GTP-004 is a fixed dose combination tablet for the treatment of the muscle weakness associated with myasthenia gravis. GTP-004 combines pyridostigmine (Mestinon®) with an antagonist of the GI side effects of pyridostigmine.

Myasthenia gravis is a chronic autoimmune disease of the neuromuscular junction characterized by muscle weakness. The disease occurs in all ethnic groups and both genders. The prevalence of the disease in the United States is estimated at 14 to 20 per 100,000 population, approximately 36,000 to 60,000 cases in the US (Howard, 2015). Myasthenia gravis most commonly affects adult women (under 40) and older men (over 60), but it can occur at any age (Myasthenia Gravis Fact Sheet; National Institute of Neurological Disorders and Stroke, 2016).

Acetylcholinesterase inhibitors (AChEIs) that do not get into the brain (do not cross the blood-brain barrier), such as Mestinon®, are used to treat the muscular weakness associated with myasthenia gravis. However, AChEIs also act at cholinergic synapses in the gut to cause gastrointestinal side effects such as diarrhea, nausea, vomiting. that are dose-limiting. The GI side effects associated with Mestinonâ are an important source of discomfort for the patient, may be the source of non-compliance, or may result in the need to decrease the dose of Mestinonâ to mitigate these side effects when these become dose-limiting. As a consequence, efficacy may be reduced. Mitigating the GI side effects of Mestinonâ with a drug that prevents diarrhea, nausea and vomiting should lead to greater patient comfort, safety, and compliance as well as to improved efficacy.

Since GTP-004 is a combination tablet of 2 approved drugs, Management anticipates that the NDA will be a 505(B)2 NDA.

Provisional patent applications protecting the combination of Mestinonâ with a number of antiemetic drugs were filed by GT Pharmaceutcals in early 2017.