- First time published in a peer-reviewed journal: birtamimab is
the only investigational drug that has shown a significant survival
benefit in patients with Mayo Stage IV AL amyloidosis in a
double-blind placebo-controlled clinical trial
- A significant improvement in time to all-cause mortality at
month 9 was observed with birtamimab versus placebo and remained
consistent across all key baseline variables in a post hoc analysis
of patients with Mayo Stage IV AL amyloidosis
- Birtamimab is currently being studied in the confirmatory Phase
3 clinical trial, AFFIRM-AL, in patients with Mayo Stage IV AL
amyloidosis; topline data is expected in 2024
Prothena Corporation plc (NASDAQ:PRTA), a late-stage clinical
biotechnology company with a robust pipeline of investigational
therapeutics built on protein dysregulation expertise, today
announced the publication of the Phase 3 VITAL clinical trial in
Blood, a journal of the American Society of Hematology (ASH). The
published data demonstrate that in a post hoc analysis of patients
with Mayo Stage IV AL amyloidosis, a statistically significant
survival benefit of 74 percent was observed for those treated with
birtamimab plus standard of care (SOC) versus 49 percent in
patients on placebo plus SOC at 9 months (HR 0.413, p=0.021). All
participants in the clinical trial received concomitant
bortezomib-containing chemotherapy regimens as part of SOC.
“For the first time, we have these important data published in a
prestigious, peer-reviewed journal which show that treatment with
birtamimab led to a survival benefit in patients with Mayo Stage IV
AL amyloidosis and affirms its potential as a safe, well-tolerated
and effective therapy,” said Morie Gertz, MD, Hematologist, Chair
emeritus Internal Medicine, Mayo Clinic. “AL amyloidosis is a rare
and life-threatening disease in which patients have no treatment
options despite the high fatality rate. We look forward to learning
more about the survival benefit of birtamimab in patients with Mayo
Stage IV AL amyloidosis from the confirmatory Phase 3 AFFIRM-AL
clinical trial.”
The article, entitled “Birtamimab plus standard of care in light
chain amyloidosis: the phase 3 randomized placebo-controlled VITAL
clinical trial”, also includes new data showing that there was no
observed difference in hematologic response rates between the
control arm and the treatment arms which suggests the survival
benefit of birtamimab was not due to improved hematologic response,
which is consistent with birtamimab’s depleter mechanism of
action.
For two secondary endpoints, birtamimab demonstrated
statistically significant improvements over placebo in a post hoc
assessment of patients with Mayo Stage IV AL amyloidosis. The
secondary endpoints were quality of life (assessed with the Short
Form-36 version 2 physical component score, SF-36v2 PCS) and
cardiac function (assessed with the 6-minute walk test). Patients
treated with birtamimab showed a slower decline in quality of life
with a mean decrease of 0.75 in the SF-36v2 PCS at 9 months
compared to a mean decrease of 5.40 in the SF-36v2 PCS for patients
on placebo at 9 months (a mean difference of 4.65 favoring
birtamimab; p=0.046). Patients treated with birtamimab after 9
months demonstrated an increase in mean distance of 15.22 meters in
the 6-minute walk test, compared to a decrease in mean distance of
21.15 meters for patients on placebo (a mean difference of 36.37
meters favoring birtamimab; p=0.022).
In safety evaluations, the rates of treatment emergent adverse
events (TEAEs) were balanced between treatment arms among patients
with Mayo Stage IV AL amyloidosis (38 TEAEs in patients treated
with birtamimab compared to 39 TEAEs in patients receiving
placebo). The rates of treatment-related TEAEs were similar or
lower with birtamimab than in the placebo arms. Cardiac disorder
was the most common class of fatal TEAEs, which is consistent with
patients who have AL amyloidosis. There were no fatal TEAEs that
were considered treatment related.
Birtamimab is a potential best-in-class amyloid depleter
treatment for AL amyloidosis. Birtamimab specifically binds to a
defined epitope on kappa and lambda AL protein involved in the
disease process. Based on the totality of data generated to date,
including results from the VITAL clinical trial, Prothena has
advanced birtamimab into the confirmatory Phase 3 AFFIRM-AL
clinical trial in patients with Mayo Stage IV AL amyloidosis under
a Special Protocol Assessment (SPA) agreement with the U.S. Food
and Drug Administration (FDA) with a primary endpoint of all-cause
mortality at a significance level of 0.10. Phase 3 AFFIRM-AL
topline data is expected in 2024. Birtamimab has also been granted
orphan drug designation for AL amyloidosis by both the FDA and the
European Medicines Agency and has been granted Fast Track
designation by the FDA.
Blood is the weekly, peer-reviewed journal published by The
American Society of Hematology (ASH). A copy of the Phase 3 VITAL
publication can be found here:
https://doi.org/10.1182/blood.2022019406
Prothena previously presented data on the Phase 3 VITAL clinical
trial in an oral presentation at the 64th ASH Annual Meeting and
Exposition.
About VITAL Phase 3 Clinical Trial
VITAL was a phase 3 multicenter, randomized, double-blind,
placebo-controlled clinical trial that evaluated the efficacy and
safety of birtamimab plus standard of care versus placebo plus
standard of care in newly diagnosed, treatment-naïve patients with
AL amyloidosis. The clinical trial was terminated early based on a
futility analysis. The primary endpoint in the full clinical trial
population was the composite of time to all-cause mortality and
cardiac hospitalization in patients with AL amyloidosis. The
primary endpoint in the overall clinical trial population favored
birtamimab over placebo, but the difference was not statistically
significant at the time of early clinical trial termination. The
primary clinical trial population included 260 patients with AL
amyloidosis, of which patients who received birtamimab and placebo
were evenly split. Approximately one-third of patients in the
clinical trial had Mayo Stage IV AL amyloidosis (n=77). Patient
demographics were generally balanced between the birtamimab and
placebo groups in the clinical trial population and the Mayo Stage
IV sub population.
About Phase 3 AFFIRM-AL Clinical Trial
The AFFIRM-AL clinical trial is a global, multi-center,
double-blind, placebo-controlled, 2:1 randomized, time-to-event
clinical trial expected to enroll approximately 150 newly
diagnosed, treatment naïve patients with AL amyloidosis categorized
as Mayo Stage IV. The clinical trial is being conducted under a SPA
agreement with FDA and supported by the significant survival
benefit observed in the previous analysis of birtamimab-treated
patients categorized as Mayo Stage IV at baseline in the VITAL
clinical trial. For more information on the clinical trial please
visit https://affirm-al.com/.
About Birtamimab
Birtamimab is an investigational, humanized monoclonal antibody
designed to specifically and selectively target and clear the
amyloid that accumulates and causes organ dysfunction and failure
in patients with AL amyloidosis. Birtamimab specifically binds to a
defined epitope on kappa and lambda AL protein involved in the
disease process. Birtamimab is the only investigational drug that
has shown a significant survival benefit in patients with Mayo
Stage IV AL amyloidosis post-hoc in a placebo-controlled clinical
trial. Birtamimab has been granted orphan drug designation for AL
Amyloidosis by both the U.S. FDA and the European Medicines Agency
and has been granted Fast Track designation by the FDA. A SPA was
agreed to between Prothena and the FDA for the AFFIRM-AL clinical
trial which represents FDA’s agreement that the design and planned
analysis for the primary endpoint of time to all-cause mortality
adequately address the objectives necessary to support a regulatory
submission. Results from the AFFIRM-AL clinical trial are
anticipated in 2024. Final marketing approval is predicated upon
FDA’s complete review of the entire application.
About AL Amyloidosis
AL amyloidosis is a rare, progressive and fatal disease where
clonal plasma cells overproduce light chain proteins that misfold,
aggregate and deposit as amyloid in vital organs such as the heart.
It is estimated that there are 60,000 – 120,000 patients worldwide
living with Mayo Stage IV AL amyloidosis. Patients with AL
amyloidosis can present with a wide range of general symptoms that
are common to other conditions such as fatigue, shortness of breath
or edema. Current treatment strategies target plasma cells to
reduce production of new amyloid, but do not address the amyloid
already deposited in organs. Mortality is driven primarily by
cardiac failure. There is an urgent unmet medical need for
therapies that improve survival in patients at risk for early
mortality due to amyloid deposition.
About Prothena
Prothena Corporation plc is a late-stage clinical biotechnology
company with expertise in protein dysregulation and a pipeline of
investigational therapeutics with the potential to change the
course of devastating neurodegenerative and rare peripheral amyloid
diseases. Fueled by its deep scientific expertise built over
decades of research, Prothena is advancing a pipeline of
therapeutic candidates for a number of indications and novel
targets for which its ability to integrate scientific insights
around neurological dysfunction and the biology of misfolded
proteins can be leveraged. Prothena’s pipeline includes both
wholly-owned and partnered programs being developed for the
potential treatment of diseases including AL amyloidosis, ATTR
amyloidosis, Alzheimer’s disease, Parkinson’s disease and a number
of other neurodegenerative diseases. For more information, please
visit the Company’s website at www.prothena.com and follow the
Company on Twitter @ProthenaCorp.
Forward-Looking Statements
This press release contains forward-looking statements. These
statements relate to, among other things, the treatment potential,
design, proposed mechanism of action, and potential administration
of birtamimab; and the expected timing of reporting data from a
clinical trial of birtamimab. These statements are based on
estimates, projections and assumptions that may prove not to be
accurate, and actual results could differ materially from those
anticipated due to known and unknown risks, uncertainties and other
factors, including but not limited to those described in the “Risk
Factors” sections of our Quarterly Report on Form 10-Q filed with
the Securities and Exchange Commission (SEC) on May 4, 2023, and
discussions of potential risks, uncertainties, and other important
factors in our subsequent filings with the SEC. We undertake no
obligation to update publicly any forward-looking statements
contained in this press release as a result of new information,
future events, or changes in our expectations.
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version on businesswire.com: https://www.businesswire.com/news/home/20230627324499/en/
Media and Investor Contact: Media Michael Bachner, Senior
Director, Corporate Communications 609-664-7308,
michael.bachner@prothena.com
Investors: IR@prothena.com
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