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:
About EPIC (Evaluation of Purified 188 In Crisis)
- 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."
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.