Item 8.01 Other Events.
On
2021 Pipeline Goals
• In the second half of 2021, the Company plans to initiate a Phase 1/2 clinical study of AOC 1001 in adults with myotonic dystrophy type 1 ("DM1"). • AOC FSHD is a therapeutic program in development for the treatment of facioscapulohumeral muscular dystrophy ("FSHD"). In 2021, the Company plans to advance the program into IND-enabling studies. In 2022, the Company plans to submit a regulatory filing to support a clinical trial. • AOC DMD is a therapeutic program in development for the treatment of Duchenne muscular dystrophy ("DMD"). The Company has expanded its efforts for this indication and is now advancing three programs for DMD which target different mutations that are amenable to skipping, including Exon 44, Exon 51 and Exon 45. The Company's lead AOC in development for DMD targets Exon 44. In 2022, Avidity plans to submit a regulatory filing to support a clinical trial. • AOC Muscle Atrophy is a therapeutic program in development for the treatment of muscle atrophy ("MA"). During 2021, Company researchers will evaluate AOCs in multiple disease models of MA to identify an optimal development path.
Research Collaboration with MyoKardia, a Wholly-Owned Subsidiary of Bristol Myers Squibb
• The Company has entered into a research collaboration with MyoKardia, a wholly-owned subsidiary of Bristol Myers Squibb, to demonstrate the potential utility of AOCs in cardiac tissue by leveraging MyoKardia's genetic cardiomyopathy platform including, among other aspects, its novel target discovery engine and proprietary cardiac disease models.
Forward-Looking Statements
The Company 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: the potential to develop a meaningful pipeline of novel AOC
therapeutics; the initiation of a Phase 1/2 clinical trial of AOC 1001 in
patients with DM1 and the timing thereof; the Company's plans to advance the AOC
FSHD and AOC DMD programs and submit regulatory filings to support clinical
trials and the expected timing thereof; the advancement of three programs for
DMD and potential development for AOC MA; the potential to identify new targets
beyond the muscle that can be targeted with the Company's AOC approach,
including cardiac tissue under the MyoKardia collaboration; the future expansion
of the Company's AOC platform; and the broad potential of AOCs to treat serious
diseases. The inclusion of forward-looking statements should not be regarded as
a representation by the Company that any of its plans will be achieved. Actual
results may differ from those set forth in this press release due to the risks
and uncertainties inherent in the Company's business, including, without
limitation: the Company is early in its development efforts and all of its
development programs are in the preclinical or discovery stage; the Company'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 clinical trials; disruption to the Company's
operations from the COVID-19 pandemic; the success of the Company's preclinical
studies and clinical trials for its product candidates; the results of
preclinical studies and early clinical trials are not necessarily predictive of
future results; the Company may not be able to show utility of its AOCs in
cardiac or other tissue and it may not realize any benefits from the MyoKardia
collaboration; the Company's dependence on third parties in connection with
preclinical testing and product manufacturing; unexpected adverse side effects
or inadequate efficacy of the Company's product candidates that may limit their
development, regulatory approval and/or commercialization, or may result in
recalls or product liability claims; regulatory developments in
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