Beren Therapeutics to Present Six Posters Highlighting Clinical and Preclinical Progress of Adrabetadex at 2026 APMRF Conference
Beren Therapeutics P.B.C., the parent company of Mandos LLC and a company focused on advancing cholesterol trafficking biology and cyclodextrin-based therapeutics, 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 football coach Ara Parseghian, is scheduled to take place from May 30 through June 2 in Tucson, Arizona.
The presentations will showcase a broad range of clinical and preclinical findings related to adrabetadex, the company’s investigational cyclodextrin-based therapy currently under review by the U.S. Food and Drug Administration (FDA) for the treatment of infantile-onset Niemann-Pick disease type C (I-NPC). The collection of data highlights the potential disease-modifying effects of adrabetadex and underscores the importance of early diagnosis and intervention in this rare and devastating neurodegenerative disorder.
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 (APMRF), during the conference. The discussion is expected to focus on the evolving NPC treatment landscape, ongoing research efforts, and the future role of adrabetadex in addressing unmet patient needs.
Focus on Early Treatment and Long-Term Outcomes
Two of the six posters focus on the impact of early and sustained treatment with adrabetadex in patients with infantile-onset NPC. These studies provide important insights into how treatment timing may influence disease progression and long-term survival.
Poster #25 examines outcomes among individuals with infantile-onset NPC who received prompt and continuous treatment with adrabetadex. According to the findings, patients treated with the investigational therapy experienced slower disease progression compared with what has been reported in published natural history studies of the disease. Researchers also observed potential differences in outcomes depending on how early treatment was initiated, suggesting that earlier intervention may provide greater clinical benefits.
Poster #26 explores survival outcomes in patients treated with adrabetadex based on their baseline disease burden. The retrospective analysis included patients who received treatment through either clinical studies or the Expanded Access Program. Results suggest that individuals who began therapy before significant neurological damage occurred may have experienced improved survival outcomes. The findings reinforce the growing consensus that early diagnosis and rapid initiation of therapy are critical in a disease characterized by progressive and irreversible neurodegeneration.
Together, these studies add to the body of evidence supporting the potential value of adrabetadex as an intervention capable of altering the natural course of infantile-onset NPC.
Evaluating Adrabetadex Alongside Approved NPC Therapies
Another major focus of the conference presentations is the potential role of adrabetadex within an evolving treatment paradigm that includes combination therapy approaches.
Poster #6 provides one of the first systematic evaluations of intrathecal adrabetadex administered alongside approved oral NPC treatments, including arimoclomol and N-acetyl-L-leucine. Data gathered through the Expanded Access Program reveal emerging treatment patterns among clinicians managing NPC patients in real-world settings.
The findings suggest that adrabetadex may serve as a foundational therapy while additional approved treatments are incorporated based on individual patient needs. Researchers believe these observations could offer valuable insights into future treatment strategies aimed at maximizing clinical benefit through complementary therapeutic mechanisms.
Poster #2 presents preclinical findings from an in vitro mouse model of NPC that investigated adrabetadex both as a standalone therapy and in combination with arimoclomol or levacetylleucine. The study evaluated effects on oligodendrocyte proliferation, synaptic network growth, and myelination—critical biological processes involved in maintaining healthy neurological function.
According to the results, adrabetadex demonstrated greater potency than currently approved NPC therapies across several measures of neuronal and oligodendrocyte health. Furthermore, combining adrabetadex with either arimoclomol or N-acetyl-L-leucine produced comparable biological effects at significantly lower concentrations than those required with monotherapy. These findings suggest potential mechanistic compatibility between adrabetadex and therapies that target different disease pathways, supporting future exploration of combination treatment approaches.
Advancing Understanding of Adrabetadex’s Mechanism of Action
Two additional posters focus on the biological mechanisms underlying adrabetadex’s therapeutic effects and its potential to modify disease progression.
Poster #18.1 examines the mechanism of action of adrabetadex in Niemann-Pick disease type C. Preclinical studies indicate that centrally administered adrabetadex achieves broad distribution throughout the central nervous system (CNS) while maintaining sustained engagement with intracellular cholesterol targets.
Researchers reported evidence supporting the restoration of intracellular cholesterol trafficking in both superficial and deep brain regions. Since defective cholesterol transport is a hallmark of NPC pathology, these findings provide important mechanistic support for the therapeutic rationale behind adrabetadex.
Poster #18.2 further explores the disease-modifying effects of centrally administered adrabetadex using NPC mouse models. The data suggest positive effects on several key markers associated with disease progression, including myelination, neuronal integrity, and neurodegenerative biomarkers.
The findings indicate that adrabetadex may influence multiple biological pathways involved in NPC pathology, potentially contributing to improved neurological outcomes. Researchers believe these mechanistic observations align with the encouraging clinical signals observed in treated patients and may help explain the therapeutic benefits seen in both clinical and Expanded Access Program settings.
Commitment to the NPC Community
Commenting on the upcoming presentations, Irene von Hennigs, PharmD, Senior Vice President of Medical Affairs at Beren Therapeutics, emphasized the importance of continued scientific collaboration and transparency within the NPC community.
She noted that the newly presented data expand the understanding of adrabetadex in infantile-onset NPC while reinforcing the critical importance of recognizing and treating the disease as early as possible. She also highlighted the company’s commitment to sharing emerging findings openly with patients, families, clinicians, and researchers who have contributed to advancing knowledge of the disease.
The company views the conference as an important opportunity to engage with the broader NPC research community and discuss the latest developments in disease biology, therapeutic innovation, and patient care.
Regulatory Progress and Future Outlook
The presentations come at a significant time for Beren Therapeutics as adrabetadex continues to advance through the regulatory review process.
In 2025, the FDA granted Breakthrough Therapy Designation to adrabetadex, recognizing its potential to provide substantial improvement over existing treatment options for patients with infantile-onset NPC. The designation is intended to facilitate the development and review of therapies that address serious or life-threatening conditions.
More recently, in February 2026, the FDA accepted the New Drug Application (NDA) for adrabetadex and granted Priority Review status. The application is being evaluated for the treatment of infantile-onset Niemann-Pick disease type C, a rare genetic disorder characterized by progressive neurological deterioration and premature mortality.
The agency has assigned a Prescription Drug User Fee Act (PDUFA) target action date of November 17, 2026. If approved, adrabetadex could become an important new treatment option for patients affected by this severe and currently underserved disease.
While adrabetadex remains investigational and has not yet been approved by the FDA or any other regulatory authority, the growing body of clinical and preclinical evidence continues to support its potential as a disease-modifying therapy.
As researchers, clinicians, and patient advocates gather at the 2026 APMRF Conference, the six presentations from Beren Therapeutics are expected to contribute meaningful new insights into both the biology of NPC and the future treatment possibilities for patients living with this challenging condition.
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.
