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
Subcutaneous MarzAA (FVIIa) prophylaxis reduced the annualized bleeding rate by more than 90% compared with pretreatment
Data presented at the 2019
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“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.
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 (
Catalyst also presented three posters at the ISTH conference. Dr.
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
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A webcast replay will be available for 30 days following the live event.
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.
This press release contains forward-looking statements that involve substantial risks and uncertainties. Forward-looking statements include statements about the potential use of MarzAA as a prophylactic therapy for patients with hemophilia A or B with inhibitors, clinical trial results, the absence of adverse events or inhibitor antibodies in patients treated with MarzAA, and immunogenicity risks of DalcA. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially, including, but not limited to, the risk that additional human trials will not replicate the results from earlier trials, that potential adverse effects may arise from the testing or use of MarzAA or DalcA, including the generation of antibodies, which has been observed in patients treated with DalcA, the risk that costs required to develop or manufacture the Company’s products will be higher than anticipated, competition and other factors that affect our ability to establish collaborations on commercially reasonable terms and other risks described in the “Risk Factors” section of the Company’s quarterly report on Form 10-Q filed with the Securities and Exchange Commission on May 2, 2019, and in other filings with the Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements, except as required by law.
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Source: Catalyst Biosciences, Inc.