Slide 1: Reach2HD Phase 2 Clinical Trial Top Line Results Investor Conference Call
Reach2HD Phase 2 Clinical Trial Top Line Results Investor Conference Call 19 th February 2014
Slide 2: Safe Harbour
This presentation may contain some statements that may be considered “ Forward - Looking Statements ” , within the meaning of the US Securities Laws . Thus, any forward - looking statement relating to financial projections or other statements relating to the Company ’ s plans, objectives, expectatio
Slide 3: Introduction
Introduction Mr Geoffrey Kempler, Chairman and Chief Executive Officer Dr Ray Dorsey, Professor of Neurology, University of Rochester; Principal Investigator Dr Rudy Tanzi, Professor of Neurology, Harvard Medical School; Prana Chief Scientific Advisor Dr Ira Shoulson, Professor of Neurology, Georget
Slide 4: Mr Geoffrey Kempler, Chairman and Chief Executive Officer
Mr Geoffrey Kempler, Chairman and Chief Executive Officer
Slide 5: Dr Ray Dorsey, Professor of Neurology, University of Rochester; Principal Investigator
Dr Ray Dorsey, Professor of Neurology, University of Rochester; Principal Investigator
Slide 6: Outline
• Huntington disease and cognition • Reach2HD study • Results Outline
Slide 7: Huntington disease (HD) is a rare, inherited neurodegenerative disorder
Huntington disease (HD) is a rare, inherited neurodegenerative disorder Who What When Where Why • Approximately 30,000 Americans and over 80,000 individuals globally • Affects both sexes equally • Inherited disorder that causes involuntary movements (chorea), behavior changes, and cognition decline
Slide 8: Preclinical and clinical data supported the study of PBT2 in HD
Preclinical and clinical data supported the study of PBT2 in HD Mechanism Preclinical study Clinical study • In Huntington disease, copper concentrations are elevated in the brain (basal ganglia) where they could promote aggregation of mutant huntingtin • PBT2 belongs to a class of metal - protein a
Slide 9: Trail Making Test Part B is a test of executive function, which is impaired in HD
Trail Making Test Part B is a test of executive function, which is impaired in HD Sources : Tabrizi SJ et al. Lancet Neurol 2013;12:637 - 49, Dorsey ER et al. JAMA Neurol 2013;310:1520 - 30, Paulsen JS et al. JNNP 2013;84:1233 - 9, Stout JC et al. Cogn Behav Neurol 2007;20:212 - 8, O’Rourke JJ et al
Slide 10: The Reach2HD: Phase 2, randomized, double-blind placebo-controlled study
The Reach2HD: Phase 2, randomized, double - blind placebo - controlled study 36 randomized to PBT2 250mg once daily Study design 109 individuals with early to mid - stage Huntington disease Study Objectives Primary: To evaluate the tolerability and safety of PBT2 Secondary: To evaluate the effect of
Slide 11: Baseline characteristics of participants were well balanced across groups
Baseline characteristics of participants were well balanced across groups Baseline characteristics of the Reach2HD study population Characteristic Placebo (N=35 ) PBT2 100mg (N=38) PBT2 250mg (N=36) All (N=109) Mean age in years (range) 51.2 (30 - 66) 54.1 ( 31 - 79) 50.3 ( 28 - 70) 51.9 ( 28 – 79)
Slide 12: PBT2 was well tolerated
PBT2 was well tolerated ... Tolerability PBT2 250mg daily PBT2 100mg daily Placebo • 32 (88.9%) of the 36 individuals randomized to PBT2 250mg completed the study • 38 (100%) of the 38 individuals randomized to PBT2 100mg completed the study • 34 (97.1%) of the 35 individuals randomized to placebo c
Slide 13: ... and generally safe in the study
... and generally safe in the study Safety of PBT2 Serious adverse events Adverse events • Ten serious adverse events occurred during the study • Nine were in the PBT2 groups (6 in PBT2 250mg and 3 in PBT2 100 mg) • Only one (on PBT2 250mg) was deemed related to study drug by the site investigator •
Slide 14: PBT2 250mg significantly improved performance on Trail Making Test Part B
PBT2 250mg significantly improved performance on Trail Making Test Part B Change in Trail Making Test Part B Improvement in Trail Making Test Part B was significant at 12 (p<0.001) and 26 weeks (p=0.042)
Slide 15: Trend toward improvement on the executive function composite z-score
Trend toward improvement on the executive function composite z - score Other cognitive outcomes Executive function composite Among all participants, there was a trend toward improvement in the composite executive function for those randomized to PBT2 250mg (p=0.069) that was significant among those
Slide 16: Cognitive improvement also accompanied by a trend toward improvement on functional capacity
Cognitive improvement was also accompanied by a trend toward improvement on functional capacity Other efficacy outcomes Total Functional Capacity • Total Functional Capacity is a key measure of function in occupation, finances, domestic chores, activities of daily living, and care level that is used
Slide 17: Small, exploratory neuroimaging study suggested decreased atrophy among those exposed to PBT2
Small, exploratory neuroimaging study suggested decreased atrophy among those exposed to PBT2 Exploratory outcome Imaging results • In a small (n=6), pilot sub - study, individuals randomized to PBT2 (n=4) had reduced brain atrophy compared to those randomized to placebo • Brain atrophy is known to
Slide 18: PBT2 is a promising therapy for a cardinal feature of HD
PBT2 is a promising therapy for a cardinal feature of HD Summary Tolerability and safety • PBT2 was well tolerated and generally safe over 26 weeks in individuals with early to mid - stage Huntington disease Efficacy • PBT2 250mg daily significantly improved cognition on a key measure of executive f
Slide 19: Dr Ira Shoulson, Professor of Neurology, Georgetown University; Chair, Huntington Study Group
Dr Ira Shoulson, Professor of Neurology, Georgetown University; Chair, Huntington Study Group
Slide 20: Q&A / Signatures
Q&A SIGNATURES Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized. PRANA BIOTECHNOLOGY LIMITED (Registrant) By /s/ Geoffrey Kempler Geoffrey Kempler, Executive Chairm