Theranexus and BBDF Announce Agreement for Juvenile Batten Disease Treatment Trial

Theranexus and BBDF Announce Agreement for Juvenile Batten Disease Treatment Trial
0
(0)

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.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
Total Posts: 14
Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.
×
Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours d’Elegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled “Belle of the Concours.”
Latest Posts
  • genetic testing and counseling

How useful was this post?

Click on a star to rate it!

Average rating 0 / 5. Vote count: 0

No votes so far! Be the first to rate this post.

As you found this post useful...

Follow us on social media!

We are sorry that this post was not useful for you!

Let us improve this post!

Tell us how we can improve this post?