Beren Therapeutics Strengthens U.S. Patent Portfolio with Eight New Issued Patents on Cyclodextrin Therapeutics

Beren Therapeutics P.B.C., a clinical-stage biotechnology company specializing in cholesterol-trafficking biology and cyclodextrin-based therapeutics, announced the issuance of eight new U.S. patents. These patents relate to Beren’s proprietary derivatized cyclodextrins, including its lead candidate adrabetadex, increasing the company’s U.S. patent portfolio to more than 30 issued patents.

The newly granted patents form part of a comprehensive global intellectual property estate that encompasses issued patents and pending applications across key pharmaceutical markets such as the United States, Europe, and Japan. This estate covers a broad range of innovations including proprietary cyclodextrin compositions, pharmaceutical formulations, manufacturing processes, biomarkers, and methods of treatment for diseases like Niemann-Pick disease, type C (NPC).

Jason Camm, Founder and Chief Executive Officer of Beren Therapeutics, emphasized the significance of these patents, stating, “These patents reflect years of innovation to advance therapeutic cyclodextrin science, chemistry, manufacturing, and clinical development for the NPC community. They establish a foundation for our long-term commitment to the NPC community and support our continued investment in the next generation of cholesterol trafficking therapeutics across additional diseases driven by defective cholesterol trafficking.”

Niemann-Pick disease, type C is a rare, autosomal-recessive neurodegenerative disorder caused by pathogenic variants in the NPC1 or NPC2 genes. These genetic mutations impair intracellular cholesterol trafficking, leading to progressive neurological decline and premature mortality. Infantile-onset NPC (I-NPC) manifests in children under six years of age, with earlier neurological onset correlating with more rapid disease progression and poorer prognosis. The mean age of death is approximately 5.6 years for early infantile-onset cases and 13.4 years for late infantile-onset cases.

Adrabetadex, Beren’s proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers, is under clinical investigation as a treatment for NPC. The therapeutic approach aims to restore intracellular cholesterol trafficking, directly addressing the underlying pathology of the disease. Clinical trial data and expanded access programs indicate that adrabetadex is generally well tolerated. The primary adverse events reported include hearing impairment, which can be managed with hearing aids, as well as post-dose fatigue and ataxia.

The U.S. Food and Drug Administration (FDA) has granted adrabetadex Breakthrough Therapy Designation. In February 2026, the FDA accepted Beren Therapeutics’ New Drug Application (NDA) for adrabetadex targeting infantile-onset NPC and assigned a Priority Review status. The Prescription Drug User Fee Act (PDUFA) target action date is set for November 17, 2026. Adrabetadex has not yet received FDA approval or authorization from any other health authority.

Beren Therapeutics is a founder-led company headquartered in Thousand Oaks, California. It is dedicated to pioneering the discovery, development, and commercialization of cyclodextrin-based therapeutics for diseases characterized by defective cholesterol trafficking. The company supports the NPC community through its Expanded Access Program, providing access to adrabetadex prior to regulatory approval.

Beren’s public benefit mission focuses on developing novel therapies that deliver optimal patient benefit by integrating the needs of patients, caregivers, clinicians, and health systems from the outset of the development process. The company maintains a long-term commitment to delivering meaningful therapies and ensuring patient access.

For more information about Beren Therapeutics, the adrabetadex program, and the company’s cholesterol-trafficking therapeutic strategy, visit https://www.BerenTx.com or Beren’s LinkedIn channel.