THX PHARMA (THERANEXUS) AND EXELTIS ACHIEVE KEY MILESTONE TOWARDS THE COMMERCIALIZATION OF TX01

14 Oct 2025 18:30 CEST

THX PHARMA (THERANEXUS) AND EXELTIS ACHIEVE KEY MILESTONE TOWARDS THE COMMERCIALIZATION OF TX01

THX Pharma (Theranexus) and Exeltis have reached a pivotal milestone, setting the stage for the regulatory submission of TX01 for Gaucher and Niemann-Pick type C diseases

Lyon, France – Madrid, Spain – October 14, 2025 – 6.30pm CEST – THX Pharma (Theranexus), a pharmaceutical company specializing in treatments for rare neurological diseases, and Exeltis, a global pharmaceutical company within the Insud Pharma group, announce the successful completion of the first major milestone in their license and supply agreement for TX01. This achievement marks a significant step towards bringing TX01 to patients with Gaucher and Niemann-Pick type C diseases.

With this milestone, both companies are on track to submit Marketing Authorization Applications for TX01 as planned, paving the way for commercialization in 2027 in Europe, followed by launches in Latin America and certain Middle Eastern countries.

In addition to the upfront payment received at the signature of the agreement – regarding which Exeltis will not exercise its right to reimbursement, foreseen in the agreement in the event of THX Pharma's failure to provide the required documentation before October 31, 2025 – THX Pharma is eligible for further regulatory and commercial milestone payments and royalties based on the product's net sales.

TX01 is a novel formulation of an already approved compound indicated in the treatment of rare neurological diseases such as Gaucher and Niemann-Pick type C. This partnership confirms the commitment of both companies to delivering new therapies and addressing significant unmet medical needs.

Mathieu Charvériat, CEO of THX Pharma, commented: "The timely completion of this milestone demonstrates our commitment to advancing TX01's regulatory journey. This step brings us closer to the global commercialization of TX01, delivering a transformative therapy for patients with Niemann-Pick type C and Gaucher diseases."

Salustiano Perez, General Manager of Exeltis, added: "We are pleased to reach this milestone with THX Pharma, the result of a fruitful collaboration. We can now confidently proceed with the plan to apply for TX01's registration in the first quarter of 2026 and, commercialization in Europe expected as early as 2027."

About THX Pharma

THX Pharma (Theranexus) is a pharmaceutical company specializing in treatments for rare neurological diseases. Its first drug, TX01, is expected to be commercialized soon—particularly in Europe by Exeltis, but also in the United States, Canada, and Australia—for Niemann-Pick type C disease and Gaucher disease. Its second drug, Batten-1, targets the juvenile form of Batten disease and could become the first approved therapy for this condition. THX Pharma also has an innovative antisense oligonucleotide platform, codeveloped with leading research laboratories, dedicated to rare neurological diseases. THX Pharma, a trade name of Theranexus, is listed on Euronext Growth Paris (FR0013286259 – ALTHX).

About the Insud Pharma Group

Insud Pharma is a pharmaceutical group with a history spanning over 45 years, present in around 50 countries, with 9,000 employees worldwide and 20 production sites. Insud Pharma operates across the entire pharmaceutical industry value chain, offering its expertise in scientific research, development, production, sales, registration and marketing of a wide range of active pharmaceutical ingredients (APIs), finished dosage forms (FDFs), and branded and innovative medicines for human and veterinary care, as well as biopharmaceutical products. Insud Pharma is committed to improving global health by providing accessible, effective, safe, and high-quality treatments through its various business units, with continuous investment in R&D and the latest technologies. Through its brand Exeltis, Insud Pharma holds a leading position in women's health and is a key player in Central Nervous System therapies.

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

Disclaimer

This press release contains forward-looking statements relating to THX Pharma (Theranexus) and its activities, 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 occur in the future, and on various risks and uncertainties, including those described in the universal registration document filed by the company with the AMF (Autorité des Marchés Financiers) on April 29, 2025, under number D.25-0350, a copy of which is available on the company's website (www.theranexus.com), and on changes in the economic situation.

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