Achillion Announces First Dosing in Phase 1 Multiple Ascending Dose Study of ACH-5228 Next-Generation Oral Factor D Inhibitor...
January 31 2019 - 6:00AM
- ACH-5228, with improved potency and half-life,
increases alternative pathway inhibition -- Achillion planning
Investigational New Drug (IND) submission in fourth quarter 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 dosing of the first healthy volunteer in a Phase 1
multiple ascending dose (MAD) study of ACH-5228, one of the
company’s next-generation oral, small-molecule factor D inhibitors.
In the completed single ascending dose study,
ACH-5228 demonstrated enhanced potency as well as improved
pharmacokinetic properties compared to the first-generation factor
D inhibitor, ACH-4471. These attributes are projected to provide
greater inhibition of the alternative pathway (AP) and reduced
dosing frequency for patients with immune-related diseases
associated with the AP of the complement system.
“This important clinical milestone for our
next-generation oral factor D inhibition program marks an exciting
time at Achillion as we pursue safe, effective and innovative
treatments for patients with complement-mediated diseases,” said
Steven Zelenkofske D.O., Executive Vice President, Chief Medical
Officer at Achillion. “With ACH-5228’s profile, we aim to evolve
our oral factor D portfolio to enable alternative pathway
inhibition throughout a variety of rare diseases. I look forward to
working with our clinical development team to potentially broaden
treatment options for patients who may benefit by increased
therapeutic availability.”
About the ACH-5228 Phase 1 MAD
Study This Phase 1 trial is a randomized,
placebo-controlled, multiple-ascending dose study of oral ACH-5228
administered to healthy volunteers outside the United States.
Approximately 38 individuals are expected to be enrolled in four
separate cohorts. The primary endpoint for the trial is an
evaluation of safety and tolerability. Secondary endpoints include
assessments of pharmacokinetics (PK), pharmacodynamics (PD), and
evaluation of alternative pathway inhibition biomarkers to
establish a PK/PD relationship for ACH-5228. Achillion anticipates
completing the study in the third quarter of 2019 and submitting an
IND to the U.S. Food and Drug Administration supporting continued
development for ACH-5228 in the fourth quarter of 2019.
About the Achillion Complement Factor D
PortfolioAchillion has leveraged its internal discovery
capabilities and a novel complement-related platform to develop
oral small molecule drug candidates that are inhibitors of
complement factor D. Factor D is an essential serine protease
involved in the AP of the complement system, a part of the innate
immune system. Achillion's complement platform is focused on
seeking to advance oral small molecules that inhibit the AP and can
potentially be used in the treatment of immune-related diseases in
which complement AP plays a critical role. Potential indications
currently being evaluated for these compounds include paroxysmal
nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G), and immune
complex-mediated membranoproliferative glomerulonephritis
(IC-MPGN).
About Achillion
PharmaceuticalsAchillion Pharmaceuticals,
Inc. (Nasdaq: ACHN) is a clinical-stage biopharmaceutical
company focused on advancing its oral factor D inhibitors
into late-stage development and commercialization. Achillion
is initially focusing its drug development activities on
alternative pathway-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 mediated membranoproliferative
glomerulonephritis (IC-MPGN). Each of the product candidates in the
Company’s oral factor D 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 Quarterly Report on Form 10-Q for the
fiscal quarter ended September 30, 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 & Media: Brian Di Donato Vice
President, Investor Relations and Corporate Communications
Achillion Pharmaceuticals, Inc. Tel. (215) 709-3032
bdidonato@achillion.com
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