THOUSAND OAKS, Calif.–(BUSINESS WIRE)–Mandos Health® by Beren Therapeutics Public Benefit Corporation (P.B.C.) announced today that new analyses detailing the clinical and biological effects of its investigational drug, adrabetadex, in Niemann-Pick Disease Type C (NPC) will be presented at the 150th annual meeting of the American Neurological Association (ANA) September 13–16 in Baltimore, Maryland.
The first abstract, recognized by ANA as an “Abstract of Distinction,” reports improved overall survival in adrabetadex-treated participants with neurological onset before 6 years of age compared to matched external controls. The second abstract provides biomarker data supporting the potential of adrabetadex as a disease-modifying therapy if approved. NPC is a rare and progressive neurodegenerative disorder characterized by impaired cholesterol trafficking, severe neurological deficits, and ultimately, premature mortality.
“This is one of the most rigorously conducted investigations comparing treated participants to matched external controls in Niemann-Pick Disease Type C (NPC),” said Dr. Elizabeth Berry-Kravis, Professor of Pediatrics, Director of RUSH Pediatric Neurosciences F.A.S.T. Center for Translational Research at Rush University Medical Center and lead study author. “For those of us who have witnessed the relentless and heart-breaking progression of this condition, we are encouraged by the potential for adrabetadex to meaningfully alter the disease course, extend survival and improve outcomes for patients with NPC and their families.”
Both analyses will be featured in poster presentations during the ANA meeting:
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Abstract Title |
Lead Author |
Presentation Details |
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* Abstract of Distinction * Substantial survival benefit with intrathecal adrabetadex in individuals with infantile-onset Niemann-Pick Disease Type C1
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Dr. Elizabeth Berry-Kravis
Rush University Medical Center
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Poster S249
Sunday, September 14, 2025
Baltimore Marriott Waterfront |
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Restoration of cholesterol trafficking results in decreased markers of neuronal damage in individuals with Niemann-Pick Disease type C1
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Dr. Forbes D. Porter
Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health |
Poster S250
Sunday, September 14, 2025
Baltimore Marriott Waterfront |
About Niemann-Pick Disease Type C (NPC)
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. 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, but the most severe and debilitating effects occur 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 NPC. By reestablishing intracellular cholesterol trafficking, adrabetadex directly addresses the core pathology of NPC.
Adrabetadex has not been approved by the FDA or any other health authority at this time.
About Mandos LLC
Mandos LLC is a wholly-owned subsidiary of Beren Therapeutics Public Benefit Corporation (P.B.C.), a biotechnology company creating the potential for possibilities for individuals living with Niemann-Pick Disease Type C (NPC), a condition characterized by defects in cholesterol trafficking. The company’s experienced team of scientists are focused on evaluating adrabetadex, a derivatized cyclodextrin, as a potential treatment that can target the underlying pathology of NPC. Since 2021, Mandos Health has supported the NPC community by providing access to adrabetadex* through its Expanded Access Program (EAP). The company will continue working closely with patients, families, researchers, regulators, and others on a path to bring forth this potentially transformative therapy for NPC and other cholesterol-trafficking diseases. For more information, please visit https://mandoshealth.com/about-us.
About Rush University Medical Center
Rush University System for Health (RUSH) is an academic health system whose mission is to improve the health of the individuals and diverse communities it serves through the integration of outstanding patient care, education, research and community partnerships. RUSH comprises Rush University Medical Center, Rush University, Rush Copley Medical Center, and Rush Oak Park Hospital, as well as numerous outpatient care facilities. Rush University, with more than 2,500 students, is a health sciences university that comprises Rush Medical College, the College of Nursing, the College of Health Sciences, and the Graduate College.
*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
[email protected]
