AstraZeneca's treatment for fatal myocardial disease has been approved for clinical trials in China.
Astrazeneca's new drug ALXN2220 injection has obtained clinical trial implied consent in China for the treatment of diagnosed transthyretin amyloid cardiomyopathy in adult patients. ALXN2220 is a monoclonal antibody against transthyretin amyloid protein, with the potential to treat late-stage ATTR-CM patients. Alexion, a subsidiary of Astrazeneca, holds the global exclusive licensing rights for this product. The safety and efficacy of the product have been validated in preclinical models and Phase 1 clinical studies. Currently, Alexion is conducting an international multicenter Phase 3 clinical trial to evaluate the efficacy and safety of ALXN2220.
Zhitong App learned recently that the China National Medical Products Administration's Drug Evaluation Center website announced that Astrazeneca's Class 1 new drug ALXN2220 injection has obtained implied approval for clinical trials, intended for the treatment of clinically diagnosed wild-type or hereditary transthyretin amyloidosis in adult patients.
ALXN2220 is a monoclonal antibody against transthyretin amyloid (ATTR), serving as an ATTR "depletor." Alexion, a subsidiary of Astrazeneca focusing on rare diseases, previously secured a over $700 million collaboration to gain global exclusive rights for the development, production, and commercialization of this product. This product specifically targets misfolded transthyretin protein, clearing amyloid fibril deposits in the heart, addressing the pathological cause of ATTR-CM, with the potential to treat late-stage ATTR-CM patients. Preclinical models have shown that this product can induce the clearance of pathological ATTR.
In June 2023, the Phase 1 clinical study of ALXN2220 for treating ATTR-CM was published in the New England Journal of Medicine. The results indicated that the safety of ALXN2220 remained good even at the highest test dose, with no significant dose-limiting toxicity or drug-related severe adverse reactions observed. The pharmacokinetic characteristics of this product are consistent with IgG antibodies, and no drug-resistant antibodies were detected. At a dose of at least 10mg/kg, the deposition of cardiac amyloid protein in patients (detected by scintigraphy or cardiac MRI) significantly decreased within 12 months. Biomarkers for measuring cardiac pressure and myocardial cell death, NT-proBNP and troponin T levels, also decreased.
According to the ClinicalTrials website, Alexion has registered an international multicenter (including China) Phase 3 clinical study (DepleTTR-CM study) to evaluate the efficacy and safety of ALXN2220 compared to a placebo in treating adult patients with ATTR-CM. This study plans to enroll 1000 participants, with an expected completion date in June 2028.
