Catalyst Biosciences, developers of protease therapeutics for serious hemostasis and anti-complement conditions, and CMC ICOS Biologics, Inc., which manufactures therapeutic proteins, have partnered for the manufacture of a next generation hemophelia drug.
Catalyst is developing CB 813d, a longer-acting Factor VIIa drug for hemophilia A and B inhibitor patients.
“We selected CMC Biologics as our manufacturing partner for CB 813d because of their demonstrated technical expertise in producing commercial coagulation factor proteins and the quality of their cGMP operations,” said Andrew Hetherington, Catalyst’s vice president of manufacturing operations in a press release. “The successful transfer and manufacture of CB 813d is critical to meeting our clinical timelines as we advance our lead candidate towards a pivotal study.”
Nassim Usman, president and chief executive officer of Catalyst, said CB 813d is being developed to potentially treat acute bleeding episodes and allow for prophylactic treatment.
“We remain on track to initiate a Phase 2/3 clinical pivotal trial of CB 813d, our most advanced hemophilia product candidate, in 2017,” Usman said.
Hemophilia is a rare genetic or acquired bleeding disorder, which is caused by a deficient protein needed for blood coagulation. Modifications in Factor VIII or Factor IX genes lead to the development of both class A and class B hemophilia.
About 20,000 people in the U.S., and 400,000 worldwide have hemophilia.
Patients often experience spontaneous and long periods of bleeding upon injury. Patients with severe hemophilia often suffer from spontaneous bleeding into muscles or joints which leads to permanent joint damages. Current treatment includes factor replacement therapy by infusion of missing Factor VIII or IX. But challenges exist because infusions schedules differ according to therapies. Access to veins for infusion can also become difficult; especially with children who require central venous infusion equipment.
Catalyst’s most advanced drug candidate is the next-generation Factor VIIa, CB 813d. Phase I was completed in in 2015.
CB 813d is primarily designed for on-demand and prophylactic therapy for severe hemophilia A and B with inhibitors. The drug combines better clot-generating activity at the place of bleeding and longer duration of action.
“We are delighted that Catalyst has selected CMC Biologics as their contract manufacturing organization for CB 813d as they prepare for a key clinical efficacy trial,” said Gustavo Mahler, president and chief executive officer of CMC Biologics. “CMC has a great deal of experience manufacturing similar blood coagulation factor products.”