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Lyon, France – Austin, Texas, United States – February 3, 2022 – Theranexus, an innovative biopharmaceutical company in the treatment of neurological diseases and a pioneer in the development of drug candidates modulating the interaction between neurons and glial cells, and the Beyond Batten Disease Foundation (BBDF), launch recruitment for a Phase I clinical trial /II to evaluate BBDF-101 in Batten disease.

Franck Mouthon, President of Theranexusnoted: “The launch of recruitment is a key step in the development of our drug candidate BBDF-101. This trial will be conducted with BBDF and in collaboration with the main North American reference centers for Batten disease, a rare and fatal genetic disease of the nervous system. Our ambition is to demonstrate the full potential of BBDF-101 in this disease for which there is currently no therapeutic option for patients.

Following the receipt of Investigational New Drug (IND) approval by the Food and Drug Administration (FDA) for its BBDF-101 program in 2021, Theranexus is actively recruiting patients for its Phase I/II clinical trial. During the 2-year trial, six patients aged 17 or older will receive BBDF-101 at increasing doses over the first 4.5 months to establish the tolerability and pharmacokinetics of trehalose, miglustat and a combination of trehalose/miglustat. The results of the titration phase will be available at the beginning of the second half of 2022.

After consultation with the FDA, Theranexus plans to start a pivotal phase III trial at the end of 2022 involving a pediatric cohort of 30 patients with regular measurements to assess disease progression and safety. The objective will be to compare the evolution of different signs and symptoms in these patients, assessed on the Unified Batten Disease Rating Scale (UBDRS, neurological and behavioral symptoms, activities of daily living, etc.), over a period of two years, to data from a study of disease progression in a cohort of untreated patients followed for many years at the University of Rochester.

This phase III trial will be conducted in collaboration with leading reference centers for Batten disease in the United States, including Texas Children’s Hospital in Houston, TX, the fourth largest pediatric hospital in the United States, the National Institutes of Health and the University of Rochester Batten Center in Rochester, NY.

“It is with great gratitude to the families of our patients and our donors that we witness today the launch of the BBDF-101 clinical program which aims to offer a therapeutic solution to patients suffering from this very rare genetic disease. Achieving this major milestone for the Foundation, in collaboration with Theranexus, is the exciting culmination of nearly 12 years of research,” concludes Craig Benson, Chairman of the Board of BBDF.

About Batten Disease

Juvenile Batten disease, also known as Spielmeyer-Vogt disease or CLN3, 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 called neuronal ceroid lipofuscinoses (NCL). More than 400 different errors in 13 genes have been attributed to various forms of NCL, 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 a slowing of 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 become confined to wheelchairs, are bedridden, and die prematurely. Psychiatric symptoms or psychosis can appear at any time. Juvenile Batten disease is always fatal; typically late teens to early twenties. In the United States and Europe, the juvenile form is the most common form of NCL, which together affect nearly 3,000 patients1 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 inception in 2008, more than $35 million has been invested in research through donations, co-funding and strategic partnerships. BBDF is spearheading a single, cohesive strategy, incorporating independent scientific resources and collaboration with related organizations to drive research into juvenile Batten disease. Today, a cure is in sight. BBDF-funded research has discovered a drug that slows disease progression2 in Batten models. More information can be found at www.beyondbatten.org

About the development of BBDF-101 for Batten disease and the partnership between BBDF and Theranexus

BBDF-funded research conducted by academics specializing in lysosomal storage disorders (including Baylor College of Medicine) led to the discovery of drug candidate BBDF-101. Following a partnership agreement between BBDF and Theranexus concluded at the end of 2019 (see below), Theranexus has supplemented the preclinical development of BBDF-101 with research to establish the safety of the product. Thanks to the research results, BBDF-101 will now be able to enter the clinical phase following the granting of IND status by the FDA in September 2021.

The clinical development path includes:

  • Phase I/II for which recruitment started today,
  • Phase III to start at the end of 2022 and include 30 patients with the objective of obtaining definitive results in the second half of 2025,

The development of BBDF-101 is the result of a partnership concluded at the end of 2019 between BBDF and Theranexus. The exclusive worldwide license agreement between BBDF and Theranexus covers the clinical development of the pending approval drug candidate BBDF-101 and its commercial use. Theranexus also plans to continue its research on the NeuroLead platform of drug candidates targeting neurological forms of other lysosomal storage diseases.

In return for this license, the agreement provides that Theranexus will pay BBDF fixed sums upon signature, approval and achievement of post-approval business objectives. In addition, the agreement provides for the payment of royalties calculated on the net sales of BBDF-101 once marketed by Theranexus

Next financial publication:

Thursday, April 28, 2022: 2021 annual financial results and cash point as of March 31, 2022

1 National Organization for Rare Diseases (NORD)/Orphanet

2 Settembre et al, TFEB links autophagy to lysosomal biogenesis, Science 2011



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