Achillion Initiates Phase 1 Study of ACH-4471, First Orally-Administered Small Molecule Complement Factor D Inhibitor
February 11 2016 - 06:05AM
Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN)
today announced the initiation of a phase 1 study with ACH-4471,
the Company’s first orally-administered, highly potent and specific
small molecule that has been shown to inhibit complement factor D.
The phase 1 study is being conducted in healthy volunteers and is
evaluating single ascending doses of ACH-4471 to assess safety,
tolerability, pharmacokinetics (PK) and pharmacodynamics (PD). The
Company expects to report interim clinical data from this study
during the second quarter of 2016.
“We believe our molecular-level understanding of
factor D, based upon proprietary high-resolution x-ray structures,
has enabled us to design ACH-4471 with the potential to be the
first orally-bioavailable, highly differentiated treatment for PNH
and other ultra-rare diseases. We look forward to advancing
ACH-4471, with the goal of demonstrating its safety, tolerability,
proof-of-mechanism and working to establish its potential benefit
for patients,” commented Milind Deshpande, Ph.D., President and
Chief Executive Officer.
Dr. Deshpande further stated, “In addition to
ACH-4471, we have developed a substantial library of complement
factor D inhibitors and are now working to advance distinct small
molecule candidates with traits appropriate for ophthalmic
administration, to potentially treat dry AMD, and for inhalation,
in order to address respiratory indications such as COPD.”
ACH-4471 Phase 1 Healthy Volunteer
Development Program
The initial phase 1 trial is a randomized,
placebo-controlled, single-ascending dose study of ACH-4471
administered to healthy volunteers. Approximately 28 subjects are
expected to be enrolled. The primary endpoint for the trial is
evaluation of safety and tolerability. Secondary endpoints include
assessments of PK, PD, and evaluation of alternative pathway
inhibition in ex vivo laboratory assessments of blood samples from
subjects in order to establish a PK/PD relationship for ACH-4471.
Interim study results are anticipated during the second quarter of
2016. In addition, a second phase 1 study evaluating
multiple-ascending doses in healthy volunteers is expected to be
initiated in the second quarter of 2016 with interim results
anticipated during the third quarter of 2016.
About Complement Factor D
Platform
Achillion has leveraged its internal discovery
capabilities and a novel complement-related platform to develop
drug candidates that are 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 seeking to advance 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), dry age-related macular
degeneration (dry AMD), and chronic obstructive pulmonary disease
(COPD). 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 treatments, as well as for patients suffering from other
alternative pathway complement-mediated diseases. Achillion
nominated ACH-4471 for clinical development in December 2015, and
initiated clinical development in February 2016 with a phase 1
healthy volunteer trial assessing single-ascending doses of
ACH-4471.
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 inhibitors. 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,”
“intend,” “plan,” “aim,” “believe,” “seek,” “ estimate,” “can,”
“focus,” “will,” “look forward,” “goal,” and “may” and similar
expressions to identify such forward-looking statements. These
forward-looking statements also include statements about the:
potential for ACH-4471 to be the first orally-bioavailable, highly
differentiated treatment for PNH and other ultra-rare diseases; the
Company’s plans to advance complement factor D inhibitor drug
candidates to treat other conditions such as dry AMD, aHUS and
COPD; the Company’s plans to release interim results from the phase
1 healthy volunteer study of ACH-4471 during the second quarter of
2016 and initiate a multiple-ascending phase 1 study during the
second quarter of 2016; the Company’s expectations about the
potential therapeutic benefits of its complement factor D inhibitor
program; and the Company’s strategic goals, plans and prospects.
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 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; 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 Quarterly Report on Form 10-Q for the quarter ended
September 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.
Investors:
Glenn Schulman
Achillion Pharmaceuticals, Inc.
Tel. (203) 624-7000
gschulman@achillion.com
Media:
Liz Power
Achillion Pharmaceuticals, Inc.
Tel: (203) 752-5509
lpower@achillion.com
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