Actinogen Medical Limited · ASX:ACW
Actinogen Medical — Bioshares Biotech Summit 2022
23 slides from Actinogen Medical's conference presentation, May 11, 2022
Slide 1: Newly confirmed cognitive enhancement data for Xanamem & next steps
For personal use only Bioshares Biotech Summit 2022: Newly confirmed cognitive enhancement data for Xanamem & next steps Tamara Miller MS, BSc, DipBus, PMP, CPPM SVP Product Development May 11, 2022 Authorised by the Board of Directors of Actinogen Medical Limited
Slide 2: Disclaimer
Disclaimer For personal use only This presentation has been prepared by Actinogen Medical Limited. (“Actinogen” or the “Company”) based on information available to it as at the date of this presentation. The information in this presentation is provided in summary form and does not contain all inform
Slide 3: Actinogen is a neurotherapeutics developer realizing a revolutionary therapy
For personal use only Actinogen is a neurotherapeutics developer realizing a revolutionary therapy so patients and their families can live their best lives Bioshares Biotech Summit 2022 3
Slide 4: Actinogen snapshot
Actinogen snapshot For personal use only Actinogen Medical (ASX:ACW) is developing a novel oral treatment with rapid onset of clinical activity to address a range of central nervous system (CNS) diseases Demonstrated target engagement in brain and HPA axis in human trials Favourable pharmaceutical
Slide 5: Xanamem: oral, low dose, once-a-day treatment with a unique mechanism
Xanamem: oral, low dose, once-a-day For personal use only treatment with a unique mechanism Brain penetrant 11β-HSD1 small molecule enzyme inhibitor reduces cortisol inside brain cells - modulating signaling pathways and underlying disease processes1,2 Potential to be: • Rapidly cognitive enhancing
Slide 6: Cognition Clinical Advisory Board
Cognition Clinical Advisory Board Renowned global thought leaders in clinical trials for assessment of Cognition For personal use only Prof. John Harrison Dr Dana C. Hilt Dr Christina Kurre Olsen Prof. Paul Maruff Metis Cognition Ltd • Expert psychologist with a special • 25+ years of drug developme
Slide 7: Established Scientific Advisory Boards
Established Scientific Advisory Boards For personal use only World-leading, premier academics involved in the development of Xanamem Alzheimer’s Disease Clinical Advisory Board Scientific Advisory Board Prof. Craig Ritchie Prof. Colin Masters Prof. Jeffrey Cummings Prof. Jonathan Seckl Prof. Brian W
Slide 8: Key implications from the new XanaMIA results
Key implications from the new XanaMIA results For personal use only • Trial met its primary safety, efficacy (cognition) and pharmacodynamic endpoints • Second consecutive double-blind trial to show improvement in attention and working memory with Xanamem • Confirms the target dose range of ≤10 mg s
Slide 9: Previously
For personal use only Previously Bioshares Biotech Summit 2022 9
Slide 10: Cognitive improvement vs. placebo in healthy, older volunteers in XanaHES trial in 2019
Cognitive improvement vs. placebo in healthy, older volunteers in XanaHES trial in 2019 For personal use only • Cogstate Cognitive Test Battery (CTB) with 20 mg daily, 12-week treatment; effect size (ES) estimated with the same MMRM statistical model as the current trial1 • Clinically significant ef
Slide 11: PET data supports exploring a lower Xanamem dose range of ≤10mg daily
PET data supports exploring a lower For personal use only Xanamem dose range of ≤10mg daily Baseline After Dose PET data demonstrates that Xanamem extensively binds 5 mg Xanamem 10 mg Xanamem 20 mg Xanamem to the 11β-HSD1 enzyme SUVRcarotid throughout the brain, with high post-treatment effects (abs
Slide 12: XanaMIA Phase 1b/2 trial design
XanaMIA Phase 1b/2 trial design For personal use only Targeting the first stages of Alzheimer’s Disease (AD) XanaMIA - Part A: XanaMIA - Part B: Double-blind Phase 1b Phase 2 H12022: minimally effective dose level 2023/24: placebo-controlled design in patients with early stages of Alzheimer’s Diseas
Slide 13: Effect size statistics measure “signal to noise”
Effect size statistics measure “signal to noise” For personal use only Ratios commonly used in trials of Alzheimer’s Disease using raw or statistical model data Cohen’s d Z-score is a standardization method for a normal distribution 1. ADASCog endpoint using ES as Cohen’s d (Aduhelm: estimated from
Slide 14: XanaMIA dose-ranging topline results
For personal use only XanaMIA dose-ranging topline results Bioshares Biotech Summit 2022 14
Slide 15: ACTH hormone response suggests similar Xanamem biological activity at 5 mg and 10 mg dose levels
ACTH hormone response suggests similar Xanamem biological activity at 5 mg and 10 mg dose levels For personal use only ACTH is the brain hormone that regulates cortisol production in the adrenal gland Mean (times) increase from baseline in ACTH hormone level at end of treatment* by dose level across
Slide 16: Attention Composite improved vs. placebo
Attention Composite improved at Week 4 and 6 vs. placebo Pooled working memory/visual attention/psychomotor speed (mean, SE) For personal use only 0.35 ON-TREATMENT VISITS 0.3 BETTER 0.25 Mean change from baseline 0.2 (Units) 0.15 0.1 0.05 0 Baseline Week 2 Week 4 Week 6 Week 10 Xanamem pooled doses
Slide 17: Xanamem was safe and well-tolerated
Xanamem was safe and well-tolerated For personal use only • No treatment-related serious adverse events • Other predominantly mild events were generally equally distributed across the three groups including placebo • No adverse events of concern, consistent with program safety database of over 300 p
Slide 18: Summary
Summary For personal use only • There is a strong unmet need for new and safer treatments for many diseases where dysregulated cortisol is a target - Xanamem is designed to reduce cortisol inside brain cells • Xanamem is potentially a rapidly-acting cognitive enhancer and, in the longer term, a dise
Slide 19: Next: Moving Xanamem trials into AD patients with a focus on cognitive enhancement
Next: Moving Xanamem trials into AD patients with a focus on cognitive enhancement For personal use only No functional impairment XanaMIA XanaMIA Functional impairment & loss of independence Part A Part B Healthy 50-80 MCI/early AD* patients years • Randomized, placebo- Mild • Safe & well-tolerated
Slide 20: Actinogen Strategy
Actinogen Strategy For personal use only Accelerate clinical development Create value from partnerships • Focus on cognitive enhancement: Pharma/biotech engagement • Early stages of Alzheimer’s Disease • Actively engage large and mid-size • Implement XanaMIA Part B potential partners with new result
Slide 21: Xanamem Clinical Development Pipeline
Xanamem Clinical Development Pipeline For personal use only Phase 2 Pathway Outlook XanaMIA (IND) Cognitive impairment in Part A: 10mg, 5mg, Placebo Older Volunteers: Cognition “Big-to-market” early Alzheimer’s disease Part B: Patients with early stage AD: Cognitive enhancement & Multiple Phase 2b/3
Slide 22: Timeline for Xanamem data & catalysts
Timeline for Xanamem data & catalysts For personal use only 2022 - Q2 XanaMIA Part A confirms cognitive enhancement - Q2 BIO in-person pharma/biotech meetings with new data - Biomarker data to assess disease-modifying potential - Human PET scan peer reviewed publication - Scientific presentation of
Slide 23: Thank you and Questions
For personal use only Thank you and Questions Bioshares Biotech Summit 2022 23