Polaryx Therapeutics Receives Orphan Drug Designation From the US FDA for the Treatment of Neuronal Ceroid Lipofuscinoses With PLX-100
PARAMUS, N.J., December 11, 2017 (Newswire.com) - Polaryx Therapeutics, Inc. is a biotech company developing patient-friendly oral small molecule therapeutics for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) and other forms of NCL, commonly known as Batten disease. Neuronal Ceroid Lipofuscinoses qualify as rare pediatric diseases under Section 529 of the Food, Drug, and Cosmetic Act.
Neuronal Ceroid Lipofuscinoses are a group of autosomal recessive neurodegenerative lysosomal storage disorders. These disorders are caused by cellular accumulation of abnormal auto-fluorescent lipoproteins, resulting in deterioration of neurons in both the brain and retina. Many patients suffer from mental deterioration, vision loss, severe seizures, and declining motor function, leading to premature death. The company advanced a unique repurposing development strategy to provide patients with a safe, effective and convenient treatment option.
“Granting PLX-100 the Orphan Drug Designation (ODD) for Neuronal Ceroid Lipofuscinoses is one of our significant development milestone achievements. This will accelerate our clinical development program with PLX-200, which has been granted ODD for Neuronal Ceroid Lipofuscinoses from the FDA, previously. Since earlier this year Polaryx had a pre-IND meeting with the FDA, we are now able to focus on the clinical development program to assess the clinical efficacies,” stated Dr. Hahn-Jun Lee, M.Sc., Ph.D., President and CEO of the company.
Dr. Kalipada Pahan, Ph.D., a Professor of Neurological Sciences, Biochemistry, and Pharmacology and the Floyd A. Davis, M.D., Endowed Chair in Neurology at the Rush University Medical Center in Chicago, jointly stated that the Orphan Drug Designation for PLX-100 was indeed a cooperative effort based on active collaboration with the company. “The FDA’s granting ODD solidly confirms the therapeutic rationale of our treatment that led to the significant extension of survival and improved motor function in a murine NCL model. As PLX-100 is a non-invasive, safe, oral small molecule therapy, it will help many patients when it reaches the clinic in the near future.”
Under the U.S. Orphan Drug Act, the FDA’s Office of Orphan Products Development provides sponsors with special status and incentives to facilitate drug development for rare diseases affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides seven years of market exclusivity if the drug candidate receives regulatory approval together with tax credits for qualified clinical trial costs, exemptions from certain FDA application fees, and assistance in clinical trial design.
About Polaryx Therapeutics, Inc.
Polaryx Therapeutics, Inc. is solely dedicated to developing drug candidates for late infantile neuronal ceroid lipofuscinosis (LINCL) and other forms of NCL, for which there is currently no patient-friendly treatment option available. Polaryx is repurposing existing safe oral medications so that the treatment is patient-friendly for a prolonged use.
PLX-100 is a combination of orally available small molecules that bind to the retinoid X receptor-α (RXRα), which then binds to PPARα thereby up-regulating the expression of TPP1 mRNA in brain cells via the PPARα/RXRα heterodimer formation. PLX-100 also enhances the production of transcription factor EB (TFEB) in brain cells via the PPARα/RXRα heterodimer pathway. TFEB then binds to the promoter of genes involved in lysosome biogenesis and activates their production. PLX- 100 also reduces inflammation and prevents cell death (apoptosis).
About Neuronal Ceroid Lipofuscinoses
Neuronal Ceroid Lipofuscinoses (NCLs) are a group of autosomal recessive rare neurodegenerative disorders caused by mutations within one of 13 genes, leading to the accumulation of abnormal auto-fluorescent, electron-dense granules in the cells. This accumulation then causes deterioration of neurons in both the brain and retina. The onset of all NCLs can range from infancy to adulthood. Patients suffer from severe seizures, progressive visual degeneration, cognitive impairment, motor dysfunction, speech difficulties, behavioral problems, cerebral atrophies, cardiac problems, and dementia prior to premature death.
Hahn-Jun Lee, M.Sc., Ph.D.
Source: Polaryx Therapeutics, Inc.
Categories: Medical Research