Catalyst Biosciences Inc (NASDAQ:CBIO) posted top-line report from its Phase 1/2 trial of subcutaneous prophylactic candidate CB 2679d/ISU304 being advanced for the cure of hemophilia B in a presentation at the 11th Annual Congress of The EAHAD in Madrid. Data from the trial demonstrated a continuous linear rise in Factor IX activity levels after daily administration of CB 2679d for six days.
Dr. John Pasi reported that these thrilling results showcase for the first time the viability of a subcutaneous FIX injection to offer notable protection from bleeding, even after merely six doses. Subsequently, this treatment option possesses the potential to regularize coagulation activity and dramatically enhance the quality of life of people affected by hemophilia B.
Dr. Howard Levy, the Chief Medical Officer of Catalyst, showcased data from the fifth cohort of the Phase 1/2 study of CB 2679d in people with severe hemophilia B. Following 6 days of once-daily SQ administration, CB 2679d enhanced FIX activity levels in five people who were each administrated daily with 140 IU per kg SQ, from extremely low levels following washout of previous treatment to a median FIX activity mark of 16%, that is into the mild hemophilia range of 5-40%, and is higher than a mark required to avert hemarthrosis.
The noted growth in FIX activity levels following the daily administration was linear, suggesting that continued SQ administration may attain high-mild hemophilia or even usual FIX clotting activity. Dr. Levy expressed that current IV therapies boasts FIX trough levels that can decline as low as 1-3% prior to repeat dosing. Daily SQ dosing of CB 2679d boasts the potential to lower the variability in FIX activity marks noted between IV doses and maintain people in the mild or normal hemophilia range. This trial exhibits that, even after merely 6 days of treatment, CB 2679d comes favorably to existing approved treatments for hemophilia B.