Updates from Pharmaceutical Companies

Quick Overview

Pharmaceutical companies Ovid Therapeutics, Roche, Biogen, and Ionis are providing updates on their progress in developing treatments for Angelman syndrome. Ovid Therapeutics has completed a Phase 2 study and is preparing to start a Phase 3 study for their molecule OV101. Roche is in the preclinical phase of testing their molecule in animals and has achieved positive results in cynomolgus monkeys. Both companies are working on endpoint development to ensure meaningful clinical trials. Collaboration between companies and the Angelman community is emphasized.


This talk provides updates from pharmaceutical companies regarding their progress in developing treatments for Angelman Syndrome. Representatives from Ovid Therapeutics, Ionis Pharmaceuticals, and Roche share information about their research and clinical trials.

Ovid Therapeutics

Tamara Agajanov from Ovid Therapeutics discusses their work in developing a potential therapy for Angelman Syndrome. Ovid Therapeutics is a small biotech company based in New York. They have conducted the first Angelman study and are preparing to start a Phase 3 study. They have two molecules they are working on, OV101 and Fragile X, and are conducting studies in collaboration with Takeda in rare epilepsies. Agajanov provides details about their Phase 2 study called STARS and their ongoing study called ELARA. She also discusses their upcoming Phase 3 study called NEPTUNE, which will be a multicenter, randomized, double-blind, placebo-controlled study.

Ionis Pharmaceuticals

Dr. Frank Bennett from Ionis Pharmaceuticals explains their approach to developing a potential therapy for Angelman Syndrome using antisense oligonucleotides (ASOs). He describes how their ASOs target the antisense RNA to unblock and silence the father’s copy of the UBE3A gene, which is missing in Angelman Syndrome. Dr. Bennett highlights the progress they have made in identifying a lead compound and testing it in cynomolgus monkeys. He also emphasizes the importance of collaboration and the need for natural history data to inform clinical trial design.


Ina Brünig-Traebert and Michelle Krishnan from Roche provide an update on their Angelman development project. They discuss Roche’s rigorous approach to drug development in rare diseases and their focus on understanding the disease and patient perspective. They explain how their molecule interacts with the antisense RNA to bring back UBE3A protein expression in neurons. Brünig-Traebert mentions that they are currently in the preclinical phase of animal testing to assess safety and tolerability. Krishnan discusses their work in endpoint development, which involves understanding what aspects of Angelman are important to families and designing clinical trials that measure these aspects. She highlights the collaboration between Roche, Genentech, Biogen, and Ionis in running a shared study to gather data.


The updates from Ovid Therapeutics, Ionis Pharmaceuticals, and Roche provide valuable insights into the progress being made in developing treatments for Angelman Syndrome. These pharmaceutical companies are working diligently to advance their research and clinical trials, with a focus on understanding the disease, ensuring safety, and addressing the needs of patients and families. Collaboration between industry and the Angelman community is key to accelerating progress and bringing potential therapies to market.

Talk details

  • Title: Updates from Pharmaceutical Companies
  • Author(s): Tamara Agajanov, Frank Bennett, Ina Brünig-Traebert, Michelle Krishnan
  • Author(s)’ affiliation: Ovid Therapeutics; Ionis Pharmaceuticals; Roche
  • Publication date: 2019-09-06
  • Collection: 2019 ASF Family Conference