Design Therapeutics reports Q4 EPS (21c), consensus (31c)

Reports cash, cash equivalents and marketable securities were $281.8M as of December 31, . Design expects its cash, cash equivalents and marketable securities as of December 31 to fund its planned operating expenses through the next five years. “At Design, our vision is to develop a portfolio of first or best-in-class small molecules capable of treating a host of degenerative diseases by working with a patient’s natural genome to restore cellular health. In recent months we have undertaken important work to evaluate the scope of our GeneTAC(TM) platform, which we believe has the potential to deliver clinical proof-of-concept in up to four programs, subject to research and development results, under our current cash runway through the next five years,” said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics. Dr. Shah continued, “Our lead program in FA has a new drug product, DT-216P2, that we have designed to have an improved pharmacokinetic and injection site safety profile, which positions us to resume clinical development for this debilitating, degenerative, neuro-muscular genetic disease. This work builds on encouraging data in FA patients from our previous clinical trial. In addition, our FECD program, DT-168, which has the potential to be the first effective treatment addressing the root cause of this degenerative corneal disease, now has an IND that was cleared by the FDA. Finally, we are highlighting an exciting new program for Huntington’s Disease, an indication of tremendous unmet medical need, which further demonstrates the depth and versatility of our differentiated approach to genomic medicines.”