Batten-1 May Preserve Vision with Juvenile Batten Disease
Findings in Phase 1/2 clinical trial, access program also support miglustat’s safety.
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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.
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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.
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 SurveyTheranexus 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.
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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
Theranexus Announces Positive Data on Efficacy and Safety in the Phase I/II Trial of Batten-1
Theranexus Announces Positive Data on Efficacy and Safety in the Phase I/II Trial of Batten-1
Stabilization of motor symptom progression in young adult patients suffering from juvenile Batten disease (CLN3) after 18 months of treatment
Lyon, France – Austin, Texas, United States – 17 April 2024 – 3pm CET – Theranexus, a biopharmaceutical company innovating in the treatment of rare neurological diseases and the Beyond Batten Disease Foundation (BBDF), today announce final positive data on efficacy and safety in the Phase I/II trial of its drug candidate Batten-1 in juvenile Batten disease (CLN3) after 18 months of treatment.
The 18-month safety and efficacy data confirm the 12-month results announced in September 2023. Batten-1 presented a good safety profile. On average, in the 6 young adult patients, treated with Batten-1, progression of motor symptoms was considerably slowed down and appeared stable compared to progression in untreated patients, as evaluated by the modified UBDRS Physical Assessment score. The mean change from baseline was +1,83 for the six subjects treated over 18 months vs +6,04 in untreated subjects from the natural history study conducted by the University of Rochester (n=46).
For Professor Gary Clark, the trial’s principal investigator and Chief of Child Neurology at Texas Children’s Hospital in Houston, “The data collected after 18 months of treatment with Batten-1 further reinforce its highly promising potential. We currently no longer observe a marked progression of motor symptoms in the 6 patients treated. These results support the prospect of a major benefit of Batten-1 for the children with this very severe disease and for their families”.
For Theranexus’ CEO, Mathieu Charvériat: “These positive results on efficacy and safety of Batten-1 are highly encouraging for the patients and their families. Together with BBDF and the investigators we are initiating an Expanded Access Program (EAP) to enable the 6 patients previously in the trial to continue receiving the treatment, considering its favorable risk-benefit profile. These results highlight the strong therapeutic potential Batten-1 on the clinical course of the disease. In this context, we are exploring different funding options to ensure we have the adequate resources to launch a phase 3 pivotal trial, which design has already received positive opinions by the FDA and the EMA”.
As a reminder, the Phase I/II trial conducted by Theranexus and BBDF to evaluate their drug candidate, Batten-1, included six patients with juvenile Batten disease (CLN3) aged 17 years and over. The patients were treated for 18 months. Following their participation in the trial, all patients are offered the possibility to continue receiving the treatment through a compassionate use program.
About Batten-1
Batten-1 is a novel and exclusive proprietary drug containing the active ingredient miglustat. The mechanism of action of this substance blocks the accumulation of glycosphingolipids and neuroinflammation, thus significantly reducing neuronal death that contributes to a progressive loss of function in patients. For patients over 17 years of age in the Phase I/II trial, the product is administered in solid form. In the Phase III trial, it will be administered in a liquid form better suited to pediatric patients.
Phase I/II trial design: this is an open-label trial involving 6 patients over 17 years of age with CLN3 Batten disease, treated with miglustat up to 600 mg/day for an 18-month period. The primary endpoint is patient safety and tolerability, assessed using reports of adverse effects, biological tests and ECG, as well as the pharmacokinetics of miglustat. The secondary endpoints include biomarkers (NfL, glycosphingolipds), efficacy monitoring: Unified Batten Disease Rating Scale, visual acuity, measurement of brain volumes by MRI and measurement of the thickness of the neuronal layer of the retina by optical coherence tomography scans. Administration of Batten-1 in escalating doses with a maximum of 600 mg/day was well tolerated, with no severe side effects observed causing treatment discontinuation. The most commonly reported adverse events are reversible gastrointestinal effects of often light to moderate severity, thus demonstrating the good tolerability profile of Batten-1 in this population. Further information about the trial is available on https://clinicaltrials.gov/ct2/show/NCT05174039.
About Batten disease
Juvenile Batten disease, also known as Spielmeyer-Vogt or CLN3 disease, is a rare, fatal, inherited disorder of the nervous system for which there is no treatment or cure. Juvenile Batten disease belongs to a group of disorders referred to as neuronal ceroid lipofuscinoses (NCLs). Over 400 different errors in 13 genes have been attributed to various forms of NCL, which differ from one another primarily by when symptoms first appear. The first symptom in the juvenile form, progressive vision loss, appears between the ages of 4 and 6 and is followed by cognitive disorders, behavioral disorders, and motor disorders. Seizures commonly appear within 2-4 years of the onset of disease. Over time, patients continue to decline mentally and physically. Eventually, those affected become wheelchair-bound, are bedridden, and die prematurely.
Juvenile Batten disease is always fatal; usually by the late teens to early 20s. In the United States and Europe, the juvenile form is the most common of the NCLs, which together, affect nearly 2,000 patients[1]. In pathophysiological terms, interactions between neurons and glial cells play key roles in the emergence and progression of all the NCLs.
About Beyond Batten Disease Foundation
Beyond Batten Disease Foundation (BBDF) is the world’s largest nonprofit organization dedicated to funding research for a treatment and cure for juvenile (CLN3) Batten disease. Since its inception in 2008, over $35 million has been invested in research by leveraging donations, co-funding and strategic partnerships. BBDF is spearheading a unique, cohesive strategy, incorporating independent scientific resources and collaboration with related organizations to drive research in juvenile Batten Disease. Today there is a treatment in sight. BBDF funded research has discovered a drug – Batten-1 – that slows the progression of the disease in Batten models. More information can be found at www.beyondbatten.org.
About Theranexus
Theranexus is an innovative biopharmaceutical company that emerged from the French Alternative Energies and Atomic Energy Commission (CEA). The company has a unique platform for the identification and characterization of advanced therapy drug candidates targeting rare neurological disorders and an initial drug candidate in clinical development for Batten disease.
Theranexus is listed on the Euronext Growth market in Paris (FR0013286259- ALTHX).
[1] National Organization for Rare Disorders (NORD)/Orphanet
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Wall Street Journal Article by Judy Stecker
Pediatric rare disease drug development relies on the commitment, motivation, and tenacity of the parents. BBDF is grateful for our partnership with rare disease families who are advocating for treatments for Batten disease. Congratulations to Batten mom, Judy Stecker, co-founder of Wheeler’s Warriors at BBDF, for articulating some of the challenges faced by rare diseases.
The FDA Could Help Save My Son From a Rare Disease
My eldest son, Wheeler, has a rare genetic disease that will steal his vision, mobility, memory and, ultimately, his life. Every morning when he leaves for preschool, I worry it may be the last time he’ll be able to see my face.
One in 10 Americans have rare diseases— defined as affecting fewer than 200,000 people in the U.S.—and 95% of these afflictions lack a Food and Drug Administration-approved treatment or therapy. Three in 10 children affected by rare diseases won’t live to see their fifth birthday. Wheeler turns 5 in May.
When my son was diagnosed with CLN3 juvenile Batten disease at 4 weeks old, research into possible therapies offered hope. That hope is all but gone. The Beyond Batten Disease Foundation’s planned 2023 Phase III clinical trial of a potential treatment is in limbo, seeking funding. (My family supports the foundation.) Earlier this month, Amicus Therapeutics abandoned its pursuit of the only proposed gene therapy.
These developments are devastating but unsurprising. Given this, the anti-innovation sentiment in Washington and unnecessary bureaucracy at the FDA have made it difficult for companies to justify investments in rare-disease therapies.
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Amicus Discontinuing Their CLN6 program
Like many in our Batten community, we are disappointed to hear that Amicus have chosen to discontinue their CLN6 program and now their CLN3 gene therapy programs. We are encouraged, however, to learn that Nationwide Children’s Hospital has expressed its commitment to continue moving both of these programs forward. We look forward to seeing the ongoing development of the programs, and plan to follow up with Nationwide to understand their plans for the CLN3 program and how BBDF might collaborate.
In the meantime, we continue working very hard to advance our Batten-1 study to Phase III enrollment and look forward to sharing another update on this with our community soon.