Audentes Therapeutics has reported that the first patient has been enrolled in INCEPTUS, a study to characterise the disease presentation in children suffering with X-Linked Myotubular Myopathy (XLMTM).

The study will evaluate patients before their enrolment in ASPIRO, which is a planned Phase 1 / 2 clinical study meant for evaluation of the safety and preliminary efficacy of AT132, a product candidate for treatment of XLMTM.

Audentes senior vice-president and chief medical officer Dr. Suyash Prasad said: "The initiation of enrolment for INCEPTUS is an important first step in the clinical development of AT132 for the treatment of XLMTM and a notable milestone for Audentes.

"The burden of the disease of XLMTM to children and families is considerable, and we are grateful to those who have agreed to participate in the study."

"The burden of the disease of XLMTM to children and families is considerable, and we are grateful to those who have agreed to participate in the study."

INCEPTUS is a study on XLMTM in boys, aged three years or younger. It is intended to characterise the individual disease presentation with a particular focus on respiratory measurements, as well as assessment of muscle strength and function.

Patients who have been enrolled in INCEPTUS will be evaluated over a three to twelve month period before their potential participation in ASPIRO.

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INCEPTUS will serve as a longitudinal baseline and within-patient control for subjects who enrol in the ASPIRO study. Furthermore, it will facilitate the operational aspects of ASPIRO once initiated.

Audentes intends to file investigational new drug applications for AT132 with regulatory authorities of North America and Europe in the first quarter of 2017.

It expects preliminary data from the ASPIRO study in the fourth quarter of 2017.