Tovesimig (ABL001), a bispecific antibody drug being developed by Compass Therapeutics, a global partner of ABL Bio (298380.KQ), has received orphan drug designation from the U.S. Food and Drug Administration (FDA), accelerating its path toward commercialization. If the drug is commercialized, ABL Bio will receive milestone payments and sales royalties.
ABL Bio said Thursday that Compass Therapeutics' bile duct cancer (cholangiocarcinoma) treatment tovesimig received orphan drug designation from the FDA. The orphan drug designation program was established to encourage development of treatments for rare diseases affecting fewer than 200,000 patients where drug development options are limited.
Orphan drug designation grants market exclusivity in the United States for a defined period, typically seven years. It also provides benefits including tax credits, partial exemption from FDA review fees, clinical trial grants, and regulatory development support.
Tovesimig is a bispecific antibody developed by ABL Bio and out-licensed to Compass Therapeutics for global rights in 2018. It works by simultaneously blocking the DLL4 and VEGF-A signaling pathways, which play critical roles in new blood vessel formation and tumor angiogenesis, thereby inhibiting tumor cell growth. According to ABL Bio, tovesimig demonstrated potent anti-cancer efficacy in both preclinical and clinical studies.
Compass Therapeutics is conducting a Phase 2/3 clinical trial combining tovesimig with the chemotherapy agent paclitaxel in second-line bile duct cancer patients. Data on overall survival (OS), progression-free survival (PFS), and other endpoints — the most critical metrics in the FDA's new drug approval review — are expected to be released this month.
"Compass Therapeutics received Fast Track designation from the FDA for tovesimig in April 2024 and has been leveraging the benefits of the program in its clinical development," ABL Bio CEO Lee Sang-hoon said. "We expect this orphan drug designation, following the Fast Track designation, to have a positive impact on the approval process."