Stopping bleeding is good – preventing bleeding is better
All currently approved medicines for hemophilia are injected into a patient’s veins (intravenous, or IV) – at Catalyst, we believe that a clotting factor that could be injected just under the skin (subcutaneous, or SQ) would simplify the treatment, and enhance the lives of individuals with hemophilia.
Catalyst is focused on the prevention of spontaneous bleeding in hemophilia, even during surgery, using our potent coagulation factors to promote blood clotting. In June of 2017, an IV and SQ dosing trial was initiated in individuals with Hemophilia B using our next-generation Factor IX, CB 2679d. We plan to initiate an additional SQ dosing clinical trial in individuals with Hemophilia A or B with inhibitors using our next-generation Factor VIIa, marzeptacog alfa (activated), in the fourth quarter of 2017.
Hemophilia is a rare but serious bleeding disorder
Hemophilia results from a genetic or acquired deficiency of a protein required for normal blood coagulation. Individuals with hemophilia suffer from bleeding episodes and substantially prolonged bleeding times that can become limb- or life-threatening following trauma or spontaneously. In cases of severe hemophilia, spontaneous bleeding into muscles or joints is frequent and often results in permanent, disabling joint damage.
Individuals with hemophilia are currently treated with replacement therapy of key coagulation proteins, Factor VIII for Hemophilia A or Factor IX for Hemophilia B. A potential complication for individuals with hemophilia receiving factor replacement therapy is the production of antibodies against the replacement factor, also called inhibitors. The overall prevalence of inhibitor formation is up to 30 percent in individuals with hemophilia A and up to 5 percent in individuals with hemophilia B.
Individuals with hemophilia who have inhibitors are treated with what are known as “bypass” agents that initiate coagulation by a pathway that is independent of Factor VIII or Factor IX. There are currently two approved types of “bypass agents”, Factor VIIa (e.g. NovoSeven™; not approved for prophylaxis) and plasma-derived FEIBA™ that requires lengthy IV infusions every other day (not approved for prophylaxis of Hemophilia B with inhibitors in Europe). However, current hemophilia drugs for individuals with inhibitors have significant limitations regarding potency and duration of action and often require multiple doses injected intravenously, and the infusions time can be lengthy, which is not ideal for patients, especially for children.
We believe that the shortcomings of currently approved therapies are barriers to prophylactic treatment strategies that, if surmounted, could provide meaningfully improved long-term clinical outcomes for individuals with hemophilia. Catalyst's engineered proteases are designed to overcome the significant limitations of current treatment options, facilitate preventative treatment, and ultimately deliver substantially better outcomes for individuals with hemophilia.