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, XX August 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.

In late 2019, Theranexus and BBDF signed an agreement granting Theranexus an exclusive, global license agreement for the development and commercial use of drug candidate BBDF-101 for juvenile Batten disease. Batten disease belongs to a group of disorders referred to as neuronal ceroid lipofuscinoses (NCLs). BBDF funded research aimed at identifying and validating BBDF-101, a proprietary combination of drugs based on the synergistic effect of two active ingredients, like the other Theranexus drug candidates already in clinical development.

“We are delighted to have been awarded Orphan Drug Designation for BBDF-101 by the FDA. This is a sign of recognition for Batten disease and raises hopes for children and teens with this orphan disorder. I would like to say a big thank you to the entire BBDF team involved in the FDA submission, as well as to our donors, volunteers and the partner families of the foundation, without whom none of this would have been possible,” explains Craig Benson, Chair of the BBDF Board of Directors.

“We are delighted to have obtained Orphan Disease Designation and Rare Pediatric Disease Designation (RPDD). This marks a new milestone for Theranexus and BBDF in the development of the drug candidate BBDF-101. These new designations will speed up the approval process and provide at least seven years of post-approval protection and exemption from filing fees, as well as qualifying Theranexus on registration of BBDF-101 for a salable and transferable priority review voucher that can be used to speed up the approval process for any other drug,” continues Franck Mouthon, Theranexus Chairman and CEO.

The legal status of orphan designation in the United States was provided by the Orphan Drug Act of 4 July 1983. It qualifies the Company for accelerated review of its drug candidate 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.

The FDA defines rare pediatric diseases as diseases that are serious or life threatening and primarily affect individuals aged under 18, with fewer than 200,000 cases in the United States. 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. When the FDA awards Rare Pediatric Disease Designation, the sponsor of the trial qualifies for a priority review voucher, which may be sold to another company or transferred to another drug candidate. In such case the length of the approval procedure is also considerably shortened, generally taking just 6 months rather than 12 months on average, which gives a very high value to this priority review voucher.

After discussions with the FDA, Theranexus is currently preparing to launch a BBDF-101 preclinical trial to confirm the preclinical safety of BBDF-101 over a long exposure time, with the aim of supplementing the data already available so that the clinical program can be launched in 2021.

Contacts:

THERANEXUS
Thierry Lambert
Financial and Administrative Director
[email protected]

FP2COM
Florence Portejoie
Media Relations
+ 33 (0)6 07 76 82 83
[email protected]

BEYOND BATTEN DISEASE FOUNDATION
Mary Beth Kiser
President and CEO
[email protected]

ACTUS Finance & Communication
Thomas Ségouin
Investor Relations
+33 (0)1 53 67 36 75
[email protected]

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.

This signing of a worldwide exclusive license follows the agreement in principle of June 27th, which granted Theranexus a six-month exclusivity period to finalize a definitive agreement.

Batten disease is a rare, fatal, inherited pediatric disorder of the nervous system for which there is no treatment. It belongs to a group of disorders referred to as neuronal ceroid lipofuscinoses (NCLs). Beyond Batten Disease Foundation (BBDF) funded research aimed at identifying and validating BBDF-101, a proprietary combination of drugs relying on the synergistic effect of two active ingredients, similar to Theranexus’ drug candidates currently in clinical development.

The exclusive, global license agreement between BBDF and Theranexus covers the clinical development of drug candidate BBDF-101 to its approval and eventually its commercialization. Theranexus also intends to expand its research on its NeuroLead platform of drug candidates targeting lysosomal disorders associated with neurological symptoms.

“We are delighted to be involved in this partnership with BBDF, enabling Theranexus to extend its approach to lysosomal disorders affecting the nervous system. 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-101 for the benefit of patients,” explains Franck Mouthon, CEO of Theranexus.

In consideration for this license, the agreement provides for Theranexus to pay BBDF fixed sums on signing, approval, and achievement of commercial objectives post-approval. Moreover, the agreement provides for payment of royalties calculated based on net sales of BBDF-101 once it is marketed by Theranexus.

The clinical trial will begin in 2020 and include efficacy measurements comparing the development of various symptoms to the natural progression of disease previously documented in patient cohorts. The trial will also assess safety and pharmacokinetics. The lead investigation center for the trial will be Texas Children’s Hospital (TCH) in Houston, which is the fourth largest pediatric hospital in the United States.

According to initial talks with the FDA, if successful, this pivotal trial alone could be sufficient for approval of this drug candidate in the United States. Once it is approved in the United States, the company intends to apply for the approval of BBDF-101 in Europe on the basis of the same trial.

“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. All the clinical teams are eager to assess the efficacy of BBDF-101 for these patients soon,” adds Gary Clark, Professor and Chief of Child Neurology at the Texas Children’s Hospital and principal investigator for the clinical trial of BBDF-101. Joining Dr. Clark on the BBDF-101 Advisory Committee are leading experts on juvenile Batten disease including: Jonathan Mink, MD and Erika Augustine, MD at the University of Rochester; Angela Schulz, MD and Miriam Nickel, MD at the University Medical Center Hamburg-Eppendorf; Forbes Porter, MD, PhD, and An Dang Do, MD, PhD, at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). NICHD will also serve as the assessment site for the trial. Together, these experts have collected the combined largest natural history dataset in the Batten disease community worldwide.

“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. We are forever grateful to our donors, volunteers and Batten family partners who made this possible.,” concludes Craig Benson, Chairman of BBDF.

About Batten disease
Juvenile Batten disease, also known as Spielmeyer-Vogt or CLN3 disease, is a rare, fatal, inherited disorder of the nervous system for which there is no treatment or cure. Juvenile Batten disease belongs to a group of disorders referred to as neuronal ceroid lipofuscinoses (NCLs). Over 400 different errors in 13 genes have been attributed to various forms of NCL, which differ from one another primarily by when symptoms first appear. The first symptom in the juvenile form, progressive vision loss, appears between the ages of 4 and 6 which is followed by personality changes, behavioral problems, and slowed learning. Seizures commonly appear within 2-4 years of the onset of disease. Over time, patients continue to decline mentally and physically. Eventually, those affected become wheelchair-bound, are bedridden, and die prematurely. Psychiatric symptoms or psychosis can appear at any time. Juvenile Batten disease is always fatal; usually by the late teens to early 20s. In the United States and Europe, the juvenile form is the most common of the NCLs, which together, affect nearly 3,000 patients. In pathophysiological terms, interactions between neurons and glial cells play key roles in the emergence and progression of all the NCLs.

About Beyond Batten Disease Foundation
Beyond Batten Disease Foundation (BBDF) is the world’s largest nonprofit organization dedicated to funding research for a treatment and cure for juvenile (CLN3) Batten disease. Since its inception in 2008, over $35 million has been invested in research by leveraging donations, co-funding, and strategic partnerships. BBDF is spearheading a unique, cohesive strategy, incorporating independent scientific resources and collaboration with related organizations to drive research in juvenile Batten Disease. Today there is a treatment in sight. BBDF funded research has discovered a drug that slows the progression of the disease in Batten models and is pursuing an FDA approved clinical trial. More information can be found at www.beyondbatten.org

About the clinical trial of BBDF-101 for Batten disease
The clinical trial will include an adolescent/adult cohort and a pediatric cohort:

  • The trial will begin with the enrollment of an adolescent/adult cohort of six patients who will all be administered the drug BBDF-101 in escalating doses, with tolerability and pharmacokinetics established over 5 months. These patients will continue to be administered BBDF-101 throughout the trial and followed up for safety.
  • Once measurements of pharmacokinetics and tolerability have been performed for the adolescent/adult cohort, a pediatric cohort of 30 patients will be enrolled in the trial and undergo regular measurements to assess disease progression (vision, cognition, motor symptoms, etc.) over a period of two years.

At the end of the trial, patients’ data will be compared to natural disease progression as measured within cohorts already followed up by American and European academic teams.

About Theranexus
Theranexus is a clinical-stage biopharmaceutical company that emerged from the French Alternative Energies and Atomic Energy Commission (CEA) in 2013. It develops drug candidates for the treatment of nervous system diseases. Theranexus identified the key role played by non-neuronal cells (also known as “glial cells”) in the body’s response to psychotropic drugs (which target the neurons). The company is a pioneer in the design and development of drug candidates affecting the interaction between neurons and glial cells. The unique, patented technology used by Theranexus is designed to improve the efficacy of psychotropic drugs already approved and on the market, by combining them with a glial cell modulator. This strategy of combining its innovations with registered drugs means Theranexus can significantly reduce development time and costs and considerably increase the chance of its drugs reaching the market. The proprietary, adaptable Theranexus platform can generate different proprietary drug candidates offering high added-value for multiple indications. Theranexus is listed on the Euronext Growth market in Paris (FR0013286259- ALTHX). More information at www.theranexus.com

Contacts:

THERANEXUS
Thierry LAMBERT
Directeur Administratif et Financier
[email protected] 

FP2COM
Florence PORTEJOIE
Relations Médias
+ 33 (0)6 07 76 82 83
[email protected]

BEYOND BATTEN DISEASE FOUNDATION
Mary Beth Kiser
President and CEO
[email protected]

ACTUS Finance & Communication
Caroline LESAGE / Théo MARTIN
Relations Investisseurs
+33 (0)1 53 67 36 79 / +33 (0)1 53 67 36 75
[email protected]

BBDF Welcomes Delaney’s Dream

BBDF is proud to partner with Delaney’s Dream from West Virginia and welcomes them to our family of donor advised funds. By connecting affected families and communities, we are able to pool resources and better position ourselves to increase awareness and accelerate research. Delaney lost her battle with juvenile Batten disease on Jan. 21, 2019—less than 4 years after her diagnosis—but her family and friends remain dedicated to raising awareness and finding a cure for juvenile Batten disease. We are grateful for their continued interest and desire to help families and children still living with this disease.

“This quote, ‘It’s the little things that make life BIG,’ describes Delaney perfectly”, Delaney’s mom, Janet, shares. “It didn’t take much to make Delaney happy.”

Read more to learn about Delaney and her family.

Thank you from the Bensons

Dear Friends,

We would like to express our deepest gratitude for your help in successfully completing the Be Project. Eleven years ago, as parents faced with a daunting diagnosis and the enormous challenge of helping our child, we simply could not have reached this important milestone without you. Your love, support and devotion to our family and mission has been overwhelming, and we are so very grateful for you and feel incredibly blessed.

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!   

Our initial meeting with the FDA was very positive, and they have given us a clear road map to advance from here to clinical trials. We are checking off the FDA’s “to do list” as quickly as possible and are actively pursuing a pharmaceutical partner who can provide the funding and drug development expertise we need to initiate the trial. We are so very close, and anticipate a start date some time very soon. 

With much love and heartfelt gratitude from our family to yours, 

Charlotte and Craig

RARE Webinar: An Educational Webinar for the Batten Disease Community

BBDF recently participated in an informational webinar hosted by Global Genes. Mary Beth Kiser, BBDF President and CEO, was among several panelists, including representatives from Amicus Therapeutics, Nationwide Children’s Hospital, and Batten Disease Support and Research Association (BDSRA), providing updated information on Batten disease research and family resources.

Click here to download the slides or click the video link below to watch the full webinar.

Dr. Marco Sardiello’s Research on Batten Disease Paves Way for New Treatments

A team led by researchers at Baylor College of Medicine has uncovered an unexpected mechanism that can explain a form of Batten disease called neuronal ceroid lipofuscinosis 8. The findings published in the journal Nature Cell Biology provide potential new targets for future therapeutic interventions for this rare and incurable disease.

Click here to read the full article. 

Our Son Ollie – A Mother’s Story

by SUSIE EGR

This is our handsome son, Oliver James Egr. He is a big piece of our “why” and our superhero. He turned the big thirteen a few months back. When he was born I remember saying “Thank you God for all the abundance in our lives and for this sweet baby boy. He is perfect in every way and has now completed our family ”. He was the typical little boy- ornery but so dang cute. When he started kindergarten we noticed he kept sitting close to the TV and he wasn’t always paying attention. We soon learned he had visual issues. That led to months of additional neuro and genetic testing. I vividly recall that horrific day we received his official diagnosis.- Batten Disease. The genetic company called me on the phone while we were driving to Iowa City for an eye specialist appt for Ollie. To say it took my breath away is an understatement. Darin pulled the car over while I continued to listen on the phone. Ollman was singing “the ants go marching one by one” in the backseat (Not a care in the world.. while ours was being turned upside down) tears instantly flowed from my face and Darin just knew the results weren’t good. I remained quiet as I didn’t want to worry Ollie. That was the longest drive of our lives. After I got the news Dare and I just sat there holding hands with tears streaming. I felt helpless. “No cure….Fatal” is all I heard and it just kept playing over and over in my head. 

Ollie lost his complete vision 6 months after that appointment. Over the years we have seen this disease not only rear it’s ugly head in our son but take the lives of so many innocent, sweet kids. These affected families have became our extended family now. THIS HAS BECAME PERSONAL. 

My faith is strong and I remind myself frequently that our loving Father loves this child more then I will ever comprehend and he walks beside us daily as we fight the good fight. We feel his presence and see him through the kindness of others. That gives us hope. As parents we will never stop advocating for Ollie and the Batten community because they need us! What also gives us hope is putting our efforts towards funding a cure. We are so close!

BBDF 101 – Next Steps to Clinical Trials

In July, BBDF sponsored the Batten Disease Support and Research Association Family Conference where we had an opportunity to update affected families on the progress of our drug development program for the combination therapy BBDF 101.
Visiting one on one with parents at the conference allowed us to share information on the next steps required by the FDA to initiate clinical trials. We were also able to acknowledge the generosity and support of the BBDF community that created the opportunity for these children to receive the first ever treatment for juvenile Batten disease. On behalf of all the families we talked with, thank you for making this treatment a reality for their kids!
VIEW INFORMATIONAL POSTERS BELOW

Go Tyler! Beat Batten! – A Sister’s Story

by JAMIE KIM

My brother, Tyler, has always been the most energetic happy little boy I have ever been around.  He never runs out of things to do or things to say.

A few years ago, he started to have seizures and began to lose his vision.  Around a year ago, our family found out that Tyler has juvenile Batten disease. Batten disease is a rare and awful neurodegenerative disease that is terminal. There is currently no treatment or cure but there are researchers who have discovered a treatment that slows the progression of the disease and others are working on a possible cure for the disease.

When I first found out he had this disease, I was a little in disbelief.  I have seen many commercials of people diagnosed with terrible diseases but it never occured to me that it could happen to Tyler.  He seemed like such a happy and healthy boy that it was hard to understand that such a devastating disease was hurting him.

In the first few months of his diagnosis, I did not want to talk about it with anyone outside our family.  I did not know how to tell people what he was going through because it is not a common disease and I knew no one would really understand what was happening.  Our family began first with telling close friends and extended family. More recently, we joined other affected families and set up a fund at Beyond Batten Disease Foundation called Tyler’s Mission.  All donations to Tyler’s Mission go to the research for a treatment and cure for juvenile Batten disease. Seeing all the people who have donated to this cause has showed me how many people care and support our family and it really means so much to us.  It did not cross my mind that so many people who do not know us were also donating and leaving comments of support. Looking at these comments made me realize that it is possible to raise the 6 million dollars needed by next year to start a clinical trial for the treatment for kids living with Batten Disease. So far, Tyler’s Mission has raised over $45,0000! 

This summer, I want to make a difference.  I want to help raise money and awareness of Batten Disease.  It is urgent to raise the funds as quickly as possible because time is not on our side. With donations and support, there is hope for a treatment and ultimately a cure.

Every day I am reminded there is hope for my little brother and others with Batten Disease.  I love Tyler more than words can explain and he brings me happiness everyday from his little kisses to his big squeeze hugs to his goofy bad jokes.  Although he has hard days and struggles sometimes, he always hold his head up high and helps me see how much there is to appreciate in our lives.

Please help us raise funds and defeat Batten Disease by donating to Tyler’s Mission at beyondbatten.org/tylers-mission or visit the Understanding Batten page to learn more about Batten disease. To stay updated with his journey, follow his Instagram, Twitter, and Facebook @tylersmission.

GO TYLER! BEAT BATTEN!

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