Caelum Biosciences, Inc. (“Caelum”), a Fortress Biotech, Inc.
(NASDAQ:FBIO) Company developing treatments for rare and
life-threatening diseases, today announced a new analysis of data
from the Phase 1b trial of CAEL-101 (mAb 11-1F4) for the treatment
of relapsed or refractory amyloid light chain (“AL”) amyloidosis,
demonstrating a correlation between a sustained decrease in
N-terminal pro-brain natriuretic peptide (NT-proBNP) levels and an
improvement in global longitudinal strain (GLS) following CAEL-101
treatment in patients with cardiac AL amyloidosis. The data were
presented today in an oral session at the 16th International
Symposium on Amyloidosis (ISA).
The Phase 1a/1b trial (ClinicalTrials.gov
Identifier: NCT02245867) examined the tolerance, safety,
pharmacokinetics and possible clinical benefit of CAEL-101 in
patients with AL amyloidosis. CAEL-101 was administered to eight
patients via a single IV infusion at week one in the Phase 1a
portion of the trial, and to 19 patients via one weekly IV infusion
for four weeks in the Phase 1b portion of the trial.
Eight of 12 (67 percent) of evaluable cardiac
patients in the Phase 1a/1b trial demonstrated a sustained decrease
in NT-proBNP levels, an important biomarker in cardiac disease,
after treatment with CAEL-101. Evaluable patients presented with
cardiac amyloidosis at baseline (NT-proBNP ≥650 pg/mL) and at least
one post-baseline NT-proBNP measure. The depth and magnitude of
response continued through four once-weekly doses of CAEL-101.
An improvement in GLS, a measure of myocardial
shortening during systole, was demonstrated in eight patients with
confirmed cardiac amyloidosis at baseline who were enrolled in the
Phase 1b portion of the trial, and was correlated with the
reduction in NT-proBNP (Pearson correlation coefficienti 0.345),
demonstrating CAEL-101’s positive impact on cardiac response of
myocardial function.
“GLS is an important biomarker for cardiac
dysfunction and response, and may be a more accurate predictor of
survival than cardiac biomarkers, which can be elevated
disproportionately to the severity of cardiac symptoms and based on
factors unrelated to worsening cardiac dysfunction,” said Camille
Edwards, M.D., Fellow in the Department of Hematology and Oncology
at Boston Medical Center, who presented the data at ISA. “CAEL-101
is a novel and promising treatment that has the potential to safely
promote amyloid resolution and improve cardiac and overall organ
function in AL amyloidosis. The presentation of a complete analysis
of cardiac data from the Phase 1b trial is planned for later this
year.”
“The correlation of GLS improvement with
NT-proBNP reduction builds upon the strong Phase 1a/1b safety and
efficacy data presented at ASH 2017, and it is important to note
that efficacy was demonstrated independent of plasma-cell directed
therapy,” said Michael Spector, President and Chief Executive
Officer of Caelum.
About AL AmyloidosisAL amyloidosis is a rare
systemic disorder caused by an abnormality of plasma cells in the
bone marrow. Misfolded amyloid proteins produced by plasma cells
cause buildup in and around tissues, nerves and organs, gradually
affecting their function. This can cause progressive and widespread
organ damage, and high mortality rates.
AL amyloidosis affects roughly 30,000 – 40,000
patients in total throughout the U.S. and Europe, and it is
estimated that there are approximately 3,000 – 4,000 new cases of
AL amyloidosis annually in the U.S., though actual incidence is
likely higher as a result of under diagnosis. Amyloidosis has a
one-year mortality rate of 47 percent, 76 percent of which is
caused by cardiac amyloidosis.
About CAEL-101 (mAb
11-1F4)CAEL-101 is a chimeric fibril-reactive monoclonal
antibody (mAb) that has completed a Phase 1a/1b clinical trial at
Columbia University for the treatment of patients with AL
amyloidosis. While current treatment with chemotherapy is aimed at
reducing production of the amyloid-forming light-chain protein,
CAEL-101 attempts to reduce and / or eliminate the amyloid
deposits.
About Caelum BiosciencesCaelum
Biosciences, Inc. (“Caelum”), a Fortress Biotech (NASDAQ: FBIO)
Company, is a clinical-stage biotechnology company developing
treatments for rare and life-threatening diseases. Caelum’s lead
asset, CAEL-101 (mAb 11-1F4), is a novel antibody for the treatment
of patients with amyloid light chain (“AL”) amyloidosis. Phase
1a/1b data presented at the American Society of Hematology’s 59th
Annual Meeting in December 2017 support CAEL-101’s potential to be
a safe and well-tolerated therapy that promotes amyloid resolution.
CAEL-101 has received Orphan Drug Designation from the U.S. Food
and Drug Administration as a therapeutic agent for patients with AL
amyloidosis, and as a radio-imaging agent in amyloidosis. For more
information, visit www.caelumbio.com.
About Fortress BiotechFortress Biotech, Inc.
(“Fortress”) is a biopharmaceutical company dedicated to acquiring,
developing and commercializing novel pharmaceutical and
biotechnology products. Fortress develops and commercializes
products both within Fortress and through certain subsidiary
companies, also known as Fortress Companies. In addition to its
internal development programs, Fortress leverages its
biopharmaceutical business expertise and drug development
capabilities and provides funding and management services to help
the Fortress Companies achieve their goals. Fortress and the
Fortress Companies may seek licensings, acquisitions, partnerships,
joint ventures and/or public and private financings to accelerate
and provide additional funding to support their research and
development programs. For more information, visit
www.fortressbiotech.com.
Forward-Looking StatementsThis
press release may contain “forward-looking statements” within the
meaning of Section 27A of the Securities Act of 1933 and Section
21E of the Securities Exchange Act of 1934, as amended. Such
statements include, but are not limited to, any statements relating
to our growth strategy and product development programs and any
other statements that are not historical facts. Forward-looking
statements are based on management’s current expectations and are
subject to risks and uncertainties that could negatively affect our
business, operating results, financial condition and stock price.
Factors that could cause actual results to differ materially from
those currently anticipated include: risks relating to our growth
strategy; our ability to obtain, perform under and maintain
financing and strategic agreements and relationships; risks
relating to the results of research and development activities;
uncertainties relating to preclinical and clinical testing; risks
relating to the timing of starting and completing clinical trials;
our dependence on third-party suppliers; our ability to attract,
integrate and retain key personnel; the early stage of products
under development; our need for substantial additional funds;
government regulation; patent and intellectual property matters;
competition; as well as other risks described in our SEC filings.
We expressly disclaim any obligation or undertaking to release
publicly any updates or revisions to any forward-looking statements
contained herein to reflect any change in our expectations or any
changes in events, conditions or circumstances on which any such
statement is based, except as required by law.
Contacts:Caelum Biosciences, Inc.Michael
Spector, President & Chief Executive Officer(212)
574-2811mspector@caelumbio.com
Fortress Biotech, Inc.Jaclyn Jaffe, Investor Relations(781)
652-4500ir@fortressbiotech.com
Fortress Biotech, Inc.Laura Bagby, Media Relations6 Degrees(312)
448-8098lbagby@6degreespr.com
i The Pearson correlation coefficient is a measure of the
linear correlation between two variables.
Fortress Biotech (NASDAQ:FBIO)
Historical Stock Chart
From Aug 2024 to Sep 2024
Fortress Biotech (NASDAQ:FBIO)
Historical Stock Chart
From Sep 2023 to Sep 2024