Polaryx Therapeutics Receives Orphan Drug Designation From The U.S. FDA For PLX-200 For Rare Pediatric Disease
PARAMUS, N.J., Aug. 7, 2017 /PRNewswire/ -- Polaryx Therapeutics, Inc, a biotech company developing oral small molecule therapeutics for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) and for other forms of NCL, commonly known as Batten disease, has been granted Orphan Drug Designation from the U.S. FDA for PLX-200 to treat LINCL. LINCL, which qualifies as a rare pediatric disease under Section 529 of the Food, Drug, and Cosmetic Act, is an autosomal recessive neurodegenerative disorder with life expectancy ranging from six to the early teenage years. Currently, no patient-friendly treatment options are available.
LINCL, which is caused by mutations in the Cln2 gene, leads to the deficiency and/or loss of function of Tripeptidyl Peptidase 1 (TPP1). The deficiency and/or loss of function of the TPP1 leads to accumulation of autofluorescent storage materials in neurons and in other cells. The symptoms start with seizures, followed by regression of development, myoclonic ataxia, and pyramidal signs. Visual impairment typically appears at four to six years of age and rapidly progresses to light/dark awareness only. Polaryx Therapeutics' development strategy is centered on repurposing an oral small molecule to provide LINCL patients with a safe, effective, and convenient treatment option.
"Granting PLX-200 the Orphan Drug Designation is one of the company's significant development milestones," says Dr. Hahn-Jun Lee, M.Sc., Ph.D., president and CEO of Polaryx Therapeutics, Inc. "We will accelerate this program into clinics as early as possible to help LINCL patients. We will confirm the efficacy of PLX-200 in humans and further confirm that it can be applied to other types of NCL disorders."
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, offered a joint statement that the Orphan Drug Designation was a significant achievement in validating the new therapeutics. He stated, "The FDA's decision recognizes the significant value of our technology that led to the extension of survival of an LINCL animal disease model and delayed the decline in motor function. As this is a patient-friendly new treatment option that is a safely-used oral small molecule, it will help a lot to patients in many aspects."
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 disease affecting fewer than 200,000 people in the U.S. 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.
To learn more about the company and PLX-200 as well as Dr. Hahn-Jun Lee's expertise and experience, visit Polaryx.com
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. Polaryx is repurposing an existing safe oral medication, so that the treatment is patient-friendly for prolonged use. For more information, visit Polaryx.com.
PLX-200 is a repurposed drug that binds to the retinoid X receptor- (RXR), which binds to PPAR thereby up-regulating the expression of TPP1 mRNA in brain cells via the PPAR/RXR heterodimer. PLX-200 also activates PPAR, which enhances production of transcription factor EB (TFEB) in brain cells. TFEB then binds to the promoter of genes involved in lysosome biogenesis and activates their production. TFEB can regulate lysosomes due to its effects on the expression of lysosomal genes.
Dr. Hahn-Jun Lee, M.Sc., Ph.D.