Achillion Receives Breakthrough Therapy Designation from FDA for Danicopan for Treatment of Paroxysmal Nocturnal Hemoglobinur...
September 25 2019 - 7:00AM
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 that the U.S. Food and Drug Administration (FDA)
has granted Breakthrough Therapy designation for danicopan
(ACH-4471) for treatment in combination with a C5 monoclonal
antibody for patients with paroxysmal nocturnal hemoglobinuria
(PNH) who are sub-optimal responders to a C5 inhibitor alone.
The FDA’s decision was based on positive safety and efficacy
data from the ongoing danicopan Phase 2 PNH combination trial.
Interim data was reported at the New Era of Aplastic Anemia and PNH
Meeting in May 2019. The top line data from this combination trial
is expected in the fourth quarter of 2019.
“The FDA’s granting of Breakthrough Therapy designation for our
lead oral factor D inhibitor, danicopan, underscores the urgent
need for new treatment options for patients living with PNH,” said
Joe Truitt, President and Chief Executive Officer at Achillion.
“Danicopan, with its demonstrated ability to limit both
intravascular and extravascular hemolysis with oral administration,
has the potential to benefit a significant number of patients with
PNH that continue to have an unmet medical need on standard of
care. We appreciate the review and decision by the FDA and plan to
work closely with the Agency in advancing the development of
danicopan into Phase 3 in early 2020.”
FDA Breakthrough Therapy designation is designed
to expedite the development and review of medicines for serious or
life-threatening conditions. Receiving Breakthrough Therapy
designation from the FDA indicates preliminary clinical evidence
has demonstrated the drug may provide substantial improvement on at
least one clinically significant endpoint compared with currently
available therapy. The benefits of this Breakthrough Therapy
designation include more intensive guidance from FDA on an
efficient drug development program, access to a scientific liaison
to help accelerate review time and eligibility for Accelerated
Approval and Priority Review if relevant criteria are met.
Danicopan (ACH-4471) has previously received orphan drug
designation for the treatment of PNH in 2017.
About Paroxysmal Nocturnal
Hemoglobinuria (PNH)
PNH is a rare, acquired blood disease caused by a somatic
mutation resulting in the absence of key receptors, CD55 and CD59,
on the surface of red blood cells (RBCs). The alternative pathway
(AP) of the complement system recognizes these unprotected RBCs as
foreign and destroys them in the circulatory system (intravascular
hemolysis) and in the liver or spleen (extravascular hemolysis).
The current standard of care for PNH targets intravascular
hemolysis by inhibiting C5 complement protein (C5), leaving some
patients with persistent extravascular hemolysis from early phases
of complement activation (AP Activity) which C5 inhibition cannot
address leaving patients with partial control of their PNH. Up to
seventy-five percent of PNH patients treated with C5 inhibitors
remain anemic during treatment, with up to one-third of those
patients reporting the need for blood transfusions within the last
year. Factor D is the critical, rate-limiting protein within the
AP. By targeting Factor D, proximal AP inhibition may disable both
downstream terminal complement activation (IVH) and upstream C3
fragment opsonization (EVH). Achillion is developing a potentially
more complete approach to PNH with factor D inhibition to
selectively block alternative pathway activity and protect against
both destructive processes of RBCs in PNH with convenient oral
therapies.
More information is available at
http://www.achillion.com/patients-and-clinicians/.
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 alternative pathway (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 small molecule complement inhibitors into
late-stage development and commercialization. Research has shown
that an overactive complement system plays a critical role in
multiple 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 achieve its goal of advancing 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 include statements about: the potential benefits of
FDA’s Breakthrough Designation for danicopan; the potential
benefits of factor D inhibition as a treatment for
complement-mediated diseases, including danicopan (ACH-4471) for
PNH; Achillion’s expectations regarding the advancement of, and
timeline for reporting results from, clinical trials of its product
candidates (including danicopan and ACH-5228); Achillion’s
expectations regarding the timing of regulatory interactions and
filings; 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: continue to meet the
clinical development program criteria for Breakthrough Designation;
accelerate the development timeline for danicopan utilizing
benefits available through the Breakthrough Designation;
demonstrate in any current and future clinical trials the requisite
safety, efficacy and combinability of its product candidates,
including danicopan and ACH-5228; 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; whether interim results from a clinical trial
will be predictive of the final results of that trial or whether
results of early clinical trials or preclinical studies will be
indicative of the results of later clinical trials; enroll patients
in its clinical trials on its projected timelines; 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 quarterly
period ended June 30, 2019, 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.
Investor Relations: Clayton RobertsonAchillion
Pharmaceuticals, Inc.Tel. 215-709-3078 crobertson@achillion.com
Media: Susanne Heinzinger Senior VP, Corporate
CommunicationsAchillion Pharmaceuticals, Inc. Tel. 215-709-3032
sheinzinger@achillion.com
Source: Achillion Pharmaceuticals, Inc.
Achillion Pharmaceuticals (NASDAQ:ACHN)
Historical Stock Chart
From Mar 2024 to Apr 2024
Achillion Pharmaceuticals (NASDAQ:ACHN)
Historical Stock Chart
From Apr 2023 to Apr 2024