Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Shareholder Meeting (June 2020)
22 slides from Neuren Pharmaceuticals's shareholder meeting presentation, Jun 29, 2020
Slide 1: Chairman's Address at 2020 Annual Meeting of Shareholders
Neuren (NEU) - ASX Announcement 29 June 2020 Chairman’s Address at 2020 Annual Meeting of Shareholders For personal use only Since the last Annual Shareholders’ Meeting Neuren has made important progress in our ambition to achieve approval of novel drug therapies for a range of serious neurodevelopm
Slide 2: Chairman's Address (continued) and About Neuren
approval is achieved. Outside North America, we chose to defer partnering in order to capture substantially greater value by selecting the best commercial outcome after the US Phase 3 trial results. For personal use only There has been much progress for NNZ-2591 with the achievement of three Orphan
Slide 3: Contact, ASX information and forward-looking statements
Contact: Jon Pilcher, CEO For personal use only jpilcher@neurenpharma.com; +61 438 422 271 ASX Listing Rules information This announcement was authorized to be given to the ASX by the board of directors of Neuren Pharmaceuticals Limited, Suite 201, 697 Burke Road, Camberwell, VIC 3124 Forward-lookin
Slide 4: Annual Shareholders Meeting June 2020
For personal use only ANNUAL SHAREHOLDERS MEETING 29 June 2020 1
Slide 5: 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 6: Neuren's Current Strong Position
NEUREN’S CURRENT STRONG POSITION For personal use only Neuren is developing 2 drugs to treat 5 debilitating childhood disorders, which have no approved therapies Drugs based on naturally occurring molecules target the underlying impairment in signalling between brain cells; no royalties payable
Slide 7: Stock Information Post-Raising
STOCK INFORMATION POST-RAISING (ASX: NEU) For personal use only Pro-forma cash post-raising: circa A$30 million Estimated share register composition (115 million quoted shares) 52 week price range: A$0.97 - A$3.04 Institutions, 33% Management, 8% Retail, 59% 4
Slide 8: 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 9: Key Value Driver: Trofinetide US Value
KEY VALUE DRIVER 1 For personal use only Achieved last year: Successful manufacturing scale-up Phase 3 trial commenced on time Rare Pediatric Disease designation Realise Neuren’s share of trofinetide value in the US through ACADIA’s Phase 3 results and New Drug Application Key future milestone
Slide 10: Key Value Driver: Trofinetide Ex-North America
KEY VALUE DRIVER 2 For personal use only Achieved last year: Deferred partnering to capture more value post-Phase 3 New patent granted in Israel Implement commercial strategy for trofinetide ex-North America, using US data for registration Key future milestones: EU national agency meetings H2
Slide 11: Key Value Driver: NNZ-2591 Phase 2
KEY VALUE DRIVER 3 For personal use only Achieved last year: 3 Orphan drug designations from FDA Compelling results in 3 animal models Successful manufacturing development Non-clinical toxicology studies Commenced Phase 1 trial New patents granted in US, Europe and Confirm efficacy of Ja
Slide 12: Trofinetide for Rett Syndrome
For personal use only TROFINETIDE FOR RETT SYNDROME 9
Slide 13: Rett Syndrome Phase 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 Lavender results expected in 2021, potential US marketing approval in 2022 180 females with Rett syndrome aged
Slide 14: 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 15: NNZ-2591 for Phelan-McDermid, Angelman and Pitt Hopkins Syndromes
For personal use only NNZ-2591 FOR PHELAN-MCDERMID, ANGELMAN AND PITT HOPKINS SYNDROMES 12
Slide 16: Advancing NNZ-2591 for Indications
ADVANCING NNZ-2591 FOR 3 INDICATIONS For personal use only FDA granted Orphan Drug designation for Phelan-McDermid, Angelman and Pitt Hopkins Compelling data package assembled in preparation for clinical trials: Clear and consistent efficacy in mouse models of each syndrome (SHANK3, UBE3A, TCF
Slide 17: 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 18: 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 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 17
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: Contact
For personal use only CONTACT Jon Pilcher, CEO jpilcher@neurenpharma.com +61 438 422 271 19
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