Neuren Pharmaceuticals Limited · ASX:NEU
Neuren Pharmaceuticals — Investor Presentation (December 2023)
33 slides from Neuren Pharmaceuticals's investor presentation, Dec 18, 2023
Slide 1: NNZ-2591 Phelan-McDermid syndrome Phase 2 trial top-line results
NNZ-2591 Phelan-McDermid syndrome Phase 2 trial top-line results 18 December 2023 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 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 expectations will prove to
Slide 3: Phelan-McDermid syndrome has overwhelming unmet medical need
Phelan-McDermid syndrome has overwhelming unmet medical need Cause of the syndrome Broad and severe impact on life From Voice of the Patient Report Intellectual impairment Externally-Led Patient-Focused Drug Deletion or variation in the Behavioural issues Development Meeting 8 Nov 2022 SHANK3 gene o
Slide 4: Consistent efficacy and clear dose response for NNZ-2591 in shank3 model
Consistent efficacy and clear dose response for NNZ-2591 in shank3 model Memory Learning Sociability In biochemical testing, NNZ-2591 was shown to normalize the abnormal length of dendritic spines that form the synapse, the excess activated ERK protein (pERK) and the depressed level of IGF-1 in shan
Slide 5: Phase 2 Clinical Trial Results Highlights
Phase 2 Clinical Trial Results Highlights • NNZ-2591 was safe and well tolerated, with no clinically significant changes in laboratory values or other safety parameters during treatment • Significant improvement was assessed by both clinicians and caregivers across multiple efficacy measures • Impro
Slide 6: Phase 2 Clinical Trial Design
Phase 2 Clinical Trial Design 6
Slide 7: Neuren's Phase 2 trial in children with Phelan-McDermid syndrome
Neuren’s Phase 2 trial in children with Phelan-McDermid syndrome First study in pediatric patients, collecting the data needed to design a registration study 4 US sites: Rush University, Massachusetts General Hospital, Boston Children’s Hospital and Texas Children’s Hospital n subjects: Up to 20 Scr
Slide 8: Participant disposition
Participant disposition 23 participants 18 dosed1 5 screen failures 3 discontinued (none 15 completers related to study drug) 1 All 18 subjects contributed to safety and efficacy data 8
Slide 9: Dosed participant demographics
Dosed participant demographics Age Sex Cognitive level (non-verbal IQ/DQ) Mean 8.6 yrs Missing Female NVIQ 6 (33%) 1 (6%) Median 8.3 yrs <50 13 (72%) Min - Max 4.4 – 13.0 yrs >=50 Male 4 (22%) 12 (67%) 9
Slide 10: Safety and Tolerability
Safety and Tolerability 10
Slide 11: Safety and tolerability summary
Safety and tolerability summary NNZ-2591 was safe and well tolerated ✓ Well tolerated ✓ Most Treatment Emergent Adverse Events (TEAE) were mild to moderate • 1 Serious TEAE (gastroenteritis) not related to study drug, occurred during safety follow-up period after end of treatment • 3 discontinuation
Slide 12: TEAEs in 2 or more subjects
TEAEs in 2 or more subjects Event NNZ-2591 (N=18) Event NNZ-2591 (N=18) n (%) n (%) Constipation 2 (11.1) Somnolence 3 (16.7) Diarrhea 2 (11.1) Pyrexia 3 (16.7) Nausea 2 (11.1) Fatigue 2 (11.1) Vomiting 2 (11.1) Aggression 2 (11.1) COVID-19 3 (16.7) Insomnia 2 (11.1) Nasopharyngitis 2 (11.1) Decreas
Slide 13: Efficacy
Efficacy 13
Slide 14: Efficacy endpoints summary
Efficacy endpoints summary Efficacy measures and p-values1 (Total/Overall scores) Global Behaviour • Statistically significant CGI-I <0.0001 Aberrant Behavior 0.0013 improvement vs baseline in CIC 0.0003 Checklist-2 total 10/14 efficacy CGI-S 0.0156 Behavior Problems Inventory total frequency 0.0326
Slide 15: Best practice implemented for CGI-I and CIC measures in PMS
Best practice implemented for CGI-I and CIC measures in PMS • Both CGI-I and CIC scores Clinical Global Impression of Caregiver Impression of reflect overall improvement Improvement (CGI-I) Change (CIC) from baseline Scoring Clinician gives an overall score Caregiver gives an overall 1 – Very Much I
Slide 16: CGI-I (clinician) results by subject and by domain
CGI-I (clinician) results by subject and by domain Mean CGI-I score of 2.4 with 16 out of 18 children showing improvement Improvement 16
Slide 17: CIC (caregiver) results by subject and by domain
CIC (caregiver) results by subject and by domain Mean CIC score of 2.7 with 15 out of 18 children showing improvement Improvement 17
Slide 18: CGI-S and Caregiver Top 3 Concerns results by domain
Clinical Global Impression of Severity (CGI-S) and Caregiver Top 3 Concerns results by domain 7 subjects improved by one point on the overall CGI-S score after 13 weeks of treatment and improvement was observed in the most common concerns of caregivers (communication, behaviour, social interaction,
Slide 19: Quality of Life Inventory-Disability results by subject and by subscale
Quality of Life Inventory-Disability results by subject and by subscale Improvement Improvement 19
Slide 20: Aberrant Behavior Checklist-2 results by subject and by subscale
Aberrant Behavior Checklist-2 results by subject and by subscale Improvement Improvement Subjects with a score of zero not shown 20
Slide 21: Child Sleep Habits Questionnaire results by subject and by subscale
Child Sleep Habits Questionnaire results by subject and by subscale Improvement Improvement Subjects with a score of zero not shown 21
Slide 22: Gastrointestinal Health Questionnaire results by subject and by subscale
Gastrointestinal Health Questionnaire results by subject and by subscale Improvement Improvement Acknowledgment: GIHQ developed by Kathleen J. Motil, MD, PhD, Baylor College of Medicine 22
Slide 23: ORCA T-Score and MB-CDI Total Vocabulary results by subject
ORCA T-Score and MB-CDI Total Vocabulary results by subject Improvements in communication observed in ORCA T-Score and MB-CDI Total Vocabulary, as well as domains/subscales in CGI-I, CGI-S, CIC and Caregiver Top 3 Concerns ORCA T-Score MB-CDI Total Vocabulary Improvement Improvement 23
Slide 24: Testimonials
Testimonials 24
Slide 25: Clinician and caregiver testimonials
Clinician and caregiver testimonials Clinicians Caregivers “Marked improvement in expressive language and moderate “Using more words while retaining eye contact… Improved improvement in socialization.” pretend play… Initiating eye contact” “Teachers noted improvement in learning new skills.” “Less s
Slide 26: PMS opportunity
PMS opportunity 26
Slide 27: PMS is historically under-diagnosed, but this is changing
PMS is historically under-diagnosed, but this is changing Estimated prevalence is 1% of people with autism - 1/8,000 to 1/15,000 males and females1 US Europe Japan China Other2 Potential PMS patients 17,000 – 32,0003 21,000 – 41,0003 5,000 - 9,0003 51,000 – 95,0003 16,000 - 31,0003 Phelan-McDermid S
Slide 28: Neuren is leading development of a first approved treatment for PMS
Neuren is leading development of a first approved treatment for PMS Phase 2 Program Status Limited products in development Neuren engaging with all stakeholders • Orphan Drug designation in US and EU • Phase 2 clinical Company Product Development Stage development in the US under an IND Phase 2 (suc
Slide 29: NNZ-2591 in development for multiple neurodevelopmental disorders
NNZ-2591 in development for multiple neurodevelopmental disorders Potential patients Disorder Gene mutation Published prevalence estimates US1 Europe1 RoW1, 2 Phelan- SHANK3 1/8,000 to 1/15,000 males and females 24,000 31,000 103,500 McDermid Pitt Hopkins TCF4 1/34,000 to 1/41,000 males and females
Slide 30: Contact
CONTACT Jon Pilcher, CEO jpilcher@neurenpharma.com +61 438 422 271 30
Slide 31: Phelan McDermid syndrome affects all genders and ages
Phelan McDermid syndrome affects all genders and ages % currently diagnosed patients by % currently diagnosed patients by age group1 gender1 36% 34% 29% 48% 17% 52% 15% 16% 13% 12% 9% 5% 5% 5% 4% 1% Male Female < 12 m 1-2 yrs 3-4 yrs 5-10 yrs 11-20 yrs21-30 yrs31-40 yrs41-60 yrs 60+ Patients current
Slide 32: Consistent efficacy in Pitt Hopkins, Angelman and Prader-Willi models
Consistent efficacy in Pitt Hopkins, Angelman and Prader-Willi models Hypoactivity & anxiety Daily living Daily living Sociability Motor Cognition Angelman Hypoactivity Daily living Learning & Memory Sociability Repetitive behavior Motor performance Pitt Hopkins Obesity Circulating IGF-1 Cognition H
Slide 33: Novel mechanisms of action – NNZ-2591
Novel mechanisms of action – NNZ-2591 Reversible binding • NNZ-2591 is a synthetic analog regulates of cyclic glycine proline, a bioavailability IGF peptide that occurs naturally in IGF-1 Binding Produce Protein‐3 the brain, designed to be more essential stable, orally bioavailable and growth factor
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