ABL Bio, a bispecific antibody specialist, announced on May 8 that its partner Compass Therapeutics has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for Tovecimig (CTX-009/ABL001), a treatment for cholangiocarcinoma.
The Orphan Drug Designation program is designed to encourage the development of treatments for rare diseases affecting fewer than 200,000 patients, where drug development is often limited.
This designation grants market exclusivity in the U.S. for a set period (typically seven years), along with benefits such as tax credits, partial FDA review fee waivers, clinical trial grants, and regulatory support.
Tovecimig, developed by ABL Bio and licensed to Compass Therapeutics, is a bispecific antibody that simultaneously blocks the Delta-like ligand 4 (DLL4) and Vascular Endothelial Growth Factor-A (VEGF-A) signaling pathways, which are crucial for new blood vessel formation and tumor vascularization.
Preclinical and clinical studies have shown that inhibiting both DLL4 and VEGF-A can significantly suppress tumor growth, resulting in potent anti-cancer effects.
Compass Therapeutics is currently conducting the Phase 2/3 COMPANION-002 trial, combining Tovecimig with the chemotherapy drug Paclitaxel for second-line cholangiocarcinoma patients. This month, they plan to release data on key indicators such as overall survival (OS) and progression-free survival (PFS), which are critical for FDA approval.
ABL Bio Chief Executive Officer (CEO) Lee Sang-hoon stated that Compass Therapeutics received Fast Track designation for Tovecimig from the FDA in April 2024 and has been leveraging its benefits in clinical development. They plan to discuss future development stages with the FDA based on COMPANION-002 data.
He added that it expects this Orphan Drug Designation, following the Fast Track designation, to positively impact Tovecimig’s approval process.
ABL Bio is developing various preclinical and clinical pipelines based on its Grabody bispecific antibody platform. Clinical projects for ten pipelines, including ABL301 (SAR446159), ABL001 (Tovecimig), ABL111 (Givastomig), ABL503 (Ragistomig), ABL105 (Nesfrotamig), ABL104 (YH32364), ABL103, ABL202 (CS5001/LCB71), ABL206 (NEOK001), and ABL209 (NEOK002), are underway in the U.S., China, Australia, and South Korea.
Sanofi will conduct follow-up clinical trials for ABL301 (SAR446159), which completed its U.S. Phase 1 trial. ABL001 (Tovecimig), in Phase 2/3 trials for cholangiocarcinoma, has received both Fast Track and Orphan Drug Designations from the FDA. ABL111 (Givastomig), co-developed with NovaBridge, has initiated a Phase 2 trial combining it with Nivolumab and chemotherapy. Additional Phase 1b data will be presented at a global conference later this year.
ABL Bio continues to research and develop several preclinical pipelines, including bispecific antibody drug conjugates (ADCs) and dual payload ADCs.
