Achillion Announces Completion of Enrollment in Phase 2 PNH Combination Trial
27 Março 2019 - 07:00AM
- Completed enrollment in ACH-4471 Phase 2 PNH
trial in combination with eculizumab -- Interim data to be released
in May 2019 - - Targeting FDA end-of-Phase 2 meeting in second half
of 2019 -
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN), a clinical-stage
biopharmaceutical company dedicated to transforming the lives of
patients and families affected by complement-mediated diseases,
today announced achievement of enrollment in their ACH-4471 Phase 2
paroxysmal nocturnal hemoglobinuria (PNH) trial in combination with
eculizumab.
In the Phase 2 trial, patients with an inadequate response to
eculizumab as monotherapy are being evaluated for increases in
hemoglobin and reduced transfusions over 24-weeks. Early data
released in December 2018 provided evidence that ACH-4471 in
combination with eculizumab had a positive impact on patient
hemoglobin and FACIT-fatigue scores while reducing transfusions,
reticulocyte counts and LDH levels.
The company expects to present interim data for
the first 10 patients on May 17, 2019 at The New Era of Aplastic
Anemia and Paroxysmal Nocturnal Hemoglobinuria meeting in Napoli,
Italy. This specialized meeting is sponsored by AIEPEN Onlus, the
Italian PNH Association.
“We are excited to present interim data on the
first 10 patients enrolled and to discuss these results with the
FDA,” said Steven Zelenkofske, D.O., Executive Vice President,
Chief Medical Officer at Achillion. “This is an important clinical
milestone for our first-generation oral factor D inhibitor program,
as it adds to the growing body of ACH-4471 data in PNH patients who
remain anemic or transfusion dependent while receiving eculizumab.
We believe this data supports the hypothesis that alternative
pathway inhibition can potentially provide additional benefit to
patients who are receiving C5 inhibitors, such as eculizumab.”
The data from the ACH-4471 Phase 2 combination
study along with the prior completed 10 patient Phase 2 monotherapy
study will be submitted for review by regulatory authorities in an
end of Phase 2 meeting in the second half of 2019.
About the ACH-4471 Phase 2 Combination
Study A Phase 2 open-label study of ACH-4471 in up to 12
patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have an
inadequate response to eculizumab as monotherapy. The purpose of
this dose-ranging study is to determine the effectiveness of
ACH-4471 in improving anemia, as measured by increased blood
hemoglobin, when given with eculizumab (a C5 inhibitor commonly
used for treatment of PNH) for 24 weeks. In December 2018,
Achillion announced interim data for the first four patients in
this trial, which showed trial participants reduced blood
transfusions to zero from an aggregate of 14 in the prior 52 weeks.
In addition, early data provided evidence that ACH-4471 in
combination with eculizumab had a positive impact on patient’s
hemoglobin, reticulocyte counts, LDH, and FACIT-fatigue scores. The
Company believes this data helps support its hypothesis that if the
alternative pathway is adequately inhibited, therapeutic benefit
can be achieved in fundamentally different ways than has been seen
with C5 inhibitors alone. Achillion expects to report updated
interim results for this Phase 2 clinical trial in May 2019 and is
targeting a meeting with the FDA in the second half of 2019.
About Achillion
PharmaceuticalsAchillion Pharmaceuticals,
Inc. (Nasdaq: ACHN) is a clinical-stage biopharmaceutical
company focused on advancing its oral small molecule
complement inhibitors into late-stage development and
commercialization. Research has shown that an overactive complement
system plays a critical role in a number of disease conditions
including the therapeutic areas of nephrology, hematology,
ophthalmology and neurology. Achillion is initially focusing its
drug development activities on complement-mediated diseases where
there are no approved therapies or where existing therapies are
inadequate for patients. Potential indications being evaluated for
its compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3
glomerulopathy (C3G), and immune complex membranoproliferative
glomerulonephritis (IC-MPGN). Each of the product candidates in the
Company’s oral small molecule portfolio was discovered in its
laboratories and is wholly owned. To advance its
investigational product candidates into Phase 3 clinical trials and
commercialization, the Company plans to work closely with key
stakeholders including healthcare professionals, patients,
regulators and payors. More information is available
at http://www.achillion.com.
Cautionary Note Regarding
Forward-Looking StatementsThis 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,” “continue,” “goal,” “strategy,”
“objective,” “may,” “potential,” and similar expressions to
identify such forward-looking statements. These forward-looking
statements also include statements about: the potential benefits of
factor D inhibition as a treatment for complement-mediated
diseases; the potential benefits of, and indications for,
Achillion’s compounds that inhibit factor D, including ACH-4471,
ACH-5228 and ACH-5548; Achillion’s belief that its portfolio
of compounds could expand factor D portfolio opportunities, provide
strategic optionality or create significant value; Achillion’s
expectations regarding the advancement of, and timeline for
reporting results from, clinical trials of its product candidates
as well as its ability to advance additional compounds; and other
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: demonstrate in any current and
future clinical trials the requisite safety, efficacy and
combinability of its product 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; enroll patients in its clinical trials
on its projected timelines; replicate in later stage clinical
trials favorable data demonstrated in preclinical and early-stage
clinical trials; obtain and maintain patent protection for its
product 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 Annual Report on Form 10-K for the fiscal
year ended December 31, 2018, 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: Brian Di Donato Chief Financial
OfficerTel. (215) 709-3032bdidonato@achillion.com
Media:Susanne HeinzingerSenior VP, Corporate
CommunicationsTel. (215) 709-3055sheinzinger@achillion.com
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