Ionis Pharmaceuticals Angelman Syndrome Program Update

Quick Overview

Ionis Pharmaceuticals provided an update on their Angelman Syndrome program during the 2021 FAST Science Summit. They announced that they have received FDA approval to initiate a clinical trial with their lead compound. They have also started important startup activities such as country and site selection for the trial. Ionis Pharmaceuticals has partnered with Roche, Biogen, and Dr. Gali Heimer to collect CSF samples in individuals with Angelman syndrome to measure the levels of UBE3A. The goal of Ionis Pharmaceuticals is to bring the best drug forward that treats the broadest population in Angelman syndrome. They provided an overview of the drug development process and the different phases of clinical trials. They also discussed the importance of informed consent and the do’s and don’ts of participating in a clinical trial. Ionis Pharmaceuticals introduced their clinical trial called Halos, which will be a combination of phase 1 and phase 2. The trial will assess the safety of the drug and measure clinical changes in participants. The trial will be global, with sites in multiple countries. The trial will initially last three months with a follow-up period, and a long-term extension will be added later. The details of the trial will be listed on clinicaltrials.gov. Ionis Pharmaceuticals expressed their commitment to continue their work on behalf of the Angelman community and thanked the participants for their commitment as well.

Introduction

Becky Crean, a representative from Ionis Pharmaceuticals, expresses gratitude to FAST for the invitation to discuss the advances made in their Angelman program since the previous year. Crean provides an update on the current status of the program and highlights the importance of measuring the response to the drug. She also acknowledges the contributions of participating families and outlines the drug development process.

Program Progress

Crean announces that Ionis Pharmaceuticals has successfully initiated a clinical trial with their lead compound in 2021. The company submitted their program package to the FDA and received approval to begin the trial in the United States. They have also started important startup activities such as country and site selection for the trial.

Collaboration and Research

Ionis Pharmaceuticals has partnered with Roche, Biogen, and Dr. Gali Heimer at Sheba Medical Center in Israel to conduct a natural history study. The study involves collecting cerebrospinal fluid (CSF) samples from individuals with Angelman syndrome to determine the levels of UBE3A protein. These studies help inform and de-risk not only Ionis and Biogen’s program but also other programs in the field. The data collected from these studies also aids in designing more efficient trials to reduce the burden on patients.

Drug Development Process

Crean provides an overview of the drug development process, starting from basic research and target identification to preclinical research in animal models. Ionis Pharmaceuticals has completed the drug discovery and preclinical work for their lead compound and received approval to begin the clinical trial. She explains the different phases of clinical trials, including Phase 1, Phase 2, and Phase 3, and emphasizes the importance of following the necessary steps to ensure the success of the trial.

Participating in Clinical Trials

Crean advises families considering participating in a clinical trial to discuss it with their family and educate themselves about the trial. It is crucial to consult with the child’s doctor to understand the drugs being studied and the expectations of participation. She explains the informed consent process and the screening visit to determine eligibility for the trial. Crean also highlights the importance of maintaining study confidentiality and not sharing trial information on social media.

Introduction to Halos Trial

Crean introduces the Halos trial, Ionis Pharmaceuticals’ first clinical trial for Angelman syndrome. The trial will involve intrathecal injections of the drug into the spinal canal and will be a combination of Phase 1 and Phase 2. The trial will enroll a broad age range, including young children and adults, and will be conducted globally. Crean provides an overview of the trial visits and timing and encourages interested individuals to check the clinicaltrials.gov website for updates on site openings and enrollment.

Conclusion

Crean expresses Ionis Pharmaceuticals’ commitment to the Angelman community and thanks the participants for their dedication. She assures the community that Ionis and Biogen will continue their work on behalf of individuals with Angelman syndrome.