Design Therapeutics reports Q3 EPS (28c), consensus (41c)

Reports Q3 cash, cash equivalents and marketable securities were $290.9M as of September 30. As a result of a strategic realignment to prioritize long-term growth, focus capital resources on program spend and implement cost savings, Design now expects its cash, cash equivalents and marketable securities as of September 30, to fund its planned operating expenses through at least the next five years. “When we started Design just five years ago, we were looking to address the problem of genetic diseases differently by working alongside a person’s genome. Our solution, GeneTAC small molecules, are thoughtfully designed to dial up or down the expression of specific genes, addressing the root cause of disease without permanently modifying their genome. We believe this technology has the potential to transform the treatment landscape for many patients suffering from devastating degenerative diseases,” said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics. “The recently reported data from our observational biomarker study and multiple-ascending dose Phase 1 trial in patients with Friedreich ataxia (FA) provided us with important proof-of-concept for our GeneTAC platform, with the first human in history with FA experiencing a restoration of mRNA to carrier levels without the need for an irreversible genetic modification. Additionally, the initial clinical results provided important learnings on the behavior and tolerability of our lead GeneTAC molecule, DT-216, in patients, leading to our efforts to further optimize the formulation of DT-216 containing excipients with improved injection site tolerability, to enable the long-term treatment of patients with FA.” Dr. Shah continued, “We believe that GeneTAC molecules can be transformative therapies for patient populations with urgent unmet medical needs, including FA, Fuchs endothelial corneal dystrophy and myotonic dystrophy type-1. To maximize our future potential, we are undertaking a close review of our business to build on the progress already made and determine the optimal paths forward and development timelines for each of our programs. Already, we have sharpened our focus to prioritize our long-term growth, allowing us to strategically deploy our capital for pipeline investment that extends our operating plans through at least the next five years. We look forward to sharing more on our near- and long-term strategic priorities as part of a comprehensive update in early 2024. This is an important time for Design, and it’s an honor to lead this incredible team as we work to translate the proof-of-concept established for our platform into meaningful treatments for patients and the treating communities.”