Catalyst Biosciences Announces Preclinical Proof-of-Concept Data of Gene Therapy Candidate CB 2679d-GT for the Treatment of Hemophilia B
Results show a four-fold reduction in bleeding time for CB 2679d-GT and a three-fold improvement in clotting activity when compared with an AAV-encoding Padua vector
Data presented at the 12th Annual
“These results are encouraging and demonstrate preclinical proof-of-concept for CB 2679d-GT, a gene therapy candidate encoding our engineered Factor IX as a potential new treatment for hemophilia B,” said
The 20-week preclinical study compared the activity of CB 2679d-GT with that of an AAV-encoding FIX-R338L Padua (FIX-Padua) in hemophilia B mice. Treatment with both CB 2679d-GT and FIX-Padua showed a reduced clotting time within the first week that remained stable up to the 20-week study endpoint. CB 2679d-GT demonstrated a statistically significant, three-fold improvement in clotting activity (p <0.04), compared with FIX-Padua. Furthermore, when evaluated at 20 weeks, there was a four-fold reduction in bleeding time after treatment with CB 2679d-GT compared to FIX-Padua at both the 5x109 vg/mouse (p < 0.01) and the 1x1010 vg/mouse (p < 0.01) dose levels. These results suggest that CB 2679d-GT exhibits a superior hemostatic potency when compared with FIX-Padua.
A copy of the poster can be accessed on the Events and Presentations section of the Catalyst website.
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 and facilitating surgery in individuals with hemophilia. For more information, please visit www.catalystbiosciences.com.
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statement of historical facts (including, but not limited to, statements about the potential for CB 2679d-GT to provide gene therapy treatment for hemophilia and plans to commence a Phase 2b study this quarter) are forward-looking statements. 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 from the forward-looking statements that the Company makes, including, but not limited to, the risk that trials and studies may be delayed and may not have satisfactory outcomes, that human trials will not replicate the results from animal studies, that potential adverse effects may arise from the testing or use of the Company’s products, including the generation of antibodies, 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 for the quarter ended
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Source: Catalyst Biosciences, Inc.