RIVIERA BEACH, Fla. – November 1st, 2017
Statistically significant results confirm the mechanism of action of Altemia™ for the treatment of SCD Primary and secondary endpoints were met.
A clinically meaningful reduction of vaso-occlusive crises (VOCs) was observed in the top line results. No treatment related serious adverse events (SAEs) were observed Majority of patients elected to continue treatment in the open label extension (OLE) Micelle Bio Pharma, Inc. today announced positive top line results from a clinical study evaluating the efficacy and safety of Altemia, an oral soft gelatin dosage form, in pediatric sickle cell patients aged 5-17 years (https://clinicaltrials.gov/ct2/show/NCT02973360).
Sickle cell disease is characterized by an imbalance of certain fatty acids in blood cell membranes resulting in an increase in blood cell adhesion, chronic inflammation, increased coagulation activity and red blood cell hemolysis, all factors that lead to pain episodes, VOCs, and organ damage. Based on research, Micelle Bio Pharma developed Altemia, a combination of specific lipids formulated with the Company’s proprietary Advanced Lipid Technologies® platform (ALT®), to restore the appropriate balance to blood cell membranes affected by the disease.
The primary endpoint was the measurement of the change from baseline compared to placebo in blood cell membranes’ fatty acids concentration. Statistical significance was achieved within 4 weeks in patients treated with Altemia.
Statistically significant improvements in markers of coagulation (D-Dimer), inflammation (C-Reactive Protein) and adhesion (E-selectin), key elements associated with the clinical manifestations of SCD, were seen after 8 weeks of treatment. A clinically meaningful reduction of VOC was also observed. No treatment related SAEs were reported.
Ninety-four percent (94%) of subjects completed the study and the majority have chosen to participate in the open label extension phase that will continue monitoring the safety and effectiveness of the drug.
“An effective and safe treatment of Sickle Cell Disease has been elusive for decades. The findings from this successful double blinded randomized controlled multi-center clinical study may lead to a new treatment which is safe and effective for patients worldwide with this devastating disease,” said Dr. Frederick D. Sancilio of Micelle Bio Pharma.
“A safe and well tolerated treatment, presented as a small soft gelatin capsule, administered once a day, will be a major and novel breakthrough in the treatment of Sickle Cell Disease. The clinical efficacy and safety profile of Altemia clearly warrants further advancement of the program,” said Adrian L. Rabinowicz, MD, former Chief Medical Officer of Micelle Bio Pharma.
Additional analysis of the data is ongoing, and Micelle Bio Pharma plans to present detailed data from this study in peer reviewed journals and upcoming scientific conferences. The Company plans to meet with the U.S. Food and Drug Administration (FDA) as well as European Medicines Agency (EMA) to address next steps for Altemia.
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 cells’ tendency to adhere to each other, resulting in episodic occlusions of blood vessels, reperfusion damage and excruciating pain. Ultimately, many children develop organ damage and strokes. There are approximately 100,000 cases of SCD in the United States and treatment options are limited. The cost of care for this group may exceed $5 billion.
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 Micelle Bio Pharma 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, VOCs 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 VOCs 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 Micelle Bio Pharma, Inc.
Micelle Bio Pharma, Inc. 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. Micelle Bio Pharma 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.