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]

2nd Annual Bind Up Batten Golf Charity

Bind Up Batten brought together the community on July 20, 2019, for the 2nd Annual Batten Disease Golf Charity Outing. Over 80 golfers came out on a beautiful, cloudy day in Waterville, Ohio to raise $12,700 for juvenile Batten Disease research.

In addition to the golfers, Bind Up Batten would like to thank the families, friends, musicians, Fallen Timbers Fairways and all the sponsors who provided their support to help make this event happen.

Special thanks and recognition to Mike’s parents TJ and John Klein as well as Donna and Paul Iwinski for their incredible effort along with many other volunteers who made this day possible.

Together we can Bind Up Batten!

Watch the video

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!

Rare Sisters Hosts First-Ever Expect Miracles 5K

Rare Sisters brought together a community of over 1,000 supporters on Sunday, April 28 for the fund’s first-ever Expect Miracles 5K. Participants were treated to a beautiful Colorado Spring day at Lake Arbor in Arvada, where runners and families enjoyed a whole host of outdoor activities as well as a crowdsourced art project and local vendor fair. The event raised nearly $40,000 to support juvenile Batten disease research, advancing us closer to a clinical trial and viable treatment. Thank you to all the runners (present and virtual), supporters, sponsors, and volunteers who came together to make the first annual Expect Miracles 5K a tremendous success. Special thanks and recognition to Beth Fries, mother and co-founder of Rare Sisters, who organized the Expect Miracles 5k and inspired good among so many.

Austin Takes a Swing at Batten

Blue skies, sunshine, and near-perfect temperatures provided the ideal setting for the BBDF Youth Leadership Committee’s (YLC) second annual Taking a Swing at Batten golf tournament. On Sunday, April 14, YLC members, together with friends, family, and local supporters, teed up at Topgolf Austin to help raise nearly $9,000 to support Batten disease research. Thank you to everyone came out and took a BIG swing at Batten and to our generous sponsors: Amicus Therapeutics, Wadsworth Golf Charities, Fosforus, Jenna Rose, Elle McCartt, Carson Kurad, and Jackson Hanson. Special thanks to President Carson Kurad, President-Elect Elle McCartt, and Member-At-Large Grace Meinzer for their hard work organizing this event.

Battlin’ Batten in Baffin Fishing Tournament Exceeds $400,000 in Total Donations!

Friends and supporters of Beyond Batten Disease Foundation gathered on Sunday, April 7 for the 11th annual Battlin’ Batten in Baffin Fishing Tournament in Baffin, TX. The event raised more than $65,000 to support Batten disease research, bringing the event’s 11-year fundraising total to more than $400,000. Thank you to Neal Meinzer, who hosted the incredibly successful event, J. McCartt, the MVP of fundraising, and to all the fishermen and supporters who donated on their behalf. We also wish to congratulate this year’s winning team, the Trout Snobs (pictured above, left to right: Len Wright, Ken Adolph, Jim Arnold, and Kelly Dwyer).

Jon and Beth Fries Share Their Family’s Story on CBS Denver

Parents and Rare Sisters founders Jon and Beth Fries appeared on CBS Denver on Monday, April 8, where they shared the story of their two daughters Cecilia and Lilly who are currently living with juvenile Batten disease. Jon and Beth also invited the community to participate in the inaugural Expect Miracles Rare Sisters 5K, taking place Sunday, April 28 at Lake Arbor in Arvada, Colorado—sign up or make a donation at https://beyondbatten.org/events/expect_miracles_5k/

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.

Inaugural Batten Bash is a Great Success!

The Jackson, MI community came out in strong support of Bind Up Batten’s inaugural Batten Bash fundraising event, which raised more than $30,000! Simply incredible. With hors d’oeuvres, drinks, and a silent auction, guests enjoyed an evening of fun and giving at the historic Cascades Manor house. Thank you and congratulations to Angie Lefere for organizing and hosting such a successful fundraiser. And thank you to all the friends and generous sponsors for your support, which allows BBDF to continue leading the way in scientific research with the hope of finding treatments and a cure for juvenile Batten disease for Charleston and children like him.

 

Get Involved: April Fundraising Events

There are ample opportunities during the month of April to get involved and support BBDF. Make sure to visit each event page for information on how to participate, including options for those who can’t physically be present. 

 

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