Lyon, France – Austin, Texas, United States – 29 September 2023 – 7.30am – CET – Theranexus, a biopharmaceutical company innovating in the treatment of rare neurological diseases, and the Beyond Batten Disease Foundation (BBDF), present very encouraging interim results for efficacy and safety after 12 months of treatment in their Phase I/II trial for Batten disease (CLN3) at NCL2023, the International Congress on Neuronal Ceroid Lipofuscinoses held in Hamburg (Germany) from 26 to 30 September 2023.

For Professor Gary Clark, the trial’s principal investigator and Chief of Child Neurology at Texas Children’s Hospital in Houston, “The Phase I/II results for Batten-1 are highly promising, as we have observed a reduction in neurofilaments and glycosphingolipids and no notable progression of motor symptoms after 12 months of treatment in our 6 patients aged 17 years and over. This is unprecedented in the indication, and Batten-1 is thus a source of great hope for children affected by the disease and for their families”. Professor Clark will be presenting the 12-month interim efficacy and safety results at the “Translational Research Clinical” session on Friday 29 September at 12.25pm (CEST).

The 12-month treatment results further support the 6-month results announced in mid-June 2023. The new results, achieved after 12 months of treatment, show an average 32% decline in neurofilament light chain (NfL) levels in patient serum (as a reminder, 17% after 6 months’ treatment). Neurofilaments are a recognized biomarker of neurodegeneration. A 64% reduction in this biomarker is also observed in the cerebrospinal fluid (CSF). After twelve months of treatment, in line with preclinical data the results also confirmed a reduction in the glycosphingolipids involved in the disease (Gb3 reduction of 45%) which, when they accumulate, are toxic to neurons. Clinically, there was notably less worsening of motor symptoms evaluated by the modified UBDRS physical assessment subscale after 12 months than what is expected in this naturally progressive disease.

For Theranexus’ CEO, Mathieu Charvériat: “We are delighted with these 12-month results which confirm and complement our initial 6-month results. The decrease of biomarker levels confirms the effect of Batten-1 on neuronal death, and the clinical course did not worsen. These consistent results represent a significant endorsement of the mechanism of action and demonstrate the strong therapeutic potential of Batten-1 in Batten disease (CLN3)”.

As a reminder, the Phase I/II trial conducted by Theranexus and BBDF to evaluate their drug candidate, Batten-1, includes six patients with Batten disease (CLN3) aged 17 years and over. The patients are being treated over a 24-month period.

The presentation of the 12-month efficacy and safety results will be available online on 29 September after market closing.

For further information about NCL2023:

Read the full press release here.

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