Announcement PDF Improving the lives of people with neurodevelopmental disabilities Neuren Pharmaceuticals Limited ANNUAL REPORT 2024 Neuren Pharmaceuticals is developing new therapies for debilitating neurodevelopmental disorders that emerge in early childhood and are characterised by impaired connections and signalling between brain cells. Incorporated in New Zealand and based in Melbourne, Australia, Neuren is listed on the ASX under the code NEU. CONTENTS 1 Neuren’s value proposition 2 Chair and CEO message 4 Operating Review 14 Board 15 Executive Team 16 Environmental, Social and Governance (ESG) 23 Directors’ Responsibilities Statement 24 Consolidated statement of Profit or Loss and Other Comprehensive Income 25 Consolidated Statement of Financial Position 26 Consolidated Statement of Changes in Equity 27 Consolidated Statement of Cash Flows 28 Notes to the Consolidated Financial Statements 48 Independent Auditor’s Report 51 Additional Information Neuren Pharmaceuticals Limited Annual Repor t 2024 arge potential upside for shareholders is enabled by financial strength N E U R E N ’ S V A LU E P R O P O S I T I O N Maximise value of NNZ-2591 as a multiple indication platform  Positive Phase 2 results for Phelan-McDermid syndrome  Positive Phase 2 results for Pitt Hopkins syndrome Value  Positive Phase 2 results for Angelman syndrome Long-term income growth from Acadia’s successful global commercialization of A$359 million pro-forma cash1 222 million cash and short-term investments at 31 December 2024, adjusted to include receipt in Q1 2025 of PRV sale proceeds, sales milestone and Q4 2024 royalty and payment in Q1 2025 of Q4 2024 tax THE 2024 NUMBERS – A RECORD YEAR OF INCOME FOR SHAREHOLDERS The 2024 numbers – a record year of income for shareholders Comprehensive income (CI) A$166m Comprehensive income (CI) A$166m CI margin A$142m NPAT Total comprehensive income (A$m) 73% 73% NPAT 157.1 166.2 FX gain A$24m A$142m R&D investment in NNZ-2591 Corporate & admin costs A$5m CI margin A$33m FX gain A$24m DAYBUE revenue 23 & 24 A$445m Pro-forma cash A$359m Corporate & admin costs 0.2 2022 2023 2024 A$5m R&D investment in NNZ-2591 A$33m 2 DAYBUETM revenue 23 & 24 A$445m Pro-forma cash1 A$359m 1 A$222 million cash and short-term investments at 31 December 2024, adjusted to include receipt in Q1 2025 of PRV sale proceeds, sales milestone and Q4 2024 royalty and payment in Q1 2025 of Q4 2024 tax. This is a non-IFRS measure and is relevant to illustrate the expected cash and short-term investments position, due to the large proportion of 2024 revenue being earned in Q4 2024 and received in Q1 2025. 1 1 Neuren Pharmaceuticals Limited Annual Repor t 2024 CHAIR AND CEO MESSAGE P AT R I C K D A V I E S & J O N P I L C H E R For NNZ-2591, we were excited that during 2024 the positive Phase 2 trial results for Pitt Hopkins syndrome and Angelman syndrome were consistent with the Phelan-McDermid syndrome results, validating our thesis that NNZ-2591 can potentially have a broad impact on neurodevelopmental conditions. We were very pleased with the outcome of our End of Phase 2 Meeting with the FDA for Phelan-McDermid syndrome, enabling us to move straight to Phase 3 with a similar program to the successful DAYBUE program in Rett syndrome. Efficacy endpoints are always the most complex issue when you are leading the way as a first treatment and there is no precedent to follow. We went through a similar journey with FDA to align on efficacy endpoints for Rett syndrome. Overall, we believe that the success of DAYBUE de-risks the NNZ-2591 programs, given the similarities in clinical profile, scientific rationale, trial design and endpoints. Building on the Rett syndrome experience, we are eager to embark on the first ever Phase 3 trial in Phelan-McDermid syndrome, aiming to provide a first treatment option to that community. 2024 was another highly successful year for the Neuren business, with record financial performance, record sales of DAYBUE™ and two more successful Phase 2 trial results for NNZ-2591. For the second year in succession Neuren received the Australian Growth Company of the Year Award for Health and Life Sciences. Across 2023 and 2024 our income from Acadia for DAYBUE was A$445 million at 100% pre-tax margin, which led to pro-forma cash of A$359 million at 31 December 2024. That cash has come from income and not from capital raising. This puts us in an enviable position to pursue the realisation of the value of NNZ-2591, which can potentially be many multiples of DAYBUE. Relative to our peers, we believe this provides a unique proposition for investors – a valuation backstop as well as the potential to add much more value using existing funds. We are excited to also now be targeting hypoxic-ischemic encephalopathy (HIE), a devastating type of brain injury in newborns. We believe that NNZ-2591 can potentially provide a highly differentiated form of treatment continuing beyond acute treatment in the neonatal intensive care unit to target both the acute effects and the long term neurodevelopmental impairments resulting from HIE. Despite this progress and “buy” recommendations from all nine analysts that cover Neuren, the share price has fallen significantly from its peak, which is frustrating and disappointing. Neuren was the best performing ASX 200 stock in 2023, a year in which DAYBUE was approved and launched, together with a first positive Phase 2 trial result for NNZ-2591. In 2024 we were impacted negatively by momentum trading, initially triggered by a switch to negative sentiment about DAYBUE sales. In the early launch period with limited experience there was heavy focus in the investment community on quarterly sales rather than the long-term opportunity for Neuren. We continue to maintain that the launch has been very successful and better than comparators, with net sales reaching US$348 million in the first full year of sales. Acadia has committed substantial additional resources to expand DAYBUE in the USA and has forecast continued growth in 2025. More than 60% of patients currently on therapy have now been treated for more than 12 months. This provides a very stable base which means that sales have become much more predictable. There are still 70% of the expanding pool of diagnosed patients in the US who have not yet tried treatment. This, together with the coming expansion into Canada, Europe, Japan and potentially other countries, provides substantial upside and a long-term growth opportunity for Neuren. Acadia has done an impressive job so far and with new commercial leadership and initiatives in place is now in an even better position to maximise that global opportunity. Most importantly, the stories from families of benefits that children and adults with Rett syndrome are experiencing on DAYBUE are heartening and extremely motivating for the Neuren team. During the past year we have been transitioning Neuren’s capabilities from Phase 2 development to Phase 3 development. That has required some changes in the skills and location of roles as well as a major focus on uplifting the sophistication of Neuren’s quality systems. We thank the Neuren team for their diligence through this transition and for all their many achievements during the year. We are also grateful to all our supportive shareholders and can assure you that we will continue to evaluate all options to maximise shareholder value. Last but not least, we thank the patient communities across the indications we are pursuing for their support, determination and courage, which is so critical to achieving the outcomes we are all striving for. Patrick Davies Chair 2 Jon Pilcher CEO Neuren Pharmaceuticals Limited Annual Repor t 2024 CHAIR AND CEO MESSAGE CONTINUED We are passionate about making a difference to the lives of patients and their families We aim to earn the respect of everyone we deal with NEUREN’S VALUES We are determined and creative to break through barriers We harness the power of collaboration and different perspectives We recognise the importance of all stakeholders and endeavour to use financial resources efficiently We apply a quality mindset to everything we do Jon Pilcher CEO Patrick Davies Chair 3 Neuren Pharmaceuticals Limited Annual Repor t 2024 O P E R AT I N G R E V I E W Treating neurodevelopmental disorders Rett Fragile X PhelanMcDermid Angelman Pitt Hopkins Prader-Willi MECP2 FMR1 SHANK3 UBE3A TCF4 15q11-q13 Impaired communication between neurons, abnormal formation/pruning of dendrites & chronic inflammation Neuren’s drugs target the critical role of IGF-1 in this upstream process, using analogs of peptides that can be taken orally as liquids Severe impact on nearly every aspect of life walking and balance issues anxiety and hyperactivity seizures impaired communication intellectual disability impaired social interaction impaired hand use sleep disturbance gastrointestinal problems NEUREN’S GROUND -BREAKING THER APIES IGF-1 and its metabolites play a significant role in regulating these changes. In the mature brain, these molecules play an important role in responding to disease, stress and injury. Neuren focuses on developing treatments for debilitating neurodevelopmental disorders that emerge in early childhood and stem from problems in brain development which lead to a wide range of serious issues affecting nearly every aspect of life. These neurodevelopmental disorders have severe life-long impact on the patients and their families. Each neurodevelopmental disorder is caused by a different genetic mutation, but in many cases, they share similar symptoms and the common characteristic of impaired connections and signalling between brain cells. Trofinetide and NNZ-2591 mimic the function of the natural molecules in the brain, however each drug is designed to have a longer half-life in circulation, be suitable for use as an oral medication, more readily cross the blood brain barrier and have better stability for longer and easier storage and shipping. Whereas many drugs typically exert a specific effect on a specific target related to one symptom, trofinetide and NNZ-2591 exert diverse effects which can help to control or normalise abnormal biological processes in the brain. This means that the target is to have a broad impact on the disorder rather than aiming to treat one symptom. An important feature is that both drugs can be administered orally in a patient-friendly liquid dose. Neuren currently has two novel patented drugs, trofinetide and NNZ-2591, which potentially have broad utility in the treatment of neurological disorders. Both drugs are synthetic analogues of important molecules that occur naturally in the brain and are involved in the biology of IGF-1, a growth factor stimulated by growth hormone. In the central nervous system, IGF-1 is produced by both of the major types of brain cells – neurons and glia. IGF-1 in the brain is critical both for normal development and to maintain or restore the biological balance required for normal functioning. During development, the brain and the cells that comprise it change rapidly and in complex ways. A critical feature of Neuren’s work to develop therapies for each of these disorders is close collaboration with the leading specialist physicians and with the well-organised patient advocacy organisations. 4 Neuren Pharmaceuticals Limited Annual Repor t 2024 O P E R AT I N G R E V I E W CONTINUED THE IMPORTANCE OF ORPHAN DRUG DESIGNATION TROFINETIDE FOR RETT SYNDROME Growing, sustainable income to Neuren from DAYBUE™ (trofinetide) in the United States The US Food and Drug Administration (FDA) and European Medicines Agency (EMA) have both granted Orphan Drug designation for trofinetide in Rett syndrome and Fragile X syndrome and for NNZ-2591 in each of Phelan-McDermid, Angelman and Pitt Hopkins syndromes. The FDA has also granted Orphan Drug designation for Prader-Willi syndrome. DAYBUE US Net Sales (US$m) + 9-16% Orphan Drug designation is a special status that the regulators may grant to a drug to treat a rare disease or condition. Amongst other incentives, Orphan Drug designation qualifies the sponsor of the drug for exclusivity periods during which the regulators will not approve a generic competitor product. These marketing exclusivity periods are extremely valuable for the commercialisation of Orphan Drugs. They provide additional protection, along with patents, against generic competitors and potentially can continue to provide protection after patent expiry. The exclusivity periods after marketing authorisation of products approved for pediatric use are 7.5 years in the United States and 12 years in the EMA region. Japan, South Korea and Taiwan also have Orphan Drug programs. + 97% 405 348 380 177 CY2023 (Apr - Dec) DAYBUE US Net Sales (US$m) CY2024 CY2025 Acadia Guidance US Royalty to Neuren(A$m) As well as the exclusivity periods, Orphan Drugs have many other commercial advantages compared existing DAYBUE US Net Saleswith (US$m) + 9-16% markets that have apparently attractive large sales in which 405 established products and companies have to be displaced. + 9-16% The serious and urgent unmet348 need results in a more 405 380 + 97%and pricing environment and supportive regulatory strong 348 engagement from the patient community and leading 380 + 97% physicians. Historical data indicates a higher probability of achieving regulatory approval and the potential for 177 immediate access to known patients means that a large sales organisation 177 is less important. US Royalty to Neuren(A$m) + 11-19% + 110% 56 + 110% 56 + 11-19% 67 67 62 62 27 27 In short, the Orphan Drug business model targets a leadership position in markets with urgent need, at an attractive price and with a higher probability of getting to market. CY2023 (Apr - Dec) CY2024 CY2025 Acadia The neurodevelopmental disorders that NeurenGuidance is aiming to (Apr - Dec) however CY2024 CY2025 Acadia treatCY2023 are “rare diseases”, they are not “ultra-rare”, Guidance and in each disorder there are tens of thousands of potential patients around the world. CY2023 CY2024 CY2025E* CY2023 CY2024 CY2025E* * Based on CY25 Acadia DAYBUE US Net Sales Guidance of US$380-405m, 10% of DAYBUE net sales up to US$250m and 12% of DAYBUE net sales between US$250m and US$500m, and AUDUSD of 0.65 COMMERCIAL EXCLUSIVIT Y In March 2023, Neuren’s partner for trofinetide, Acadia Pharmaceuticals (NASDAQ: ACAD), received FDA approval of DAYBUE™ (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. On 17 April 2023, Acadia launched DAYBUE™ (trofinetide) in the United States as the first ever approved treatment for Rett syndrome. Access to DAYBUE has been well supported by Medicaid and private health insurance payors. In addition to the primary protection of the important exclusivity periods from Orphan Drug designation explained above, Neuren has additional commercial protection from issued patents and pending patent applications, which extend as far as 2041. Since trofinetide and NNZ-2591 are new chemical entities, following the first marketing authorisation for each drug, the term of one patent may potentially be extended by up to 5 years in many countries, including the United States, Europe and Japan. 5 C Neuren Pharmaceuticals Limited Annual Repor t 2024 O P E R AT I N G R E V I E W CONTINUED Net sales of DAYBUE in 2024 were US$348.4 million, with sequential growth in each quarter and record net sales of US$96.7 million in Q4 2024. These sales generated royalty income for Neuren in 2024 of A$56 million. US Net Sales (US$m) CY23 DAYBUE US$177m CY24 DAYBUE US$348m (up 97% y-o-y) 87 67 76 85 91 97 23 Q2 2023 Q3 2023 Q4 2023 Q1 2024 Q2 2024 Q3 2024 Q4 2024 DAYBUE US Comparable Drug Launch (US)^ ^ For illustrative purposes only. Comparable Orphan Drug has different patient/clinician experience, approval and distribution/logistical dynamics 1. Latest stats based on Acadia 4Q and full year 2024 Earnings Presentation 26 February 2025 and 43rd Annual JP Morgan Healthcare Conference Presentation 14 January 2025 ^ For illustrative purposes only. Comparable Orphan Drug has different patient/clinician experience, approval and distribution/logistical dynamics 920 patients received DAYBUE in Q4 2024 and 62% of currently active patients had been on therapy for 12 months or longer. Acadia continues to collect and report realworld experience in the LOTUS study, with the majority of caregivers reporting meaningful improvements in patients. The number of diagnosed Rett patients in the US has grown from approximately 4,500 at the launch of DAYBUE to between 5,500 and 5,800. Prevalence studies suggest the total number of patients may be 6,000 to 9,000. Acadia has provided guidance for growth in US net sales in 2025 to between US$380 million and US$405 million. Q1 2025 net sales will be lower than Q4 2024 due to seasonal impacts, which is consistent with the previous year. With 70% of the diagnosed patients yet to try DAYBUE, there is substantial potential for growth in the US. In January 2025, Acadia announced initiatives to accelerate adoption, in particular among the 65% of patients that are treated outside the Rett syndrome Centers of Excellence. Acadia is expanding its field force by approximately 30%, optimizing patient support, launching branded Direct-to-Consumer campaigns to showcase DAYBUE benefits and utilizing a range of communication channels to bring the DAYBUE clinical data to life for both physicians and families. A characteristic of all long-term medicines is that not all patients who commence treatment will persist with treatment. Furthermore, for patients and caregivers, adjusting to a novel treatment regimen can take time, especially when it is the first treatment ever to become available. The number of patients commencing treatment and the proportion that persist with treatment long-term are key factors in the sales outcome. That persistency rate in the real world has consistently tracked at more than 10 percentage points above the clinical trial experience and has been stable at approximately 50% after 12 months of treatment. In Q4 2024 discontinuations improved by approximately 15% compared with Q3. The LOTUS study indicates that initial dose titration improves tolerability, suggesting that the future persistence rate for new patients can be higher than the early experience after launch. Further information about DAYBUE, including prescribing information can be accessed at www.DAYBUE.com 6 Neuren Pharmaceuticals Limited Annual Repor t 2024 O P E R AT I N G R E V I E W CONTINUED NEUREN’S ATTR ACTIVE ECONOMICS FROM DAYBUE (TROFINETIDE) IN NORTH AMERIC A In October 2024, Health Canada approved Acadia’s New Drug Submission for DAYBUE and Acadia anticipates first sales in Q3 2025, pending price negotiations. Canada net sales will be added to US net sales to give total net sales for calculation of Neuren’s North America royalties and sales milestone payments. In Canada, the prevalence of Rett Syndrome is estimated to be 600 to 900 patients. In 2024, Neuren earned income from DAYBUE of A$213 million. In addition to royalties of A$56 million, Neuren received a sales milestone payment of A$80.5 million earned on achievement of the first in a series of four thresholds of total annual net sales of DAYBUE in North America, due to net sales for 2024 exceeding US$250 million. LONG TERM GROW TH OPPORTUNIT Y FOR TROFINETIDE THROUGH GLOBAL EXPANSION Neuren also earned A$76.5 million from Acadia, being one third of the market value of the Rare Pediatric Disease Priority Review Voucher (PRV) that was awarded to Acadia by the FDA upon marketing authorisation of DAYBUE. Acadia completed the sale of the PRV for US$150 million in December 2024.     In July 2023 Neuren and Acadia expanded their partnership for trofinetide from North America to worldwide. Neuren received US$100 million up-front and is eligible to receive milestone payments and royalties related to development and commercialization of trofinetide outside North America as set out in the table below. Neuren is eligible to receive ongoing royalties on net sales of trofinetide in North America, plus milestone payments of up to US$350m on achievement of a series of four America thresholds of totalNorth annual net sales. No royalties or similar costs are payable by Neuren to third parties, which means US$10m U upfront in 2018 Neuren’s revenue from Acadia flows through to pre-tax U US$10m 2022 following of NDA for payments review profit. in The royalty ratesacceptance and sales milestone are related to the total amount of annual net sales ofUS trofinetide U US$40m in 2023 following 1st commercial sale in the in all indications in North America, as set out in the U US$50m In 2024 one third share of Priority Review Voucher following tables: Outside North America  US$100m U upfront in 2023 US$35m following 1st commercial sale in Europe US$15m following 1st commercial sale in Japan US$10m following 1st commercial sale of a 2 nd indication Europe awarded to Acadia (sold for US$150m) US$55m following 1st commercial sale of a 2 nd indication Outside North America Japan US$100m upfront in 2023  U Sales milestones On achievement of escalating annual net 1st commercial sale in Europe US$35m following sales thresholds: Europe: up to US$170m US$15m following 1st commercial sale in Japan Japan: up to US$110m : up to US$83m US$10m following RoW 1st commercial sale of a 2 nd indication Europe Tiered royalties Mid - teens to low - 20s % of net sales US$4m following 1st commercial sale of a 2 nd indication Japan Milestone payments related to Fragile X US$4m North America Tiered U Royalty Rates Sales Milestones US$10m upfront in 2018 (% of net sales) Net Sales onefor review US$10m in 2022 following acceptance ofinNDA  U Annual Net Sales Rates calendar year US$m US$40m in 2023 following 1st commercial sale in the US  U ≤US$250m 10% ≥US$250m  50 US$50m In 2024 one third share of Priority Review Voucher  U >US$250m, ≤US$500m 12% ≥US$500m 50 awarded to Acadia (sold for US$150m) >US$500m, ≤US$750m ≥US$750m US$55m Milestone14% payments related to Fragile X 100 >US$750m Tiered Royalty Rates 15% (% of net sales) ≥US$1bn 150 Sales Milestones Annual Net Sales Rates Net Sales in one calendar year US$m ≤US$250m 10% ≥US$250m  50 >US$250m, ≤US$500m 12% ≥US$500m 50 >US$500m, ≤US$750m 14% ≥US$750m 100 >US$750m 15% ≥US$1bn 150 Salesversion milestones On achievement of escalating annual net A redacted of the expanded licence agreement sales thresholds: between Neuren and Acadia was filed with the US Securities Europe: up to US$170m and Exchange Commission as a material contract exhibit to Japan: up to US$110m Acadia’s 2023 10-K Annual Report, which is available to view RoW : up to US$83m via the SEC Filings section of Acadia’s website. Tiered royalties Mid - teens to low - 20s % of net sales In January 2025, Acadia submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for trofinetide for the treatment of Rett syndrome in adults and pediatric patients two years of age and older. Acadia anticipates potential approval in Q1 2026. If granted marketing authorization, trofinetide will be the first and only approved therapy for Rett syndrome in the European Union. In the meantime, Acadia anticipates initiating Managed Access Programs in Europe in Q2 2025, which will provide valuable real-world experience of treatment for European families and physicians in advance of full commercial launch. 7 Neuren Pharmaceuticals Limited Annual Repor t 2024 O P E R AT I N G R E V I E W CONTINUED For Japan, Acadia has had productive discussions with the regulatory agency (PMDA) and plans to initiate a small clinical study by Q3 2025 to support a marketing application. There is urgent unmet need for a treatment for Rett syndrome around the world, evidenced by communications received from families, patient support groups and physicians. Acadia is assessing a strategic approach to make DAYBUE available in select markets through named patient programs in 2025. Canada 600 - 900 Rett patients 1 Approved in Oct 2024 First sales in Q3 2025 US 6,000 - 9,000 Rett patients 1 Launched in Apr 2023 Europe 9,000 - 12,000 Rett patients 1 MAA filed with potential approval Q1 2026 Initiation of Managed Access Program Q2 2025 Acadia building EU leadership and launch teams 1 Japan 1,000 - 2,000 Rett patients 1 PMDA discussions ongoing; clinical study start by Q3 2025 to support marketing application Acadia estimates i ABOUT RETT SYNDROME Rett syndrome is a seriously debilitating and life-threatening neurological disorder. It is first recognized in infancy and seen predominantly in girls, but can occur very rarely in boys. At diagnosis, Rett syndrome has often been misdiagnosed as autism, cerebral palsy, or non-specific developmental delay. Most cases of Rett syndrome are caused by mutations on the X chromosome on a gene called MECP2. Rett syndrome strikes all racial and ethnic groups and has been estimated to occur worldwide in 1 of every 10,000 to 15,000 female births, causing problems in brain function that are responsible for cognitive, sensory, emotional, motor and autonomic function. These problems can include learning, speech, sensory sensations, mood, movement, breathing, cardiac function, and even chewing, swallowing, and digestion. Rett syndrome symptoms appear after an early period of apparently normal or near normal development until six to eighteen months of life, when there is a slowing down or stagnation of skills. A period of regression then follows, with loss of communication skills and purposeful hand use, loss or impairment of walking, and the onset of stereotypic hand movements. Other problems frequently include seizures and erratic breathing patterns, an abnormal side-to-side curvature of the spine (scoliosis), and sleep disturbances. 8 Neuren Pharmaceuticals Limited Annual Repor t 2024 O P E R AT I N G R E V I E W CONTINUED NNZ-2591 FOR MULTIPLE NEURODEVELOPMENTAL DISORDERS Successful Phase 2 clinical trials across three syndromes Neuren is developing NNZ-2591 for multiple serious neurodevelopmental disorders with different genetic origins that emerge in early childhood and have no or limited approved treatment options. The estimated number of potential patients being targeted across these disorders is more than five times larger than Rett syndrome. In May 2024, Neuren announced positive top-line results from the Phase 2 clinical trial of NNZ-2591 in children with PTHS. After treatment for 13 weeks, 9 out of 11 children showed improvement assessed by clinicians and significant improvement was observed by both clinicians and caregivers in clinically important aspects of PTHS, including communication, social interaction, cognition and motor abilities. NNZ-2591 was well tolerated and demonstrated a good safety profile. Neuren recently announced that the FDA has granted Fast Track designation for NNZ-2591 for the treatment of PTHS. Fast Track is designed to facilitate the development and expedite the review of drugs to treat serious conditions. Neuren’s programs for Phelan-McDermid syndrome (PMS), Pitt Hopkins syndrome (PTHS), Angelman syndrome (AS) and Prader-Willi syndrome (PWS) have all been granted Orphan Drug designation by the FDA and are being developed under Investigational New Drug (IND) applications. In designing and executing the NNZ-2591 development program, Neuren has been able to leverage the extensive and highly relevant experience the Neuren team has gained from the trofinetide Rett syndrome program across manufacturing, non-clinical, clinical and regulatory. In August 2024, Neuren announced positive top-line results from the Phase 2 clinical trial of NNZ-2591 in children with AS. After treatment for 13 weeks, 11 out of 13 children showed improvement assessed by clinicians, with improvements seen in clinically important aspects of AS. In the 3-12 years age group all 8 children showed improvement. The FDA has also granted Rare Pediatric disease Designation for NNZ-2591 in each of PMS, PTHS and AS. With this designation in place, Neuren may be awarded a PRV if the Rare Pediatric Disease PRV program is reauthorized by the US Congress and NNZ-2591 receives marketing authorisation for any of these indications by the FDA. The Rare Pediatric Disease PRV program is designed to incentivize drug development for serious rare pediatric diseases. If awarded, a PRV can be redeemed to receive priority review for a different product or sold to another sponsor. As noted above, Neuren’s partner Acadia received a PRV on marketing authorization of DAYBUE in Rett syndrome and sold the PRV for US$150 million. The positive results of NNZ-2591 in PTHS and AS followed the announcement of positive top-line results from the Phase 2 clinical trial of NNZ-2591 in children with PMS. Phase 2 trial results validating multi-indication platform Phase 2 trial results validating multi-indication platform # ')Ҋ -($
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