Theranexus and BBDF Finalize Recruitment For Phase I/II Trial to Evaluate Batten-1 in Batten Disease

Lyon, France – Austin, Texas, United States – 6 September 2022 – 6 pm CEST – 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), have today announced finalization of recruitment of the last patient for the Phase I/II clinical trial to evaluate the tolerability, pharmacokinetics of the Batten-1 drug candidate in juvenile (CLN3) Batten disease.

A total of six patients with juvenile Batten disease aged 17 and over are enrolled in the Phase I/II clinical trial. The Batten-1 drug candidate containing the active ingredient miglustat is administered in escalating doses. The initial tolerability and pharmacokinetic data for miglustat obtained after the first 9 weeks of treatment in this population will serve to initiate Phase III, and Phase I/II patients are being treated and evaluated over a 24-month period.

Franck Mouthon, Chairman of Theranexus, said: “Finalization of recruitment for our Phase I/II trial is an important milestone in the development of Batten-1, a drug candidate that aims to significantly slow progression of juvenile Batten disease for which there is currently no available treatment. Our goal for the next stages in the development process is to launch an international pivotal Phase II/III trial in 2023.”

Craig Benson, Chair of the Beyond Batten Disease Foundation, added: “As parents and representatives of patients with juvenile Batten disease and their families, we are delighted with the enrollment of the last patient in the Phase I/II trial. This new Batten-1 treatment offers tremendous hope for our children affected by the disease.”

The Phase I/II trial is being conducted by referral centers for Batten disease in the United States, with Dr. Gary Clark, Chief of Child Neurology at Texas Children’s Hospital in Houston, as principal investigator.

About Batten-1
Batten-1 is a novel and exclusive proprietary drug containing the active ingredient miglustat. The mechanism of action of this substance blocks the accumulation of glycosphingolipids and neuroinflammation to prevent brain cell death. In the Phase I/II trial, Batten-1 is administered to patients over 17 years of age in solid form, and in Phase III it will be administered in a liquid form better suited to children.

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 – Batten-1 – that slows the progression of the disease in Batten models. More information can be found at www.beyondbatten.org

About Theranexus
Theranexus is an innovative biopharmaceutical company that emerged from the French Alternative Energies and Atomic Energy Commission (CEA). It specializes in the treatment of central nervous system disorders and is a pioneer in the development of drug candidates targeting both neurons and glial cells.

The company has a unique platform for the identification and characterization of advanced therapy drug candidates targeting rare neurological disorders and an initial drug candidate in clinical development for Batten disease.
Theranexus is listed on the Euronext Growth market in Paris (FR0013286259- ALTHX).

Next financial publication:
Tuesday 27 September 2022: First half 2022 financial results

More information at http://www.theranexus.com

Contacts
THERANEXUS
Thierry Lambert
Chief Financial Officer
contact@theranexus.com

NEWCAP
Théo Martin/Pierre Laurent
Investor Relations
+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

Disclaimer
This press release contains certain forward-looking statements concerning Theranexus and its business, including its prospects and product candidate development. Such forward-looking statements are based on assumptions that Theranexus considers to be reasonable. However, there can be no assurance that the estimates contained in such forward-looking statements will be verified, which estimates are subject to numerous risks including the risks set forth in the universal registration document of Theranexus filed with the AMF on 28 April 2021 under number D.21-0379 (a copy of which is available on www.theranexus.com) and to the development of economic conditions, financial markets and the markets in which Theranexus operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Theranexus or not currently considered material by Theranexus. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Theranexus to be materially different from such forward-looking statements. Theranexus expressly declines any obligation to update such forward-looking statements.

 

[1] National Organization for Rare Disorders (NORD)/Orphanet
[2] Settembre et al, TFEB links autophagy to lysosomal biogenesis, Science 2011

Craig Benson Interview with KHN

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.

BBDF 101 Update

BBDF 101 Update

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.

Read the press release here

BBDF-101 PHASE I/II TRIAL UNDERWAY

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:

  • Phase I/II for which recruitment began today,
  • Phase III due to start by the end of 2022 and to include 30 patients with the aim of achieving final results in the second half of 2025,

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

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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

Foundation Helps Sponsor Drafting of Guidelines for Juvenile Batten

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.

USNA Class of 1986 Fundraiser for Tyler’s Mission

USNA Class of 1986 Fundraiser for Tyler’s Mission

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

 

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.

www.carebeyonddiagnosis.org

BBDF 101 Wins FDA Investigational New Drug Approval to Pursue Clinical Trials

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).

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