BBDF Co-Founder and Board Chair Craig Benson was interviewed by Kaiser Health News for a story focused on family foundations taking greater control in drug development. The article shares some of the challenges faced by rare disease groups and highlights organizations, like BBDF, who are overcoming those obstacles.
Theranexus announced yesterday that it is now, more than ever, committed to support patients with rare neurological disorders by focusing all its research efforts on these diseases, starting with CLN3. In this context, the project with Beyond Batten Disease Foundation (BBDF) becomes their flagship project and they have decided to dedicate all resources and efforts to make this project a success to benefit of the CLN3 disease community. Following the initiation of the ongoing Phase l/ll clinical study involving 6 young adult patients, Theranexus and BBDF are in the process of finalizing discussions with the FDA to initiate Phase III.
Lyon, France – Austin, TX, USA – 3 February 2022 – 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, and the Beyond Batten Disease Foundation (BBDF), are starting recruitment for Phase I/II clinical trial to evaluate BBDF-101 in Batten disease.
Franck Mouthon, Chairman of Theranexus, said: “The launch of recruitment is a key step in the development of our drug candidate BBDF-101. This trial will be conducted with BBDF and in collaboration with leading North American referral centers for Batten Disease, a rare, fatal, genetic disorder of the nervous system. Our ambition is to demonstrate the full potential of BBDF-101 in this disease for which there are currently no therapeutic options for patients.”
Following on from the award of Investigational New Drug (IND) approval from the Food and Drug Administration (FDA) for its BBDF-101 program in 2021, Theranexus is now actively recruiting patients for its Phase I/II clinical trial. During the 2-year trial, six patients 17-year-old or older will receive BBDF-101 in escalating doses during the first 4.5 months to establish tolerability and pharmacokinetics of trehalose, miglustat and a combination of trehalose/miglustat. The results of the titration phase will be available at the start of the second half of 2022.
After consulting with the FDA, at the end of 2022 Theranexus plans to start a pivotal Phase III trial involving a pediatric cohort of 30 patients with regular measurements to assess disease progression and safety. The aim will be to compare the development of various signs and symptoms in these patients, as rated on the Unified Batten Disease Rating Scale (UBDRS, neurological and behavioral symptoms, activities of daily living, etc.), over a period of two years, to data from a study of disease progression in a cohort of untreated patients followed for many years at the University of Rochester.
This Phase III trial will be conducted in collaboration with the leading referral centers for Batten disease in the United States which include the Texas Children’s Hospital in Houston, TX, the fourth largest pediatric hospital in the United States, the National Institutes of Health, and the University of Rochester Batten Center in Rochester, NY.
“It is with great gratitude for our patients’ families and donors that we now see the launch of the BBDF-101 clinical program which aims to offer a therapeutic solution for patients suffering from this very rare genetic disease. Achieving this major milestone for the Foundation, in collaboration with Theranexus, is an exciting culmination of almost 12 years of research,” concludes Craig Benson, Chair of the BBDF Board of Directors.
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[1] 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[2]. Today there is a treatment in sight. BBDF funded research has discovered a drug that slows the progression of the disease in Batten models. More information can be found at www.beyondbatten.org
About development of BBDF-101 for Batten disease and the partnership between BBDF and Theranexus
The research funded by BBDF and conducted by academics specializing in lysosomal storage disorders (particularly Baylor College of Medicine) led to the discovery of the drug candidate BBDF-101. Following a partnership agreement between BBDF and Theranexus concluded in late 2019 (see below), Theranexus supplemented preclinical development of BBDF-101 with research to establish product safety. Thanks to the research findings, BBDF-101 will now be able to enter the clinical phase following the grant of IND status by the FDA in September 2021.
The clinical development pathway includes:
The development of BBDF-101 is the result of a partnership concluded between BBDF and Theranexus in late 2019. The exclusive, global license agreement between BBDF and Theranexus covers the clinical development of drug candidate BBDF-101 pending approval and its commercial use. Theranexus also plans to continue its research on the NeuroLead platform of drug candidates targeting neurological forms of other lysosomal disorders.
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
| 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. Thanks to its knowledge of neuron and glial cell interactions, THERANEXUS is a pioneer in the design and combination of approved substances and has a solid and diversified portfolio of drug candidates in clinical-phase testing. The company’s combined drug repurposing strategy based on a solid commercial footing and a capability to rapidly demonstrate its clinical worth, enables it to produce different high-value-added proprietary drug candidates, significantly reduce development time and costs, and considerably increase the chance of its drugs reaching the market. Accordingly, THERANEXUS is well-positioned in several indications, including for Parkinson’s and Batten disease, for which there is currently no treatment available. Theranexus is listed on the Euronext Growth market in Paris (FR0013286259- ALTHX). More information on: http://www.theranexus.com Click and follow us on Twitter and Linkedln Contacts THERANEXUS Thierry LAMBERT Financial and Administrative Director investisseurs@theranexus.fr
NEWCAP Théo MARTIN/Pierre LAURENT Relations Investisseurs +33 (0)1 44 71 94 97 theranexus@newcap.eu
FP2COM Florence PORTEJOIE Media Relations + 33 (0)6 07 76 82 83 fportejoie@fp2com.fr |
[1] National Organization for Rare Disorders (NORD)/Orphanet
[2] Settembre et al, TFEB links autophagy to lysosomal biogenesis, Science 2011
The Beyond Batten Disease Foundation (BBDF) is sponsoring Care Beyond Diagnosis in its efforts to draft international guidelines to help clinicians counsel and treat patients with CLN3 disease, or juvenile Batten disease.
Based on clinical evidence and consensus-based recommendations, the guidelines are designed to improve patient care and disease management, as well as facilitate reimbursements.
“Having the support of BBDF is a huge achievement for our team,” Jeffrey Donohue, co-CEO of Care Beyond Diagnosis, said in a press release. “Obviously, the financial commitment is important for us to keep the program going, but looking ahead towards the publication of the guidelines, BBDF will be instrumental in dissemination of the guidelines into family/caregivers’ hands. Engagement from the patient community is a necessity in guideline development.”
Read the full article here.
The weekend of 7-10 October marked the 35th Reunion for the Naval Academy Class of 1986. The first event of the weekend was a golf outing at Compass Pointe Golf Course in Pasadena, MD. Seventy-seven classmates gathered to play golf and swap seas stories. But more importantly, the occasion was an opportunity to give back to a classmate in need. Through the selling of various on course items (mulligans, 2′ of string, opportunity to tee off from the fairway on one hole), and a raffle event after the tournament featuring items donated by classmates and local businesses, the Class raised $4862 for Tyler’s Mission in support of the son on classmate Jim Kim. It was a perfect ending to a perfect day. The Class could not be more inspired by the way Tyler lives his life and was honored to make this donation to his fund. Go Tyler! Beat Batten!
Beyond Batten Disease foundation announces formal sponsorship of Care Beyond Diagnosis in effort to establish Clinical Management Guidelines for CLN3 Disease.
Austin, TX – Raleigh, NC, September 27, 2021 – Beyond Batten Disease Foundation (BBDF) is pleased to announce a sponsorship agreement with Care Beyond Diagnosis (CBD) to establish internationally validated management guidelines for CLN3 Disease, an ultra-rare, fatal neurodegenerative genetic disorder.
Guidelines which have been methodically and transparently developed can both establish and improve upon a standard of care for patient management. Evidence and consensus-based recommendations for care can improve access to effective management and improve the reimbursement process by recognizing medical necessity.
“Having the support of BBDF is a huge achievement for our team. Obviously, the financial commitment is important for us to keep the program going, but looking ahead towards the publication of the guidelines, BBDF will be instrumental in dissemination of the guidelines into family/caregivers’ hands” says Care Beyond Diagnosis Co-CEO, Jeffrey Donohue. “Engagement from the patient community is a necessity in guideline development.”
President and CEO Mary Beth Kiser says, “BBDF has been keenly interested in guidelines for the CLN3 community for many years. Thanks to the work of Care Beyond the Diagnosis we are able to meet that need and know this will be a valuable resource for our dedicated community of physicians.”
The development program which began in October 2020 is on track to be completed by year end – 2022 ready for submission to a medical journal. CBD predicts a publication will be available for review by Q2 2023.
The effectiveness of clinical guidelines depends on editorial independence, transparency of process and a validated design. CBD organizes its guideline programs in accordance with the Appraisal of Guidelines for Research and Evaluation’s AGREE II Tool, a 23-point assessment used to assess clinical guidelines.
In the rare disease community, research is by nature, is lacking. A guideline development program identifies evidence gaps in existing literature, but through a Modified Delphi Consensus Program, generates new evidence where those gaps exist.
Guidelines for Rare Disease is currently an unmet need within the community. With over 7,000 rare diseases and a vast majority currently without a disease altering therapy, guidelines can offer caregivers and clinicians a tool to provide patients with the highest QoL possible.
CBD published guidelines for CLN2 Disease in April of 2021. The program was assessed by independent reviewers according to the AGREE II Tool and received a 5.7 out of 7, according to Donohue, was the highest assessment for a rare disease guideline publication.
About Beyond Batten Disease Foundation: Beyond Batten Disease Foundation (BBDF) is the world’s largest non-profit 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, coordinated strategy that incorporates independent scientific resources and collaborates with related organizations to drive research in CLN3 disease. More information can be found at www.beyondbatten.org.
About Care Beyond Diagnosis: Care Beyond Diagnosis (CBD) is a non-profit organization dedicated towards supporting patients, families and communities following a rare disease diagnosis. CBD has a vision for improved patient-outcomes through both a small scale, patient specific approach and through large scale, internationally collaborated Rare Disorder guideline development programs.
A big thank you to Drew, Cara, Mike, and all of the Batten 4 Drew supporters who made a $10,000 donation to Beyond Batten Disease Foundation possible!
Lyon, 7 September 2021 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and Beyond Batten Disease Foundation (BBDF), announce receipt of Investigational New Drug (IND) approval from the Food and Drug Administration (FDA) to launch a Phase I/II clinical trial of their drug candidate BBDF 101 for juvenile Batten disease, a rare, fatal, genetic disorder.
Batten disease belongs to a group of disorders referred to as neuronal ceroid lipofuscinoses (NCLs).
Juvenile Batten disease is a rare and terminal condition that causes blindness and seizures and is mentally and physically incapacitating with an average life expectancy of late teens or early twenties.
If you never believed in fairy tales, this may change your mind. On June first, my daughter, Christiane, who suffers from Juvenile Batten disease graduated from Austin High School. Against all odds, she walked in her class processional and made her way across the stage to claim her diploma. It was a milestone we could have only dreamed of 13 years ago when we received her diagnosis. We never imagined that the happy day of her high school graduation would come and that our tears would be tears of overwhelming joy and gratitude.
When Christiane came home from school one day before graduation, she announced that she was going to the Senior Prom. She had already imagined her dress and reported that she only needed help narrowing down her choices for an escort. Christiane had always been self-determined and independently-minded, so I flashed back to her sixth-grade year on a similar afternoon when she came home from school and pulled a yellow sheet of paper from her backpack announcing that she would be trying out for cheerleader.
A thousand logistical questions raced through my mind as she confidently reeled off a Rolodex of possible escorts ranging from childhood schoolmates and family friends, to a very complete list of every accomplished and handsome boy on the Austin High football team. After careful deliberation, she landed on Beau Beathard, a handsome two-time Westlake State Champion football player, Eagle Scout, and a schoolmate she’s known since she was a child. “Wow,” I thought giggling to myself in amusement, “I better check it out with his mom.” After all, why shoot for the stars when you can land on the moon!? I decided to float the idea past his mother first, in an effort to avoid overwhelming Beau or putting him on the spot. The next day I received this text that read, “Hi, Mrs. Benson, it’s Beau Beathard. I would be honored to go to prom with Christiane! Just let me know when. So happy to do this and I can’t wait. Thank y’all!”
I will never forget her broad smile and the complete delight that illuminated her face when I told her that Beau would be taking her to the dance. Every morning after that, the first thing she asked when I woke her up, was, “Is today Prom”? Christiane spent the next month imagining the perfect dress and spent countless hours imagining her first date. She very specifically wanted a dress that didn’t touch the ground “so she wouldn’t trip while she was dancing.” Of course, she was imagining that, too!
When the day finally came, Beau came to pick up Christiane and I watched from behind as he guided her to the car. He so intuitively held her flowers so she had a free hand to use her cane, plus he seamlessly offered his arm to guide her as they made slow but eventual progress down the sidewalk to the car. There was absolutely not a single awkward moment or hesitation in initiation or leadership. With unflappable confidence, Beau spent the entire evening patiently and attentively attending to Christiane in every possible way with his fixed attention solely on her happiness. As I was a chaperone at the dance that night, I watched in tearful amazement at the incredible selflessness of The Boy with the Golden Heart. Knowing that Christiane loves to dance and sitting face to face in two chairs, Beau sweetly took Christiane’s hands and with absolutely no regard for how he may appear, he began dancing with her while she sat in her chair. After the first song, Christiane leaned over to me and while handing me her cane, she said “Hold this mom, I’m going to the dance floor!” Determinedly, she inched herself forward to the edge of her seat and while using the table for support, she pulled herself out of her chair with unprecedented enthusiasm. He held her hand over her head and in spite of her vision loss and coordinated efforts, Beau steadied her as she twirled around exactly as she imagined she would.
For the grand finale, Christiane was surprised when Beau escorted her to the stage to receive a crown and title of “Prom Royalty.”
The next day, I sent Beau a text to tell him that the evening had been the highlight of Christiane’s life, what a special person he was and that I believed God had used him in a special way inspiring many by his example. He texted back and said, “I’m just really happy Christiane got to have a fun night. Thank y’all so much for letting me go to prom with her. I’m always around if you ever need anything at all, so please do not hesitate to text me. I would be beyond proud if I inspired one boy to do the right thing!” I am sure he did. I know he inspired me and I am humbled to know an angel among us who could show us all what fairy tales are really made of.
I never saw Christiane’s smile leave her face that evening. Not once. I can confidently say it was the happiest occasion of her life and for an evening, she lived her own “happily ever after” in a beautiful story that only God could write. It was her very own fairytale that will forever be etched in her memory and The Boy with the Golden Heart will forever be etched in mine.
Written by Charlotte Benson, Christiane’s mother.
