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  • · New milestone for NeuroLead, the active agent screening platform
  • · New publications in scientific journals
  • · Start of the phase I/II clinical trial in Batten disease

Lyon, February 28, 2022 – On the occasion of World Rare Disease Day 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, takes the opportunity to report on its latest scientific developments with new advances on its NeuroLead active agent screening platform in rare neurological diseases in collaboration with the Collège de France and the French Alternative Energies and Atomic Energy Commission (CEA), and new academic publications .

NeuroLead, the platform for generating new drug candidates for innovative therapies in orphan neurological indications

Theranexus, a CEA spin-off, which coordinates the NeuroLead program, in collaboration with the Collège de France and the CEA, has just completed the first scientific phase of the platform, based on the implementation and validation of models of interactions between neurons and astrocytes and the study of their functions. These models will serve as a basis for identifying and characterizing new drug candidates in orphan neurological indications. With the achievement of this new milestone, Theranexus received in February 2022 a payment of €1.7 million from Bpifrance as part of the Future Investments program (PIA). To this amount is added financing of €800,000 through a Bpifrance R&D Innovation Loan to accelerate the development of the platform.

As a reminder, in 2019, Theranexus and its partners obtained total funding of €6.2 million via the PIA program. Of the 4 million euros granted to Theranexus, the Company has received a total of 2.7 million euros to date.

We are very pleased to have succeeded in this first scientific phase which will allow us to continue the identification and development of new drug candidates in rare diseases of the nervous system, and to expand our portfolio in this area beyond our active BBDF-101 for Batten disease. We welcome the new perspectives offered by the NeuroLead platform“, declared Mathieu Charvériat, Deputy CEO and Scientific Director of Theranexus.

With the NeuroLead platform, we now have new preclinical models to better target the interactions between neurons and astrocytes in a physiological setting, and we are currently working on rare disease models.“, continued Dr Nathalie Rouach from the Center for Interdisciplinary Research in Biology at the Collège de France.

We are working on the co-culture of induced pluripotent human stem cells, differentiated into neurons and astrocytes, which constitute an ideal human tissue to identify new drugs in orphan neurological diseases.“, concluded Dr Anselme Perrier from the François Jacob Institute of Biology, Molecular Imaging Research Center (MIRCen) at the CEA.

Three new publications in scientific journals

In January 2022, Theranexus, the Collège de France and the CEA published a state of knowledge on the interactions between astrocytes and neurons in orphan indications and their modeling by the use of stem cells in the International Journal of Molecular Sciencesentitled “Modeling and targeting neuroglial interactions with human pluripotent stem cell models“.

In December 2021, Theranexus published another article titled “Automated assays to identify modulators of transcription factor EB translocation and autophagy” in ASSAY and drug development technologiespresenting his research on the identification of new molecules in the treatment of lysosomal storage disorders, a group of 50 orphan conditions that affect more than 3,000 adults and children in France.

Theranexus also recently presented its new research in neuroimaging, with the very first demonstration of functional ultrasound imaging for the characterization of disease models, paving the way for the use of such models in orphan diseases. The article titled “Impaired local and long-range brain connectivity and visual response in a rat genetic model of hyperactivity revealed by functional ultrasound” was published in Frontiers in Neuroscience in February 2022.

Start of Phase I/II clinical trial for Batten disease, a rare degenerative genetic disease

Finally, in early February 2022, Theranexus and BBDF began recruiting for a Phase I/II clinical trial to evaluate BBDF-101 in Batten disease, a rare and fatal genetic disorder of the nervous system for which there is no currently no treatment or cure. The first results of the titration phase will be available at the beginning of the second half of 2022 and, after consultation with the FDA, at the end of 2022, Theranexus aims to start pivotal Phase III trials.

About the NeuroLead Platform

In early 2019, the NeuroLead platform was granted a funding envelope of €6.2 million as part of the Future Investments program (PIA), led by Bpifrance (Structuring Research and Development Projects for Competitiveness, PSPC) to support its development. The four-year NeuroLead program is coordinated by Theranexus in collaboration with the Collège de France and the Alternative Energies and Atomic Energy Commission (CEA). Combining the latest innovations in neuroscience and artificial intelligence, the program aims to design and industrialize a unique platform for the identification and characterization of drug candidates targeting the interactions between the two major populations of brain cells, neurons and glial cells, to the treatment of neurological disorders.

About Theranexus

Theranexus is a clinical-stage biopharmaceutical company spun off from the Commissariat aux Energies Alternatives et à l’Energie Atomique (CEA) in 2013. It develops drug candidates for the treatment of diseases of the nervous system. Thanks to its knowledge of the interactions between neurons and glial cells, Theranexus is a pioneer in the design and combination of approved substances and has a strong and diverse portfolio of clinical-stage drug candidates. The company’s combined drug repositioning strategy, based on a solid commercial base and an ability to quickly demonstrate its clinical value, enables it to produce various high value-added proprietary drug candidates, significantly reduce the time and costs of development and greatly increase the chances of its drugs reaching the market. Thus, Theranexus is well positioned in several indications, including Batten’s Parkinson’s disease, for which no treatment is currently available. Theranexus is listed on the Euronext Growth market in Paris (FR0013286259-ALTX).

About the College de France

The Collège de France is a public institution of higher education unique in France and without equivalent abroad. Since the 16th century, the Collège de France has fulfilled a dual mission: to carry out the most innovative research and to teach it. Dedicated to fundamental research, the Collège de France has the sole mission of “teaching the knowledge in the making in all fields of literature, the arts and the sciences”. It works in partnership with the National Center for Scientific Research (CNRS), the National Institute of Health and Medical Research (INSERM) and several other leading institutions. The Interdisciplinary Research Center in Biology (CIRB) of the Collège de France, specific partner of the Neurolead program, occupies a prominent place on critical themes and research priorities, under the direction of Marie-Hélène Verlhac. Nathalie Rouach’s team, actively involved in Neurolead, is an internationally recognized authority on these cellular interactions.

About CEA and MIRCEN

The CEA is a major player in research, serving the State, the economy and citizens. It provides concrete responses to their needs in four major areas: energy transition, digital transition, technologies for the medicine of the future, defense and security. The CEA/MIRCen is a department of the CEA’s François Jacob Institute of Biology directed by Dr Philippe Hantraye, member of the Neurolead project. This research center, unique in Europe, welcomes researchers from various organisations, conducts translational research on neurodegenerative diseases and develops innovative therapies in an optimal scientific, technological and methodological framework. The main objective is to improve the transfer of knowledge and technology from basic neuroscience research to clinical trials in patients with neurodegenerative disorders. The MIRCen also oversees a National Biology and Health Infrastructure (INBS), NeurATRIS, bringing together prestigious research centers in the Paris region.

Next financial publication:

Monday, April 25, 2022: 2021 annual financial results and cash point as of March 31, 2022

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