To all, 2 notes from Tuesday's conference call:
1. RRP trial endpoints
RRP is Recurrent Respiratory Papillomatosis, which is warts of the upper airways. It's caused by HPV types 6 and 11, which also cause genital and anal warts.
The RRP trial had the endpoint of increasing the length of time between surgeries for RRP patients, who were 27 children between the ages of 2 and 18.
The patients were difficult. One half had "severe" disease according to guidelines. Seven of the patients had disease in the lung, which is dire indeed. Disease is often around the vocal chords and entrance to the airways. The group was "a difficult challenge".
RRP patients can have 60 to 100 surgeries in their lifetimes. A patient commonly has surgery 5 times a year, every 70 days. These are children. Its painful. Their voice quality is affected and the warts obstruct air flow. $7,500 per surgery. Typically they have 60 to 100 surgeries in their lifetime. They go under general anesthesia. Some patients have hundreds of surgeries. For them it stinks big time.
The endpoint of increasing time between surgeries was easily met. HspE7 will go on to pivotal Phase III trials.
The secondary endpoint of doubling the time between surgeries was not quite met. The time between surgeries increased by 78% instead of 100%. Considering the really difficult patients, the result was great. Also, HspE7 in other trials has shown greater efficacy over time, so comparing a time immediately after administering the therapy was tough. They had to compare this way because these were children, so the airways would grow bigger over a longer period of time and might result in less frequent surgeries for that reason. The secondary endpoint, "if achieved, could further accelerate" the clinical trials program. Now they have to settle for orphan drug status, which is excellent.
2. HspE7 manufacturing decision
Manufacturing process "A" was used to make HspE7 material for pre-clinical and early clinical trials. The plan was for process "B" to be used for RRP Phase III and Roche's process "C" for commercial scale production. Those who follow biotech know each manufacturing change has a time delay penalty, because of understandable FDA requirements. There would have been 2 time hits- from A to B and from B to C.
The Stressgen/Roche Joint Development Team made the decision to go from process "A" to process "C" directly and to do it right now, before RRP Phase III. Material from Roche's commercial scale process will be used in RRP Phase III trials and all other HspE7 trials for genital warts, anal dysplasia, etc. The manufacturing change and FDA requirements will result in a single 6-month time hit, so RRP phase III will begin in the second half of 2004, instead of the first half.
The time hit was going to happen anyway. The decision was to shorten the time hit and take it now, instead of just before commercialization, a good decision IMHO.
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