Avidity Biosciences Touts Encouraging Functional Gains And Biomarker Reductions In Rare Muscular Weakness Disease Trial

Avidity Biosciences, Inc. (NASDAQ:RNA) revealed topline data from the dose escalation cohorts of the delpacibart braxlosiran (del-brax) Phase 1/2 FORTITUDE program in Facioscapulohumeral Muscular Dystrophy (FSHD).

FSHD is a genetically acquired disease that leads to progressive muscle weakness and severely decreased functional capacity.

The data will be presented at the 32nd Annual FSHD Society International Research Congress.

Also Read: Avidity Biosciences Touts Positive Data From Early-Stage Duchenne Muscular Dystrophy Study Data, FDA Submission Expected By End Of 2025

Del-brax is the first investigational therapy designed to treat the underlying cause of FSHD by directly targeting the disease-causing gene, double homeobox 4 (DUX4).

FSHD affects approximately 45,000 to 87,000 people in the United States and Europe.

Topline data from these cohorts for del-brax treated participants, compared to placebo, demonstrated:

Consistent improvement of functional mobility and muscle strength as measured by 10-meter Walk-Run test (10MWRT), Timed Up and Go (TUG), and quantitative muscle testing (QMT) as compared to placebo;
Consistent improvement in multiple measures of quality of life as measured by patient-reported outcomes and compared to placebo;
Rapid and significant reductions in levels of KHDC1L and creatine kinase, a biomarker of muscle damage.
Favorable long-term safety and tolerability with most adverse events (AEs) mild or moderate, with no related serious or severe adverse events and no discontinuations.

Topline data from the ongoing, fully enrolled del-brax Phase 1/2 FORTITUDE biomarker cohort are anticipated in Q2 2026.

The primary endpoint of the FORTITUDE biomarker cohort is reduction of KHDC1L, a novel DUX4-regulated circulating biomarker.

Avidity also announced that the accelerated approval regulatory pathway in the U.S. is open for del-brax and that the company has initiated the global, confirmatory Phase 3 FORWARD study in FSHD.

In a recent research note, Chardan Capital analyst Keay Nakae set a 12-month price target of $75 per share for Avidity Biosciences. The valuation stems from a Net Present Value (NPV) model of the company’s projected revenues from its RNA therapeutics pipeline, forecasted out to the year 2034.

Chardan maintained its Buy rating and increased its price forecast from $65 to $75. According to Nakae’s analysis, the valuation incorporates specific “probability of success” rates for each of Avidity’s drug candidates to account for clinical and regulatory risks.

His model assigns a 60% chance of success for the company’s FSHD candidate, 50% for its DM1 and DMD exon 44 candidates, and lower probabilities for other assets. The analyst applied a 14% Weighted Average Cost of Capital (WACC) as the discount rate to reflect the high-risk profile of the development-stage company.

Nakae also highlighted several significant risks that could impede Avidity from reaching the $75 price target. He noted that the company, which currently has no revenue-generating products, will likely require additional financing, potentially leading to shareholder dilution.

The analyst also pointed to the risks of intellectual property challenges, the critical nature of upcoming clinical trial outcomes, and the intense competition from larger, better-resourced companies in the biotechnology sector.

Price Action: RNA stock is trading lower by 11.6% to $32.03 at last check Monday.

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