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Biopharmaceutical company Theranexus has entered into an exclusive global $ 20 million agreement with Beyond Batten Disease Foundation (BBDF) for an upcoming clinical trial and potential commercialization of juvenile Batten disease treatment candidate BBDF-101.

The pact follows a tentative June 27 agreement, in which Theranexus was given six months to finalize the details of the contract.

The latest agreement calls for Theranexus to pay specific amounts to BBDF for key milestones, including signing the contract, federal approval of treatment, and achieving commercialization targets. It also provides for royalty payments based on net sales.

Batten disease is a rare inherited neurodegenerative disease belonging to a group of disorders called neuronal ceroid lipofuscinosis (NCL). People with Batten disease have inefficient cellular waste centers called lysosomes; over time, brain cells fill up with toxic waste and die. In the United States and Europe, the juvenile form is the most common form of NCL, affecting nearly 3,000 individuals. Characteristics of the disease include vision loss, personality changes, behavioral problems, and slow learning.

BBDF-101 is a proprietary combination therapy intended to slow the progression of juvenile Batten disease (CLN3). It works by activating a molecule known as EB transcription factor (TFEB), a primary controller of lysosome production, which in theory could help cells remove toxic waste and slow disease progression. Similar to other Theranexus therapeutic candidates, the treatment is based on the synergistic effect of the active ingredients.

“Following the success of research sponsored by BBDF to improve understanding of disease mechanisms and identify BBDF-101 as a drug candidate, I am delighted with this partnership with Theranexus, which will allow the clinical development of the drug with a view to finally bringing a medical solution offering significant benefits to children and adolescents with this disease, ”said Craig Benson, president of BBDF, in a press release.

The trial, due to start this year, will compare the effectiveness of the candidate treatment to the natural progression of Batten, as documented in previous patient groups. Texas Children’s Hospital will be the primary investigative center for the trial, which will also assess safety and pharmacokinetics – how the drug travels through the body.

If the treatment is approved in the United States, the European company at the clinical stage plans to use the results of the trials to seek authorization for the therapy in Europe.

“As a pediatric neurologist, I am faced with cases of Batten disease in children and adolescents for which I unfortunately have no treatment at the present time,” said Gary Clark, head of childhood neurology at Texas Children’s Hospital and principal investigator of the trial. “All clinical teams are eager to assess the effectiveness of BBDF-101 for these patients in the near future. “

The clinical trial will include adolescent / adult and pediatric groups. It will begin with the recruitment of six adolescent / adult patients, who will receive BBDF 101 in escalating doses, with a tolerance and pharmacokinetics established over five months. Participants will be followed for security reasons. After safety and pharmacokinetic measurements, 30 pediatric patients will be recruited and evaluated for two years for disease progression.

“We are delighted to be involved in this partnership with BBDF, allowing Theranexus to extend its approach to lysosomal disorders affecting the nervous system,” said Frank Mouthon, CEO of Theranexus. “This asset is a constant addition to our portfolio, with considerable potential for value creation. We would like to thank BBDF and the patient families who support it for trusting Theranexus to lead the development and commercialization of BBDF for the benefit of patients.

Theranexus plans to enrich its platform with therapeutic candidates for lysosomal diseases associated with neurological conditions.

Theranexus is a biopharmaceutical company that designs and develops potential treatments for central nervous system disorders.

BBDF is a non-profit organization that funds studies on juvenile Batten disease.

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