THX Pharma Reports Its Cash Position as of September 30, 2026

Lyon, France – October 22, 2026 – 6:00 p.m. CEST – THX Pharma (Theranexus), an innovative biopharmaceutical company developing treatments for rare neurological diseases, today announced its cash position as of September 30, 2026.

As of September 30, 2026, cash and cash equivalents amounted to €16.0 million, compared with €17.5 million as of June 30, 2026. Cash consumption in the third quarter reflects the operational preparation of the international pivotal Phase 3 trial of Batten-1, as well as the repayment of bank loan installments. Clinical development costs for Batten-1 are fully funded by Biocodex. However, the time lag between costs incurred and their recharge may result in quarter-to-quarter fluctuations in the Company's cash position.

Third-Quarter 2026 Highlights

• Regulatory submissions for the pivotal Phase 3 trial of Batten-1 – On September 1, 2026, THX Pharma, the Beyond Batten Disease Foundation (BBDF) and Biocodex submitted applications for authorization of the Batten-1-02 trial to the U.S. Food and Drug Administration (FDA), the UK Medicines and Healthcare products Regulatory Agency (MHRA) and, through the Clinical Trials Information System (CTIS), to six European Union Member States (France, Germany, the Netherlands, Denmark, Sweden and Italy). This open-label, multicenter trial, to be conducted in 10 reference centers in the United States and Europe, is expected to enroll approximately 27 patients aged 4 to 17 years, with visual acuity as the primary endpoint. Subject to regulatory approvals, enrollment of the first patients is expected by the end of 2026.
• Grant of a European patent protecting Batten-1 through 2041 – The European Patent Office (EPO) granted European patent No. EP 4 188 371, which protects the use of miglustat, the active ingredient in Batten-1, for the treatment of juvenile Batten disease (CLN3) through July 30, 2041, in 38 contracting states. THX Pharma holds an exclusive worldwide license to this patent granted by BBDF. The patent strengthens Batten-1's protection, which is complemented by the market exclusivities associated with the orphan drug designations granted in the United States and the European Union.
• First significant revenue in the first half of 2026 – Half-year results, published on September 29, 2026, showed revenue of €3.2 million and a net loss reduced to €0.1 million, compared with €1.1 million in the first half of 2025.

Mathieu Charvériat, Chairman and Chief Executive Officer of THX Pharma, said: “With TX01 and Batten-1, THX Pharma now has two advanced assets in rare neurological diseases, each supported by leading partners. Our cash position of €16.0 million, combined with Biocodex's full funding of Batten-1's clinical development, enables us to pursue our roadmap. We remain focused on our upcoming milestones: the initiation of the pivotal Phase 3 trial of Batten-1, subject to regulatory approvals, and the preparation, together with Exeltis, of the marketing authorization application for TX01 in Europe under the PUMA procedure.”

Next financial publication:
February 18, 2027: Cash position as of December 31, 2026

About THX Pharma (Theranexus)

THX Pharma (Theranexus, Euronext Growth: ALTHX) is a biopharmaceutical company specializing in the development of innovative treatments for rare neurological diseases. The company is developing in particular TX01, a new formulation intended for the treatment of Gaucher disease and Niemann-Pick disease type C, and Batten-1, a drug candidate targeting juvenile Batten disease (CLN3). TX01 is the subject of several licensing agreements with international pharmaceutical partners, including Exeltis and Biocodex, for its development and commercialization in different regions of the world. Batten-1 is covered by a global licensing agreement with Biocodex for its development and commercial exploitation. THX Pharma also has an innovative antisense oligonucleotide platform, co-developed with leading research laboratories, dedicated to the development of new therapeutic approaches for rare neurological diseases. THX Pharma is listed on the Euronext Growth market in Paris (ISIN: FR0013286259 – ALTHX).

For more information: http://www.thxpharma.com

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Contacts:

THX PHARMA
Christine PLACET
Chief Financial Officer
contact@thxpharma.com

FP2COM
Florence PORTEJOIE
Media Relations
+33 (0)6 07 76 82 83
fportejoie@fp2com.fr

Forward-Looking Statements

This press release contains forward-looking statements relating to THX Pharma (Theranexus) and its activities, including its prospects and the development of its products. THX Pharma believes that these forward-looking statements are based on reasonable assumptions. However, forward-looking statements do not constitute guarantees of future performance, as they relate to future events and depend on circumstances that may or may not occur in the future, as well as on various risks and uncertainties, including those described in the Company's Universal Registration Document filed with the AMF (Autorité des marchés financiers) on April 29, 2026, under number D.26-0321, a copy of which is available on the Company's website (www.thxpharma.com), and on developments in the economic environment, financial markets and the markets in which THX Pharma operates.

The forward-looking statements contained in this press release are also subject to risks that are unknown to THX Pharma or that THX Pharma does not consider material as of the date hereof. The occurrence of all or part of these risks could cause THX Pharma's actual results, financial condition, performance or achievements to differ materially from the results, financial condition, performance or achievements expressed in these forward-looking statements. THX Pharma assumes no obligation to update these forward-looking statements.