Waltham, Mass. — Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, reported financial results for the fourth quarter and full year 2022 and business highlights.
“2022 was a momentous year for Dyne and our commitment to the rare muscle disease community as we advanced not one, but two programs from our FORCE™ platform into clinical trials. This exciting transition into a clinical-stage company required important work across the organization to engage with individuals and families living with DM1 and DMD, clinicians treating these diseases, regulators and other stakeholders,” said Joshua Brumm, president and chief executive officer of Dyne. “We remain focused on driving to meaningful clinical data readouts for our ACHIEVE and DELIVER trials anticipated in the second half of 2023, including evaluating key biomarkers of dystrophin in DMD and splicing in DM1. With cash runway expected through 2024 and strong fundamentals across the business, we believe we are well positioned to achieve our near-term milestones and to advance our mission of delivering life-transforming therapies for people with serious muscle diseases.”
- Enrollment continues in ACHIEVE, a Phase 1/2 global clinical trial evaluating DYNE-101 in adult patients with myotonic dystrophy type 1 (DM1). ACHIEVE, which is designed to be a registrational trial, consists of a 24-week multiple ascending dose (MAD) randomized, placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension. The primary endpoints are safety and tolerability, with secondary endpoints of pharmacokinetics and pharmacodynamics, including change from baseline in splicing, as well as measures of muscle strength and function.
- Enrollment continues in DELIVER, a Phase 1/2 global clinical trial evaluating DYNE-251 in males with Duchenne muscular dystrophy (DMD) who have mutations amenable to exon 51 skipping therapy. DELIVER, which is designed to be a registrational trial, consists of a 24-week MAD randomized, placebo-controlled period, a 24-week open-label extension and a 96-week long-term extension. The primary endpoints are safety, tolerability and change from baseline in dystrophin levels as measured by Western blot. Secondary endpoints include measures of muscle function, exon skipping and pharmacokinetics.
- In recognition of Rare Disease Day, held annually on the last day of February, Dyne employees hosted members of the rare muscle disease community, and their perspectives are featured in a video available at https://www.dyne-tx.com/rare-disease-day-2023/.
Upcoming Events & Key 2023 Milestones
- Dyne anticipates reporting initial data in the second half of 2023 from:
- the MAD placebo-controlled portion of the ACHIEVE trial of DYNE-101 in DM1 on safety, tolerability and splicing
- the MAD placebo-controlled portion of the DELIVER trial of DYNE-251 in DMD on safety, tolerability and dystrophin
- Additional preclinical data supporting Dyne’s DMD program as well as overviews of the DELIVER and ACHIEVE trial designs will be featured in poster presentations during the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 19-22, 2023.
Fourth Quarter and Full Year 2022 Financial Results
Cash position: Cash, cash equivalents and marketable securities were $256.0 million as of December 31, 2022, which is anticipated to fund operations through 2024.
Research and development (R&D) expenses: R&D expenses were $33.2 million and $42.3 million for the quarters ended December 31, 2022 and 2021, respectively. R&D expenses were $142.8 million and $121.3 million for the years ended December 31, 2022 and 2021, respectively.
General and administrative (G&A) expenses: G&A expenses were $7.0 million and $9.7 million for the quarters ended December 31, 2022 and 2021, respectively. G&A expenses were $28.2 million and $28.7 million for the years ended December 31, 2022 and 2021, respectively.
Net loss: Net loss for the quarter ended December 31, 2022 was $38.8 million, or $0.74 per basic and diluted share. This compares with a net loss of $51.8 million, or $1.00 per basic and diluted share, for the quarter ended December 31, 2021. Net loss for the year ended December 31, 2022 was $168.1 million, or $3.23 per basic and diluted share. This compares with a net loss of $149.3 million, or $2.93 per basic and diluted share, for the year ended December 31, 2021.
About Dyne Therapeutics
Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases. With its proprietary FORCE™ platform, Dyne is developing modern oligonucleotide therapeutics that are designed to overcome limitations in delivery to muscle tissue. Dyne has 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). For more information, please visit https://www.dyne-tx.com/, and follow us on Twitter, LinkedIn and Facebook.