Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (August 2020)
32 slides from Neuren Pharmaceuticals's investor presentation, Aug 18, 2020
Slide 1: Corporate Presentation
For personal use only CORPORATE PRESENTATION 18 August 2020 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: Neuren's Current Strong Position
NEUREN’S CURRENT STRONG POSITION For personal use only Developing 2 drugs to treat 5 debilitating childhood disorders, which have no approved therapies – lead program in Phase 3 Orphan Drug provides regulatory incentives and commercial protection Trofinetide partnered with ACADIA (NASDAQ:ACAD)
Slide 4: The Future - Three Key Value Drivers
THE FUTURE - THREE KEY VALUE DRIVERS 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 in
Slide 5: Stock Information
STOCK INFORMATION (ASX: NEU) For personal use only Current risk-adjusted valuations: Bell Potter - $3.05, MST Access - $3.32 Share register composition (114 million quoted shares – 52 week price range: A$0.97 - A$3.04 top 20 hold 51%) Institutions, 33% Management, 7% Retail, 60% A$28 million cash at
Slide 6: Key Messages
KEY MESSAGES For personal use only ACADIA’s Q2 earnings call on 5 August re-confirmed trofinetide Phase 3 trial continuing and results expected in H2 2021 $28 million cash following successful capital raise supported by international institutions Funding now in place to generate Phase 2 data f
Slide 7: Product Pipeline
PRODUCT PIPELINE For personal use only Compound Indication Preclinical / Phase 1 Phase 2 Phase 3 Commercial Partner Rett syndrome1 (North America) Trofinetide Fragile X syndrome1 (North America) Phelan- McDermid syndrome2 Angelman NNZ-2591 syndrome2 Pitt Hopkins syndrome2 1 Orphan Drug designation i
Slide 8: 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 9: Estimates of Patient Populations Aged <60
ESTIMATES OF PATIENT POPULATIONS AGED <60 For personal use only Disorder Gene Published Potential Potential mutation prevalence estimates patients US1 patients EU/JP1 Rett MECP2 1/10,000 to 1/15,000 10,000 16,000 females Fragile X FMR1 1/4,000 to 1/7,000 30,000 48,000 males 1/12,000 to 1/22,000 fema
Slide 10: 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 11: Trofinetide for Rett Syndrome
For personal use only TROFINETIDE FOR RETT SYNDROME 11
Slide 12: Rett Syndrome Overview
RETT SYNDROME OVERVIEW For personal use only Rett syndrome is a debilitating and life-threatening neurological disorder with no approved medicines It is caused by a non-inherited mutation on the X chromosome. Estimated incidence of 1 in 10,000 – 15,000 live female births After normal developme
Slide 13: 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 14: 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 15: 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 16: NNZ-2591 for Phelan-McDermid, Angelman and Pitt Hopkins Syndromes
For personal use only NNZ-2591 FOR PHELAN-MCDERMID, ANGELMAN AND PITT HOPKINS SYNDROMES 16
Slide 17: Advancing to Phase 2 in 3 Indications
ADVANCING TO PHASE 2 IN 3 INDICATIONS For personal use only Clear and consistent efficacy in mouse models of Phelan-McDermid, Angelman and Pitt Hopkins syndromes Biochemical effects in the brain and dose response confirmed in Phelan- McDermid model Following review of data, FDA granted Orphan
Slide 18: Clinical Profile of PMS, AS and PTHS
CLINICAL PROFILE OF PMS, AS and PTHS For personal use only Characteristic Phelan-McDermid Angelman Pitt Hopkins (PMS) (AS) (PTHS) Intellectual disability √ √ √ Anxiety and hyperactivity √ √ Speech impairment √ √ √ Motor and balance problems √ √ Sleep disturbance √ √ √ Seizures √ √ √ Breathing irregu
Slide 19: 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 20: 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 20
Slide 21: 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 22: NNZ-2591 Efficacy in AS Mouse Model (UBE3A)
NNZ-2591 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
Slide 23: NNZ-2591 Efficacy in PTHS Mouse Model (TCF4)
NNZ-2591 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-25
Slide 24: Contact
For personal use only CONTACT Authorised by: Jon Pilcher, CEO jpilcher@neurenpharma.com +61 438 422 271 Neuren Pharmaceuticals Limited Suite 201, 697 Burke Road Camberwell, VIC 3124 24
Slide 25: Appendix
For personal use only APPENDIX 25
Slide 26: Neuren's Business Summary
NEUREN’S BUSINESS SUMMARY For personal use only Significant commercial opportunities with no approved drug therapies Strong support from leading physicians and patient advocacy groups Using regulatory incentives – Orphan Drug, Fast Track, Priority Review Protected by Orphan Drug exclusivity
Slide 27: Management Team
MANAGEMENT TEAM For personal use only Team responsible for devising and executing Neuren’s Orphan Drug programs since 2013 Extensive international pharmaceutical business experience Successfully developed drugs from pre-clinical through to FDA approval Executed multiple partnering transactio
Slide 28: Market Exclusivity and Patents
MARKET EXCLUSIVITY AND PATENTS For personal use only Regulators provide exclusivity periods for products with orphan drug designation: US – 7 years from marketing authorization, plus 6 months if approved for pediatric use European Union – 10 years from marketing authorization, plus 2 years if
Slide 29: 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 30: Rett Syndrome Phase 2 Highlights
RETT SYNDROME PHASE 2 HIGHLIGHTS For personal use only High dose of trofinetide (n=27) achieved statistically significant and clinically meaningful efficacy compared with placebo (n=24) for each of the two Phase 3 trial primary endpoints Published in Neurology® Open access: https://n.neurology
Slide 31: Rett Syndrome Phase 2 Trial - RSBQ Items
RETT SYNDROME PHASE 2 TRIAL - RSBQ ITEMS For personal use only RSBQ items with largest Cohen’s D effect size in favour of active 31
Slide 32: Rett Syndrome Phase 2 Trial - RSBQ Subscales
RETT SYNDROME PHASE 2 TRIAL - RSBQ SUBSCALES For personal use only Cohen’s D effect size 32
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