Nine of 10 cardiac patients on CAEL-101 showed
improvement in global longitudinal strain
Caelum Biosciences, Inc. (“Caelum”), a Fortress Biotech, Inc.
(NASDAQ:FBIO) Company developing treatments for rare and
life-threatening diseases, today announced a complete analysis
of cardiac data from Columbia University’s (“Columbia”) Phase 1b
trial that supports CAEL-101’s (mAb 11-1F4) potential to improve
myocardial function as assessed by global longitudinal strain
(“GLS”) and generate a sustained decrease in N-terminal pro-brain
natriuretic peptide (NT-proBNP) levels in amyloid light chain
(“AL”) amyloidosis patients experiencing cardiac involvement. These
data were presented at the American Society of Echocardiography
(ASE) 29th Annual Scientific Sessions.
“Cardiac involvement can indicate a poor
prognosis in patients with AL amyloidosis, and is the cause of more
than 75 percent of one-year mortality,” said Sofia Shames, M.D.,
Assistant Professor at Columbia University Medical Center and
investigator on the Phase 1b trial. “The Phase 1b trial of CAEL-101
is the first clinical study to show a significant improvement in
GLS after exposure to an anti-fibril specific mAb in people with AL
amyloid cardiac involvement. This is important, as
echocardiographical GLS may be more effective in predicting overall
and cardiovascular mortality than traditional ejection fraction,
which warrants further evaluation in clinical trials.”
Myocardial response was assessed using GLS as
part of a long-term analysis of data from the Phase 1b trial. GLS
is a measure of myocardial shortening during systole, which may
detect early cardiac functional improvement and be a predictor of
cardiac survival.
Nineteen patients with relapsed or refractory AL
amyloidosis enrolled in the Phase 1b trial had a baseline
echocardiogram taken at their initial screening visit. Ten of 19
patients (52 percent) had cardiac involvement at the time of their
baseline echocardiogram reading as defined by NT-proBNP levels
(>650 pg/ml), an important biomarker in cardiac disease.
CAEL-101 was administered weekly for four weeks in the
multi-ascending dose trial. The dose escalation included the doses
0.5, 5, 10, 50, 100, 250 and 500 mg/m2. Clinical echocardiographic
examinations at baseline and 12 weeks post therapy were
compared.
Patients with cardiac involvement demonstrated
an improvement in GLS (-15.58 ± -4.14 percent pre-treatment /
-17.37 ± -3.53 percent post-treatment, p=0.004). Patients with both
kappa and lambda amyloid light chain subtypes were studied. The
change in GLS patients with a p-value of 0.004 and the lack of
response in patients without cardiac involvement supports further
clinical development in patients with cardiac AL amyloidosis.
In addition, mean NT-proBNP reduction was
presented, which demonstrated that there was an improvement in
NT-proBNP in eight evaluable patients with cardiac amyloid
involvement after four weekly doses. The NT-proBNP patient response
criteria were a 30-percent reduction and a 300 pg/ml reduction from
baseline.
The full abstract was published in the June 2018
edition of the Journal of the American Society of
Echocardiography.
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 trial at Columbia
University for the treatment of patients with relapsed or
refractory 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 the Phase 1a/1b trial The
Phase 1a/1b trial (ClinicalTrials.gov Identifier: NCT02245867)
examined the tolerance, safety, pharmacokinetics and possible
clinical benefit of CAEL-101 (mAb 11-1F4) in patients with relapsed
or refractory AL amyloidosis. CAEL-101 was administered weekly for
four weeks with sequential doses of 0.5, 5, 10, 50, 100, 250 and
500 mg/m2. 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.
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) 574-2811
mspector@caelumbio.com
Fortress Biotech, Inc. Jaclyn Jaffe, Investor Relations (781)
652-4500 ir@fortressbiotech.com
Fortress Biotech, Inc. Tony Plohoros, Media Relations 6 Degrees
(908) 591-2839 tplohoros@6degreesPR.com
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