- Accepted abstracts include new Phase 2 safety
and tolerability data for CAEL-101 in AL amyloidosis - - Phase 3
studies of CAEL-101 in AL amyloidosis are underway -
Caelum Biosciences and Alexion Pharmaceuticals, Inc.
(NASDAQ:ALXN) today announced that two abstracts on CAEL-101, a
potentially first-in-class amyloid fibril targeted therapy, have
been accepted for e-poster presentation at the European Hematology
Association (EHA) Congress 2021, taking place virtually from June 9
to 17, 2021. Both presentations will include new data from the
Phase 2 open-label dose escalation study evaluating the safety and
tolerability of CAEL-101 in combination with standard-of-care (SoC)
therapy in AL amyloidosis. One will feature data studying safety
and tolerability in AL amyloidosis patients treated with 1000 mg/m2
CAEL-101 in combination with
cyclophosphamide-bortezomib-dexamethasone plus daratumumab. The
second will include new data further supporting the selection of
the 1000 mg/m2 dose for the ongoing Phase 3 study and demonstrating
the possible stabilization of cardiac disease as assessed by Mayo
staging.
This press release features multimedia. View
the full release here:
https://www.businesswire.com/news/home/20210512006005/en/
The abstracts accepted for e-poster presentations are listed
below and are now available on the EHA website. All e-poster
presentations will be made available on the virtual congress
platform on June 11, 2021 at 09:00 a.m. CEST (3:00 a.m. EDT) and
will be available throughout the duration of the Congress.
Safety and Tolerability of CAEL-101 in Combination with
Cyclophosphamide-Bortezomib-Dexamethasone and Daratumumab in
Patients with AL Amyloidosis. Abstract #EP1017– e-poster
presentation; session title: Myeloma and other monoclonal
gammopathies – Clinical.
Safety and Tolerability of CAEL-101 in Patients with AL
Amyloidosis in a Phase 2 Study for a Median of 38 Weeks. Abstract
#EP1018 – e-poster presentation; session title: Myeloma and other
monoclonal gammopathies – Clinical.
As was previously announced, the Cardiac Amyloid Reaching for
Extended Survival (CARES) Phase 3 clinical program to evaluate
CAEL-101 in combination with SoC therapy in AL amyloidosis has
begun. Enrollment is underway in two parallel Phase 3 studies – one
in patients with Mayo stage IIIa disease (ClinicalTrials.gov
Identifier: NCT04512235) and one in patients with Mayo stage IIIb
disease (ClinicalTrials.gov Identifier: NCT04504825) – and will
collectively enroll approximately 370 patients globally. The Phase
2 program continues with the ongoing evaluation of CAEL-101 in
combination with SoC therapy plus daratumumab.
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 potential 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
systemic 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 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. Caelum was founded by Fortress
Biotech, Inc. (NASDAQ: FBIO). For more information, visit
www.caelumbio.com.
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 hematology, nephrology, neurology,
metabolic disorders, cardiology, ophthalmology and acute care.
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]
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 its products, including
statements related to: the anticipated benefits of CAEL-101
(including possible stabilization of cardiac disease as assessed by
Mayo staging); CAEL-101 is designed to improve organ function by
reducing or eliminating amyloid deposits in the tissues and organs
of patients with AL amyloidosis; CAEL-101 is designed to bind to
misfolded light chain protein and amyloid and shows binding to both
kappa and lambda subtypes; characteristics of clinical trials for
CAEL-101 including patients expected to be enrolled in the trials;
the Company continues to progress efforts to innovate for patients.
Forward-looking statements are subject to factors that may cause
Alexion's or Caelum’s results and plans to differ materially from
those expected by these forward looking statements, including for
example: CAEL-101 may not generate the expected benefits to
patients that are anticipated; anticipated regulatory approvals may
be delayed or refused; results of clinical trials may not be
sufficient to satisfy regulatory authorities to approve CAEL-101 as
a treatment for AL amyloidosis or other indication (or they may
request additional trials or additional information); results in
clinical trials may not be indicative of results from later stage
or larger clinical trials (or in broader patient populations once
the product is approved for use by regulatory agencies); the
possibility that results of clinical trials are not predictive of
safety and efficacy and potency of CAEL-101 (or failure to
adequately operate or manage clinical trials) which could cause us
to discontinue sales of the product (or halt trials, delay or
prevent submission of regulatory approval filings or result in
denial of approval of product candidates); the severity of the
impact of the COVID-19 pandemic on the businesses, including on
commercial and clinical trial and clinical development programs;
unexpected delays in clinical trials; unexpected concerns regarding
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 products; inability to timely submit (or failure to
submit) future applications for regulatory approval for 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); Alexion dependence on
sales from complement inhibitors; future competition from
biosimilars and novel products; decisions of regulatory authorities
regarding the adequacy of the research, marketing approval or
material limitations on the marketing of products; 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 products and product
candidates; failure to satisfactorily address matters raised by
regulatory agencies regarding 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 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 (including intellectual property
lawsuits relating to ULTOMIRIS brought by third parties); the risk
that third party payors (including governmental agencies) will not
reimburse or continue to reimburse for the use of our products at
acceptable rates or at all; failure to realize the benefits and
potential of investments, collaborations, licenses and
acquisitions; the possibility that expected tax benefits will not
be realized; potential declines in sovereign credit ratings or
sovereign defaults in countries where products are sold; 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 our supply
chain, clinical trials, manufacturing operations, financial
results, liquidity, hospitals, pharmacies and health care systems
from natural disasters and global pandemics, including the
coronavirus; uncertainties surrounding legal proceedings, company
investigations and government investigations; the risk that
estimates regarding the number of patients with AL amyloidosis and
other indications we are pursuing are inaccurate; the risks of
changing foreign exchange rates; risks relating to the potential
effects of the Company's restructurings; 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 quarter ended March 31, 2021
and in our 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.
Forward-Looking Statement Regarding Acquisition of Alexion by
AstraZeneca
This communication contains forward-looking statements within
the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended. You can generally identify forward-looking statements by
the use of forward-looking terminology such as “anticipate,”
“believe,” “continue,” “could,” “estimate,” “expect,” “explore,”
“evaluate,” “intend,” “may,” “might,” “plan,” “potential,”
“predict,” “project,” “seek,” “should,” or “will,” or the negative
thereof or other variations thereon or comparable terminology.
These forward-looking statements are only predictions and involve
known and unknown risks and uncertainties, many of which are beyond
Alexion’s and AstraZeneca plc’s “AstraZeneca”) control. Statements
in this communication regarding Alexion, AstraZeneca and the
combined company that are forward-looking, including anticipated
benefits of the proposed transaction, the impact of the proposed
transaction on Alexion’s and AstraZeneca’s businesses and future
financial and operating results, the amount and timing of synergies
from the proposed transaction, the terms and scope of the expected
financing for the proposed transaction, the aggregate amount of
indebtedness of the combined company following the closing of the
proposed transaction, are based on management’s estimates,
assumptions and projections, and are subject to significant
uncertainties and other factors, many of which are beyond Alexion’s
and AstraZeneca’s control. These factors include, among other
things, market factors, competitive product development and
approvals, pricing controls and pressures (including changes in
rules and practices of managed care groups and institutional and
governmental purchasers), economic conditions such as interest rate
and currency exchange rate fluctuations, judicial decisions, claims
and concerns that may arise regarding the safety and efficacy of
in-line products and product candidates, changes to wholesaler
inventory levels, variability in data provided by third parties,
changes in, and interpretation of, governmental regulations and
legislation affecting domestic or foreign operations, including tax
obligations, changes to business or tax planning strategies,
difficulties and delays in product development, manufacturing or
sales including any potential future recalls, patent positions and
the ultimate outcome of any litigation matter. Additional
information concerning these risks, uncertainties and assumptions
can be found in Alexion’s and AstraZeneca’s respective filings with
the SEC, including the risk factors discussed in Alexion’s most
recent Annual Report on Form 10-K, as updated by its Quarterly
Reports on Form 10-Q, in AstraZeneca’s most recent Annual Report on
Form 20-F and in each company’s future filings with the SEC.
Important risk factors could cause actual future results and other
future events to differ materially from those currently estimated
by management, including, but not limited to, the risks that: a
condition to the closing the proposed acquisition may not be
satisfied; a regulatory approval that may be required for the
proposed acquisition is delayed, is not obtained or is obtained
subject to conditions that are not anticipated; AstraZeneca is
unable to achieve the synergies and value creation contemplated by
the proposed acquisition; AstraZeneca is unable to promptly and
effectively integrate Alexion’s businesses; management’s time and
attention is diverted on transaction related issues; disruption
from the transaction makes it more difficult to maintain business,
contractual and operational relationships; the credit ratings of
the combined company declines following the proposed acquisition;
legal proceedings are instituted against Alexion, AstraZeneca or
the combined company; Alexion, AstraZeneca or the combined company
is unable to retain key personnel; and the announcement or the
consummation of the proposed acquisition has a negative effect on
the market price of the capital stock of Alexion or AstraZeneca or
on Alexion’s or AstraZeneca’s operating results. No assurances can
be given that any of the events anticipated by the forward-looking
statements will transpire or occur, or if any of them do occur,
what impact they will have on the results of operations, financial
condition or cash flows of Alexion or AstraZeneca. Should any risks
and uncertainties develop into actual events, these developments
could have a material adverse effect on the proposed transaction
and/or Alexion or AstraZeneca, AstraZeneca’s ability to
successfully complete the proposed transaction and/or realize the
expected benefits from the proposed transaction. You are cautioned
not to rely on Alexion’s and AstraZeneca’s forward-looking
statements. These forward-looking statements are and will be based
upon management’s then-current views and assumptions regarding
future events and operating performance, and are applicable only as
of the dates of such statements. Neither Alexion nor AstraZeneca
assumes any duty to update or revise forward-looking statements,
whether as a result of new information, future events or otherwise,
as of any future date.
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version on businesswire.com: https://www.businesswire.com/news/home/20210512006005/en/
Caelum Contacts: Company Michael Spector,
President & Chief Executive Officer mspector@caelumbio.com
Jaclyn Jaffe and Bill Begien Investor Relations (781) 652-4500
info@caelumbio.com
Media Tony Plohoros 6 Degrees (908) 591-2839
tplohoros@6degreespr.com
Alexion Contacts: Media Megan Goulart,
857-338-8634 Executive Director, Corporate Communications
Investors Chris Stevo, 857-338-9309 Head of Investor
Relations
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