AstraZeneca and Alexion Launch Phase III Clinical Trial of Ravulizumab in China for Treatment-Naïve Adult PNH Patients
AstraZeneca
Biopharmaceutical Manufacturer
Alexion
Developer of New Drugs for Rare Disease Treatment
On July 31, according to the Drug Clinical Trial Registration and Information Disclosure Platform, AstraZeneca and Alexion initiated a Phase III clinical trial in China. This trial is a single-arm, open-label, multi-center study designed to evaluate Relizumab (Ravulizumab) in treatment-naive adult patients with paroxysmal nocturnal hemoglobinuria (PNH) Efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity in patients.
Screenshot source: Drug Clinical Trial Registration and Information Disclosure Platform
Ravulizumab is a long-acting C5 monoclonal antibody developed by Alexion. Its unique design gives it a longer half-life than first-generation C5 complement inhibitors. When treating PNH patients, it only needs to be administered intravenously once every 8 weeks to effectively control the occurrence of hemolysis. Currently, Ravulizumab has been approved abroad for various autoimmune diseases, including PNH and atypical hemolytic uremic syndrome (aHUS), Myasthenia Gravis, and Neuromyelitis Optica.
In June 2021, AstraZeneca acquired Alexion and announced its official entry into the rare disease field. According to the currently published two-year follow-up data, during the treatment of PNH patients with Ravulizumab, the minimum concentration of serum Ravulizumab remained ≥175 μg/mL, and lactate dehydrogenase (LDH) The average change in level from baseline to Day 743 was +2.3%. Hemolytic episodes (BTH) The incidence rate was 3.9%, indicating that the treatment method maintained effective drug concentration and therapeutic effects during the long-term follow-up period.
In March this year, another Phase III clinical study result of Ravulizumab was announced. The data showed that among 13 pediatric PNH patients, the minimum serum concentration of Ravulizumab in treatment-naïve patients was 610.50 ± 201.53 μg/mL, and in pre-treated patients, it was 518.29 ± 109.67 μg/mL. The serum free C5 concentrations in the two groups were 0.061 ± 0.021 μg/mL and 0.061 ± 0.018 μg/mL, respectively. The study demonstrated that Ravulizumab maintained high serum concentrations in both groups and effectively reduced serum free C5 levels, proving its efficacy and safety in pediatric PNH patients.
Screenshot source: Insight Database official website
PNH is a disease caused by glycosylphosphatidylinositol class A at the level of pluripotent hematopoietic stem cells (PIGA) Rare diseases caused by somatic mutations in the genome. Although considered benign, the survival rate of patients still needs urgent improvement under traditional treatment methods.
In addition to Ralizumab, another C5 monoclonal antibody from AstraZeneca is Eculizumab (Originally developed by Alexion) As early as 2018, it was approved for marketing in China for the treatment of PNH, and subsequently, it also received approval for anti-acetylcholine receptor (AChR) Antibody-positive refractory generalized myasthenia gravis (gMG) and aHUS, two rare disease indications.
In February and April this year, the PNH treatment field welcomed the approval of two new drugs, respectively Roche's Crovalimab and Novartis' Iptacopan. In addition, an increasing number of pharmaceutical companies are also stepping up their efforts in the PNH treatment area. On May 28, 2024, Zai Lab and its partner Regeneron initiated a Phase III clinical trial for the combination therapy of C5 antibody Pozelimab and C5 siRNA therapy Cemdisiran in the treatment of PNH.
Chinese pharmaceutical companies are also actively engaged in the PNH treatment field. Currently, HRS-5965 from Hengrui Medicine, KP104 from Kexing Biopharma, LP-005 from Tianchen Bio, and MY008211A from Meiyue Bio have all entered Phase II clinical trials.
