Data Analyses Show Benefit for Individuals With Infantile-Onset Niemann-Pick Disease Type C Treated With Investigational Drug Adrabetadex
News > Health News

Audio By Carbonatix
2:28 PM on Thursday, October 9
The Associated Press
THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Oct 9, 2025--
Mandos Health ® by Beren Therapeutics Public Benefit Corporation (P.B.C.) announced today that findings from analyses of its investigational therapy adrabetadex will be presented at the 54 th Child Neurology Society (CNS) Annual Meeting, taking place on October 8-11, 2025 in Charlotte, North Carolina.
Two analyses detail the clinical and biological effects of adrabetadex in Niemann-Pick disease type C (NPC), a rare and progressive neurodegenerative disorder characterized by impaired cholesterol trafficking, severe neurological deficits and premature mortality. The first abstract reports improved overall survival in adrabetadex-treated participants with neurological symptom 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.
“These analyses include long-term treatment data allowing evaluation of the effect on survival. They represent the most comprehensive clinical assessment of adrabetadex to date and show its therapeutic potential for individuals with infantile-onset Niemann-Pick disease type C (NPC) and their families. If approved, adrabetadex could become the first treatment option that improves survival in patients with infantile-onset NPC,” said Jason Camm, Chief Executive Officer, Mandos Health by Beren Therapeutics Public Benefit Corporation (P.B.C.).
Clinically meaningful survival benefit
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, will present the survival analysis comparing adrabetadex-treated participants from clinical trials and an ongoing Expanded Access Program (EAP) to controls developed from four major disease databases or publications. Participants with infantile-onset NPC (neurological symptoms before 6 years of age) treated with intrathecal adrabetadex had a statistically significant increase in survival compared to matched, untreated external controls.
"These data represent an important step forward in our understanding of how treatment with adrabetadex may impact disease progression in individuals with infantile-onset Niemann-Pick disease type C," said Berry-Kravis. "The rigorous methodology we employed in comparing treated participants to matched external controls provides valuable insights into how treatment can potentially extend the lives of these patients.”
Evidence of disease-modifying potential
Results from a biomarker analysis of cerebrospinal fluid (CSF) samples banked during a previously completed Phase 2b/3 clinical trial show that adrabetadex addresses the core pathology of NPC and is associated with reductions in biomarkers of neuronal damage and cell death. Specifically, treatment with adrabetadex increased CSF levels of 24(S)-hydroxycholesterol, which is low in individuals with NPC, reflecting the restoration of intracellular cholesterol trafficking that is impaired in NPC. Adrabetadex treatment also decreased levels of both calbindin D and fatty acid-binding protein 3 (FABP3), markers associated with neuronal injury and death.
"These biomarker findings provide compelling evidence that adrabetadex addresses the underlying pathophysiology of Niemann-Pick disease type C (NPC). These data strengthen our understanding of how adrabetadex works at the molecular level and support its potential as a disease-modifying investigational therapy for individuals with NPC," said Dr. Forbes D. Porter, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, who led the early Phase 1 and 2 clinical trials and whose team provided natural history data to support the studies.
Adrabetadex, which is not approved by the FDA, was 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.
Both of these analyses, previously presented at the 150 th annual meeting of the American Neurological Association, will be featured in encore poster presentations during the CNS meeting:
Abstract Title | Lead Author | Presentation Details |
Substantial survival benefit with intrathecal adrabetadex in children with infantile-onset Niemann-Pick disease type C1 (NPC1)
| Dr. Elizabeth Berry-Kravis
Rush University Medical Center
| Poster 100
Thursday, October 9 12:30 p.m. ET
Charlotte Convention Center
|
Decreased markers of neuronal damage in children with
| Dr. Forbes D. Porter
Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health
| Poster 96
Thursday, October 9 5:30 p.m. ET
Charlotte Convention Center |
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. 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). By re-establishing intracellular cholesterol trafficking, adrabetadex directly addresses the core pathology of NPC.
Adrabetadex has not been approved by the U.S. Food and Drug Administration (FDA) or any other health authority at this time.
About Mandos Health
Mandos LLC, a wholly-owned subsidiary of Beren Therapeutics Public Benefit Corporation (P.B.C.), is a biotechnology company 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. 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 an 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, investigational therapy for NPC and other cholesterol-trafficking diseases. For more information, please visit 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.
View source version on businesswire.com:https://www.businesswire.com/news/home/20251009444024/en/
CONTACT: Amanda Eckel
BGB Group
KEYWORD: UNITED STATES NORTH AMERICA CALIFORNIA NORTH CAROLINA
INDUSTRY KEYWORD: HEALTH CLINICAL TRIALS RESEARCH PHARMACEUTICAL SCIENCE BIOTECHNOLOGY
SOURCE: Mandos Health
Copyright Business Wire 2025.
PUB: 10/09/2025 05:28 PM/DISC: 10/09/2025 05:26 PM
http://www.businesswire.com/news/home/20251009444024/en