Avidity Biosciences, Inc. ( RNA Quick Quote RNA - Free Report) provided an update on its phase I/II study of AOC 1001 in adults with myotonic dystrophy type I (DM1). The company reported that it is engaged in ongoing discussions with the FDA regarding the partial clinical hold on new participant enrollment in the MARINA study. The company continues to share new AOC 1001 data with the regulatory body as it emerges from the MARINA study. However, the stock of the company dropped about 17% on Thursday, following the company’s decision to conclude the MARINA study with the 38 participants enrolled at 1 mg/kg, 2 mg/kg and 4 mg/kg of AOC 1001 and will not move forward with the 8 mg/kg dose of AOC 1001.
AOC 1001, Avidity’s lead product candidate, is part of the company’s new class of RNA therapy candidates called Antibody Oligonucleotide Conjugates (AOC). The mechanism of action of AOC 1001 includes a proprietary monoclonal antibody that binds to the transferrin receptor 1 (TfR1) conjugated with a siRNA targeting DMPK mRNA.
In the past year, shares of Avidity have declined 16% compared with the
industry’s 14.3% fall. Image Source: Zacks Investment Research
DM1 is a rare, progressive, neuromuscular disease with an extremely high mortality rate. DM1 primarily affects skeletal and cardiac muscles. However, it can cause a plethora of complexities, like myotonia and muscle weakness, respiratory problems, fatigue, hypersomnia, cardiac abnormalities, severe gastrointestinal complications and cognitive and behavioral impairment.
We would like to remind the investors that in September 2022, a partial clinical hold was issued by the FDA on new participant enrollment in Avidity’s phase I/II MARINA study. This partial hold was the consequence of a serious complexity reported in a single participant in the 4 mg/kg cohort of the MARINA study. The study investigator then concluded that the adverse event was related to the AOC 1001. However, post thorough analysis, Avidity reported that the unfortunate occurrence was a result of an unforeseen rare complication without a plausible biological link to any component of AOC 1001, the AOC platform, the transferrin receptor delivery mechanism or the reduction of DMPK. The company emphasized the fact that AOC 1001 does not cross the blood-brain barrier.
In December 2022, the company reported positive data from a preliminary assessment of the phase I/II MARINA study of AOC 1001. Data showed that the mechanism of action was successfully carried out with early signs of clinical activity with improvement in myotonia after just one or two doses of the candidate. Data from the preliminary assessment reportedly supports that the targeted dose range is between 2 mg/kg and 4 mg/kg of AOC 1001.
Following these findings, Avidity also announced its decision to continue dosing in both the patient cohorts, receiving 2 mg/kg and 4 mg/kg of AOC 1001 in the MARINA-OLE study. The study will evaluate the long-term safety and tolerability of AOC 1001 in participants with DM1 who were previously enrolled in the MARINA phase I/II study. The company expects to share top-line data from the study toward the end of 2023.
Management is focused on resolving the partial clinical hold and remains confident about AOC 1001’s benefit/risk profile.
Zacks Rank and Stocks to Consider
Avidity currently has a Zacks Rank #3 (Hold).
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