Neuromuscular Focused-Avidity Biosciences Has 'Complete Package' - Analyst Predicts Stellar 142% Stock Surge

Cantor Fitzgerald has initiated coverage on Avidity Biosciences Inc RNA, noting the company has the complete package, including a strong management team, a proven platform, a robust pipeline, and a blockbuster product in deldesiran (formerly AOC 1001) for Myotonic dystrophy type 1 (DM1).

The analyst reports that Avidity has accumulated extensive biomarker and clinical data, significantly reducing the risks associated with their upcoming Phase 3 study. 

Additionally, Dyne Therapeutics Inc DYN, a competitor trailing by approximately 12 months, is also generating similar data using a comparable approach, thereby offering further validation. 

Also Read: Avidity Biosciences Says Lead Program Shows Reversal Of Disease Progression In Neuromuscular Disease.

Despite the potential variability in biomarker data obtained from muscle biopsies, Avidity’s findings offer mechanistic support by demonstrating that del-desiran reduces DMPK RNA levels by 40-50% across various tested doses and has a dose-dependent effect on splicing mRNA.

Cantor analyst notes that Del-desiran focuses on addressing the core issue of disease progression, specifically targeting the toxic accumulation of the DMPK transcript. 

Phase 2 trials have demonstrated promising results, paving the way for a Phase 3 trial to commence in the second quarter of 2024. 

Based on these advancements, projections anticipate U.S. sales reaching $1.3 billion by 2031.

Cantor initiates with an Overweight rating and a price target of $60.

Avidity has two new clinical programs in Phase 1 development. One, AOC 1044, focuses on Duchenne Muscular Dystrophy that can be treated by skipping Exon 44, while the other, AOC 1020, targets facioscapulohumeral muscular dystrophy (FSHD).

Cantor notes that both programs utilize the same delivery mechanism as del-desiran, aim to tackle well-understood biological pathways, and hold the promise of becoming the first approved therapies for currently underserved patient groups.

Price Action: RNA shares are down 3.54% at $23.95 on the last check Thursday.

Illustration of Phrama lab worker created with MidJourney.