- Avidity Biosciences announced a **positive pre-BLA meeting** with the U.S. Food and Drug Administration (FDA) for **delpacibart zotadirsen (del-zota)**, an investigational drug for Duchenne muscular dystrophy (DMD44).  **[1599901_20251013LA95734:0]** 
- The BLA submission for del-zota has been updated to **Q1 2026** from the previous guidance of year-end 2025.  **[1599901_20251013LA95734:0]** 
- This timing adjustment is to ensure the FDA receives **additional chemistry, manufacturing, and controls (CMC) data** at the time of submission.  **[1599901_20251013LA95734:0]** 
- Del-zota has received **Breakthrough Therapy designation** and is the first of three planned BLA submissions for the company over a 12-month period.  **[1599901_20251013LA95734:0]**

Avidity Biosciences Updates BLA Submission Timeline for del-zota

- Avidity Biosciences will deliver late-breaking oral and poster presentations on **delpacibart zotadirsen (del-zota)** at the **30th Annual International Congress of the World Muscle Society (WMS)**, held from **October 7-11, 2025**, in Vienna, Austria.  **[1599901_20251006LA90465:0]** 
- The data to be presented will reinforce and build on positive results announced in **September 2025**, which showed **reversal of disease progression and unprecedented improvement** in functional measures for participants with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) treated with del-zota for one year in the Phase 1/2 EXPLORE44® and Phase 2 EXPLORE44-OLE™ trials.  **[1599901_20251006LA90465:0]** 
- Presentations will cover trends toward improvement in functional and patient-reported outcomes, and increased dystrophin and improved muscle integrity markers in individuals with DMD44.  **[1599901_20251006LA90465:0]** 
- Del-zota is an investigational Antibody Oligonucleotide Conjugate (AOC™) therapeutic, and its safety and efficacy have not yet been established or approved by the FDA.  **[1599901_20251006LA90465:0]**

Avidity Biosciences to Present Del-Zota Data at World Muscle Society Congress

- Avidity Biosciences, Inc. has closed its underwritten public offering, which included the full exercise of the underwriters' option to purchase additional shares, totaling **17,250,000 shares** of common stock.  **[1599901_20250915LA73565:0]** 
- The shares were sold at a price of **$40.00 per share**, generating **gross proceeds of $690.0 million** for Avidity.  **[1599901_20250915LA73565:0]** 
- The company intends to use the net proceeds to advance its three late-stage clinical programs, build commercial inventory, expand commercial infrastructure, progress research and development for its AOC platform, and for general corporate purposes.  **[1599901_20250915LA73565:0]**

Avidity Biosciences Announces Closing of Public Offering

- Avidity Biosciences, Inc. (RNA) announced the **pricing of an upsized underwritten public offering** of **15,000,000 shares** of its common stock at **$40.00 per share** on September 11, 2025.  **[1599901_0001193125-25-202371_d25770dex992.htm:0]** **[1599901_0001193125-25-202371_d25770d8k.htm:1]** **[1599901_0001193125-25-202371_d25770dex11.htm:47]** 
- The offering is expected to generate **gross proceeds of $600.0 million** and **net proceeds of approximately $564.0 million** for the company, with the potential for net proceeds to reach **approximately $648.8 million** if the underwriters' option to purchase an additional **2,250,000 shares** is fully exercised.  **[1599901_0001193125-25-202371_d25770dex992.htm:0]** **[1599901_0001193125-25-202371_d25770d8k.htm:1]** 
- Avidity intends to use the net proceeds to **advance its three late-stage clinical programs**, build commercial inventory, expand commercial infrastructure, progress research and development for its AOC platform, and for general corporate purposes.  **[1599901_0001193125-25-202371_d25770dex991.htm:0]** **[1599901_0001193125-25-202371_d25770dex992.htm:0]**

Avidity Biosciences Prices Upsized Public Offering

- Avidity Biosciences is on track to file its first Biologics License Application (BLA) for **Dalzosia** (for boys and young men amenable to exon 44 skipping) by the end of **2025** for accelerated approval, with clinical updates expected in the next couple of weeks.  **[1599901_tx_380786_0]** **[1599901_tx_380786_2]** **[1599901_tx_380786_4]**  The company estimates approximately **900 boys and young men** in the U.S. are amenable to Exon 44 skipping.  **[1599901_tx_380786_3]** 
- Enrollment for the HARBOR study, a global Phase 3 pivotal study for **Daldistarone** (for myotonic dystrophy), was completed in **July**.  **[1599901_tx_380786_0]**  Data readout is guided for **Q2 2026**, with two-year follow-up data from the Mirena OLE study expected in **Q4 2025**.  **[1599901_tx_380786_0]** **[1599901_tx_380786_4]**  Myotonic dystrophy is estimated to affect **40,000 patients** in the U.S., with **10,000** already identified and genetically confirmed.  **[1599901_tx_380786_7]** 
- Enrollment for the FORTITUDE biomarker study for **Delbrax** (for FSHD) was completed in **March**.  **[1599901_tx_380786_0]**  Data readout is also guided for **Q2 2026**, and the company has reached alignment with the FDA on cDUX as the primary endpoint for accelerated approval.  **[1599901_tx_380786_0]** **[1599901_tx_380786_8]**  The opportunity size for FSHD is considered similar to, if not larger than, myotonic dystrophy.  **[1599901_tx_380786_8]** 
- Avidity Biosciences plans to globally commercialize its drugs itself, leveraging synergies across the programs as they all target muscle diseases and use the same antibody oligonucleotide conjugates (AOCs) platform.  **[1599901_tx_380786_0]** **[1599901_tx_380786_1]**

Avidity Biosciences (RNA)