Microsoft Word Roche global HD community letter Jan 2019.docx

Transcript

1 28 January 2019 Update on RG6042 Huntington’s disease (HD) programme: First pat ients enrolled in HD Natural History and Phase III GENERATION HD1 clinical studie s Dear Global Huntington’s Community, Just over one year ago, we and our partner Ionis Pharmaceutical s announced the results of the first ever study that tested th e huntingtin-lower ing scientific hypothesis. Today, we are pleased to announce that the first patient has entered into the GENERATION HD1 clinical study – a pivotal, global Phase ficacy and safety of RG6042 III study to investigate the ef (formerly known as IONIS-HTT ). If this clinical study is successful, it is our hope that Rx RG6042 will be approved by health authorities and made availabl e for the treatment of manifest Huntington’s disease. We are grateful to the patients and family members participatin g in clinical research, as well as everyone supporting them in the broader HD community. Scient ific progress is only ion and participation. possible with your collaborat Recent updates Two global clinical studies have started and first patients are • enrolled – an important achievement for the research pr le on ClinicalTrials.gov ogramme; study status is availab o HD Natural History study (NCT03664804)  This observational study for early manifest HD will run in the USA, Canada, Germany and the UK; planned study sites were announced in late 2018. Initial sites have opened and the first patients have enrolled;  our team continues to work to open recr ckly as uitment at all study sites as qui possible. o Phase III GENERATION HD1 study (NCT03761849)  We received health authority approvals in the USA and Canada to start this pivotal study for manifest HD. Planned study sites for those co untries were lled. announced at the end of 2018 and the first patient has now enro ently working  This study will run in approximately 15 countries; we are dilig aining nd receive approvals in the rem to set up study infrastructure a countries. 1 4 /

2 • Ongoing open-label extension of t he Phase I/IIa study: sponsors hip has been transferred from Ionis to Roche o Patients who completed the Phas e I/IIa study have been particip ating in an open- label extension study sponsored by Ionis (NCT03342053). Respons ibility of this study has now transferred to Roche. Moving forward these patien ts will roll into a new Roche-sponsored, open-label extension study called GEN-EXTE ND. GEN- EXTEND will allow us to continue to study longer-term effects o f RG6042 in those participants who have previously c ompleted a clinical trial for the investigational molecule. Important to note • At this time, access to RG6042 is only through clinical studies because the benefits and risks of RG6042 are not yet fully understood. Information about additional Additional countries/sites for the Phase III study: • sites are nearly ready to countries/sites will be announced on a progressive basis, once s.gov enrol participants; information will be posted on ClinicalTrial • Communications about study data: Roche is committed to transparent and timely communications, as well as ensuring the integrity of ongoing cl inical trial operations and data collection. In line with our Global Policy on Sharing of C linical Study Information, we will share overall programme updates and relevant data from completed and ongoing clinical studies with the scientific community via appropriate channels (e.g., scientific meetings, peer-reviewed journals, etc.). Our team recognises that the need in HD is greater than the cap acity of the RG6042 development programme, and that n ble HD centre interested in ot every person nor every capa these clinical studies will be able to participate. We can assu re you that the studies are o that the benefit-risk of designed to provide health authorities with the required data s RG6042 can be determined as quickly as possible. The ultimate g oal is that this investigational medicine can b e approved by health authorities and made accessible to the broader HD community. munities around the world Our team continues to engage with health authorities and HD com on the RG6042 research programme. We look forward to providing you with further updates. Sincerely, Mai-Lise Nguyen, on behalf of the Roche & Genentech HD team Patient Partnership Director, Rare Diseases ntre Basel, Switzerland Roche Pharma Research & Early Development / Roche Innovation Ce 2 4 /

3 Frequently asked questions and answers What is the HD Natural History study? ims to further understand th e role of mutant huntingtin This 15-month observational study a protein in disease progression, i ncluding how levels of mHTT ch ange over time in the absence of any drug treatment. T here is no drug treatment in th is study. This study will arly manifest (Stage I and II) HD. For all patients who include up to 100 participants with e y study, an open-la bel extension study with the option of complete the HD Natural Histor receiving RG6042 (no placebo control) is planned, pending eligi bility, approval by Authorities and Ethics Committees/Institutional Review Boards a nd if data support the continued development of RG6042. da, Germany, the United The HD Natural History Study will run at up to 17 sites in Cana Kingdom and the United States. For more information about the s tudy/trial sites visit ClinicalTrials.gov or contact the l ocal Roche Medical Informati on team: ● Germany: (+49) 07624-14-2015 ● UK: (+44) 0800 3281629 or [email protected] ● Canada & United States of America: (+1) 888-662-6728 What is the Phase III GENERATION HD1 study? of RG6042 treatment for The GENERATION HD1 study will ev aluate the efficacy and safety rox. two years). ver a period of 25 months (app manifest HD. The study will run o and effective, and ermine whether RG6042 is safe GENERATION HD1 is designed to det therefore includes a comparis on to placebo. Participants will b e randomised to one of three treatment study arms: RG6042 m onthly, RG6042 once every two mon ths (bi-monthly) or placebo monthly. This means for ev ery two participants randomis ed to RG6042, one will receive placebo. The study is “ double-blinded,” meaning neither the participant nor his/her investigator or site staff will know which study arm the partic ipant is assigned. -label extension study For all patients who complete the GENERATION HD1 study, an open with the option of receiving RG6042 nned, pending eligibility, (no placebo control) is pla iew Boards and if data approval by Authorities and Ethics Committees/Institutional Rev support the continued d evelopment of RG6042. The GENERATION HD1 study will enrol up to 660 patients with man ifest HD at 80-90 sites in approximately 15 countries ar ound the world. Planned sites h ave been announced for Canada and the United States of America. For more information a bout the study/trial sites visit ClinicalTrials.gov or contac t our Clinical Trial Informat ion Support Line for the USA nformation about additional c and Canada at (+1) 888-662-6728. I ountries/sites involved in the e details are finalised. study will be announced, as thos How are the clinical study sites selected? A variety of factors influence site selection, including assess ments on experience with HD e capacity to run the study as wel studies, clinic infrastructur l as usual site activities, ability to 3 4 /

4 operationalise the study as quick patient population, and ly and completely as possible, geographic location. What if there is not a study sit e near where I live? Can I relo cate to participate in a study? international , national and loc al laws and regulations. Clinical studies are subject to policies, heal stitutional site th insurance and travel burden may Additionally, factors such as in impact your ability to relocate and be accepted into one of the study sites. Eligibility and enrolment are decided by the study investigator at each site, w ho takes into account all these factors and may also wish to speak to you or your local HD spec ialist for more information. Whether your HD centre is selec ted for participation or not, th is is no reflecti on on the quality of the many outstanding HD clin ics and dedicated care providers around the world. The need in HD is greater than the cap . We have designed the acity of our development programme that the benefits and risks of authorities so d data to health programme to provide the require RG6042 can be determined as quickly as possible. Our ultimate g oal is that this n be approved by health authorities, investigational medicine ca and made accessible to the broader HD community. Can I access RG6042 outside of clinical studies? Currently, access to RG6042 is only through clinical study part icipation because the benefits and risks of RG6042 are not yet fu at we are not able to grant lly understood. This means th pre-approval, compassionate use or “right-to-try” requests at t his time. As our understanding 6042 grows, we will regularly ev of the benefits and risks of RG aluate this position. ll you study RG6042 in Your clinical studies are in ear ly manifest and manifest HD. Wi other patient populations (e.g., juvenile onset HD or prodromal HD)? especially for people living need for a treatment for HD, We recognise the critical medical with severe forms like juvenile onset HD. Once there is suffici ent scientific and safety explore the pot with HD community experts and rationale, our team will consult ential use of RG6042 in populations beyond manifest HD. 4 4 /

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