Catalyst Biosciences Meets the Primary Endpoint of Reduction in Annualized Bleeding Rate in the Phase 2 Trial of Subcutaneous Marzeptacog Alfa (Activated) in Patients with Hemophilia A or B with Inhibitors
- Category: Proteins and Peptides
- Published on Monday, 08 July 2019 17:54
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Subcutaneous MarzAA (FVIIa) prophylaxis reduced the annualized bleeding rate by more than 90% compared with pretreatment
Data presented at the 2019 Congress of the International Society on Thrombosis and Haemostasis
SOUTH SAN FRANCISCO, CA, USA I July 08, 2019 I Catalyst Biosciences, Inc. (NASDAQ: CBIO) today announced that the Phase 2 trial of its subcutaneous (SQ) Factor VIIa (FVIIa) variant marzeptacog alfa (activated) (MarzAA) for prophylaxis met the primary endpoint of significantly reducing the annualized 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. The results were presented in an oral presentation at the 2019 Congress of the International Society on Thrombosis and Haemostasis on July 7, 2019, in Melbourne, Australia by Johnny Mahlangu, M.D., Professor of haematology, faculty of health sciences, head of the School of Pathology at the University of Witwatersrand in Johannesburg, South Africa, and a principal investigator in the trial.
“The results from the Phase 2 trial of MarzAA showed that SQ MarzAA achieved statistical significance in the study’s primary efficacy endpoint, with an excellent reduction in annualized bleeding rates,” said Dr. Nassim Usman, Chief Executive Officer of Catalyst Biosciences. “Reducing the median bleeds to zero with daily SQ prophylactic MarzAA therapy clearly demonstrated improved prophylactic therapy for patients with hemophilia A or B with inhibitors and the potential for leading normal active lives.”
Daily SQ administration for 50 days at an individual’s final dose of MarzAA significantly reduced the mean 6-month pre-study ABR from 19.8 to 1.6 during treatment (p<0.01). Additionally, the Proportion of Days with Bleeding (PDB), was significantly reduced from a 6-month pretreatment mean of 12.3% to 0.8% during treatment (p<0.01). The median ABR and PDB were both reduced to zero during treatment, with seven of nine subjects experiencing no bleeds, either traumatic or spontaneous, at their final dose level. Subcutaneous treatment with MarzAA was safe and well-tolerated. Six mild to moderate localized skin reactions were observed in 2 subjects. No anti-drug antibodies or inhibitors to MarzAA were detected after administration of a total of 517 SQ doses. Subcutaneous administration prolonged the half-life of MarzAA to 16.6 hours so that trough levels of MarzAA before the next SQ dose were sufficient to provide bleed prevention.
The Phase 2 open-label trial in patients with hemophilia A or B with inhibitors was designed to evaluate the efficacy of MarzAA in reducing total bleeding episodes. The primary endpoint was to assess the effect of MarzAA on annualized bleed rate at the final dose level, with each patient’s historic annualized bleeding rate serving as his own control. MarzAA has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for routine prophylaxis to prevent bleeding episodes in individuals with hemophilia A or B with inhibitors.
Catalyst also presented three posters at the ISTH conference. Dr. Howard Levy, Chief Medical Officer, presented data on assessment of quality of life parameters from the Phase 2 MarzAA trial described above. Dr. Grant Blouse, Vice President of Translational Research, presented a comprehensive in silico and in vitro immunogenicity risk assessment of the Company’s next-generation engineered SQ coagulation Factor IX (FIX) dalcinonacog alfa (DalcA) compared with wildtype FIX. Dr. Mahlangu presented the trial design of the ongoing Phase 2b study of DalcA.
A copy of the presentation materials can be accessed on the Events and Presentations section of the Catalyst website. Catalyst will host an investor call on Monday, July 8, at 8:00 a.m. EDT.
About Catalyst Biosciences
Catalyst is a clinical-stage biopharmaceutical company developing novel medicines to address hematology indications. Catalyst is focused on the field of hemostasis, including the subcutaneous prophylaxis of hemophilia. For more information, please visit www.catalystbiosciences.com.
SOURCE: Catalyst Biosciences