The study assessed the safety and effectiveness of Altemia, a combination of lipids the company formulated with its Advanced Lipid Technologies platform specifically to treat SCD. Lipids are fats and fatty substances that the body uses as energy sources.
SCD refers to a group of red blood cell disorders that leads to faulty hemoglobin, a protein in the red cells that carries oxygen. The dysfunction destroys certain lipids in the walls of red blood cells that are essential to the body’s normal functioning.
Red blood cells that contain normal hemoglobin are disc-shaped, like a doughnut, but red blood cells with defective hemoglobin look like a sickle, or crescent shape.
The abnormalities lead to inflammation and increase the tendency of red and white blood cells to stick to each other. This results in episodes of vaso-occlusion (VOC), or obstructed blood vessels, pain and organ damage.
Altemia replenishes the lost lipids to prevent the cascade from starting. The lipids restore balance and fluidity to red blood cells and other cells impacted by SCD. They may also decrease chronic inflammation, prevent blood cells from sticking to each other, and reduce the rupturing of red blood cells. The bursting, known as hemolysis, releases hemoglobin into blood plasma.
By preventing the cascading, Altemia pre-empts VOC crises, pain and organ damage.
The therapy is in a small soft gelatin capsule that is intended to be taken once a day. Altemia’s formulation and mechanism of action delivers a narrow dose of crucial lipids directly to the membrane of SCD patients’ red blood cells, Sancilio says.
Patients who complete the Phase 2 trial are eligible to continue receiving Altemia in an open-label extension study. It will collect long-term safety data and continue tracking the drug’s effectiveness.
Most patients who completed the trial have chosen to continue receiving Altemia in the extension study.
“I want to thank the many doctors, nurses and researchers who have flawlessly executed this challenging protocol,” Frederick D. Sancilio, president and chief executive officer of Sancilio Pharmaceuticals, said in a press release.
“We are very pleased to have had 97% of the randomized patients complete the trial,” he said. “Additionally, we are encouraged to see a high degree of engagement and enthusiasm by patients to enroll in the open-label extension study.”
Sancilio completed enrollment in the SCOT trial in July 2017. In September, the U.S. Food and Drug Administration granted Rare Pediatric Disease designation to the soft gelatin Altemia capsules. The designation supports pharmaceutical companies that are developing drugs and biologics for rare diseases.
The FDA defines a rare pediatric disease as a serious or life-threatening disorder that primarily affects people younger than 18 and that impacts fewer than 200,000 individuals in the U.S.