Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Shareholder Meeting (June 2021)
24 slides from Neuren Pharmaceuticals's shareholder meeting presentation, Jun 22, 2021
Slide 1: Chairman's Address at 2021 Annual Meeting of Shareholders
Neuren (NEU) - ASX Announcement 22 June 2021 Chairman’s Address at 2021 Annual Meeting of Shareholders For personal use only There is no doubt that Neuren has never been in a better position to deliver substantial value for our shareholders. Our lead drug trofinetide is in the last stages of enrolme
Slide 2: Chairman's Address (continued): NNZ-2591
Turning now to our second product NNZ-2591, our confidence in the future of this drug continues to grow. At this stage we have identified 4 neurodevelopmental disorders as the first indications for NNZ-2591, namely Angelman syndrome, Phelan-McDermid syndrome, Pitt Hopkins syndrome and For personal u
Slide 3: About Neuren
About Neuren Neuren is developing two new drug therapies to treat multiple serious neurological disorders that emerge in early childhood, none of which have any approved medicines. For personal use only The lead drug compound, trofinetide, is currently in a Phase 3 clinical trial for Rett syndrome w
Slide 4: Annual Shareholders' Meeting June 2021
For personal use only ANNUAL SHAREHOLDERS’ MEETING 22 June 2021 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: Fighting Neurodevelopmental Disorders
For personal use only FIGHTING NEURODEVELOPMENTAL DISORDERS 22 June 2021 2
Slide 7: 2021 Is a Pivotal Year for Neuren
2021 IS A PIVOTAL YEAR FOR NEUREN For personal use only 2 drugs in clinical development for 6 Orphan Drug indications: 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 8: 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 9: Estimates of Target Patient Populations
ESTIMATES OF TARGET PATIENT POPULATIONS For personal use only Disorder Gene Published prevalence Potential patients 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 10: 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 11: 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 12: Trofinetide for Rett Syndrome
For personal use only TROFINETIDE FOR RETT SYNDROME 8
Slide 13: Rett Syndrome Phase Program
RETT SYNDROME PHASE 3 PROGRAM For personal use only Double-blind 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 Multiple Neurodevelopmental Disorders
For personal use only NNZ-2591 FOR MULTIPLE NEURODEVELOPMENTAL DISORDERS 12
Slide 17: 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 18: 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 19: 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 20: 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 21: Contact
For personal use only CONTACT Jon Pilcher, CEO [email protected] +61 438 422 271 17
Slide 22: Resolutions Proxy Votes Received: Resolution 1
RESOLUTIONS PROXY VOTES RECEIVED For personal use only Total % Lodged For Lodged Open Lodged Against Available issued Votes capital Resolution 1 Votes % Votes % Votes % RE-ELECTION OF PATRICK DAVIES AS A DIRECTOR 34,109,026 99.06 321,893 0.93 2,499 0.01 34,433,418 29.28 1
Slide 23: Resolutions Proxy Votes Received: Resolution 2
RESOLUTIONS PROXY VOTES RECEIVED For personal use only Total % Lodged For Lodged Open Lodged Against Available issued Votes capital Resolution 2 Votes % Votes % Votes % AUTHORISATION TO FIX AUDITOR FEES AND EXPENSES 34,153,329 98.59 321,893 0.93 167,002 0.48 34,642,224 29.46 2
Slide 24: Resolutions Proxy Votes Received: Resolution 3
RESOLUTIONS PROXY VOTES RECEIVED For personal use only Total % Lodged For Lodged Open Lodged Against Available issued Votes capital Resolution 3 Votes % Votes % Votes % APPROVAL OF ADDITIONAL 10% PLACEMENT CAPACITY UNDER LISTING RULE 7.1A 33,949,238 98.00 347,143 1.00 344,943 1.00 34,641,324 29.45 3
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