Pilot programme on a Clinical Compound Bank for Repurposing

Transcription

1 Pilt prgramme n a Clinical Cmpund Bank fr Repurpsing Tpic: Cardivascular diseases and diabetes Tpic: Respiratry diseases Tpic: Neurdegenerative diseases Tpic: Rare/rphan diseases All infrmatin regarding future IMI Call tpics is indicative and subject t change. Final infrmatin abut future IMI Calls will be cmmunicated after apprval by the IMI Gverning Bard. Tpic details Actin type Submissin & evaluatin prcess Research and Innvatin Actin (RIA) 2 Stages Specific challenges t be addressed On average it takes abut 14 years fr a new drug t travel frm the research lab t market apprval at an average cst f 2 billin. Only 10% f cmpunds that enter preclinical testing ever make it int clinical trials, with nly 20% f these achieving marketing apprval [1][2]. A number f innvative prgrammes have been established ver the last few years between research funding agencies and industry t prvide academic researchers with access t high-quality pharmaceutical industry cmpunds that have stalled at sme stage during research r develpment. Many f these cmpunds have already undergne preliminary testing in humans, but have nt been prgressed further because they were nt fund t be sufficiently effective in the indicatin fr which they were riginally develped. These cmpunds represent valuable tls that researchers can use t test their nvel hyptheses fr alternative therapeutic indicatins, with the ultimate aim f identifying alternative uses fr these cmpunds in ther indicatins ( repurpsing, repsitining ). Since partial preclinical and clinical dcumentatin packages have been develped fr these assets, any psitive findings hld the pprtunity t prgress twards the Tel +32 (0) Fax +32 (0) Pstal address: IMI JU TO Brussels Belgium infdesk@imi.eurpa.eu Visiting address: Ave de la Tisn d Or Brussels Belgium

2 market mre quickly and cst-effectively, with the ultimate gal f benefiting patients in diseases f high unmet need. Examples f nging pen innvatin schemes include the Natinal Institute f Health/Natinal Center fr Advancing Translatinal Sciences (NIH/NCATS), the New Therapeutic Uses prgram in the US 1 and the Medical Research Cuncil (MRC) industry asset sharing initiative in the UK 2 in which many EFPIA members already prvide previusly unprecedented access t a subset f their assets. Expanding this assetsharing repurpsing prgramme thrugh IMI2 JU aims t prvide researchers acrss the EU with the same pprtunity t frm hypthesis, t engage in cllabrative research with industry and t access discntinued cmpunds that have already passed thrugh several stages f the drug develpment prcess. In this call, a number f cmpunds are made available fr explratin in specific therapy areas: cardivascular diseases and diabetes, respiratry diseases, neurdegenerative diseases and rare/rphan diseases. The pharmaceutical small mlecule cmpunds made available are listed in the Appendix, tgether with key infrmatin n mechanism f actin, pharmaclgy, safety, tlerability and exclusins relating t these cmpunds. The applicants will submit prpsals t utilise these assets t test their hyptheses fr alternative indicatins within the abve-mentined therapeutic areas, t generate clinical data and, if needed, prerequisite preclinical data, with the ultimate aim f taking these assets t the market in alternative indicatins t thse that they were riginally develped fr. If these pilt tpics n drug repurpsing are successful, the prgramme will be expanded in future calls. Need and pprtunity fr public-private cllabrative research The Eurpean research rganisatins, including universities, hspitals and Small- and Medium-sized Enterprises (SMEs) are renwned fr their cutting edge science and innvative spirit, yet ften d nt have the tls nr the expertise t develp their discveries twards the clinic and regulatry apprval. The pharmaceutical industry has built up significant experience, knwledge and research and develpment (R&D) infrmatin fr a large number f depriritised r disused cmpunds arising frm terminated prgrammes. Public-private cllabratin enables the investigatin f scientific advances within research rganisatins using drugs and drug candidates frm industry. Academic rganisatins benefit frm access t clinic-ready assets and prir R&D infrmatin. This includes predefined preclinical and clinical dsing regimens, txiclgical and pharmakkinetic/pharacdynamic data packages which help t ensure their studies are designed with the best pssible chance f success, alngside the industrial guidance n the data package needed t supprt develpment and transitin twards the market. By cllabrating and bringing the strengths f Eurpean research cmmunities and pharmaceutical cmpanies tgether, it may be pssible t accelerate the research in drug repurpsing and ptentially speed up the develpment f new treatments and giving patients access t these new therapies. Parallel grant-funding schemes with the NIH/NCATS, MRC and selected institutins arund the wrld, cntain many examples f preclinical and clinical prf-f-cncept studies in which the academic and industry cllabratin has prvided the pprtunity fr a rapid transitin twards clinical develpment. This mdel has als previusly prvided the pprtunity t spin ut new SMEs based n psitive repsitining data, enabling funds t be raised t prgress t later stages f clinical develpment. All f these advantages have the lngterm effect f getting drugs t the market quicker and mre cst effectively fr the benefit f patients. A private-public partnership like IMI2 JU prvides the pprtunity t test interesting cmpunds in new indicatins that may nt be therwise tested. In additin, IMI2 JU prvides an exciting pssibility fr translatinal research funding accessible t researchers acrss the EU and H2020 Assciated Cuntries Page 2

3 Scpe The verall bjective f this pilt prgramme is t take ne f the ten previusly depriritised clinical cmpunds listed in the Appendix and investigate their therapeutic ptential in new clinical indicatins in areas f high unmet need. Under the Clinical Cmpund Bank fr Repurpsing pilt prgramme, there will be fur separate tpics, ne fr each disease area listed belw: Cardivascular diseases and diabetes Respiratry diseases Neurdegenerative diseases Rare/rphan diseases Ptential applicants must be aware that nly the cmpunds identified in the Appendix are within the scpe f these fur tpics. These cmpunds are listed therein tgether with key infrmatin including mechanism f actin, riginal indicatin, rute f administratin pharmaclgy, safety, tlerability and links t previus clinical studies and publicatins, t facilitate idea generatin by investigatrs with hyptheses fr nvel uses. The listed cmpunds have all been thrugh clinical phase 1 studies. All prpsals submitted under ne tpic will be evaluated by a panel f independent experts and ranked tgether. Fr each tpic, nly ne prpsal will be eventually retained and a grant agreement will be signed. Prpsals shuld cver clinical Phase 2A prf f cncept studies, thugh larger Phase 2 studies are als in scpe if these are within the budget. Clinical submissins shuld aim at mving twards the next stage f develpment and psitive data shuld be a starting pint fr further investment int develping a drug twards clinic and regulatry apprval; If preclinical wrk is deemed necessary t prvide additinal supprt and cnfidence befre mving int a clinical study in an alternative indicatin, prf-f-cncept/feasibility preclinical studies f up t a year in duratin can be included in the prpsal. These studies shuld have clear g/n-g criteria fr prgressing in t the clinical phase f the prject. Imprtant nte: This prgramme intends t supprt nly innvative clinical develpment fr the cmpunds listed in the Appendix. This means that prpsals fr clinical develpment shuld nt be cnsidered in an indicatin which has been already tested (i.e. riginal primary indicatin r additinal studies) r if there are already nging r planned clinical studies n identical r related disease indicatins with the cmpund r with a cmpund with verlapping mechanism f actin that impacts the nvelty f a given prpsal. Infrmatin n riginal primary indicatins, already tested indicatins, nging and/r planned clinical studies fr each f these ten cmpunds can be fund in the Appendix. Therefre, applicants must demnstrate in their stage 1 applicatin (shrt prpsal) that the prpsed study is innvative fr the chsen cmpund. Expected key deliverables Prpsals shuld have the ptential t identify a new indicatin fr the cmpund chsen amng thse made available within this pilt prgramme. Each selected prject must aim at making clear scientific advances within a given disease area. Key deliverables fr each selected prject include: Initiatin and cmpletin f new Phase 2A clinical prf-f-cncept study in the chsen indicatin which was nt previusly investigated with the specific cmpund; Preclinical data t supprt a g/n g decisin fr initiatin f the clinical study in the new indicatin, if this is deemed necessary fr the selected prject; Page 3

4 Disseminatin f the results in high impact publicatins. Expected impact Achieving early prf-f-cncept fr new mechanisms with the ptential t rapidly bring nvel drugs t patients in areas f high unmet need and/r thse with greatest disease burden; Generatin f ideas and/r data licensed frm the research rganisatin, leading t further develpment f the cmpund in the new indicatin; Added value by repurpsing pharmaceutical assets which have already passed thrugh several stages f the R&D prcess. This can ffer significant time, cst and risk savings ver embarking n discvery prgrammes with nvel targets; Supprting EU academic institutins t cnduct well-designed and high standard translatinal and drug develpment research with quality cmpunds under GCP cnditins, resulting in high impact publicatins and patents when pssible; Pling f resurces and greater cllabratin between the public and private sectrs, with the ptential fr pharmaceutical invlvement r establishment f SMEs fllwing in/ut-licencing; Bsting the discvery and develpment f therapeutics in the areas f cardivascular diseases and diabetes, respiratry diseases, neurdegenerative diseases and rare/rphan diseases using a mre csteffective apprach t drug develpment; Advancing science and knwledge f disease (path)physilgy thrugh testing f new hypthesis; Bsting Eurpean cmpetiveness by cntributing t the establishment f clser links between industry and academia acrss the EU, and ensuring Eurpe is cmpetitive in line with initiatives already in place in ther leading scientific regins arund the wrld. Ptential synergies with existing cnsrtia Applicants shuld take int cnsideratin, while preparing their shrt prpsal, relevant natinal, Eurpean (bth research prjects as well as research infrastructure initiatives), and nn-eurpean initiatives. Synergies and cmplementarities shuld be cnsidered in rder t incrprate past achievements, available data and tls/mdels and lessns learned where pssible, thus aviding unnecessary verlap and duplicatin f effrt. The prjects generated frm this tpic will be cmplementary t ther nging similar initiatives in ther parts f the wrld. Industry cnsrtium The industry cnsrtium will cntribute the fllwing expertise and assets: Cmpunds fr these pilt tpics. The EFPIA cmpanies will cver the csts assciated with manufacturing, supply and delivery f Active Pharmaceutical Ingredient (API) required fr a given study. Clinical studies csts assciated with supply, packaging and distributin f drug prduct will als be cvered by the EFPIA cmpanies. The EFPIA cmpanies will prvide expert supprt fr e.g. study design, prtcl writing, study versight, pharmacvigilance, as well as expert supprt thrughut the duratin f the funded studies with the aim f wrking with the cnsrtium t mve psitive data twards the next stage f develpment. Under each tpic, nly ne f the tw cntributing cmpanies listed abve will be invlved in the full prpsal and selected prject. This will depend n the clinical cmpund which will be develped under a new indicatin. Page 4

5 Indicative duratin f the actin The indicative duratin f the actin is 48 mnths. Clinical studies are anticipated t have a maximum duratin f 36 mnths per actin. If preceded by a preclinical study, this study shuld have a maximum duratin f 12 mnths, giving a maximum f ttal 48 mnths per actin. Applicant cnsrtium The applicant cnsrtium will be selected n the basis f the submitted shrt prpsals. Applicants are expected t frm cnsrtia with ther investigatrs t bring in relevant expertise and ensure study recruitment targets are met in the clinical studies. All experimentatin shuld be undertaken within the investigatrs research institutins and/r their linked third parties. The prpsals shuld be based n a strng scientific ratinale frm prir preclinical and/r clinical data. The planned studies shuld have the ptential fr imprvement f currently available treatments and it is als imprtant that the prpsed clinical studies in the prjects have a clear feasibility fr further clinical develpment and cmmercialisatin f the cmpunds in the suggested indicatins, beynd the prject duratin. The applicant cnsrtium is expected t address all the research bjectives and make key cntributins t the defined deliverables in synergy with the relevant cntributing cmpany frm the industry cnsrtium which will jin the selected applicant cnsrtium fr preparatin f the full prpsal in stage 2. This may require mbilising, as apprpriate, the fllwing expertise and resurces: experience and capability t cnduct all aspects f a clinical trial using an investigatinal medicinal prduct (including data analysis and reprting) under Gd Clinical Practice (GCP) in the prpsed indicatin; clinical and preclinical expertise as necessary fr the scpe f a given study; expertise in the science f drug develpment including aspects f clinical pharmaclgy, study design and cnduct; experience and capability t submit an applicatin fr clinical trial authrisatin with the Eurpean Medicines Agency (EMA)/ natinal regulatry authrities in all member cuntries f a given cnsrtium; strng prject management and cmmunicatin expertise. Suggested architecture f the full prpsal The applicant cnsrtium shuld submit a shrt prpsal which includes their suggestins fr creating a full prpsal architecture, taking int cnsideratin the cntributing cmpany participatin including its cntributins and expertise. In the spirit f the partnership, and t reflect hw IMI2 JU call tpics are built n identified scientific pririties agreed tgether with EFPIA beneficiaries/large industrial beneficiaries, these beneficiaries intend t significantly cntribute t the prgramme and prject leadership as well as prject financial management. The final architecture f the full prpsal will be defined by the participants in cmpliance with the IMI2 JU rules and with a view t the achievement f the prject bjectives. The allcatin f a leading rle within the cnsrtium will be discussed in the curse f the drafting f the full prpsal t be submitted at stage 2. T facilitate the frmatin f the final cnsrtium, until the rles are frmally appinted thrugh the cnsrtium agreement, the prpsed prject leader frm amng EFPIA beneficiaries/large industrial beneficiaries shall Page 5

6 facilitate an efficient negtiatin f prject cntent and required agreements. All beneficiaries are encuraged t discuss the prject architecture and gvernance and the weighting f respnsibilities and pririties therein. References [1] Frail DE, Brady M, Esctt KJ, Hlt A, Sanganee HJ, Pangals MN, Watkins C, Wegner CD. Pineering gvernment-spnsred drug repsitining cllabratins: prgress and learning Nat Rev Drug Discv:14(12): [2] Hughes JP, Rees S, Kalindjian SB, Philptt KL. Principles f early drug discvery Br J Pharmacl: 162(6): Page 6

7 Appendix Cmpund infrmatin template Cmpund name Mechanism f actin Overview Safety/Tlerability Additinal infrmatin Suitable fr and Exclusins - Clinical Trials - Previus, nging and planned Link t clinical trials.gv Additinal Characteristics: CNS penetrance Pediatric Diseases Other Publicatins Page 7