Goldman Sachs has initiated coverage on Avidity Biosciences Inc (NASDAQ:RNA) based on multi-blockbuster, first-to-market commercial opportunities for its investigational candidates.
Avidity Biosciences is a clinical-stage biotechnology company that develops RNA therapeutics (antibody oligonucleotide conjugates) for muscular dystrophies.
The company's lead asset, del-desiran (AOC 1001), is currently in the Phase 3 HARBOR trial in patients with myotonic dystrophy type 1 (DM1).
The company's pipeline includes Del-brax in the Phase 1/2 Fortitude trial for facioscapulohumeral muscular dystrophy (FSHD) and del-zota in a Phase 1/2 Explore44 trial in Duchenne muscular dystrophy (DMD) for patients amenable to exon 44 skipping.
Goldman Sachs has initiated with a Buy rating and a price target of $59.
The analyst sees a blockbuster commercial opportunity for del-brax in FSHD and a positive view of the clinical outlook and registrational path, with a 60% probability of success.
Del-brax is well positioned to be first-to-market, and the analyst highlights significant revenue opportunity, with an estimated peak sales of $2.7 billion.
Goldman Sachs analyst also says Del-desiran's Phase 1/2 data positions the company to potentially be a first-to-market drug in myotonic dystrophy. The analyst projects unadjusted peak sales of $4 billion and is optimistic about the trial's outcome, with a probability of success of 75%.
In August, Avidity Biosciences released Del-zota (AOC 1044) 5 mg/kg data that demonstrated a statistically significant increase of 25% of normal dystrophin production and 37% in exon 44 skipping in people living with Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44) in the Phase 1/2 EXPLORE44 trial.
In addition, AOC 1044 5 mg/kg reduced creatine kinase levels to near normal with greater than 80% reduction compared to baseline.
Price Action: RNA stock is down 0.07% at $43.47 at last check Tuesday.
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