GT Biopharma has completed dosing patients in a Phase l trial of GTP-004 for the treatment of myasthenia gravis symptoms.
The trial is designed to demonstrate that gastro-intestinal (GI) side-effects can be safely reduced with GTP-004.
It has enrolled five healthy volunteers, who have received single increasing oral doses of pyridostigmine ranging from 30mg to 120mg administered once-daily in the morning.
When the enrolled subjects reached first intolerable dose (FID1) as determined by protocol criteria, upward dose escalation of pyridostigmine was stopped and the subjects were washed-out for two to seven days.
Subjects who reached FID level were administered with daily increasing doses of pyridostigmine in combination with ondansetron.
The results showed that GTP-004 allows the safe and well-tolerated administration of doses of pyridostigmine.
In addition, three subjects in two males and one female reached FID with pyridostigmine alone.
GT Biopharma CEO Dr Kathleen Clarence-Smith said: “These are early results, but the data provide evidence of the ability of GTP-004 to avoid the GI side-effects of administering pyridostigmine alone and offer hope to all those suffering from myasthenic syndromes.
“We expect to be in a position to begin a Phase ll clinical trial in patients in the second half of 2018.”
GT Biopharma further noted that its GTP-004 solution combines pyridostigmine with ondansetron.
It intends to lessen the GI side-effects of pyridostigmine alone, providing the potential for a fully efficacious dose of pyridostigmine to be safely used.