Batten-1 Advances Toward Pivotal Phase 3 Trial as THX Pharma, Biocodex, and BBDF Unite on International Batten Disease Awareness Day
Key Insights
THX Pharma (search), Biocodex, and the Beyond Batten Disease Foundation jointly serve as Platinum Sponsors of the 2026 BDSRA Annual Family Conference, marking International Batten Disease Awareness Day on June 9.
The partners will deliver an update on Batten-1, an oral miglustat-based investigational therapy, and the planned initiation of an international pivotal Phase 3 study later in 2026.
Phase 1/2 results demonstrated a favorable safety profile, slowed motor symptom progression, reduced neurofilament light chain (search) levels, and real-world data suggesting clinical benefit on visual acuity.
On International Batten Disease Awareness Day, June 9, 2026, THX Pharma (search) (Theranexus), Biocodex, and the Beyond Batten Disease Foundation (BBDF) reaffirmed their shared commitment to the CLN3 Batten disease (search) community with a landmark announcement: the three partners will jointly serve as Platinum Sponsors of the 2026 BDSRA Annual Family Conference, where they will present an update on the Batten-1 program and the imminent launch of an international pivotal Phase 3 clinical study.
The BDSRA Annual Family Conference, hosted by the Batten Disease Support, Research and Advocacy Foundation, will take place from July 10 to 12, 2026, at The Westin Chicago Lombard in Illinois. As the largest gathering of Batten disease families, clinicians, researchers, and industry partners in North America, the conference serves as a critical forum for community connection and the exchange of scientific advances.
A Disease Without Approved Treatment
CLN3 Batten disease (search), also known as juvenile Batten disease, is a rare inherited neurodegenerative disorder that primarily affects children. Families typically witness the onset of vision loss in the first decade of life, followed by seizures, progressive motor decline, cognitive deterioration, and premature death. To date, no approved disease-modifying treatment exists for CLN3 Batten disease, leaving patients and families with extremely limited therapeutic options.
Batten-1: Building the Evidence Base
Batten-1 is an investigational, orally administered, miglustat-based therapy designed to slow the progression of CLN3 Batten disease (search). The 18-month results from the Phase 1/2 Batten-1-01 study (NCT05174039), conducted in six young adult patients, demonstrated a favorable safety and pharmacokinetic profile alongside encouraging biomarker and clinical signals. Notably, the study showed a slowdown of motor symptom progression as measured by the modified UBDRS Physical Assessment score and a reduction in neurofilament light chain (search) (NfL) levels, a recognized biomarker of neurodegeneration.
These findings were further reinforced in 2025 by real-world data generated in collaboration with BBDF, which suggested a clinical benefit of miglustat on visual acuity in CLN3 patients.
The Road to Phase 3
Building on this body of evidence, the partners are now preparing to initiate the international pivotal Phase 3 clinical study of Batten-1 later in 2026. The study is funded by Biocodex under the terms of a global licensing agreement announced in February 2026 and is intended to support future Marketing Authorization Applications in major regions, with the objective of bringing Batten-1 to patients by 2029.
Mathieu Charvériat, President and Chief Executive Officer of THX Pharma (search), underscored the significance of the moment: "International Batten Disease Awareness Day is, for all of us at THX Pharma, a moment of unity with the families and patients and motivation for us. The progress made from the positive Phase 1/2 results, through the data confirming a clinical benefit on visual function, to the imminent launch of our phase 3 study — has only been possible thanks to the trust and commitment of the CLN3 community and of our partners BBDF and Biocodex."
Nicolas Coudurier, Chief Executive Officer of Biocodex, highlighted the company's three-decade commitment to rare diseases: "We are deeply committed to advancing Batten-1 with the ambition of making this treatment available to patients by 2029, with the scientific rigor that the families affected by CLN3 disease deserve."
Craig Benson, Founder and Chairman of BBDF, reflected on the foundation's 18-year journey: "For our families, every June 9 carries both grief and resolve. Eighteen years after BBDF was founded, we are now on the verge of something we could only dream of in 2008: an international pivotal Phase 3 trial for a candidate therapy aimed at slowing CLN3 Batten disease (search)."
A Partnership Rooted in Patient Advocacy
BBDF, the world's largest nonprofit organization dedicated to funding research for juvenile Batten disease, has invested over $35 million in research since its founding in 2008. Biocodex, an independent French pharmaceutical group with recognized expertise in pediatric neurology and orphan diseases — most notably through Diacomit (stiripentol) in Dravet syndrome (search) — brings decades of experience in rare pediatric conditions. THX Pharma (search), listed on Euronext Growth Paris, specializes in innovative treatments for rare neurological diseases and also develops TX01 for Gaucher disease (search) and Niemann-Pick disease type C (search).
Representatives from all three organizations will be present throughout the July conference, engaging directly with families and delivering a joint update on the Batten-1 program and the Phase 3 study plans.