Polaryx Announces Key Pre-Clinical Data Related to SOTERIA at the 22nd Annual WORLDSymposium™
Rhea-AI Summary
Polaryx (Nasdaq: PLYX) presented preclinical PLX-200 (reformulated gemfibrozil) data for Krabbe disease at WORLDSymposium on February 6, 2026. Findings in GALC-/- mice showed reduced astrogliosis and psychosine, myelin protection, restored motor function and increased lifespan. Polaryx received an FDA safe-to-proceed letter in October 2025 and plans to start the Phase 2 SOTERIA basket trial in H1 2026 to evaluate PLX-200 across multiple lysosomal storage disorders.
Positive
- Preclinical efficacy: restored motor function in GALC-/- mice
- Reduced psychosine and neuroinflammation in Krabbe disease model
- FDA safe-to-proceed letter received in October 2025
- Planned Phase 2 SOTERIA trial initiation targeted H1 2026
Negative
- Evidence limited to preclinical mouse data; no human efficacy yet
- SOTERIA start depends on CRO timelines and operational execution
Market Reaction
Following this news, PLYX has gained 5.55%, reflecting a notable positive market reaction. Our momentum scanner has triggered 21 alerts so far, indicating elevated trading interest and price volatility. The stock is currently trading at $4.64. This price movement has added approximately $11M to the company's valuation. Trading volume is very high at 3.3x the average, suggesting strong buying interest.
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Key Figures
Market Reality Check
Historical Context
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| 2026-02-05 | Board changes | Positive | -28.9% | Added three new directors as PLX-200 advances into Phase 2 SOTERIA. |
| 2026-02-03 | Clinical data update | Positive | -37.3% | Announced upcoming late-breaker PLX-200 data and SOTERIA trial plans. |
Recent ostensibly positive corporate and clinical updates have coincided with large negative price reactions, suggesting a pattern of selling into news.
In the past week, Polaryx announced new board appointments and highlighted progress toward the IND-approved Phase 2 SOTERIA basket trial, yet shares fell 28.89% after the board news and 37.29% after the late-breaker presentation announcement. Today’s detailed preclinical Krabbe disease data and planned SOTERIA launch in the first half of 2026 extend this clinical narrative, but the stock’s pre-news position remained depressed near its 52-week low.
Market Pulse Summary
The stock is up +5.5% following this news. A strong positive reaction aligns with the constructive clinical narrative around PLX‑200, which already had FDA safe-to-proceed clearance in October 2025 and a planned Phase 2 basket trial in the first half of 2026. Investors reviewing past events should note prior large moves around seemingly favorable news and consider whether elevated expectations or profit-taking previously led to volatility.
Key Terms
lysosomal storage disorders medical
Krabbe disease medical
proliferator activated receptor beta (PPARβ) medical
proliferator activated receptor alpha (PPARα) medical
transcription factor EB (TFEB) medical
autophagy medical
contract research organization (CRO) technical
AI-generated analysis. Not financial advice.
Oral presentation highlighted the effect of gemfibrozil on Krabbe disease, one of four LSDs in the upcoming Phase 2 SOTERIA basket trial evaluating PLX-200 (reformulated gemfibrozil)
PARAMUS, NJ, Feb. 10, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying therapies for rare, pediatric lysosomal storage disorders (“LSDs”), announced that the late-breaking presentation of new data for PLX-200, an investigational therapy for the treatment of Krabbe disease (globoid cell leukodystrophy) at the 22nd Annual WORLDSymposium™, was given on February 6, 2026 in San Diego, Calif.
Key Findings and Scientific Perspective
At the Contemporary Forum, Late-breaking Science and the Rapid Fire Competition, Shrijay Vijayan, Ph.D., MBA, Polaryx Therapeutics’ Chief Scientific and Business Development Officer, presented preclinical findings highlighting that orally administered gemfibrozil in a mouse model of Krabbe disease (GALC-/-):
-
- Reduces astrogliosis and neuro-inflammation
- Protects myelin
- Reduces psychosine accumulation, a hallmark toxic metabolite found in Krabbe disease patients
“Molecular mechanism involved recruitment of proliferator activated receptor beta (PPARβ) to the myelin-specific gene promoters in addition to proliferator activated receptor alpha (PPARα) mediated increase in lysosome biogenesis, autophagy and suppression of neuroinflammation,” noted Dr. Vijayan. “Most importantly, gemfibrozil restored motor functions and increased lifespan in GALC-/-mice.”
CEO Perspective
“These findings on PLX-200 as a potential novel therapeutic approach for Krabbe disease were encouraging as we prepare for the launch of the SOTERIA trial this year. The data deepen our confidence in the potential to translate PLX‑200’s strong preclinical evidence toward potential benefit for patients and families living with LSDs,” said Alex Yang, Chairman and Chief Executive Officer, Polaryx Therapeutics, Inc.
“Along with the data presentation, the Polaryx team, including Dr. Vijayan, Dr. Lisa Bollinger, Polaryx’s Chief Medical Officer, Dr. Minsu Kang, Polaryx’s Director of Regulatory and Clinical Affairs and myself, was delighted to engage with dedicated patient advocacy groups and clinicians who attended WORLDSymposium™ 2026. We were greatly encouraged by the positive feedback received from the LSD community, both in the excitement around the PLX-200 data and the forthcoming launch of the SOTERIA trial,” concluded Mr. Yang.
Connection to Prior Findings
These findings continue to build upon PLX-200’s existing base of preclinical data across a number of LSDs and highlight the drug’s multi-modal approach. As a PPARα activator, gemfibrozil promotes the expression of transcription factor EB (TFEB) regulated genes. TFEB is a master regulator known to be involved in coordinated regulation of a large number of lysosomal genes including CLN2, CLN3, HexB, GalC and others via coordinated lysosomal expression and regulation (CLEAR) elements leading to lysosomal biogenesis and improvement in the cellular clearance of accumulated storage materials in neurodegenerative diseases.
PLX-200 promotes expression of anti-inflammatory genes such as interleukin-1 receptor antagonist (IL-1Rα) and suppressor of cytokine signaling 3 (SOCS3) reducing neuro-inflammation. As a PPARα activator, PLX-200 has also been shown to suppress neuronal cell death and promote neuronal survival via expression of growth factors such as glial cell line-derived neurotrophic factor (GDNF).
Next Steps
The encouraging findings announced in WORLDSymposium™ 2026 for Krabbe disease continue to support the rationale for advancing PLX‑200 in the SOTERIA trial. The SOTERIA trial represents a flexible and resource‑efficient opportunity to further evaluate PLX‑200’s preclinical science across multiple LSDs while generating data to inform its future development pathway. Polaryx received a safe‑to‑proceed letter from the FDA in October 2025 and is actively working with its contract research organization (CRO) to initiate the trial in the first half of 2026.
About Krabbe Disease
Krabbe disease, also known as globoid cell leukodystrophy, is caused by mutations in the galactosylceramidase (“GALC”) gene, leading to galactosylceramidase deficiency and an inability to break down certain lipids in the body. This results in accumulation of the toxic metabolite psychosine in the brain and other areas of the nervous system, causing demyelination and severe neurological decline.
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. 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
FAQ
What did Polaryx (PLYX) present about PLX-200 for Krabbe disease at WORLDSymposium 2026?
Does Polaryx say PLX-200 improved survival in Krabbe disease mice (PLYX)?
What regulatory progress has Polaryx (PLYX) reported for the SOTERIA Phase 2 trial?
Which lysosomal storage disorders will PLX-200 target in the SOTERIA basket trial (PLYX)?
How does PLX-200 (gemfibrozil) purportedly work biologically according to Polaryx (PLYX)?
