Category Archives: Press release

‘Every time it’s a battle’: In excruciating pain, sickle cell patients are shunted aside

Amy Mason had toughed it out for hours one day this past July, trying warm soaks and heating pads and deep breathing to soothe pain that felt like her bones were being sawed with a rusty blade.

She knew this was a life-threatening emergency of sickle cell disease, in which her misshapen red blood cells were getting stuck in her blood vessels like tree limbs in a storm sewer. But she delayed going to the emergency room; previous visits hadn’t gone well.

Just before midnight, Mason, 34, finally had her boyfriend drive her to a Mobile, Ala., hospital. She told the triage nurse that she was having one of the worst sickle cell crises of her life and that she was off the far end of the 1-to-10 pain scale. She was told to wait.

As the hours passed, Mason begged her boyfriend to take her to another hospital, then passed out from the pain. She awoke, she told STAT, to her boyfriend’s shaking her and pleading with her to stay with him. I can’t do this anymore, Mason whispered.

Daylight broke. Around mid-morning, an ER nurse told her, you know, we’re not a pain clinic. She gave Mason a small dose of the narcotic Dilaudid.

“Every time, it’s a battle,” Mason said about the ER, where the staff suspected her of faking her condition in order to score opiates and viewed her as a non-emergency because she “only” had pain. “Nobody cares about people with sickle cell.”

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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 Altemia™ 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) Micelle Bio Pharma, Inc. today announced positive top line results from a clinical study evaluating the efficacy and safety of Altemia, 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, Micelle Bio Pharma 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 Altemia.

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 of Micelle Bio Pharma.

“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 Altemia clearly warrants further advancement of the program,” said Adrian L. Rabinowicz, MD, former Chief Medical Officer of Micelle Bio Pharma.

Additional analysis of the data is ongoing, and Micelle Bio Pharma 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 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 Micelle Bio Pharma 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 Micelle Bio Pharma, Inc.

Micelle Bio Pharma, Inc. 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. Micelle Bio Pharma 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.

Micelle BioPharma 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 Altemia™

RIVIERA BEACH, Fla. – April 2018

Micelle BioPharma 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.

“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 Micelle BioPharma. “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 Micelle BioPharma 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.

Micelle BioPharma 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

Micelle BioPharma today announced that the European Commission has granted Orphan Designation in the European Union (EU) 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).
Micelle BioPharma reported positive topline data from its Phase 2 study in November 2017. “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 Micelle BioPharma.

Micelle BioPharma 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 Micelle Bio Pharma 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 Micelle Bio Pharma, Inc.

Micelle Bio Pharma 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. Micelle BioPharma 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.