SAN DIEGO, CA, USA I April 30, 2015 I Retrophin, Inc. (NASDAQ:RTRX) today announced that the U.S. Food and Drug Administration (FDA) has reviewed the Company’s Investigational New Drug (IND) application for RE-024, a novel phosphopantothenate replacement therapy, and has granted clearance to begin clinical studies. The Company is initiating a Phase 1 trial in healthy adult subjects to evaluate safety, tolerability, and pharmacokinetics, with enrollment expected to begin in the coming weeks. RE-024 is being developed for the treatment of pantothenate kinase-associated neurodegeneration (PKAN), a rare and lethal autosomal recessive neurodegenerative disorder believed to affect approximately one to three persons per million worldwide, with no approved treatment currently available.
“We are excited to see RE-024 move forward into the clinic and get one step closer to reaching patients who are suffering from this rare and debilitating disease,” said Dr. Alvin Shih, Executive Vice President and Head of Research & Development for Retrophin. “With the continued support of the scientific and patient communities, our research and development team will continue to rapidly advance RE-024 through clinical development with the hope of providing these patients with a much needed treatment option.”
The Phase 1 trial is a randomized, double-blind, placebo-controlled single ascending dose study of orally administered RE-024 in healthy volunteer subjects. The primary objective of the study is to assess safety and tolerability of single oral doses of RE-024. As a secondary objective, pharmacokinetics of RE-024 will be assessed at multiple time points. Pending study completion, safety and pharmacokinetic data will be submitted for publication.
About RE-024 & PKAN
RE-024 is a small molecule discovered by Retrophin that is being developed as a replacement therapy for phosphopantothenate. Preclinical studies in PANK-deficient animal models and cell lines have demonstrated the ability of RE-024 to restore Coenzyme A (CoA) levels. PKAN is caused by a mutation in the PANK2 gene, which encodes a critical protein that metabolizes vitamin B5 (pantothenate) to phosphopantothenate. The disruption of this metabolic pathway ultimately leads to decreased levels of CoA, an important substrate for many functions such as mitochondrial energy metabolism. Clinical manifestations of PKAN include developmental delay in children, dystonia sometimes causing intractable pain, parkinsonism with Parkinson’s-like freezing and bradykinesia, choreoathetosis, status dystonicus, dysarthria, spasticity, rigidity, and dysphagia often leading to feeding tube placement.
About Retrophin
Retrophin is a pharmaceutical company focused on the development, acquisition and commercialization of drugs for the treatment of serious, catastrophic or rare diseases for which there are currently no viable options for patients. The Company’s approved products include Chenodal®, Cholbam™, and Thiola®, and its pipeline includes compounds for several catastrophic diseases, including focal segmental glomerulosclerosis (FSGS), pantothenate kinase-associated neurodegeneration (PKAN), infantile spasms, nephrotic syndrome and others. For additional information, please visit www.retrophin.com.
SOURCE: Retrophin
Post Views: 77
SAN DIEGO, CA, USA I April 30, 2015 I Retrophin, Inc. (NASDAQ:RTRX) today announced that the U.S. Food and Drug Administration (FDA) has reviewed the Company’s Investigational New Drug (IND) application for RE-024, a novel phosphopantothenate replacement therapy, and has granted clearance to begin clinical studies. The Company is initiating a Phase 1 trial in healthy adult subjects to evaluate safety, tolerability, and pharmacokinetics, with enrollment expected to begin in the coming weeks. RE-024 is being developed for the treatment of pantothenate kinase-associated neurodegeneration (PKAN), a rare and lethal autosomal recessive neurodegenerative disorder believed to affect approximately one to three persons per million worldwide, with no approved treatment currently available.
“We are excited to see RE-024 move forward into the clinic and get one step closer to reaching patients who are suffering from this rare and debilitating disease,” said Dr. Alvin Shih, Executive Vice President and Head of Research & Development for Retrophin. “With the continued support of the scientific and patient communities, our research and development team will continue to rapidly advance RE-024 through clinical development with the hope of providing these patients with a much needed treatment option.”
The Phase 1 trial is a randomized, double-blind, placebo-controlled single ascending dose study of orally administered RE-024 in healthy volunteer subjects. The primary objective of the study is to assess safety and tolerability of single oral doses of RE-024. As a secondary objective, pharmacokinetics of RE-024 will be assessed at multiple time points. Pending study completion, safety and pharmacokinetic data will be submitted for publication.
About RE-024 & PKAN
RE-024 is a small molecule discovered by Retrophin that is being developed as a replacement therapy for phosphopantothenate. Preclinical studies in PANK-deficient animal models and cell lines have demonstrated the ability of RE-024 to restore Coenzyme A (CoA) levels. PKAN is caused by a mutation in the PANK2 gene, which encodes a critical protein that metabolizes vitamin B5 (pantothenate) to phosphopantothenate. The disruption of this metabolic pathway ultimately leads to decreased levels of CoA, an important substrate for many functions such as mitochondrial energy metabolism. Clinical manifestations of PKAN include developmental delay in children, dystonia sometimes causing intractable pain, parkinsonism with Parkinson’s-like freezing and bradykinesia, choreoathetosis, status dystonicus, dysarthria, spasticity, rigidity, and dysphagia often leading to feeding tube placement.
About Retrophin
Retrophin is a pharmaceutical company focused on the development, acquisition and commercialization of drugs for the treatment of serious, catastrophic or rare diseases for which there are currently no viable options for patients. The Company’s approved products include Chenodal®, Cholbam™, and Thiola®, and its pipeline includes compounds for several catastrophic diseases, including focal segmental glomerulosclerosis (FSGS), pantothenate kinase-associated neurodegeneration (PKAN), infantile spasms, nephrotic syndrome and others. For additional information, please visit www.retrophin.com.
SOURCE: Retrophin
Post Views: 77
