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 acting on the interaction between neurons and glial cells and the Beyond Batten Disease Foundation (BBDF), initiate the recruitment of the phase I clinical trial /II evaluating BBDF-101 in Batten disease
1
.
Franck Mouthon, President of Theranexus
, says:
“The launch of recruitment is a key step for the development of our drug candidate BBDF-101. This study will be conducted with the BBDF Foundation and in collaboration, in the United States, with 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 a pathology for which there is currently no therapeutic solution for patients. »
Following the Food and Drug Administration (FDA) obtaining Investigative New Drug (IND) status for its BBDF-101 program in mid-2021, Theranexus is now actively recruiting patients for its phase 1 clinical trial. I/II. This 2-year trial plans for the six patients aged 17 or over to receive the drug BBDF-101 in dose escalation for the first 4.5 months, with the establishment of the tolerance and pharmacokinetics of Trehalose, Miglustat and the Trehalose/Miglustat combination. The results of this titration phase will be available at the start of the second half of 2022.
Theranexus plans, after consultation with the FDA, to start a pivotal phase III study at the end of 2022 which will include a pediatric cohort of 30 patients with regular measures to assess disease progression and tolerance. It will be a question of comparing the progression over two years of the various signs and symptoms in these patients, evaluated according to the scale of the Unified Batten Disease Rating Scale (UBDRS, neurological, behavioral symptoms, activities of daily living, etc.) , 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 study will be conducted in collaboration with the main reference centers for Batten disease in the United States, which includes the Texas Children’s Hospital in Houston, on the 4
th
the largest pediatric hospital center in the United States, the National Institute of Health and the University of Rochester Batten Center in Rochester, New York.
”
It is with immense gratitude to the families of our patients and our donors that we see the start of the BBDF-101 clinical program with the objective of finding a therapeutic solution for patients suffering from this very rare genetic disease. The achievement of this major milestone for the Foundation, in collaboration with Theranexus, is the exciting culmination of nearly 12 years of research work. »
concludes
Craig Benson, Chairman of the Board of the BBDF Foundation.
About Batten disease
The juvenile form of Batten disease or Spielmeyer-Vogt disease, or CLN3 disease, is a genetic disease of the nervous system, rare and fatal, for which there is no treatment. It belongs to the group of neuronal ceroid-lipofuscinoses (CLN). The first symptom of the juvenile form, a progressive loss of vision, appears between the ages of 4 and 6 years. It is followed by personality changes, behavioral problems and learning difficulties. Seizures may appear within 2 to 4 years after the onset of the disease. Gradually, patients decline physically and mentally. Eventually, they move around in a wheelchair or even remain bedridden before dying prematurely. Psychiatric disorders can arise at any time. This disease is always fatal – usually in late adolescence or early adulthood. In the United States and Europe, the prevalence of Batten disease is the highest among NLCs, affecting nearly 3,000 patients in these territories. From a pathophysiological point of view, interactions between neurons and glial cells play a key role in the appearance and progression of all NLCs.
About the Beyond Batten Disease Foundation (BBDF)
The Beyond Batten Disease Foundation (BBDF) is the world’s largest non-profit organization dedicated to funding research to treat and cure 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 and cohesive strategy, integrating independent scientific resources and collaboration with related organizations to conduct research into juvenile Batten disease. The work sponsored by BBDF initially elucidated the mechanisms of the disease. Today, a cure is in sight. BBDF-funded research has found a drug that slows disease progression in models of Batten disease. www.beyondbatten.org
About the development of BBDF101 in Batten disease and the partnership between BBDF and Theranexus
Work funded by BBDF and carried out by academic specialists in lysosomal storage diseases (in particular the Baylor College of Medicine) led to the discovery of the drug candidate BBDF-101. Following a partnership agreement between BBDF and Theranexus concluded at the end of 2019 (see below), Theranexus completed the preclinical development of BBDF-101 with work to establish the safety of the product. The result of this work now allows BBDF-101 to enter the clinic, after obtaining an IND from the FDA in September 2021.
The clinical development path includes:
Phase I/II for which recruitment began today and,
A phase III which will start by the end of 2022, which will include 30 patients and with the objective of obtaining definitive results in the second half of 2025.
The development of BBDF101 is the result of a partnership concluded at the end of 2019 between BBDF and Theranexus. The exclusive and worldwide license agreement between BBDF and Theranexus provides for the clinical development of the drug candidate BBDF-101 until its registration, as well as its commercial exploitation. Theranexus also plans to continue research on the NeuroLead platform of drug candidates targeting neurological forms of other lysosomal storage diseases.
In return for this license, the agreement provides for the payment by Theranexus to BBDF of lump sums upon signature, registration and the achievement of post-registration commercial objectives. In addition, the contract provides for the payment of royalties calculated on the basis of net sales of BBDF-101 once it is marketed by Theranexus.
Next financial publication:
Thursday, April 28, 2022
: 2021 Annual Financial Results and Cash Update as of March 31, 2022
1
National Organization for Rare Disorders (NORD) / Orphanet
2
Settembre et al, TFEB links autophagy to lysosomal biogenesis, Science 2011
ABOUT THERANEXUS
Founded in 2013, THERANEXUS is a clinical-stage biopharmaceutical company spun off from the CEA that develops drug candidates for the treatment of diseases of the nervous system.
Thanks to its knowledge of neuro-glial interactions, THERANEXUS is a pioneer in the design and combination of approved substances and has a solid and diversified portfolio of clinical-stage active ingredients. This repositioned drug combination strategy based on a solid commercial case and with a real ability to quickly demonstrate their clinical value allows the company to generate various proprietary drug candidates with high added value, to significantly reduce the time and costs of development and significantly increase the chances of market access for its drugs.
To this end, THERANEXUS is positioned in several indications, including Parkinson’s disease and Batten’s disease, for which no treatment is currently available on the market.
THERANEXUS is listed on the Euronext Growth market in Paris (FR0013286259-ALTHX).
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