-- CB 2679d/ISU304 Demonstrates Beneficial Effect
in Hemophilia B Murine Models Using Subcutaneous Dosing --
Catalyst Biosciences, Inc. (NASDAQ:CBIO), a clinical-stage
biopharmaceutical company focused on developing novel medicines to
address hematology indications, today 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
About Factor IX CB 2679d/ISU304 is a
next-generation coagulation Factor IX variant that is in advanced
preclinical development. CB 2679d/ISU304 has exhibited enhanced
procoagulant activity, improved efficacy in inhibiting blood loss,
and prolonged duration of action in bleeding and non-bleeding
preclinical models compared to other Factor IX products on the
market and in development. Based on these findings, Catalyst
believes that CB 2679d/ISU304 may represent a novel, high-potency
FIX variant. Catalyst has a collaboration with ISU Abxis to advance
the development of CB 2679d/ISU304 through a Phase 1/2
proof-of-concept study in individuals with hemophilia B. After
Phase 1, ISU Abxis retains exclusive commercial rights in South
Korea while Catalyst retains full development and commercial rights
for CB 2679d/ISU304 outside of South Korea.
About Hemophilia and Factor Replacement
TherapyHemophilia, for which there is no cure, is a rare
but serious bleeding disorder that results from a genetic or an
acquired deficiency of a protein required for normal blood
coagulation. There are two major types of hemophilia, A and B, that
are caused by alterations in Factor VIII or Factor IX genes,
respectively, with a corresponding deficiency in the affected
proteins. The prevalence of hemophilia A and B in the United States
is estimated to be around 20,000 people, with more than 400,000
cases worldwide. Individuals with hemophilia suffer from
spontaneous bleeding episodes as well as substantially prolonged
bleeding times upon injury. In cases of severe hemophilia,
spontaneous bleeding into muscles or joints is frequent and often
results in permanent, disabling joint damage and can become life
threatening. Treatment usually involves management of acute
bleeding episodes or prophylaxis through factor replacement therapy
by infusion of patients’ missing Factor VIII or IX. With the
frequent infusion schedule of current therapies, adherence is
difficult. In addition, convenient access to peripheral veins is
often a problem, and many children require use of central venous
access devices, with the concomitant risks of infection and
thrombosis.
About Catalyst Catalyst is a clinical-stage
biopharmaceutical company focused on 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. Catalyst’s
most advanced program is an improved next-generation coagulation
Factor VIIa variant, marzeptacog alfa (activated), that has
successfully completed an intravenous Phase 1 clinical trial in
individuals with severe hemophilia A or B. Catalyst is also
developing a next-generation Factor IX variant, CB 2679d/ISU304,
that is in advanced preclinical development. For more information,
please visit www.catbio.com.
Forward-Looking StatementsThis press release
contains forward-looking statements that involve substantial risks
and uncertainties. All statements, other than statement of
historical facts, included in this press release regarding our
strategy, future operations, and plans are forward-looking
statements. Examples of such statements include, but are not
limited to, statements relating to Catalyst’s clinical trial
timelines, including the anticipated initiation of a Phase 1/2
clinical trial for Factor IX CB 2679d/ISU304 in the first quarter
of 2017 and the potential uses and benefits of subcutaneously dosed
CB 2679d/ISU304. 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 Catalyst 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 Catalyst’s products,
including the generation of antibodies, the risk that costs
required to develop or manufacture Catalyst’s products will be
higher than anticipated, competition and other factors that affect
our ability to successfully develop and commercialize our product
candidates described in the “Risk Factors” section of the Company’s
Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed
with the SEC. Catalyst does not assume any obligation to update any
forward-looking statements, except as required by law.
Contacts:
Investors:
Fletcher Payne, CFO
Catalyst Biosciences, Inc.
650.871.0761
investors@catbio.com
Media:
Denise Powell
Red House Consulting, LLC
510.703.9491
denise@redhousecomms.com
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