Dyne Therapeutics, Inc. (NASDAQ: DYN) has reported its financial results for the fourth quarter and full year of 2023. The company highlighted positive initial clinical data from its Achieve trial in DM1 patients and Deliver trial in DMD patients, demonstrating proof-of-concept and validating the promise of the Force™ platform and targeted delivery to muscles. These achievements have led to a strengthened balance sheet following a $345 million public offering, extending the projected cash runway through 2025.
In the Phase 1/2 Achieve trial of Dyne-101 in DM1, initial efficacy assessment data showed a dose-dependent splicing correction, an increase in muscle delivery, and DMPK knockdown. Patients treated with 1.8 mg/kg of Dyne-101 experienced functional improvement in myotonia as well as an overall improvement in the myotonic dystrophy health index, suggesting potential benefits in the central nervous system. The trial is fully enrolled through the 5.4 mg/kg q8w cohort and is currently enrolling participants in the 6.8 mg/kg q8w cohort.
Regarding the Phase 1/2 Deliver trial of Dyne-251 in DMD, initial efficacy assessment data showed that once every 4-week administration of Dyne-251 reached levels of dystrophin expression, exon skipping, and percent dystrophin positive fibers that exceeded levels reported in a clinical trial for the current weekly standard of care for DMD exon 51, with a 24-fold lower total pmo dose. The trial is fully enrolled through the 20 mg/kg q4w cohort and is currently enrolling participants in the 40 mg/kg q8w cohort.
In terms of financial results, as of December 31, 2023, Dyne Therapeutics had $123.1 million in cash, cash equivalents, and marketable securities. Following the completion of an underwritten public offering of 19,722,500 shares of its common stock at a public offering price of $17.50 per share in January 2024, the company expects its cash, cash equivalents, and marketable securities to be sufficient to fund its operations through 2025. Research and development (R&D) expenses were $58.8 million and $33.2 million for the quarters ended December 31, 2023 and 2022, respectively, and $210.8 million and $142.8 million for the years ended December 31, 2023 and 2022, respectively. General and administrative (G&A) expenses were $8.8 million and $7.0 million for the quarters ended December 31, 2023 and 2022, respectively, and $31.4 million and $28.2 million for the years ended December 31, 2023 and 2022, respectively.
Dyne Therapeutics anticipates reporting data from multiple, higher dose cohorts from both the Achieve and Deliver trials in the second half of 2024, with the goal of initiating registrational cohorts by the end of 2024. The company is focused on leveraging the favorable safety profiles for both Dyne-101 and Dyne-251 and the adaptive nature of its trials to optimize dose and dose regimen, with plans to provide updates from both trials in the second half of 2024.
Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, with a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) and a preclinical program for facioscapulohumeral muscular dystrophy (FSHD).
The market has reacted to these announcements by moving the company's shares -7.2% to a price of $25.71. If you want to know more, read the company's complete 8-K report here.
