Catalyst Biosciences (CBIO) Announces Strong Preclinical Data on CB 2679d/ISU304 in Hemophilia B

November 14, 2016 8:02 AM EST
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Catalyst Biosciences, Inc. (Nasdaq: CBIO) announced positive results from a preclinical study of CB 2679d/ISU304 in well-validated models of hemophilia B. The study highlighted the attractive pharmacokinetic profile of CB 2679d/ISU304 based on bioavailability, potency, time to maximal concentration, and half-life, which resulted in the ability to dose CB 2679d/ISU304 subcutaneously while also achieving steady-state levels of procoagulant activity that moved hemophilia B mice from the severe range to the mild range.

Catalyst is focused on the prevention of spontaneous bleeding in hemophilia through the development of clotting factors that may be injected subcutaneously. Hemophilia B is a chronic disease caused by a genetic deficiency in coagulation Factor IX. The current approach to treating acute bleeding episodes in individuals with hemophilia B includes the use of recombinant human Factor IX, which has low potency and is therefore not appropriate for subcutaneous prophylactic treatment.

“These results support our earlier preclinical findings that CB 2679d/ISU304 has significantly higher potency compared with other Factor IX products that cannot be practically dosed by subcutaneous injection to achieve satisfactory levels of drug. If these preclinical results can be duplicated in patients, not only would CB 2679d/ISU304 provide greater convenience, but could potentially provide better efficacy since CB 2679d/ISU304 may result in steady state FIX activity levels consistent with normal coagulation,” said Nassim Usman, Ph.D., President and Chief Executive Officer of Catalyst. “Based on the totality of our preclinical data, we and our partner, ISU Abxis, are preparing to initiate a Phase 1/2 proof-of-concept clinical trial in individuals with hemophilia B in the first quarter of 2017. We believe that CB 2679d/ISU304 may ultimately provide individuals with hemophilia B prophylaxis by subcutaneous injection.”

The results, being presented in a poster session at the American Society of Hematology (ASH) 58th Annual Meeting in San Diego, Calif. from December 3 to 6, 2016, are summarized below:

Pharmacokinetics of Subcutaneously Administered CB 2679d/ISU304 in Wild-Type and Hemophilia B Mice (Poster abstract #1389, Session: 321 Blood Coagulation and Fibrinolytic Factors)Seung-Beom Hong, PhD, Howard Levy, MBBChir, PhD, Jae Yong Jung, MS, Minkyung Park, AS, A Rim Seo, AS, So Hyeon Seo, MS and Ed Madison, PhD

The authors tested subcutaneous doses of CB 2679d/ISU304 in both hemophilia B and wild-type mice. The wild-type mice also received subcutaneous doses of BeneFIX. Factor IX antigen and activity were measured at various time points. The following conclusions were made:

  • There was a dose-dependent increase in plasma Factor IX antigen with subcutaneous CB 2679d/ISU304;
  • The pharmacokinetic profile of CB 2679d/ISU304 was similar to BeneFIX when dosed using the same mass, however, CB 2679d/ISU304 has approximately 17-times greater potency;
  • Due to the high specific activity of CB 2679d/ISU304, a subcutaneous dose of CB 2679d/ISU304 yields much higher Factor IX activities in mouse plasma compared with the same mass dose of BeneFIX; and
  • Daily subcutaneous dosing of CB 2679d/ISU304 demonstrated the effects of the bioavailability, potency, time to maximal concentration, and half-life by reaching a steady-state activity after three days, sufficient to correct severe hemophilia to mild hemophilia in hemophilia B mice.

For more information about Catalyst Biosciences, including a recent corporate update, please see the webcast replay of an expert panel meeting titled Advances in Hemophilia Treatment that occurred on November 10, 2016. Advances in Hemophilia: Catalyst Biosciences Webcast

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