Phase 1 Trial of GTP-004 Initiated as Therapy for Myasthenia Gravis Treatment Side Effects

GT Biopharma has launched a proof-of-concept Phase 1 trial of its investigational therapy GTP-004 for the treatment of myasthenia gravis. The trial’s primary goal is to demonstrate the therapy improves the gastrointestinal side effects of current treatments.

“I am very pleased that GT Biopharma’s first clinical trial in patients with myasthenia gravis has started and I have high hopes that the GTP-004 drug will bring substantial help to patients. The start of this trial underscores our commitment to patients with neurological disease,” Dr. Kathleen Clarence-Smith, CEO of GT Biopharma, said in a press release.

Currently only two medicines – Mestinon (pyridostigmine) and Prostigmin (neostigmine) – are approved to treat the muscle weakness and fatigue symptoms in patients with myasthenia gravis. Both drugs belong to the class of acetylcholinesterase inhibitors, meaning they work by blocking an enzyme called acetylcholine esterase.

While acetylcholinesterase inhibitors have been shown to be effective in halting muscle weakness, they also act on the gut, causing gastrointestinal side effects: diarrhea, nausea, and vomiting. These side effects are a considerable source of discomfort to patients and are dose-dependent, which means that often the therapeutic dose needs a decrease. As a consequence, effectiveness may be reduced.

GTP-004 is a fixed-dose combination tablet of two approved therapies – Mestinon (pyridostigmine) and an antagonist to Mestinon’s gastrointestinal side effects.

Blocking the adverse effects of Mestinon will improve patients’ comfort and safety and enhance their compliance to follow the treatment. Also, mitigating the side effects ensures drug efficacy at its full potential, since it wouldn’t be necessary to decrease the drug’s dose.

“We continue to move our product portfolio forward with major milestones. The start of our clinical trial of GTP-004 (for myasthenia gravis) represents another biotech asset that we believe could bring significant value to our shareholders,” said Anthony J. Cataldo, executive chairman of GT Biopharma.

“This represents not only another significant step for GT Biopharma, but also for the thousands of patients that suffer with this often debilitating disease,” said Dr. Raymond Urbanski,the company’s chief marketing officer.

GT Biopharma is a clinical-stage biopharma that developes novel immunotherapies for cancers and diseases affecting the central nervous system.