AMO Pharma has announced its plans to initiate a Phase III clinical trial for its investigational therapy, AMO-02 (tideglusib), aimed at treating type 1 myotonic dystrophy (DM1).
This decision follows a meeting with the US Food and Drug Administration (FDA), where the design and objectives of the upcoming trial were discussed.
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The trial will evaluate the efficacy and safety of AMO-02 in adult patients with adult-onset DM1 and will support future marketing authorisation submissions for the treatment across various age groups.
The company’s previous REACH-CDM Phase II/III clinical study, which involved children and adolescents with congenital-onset DM1, found that AMO-02 was generally safe and well-tolerated.
There were no severe adverse events related to the treatment reported.
However, the study’s primary and secondary outcomes, based on clinician-administered subjective ratings, revealed an unexpected placebo effect. This effect may have obscured some of the treatment’s beneficial impacts.
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By GlobalDataFurther post hoc analysis, as assessed by the regulator, suggested that there is ample evidence to warrant the continued development of tideglusib for DM1 treatment.
The FDA then agreed that AMO Pharma should proceed with a Phase III study in adults with the adult-onset form of DM1.
The outcome measures for the upcoming study in adult-onset DM1 will be similar to those that indicated benefits in the REACH-CDM Study.
Following discussions with the FDA, AMO Pharma plans to compile a comprehensive package of safety and efficacy data from the new Phase III trial in adults.
This package will also include efficacy, pharmacokinetic, and safety data from the REACH-CDM Study to support a future submission for approval in children, adolescents, and adults with myotonic dystrophy.
AMO Pharma executive chair Alan Rubino said: “We are pleased with the outcome of the productive discussions we had with the FDA regarding the next steps for AMO-02 and continue to feel strongly that this innovative therapy can represent a significant advance in the treatment of patients with DM1, whether they are children, adolescents or adults.
“We are now focused on progressing a new clinical study in adults with adult-onset DM1.
“We will make an announcement regarding the start date and site locations for the adult-onset DM1 study shortly.”
