Data from the Phase 2 SCOT trial (NCT02973360) trial were given at the 2018 American Society Pediatric Hematology and Oncology (ASPHO) meeting, held in Pittsburgh in early May, in two presentations.
The presentations were titled “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, parallel group, multi-center study,” and “Clinical effects 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.”
Altemia is a gelatin-capsule treatment that consists of a complex mixture of lipids (fats) — specifically, docosahexaenoic acid, an omega-3 fatty acid — formulated using Sancilio’s Advanced Lipid Technology (ALT). It is intended for once daily use.
The medication is designed to replenish the lipids destroyed by the abnormal hemoglobin (HbS) that is characteristic of SCD. The disease causes blood cells to deform and stick together, clogging vessels and resulting in the intensely painful and damaging episodes, and bouts of anemia, dizziness, and fatigue, known as sickle cell crises.
SCOT, a two-part trial due to conclude in July 2019, is evaluating the safety and tolerability of three different doses of the medicine in SCD children compared to a placebo group. It’s primary goal, or endpoint, is changes from study start (baseline) in the omega-3 fatty acids index in patients’ blood cells after four weeks of treatment.
Patients in part A were given Altemia at 20 mg/kg, 36 mg/kg or 60 mg/kg — or placebo — for eight weeks. For part B, they are invited to continue or start treatment in an open-label extension of the study.
Data reported increases in docosahexaenoic acid and eicosapentaenoic acid — both omega-3 fatty acids — on the membrane of blood cells in patients given any of the three Altemia doses. Whether these increases were significant was not evident in a company press release about them.
Fewer sickle cell crises — a 54 percent drop in the crisis rate — were seen in Altemia-treated patients compared to those given placebo.
A significant decrease in disease-related pain, use of pain treatments (analgesics and opioids), and missed school days were reported in patients given one of the two higher Altemia doses (36 mg/kg or 60 mg/kg) compared to placebo patients. A significant increase in hemoglobin levels in treated patients (all doses) at eight weeks was also seen.
One patient had two adverse events related to treatment, nausea and abdominal pain, but overall safety appeared good. In total, 93 percent of children in part A (62 patients) completed the study, and 41 of those 62 chose to continue in its open-label extension that runs for 19 months.
These and further trial data will be published in peer-reviewed journals and reported at upcoming scientific events, the company said.
“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 that help inform our selection of endpoints for a pivotal study in children afflicted with this devastating disease,” Geoffrey Glass, Sancilio’s chief executive officer, said in the release.
An ideal dose for a soon-to-open Phase 3 trial, also called SCOT (NCT02604368), has been selected, the company announced. That trial plans to enroll about 210 SCD patients, ages 5 to 17, at sites yet to be announced; patients will be given either oral Altemia or placebo once a day. More information, including enrollment information, is available here.
“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, Sancilio’s chief medical officer.