Alexion Completes Acquisition of Achillion
January 28 2020 - 9:29AM
Business Wire
– Acquisition adds two clinical-stage Factor D
inhibitors to Alexion’s pipeline and provides promising development
platform for additional complement-mediated diseases –
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced it
has completed its acquisition of Achillion Pharmaceuticals, Inc.
The acquisition adds two clinical-stage oral small molecule Factor
D inhibitors to Alexion’s pipeline and provides the foundation and
expertise for a broader oral Factor D inhibition development
platform with the potential to treat numerous additional
complement-mediated diseases.
“The acquisition of Achillion adds two clinical-stage Factor D
inhibitors to our growing pipeline, representing important
continued momentum in expanding and diversifying our portfolio and
advancing our mission of transforming the lives of people with rare
diseases,” said Ludwig Hantson, Ph.D., Chief Executive Officer of
Alexion. “We believe oral Factor D inhibition holds great promise
in treating people with multiple rare, complement-mediated
diseases, providing the opportunity to significantly expand our
portfolio into new therapeutic areas and to help many more
patients. We look forward to the expertise the Achillion team
brings to Alexion, which, combined with our own complement biology
and rare disease development and commercialization experience, will
enable us to collectively accelerate progress of the development
programs. We are committed to maintaining continuity in the
programs currently underway and will be moving quickly to advance
Achillion's efforts.”
Alexion will continue development of Achillion’s oral Factor D
inhibitor portfolio, which includes two clinical-stage
medicines-in-development – danicopan (ACH-4471) and ACH-5228 – as
well as multiple compounds in preclinical development. Phase 3
development is being initiated for danicopan as an add-on therapy
for PNH patients with extravascular hemolysis (EVH). Danicopan is
also in Phase 2 development for C3G, and ACH-5228 is in Phase 2
development for PNH.
About Factor D Factor D is an essential serine protease
and critical control point in the alternative pathway (AP) of the
complement system, a part of the innate immune system. Achillion’s
complement platform is focused on advancing oral small molecules
that inhibit the AP and can potentially be used in the treatment of
immune-related diseases in which complement AP plays a critical
role. Potential indications currently being evaluated for these
compounds include paroxysmal nocturnal hemoglobinuria (PNH) and C3
glomerulopathy (C3G).
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic,
progressive, debilitating and life-threatening ultra-rare blood
disorder characterized by hemolysis (destruction of red blood
cells) that is mediated by uncontrolled activation of the
complement system, a component of the body’s immune system.
Patients with PNH may experience a wide range of signs and
symptoms, such as fatigue, difficulty swallowing, shortness of
breath, abdominal pain, erectile dysfunction, dark-colored urine
and anemia. The most devastating consequence of chronic hemolysis
is thrombosis, which can occur in blood vessels throughout the
body, damaging vital organs and causing premature death. PNH is
primarily a disease of intravascular hemolysis (IVH), where the red
blood cell destruction occurs within the blood vessels. C5
inhibition addresses the complications of IVH and the increases in
LDH that cause thrombosis and even death in patients with PNH.
However, a small portion of patients – less than 10 percent –
receiving a C5 inhibitor continue to experience clinical
extravascular hemolysis (EVH), where the red blood cell destruction
occurs outside the blood vessels. As a result, these patients are
transfusion dependent despite treatment but do not have bone marrow
failure or aplastic anemia. Inhibiting Factor D in the alternative
pathway (AP) of the complement system offers the possibility of
selectively blocking AP activity and protecting against the
destruction of red blood cells, while leaving the rest of the
complement system intact to fight infection.
About C3 Glomerulopathy (C3G) C3G is an ultra-rare kidney
disease for which there is no approved treatment. The disease is
characterized by the deposition of C3 protein fragments in the
filtering units (glomeruli) of the kidney, caused by overactivation
of the complement alternative pathway (AP). Over time, the chronic
deposition of C3 fragments results in permanent kidney damage and
kidney failure. Today, C3G patients are treated with steroids and
broad-acting immunosuppressants to slow the progression of kidney
damage. Oral Factor D inhibitors have demonstrated
proof-of-mechanism to interrupt the overactivation of the AP and
reduce C3 fragment deposition, providing a potential treatment
approach for targeting the underlying cause of C3G.
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 medicines. 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) 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). 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 anti-FcRn therapy, 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-G]
Forward-Looking Statement This press release includes
forward-looking statements related to Alexion’s acquisition of
Achillion, including: the acquisition provides the foundation for a
broader oral Factor D inhibition development platform with the
potential to treat numerous additional complement-mediated
diseases; the acquisition of Achillion represents important
continued momentum in expanding and diversifying our portfolio;
oral Factor D inhibition holds great promise in treating multiple
rare, complement-mediated diseases, providing the opportunity to
significantly expand our portfolio into new therapeutic areas; we
are committed to maintaining continuity in the programs currently
underway and will be moving quickly to accelerate Achillion’s
efforts; and Alexion will continue development of Achillion’s oral
Factor D inhibitor portfolio. A number of important factors could
cause actual results to differ materially from those indicated by
such forward-looking statements, including: the anticipated
benefits of the Achillion platform and therapies not being
realized; future clinical trials of Achillion products not proving
that the therapies are safe and effective to the level required by
regulators; decisions of regulatory authorities regarding the
adequacy of the research and clinical tests, marketing approval or
material limitations on the marketing of Achillion products; delays
or failure of product candidates to obtain regulatory approval;
delays or the inability to launch product candidates due to
regulatory restrictions; unanticipated expenses; interruptions or
failures in the manufacture and supply of products and product
candidates; failure to satisfactorily address matters raised by the
FDA and other regulatory agencies; the possibility that results of
clinical trials are not predictive of safety and efficacy results
of products in broader patient populations; the possibility that
clinical trials of product candidates could be delayed or
terminated prior to completion for a number of reasons; the
adequacy of pharmacovigilance and drug safety reporting processes;
and a variety of other risks set forth from time to time in
Alexion’s or Achillion’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 September 30, 2019 and in its other
filings with the SEC and the risks discussed in Achillion’s
Quarterly Report on Form 10-Q for the period ended September 30,
2019 and in its 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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version on businesswire.com: https://www.businesswire.com/news/home/20200128005516/en/
Alexion Contacts: Media Megan Goulart,
857-338-8634 Senior Director, Corporate Communications
Investors Susan Altschuller, Ph.D., 857-338-8788 Vice
President, Investor Relations
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