Beren Therapeutics Presents Data Supporting the Efficacy of Adrabetadex in Infantile-Onset NPC at 2026 ACMG Clinical Genetics Meeting

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THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Mar 12, 2026--

Beren Therapeutics P.B.C. ®, the parent company of Mandos LLC ® and a leader in cholesterol trafficking biology and cyclodextrin-based therapeutics, today announced that data for adrabetadex, an investigational cyclodextrin-based therapy in development for infantile-onset Niemann-Pick disease type C (NPC), were presented at the 2026 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Baltimore, MD. The presentation included data relating to disease progression, survival and biomarker data.

Elizabeth M. Berry-Kravis, M.D., Ph.D., Professor of Pediatrics at Rush University Medical Center, presented evidence that adrabetadex-treated, infantile-onset NPC patients (n=79) had:

• A significantly reduced annual rate of disease progression of 43% (p=0.0055) over 52 weeks compared with pre-treatment rates of disease progression.

Neurological disease progression was assessed pre- and post-adrabetadex-treatment using the 4-Domain Composite NPC Clinical Severity Scale (4DNPCCSS) of ambulation, speech, fine-motor function and swallowing.

In the previously reported analysis, a comparison of survival in individuals with infantile-onset NPC treated with adrabetadex to matched external controls showed a 71% reduction in the risk of mortality (p<0.0001). The 5-year survival rate was 84% for those treated with adrabetadex versus 42% for external controls. The survival analysis compared adrabetadex-treated individuals (n=72) with infantile neurological onset NPC who participated in a Phase 2b/3 trial and open-label extension period and/or in an Expanded Access Program (EAP) to 119 matched external controls from the NIH Natural History study and three other major disease databases or publications.

Additionally, patients treated with adrabetadex showed significant changes in biomarkers associated with improved neuronal cholesterol trafficking – CSF levels of 24(S)-OHC increased 27.7% from baseline (p=0.0012). These findings are based on longitudinal biomarker analyses from subsets of the previous 12-month Phase 2b/3 prospective, randomized, placebo-controlled clinical trial (n=22).

“There are limited therapies for patients with rapidly progressive and fatal infantile-onset NPC, none of which have demonstrated survival benefit,” said Dr. Berry-Kravis. “In view of this data, adrabetadex may potentially slow disease progression and improve survival, offering disease-modifying therapy.”

The FDA granted adrabetadex Breakthrough Therapy Designation in 2025, and in February 2026, the NDA for adrabetadex in infantile-onset NPC was accepted by the FDA for Priority Review. The agency assigned a Prescription Drug User Fee Act (PDUFA) target action date of August 17, 2026. Adrabetadex has not been approved by the FDA or any other health authority at this time.

“These biomarker data support Beren’s cholesterol trafficking-based approach, which is grounded in disease biology. If adrabetadex is approved, it would be the first therapeutic option for patients and families designed to target the underlying pathophysiology of NPC with the goal to extend lifespan and slow the progression of this rare and rapidly fatal pediatric neurodegenerative disorder,” said Alex Gold, M.D., Chief Medical Officer of Beren Therapeutics P.B.C. “We remain focused on working with the FDA to advance our NDA for adrabetadex.”

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). Children with infantile-onset NPC can experience delayed or missed development milestones, loss of previously acquired skills, sudden and temporary loss of muscle tone, clumsiness and vertical supranuclear gaze palsy (inability to voluntarily move eyes upward or downward).

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.

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 August 17, 2026. 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: https://www.BerenTx.com 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.

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PUB: 03/12/2026 04:00 PM/DISC: 03/12/2026 04:00 PM

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