2129 N Congress Ave, Riviera Beach, FL 33404 [email protected] 561.847.2302

World Sickle Cell Day

In support of World Sickle Cell Day, the Sickle Cell Disease Coalition is raising awareness of SCD in Africa and the rest of the world in an effort to improve health outcomes for people with the disease from infancy to adulthood.

SCD continues to be a major global public health issue. It remains a major killer of infants and children in the developing world, particularly in sub-Saharan Africa and India, where an estimated 50–90 percent of infants born with SCD will die before their fifth birthday.

The SCDC PSA and the global library can be found by visiting http://www.scdcoalition.org/priorities/global.html.

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

RIVIERA BEACH, Fla. – April 30th, 2018 – 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.

Contacts

Sancilio Pharmaceuticals Company, Inc.
Geoffrey Glass, 561-899-4131
Chief Executive Officer

Sancilio Pharmaceuticals Company Announces Altemia™ Receives Orphan Drug Designation from the European Medicines Agency for the Treatment of Pediatric Patients with Sickle Cell Disease (SCD)

This follows the Orphan Drug Designation for Altemia granted by the U.S. Food and Drug Administration (FDA) in 2015

RIVIERA BEACH, Fla. – April 3rd, 2018 – Sancilio Pharmaceuticals Company, Inc. (SPCI) today announced that the European Commission has granted Orphan Designation in the European Union to Altemia, the Company’s novel oral formulation being developed for the treatment of SCD in pediatric patients. This approval follows a positive opinion in February 2018 from the European Medicine Agency’s (EMA) Committee for Orphan Medicinal Products (COMP).

SPCI reported positive topline data from its Phase 2 study in November 2017.  “The European Commission designating Altemia as an Orphan Medicine for the EU continues to advance the pathway for our lead clinical compound,” said Geoffrey Glass, CEO of SPCI. “The pediatric population of Sickle Cell Disease patients need, and deserve, more therapeutic options, and we are excited about the opportunity to gain advice from the European Commission on advancing Altemia in the EU markets.”

Orphan Designation is granted by the European Commission to drugs that are intended for the treatment, prevention or diagnosis of life-threatening or chronically debilitating rare diseases. Rare diseases are those defined as having a prevalence of not more than five per 10,000 population in the European Union. The designation potentially qualifies the sponsor for ten years of marketing exclusivity upon approval, as well as fee reduction for various centralized activities, including applications for marketing authorization, inspections and protocol assistance.

Altemia had previously received Orphan Drug Designation from U.S. Food and Drug Administration (FDA) for the US market.

“We are extremely encouraged to receive this designation, further supporting the significant clinical and regulatory progress made in advancing Altemia’s development program,” said Adrian L. Rabinowicz, MD, Chief Medical Officer of SPCI.

SPCI plans to present detailed 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.

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: www.sancilio.com.

Contacts
Sancilio Pharmaceuticals Company, Inc.
Marc Wolff, 561-847-2302
Executive Vice President & Chief Financial Officer

 

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

RIVIERA BEACH, Fla. – November 1st, 2017

  • 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 (https://clinicaltrials.gov/ct2/show/NCT02973360).

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 worldwide 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: www.sancilio.com.

Contacts
Sancilio Pharmaceuticals Company, Inc.
Marc Wolff, 561-847-2302
Executive Vice President & Chief Financial Officer

 

Sancilio Pharmaceuticals Company, Inc. to Present an Update on Research in Sickle Cell at Upcoming National SCDAA Convention

RIVIERA BEACH, Florida. – October 26th, 2017

Sancilio Pharmaceuticals Company, Inc. (SPCI) will present an update on the research program in the field of sickle cell disease, at the National Sickle Cell Disease Association of America (SCDAA) convention, being held in Atlanta from October 25-28, 2017. SPCI is developing Altemia™ as a treatment for pediatric patients with sickle cell disease.

The Company previously announced enrollment completion of the clinical trial, highlighting high retention rates and the possibility for patients to enroll into an open label extension that will collect long term safety data while continuing to monitor the effectiveness of the drug (https://clinicaltrials.gov/ct2/show/NCT02973360).

“We are excited that database lock, another key milestone for the study, is being achieved. We will discuss progress made in our sickle cell research initiatives with the scientific community at SCDAA. We expect to present all data available at future scientific events. Altemia™ has the potential to be an important new treatment option for people with sickle cell disease who experience devastating vaso-occlussive events, which can be extremely disruptive in their lives”, said Dr. Frederick D. Sancilio, President and Chief Executive Officer of SPCI.

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 cell’s 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. Treatment options are limited. The cost of care for this group may exceed $5BB.

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, VOC crises 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 VOC 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.

Contacts
Sancilio Pharmaceuticals Company, Inc.
Marc Wolff, 561-847-2302
Executive Vice President & Chief Financial Officer

Sancilio Pharmaceuticals Company, Inc. Announces the Successful Completion of a Randomized, Double Blind, and Placebo Controlled Phase of a Clinical Trial in Pediatric Patients with Sickle Cell Disease using AltemiaTM

RIVIERA BEACH, Fla. – October 03, 2017

(BUSINESS WIRE)–Sancilio Pharmaceuticals Company, Inc. (SPCI) today announced the successful completion of a randomized, double blinded, placebo controlled clinical trial in pediatric sickle cell patients aged 5 – 17 years old. The clinical trial is a multi-center study designed to evaluate treatment effects and safety of AltemiaTM, a novel pharmaceutical preparation utilizing the Company’s Advanced Lipid Technologies (ALT®) platform. The Company expects to announce top-line data, including Altemia’s impact on the frequency of vaso-occlusive events, in the fourth quarter of 2017. Patients who have completed the study are eligible to continue active drug therapy by entering an open label extension program that will collect long term safety data while continuing to monitor the effectiveness of the drug (https://clinicaltrials.gov/ct2/show/NCT02973360). At present, the majority of patients completing the study have chosen to continue to participate in the open label extension.

Sancilio Pharmaceuticals achieves key milestone for Altemia in pediatric trial in sickle cell disease

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“I want to thank the many doctors, nurses and researchers who have flawlessly executed this challenging protocol,” said Dr. Frederick D. Sancilio, President and Chief Executive Officer of SPCI. “We are very pleased to have had 97% of the randomized patients complete the trial. Additionally, we are encouraged to see a high degree of engagement and enthusiasm by patients to enroll in the open label extension study.”

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 cell’s tendency to adhere to each other, resulting in episodic occlusions of blood vessels, reperfusion damage and excruciating pain. Ultimately, many children develop end organ damage and strokes. There are approximately 100,000 cases of SCD in the United States. Treatment options are limited. The cost of care for this group may exceed $5BB.

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, VOC crises 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 VOC 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.

Contacts

Sancilio Pharmaceuticals Company, Inc.
Marc Wolff, 561-847-2302
Executive Vice President & Chief Financial Officer

Sancilio Pharmaceuticals Company, Inc (SPCI) Receives Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for Altemia(TM) a Treatment of Sickle Cell Disease (SCD) in Children

RIVIERA BEACH, Fla. – October 03, 2017

(BUSINESS WIRE)–Sancilio Pharmaceuticals Company, Inc. (SPCI) today announced the successful completion of a randomized, double blinded, placebo controlled clinical trial in pediatric sickle cell patients aged 5 – 17 years old. The clinical trial is a multi-center study designed to evaluate treatment effects and safety of AltemiaTM, a novel pharmaceutical preparation utilizing the Company’s Advanced Lipid Technologies (ALT®) platform. The Company expects to announce top-line data, including Altemia’s impact on the frequency of vaso-occlusive events, in the fourth quarter of 2017. Patients who have completed the study are eligible to continue active drug therapy by entering an open label extension program that will collect long term safety data while continuing to monitor the effectiveness of the drug (https://clinicaltrials.gov/ct2/show/NCT02973360). At present, the majority of patients completing the study have chosen to continue to participate in the open label extension.

Sancilio Pharmaceuticals achieves key milestone for Altemia in pediatric trial in sickle cell disease

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“I want to thank the many doctors, nurses and researchers who have flawlessly executed this challenging protocol,” said Dr. Frederick D. Sancilio, President and Chief Executive Officer of SPCI. “We are very pleased to have had 97% of the randomized patients complete the trial. Additionally, we are encouraged to see a high degree of engagement and enthusiasm by patients to enroll in the open label extension study.”

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 cell’s tendency to adhere to each other, resulting in episodic occlusions of blood vessels, reperfusion damage and excruciating pain. Ultimately, many children develop end organ damage and strokes. There are approximately 100,000 cases of SCD in the United States. Treatment options are limited. The cost of care for this group may exceed $5BB.

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, VOC crises 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 VOC 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.

Contacts

Sancilio Pharmaceuticals Company, Inc.
Marc Wolff, 561-847-2302
Executive Vice President & Chief Financial Officer