Arimoclomol

A case study presented at Child Neurology Society Annual Meeting demonstrates preservation of neurological function in sisters with infantile-onset Niemann-Pick disease, type C Additional data presentations reinforce benefit of early and sustained adrabetadex treatment in preserving function and improving long-term outcomes THOUSAND OAKS, Calif.--(BUSINESS WIRE)-- Beren Therapeutics P.B.C. , a leader in cholesterol-trafficking biology and novel cyclodextrin-based therapeutics, today announced new long-term data demonstrating neurological function was preserved for more than 10 years following initiation of treatment with adrabetadex, an investigational cyclodextrin-based medicine under review by the U.S. Food and Drug Administration (FDA) for infantile-onset Niemann-Pick disease, type C (I-NPC). A case study containing these data will be presented at the 55th Annual Meeting of the Child Neurology Society (CNS 2026), taking place October 14–17, 2026, in Montréal, Québec, Canada. Additional poster presentations highlight data showing that adrabetadex was associated with improved survival and slower disease progression and that it was generally well tolerated alone and in combination with other NPC treatments. “Infantile-onset NPC is associated with the most rapid neurodegeneration, accelerated disease progression, and markedly increased risk of premature death,” said Kristina Julich, M.D., Chief of the Pediatric Neurogenetics Center at the Comprehensive Pediatric Epilepsy Center within UT Health Austin Pediatric Neurosciences at Dell Children’s and treating physician for the case study. “Given this natural history of the disease, it is especially impressive to see that neurological function was preserved for more than 10 years in sisters with infantile-onset NPC who began treatment with adrabetadex at very different stages of disease.” Case report shows benefits of early treatment for I-NPC Poster 284: Case Report of Siblings With Infantile-Onset Niemann-Pick Type C (NPC) Initiating Adrabetadex at Different Ages and Intervals From Disease Onset (presented October 15, 2026) This case study follows two sisters who have I-NPC through 10 years of adrabetadex treatment. The older sister began adrabetadex more than four years after the onset of symptoms, which began before she was six years old and included ataxia, gross motor decline, and tremors/dysmetria, when she was classified as having moderate disease. Despite this delayed treatment initiation, she still experienced stabilization or improvement of her symptoms and she maintains moderate disease symptoms after approximately 10 years on adrabetadex. The younger sister began treatment with adrabetadex approximately 10 months after her first neurological symptoms appeared, which has resulted in maintenance of minimal disease activity and symptoms over 10 years. Importantly, treatment with adrabetadex has preserved neurological function for more than 10 years in both sisters, regardless of their stage of disease when they began adrabetadex. “These findings are meaningful in showing that neurological function was preserved for these patients for a decade,” said Irene von Hennigs, PharmD, SVP of Medical Affairs of Beren Therapeutics P.B.C. “In a disease where children can progressively lose the ability to walk, communicate, and develop, preserving function over many years represents an important clinical outcome. These sisters also illustrate why early diagnosis and treatment matter.” Additional Poster Presentations The following posters will also be presented during the conference poster sessions on Thursday, October 15, 2026. Poster 283: Survival outcomes by baseline disease burden in participants with infantile-onset Niemann-Pick disease type C treated with adrabetadex ​ Among 76 individuals treated with adrabetadex for up to 11 years, those with the lowest baseline disease severity (as assessed by NPC Clinical Severity Scale, NPCCSS) demonstrated the longest survival observed (n=29; 100% survival at 5 years), supporting the importance of initiating adrabetadex treatment before advanced disease progression. The findings suggest that earlier restoration of cholesterol trafficking, before substantial neurological disease burden accumulates, may improve long-term outcomes. Poster 281: Outcomes in participants with infantile-onset Niemann-Pick disease type C receiving prompt and sustained treatment with adrabetadex ​ Among 25 patients who received adrabetadex for at least five years, both prompt and delayed treatment initiation were associated with slower disease progression relative to published natural history data. Participants who initiated adrabetadex within one year of neurological symptom onset demonstrated less progression from baseline in NPCCSS scores compared with later treatment initiation, suggesting that earlier initiation of treatment with adrabetadex may result in greater long-term clinical benefit in I-NPC. Poster 275: Early evidence of an emerging NPC treatment paradigm: adrabetadex use alongside arimoclomol and/or N-acetyl-L-leucine ​ The safety pro adrabetadex was generally similar with and without the use of additional therapies, supporting its use as a foundational therapy that can be safely combined with other approved NPC treatments. Most participants initiated adrabetadex prior to complementary therapies, with arimoclomol and/or levacetylleucine frequently added after adrabetadex (when they became commercially available) rather than used instead of adrabetadex. Together, these findings add to the growing body of long-term clinical evidence supporting the potential for adrabetadex to slow progression of I-NPC by addressing impaired cholesterol trafficking, the underlying cause of the disease. About 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 an investigational cyclodextrin-based therapy designed to address the underlying cause of Niemann-Pick disease, type C (NPC) by restoring impaired intracellular cholesterol trafficking. By targeting the biological defect responsible for cholesterol accumulation and downstream neurological injury, adrabetadex is being developed as a potential disease-modifying treatment for infantile-onset NPC. Clinical studies and expanded access experience have generated long-term data on survival, neurological function, and safety, including treatment extending beyond a decade in some individuals. The main adverse events associated with adrabetadex include hearing impairment that can be managed with hearing aids when necessary, and post-dose fatigue and/or ataxia. The U.S. Food and Drug Administration (FDA) granted adrabetadex Breakthrough Therapy Designation. In February 2026, the FDA accepted Beren Therapeutics’ New Drug Application (NDA) for adrabetadex in infantile-onset NPC for Priority Review. The agency assigned the NDA a Prescription Drug User Fee Act (PDUFA) target action date of November 17, 2026. Adrabetadex has not been approved by the FDA or any other health authority at this time. About Beren Therapeutics 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. Beren is headquartered in Thousand Oaks, Calif. To learn more about Beren, the adrabetadex program, and Beren’s cholesterol-trafficking focused therapeutic strategy, visit the company’s website , LinkedIn channel or Facebook page . Forward-Looking Statement This press release contains forward-looking statements regarding Beren’s clinical development program, regulatory plans, and the potential of adrabetadex, which is investigational and not yet approved by any regulatory authority. These statements are based on current data — including data from small, uncontrolled case reports and observational analyses — and are subject to risks and uncertainties inherent in drug development. Actual results may differ materially. Contacts Media Contact Beren Therapeutics Media Relations media@berentx.com