The Center for Drug Evaluation (CDE) website has indicated that China-based Ascentage Pharma’s (HKG: 6855) lisaftoclax (APG-2575) and AceLink Therapeutics(Suzhou)’s AL01211 are on track to receive priority review and breakthrough therapy designation (BTD) statuses respectively from the US Food and Drug Administration (FDA). Lisaftoclax, an oral BCL-2 inhibitor, is indicated for the treatment of refractory or recurrent (R/R) chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL), while AL01211, a glucosylceramide synthase (GCS) inhibitor, is aimed at treating Fabry disease.
Clinical Data and Drug Profiles
Existing clinical data has demonstrated that APG-2575 can achieve a 68.3% objective remission rate in patients with R/R CLL/SLL, along with good tolerability. The drug is also being evaluated in combination with BTK inhibitors and other drugs for indications such as Waldenström’s macroglobulinemia (WM), acute myeloid leukemia, and multiple myeloma (MM). In China, the commercially available BCL-2 inhibitor is AbbVie’s venetoclax .
AL01211’s Mechanism and Impact on Fabry Disease
AL01211, a GCS inhibitor, can block the enzymatic reaction between ceramide and glucose, reducing the production of GL3. Fabry disease, a rare genetic metabolic disease caused by mutations in the GLA gene linked to the X chromosome, results in reduced or deficient alpha galactosidase A (alpha GalA) activity, leading to the accumulation of spherical tricarboxylic acid ceramide (GL3) and organic lesions.-Fineline Info & Tech
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