and development of many serious diseases.
Pegcetacoplan is a synthetic cyclic peptide that binds to a polyethylene glycol polymer and specifically binds to C3 and C3b.
Currently, pegcetacoplan is being developed to treat a variety of diseases, including PNH, geographic atrophy (GA) and C3 glomerulopathy.
In the United States, the bioon.
com/fda/" target="_blank">FDA has granted pegcetacoplan fast track qualification for the treatment of PNH and GA.
bioon. Pegcetacoplan is a synthetic cyclic peptide that binds to a polyethylene glycol polymer and specifically binds to C3 and C3b.
Currently, pegcetacoplan is being developed to treat a variety of diseases, including PNH, geographic atrophy (GA) and C3 glomerulopathy.
In the United States, the bioon.
com/fda/" target="_blank">FDA has granted pegcetacoplan fast track qualification for the treatment of PNH and GA.
com/fda/" target="_blank">FDA
Soliris is a drug sold by Alexion.
This is a pioneering complement inhibitor that works by inhibiting the C5 protein in the terminal part of the complement cascade.
The complement cascade is part of the immune system, and its uncontrolled activation plays an important role in a variety of serious rare diseases and super rare diseases.
Soliris was first approved for marketing in 2007, and has been approved for a variety of super rare diseases before: PNH, atypical hemolytic bioon.
com/tags/%E5%B0%BF%E6%AF%92%E7%97%87/">uremic syndrome (aHUS), anti-AchR antibody-positive systemic myasthenia gravis (gMG), anti-aquaporin-4 (AQP4) Antibody-positive neuromyelitis optica spectrum disorder (NMOSD).
bioon. This is a pioneering complement inhibitor that works by inhibiting the C5 protein in the terminal part of the complement cascade.
The complement cascade is part of the immune system, and its uncontrolled activation plays an important role in a variety of serious rare diseases and super rare diseases.
Soliris was first approved for marketing in 2007, and has been approved for a variety of super rare diseases before: PNH, atypical hemolytic bioon.
com/tags/%E5%B0%BF%E6%AF%92%E7%97%87/">uremic syndrome (aHUS), anti-AchR antibody-positive systemic myasthenia gravis (gMG), anti-aquaporin-4 (AQP4) Antibody-positive neuromyelitis optica spectrum disorder (NMOSD).
com/tags/%E5%B0%BF%E6%AF%92%E7%97%87/">Uremia
Soliris is one of the best-selling orphanages in the world.
Currently, Alexion is also developing an upgraded version of Soliris, Ultramiris, which was approved by the FDA for PNH indications in December 2018.
In October 2019, Ultomiris was approved by the bioon.
com/fda/" target="_blank">FDA for a new indication: the treatment of children and adults with aHUS .
Ultomiris is the first and only long-acting C5 complement inhibitor to be administered every 8 weeks.
In a phase III clinical study for the treatment of PNH, Ultomiris is infused every 2 months (8 weeks) with Soliris every 2 The weekly infusion achieved non-inferiority in all 11 endpoints.
()
bioon. Currently, Alexion is also developing an upgraded version of Soliris, Ultramiris, which was approved by the FDA for PNH indications in December 2018.
In October 2019, Ultomiris was approved by the bioon.
com/fda/" target="_blank">FDA for a new indication: the treatment of children and adults with aHUS .
Ultomiris is the first and only long-acting C5 complement inhibitor to be administered every 8 weeks.
In a phase III clinical study for the treatment of PNH, Ultomiris is infused every 2 months (8 weeks) with Soliris every 2 The weekly infusion achieved non-inferiority in all 11 endpoints.
()
com/fda/" target="_blank">FDA
Original source: The NEJM publishes phase 3 PEGASUS study results comparing pegcetacoplan to eculizumab for PNH
