GT Biopharma ends dosing in Phase l trial of GTP-004

11th January 2018 (Last Updated January 11th, 2018 00:00)

GT Biopharma has completed dosing patients in a Phase l trial of GTP-004 for the treatment of myasthenia gravis symptoms.

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.

"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."

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.