Lyon, France – Austin, Texas, United States – Mai 13, 2025 – 6.00 pm CET –Theranexus, a
biopharmaceutical company developing drug candidates for rare neurological diseases and the Beyond
Batten Disease Foundation (BBDF), today announced new real-world data strongly supporting the efficacy of
Batten-1 (miglustat) as a treatment for CLN3 disease, also known as juvenile Batten disease.
The analysis evaluated visual acuity outcomes in CLN3 patients treated with Batten-1 (miglustat, n=11),
versus untreated patients (n=22), using real-world data collected from natural history studies, family
interviews, published reports, and prescribers’ personal communications. This analysis focused on patients
with measurable visual acuity at baseline (visual acuity score ≤ 1.9 LogMAR) and compared the progression
of visual function between groups over a 12-month period. To ensure an appropriate comparison between the
treated and untreated groups, a propensity score methodology was applied.
The analysis demonstrated a statistically significant and clinically meaningful difference in favor of
Batten-1 treatment on visual function with a notable preservation of visual acuity in treated patients
compared to untreated patients. The notable preservation of visual acuity in treated patients compared to
untreated patients add to the positive results from the Phase 1/2 study of Batten-11 and underscore the
potential of miglustat to address critical unmet needs in Batten disease. The visual acuity endpoint used in
the real-world analysis is the same as that selected for the planned pivotal Phase 3 clinical trial of Batten-1,
a design endorsed by the U.S. Food and Drug Administration (FDA)2 and the European Medicines Agency
(EMA)3.
“We are deeply grateful to the patients, their families, and the healthcare providers who made this important
analysis possible by agreeing to share their experiences and those unique clinical data. This collaboration has
been essential in demonstrating Batten-1 potential to change the course of CLN3 disease” explains Craig
Benson, Chairman of the Beyond Batten Disease Foundation.
“Achieving near stabilization of visual acuity over 12 months in CLN3 patients is truly remarkable and
unprecedented. In this population, we would typically expect a relentless decline in vision. These results offer
real hope for altering the natural progression of the disease,” commented Dr. Gary Clark, MD, Chief of
Neurology and Developmental Neuroscience at Baylor College of Medicine, Houston, Texas.
“These results have been observed using the primary efficacy endpoint defined in our Phase 3 trial, this is
highly reassuring. These findings strongly support our development strategy and reinforce the relevance of our
endpoint in demonstrating Batten-1 potential efficacy” concluded Marie Sebille, Chief Medical Officer at
Theranexus.
View the Full Press Release