• Breakthrough Therapy Designation is based on FDA’s review of survival analyses comparing adrabetadex-treated patients with external controls and marks a significant regulatory milestone ahead of an NDA submission.

Beren Therapeutics P.B.C.®, a Public Benefit Corporation and parent company of Mandos LLC®, today announced that Mandos received Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for adrabetadex, an investigational drug for infantile-onset Niemann-Pick disease type C (NPC).

BTD is granted by the FDA to expedite the development of drugs for serious or life-threatening conditions when preliminary clinical evidence may indicate a substantial improvement over existing therapy. The FDA decision was informed by an externally controlled survival analysis that showed adrabetadex improves survival in individuals with infantile-onset NPC and was reviewed alongside supportive biomarker and nonclinical data.

Adrabetadex previously received BTD in 2016 under a prior sponsor, and the FDA rescinded the designation based on data from a 12-month Phase 2b/3 clinical trial. Beren, through its subsidiary Mandos, acquired the adrabetadex program from Mallinckrodt in 2021 and has worked closely with the FDA and NPC community to advance adrabetadex’s development and generate the data and analyses that supported the new BTD designation.

“This newly granted designation represents an important milestone in the evaluation of adrabetadex for people living with infantile-onset NPC,” said Jason Camm, Chief Executive Officer of Beren Therapeutics P.B.C. “We are grateful to the people living with NPC and their caregivers, clinicians, and advocates who have worked with us through a long development path.”

Dr. Elizabeth Berry-Kravis, Professor of Pediatrics at Rush University Medical Center and principal EAP investigator, noted: “Infantile-onset NPC is a devastating, rapidly fatal diagnosis. Seeing statistically significant improvements in survival signals a meaningful shift in what is achievable for these patients.”

Adrabetadex also has Orphan Drug and Rare Pediatric Disease designations. Beren plans to submit an NDA in the near future and expects adrabetadex to be eligible for Priority Review.

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). 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 out of stealth mode 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.

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