Atsena Therapeutics released positive 6-month safety and efficacy data from its ongoing phase 1/2 trial on ATSN-101 for the treatment of autosomal recessive guanylate cyclase 2D (GUCY2D) -associated Leber Congenital Amaurosis (LCA1).
Refresh me on Atsena.
The clinical-stage gene therapy company targets the development of novel treatments for inherited forms of blindness, including LCA1, X-linked retinoschisis (XLRS), and MYO7A-associated Usher syndrome (USH1B).
Atsena’s next-generation, adeno-associated virus (AAV) technologies feature laterally spreading capsids, dual vectors, and intravitreal capsids.
Talk about ATSN-101.
ATSN-101 is an investigational gene therapy candidate developed to introduce the functional human GUCY2D to photoreceptors—potentially treating LCA1.
Administered via a single subretinal injection, the therapy is an AAV5 capsid and human rhodopsin promoter.
Now talk about this trial.
The phase 1/2 trial (NCT03920007) enrolled 15 patients (including 3 pediatric) that were divided into three adult cohorts (with 3 patients each) to be treated with three ascending doses of ATSN-101 via subretinal injection.
Of note, an additional 6 patients were given the highest dose treatment in a dose expansion phase.
Findings?
For the 9 patients receiving the high dose in the main trial, the mean change from baseline in retinal sensitivity was reported to be significantly greater in treated eyes vs untreated eyes at Day 28 (as well as later follow-up visits) using dark-adapted, full-field stimulus testing (FST).
Further, 2 high-dose patients achieved an improvement in best-corrected visual acuity (BCVA) that was greater than 0.3 logMAR (compared to none in untreated eyes).
Anything else?
For the 5 high-dose patients tested with the multi-luminance mobility test (MLMT), 4 presented with a maximum MLMT score of 6 or a ≥2 level improvement compared to baseline.
Any adverse reactions?
The company reported no drug-related serious adverse events were reported; ocular inflammation was noted as infrequent, minimal, and reversible with steroid treatment.
What’s next?
According to Atsena CMO Kenji Fujita, MD, the company is “looking forward to reporting 12-month data later this year and are exploring options to advance ATSN-101 into a pivotal trial.”
Per Clinical Trials, the study is expected to be complete by May 2027.