Design Therapeutics Reports Q1 2024 Financials and Upcoming Milestones

Design Therapeutics, Inc., a biotechnology firm listed on Nasdaq under the ticker DSGN, has unveiled its first quarter financial results for 2024 and provided updates on its ongoing projects. The company is pioneering treatments for severe degenerative genetic ailments through innovative small molecules that interact with a patient’s natural genome.

Dr. Pratik Shah, the company's chairperson and CEO, emphasized the company's commitment to advancing therapies for significant genetic disorders. He highlighted the progress in Friedreich Ataxia (FA) with DT-216P2, which has shown promise in increasing levels of endogenous frataxin. Patient trials for DT-216P2 are slated to kick off next year.

Additionally, the company is making strides with its observational study for Fuchs Endothelial Corneal Dystrophy (FECD). This study aims to confirm FECD characteristics and evaluate potential treatment endpoints. Enrollment of 200 patients is ongoing, with a planned two-year follow-up. The initiation of Phase 1 development for DT-168 is expected later this year. DT-168 could become the inaugural treatment targeting the root cause of this degenerative corneal disease.

Design Therapeutics is also pushing forward its GeneTAC™ pipeline programs for Huntington’s Disease (HD) and Myotonic Dystrophy Type-1 (DM1). Preclinical studies are progressing, with promising results indicating a significant reduction in disease-causing gene expressions. These programs, encompassing four potential projects, could yield clinical proof-of-concept in the coming years, sustained by the company's strong financial position.

Business Highlights and Anticipated Milestones

Friedreich Ataxia (FA)
Design’s drug candidate for FA, DT-216P2, has shown an improved pharmacokinetic profile, better injection site reactions, and sustained drug exposure in nonclinical studies compared to previous formulations. The company is on track to complete GLP studies for DT-216P2 by the end of 2024, aiming to begin patient trials in 2025.

Fuchs Endothelial Corneal Dystrophy (FECD)
An observational study is being conducted by Design to confirm disease characteristics and evaluate potential progression endpoints before starting interventional treatment trials. The study aims to enroll 200 patients with a planned two-year follow-up. Phase 1 development for DT-168 is expected to begin in 2024.

Pipeline Programs
Design is advancing the preclinical characterization of lead molecules toward selecting development candidates for HD and DM1. In preclinical studies, GeneTAC™ molecules have selectively reduced the expression of the mutant HTT gene in the brain striatum by over 50% with systemic administration. Similarly, for DM1, these molecules have shown potent reduction in mutant DMPK gene expression in patient cells, eliminating foci and restoring normal splicing.

First Quarter 2024 Financial Results

R&D Expenses: The research and development expenses stood at $9.8 million for the quarter ending March 31, 2024.

G&A Expenses: General and administrative expenses were reported at $4.6 million for the same period.

Net Loss: The company reported a net loss of $11.1 million for the quarter.

Cash Position: As of March 31, 2024, Design Therapeutics had cash, cash equivalents, and marketable securities totaling $270.7 million. This financial reserve is expected to support the company’s operations into 2029, enabling the advancement of up to four programs to clinical proof-of-concept stages.

About Design Therapeutics
Design Therapeutics is a biotech company developing a new class of therapies based on its GeneTAC™ gene-targeted chimera small molecules platform. These molecules are designed to modulate the expression of specific disease-causing genes to address the underlying causes of diseases. Besides its lead molecule, DT-216, for Friedreich ataxia, the company is also progressing with programs for Fuchs endothelial corneal dystrophy, Huntington’s disease, and myotonic dystrophy type-1. Discovery efforts are ongoing for multiple genomic medicines.

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