CANbridge, targeting gaps in AstraZeneca's rare disease coverage, posts early-phase data on C5 inhibitor

CANbridge is coming for the anti-C5 market. With access to Soliris and Ultomiris limited in parts of the world, CANbridge has taken a rival C5 inhibitor through a phase 1 study, teeing it up to start an early test of the efficacy of the molecule.

Alexion won approval for Soliris in China in 2018, shortly after the regulator included the drug on a list of high-priority products approved overseas that it would authorize on the strength of foreign clinical data. But China is still without a long-acting anti-C5 therapy, with AstraZeneca’s Alexion yet to win approval for Ultomiris in the country, and other parts of the world lack access to the notoriously expensive Soliris.

CANbridge sees the limited market access to existing C5 inhibitors as an opportunity. Having worked with WuXi Biologics to develop CAN106, the Chinese biotech is now pushing the asset deeper into the clinic to capitalize on the gaps in the market.

CAN106 is moving into a Chinese phase 1b/2 clinical trial in paroxysmal nocturnal hemoglobinuria (PNH), a rare disease characterized by the destruction of red blood cells, on the strength of data captured on 31 healthy volunteers. The single ascending dose study provided early clinical evidence that CAN106 is safe, tolerable and reduces free C5, a protein required for activation of the terminal complement pathway.

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At the highest two doses, 8 and 12 mg/kg, all subjects experienced a 99% or greater reduction in free C5. CANbridge also found 90% or greater inhibition of an assay that measures the activity of the classical and terminal complement pathways. 

“The results of this phase 1 compare well with other anti-C5 therapies and support the further clinical development of CAN106 in patients with PNH,” Gerry Cox, M.D., Ph.D., chief development strategist and acting chief medical officer at CANbridge, said in a statement. “CAN106 appears to have a favorable safety profile, high potency, and a long half-life supporting a potential extended dosing interval in PNH patients.” 

Chinese authorities have signed off on the phase 1b/2 PNH study.