In early January 2021, biopharmaceutical company Avidity Biosciences (“Avidity”) announced updates from its 2021 pipeline. Currently, Avidity is working to create a line of Antibody Oligonucleotide Conjugates (AOCs), therapeutic options for a wide range of conditions. Included in the pipeline are drug updates for patients with myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), muscular atrophy (MA), and Duchenne muscular dystrophy (DMD).
Information included in the updates highlights:
- A Phase 1/2 clinical trial which will evaluate AOC 1001 in adult patients with DM1. Avidity hopes to be able to launch this study within the 2nd quarter of 2021.
- Avidity is going to expand three programs evaluating potential treatments for patients with DMD. Specifically, the programs will target gene mutations associated with this condition. The leading therapeutic focuses in on Exon 44.
- Currently, Avidity is running one program to develop a therapy for patients with FSHD. By the end of 2021, Avidity hopes to begin studies to evaluate potential treatment options. However, clinical trials will most likely not be held until 2022-2023.
- Over the next year, Avidity will work to develop a therapeutic program for MA to determine potential drug development options.
- Currently, Avidity and Myokardia partnered to research the use of AOCs, in conjunction with MyoKardia’s proprietary cardiomyopathy technology, in patients with cardiac conditions.
Duchenne Muscular Dystrophy (DMD)
Gene mutations prevent patients with Duchenne muscular dystrophy (DMD) from producing enough dystrophin. Without these proteins, muscles become weak and fragile. DMD is characterized by progressive muscle weakness which can cause heart and respiratory issues. DMD is much more common in males, present in 1 in 3500 male births and only 1 in 50 million female births. Symptoms usually appear before 6 years old. These include:
- Difficulty walking or moving positions
- Frequent falls
- Muscle weakness beginning in the lower extremities
- Enlarged calves
- Foot drop
- Developmental and intellectual delays
- Delayed growth
- Waddling gait
- Heart and respiratory damage
Typically, myotonic dystrophy, a rare disease characterized by progressive muscle wasting, is caused by specific gene mutations. Patients with myotonic dystrophy type 1 (DM1) have a DMPK mutation, and their condition is usually more severe. DM1 usually affects the lower extremities, hands, neck, and face. Alternately, ZNF9 gene mutations cause myotonic dystrophy type 2 (DM2), which often affects the hips, shoulders, elbows, and neck. Myotonic dystrophy is one of the most common forms of muscular dystrophy in which symptoms begin in adulthood. Some symptoms include:
- Difficulty swallowing
- Muscle weakness
- Difficulty breathing
- Prolonged muscle contractions with the inability to relax muscles
- Pregnancy and labor-related complications
- Insulin resistance
- Abnormal heartbeat
- Learning delays
Learn more about myotonic dystrophy.