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Design Therapeutics to Present Phase 1 Data for Fuchs Endothelial Corneal Dystrophy Program at Eyecelerator @ Park City 2025

1. Design Therapeutics to present DT-168 progress at Eyecelerator on May 2. 2. Safety findings from Phase 1 trials will be shared, targeting FECD treatment. 3. DT-168 is a GeneTAC® eye drop aimed at improving corneal endothelial function. 4. No disease-modifying treatments exist for FECD, highlighting DT-168's potential impact. 5. Next conference coincides with ARVO meeting, indicating industry interest.

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Why Bullish?

The advancement of DT-168 could significantly enhance its market position, similar to how other biotech advancements have led to price surges.

How important is it?

The article highlights significant clinical advancements for DSGN, impacting future market potential.

Why Long Term?

Successful Phase 2 trials can establish DT-168 as a viable market treatment, attracting long-term investment.

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April 21, 2025 16:01 ET  | Source: Design Therapeutics, Inc. CARLSBAD, Calif., April 21, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that it will present an update on the progress of its DT-168 program for Fuchs endothelial corneal dystrophy (FECD) at Eyecelerator @ Park City 2025 on Friday, May 2, 2025, at 1:30 p.m. MT in Park City, UT. The presentation will include safety findings from the Phase 1 single- and multiple-ascending dose trial of DT-168 in healthy adult volunteers and plans for Phase 2 clinical development in FECD patients. DT-168 is a GeneTAC® small molecule, formulated as an eye drop, that is designed to selectively target the CTG repeat expansion in the TCF4 gene and reduce the expression of the mutant gene product that causes corneal endothelial cell dysfunction leading to FECD. “We are pleased for the opportunity to showcase our Phase 1 results for DT-168 at Eyecelerator, a forum dedicated to highlighting innovation in the field of ophthalmology,” said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics. “FECD is a progressive corneal disease with no disease-modifying treatments currently available, ultimately leading to corneal transplant surgery for many patients. We believe DT-168, formulated as an eye drop, has the potential to restore endothelial function, representing a significant advancement in the treatment of FECD.” Eyecelerator conferences, backed by the American Academy of Ophthalmology, highlight industry advancements and innovative new products disrupting eye care. Eyecelerator @ Park City 2025 is adjacent to the Association for Research in Vision and Ophthalmology (ARVO) meeting being held May 4 - 8, 2025, in Salt Lake City, UT. About Design TherapeuticsDesign Therapeutics is a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC® gene targeted chimera small molecules. The company’s GeneTAC® molecules are designed to either dial up or dial down the expression of a specific disease-causing gene to address the underlying cause of disease. In addition to its GeneTAC® programs, DT-216P2, in development for patients with Friedreich ataxia, and DT-168, for Fuchs endothelial corneal dystrophy, the company is advancing programs in myotonic dystrophy type-1 and Huntington’s disease. Discovery efforts are underway for multiple genomic medicines. For more information, please visit designtx.com. Forward-Looking StatementsStatements in this press release that are not purely historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to the potential for DT-168 to restore endothelial function and its impact on the treatment of FECD; Design's ability to advance its pipeline of GeneTAC® small molecules; and the capabilities and potential advantages of Design’s pipeline of GeneTAC® small molecules. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “designed to,” “anticipates,” “capable of,” “plans to,” “expects,” “estimate,” “intends,” “will,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Design’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with: the acceptance of INDs by the FDA or similar applications by foreign regulatory agencies for the conduct of planned clinical trials of our product candidates and our proposed design of future clinical trials; nonclinical development activities and results of nonclinical studies; conducting a clinical trial and patient enrollment, which are affected by many factors, and any difficulties or delays encountered with such clinical trial or patient enrollment may delay or otherwise adversely affect Design’s clinical development plans; the process of discovering and developing therapies that are safe and effective for use as human therapeutics and operating as a development stage company; undesirable side effects or other undesirable properties, which could cause Design or regulatory authorities to suspend or discontinue clinical trials and thereby delay or prevent Design’s product candidates’ development or regulatory approval; Design’s ability to develop, initiate or complete nonclinical studies and clinical trials for its product candidates; whether promising early research or clinical trials will demonstrate safety and/or efficacy in later nonclinical studies or clinical trials; changes in Design’s plans to develop its product candidates; reliance on third parties to successfully conduct clinical trials and nonclinical studies; competitive products, which may make any products we develop or seek to develop obsolete or noncompetitive; Design’s reliance on key third parties, including contract manufacturers and contract research organizations; Design’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; regulatory developments in the United States and foreign countries; Design’s ability to obtain and maintain intellectual property protection for its product candidates; Design’s ability to recruit and retain key scientific or management personnel; and market conditions. For a more detailed discussion of these and other factors, please refer to Design’s filings with the Securities and Exchange Commission (SEC), including under the “Risk Factors” heading of Design’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024, as filed with the SEC on March 10, 2025. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Design undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, except as required by law. Contact:Renee LeckTHRUST Strategic Communicationsrenee@thrustsc.com

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