Item 7.01. Regulation FD Disclosure.
On
The information contained in this Item 7.01, including in Exhibit 99.1 hereto, is being "furnished" and shall not be deemed "filed" for the purposes of Section 18 of the Securities Exchange Act of 1934, as amended, is not subject to the liabilities of that section and is not deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Securities Exchange Act of 1934, as amended, except as shall be expressly set forth by specific reference in such a filing.
Item 8.01. Other Events.
On
Interim data from the preliminary assessment demonstrated:
• Targeted delivery of siRNA to muscle, a tissue previously untreatable with existing RNA therapeutics; • Meaningful DMPK reduction in 100% of participants treated with AOC 1001; • Mean reduction of 45% in DMPK after only a single dose of 1 mg/kg or two doses of 2 mg/kg of AOC 1001; • Splicing improvement of 31% in a key set of muscle-specific genes and splicing improvement of 16% across a broad 22-gene panel in the 2 mg/kg cohort (excluding one splicing sample that will be evaluated in the next batch analysis). Splicing improvements demonstrated AOC 1001 activity in the nucleus; • Early signs of clinical activity with improvement in myotonia in some participants. Myotonia was measured by video hand opening time (vHOT) and is a hallmark of DM1 where relaxation of key muscle groups is impaired; and • Safety and tolerability data with majority of adverse events ("AEs") mild or moderate as of theNovember 17, 2022 data cutoff.
The most common treatment emergent AEs observed in the study through the
Serious adverse events ("SAEs") were observed in two participants. One participant in the 4 mg/kg cohort experienced a SAE that resulted in the previously disclosed partial clinical hold on new participant enrollment in the MARINA trial. One participant in the 2 mg/kg cohort experienced an SAE deemed unrelated to treatment by the investigator. The SAE was in reaction to opioid pain medication following an elective surgery.
In addition to these interim data from the preliminary assessment, Avidity announced that the Company anticipates sharing an update on the partial clinical hold, including more information about the drug-related SAE, by the end of the first quarter in 2023. No similar AEs have been observed in other participants in either the MARINA trial or the MARINA open label extension study ("MARINA-OLE"), and 100% of participants who have completed the MARINA trial have opted to roll over into MARINA-OLE.
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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 related to Avidity's ability to resolve the partial
clinical hold and resume enrollment in and complete the MARINA trial; Avidity's
plans to announce further details regarding the drug-related SAE and the timing
thereof; the continuation of participants in the MARINA trial and enrollment of
participants into the MARINA-OLE and the timing thereof; the potential of
Avidity's product candidates to treat rare diseases including DM1; and the
potential of AOCs to target a range of different cells and tissues beyond the
liver. 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 press release 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 serious adverse event may result in
delays in the MARINA study or an inability to compete the study; the Phase 1/2
MARINA trial results are based on a preliminary analysis of interim data
available as of the data cutoffs, and the interim results do not predict final
results of the trial, and one or more of the safety or biomarker results may
materially change following more comprehensive reviews of the data, as follow-up
on the outcome of any particular patient continues, as and if additional
patients enroll in the trial and as more patient data become available, any of
which may materially alter the findings and conclusions from Avidity's
preliminary analysis; 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
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits Exhibit Number Description 99.1 Slide Presentation 104 Cover Page Interactive Data File (embedded within the Inline XBRL document)
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