Goldman Sachs has shown strong confidence in Avidity Biosciences, a clinical-stage biotechnology company specializing in RNA therapeutics for muscular dystrophies. The investment firm initiated coverage with a Buy rating and a $59 price target, citing the company’s potential to develop multi-blockbuster, first-to-market treatments.
Avidity’s lead asset, del-desiran (AOC 1001), is currently in a Phase 3 trial (HARBOR) for patients with myotonic dystrophy type 1 (DM1). The company’s pipeline also includes del-brax, in a Phase 1/2 trial (Fortitude) for facioscapulohumeral muscular dystrophy (FSHD), and del-zota, in a Phase 1/2 trial (Explore44) for Duchenne muscular dystrophy (DMD) patients amenable to exon 44 skipping.
Goldman Sachs’ analyst highlights the significant revenue potential of del-brax in FSHD, with an estimated peak sales of $2.7 billion. They view the clinical outlook and registration pathway for del-brax as positive, with a 60% probability of success. The analyst believes del-brax is well-positioned to be the first-to-market treatment for FSHD.
Del-desiran’s Phase 1/2 data has positioned Avidity to potentially be a first-to-market drug for myotonic dystrophy. The analyst projects unadjusted peak sales of $4 billion for del-desiran and expresses optimism about the HARBOR trial’s outcome, with a 75% probability of success.
Avidity Biosciences recently released data from their Phase 1/2 EXPLORE44 trial for Del-zota (AOC 1044) in DMD44 patients. This data showed a statistically significant increase of 25% in normal dystrophin production and 37% in exon 44 skipping. Additionally, Del-zota (at 5 mg/kg) reduced creatine kinase levels to near normal, representing a greater than 80% reduction from baseline.
This positive outlook from Goldman Sachs, coupled with promising clinical trial results, indicates that Avidity Biosciences is well-positioned to make a significant impact in the fight against muscular dystrophies. The company’s commitment to developing innovative RNA therapeutics offers hope for patients and their families seeking effective treatments for these debilitating diseases.
