Analyst Conference Summary

biotechnology

conference date: November 8, 2022 (press release only)
for quarter ending: September 30, 2022 (third quarter, Q3 2022)

Forward-looking statements

Overview: First three clinical trials are enrolling.

Basic data (GAAP):

Revenue was $2.5 million, up sequentially from $2 million, and up from $2 million year-earlier. All revenue was from collaborations.

Net loss was $44 million, up sequentially from loss of $46 million, and down from loss of $29 million year-earlier.

EPS loss $0.82, up sequentially from loss of $0.92, and down from loss of $0.68 year-earlier.

Guidance:

Cash runway through 2024

Conference Highlights:

Sarah Boyce, CEO said "Consistent with our guidance for 2022, we have progressed three programs addressing three distinct rare diseases into clinical development, and we remain on track for our preliminary assessment of the MARINA trial this quarter. Our team continues to execute on our plan as we advance AOC 1020 for FSHD in the FORTITUDE trial and AOC 1044 for DMD in the EXPLORE44 trial. With close to 40 participants enrolled in the MARINA trial, we continue to gather data on AOC 1001 as we work to resolve the recent partial clinical hold on new participant enrollment as swiftly as possible." AOCs are Antibody Oligonucleotide Conjugates, a method of delivering RNA to target cells.

In Q3 2022 raised $18.7 million cash through an at the market program for stock sales (WM: and dilution). Also raised more cash in Q4.

In Q3 2021 initiated a Phase 1/2 trial for AOC 1001 for DM1 (myotonic dystrophy type 1). It is in a collaboration with Myotonic Dystrophy Clinical Research Network supporting END-DM1, a natural history study to advance the understanding of disease progression in patients with DM1. The FDA placed a partial clinical hold in response to one serius adverse event. Current participants continue dosing. Also added a Phase 2 open label extension study of patients who completed the orignial trial.

AOC 1044 for DMD (Duchenne Muscular Dystrophy) began enrollment in Q3. The EXPLORE44 trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial to evaluate AOC 1044 in approximately 40 healthy volunteers and 24 participants with DMD44, ages seven to 27 years old.

AOC 1020 for the FSHD (facioscapulohumeral muscular dystrophy) Phase 1 trial began in Q3 2022. The FORTITUDE trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial designed to evaluate AOC 1020 in 68 adult participants with FSHD. Avidity entered a collaboration with the FSHD Clinical Trial Research Network (FSHD CTRN) to support a natural history study called the Motor Outcomes to Validate Evaluations Plus (MOVE+) Study to enhance the understanding of how to utilize whole-body MRI and other tools to identify specific biomarkers for FSHD that can potentially accelerate and support future clinical trial design. Avidity presented data supporting directly targeting DUX4 with AOC 1020 at the 29th Annual FSHD Society International Research Congress. Data from a FSHD mouse model showed that a single intravenous dose with the murine version of AOC 1020 prevented development of muscle weakness demonstrated by three functional assays: treadmill running, in vivo force, and compound muscle action potential.

Cash and equivalents ended at $406 million, down sequentially from $398 million. No debt.

Operating expense of $47 million consisted of $37 million for R&D and $10 million for G&A. Other income $1 million.

Q&A:

none

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