Replies to post #59544 on Biotech Values

[quantumdot]

PRAN. Here is the trial info from the Appendix mentioned in the PR.

i own the stock and am somewhat pleased with these results although the fact they missed on ADAS-cogs and also do not mention the other biomarkers (besides the very strong result against Abeta42) is a little troubling. so little really understood about early stage disease markers though.

Number of patients 
 78 patients randomized (Intent-to-Treat population); 74 patients completed study. 
  
 Key patient selection criteria 
 -Fulfill National Institute of Neurological and Communication Disorders and Stroke (NINCDS)/Alzheimer's Disease and Related Disorders Association (ADRDA) criteria for probable AD and International Classification of Diseases (ICD)-10 criteria for AD. 
 -Age over 55 years 
 -Mini-mental Status Examination (MMSE) score between 20 and 26 inclusive or ADAS-Cog score of 10-25. 
 -Modified Hachinski score ≤ 4 
 -Computerised tomography (CT) or magnetic resonance imaging (MRI) scan within the last 24 months which, in the opinion of the local investigator, does not alter the diagnosis of AD to vascular or multi-infarct dementia. 
 -Stable dose of donepezil, rivastigmine or galantamine for at least 4 months with clinical evidence of deterioration. 
  
 Rationale 
 The interactions between key metals and beta amyloid (Abeta) in the brains of patients suffering from AD may be related to both the pathogenesis and devastating toxicity of the disease. Because PBT2 interferes with this interaction and inhibits toxic Abeta oligomer production, PBT2 is being developed as a potential disease modifying treatment for AD. 
  
 Blinding 
 Double-blind 
  
 Placebo controlled 
 Yes 
  
 Route of administration 
 Oral (capsules) 
  
 Study design 
 Randomised, double-blind, placebo-controlled, parallel three-group study to assess the safety and tolerability of PBT2 in patients with early AD. A modified dose-escalation element with ongoing Data Safety Monitoring Board (DSMB) review was included at the start of the study. An initial cohort of patients were allocated to either 50mg or placebo and data was monitored by the DSMB. Further patients randomised were allocated to either 50mg, 250mg or placebo, again with ongoing DSMB monitoring. 
  
 Dose groups 
 0mg (placebo capsules of identical appearance), 50mg and 250mg 
 Duration 
 12 weeks once daily dosing with 2 week follow-up 
  
 Primary endpoints 
 Safety and tolerability 
  
 Secondary endpoints 
 CSF and plasma biomarkers (including Abeta42, Abeta40, total Tau, P-Tau); cognition readouts 
  
 Trial sites 
 15 clinical trial sites in Australia (7) and Sweden (8) 
  
 Contract Research Organisation 
 Quintiles Limited, Berkshire, UK 
  
 Patient demographics 
 • Mean age 72 years (range 58 – 83 years) 
 • 50% male, 50% female 
 • ApoE e4 genotype 75.6% 
 • Mean ADAScog score at entry 
 • 50mg 18.9 
 • 250mg 18.7 
 • Placebo 18.9 
 • Mean MMSE score at entry 
 • 50mg 23.2 
 • 250mg 23.5 
 • Placebo 22.2 
 All characteristics similar across dose groups 
  
 Primary endpoint 
 PBT2 in this study of early AD patients was safe and well tolerated, with no significant findings or trends in any of the safety parameters measured. The safety and tolerability profile of either dose of PBT2 was indistinguishable from placebo. 
 Secondary endpoints - biomarkers 
 PBT2 250mg showed a statistically significant reduction of CSF Abeta42 after 12 weeks of treatment compared with placebo (p=0.006, ITT). 
  
 Secondary endpoints - cognition 
 PBT 250mg showed statistically significant improvements in both the Trail Making Test part B and the Category Fluency Test (NTB sub-tests) after 12 weeks of treatment compared with placebo(p=0.005 and p=0.028, respectively (ITT)). 
  
 

[Kadaicher1]

Pran, he did say those tests were better able to evaluate mild stage Alzheimers patients. I am just happy to get any cognition improvement and beat the placebo, aricept, a billion dollar drug.