Alexion Completes Acquisition of Syntimmune
Business Wire 2-Nov-2018 11:07 AM
- Acquisition adds clinical-stage anti-FcRn antibody SYNT001 to
company's growing pipeline -
- SYNT001 is first and only anti-FcRn asset currently in clinical
development for warm autoimmune hemolytic anemia (WAIHA) -
Alexion
Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the
previously announced acquisition of Syntimmune has been successfully
completed. The acquisition adds to the company's growing pipeline with
the addition of clinical-stage SYNT001, a humanized monoclonal antibody
that inhibits the interaction of neonatal Fc receptor (FcRn) with
Immunoglobulin G (IgG) and IgG immune complexes with the potential to
treat a number of rare IgG-mediated diseases.
"We've made significant progress rebuilding our pipeline this year, and
the acquisition of Syntimmune is another critical step in continuing to
expand and diversify our portfolio," said Ludwig Hantson, Ph.D., Chief
Executive Officer of Alexion. "We're very excited to continue the
development of SYNT001, which we believe holds great promise in
transforming patient care across a number of rare IgG-mediated diseases.
We are rapidly advancing the current development programs and look
forward to beginning two pivotal programs next year, including one in
warm autoimmune hemolytic anemia."
SYNT001 is currently being evaluated in Phase 1b/2a studies in patients
with warm autoimmune hemolytic anemia (WAIHA) and in patients with
pemphigus vulgaris (PV) or pemphigus foliaceus (PF) and has demonstrated
proof of mechanism showing rapid IgG reduction. Alexion plans to
initiate two pivotal trials in 2019 – one in WAIHA, following successful
completion of the current Phase 1b/2a study, and one in an undisclosed
indication.
About FcRn
Antibodies play an important role in a healthy body's defense by
fighting infections from bacteria and other invaders. In autoimmune
diseases, however, the body mistakenly attacks itself through the
production of pathogenic (disease-causing) antibodies of the
Immunoglobulin G (IgG) subtype. Neonatal Fc receptor (FcRn) rescues IgGs
from lysosomal degradation by binding them to endosomes and returning
them to the bloodstream. This helps prolong the half-life of IgG. In
healthy individuals, this function contributes to a normal immune
response. In many autoimmune conditions, however, FcRn prevents
lysosomal degradation of pathogenic IgGs associated with driving the
disease. Therefore, blocking the FcRn-IgG interaction has the potential
to drive degradation of IgG within cells and rapidly reduce circulating
pathogenic IgG.
About WAIHA
Warm autoimmune hemolytic anemia (WAIHA) is a rare autoimmune disorder
caused by pathogenic Immunoglobulin G (IgG) antibodies that react with
and cause the premature destruction of red blood cells at normal body
temperature. The disease is often characterized by profound, and
potentially life-threatening anemia and other acute complications,
including severe and life-threatening hemolysis, severe weakness,
enlarged spleen and/or liver, rapid heart rate (tachycardia), chest
pain, heart failure and fainting (syncope). There are approximately
65,000 patients across the United States, France, Germany, Italy, Spain
and the United Kingdom. There are currently no approved treatments for
WAIHA.
About SYNT001
SYNT001 is an investigational humanized IgG4 monoclonal antibody
optimized to inhibit FcRn binding to IgG at both neutral and acidic pH.
Studies have shown that SYNT001 rapidly facilitates clearance of IgG and
IgG circulating immune complexes (CICs), with the potential to block
innate immune responses induced by IgG and CIC, as well as inhibit
T-cell and B-cell activation in response to CIC. Additionally, studies
suggest that SYNT001 accomplishes its effects on IgG without destroying
immune cells or impacting other types of immunoglobulin. SYNT001 has the
potential to exert a rapid therapeutic effect in a wide range of
IgG-mediated autoimmune diseases.
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 the first and only
approved complement inhibitor to treat patients with paroxysmal
nocturnal hemoglobinuria (PNH), 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 Forbes list of the World's Most Innovative Companies seven
years in a row and is headquartered in Boston, Massachusetts' Innovation
District. The company also 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 contains forward-looking statements for purposes of
the Private Securities Litigation Reform Act of 1995, including
statements related to: the benefits of SYNT001, the potential of SYNT001
to treat a number of rare IgG-mediated diseases, the potential benefits
of targeting FcRn, SYNT001 holds great promise in transforming patient
care across a number of rare IgG-mediated diseases, the acquisition of
Syntimmune is a critical step in expanding and diversifying Alexion's
portfolio, Syntimmune provides the opportunity to transform patient care
in diseases like WAIHA, Alexion's clinical development plans, and the
potential benefits of the transaction. Forward-looking statements are
subject to factors that may cause Alexion's results and plans to differ
materially from those forward-looking statements, including for example,
the technology acquired from Syntimmune may not confer the expected
therapeutic benefits (particularly with respect to treatment of
IgG-mediated diseases), future clinical trials of SYNT001 may not prove
that the therapy is safe and effective to the level required by
regulators, decisions of regulatory authorities regarding the adequacy
of our and Syntimmune's research and clinical tests, marketing approval
or material limitations on the marketing of products, delays, 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 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 our product
candidates could be delayed or terminated prior to completion, the
adequacy of our pharmacovigilance and drug safety reporting processes,
delay of collection or reduction in reimbursement due to adverse
economic conditions or changes in government and private insurer
regulations and approaches to reimbursement, uncertainties surrounding
legal proceedings, company investigations and government investigations,
including investigations of Alexion by the U.S. Securities and Exchange
Commission (SEC) and U.S. Department of Justice, the risk that
anticipated regulatory filings are delayed, the risk that estimates
regarding the number of patients with IgG-mediated diseases (including
WAIHA) are inaccurate, risks related to the acquisition of Syntimmune
and other acquisitions and co-development efforts, 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 September 30, 2018
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.
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