Goldman Sachs Bullish On Avidity Biosciences, Cites Scalable RNA Therapeutics Platform For Rare Diseases

Goldman Sachs has initiated coverage on Avidity Biosciences Inc 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.

Photo via Shutterstock

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