Prior to launching a clinical trial, the FDA must clear an Investigational New Drug (IND) application. This allows the drug to be distributed across state lines, which is important as clinical studies often have multiple sites. According to Muscular Dystrophy News, the FDA recently cleared the IND for Benitec Biopharma (“Benitec”). This will allow the company to begin a Phase 1b/2a clinical trial evaluating BB-301 for oculopharyngeal muscular dystrophy (OPMD). 

BB-301 is an investigational gene therapy that silences and replaces the mutated gene associated with OPMD using RNAi technology. The treatment has already received Orphan Drug designation, which is granted to drugs or biologics intended to treat conditions affecting fewer than 200,000 people nationwide. So far, preclinical studies of BB-301 found that the therapy could be delivered intramuscularly, improved muscle strength and healthy protein, and reduced the levels of mutant protein caused by the gene mutations.

Now that the FDA has cleared Benitec’s IND, the company can begin the clinical study. Currently, the plan is to launch the study later this year. Participants in an ongoing natural history study can come over to the clinical trial upon completion. Researchers will evaluate the drug’s safety, efficacy, tolerability, and ability to improve swallowing. 

About Oculopharyngeal Muscular Dystrophy (OPMD)

Oculopharyngeal muscular dystrophy is a rare genetic disorder caused by PABPN1 mutations that often manifests between ages 40 to 60. It is one of the nine forms of muscular dystrophy. This slowly progressing myopathy (muscle disease) causes ocular and pharyngeal (eye and throat) muscle weakness. The gene mutations cause the production of abnormal protein which clumps in muscles and causes issues. Symptoms associated with OPMD can include:

• Difficulty swallowing (dysphagia)
• Drooping eyelids (ptosis)
• Tongue weakness
• Muscle weakness of the lower extremities
• A “breathy” voice
• Double vision
• Difficulty speaking
• Limited upward gaze

Treatment includes orthopedic interventions, blepharoplasties, and cricopharyngeal myotomies.