Rare Daily Staff
GT Biopharma said it has initiated the proof of concept trial for GTP-004, a novel treatment for the symptoms of myasthenia gravis, a rare autoimmune disease caused by antibodies that attack muscles causing weakness and fatigue.
Acetylcholinesterase inhibitors, such as Mestinon, are used to treat muscle weakness in patients with myasthenia gravis. However, these drugs can cause gastrointestinal side effects including diarrhea, nausea, vomiting. Those side affect the dose of the drug that can be used and can also cause patients to fail to adhere to their medication requirements or discontinue use.
GTP-004 is a fixed dose combination Mestinon with a second drug that prevents diarrhea, nausea and vomiting should lead to greater patient comfort, safety, and compliance as well as to improved efficacy.
The goal of the early-stage study is to allow for the fully efficacious dose of pyridostigmine to be safely used while reducing side effects.
December 6, 2017