THERANEXUS: New progress for the Batten-1 Program of Theranexus and the BBDF foundation in the juvenile form of Batten disease (CLN3) – 01/05/2023 at 18:00


Lyon, France – Austin, Texas, United States – January 5, 2023 – 6:00 p.m. CET –

Theranexus, an innovative biopharmaceutical company in the treatment of neurological diseases and a pioneer in the development of drug candidates acting on the interaction between neurons and glial cells and the Beyond Batten Disease Foundation (BBDF), announce today the first results of their clinical trial phase 1/2 in the juvenile form of Batten disease (or CLN3).

After 9 weeks of treatment at the maximum dose of 600 mg/d of Batten-1, the first results of the phase 1/2 study involving six patients with the juvenile form of Batten disease CLN3, aged 17 years and over, have shown a good tolerance and safety profile, as well as a pharmacokinetic profile in line with expectations.

Craig Benson, President of the Beyond Batten Disease Foundation

,

Explain :

“We are very pleased that Batten-1 was able to demonstrate a good tolerability and safety profile for our patients suffering from Batten disease. These results are very encouraging and bear immense hope for all the families facing this neurodegenerative disease for which there is no treatment today”.

To conclude, Marie Sebille, Medical Director at Theranexus, adds:



We have just reached an important milestone with these Phase 1/2 results in CLN3 patients, allowing us to prepare the Phase 3 study protocol to launch our pivotal clinical trial in a pediatric population in 2023

“.

As a reminder, as part of the 6

th

Annual Neuroscience Innovation Forum by Sachs Associates, to be held January 8, 2023 in San Francisco, Theranexus

[1]

will present the latest developments of the Batten-1 program and will also participate in a round table on the theme

“Rare and orphan diseases new modalities panel”

. In addition, the company will also be present at the Biotech Showcase and will participate, in parallel, in the 41

th

Annual JP Morgan Healthcare Conference bringing together all the key healthcare players.

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 blocks the accumulation of glycosphingolipids and neuro-inflammation. 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 parameters: Unified Batten Disease Rating Scale, visual acuity, cerebral magnetic resonance imaging and optical coherence tomography. 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 personality changes, behavioral problems and learning 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. Psychiatric or psychotic disorders can occur at any time.

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 3,000 patients

[2]

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 has initially elucidated the mechanisms of the disease

[3]

. 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 neurological diseases and a pioneer in the development of drug candidates targeting both neurons and glial cells. T he 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).

Next financial publication:

January 18, 2023 (before market)

: cash update as of December 31, 2022

More information at http://www.theranexus.com

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contacts

THERANEXUS

Thierry LAMBERT

Financial and Administrative Director

[email protected]

NEWCAP

Théo MARTIN/Pierre LAURENT


Investor Relations

+33 (0)1 44 71 94 97

[email protected]

FP2COM

Florence PORTEJOIE


Media relations

+ 33 (0)6 07 76 82 83

[email protected]

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 28, 2021 under number D. 21-0379, 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_Conference_JP_Morgan_FR.pdf

[2]

National Organization for Rare Disorders (NORD)/Orphanet

[3]

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


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