SAN DIEGO, Jan. 30, 2013 /PRNewswire/ -- ADVENTRX
Pharmaceuticals, Inc. (NYSE MKT: ANX) today announced that it has
initiated patient recruitment in its pivotal phase 3 clinical study
of ANX-188 (purified poloxamer 188) in sickle cell
disease.
Santosh Vetticaden, Chief Medical Officer, said: "The
substantial effort that went into the design of this study is
reflected by the enthusiasm that we have heard from thought leaders
and clinical sites who wish to participate. In particular,
the study's primary endpoint, in which crisis resolution is based
on the time at which subjects receive their last dose of parenteral
opioid analgesic, provides a level of objectivity, specificity and
reliability that is unattainable with endpoints based on pain
scores or time to hospital discharge. We believe that ANX-188 has
the potential to be the first FDA-approved product for sickle cell
disease vaso-occlusive crisis in fifteen years."
Brian M. Culley, Chief Executive
Officer, said: "ADVENTRX now is the only company with a new
molecular entity in phase 3 for the treatment of sickle cell
disease, a disease with significant unmet needs and which has been
experiencing unprecedented levels of interest from both strategic
partners and financial investors. We believe ANX-188 is
well-positioned for regulatory and commercial success due in part
to:
- Orphan drug designation by FDA for sickle cell disease, which
is expected to provide ANX-188 with seven years of post-approval
exclusivity in the U.S.;
- Fast track designation by FDA for sickle cell disease, which
makes ANX-188 eligible for accelerated approval and rolling review
and likely to be considered appropriate for priority review;
- A competitive landscape in which there are no drugs approved to
treat an on-going vaso-occlusive crisis;
- The potential to receive a priority review voucher under the
rare pediatric disease incentive program, which voucher entitles
the holder to priority (6-month) review of a new drug or biologics
license application and which voucher can be sold prior to being
used. This year, we plan to seek designation of ANX-188 as a
drug for a rare pediatric disease."
About EPIC (Evaluation of Purified 188 In Crisis)
The EPIC study is a randomized, double-blind, two-arm,
placebo-controlled study that will be conducted at approximately 40
sites, primarily in the U.S. The primary objective is to
demonstrate that ANX-188 reduces the duration of vaso-occlusive
crisis in patients with sickle cell disease. The duration of
vaso-occlusive crisis will be measured from the time a subject is
randomized to the time at which the subject receives the last dose
of parenteral opioid analgesic for the treatment of vaso-occlusive
crisis prior to hospital discharge. A total of 388 subjects
ages 8 to 17 who have sickle cell disease and are experiencing
acute pain typical of vaso-occlusive crisis will be enrolled.
Using a two-sided alpha of 0.05, the study has approximately 90%
power to detect a 16-hour difference between treatment arms.
Secondary endpoints will compare re-hospitalization rate (for
vaso-occlusive crisis) within 14 days of initial discharge from the
hospital and the occurrence of acute chest syndrome within 120
hours of randomization.
About ADVENTRX Pharmaceuticals
ADVENTRX Pharmaceuticals is a biopharmaceutical company
developing proprietary product candidates to treat various diseases
and conditions. The Company's lead product candidate, ANX-188, has
potential to reduce ischemic tissue injury and end-organ damage by
restoring microvascular function, which is compromised in a wide
range of serious and life-threatening diseases and
conditions. The Company initially is developing ANX-188 as a
treatment for complications arising from sickle cell disease. More
information can be found on the Company's web site at
www.adventrx.com.
Forward Looking Statements
ADVENTRX cautions you that statements included in this press
release that are not a description of historical facts are
forward-looking statements that are based on ADVENTRX's current
expectations and assumptions. Such forward-looking statements
include, but are not limited to, statements regarding the prospects
for ANX-188's clinical, regulatory and commercial success in sickle
cell disease, including that ANX-188 may be the first FDA-approved
product for sickle cell disease in several years, that the
orphan-drug designation for poloxamer 188 (purified) is expected
provide ANX-188 with seven years of post-approval exclusivity in
the U.S., that fast track designation may result in a shortened
timeframe for FDA review of a new drug application for ANX-188, and
the potential for ANX-188 to be designated a drug for a rare
pediatric disease and for ADVENTRX to receive a priority review
voucher. Among the factors that could cause or contribute to
material differences between ADVENTRX's actual results and those
indicated from the forward-looking statements are risks and
uncertainties inherent in ADVENTRX's business, including, but not
limited to: the risk that the rate of enrollment in the EPIC study
is slower than was anticipated prior to the study's initiation,
including as a result of delays in opening study sites and
difficulties in recruiting study subjects; the potential for
further delays in the commencement or completion of planned
clinical studies, including the phase 3 study of ANX-188 in sickle
cell disease, including as a result of difficulties in completing
manufacturing process development activities, manufacturing
sufficient quantities of clinical trial material, meeting
applicable regulatory requirements for clinical trial material,
meeting applicable requirements of institutional review boards
overseeing clinical study sites, negotiating agreements with
potential clinical study sites, enrolling study subjects or being
subject to a "clinical hold"; the impact of missing or imputed data
on the treatment effect observed in the prior phase 3 study of
ANX-188 in sickle cell disease; the risk of suspension or
termination of a clinical study, including due to lack of adequate
funding or patient safety concerns; ADVENTRX's reliance on contract
research organizations (CROs) and other third parties to assist in
the conduct of important aspects of EPIC and other clinical
studies, and that such third parties may fail to perform as
expected; the risk that planned clinical studies, including EPIC,
are not successfully executed and/or do not successfully
demonstrate the safety or efficacy of the investigational drug; the
risk that, even if clinical studies are successful, the FDA
determines they are not sufficient to support a new drug
application; the risk that even if clinical studies of an
investigational drug in one indication are successful, clinical
studies of the same investigational drug in another indication may
not be successful; ADVENTRX's ability to obtain additional funding
on a timely basis or on acceptable terms, or at all; the potential
for ADVENTRX to delay, reduce or discontinue current and/or planned
development activities, including clinical studies, partner its
product candidates at inopportune times or pursue less expensive
but higher-risk and/or lower-return development paths if it is
unable to raise sufficient additional capital as needed; the risk
that the FDA does not grant marketing approval of ADVENTRX's
product candidates, including ANX-188, on a timely basis, or at
all; and other risks and uncertainties more fully described in
ADVENTRX's press releases and periodic filings with the Securities
and Exchange Commission. ADVENTRX's public filings with the
Securities and Exchange Commission are available at
www.sec.gov.
You are cautioned not to place undue reliance on forward-looking
statements, which speak only as of the date when made. ADVENTRX
does not intend to revise or update any forward-looking statement
set forth in this press release to reflect events or circumstances
arising after the date hereof, except as may be required by
law.
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SOURCE ADVENTRX Pharmaceuticals, Inc.