Investor presentation, 27 May 2024

PPaaggee□□11 27 May 2024 IMPROVING THE LIVES OF PEOPLE WITH NEURODEVELOPMENTAL DISABILITIES NNZ-2591 Pitt Hopkins syndrome Phase 2 trial top-line results 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 be correct. Actual results could differ materially from those anticipated. Reasons may include risks associated with drug development and manufacture, risks inherent in the regulatory processes, delays in clinical trials, risks associated with patent protection, future capital needs or other general risks or factors. 2 Pitt Hopkins syndrome has overwhelming unmet medical need Deletion or variation in the TCF4 gene on chromosome 18 TCF4 protein plays a role in the formation, maintenance and function of dendrites and synapses Cause of the syndrome Broad and severe impact on life Intellectual impairment Behavioural issues Sensory processing disorder Sleep disorders Seizures Vision impairment (severe myopia) Language deficits Breathing problems (hyperventilation, apnea, breath-holding) Feeding difficulties GI dysfunction (gastroesophageal reflux and constipation) Motor impairments including hypotonia (low muscle tone) and gross and fine motor delays Walking abnormalities “She was tested earlier for Angelman and Rett Syndrome, but they were of course negative. I had a strange feeling that something was wrong with her already when she was a newborn…I started to see different doctors with her, but they just told me nothing was wrong, until we met a Neurologist who told us that she had Cerebral Palsy and that she would not able to walk, ever…She doesn’t talk but when she was about one year old she was saying a few words that never ever came back...” “Caleb is currently 10 months old and he does not sit or roll yet and is not really interested in toys. He is currently in an early intervention program and is going through physical therapy, and sees a vision teacher and special education teacher…It has not been an easy journey thus far. I still do not how and where I get all my strength from. I know things will only get harder as he gets older but I am ready to accept the challenge and take each day as it comes.” Patients stories Pitt Hopkins Research Foundation 3 Consistent efficacy observed for NNZ-2591 in TCF4 mouse model 4 Hypoactivity Daily living Motor performanceSociability Repetitive behavior Learning & Memory Phase 2 clinical trial results highlights 5 • NNZ-2591 was safe and well tolerated, with no meaningful trends in laboratory values or other safety parameters during treatment • Statistically significant improvement from baseline assessed by both clinicians and caregivers in all 4 efficacy measures specifically designed for Pitt Hopkins syndrome (p<0.05)1 • Clinician and caregiver global efficacy measures showed a level of improvement considered clinically meaningful: • PTHS Clinical Global Impression of Improvement (CGI-I) – mean score of 2.6 with 9 out of 11 children showing improvement assessed by clinicians • PTHS Caregiver Overall Impression of Change (CIC) – mean score of 3.0 with 8 out of 11 children showing improvement assessed by caregivers • Improvements were seen in clinically important aspects of Pitt Hopkins syndrome, including communication, social interaction, cognition and motor abilities 1 Wilcoxon signed rank test Phase 2 Clinical Trial Design 6 Neuren’s Phase 2 trial in children with Pitt Hopkins syndrome 7 Screening /Baseline Week 4 Up-titration to 12 mg/kg BID Week 17 Follow-up Week 19 Week 10 NNZ-2591 treatment Week 0 5 US sites: Rush University, UTSW, UCSF, UAB, Colorado Children’s Hospital 16 subjects, age 3-17 Endpoints • Primary endpoints were safety, tolerability and PK • Secondary endpoints included 14 efficacy measurements, including 4 specifically designed for PTHS • A key objective is selection of the best primary efficacy endpoint or endpoints for a registration study Global • CGI-I • Caregiver Impression of Change (CIC) • CGI-S Symptom Specific • Caregiver Top 3 Concerns First study in pediatric patients, collecting the data needed to design a registration study PTHS specific efficacy measurements 10 other non PTHS specific measures that had been used in other conditions + Participant Disposition 8 16 enrolled 15 1 discontinued due to inability to complete safety monitoring procedures 11 completers 4 discontinued due to TEAE • Discontinuations due to TEAEs, all mild/moderate, all resolved: • 2 due to TEAEs unrelated to drug (COVID- 19 and mild vomiting, diarrhea, lethargy) • 1 due to moderate constipation, self injury, abdominal distention, fatigue • 1 due to mild sleep disorder, constipation Participant demographics 9 Age Mean 9.1yrs Median 9.5yrs 3-12yrs: N = 11 13-17yrs: N = 5 Sex Male, 8, 50% Female, 8, 50% Cognitive level (non-verbal IQ/DQ) <35, n=15, 94% >=35, n=1, 6% CGI-S (at Baseline) Mean (SD) 5.0 (0.69) Low IQ/DQ reflecting severity of the syndrome Completers average DQ: 12 Safety and Tolerability 10 Safety and tolerability summary 11 NNZ-2591 was safe and well tolerated Event N=16 n (%) Event N=16 n (%) Constipation 3 (19) 2 mild, 1 mod Contusion 2 (13) all mild Diarrhea 4 (25) all mild Gastroenteritis-viral 2 (13) 1 mild, 1 mod Vomiting 2 (13) all mild Nasopharyngitis 3 (19) all mild Fatigue 4 (25) 3 mild, 1 mod Cough 2 (13) all mild Somnolence 2 (13) all mild Rhinorrhea 2 (13) all mild Irritability 2 (13) all mild Decreased appetite 2 (13) all mild TEAEs in 2 or more subjects✓ Well tolerated ✓ All Treatment Emergent Adverse Events (TEAE) were mild to moderate, mostly not drug related • 0 Serious TEAE • 4 discontinuations due to TEAEs, all mild/moderate, all resolved ✓ No meaningful trends in laboratory values, electrocardiogram (ECG) or other safety parameters were observed during treatment Efficacy 12 Efficacy endpoints summary 13 • Mean CGI - I of 2.6 and Median of 3.0 with p-value = 0.0039 • Mean CIC of 3.0 and Median of 3.0 with p-value =0.0234 • Statistically significant improvement vs baseline in 4/4 PTHS specific endpoints PTHS Specific Endpoints Completers (MITT) N=11 Including discontinued N=15 CGI-I 0.0039 0.0205 CIC 0.0234 0.0137 CGI-S 0.0313 0.0078 Caregiver Top 3 Concerns 0.0077 0.0024 Efficacy measures and p-values1 (Total/Overall scores) 1 Wilcoxon signed rank test • Changes from baseline for the measures that were not designed for PTHS were not statistically significant Best practice implemented for PTHS-specific CGI-I and CIC measures 14 • Both CGI-I and CIC scores reflect overall improvement from baseline 1 – Very Much Improved 2 – Much Improved 3 – Minimally Improved 4 – No Change 5 – Minimally Worse 6 – Much Worse 7 – Very Much Worse • All clinician raters completed training to calibrate scoring and interpretation of the scoring anchors amongst raters • Training was done at study start up and a follow-up calibration training was done during the study Clinical Global Impression of Improvement (CGI-I) Caregiver Impression of Change (CIC) Scoring Clinician gives an overall score and scores each domain Caregiver gives an overall score and scores each domain Also identifies the one symptom area that has most influenced his or her rating of the child’s overall function Domain Anchors • Language/Communication • Social Interaction • Ambulation/Gross Motor • Fine Motor/Self-Help • GI Issues • Autonomic/Breathing Abnormalities • Challenging Behaviors • Communication • Social interaction • Motor abilities • Self-care skills • GI Problems • Breathing Problems • Behavior • Seizures • Cognitive abilities/ability to learn PTHS CGI-I (clinician) results by subject and by domain 15 Mean CGI-I score of 2.6 with 9 out of 11 children showing improvement Improvement CGI-I Overall Score by subject MITT Population Forest Plot of mean CGI-I Domain Scores MITT Population Subject PTHS CIC (caregiver) results by subject and by domain 16 Mean CIC score of 3.0 with 8 out of 11 children showing improvement Improvement CIC Overall Score by subject MITT Population Forest Plot of mean CIC Domain Scores MITT Population Subject PTHS Clinical Global Impression of Severity (CGI-S) and Caregiver Top 3 Concerns results by domain 17 6 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, self care, behaviour, motor skills) CGI-S Scores Caregiver Top 3 Concerns (Domains and frequency of nomination) Improvement MITT Population Forest Plot of Mean Change from Baseline in Top 3 Concerns Domain Severity MITT Population 0 1 2 3 4 5 6 7 Baseline End of Treatment Improvement Improvement Social Interest/Avoidance (n=1) Self Care (n=7) Fine Motor Skills (n=3) Language/Communication (n=11) Gross Motor Skills (n=4) Gastrointestinal Problems (n=2) Challenging Behaviour (n=4) Testimonials 18 Clinician and caregiver testimonials 19 “Is now able to explore environment… can move towards people to initiate contact and… can seek out whatever … wants to play with.” Caregivers “Stability when walking improved.” “Listen to conversation + follow some discussions, able to understand when we’re talking about…” “Far less hyper and easily able to concentrate better… is able to concentrate and master tasks that … has been working on for years (getting in and out of car independently, catching a ball).” “More intentional movements… been more gentle with almost all interactions.” Clinicians “Increased babbling and jargoning….More inflections with eye contact and consonant sounds rather than just noises.” “Decreased frequency and intensity of smacking and hairpulling.” “Supported stepping increased over last few months…Now taking steps without trainer with parent support.” “Improved expressive communication: 2 additional words, uses AAC device to ask for food. Increase vocalization.” “Less breath holding. More opinionated. More social interest.” “Able to match items/pictures…moved from 4 pictures to 6 pictures.” “Improved motor skills. Better motor coordination getting in car.” “Almost constant babbling and even has said “hi” and “more.”” “More calm and attentive, especially looking at faces and eyes.” “Can seem to hold on to things for longer periods without letting go.” PTHS opportunity 20 Autism US ADDM tracks 440k children with autism spectrum disorder PTHS is historically under-diagnosed, but this is changing 21 Estimated prevalence is 1/34,000 to 1/41,000 males and females1 1 Pitt Hopkins Research Foundation (PHRF) (pitthopkins.org) 2 Brazil, Israel, South Korea, Australia and New Zealand 3 Estimates based on United Nations population data 2022, derived by applying the estimated prevalence range to the populations under 60 years (urban population only for China) 4 Takano et al, “Two percent of patients suspected of having Angelman syndrome have TCF4 mutations” Clin Genet. 2010 Sep;78(3):282-8; Armani et al, “Transcription factor 4 and myocyte enhancer factor 2C mutations are not common causes of Rett syndrome” Am J Med Genet A. 2012;158A(4):713–9 US Europe Japan China Other2 Potential PTHS patients 6,000 – 7,0003 8,000 – 9,0003 1,000 - 2,0003 18,000 – 22,0003 6,000 - 7,0003 Currently 497 in US & Canada, 329 in EMA & UK, 248 in China, 60 in ANZ Opportunity to accelerate diagnosis • Rising awareness • ICD code assigned in 2020 • Enhanced genetic testing technologies • Expanding ADDM network sites Pitt Hopkins Syndrome Census – initiated Q1 20231 Clinical similarities between PTHS, Rett and Angelman syndromes calling for TCF4 screening in suspected Rett or Angelman patients4 1,083 1,188 1,237 1,318 1,391 Q1 2023 Q2 2023 Q3 2023 Q4 2023 Q1 2024 Neuren is leading development of a first approved treatment for PTHS Company Product Development Stage Successful Phase 2 #2 Phase 2 (research institute sponsored, focusing on GI symptoms) #3 Phase 1/2a trial (not yet recruiting) #4 Preclinical • Positive Phase 2 trial • Clinical development in the US under an IND • Orphan Drug designation in US and EU • Eligible for Rare Pediatric Disease Designation Priority Review Voucher program Neuren Program Status Limited products in development Neuren engaging with all stakeholders Leading clinicians 22 NNZ-2591 as multi-indication platform 23 Regulating IGF-1 in the brain 24 Produce essential growth factor Activates PI3K-Akt- mTOR and Ras-MAPK- ERK signaling pathways in neurons, regulating formation of new synapses IGF Binding Protein‐3 Reversible binding regulates bioavailability NNZ-2591 Competitively binds to binding protein, regulating IGF-1 binding1 1 doi: 10.1038/srep04388: Guan et al, 2017: Cyclic glycine -proline ( cGP) regulates IGF-1 homeostasis by altering the binding of IGFBP -3 to IGF-1 IGF-1 IGF-1 receptor on cell surface • NNZ-2591 is a synthetic analog of cyclic glycine proline, a peptide that occurs naturally in the brain, designed to be more stable, orally bioavailable and readily cross the blood-brain barrier • NNZ-2591 can regulate the amount of IGF-1 that is available to activate IGF-1 receptors • The effects of NNZ-2591 are “state-dependent” – correcting impairment, but not impacting normal cells Phase 2 trial results validating multi-indication platform 25 Phelan-McDermid syndrome N=18, 13 weeks Pitt Hopkins syndrome N=11, 13 weeks General safety & tolerability Safe and well tolerated, with no meaningful trends in laboratory values or other safety parameters during treatment Safe and well tolerated, with no meaningful trends in laboratory values or other safety parameters during treatment Serious TEAEs 1 unrelated to drug 0 Mean CGI-I 2.4 (89% shown improvement) 2.6 (82% shown improvement) Mean CIC 2.7 (83% shown improvement) 3.0 (73% shown improvement) # patients had CGI-S improvement of 1 7 (39%) 6 (55%) # syndrome-specific efficacy measures statistically significant1 5/5 4/4 1 Wilcoxon signed rank test Good safety profile supports review of trial protocol for future indications 26 • Positive data from Phelan McDermid syndrome and Pitt Hopkins syndrome Phase 2 trials support review and potential optimisation of existing Phase 2 trial protocol for Prader-Willi syndrome and future indications, subject to FDA agreement • To reduce excessive burden on patients and their families • To enhance competitiveness vs ongoing Prader-Willi syndrome trials in the US • To expedite future development in other indications • Prader-Willi syndrome Phase 2 trial has been paused, pending review of the trial protocol post planned End of Phase 2 Meeting with the FDA in Q3 2024 for Phelan-McDermid syndrome • Pre-clinical studies are ongoing for other indications that could potentially move into Phase 2 with an optimised protocol Multiple indications opportunity for NNZ-2591 27 NNZ-2591 Phelan- McDermid syndrome Pitt Hopkins syndrome Angelman syndrome (Q3 2024) Prader-Willi syndrome Other undisclosed indications Rett syndrome (Acadia) Fragile X syndrome (Acadia) • Positive results from Phelan McDermid syndrome and Pitt Hopkins syndrome Phase 2 trials • Top-line results from Angelman syndrome Phase 2 trial expected in Q3 2024 • End of Phase 2 meeting with FDA for Phelan McDermid syndrome planned Q3 2024 • The mechanism of action of NNZ-2591 is relevant for many other neurodevelopmental synaptopathies • Rett and Fragile X syndromes are licensed to Acadia, with same economics to Neuren as trofinetide; Neuren retains worldwide rights to all other indications CONTACT Jon Pilcher, CEO [email protected] +61 438 422 271 28

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