Catalyst Biosciences Announces Updated Positive Interim Data from Its Phase 2/3 Study of Marzeptacog Alfa (Activated) in Indi...
December 01 2018 - 12:00PM
Results demonstrate efficacy of subcutaneous
prophylaxis with MarzAA, Catalyst’s high potency engineered
FVIIa
Catalyst Biosciences, Inc. (NASDAQ: CBIO), a clinical-stage
biopharmaceutical company focused on developing novel medicines to
address hematology indications, today announced additional positive
interim data from its Phase 2/3 study of subcutaneous prophylactic
Factor VIIa (FVIIa) variant marzeptacog alfa (activated) (MarzAA),
currently being developed for the treatment of hemophilia A or B
with inhibitors. The data were presented in a poster at the 60th
American Society of Hematology (ASH) Annual Meeting &
Exposition on December 1, 2018 in San Diego.
“These results support the efficacy of MarzAA as a subcutaneous
prophylactic treatment option for individuals with hemophilia A or
B with inhibitors,” said Nassim Usman, Ph.D., chief executive
officer of Catalyst. “We observed that bleeding in individuals with
high annualized bleed rates is significantly reduced or eliminated
during MarzAA treatment. With these new data from two additional
individuals who have successfully completed the trial, we are
optimistic about the potential for MarzAA treatment to achieve
extremely low annualized spontaneous bleed rates with
individualized, subcutaneous daily dosing.”
Dr. Howard Levy, chief medical officer of Catalyst, presented
the updated results, including the new data from two additional
subjects who have completed the Phase 2/3 MarzAA trial. The first
subject, who had an annualized bleed rate (ABR) of 15.2, had no
bleeds during 50 days of treatment with 30 µg/kg MarzAA. The second
subject, who had an ABR of 22.2, experienced a bleed on Day 4 that
did not require treatment. The subject continued on the 30 µg/kg
dose level, as the bleed occurred within the first five days of
dosing when FVIIa levels are still increasing to therapeutic levels
and completed the trial with no additional bleeds during the
treatment period.
To date, 13 subjects have consented and five have completed
dosing in the Phase 2/3 MarzAA trial. These five subjects had an
ABR range of 15.2-26.7 before MarzAA treatment. Three participants
experienced no bleeds with individualized dosing of either 30 µg/kg
or 60 µg/kg MarzAA and two others had clinically significant
reductions in ABR. The median proportion of days with bleeding
during the pre-study period was 11.9% and this was significantly
reduced to a median of 0.5% during the treatment period. Through a
total of more than 300 days of subcutaneous dosing, no injection
site reactions or anti-drug antibodies to MarzAA have been
detected. In one subject, two subcutaneous injections resulted in a
mild localized hematoma that resolved without sequelae or
treatment. Final data from the study are expected in the first half
of 2019.
A copy of the presentation materials can be accessed on the
Events and Presentations section of the Catalyst website.
About the FVIIa Phase 2/3 TrialMarzeptacog alfa
(activated) (MarzAA) is a potent, subcutaneous Factor VIIa therapy
being developed for prophylaxis in hemophilia A or B with
inhibitors. The Phase 2/3 open-label, subcutaneous efficacy trial
in individuals with hemophilia A or B with inhibitors will evaluate
the ability of MarzAA to eliminate, or minimize, spontaneous
bleeding episodes. The primary endpoint is a reduction in
annualized bleed rate that will be compared with each individual’s
recorded historical annualized bleed rate as the control. The trial
will enroll up to 12 individuals with hemophilia and an inhibitor
across approximately ten clinical trial sites globally. MarzAA has
been granted orphan drug designation by the U.S. Food and Drug
Administration (FDA) for routine prophylaxis to prevent bleeding
episodes in individuals with hemophilia A or B with inhibitors.
About Catalyst BiosciencesCatalyst 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.
Forward-Looking StatementsThis press release
contains forward-looking statements that involve substantial risks
and uncertainties. Forward-looking statements include statements
about the potential for marzeptacog alfa (activated) (MarzAA) to
provide prophylaxis therapy in patients with hemophilia A or B with
inhibitors, Catalyst’s plans to complete a Phase 2/3 open label
trial of this product candidate, and Catalyst’s plans to continue
development of this and other product candidates. 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 additional human trials will not
replicate the results from earlier trials, including interim data,
that potential adverse effects may arise from the testing or use of
MarzAA or other Catalyst product candidates, 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
September 30, 2018 filed with the Securities and Exchange
Commission on November 1, 2018, and with 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.
Contacts:Investors:Fletcher
Payne, CFOCatalyst Biosciences,
Inc.1.650.871.0761investors@catbio.com
Media:Josephine Belluardo, Ph.D.LifeSci Public
Relations1.646.751.4361jo@lifescipublicrelations.com
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