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Avidity Biosciences (RNA) announced positive topline data from Phase 1/2 FORTITUDE trial for delpacibart braxlosiran (del-brax) in treating Facioscapulohumeral Muscular Dystrophy (FSHD). Del-brax, the first therapy targeting the disease-causing DUX4 gene, showed consistent improvements in functional mobility, muscle strength, and quality of life compared to placebo. The trial evaluated 39 participants across two dose cohorts (2mg/kg and 4mg/kg) over 12 months. Key findings include improved 10-Meter Walk-Run test, Timed Up and Go, and quantitative muscle testing scores, along with significant reductions in KHDC1L and creatine kinase biomarkers. The company plans to submit a BLA for accelerated approval in H2 2026 and has initiated the Phase 3 FORWARD study. FSHD affects 45,000-87,000 people in the US and Europe, with no currently approved treatments.

Avidity Biosciences (RNA) announced that del-brax, its investigational therapy for facioscapulohumeral muscular dystrophy (FSHD), has received FDA clearance for the accelerated approval pathway. The company plans to submit a BLA in H2 2026 following topline data from the FORTITUDE biomarker cohort. Additionally, Avidity has initiated FORWARD, a global Phase 3 confirmatory study of del-brax 2mg/kg administered every six weeks in approximately 200 FSHD patients. Del-brax is the first therapy targeting DUX4, the disease-causing gene in FSHD, which affects 45,000-87,000 people in the US and Europe. The FORWARD study will evaluate functional mobility, strength, and patient-reported outcomes over 18 months. Currently, there are no approved treatments for FSHD, a rare hereditary disorder characterized by progressive muscle weakness and disability.

Avidity Biosciences (RNA) has granted equity-based inducement awards to 16 new non-executive employees under its 2022 Employment Inducement Incentive Award Plan. The awards include 71,000 non-qualified stock options with an exercise price of $31.64 per share and 35,000 restricted stock units (RSUs).

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. All awards are subject to continued employment and were granted in accordance with Nasdaq Listing Rule 5635(c)(4) as inducements for joining the company.

Avidity Biosciences (RNA) reported Q1 2025 financial results and progress across its clinical programs. The company maintains a strong financial position with $1.4 billion in cash, providing runway into mid-2027. Key highlights include positive topline del-zota data for DMD44 treatment, showing significant improvements in dystrophin production and creatine kinase levels. Avidity plans its first BLA submission by end of 2025. The company expects multiple Q2 updates for del-brax, including potential accelerated approval path and Phase 3 trial initiation. Financial results show collaboration revenues of $1.6 million (down from $3.5M in Q1 2024), R&D expenses of $99.5 million (up from $66.8M), and G&A expenses of $33.6 million (up from $13.9M). Avidity is preparing for potential commercial launches in 2026 while advancing three late-stage clinical programs.

Avidity Biosciences (Nasdaq: RNA), a biopharmaceutical company focused on developing Antibody Oligonucleotide Conjugates (AOCs™), has announced its participation in the upcoming Bank of America Securities 2025 Health Care Conference. The company's management will engage in a fireside chat scheduled for Tuesday, May 13, 2025, at 3:00 p.m. PT / 6:00 p.m. ET. Investors can access the live webcast and replay of the presentation through Avidity's investor relations website.

Avidity Biosciences (Nasdaq: RNA) has announced new equity grants under its 2022 Employment Inducement Incentive Award Plan. The company granted 263,000 non-qualified stock options and 131,450 restricted stock units (RSUs) to 33 new non-executive employees as employment inducements, complying with Nasdaq Listing Rule 5635(c)(4).

The stock options have an exercise price of $26.66 per share, matching Avidity's closing price on Nasdaq Global Market on April 17, 2025. The options will vest over four years, with 25% vesting after one year and the remainder monthly over 36 months. The RSUs will vest in four equal annual installments. Both grants require continued employment for vesting.

Avidity Biosciences (RNA) has achieved a significant milestone as its drug delpacibart etedesiran (del-desiran) received Orphan Drug designation in Japan for treating myotonic dystrophy type 1 (DM1). This marks the first DM1 treatment to receive this designation in Japan.

Del-desiran, designed to target the root cause of DM1 (a progressive and often fatal neuromuscular disease), has already secured multiple regulatory designations including Breakthrough Therapy, Orphan Drug, and Fast Track from the FDA, along with Orphan designation from the EMA.

The company reports promising results from the MARINA and MARINA-OLE studies, showing favorable long-term safety, disease progression reversal, and sustained clinical improvements. Avidity expects to complete enrollment in the Phase 3 HARBOR trial by mid-2025, with marketing applications planned for submission starting 2026 in the U.S., EU, and Japan.

Avidity Biosciences (Nasdaq: RNA), a developer of Antibody Oligonucleotide Conjugates (AOCs™), has announced its participation in the upcoming 24th Annual Needham Virtual Healthcare Conference. The company's management will engage in a fireside chat scheduled for Tuesday, April 8, 2025, at 3:00 p.m. ET.

Investors and interested parties can access the live webcast and replay of the presentation through the 'Events and Presentations' section on Avidity's investor relations website at aviditybiosciences.investorroom.com/events-and-presentations.

Avidity Biosciences (Nasdaq: RNA) has completed enrollment of 51 participants in the biomarker cohort of its Phase 1/2 FORTITUDE™ trial for delpacibart braxlosiran (del-brax), targeting facioscapulohumeral muscular dystrophy (FSHD). The company aims to be first to market with an approved FSHD treatment.

Del-brax, at 2 mg/kg dosage, has shown promising results including:

• Unprecedented reductions in DUX4-regulated genes
• Significant decreases in novel circulating biomarker and creatine kinase
• Trends of functional improvement
• Favorable safety and tolerability

Key Q2 2025 milestones include:

• Updates on potential U.S. accelerated approval pathway
• Global Phase 3 trial design alignment and initiation
• Topline data from FORTITUDE dose escalation cohorts