Shanghai-based biotech Abbisko Therapeutics Co., Ltd (HKG: 2256) has announced that it has received orphan drug designation (ODD) status from the US Food and Drug Administration (FDA) for its next-generation FGFR4 mutant inhibitor, ABSK012, intended for the treatment of soft-tissue sarcoma.
Preclinical Efficacy of ABSK012 Against FGFR4 Mutants
Preclinical studies have demonstrated that ABSK012 exhibits excellent activity against wild-type FGFR4 as well as clinically developed FGFR4 inhibitor-resistant mutants, both in vitro and in cellular models. Furthermore, the compound has shown significant in vivo efficacy in models driven by FGF19 and FGFR4 mutations, reinforcing its potential as a therapeutic option for patients with soft-tissue sarcoma.
Addressing the Need for New Therapies in Soft Tissue Sarcoma
Soft tissue sarcomas account for 7% of malignant tumors in children and 1% of malignant tumors in adults, with annual incidence rates of 2.91 per 100,000 in China and 3.4 per 100,000 in the US. The average five-year overall survival rate for patients with soft tissue sarcoma is 65%, but those with the rhabdomyosarcoma subtype face a significantly lower survival rate, highlighting the urgent need for new therapies. FGFR4 overexpression or mutations are prevalent in rhabdomyosarcoma, making ABSK012 a promising candidate for addressing this critical gap in treatment options.-Fineline Info & Tech
