BBDF Blog

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.

Lyon, France – Austin, Texas, United States – November 19, 2024 – 6 PM CET – Theranexus, a biopharmaceutical

company innovating in the treatment of rare neurological diseases, and the Beyond Batten Disease Foundation

(BBDF) presented the final and positive results of the Phase I/II trial to evaluate Batten-1 in 6 young adult patients

with CLN3 Batten’s disease after 18 months of treatment, at the Child Neurology Society Annual Meeting in San

Diego, 11 to 14th November.

This presentation highlights the strong safety profile of miglustat in CLN3 Batten disease patients and underscores

the positive efficacy data gathered during this trial. The findings demonstrate clear target engagement, a

biological effect, and are suggestive of a stabilization of disease progression over the treatment period. All trial

participants chose to continue receiving miglustat through the BBDF’s early access program, with no safety

concerns reported after two years of ongoing treatment.

Lyon, France – Austin, Texas, United States – 6 June 2024 – 7:30am CT – Theranexus, a biopharmaceutical company innovating in the treatment of rare neurological diseases, and the Beyond Batten Disease Foundation (BBDF), confirm positive results in their Phase I/II trial to evaluate Batten-1 in Batten disease (CLN3) after 18 months of treatment. The results indicate a decline in serum neurofilament light chain (NfL), a biomarker of neuronal death, and confirm the therapeutic potential of the Batten-1 drug candidate in juvenile (CLN3) Batten disease.

Measurements of the concentration of neurofilament light chains (NfL), a recognized biomarker of neurodegeneration, after 18 months of treatment confirm the 12-month results presented at the International NCL2023 Congress in September 2023. This biomarker, measured in the serum of patients compliant to protocol, decreased by an average of 33% after 18 months of treatment (as a reminder, 32% after 12 months of treatment) compared to pre-treatment level.