Pfizer Announces Enrollment Of First Patient In Phase 3 Trial In Sickle Cell Disease
RESET Trial to Assess Effectiveness and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Individuals with Sickle Cell Disease
Pfizer Inc. (NYSE:PFE) today announced that the first patient has been enrolled in the RESET (Rivipansel: Evaluating Safety, Efficacy and Time to Discharge) study – a Phase 3 clinical trial assessing the efficacy and safety of rivipansel for the treatment of vaso-occlusive crisis in hospitalized individuals with sickle cell disease who are six years of age or older.
Sickle cell disease is one of the most prevalent genetic disorders in the U.S. It is a rare and debilitating chronic disease with lifelong clinical impact and reduced life expectancy; life expectancy is 48 years for females and 42 years for males with sickle cell disease. There are more than 100,000 people in the U.S. living with sickle cell disease1, and many of them experience multiple vaso-occlusive crises each year. These painful crises result in more than 75,000 hospitalizations per year in the U.S., with an average hospital stay of approximately six days.
“Scientific innovation cannot forge ahead without the patients that are willing to work with the scientific community by participating in clinical trials,” said Sonja L. Banks, president and chief operating officer of the Sickle Cell Disease Association of America. “Patients should speak with their healthcare providers if they are interested in learning more about how to participate in a trial.”
About the RESET Trial
This phase 3, multicenter, randomized, double-blind, placebo-controlled, parallel-group study is planning to enroll at least 350 people with sickle cell disease, aged six and older who are hospitalized for a vaso-occlusive crisis and will evaluate the efficacy and safety of treatment with rivipansel. Study participants must be receiving treatment with intravenous opioids for their vaso-occlusive crisis and must be able to receive the first dose of study drug within 24 hours of initiation of intravenous opioid therapy.
The primary endpoint for the study will be time to readiness-for- discharge. Key secondary endpoints will include time to discharge, cumulative IV opioid consumption and time to discontinuation of IV opioids.
“We are pleased to enroll the first patient in the RESET trial, which will further our understanding of the potential role of rivipansel for the treatment of painful crises in patients with sickle cell disease,” said Brenda Cooperstone, MD, vice president and medicine team lead, Rare Diseases, Global Innovative Pharmaceuticals Business, Pfizer Inc. “Pfizer has a deep history in researching and bringing to market therapies for hematologic rare diseases and is committed to working to find innovative solutions to the sickle cell patient community’s unmet needs.”
About Rivipansel
Rivipansel (GMI-1070) is an investigational pan-selectin inhibitor. Selectins are a family of molecules believed to play a key role in regulating cellular interactions within blood vessels. Rivipansel is not a narcotic or a pain medication.
The intense pain associated with sickle cell vaso-occlusive crisis is believed to be due to local tissue ischemia consequent upon blockage occurring in microvascular beds and capillaries. This results from occlusion or clogging of vessels with sickled red blood cells (which become rigid and inflexible when sickled) and subsequent, selectin-driven adhesion of white blood cells (leukocytes) to the inner wall of the blood vessel (endothelium) and the recruitment of platelets; events which collectively restrict blood flow.
In 2011, Pfizer entered into a worldwide license agreement with GlycoMimetics (NASDAQ: GLYC) for the development and, if approved by applicable regulatory authorities, commercialization of rivipansel. GlycoMimetics was responsible for development up to phase 2 and Pfizer will be responsible for all future clinical development of rivipansel.
Rivipansel has received Orphan Drug and Fast Track status from the U.S. Food and Drug Administration (FDA), and this study is being conducted under a Special Protocol Assessment (SPA), in agreement with the FDA.
Pfizer and Rare Diseases
Rare diseases are among the most serious of all illnesses and impact millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact in addressing unmet medical needs. The Pfizer focus on rare diseases builds on more than a decade of experience and a global portfolio of more than 20 medicines approved worldwide that treat rare diseases in the areas of hematology, neuroscience, inherited metabolic disorders, pulmonology, and oncology.
Pfizer Inc.: Working together for a healthier world®
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world’s best-known consumer health care products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, Pfizer has worked to make a difference for all who rely on us.
DISCLOSURE NOTICE: The information contained in this release is as of June 23, 2015. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.
This release contains forward-looking information about rivipansel, including its potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including, without limitation, the ability to meet anticipated clinical trial commencement and completion dates and regulatory submission dates, as well as the possibility of unfavorable clinical trial results, including unfavorable new clinical data and additional analyses of existing clinical data; whether and when any new drug applications may be filed with any jurisdiction for rivipansel; whether and when any such applications may be approved by regulatory authorities, which will depend on the assessment by such regulatory authorities of the benefit-risk profile suggested by the totality of the efficacy and safety information submitted; decisions by regulatory authorities regarding labeling and other matters that could affect the availability or commercial potential of rivipansel; and competitive developments.
25 – June – 2015