A Joint Community Webinar with Dr. Berry-Kravis on Roche updates

Quick Overview

Dr. Elizabeth Berry-Kravis, a professor of Pediatrics and Neurological Science and Biochemistry, joined a community webinar to discuss the recent news about the TANGELO study by Roche. The webinar addressed questions from the community and reflected on Roche’s responses. Dr. Berry-Kravis highlighted the importance of dosing timing and the need for more frequent dosing intervals in clinical trials. She also emphasized the need for improved outcome measures that capture the full range of improvements seen in Angelman syndrome. Dr. Berry-Kravis acknowledged the challenges faced by rare disease trials and the importance of persistence in advancing drug development. The webinar concluded with a commitment to transparency and continued support for the community.

Hi everyone! Welcome to today’s webinar hosted by ASF and FAST. We have some important updates to share regarding the TANGELO study. I’m Ryan Fisher, the Chief Operating Officer of FAST, and I’m joined by my co-host and co-moderator, Amanda Moore, CEO of ASF. Our distinguished speaker today is Dr. Elizabeth Berry-Kravis, a renowned expert in clinical trials for rare diseases.

Logistics and Q&A

Before we begin, let’s go over some logistics. We received a number of questions for today’s discussion with Dr. Berry-Kravis. While we were unable to have a community webinar with Roche, we did receive written responses from them, which we have posted online. We will address some of those responses today and continue to advocate for more answers on your behalf. You can submit questions during the webinar using the chat function or the Q&A function on your screen. We will try to address as many questions as possible, but if we don’t get to yours, we will follow up afterwards.

Introduction to Dr. Berry-Kravis

Dr. Elizabeth Berry-Kravis is a professor of Pediatrics and Neurological Science and Biochemistry at Rush Medical Center. She is also the lead of many rare disease clinics, including the Angelman Syndrome Clinic. Dr. Berry-Kravis has extensive experience in clinical trials for Angelman Syndrome and other rare diseases. It’s important to note that she is speaking today based on her expertise and not on behalf of any industry or company.

Families’ Reactions to the News

The recent news about Roche pulling out of the ASO trials has been difficult for families involved in the study. Dr. Berry-Kravis has been in touch with many families and has witnessed their disappointment and concerns. The way the news was communicated, with some families hearing about it through social media before being officially informed, added to the pain and confusion. Dr. Berry-Kravis acknowledges that the situation could have been handled better by Roche.

Impact on Other Trials and Patient Care

The closure of the TANGELO trial has not had any immediate impact on other ASO trials by Ionis and Ultragenyx. These trials are moving forward as planned. However, there are questions about whether patients from TANGELO can transition to these other trials. Dr. Berry-Kravis explains that it may not be possible due to the limited time and the need to protect the integrity of the trials. However, she reassures families that patient care will continue and adjustments may be made as needed.

Washout Period and Potential Partners

Roche has recommended a 9.5-month washout period for patients transitioning to another study. Dr. Berry-Kravis explains that this recommendation is based on the half-life of the ASO and the need to ensure a sufficient washout period. However, the decision to accept TANGELO patients in other trials will ultimately depend on the new partner and their specific requirements. Dr. Berry-Kravis believes that it is unlikely for a partner to take over the program and cancel the ongoing trial, but the final decision will depend on the partner.

Sharing Study Results and Individual Patient Data

Roche has stated that they plan to share study results with the scientific and patient community through various channels, including manuscripts and late summaries. However, the timing of these publications is uncertain and will depend on the partner. As for individual patient data, it is unlikely that families will receive their own child’s data from the trial. Dr. Berry-Kravis explains that it is not common practice to share individual data from clinical trials. However, efforts are being made to enter the baseline data into a database for future analysis.

Lessons Learned and Future Research

The closure of the TANGELO trial has provided valuable lessons for future research and trials. Dr. Berry-Kravis emphasizes the importance of flexibility in study design and the need to listen to investigators and families. She suggests that a stronger advisory body could be established to provide guidance to industry and advocate for the community. Dr. Berry-Kravis also highlights the need for more granular outcome measures that capture the full range of improvements seen in Angelman Syndrome. She encourages ongoing discussions with the FDA to find ways to capture the entire disease in a primary outcome measure.

Future Options for Participating Families

The options available to families who participated in the TANGELO trial are uncertain at this time. Dr. Berry-Kravis explains that if a partner is found, ongoing dosing may be possible. However, the decision to continue the trial or provide access to the drug through expanded access programs will depend on Roche and the FDA. Dr. Berry-Kravis acknowledges the challenges families may face in accessing the drug and suggests that support from the community may be necessary to navigate these challenges.

Persistence and Learning from Setbacks

Dr. Berry-Kravis reassures the community that setbacks like the closure of the TANGELO trial are not uncommon in rare diseases. She emphasizes the importance of persistence and learning from each trial to improve future research and drug development. Dr. Berry-Kravis encourages companies to consider dosing timing, listen to families, and explore more comprehensive outcome measures. She also highlights the need for consistency and collaboration within the rare disease community to overcome the unique challenges faced in drug development.