Biogen Ends AGTC Collaboration After Data from Gene Therapy Study in Retinoschisis Shows No Sign of Clinical Activity

December 14, 2018

Rare Daily Staff

Applied Genetic Technologies Corporation said Biogen terminated its collaboration after it reported topline interim data from its phase 1/2 clinical trial of its experimental AAV-based gene therapy for X-linked retinoschisis (XLRS), a rare genetic eye disease, showed no clinical activity.

AGTC said the study did show its gene therapy, known as rAAV2tYF-CB-hRS1, is generally safe and well-tolerated. The company said it will continue to monitor enrolled patients at scheduled visits through the end of the study.

As a result of Biogen’s decision to terminate the collaboration with the company, AGTC will regain full rights to the XLRS and X-linked retinitis pigmentosa (XLRP) programs and the three other partnered discovery programs. AGTC said it will complete patient monitoring activities on the XLRS program according to the clinical protocol but will not further develop the product.

AGTC plans to advance its XLRP program as previously disclosed and will determine next steps for the three discovery programs over the next several months.

“The data from the XLRS trial support the general safety and tolerability of our gene delivery platform and further reinforce our confidence in our ongoing phase 1/2 trials in achromatopsia (ACHM) and XLRP,” said Sue Washer, president and CEO of AGTC. “Through this study, we have deepened our understanding of the challenges that patients with inherited ophthalmic diseases face every day. It has also provided us with valuable information and insights in designing and analyzing clinical trials for inherited retinal diseases.”

AGTC’s two ongoing clinical programs in ACHM are proceeding as previously disclosed and the company is expecting data readouts from all three trials in 2019. We will also regain control of the preclinical programs in adrenoleukodystrophy (ALD) and two ophthalmic programs. After reviewing all available data for these programs, we will decide which to independently move forward into IND-enabling studies.”

As of September 30, 2018, the company’s cash, cash equivalents and investments totaled $105.4 million. The company believes these funds will be sufficient to allow AGTC to generate data from its ongoing clinical programs, to move the preclinical optogenetic program in collaboration with Bionic Sight into the clinic and fund the currently planned research and discovery programs for at least the next two years.


December 14, 2018
Photo: Sue Washer, president and CEO of AGTC

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