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BBDF 101

Lyon, September 9, 2021 – Theranexus, an innovative biopharmaceutical company in the treatment of neurological diseases, and Beyond Batten Disease Foundation (BBDF), announce that they have received Investigational New Drug (IND) approval from the Food and Drug Administration (FDA) to launch a study Phase I / II clinical trial of their BBDF 101 drug candidate for juvenile Batten disease1, a rare and fatal genetic disease.

With the IND FDA approval for BBDF 101, we have taken an important step in the drug’s regulatory development plan. This approval will allow us to start the clinical development of BBDF 101 in Batten disease, for which there is currently no treatment. Our goal is to be able to offer this treatment to patients with Batten disease as quickly as possible ”, explains Franck Mouthon, CEO of Theranexus.

At the end of 2019, Theranexus and BBDF signed an exclusive, worldwide license agreement for the development and commercial use of the BBDF 101 drug candidate for Batten disease by the company. BBDF 101, which received the orphan designation in Europe and the United States, is a drug candidate combining trehalose and miglustat, two active substances each having its own specific activity of interest for the disease.

The clinical program will begin with Phase I / II by the end of 2021. This will involve the recruitment of an initial adolescent / adult cohort of six patients who will receive the drug BBDF 101 as a dose exploration, with the tolerability and pharmacokinetics of trehalose, miglustat and a combination of trehalose / miglustat established on

4.5 months. The initial phase of the trial will be followed by a maintenance phase assessing patient safety and efficacy for a period of up to two years during which they will continue to receive BBDF 101 and will do the same. subject to security monitoring.

Phase III will begin after the evaluation of pharmacokinetics and tolerability in phase I / II and after consultation with the FDA. A pediatric cohort of 30 patients will be included in the trial and will undergo regular examinations to assess disease progression. The objective will be to compare the evolution of various symptoms in these patients, according to the evaluation of the UBDRS2 (neurological and behavioral symptoms, activities of daily living, etc.), over a period of two years, to a natural course of the disease documented on the basis of cohorts of patients already followed, over a period of two years. The phase III trial will begin in 2022.

The primary investigative centers for the trial will be Texas Children’s Hospital (TCH) in Houston, which is the fourth-largest pediatric hospital in the United States, the National Institutes of Health (NCIHD3 – Bethesda, Maryland), and the University of Rochester Batten Center (URBC), New York. These institutions are the primary referral centers for Batten disease in the United States. URBC and NIH have previously established Batten disease patient cohorts to assess the natural history of the disease.

  • Batten’s disease belongs to a group of disorders called neuronal ceroid lipofuscinosis (NCL).
    2 Unified Slat Disease Rating Scale
    3 National Institute of Child Health and Human Development (NICHD)

This approval offers immense hope for all patients – children, adolescents and adults – with Batten disease and their families for whom no medical solution currently exists. We are extremely grateful for the support of the Batten families and our communities in making this milestone a reality, Craig Benson concludes, Co-founderand Chairman of the Board of Directors of BBDF.

About Batten disease

Juvenile Batten disease, also known as Spielmeyer-Vogt disease or CLN3, is a rare, fatal, inherited disease of the nervous system for which there is no treatment or cure. Juvenile Batten disease belongs to a group of disorders called neuronal ceroid lipofuscinosis (NCL). Over 400 different errors in 13 genes have been attributed to various forms of CNL, which differ from each other primarily in when symptoms first appear. The first symptom of the juvenile form, progressive vision loss, appears between the ages of 4 and 6, followed by personality changes, behavioral problems and slow learning. Seizures usually appear within 2 to 4 years of the onset of the disease. Over time, patients continue to decline mentally and physically. Eventually, those affected end up in wheelchairs, are bedridden and die prematurely. Psychiatric symptoms or psychosis can appear at any time. Juvenile Batten disease is always fatal; usually in their late teens to early twenties. In the United States and Europe, the juvenile form is the most common NCL which, together, affects nearly 3,000 patients4 In pathophysiological terms, interactions between neurons and glial cells play a key role in the emergence and progression of all NCLs.

About the Beyond Batten Disease Foundation

Beyond Batten Disease Foundation (BBDF) is the world’s largest non-profit organization dedicated to funding research for a treatment and cure for juvenile Batten Disease (CLN3). Since its creation in 2008, more than $ 35 million has been invested in research by mobilizing donations, cofinancing and strategic partnerships. BBDF is spearheading a unique and cohesive strategy, integrating independent scientific resources and collaboration with related organizations to spur research on juvenile Batten disease5. Today, a cure is in sight. BBDF-funded research discovered a drug that slows disease progression in Batten models. More information can be found at

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Thursday, September 30, 2021: Financial results for the first half of 2021


Theranexus is a clinical stage biopharmaceutical company created by the French Alternative Energies and Atomic Energy Commission (CEA) in 2013. It develops drug candidates for the treatment of diseases of the nervous system. Theranexus has identified the key role that non-neuronal cells (also called “glial cells”) play in the body’s response to psychotropic drugs (which target neurons). The company is a pioneer in the design and development of drug candidates affecting the interaction between neurons and glial cells. The unique and patented technology used by Theranexus is designed to improve the effectiveness of psychotropic drugs already approved and on the market, by combining them with a glial cell modulator. This strategy of combining its innovations with registered drugs allows Theranexus to significantly reduce development time and costs and significantly increase the chances of its drugs getting to market.

Theranexus’ proprietary and adaptable platform enables the generation of different proprietary drug candidates with high added value for multiple indications.

Theranexus is listed on the Euronext Growth market in Paris (FR0013286259-ALTHX). More information at:

  • National Organization for Rare Diseases (NORD) / Orphanet
  • Settembre et al, TFEB links autophagy to lysosomal biogenesis, Science 2011

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Theranexus SA published this content on September 09, 2021 and is solely responsible for the information it contains. Distributed by Public, unedited and unmodified, on 09 September 2021 04:11:01 PM UTC.

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