Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
today announced initiation of patient dosing in a Phase 2
open-label study of ACH-4471, Achillion’s first
orally-administered, small molecule factor D inhibitor, for
patients with paroxysmal nocturnal hemoglobinuria (PNH). This proof
of concept study will assess the efficacy, safety, and
pharmacokinetics of ACH-4471 in untreated patients with PNH.
“We are pleased to have begun this trial in PNH
patients to evaluate ACH-4471, the first orally-administered factor
D inhibitor to have demonstrated complement alternative pathway
(AP) inhibition in humans, which represents a potentially novel and
unique mechanistic approach to treating this life-threatening
disease,” commented Milind Deshpande, Ph.D., President and Chief
Executive Officer of Achillion.
"As an oral, small molecule inhibitor of factor
D, ACH-4471 has a unique mechanism of action to address the needs
of PNH patients. By blocking the amplification loop of the
complement alternative pathway, ACH-4471 has the potential to
control intravascular hemolysis as well as prevent the development
of extravascular hemolysis," said Professor Peter Browett, BMedSci,
MBChB, FRACP, FRCPA; Department of Molecular Medicine and
Pathology, University of Auckland, Auckland, New Zealand.
The primary objective of the study is to assess
the change-from-baseline in serum lactate dehydrogenase (LDH)
levels, a sensitive biomarker for intravascular hemolysis, during
28 days of dosing. The protocol allows for intra-patient
dose-escalation with patients initially receiving 100 mg three
times daily of ACH-4471 with the ability to increase dosage during
the treatment period. Secondary endpoints being assessed include
changes in hemoglobin and red blood cell levels, complement pathway
biomarkers, such as Bb and factor D, levels, pharmacokinetics, and
safety. Interim results from this study are anticipated during the
second quarter of 2017.
David Apelian, M.D., Ph.D., Chief Medical
Officer, commented, "In addition to PNH, we look forward to
evaluating ACH-4471 in a Phase 2 trial for patients with C3
glomerulopathy, a disease due to over activation of the AP, that we
plan initiate during the second half of this year. Furthermore, as
we continue to evaluate the PK/PD profile of ACH-4471, we are also
pursuing extended release formulations that have the potential of
achieving desired exposures with less frequent dosing, potentially
just once daily.”
Further information on this study can be found
on www.clinicaltrials.gov. Study identifier NCT03053102.
About Achillion’s Complement Alternative
Pathway (AP) Factor D Inhibitor Platform
Achillion has leveraged its internal discovery
capabilities and a novel complement-related platform to develop
small molecule factor D inhibitor compounds that target the
complement AP. Factor D is an essential serine protease involved in
the AP, a part of the innate immune system. Achillion's complement
platform is focused on seeking to advance small molecule compounds
that inhibit factor D and can potentially be used in the treatment
of immune-related diseases in which the AP plays a critical role.
Potential indications currently being evaluated for these compounds
include paroxysmal nocturnal hemoglobinuria (PNH), C3
glomerulopathy (C3G), and geographic atrophy (GA), an advanced form
of age-related macular degeneration (AMD).
About Paroxysmal Nocturnal
Hemoglobinuria (PNH)
PNH is thought to be caused by a mutation
resulting in the absence of receptors normally present on red blood
cells (RBCs) that interact with the AP. The AP of the complement
system typically functions normally in these patients but due to
the lack of key receptors, known as CD55 and CD59, on the surface
of PNH RBCs, the AP treats these cells as foreign and destroys them
via hemolysis in the circulatory system (intravascular) and in the
liver or spleen (extravascular). Complement factor D is a critical
protein within the amplification loop of the AP and it is believed
that inhibiting it could control the AP response. Furthermore, this
mechanism of action represents a potentially distinct and unique
therapeutic approach for controlling intravascular and
extravascular hemolysis associated with PNH.
About Achillion
Pharmaceuticals
Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) is
a science-driven, patient-focused company seeking to leverage its
strengths across the continuum from discovery to commercialization
in its goal of providing better treatments for people with serious
diseases. The company employs a highly-disciplined discovery and
development approach that has allowed it to pursue best-in-class
oral antiviral therapy for chronic hepatitis C (HCV) and build a
platform of potent and specific complement factor D inhibitors for
AP-mediated diseases. Achillion is rapidly advancing its efforts to
become a fully-integrated pharmaceutical company with a goal of
bringing life-saving medicines to patients with rare diseases. 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.
Achillion may use words such as “expect,” “anticipate,” “project,”
“target,” “intend,” “plan,” “aim,” “believe,” “seek,” “estimate,”
“can,” “could,” “focus,” “will,” “look forward,” “goal,” “may,”
“potential,” and similar expressions to identify such
forward-looking statements. These forward-looking statements also
include statements about: the potential benefits of, and potential
indications for, Achillion’s compounds that inhibit factor D,
including the potential for ACH-4471 to treat PNH and C3G; the
timing for interim results from the Phase 2 study of ACH-4771 in
PNH; and statements concerning Achillion’s strategic goals,
efforts, plans, and prospects, including those relating to ACH-4771
and its complement factor D inhibitor program. 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 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 third-parties, including the current collaboration
with Janssen; compete successfully in the markets in which it seeks
to develop and commercialize its product candidates and future
products; 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 Annual Report on
Form 10-K for the fiscal year ended December 31, 2016, and any
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.
Investors & Media:
Glenn Schulman, PharmD, MPH
Executive Director, Investor Relations
Achillion Pharmaceuticals, Inc.
Tel. (203) 752-5510
gschulman@achillion.com
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