Avidity Biosciences Announces Q3 2024 Financial Results and Updates

Avidity Biosciences, Inc. recently disclosed its financial performance for the third quarter ended September 30, 2024, and provided updates on significant corporate advancements. The biopharmaceutical company, which specializes in Antibody Oligonucleotide Conjugates (AOCs™), is progressing with three pivotal clinical trials for rare diseases with limited or no therapeutic options.

Sarah Boyce, President and CEO of Avidity, emphasized the company's commitment to advancing their clinical trials. Positive data was reported from the 5 mg/kg cohort of the Phase 1/2 EXPLORE44™ trial for DMD44, and the enrollment for the EXPLORE44 Open-Label Extension (OLE) has begun. Additionally, the enrollment for the global Phase 3 HARBOR™ trial for del-desiran in DM1 is proceeding as planned. The biomarker cohort of the FORTITUDE™ study for FSHD has also been initiated, marking a significant stride towards a potential accelerated approval path for del-brax.

Mike MacLean, CFO and Chief Business Officer, highlighted the company's strong financial standing, with a cash position of approximately $1.6 billion. This financial robustness is attributed to a successful public offering in August, following positive clinical data from the EXPLORE44 program. The funds are directed towards advancing current programs, developing additional candidates from the DMD pipeline, and expanding the company's global commercial infrastructure.

Key developments include:

1. **Del-zota (AOC 1044) for DMD44**:
- Positive initial data from the 5 mg/kg cohort of the Phase 1/2 EXPLORE44™ trial demonstrated exceptional delivery to skeletal muscle, with a 25% increase in near full-length dystrophin production and a significant reduction in creatine kinase levels. Del-zota showed favorable safety and tolerability. The trial is fully enrolled, and the enrollment for the EXPLORE44-OLE has started.

2. **Del-brax (AOC 1020) for FSHD**:
- The biomarker cohort in the Phase 1/2 FORTITUDE™ trial has commenced, with 2 mg/kg of del-brax being administered every six weeks to ensure continuous suppression of DUX4. Positive initial data from the Phase 1/2 FORTITUDE trial showed significant reductions in DUX4-regulated genes and novel circulating biomarkers, along with trends of functional improvement and favorable safety. The functional cohort is set to begin in the first half of 2025.

3. **Del-desiran (AOC 1001) for DM1**:
- Enrollment for the global Phase 3 HARBOR™ trial is ongoing, and the U.S. FDA has lifted the partial clinical hold on del-desiran, indicating progress in the trial.

Additional advancements include the promotion of Kathleen Gallagher to Chief Program Officer and plans to advance candidates from the DMD franchise following positive data on del-zota. Avidity aims to extend their research and development capabilities, including exploring precision cardiology and next-generation technology innovations.

Financially, Avidity reported collaboration revenues of $2.3 million for the third quarter of 2024, primarily from partnerships with Bristol Myers Squibb. R&D expenses increased to $77.2 million due to advancements in their pipeline and expansion of research capabilities. General and Administrative expenses rose to $23.3 million, reflecting higher personnel costs to support expanded operations.

Avidity Biosciences is dedicated to transforming the field of RNA therapeutics with its proprietary AOC platform. The company's efforts are focused on delivering innovative therapies for muscle diseases and expanding their reach into cardiology and immunology.