- Phase 2 study met primary objective,
supporting initiation of two parallel Phase 3 studies that will
enroll ~370 AL amyloidosis patients -
- Positive long-term Phase 1a/1b data presented
at the International Symposium on Amyloidosis (ISA) 2020
demonstrate prolonged overall survival (78% at 37 months) and
durable organ response -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) and Caelum
Biosciences today announced the initiation of the Cardiac Amyloid
Reaching for Extended Survival (CARES) Phase 3 clinical program to
evaluate CAEL-101, a first-in-class amyloid fibril targeted
therapy, in combination with standard-of-care (SoC) therapy in AL
amyloidosis. The CARES clinical program includes two parallel Phase
3 studies – one in patients with Mayo stage IIIa disease and one in
patients with Mayo stage IIIb disease – and will collectively
enroll approximately 370 patients globally. Enrollment is underway
in both studies. The primary objective of the clinical program is
to assess overall survival.
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“In AL amyloidosis, misfolded amyloid proteins can build up in
many organs throughout the body, including the heart and kidneys,
causing significant damage to these organs and impairing their
function. While current treatments address the bone marrow disorder
that creates the misfolded amyloid proteins, there are no approved
therapies for the significant organ damage the disease causes,”
said John Orloff, M.D., Executive Vice President and Head of
Research and Development at Alexion. “CAEL-101 has the potential to
be the first treatment to target and remove the amyloid deposits
from these organs. Data from Phase 1 studies suggest that this
treatment approach may improve organ function and long-term
survival. We look forward to investigating this further in the
Phase 3 clinical program.”
“AL amyloidosis is particularly devastating when it affects the
heart, with median survival in these patients of less than one year
following diagnosis,” said Michael Spector, President and Chief
Executive Officer of Caelum. “Long-term survival data from AL
amyloidosis patients treated with CAEL-101 in the Phase 1a/1b study
showed that 78 percent were still alive after a median follow-up
time of more than three years. We recognize the urgent need for new
treatments that address the organ damage caused by AL amyloidosis
and are working together with the AL amyloidosis community and
Alexion to advance the Phase 3 clinical program as quickly as
possible.”
About the CARES Phase 3 Clinical Program
The CARES clinical program consists of two parallel
double-blind, randomized, event-driven global Phase 3 studies,
which are evaluating the efficacy and safety of CAEL-101 in AL
amyloidosis patients who are newly diagnosed and naïve to standard
of care (SoC) treatment (cyclophosphamide-bortezomib-dexamethasone
(CyBorD) chemotherapy). One study is enrolling approximately 260
patients with Mayo stage IIIa disease and one study is enrolling
approximately 110 patients with Mayo stage IIIb disease. The
studies will be conducted at approximately 70 sites across North
America, the United Kingdom, Europe, Israel, Japan, and
Australia.
In each study, participants are being randomized in a 2:1 ratio
to receive either CAEL-101 plus SoC or placebo plus SoC once weekly
for four weeks. This will be followed by a maintenance dose
administered every two weeks until the last patient enrolled
completes at least 50 weeks of treatment. Patients will continue
follow-up visits every 12 weeks.
The primary study objectives are overall survival and the safety
and tolerability of CAEL-101. Key secondary objectives will assess
functional improvement in the six-minute walk test (6MWT), quality
of life measures (Kansas City Cardiomyopathy Questionnaire Overall
Score & Short Form 36 version 2 Physical Component Score) and
cardiac improvement (Global Longitudinal Strain, or GLS).
Phase 2 Study Results
The Phase 2 open-label dose escalation study was conducted to
investigate higher doses of CAEL-101 than had been evaluated in
Phase 1 studies with a primary objective to identify the best dose
to advance into Phase 3 development. The study evaluated the safety
and tolerability of CAEL-101 in 13 AL amyloidosis patients at three
study sites who received up to 1000 mg/m2 of CAEL-101 (two times
the Phase 1 dose) administered in combination with SoC treatment.
The study met its primary objectives, supporting the safety and
tolerability of CAEL-101 and the selection of the 1000 mg/m2 dose
for the Phase 3 study.
Phase 1a/1b Long-Term Follow-Up Results Presented at ISA
2020
As previously reported, the Phase 1a/1b study of CAEL-101 was
the first clinical trial to demonstrate improvement in cardiac
function via GLS after treatment with an amyloid fibril targeted
therapy in AL amyloidosis patients with amyloid cardiac
involvement. New long-term follow-up data from the Phase 1a/1b
study will be presented at the virtual International Symposium on
Amyloidosis (ISA), September 14 to 18, 2020, in the poster titled,
“Long term follow-up of patients with AL amyloidosis treated on a
phase 1 study of Anti-Amyloid Monoclonal Antibody CAEL-101”
(Abstract #342, Divaya Bhutani, M.D., et. al, Columbia University
Medical Center). These data demonstrate 78 percent survival (15/19)
at a median follow-up of more than three years (37 months) in AL
amyloidosis patients treated with CAEL-101 as well as durable organ
response among evaluable patients, further supporting the
advancement of CAEL-101 into Phase 3 development.
About CAEL-101
CAEL-101 is a first-in-class monoclonal antibody (mAb) designed
to improve organ function by reducing or eliminating amyloid
deposits in the tissues and organs of patients with AL amyloidosis.
The antibody is designed to bind to misfolded light chain protein
and amyloid and shows binding to both kappa and lambda subtypes. In
a Phase 1a/1b study, CAEL-101 demonstrated improved organ function,
including cardiac and renal function, in 27 patients with relapsed
and refractory AL amyloidosis who had previously not had an organ
response to standard of care therapy. CAEL-101 has received Orphan
Drug Designation from both the U.S. Food and Drug Administration
and European Medicine Agency as a therapy for patients with AL
amyloidosis.
About AL Amyloidosis
AL amyloidosis is a rare systemic disorder caused by an
abnormality of plasma cells in the bone marrow. Misfolded
immunoglobulin light chains produced by plasma cells aggregate and
form fibrils that deposit in tissues and organs. This deposition
can cause widespread and progressive organ damage and high
mortality rates, with death most frequently occurring as a result
of cardiac failure. Current standard of care includes plasma cell
directed chemotherapy and autologous stem cell transplant, but
these therapies do not address the organ dysfunction caused by
amyloid deposition, and up to 80 percent of patients are ineligible
for transplant.
AL amyloidosis is a rare disease but is the most common form of
amyloidosis. There are approximately 22,000 patients across the
United States, France, Germany, Italy, Spain and the United
Kingdom. AL amyloidosis has a one-year mortality rate of 47
percent, 76 percent of which is caused by cardiac amyloidosis.
About Alexion
Alexion is a global biopharmaceutical company focused on serving
patients and families affected by rare diseases and devastating
conditions through the discovery, development and commercialization
of life-changing medicines. As a leader in rare diseases for more
than 25 years, Alexion has developed and commercializes two
approved complement inhibitors to treat patients with paroxysmal
nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic
syndrome (aHUS), as well as the first and only approved complement
inhibitor to treat anti-acetylcholine receptor (AchR)
antibody-positive generalized myasthenia gravis (gMG) and
neuromyelitis optica spectrum disorder (NMOSD). Alexion also has
two highly innovative enzyme replacement therapies for patients
with life-threatening and ultra-rare metabolic disorders,
hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D)
as well as the first and only approved Factor Xa inhibitor reversal
agent. In addition, the company is developing several
mid-to-late-stage therapies, including a copper-binding agent for
Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for
rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D
inhibitor as well as several early-stage therapies, including one
for light chain (AL) amyloidosis, a second oral Factor D inhibitor
and a third complement inhibitor. Alexion focuses its research
efforts on novel molecules and targets in the complement cascade
and its development efforts on the core therapeutic areas of
hematology, nephrology, neurology, metabolic disorders and
cardiology. Headquartered in Boston, Massachusetts, Alexion has
offices around the globe and serves patients in more than 50
countries. This press release and further information about Alexion
can be found at: www.alexion.com.
[ALXN-P]
About Caelum Biosciences
Caelum Biosciences, Inc. (“Caelum”) is a clinical-stage
biotechnology company developing treatments for rare and
life-threatening diseases. Caelum’s lead asset, CAEL-101, is a
novel antibody for the treatment of patients with amyloid light
chain (“AL”) amyloidosis. In 2019, Caelum entered a collaboration
agreement with Alexion under which Alexion acquired a minority
equity interest in Caelum and an exclusive option to acquire the
remaining equity in the company based on Phase 3 CAEL-101 data.
Caelum was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For
more information, visit www.caelumbio.com.
Forward-Looking Statement
This press release contains forward-looking statements that
involve risks and uncertainties relating to future events and the
future performance of Alexion and Caelum, including statements
related to: the safety and efficacy CAEL-101 as a treatment for AL
amyloidosis; CAEL-101 has the potential to be the first treatment
to target and remove the amyloid deposits from the heart, kidney
and other organs; data from the Phase 1 studies suggest that the
treatment approach may improve organ function and long-term
survival and enrollment of the Phase 3 trials. Forward-looking
statements are subject to factors that may cause Alexion's and
Caelum’s results and plans to differ materially from those expected
by these forward looking statements, including for example: the
anticipated safety profile and the benefits of the CAEL-101 may not
be realized (and the results of the clinical trials may not be
indicative of future results); the inability to enroll and complete
the Phase 3 trial; results of clinical trials may not be sufficient
to satisfy regulatory authorities; results in clinical trials may
not be indicative of results from later stage or larger clinical
trials (or in broader patient populations); the possibility that
results of clinical trials are not predictive of safety and
efficacy and potency of our products (or we fail to adequately
operate or manage our clinical trials) which could cause us to
discontinue sales of the product (or halt trials, delay or prevent
us from making regulatory approval filings or result in denial of
approval of our product candidates); the severity of the impact of
the COVID-19 pandemic on Alexion’s or Caelum’s business, including
on commercial and clinical development programs; unexpected delays
in clinical trials; unexpected concerns regarding products and
product candidates that may arise from additional data or analysis
obtained during clinical trials or obtained once used by patients
following product approval; future product improvements may not be
realized due to expense or feasibility or other factors; delays
(expected or unexpected) in the time it takes regulatory agencies
to review and make determinations on applications for the marketing
approval of our products; inability to timely submit (or failure to
submit) future applications for regulatory approval for our
products and product candidates; inability to timely initiate (or
failure to initiate) and complete future clinical trials due to
safety issues, IRB decisions, CMC-related issues, expense or
unfavorable results from earlier trials (among other reasons);
future competition from biosimilars and novel products; decisions
of regulatory authorities regarding the adequacy of our research,
marketing approval or material limitations on the marketing of our
products; delays or failure of product candidates to obtain
regulatory approval; delays or the inability to launch product
candidates due to regulatory restrictions, anticipated expense or
other matters; interruptions or failures in the manufacture and
supply of our products and our product candidates; failure to
satisfactorily address matters raised by regulatory agencies
regarding our products and product candidates; uncertainty of
long-term success in developing, licensing or acquiring other
product candidates or additional indications for existing products;
the adequacy of our pharmacovigilance and drug safety reporting
processes; failure to protect and enforce our data, intellectual
property and proprietary rights and the risks and uncertainties
relating to intellectual property claims, lawsuits and challenges
against us; the risk that third party payors (including
governmental agencies) will not reimburse for the use of our
products at acceptable rates or at all; delay of collection or
reduction in reimbursement due to adverse economic conditions or
changes in government and private insurer regulations and
approaches to reimbursement; adverse impacts on supply chain,
clinical trials, manufacturing operations, financial results,
liquidity, hospitals, pharmacies and health care systems from
natural disasters and global pandemics, including COVID-19 and a
variety of other risks set forth from time to time in Alexion's
filings with the SEC, including but not limited to the risks
discussed in Alexion's Quarterly Report on Form 10-Q for the period
ended June 30, 2020 and in their other filings with the SEC.
Alexion disclaims any obligation to update any of these
forward-looking statements to reflect events or circumstances after
the date hereof, except when a duty arises under law.
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Alexion Contacts: Media Megan Goulart,
857-338-8634 Executive Director, Corporate Communications
Investors Chris Stevo, 857-338-9309 Head of Investor
Relations
Caelum Contacts: Company Michael Spector,
President & Chief Executive Officer mspector@caelumbio.com
Jaclyn Jaffe and William Begien Investor Relations (781)
652-4500 info@caelumbio.com
Media Tony Plohoros 6 Degrees (908) 591-2839
tplohoros@6degreespr.com
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