Avidity Biosciences, Inc. Stock Price, News & Analysis

Avidity Biosciences (Nasdaq: RNA) announced positive topline data from its Phase 1/2 EXPLORE44® trial for del-zota in treating Duchenne muscular dystrophy amenable to exon 44 skipping (DMD44). The trial demonstrated statistically significant improvements across key biomarkers:

• Approximately 40% increase in exon 44 skipping
• 25% of normal dystrophin production with total dystrophin restored up to 58% of normal
• Over 80% reduction in creatine kinase levels compared to baseline
• Favorable safety and tolerability across dose cohorts

Based on consistent results between dosing groups, Avidity selected 5 mg/kg every six weeks for their Biologics License Application (BLA) submission, planned for year-end 2025. The drug has received FDA's Orphan, Rare Pediatric Disease, and Fast Track designations, as well as EMA's Orphan designation.

Avidity Biosciences (Nasdaq: RNA) announced multiple presentations at the 2025 MDA Clinical & Scientific Conference in Dallas, Texas from March 16-19, 2025. The highlight includes presentation of topline data for del-zota from the Phase 1/2 EXPLORE44 trial, showing statistically significant increases in exon skipping and dystrophin levels in DMD44 patients.

Key events include:

• An oral presentation by Dr. Aravindhan Veerapandiyan on March 19
• Two poster presentations on del-zota results and the HARBOR™ trial design
• An industry forum breakfast on March 17 titled 'Advancing RNA Therapeutics' featuring expert panel discussion
• An investor and analyst webcast on March 17 to discuss EXPLORE44 trial data

The company will make presentations and posters available on their website following the conference.

Avidity Biosciences (Nasdaq: RNA) joins the global community in observing Rare Disease Day® 2025, demonstrating its commitment to raising awareness for rare neuromuscular diseases and the need for approved treatments. The company, which develops Antibody Oligonucleotide Conjugates (AOCs™), is participating in several initiatives including:

• Supporting the EveryLife Foundation's Rare Disease Week on Capitol Hill (February 24-26, 2025)
• Sponsoring Jett Foundation's 'Thriving with Duchenne' webinar on February 28, 2025
• Participating in EURORDIS 'More Than You Can Imagine' campaign

The company focuses on rare progressive disorders including DMD, DM1, and FSHD as well as rare cardiomyopathies. Rare Disease Day, established by EURORDIS in 2008, is observed on the last day of February annually, coordinating with over 70 national alliance patient organizations to honor those affected by rare diseases.

Avidity Biosciences (RNA) reported Q4 2024 financial results and milestones for its three clinical programs in rare neuromuscular diseases. The company ended 2024 with a strong balance sheet of $1.5 billion and is preparing for potential product launches starting in 2026.

Key financial metrics for Q4 2024 include: collaboration revenue of $3.0 million, R&D expenses of $95.6 million (up from $52.8M in Q4 2023), and G&A expenses of $28.3 million (up from $16.1M in Q4 2023).

Major 2025 milestones include:

• Del-zota: BLA submission planned for year-end 2025 with completed enrollment in EXPLORE44-OLE study
• Del-desiran: Phase 3 HARBOR trial enrollment completion expected mid-2025
• Del-brax: Global Phase 3 trial initiation and FORTITUDE biomarker cohort data in Q2

Avidity Biosciences (Nasdaq: RNA), a developer of Antibody Oligonucleotide Conjugates (AOCs™), has announced its participation in three major upcoming investor conferences in March 2025:

• TD Cowen 45th Annual Health Care Conference - March 4, 2025, at 3:10 p.m. ET
• Leerink Partners Global Healthcare Conference - March 11, 2025, at 1:00 p.m. ET
• Barclays 27th Annual Global Healthcare Conference - March 12, 2025, at 11:30 a.m. ET

The company will participate in fireside chats at each event. Live webcasts and archived replays will be available through Avidity's investor relations website.

Avidity Biosciences (RNA) has granted equity awards to 22 new non-executive employees under its 2022 Employment Inducement Incentive Award Plan. The awards, approved on February 20, 2025, include non-qualified stock options to purchase 107,000 shares and 53,500 restricted stock units (RSUs).

The stock options have an exercise price of $32.53 per share, matching Avidity's closing price on Nasdaq on February 20, 2025. The options will vest over four years, with 25% vesting after one year and the remainder vesting monthly over 36 months. The RSUs will vest in four equal annual installments. Both awards require continued employment with Avidity and were granted as employment inducements under Nasdaq Rule 5635(c)(4).

Avidity Biosciences (RNA) has granted equity awards to one new non-executive employee under its 2022 Employment Inducement Incentive Award Plan. The grants include a non-qualified stock option to purchase 10,500 shares of common stock at $28.92 per share and 5,250 restricted stock units (RSUs).

The stock options will vest over four years, with 25% vesting after one year from December 20, 2024, and the remaining vesting monthly over 36 months. The RSUs will vest in four equal annual installments. These awards were granted as employment inducements in accordance with Nasdaq Listing Rule 5635(c)(4) and are subject to continued employment with Avidity.

Avidity Biosciences (Nasdaq: RNA) has announced inducement grants to its new Chief Technical Officer, Charles Calderaro III. The grants include a non-qualified stock option to purchase 80,000 shares of common stock and 40,000 restricted stock units (RSUs) under the company's 2022 Employment Inducement Incentive Award Plan.

The stock option has an exercise price of $31.42 per share, matching Avidity's closing price on Nasdaq on January 6, 2025. The stock options will vest over four years, with 25% vesting after one year and the remainder vesting monthly over 36 months. The RSUs will vest in four equal annual installments. Both grants are subject to Mr. Calderaro's continued employment with Avidity.

Avidity Biosciences (RNA) announced significant plans for 2025, including its first Biologics License Application (BLA) submission for del-zota in treating Duchenne muscular dystrophy. The company confirmed an accelerated approval path and plans for multiple product launches in DMD44, DM1, and FSHD rare muscle diseases.

The company is expanding its leadership team with Eric Mosbrooker as Chief Commercial Officer, Charles Calderaro III as Chief Technical Officer, and Kat Lange as Chief Business Officer to support global commercialization efforts.

Key 2025 milestones include: BLA submission for del-zota by year-end, completion of enrollment in del-desiran HARBOR Phase 3 trial and del-brax biomarker cohort by mid-2025, and initiation of a global pivotal trial for del-brax. Marketing applications for del-desiran are planned for 2026 in the U.S. and European Union.