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Webinar: SANRECO Phase 2 Results in Polycythemia Vera | Silence Therapeutics

Global MPN Scientific Foundation

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Webinar: SANRECO Phase 2 Results in Polycythemia Vera | Silence Therapeutics

18 просмотров · 5 дн. назад
Global MPN Scientific Foundation
141 подписчик
18 просмотров · 5 дн. назад
How is a new medicine created, and how long does it take to reach patients? In this Global MPN webinar, Prof. Martínez, from Silence TheHow is a new medicine created, and how long does it take to reach patients? In this Global MPN webinar, Prof. Martínez, from Silence Therapeutics, presents the results of the SANRECO Phase 2 clinical trial in polycythemia vera (PV) and walks us, step by step and in plain language, through the full journey of a drug from the laboratory to the pharmacy. In this video you will learn what a small interfering RNA (siRNA) is, how the study was designed with 48 patients across several countries, what the results showed in hematocrit control and phlebotomy reduction, and what the Phase 3 trial planned for next year will look like. The conversation with Peter Löffelhardt closes with the questions the community cares about most: how the treatment will be given, how often, for how long, and what it may mean for iron levels and the spleen. 👉 Interested in MPN research? Subscribe and turn on notifications so you never miss a webinar. 💬 Leave your questions in the comments. We use them to shape future sessions. 🔗 Find more resources for patients and caregivers on our website. This content is for information purposes only and does not replace advice from your medical team. Chapters 00:00 Welcome and opening 00:25 Introducing Prof. Martínez 01:10 Why this study matters to patients 02:02 What it takes to develop a medicine 02:49 From the laboratory to the first candidate 03:26 First studies in healthy volunteers 04:25 Studies in patients: what Phase 2 means 05:41 What changes in Phase 3 06:15 The programme timeline, year by year 08:02 How long and how much a new drug costs 09:15 Why many medicines never reach the finish line 11:12 The role of patients in research 12:12 The SANRECO study and the story behind its name 12:45 Study design: 48 patients, double blind 13:49 The primary endpoint: hematocrit and phlebotomy 14:24 Trial sites around the world 15:06 Key results: 88% versus 19% 16:32 Number of phlebotomies over nine months 19:25 Symptoms and quality of life 20:00 Conclusions: administration, tolerability and iron 22:12 Moving into Phase 3 24:18 Regulators and global reach 25:30 When the new study is expected to start 26:15 Q&A: how the medicine is given 27:49 One fixed dose for everyone, every three months 30:46 Do patients respond the same way across countries? 33:09 How long does treatment need to continue? 35:43 Long term follow up 37:05 Iron, phlebotomy and the spleen 41:52 What patients can hope for next 42:02 Closing thanksrapeutics, presents the results of the SANRECO Phase 2 clinical trial in polycythemia vera (PV) and walks us, step by step and in plain language, through the full journey of a drug from the laboratory to the pharmacy. In this video you will learn what a small interfering RNA (siRNA) is, how the study was designed with 48 patients across several countries, what the results showed in hematocrit control and phlebotomy reduction, and what the Phase 3 trial planned for next year will look like. The conversation with Peter Löffelhardt closes with the questions the community cares about most: how the treatment will be given, how often, for how long, and what it may mean for iron levels and the spleen. 👉 Interested in MPN research? Subscribe and turn on notifications so you never miss a webinar. 💬 Leave your questions in the comments. We use them to shape future sessions. 🔗 Find more resources for patients and caregivers on our website. This content is for information purposes only and does not replace advice from your medical team. Chapters 00:00 Welcome and opening 00:25 Introducing Prof. Martínez 01:10 Why this study matters to patients 02:02 What it takes to develop a medicine 02:49 From the laboratory to the first candidate 03:26 First studies in healthy volunteers 04:25 Studies in patients: what Phase 2 means 05:41 What changes in Phase 3 06:15 The programme timeline, year by year 08:02 How long and how much a new drug costs 09:15 Why many medicines never reach the finish line 11:12 The role of patients in research 12:12 The SANRECO study and the story behind its name 12:45 Study design: 48 patients, double blind 13:49 The primary endpoint: hematocrit and phlebotomy 14:24 Trial sites around the world 15:06 Key results: 88% versus 19% 16:32 Number of phlebotomies over nine months 19:25 Symptoms and quality of life 20:00 Conclusions: administration, tolerability and iron 22:12 Moving into Phase 3 24:18 Regulators and global reach 25:30 When the new study is expected to start 26:15 Q&A: how the medicine is given 27:49 One fixed dose for everyone, every three months 30:46 Do patients respond the same way across countries? 33:09 How long does treatment need to continue? 35:43 Long term follow up 37:05 Iron, phlebotomy and the spleen 41:52 What patients can hope for next 42:02 Closing thanks