Avidity Biosciences, Inc. (Nasdaq: RNA), has announced new positive AOC 1001 data demonstrating improvement in multiple functional endpoints and favorable long-term safety and tolerability in people living with myotonic dystrophy type 1 (DM1). Avidity is a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™).
Preliminary data presented at the World Muscle Society conference last week “demonstrate improvement in additional functional measures including hand grip, muscle strength and patient reported outcomes, augmenting previously reported positive data showing improvements in myotonia, muscle strength and mobility,” states the company’s press release. In addition, “new long-term safety data of AOC 1001 continue to demonstrate favorable safety and tolerability with over 200 infusions totaling 46.2 patient-years of exposure.”
What does this mean for FSHD?
The company is developing a similar drug, AOC 1020, for FSH muscular dystrophy, which is currently in a Phase 1/2 trial called FORTITUDE. We therefore have an avid (so to speak) interest in any AOC 1001 updates. While the two drugs take aim at different genes, the “vehicle” that delivers these genes to muscles is the same. Thus, if the myotonic dystrophy trial reveals any adverse side effects, these might also occur in FSHD patients. On the positive side, if AOC 1001 is improving symptoms for DM1 patients, this suggests that the vehicle is effective in delivering therapeutic levels of RNA to muscles.
In sum, Avidity’s announcement is encouraging news for the FSHD community. The latest data indicate that the company’s drug for myotonic dystrophy may be providing benefits to patients. The news gives us hope that AOC1020 will similarly be found to be well-tolerated and effective at delivering its anti-DUX4 drug to the muscles.