Design Therapeutics, Inc. (DSGN) Q1 2026 Earnings Call April 28, 2026 4:30 PM EDT

Company Participants

• Pratik Shah - Co-Founder, President, CEO, Principal Financial Officer & Executive Chairperson

Presentation

Design Therapeutics Advances DT-216 as Potential Friedreich's Ataxia Breakthrough

I'm Pratik Shah, CEO of Design Therapeutics. During this presentation, we will use forward-looking statements with regards to our business, R&D activities and financial conditions, which are subject to known and unknown risks and uncertainties. Actual results may differ materially due to various important factors, including those described in the Risk Factors section of our most recent Form 10-Q filed with the SEC.

Given the nature of the interest in the FA program and the design of the RESTORE-FA trial, we're delighted to provide a general update on our FA program, the key objectives of the RESTORE-FA study and the multiple biomarker endpoints being evaluated. And we're using this quarter's update to provide further context on how we're thinking about the study. The trial is proceeding well, and we're looking forward to data in the second half of 2026.

As a reminder, FA is a debilitating condition, which is caused by mutation in a single gene called the frataxin gene. The mutation causes low levels of production of frataxin, which causes all kinds of downstream dysfunction in multiple organs. There is an approved drug for FA called omaveloxolone, which targets Nrf2 and not frataxin. The mutation causing FA is a GAA-GAA repeat expansion in the first intron of the frataxin gene, which causes the level of normal mRNA to be low and therefore, levels of protein to be low. DT-216 is a heterobifunctional gene-targeted chimera or GeneTAC small molecule that is designed to recognize these long GAA repeat expansions and dial up normal frataxin RNA transcription.

Cellular data suggests a