Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced its 2024 corporate priorities and catalysts for the next stage of growth. In mid-2024, Avidity plans to initiate the global Phase 3 HARBOR™ trial of AOC 1001 for adults living with myotonic dystrophy type 1 (DM1). The robust data package of AOC 1001 from the Phase 1/2 MARINA® trial and open-label extension study, MARINA-OLE™, has demonstrated consistent improvements across multiple functional endpoints including myotonia, muscle strength and mobility, and long-term favorable safety and tolerability results in people living with DM1. Avidity also plans to report data in 2024 from all three of its ongoing clinical programs targeting three distinct rare muscle diseases: DM1, Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44) and facioscapulohumeral muscular dystrophy (FSHD) while advancing its cardiology programs toward clinical development. In addition, Avidity announced the appointment of Eric B. Mosbrooker as Chief Strategy Officer.
2024 Upcoming Milestones & Key Highlights
- Initiation of global Phase 3 HARBOR™ trial of AOC 1001 for DM1 in mid-2024
- Data readouts from all three clinical programs for three distinct muscle diseases
- Q1 2024: First look at AOC 1001 efficacy data from MARINA-OLE™ trial in people living with DM1
- Q2 2024: AOC 1020 data from a preliminary assessment in approximately half of patients in Phase 1/2 FORTITUDE™ trial in FSHD
- 2H 2024: First look at AOC 1044 data in people living with DMD44 from Phase 1/2 EXPLORE44™ trial
- Dose-escalation of remaining study participants from 2 mg/kg to 4 mg/kg of AOC 1001 in the MARINA-OLE trial
- Advance wholly-owned and partnered cardiology programs toward clinical development following recent expansion of cardiology collaboration with Bristol Myers Squibb
- Strong cash balance with funding through 2025
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