Beren Therapeutics to Present Comprehensive Adrabetadex Data at 2026 NPC Research Conference, Highlighting Early Treatment Benefits and Mechanistic Insights
Beren Therapeutics P.B.C., the parent company of Mandos LLC and a biotechnology company focused on cholesterol trafficking biology and cyclodextrin-based therapeutic approaches, has announced that it will present six scientific posters at the 2026 Michael, Marcia, and Christa Parseghian Scientific Conference for Niemann-Pick Type C (NPC) Research. The conference, sponsored by the family of former Notre Dame head football coach Ara Parseghian, will take place from May 30 to June 2 in Tucson, Arizona.
The presentations will feature new clinical and preclinical findings on adrabetadex, an investigational cyclodextrin-based therapy currently under regulatory review by the U.S. Food and Drug Administration (FDA) for the treatment of infantile-onset Niemann-Pick disease type C (I-NPC). In addition to the poster presentations, Beren Therapeutics’ Chief Executive Officer Jason Camm will participate in a fireside chat with Sean Kassen, Director of the Ara Parseghian Medical Research Fund, on May 31 at 4 p.m. MST.
The company’s presence at the conference underscores its ongoing commitment to advancing research in NPC, a rare and devastating neurodegenerative disorder with significant unmet medical need.
Advancing Understanding of Niemann-Pick Disease Type C
Niemann-Pick disease type C is a rare, inherited lysosomal storage disorder characterized by the abnormal accumulation of cholesterol and other lipids within cells. This buildup disrupts normal cellular function, particularly in the central nervous system, leading to progressive neurological decline.
Infantile-onset NPC represents one of the most severe forms of the disease, with early onset symptoms and rapid neurodegeneration. Despite advances in supportive care and emerging therapies, treatment options remain limited, and the disease continues to pose a significant clinical challenge.
Beren Therapeutics is developing adrabetadex as a potential disease-modifying therapy designed to restore intracellular cholesterol trafficking and reduce pathological lipid accumulation. The investigational treatment is based on cyclodextrin chemistry and is administered centrally to target neurological manifestations of NPC.
The upcoming conference presentations will provide new insights into both the clinical outcomes and underlying biological mechanisms associated with adrabetadex therapy.
Six Poster Presentations Highlight Clinical and Preclinical Findings
At the conference, Beren Therapeutics will present six posters spanning clinical outcomes, treatment paradigms, combination therapy approaches, and mechanistic studies. Together, these data sets aim to deepen scientific understanding of how adrabetadex may impact disease progression in infantile-onset NPC.
Early Treatment and Long-Term Outcomes
Two posters focus on the importance of early intervention and long-term clinical outcomes in patients treated with adrabetadex.
Poster #25 examines outcomes in participants with infantile-onset NPC who received prompt and sustained treatment with adrabetadex. The findings suggest that earlier administration of the therapy may be associated with slower disease progression compared with published natural history data. These observations reinforce the importance of early diagnosis and timely initiation of treatment.
Poster #26 evaluates survival outcomes based on baseline disease burden in patients treated with adrabetadex, including those enrolled in clinical studies and the Expanded Access Program. Retrospective analyses indicate that patients with lower disease burden at baseline tend to experience improved survival outcomes, highlighting the critical role of early therapeutic intervention before irreversible neurological damage occurs.
Combination Therapy and Emerging Treatment Paradigms
Two additional posters explore the use of adrabetadex in combination with other approved or investigational NPC therapies.
Poster #6 presents early evidence of an evolving treatment paradigm involving the use of adrabetadex alongside oral therapies such as arimoclomol and N-acetyl-L-leucine. This represents the first systematic characterization of intrathecal adrabetadex use in combination with these agents. Data from the Expanded Access Program suggest that adrabetadex may function as a foundational therapy that can be complemented by additional treatments based on individual patient needs.
Poster #2 provides preclinical data from an in vitro mouse model of NPC evaluating adrabetadex alone and in combination with arimoclomol and levacetylleucine. The results indicate that adrabetadex promotes oligodendrocyte proliferation, synaptic network development, and myelination. In comparative analyses, adrabetadex demonstrated greater potency than approved NPC therapies across several measures of neuronal and glial health. Importantly, combination treatments achieved similar biological effects at lower concentrations, suggesting potential mechanistic compatibility between adrabetadex and other therapeutic agents.
Mechanism of Action and Disease Modification
The final two posters focus on the mechanism of action and potential disease-modifying effects of adrabetadex.
Poster #18.1 examines the mechanism of action of centrally administered adrabetadex in NPC. Preclinical data indicate broad distribution within the central nervous system and sustained engagement with intracellular cholesterol. These findings support the hypothesis that adrabetadex may help restore impaired cholesterol trafficking in both superficial and deeper brain regions.
Poster #18.2 explores the disease-modifying potential of adrabetadex in NPC mouse models. Results suggest improvements in key neurological parameters, including myelination, neuronal integrity, and biomarkers associated with neurodegeneration. These mechanistic findings provide additional support for the potential clinical benefits observed in human studies.
Expert Commentary Highlights Importance of Early Intervention
Beren Therapeutics emphasized the significance of the new data in advancing understanding of infantile-onset NPC and reinforcing the importance of early treatment.
Dr. Irene von Hennigs, PharmD, Senior Vice President of Medical Affairs at Beren Therapeutics P.B.C., noted that the company is committed to sharing scientific findings openly and in collaboration with the broader NPC research community.
She stated that the emerging data deepen understanding of adrabetadex’s role in infantile-onset NPC and reinforce the critical importance of early diagnosis and early treatment initiation. She also highlighted the company’s ongoing collaboration with families and clinicians who have contributed to the development of the program from its earliest stages.
Leadership Engagement at the Conference
In addition to the poster presentations, Beren Therapeutics Chief Executive Officer Jason Camm will participate in a fireside chat with Sean Kassen, Director of the Ara Parseghian Medical Research Fund at the University of Notre Dame. The discussion is scheduled for May 31 at 4 p.m. MST and is expected to focus on advances in NPC research, clinical development strategies, and the future of adrabetadex.
The company has made the full set of poster presentations available to healthcare professionals through its medical information website at https://medical.berentx.com/.
Regulatory Progress and Development Outlook
Adrabetadex has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration, reflecting its potential to address a serious and life-threatening condition with limited treatment options. In February 2026, the New Drug Application (NDA) for adrabetadex in infantile-onset NPC was accepted by the FDA for Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date set for November 17, 2026.
While the therapy has not yet been approved by the FDA or any other regulatory authority, these designations indicate that the product is being evaluated under expedited review pathways due to its potential clinical significance.
Beren Therapeutics’ participation in the 2026 Parseghian NPC Research Conference highlights the company’s ongoing efforts to advance both the scientific understanding and clinical development of adrabetadex. The combination of clinical outcomes, preclinical mechanistic insights, and emerging combination therapy data provides a comprehensive view of the program’s progress.
As regulatory review continues and additional clinical data emerge, adrabetadex remains an important investigational candidate in the field of rare neurodegenerative disease. The company’s focus on early intervention, disease modification, and combination strategies reflects a broader effort to improve outcomes for patients with infantile-onset Niemann-Pick disease type C, a condition where therapeutic advances are urgently needed.
About Infantile-Onset Niemann-Pick Disease, Type C
Niemann-Pick disease, type C (NPC) is a rare, autosomal-recessive, severe, heterogeneous, neurodegenerative disorder caused by pathogenic variants in the NPC1 (~95% of cases) or NPC2 genes, leading to impaired intracellular cholesterol trafficking resulting in progressive neurological decline and premature mortality. Infantile-onset NPC (I-NPC) refers to NPC in infants and children who first experience neurological symptoms <6 years of age. Earlier neurological onset is associated with more rapid progression and poorer prognosis, with mean ages of death of ~5.6 years for early infantile-onset (age of neurological onset <2 years) and ~13.4 years for late-infantile onset (2 to <6 years).
About Adrabetadex
Adrabetadex is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers suitable for intrathecal delivery, under investigation as a treatment for Niemann-Pick disease, type C (NPC). Clinical and nonclinical data demonstrate that adrabetadex directly targets the underlying pathophysiology of NPC by re-establishing intracellular cholesterol trafficking. Adrabetadex is generally well tolerated, with a well-characterized safety profile established over more than a decade of clinical development. The most common adverse events are hearing impairment (manageable with hearing aids when necessary) and post-dose fatigue and ataxia. Adrabetadex has not been approved by the FDA or any other health authority at this time.
About Beren Therapeutics P.B.C.
Beren Therapeutics P.B.C.® is a founder-led, clinical-stage biotechnology company pioneering the discovery, development, and commercialization of cyclodextrin-based therapeutics for conditions characterized by defective cholesterol trafficking. Beren and its subsidiary Mandos LLC® are committed to the development of adrabetadex for individuals living with Niemann-Pick disease, type C (NPC) and have supported the NPC community by providing access to adrabetadex through an Expanded Access Program (EAP).
Beren’s public benefit purpose is to discover, develop, and deliver novel therapies that provide optimal benefit for patients, and to do so by integrating the needs of patients, caregivers, clinicians, and health systems from the beginning of the development process and maintaining a long-term focus on delivering meaningful therapies and access.
