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We are focused on the treatment of hemophilia using our high potency coagulation factors that promote blood clotting. We have two candidates in clinical development: marzeptacog alfa (activated) or MarzAA, and dalcinonacog alfa, or DalcA. MarzAA is our next-generation subcutaneous coagulation Factor VIIa (FVIIa) for individuals with hemophilia A or B with inhibitors. We have completed enrollment and dosing of our Phase 2 open-label subcutaneous efficacy trial. Final results from all nine subjects who completed dosing in the Phase 2 open-label trial (NCT03407651) were presented at the International Society for Thrombosis & Hemostasis (ISTH) meeting in July 2019. The Phase 2 trial of our subcutaneous (SQ) Factor VIIa (FVIIa) variant marzeptacog alfa (activated) (MarzAA) for prophylaxis met the primary endpoint of significantly reducing the annualize bleed rate (ABR) in patients with hemophilia A or B with inhibitors. The study also met all secondary endpoints of safety, tolerability and lack of anti-drug antibody or inhibitor formation.

Dalcinonacog alfa (DalcA), is our next-generation subcutaneously-administered Factor IX (FIX) therapy being developed for the treatment of hemophilia B.

Both MarzAA and DalcA have received orphan drug designation in the U.S. and in the E.U.

Additional Pipeline Asset

The Factor IX gene therapy construct CB 2679d-GT has demonstrated 3-fold higher activity and 4-5-fold faster clotting time in a preclinical hemophilia B mouse model compared with the Padua variant of Factor IX that is in clinical development by others. Pfizer/Spark (fidanacogene elaparvovec) and uniQure (AMT-061) use the Padua variant as the transgene in their AAV based gene therapy clinical programs and both have demonstrated encouraging Factor IX levels in their respective Phase 1/2 and Phase 2/3 studies with median Factor IX levels of approximately 30%. We are currently optimizing the construct and will make a strategic decision on its future development at the end of this year.