ADVENTRX Initiates Pivotal Phase 3 Study of ANX-188
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.
SOURCE ADVENTRX Pharmaceuticals, Inc.