The therapy, AMO-02 (tideglusib), also improved patients’ autism symptoms. In addition, it was safe and patients tolerated it well.
Dr. Joseph Horrigan, the company’s chief medical officer, presented the findings at the Muscular Dystrophy Association Clinical Conference in Arlington, Va., March 11-14.
The trial (NCT02858908) involved 16 patients, aged 13 to 34 years, with congenital and juvenile-onset myotonic dystrophy type 1. Patients took daily doses of 400 mg or 1,000 mg of AMO-02 or a placebo.
Those on the 1,000-mg doses responded better than those on the 400-mg doses.
Both doctors and caregivers reported that the compound improved patients’ condition throughout the treatment period.
“These significant data are an important step in the development of AMO-02 as a potentially safe and effective treatment option for many patients living with congenital and childhood onset myotonic dystrophy type 1,” Michael Snape, the CEO of AMO Pharma, said in a press release.
“We look forward to advancing the clinical development program for AMO-02, and are grateful to the clinicians [doctors], caregivers and patients who participated in this landmark trial,” Snape added.
AMO is developing the treatment for a severe form of congenital myotonic dystrophy known as myotonic dystrophy type 1 (DM1) or Steinert disease. DM1 is an inherited type of muscular dystrophy that affects the muscles, heart, central nervous system, eyes, and endocrine system.
Signs of congenital myotonic dystrophy typically show up at birth. Diagnosis is done through medical evaluations or genetic tests.
There are no approved therapies that can address the cause of the disease, so patients are treated with medications that can improve their symptoms. Physical and speech therapy, as well as special education programs, are also commonly used.
Preclinical-trial studies have linked DM1 and Duchenne muscular dystrophy to higher than normal activity of an enzyme called glycogen synthase kinase 3 beta (GSK3beta). AMO-02 inhibits the enzyme, improving muscle characteristics in tissue samples from DM1 patients.
“As the first clinical trial devoted to this patient population, these results are especially encouraging,” Horrigan said. “We look forward to continuing to expand our research and further evaluate the efficacy of AMO-02 in larger multi-site clinical trials in the U.S., Canada and the U.K.”