Clinical Trials

Our Clinical Trials

Polaryx is approved to examine our lead candidate PLX‑200, a novel and patient‑friendly therapy, through three IND‑approved trials: SOTERIA, a Phase 2, open‑label proof‑of‑concept basket trial for CLN2, CLN3, Krabbe disease, and Sandhoff disease, along with separate pivotal trials for CLN2 and CLN3. We plan to prioritize the launch of SOTERIA, which is expected to initiate in the first half of 2026.

Preclinical studies of PLX-200 has demonstrated potential therapeutic value beyond CLN2 and CLN3 to include multiple lysosomal storage disorders. We plan to align our CLN2 and CLN3 trial timelines with emerging data from SOTERIA, as data readouts from SOTERIA are expected to provide guidance and a clear pathway for each of the four indications towards potentially registrable trials, including CLN2, CLN3, Krabbe disease and Sandhoff disease.

Phase 2 SOTERIA Basket Trial

SOTERIA is a small-scale, proof-of-concept, open-label Phase 2 basket trial intended to assess the safety, tolerability, and clinical activity of PLX-200 for the treatment of certain LSDs, which we believe represent approximately one quarter of the LSD population, including CLN2 and CLN3 subtypes of the 13 ODD-approved NCLs, as well as Krabbe disease and Sandhoff disease. Designed with a high degree of flexibility, SOTERIA represents a resource-efficient opportunity to validate PLX-200’s preclinical science across multiple LSDs while gathering data that we believe will be invaluable in planning PLX-200’s future development pathway, including the initiation of potentially pivotal trials. Polaryx received a safe to proceed letter in October 2025 and expects to initiate the trial in the first half of 2026.

Single Pivotal Trial For CLN2

Our IND-approved CLN2 trial is a Phase 2 clinical study of PLX-200 for the treatment of CLN2. This potentially single pivotal trial is designed as a single arm, open-label, synthetic control clinical trial in participants with mild-to-moderate “classic” CLN2 disease. The primary endpoints of the study aims to primary endpoint aims to measure the mean difference in the rate of decline in the Total Disability Score of the Adapted Hamburg Rating Scale, which contains the motor, visual, and language domains, between matched populations, at Week 96 of treatment exposure. The current total trial duration is expected to be approximately two years, although we may amend the clinical trial design to improve patient/caregiver experience in the trial, reduce the length of the trial, and ensure that patients are exposed to an effective and safe treatment.

Single Pivotal Trial For CLN3

STARLIGHT is an IND-approved Phase 3 clinical trial of PLX-200 for the treatment of CLN3. The trial is a single registrable, randomized, double-blind, placebo-controlled clinical trial for participants with mild to moderate CLN3 disease. The primary objectives are to evaluate the safety and tolerability of PLX-200 compared with the placebo group and to evaluate the efficacy of PLX-200 using the motor score of Hamburg Rating Scale compared with the placebo group after 60 weeks of maintenance therapy. We plan to continue to optimize the STARLIGHT clinical trial design based on the findings from SOTERIA and inputs from clinical experts in the CLN3 landscape before continuing to actively align with the FDA. If the SOTERIA trial generates evidence of safety and effectiveness, it may help support the regulatory pathway and NDA submission for PLX-200 as the treatment of CLN3.

More information on the trial can be found at Clinicaltrials.gov NCT04637282.

If you need more information about the clinical trial, please contact us at info@polaryx.com.