Sancilio Pharmaceuticals Company, Inc. Announces The Successful Completion Of A Randomized, Double Blind, And Placebo Controlled Phase Of A Clinical Trial In Pediatric Patients With Sickle Cell Disease Using Altemia
Published: Oct 03, 2017
Open Label Extension Phase Continues
RIVIERA BEACH, Fla.--(BUSINESS WIRE)--Sancilio Pharmaceuticals Company, Inc. (SPCI) today announced the successful completion of a randomized, double blinded, placebo controlled clinical trial in pediatric sickle cell patients aged 5 – 17 years old. The clinical trial is a multi-center study designed to evaluate treatment effects and safety of AltemiaTM, a novel pharmaceutical preparation utilizing the Company’s Advanced Lipid Technologies (ALT®) platform. The Company expects to announce top-line data, including Altemia’s impact on the frequency of vaso-occlusive events, in the fourth quarter of 2017. Patients who have completed the study are eligible to continue active drug therapy by entering an open label extension program that will collect long term safety data while continuing to monitor the effectiveness of the drug (https://clinicaltrials.gov/ct2/show/NCT02973360). At present, the majority of patients completing the study have chosen to continue to participate in the open label extension.
“I want to thank the many doctors, nurses and researchers who have flawlessly executed this challenging protocol”
"I want to thank the many doctors, nurses and researchers who have flawlessly executed this challenging protocol," said Dr. Frederick D. Sancilio, President and Chief Executive Officer of SPCI. "We are very pleased to have had 97% of the randomized patients complete the trial. Additionally, we are encouraged to see a high degree of engagement and enthusiasm by patients to enroll in the open label extension study.”
About Sickle Cell Disease (SCD)
Sickle Cell Disease (SCD) is a group of genetic disorders that results in dysfunctional hemoglobin (HbS) and a depletion of certain lipids in the walls of blood cells. These abnormalities create an inflammatory state and an increase in the red and white blood cell’s tendency to adhere to each other, resulting in episodic occlusions of blood vessels, reperfusion damage and excruciating pain. Ultimately, many children develop end organ damage and strokes. There are approximately 100,000 cases of SCD in the United States. Treatment options are limited. The cost of care for this group may exceed $5BB.
Altemia™ is our proprietary product candidate that is being developed for the treatment of SCD. Altemia™ consists of a complex mixture of lipids formulated using Advanced Lipid Technologies® (ALT®) specifically to address the treatment of the disease. The drug is encapsulated in a small soft gelatin capsule and intended to be taken once daily to reduce VOC episodes, anemia, organ damage and other disease complications in sickle cell patients.
HbS destroys specific lipids, creating a cascade that culminates in VOC episodes. Altemia™ is designed to replenish those lipids in order to prevent the cascade effect from initiating.
Based on research performed by Sancilio Pharmaceuticals Company, Inc. (SPCI) and others, the specific lipids contained in Altemia™, may restore balance and fluidity to red blood cells and other cells impacted by the disease. We believe that Altemia™ will treat sickle cell disease by decreasing blood cell adhesion, chronic inflammation and red blood cell hemolysis, the factors that lead to reduction in pain episodes, VOC crises and organ damage. Based on its formulation and mechanism of action, we believe that Altemia™ is well-positioned to deliver a narrow, therapeutic dose of certain lipids directly to the membrane of red blood cells of sickle cell patients. The combination of ALT® drug delivery technology and highly purified lipids may reduce VOC significantly. We also believe that Altemia™ has the potential to address the inflammatory symptoms of SCD and to assist in reducing sickle cell events in general. By minimizing damage, Altemia™ may be able to reduce sickle cell crisis events and related mortality.
About Sancilio Pharmaceuticals Company, Inc.
Sancilio Pharmaceuticals Company, Inc. (SPCI) is a fully integrated, specialty pharmaceutical company focused on developing, manufacturing and commercializing pharmaceutical products, including those based on our proprietary Advanced Lipid Technologies® (ALT®) platform. SPCI is pursuing treatments for sickle cell disease, short bowel syndrome and severe hypertriglyceridemia. We utilize our cGMP compliant facility to develop and manufacture our products. Our ALT® platform is designed to enhance the bioavailability, reduce the food effect and improve the efficacy of lipids and lipophilic active pharmaceutical ingredients (APIs). Lipids are hydrophobic or amphipathic molecules, including fatty acids, steroids (including hormones) and fat-soluble vitamins (such as vitamins A, D, E and K). Our business model is to apply our ALT® platform to lipids or lipophilic APIs to create unique product candidates that address the disorders and diseases resulting from imbalances of lipids in the body. In addition to our primary focus of developing our proprietary products using the ALT® platform, we make use of, and license rights to, our proprietary ALT® platform and other technologies to third parties, providing both development and subsequent soft gelatin encapsulation services. More information is available at: www.sancilio.com.
Sancilio Pharmaceuticals Company, Inc.
Marc Wolff, 561-847-2302
Executive Vice President & Chief Financial Officer