Polaryx Therapeutics (PLYX)
Develops PLX-200 and other candidates for rare pediatric lysosomal storage disorders.
Something off on this page? Send us feedback.
Develops small-molecule and gene-therapy candidates for rare pediatric lysosomal storage disorders. Its lead candidate, PLX-200 (gemfibrozil), is a repurposed, reformulated oral therapy being advanced through the SOTERIA Phase 2 basket study; near-term work centers on trial initiation and clinical evidence.
Develops small-molecule and gene-therapy candidates for rare pediatric lysosomal storage disorders. Its lead candidate, PLX-200 (gemfibrozil), is a repurposed and reformulated oral small molecule.
PLX-200 is being advanced through SOTERIA, an open-label, single-arm Phase 2 basket study assessing safety, tolerability, and clinical activity in CLN2, CLN3, Krabbe disease, and Sandhoff disease. The program follows the FDA's 505(b)(2) pathway.
Trial initiation was planned in the United States, with potential expansion to Europe, Asia, or other jurisdictions. The current operating path centers on initiating SOTERIA and generating clinical evidence from the lead program.
Company facts
Unavailable right now.
EPSReportedEstimate
RevenueReportedEstimate
Balance sheet, Jun 30, 2026Cash & short-term investments $11.5M · Total debt $0 · Total assets $11.8M · Shareholders' equity $10.9M
- Revenue
- $0
- Operating income
- −$1.4M
- Net income
- −$1.4M
No dividends on record.
| Institutions14 holders · as of Mar 31, 2026 | 4.55% | 2.27M shares | |
| Otherremainder | 95.45% | 47.58M shares |
49.85M company shares as of Aug 21, 2026 · institutions as of Mar 31, 2026 · holders as of their latest filing
The share count has not been confirmed by a second filing; stakes use it as it stands.
Top shareholders13F · 13D/G · Form 4
| Holder | Stake | Shares | Evidence |
|---|---|---|---|
Vanguard Capital Management LLCInstitution | 3.33% | 1.66M | 13Fas of Mar 31, 2026 |
Geode Capital Management, LLCInstitution | 0.34% | 170,673 | 13Fas of Mar 31, 2026 |
Millennium Management LLCInstitution | 0.26% | 127,525 | 13Fas of Mar 31, 2026 |
Vanguard Fiduciary Trust CoInstitution | 0.21% | 104,556 | 13Fas of Mar 31, 2026 |
State Street CorpInstitution | 0.12% | 58,104 | 13Fas of Mar 31, 2026 |
| See all 14 → | |||
13F — a manager's quarterly holdings
Insider tradesForm 4
Nothing filed in the last 12 months.
- BioMarin
Approved CLN2 enzyme replacement therapy
- Sanofi (ADR)
Approved treatment in Niemann–Pick A/B disease
- Ultragenyx Pharmaceutical
MPS therapies and neuro-LSD gene-therapy development
- REGENXBIO
One-time gene therapies for pediatric LSDs
ComparePre-market
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| Polaryx Therapeutics | $2.02 | −0.49% | $101.7M | — | — | — | — |
| BioMarin | $67.20 | +0.28% | $12.94B | 171.7 | 10.8 | 3.8 | +19.9% |
| Sanofi (ADR) | $44.52 | +0.12% | $106.78B | 78.2 | — | 1.9 | +11.2% |
| Ultragenyx Pharmaceutical | $14.75 | −44.45% | $2.61B | — | — | 3.6 | +28.5% |
| REGENXBIO | $9.60 | +3.00% | $496.3M | — | — | 2.8 | +405.7% |
| Median | 125.0 | 10.8 | 3.2 | +24.2% |






