Summary of Design Therapeutics FY Conference Call Company Overview - **Company**: Design Therapeutics (NasdaqGS:DSGN) - **Event**: FY Conference on December 03, 2025 Key Updates on Products and Programs DT-818 for DM1 - **Clinical Development**: DT-818 is set to enter clinical studies in the first half of 2026 for the DM1 program [3][16] - **Mechanism of Action**: DT-818 is a gene-targeted chimera designed to reduce the expression of mutant toxic DMPK RNA, which is the genetic cause of DM1. It targets the long CTG repeats in the mutant allele, aiming to restore cellular health [3][4] - **Differentiation**: Unlike other oligonucleotide-based therapies, DT-818 distributes widely to all affected tissues, including the CNS, potentially offering broader therapeutic benefits [5][6] - **Efficacy in Preclinical Models**: DT-818 demonstrated over 90% reduction in mutant RNA foci in preclinical models, significantly outperforming competitors that achieved only 30%-55% reduction [6][8] - **Clinical Translation**: There is a correlation between toxic foci reduction and clinical benefits, with existing literature supporting the link between splicing improvements and clinical outcomes [13][14] DT-216P2 for Friedreich's Ataxia (FA) - **Clinical Hold Lifted**: The company is off clinical hold and is conducting the RESTOR-FA study to evaluate DT-216P2's effect on increasing endogenous frataxin expression [22][23] - **Study Design**: The study is a multiple ascending dose study, measuring frataxin levels in whole blood and muscle tissue [30][32] - **Expected Data**: Results from the RESTOR-FA study are anticipated in the second half of 2026, with a focus on significant increases in frataxin levels being a potential regulatory endpoint [28][38] DT-168 for Fuchs' Dystrophy - **Study Design**: A biomarker phase two study is ongoing, where patients scheduled for corneal transplants will use DT-168 eye drops to assess splicing effects in corneal endothelial cells [40][41] - **Innovative Approach**: The study utilizes discarded corneal tissue to measure the drug's efficacy, marking a novel approach in the field [41] - **Observational Study**: Concurrently, an observational study is evaluating endpoints like visual quality and corneal edema to inform future clinical studies [42] Financial Position - **Cash Balance**: The company reported over $200 million in cash, providing a runway into 2029 [43] Conclusion - Design Therapeutics is advancing multiple innovative therapies targeting rare genetic diseases, with significant clinical studies planned for 2026. The company maintains a strong financial position to support its research and development efforts.

Core Insights - Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for serious degenerative genetic diseases [2] - The company utilizes its GeneTAC platform to create therapies that target specific disease-causing genes [2] - Upcoming investor conferences will feature management participation in fireside chats, with live webcasts available [1][3] Company Overview - Design Therapeutics is developing a new class of therapies based on GeneTAC gene targeted chimera small molecules [2] - Current clinical-stage programs include DT-216P2 for Friedreich ataxia, DT-168 for Fuchs endothelial corneal dystrophy, and DT-818 for myotonic dystrophy type-1 [2] - The company is also advancing a program for Huntington's disease and exploring multiple genomic medicine discovery efforts [2]

Core Insights - Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for serious degenerative genetic diseases [3] Company Overview - The company utilizes a platform of GeneTAC gene targeted chimera small molecules to create a new class of therapies aimed at modifying the expression of disease-causing genes [3] - Current clinical-stage programs include DT-216P2 for Friedreich ataxia and DT-168 for Fuchs endothelial corneal dystrophy, with additional programs in myotonic dystrophy type-1 and Huntington's disease [3] - Design Therapeutics is also engaged in discovery efforts for multiple genomic medicines [3] Upcoming Events - Management will participate in a fireside chat at the 2025 Cantor Global Healthcare Conference on September 4, 2025, at 10:55 a.m. ET in New York [1] - A live webcast of the event will be available on the company's website and archived for at least 30 days [2]

Core Insights - Design Therapeutics, Inc. announced favorable data from a Phase 1 trial of DT-168, a treatment for Fuchs endothelial corneal dystrophy (FECD), which will be presented at an upcoming industry event [1][3] - DT-168 is a GeneTAC® small molecule eye drop targeting the mutant TCF4 gene responsible for FECD, a condition affecting millions with no approved disease-modifying therapies [2][3] - The company plans to initiate a Phase 2 biomarker trial later in 2025 to further evaluate DT-168's safety and efficacy in FECD patients [5] Group 1: Clinical Trial Results - The Phase 1 trial was a double-masked, placebo-controlled study involving 24 healthy volunteers who received either placebo or DT-168 eye drops twice daily for seven days [3][8] - DT-168 was well-tolerated with no serious adverse events or ocular adverse events reported, and systemic exposure was below the limit of quantitation across all participants [8] Group 2: Future Development Plans - Design Therapeutics plans to conduct a Phase 2 biomarker trial to assess DT-168's safety and corneal endothelium biomarkers in FECD patients scheduled for corneal transplant surgery [5] - The Phase 2 trial will involve administering 0.5% DT-168 eye drops twice daily for approximately four weeks before surgery, with results expected in 2026 [5] Group 3: Company Overview - Design Therapeutics is focused on developing a new class of therapies using its GeneTAC® platform, which targets disease-causing genes [6] - The company is also advancing other programs for conditions such as Friedreich ataxia, myotonic dystrophy type-1, and Huntington's disease [6]

Core Insights - Design Therapeutics, Inc. is advancing its DT-168 program for Fuchs endothelial corneal dystrophy (FECD) and will present updates at Eyecelerator @ Park City 2025 on May 2, 2025 [1] - DT-168 is a GeneTAC® small molecule eye drop targeting the CTG repeat expansion in the TCF4 gene to reduce mutant gene expression linked to FECD [2] - The Phase 1 trial results for DT-168 will be showcased, highlighting its potential to restore endothelial function in a disease with no current disease-modifying treatments [3] Company Overview - Design Therapeutics is a clinical-stage biotechnology company focused on developing GeneTAC® therapies aimed at addressing serious degenerative genetic diseases [4] - The company is also developing DT-216P2 for Friedreich ataxia and has programs targeting myotonic dystrophy type-1 and Huntington's disease [4]