Pivotal Trial Begins for AMO-02 in Steinert Disease

AMO Pharma has announced the initiation of REACH-CDM, a pivotal Phase 2/3 clinical trial that will assess the efficacy and safety of the investigational medication AMO-02 (tideglusib) in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), also known as Steinert disease.

The double-blind trial (NCT03692312) is intended to support future submissions for marketing authorization, according to AMO Pharma, which is sponsoring the study.

The trial intends to enroll 56 children and adolescents with CDM1. Recruitment initially will take place at sites in the U.S. and Canada; additional sites in Australia, New Zealand and other countries will be added pending local approvals. More information about locations is available here.

“We are extremely excited to start enrolling patients for REACH-CDM, a pivotal therapeutic trial in the space of congenital myotonic dystrophy,” Aravindhan Veerapandiyan, MD, principal investigator of REACH-CDM at Arkansas Children’s Hospital, said in a press release.

The initiation of REACH-CDM follows the completion of discussions with the U.S. Food and Drug Administration (FDA) regarding changes to the trial’s protocol in order to address potential complications caused by the COVID-19 pandemic. Specifically, the trial will use telehealth (medical visits conducted via phone or computer call) in order to minimize in-person contact. Health Canada also has approved the revised study protocol.

“COVID-19 has caused disruption in clinical research for almost every drug developer around the world,” said Joseph Horrigan, MD, chief medical officer at AMO Pharma. “Based on our experience with clinical studies in pediatric neurological disorders, we were able to outline a path forward that both optimizes safety for participants in the REACH-CDM study and positions us to provide regulators with a clear and unambiguous assessment of outcomes.

“We are grateful that FDA has agreed to our approach of using telehealth as a strategy to manage the potential impact of COVID-19 on the progression of this important study,” Horrigan added.

AMO-02 is an oral medication that blocks the activity of an enzyme called glycogen synthase kinase 3 beta. This enzyme is overly active in people with certain kinds of muscular dystrophy, including CDM1 and Duchenne muscular dystrophy. By inhibiting the enzyme’s activity, AMO-02 is expected to improve the muscle health of patients.

Prior results from a Phase 2 clinical trial (NCT02858908) indicated that AMO-02 eased muscle- and nervous system-related symptoms in people with CDM1. The therapy also was found to be generally safe and well-tolerated.

The REACH-CDM study was designed based on these previous results. Its primary outcome (the main measurement of effectiveness) will be changes in the Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS), an 11-item rating scale completed by a clinician that scores the severity of features associated with CDM1. The FDA has agreed to this primary outcome measure.

Participants will be assigned randomly to receive either AMO-02, adjusted to weight at 400 mg, 600 mg or 1,000 mg dose levels, or a placebo once daily. Other outcome measures include assessments by clinicians and caregivers.

“The unique aspects of this research trial that interest me are utilization of telehealth as well as the primary outcome measure used to assess the treatment response,” Veerapandiyan said. “I am hopeful that this investigational product can be a life changer for patients living with this ultra-rare severe muscular dystrophy and their families.”

Added Ibs Mahmood, AMO Pharma’s CEO: “We are very encouraged by the levels of interest in the REACH-CDM trial from investigators and researchers and from families who are so anxious for a treatment.

“We are dedicated to advancing the development program for AMO-02 as rapidly as possible and are grateful to the regulators, investigators and leaders in patient advocacy for their support,” Mahmood said.

AMO-02 recently was given rare pediatric disease status by the FDA. The agency also granted fast track status to the investigational medication in 2017.