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PARAMUS, NEW JERSEY, USA, May 5, 2022 / —

• Focused on the initiation of the STARLIGHT Phase 3 clinical trial for the lead drug candidate, PLX-200, in the treatment of CLN3; IND-approved CLN2 trial also in preparation

• Completed the development of a new patient-friendly oral solution and continued research and development of the buccal formulation

• Explore partnership opportunities to accelerate clinical development

Polaryx Therapeutics (“Polaryx”), a clinical biopharmaceutical company developing effective and user-friendly therapies for rare and inherited lysosomal storage disorders (“LSD”), today provides an update on clinical development plans and business.


Polaryx’s pipeline of therapies – PLX-100, PLX-200 and PLX-300 – focuses on a range of rare inherited lysosomal storage disorders such as Batten’s disease (neuronal Ceroid Lipofuscinosis), Krabbe’s disease, Tay Sachs/Sandhoff disease and Niemann-Pick disease Type A/B disease. As many of these diseases currently have no available treatment options, the development of Polaryx’s potentially disease-modifying therapies has a high unmet medical need.


In the near term, Polaryx continues to focus on PLX-200, its lead drug candidate, and the initiation of STARLIGHT, a pivotal Phase 3 clinical trial evaluating PLX-200 in 39 participants with mild to moderate CLN3 ( Juvenile NCL). CLN3 has no current treatment and is marked by lysosomal dysfunction leading to significant comorbidities and possibly premature death.

Polaryx is also continuing to prepare PLX-200 for a pivotal Phase 3 trial in 26 mild to moderate CLN2 (Late Infantile NCL) children. CLN2 has a treatment available that is delivered at high cost in a hospital setting by a distinctly patient-unfriendly intracerebroventricular delivery.


In line with Polaryx’s commitment to patient-friendly administration as part of today’s standard of care, Polaryx has completed development of a highly compliant oral solution that increases dosage control, palatability, and bioavailability. Research and development of formulations of buccal and oral solutions and suspensions with improved bioavailability will continue.

“Polaryx is intensely focused on providing patient-tailored therapies for lysosomal storage disorders. Our proprietary oral liquid dosage forms are central to our ability to provide accurate and safe dosing of our therapies, which is especially critical as many LSDs affect pediatric patients,” commented Hahn-Jun Lee, M.Sc., Ph.D., President and Director of Polaryx. Chief executive officer.


As PLX-200 is poised to launch pivotal Phase 3 trials in two indications, Polaryx is set to identify partners to advance the development timeline and bring therapies closer to market. In support of this objective, MTS Health Partners was retained as financial advisor to assist in the search for a partner.

“We are very pleased with the clinical development of Polaryx at this stage with two indications already approved for pivotal trials. Currently, we are also exploring partnership opportunities that will catalyze accelerated clinical development and bring us closer to delivering our therapies to patients in need,” said Alex Yang, JD, LLM, Executive Chairman of the Board of Polaryx.


Polaryx Therapeutics, Inc. is a late-stage, multi-asset clinical biopharmaceutical company developing patient-tailored small molecule therapies for rare and inherited lysosomal storage disorders such as neuronal ceroid lipofuscinosis, Tay Sachs disease , Krabbe disease and Niemann-Pick disease type A. /B. Polaryx’s drug candidates exhibit multiple mechanisms of action to enhance cellular clearance pathways to remove cellular waste and further support neuronal survival.

Polaryx’s largest shareholder is Mstone Partners, an entrepreneurial biotechnology incubator in the form of a holding company that owns and manages a portfolio of drug development companies. Mstone focuses on rare, pediatric and repurposed drugs, neurodegenerative disorders and innovative technologies for targeted indications. Since its inception in 2016, Mstone has invested in two US companies and one in Hong Kong, which are now in advanced clinical stages with the US FDA. Mstone has also created a number of portfolio companies under the Curestone platform, which manages a portfolio of drug development companies in a centralized hub-and-spoke model.

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Andrew O
Polaryx therapeutics
[email protected]