Theranexus and BBDF Announce Agreement for Juvenile Batten Disease Treatment Trial

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by Mary Chapman |

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Biopharmaceutical company Theranexus has reached an exclusive $20 million global 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 June 27 agreement in principle, in which Theranexus was granted six months to finalize contract details.

The latest agreement calls for Theranexus to pay BBDF specific amounts for milestones including the contract’s signing, the treatment’s federal approval, and achievement of marketing goals. It also provides for royalty payments based on net sales.

Batten disease is a rare, inherited neurodegenerative disease, belonging to a group of disorders known as neuronal ceroid lipofuscinoses (NCLs). People with Batten disease have inefficient cellular waste centers called lysosomes; over time, brain cells fill with toxic waste material and die. In the United States and Europe, the juvenile form is the most common of the NCLs, which affect nearly 3,000 individuals. Disease hallmarks include vision loss, personality changes, behavioral problems, and slowed learning.

BBDF-101 is a proprietary combination therapy intended to slow progression of juvenile Batten disease (CLN3). It works by activating a molecule known as transcription factor EB (TFEB), a master controller of lysosome production, which, in theory, could help cells clear toxic waste material and slow disease progression. Similar to other Theranexus therapeutic candidates, the treatment is based on the synergistic effect of 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 thrilled about this partnership with Theranexus, which will enable the drug’s clinical development with a view to finally providing a medical solution offering considerable benefits to children and teens with this disease,” Craig Benson, chairman of BBDF, said in a press release.

Set to begin this year, the trial will compare the treatment candidate’s effectiveness to the natural progression of Batten as documented in previous groups of patients. Texas Children’s Hospital will be the lead investigation center for the trial, which also will assess safety and pharmacokinetics — how the medicine moves through the body.

If the treatment is approved in the U.S. the clinical-stage European company plans to use trial results to apply for the therapy’s licensing in Europe.

“As a pediatric neurologist, I am faced with cases of Batten disease in children and teens for whom I sadly have no treatment at present,” said Gary Clark, chief of child neurology at Texas Children’s Hospital, and the trial’s principal investigator. “All the clinical teams are eager to assess the efficacy of BBDF-101 for these patients soon.”

The clinical trial will include adolescent/adult and pediatric groups. It will begin with the enrollment of six adolescent/adult patients, who will be administered BBDF 101 in escalating doses, with tolerability and pharmacokinetics established over five months. Participants will be followed up for safety. Following tolerability and pharmacokinetics measurements, 30 pediatric patients will be enrolled and assessed for two years for disease progression.

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

Theranexus plans to enhance its platform of treatment candidates aimed at 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 nonprofit organization funding studies of juvenile Batten disease.