RIVIERA BEACH, Fla.--(BUSINESS WIRE)--Sancilio Pharmaceuticals Company, Inc (SPCI) will present new research data from the Phase 2 study (SCOT trial) on SC411 (Altemia™) at the 2108 American Society Pediatric Hematology and Oncology (ASPHO) meeting, being held in Pittsburgh, PA , May 2-5, 2018.
“The results of the SCOT study being presented at ASPHO improve our understanding of how Altemia may address several key manifestations of Sickle Cell Disease (SCD) that help inform our selection of endpoints for a pivotal study in children afflicted with this devastating disease,” said Geoffrey Glass, SPCI’s Chief Executive Officer.
Data being presented on Altemia include:
EFFECTS OF SC411 (Altemia™) ON BLOOD CELL MEMBRANE OMEGA-3 INDEX AND SELECT SICKLE CELL DISEASE BIOMARKERS IN THE SCOT TRIAL: A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, PARALLELGROUP, MULTI-CENTER STUDY being presented Thursday, May 3, 2018 at 6:30 pm
CLINICAL EFFECT OF SC411 (Altemia™) ON CHILDREN WITH SICKLE CELL DISEASE IN THE SCOT TRIAL: A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED, PARALLEL-GROUP, DOSE-FINDING MULTI-CENTER STUDY being presented Thursday, May 3, 2018 at 3:45 pm - 4:45 pm
Key Highlights from these presentations are:
- Sixty-two subjects (93%) completed the blinded portion of the study, with 41 opting to participate in the Open Label Extension (OLE) study
- The rate of clinical Sickle Cell Crisis (SCC) was 54% lower in the SC411 treatment groups combined versus placebo (rate ratio, 0.46; 95% confidence interval [CI]: 0.20 to 1.04; p=0.07)
- Docosahexaenoicfacid (DHA) and eicosapentaenoic acid (EPA ) levels on blood cell membrane significantly increased against baseline in all doses 4 weeks post-treatment (p<0.01)
- The optimal dose that was selected for the pivotal study and the ongoing Open Label Extension (OLE) study showed a significant reduction 8 weeks post-treatment vs. placebo in sE-selectin (p: 0.0219) and D-dimer (p: 0.025)
- Significant increases in Hemoglobin 8 weeks post treatment vs. baseline was observed in all active doses
- Significant reductions in eDiary-recorded sickle cell crises, analgesic use, opioid use, and school absences due to SCD pain were observed with SC411 treatment dose levels of 36 mg/kg and 60 mg/kgand pooled SC411 treated subjects compared to placebo
- Two treatment-related adverse events (nausea and abdominal pain) were observed in one patient. No other treatment related adverse events were observed
More detailed information on scientific sessions and data presentations at the ASPHO annual meeting can be found on the conference website (http://aspho.org/meetings/conference/overview)
SPCI plans to present additional data from the recently completed SCOT Phase 2 study in peer reviewed journals and upcoming scientific conferences. The Company plans to meet with the U.S. FDA as well as European Medicines Agency (EMA) to address next steps for Altemia.
“The pediatric population of Sickle Cell Disease patients around the world need, and deserve, more therapeutic options, and we are excited about the opportunity to gain advice from regulatory authorities on advancing Altemia in global markets,” said Adrian L. Rabinowicz, M.D, Chief Medical Officer of SPCI.
About Sickle Cell Disease (SCD)
Sickle Cell Disease (SCD) is a group of genetic disorders that results in dysfunctional hemoglobin (HbS), oxidative stress, chronic inflammation, and a depletion of certain lipids in the walls of blood cells. These abnormalities lead to an increased tendency of red and white blood cell 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 SCC episodes, anemia, organ damage and other disease complications in sickle cell patients.
HbS destroys specific lipids, creating a cascade that culminates in pain 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, SCC and organ damage. Based on its formulation and mechanism of action, we believe that Altemia™ is well-positioned to deliver an optimal 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 SCC 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.