IIIRIS Digitally Signed Document Document ID.. 154B612ACA5459 Document Details FiienamL: INP003-Chatity accounts (final)-CS-20241224- 103357-315.PDF cliLnt of: James Anderson & Co Signature Details Jim Green Email: Jitn.Gre¢n@inpdrgaleway.coin Date & Tijne: 2411212024 11:13..55 AM (GMT) IP Address: 109.146.128.134 Signing Statement: International Niemann-Pick Di5C8se Regis agrees and approvcs thc contcnts of this document. Digital Certificate The appi'oved PDF file has been digltally cerlified.Please check tlie Digiti¢ I Cerlificiile information in yoiir l)Dl.' viewer to verify tlie Digital Certificate aiithenticity and the PDI". ha5 llot bee lampered ivith. On bi.hAlf vf: Jaines Anderson & Co PDF digital c¢rtifieat¢: IRIS Sofiwai'e Group Liinited Digital ccrtificate l$$ued by: Globalsign Please keep a copy of this document foi. your itcoi'ds.
REGISTERED COMPANY NUMBER.. 10646129 (England and Wales REGISTERED CHARITY NUMBER.. 1175311 INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY IA COMPANY LIMITED BY GUARANTEEI Report ofthe Trustses and Financial Ststements for the Year Ended 31 March 2024
INTERNATIONAL NIEMANN.PICK DISEASE REGISTRY Cont¥nts of the Financial Statements for the Year Ended 31 March 2024 Page Rèlerènce and Administrativè Detalls Report of th& Trustees Independent Examlnels Report Statement of Financial Activltles 10 Balance Sheet Notes to the Financial Statements 12 10 18 Dètall•d Statement ol FnanCIal A¢tlvltles 19
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY Reference and Administrative D8tai15 for the Y8ar End8d 31 March 2024 Trustees J E Green T Hwot Mts T A Malhieson Dr A Dsrdis Dr M Vaniei CtroptedTwste8- Glty1 Company secretary Mrs T A Malhieson Reglstered offlce Suitè 2 Veirnont House Washington Tyne and Wear NE37 2SQ Reglstered Company number 1C646129 (England and Wales) Reglstered eharfty number 1175311 Independent examlner James Anderson & Co Chartered Accountsnts Pentland Eslale Slraiton Edinburgh EH20 9QH Chief oxecutlvB officer Conan DonrEIIy Page 1
INTERNATIONAL NIEMANN-PIGK DISÉASE REGISTRY Report of th8 Trustees fortho Year Ended 31 March 2024 The trustees who are also director5 of the Charity for the purposes ol the Companies Ad 2006, present their report with financial slatements ol charity for the year 8nded 31 March 2D24. The trustees have adopted the provisions ol Accounting and Reporbng by Charili8s.' Statement of Recommended Practice 8pplic8bb to charities prep8ritvJ their accounts accordan[ with the Financial Rèportirg Stsndaid applicable in tIE UK and RepublTe of Ireland IFRS 1021 leffeclive 1 January 20191. Objectives and activities Backgiound Th8 International Niemann-Pi¢k Disease Registry IINPDRI is an ind8pendant. not-for-profit charitable company regiSt8d in the UK Cleated by a collabofalion of palienls. health professionals and medirAI iesearchers. it offers a single. di5ease-specific global regislry data r&sou that indudes both Clin81 data and patient reported data for Aod Sphingomy81inase Deficiency IASMDI Niem8nn-Pick disease. and Niemann-Pick disease type C. Due to its rarity. r8gi51ration for Niemann-PKk dk8e8se5 is partKularfy important. Registration can provid8 Sufficient data lo understand its cOp1eX natural history, support acc18[ated recruitment to trials facilitate the devdopment and monitori9 of care standards. Mission, Vlslon and Princlples The mission of Ihe INPDR is to document the Ni8m8nn-Pick patpnl experieTh'. to improve diagnosis, trealment arKI cai8, to advance r8search and ultimat81y, to improve health outcomes for 811 those affected Our vision Is the cr8akn.on aThS ong(Mng devdopmenl of a comprehensive. international (J8ta resource, sperific to Niernann-Pick diseases which benefits patients by increasir¥J understanding ol these rare conditions, en(x>urag&s effluent and timely diagnosis. enabtes progress in research and clinical Irials. and laulitates the development of therapeutic in18tventions. INPDR advan¢es ourwsion by adhering to following prines. owe respèct and protect patient piivacy, arKI build trust among our patierit c(xHmunity. o We striv8 for excellence through ¢tInUOuS improvement. professionalism and expert support. o We operats lo the hvahest leve15 of suentific integity arKI promote high-quality research. o We a transparent in communilxtions and lake respoTrsibilily lor our action5 and decisions. owe foster an environrnent olcollaboration to itnprove outcomes in Niemann-Pidt diseases. o We embra¢* Innovation and change as appropriate to our need$. We shall remain indetend8nt of Voca¢y, policy and commercial interests and bl the data tell the story of Niemann-Pick diseasès. A key strength ol the INPDR is the collaborative relationships developed trom the outset and enabling the powerful combination ol patient adershiP and professional imp4emènialion. This ensures the leort5macy ol the patient perspective and ensures Ih8t il is at the heart of our organisation and mission. Page 2
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY Report of the Trustees for the Year End8d 31 March 2024 The INPDR will work to eThh8n and falItate progress through the following airns and objèctiV8S'. Alms o To build a high qu81ily, complete Inleinalional populalion-based Niemann-Pick Disease Registry o Proted patient privacy and ènsure (xrnpI1an wth data protection an£J the highest Clinical stsndards o To f8cili18te clinical rtsearch through data sharing provision ol analytical 8eNces o Provide evidence to facilitate advocacy and inform publK debate Objectlves o To describe Ihe epidemiology and natural hislory ol ASMD Ni8rn8nn-Pid( diseasè and Niemann-Pick Typè C o To SUPPDrt development of stndaids of diagnosis, clini(xl rnanagement and quahty ol care o To e5t8blish genotype-phenotype correlat'ons o To piowde support lor equal accB$5 to biochemic81 gr gerElic testing To faTrlitate accelerated r8(Yuilrnenl to intetvention81 stlIeS o To prowide a regulatory compliant registry 10 satisfy posl-markeling oblvJation5 o To support regulatory interaction between nalionallsupranation81 rnedicines agenc$ and ph8rmaceutical compani85 o To alerate progress in underslandiw NPD, by encouraging 8duc2tion ol health professionals and researchèts Background The INPDR is a regulatory-compliant. secure dalab8se ol Consenting pa118nts, with appropriqte security, manageffl8nt, and controls. A secure web-based electronic dats capture IEDCI 8orM is used for the 5yStemalic and uniform collection of clinical d8ta ol patients diagnosed with either Acid Sphingomyelinase Defiaency IASMDI or Nlemann-pick Type C INPCI. The 8clive participalion of clinicians and pabents from around th8 wodd facilitates the collection ol demogfaphie inf(Kmation. diagnosknc ie8ulls, treatment, 8nd h8Oh outcomes data The INPDR is a long-temi initiative that will continue to grow and to create mechanisms to ensure that the data (x>1ckd woiks hardest to 5erye patient needs. Siqnlll¢ant activitie5 In the 18st year we have worked to implement the INPDR'S global reciuitmenl plan, lo acc818r8te data collectK)n, improve clala quality assurance and to facililaie research with Ihe ultimate goal ol improving outcomes in Niemann-Pick diseases, Clinlcal Data Colleetlon In 2023124, INPDR collaborated wth 22 clinical site8 across Europe, North Arnera. and South Arnerica to collect data on 450 pstients 1350 with NPC 8nd 1C() with ASMDI for clinical research. Th8 r89islry is expanding with 44 new clinic81 site5 In Europe, Australia, and th8 US. Patlent Reported Dats A revised patnt-reported database IPRDI launched in 2023 saw 144 patients enrolkgd by March 31, 2024. mathng a 40¥. growth. Vvhile growth has b&t*n slower than expected, Improvements including promotional aclivilies and user interfats enhanMentS We Inilialed. By March 31, 2024. several qUestnnaireS were completed, including 96 quality ol life foFrn5 and 102 symptomslsev8rity assessment5. The PRD is currently in English, bul a mulb-language solution is in develoKKnenl for 2025. Data quality monitoring was strengthened in the US and UK. SUPFK)rtaY by the Ara Parseghi£n MedicAI Research Fund, wrth wsits to clinical sile5 to ensure data accuracy INPDR also achieved IS09001 a¢credilation for Qualty Managem8nt Systems, confirning ils strong registry and 18588rch support cap8bililK95. Page 3
INTERNATIONAL NIEMANN.PI¢K DISEASE REGISTRY Roport of the Tru8tee5 for the Year Ended 31 Mareh 2024 Obje¢tive8 and aetlvltles Anonymlged inforrnatlon $harlng INPDR suppNted multiple research projects focusing on various a5PeCts ol Niemann-Pick Disease, Including disea88 pl0g$s1on, qu81ity of life for patients 8nd carer5, seizure burden, and the management of associ8ted conditions like infiammatory ix)wel di563se. S8v8ral antsnymised d8ta requests were proSsed, including genotype reports for the NPC population. 8n anonymized dataset was prOVed to C-P8th, and a VIeW ol the All-stripès database to assess ils ulilily for a Us-led sibling study was undertaken. Publlcations, Reports and Poster8 INPDR were also invofv8d in reports and publicth.ons Induding, a 2024 publication in Orphanet Journal ol Rare Di4e858s expk)ring the re81-lif8 impacts ol olipudase alfa on ASMO patients and their families. In add110, INPDR published both quanlilalive and qualilats.ve reports on th8 impact ol olipudasg £lfa on adutts with ASMD, contribukn'ons from INPDA, NNPDF and NPUK. INPDR also contributed to posters al several cOnfen$ indudiry update5 on Niemann-Pick Typ8 C and ils research al the WORLD Confer8ntE in February 2024. including findings on impad of olipudase alfa on 8cSu115 with ASMD and real-world study from the Early A¢$$ Program with Arimodomol. Public benefit In shaping our objectives for th8 yèar and annIng our activities, the trustees have con&ded the Charity Commission's guidan on publi¢ befil. including the gUidan.pUb1iC benefit.. running a ch2rily IP821'. Achlevement and performance Charltsble a¢tivltles Grant funding in Ihe form of unrestricted educats'onal grants enabled the charty to carry out ts acliwties. In addition, INPDR Gateway provided payment io INPDR lo facilitate its ser$. Further funding came trom the Ara P8r58ghian Medral Research Fund. Vve have conlinu8d to maintain a strong pr85ence in the field of research have collaborated on a global basis Wth other rare disease oiganisalions, research and academic institutions, regulators, and industry to pri[@s$ and further dgvalop the registry, and to faalftaie research and diniral development. We continue lo sustain appropriate relationships wth the pharmaUl1CaI industry, particulady those enged in elinieal ogiarnmes and activities in the field of Niemann-Pick disease5 and lo work dosely with the global patient advocacy leaders supportitvJ the NM8nn-pk community. This indudes the International Niemann-Pid( Dlsease Allian. whos8 presenlalives ale appointed as INPDR Board and Scientific Advisory Comrnittee Mernbers. Wè continued to engage with the global Niemann-Pick community through irtUal in-person conferen$ and focus groups. pe4
INTERNATIONAL NIEMANN.PICK DISEASE REGISTRY Report ofthe Trustees for th8 Year Ended 31 March 2024 Achlevement and performanc¢ Achlevement and perfomTrance In the last year we have worked to impkment the INPDR'S global reeruitm8nl plan, to arat8 data colledion. improve data qualty assu[ant and to facilitsle research with the ulllmate goal of improving outcomes in Niemann-Pid diseases. During 2023124 INPDR worked with 22 clinical siles in Eurq)e. Norlh America, and South America to collect clinical data on 450 patients that will be used to support 8 r8ng8 of clinrAI research studies. This included 350 people with NPC and 100 with ASMD. With increasing engagement from the global dinical community, we are slso onboarding a further 44 clinical sites in s8vgral European countries, Australia, arKI the United Slates. This L% the first step toward growing the giStry and (aplurirvj cntically Important patsent d8ts. -The registry a150 10118d out a revised patient-reported database IPRDI in 2023. He patients can share thèir experien of Ni8n18nn-Pick Disease through a seri85 of questionnaires. There were 144 patients in PRO by March 31st 2024 133 people affected by ASMD ancl 111 with NPCI, over 40% growth on th6 pfftwous yèar. lllcieases were ObSerd across sever81 regions but rCUlarty in US. Europ8 and UK. However, grh of the PRD has been Slower than anlicipaled. Consequently, a number of improvements were initiated during the period including a range ol piomolional activities and user int8rf8c8 enhancmentS. Not all people gistered in Ihe PRD have ¢ornpleted oueslionnsires and som& h8ve CoMpted questionnai$ acr055 different lime points. By March 315t 2024, 96 quality ol life questionna15 have been completed 181 NPC & 15 ASMDI. 78 Caier qu81ity of life questionnaires h8ve been completed and 78 NPC quality of life forms have been eomplèt8d. 102 syrnptoms I saveiity qu8Stionnaires have been completed186 for NPC and 16 frir ASMDI. Vthile the PRD iq currently available in English 18ThJuage, work has progiessed swn1ficanY with our te¢hnolcvJy partner. OpenApp, to develop a mulliple-langu8ge Solution wth the expectation that thi5 will be iolled out in 2025. During the year. we embedded our d8t8 Quality monitoring function In the US 8nd UK with the support ol the Ara Parseghian MeLlical Research Fund. Our Clinical Research Assooat8s continue Io wi51t ¢lini¢al siles in the UK and the USA lo undertake data monitori. Th8se visits are instrument81 in building clo$e rel81ions wlh site staff and to ensure there has been an ass&ssment of the accuracy ol dinical data in the regislry. In addition, we have establi5h8d centtal data monitoring process85 lo provide a cosl-effectiV6 monitoring of dalg CoMetene$s, Iirneliness and consistency. The INPDR achieved IS09001 accreditation for Quality Management Systems by the 8rit15h Standards In51ilule during thi5 period for 811 areas related to data collection and management. This accrecfitation dernon5tr8les the INPDR has robust system for managing the registry and supporting dinical research. In terms of data use in the INPDR, there have been several approved 5tudie5, r858arch sludE$ and data requests. These are summar1$6d below. Research Studlos St Mary'5 H05Pital M8nchester, UK,"Defining the 58izure burden in Niemann Pick C. Aston Univ8rsity. UK."Quality ol life (QOLI in patients with Niemann-Pick D1sease Type C INPCI and their carers.. Univer511y College Lordon, Disease piogiession in early and late infantile Niemann-Pick. University ol Oxford, UK, 'The manif8Stations and rnana9ement ol Inflammatory bNel dise83e in Niemann-Pick disease type C INPCI patients." - University ol Oxlord,"Incorpoiation of ASIS into IfE INPDR for NPC cases. Shared Informatlon - Genotype reports published for NPC population by Country- NNPDF, Marth 2024. - Anonymised NPC 8Xtract released to C-Palh, February 2024. Note review performed on All-slripes database Shared with INPDR to 8s$e$s utility for Us-led Slbling study. Peer.revlewed Publications R8eb81. E.M . Wseman, S., Donnelly, C., Malhieson. T. Pounlney. J., Crowe, J. and Hopkin, J., 2024. Real-life impacts of 011plase alfa.. The experienc8 of patnIs and families taking an enzyme repl8ment Iherapy lor acKI SphiOMY8l1Thase deficiency. Orphan8t Joumal of Rare Diseases. 19111, p.36. Reports The Impact of olipudase alfa on adults with ASMD- Part 1 Qu3nlitaliv8 Report. Solomon Mbua, INPOR Justin Hopkin. NNPDF Toni Mathieson, NPUK Joslyn Crowe, NNPDF Conan Donnelly. INPDR The impact of treatment with dipudase alfa on adult patients with ASMD - Part 2 Qualitative Repoft Rare Disease R65earch Partngts. Page S
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY R8POrt of the Trustees for the Year Ended 31 March 2024 Achlevement and porforman¢& Posters WIORLD COnfgf8n, February 2024. Ni8mann-Pick Type C.. An Update by The International Niemann-PicJ( Disease Registry. WORLD Conferenc£, F6bruary 2if24, Th8 impacts ol olipudase alfa on adults with ASMD.. palient-reported experien. - WORLD Conferen, February 2024, Real World Data Collection in Niemann-Piek Disease Type C - Data from Early A¢$$ Pragr8m with Arimwbmd. Communleatlon and engagement INPDR also wllaborated internationally to undertake research in Niem8nn-Pick diseases. Resutt5 were sented at national and International meetings Includ1no'. Niemann-Pid( UK, The International Niemann-Pick Di88as6s Allian. - WORLD (We'r8 Organising Research on Lysosomal Disease) Symposium. Florida, USA National Niemann-Pick Disease Foundation Conferen, Florida, USA - Ala ParseghBn Research Conferen, Arizona, USA The iegistry has worked hard to strengthen ils profi18 and Increase inlernalional engagement during th8 last year, in addition lo pièsenting at international meetiNJs, we established the INPDR Ambassador Programme, where communlty leaders work to promote re¢ruilm8rit to the registry in their country and to provide 360. feedback lo INPDR with vW to improving INPOR engagemenl and perf0mlan. Flnanclal review Flnancial posltion Uniesliicled income in th8 year was £39.143 12023 £109,381) arKI unrestridèd expendilure vas £183,249 12023 £348.3701. A iestricled giant totalling £81.321 12023 £63,980) w8s awsrded by the University ol Notre Dame. thr¢)ugh its Ara Parseghian Medical Research Fund and Michael, Marria. arKI Christa Parseghian Endowtnent for Excellence in Niemann Pick Type C Research. The funding re1Ve has en8bled the continuing dev81oprnent ol the Reg151ry. Alongside the high level of 5UPPOrt and inler8Gtion from the patient and Clinal communities. d8mon51rates the potenti81 of the Registry lo make a posrtive diffe nc8 for the global NMann-PiCk patient community. A iestricted giant of £11.534 was 1Ved Iiom Sanofi Genzyme loward5 the INPDR forum 2023 8nd the National Lottery Communty Fund adva[ed £19,500 for telling the story of NPDS in the UK. Deficit on unrestrf¢ted fund The Is a deficit on the nstrICted funds. This reflected increased activity and COrspOndIng expenditu following an UnderSper in these funds In PvIouS years due to slower activity during COVID. 11 is proposed to address the deft in the UnstrICted funds by increasing the contiibutv)ns from INPDR G2Mval'. Reserves pollcy There were no fve8 teserves on 31 March 2024 as there was 8 defiat of £7,429 12023 £136,677). The board aim is to hold six months running costs in feserwe which WOU be £91.624. Further as the registry acb"irities glow a higher Ve1 of r8serve will be reqviied. Sustslnablllty & INPDR Gatèway Ltd A sustainability plan h8$ be8n introduced with th8 aim ol diversifyiro income and providing finanu81 stability. This includes a commercial subscriplK)n model, a costing miKJel for the non-c(xMmertsal sectors alongsid9 a trust and corpor8te g18nt strategy. To support th8 commercial subscription, a commer¢i81 cnmpgny, INPDR Gateway Ltd, was estsblished. An 8PPlOPriata gtsvernan rne1 was established to allow the Board of Directors ol INPDR Gateway to manage commercial relaknonships on behalf ol INPOR and ensure that income gener8ted through these activiti85 IS utilised by INPDR to serve interests of th8 International Ni8mann-Pick community. INPDR Gateway contribuled lo £20,0(YJ 12023 £104,2761 to INPDR. Page 6
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY Report tsf the Trustees for the Ygar Ended 31 March 2024 Future plans The INPDR has devgloped and embe(IcSed a compreh8n3iV8 model for collectiry both rjinician and patient-reported data, support8d by systems that imprtsve data quality and facilities for clinical and 8pid8miok)gi¢al research. Our 5USt8in8bility p18n, powered by the INPDR G8teway. offers commercial organiz31ions the opportunity tts invest in the registry, driving growth and broadening th8 servi8 we provide. In the coming year, the INPDR will txjild on Ikese Systems to lurther adv8nc8 our mission, with eonts"nu8d inv8Strnent in the following key areas". 1. Communication A variety of resour$ will be developed 10 51r8ngthen communication with all INPDR stakehoklers. The registry will invest in communication expertise and enhancs its presence at nallonal and intemational Conferen$. We will 8180 focus on building stronger partne15hips wth the global Niemann-Pick community to promote the INPDR among clinicians patients. 2. Data Quallty and Cornplfrten855 The INPDR tearn will ¢ollaborale with dinical sit•s woiklwKI8 to improve data COmptene58 and accuracy, ensuring the gistry effectively Supports a wide range of clinical research iniliatwes. The r8gistry tnanagement team will furth8r develop this fun¢lion and undertake further initiatives to enhan ntral data tnonitoring pr(Kesses at INPDR. 3. Research Support In the coming year, the INPDR aims to expand its portfolio 015UPPOrted research studies. In partnership with our collaborators, we will facilitat8 approved research by offerirKJ clinicians, researchers. and Industry aec8SS to 8nonymized data and an8lyses. Addbtionally. we will engage in 5pecffic research projects with clinicians 8nd natronal and intemational patient organEz8tions to deepen the understanding of th8 Niernann-PKk dise8Sè expernc?. Structure, goyemance and management Statu5 and Govemlng Document INPDR is a company limited by guarantee governed by rts Memorandurn and Artides of ASSOCIat)n. The cotnp8ny has ore member, the International Ni8m8nn-Pid< Disease Alliance. INPDR is also iegistered as a charity with the Charity Comtnk55ion. Re¢rultm¥nt and appointment of new trustees Future Tru51ees shall be appointed by th8 Truslees frThr time to lime following 2 nornin8tion 1Ved Irotn Ihe Nominations Committee. Trustees ai? VOner$ and e8ch takes on responsibilities wthin the organlsatK)n to co-or(lin8telsupport an aspect of its fundion. Inductlon and tralnlng of new tn10*9 New Trustees are bnefed on their legal obligations, the content5 of memorandum & artidès ol association. the board ancl dècision tnaking processes. the business p13n and the recent financial performance of the charity. During the Induction, they are mentored by other Trustees. Oragnlsatlonal Structur8 The Tiustees are directly responsib16 lor the effeclive goVerTran ol the organis8tion in acrdanCe with statutory guidance ano legislath)n as provided by the Charity Commissioners and Companies House. The Trustees meet quarterly with additional contact as requir1. in order lo VIeW ièports and lo carry out management and financial rewews. Trustees follow a code of conduct. which provides clear guidelines 8$ lo the standards of behaviour, r85pon3ibilities, and best practi exp8Cted of those involved with the INPOR. Trusteas 818 the day-lo-day management of th8 organisalion to the Registry Management Team. led by the Chief Executive Officer, who reports r4ularty to the Board at quarterly virtual meeting5. Key managemènt remuneration All trustees give ol their lime IreeFy and no Iiustee received remuneralK)n in the y8ar. The board i8 responsibl8 for setting employee salary levels. All salaries are b8nchm3rked with similar roles in the volunlary sector, not wth lh8 publ (K priv8te sectors. The size ol the organisation is also consKJered. Pag8 7
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY R8POrt ofthe Trustees for the Year Ended 31 Mareh 2024 Stru¢ture, governance and management WSder nelwork The eh8rity's member is the Internation81 Niemann-Pick Di58a8e Allian IINPDAI which is an alliance ol non-profit support oiganisations who ar8 associaled Mrith Ihe rare group of genetic diseases known collectively 88 Niemann-Pick Diseases. The Alliance provides a forum where, throuuh the exchange ol inlormatK)n, experien and knthedge. global progress can be accelerated Rlsk management As part of the Quality Management System, the INPDR rnaintains a risks and issues log. Risks are managed on 8n orooing basts by the RiStry Management Team and reviewed by Board Mernbers 81 a quarterly Man8getnenl Review Meeting. The registry OP8rates in a highly regulated dinical research environment arvj musl ensurè mplianCe vrilh data protection law and good ¢lini¢al practice. INPDR operale5 In compliance with the European Medicine Agency IEMAI Guidelinès on the Management of Patient Registries and the ieuistry has reCVed a letter ol support trom EMA as g resource for dinical res8arch. To manage ii5k5 lated to data protection and ensuie the privacy of gistry partlC4P8nts. INPDR employs the servi ol a ¢on$ultant Oala Prote¢tK)n offir. In addition. durino the year 2022123, our tschnology partner, OpenApp was awarded IS027001 acciedilalion for data se¢urity, provKling further Confiden that the INPDR are well-placed lo en8uie patient privacy th regard lo data protection, there were no adverse events, incidents or breaches reported during 2ry23124 and no data $ubj8ct ligh15 Nuesls. Recognising the contribution of voluntsers We a extremely grateful to those who give their tirne as volunteers in su5WQrt of the INPDR. Without Iheii d8dicated and continued 5UPPOrt, we woukj not be gb lo achieve 811 that is quired on behsll ol our oryanisstion and community statement of tnJ8tètrs' respon8ibilitles The tru51e8s (who are also the directors of Inlemational Niem8nn-Pick Dkqease Registry lor the purposes ol company wl are r8sponsible for preparing the Report of the Trustees th8 financial slalements in accordance wrth applicab law and Unit8d Kingdom Accounting Standards (United Kingdom Genet81ty Apted Accounting Practi1. Company law requiie5 the trustee5 to prepare financial statements lor each financial year which give a true and fair view of the 5tste of affairs ol the tharitsb company and of the incoming resource5 and application of osources, including th8 income and expenditure, ol the th8rltsb company for that pefiod. In prepang those finan81 statements, the trustees are required to select suitable accounting pdicies and then apply them consistently., obseNe the methods and principles in the Ch8rity SORP", makejudg8ments and estimates that are aSOnable and PDJdent'. prepare the finanoal statements on the going concem basis unless il is inappropriate to piosume that the charitable company will continue In business. The trustees are responsible for keeping proper accounting records which disclose with reasonable accuracy at any time the Iinancial position of the eharilable company 2nd lo enabl8 thèm to ensure that thè financial st8lemen15 Comply with the Companies Act 2006. They a also responsible lor S8f&[dIng the assets of Ihe chantabb (x>mpany and hen f¢w taking reasonable step5 ft)rthe prevention 8nd detection of fraud other irregul8ritie5. This report has been prepared in 8Cwrdan with th8 swal provisions of Part 15 01 the Companles Act 28 r81ating to srnall companies. Approved by order of the board ol trust8es on 24 December 2024 arKI signed on I behalf by.. J E Green- Tiusteè Page 8
Indèpendent Examlner's Report to the Tru8tees of International Niemann.Pick Dis8ase Reglstry Independent examlnerfs report to the trustees of International Nlemann.Pick Disease Registry I'the Company'l l port to the charity trustees on my examination ol the accounts ol the Company for the year ended 31 Maich 2024. Responsibi15ties and basls of report As Ihg charity's trustees of th8 Company land alsts its directors for the purposes ol company lawl you are responsible for Ihe preparation ol the account5 in arordanTr with th8 requirements ol the Compan5 Act 20061'the 20W Act'l. Having satisfied mysew that accounts ol the Company are not required to be audited urKJer Part 16 of the 2CO6 Acl and are eligible for independent examination. I report in respect DI tny examination of your charity's accounts as carried out undei Se¢lion 145 01 the Chaiitie5 Act 2011 I'the 2011 Acl'l. In carryin9 out my examinat I have followed the Directions given by the Charity Comrni5siDn under Seclion 145151 Ibl of the 2011 Act. Independent examinels stalemtrnt I have completed my 8x8minalion. I confim that no matters have come to my 8ttenty.on in COn¢(lon with the examination giving me cause to believe.. aceounling records were not kept in r8spect ol the Company a5 required by Section 386 olthe 2006Act', or the 8ccounls do not accoid with Ihose records," or the a¢counls do not compw with the accoUnng requirements ol Secknon 396 of the 2006 Act other than any reqU1ment that the accounts give a tIU8 and f8ir view which is not a mattor consider1 as part of an independenl examination.. or the accoun15 have not been pPared In accordance with the tnelhods and principles ol the Statement of Recommended Pra¢U'ce for accounting and repon9 by charibe8 (applicab to charities preparing their account5 in gccordance with the Financial Reporting Standard applicable in the UK and Republic of Ireland IFRS 10211. I have no concerns and have come across no other ma118r5 in connection wilh the examination to which attention should be drawn in this report in ord81 to engbEe a proper understanding of the accounts to be reached. Christopher T Spalding James Ancserson & Co Charterecl Accountants Ptrntland E5tste Str&iton Edinbuigh EH20 9QH 24 Dember 2024 Pag8 9
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY ststement of Financlal Actlvltl85 Ilncorporatlng an Income and Exp8ndlture Accounti for Ihe Year Ended 31 March 2024 2024 Totsl funds 2023 Totsl funds Unre8tricted fund Restricted fund¥ Noteg Income and endowments from Donations 8nd 0a¢re$ 520 92,355 92,875 69,085 Charitable actlvltles Charitabk acliwties 38,623 38,623 104,276 Total 39,143 92.355 131,498 173,361 Expenditure on Charitable actlvltles charitab activities 183.249 124,437 307,686 415.907 NET INCOMEIIEXPENDITUREI 1144.1061 132.0821 1176,1881 1242,5461 Reconciliation of ndS Total funds brought fotward 136,677 50,898 187,575 430,121 Total funds ¢ar¥ied forward 17,4291 18,816 11,387 187,575 Th8 notes form part ol these financial statements P8ge 10
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY Balance Sheet 31 March 2024 2024 2023 Notes Flxed assets Inveslmenls Current a¥sets Deblo Cash at bank 10 22 25,363 90.056 116.772 25,385 2[,828 Crgditors Amounts falliro due Within on8 year 113,9991 119.2541 Net Current assots 11,386 187.574 Total ass8ts lass current Ilabllltles 11.387 187,575 NET ASSETS 11,387 187,575 Funds Uni881iicled fijnds Restricted ndS 13 17,4291 18,816 136,677 50,898 Totsl funds 11.387 187.575 The charitablè company is entild to exemptron from aLKJit under Section 477 of the CoMnieS Act 20C6 for the year ended 31 Mareh 2024. Th8 mgrnbets have not quired the company lo obtain an audit ol ils financial Statemen for the year ended 31 March 2024 in accordano wrth Section 476 of the Compani&s Act 2006. TIE Iruslees acknowledge their responsibilrties for ensuring that th8 Charitsb company keeps accounting records thai 1X)mp wrth Sections 386 and 387 01 the Companie5 Act 2006 and preparing financial statements which gwe a true and fair view of th8 stale of affairs of the charitab company as st th8 end of e8ch finala1 year arKI of ils surplus or deficrt for each financial year In accordan with the requirements ol Sections 394 and 395 and which othetwise comply with the requirements of the Cornpanies Act 2006 relating to financi81 statetnents. so lar as appllcable to th8 charitatde company. These financial slatetnents have been prepared in accordan with the provisK)ns applic8ble lo charitable compani8S subject lo the sm811 cotnp8nie5 r8gime. The financlsl 5ta18m8nts We approved by the Board ol Trustees and 8UtFK>fi8ed for issue on 24 Dernb8r 2024 and were signed on its behalf by.. J E Green- Trustee T1 notes fom part ol thès8 financial statements Page11
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY Notes to the Financigl Statements forth& Year Ended 31 Mar¢h 2024 Accounting pollcies Ba$ls of preparing the financial ststements The financial slat8m8nts ol the ¢h8ri1ab company, which is a public benefit enb.ty under FRS 102. have been prepared in ac¢ord8nce wrth the Charities SORP IFRS 1021 'Accounling and Reporting by Charities.. Staletnent of R8commended Practice applicable lo charitie5 PparIng their accounts in accordan wrth the Financial Reporting Standard applicable In the UK and R8public of Ireland IFRS 1021 leffe¢live 1 January 20191,. Financial Reporting Stand3rd 102 'The Finanoal Reporting Standard applicable in the UK and Republic of Ireland, and the Companies Act 2006. The financial Statements hav8 been prepared urKJer the historical cost convention, wrth the &XptIOn ol investtnents which are included at market walu8. In18mational Nianann-Pick Disease Regk3try meets the definilion of a PLblic benefit entity undèr FRS 102. Income All in¢L¥ne is recognised ¢8 the charity has enti118ment to the incomg. there i% suffKient cert8inty of reIpt and so it is probable that the in(x>m8 wll be 1ved. arKI tha amount of incotne reNable c8n measured Donations and grants are recognised when they have been communicated In vrriling with notification of both the amount and settlernenl dal8 In the event that 8 donation or grant is subject to conditions that require 8 level of performance befo the ¢h8rity is entitled to th8 funds. the income is d8ferred and nol recogniseis until either those corKlilions are fully met, or th8 fijifilment of Ih05e conditions is wholty within the control of the charity and il is probable that those conditions will be lulfilled in the reporting period. ExpendItre Expenditure is recognis8d as soon as th8re is a legal or constiudive oblualion c0tntnitt1 the charity to that expenditure, il is probabl8 that a transfer of economic benefits wll be reqUId in sett18m&nt and the amount of OblatIon Can be measured Trliably. All eXperlture is accounted for on an accruals b8sis. All expenses, including support ¢0515 gtsvernan costs, are alloca18d or apportioned to the applicable expenditurt headings in the Statement ol fin8nci81 acuvities. ExpetKfiture on ¢harilable aclivib'es includes any VAT which txnnot be recovered and is reported a5 part of the expenditure to which it relates and compri58S those Costs incurred by the Charity in the delivery ol ils 8CtMties and services for its benefickanes. It includes both costs that can be allocated diTect1y to such actwities and those costs ol an Indiffjct nature ne[Ssary lo support them. Taxatlon The tharty is exemptfrom corporation tax on its charitabb activitiès. Fund ac¢ountÈng Unrestricted lurKls can be used in accordance wrth the charitable objectwes at the discretion of the trustees. Reslricled fund5 can only be used foi particular restricted purposes within the objects ol the charity. RestriclDns are when specified by the donoi 01 when lunds are ra$ lor particular r8Stricted pury)oses. Further exanatIOn aft nature and purpose ol each fund is induded in the notes to the finanual ststetnen P•nslon cost$ The company operates a defined eontribulion $ch8me, the assels of which are hdd separatety from those of the charity Th8 PenSn cost ¢harge represents Contributions payab by the charity to the schemg. Investments The investment in the group undertaking Is measured at cost less impairment on the basis that it repiB5ernts share5 In an entity Ihat is not publidy traded arKJ the lair value cannot othemise be rne8sured r81iably. Debtors and creditors recelvable I payable wlthln ono yoar Debtors ancl creditors with no stated interest rate arvj re1Vble or payable wrythin one year are re¢ord8d at transaction pri. Any1085es 8rising from impairtnenl are reco9nised In expenditure. Ca8h in bank and In hand Cash at bank and cash in hand includes cash and short term highly IiquKI invèstments. Page 12 (x>nkn'nued...
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY Notes to the Financial Statements contlnu8d for the Year Ended 31 Maich 2024 A¢¢ountlng pollcles- continued Golng concern Th8 financi81 statements have been ppared on 8 going Contsrn basis as the trustees balv8 that no material uncertainties exi51. The company has a defi8cY of as*ts on ils unrestricled fvnd. In response plans have been drawn up to address this. The trustees have considered t1 level of funds helij and the expected 18v81 of income arKI expe[llUre for 12 months from 8ulhorising thes8 financial statement5. The budgeted income and expendilurE 15 sufficient wth the level of ieseNes lor the charity to be able lo conlnue as a going contsm. Donations and legacles 2024 Total funds 2023 Total funds Unrestricted funds Restrlcted funds Donations Grants 520 520 92.355 5,105 63.980 92.355 520 92,355 92,875 69.085 Grants re1Ve(l, Pruded in the above, are as follows.. 2024 2023 Sanofi Genzyme University of Nolre Dame du Lac National Lottery Community Fund 11.534 61,321 19,500 63,980 92,355 63.980 Income from charitab18 a¢tivities 2024 2023 Activity charitab actiwtie5 Contribution lo Research Recharge to INPDR Gateway Ltd Third party reirnbutsernenl 18,459 20,og2 ChaiitalAe 8cli¥iti8s Charitable 8cliwties 20,)0 164 84.184 38.623 104,276 Charitable activities costs Direct Costs Isee note 5 Charrtable activiti8 307.686 Page 13 continued...
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY Notes to the Flnancial Statements. continuetl for the Year Ended 31 March 2024 Direct costs of charltsble actlvltles 2024 2023 Staff costs Equipment costs Registry hosting Travel & meeting5 Website ¢o$ls other staff costs Offi(E expenses Promolional expenses Executive rneetings Independent exatninels fee other regulatory eosts Consultan¢y Legal fees Rebrgnding Research HIC Maint8nan & support 129,563 103,305 1,299 47.913 38.583 1,070 689 8,184 158 2.426 852 60.001 89,619 23,572 9,024 29,212 21,740 29.722 1.027 9,520 5,826 2,424 1.974 3.713 71.669 20,068 10,440 307,6e6 415,907 Tru$tee$' remuneratlon and benefits There were no trustees, remuneration or other benefits for the period ended 31 March 202412023- nil) Trustees. expenses During the period one trustee was wrnbutsed £ 58312023 £1031 for travel exp8nses.12023- 11 staff costs 2024 2023 Wages and salaii85 Social security costs Oth8r pension costs 121,223 S.993 2.347 97.527 3,897 1,881 129,563 103,305 The average monthly number of employees during Ihe year was as fdlows.. 2024 2023 Coordination and 8dministration Registry Manager The Chief Executs"ve OffKer is k8y Man4emeDt and his total ernployee benefits lindud1 Nl and Pension) re £65,455. 12023 £53,915) Page 14 continued...
INTERNAfioNAL NIEMANN-PICK DISEASE REGISTRY Notes to the Financial Statements- continued for the Y8ar Ended 31 March 2024 Comparatlvès for the statoment of financlal adlvitles Unrestrlctgd fund R8Stricted funds Totsl funds Income and endowments from Donations and legarjes 5,105 63,980 69,085 Charltsble activities Chaiitable activities 104,276 104,276 Total 109,381 63,980 173,361 Expènditure on Charitable actlvltles Charitable acb¥rylies 348.370 67,537 415,7 NET INCOMEIIEXPENDITUREI 1238,9891 13,5571 1242,5461 Reconclllatlon of funds Total lunds brought forward 375,6e6 54.455 430.121 Total fund8 Carried forward 136.677 9).898 187,575 FIx8d asset Investmonts shares In group undertakings Markèt value Al 1 Apri12023 and 31 March 2024 Nèt book value Al 31 Msrch 2024 At 31 March 2023 Ther8 were no investment assots outside the UK. Th8 companls inveslrn8nts al the balance sheet d8te in share capital of companies include the following.. INPDR Gateway Ltd Registered office.. United Kingdom 12706306 Nature ol busin8ss'. Data provider lor research Clas$ of share.. holdlng 100 31.3.24 31.3.23 Aggr8gate capital and reseN8s (Lossllprofit lor the yebr 149,4461 152,1601 2,715 2.714 Page 15 continued...
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY Not&s to the Financial Statements- contlnued for the Year Ended 31 March 2024 10. Debtors.. amounts falllng due within one year 2024 2023 Trade debtors Prepayments 22 84.184 5,872 22 J.056 11. Creditors: amounts falling due wlthin one year 2024 2023 Srtial security and other taxes Accruals and deferred income 2.854 11.145 2,532 16,722 13,9 19,254 12. Analysls of net assets befven lund8 2024 Total fvnd8 2023 Total funds Unrestrlcted fund R&strS¢ted fvnds Inveslm8nts Curienl assets Current liabilities 6,569 113,9991 18,816 25,385 113,9991 206.828 119,2541 17,4291 18,816 11,387 187,575 13. Movement In funds Net mov6ment in funds At 3113124 At 114123 Unrestrl¢t¢d funds Gera[ fund 136,677 1144.1(bl 17,4291 Restricted funds APMRF National Lottery Community Fund .898 150.3641 18.282 534 18,282 50,898 132,0821 18.816 TOTAL FUNDS 187,575 1176,1881 11,387 Pag8 16 continued...
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY Notes to the Financial Stat0wrtents- contlnuèd for the Year End8d 31 March 2024 13. Movement In funds - contfjnued Net mov8mgnt in funds. included in the ebove are a5 follow$'. Incomlng r&$ources Resouices expended Movement In funds Unrestrlcted funds General fvnd 39,143 {183.2491 1144,1061 Rgstrlcted fund8 APMRF Sanofi Nstional Lottery Cornmunity Fund 61,321 11.534 19,500 1111,6851 111,5341 I,384) 18,282 92,355 1124.4371 132,0821 TOTAL FUNDS 131.498 1307,6861 1176.1881 Comparatlves for movement In funds Ntrt movement in funds At 3113123 At 114122 Unrestricted funds General fund 375,666 1238,9891 136.677 Rèstrlcted funds APMRF 54.455 13,5571 50,898 TOTAL FUNDS 430,121 1242,5461 187.575 omparatsve nel tnovement in funds. inc4ud8d in the above ar8 as follows.. Incomlng resouree8 Resource$ eXP8nded Movement In funds Unrestilcted fund8 General fund 109,381 1348,3701 1238,9891 Restricted funds APMRF 63,980 167,5371 13,5571 TOTAL FUNDS 173,361 1415,9071 1242,5461 The general fund is ftsa lo use in accordan with the objects olthe charity. The APMRF fund is to accelerate prc¥ress by facilitating iegislry recruitment in the USA. The Sanofi furKI w8S towards the INPDR 2023 Forurn The National Lottery Community Fund is lor telling the story ol Niem?nn-Pk Dis&ases in thè UK Page17 continued...
INTERNATIONAL NIEMANN-PICK DISEASE REGISTRY Notes to the Flnancial Statements- contlnued for thp Year Ended 3q March 2024 14. Relatsd party dlsclosure Tha International Niemann-Pick Disease Alliance IINPDAI is the only memb8r ol the company. Duriro the year costs were charged by INPDA to INPDR of £3.014 12023 £2,920). INPDA paid shared costs of £4,422 12023 £5781. At 31 Marth 2024 there was a balance due to INPDA ol £Nil 12023 £5781. Niemann-Pick UK INPUKI 15 a member of SNPDR. During the year there were rechaiges for shad staff, travel and oveiheads etc by NPUK lo INPDR of £27,142 12023 £20.9911. INPDR received inwre in the year from NPUK of £7,981 12023 - £10,89gi. Al 31 March 2024 there was a balance due to NPUK of £2.650 12023 The INPDR (Registry) fully owns a company, INPDR Gateway Ltd (Gatewayl. which carries out the comrneicial operations of the RlsIry. Ouring the year 2 sum ol £Nil12023 £5.0001 was nI by Registry lo the Gateway. In addilion iecharges and setvice charges weie made to the Gateway in the year of £20,761 12023- £84,184>. A sutn of £22 was due from the Registry at 31 March 202412023 due to £84.1841. Page 18
INTERNATIONAL NIEMANN.PI¢K DISEASE REGISTRY Detailed Ststement of Financlal Activitles lor the Year Endad 31 Maich 2024 2024 Total funds 2023 Total funds Unrestricted funds R8Strlcted fund5 Income and endowments Donallons and legacies Donab"ons Gr8nts 520 520 5.105 92,355 520 92,355 92,875 Charltablg actlvltlè8 Contribution lo Research Recharge to INPDR Gateway Ltd Third paty reimbursernent 18.459 20,0 164 18,459 20,000 164 20.092 84.184 38,623 38,623 104,276 Total Incomlng r8sour¢es 39,143 92,355 131,498 173.361 Expenditure Charitable actlvltles W8ges Social security Employer pEn5ion Equipment costs Registry hosting Travel & meetings Website costs Olhgr staff costs Office expenses Promots"onal exp8nses Executive meetings Independenl examinerts lea Other iegu18tory costs Consultancy Legal fees Rebranding Resear¢h HIC mainlenarice & support 74.666 5.993 2.347 46.557 121.223 5.993 2.347 97,527 3,8g7 1.881 1.299 47.913 38,583 1,070 21,740 11.921 1,027 21,740 29,722 1,027 17.801 9,520 5.826 2,424 1,974 3,713 11.590 9,520 5.826 2.424 1,974 3,713 71,669 8,184 158 2,426 852 60.001 89.619 23,512 9,024 29,212 60.079 20,th8 10,440 20,068 10,440 183,249 124,437 307,686 415.907 Total rèsources eXpded 183.249 124.437 307.686 415,907 Net loxpendlturglllncome I144.1) 132.0821 1176, 1881 1242.5461 This page does Th)t forrn part of the statutory finan(xal staten18nl Page 19