Avidity Biosciences Says Lead Program Shows Reversal Of Disease Progression In Neuromuscular Disease

Monday, Avidity Biosciences Inc RNA revealed new long-term AOC 1001 data from the MARINA open-label extension (MARINA-OLE) trial showing reversal of disease progression in myotonic dystrophy type 1 (DM1) patients across multiple endpoints compared to END-DM1 natural history data. 

These are the same key endpoints used in the global Phase 3 HARBOR trial for DM1. 

Also Read: Avidity Biosciences’ Muscle Disease Candidate Shows Improvement in Multiple Additional Functional Endpoints, Safety.

The primary endpoint in the Phase 3 HARBOR trial is video hand opening time (vHOT), and key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score and activities of daily living. 

Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024.

Avidity also announced delpacibart etedesiran as the approved international nonproprietary name of AOC 1001, abbreviated as del-desiran.

In the MARINA-OLE study, del-desiran (AOC 1001) 4 mg/kg provided consistent and durable improvements in the following:

• Myotonia (video hand opening time, or vHOT), hand grip, Quantitative Muscle Testing (QMT) total score includes hand grip, elbow extension and elbow flexion, knee extension and knee flexion, and ankle dorsiflexion.
• DM1-Activ is a patient-reported outcome (PRO) that measures activities of daily living (e.g., taking a shower, visiting family or friends, and walking upstairs).

With over 265 infusions totaling 61.1 patient-years of exposure, del-desiran (AOC 1001) continues demonstrating favorable safety and tolerability. 

Price Action: RNA shares are down 9.75% at $18.15 on the last check Monday.

Photo by Darko Stojanovic from Pixabay