Press Releases April 15, 2026 09:15 AM

Polaryx Therapeutics Recognized with Pinnacle Award for Excellence in Pediatric Care

Polaryx Therapeutics Wins 2026 Pinnacle Award for Excellence in Pediatric Care, Recognizing Innovation in Treatment of Rare Lysosomal Storage Disorders

By Leila Farooq PLYX
Polaryx Therapeutics Recognized with Pinnacle Award for Excellence in Pediatric Care
PLYX

Polaryx Therapeutics, a Nasdaq-listed clinical-stage biotech company focused on rare pediatric lysosomal storage disorders, has been honored with the 2026 Pinnacle Award for Excellence in Pediatric Care. The award recognizes Polaryx's innovative and scalable platform targeting shared biological mechanisms underlying multiple LSDs, potentially transforming treatment for these rare diseases. The company is advancing PLX-200, a promising small molecule candidate, with plans for a Phase 2 basket trial to evaluate safety and efficacy.

Key Points

  • Polaryx Therapeutics received the 2026 Pinnacle Award as a Diamond honoree for excellence in pediatric care, highlighting its innovative approach to treating lysosomal storage disorders (LSDs).
  • The company employs a platform targeting common disease mechanisms like lysosomal dysfunction and neuroinflammation, aiming to address multiple rare diseases simultaneously rather than developing treatments in isolation.
  • Polaryx plans to initiate the SOTERIA Phase 2 basket trial for its lead candidate PLX-200, emphasizing its commitment to advancing clinical development in rare pediatric diseases.

PARAMUS, NJ, April 15, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company focused on developing disease-modifying therapies for rare, pediatric lysosomal storage disorders (LSDs), announces that it has been named a winner of the 2026 Pinnacle Awards for Healthcare, being selected as a Diamond honoree for Excellence in Pediatric Care.

Polaryx was recognized for its innovative and scalable approach to treating lysosomal storage disorders (LSDs), a group of rare genetic diseases that cause progressive neurological decline and early mortality. Rather than developing therapies for individual conditions in isolation, the company is advancing a platform designed to target shared biological mechanisms, including lysosomal dysfunction and neuroinflammation, enabling a broader and more efficient approach to rare disease treatment.

"While the limited existing therapies across these diseases has advanced care for lysosomal storage disorders, meaningful unmet need remains—particularly around reducing treatment burden for children and their families,” said Polaryx Therapeutics Chief Executive Officer Alex Yang. “Our work is focused on developing effective patient‑centered options that can broaden the treatment landscape for patients and their families."

The Pinnacle Awards are a prestigious annual program designed to acknowledge outstanding cross-industry achievements. Its core mission is to highlight companies, products, and professionals demonstrating unparalleled innovation, creativity, and industry impact. All nominees undergo a meticulous evaluation by a distinguished panel of industry authorities, media members, and consultants to guarantee that recognition is awarded strictly to the most deserving candidates.

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 most recently filed Form 10K with the SEC 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
[email protected]

Investor Contacts:
CORE IR
(212) 655-0924
[email protected]


Risks

  • Clinical trial risks including potential delays or failures in the SOTERIA Phase 2 basket trial assessing PLX-200 efficacy and safety, impacting development timelines.
  • Regulatory risks related to the timing, completion, and outcome of regulatory filings and approvals necessary for product commercialization.
  • Market and financial uncertainties such as liquidity and capital resources constraints, and macroeconomic conditions that may impact the company's operations and clinical progress.

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