- Highly potent, specific, and oral factor D
inhibitors effectively block hemolysis of paroxysmal nocturnal
hemoglobinuria (PNH) cells and mitigate the accumulation of C3
fragments –
Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
today announced that a submitted abstract has been accepted for
oral presentation at the 57th Annual Meeting of the American
Society of Hematology (ASH) in Orlando, FL, December 5 – 8, and for
inclusion in the 2016 Highlights of ASH. The abstract entitled,
“Small Molecule Factor D Inhibitors Block Complement Activation in
Paroxysmal Nocturnal Hemoglobinuria and Atypical Hemolytic Uremic
Syndrome,” details research conducted by Dr. Robert Brodsky and his
team at Johns Hopkins University School of Medicine, with highly
potent and specific factor D inhibitors from Achillion’s complement
inhibitor platform.
Abstracts for the conference are available
online at http://www.hematology.org/Annual-Meeting/.
Milind Deshpande, Ph.D., President and Chief
Executive Officer of Achillion, commented, “The alternative pathway
within the complement system plays a significant role in multiple
serious and underserved diseases. This research conducted by Dr.
Brodsky and his associates demonstrates that Achillion’s compounds
effectively block the breakdown of PNH cells and mitigate the
accumulation of C3 fragments on cells which leads to extravascular
hemolysis. Furthermore, the effect observed with these inhibitors
for aHUS was observed in the first reliable human, in vitro model
of the disease, which was developed by Dr. Brodsky and his
colleagues.”
Dr. Deshpande further stated, “Achillion’s
factor D inhibitor compounds represent a novel approach to
potentially treat patients with complement-related rare diseases,
many of which are directly caused by dysregulation of the
alternative pathway, including orphan diseases such as PNH and
aHUS, as well as for higher prevalence indications such as dry
age-related macular degeneration.”
Abstract #275
Title: Small Molecule Factor D
Inhibitors Block Complement Activation in Paroxysmal Nocturnal
Hemoglobinuria and Atypical Hemolytic Uremic
SyndromeSession Name: 101. Red Cells and
Erythropoiesis, Structure and Function, Metabolism, and Survival,
Excluding Iron: Heme and Anemia Session Date:
Sunday, December 6, 2015 Session Time: 4:30 PM -
6:00 PM Presentation Time: 5:30 PM
Room: Orange County Convention Center, W414AB
Complement Symposium at ASH
2015
The Company will be hosting a Complement
Symposium on Sunday, December 6, 2015 in Orlando, FL, during the
2015 ASH Annual Meeting. A webcast replay of the event will be
available from Achillion’s investor website,
http://ir.achillion.com beginning Sunday, December 6, 2015 at 11:00
p.m. Eastern time.
Reprints of the oral presentation and symposium
slides will all be made available for download from the Company’s
website, http://www.achillion.com following the presentations at
ASH.
About Complement Factor D
Platform
Achillion has leveraged its internal discovery
capabilities and a novel complement-related platform to develop
oral inhibitors of complement factor D. Factor D is an essential
serine protease involved in the complement pathway, a part of the
innate immune system. Achillion’s complement platform is focused on
advancing compounds that inhibit factor D, can be
orally-administered, and can potentially be used in the treatment
of immune-related diseases in which complement plays a critical
role. Potential indications being evaluated for these compounds
include paroxysmal nocturnal hemoglobinuria (PNH), atypical
hemolytic uremic syndrome (aHUS), myasthenia gravis, and dry
age-related macular degeneration (dry AMD). Achillion anticipates
that its platform could play a role in addressing the needs of all
PNH patients, including patients who have suboptimal response to,
or fail to respond to, the currently available treatment, as well
as for patients suffering from other complement-mediated
diseases.
About Achillion
Pharmaceuticals
Achillion is seeking to apply its expertise in
biology and structure-guided design with a deep understanding of
patient and clinician needs to develop innovative treatment
solutions aimed at improving patients’ lives. The company’s
scientific excellence, integrated capabilities and experienced team
position it to successfully achieve its goal of advancing new
products along the entire continuum from the bench to the patient.
Achillion’s pipeline is currently focused on small molecule
therapeutics for chronic hepatitis C viral infection (HCV) and
complement-related diseases through the inhibition of complement
factor D. More information is available at
http://www.achillion.com.
Cautionary Note Regarding
Forward-Looking Statements
This press release includes forward-looking
statements within the meaning of the Private Securities Litigation
Reform Act of 1995 that are subject to risks, uncertainties and
other important factors that could cause actual results to differ
materially from those indicated by such forward-looking statements,
including statements with respect to the Company’s plans to present
poster presentations about its complement factor D inhibition
program at the ASH annual meeting in 2015 and the potential
therapeutic benefits of its complement factor D compounds under
development. Achillion may use words such as “expect,”
“anticipate,” “project,” “intend,” “plan,” “aim,” “believe,”
“seek,” “ estimate,” “can,” “focus,” “will,” and “may” and similar
expressions to identify such forward-looking statements. Among the
important factors that could cause actual results to differ
materially from those indicated by such forward-looking statements
are risks relating to, among other things Achillion’s ability to:
advance the preclinical and clinical development of its drug
candidates, including complement factor D inhibitors, under the
timelines it projects in current and future preclinical studies and
clinical trials; obtain and maintain patent protection for its drug
candidates and the freedom to operate under third party
intellectual property; demonstrate in any current and future
clinical trials the requisite safety, efficacy and combinability of
its drug candidates; obtain and maintain necessary regulatory
approvals; establish commercial manufacturing arrangements;
identify, enter into and maintain collaboration agreements with
appropriate third-parties; compete successfully with other
companies that are seeking to develop improved therapies for the
treatment of HCV; manage expenses; manage litigation; raise the
substantial additional capital needed to achieve its business
objectives; and successfully execute on its business strategies.
These and other risks are described in the reports filed by
Achillion with the U.S. Securities and Exchange Commission,
including its Quarterly Report on Form 10-Q for the quarter ended
June 30, 2015, and its subsequent SEC filings.
In addition, any forward-looking statement in
this press release represents Achillion’s views only as of the date
of this press release and should not be relied upon as representing
its views as of any subsequent date. Achillion disclaims any duty
to update any forward-looking statement, except as required by
applicable law.
Company Contact:
Glenn Schulman
Achillion Pharmaceuticals, Inc.
Tel. (203) 624-7000
gschulman@achillion.com
Investors:
Mary Kay Fenton
Achillion Pharmaceuticals, Inc.
Tel. (203) 624-7000
mfenton@achillion.com
Investors:
Tricia Truehart
The Trout Group, LLC
Tel. (646) 378-2953
ttruehart@troutgroup.com
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