Polaryx Therapeutics Received Orphan Drug Designation From the European Medicines Agency (EMA) for the Treatment of Neuronal Ceroid Lipofuscinosis With PLX-200

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 Lipofuscinosis qualifies as rare pediatric disease under Section 529 of the Food, Drug, and Cosmetic Act.

Neuronal Ceroid Lipofuscinosis is a group of autosomal recessive neurodegenerative lysosomal storage disorders. This disorder is caused by cellular accumulation of abnormal auto-fluorescent lipoproteins, resulting in deterioration of neurons in both the brain and retina. Because many patients suffer from vision loss, severe seizures, and declining motor function, leading to premature death, treatment for these diseases is indeed an unmet medical need. The company has advanced a unique repurposing drug development strategy to provide patients with a safe and effective oral treatment option.

"Granting PLX-200 the Orphan Drug Designation (ODD) for Neuronal Ceroid Lipofuscinosis from the European Medicines Agency (EMA) indicates that the EMA highly regarded the significant value of the PLX-200 program, especially its safety and many potential benefits to patients. This EMA designation will clearly facilitate PLX-200 clinical development, which also was granted ODD for Neuronal Ceroid Lipofuscinoses from the FDA, last year. Polaryx will file an IND for PLX-200 to the FDA this year, 2018, to assess its clinical efficacy and benefits to LINCL patients, first. We will initiate this clinical testing under the right settings and criteria as early as possible," 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 EMA’s granting of ODD to PLX-200 reconfirms the excellence of our science that led to the significant extension of survival and improved motor function in a murine model of LINCL. Our research also demonstrated the potential of this therapeutic to treat other subtypes of NCL based on its mechanisms of action. This treatment will help many patients who suffer from this devastating disease when it reaches the clinic.”

EMA Orphan Drug Designation (ODD)

In Europe, to qualify for orphan drug designation, a medicine must be intended for the treatment, prevention, or diagnosis of a disease that is life-threatening or chronically debilitating. In addition, the disease prevalence in the EU must not be more than 5 in 10,000, and when no satisfactory method of diagnosis, prevention, or treatment of the condition exists, or, if such method exists, the medicine must be of significant benefit to those affected by the condition. As incentives to encourage the development of orphan medicines, the EU offers protocol assistance specific for designated orphan medicine, 10 years of market exclusivity once the medicine is on the market, and fee reductions depending on the status of the sponsor and the type of service required.

Polaryx Therapeutics, Inc

Polaryx Therapeutics, Inc is solely dedicated to developing drug candidates for late infantile neuronal ceroid lipofuscinosis (LINCL) and other types of NCL, such as the juvenile forms of NCL, for which there is currently no safe and patient-friendly treatment option available. Polaryx is repurposing existing safe oral medications, so that the treatment is patient-friendly for a prolonged use.


PLX-200 is a repurposed drug that binds to the retinoid X receptor-α (RXRα), which binds to PPARα thereby up-regulating the expression of TPP1 mRNA in brain cells via the PPARα/RXRα heterodimer. PLX-200 also activates PPARα, 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. PLX-200 also reduces inflammation and prevents cell death (apoptosis).

Neuronal Ceroid Lipofuscinosis

Neuronal Ceroid Lipofuscinosis (NCL) is 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 atrophy, cardiac problems, and dementia prior to premature death.

Media Contact

Hahn-Jun Lee, M.Sc., Ph.D.

Source: Polaryx Therapeutics, Inc.

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 Therapeutics, Inc
140 E. Ridgewood Avenue, Suite 415, South Tower,
Paramus, NJ

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