Chinese pharmaceutical company Akeso Inc (HK:9926) announced on Monday that its proprietary next-generation humanised IgG4 monoclonal antibody targeting CD47, ligufalimab (AK117), has been granted Orphan Drug Designation (ODD) by the US FDA for the treatment of acute myeloid leukaemia (AML).

Akeso says that it is actively advancing the international clinical development for ligufalimab, which is being evaluated in both haematologic malignancies and solid tumours. In addition to its application in AML, patient enrolment has been completed in a randomised, double-blind, multicentre Phase II study assessing ligufalimab combined with azacitidine in higher-risk myelodysplastic syndromes (HR-MDS).

Ligufalimab is also the first CD47 monoclonal antibody to enter registrational Phase III trials in solid tumours.

According to Akeso, preclinical studies have shown that ligufalimab, when combined with azacitidine or venetoclax, synergistically enhances the expression of eat me signals (such as calreticulin), leading to more efficient activation of phagocytic immune responses. This combination may thus offer a promising treatment option for AML patients ineligible for standard induction chemotherapy.

Clinical trials have demonstrated that ligufalimab combined with azacitidine shows a favorable safety profile and promising efficacy in first-line AML treatment. Building on these encouraging results, Akeso has launched a Phase II study to further investigate the safety and efficacy of ligufalimab in combination with venetoclax and azacitidine for first-line AML patients ineligible for intensive chemotherapy.