Audentes Therapeutics is currently developing a gene therapy drug, named AT132, for the potential treatment of X-linked myotubular myopathy (XLMTM). AT132 uses a harmless adeno-associated virus to deliver a healthy copy of the MTM1 gene to the body. AT132 is currently being tested in the US, in a phase 1/2 trial named ASPIRO.
Audentes Therapeutics recently announced that the Medicines and Healthcare Products Regulatory Agency (MHRA) has approved the Clinical Trial Authorisation (CTA) application for testing AT132 in the UK. This means Audentes Therapeutics can start working with UK based clinical study sites to start enrolling individuals with XLMTM into their ongoing ASPIRO trial.
Mr Matthew R Patterson, President and Chief Executive Officer at Audentes Therapeutics, said in a press release:
This CTA approval represents another important milestone for our AT132 program. We recently announced dosing of the first patient in ASPIRO at a U.S. clinical study site, and we are pleased to be working closely with the European XLMTM community as we continue to execute on our global plans to develop AT132 as a potentially transformative product to treat this devastating rare disease.
ASPIRO is a multi-centre, open-label, dose-ascending study which will enrol 12 children who are less than five years old with XLMTM. The study will compare the safety and efficacy of three different doses of AT312 in nine of the twelve participants. The remaining three participants will be part of a delayed-treatment control group, who will be treated once the optimal dose of AT132 has been established. Primary efficacy analysis will be assessed twelve months after treatment, with participants being monitored for a further four years to assess long-term safety and developmental progression. Preliminary results from the ongoing ASPIRO study are expected by the end of the year.
In September 2017 Audentes Therapeutics announced the US Food and Drug Administration (FDA) granted Rare Paediatric Disease and Fast Track status for AT132. In addition, AT132 has also received Orphan Drug status from both the FDA and the European Medical Agency. These statuses will help speed-up the development and review process of AT132, which will help get the therapy to patients quicker.