Adrabetadex

--Accepted for priority review with Prescription Drug User Fee Act (PDUFA) action date of August 17, 2026 --If approved, adrabetadex would be the only therapy to directly target the underlying pathophysiology of Niemann-Pick disease type C (NPC) --Comprehensive NDA application includes data showing long-term survival benefit, slowed disease progression, confirmatory biomarkers and nonclinical evidence THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Beren Therapeutics P.B.C. ® , the parent company of Mandos LLC ® and leader in cholesterol trafficking biology and cyclodextrin-based therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review its New Drug Application (NDA) for adrabetadex, an investigational cyclodextrin therapy designed to increase intracellular cholesterol trafficking in Niemann-Pick disease type C (NPC). If approved, adrabetadex would represent a first-in-class, disease-modifying approach to treat infantile-onset NPC, a rare and rapidly fatal pediatric neurodegenerative disorder. The FDA assigned adrabetadex a Prescription Drug User Fee Act (PDUFA) target action date of August 17, 2026. “The FDA’s acceptance of our NDA submission for review is an important milestone, taking us another step closer to a new treatment option for children living with infantile-onset NPC,” said Jason Camm, Chief Executive Officer of Beren Therapeutics P.B.C. “The FDA’s granting of a Priority Review further reinforces the high unmet need in infantile-onset NPC. Our rigorous and comprehensive NDA package incorporated feedback from the FDA and the NPC community, and we remain focused on advancing our application. We are grateful to the patients, families, clinicians and advocates who made this submission possible.” The NDA for adrabetadex is supported by data demonstrating a clinically meaningful survival benefit in infantile-onset patients. This evidence is from an externally controlled analysis intended to serve as a single adequate and well-controlled study. The comprehensive submission also includes data showing slowed disease progression, confirmatory biomarker and nonclinical findings and patient experience narratives. Key findings were recently presented at WORLD Symposium ™ 2026, the largest annual scientific meeting in the field of lysosomal storage disease, including survival data in infantile-onset NPC: a 71% reduction in the risk of mortality in adrabetadex-treated patients compared with matched external controls (HR 0.289; 95% CI, 0.141–0.593; P < 0.0001). “Infantile-onset NPC is the fastest progressing subtype and has limited treatment options,” said Elizabeth Berry-Kravis, M.D., Ph.D., professor of Pediatrics at Rush University Medical Center and principal investigator of the Expanded Access Program. “The approval of adrabetadex would mark the first time we have a disease-modifying therapeutic option, which is urgently needed for these young children and their families.” Beren previously announced that in 2025 the FDA granted adrabetadex Breakthrough Therapy Designation, a status that accelerates the development of drugs for serious or life-threatening conditions when early evidence suggests a substantial improvement over existing therapy. About Niemann-Pick Disease Type C Niemann-Pick disease type C (NPC) is a rare, autosomal-recessive, severe, neurodegenerative disorder caused by pathologic variants in the NPC1 (~95% of cases) or NPC2 genes, leading to impaired cholesterol trafficking resulting in progressive neurological decline and premature death. Infantile-onset NPC refers to NPC in infants and children who first experience neurological symptoms between 0 and 6 years of age. Earlier neurological onset is associated with more rapid progression and poorer prognosis, with mean survival 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 under investigation as a treatment for Niemann-Pick disease type C (NPC). The data suggest that by re-establishing intracellular cholesterol trafficking, adrabetadex is designed to directly address the underlying pathology of NPC. Data from clinical trials and expanded access programs suggest that adrabetadex is generally well tolerated. 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. 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. 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 newly launched website at: or visit Beren’s LinkedIn channel. Forward-Looking Statements This press release contains forward-looking statements, including, but not limited to, statements regarding the FDA’s review of the NDA for adrabetadex; the timing and outcome of regulatory review; the potential for adrabetadex to provide clinical benefit or to address the underlying pathology of Niemann-Pick disease type C; the potential for approval or commercialization; the ability to continue providing access through expanded access; and Beren’s plans to expand its pipeline. Actual results may differ materially due to risks and uncertainties including those associated with clinical development, regulatory review, manufacturing, safety and efficacy outcomes and other factors. Contacts Media Campbell O’Connor Real Chemistry coconnor@realchemistry.com

Comprehensive submission package includes evidence of substantially improved survival among adrabetadex-treated patients with infantile-onset Niemann Pick disease type C (NPC), intended to serve as a single adequate and well-controlled study, and confirmatory evidence of biomarkers and nonclinical data demonstrating effects on the underlying pathology Priority Review was requested on the basis that the application meets the qualifying criteria THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Beren Therapeutics P.B.C. ® (“Beren”) today announced that its subsidiary Mandos LLC ® (“Mandos”) has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for adrabetadex, an investigational therapy for the treatment of infantile-onset Niemann-Pick disease type C (NPC). The application was based on data supporting a finding of improved survival in an externally controlled survival analysis, biomarker and nonclinical confirmatory evidence, and patient experience narratives. Adrabetadex is an investigational, proprietary cyclodextrin therapy designed to increase intracellular cholesterol trafficking to target the underlying pathology of NPC. “The submission of the NDA marks an important milestone for adrabetadex,” said Dr. Alexander Gold, Chief Medical Officer, Beren Therapeutics P.B.C. “The totality of the evidence – including a rigorous survival analysis compared to external controls – supports the potential of adrabetadex to provide meaningful clinical benefit for patients living with NPC. We are grateful to the patients with NPC and their caregivers, clinicians, and advocates who have worked with the Mandos development team to make this possible.” At the American Neurological Association (ANA) and Child Neurology Society (CNS) annual meetings in 2025, Beren/Mandos presented analyses that were featured in the NDA submission, including: Survival analyses that support improved outcomes in adrabetadex-treated patients with infantile-onset NPC compared to matched external controls Biomarker analyses demonstrating reductions in markers of neuronal injury, including fatty acid-binding protein 3 (FABP3) and calbindin D, and increases in a marker of neuronal cholesterol trafficking (24S-OHC), supporting a mechanistic rationale for the observed clinical outcomes The survival analysis received Abstract of Distinction recognition at ANA. Beren/Mandos will present additional data at the WORLD Symposium TM in San Diego, CA, February 2-6, 2026. “Submitting this NDA reflects our commitment to the NPC community and to advancing therapies grounded in rigorous science,” said Jason Camm, Chief Executive Officer, Beren Therapeutics P.B.C. “We believe the data included in this submission demonstrate adrabetadex has the potential to alter the course of disease. We are proud to take this step toward making a potential new treatment option available to families affected by NPC.” Data from the clinical trials and expanded access programs suggest that adrabetadex is well-tolerated. The most common adverse events associated with treatment include hearing impairment (manageable with hearing aids if needed) and transient post-dose fatigue and/or ataxia. The FDA granted Breakthrough Therapy Designation (BTD) in 2025 for adrabetadex in infantile-onset NPC, recognizing the potential to address a serious condition with substantial improvement over existing options. The FDA has a 60-day filing review period to determine whether the NDA is complete and accepted for review. Mandos intends to continue providing adrabetadex to eligible patients through an Expanded Access Program (EAP) during the FDA review process. About Niemann-Pick Disease Type C Niemann-Pick disease type C (NPC) is a rare, autosomal-recessive, severe, neurodegenerative disorder caused by pathologic variants in the NPC1 (~95% of cases) or NPC2 genes, leading to impaired cholesterol trafficking resulting in progressive neurological decline and premature death. Infantile-onset NPC refers to NPC in individuals who first experience neurological symptoms between 0 and 6 years of age. Earlier neurological onset is associated with more rapid progression and poorer prognosis, with mean survival 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). Individuals with early- and late-infantile-onset NPC typically present with manifestations affecting multiple organs, with the most severe and debilitating effects occurring in the brain. About Adrabetadex (VTS-270) Adrabetadex (VTS-270) is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers under investigation as a treatment for Niemann-Pick disease type C (NPC). The data suggests that by re-establishing intracellular cholesterol trafficking, adrabetadex directly addresses the underlying pathology of NPC. Adrabetadex is generally well tolerated. 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. Adrabetadex has not been approved by the FDA or any other health authority at this time. About Beren Therapeutics P.B.C. and Mandos 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, are committed to the development of adrabetadex for individuals living with Niemann-Pick disease type C (NPC), a condition characterized by defects in intracellular cholesterol trafficking. Beren and Mandos have supported the NPC community by providing access to adrabetadex* through an Expanded Access Program (EAP). Beren will continue working closely with patients, families, researchers, regulators, and others on a path to bring forth this potentially transformative, investigational therapy for NPC. Beren is headquartered in Thousand Oaks, CA, and will launch its website in Q1 2026. For more information, please visit our public-facing subsidiary Mandos . * Adrabetadex is an investigational drug that has not been approved by the U.S. Food and Drug Administration and has not been found safe and effective to treat NPC or any other condition. Contacts Amanda Eckel BGB Group Aeckel@bgbgroup.com