Highly Promising 12-month results in the Phase I/II trial of Batten-1

September 29, 2023
News, Press Releases

Lyon, France – Austin, Texas, United States – 29 September 2023 – 7.30am – CET – Theranexus, a biopharmaceutical company innovating in the treatment of rare neurological diseases, and the Beyond Batten Disease Foundation (BBDF), present very encouraging interim results for efficacy and safety after 12 months of treatment in their Phase I/II trial for Batten disease (CLN3) at NCL2023, the International

For Professor Gary Clark, the trial’s principal investigator and Chief of Child Neurology at Texas Children’s Hospital in Houston, “The Phase I/II results for Batten-1 are highly promising, as we have observed a reduction in neurofilaments and glycosphingolipids and no notable progression of motor symptoms after 12 months of treatment in our 6 patients aged 17 years and over. This is unprecedented in the indication, and Batten-1 is thus a source of great hope for children affected by the disease and for their families”. Professor Clark will be presenting the 12-month interim efficacy and safety results at the “Translational Research Clinical” session on Friday 29 September at 12.25pm (CEST).

The 12-month treatment results further support the 6-month results announced in mid-June 2023. The new results, achieved after 12 months of treatment, show an average 32% decline in neurofilament light chain (NfL) levels in patient serum (as a reminder, 17% after 6 months’ treatment). Neurofilaments are a recognized biomarker of neurodegeneration. A 64% reduction in this biomarker is also observed in the cerebrospinal fluid (CSF). After twelve months of treatment, in line with preclinical data the results also confirmed a reduction in the glycosphingolipids involved in the disease (Gb3 reduction of 45%) which, when they accumulate, are toxic to neurons. Clinically, there was notably less worsening of motor symptoms evaluated by the modified UBDRS physical assessment subscale after 12 months than what is expected in this naturally progressive disease.

Dang Do AN, et al. Neurofilament light chain levels correlate with clinical measures in CLN3 disease. Genet Med. 2021 Apr;23(4):751-757

 

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Rare Sibling Stories Looking for Stories!

August 15, 2023
News

Rare Sibling Stories is looking for siblings to highlight on their platforms! They would love to feature siblings of individuals with Batten disease. Siblings (any age) just need to fill out a short survey with this link: https://forms.gle/SbogDffzfphoAFjj7

They created Rare Sibling Stories to provide a space for siblings of individuals with rare diseases to share their stories and connect with others like themselves. Rare Sibling Stories is gathering information from siblings to highlight on the Rare Sibling Stories website and social media!

NORD’s Batten Disease Patient Assistance Program

July 17, 2023
News

Having a rare disease is difficult. Adding in the complex care required to treat or manage that disease and figuring out how to pay for it makes a rare diagnosis even harder. 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 as well as Emergency Relief (critical, non-medical needs). Eva’s Butterfly Wishes for rare children may also be available. Learn more below.

 

PREMIUM & COPAY PROGRAM

 

EVA’S BUTTERFLY WISHES® FOR RARE CHILDREN

 

EMERGENCY RELIEF PROGRAM

 

BBDF Grants $60,000 Over 3-Years to Support Ineka Whiteman, Ph.D., Head of Research and Medical Affairs

July 17, 2023
Research

BBDF grants $60,000 over 3-years to the BDSRA Foundation to support Ineka Whiteman, Ph.D. to serve as the Head of Research and Medical Affairs. With this support, Dr. Whiteman is able to focus on Batten research full-time. In addition, Ineka serves as a scientific consultant to BBDF and the Head of Research and Medical Affairs for BDSRA Australia. Her leadership brings a more cohesive approach and fosters greater partnership and collaboration across all CLN3 initiatives. Click here to read her bio.

Encouraging preliminary 6-month results in the Phase I/II trial of Batten-1 in Batten disease (CLN3)

June 14, 2023
Research, News, Press Releases

Lyon, France – Austin, Texas, United States – 14 June 2023 – 7.30am CEST – Theranexus, a biopharmaceutical company innovating in the treatment of rare neurological diseases, and the Beyond Batten Disease Foundation (BBDF), have today announced encouraging preliminary results achieved with their drug candidate, Batten-1, in juvenile Batten disease as part of the Phase I/II ongoing trial after 6 months of treatment. These preliminary results suggest an effect of Batten-1 on neuronal death and initial signs of clinical efficacy.

Theranexus and BBDF are conducting a Phase I/II trial of their drug candidate in six patients with Batten disease (CLN3) aged 17 years and over who are due to receive treatment over a 24-month period. The first results of this Phase I/II trial after 9 weeks of treatment showed a good tolerability and pharmacokinetic profile for Batten-1. The new preliminary results, achieved after 6 months of treatment, show an average 17% decline in neurofilament light chain (NfL) levels in the blood of dosed patients. Neurofilaments are a recognized biomarker of neurodegeneration. Moreover, motor symptoms assessed by the modified physical subscale of the disease- specific UBDRS did not progress over the same period (mean score of 31.8 after 6 months of treatment as against 32.4 at baseline), whereas according to data available describing the natural course of the disease, this clinical score should have progressedby around one point in these patients. Lastly, the drug candidate continues to show a good tolerability profile after six months of treatment.

Read the full press release here.

Expert Insights on Gene Therapy for International Batten Awareness Day

June 10, 2023
Research, News

Neuronal ceroid lipofuscinosis (NCL) is a group of 13 rare inherited lysosomal disorders affecting the nervous system which are collectively referred to as Batten disease.1 According to the Batten Disease Support and Research Association (BDSRA), Batten disease is estimated to occur in approximately 2 to 4 in 100,000 births in the United States; however, the BDSRA indicates that the actual occurrence may be even higher.

Several companies are currently developing gene therapies intended to treat specific forms of Batten disease. Among these are REGENXBIO, which is developing RGX-181 and RGX-381 for the CLN2 subtype; Lexeo Therapeutics, which is developing LX1004 for the CLN2 subtype; and Taysha Gene Therapies, which is developing TSHA-118 for the CLN1 subtype.2-4 RGX-381 is specifically intended to address the ocular manifestations of CLN2; as of March 30, 2023, a phase 1/2 clinical trial (NCT05791864) for the therapy is active, but not yet recruiting. RGX-181 remains in preclinical development. According to Lexeo and Taysha’s respective pipeline overviews, LX1004 and TSHA-118 have entered clinical development.

Read the full article here.

Theranexus and BBDF granted positive opinion by EMA for the design of Phase III trial to evaluate Batten-1 in CLN3 Batten disease

June 7, 2023
Research, News, Press Releases

Lyon, France – Austin, Texas, United States – 7 June 2023 – 7.30 am CEST – Theranexus, a biopharmaceutical company innovating in the treatment of rare neurological diseases, and the Beyond Batten Disease Foundation (BBDF), have today announced receipt of a positive opinion from the European Medicines Agency (EMA) for the design of the pivotal Phase III trial to evaluate their Batten-1 drug candidate for Batten disease.

Read the full press release here.

Families raised millions and handed rare disease therapies off to biotechs. But companies have backed out

May 16, 2023
News

When Julia Taravella’s two sons were diagnosed with an ultra-rare disease, she did something extraordinary but not uncommon among parents like her: She formed a group that raised $1.2 million to try and find a treatment.

“Without a therapy, my sons will die,” Taravella said.

The money helped launch work on a potential gene therapy at the University of North Carolina, which was then handed off to a Texas-based company called Neurogene.

But what sounds like a victory propelled by a parent’s perseverance is being overshadowed by a lesserknown but increasingly common trend: Drugmakers are shelving many of those family-funded therapies.

In 2022, almost four years after Neurogene took over, the company stopped the work. The program is back with the University of North Carolina, and Taravella and other parents are trying to raise another $2.3 million for a clinical trial.

Read the full article here.

Theranexus and BBDF win FDA approval on efficacy endpoints for the Phase III trial to evaluate Batten-1 in CLN3 Batten disease

May 9, 2023
Research, News, Press Releases

Theranexus and BBDF win FDA approval on efficacy endpoints for the Phase III trial to evaluate Batten-1 in CLN3 Batten disease

  • Phase III’s primary endpoint will be visual acuity, with secondary endpoints including assessment of cognitive and motor functions,
  • The FDA confirmed that this sole Phase III trial would secure Batten-1 approval for Batten CLN3 disease.

Lyon, France – Austin, Texas, United States – 9 May 2023 – 7.30am CEST – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and the Beyond Batten Disease Foundation (BBDF), have today announced receipt of approval from the Food and Drug Administration (FDA) for the design and primary and secondary endpoints of the pivotal Phase III trial for Batten disease CLN3, at a meeting with the Division of Rare Diseases and Medical Genetics (DRDMG) held in mid-April.

“We are delighted with the constructive discussions we had with the FDA on defining the endpoints of Batten-1 and the design of our pivotal Phase III trial. Its valuable guidance allows us to get fully prepared for the trial launch,” explained Theranexus’ Chief Medical Officer Marie Sebille.

“We would like to thank the FDA for its support, which is fundamental to the development of our Batten-1 drug candidate, the only asset in active clinical development for Batten disease (CLN3). Our pivotal Phase III trial will enable us to gain approval for Batten-1, and also deliver rich news flow throughout the duration of the Phase III trial thanks to the parallel open-label patient cohort. More generally, we have no doubt that we can deliver a therapeutic response for patients suffering from Batten disease,” added Theranexus’ CEO Mathieu Charvériat.

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My son’s time is running out due to a rare disease. The FDA needs to add more clinical trial flexibility

March 2, 2023
Family Stories

By Judy Stecker
Feb. 28, 2023

My toddler, Wheeler, will probably not live to adulthood. Juvenile Batten disease — he has the type known as CLN3 — is stealing his childhood. And then this rare disease will steal my child.

Wheeler is missing the DNA needed to recycle a waste product called lipofuscin that his cells naturally produce. As lipofuscin builds up, his condition will get worse, robbing him of his eyesight, his speech, his mobility, and ultimately his life.

Our only hope is finding a treatment to cure, or at least a way to slow down this unrelenting disease. At age 3, Wheeler already has sleep and behavior problems, as well as delays in his speech and fine motor development. He recently began moving closer to the TV and rubbing his eyes, signals that in a matter of months or weeks he may soon live the rest of his life blind.

 

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