Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Technical Presentation (August 2024)
26 slides from Neuren Pharmaceuticals's technical presentation, Aug 9, 2024
Slide 1: NNZ-2591 Angelman syndrome Phase 2 trial top-line results
For personal use only NNZ-2591 Angelman syndrome Phase 2 trial top-line results 9 August 2024 IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES Page□1
Slide 2: Forward looking statements
Forward looking statements This presentation contains forward looking statements that involve For personal use only 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: Angelman syndrome (AS) overview
Angelman syndrome (AS) overview Cause of the syndrome Broad and severe impact on life Patients stories For personal use only Deletion or variation in the Intellectual impairment “It has impacted our lives in almost every aspect. maternal allele of the UBE3A Behavioural issues Day-to-day challenges a
Slide 4: Consistent efficacy observed for NNZ-2591 in Ube3am-/p+ mouse model of AS
Consistent efficacy observed for NNZ-2591 in Ube3am-/p+ mouse model of AS Hypoactivity & anxiety Daily living Daily living For personal use only Sociability Motor Cognition 4
Slide 5: Phase 2 clinical trial results highlights
Phase 2 clinical trial results highlights • NNZ-2591 was safe and well tolerated, with no serious adverse events and no meaningful trends in For personal use only laboratory values or other safety parameters during treatment • Clinician and caregiver global efficacy measures specifically designed fo
Slide 6: Phase 2 Clinical Trial Design
For personal use only Phase 2 Clinical Trial Design 6
Slide 7: Neuren's Phase 2 trial in children with Angelman syndrome
Neuren’s Phase 2 trial in children with Angelman syndrome First study in pediatric patients, to inform future development For personal use only 3 AU sites: Austin Health, Screening NNZ-2591 treatment Follow-up Sydney Children’s Hospital, Children’s Health Queensland /Baseline Up-titration to Hospita
Slide 8: Participant Disposition
Participant Disposition 17 participants For personal use only 16 enrolled 1 screen failure (ITT1 Population) Discontinuations: 1 due to inability to comply with study related safety measures; 1 due to protocol deviation (COVID-19 during screening); 1 due to Treatment Emergent Adverse Event (TEAE) (C
Slide 9: Enrolled demographics
Enrolled demographics Low Developmental Quotient (DQ) score reflecting severity of the syndrome For personal use only Age Sex Genotype Cognitive level CGI-S (non-verbal DQ) (at Baseline) Non Deletion, >=20, Female, 7, 44% n=4, 9, 56% Mean 10.1yrs 25% Median 9.5yrs Mean (SD): 4.8 (0.75) 3-12yrs: N =
Slide 10: Safety and Tolerability
For personal use only Safety and Tolerability 10
Slide 11: Safety and tolerability summary
Safety and tolerability summary NNZ-2591 was safe and well tolerated ✓ Well tolerated TEAEs in 2 or more subjects For personal use only ✓ Most TEAEs were mild to Event N=16 Event N=16 moderate, and not drug n (%) n (%) related • 0 Serious TEAE Viral Infection 5 (31) Drooling 2 (13) • 1 discontin
Slide 12: Efficacy
For personal use only Efficacy 12
Slide 13: Best practice implemented for AS-specific CGI-I and CIC measures
Best practice implemented for AS-specific CGI-I and CIC measures • Both CGI-I and CIC scores Clinical Global Impression of Caregiver Impression of reflect overall improvement Improvement (CGI-I) Change (CIC) For personal use only from baseline Scoring Clinician gives an overall score Caregiver gives
Slide 14: AS CGI-I (clinician) results by subject and by domain
AS CGI-I (clinician) results by subject and by domain Mean CGI-I score of 3.0 (p=0.0010) with 11 out of 13 children showing improvement For personal use only CGI-I Overall Score by subject Forest Plot of mean CGI-I Domain Scores mITT Population mITT Population Subject Improvement 14
Slide 15: AS CIC (caregiver) results by subject and by domain
AS CIC (caregiver) results by subject and by domain Mean CIC score of 3.2 (p=0.0273) with 8 out of 121 children showing improvement For personal use only CIC Overall Score by subject Forest Plot of mean CIC Domain Scores mITT Population mITT Population Subject Improvement 1 Score for one subject ina
Slide 16: All children in 3 to 12 years age group improved
All children in 3 to 12 years age group improved Mean CGI-I of 2.8 (p=0.0078) and mean CIC of 2.6 (p=0.0078) with all children showing improvements For personal use only CGI-I Overall Score by subject CIC Overall Score by subject 3-12 year old age group 3-12 year old age group mITT Population mITT P
Slide 17: AS Clinical Global Impression of Severity (CGI-S) results
AS Clinical Global Impression of Severity (CGI-S) 4 subjects improved by one point on the overall CGI-S score after 13 weeks of treatment For personal use only Forest Plot of Change from Baseline in CGI-S Domain Scores mITT Population Improvement 17
Slide 18: Bayley-4 scales (raw scores)
Bayley-4 scales (raw scores) Improvements observed despite treatment period of only 13 weeks For personal use only Cognitive Score by subject Fine Motor Score by subject Gross Motor Score by subject mITT Population mITT Population mITT Population Improvement Improvement Improvement 54% shown improve
Slide 19: Clinician and caregiver testimonials
Clinician and caregiver testimonials Clinicians Caregivers “Sleeps longer hours greater than 8 per night. Night “…is more settled and focus on what they are doing …also awakenings only to be covered but back to sleep on their much more calmer than before.” For personal use only own. Wakes up much le
Slide 20: AS opportunity
For personal use only AS opportunity 20
Slide 21: AS has a well established global clinical environment
AS has a well established global clinical environment Estimated prevalence is 1/10,000 to 1/20,000 males and females1 For personal use only US Europe Japan China Other2 Potential AS patients 12,000 – 25,0003 16,000 – 32,0003 3,000 - 7,0003 38,000 – 76,0003 12,000 - 25,0003 Natural history study sinc
Slide 22: Neuren has one of the leading programs for AS
Neuren has one of the leading programs for AS Neuren Program Status Leading products in development Neuren engaging with all For personal use only stakeholders • Orphan Drug designation in US and Delivery Product EU Company Modalities Mechanism Development Stage • Phase 2 clinical Phase 3 to Spinal
Slide 23: NNZ-2591 as a multi-indication platform
For personal use only NNZ-2591 as a multi-indication platform 23
Slide 24: Phase 2 trial results validating multi-indication platform
Phase 2 trial results validating multi-indication platform Phelan-McDermid syndrome Pitt Hopkins syndrome Angelman syndrome N=18, 13 weeks N=11, 13 weeks N=13, 13 weeks For personal use only General safety & tolerability Safe and well tolerated, with no Safe and well tolerated, with no Safe and well
Slide 25: Multiple indications opportunity for NNZ-2591
Multiple indications opportunity for NNZ-2591 For personal use only • Positive results from Phelan McDermid syndrome, Pitt Hopkins Prader-Willi syndrome and Angelman syndrome syndrome Phase 2 trials • End of Phase 2 meeting with FDA Other Pitt Hopkins for Phelan McDermid syndrome undisclosed syndrom
Slide 26: Contact
For personal use only CONTACT [email protected] 26
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