- Orphan drug designation for the treatment of
C3G granted by the U.S. Food and Drug Administration (FDA) –
Achillion Pharmaceuticals, Inc. (Nasdaq:ACHN), a
pharmaceutical company focused on advancing small molecule
inhibitors of factor D in the complement alternative pathway, today
announced the U.S. Food and Drug Administration (FDA) has granted
orphan drug designation to ACH-4471 for the treatment of patients
with C3 Glomerulopathy (C3G).
C3G is a devastating renal disease for which
there is no approved therapy. There are estimated to be
approximately 4,000 C3G patients in the United States,
approximately 4,000 in Europe, and more than 1,000 patients with
this devastating disease in Japan.
The FDA Orphan Drug Designation program provides
incentives for the development of potentially promising drugs to
treat, diagnose or prevent orphan diseases and disorders that
affect fewer than 200,000 people in the U.S. This designation may
provide, under specified conditions, for a seven-year marketing
exclusivity period, as well as certain incentives, including
federal grants, tax credits and a waiver of PDUFA filing fees.
On November 14, 2017, Achillion announced
interim data from the first two patients in an on-going Phase 2
study in C3G patients demonstrated that the current formulation of
ACH-4471 achieved complement alternative pathway inhibition
resulting in greater than 50% reduction in proteinuria over the
14-day treatment period and a favorable tolerability profile.
Additionally, Achillion announced the initiation
of bioavailability study of extended release formulations of
ACH-4471 in healthy volunteers. The extended release oral tablet
formulations may have the potential to achieve once or twice daily
dosing in patients.
“We are pleased that the FDA has granted orphan
drug designation to ACH-4471 for treatment of C3G, and we look
forward to additional data from the ongoing extended release
formulation study. We are keenly aware of the unmet need for
patients and we are committed to advancing ACH-4471 for C3G as we
believe we have an opportunity to develop a potentially
disease-modifying therapy, based on the unique mechanism of action,
for ACH-4471,” commented Milind Deshpande, Ph.D., President and
CEO.
ACH-4471: Phase 1 Bioavailability Study
of Extended-Release Formulations
The purpose of this study is to evaluate and
compare the pharmacokinetic profiles of multiple extended-release
formulations of ACH-4471 in healthy subjects, after oral
administration, with the goal of identifying an extended-release
formulation to enable once or twice daily dosing regimens of
ACH-4471. Interim results are anticipated during the second quarter
of 2018. (EudraCT: 2017-003525-15)
About the Achillion Complement Factor D
Platform
Achillion has leveraged its internal discovery
capabilities and a novel complement-related platform to develop
small molecule 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 small molecule compounds that inhibit factor D and can
potentially be used in the treatment of immune-related diseases in
which complement alternative pathway plays a critical role.
Potential indications being evaluated for these compounds include
paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G),
immune complex-mediated membranoproliferative glomerulonephritis
(IC-MPGN), and geographic atrophy (GA).
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 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,”
“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 expected benefits of orphan drug
designation; the potential for development of an extended release
formulation for ACH-4471; the potential benefits of, and potential
indications for, Achillion’s compounds that inhibit factor D,
including ACH-4771; and statements concerning Achillion’s strategic
goals, efforts, 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 complement factor D inhibitors
under the timelines it projects in current and future preclinical
studies and clinical trials; avail itself of the benefits of orphan
drug designation for ACH-4771, because, for example, orphan drug
designation exclusivity may not prevent the FDA or other regulatory
authorities from approving competing products; develop an extended
release formulation of ACH-4471 that is able to achieve once or
twice daily dosing regimens; demonstrate in any current and future
clinical trials the requisite safety, efficacy and combinability of
its drug candidates; 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; obtain and maintain necessary regulatory
approvals, and the granting of orphan designation does not alter
the standard regulatory requirements and process for obtaining such
approval; establish commercial manufacturing arrangements;
identify, enter into and maintain collaboration and other
commercial 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
fiscal quarter ended September 30, 2017, and any other SEC filings
that Achillion makes from time to time.
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, MPHExecutive Director, Investor
RelationsAchillion Pharmaceuticals, Inc.Tel. (203)
752-5510gschulman@achillion.com
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