BBDF 101

Combination treatment to slow the progression of juvenile Batten (CLN3) disease

History of BBDF 101


Finding a solution to the problem
and identifying potential drugs for treatment

  • Beyond Batten Disease Foundation (BBDF) opens its doors and begins looking for a solution to fix what goes wrong in Batten-affected cells. Children with Batten have cellular waste centers (lysosomes) that work, but they work inefficiently. Over time, brain cells fill with toxic waste material and die.
  • Transcription Factor –EB (TFEB), a master controller of lysosome production is discovered. If researchers can activate TFEB in Batten-affected cells, maybe each  cell could clear itself, staving off its own death and slowing disease.
  • BBDF grants TFEB discovery team $2.5 million to join the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital to apply TFEB activation to juvenile Batten (CLN3 disease). Housed on the largest medical center campus in the world, researchers have access to unprecedented resources.


  • NRI investigators, together with international collaborators, demonstrate TFEB activated lysosome production is safe and effective at clearing accumulated waste material across multiple healthy and diseased animals throughout their development.
  • Three years and an additional $1.8 million in BBDF-funding for labor-intensive searches and 100s of experiments lead to the identification of 6 potential drugs to treat CLN3 disease via TFEB activation.


100s of experiments for dosing, safety and efficacy

  • $2.2 million in BBDF-funding to German pharmaceutical-grade contract research organization, Evotec, leads to the prioritization of Drug #1, the most promising compound with little to no side-effects.
  • Evotec and others conduct 125 experiments to determine that the best way for Drug #1 to reach the brain is through intravenous administration.
  • Leveraging BBDF’s investments with over $3 million in nonprofit and NIH-funding leads to the addition of Drug #2. Drug #2 enhances the ability of Drug #1 to reach the brain, therefore, creating the combination therapy BBDF 101.


  • NRI researchers learn that, independent of Drug #1, Drug #2 clears a subset of waste material and inhibits the harmful effects of chronic inflammation in the brain, resulting in additional unexpected benefits of the drug combination.
  • Evotec scientists conduct hundreds of experiments to determine the correct dosage for each drug independently, and in-combination with one another.


Preparing for clinical trial success

  • Camargo Regulatory Strategists join BBDF to bring BBDF-101 to the U.S. Federal Drug Administration (FDA) for permission to enter into a clinical trial.
  • After almost 9 years and over $23 million invested in overall research, the teams from BBDF and Camargo, along with key opinion leaders in Batten disease from University of Rochester, University Medical Center Hamburg-Eppendorf, Texas Children’s Hospital and BDSRA, meet with the FDA for BBDF 101’s Pre Investigational New Drug (PreIND) meeting. This is the first formal step in clinical trial development.


Major BBDF Announcement

  • Theranexus, a publicly-traded European pharmaceutical company, has committed the $20 million and expertise necessary to complete the clinical trial and commercialization for our drug discovery, BBDF-101.
Press Release »


Major BBDF Announcement

  • The FDA has awarded Orphan Drug and Rare Pediatric Disease designations to Beyond Batten Disease Foundation for BBDF-101
Press Release »


Major BBDF Announcement

  • BBDF 101 Wins FDA Investigational New Drug Approval (IND)
    The clinical trial program will start with Phase I/II by the end of 2021.

Press Release >>

BBDF 101 Now

Pre Investigational New Drug (Pre IND) Meeting
at the FDA on May 10, 2018

This spring BBDF had its Pre IND meeting with the FDA to get input and direction on our plan for clinical trials. This meeting was a huge milestone and an important first step in the process to develop BBDF’s drug combination, BBDF 101. (Pictured above: key opinion leaders and BBDF team at the FDA). The FDA gave very positive feedback and it was clear they want to help us reach our goal. The FDA guidelines provide a clear path forward for our program and do include some action items for us to address which will help set us up for success when we submit BBDF 101 for New Drug Approval. Read on for more detailed information about each step in the process:

Toxicity Study

A Juvenile Toxicity Study is required by the FDA prior to testing BBDF 101 in children. Even though there is a great deal of safety data on the two drugs, the FDA would like to see more on the combination to determine safety in a younger population. This study will be conducted in animals and should take approximately 6 weeks to complete.


Pre Market

The FDA will require a safety study in healthy adults prior to New Drug Approval.

A Mouse Factorial Study will be required by the FDA for New Drug Approval to better correlate animal endpoints to humans. Both studies will be run in parallel with Phase III.


FDA New Drug
Approval (NDA)

After completing the steps outlined above, BBDF can apply for NDA by the FDA. Once approved this treatment will be made available to the public and should be eligible for coverage by insurance.

*Expanded Access/Compassionate Use

Based on the FDA’s feedback, BBDF is pursuing Expanded Access, also known as Compassionate Use, in patients over 18 years of age. This could start prior to Phase III.


The FDA encouraged the application for Orphan Drug Designation (ODD) and Rare Disease Pediatric Review Voucher (RDPRV). These two special drug designations could make BBDF 101 attractive for pharmaceutical investment.

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