September 21, 2015 11:54am
QURE is confirming these encouraging trends on cognitive development over the next 6-12 months … based on these results, QURE is negotiating an agreement with the consortium to take over the sponsorship of the program and intends to advance the program into the pivotal stage of development. QURE is trading DOWN -$4.03 <-12.06%> in Monday’s am session having closed at $33.41 on 9/18.
In four Sanfilippo B syndrome patients treated with a novel gene therapy, AMT-110; positive signs of efficacy demonstrated in all four patients.
QURE announced the topline results of one-year follow-up data from a P1/2 clinical trial conducted by Institut Pasteur in partnership with the French Muscular Dystrophy Association and Vaincre les Maladies Lysosomales in four Sanfilippo B syndrome patients treated with a novel gene therapy, AMT-110. In all four patients, researchers verified the restoration of catalytical activity of the NaGlu protein in the cerebrospinal fluid from 0% at baseline up to 14-17% of normal at 3 months with persistent effect at 12 months.
In the trial, four patients received a one-time administration of AAV5 gene therapy dosed over two hours directly into the brain. All patients were maintained under coverage of a continuous immune suppression regimen. No local inflammation or other safety concerns related to the therapy or the procedure have been identified. In addition to establishing the safety of the procedure and the AAV5 viral vector, the most important result was the presence of catalytically active NaGlu protein in the CSF measured at 1, 3, and 12 months after treatment.
The Bottom Line: These results validate the effective transmission of the NAGLU gene with the AAV5 vector. The trial demonstrated that incremental cognitive development was maintained in all four (4) patients, aged 20 to 53 months at study onset.
Selling it seems is based on strength and positive news flow.
