Analyst Conference Summary

biotechnology

conference date: February 28, 2023 (press release only)
for quarter ending: December 31, 2022 (fourth quarter, Q4 2022)

Forward-looking statements

Overview: First three clinical trials are enrolling.

Basic data (GAAP):

Revenue was $2.8 million, up sequentially from $2.5 million, and up from $1.9 million year-earlier. All revenue was from collaborations.

Net loss was $50 million, down sequentially from loss of $44 million, and down from loss of $39 million year-earlier.

EPS loss $0.88, down sequentially from loss of $0.82, and down from loss of $0.82 year-earlier.

Guidance:

Cash runway through mid 2025

Conference Highlights:

Sarah Boyce, CEO said "In 2022, we executed on our goal of advancing three rare disease programs into clinical development and demonstrated the first-ever successful targeted delivery of RNA into muscle, a revolutionary advancement for the field of RNA therapeutics. Building on our AOC proof of platform data, 2023 will be another important year for Avidity. We look forward to Phase 1/2 MARINA and MARINA-OLE data as well as results from healthy volunteers in the Phase 1/2 EXPLORE44 trial while continuing to expand the broad utility of our AOC platform." 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.

AOC 1001 for DM1 (myotonic dystrophy type 1) reported positive interim results. AOC 1011 demonstrated first-ever successful targeted delivery of RNA to skeletal muscle, DMPK reduction, splicing improvements and early signs of clinical activity with improvement in myotonia in some participants. Expects to report top-line data in 2023 despite the partial clinical hold. 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. Added a Phase 2 open label extension study of patients who completed the orignial trial, with preliminary data due in late 2023.

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. Data from healthy volunteers is expected in 2H 2023.

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. Data from healthy volunteers is expected in 1H 2024. Granted Fast Track status by the FDA in January 2023 and Orphan status in the EU in February 2023.

Cash and equivalents ended at $611 million, up sequentially from $406 million, reflecting $223.8 million follow-up financing in the quarter. No debt.

Operating expense of $56 million consisted of $46 million for R&D and $10 million for G&A. Other income $3 million. Owed $47 million in accounts payable.

Q&A:

none

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