Prana Biotechnology (PRAN)

[kookiekook]

Please read the full test results for yourself before making any determination as to whether PBT2 shows promise. The results are posted at http://pranabio.com/news/prana-announces-successful-phase-2-results-huntington-disease-trial#.UwNjNH7nZLN . It is great the it shows that the drug is safe, but it does not show any significant health improvements, that I can see.


Primary Objective: Safety and Tolerability

PBT2, in this study of early- to mid-stage HD patients, was safe and well tolerated, with no significant findings or trends in any of the safety parameters measured.
Tolerability: PBT2 was well tolerated during this study as demonstrated by no difference in the Kaplan-Meier estimates of time-to-withdrawal between the 100mg dose and placebo groups (p=0.297) or between the PBT2 250mg and placebo groups (p=0.173). Of the 109 patients randomized, 104 patients completed the study (95.4% retention rate). Of the five participants who withdrew from the study, one participant in the placebo group and 3 participants in the 250mg group withdrew due to adverse events. One participant did not return for their follow up visit.
Safety: Of the 10 SAEs reported, one was in the placebo group, 3 in the 100mg PBT2 group and 6 in the 250mg group. With the exception of one SAE in the 250mg group, all SAEs were deemed to be not related to Study Drug by the site investigators. The participant who had an SAE deemed related to Study Drug reported a worsening of their HD symptoms during the 4 week follow up period (i.e. no Study Drug administered), after completing the 6 month treatment.
The safety and tolerability profile of either dose of PBT2 was similar to placebo. There were no significant differences in the numbers of participants reporting any particular AE between PBT2 and placebo groups. The most common AE was diarrhea, with 16 participants reporting a total of 20 events. The frequency of this AE was similar across PBT2 and placebo groups.


Secondary Objectives: Efficacy
Main/Primary Efficacy Endpoint: Cognition


Main Composite Cognition z-score and Exploratory Composite Cognition z-score. No statistically significant changes.
Executive Function Composite z-score: PBT2 250mg showed a significant improvement at 12 weeks (p=0.005) and trend at 26 weeks (p=0.069) compared with placebo. On a pre-specified subgroup analysis of early-stage HD (TFC 11-13), the change in Executive Function Composite z-score from baseline at 26 weeks was significantly improved in participants receiving 250mg PBT2 compared with placebo (p=0.038).
Of the two tests within the Executive Function Composite, there was a statistically significant improvement in the Trail Making Test Part B after 12 weeks of treatment compared with placebo (p<0.001) and at 26 weeks (p=0.042). The effect of PBT2 at 26 weeks was dose-dependent (p=0.035).
There were no statistically significant differences between either dose of PBT2 and placebo in other individual tests of cognition over 26 weeks.


Secondary Objective: Efficacy

Motor, Behaviour, Function, Global Endpoints

No significant changes were seen in motor, functional, behavioural or global assessments in either PBT2 treatment group compared to placebo over the 26 week treatment period.
A small but positive signal in TFC was observed on the 13 point scale across the PBT2 groups relative to placebo (mean changes from baseline:
250mg PBT2 = -0.3; 100mg PBT2 = -0.5; placebo= -0.6.


Secondary Objective: Efficacy–Biomarker Endpoint

There were no significant changes in the urine or blood biomarkers assessed at week 26 with PBT2 treatment compared to placebo.


Secondary Objective:
MRI brain volumes and function

Changes in cortical thickness (mm) were mapped at week 26 for the combined treatment group (n=2 250mg and n=2 100mg) compared to placebo (n=2). The rate of thinning in the placebo group was faster than in the treated groups; however the effects did not reach statistical significance.