Atsena Therapeutics LCA1 Gene Therapy Shows Early Promise
By Alex Keown
May 1, 2023
Durham-based Atsena Therapeutics posted positive six month safety and efficacy data from its ongoing Phase I/II trial assessing the company’s investigational gene therapy for the treatment of GUCY2D-associated Leber congenital amaurosis (LCA1).
LCA1 is a monogenic eye disease that disrupts the function of the retina and results in early and severe vision impairment or blindness. It is the most common cause of blindness in children, impacting two to three per 100,000. Caused by mutations in the GUCY2D gene, GUCY2D-LCA1 is one of the most common forms of LCA, affecting roughly 20% of patients. There are currently no approved treatments for LCA1 or its different forms.
The six-month readout, presented at the 2023 Association for Research in Vision and Ophthalmology meeting in New Orleans showed ATSN-101 demonstrated clinically meaningful improvements in vision at the highest dose. No drug-related adverse events were reported, Atsena Therapeutics announced.
ATSN-101 is a subretinal gene therapy designed to introduce the functional human GUCY2D to photoreceptors. In 2020, Atsena secured rights to the gene therapy from Sanofi, which originally licensed it from the University of Florida. In 2021, ATSN-101 received orphan drug designation from the FDA for the treatment of GUCY2D-associated LCA1.
In the Phase I/II ascending-dose study, six of the 15 patients were provided with the highest dose treatment. Data from those patients showed the mean change from baseline in retinal sensitivity by dark-adapted full-field stimulus testing was significantly greater in treated eyes compared with untreated eyes after 28 days. Two of the patients demonstrated best corrected visual acuity (BCVA) improvement greater than 0.3 logMAR. No treated eyes had a decrease in BCVA, the company said.
Additionally, Atsena reported that five of the high-dose patients underwent a multi-luminance mobility test (MLMT), which has participants navigating an obstacle course under different levels of illumination. Four of the patients who participated in the MLMT saw a maximum score compared to baseline, the company noted.
Kenji Fujita, chief medical officer of Atsena Therapeutics, expressed excitement about the positive results seen at the six month mark.
“The latest data reinforce our confidence in the potential of ATSN-101 to improve vision in patients with GUCY2D-associated LCA1, which results in early and severe vision impairment or blindness and lacks an approved treatment,” Fujita said in a brief statement. “We look forward to reporting 12-month data later this year and are exploring options to advance ATSN-101 into a pivotal trial.”
Atsena’s six-month data builds on previous reports regarding the safety and efficacy of ATSN-101. In October 2022, the company announced significant improvements in retinal sensitivity and BCVA in the treated eyes of trial patients dosed that summer. The majority of patients who took part in MLMT studies saw at least two-levels of improvement from baseline, the company noted.
The Durham-based company is now awaiting 12 month data from the Phase I/II study of ATSN-101. That data is expected at the end of the year.
ATSN-101 is the most advanced developmental asset in Atsena’s pipeline. The company is also planning to initiate clinical studies of two other gene therapies. ATSN-201 is being developed for X-linked retinoschisis, an inherited disease that causes loss of central and peripheral vision. ATSN-301 is being developed to prevent blindness due to MYO7A-associated Usher syndrome, an inherited disease that affects the retina and the inner ear.
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Alex Keown is a freelance journalist who writes about a variety of subjects including the pharma, biotech, and life science industries. Prior to freelancing, Alex has served as a staff writer and editor for several publications.
