July/
August 2019

Positive 2-Year Results Announced in LHON Gene Therapy Trial

In the phase 3 REVERSE clinical trial, patients with Leber hereditary optic neuropathy (LHON) treated with GS010 (GenSight Biologics) showed continued efficacy at nearly 2 years after initial treatment, according to the company. The 96-week top-line data were announced in a press release.

Mean improvement in BCVA from baseline was +15.4 ETDRS letters in GS010-treated eyes, according to GenSight. Patients were treated with a single injection of the gene therapy in one eye within 6 to 12 months of disease onset.

Of note, bilateral improvement in both BCVA and contrast sensitivity has been noted.

“The data show that both the treated and the sham eye improved in both high and low contrast, defying the accepted natural history of this disease and improving upon it,” said Robert C. Sergott, MD, chief of the Wills Eye Hospital neuro-ophthalmology service and director of the new Annesley EyeBrain Center at Thomas Jefferson University, both in Philadelphia. “The behavior of the untreated eye must also make us re-examine what we thought we knew as possibly dogma and be open to the idea that gene therapy delivered into one eye may be able to access the contralateral eye.”

Results of a second phase 3 trial, RESCUE, which includes patients who have had LHON for 6 months or less, will be available by the end of the third quarter of this year, according to the company. A third phase 3 study for the same condition, REFLECT, is ongoing. Patients in that trial will be treated in both eyes.

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Congressional Bill Aims to Streamline and Improve Transparency in Prior Authorization

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