uniQure NV (NASDAQ:QURE) has been slumping since the beginning of the week and in the previous trading session its stock lost 3.21% to close the day at $33.18 per share.

The stock has continued to fall at the back of the announcement that the United States Food and Drug Administration had finalized uniQure’s AMT-130 application for Investigational New Drug.

Second phase AMT-130 study

Following the approval for Investigational New Drug the company can now begin their planned Phases I/II trials. The AMT-130 has a recombinant vector AAV5 that carries a DNA encoding cassette microRNA which non-selectively decreases the human huntingtin proteins in patients with Huntington’s disease.

Matt Kapusta, the Chief Executive Officer, stated that the FDA clearance of uniQure’s IND for AMT-130 is a critical milestone for patients with Huntington’s disease as well as a significant event in in the development of gene therapy. The CEO indicated that they expected AMT-130 to be the first AAV gene therapy administered one time to make it through clinical testing in treating Huntington’s disease. Huntington’s disease is a neurodegenerative disorder that is very devastating and it has no approved treatment.

AMT-130 silences abnormal genes

Mr Kapusta said the AMT-130 study equally represents the initial clinical stage for AAV based therapy designed precisely to silence the abnormal genes in the brain with just one administration through the miQURE platform. The CEO believes that their proprietary miQURE technology platform form gene silencing can potentially get applied in management of other diseases like type III spinocelebellar ataxia.

Approval of IND application is a significant milestone for their company’s research organization considering they have dedicated many years of resources towards the development of an effective therapy.

With the FDA approval of the IND the company can now progress to initiate the initial Phase I/II controlled dose escalation and randomized clinical trials planned for the assessment of tolerability, efficacy and safety of AMT-130 as a one-time treatment for Huntington’s disease patients. uniQure anticipates opening numerous clinical sites across the United States and initiating dosing of the patients by the second half of 2019.

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