Roche Genentech Webinar: Informing the Angelman Syndrome Community

Quick Overview

Roche and Genentech held a webinar to provide information about their Angelman Syndrome program. The webinar discussed the history of the program, the ongoing observational study called FREESIAS, and the upcoming Phase 1 study called TANGELO. The webinar also addressed questions from participants, including topics such as eligibility criteria, reimbursement for participation, and the potential efficacy of the investigational therapy. The presenters emphasized the importance of collaboration with the Angelman Syndrome community and expressed their commitment to developing breakthrough treatments for rare diseases. The webinar was recorded and will be available on the Angelman Syndrome Foundation’s clinical trials website.

Introduction

Hello, everyone! We are delighted to have so many of you joining us today from all over the world. We understand that these are challenging times with the COVID-19 pandemic and the Black Lives Matter movement, but we appreciate you taking the time to learn more about Roche and Genentech’s Angelman’s program. Today, our goal is to inform you about Roche’s Angelman Syndrome program and provide updates on our ongoing observational study, FREESIAS, and our Phase 1 TANGELO study. We would like to thank the Angelman Syndrome Foundation for hosting this webinar and for their continued support.

Introductions

• Zeena Chi: Patient Advocacy Relations, Genentech
• Shady Sedhom: Global Patient Partnership Director, Roche
• Brenda Vincenzi: Medical Lead, Angelman Syndrome Program, Roche

Agenda

1. Introduction to Roche and Genentech
2. History of the Angelman Syndrome Program
3. Overview of the FREESIAS observational study
4. Introduction to the Phase 1 TANGELO study
5. Q&A session

Roche and Genentech: Transforming Lives

Roche and Genentech are one company with a mission to transform the lives of people with serious diseases. Over the years, we have developed 38 FDA-approved medicines, 22 of which have been breakthrough designations. Our commitment to rare diseases dates back to 1985 with the approval of our first growth hormone product. We continue to focus on rare diseases through our three main pillars: following the science, achieving a deep understanding of the disease, and forming strong collaborations with the community.

History of the Angelman Syndrome Program

Our journey in Angelman Syndrome began in 2015 when we started listening to the community and attending conferences. In 2017, we created the disease concept model in collaboration with ABOM, which guided the development of our molecule. In 2018, we co-created the FREESIAS study with the help of families and the scientific community. Last year, we began recruiting patients for FREESIAS, and despite some delays due to COVID-19, we have been working on designing the TANGELO study in collaboration with the patient community. Our goal is to continue recruiting for FREESIAS and have our first patient enrolled in the TANGELO study this summer.

FREESIAS Observational Study

FREESIAS is an observational study co-founded by Roche, Ionis, and Biogen. It aims to assess the feasibility and value of novel endpoints and biomarkers in Angelman Syndrome. The study is decentralized, with only two in-clinic visits and the rest of the assessments done at home. It is currently running in the US and focuses on digital biomarkers such as sleep EEG, sleep mat, and e-diaries. Despite some delays caused by COVID-19, we are almost ready to resume full activities and would like to thank all the families and study teams involved.

Phase 1 TANGELO Study

TANGELO is a Phase 1 study that aims to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of our drug, UBE3A-LNA. The study will enroll up to 66 individuals with Angelman Syndrome aged 1 to 12, with various genotypes. It will be conducted at multiple centers in the US and other countries, with a study duration of up to 58 weeks. TANGELO is an open-label study, meaning that all participants will receive the drug. The primary objective is to assess the safety and explore the efficacy of UBE3A-LNA.

Q&A Session

During the Q&A session, participants had the opportunity to ask questions about the exclusion criteria, eligibility for future trials, reimbursement for participation, the impact of COVID-19 on the trial, potential side effects, primary endpoints for efficacy, and more. The presenters provided detailed answers and assured participants that their questions would be considered even if they couldn’t be answered during the webinar.

Stay Updated

For families interested in staying updated with the latest information about the clinical trials, the protocol for TANGELO will be posted on ct.gov and Roche’s patient website, forpatients.roche.com, by the end of next week. Additionally, Roche is working on a communication plan to ensure that information is easily accessible to everyone, regardless of their participation in the trials.

Conclusion

We would like to thank everyone for their participation and for the insightful questions. The webinar recording will be available on the Angelman Syndrome Foundation clinical trial website for those who missed any portion of it or want to share it with others. We appreciate the support of the Angelman Syndrome Foundation and other organizations in the community and look forward to continuing our collaboration to advance the understanding and treatment of Angelman Syndrome.