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 reported interim data from a Phase 2 paroxysmal nocturnal
hemoglobinuria (PNH) trial assessing the safety and effectiveness
of its oral small molecule factor D inhibitor ACH-4471 in
combination with intravenous eculizumab at The New Era of Aplastic
Anemia and PNH Meeting in Naples, Italy.
“Anemia is a persistent problem in the majority
of patients with PNH treated with standard and even high doses of
eculizumab. These interim data are encouraging and demonstrate
that ACH-4471, when used in combination with a C5 inhibitor, such
as eculizumab, has the potential to improve anemia, decrease
transfusions and lead to improvement in important clinical
parameters of hemolysis as well as quality of life measurements for
patients with this devastating condition,” stated Joseph Truitt,
Chief Executive Officer at Achillion.
ACH-4471 Interim Data This
proof-of-concept, 24-week trial is ongoing. Interim data in 11
enrolled patients were assessed between 4 to 24 weeks, depending on
the patient’s current treatment duration in the dose escalating
trial. The oral presentation features the following data:
- Increases in mean hemoglobin of approximately 2 g/dL at week 4
(n=11); for the 4 patients that have reached 24 weeks their mean
rise in hemoglobin is 2.6 g/dL;
- A reduction in blood transfusions from 34 transfusions totaling
58 units in the 24 weeks prior to screening to only 1 transfusion
of 2 units during treatment with ACH-4471;
- Meaningful improvement in Functional Assessment of Chronic
Illness Therapy (FACIT) fatigue scores versus baseline, with a mean
score increase of 11 at week 4;
- Increase in the percentage of PNH RBC Type III clone size from
40% at baseline to 71% at week 12 (n=8);
- Reduction in total bilirubin from a mean of 2.17 mg/dL to 1.21
mg/dL at week 16 (n=8);
- Reduction in mean reticulocytes from 219 10^9/uL at baseline to
153 10^9/uL at week 16 (n=8);
- Further reduction of LDH into the normal range;
- Four patients are currently receiving the lowest study dose of
100 mg three times a day;
- ACH-4471 was generally well tolerated when added to eculizumab
in patients with PNH.
“In patients treated with C5 inhibitors alone,
extravascular hemolysis (EVH) is an ongoing problem resulting in
anemia with an impact on most of the clinical parameters of
hemolysis. These interim findings from this important
proof-of-concept trial show that if the alternative pathway is
adequately inhibited, important parameters of EVH can be improved,
including and most importantly hemoglobin, transfusion, bilirubin,
reticulocyte count, and FACIT fatigue scores, when added to stable
optimal dose eculizumab therapy. We look forward to completing
the Phase 2 trial this summer and future discussions with the
regulatory authorities. After completion of regulatory discussions,
we hope to initiate a Phase 3 PNH combination trial of ACH-4471
with C5 inhibitors in the first half of 2020,” stated Steven
Zelenkofske, D.O. Chief Medical Officer at Achillion.
Slides from the oral presentation will be
available on the Company’s website, today, May 17, at approximately
9:00 a.m. EST at
http://ir.achillion.com/events-and-presentations.
ACH-4471 Phase 2 Trial in Combination
with EculizumabACH-4471 is being evaluated in combination
with eculizumab, an intravenous C5 inhibitor that is currently
approved as monotherapy for PNH. This is a Phase 2, open-label,
multiple dose trial in adult patients on stable eculizumab
treatment with blood transfusion dependent anemia, defined as
receiving at least one transfusion in the 12 weeks prior to the
study and a hemoglobin level below 10 g/dL. In addition to their
usual dose of eculizumab, patients are administered ACH-4471 orally
three times a day at a dose determined by patient clinical
response. The primary outcome of the trial is the change in
hemoglobin at 24 weeks compared to baseline. Secondary
outcomes include the number of blood transfusions required, impact
on selected clinical parameters, and safety. The trial will be
followed by a long-term extension phase.
About Paroxysmal Nocturnal Hemoglobinuria
(PNH)
PNH is thought to be caused by a mutation resulting in the
absence of receptors normally present on red blood cells (RBCs)
that interact with the complement system. The complement system
typically functions normally in these patients but due to the lack
of key receptors, known as CD55 and CD59, on the surface of PNH
RBCs, the complement system treats these cells as foreign and
destroys them via hemolysis in the circulatory system
(intravascular) and in the liver or spleen (extravascular). The
complement alternative pathway (AP) is a critical factor in the
development of extravascular hemolysis. Complement factor D is a
critical protein within the amplification loop of the AP and it is
believed that inhibiting it could control the AP response.
Furthermore, this mechanism of action represents a potentially
distinct and unique therapeutic approach for controlling
intravascular and extravascular hemolysis associated with PNH.
More information is available
at http://www.achillion.com/patients-and-clinicians/.
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 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, including ACH-4471 for PNH in combination with
eculizumab; 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; the status of
enrollment in Achillion’s ongoing clinical trials; 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; Achillion’s
expectations regarding the timing of regulatory interactions and
filings; Achillion’s anticipated cash expenditures for 2019 and the
sufficiency of its existing cash resources; 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; 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 March 31, 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.
Investors: Brian Di Donato Senior VP, Chief
Financial OfficerTel. 215-709-3032 bdidonato@achillion.com
Media: Susanne Heinzinger Senior VP, Corporate
Communications Tel. 215-709-3055 sheinzinger@achillion.com
Source: Achillion Pharmaceuticals, Inc.
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