China-based rare disease firm CANbridge Pharmaceuticals Inc. (HKG: 1228) has announced receiving Orphan Drug Designation (ODD) status for its pipeline drug candidate CAN106, a long-acting monoclonal antibody (mAb) targeting C5 of the complement system, for use in myasthenia gravis (MG). Myasthenia gravis is a rare and chronic autoimmune disease caused by the dysfunction of nerve muscle junction transmission.
CAN106 Profile
CAN106 prevents the formation of the membrane attack complex (MAC) by binding and neutralizing C5, a complement system protein, which causes cell lysis (destruction) and other conditions associated with paroxysmal nocturnal hemoglobinuria (PNH)-related symptoms. When the C5 protein is cleaved into C5a and C5b, the MAC is activated, and the specific binding of CAN106 to C5 can block this process. CAN106 exhibits a favorable pharmacokinetic/pharmacodynamic (PK/PD) profile, safety, and tolerability, and can effectively inhibit certain complement-mediated diseases.
Partnerships and Development
CANbridge struck partnerships with Wuxi Biologics and Privus Biologics in 2019 and 2020, respectively, to develop rare disease drugs. The company holds exclusive development and commercialization rights to CAN106 globally. CAN106 initiated a Phase Ib/II study in PNH in China in March 2022, marking a significant step in its clinical development.
Future Implications
The Orphan Drug Designation for CAN106 underscores its potential as a novel treatment for myasthenia gravis, a rare and debilitating disease. This designation is expected to facilitate the drug’s development and regulatory approval process, positioning CANbridge to address significant unmet medical needs in the rare disease space.-Fineline Info & Tech
