View all newsletters
Receive our newsletter – data, insights and analysis delivered to you
In association with Cytiva for Cell & Gene Therapy coverage
  1. News
June 15, 2021

Biogen’s rare eye disease gene therapy falls short at Phase III

Biogen's late-stage study evaluating timrepigene emparvovec to treat choroideremia has failed to hit its main goals.

Biogen’s Phase III trial of its gene therapy, timrepigene emparvovec, did not meet its primary or key secondary endpoints in patients with the rare inherited eye disease choroideremia, the firm has announced.

The Phase III STAR study, a multicenter, randomized, three-arm, parallel-controlled group trial, evaluated the safety and efficacy of a single subretinal injection of the investigational gene therapy. It enrolled 169 adult males with a genetically confirmed diagnosis of choroideremia, a rare inherited retinal disease that results in progressive vision loss and eventually leads to blindness.

Timrepigene emparvovec is an investigational recombinant AAV2 vector ‘designed to deliver a functional version of the human choroideremia gene into the retinal pigment epithelium and photoreceptor cells that aims to address the underlying genetic cause of choroideremia’.

The primary endpoint of the study was the proportion of patients with an improvement of at least 15 letters from baseline in best-corrected visual acuity (BCVA) at 12 months post-treatment, as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart.

The study failed to meet this primary endpoint and saw that the gene therapy did not increase the number of patients demonstrating an improvement on this measure.

Additionally, the Phase III STAR study did not demonstrate efficacy on key secondary endpoints, although the safety results were consistent with earlier studies evaluating timrepigene emparvovec.

“We extend our deepest gratitude to all those who contributed to the STAR study, including the participants, investigators, site staff, and the broader choroideremia community,” said Biogen Therapeutics Development Unit head Katherine Dawson.

“While we are disappointed by the results of the STAR study, we are hopeful that the clinical insights gleaned from this study may help to shape therapeutic innovation for inherited retinal diseases including choroideremia, so that in the future there may be treatment options for the community affected by these debilitating disorders.”

Biogen said it will evaluate the complete data set before confirming next steps for the timrepigene emparvovec clinical development program. Detailed results of the study will be made available at a future scientific forum.

Cell & Gene Therapy Coverage on Clinical Trials Arena supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

Free Whitepaper

Secure the cell therapy supply chain from bench to bedside

The development of cell therapies is changing healthcare, delivering new hope to thousands of patients around the world. The vein-to-vein workflow for these therapies, however, is not without challenges, many of which will increase as we scale up to treat more patients. Download this free guide from Cytiva to learn more about the challenges and risks associated with the cryogenic supply chain for cell therapies, and how supply chain disruptions can best be mitigated.
by Cytiva Thematic

By clicking the Download Free Whitepaper button, you accept the terms and conditions and acknowledge that your data will be used as described in the Cytiva Thematic privacy policy By downloading this Whitepaper, you acknowledge that we may share your information with our white paper partners/sponsors who may contact you directly with information on their products and services.

Visit our privacy policy for more information about our services, how we may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

Topics in this article: ,
NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. Key drug pipeline and competitive landscape changes based on the latest clinical activity, sent every Tuesday. Curated analysis and data-driven insights on clinical trials strategy and operations, sent every Thursday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy


Thank you for subscribing to Clinical Trials Arena