Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (February 2021)
30 slides from Neuren Pharmaceuticals's investor presentation, Feb 24, 2021
Slide 1: Corporate Presentation
For personal use only CORPORATE PRESENTATION 24 February 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: Pivotal Year for Neuren in 2021
PIVOTAL YEAR FOR NEUREN IN 2021 5 Orphan Drug indications in clinical development: For personal use only Rett syndrome in Phase 3 funded by US partner, results in H2 2021 Phase 2 trials in 3 disorders commencing in 2021 Serious neurological disorders in children, no approved therapies Orph
Slide 4: Product Pipeline
PRODUCT PIPELINE For personal use only Commercial Compound Indication Preclinical Phase 1 Phase 2 Phase 3 Partner Rett Results syndrome1 H2 2021 (North America) Trofinetide Fragile X syndrome1 (North America) Phelan- Commence McDermid 2021 syndrome2 Angelman Commence syndrome2 2021 NNZ-2591 Pitt Hop
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: 2021 Milestones
2021 MILESTONES 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 Submit NNZ-2591 INDs to FDA Com
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 NEURODVELOPMENTAL DISORDERS 14
Slide 15: NNZ-2591 Foundations in Place to Realise Value
NNZ-2591 FOUNDATIONS IN PLACE TO REALISE VALUE For personal use only 2022 Phase 2 data in multiple indications Phase 2 trials in 2021 INDs approval by FDA multiple indications Identify Initiate GMP Non-clinical Scale-up Phase 1 3 EU Orphan 2020 optimum manufacturing manufacturing safety trial design
Slide 16: Commencing Phase 2 in Multiple Indications
COMMENCING PHASE 2 IN MULTIPLE INDICATIONS For personal use only Neuren is leveraging experience from Rett and Fragile X programs across CMC, non-clinical, clinical and regulatory to execute rapid development Clear and consistent efficacy in mouse models of Phelan-McDermid, Angelman, Pitt Hopkins
Slide 17: 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 18: Efficacy and Optimum Dose in PMS Model (SHANK3)
EFFICACY AND OPTIMUM DOSE IN PMS MODEL (SHANK3) 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 treated with pl
Slide 19: Efficacy and Optimum Dose in PMS Model (SHANK3)
EFFICACY AND OPTIMUM DOSE IN PMS MODEL (SHANK3) For personal use only Motor function Anxiety Repetitive behavior Daily living Daily living 19
Slide 20: Biochemical Effects Confirmed in SHANK3 Model
BIOCHEMICAL EFFECTS CONFIRMED IN SHANK3 MODEL For personal use only In additional 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
Slide 21: Efficacy in AS Mouse Model (UBE3A)
EFFICACY IN AS MOUSE MODEL (UBE3A) 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 placebo o
Slide 22: Efficacy in PTHS Mouse Model (TCF4)
EFFICACY IN PTHS MOUSE MODEL (TCF4) 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 for 6
Slide 23: Efficacy in PWS Mouse Model (Magel2-null)
EFFICACY IN PWS MOUSE MODEL (Magel2-null) 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 24: Efficacy in PWS Mouse Model (Magel2-null)
EFFICACY IN PWS MOUSE MODEL (Magel2-null) For personal use only 24
Slide 25: Appendix
For personal use only APPENDIX 25
Slide 26: 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 27: Normal Biology of IGF-1 in the Brain
NORMAL BIOLOGY OF IGF‐1 IN THE BRAIN For personal use only Neuren’s trofinetide and NNZ‐2591 are synthetic analogues of GPE and cGP which occur naturally in the brain: • Replicate the activity of the natural molecules • More stable and orally bioavailable • Readily cross the blood‐brain barrier Prod
Slide 28: Stock Information
STOCK INFORMATION (ASX: NEU) For personal use only Current risk-adjusted valuations: Bell Potter - $3.10, MST Access - $3.93 Share register composition (114 million quoted shares – 52 week price range: A$0.97 - A$2.82 top 20 hold 50%) Institutions, 30% Management, 7% Retail, 63% A$24 million cash at
Slide 29: 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 30: Contact
For personal use only CONTACT Jon Pilcher, CEO [email protected] +61 438 422 271 30
Neuren Pharmaceuticals — Investor Presentation (February 2021) on 6ix