BBDF Announces Agreement with Actelion Pharmaceuticals Ltd to Provide Drug Product for BBDF-101

Austin, Texas, March 2, 2021

Beyond Batten Disease Foundation (BBDF) is pleased to announce an agreement with Actelion Pharmaceuticals Ltd, a Janssen Pharmaceutical Company of Johnson & Johnson, to provide Zavesca® (miglustat) for the development of BBDF-101, a proprietary combination of miglustat and trehalose to treat juvenile Batten disease (CLN3). Janssen will provide drug supply for the clinical trials and Extended Access Program, and right of reference to data within the Zavesca New Drug Application (NDA) to support the development of BBDF-101.

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BBDF and Theranexus collaborate to publish journal

BBDF and Theranexus collaborate to publish “Evaluating and modulating TFEB in the control of autophagy: toward new treatments in CNS disorders” in the journal Fundamental & Clinical Pharmacology

Intense scrutiny of patient cells and animal models of CLN3 disease over the past 3 decades demonstrates that the loss of CLN3 protein impacts multiple cellular functions. The majority of dysfunction is in cellular compartments responsible for cellular recycling and waste management. This review, co-authored by Beyond Batten Disease Foundation and Theranexus, describes how drug-mediated activation of transcription factor EB (TFEB) clears toxic waste from Batten and other neurodegenerative disease models and describes ongoing pharmaceutical plans to use TFEB activators to treat these diseases.

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Manuscript accepted by the journal

NEUROPHARMA: Evaluating and modulating TFEB in the control of autophagy: towards new treatments in CNS disorders

Manuscript by da Costa, Anaelle; Metais, Thibaud; Mouthon, Franck; Kerkovich, Danielle; Charvériat, Mathieu, was successfully accepted by the journal “Fundamental & Clinical Pharmacology.”

BBDF-101 Announcement

We are excited to announce the following major milestone for BBDF!

The FDA has awarded Orphan Drug and Rare Pediatric Disease designations to Beyond Batten Disease Foundation for BBDF-101. The aim of the program is to facilitate the development of new drugs and biological products for the prevention and treatment of rare pediatric diseases. These designations provide accelerated review for approval, support with the FDA regulatory process and at least seven years of post-approval protection, as well as exemption from filing fees that normally have to be paid to the FDA. These designations do not impact the clinical trial, but are beneficial once the therapy receives New Drug Approval. We are grateful to Theranexus for their continued partnership and commitment to initiating a clinical trial for BBDF-101 as quickly as possible.

THERANEXUS AND BBDF OBTAIN ORPHAN DRUG DESIGNATION (ODD) AND RARE PEDIATRIC DISEASE DESIGNATION (RPDD) FROM THE FOOD AND DRUG ADMINISTRATION (FDA) FOR BBDF-101 FOR BATTEN DISEASE

  • Orphan Drug Designation (ODD) is a status that provides seven years of additional post-approval protection and exemption from filing fees
  • Rare Pediatric Disease Designation (RPDD) qualifies the sponsor at the time of registration for a salable, transferable priority review voucher which can be used to speed up the approval process for another drug candidate

Lyon, August 28, 2020 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and Beyond Batten Disease Foundation (BBDF) today announced the decision by the Food and Drug Administration (FDA) to award Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to the drug candidate BBDF-101 for Batten disease, a rare, fatal, genetic disorder of the nervous system for which there is no treatment.

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BBDF-101 Announcement

We are excited to announce the following major milestone for BBDF! 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.

Theranexus and Beyond Batten Disease Foundation (BBDF) announce the signing of a worldwide exclusive license for Batten disease drug candidate BBDF-101
  • The agreement covers the development and marketing of the drug following a single clinical trial due to begin in 2020. 
  • If successful, this trial will lead directly to the drug’s approval.

Lyon, 12 December 2019 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases and pioneer in the development of drug candidates modulating the interaction between neurons and glial cells, is pleased to announce the signing of an agreement with Beyond Batten Disease Foundation, granting it a worldwide exclusive license to develop and commercialize the drug candidate BBDF-101 for juvenile Batten disease.

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Beyond Batten Funded Research Professor Awarded

Congratulations to Marco Sardiello, Ph.D. Assistant Professor Molecular &Human Genetics Department, Baylor College of Medicine and BBDF-funded researcher on winning the 2019 Michael E. DeBakey M.D. Award for Research Excellence.  Dr. Sardiello is being recognized for his exemplary work in Batten disease.

We are grateful to his dedication to our families and are thrilled that he has been recognized with this prominent award.

Well done and well deserved!

BE Project – Thank You from the Bensons

BE Project – Thank you from the Bensons from Beyond Batten Disease Foundation on Vimeo.

In 2016, after 8 years of funding research for Batten disease, our research team was ready to take the next steps to advance our discovery of a treatment to slow the progression of Batten disease. As you know, The Be Project was launched to raise $6 million in 24 months in order to validate their findings, create patient registries, and advance a host of other necessary steps to prepare for FDA engagement and clinical trials. And, we did just that. WE DID IT!!! We achieved our goal, and we hope you feel so proud and take ownership in this enormous accomplishment!