Item 8.01. Other Events.
On
The partial clinical hold is in response to a serious adverse event ("SAE") reported in a single participant in the 4mg/kg cohort of the MARINA study. Avidity is working closely with the FDA and the investigators to assess the cause of this SAE. The Company plans to take all necessary steps to resolve the partial clinical hold on new participant enrollment as quickly as possible. Avidity remains on track to conduct a preliminary assessment of safety, tolerability and key biomarkers in approximately half of the study participants in the MARINA trial in the fourth quarter of 2022.
Additionally, Avidity announced that the FDA has cleared the Company to proceed with clinical trials under two INDs. These clinical trials will evaluate AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy ("FSHD") and AOC 1044 for the treatment of Duchenne muscular dystrophy ("DMD") with mutations amenable to exon 44 skipping.
Forward-Looking Statements
Avidity cautions readers that statements contained in this report regarding
matters that are not historical facts are forward-looking statements. These
statements are based on the Company's current beliefs and expectations. Such
forward-looking statements include, but are not limited to, statements
regarding: expectations for Avidity's interactions with the FDA, the ongoing
investigation into the underlying cause of the SAE for the affected patient, and
the anticipated impact of, and Avidity's ability to resolve, the partial
clinical hold and resume enrollment in and complete the MARINA study, and to
conduct and present data from the preliminary assessment of the MARINA study and
the timing thereof; the progression of clinical programs for AOC 1001, AOC 1044
and AOC 1020 and timing thereof; and the potential of AOC 1001 to treat DM1. The
inclusion of forward-looking statements should not be regarded as a
representation by Avidity that any of these plans will be achieved. Actual
results may differ from those set forth in this report due to the risks and
uncertainties inherent in the business, including, without limitation: Avidity
may not be able to resolve the partial clinical hold and the analysis related to
the underlying cause of the SAE may result in delays in the MARINA study or an
inability to compete the study; unexpected adverse side effects or inadequate
efficacy of its product candidates that may delay or limit their development,
regulatory approval and/or commercialization, or may result in clinical holds,
recalls or product liability claims; Avidity is early in its development
efforts; Avidity's approach to the discovery and development of product
candidates based on its AOC platform is unproven, and the Company does not know
whether it will be able to develop any products of commercial value; potential
delays in the commencement, enrollment and completion of preclinical studies or
clinical trials; the success of its preclinical studies and clinical trials for
the company's product candidates; the results of preclinical studies and early
clinical trials are not necessarily predictive of future results; Avidity's
dependence on third parties in connection with preclinical and clinical testing
and product manufacturing; regulatory developments in
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