Actinogen Medical Limited · ASX:ACW
Actinogen Medical — ASX Small & Mid-Caps Conference 2025
23 slides from Actinogen Medical's conference presentation, Mar 26, 2025
Slide 1: ASX Announcement: Actinogen presentation to ASX small & mid-caps conference
ASX ANNOUNCEMENT Actinogen presentation to ASX small & mid-caps conference Sydney, 26 March 2025. Actinogen Medical ASX: ACW (“ACW” or “the Company”) is pleased to announce that its CEO, Dr Steven Gourlay, will be a speaker this afternoon at the ASX small & mid-caps conference in Sydney. Dr Gourlay’
Slide 2: Announcement continued: clinical trials and about Xanamem
behavioural abnormalities, and neuropsychological burden associated with these conditions is debilitating for patients, and there is a substantial unmet medical need for new and improved treatments. Clinical Trials The XanaMIA Phase 2b/3 Alzheimer’s disease trial is a double-blind, 36-week treatment
Slide 3: Oral Xanamem (emestedastat): Controlling brain cortisol in Alzheimer's disease and depression
For personal use only Oral Xanamem® (emestedastat) Controlling brain cortisol to slow progression in Alzheimer’s disease and treat depression: enrolling pivotal Phase 2b/3 trial in Alzheimer’s ASX SMIDCaps conference 26 March 2025 ® Xanamem is a registered trademark of Actinogen Medical Limited
Slide 4: Disclaimer
Disclaimer • 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 information necessary to m
Slide 5: Corporate snapshot
Corporate snapshot For personal use only ASX-listed company founded in 2014 • Market Cap ~$100 million • Cash balance of $22.9 million at Dec 31 2024 provides runway to at least mid 2026 • Conducted three phase 1 (Australia) and four phase 2 trials (Australia, US and UK) Key shareholders • Biotech V
Slide 6: Neuroscience is hot for late-stage programs
Neuroscience is hot for late-stage programs For personal use only M&A Ph2 M&A Ph3 M&A Ph2/3 Principia1/Sanofi Karuna/BMS Cerevel/Abbvie MS, Immunol. Schizophrenia Schizophrenia $3.7B 2020 $14B 2023 $8.7B 2023 M&A Ph2 License Ph2 Longboard/ License Ph1 PTC/Novartis Lundbeck Voyager/Novartis Huntingto
Slide 7: Experienced board and management team
Experienced board and management team Board of Directors For personal use only Dr. Geoff Brooke Dr. Steven Gourlay Mr. Malcolm McComas Dr. George Morstyn Dr. Nicki Vasquez Chairman CEO & MD Non-Executive Director Non-Executive Director Non-Executive Director MBBS; MBA MBBS; FRACP; PhD; MBA BEc, LLB;
Slide 8: Xanamem is now in advanced stages of development
Xanamem is now in advanced stages of development Novel 11β-HSD1 cortisol control mechanism, oral, attractive safety profile • Brain cortisol has long been proposed as a pathogenic mechanism in Major Depressive Disorder (MDD) and Alzheimer’s (AD) For personal use only • Unique brain-penetrant tissue
Slide 9: Xanamem controls cortisol by inhibition of 11β-HSD1
Xanamem controls cortisol by inhibition of 11β-HSD11 Controlling brain cortisol2 has potential durable benefits For personal use only Reduction of “stress response” in brain RAPID changes in kinases, cell function, “Lower stress” shorter term e.g. neurotransmitters over hours to days • Reducing infl
Slide 10: Human PET study shows full target engagement
Human PET study shows full target engagement Other 11β-HSD1 enzyme inhibitors have not achieved adequate brain levels For personal use only Baseline After 7 days of daily dosing 5 mg Xanamem 10 mg Xanamem 20 mg Xanamem Xanamem extensively binds SUVRcarotid to the 11β-HSD1 enzyme throughout the brain
Slide 11: Alzheimer's disease
Alzheimer’s disease Strong cortisol control scientific rationale to address huge unmet medical need For personal use only Rationale Growing Alzheimer’s Disease market – U.S. • Cortisol levels are elevated in brain fluid in Large, unsatisfied and growing market early AD • Chronic corticosteroid treat
Slide 12: Anti-amyloid therapy modestly slows AD progression
Anti-amyloid therapy modestly slows AD progression Ideally patients with AD would not worsen on treatment at all 0.5 For personal use only Ideal trajectory Worsening of 0 with disease CDR-SB stabilization over 18 months -0.5 Unmet need -1 despite amyloid therapy -1.5 -2 Benefit of donanemab over 18
Slide 13: Xanamem benefit in pTau181-positive AD patients
Xanamem benefit in pTau181-positive AD patients Phase 2a biomarker study: major slowing of CDR-SB decline (n=34) For personal use only Xanamem Placebo 0 0.2 0.4 0.36 Xanamem vs. Placebo CDR-SB units 0.6 • Mean 0.6 units Change from • Median 0.8 units Baseline (SE) • p = 0.09 0.8 • Cohen’s d = 0.4 1
Slide 14: XanaMIA phase 2b/3 trial in Alzheimer's disease
XanaMIA phase 2b/3 trial in Alzheimer’s disease Initial, interim results in Q4 2025, final results H2 2026 For personal use only 10mg Xanamem Follow-up & open label Screening from Dec 23 Placebo -4 0 36 Weeks 40 Weeks Double-blind, randomised treatment period (N=220) Interim analysis N=100 @ 24 week
Slide 15: Six different datasets validate Xanamem 10mg dose
Six different datasets validate Xanamem 10mg dose For personal use only Data source Conclusion Safety data n > 400 No concerning safety signals Phase 1 Adequate blood levels Phase 1 Adequate brain fluid levels Human PET study High target binding in the brain Phase 1b Improved cognition in healthy pe
Slide 16: Building positive momentum
Building positive momentum Evidence of durable benefit on depression from control of brain cortisol validates For personal use only the Xanamem program in terms of: ✓ “Cortisol control” mechanism of action ✓ 10 mg daily proof-of-concept dose being used in Alzheimer’s phase 2b/3 trial ✓ 10 mg daily d
Slide 17: A busy 2025 of milestones and announcements
A busy 2025 of milestones and announcements Milestone Likely Timing For personal use only First patient randomized and treated in US, XanaMIA trial Q4 24 Meetings at JP Morgan Healthcare conference week, San Francisco Q1 25 Clinical pharmacology manuscript peer-reviewed publication Q1 25 FDA Type C
Slide 18: Actinogen investment highlights
Actinogen investment highlights Novel 11β-HSD1 cortisol control mechanism, oral, attractive safety profile For personal use only Potential to be the “holy grail” for Alzheimer’s Positive phase 2a clinical data in two diseases has proven the “cortisol hypothesis” Phase 2b and 3 trials significantly d
Slide 19: Appendix
For personal use only Appendix ASX SMIDCaps conference 26 March 2025 17
Slide 20: Key references
Key references Other references see also https://actinogen.com.au/xanamem 11β-HSD1 inhibition Alzheimer’s disease and cortisol • Seckl J. 11β-Hydroxysteroid dehydrogenase and the brain: Not (yet) lost in translation. J Intern Med. 2024 Jan;295(1):20-37. doi: 10.1111/joim.13741. Epub 2023 Nov 8. • Pl
Slide 21: Selected Glossary
Selected Glossary 1 • 11β-HSD1 – 11 beta HydroxySteroid Dehydrogenase-1 enzyme. Selectively expressed in brain, liver, adipose. • Aβ – Amyloid beta – a type of amyloid protein associated with Alzheimer’s Disease, 42 and 40 are different forms For personal use only • ACTH – Adrenocorticotropic hormon
Slide 22: Selected Glossary
Selected Glossary 2 • IQCODE – Informant Questionnaire on Cognitive Decline in the Elderly • MCI – Mild Cognitive Impairment – memory, executive function deterioration with retained functional abilities For personal use only • MDD – Major Depressive Disorder • MMSE – Mini Mental State Examination –
Slide 23: Contacts
Contacts For personal use only Michael Roberts Steven Gourlay Investor Relations CEO & Managing Director P: +61 2 8964 7401 P: +61 2 8964 7401 M: +61 423 866 231 E. steven.gourlay@actinogen.com.au E. michael.roberts@actinogen.com.au ASX SMIDCaps conference 26 March 2025 21
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