Pharma and Biotech Industry Update Aug 2021

Quick Overview

The Pharma and Biotech Industry Update for August 2021 focused on the development of potential treatments for Angelman syndrome. Ionis Pharmaceuticals is developing antisense oligonucleotides (ASOs) for the disease and plans to initiate a clinical trial by year-end. Roche’s drug, TANGELO, is in Phase 1 and has shown improvements in some participants. GeneTX’s GTX-102 has completed Phase 1 and is planning for Phase 2. Neuren Pharmaceuticals’ NNZ-2591 has shown potential to improve impairments in preclinical and Phase 1 studies. PTC Therapeutics is leading a study to investigate UBE3A protein as a biomarker for Angelman syndrome. Taysha Gene Therapies is focused on treatments for monogenic diseases of the central nervous system. The Angelman Syndrome Natural History Study is collecting data to understand the disease over time.

Introduction

Welcome to the Pharma and Biotech Industry Update for August 2021. In this talk, we will provide a summary of the key points discussed during the scientific talk at the 2021 ASF Virtual Family Conference.

Conference Information

• The conference organizers are not able to have a live Q&A session, but attendees can submit questions in the Q&A box or chat.
• Attendees are encouraged to provide feedback by texting ASF to 90412, as it will result in a donation to the Angelman Syndrome Foundation.

Ionis Pharmaceuticals

• Ionis Pharmaceuticals is focused on developing antisense oligonucleotides (ASOs) as therapies for various diseases, including Angelman syndrome.
• Ionis has a team dedicated to their Angelman Drug Development Program, led by Becky Crean.
• Ionis has been working on Angelman syndrome since 2012 and has validated the ASO approach in animal models.
• Ionis is planning to initiate a clinical trial with their lead compound by the end of the year.
• Ionis is partnering with Roche and Biogen on a natural history study to collect cerebrospinal fluid samples from individuals with Angelman syndrome.
• Ionis aims to bring the best drug forward and is committed to delivering an ASO to the Angelman community.

Roche

• Roche is also involved in the development of a drug for Angelman syndrome called TANGELO.
• TANGELO is currently in Phase 1 and has enrolled 32 participants.
• TANGELO has shown improvements in communication and other functional domains in some participants.

GeneTX

• GeneTX is developing GTX-102, an antisense oligonucleotide for Angelman syndrome.
• GTX-102 has completed a Phase 1 study and is planning to start a Phase 2 study.
• GTX-102 targets the RNA that silences the paternal copy of the UBE3A gene, aiming to increase UBE3A production in the brain.
• Preliminary data from GTX-102 showed improvements in communication, motor skills, and behavior in some participants.

Neuren Pharmaceuticals

• Neuren Pharmaceuticals is developing NNZ-2591 as a potential treatment for Angelman syndrome.
• NNZ-2591 is a synthetic analog of cyclic glycine proline, which regulates IGF-1 levels in the brain.
• Preclinical studies have shown that NNZ-2591 can correct deficits in synaptic plasticity and improve symptoms in a mouse model of Angelman syndrome.
• Neuren has completed a Phase 1 study in healthy adults and is planning to start a Phase 2 study for Angelman syndrome.
• NNZ-2591 has shown potential to improve impairments across a range of symptoms in preclinical and Phase 1 studies.

PTC Therapeutics

• PTC Therapeutics is a multinational biopharmaceutical company focused on developing treatments for orphan diseases.
• PTC’s gene therapy program in Angelman syndrome is part of a collaborative research partnership with Ionis and Roche.
• The AS CSF collection study is being led by PTC and aims to investigate the use of UBE3A protein as a biomarker for Angelman syndrome.
• The study requires participants to have a genetically confirmed diagnosis of Angelman syndrome and a planned procedure requiring anesthesia or conscious sedation.
• The study is being conducted at five centers in the US and aims to enroll approximately 20 participants.

Taysha Gene Therapies

• Taysha Gene Therapies is focused on finding potential treatments for patients with monogenic diseases of the central nervous system.
• Taysha has deep-rooted experience in drug development and a partnership with UT Southwestern.
• The company is using proven approaches to bridge the gap between innovation and treatment.
• The name “Taysha” means ally or friend, reflecting the company’s commitment to partnerships and the rare disease community.

Angelman Syndrome Natural History Study

• The Angelman Syndrome Natural History Study is important for the development of therapeutic products and understanding the disease over time.
• The study has made changes to streamline the visit process and reduce the burden on participants and caregivers.
• The study is collecting data on outcome measures used in current and future clinical trials, including the Bayley scales, Vineland scales, and ORCA.
• An off-site study visit will be conducted in Princeton, NJ in September 2021.
• Participants can contact the study coordinator for more information and to express interest in participating.
• The study has enrolled 159 participants across multiple sites in the US and Canada.
• The study is still heavily biased towards younger participants, and more adults with Angelman syndrome are needed in the study.
• The study is grateful for the support of the Angelman Syndrome Foundation, Foundation for Angelman Syndrome Therapeutics, and RTI International.
• The study staff at Boston Children’s Hospital includes Katie Anderson, Johnna O’Sullivan, and Liana Cole.

Conclusion

In conclusion, the Pharma and Biotech Industry Update for August 2021 highlighted the progress and ongoing research in the development of potential treatments for Angelman syndrome. The collaboration between pharmaceutical companies, research institutions, and patient advocacy groups is crucial in advancing the understanding and treatment of this rare genetic disorder.