Avidity Biosciences granted FDA Fast Track Designation for Duchenne Muscular Dystrophy treatment
AOC 1044 is being developed for the treatment of Duchenne muscular dystrophy (DMD) in people with mutations amenable to exon 44 skipping (DMD44). The candidate is being assessed in the Phase 1/2 EXPLORE44 clinical trial for people living with DMD44 and is the first of multiple AOCs the company is developing for DMD. The first data from the trial are expected later this year.
Fast Track designation enables more frequent interactions with the FDA to expedite the development and review process for drugs intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs (While there are approved treatments for DMD, there are no therapies approved targeting exon 44).
3 programs with Fast Track status
DMD is a rare genetic condition that is characterized by progressive muscle damage and weakness due to the loss of dystrophin protein that typically starts in males at a very young age.
Those living with the condition often require special aid and assistance throughout their lives and have significantly shortened life expectancy.
DMD is a monogenic, X-linked, recessive disease that primarily affects males, with 1 in 3500 to 5000 boys born worldwide having Duchenne.
Avidity's proprietary AOCs are designed to combine the specificity of monoclonal antibodies (mAbs) with the precision of oligonucleotide therapies to target the root causes of diseases previously untreatable with RNA therapeutics.
In the case of DMD, the disease is caused by a genetic mutation that prevents the body from producing the dystrophin protein, which protects muscle cells from injury during contraction. The lack of functional dystrophin leads to stress and tears of muscle cell membranes, resulting in muscle cell death and progressive loss of muscle function. AOC 1044 is designed to deliver phosphorodiamidate morpholino oligomers (PMOs) to skeletal muscle and heart tissue. The PMOs circumvent the mutation by causing exon 44 of the dystrophin gene to be skipped, which enables production of functional dystrophin protein.
"It is very encouraging to receive FDA Fast Track designation as it further validates the potential of AOC 1044 to target the underlying cause of DMD44 and the importance of bringing people living with this devastating disease an effective treatment option,” said Steve Hughes, M.D., chief medical officer at Avidity.
“This recognition also means that now all three of our clinical-stage programs have Fast Track status, further reinforcing our efforts to make a profound difference in people's lives.
"We will continue to work closely with the FDA as we advance AOC 1044 and look forward to the anticipated data readout from the healthy volunteer portion of our Phase 1/2 EXPLORE44 clinical trial later this year."
Headquartered in San Francisco, Avidity has three programs in clinical development: all currently in Phase 1/2 trials.
AOC 1001 is designed to treat people with myotonic dystrophy type 1 (DM1); while AOC 1020 is designed to treat people living with facioscapulohumeral muscular dystrophy (FSHD).
AOC 1044 is the first of multiple AOCs the company is developing for DMD.
