ISU Abxis, an orphan drug developer, said Wednesday that its U.S. partner, Catalyst Biosciences, has secured encouraging drug efficacy and safety data on their new hemophilia treatment, DalcA (Dalcinonacog Alfa), during phase 2b clinical trials.
The U.S. company released the results at the 13th European Association for Haemophilia and Allied Disorders (EAHAD) in The Hague, Netherlands.
DalcA is a second-generation subcutaneous injection type Factor IX (FIX), the blood clotting factor nine produced in the liver, the deficiency of which causes hemophilia. ISU Abxis conducted phase 1 trials.
According to the research, blood FIX concentration remained above the target level of 12 percent when DalcA was injected once a day for 28 days.
After 14 days of the administration, FIX levels could be maintained at a maximum of 27 percent without bleeding. The result proved that it is effective bleeding prophylaxis and confirmed the possibility to reduce the doses.
The injection also did not form an anti-drug antibody (ADA) with no significant side effects reported. Three subjects only had mild injection site reactions and recovered without aftereffects.
The target of the open-label clinical trial phase 2b is to evaluate whether DalcA can make FIX activity of six severe hemophilia B patients remain above 12 percent. The researchers monitor pharmacokinetics, safety, drug tolerance, and ADA of DalcA on the subjects who were subcutaneously administered for 28 days after one intravenous injection.
"The results of the clinical phase 2b reported at the conference showed the potential for DalcA to change the treatment paradigm for hemophilia B patients completely," Catalyst Biosciences CEO Nassim Usman said. “We expect the final result will come out within the second quarter of this year.”