Theranexus and Beyond Batten Disease Foundation Announce Strong Positive Real-World Data Supporting Batten-1 Efficacy for the Treatment of Batten Disease

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

 

Family Research Calls – May 13 & 15

Dear BBDF Family Partners,

Please join us on our next two family research calls. You will need to register for each call separately.

Tuesday, May 13 at 3 p.m. CST – we will be sharing an update on the Batten-1 clinical trial.

Please register to participate in the call here.

Thursday, May 15 at 2 p.m. CST – we will be sharing an update on the broader CLN3 research landscape, including current preclinical and clinical studies.

Please register to participate in the call here.

If you are not able to attend, we will make a recording available after the meeting.

Thanks so much for your continued support and partnership,

Mary Beth

 

Mary Beth Kiser

President & CEO

512.275.2600

Batten Disease Global Research Initiative Announces Inaugural Grant Recipients

March 18, 2025

Batten Disease Global Research Initiative Announces Inaugural Grant Recipients

The Batten Disease Global Research Initiative (BDGRI) is proud to announce the recipients of its inaugural Research Grant round, awarding a total of USD $320,000 to six groundbreaking projects worldwide.

Following a robust selection process by a Grant Review Panel of eight international Batten disease experts, the nominated projects each address one or more of the “Top 10 Research Priorities for Batten Disease” as identified in the BDGRI Research Strategy.

View the Full Press Release

Update on the Theranexus’ Batten-1 project in CLN3 Batten’s disease

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.

 

Read Full Press Release

 

 

NORD Batten Disease Patient Assistance Program Reminder

Reminder that NORD’s Batten Disease Premium & Copay Patient Assistance Program is open and accepting applications for assistance. NORD’s Batten Disease Patient Assistance Program offers eligible individuals diagnosed with Batten Disease financial support to pay for out-of-pocket healthcare costs that are directly related to the care and treatment of Batten. These Patient Assistance Programs for individuals diagnosed with Batten disease have been expanded. Eligible individuals may now apply for financial support with health insurance premiums and copay expenses. Learn more below.

PREMIUM & COPAY PROGRAM

We Need Your Help Re: Off-Label Miglustat Use

As we have previously reported, the Phase III trial for Batten-1 is experiencing some unanticipated delays due to funding issues. However, the program continues to be the highest priority for BBDF and Theranexus. Together, we are working hard to secure the significant financial resources necessary to see this program through to potential regulatory approval.

The delay in the Phase III portion of the Batten-1 clinical trial is providing an opportunity for us to further review the FDA’s feedback on the study design and discuss and consider alternative regulatory strategies that will seek to bring the drug to patients within the shortest possible time frame. To this end, we are working with Engage Health to collect information from patients who are currently using off-label miglustat in consultation with their medical practitioner. Please note that taking miglustat off-label will not exclude a patient from participating in a future trial, and this data may be helpful in informing our efforts and potentially revising the study design.

If your child is currently using off-label miglustat in consultation with your medical practitioner, we would be so grateful for your participation.

If you know of other families using miglustat off-label, please share this information with them.

We are anxious to get started on Phase lll. If you have any questions, please don’t hesitate to reach out to me.

Many thanks,
Mary Beth Kiser
President and CEO

Batten Disease Research Priorities (Phase 2) – YOUR HELP REQUESTED

Dear Batten community,

Phase 2 of the Batten disease research priority-setting process is underway and we need YOUR input.

Our colleagues at the BDSRA Foundation and Kennedy Krieger Institute in the USA are conducting an important 2-phase community survey to help inform and drive research priorities and investment in Batten disease.

Following the Batten community’s responses in Survey 1, a summary list of research questions about Batten disease has now been created. Please review the summary list and select the 10 research questions you think are most important for researchers to study. This anonymous survey is open to the ENTIRE Batten community and takes 10-15 minutes to complete.

Learn more by reading this flyer, and complete the survey by clicking the button below.

Please complete the survey by July 17, 2024.

Our sincerest thanks for your input into this important initiative.

Take the Survey

Theranexus and BBDF Confirm Positive 18-Month Results for Batten-1 in Phase I/II Trial Based on Neuronal Death Biomarker Values

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.

Read Full Press Release

 

 

Batten Disease Research Priorities – YOUR HELP REQUESTED

Dear Batten community,

The BDSRA Foundation and Kennedy Krieger Institute are conducting an important community-wide survey to help inform and drive research priorities and investment in Batten disease.

Parents, carers, clinicians, researchers, industry, policymakers, allied health, support workers, educators or anyone with an interest in Batten disease are warmly invited to participate in this anonymous survey and to share with their networks. Hurry! the deadline to take the survey is this week.

Please complete the survey by May 24, 2024.

Learn more by reading this flyer, and complete the survey by clicking the button below.

Our sincerest thanks for your input into this important initiative.

Warm regards,

Mary Beth Kiser

 

Take the Survey
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