Columbia University reports Caelum Biosciences’
CAEL-101 was well tolerated, demonstrated early and clinically
efficacious organ response in Phase 1a/1b trial in AL
amyloidosis
Fortress Biotech, Inc. (NASDAQ:FBIO) (“Fortress”), a
biopharmaceutical company dedicated to acquiring, developing and
commercializing novel pharmaceutical and biotechnology products,
today announced positive clinical data on therapies under
development at its Fortress Company subsidiaries Caelum
Biosciences, Inc. (“Caelum”), and Mustang Bio, Inc. (“Mustang”)
(NASDAQ:MBIO), were presented in oral sessions at the 59th American
Society of Hematology (ASH) Annual Meeting.
Dr. Lindsay A. Rosenwald, Fortress Biotech’s
Chairman, President and Chief Executive Officer, said, “We are
delighted to report that data from two of our Fortress Companies
were presented in oral sessions at ASH, which is a testament to our
top-notch business development engine’s expertise in securing
compelling assets and our corporate strategy of partnering with
first-rate academic and commercial entities.”
Dr. Rosenwald added, “Trial investigators at
Columbia concluded that Caelum’s CAEL-101 dosed once weekly
demonstrated early and clinically efficacious organ responses
throughout a Phase 1a/1b trial, underscoring its potential to be a
best-in-class treatment in AL amyloidosis and providing signals to
support advancement into a Phase 2b/3 trial in the second
half of 2018. In addition, trial investigators at City of Hope
found that Mustang Bio’s MB-102 CAR T therapy was safe, well
tolerated and achieved a complete response in acute myeloid
leukemia and blastic plasmacytoid dendritic cell neoplasm in an
ongoing Phase 1 trial. According to City of Hope, this is the first
BPDCN patient to achieve a complete response to a CAR T cell
therapy.”
Caelum’s CAEL-101 improves organ
function in AL amyloidosis
Twenty-seven patients were treated with CAEL-101
in this open-label, dose-escalation trial. In the Phase 1a trial,
CAEL-101 was administered to eight patients via a single IV
infusion at week one. In the Phase 1b trial, CAEL-101 was
administered to 19 patients via one weekly IV infusion for four
weeks. Trial investigators at Columbia University (“Columbia”)
determined the study achieved its primary objective of establishing
maximum tolerated dose of up to 500mg/m2 of CAEL-101.
Trial investigators presented organ response
rates in the Phase 1a and the Phase 1b, with 63 percent (14 of 24)
overall organ response rate, 67 percent (8 of 12) overall cardiac
response rate and 50 percent (5 of 10) overall renal response
rate.1 Early organ response was demonstrated in a high-mortality
population (21 days median time to cardiac response in Phase 1b; 28
days median time to renal response in Phase 1b2).
Trial investigators found that CAEL-101 achieved
and demonstrated organ response at multiple points in time
throughout the duration of treatment; all patients showed an organ
response or were stable, and no patients showed organ progression.
Organ response independent of a chemotherapy-free light chain
response was demonstrated. No drug-related grade 4 or 5 adverse
events or dose-limiting toxicities were seen in the trial. There
was no mortality during the study. The investigators followed
patients beyond the study and reported an overall survival rate of
93 percent (median follow-up period of 18.6 months).
A copy of the presentation can be viewed online
on the Publications page of the Caelum website
at www.caelumbio.com/pipeline/publications.
Mustang’s MB-102 (CD123 CAR) CAR T
therapy achieves complete response in AML and
BPDCN
This ongoing, single center, first-in-human
Phase 1 dose-escalation clinical trial (NCT02159495) at City of
Hope is evaluating the safety and activity of escalating doses of
MB-102 in patients with relapsed or refractory acute myeloid
leukemia (AML) (cohort 1) and blastic plasmacytoid dendritic cell
neoplasm (BPDCN) (cohort 2). Patients receive a single dose of
MB-102 with an option for a second infusion if they continue to
meet safety and eligibility criteria and still have CD123+ disease.
To date, 14 patients have been enrolled and seven have been treated
(six with AML, one with BPDCN) in this first in-human trial for AML
and BPDCN patients using a CD123 CAR T therapy.
In the AML cohort, two patients were treated at
dose level 1 (50M CAR+ T). Trial investigators reported that one
achieved a morphologic leukemic-free state at day 28 post-infusion.
Four patients received dose level 2 (200M CAR+ T), with a complete
response (CR) observed at day 28 in one patient, and a CR with
incomplete blood count recovery demonstrated at day 28 in a second
patient. Both patients proceeded to a second allogeneic
hematopoietic stem cell transplantation.
In the BPDCN cohort, one patient received a
single dose of 100M CAR+ T and achieved a CR at day 28, which
lasted at least 60 days, according to investigators. Of note, this
patient had previously experienced disease progression following
five cycles of treatment with a CD123-targeted recombinant fusion
protein.
Investigators found MB-102 infusions of up to
200M CAR T cells were safe, with no graft-versus-host disease,
myeloablative effects, neurologic toxicity or dose-limiting
toxicities. Adverse events (AEs) included: cytokine release
syndrome (six grade 1, one grade 2), neurotoxicity (dizziness: one
grade 1, two grade 2; headache (five grade 1, two grade 2);
somnolence (one grade 1, two grade 2), three cases of infection
(lung infection: two, other: one). The most common ≥ grade 3
AEs included lymphopenia (seven), thrombocytopenia (seven) and
febrile neutropenia (six).
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 Mustang BioMustang Bio, Inc., a
subsidiary of Fortress Biotech, Inc., is a clinical‐stage
biopharmaceutical company focused on the development and
commercialization of novel cancer immunotherapy products designed
to leverage the patient’s own immune system to eliminate cancer
cells. Mustang aims to acquire rights to these technologies by
licensing or otherwise acquiring an ownership interest, funding
research and development, and outlicensing or bringing the
technologies to market. Mustang has partnered with the City of Hope
National Medical Center (“COH”) and the Fred Hutchinson Cancer
Research Center in the development of proprietary chimeric antigen
receptor (“CAR”) engineered T cell (“CAR T”) therapies across many
cancers, and with Harvard Medical School’s Beth Israel Deaconess
Medical Center and the Harvard Stem Cell Institute for the
development of CRISPR/Cas9-enhanced CAR T therapies in hematologic
malignancies and solid tumors. Mustang’s lead programs are in Phase
1 clinical trials at COH: MB-101 for the treatment of brain cancer
and MB-102 as a therapeutic agent in acute myeloid leukemia and
blastic plasmacytoid dendritic cell neoplasm. Mustang is registered
under the Securities Exchange Act of 1934, as amended, and files
periodic reports with the U.S. Securities and Exchange Commission.
For more information, visit www.mustangbio.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 licensing arrangements, 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, each 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 value.
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;
risks relating to the timing of starting and completing clinical
trials; uncertainties relating to preclinical and clinical testing;
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:Fortress Biotech, Inc.Jaclyn Jaffe,
Investor Relations(781) 652-4500ir@fortressbiotech.com
Fortress Biotech Media RelationsLaura Bagby6 Degrees(312)
448-8098lbagby@6degreespr.com
______________________________
1 Response rates are based on the number of evaluable
patients.
2 First renal response evaluation point was 28 days for all but
one patient, who was evaluated at 21 days.
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