Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (June 2021)
31 slides from Neuren Pharmaceuticals's investor presentation, Jun 15, 2021
Slide 1: Fighting Neurodevelopmental Disorders
For personal use only FIGHTING NEURODEVELOPMENTAL DISORDERS 15 June 2021 1
Slide 2: Forward Looking Statements
FORWARD LOOKING STATEMENTS For personal use only This presentation contains forward looking statements that involve risks and uncertainties. Although we believe that the expectations reflected in the forward looking statements are reasonable at this time, Neuren can give no assurance that these expe
Slide 3: 2021 Is a Pivotal Year for Neuren
2021 IS A PIVOTAL YEAR FOR NEUREN 2 drugs in clinical development for 6 Orphan Drug indications: For personal use only All serious neurological disorders in children, no approved therapies Orphan drugs have higher probability of approval and significant commercial advantages Results in Q4 20
Slide 4: Product Pipeline
PRODUCT PIPELINE For personal use only Commercial Compound Indication Preclinical Phase 1 Phase 2 Phase 3 Partner Rett Results syndrome1 Q4 2021 (North America) Trofinetide Fragile X syndrome1 (North America) Phelan- Commence McDermid H2 2021 syndrome2 Angelman Commence syndrome2 H2 2021 NNZ-2591 Pi
Slide 5: Commercial Advantages of Orphan Drugs
COMMERCIAL ADVANTAGES OF ORPHAN DRUGS Compared with existing markets that have apparently attractive large $ For personal use only sales in which established products have to be displaced, Orphan Drugs have many commercial advantages: Ability to target a leadership position with little competition
Slide 6: Estimates of Target Patient Populations
ESTIMATES OF TARGET PATIENT POPULATIONS Disorder Gene Published prevalence Potential patients For personal use only mutation estimates US1 Europe1 Asia1, 2 Trofinetide: Rett MECP2 1/10,000 to 1/15,000 females 10,000 13,000 37,000 Fragile X FMR1 1/4,000 to 1/7,000 males 30,000 38,000 112,000 1/12,000
Slide 7: Three Key Drivers of Future Value
THREE KEY DRIVERS OF FUTURE VALUE For personal use only Realise Neuren’s share Implement commercial Confirm efficacy of of trofinetide value in strategy for trofinetide NNZ-2591 in Phase 2 the US through ACADIA’s ex-North America, trials for 3 valuable Phase 3 results and New using US data for indic
Slide 8: Ten Catalysts in 2021 Driving Long Term Value
TEN CATALYSTS IN 2021 DRIVING LONG TERM VALUE For personal use only EU Orphan designations for Phelan-McDermid, Angelman, and Pitt Hopkins Successful Phase 1 trial results for NNZ-2591 Prader-Willi syndrome added to NNZ-2591 pipeline Complete drug substance manufacturing for NNZ-2591 Phase 2
Slide 9: Trofinetide for Rett Syndrome
For personal use only TROFINETIDE FOR RETT SYNDROME 9
Slide 10: Trofinetide Licence Agreement with ACADIA
TROFINETIDE LICENCE AGREEMENT WITH ACADIA For personal use only Partnership commenced in August 2018, providing the necessary funding and capabilities to execute Phase 3 and commercialise trofinetide in the US Redacted agreement is available in ACADIA’s 2018 10K filing Territory North America (N
Slide 11: Rett Syndrome Phase 3 Program
RETT SYNDROME PHASE 3 PROGRAM Double-blind For personal use only Open-label Continued Access LAVENDER LILAC LILAC-2 TROFINETIDE TROFINETIDE TROFINETIDE PLACEBO Baseline Week 12 Week 52 180 females aged 5 to 20 years RSBQ (caregiver) and CGI-I (physician) at 12 weeks are co-primary efficacy endpo
Slide 12: Maximising Probability of Success
MAXIMISING PROBABILITY OF SUCCESS For personal use only The Phase 3 co-primary endpoints were both positive in the Phase 2 trial In the Phase 2 trial clinical improvement continued increasing through to end of treatment - the Phase 3 trial at 12 weeks is twice the duration of the Phase 2 trial
Slide 13: Rett Syndrome Phase 2 - RSBQ and CGI-I
RETT SYNDROME PHASE 2 - RSBQ AND CGI-I For personal use only Clinical improvement measured by RSBQ Clinical improvement measured by CGI-I p=0.042 p=0.029 RSBQ is a caregiver rating, reflecting the CGI-I is a clinician rating of how much the severity of the syndrome. Mean subject’s overall illness ha
Slide 14: NNZ-2591 for Multiple Neurodevelopmental Disorders
For personal use only NNZ-2591 FOR MULTIPLE NEURODEVELOPMENTAL DISORDERS 14
Slide 15: NNZ-2591 Mechanism of Action
NNZ-2591 MECHANISM OF ACTION For personal use only NNZ‐2591 is a synthetic analog of a peptide that occurs naturally in the brain, designed to be more stable, orally bioavailable and readily cross the blood‐brain barrier NNZ-2591 can regulate the amount of IGF-1 that is available to activate IGF-1 r
Slide 16: Foundations in Place for Multiple Indications
FOUNDATIONS IN PLACE FOR MULTIPLE INDICATIONS For personal use only Neuren is leveraging experience from Rett syndrome program across all areas to execute much faster development for NNZ-2591 2022 Phase 2 data in multiple indications Commence Phase 2 trials 2021 INDs approval by FDA in multiple indi
Slide 17: Ideal Attributes Leading into Phase 2
IDEAL ATTRIBUTES LEADING INTO PHASE 2 For personal use only Unique mechanism of action Clear and consistent efficacy in mouse models of each syndrome Biochemical effects in the brain and optimum dose confirmed Demonstrated high oral bioavailability and blood-brain barrier penetration Orpha
Slide 18: Key Features of Phase 2 Trials Commencing H2 2021
KEY FEATURES OF PHASE 2 TRIALS COMMENCING H2 2021 For personal use only Prioritising speed to data – results in H2 2022: Angelman syndrome trial in Australia Phelan-McDermid and Pitt Hopkins trials in US 10-20 patients in each trial Maximising opportunity to demonstrate effects: Pediatri
Slide 19: Phase 1 Clinical Trial Highlights
PHASE 1 CLINICAL TRIAL HIGHLIGHTS For personal use only Twice daily dosing for 7 days was safe and well tolerated at all dose levels tested in healthy volunteers No SAEs, no clinically significant findings in lab or cardiac tests All AEs mild or moderate and resolved during the trial At high
Slide 20: Consistent Efficacy and Dose Response in Phelan-McDermid Model
CONSISTENT EFFICACY AND DOSE RESPONSE IN PHELAN- MCDERMID MODEL PMS is caused by a deletion or other change in the 22q13 region of chromosome 22, For personal use only which includes the SHANK3 gene, or a mutation of the gene. In the shank3 knockout mouse model, wild type mice and knockout mice were
Slide 21: Consistent Efficacy and Dose Response in Phelan-McDermid Model
CONSISTENT EFFICACY AND DOSE RESPONSE IN PHELAN- MCDERMID MODEL For personal use only Motor function Anxiety Repetitive behavior Daily living Daily living 21
Slide 22: Biochemical Effects Confirmed in Shank3 Model
BIOCHEMICAL EFFECTS CONFIRMED IN SHANK3 MODEL For personal use only In biochemical testing, NNZ-2591 was shown to normalise the abnormal length of dendrite spines between brain cells, the excess activated ERK protein (pERK) and the depressed level of IGF-1 in shank3 knockout mice. 2000 4 16000 Dendr
Slide 23: Consistent Efficacy in Angelman Model
CONSISTENT EFFICACY IN ANGELMAN MODEL AS is caused by a deletion or mutation in the ubiquitin protein ligase E3A (UBE3A) gene For personal use only on chromosome 15. In the ube3a knockout mouse model, which resembles features of AS in humans, wild type and knockout mice were each treated with placeb
Slide 24: Consistent Efficacy in Pitt Hopkins Model
CONSISTENT EFFICACY IN PITT HOPKINS MODEL PTHS is caused by the loss of one copy or a mutation of the TCF4 gene on For personal use only chromosome 18. In the tcf4 mutation mouse model, which exhibits features of PTHS in humans, wild type mice and knockout mice were treated with placebo or NNZ-2591
Slide 25: Consistent Efficacy in Prader-Willi Model
CONSISTENT EFFICACY IN PRADER-WILLI MODEL PWS is caused by mutations in the 15q11-q13 region of chromosome 15. In the For personal use only Magel2-null mouse model, which exhibits features of PWS in humans, wild type mice and knockout mice were treated with placebo or NNZ-2591 for 6 weeks. Treatment
Slide 26: Consistent Efficacy in Prader-Willi Model
CONSISTENT EFFICACY IN PRADER-WILLI MODEL For personal use only 26
Slide 27: Appendix
For personal use only APPENDIX 27
Slide 28: Correcting Impaired Signaling in Neurons
CORRECTING IMPAIRED SIGNALING IN NEURONS For personal use only Neurodevelopmental disorders result from different gene mutations, but all feature impaired signaling between neurons, with abnormal length and density of the dendritic spines that connect the neurons via synapses This impaired signa
Slide 29: Stock Information
STOCK INFORMATION (ASX: NEU) For personal use only Current risk-adjusted valuations: MST Access - $3.93, Bell Potter - $3.10 Share register composition (114 million quoted shares – 52 week price range: A$1.00 - A$1.74 top 20 hold 50%) Institutions, 30% Management, 7% Retail, 63% A$22.6 million cash
Slide 30: Management and Board
MANAGEMENT AND BOARD Management team has devised and executed Neuren’s Orphan Drug programs since 2013 For personal use only Extensive international pharmaceutical business experience Successfully developed drugs from pre-clinical through to FDA approval Executed multiple partnering transact
Slide 31: Contact
For personal use only CONTACT Jon Pilcher, CEO jpilcher@neurenpharma.com +61 438 422 271 31
Neuren Pharmaceuticals — Investor Presentation (June 2021) on 6ix