Market Overview

Sancilio Pharmaceuticals Company to Present New Research on Altemia™ to Treat Sickle Cell Disease at the ASPHO Conference

Share:

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.

About Altemia™

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.

View Comments and Join the Discussion!