Sodium Feredetate

The U.S. Food and Drug Administration (FDA) approved Chiesi Global Rare Diseases’ Ferriprox (deferiprone) for treatment of transfusional iron overload caused by sickle cell disease (SCD) or other anemias in adults and children ages three years and older. This approval expands the use of the drug for patients with SCD or other anemias in addition to patients with thalassemia without regard for previous iron chelation exposure. Ferriprox is a synthetic iron-chelating agent that can be taken orally. It decreases iron concentrations by penetrating cell membranes and latching onto and removing toxic iron from organs and extracellular fluids. The drug is available as a 1000mg twice-daily oral tablet. SCD affects approximately 100,000 people in the U.S. In it, the red blood cells take on a C-shaped, sickle-like formation. They do not carry oxygen as effectively and die early, resulting in a shortage of red blood cells (anemia). Because they are stickier than normal red blood cells, they also cause more blood clots in small blood vessels, which can cause pain and other problems like infection, acute chest syndrome and stroke. Iron overload results from the number of blood transfusions SCD patients require. “People who are living with SCD face significant challenges with pain and organ damage that can greatly impact their quality of life, and most who need blood transfusions also need iron chelation therapy including those with known kidney issues who have limited treatment options,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases. “We believe that delivering an iron chelation therapy that has no dosage adjustment required for patients with mild to severe renal impairment may address a significant unmet need in SCD.” Chiesi went on to say, “We have a long history of commitment to the rare disease community and this FDA approval is a testament to the investments we continue to make in scientific research and development with patients at the center of everything we do. We would like to thank the patients and the SCD community for their participation in clinical studies because without their support this would not have been possible.” Just last week, Chiesi and its development partner Protalix Biotherapeutics received a Complete Response Letter (CRL) from the FDA for their Biologics License Application (BLA) for pegunigalsidase alfa (PRX-102) for adults with Fabry disease. Fabry disease is an X-linked inherited disease caused by insufficient activity of the enzyme lysosomal alpha-Galactosidase-A. This causes the progressive accumulation of abnormal deposits of globotriaosylceramide (Gb3). Gb3 is a fatty substance that accumulates in the blood and blood vessel walls. Symptoms are pain, gastrointestinal issues, fatigue, angiokeratoma, dark spots on the skin caused by dilated capillaries, abnormal sweating, and cardiovascular, renal, and cerebrovascular events. PRX-102 is a plant cell culture-expressed and chemically modified type of the recombinant alpha-Galactosidase-A enzyme. The submission was based on data from completed Phase I/II clinical tests, an extension study, and interim data from the Phase III BRIDGE switch-over trials, as well as safety data from other ongoing trials. It was submitted under the agency’s Accelerated Pathway program. Giacomo Chiesi said at that time, “Based on extensive clinical data including results from the Phase III BRIDGE clinical trial of PRX-102 for the proposed treatment of Fabry disease, we continue to feel strongly that PRX-102 is an important option for the treatment of Fabry disease in adult patients, and we are continuing with our efforts to make this therapy available to patients.”

HARWELL, England, Feb. 17, 2021 /PRNewswire/ -- AbFero Ltd., a subsidiary of AbFero Pharmaceuticals, Inc., a privately-held clinical stage pharmaceutical company dedicated to treating diseases of iron overload, today announced the award of a grant from Cure Parkinson's for the development of their lead, brain-penetrant iron chelating agent, SP-420, as a disease modifying therapy for Parkinson's disease (PD). The funding will be used to develop biomarkers to demonstrate that SP-420 is an effective targeted therapy for Parkinson's. This is the second grant AbFero has received to support development of SP-420 for the condition, following recent funding from Eureka EUROSTARS. "The role of iron in neurodegenerative diseases grows more compelling every day. Cure Parkinson's has been a strong proponent of iron chelation for Parkinson's, so this grant is particularly meaningful. It further accelerates AbFero's drive towards our first clinical study in Parkinson's," said AbFero CEO Thomas Neenan. Removal of excess brain iron via chelation has long been considered an attractive disease modifying approach, but a therapy with sufficient safety and proven efficacy remains lacking. AbFero expects SP-420 will limit iron accumulation in the brain, reducing iron-related oxidative stress, iron related neuronal death (i.e., ferroptosis), and alpha-synuclein aggregation, well-known neuropathological hallmarks of Parkinson's. Dr Richard Wyse, Director of Research & Development at Cure Parkinson's said: "Iron chelation is an extremely promising line of investigation in Parkinson's. Our funding for this project adds new impetus and expands our long-standing interest in the future relevance of this approach in slowing or stopping long-term neurodegeneration in patients with Parkinson's disease." The grant will fund ongoing development of the small molecule in the laboratory of Professor David Devos, MD, PhD, University of Lille, CHU of Lille, INSERM. "We believe SP-420 is one of the greatest iron chelators, given it has excellent kinetics, high oral bioavailability and biodistribution, and no evidence to date of inappropriate competition with the native iron transport and storage mechanisms of the body," Dr. Devos notes. "AbFero is working urgently to develop best-in-class, next generation iron chelators that will slow progression and improve the quality of life for the nearly 10 million people living with Parkinson's today," Neenan adds. "We're grateful to Cure Parkinson's for their support as we press forward." About AbFero, Ltd. AbFero, Ltd., is a subsidiary of AbFero Pharmaceuticals, Inc., a privately-held clinical stage pharmaceutical dedicated to treating diseases of iron overload. AbFero's technology is based upon the discoveries of a pioneer in the field of iron chelator medicinal chemistry, Professor Raymond Bergeron, University of Florida. Our therapeutic platform addresses transfusional iron overload (TIO) and iron accumulation associated with retinal and neurologic diseases including age-related macular degeneration (AMD), Parkinson's disease (PD), and traumatic brain injury (TBI). AbFero has completed three clinical trials with the company's lead chelator, SP-420. AbFero Ltd., is located in Harwell, UK. For more information, visit . Investor & Media Contact: Mih-Ho Cha mcha@abfero.com