Market Overview

AltemiaTM Achieves Successful Clinical Results in Pediatric Patients with Sickle Cell Disease (SCD)

  • Statistically significant results confirm the mechanism of action of
    AltemiaTM 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)

Sancilio Pharmaceuticals Company, Inc. (SPCI) today announced positive
top line results from a clinical study evaluating the efficacy and
safety of AltemiaTM, an oral soft gelatin dosage form, in
pediatric sickle cell patients aged 5-17 years (

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, SPCI 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 AltemiaTM.

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 wordlwide with this devastating
disease," said Dr. Frederick D. Sancilio, President and Chief Executive
Officer of SPCI.

"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 AltemiaTM clearly warrants further
advancement of the program," said Adrian L. Rabinowicz, MD, Chief
Medical Officer of SPCI.

Additional analysis of the data is ongoing, and SPCI 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 AltemiaTM.

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.

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 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 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,
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 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:

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