THERANEXUS: Encouraging preliminary results at 6 months in the phase 1/2 study of Batten-1 in Batten disease (CLN3) – 06/14/2023 at 07:30

• Reduction after 6 months of treatment of neurofilaments, a biomarker of neuronal death
  

• Stabilization of motor symptoms for treated patients compared to an expected deterioration according to the natural history of the disease
  

Lyon, France – Austin, Texas, United States – June 14, 2023 – 7:30 a.m. CEST –

Theranexus, an innovative biopharmaceutical company in the treatment of rare neurological diseases, and the Beyond Batten Disease Foundation (BBDF), today announce encouraging preliminary results obtained by their drug candidate,

Batten-1, in the juvenile form of Batten disease, in the ongoing phase 1/2 study after 6 months of treatment. These preliminary results suggest an effect of Batten-1 on neuronal death and first signals of clinical efficacy.

Theranexus and BBDF are conducting a Phase 1/2 study of their drug candidate in six patients with Batten’s disease (CLN3) aged 17 and older to be treated for 24 months. The first results of this phase 1/2 study, after 9 weeks of treatment showed a good safety and pharmacokinetic profile

[1]

of Batten-1. The new preliminary results, obtained after 6 months of treatment, show

an average 17% decrease in neurofilament light chain (NfL)

in the blood of patients assayed, neurofilaments being a recognized biomarker of neuronal degeneration

[2]

. Moreover,

motor symptoms assessed using a modified UBDRS subscale
[3]
specific for the CLN3 form of the disease, did not progress during the same period

(average score of 31.8 after 6 months of treatment compared to 32.4 at entry into the study), whereas according to the available data describing the natural history of the disease, this clinical score should have increased

[4]

by about one point in these patients. Finally, the drug candidate still demonstrates a good safety profile after six months of treatment.

For

Mathieu Charvériat, CEO of Theranexus

In our phase 1/2 study with our drug candidate Batten-1, on patients aged 17 and over and unfortunately already advanced in the disease, we measure the levels of NfL neurofilaments and motor symptoms via the modified UBDRS scale , which gives us data indicative of the effectiveness of Batten-1. We are now presenting our first results at six months, which confirm the strong therapeutic potential of Batten-1 in Batten disease, driven by its ability to reduce neuronal death and consequently the progression of the disease”.

For Marie Sebille, Medical Director at Theranexus

”

These preliminary results are very promising in this devastating disease for which there is currently no treatment. We have a convergence of positive signals with the reduction of neurofilaments and the stabilization of motor symptoms after six months of treatment. This is a great hope for children with this disease and for their families.”

.

The preliminary results at six months will be presented to the scientific and medical community at the NCL2023 – 18th International Congress on Neuronal Ceroid Lipofuscinoses conference which will take place from September 26 to 30, 2023 in Hamburg, Germany.

About Batten-1

Batten-1 is a new proprietary and exclusive pharmaceutical specialty whose active ingredient is miglustat. The mechanism of action of this active ingredient makes it possible to block the accumulation of glycosphingolipids and neuro-inflammation, and thus significantly reduce neuronal death, the cause of progressive loss of function in patients. For patients over 17 years of age in the Phase 1/2 study, the product is administered in solid form. In the Phase 3 study, it will be administered in a liquid form, more suitable for a pediatric population.

Design of the Phase 1/2 study: this is an open-label study in 6 patients over 17 years of age with Batten CLN3 disease, treated with miglustat up to 600 mg/day for a duration of 2 years. The primary endpoint is the tolerance and safety of use, assessed by the collection of adverse effects, biological tests and ECG, as well as the pharmacokinetics of miglustat. Secondary endpoints include efficacy endpoints: Unified Batten Disease Rating Scale, visual acuity, measurement of brain volumes by magnetic resonance imaging, and measurement of retinal neuronal layer thickness by coherence tomography optical. Administration in dose escalation to a maximum of 600 mg/d of Batten-1 was well tolerated, with no serious adverse effects or leading to discontinuation of treatment. The most frequently reported adverse effects are of a gastrointestinal nature, reversible and most often of mild to moderate intensity, thus demonstrating the good tolerance profile of Batten-1 in this population. Batten-1 will continue to be evaluated in these treated patients for a total of 24 months. Additional information is available at https://clinicaltrials.gov/ct2/show/NCT05174039.

About Batten disease

The juvenile form of Batten disease or Spielmeyer-Vogt disease, or CLN3 disease, is a genetic disease of the nervous system, rare and fatal, for which there is no treatment. It belongs to the group of neuronal ceroid-lipofuscinoses (CLN). More than 400 mutations in 13 genes have been associated with CLN variants, which essentially differ from each other in the age at onset of first symptoms. The first symptom of the juvenile form, a progressive loss of vision, appears between the ages of 4 and 6 years. It is followed by cognitive disorders, behavioral disorders and motor difficulties. Seizures appear within 2 to 4 years after the onset of the disease. Gradually, patients decline physically and mentally. Eventually, they move around in a wheelchair or even remain bedridden before dying prematurely.

This disease is always fatal – usually in late adolescence or early adulthood. In the United States and Europe, the prevalence of Batten disease is the highest among NLCs, affecting nearly 2,000 patients

[5]

on these territories. From a pathophysiological point of view, interactions between neurons and glial cells play a key role in the appearance and progression of all NLCs.

About the Beyond Batten Disease Foundation (BBDF)

The Beyond Batten Disease Foundation (BBDF) is the world’s largest non-profit organization dedicated to funding research to treat and cure juvenile Batten disease (CLN3). Since its inception in 2008, more than $35 million has been invested in research through donations, co-funding and strategic partnerships. BBDF is spearheading a single and cohesive strategy, integrating independent scientific resources and collaboration with related organizations to conduct research into juvenile Batten disease. BBDF-sponsored work first uncovers disease mechanisms

[6]

. 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 at www.beyondbatten.org.

About Theranexus

Theranexus is an innovative biopharmaceutical company, spun off from the CEA and specializing in the treatment of rare neurological diseases. The company has a unique platform for the identification and characterization of drug candidates for innovative therapy in the field of rare neurological disorders and a first drug candidate in development and clinically in Batten disease.

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

Warning

This press 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 occur 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 27, 2023 under number D.23-0345, a copy of which is available on the company’s website (www.theranexus.com), and changes in economic conditions, financial markets and markets in which Theranexus is present. The forward-looking statements contained in this press release are also subject to risks unknown to Theranexus or that Theranexus does not consider material at this date. The realization of all or part of these risks could cause the actual results, financial conditions, performances or achievements of Theranexus to differ materially from the results, financial conditions, performances or achievements expressed in these forward-looking statements. Theranexus assumes no responsibility for updating these forward-looking statements.

[1]

https://www.theranexus.com/images/pdf/Theranexus_CP_Resultats_Programme_Batten-1_VDEF.pdf

[2]

Dang DoAN, et al. Neurofilament light chain levels correlate with clinical measures in CLN3 disease. Genet Med. 2021 Apr;23(4):751-757

[3]

Unified Batten Disease Rating Scale

[4]

An increase in the score corresponds to a worsening of the disease

[5]

National Organization for Rare Disorders (NORD)/Orphanet

[6]

Settembre et al, TFEB links autophagy to lysosomal biogenesis, Science 2011

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