Polaryx to Present Late-Breaker Data Related to SOTERIA at the 22nd Annual WORLDSymposium™ as Company Prepares for SOTERIA Trial Launch
Rhea-AI Summary
Polaryx (NASDAQ: PLYX) will present late-breaking preclinical data on PLX-200 for Krabbe disease at the 22nd Annual WORLDSymposium™ (Feb 2-6, 2026).
Data support advancing the Phase 2 SOTERIA basket trial (CLN2, CLN3, Krabbe, Sandhoff). The company received an FDA safe to proceed letter in October 2025 and plans trial initiation in the first half of 2026. Presentation: Feb 6, 2026, 9:00am PST; presenter Shrijay Vijayan, Ph.D., MBA.
Positive
- Late-breaking abstract accepted at WORLDSymposium signaling external scientific interest
- FDA issued a safe to proceed letter in October 2025
- Company plans SOTERIA Phase 2 initiation in the first half of 2026
- Preclinical mouse-model data for PLX-200 supporting Krabbe disease development
Negative
- SOTERIA is an open-label, single-arm Phase 2 basket trial, limiting controlled efficacy comparisons
- Current evidence is preclinical; no human efficacy data from PLX-200 published yet
News Market Reaction – PLYX
In the Feb 3 session, PLYX declined 37.29%, reflecting a significant negative market reaction. Argus tracked a trough of -60.7% from its starting point during tracking. Our momentum scanner triggered 14 alerts that day, indicating notable trading interest and price volatility.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Key Terms
lysosomal storage disorders medical
basket trial medical
open-label medical
single arm medical
contract research organization technical
mouse model medical
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Oral presentation provides additional scientific rationale on Krabbe disease, one of four LSDs in the Phase 2 SOTERIA basket trial designed to evaluate the safety and clinical activity of lead drug candidate PLX-200
PARAMUS, NJ, Feb. 03, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying therapies for rare, pediatric lysosomal storage disorders (“LSDs”), announces it will be presenting a late-breaking abstract on new data for PLX-200, an investigational therapy for the treatment of Krabbe disease (globoid cell leukodystrophy) at the 22nd Annual WORLDSymposium™, held February 2-6, 2026 in San Diego, Calif. These data support the continued advancement on progressing the launch of the SOTERIA trial, a Phase 2, open-label, single arm trial intended to assess the safety, tolerability, and clinical activity of PLX-200, in CLN2, CLN3, Krabbe disease, and Sandhoff disease.
“These data, which we were honored to learn was accepted as a late-breaking abstract the prestigious WORLDSymposium™ 2026, form the foundation of our work on PLX-200 in Krabbe disease,” said Lisa Bollinger, M.D., Polaryx Therapeutics Chief Medical Officer. “As one of the four indications in our Phase 2 SOTERIA basket trial, we continue to reinforce our commitment to addressing significant unmet needs across lysosomal storage disorders and are excited about the ongoing preparations of SOTERIA.”
The SOTERIA trial represents a flexible and resource‑efficient opportunity to validate PLX‑200’s preclinical science across multiple LSDs while gathering data important in planning PLX‑200’s future development pathway. Polaryx received a safe to proceed letter in October 2025 from the FDA and is actively planning with its contract research organization (CRO) to initiate the trial in the first half of 2026.
Shrijay Vijayan, Ph.D., MBA, Polaryx Therapeutics Chief Scientific and Business Development Officer, will present preclinical findings in an oral presentation on PLX-200 in a mouse model of Krabbe disease. The presentation details are as follows:
Title: Gemfibrozil attenuates disease progression in mouse model of Krabbe disease: Basis for the initiation of SOTERIA, a phase 2 clinical trial of PLX-200 (newly formulated gemfibrozil) in lysosomal disorders
Presentation Type: Oral
Presenter: Shrijay Vijayan, Ph.D., MBA, Chief Scientific and Business Development Officer, Polaryx Therapeutics, Inc.
Date: Friday, February 6, 2026
Time: 9:00am PST
About PLX-200
Polaryx’s lead drug candidate, PLX-200, is an orally available compound comprised of gemfibrozil. Gemfibrozil is an FDA-approved lipid regulating agent in the fibrate family which has only been approved in a capsule form for adult patients with very high elevations of serum triglyceride levels to decrease serum triglycerides and very low-density lipoprotein cholesterol and increase high density lipoprotein cholesterol. The ability of gemfibrozil to cross the blood-brain barrier (“BBB”) has also been documented in third-party preclinical trials and safe use of gemfibrozil in adults has also been well-established over several decades of clinical investigation and commercial use, which we believe accelerates clinical development and reduces associated costs. Although pediatric patients have been treated with gemfibrozil through small-scale compassionate use programs across several pediatric indications, including CLN2, gemfibrozil has never been approved to treat any indications in pediatric patients. We believe the unique ability of PLX-200 to cross the BBB, along with its widely applicable mechanism of action, positions PLX-200 to potentially address the immense unmet need in multiple rare, catastrophic LSD indications.
About the SOTERIA Trial
SOTERIA is a Phase 2, open-label, single arm trial intended to assess the safety, tolerability, and clinical activity of Polaryx’s lead drug candidate, PLX-200, in CLN2, CLN3, Krabbe disease, and Sandhoff disease, four different LSDs whose patient populations Polaryx believes represent approximately one quarter of the LSD population. SOTERIA is designed to be flexible, resource-efficient, and provide important data and information important to PLX-200’s future clinical development. Polaryx received a safe to proceed letter in October 2025 from the FDA and plans to initiate SOTERIA in the first half of 2026 in trial sites in the United States as well as in Europe and Asia or other foreign jurisdictions. 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 will be invaluable in planning PLX-200’s future development pathway, including the initiation of potentially pivotal trials. For the CLN2 and CLN3 cohorts, although the entire trial is open label, these cohorts will incorporate analyses comparing natural history data as a control arm to PLX-200’s treated arm. A natural history study is a preplanned observational study intended to track the course of the disease. Should the data demonstrate compelling clinical activity, Polaryx may seek conditional marketing authorization.
About Polaryx Therapeutics
Polaryx Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing patient-friendly small molecule and gene therapy treatments for rare orphan lysosomal storage disorders (LSDs). Founded in 2014, Polaryx seeks to deliver safe, effective, and patient-friendly treatments that address the underlying pathophysiology of these catastrophic diseases and their significant unmet need. Our approach integrates small molecule therapies, including a combination therapy, and a gene therapy, positioning us to potentially address both the genetic and downstream pathological features of LSDs. Our small molecule drug candidates share similar modes of action that have been demonstrated to address lysosomal dysfunction, neuroinflammation, and neuronal loss in our validated animal models that closely mimic human clinical phenotypes. Our most advanced product candidate, PLX-200, targets several LSDs and we intend to launch SOTERIA, a Phase 2 basket trial, to evaluate PLX-200’s safety and efficacy. For more information, please visit www.polaryx.com.
Forward-Looking Statements
Certain statements in this press release may constitute “forward-looking statements” within the meaning of the federal securities laws, including, but not limited to, statements regarding: Polaryx’s clinical development plans for PLX-200, including the timing for initiation of the SOTERIA trial. Words such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “develop,” “plan” or the negative of these terms, and similar expressions, or statements regarding intent, belief, or current expectations, are forward-looking statements. While Polaryx believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to the company on the date of this release. These forward-looking statements are based upon current estimates and assumptions and are subject to various risks and uncertainties (including, without limitation, those set forth in Polaryx’s filings with the U.S. Securities and Exchange Commission (the SEC), many of which are beyond the company’s control and subject to change. Actual results could be materially different. Risks and uncertainties include: global macroeconomic conditions and related volatility, expectations regarding the initiation, progress, and expected results of Polaryx’s clinical trials; expectations regarding the timing, completion and outcome of Polaryx’s clinical trials; the timing or likelihood of regulatory filings and approvals; liquidity and capital resources; and other risks and uncertainties identified in Polaryx’s Registration Statement on Form S-1, as amended, filed with the SEC on January 27, 2026 and subsequent disclosure documents Polaryx may file with the SEC. Polaryx claims the protection of the Safe Harbor contained in the Private Securities Litigation Reform Act of 1995 for forward-looking statements. Polaryx expressly disclaims any obligation to update or alter any statements whether as a result of new information, future events or otherwise, except as required by law.
Media Contact:
Jules Abraham
Managing Director, Communications
CORE IR
(212) 655-0924
Julesa@coreir.com
Investor Contacts:
CORE IR
(212) 655-0924
investor@polaryx.com
