2024-03-05 13:27:40 ET
Summary
- Avidity Biosciences stock has gained more than 20% in the past five days due to positive long-term data for its lead candidate, AOC 1001, in the MARINA-OLE trial in DM1 patients.
- The company's market cap valuation is approximately $1.5 billion. Its IPO raised $259 million in June 2020 at $18 per share.
- Avidity has attracted major pharma companies for collaborations and licensing agreements, potentially worth up to $2.2 billion and $405 million in milestone payouts, respectively.
- Lead candidate AOC-1001 - known as del desiran - has been subject to a partial clinical hold, which was partially lifted last year, allowing the Marin study to progress.
- Management wants to move del desiran into a pivotal study this year, in DM1, targeting approval in 2026. The market opportunity, efficacy and safety of the drug needs more clarification to derisk this potentially promising investment opportunity.
Avidity Biosciences Overview
San Diego-based Avidity Biosciences ( RNA ) stock has gained >20% during the past five days, primarily due to the company's release of positive long-term data for its lead candidate, AOC 1001, otherwise known as delpacibart etedesiran, from its MARINA open-label extension ("MARINA-OLE") trial. According to a press release issued March 4th, the drug showed:
Reversal of disease progression in people living with myotonic dystrophy type 1 (DM1) across multiple endpoints including vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data.
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For further details see:
Avidity Biosciences: Targeting A First Ever Approval In DM1 With Improving Data