- Collaboration provides opportunity to diversify Alexion’s clinical-stage rare hematology portfolio -
- Caelum to receive up to
- Alexion has option to acquire Caelum based on Phase 2 data -
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“With a median survival time of less than 18 months following diagnosis
and no approved therapies to address the organ damage caused by AL
amyloidosis, there is a significant need for new treatments for this
devastating disease,” said
Under the terms of the agreement, Alexion will acquire a minority equity
interest in Caelum and an exclusive option to acquire the remaining
equity in the company based on Phase 2 data for pre-negotiated
economics. Alexion will make payments to Caelum totaling up to
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 insoluble light chain amyloid protein, including
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 the
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, gradually affecting their function. This can cause progressive and widespread 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
About Alexion
Alexion is a global biopharmaceutical company focused on serving
patients and families affected by rare diseases through the discovery,
development and commercialization of life-changing therapies. As the
global leader in complement biology and inhibition for more than 20
years, Alexion has developed and commercializes two approved complement
inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria
(PNH), as well as the first and only approved complement inhibitor to
treat atypical hemolytic uremic syndrome (aHUS) and anti-acetylcholine
receptor (AchR) antibody-positive generalized myasthenia gravis (gMG),
and is also developing it for patients with 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). In addition, the company is developing several
mid-to-late-stage therapies, including a second complement inhibitor, a
copper-binding agent for Wilson disease and an anti-neonatal Fc receptor
(FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases.
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 and metabolic
disorders. Alexion has been named to the
[ALXN-G]
About Caelum Biosciences
Caelum Biosciences, founded by
Forward-Looking Statement
This press release includes forward-looking statements, including
statements related to the therapeutic benefits of CAEL-101, the
potential of CAEL-101 as a treatment for AL amyloidosis and the
potential benefits of the collaboration. Such forward-looking statements
are subject to risks and uncertainties that could cause actual results
to differ materially from those expressed or implied in such statements.
The process by which an early stage product such as CAEL-101 could
potentially lead to an approved product for the treatment of AL
amyloidosis is long and subject to highly significant risks, including
for example, decisions of regulatory authorities regarding the adequacy
of our research, marketing approval or material limitations on the
marketing of products, delays, interruptions or failures in manufacture
and supply, failure to satisfactorily address matters raised by the
View source version on businesswire.com: https://www.businesswire.com/news/home/20190131005293/en/
Source:
Alexion:
Media
Megan Goulart, 857-338-8634
Senior
Director, Corporate Communications
Investors
Susan
Altschuller, Ph.D., 857-338-8788
Vice President, Investor Relations
Caelum:
Michael
Spector, 609-845-7088
President & Chief Executive Officer
mspector@caelumbio.com
Media
Tony
Plohoros, 908-940-0135
6 Degrees
tplohoros@6degreesPR.com