ACH-4471, First Generation Oral factor D Inhibitor- Proof of
Concept Validated in both PNH as Monotherapy and in Combination
w/C5 Inhibitor - Proof of Mechanism Validated in C3G, End of
Phase 2 Meeting Targeted for 4Q 2019
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN), a clinical-stage
biopharmaceutical company dedicated to transforming the lives of
patients and families affected by complement mediated disorders,
today reported interim results for the Company’s Phase 2 trials of
its first-generation oral factor D inhibitor, ACH-4471, as well as
Phase 1 pharmacokinetics and potency data for its next-generation
factor D inhibitors, ACH-5228 and ACH-5548.
“These compelling findings underscore the momentum for Achillion
and our factor D portfolio as we advance three oral small molecule
compounds through clinical development. Our oral factor D
inhibitors have the potential to treat patients suffering from
alternative pathway mediated diseases,” said Joe Truitt, President
and Chief Executive Officer at Achillion. “In C3G, we have
completed patient dosing in our 14-day biomarker/dose ranging study
and we are currently enrolling patients in our 6 and 12-month PoC
trials. We now have 18 clinical sites open globally and plan to
open additional sites in 2019. We are also delighted that the FDA
has recently agreed to allow adolescents in our trials as C3G is a
disease with unmet medical needs that often begins in childhood.
Our C3G plan is to enroll up to 20 patients in our 6 and 12-month
trials and to present our data to the FDA at an End of Phase 2
meeting in Q4 2019.”
Truitt continued, “The PNH trial data show that factor D
inhibition may play an important role in the future treatment
paradigm for PNH patients. Our trials have demonstrated a positive
impact on patient’s hemoglobin, reticulocyte counts, LDH,
FACIT-fatigue scores and reduced blood transfusions both as a
monotherapy and in combination with a C5 inhibitor. Our hypothesis
has been reinforced that if the alternative pathway is adequately
inhibited then patient benefit can be achieved in fundamentally
different ways than has been seen with C5 inhibitors. We believe
this is an unmet medical need and a market segment we will continue
to evaluate. Additionally, based on the Phase 1 pharmacokinetic and
potency data, our next generation compounds, ACH-5228 and ACH-5548,
allow for higher alternative pathway inhibition along with a
reduced dosing frequency. These compounds have the potential to be
transformative both for patients and Achillion and to deliver on
the promise of alternative pathway inhibition across a wide
spectrum of diseases.”
The Company also confirmed today an expected YE 2018 cash and
marketable securities balance of approximately $270 million and a
projected 2019 cash burn of $80-85 million.
ACH-4471, Complement Factor D Inhibitor
for PNH and C3G
The Company’s first-generation oral complement factor D
inhibitor, ACH-4471, is being evaluated for safety and efficacy
with Phase 2 clinical programs in both PNH and C3G and has
demonstrated preliminary proof-of-concept in both indications.
The C3G program consists of two Phase 2 clinical
trials which are currently recruiting, a six-month blinded,
placebo-controlled study, and a 12-month open label study.
The PNH program consists of a Phase 2 clinical
trial evaluating ACH-4471 in patients who are inadequately
controlled or sub-optimally responding to eculizumab. Additionally,
we continue to dose patients in our PNH monotherapy extension
trial.
More information is available at
http://www.achillion.com/patients-and-clinicians/
ACH-5228 and ACH-5548, Next Generation
Complement Factor D Inhibitors
ACH-5228 and ACH-5548 are our next-generation
oral factor D inhibitors currently in Phase 1 clinical trials.
These compounds demonstrated enhanced potency as well as improved
pharmacokinetic properties that allow for higher alternative
pathway inhibition along with a reduced dosing frequency.
Achillion to Host Conference Call and Webcast Today at
4:30 p.m. EST
The live audio and subsequent archived webcasts
of the Company's presentations will be accessible from the
Company's investor relations website:
http://ir.achillion.com. The audio recording will be
archived for 30 days following the live presentation. Please
connect to Achillion's website several minutes prior to the start
of the presentation to ensure adequate time for any software
downloads that may be necessary.
Alternatively, the webcast and audio can be
accessed directly as follows:
Webcast Link: https://edge.media-server.com/m6/p/kd6w84c6 US
Toll-Free Dial-In Number: (866) 205-4820International Dial-In
Number: (419) 386-0004Conference ID# 5987613
About Achillion
Pharmaceuticals
Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN)
is a clinical-stage biopharmaceutical company focused on
advancing its oral factor D inhibitors into late-stage
development and commercialization. Factor D is an essential
serine protease involved in the alternative pathway of the
complement system, a part of the innate immune system. 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 patients, payors,
regulators and healthcare professionals. 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,” “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; 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 DonatoVice
President, Investor Relations and Corporate CommunicationsAchillion
Pharmaceuticals, Inc.Tel. (215) 709-3032bdidonato@achillion.com
Achillion Pharmaceuticals (NASDAQ:ACHN)
Historical Stock Chart
From Apr 2024 to May 2024
Achillion Pharmaceuticals (NASDAQ:ACHN)
Historical Stock Chart
From May 2023 to May 2024