Slide 1: ASX Announcement: Investor Presentation by Cogstate Executives
ASX Announcement 13 October 2023 Investor Presentation by Cogstate Executives Cogstate (ASX.CGS) advises that senior executives of the company will be hosting a 90-minute investor briefing session on Friday 13 October at 11:00 AM Australian Eastern Summer Time. For personal use only DESCRIPTION Inve
Slide 2: About Cogstate
About Cogstate Cogstate Ltd (ASX:CGS) is a leading neuroscience technology company optimising brain health assessments to advance the development of new medicines and to enable earlier clinical insights in healthcare. Cogstate technologies provide rapid, reliable and highly sensitive computerised co
Slide 3: Cogstate Investor Presentation
Cogstate Investor Presentation For personal use only Alzheimer’s Disease Landscape | Rare Disease Landscape | Clinical Trials Capabilities 13 October 2023 Brad O’Connor Chris Edgar, PhD Rachel Colite Pam Ventola, PhD CEO Chief Science Officer EVP, Clinical Trials VP, Science © 2023 Cogstate Ltd. All
Slide 4: Alzheimer's Disease Clinical Trials Landscape
For personal use only Alzheimer’s Disease Clinical Trials Landscape © 2023 Cogstate Ltd. All rights reserved.
Slide 5: Landmark Approvals with More Progress to Come
Landmark Approvals with More Progress to Come For personal use only Tacrine approved by Food and Drug Administration (FDA) in 1993, donepezil 1996, rivastigmine 1998, galantamine 2001, and memantine 2003 Aduhelm 2022 and Lequembi 2023 first new FDA approvals in almost 20 years Modest efficacy (≈30%
Slide 6: Revised Clinical Staging Criteria: Increasing Clarity Around Predementia AD
Revised Clinical Staging Criteria: Increasing Clarity Around Predementia Alzheimer’s Disease (AD) For personal use only FDA Draft Early AD Guidance NIA-AA 2018 Clinical Stages Measurement concepts: Stage 1: Stage 1: Patients with characteristic pathophysiologic changes of AD but no • Performance in
Slide 7: Clinical Trials Landscape
Clinical Trials Landscape For personal use only How to increase effectiveness and access? Active Research Area • Investigation of non-amyloid mechanisms of action • As of January 1, 2023, (combination therapy) • 187 trials assessing 141 unique treatments for AD • Routes of administration not • Phase
Slide 8: Preclinical AD: An Opportunity for Disease Modifying Therapies
Preclinical AD: An Opportunity for Disease Modifying Therapies For personal use only Prevention trials in patients with pathophysiologic changes of AD and no or only subtle cognitive abnormalities • Opportunities – Larger market – Greater potential effectiveness • Challenges – Larger sample size – L
Slide 9: Decentralized Clinical Trials (DCT) in Preclinical AD
Decentralized Clinical Trials (DCT) in Preclinical AD For personal use only Greater flexibility in • Patients and study partners are younger and more study design and cognitively able to engage with DCT technology conduct • Longer trial duration and greater likelihood of work commitment drives need
Slide 10: Multiple Opportunities for Digital Tools to Address Challenges
Multiple Opportunities for Digital Tools to Address Challenges For personal use only Hampel et al, Nat Aging 2022 Cognitive Screening and Early Detection | Brain Health Awareness | Home-based Computerized Cognitive Assessments | Clinical Trial Architecture in the Community | Digital Tools | Digital
Slide 11: Rare Disease Clinical Trials Landscape
For personal use only Rare Disease Clinical Trials Landscape © 2023 Cogstate Ltd. All rights reserved.
Slide 12: What is a Rare Disease? Central Nervous System Rare Disease Examples
What is a Rare Disease? Central Nervous System Rare Disease Examples For personal use only • Rare Disease: Affects less than 1 in 2,000 people • Angelman syndrome • Autism spectrum disorder • Ultra-Rare Disease: • Batten disease Affects less than 1 in 50,000 • CDKL5 deficiency disorder • Down syndro
Slide 13: Why is Rare Disease an Attractive Market?
Why is Rare Disease an Attractive Market? For personal use only • In the 1970s, only 10 orphan drugs were approved. • Between 1983 until 2019, there were 564 orphan drugs approved by the FDA. 11
Slide 14: Focus on Rare: Accelerated Approval for Rare Disease Trials
Focus on Rare For personal use only Accelerated Approval for Rare Disease Trials 12
Slide 15: Focus on Rare: Probability of Success for New Drugs in the US
Focus on Rare For personal use only Probability of Success for New Drugs in the US 25 20 15 10 5 0 All Diseases Chronic High Prevalence Rare Diseases 13
Slide 16: Biotech Strategy: Moving Forward Strategically and Quickly
Biotech Strategy For personal use only Moving Forward Strategically and Quickly Strategy for Biotechs • One compound can target multiple diseases • One pivotal trial; multi-phase trials (combine Ph2/3 into one study) • Serious diseases with no treatment • Biotechs are able to move quickly and with a
Slide 17: Recent Rare Disease Success
Recent Rare Disease Success For personal use only First Approval of Rare Genetic Neurological Disease Trofinitide: Rett Syndrome • Neuren licensed compound to Acadia • Cogstate authored publication with Acadia providing evidence for the clinical meaningfulness of the change seen in the trial 15
Slide 18: Reata: Skyclarys approval and Biogen acquisition
For personal use only 7,000 6,000 1st drug approval in Reata’s 21-year history. Follows rejection in 2020, when FDA asked to see more data. Biogen announce definitive 5,000 agreement to acquire Reata “Skyclarys” is the first drug approved for Market Cap US$m Friedreich’s ataxia. 4,000 5,000 patients
Slide 19: Acadia: Daybue approval for Rett syndrome
Acadia acquires expanded For personal use only rights to trofinetide and Acadia announces FDA approval. global rights to NNZ-2591 2,000 “Daybue” is the first drug approved for Rett 1,800 syndrome. 4,500 patients diagnosed in the USA (expected 1,600 to affect 6,000 – 9,000 & approx. 100,000 Market Ca
Slide 20: Acadia acquires expanded rights to trofinetide and NNZ-2591
Acadia acquires expanded rights to trofinetide and global rights to NNZ-2591 For personal use only Upgraded projections for sales of “Daybue”. 6,000 - 2Q23 $21 - $23m (vs consensus of $2.3m) - 3Q23 $45 - $55m 5,500 Note that guidance also included upgrade for Parkinson’s disease psychosis therapy Nu
Slide 21: Why Rare Disease for Cogstate
Why Rare Disease for Cogstate For personal use only • Vast experience (~40 trials, across 25 indications) • Natural History Studies experience • Development of indication-specific outcome measures • No clear precedence for measures or methodology • Fast fail/ fast approval • Iterate on multiple smal
Slide 22: Why Rare Disease for Cogstate: Natural History Studies
Why Rare Disease for Cogstate For personal use only Expertise in Natural History Studies • Natural course of disease; Inform endpoints; Control condition of a treated group (that is, can replace the need for a placebo group) • Cogstate currenting supporting four natural history studies • Example: Lo
Slide 23: Central Rating and Central Scoring Reduces Error and Site Burden
Why Rare Disease for Cogstate For personal use only Central Rating and Central Scoring: Reduces Error and Decreases Site Burden 37% of administrations had errors that were corrected by Central Rater Use of Central Raters improves accuracy; reduces variance; increases efficiency 21
Slide 24: Recent Publications in Rare Disease
Recent Publications in Rare Disease For personal use only 22
Slide 25: Clinical Trials Capabilities Overview
For personal use only Clinical Trials Capabilities Overview © 2023 Cogstate Ltd. All rights reserved.
Slide 26: CNS Clinical Trial Vendor Ecosystem
1 Contract Research Organisations (CRO) For personal use only 2 Clinical Trial Sites 3 Electronic Data Capture (EDC) 4 Electronic Clinical Outcome Assessment (eCOA) CNS Clinical Trial Vendor Ecosystem 5 Rater Training / Monitoring / Central Rating Organisations 6 Digital Assessments (Performance-bas
Slide 27: Clinical science expertise, technology and operational excellence
Cogstate brings together For personal use only clinical science expertise, innovative technology, and operational excellence to help clinical trial teams understand drug safety and efficacy. 25
Slide 28: Strategic guidance on cognitive measures
For personal use only Strategic guidance on the selection, execution and analysis of cognitive measures. • Access to thousands of cognitive data profiles generated in different study populations, interventions, mechanisms • Endpoint selection, powering decisions, robust statistical analysis and inte
Slide 29: Licensing and translating rating scale instruments
For personal use only Licensing and translating rating scale instruments for a study is a meticulous process that can dramatically impact trial start-up timelines and data quality. • Licensing agreements, translations and linguistic validation, development of master source documents • Migration to e
Slide 30: Electronic clinical outcome assessments (eCOA)
For personal use only Conventional paper and pencil assessments allow missing data and erroneous transcription. Electronic clinical outcome assessments (eCOA) allow for digital capture of study data with pre-set workflows, real-time edit checks and algorithmic flags to improve data quality. • Patien
Slide 31: Rater training
For personal use only Clinical trial sponsors must ensure raters administer scales in the most accurate and standardized way possible. • Prepare raters to administer scales to high standards in the timeliest way possible. • Eliminate unnecessary training based on rater experience and leverage custom
Slide 32: Central monitoring and rating
For personal use only Rater variability and error can be detrimental to a study, adding noise and diminishing signal detection. • Develop and execute strategic, risk-based central monitoring programs to ensure the reliability and validity of clinical measures. • 200+ highly trained clinical and scie
Slide 33: Digital cognitive assessments
For personal use only Increases the sensitivity and specificity of the measurement of human cognition. • Designed and validated to assess memory, verbal learning, attention, psychomotor function, motor function, executive function, vigilance, and emotional recognition • Batteries are customized for
Slide 34: Remote and at-home data collection
For personal use only At-home data collection is increasing in Clinical Trials. Cogstate is expertly positioned to support remote administration of conventional clinical assessments as well as Cogstate proprietary digital assessments. • Self-administered digital assessments • Central rating via tele
Slide 35: Case Example: Phase 3 AD Study
The need to enroll thousands of cognitively normal pre- Case Example: clinical Alzheimer’s participants meant innovative, patient- For personal use only centric trial design was necessary (at-home cognitive Phase 3 AD Study assessment leveraging self-administered digital tests) DETAILS SERVICES Phas
Slide 36: For personal use only
For personal use only