Theranexus: Theranexus provides an update on its Batten-1 program in Batten disease CLN3

• Presentation of promising results from the Phase I/II trial of Batten-1 by the BBDF Foundation and Theranexus at the Child Neurology Society Annual Meeting in San Diego,
• Positive signals on visual acuity in younger patients with CLN3 in the context of compassionate use

Lyon, France – Austin, Texas, United States – November 19, 2024 – 6:00 p.m. CET – Theranexus, an innovative biopharmaceutical company in the treatment of rare diseases, and the Beyond Batten Foundation (BBDF) presented the final and positive results of the phase I/II trial evaluating Batten-1 in six young adults with Batten disease CLN3, after 18 months of treatment, at the Annual Meeting of the Society of Pediatric Neurology held from November 11 to 14 in San Diego, United States.

This presentation highlighted the strong safety profile of miglustat in patients with CLN3 and highlighted the positive efficacy data collected during this trial. The results demonstrate clear target activation, biological effect and suggest stabilization of disease progression during the treatment period. All trial participants opted to continue receiving miglustat through the BBDF Early Access Program. After two years of continuous treatment, no tolerability problems have been reported.

Additionally, case reports from the compassionate use of miglustat in CLN3 Batten disease reinforce its potential as a disease-modifying treatment. They suggest a possible stabilization of visual acuity, a function severely impacted in CLN3 Batten disease and often associated with legal blindness before the age of 12. This information is encouraging, particularly in the context of discussions between Theranexus, BBDF, the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the consideration of visual acuity as a main criterion for the next phase III trial, with a view to registration of the drug in the United States and Europe.

Clinicians who have treated these and other younger patients with miglustat are enthusiastic about these promising data. For Professor Gary Clark, principal investigator of the trial and chief of pediatric neurology at Texas Children’s Hospital in Houston : « These case reports on visual acuity, in addition to other positive personal observations on visual experience in younger patients, complement our Phase I/II trial results that support the prospective benefit of Batten-1 in treatment of Batten disease CLN3. After confirmation in a larger trial, these results with Batten-1 will represent a major advance in the management of this disease. »

Mathieu Charvériat, President and CEO of Theranexus, concludes: « Our positive phase I/II study, as well as additional very promising case reports of compassionate use of miglustat with positive results on vision, strongly reinforce the launch of our phase III as well as the selection of visual acuity as main criterion. As previously noted, we are exploring options to secure the resources necessary for this pivotal Phase III trial, including through the future commercialization of our liquid formulation in Niemann-Pick Type C disease, as recently announced.. »

About the Beyond Batten Disease Foundation (BBDF)

The Beyond Batten Disease Foundation (BBDF) is the world’s largest nonprofit organization dedicated to funding research to treat and cure juvenile Batten disease (CLN3). Since its creation in 2008, more than $35 million has been invested in research through donations, co-funding and strategic partnerships. BBDF spearheads a unique 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 made it possible to discover the mechanisms of the disease. Today, a cure is in sight. BBDF-funded research has discovered a drug, Batten-1, that slows disease progression in models of Batten disease. More information on www.beyondbatten.org.

About Theranexus

Theranexus is an innovative biopharmaceutical company, spun out from the CEA and specialized in the treatment of rare neurological diseases. The Company has a unique platform for identifying and characterizing innovative therapy drug candidates in the field of rare neurological disorders and a first drug candidate in clinical development in Batten disease.

THERANEXUS is listed on the Euronext Growth market in Paris (FR0013286259- ALTHX).

More information on:

http://www.theranexus.com

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Contacts :

THERANEXUS

Christine likes it
Administrative and Financial Director

[email protected]

FP2COM

Florence PORTEJOIE

Media relations

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Warning

This release contains forward-looking statements relating to Theranexus and its business, including its prospects and product development. Theranexus believes that these forward-looking statements are based on reasonable assumptions. However, forward-looking statements are not guarantees of future performance, as they relate to future events and depend on circumstances that may or may not materialize in the future, and on various risks and uncertainties, including those described in the company’s universal registration document filed with the AMF on April 30, 2024 under number D.24-0382, a copy of which is available on the company’s website (www.theranexus.com), and developments in the economic situation, financial markets and markets in which Theranexus is present. Forward-looking statements in this release are also subject to risks that are unknown to Theranexus or that Theranexus does not consider to be material as of this date. The realization of all or any of these risks could cause the actual results, financial conditions, performance or achievements of Theranexus to differ materially from the results, financial conditions, performance or achievements expressed in these forward-looking statements. Theranexus disclaims any responsibility to update these forward-looking statements.

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