The trial is being conducted in six CLN3 patients aged 17 years and above. Credit: National Cancer Institute on Unsplash.

Theranexus, along with Beyond Batten Disease Foundation (BBDF), has revealed promising preliminary findings from a Phase I/II trial of Batten-1 to treat juvenile Batten disease (CLN3).

The results are based on the effect of Batten-1 on the patients after six months of treatment.

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They demonstrate an impact of the drug candidate on neuronal death and early clinical efficacy signs.

In addition, the data showed an average 17% reduction in neurofilament light chain (NfL) levels in the blood of the patients treated with Batten-1.

Motor symptoms evaluated by the improved physical subscale of the disease-specific UBDRS3 did not advance during the six-month period, with the natural course of the disease being observed.

This clinical score should have advanced by approximately one point in these patient groups.

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Furthermore, Batten-1 continues to demonstrate a good tolerability profile following the six-month treatment period.

The Phase I/II trial is currently being conducted in six CLN3 patients aged 17 years and above who are expected to get a 24-month treatment.

Initial data from the trial after nine weeks of treatment demonstrated a good tolerability and pharmacokinetic profile for Batten-1, which comprises the active ingredient miglustat.

As part of the open-label trial, patients will receive miglustat up to 600mg per day for two years.

The study’s primary endpoint is safety and tolerability, assessed using reports
regarding adverse effects, biological tests and electrocardiogram (ECG), along with the pharmacokinetics of miglustat.

Its secondary endpoints are efficacy monitoring, Unified Batten Disease Rating Scale, visual acuity and others.

Theranexus CEO Mathieu Charvériat said: “In our Phase I/II trial on Batten-1 in patients aged 17 and over and unfortunately at a rather advanced stage of the disease, we measure the levels of neurofilaments NfL and motor symptoms via the modified UBDRS, which provide data indicative of Batten-1’s efficacy.”