Lysosomal storage disorders are a group of rare inherited metabolic disease.
Transcription Factor EB (TFEB) is a master regulator of lysosome biogenesis. Increasing evidence has defined the crucial role of TFEB-dependent lysosome biogenesis on lysosomal storage disorders (LSDs). An attractive approach to eliminate toxic aggregates in LSDs is to upregulate the cellular clearance of these wastes through induction of the lysosomal machinery.
Polaryx® Therapeutics utilizes either new drugs or repurposed drugs to tackle these diseases, and all of them are uniquely formulated. The company is currently preparing to perform a clinical trial to confirm the safety and efficacy of its drug candidates.
Why Polaryx?®
There is an urgent need for novel therapeutic interventions for lysosomal storage disorders, including neuronal ceroid lipofuscinosis, Krabbe disease, Sandoff disease, and Niemann Pick Type A/B diseases. That’s where Polaryx® comes in.
Learn MoreAbout PolaryxThe Technology
Discover our unique investigational methodology that Polaryx® is pursuing to treat lysosomal storage disorders, including neuronal ceroid lipofuscinosis, Krabbe disease, Sandoff disease, and Niemann Pick Type A/B diseases, non-invasively.
Learn MoreThe TechnologyThe Pipeline
Learn about key mechanism of action of our drug candidates development strategy.
Learn MoreThe PipelineMeet the team
With over 100 years of combined experience, we are ready to take on lysosomal storage disorders, including neuronal ceroid lipofuscinosis, Krabbe disease, Sandoff disease, and Niemann Pick Type A/B diseases.
Learn MoreAbout Our TeamThe Science
Learn more about TFEB-dependent lysosome biogenesis, our key mechanism of action (MOA) to clear cellular wastes.
Learn MoreThe SciencePolaryx® Highlights
Diversified pipeline
with novel and
repurposed drugs
Pursuing pediatric
dosing options
Backed by an
experienced team
Expanding to neuronal ceroid lipofuscinosis, Krabbe disease, Sandoff disease, and Niemann Pick Type A/B diseases.
Completely
non-invasive
(taken orally)
Cost-effective drug
repurposing strategy
Latest News
Polaryx® Therapeutics Provides Corporate Update
PARAMUS, NEW JERSEY, UNITED STATES, May 5, 2022 /EINPresswire.com/ --
- Focused on launching STARLIGHT Phase 3 clinical trial for lead drug candidate, PLX-200, in treatment of CLN3; IND-approved CLN2 trial also in prep
- Completed development of novel, patient-friendly oral solution and continuing research and development of buccal formulation
- Exploring partnership opportunities to accelerate clinical development
Polaryx® Therapeutics Receives FDA Orphan Drug Designation for PLX-200 to Treat Krabbe Disease
PARAMUS, N.J., September 2, 2021 (Newswire.com) - Polaryx Therapeutics, Inc. ("Polaryx"), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat Krabbe disease.
Polaryx® Therapeutics Announces FDA Grants Orphan Drug Designation for PLX-200 in GM2 Gangliosidoses
PARAMUS, N.J., Aug. 30, 2021 /PRNewswire/ -- Polaryx Therapeutics, Inc. ("Polaryx"), a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration ("FDA") has granted Orphan Drug Designation for PLX-200 to treat GM2 gangliosidoses.
Polaryx® Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of Krabbe Disease With PLX-300
PARAMUS, N.J., Feb. 10, 2021 /PRNewswire/ -- Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that it has received both Rare Pediatric Disease and Orphan Drug Designations for the treatment of Krabbe Disease (a.k.a. Globoid Cell Leukodystrophy) with PLX-300 from the U.S. Food and Drug Administration (FDA).
Polaryx® Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of Niemann Pick Disease Types A and B With PLX-300
PARAMUS, N.J., Dec. 18, 2020 /PRNewswire/ -- Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that it has received from the U.S. Food and Drug Administration (FDA) both Rare Pediatric Disease and Orphan Drug Designations for the treatment of Niemann Pick Disease Types A and B, known as Acid Sphingomyelinase Deficiency with PLX-300.
Polaryx® Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of GM2 Gangliosidosis With PLX-300
PARAMUS, N.J., Nov. 25, 2020 /PRNewswire/ -- Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that it has received from the U.S. Food and Drug Administration (FDA) both Rare Pediatric Disease and Orphan Drug designations for the treatment of GM2 gangliosidosis with PLX-300.
Polaryx® Therapeutics Receives FDA Fast Track Designation to PLX-200 for the Treatment of Patients With Juvenile Neuronal Ceroid Lipofuscinosis
PARAMUS, N.J., Aug. 20, 2020 /PRNewswire/ -- Polaryx Therapeutics Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to PLX-200 for the treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JNCL/CLN3) patients.
Polaryx® Therapeutics Receives IND Approval From the FDA to Study PLX-200 Treatment for Patients With Juvenile Neuronal Ceroid Lipofuscinosis
PARAMUS, N.J., April 8, 2020 /PRNewswire/ -- Polaryx Therapeutics, a biotech company developing patient-friendly, small molecule therapeutics for lysosomal storage disorders, announced today that the Company has received an Investigational New Drug Application (IND) approval from the U.S. Food and Drug Administration (FDA) to study PLX-200 treatment on Juvenile Neuronal Ceroid Lipofuscinosis (JNCL or CLN3) patients.
Polaryx® Therapeutics Receives IND Approval for PLX-200 From the FDA for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis
PARAMUS, N.J., Jan. 20, 2020 /PRNewswire/ -- Polaryx Therapeutics, a biotech company developing patient-friendly small molecule therapeutics for lysosomal storage disorders announced today that the Company has received an Investigational New Drug Application (IND) approval from the U.S. Food and Drug Administration (FDA) for the treatment of LINCL patients with PLX-200.
Polaryx® Therapeutics Submits Investigational New Drug Application to the U.S. FDA for the Treatment of Late Infantile Neuronal Ceroid Lipofuscinosis With PLX-200
This new drug candidate will be used for the treatment of patients with Late Infantile Neuronal Ceroid Lipofuscinosis.
Polaryx Therapeutics Boosts Lysosome Biogenesis to Treat Neurodegenerative Disorders
Hahn-Jun Lee, President and CEO of Polaryx Therapeutics Inc., speaks at BIO-Europe® 2019 in Hamburg about how the company accelerates the availability of treatment options for underserved patients, and what the scientific rationale is behind their products.
Polaryx® Therapeutics Received Orphan Drug Designation From the European Medicines Agency (EMA) for the Treatment of All Neuronal Ceroid Lipofuscinosis With PLX-200
Click "read more" now to view the press release.
Polaryx® Featured in June 2017 Issue of Biopharma Dealmakers
Renowned pharmaceutical industry publication, Biopharma Dealmakers, has featured Polaryx® Therapeturics in their June 2017 edition. Click "read more" now to view the publication.
Polaryx® interviewed by and featured in GHP magazine
To introduce Polaryx® and its technology, GHP magazine has interviewed the Polaryx® team. Click now to see the June 2017 GHP magazine edition. Polaryx® is featured on pages 26-27.
Polaryx® Therapeutics Receives Orphan Drug Designation From the U.S. FDA for PLX-200 for Rare Pediatric Disease
This new drug candidate will be used for the treatment of patients with Late Infantile Neuronal Ceroid Lipofuscinosis.
Polaryx® Therapeutics Receives Orphan Drug Designation From the US FDA for the Treatment of Neuronal Ceroid Lipofuscinoses With PLX-100
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.
Polaryx® Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of Niemann Pick Disease Types A and B With PLX-300
PARAMUS, N.J., Dec. 18, 2020 /PRNewswire/ -- Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that it has received from the U.S. Food and Drug Administration (FDA) both Rare Pediatric Disease and Orphan Drug Designations for the treatment of Niemann Pick Disease Types A and B, known as Acid Sphingomyelinase Deficiency with PLX-300.