next Biotech, page-3

At the start of the trial, expectations of a clinical benefit using the low dosage rate of 25mg once weekly, were rather subdued. We could hope for the best in regards to efficacy but it was the safety and tolerability that appeared key to the advancement to the next clinical stage. Hold on, lets see what has happened!

According to Professor Voit, "disease stabilisation or indeed improvement in functional scores in non-ambulant DMD boys is almost unheard of and a very encouraging result.....even more meaningful....over a relatively short trial time of 24 weeks". Voit is not talking about expected results from a higher dosage but the actual results from the current trial using the low dosage rate.

It might explain the comment by William Goolsbee, "we now look to have a drug".

Indeed, we do look to have a drug. Yes, the proposed phase 11b clinical trial will seek to optimise the clinical benefit at a higher dosage but even the fall back dosage rate of 25mg once weekly could well justify approval in an area of relative unmet need.

We look to have a drug and we know a lot about a number of key aspects critical to a successful pivotal trial. For a start we understand the mechanism of action. Secondly, there is general acceptance by the agencies of the study endpoints. All in all there appears to be a clear pathway to approval.