Beren Therapeutics Expands Cyclodextrin Intellectual Property Estate with Eight New U.S. Issued Patents

• Eight newly issued U.S. patents expand Beren's intellectual property estate covering therapeutic cyclodextrin compositions, pharmaceutical formulations, manufacturing processes, and methods of treatment, increasing the company’s U.S. portfolio to more than 30 issued patents.
• Global intellectual property estate includes issued patents and pending applications supporting adrabetadex and Beren’s broader cholesterol-trafficking pipeline with patent protection through 2046, excluding potential patent term extensions, patent term adjustments, and supplementary protection certificates.

THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Beren Therapeutics P.B.C., a leader in cholesterol-trafficking biology and novel cyclodextrin-based therapeutics, today announced that the U.S. Patent and Trademark Office has issued eight new U.S. patents related to Beren’s proprietary derivatized cyclodextrins, including adrabetadex, increasing Beren’s U.S. patent portfolio to more than 30 issued patents.

The new patents are part of a broader global intellectual property estate, which includes issued patents and pending applications in major pharmaceutical markets, including the U.S., Europe, and Japan. The estate spans proprietary derivatized cyclodextrin compositions, formulations, manufacturing processes, biomarkers, and methods of treatment, including for Niemann-Pick disease, type C (NPC).

“These patents reflect years of innovation to advance therapeutic cyclodextrin science, chemistry, manufacturing, and clinical development for the NPC community,” said Jason Camm, Founder and Chief Executive Officer of Beren. “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.”

About Infantile-Onset Niemann-Pick Disease, Type C

Niemann-Pick disease, type C (NPC) is a rare, autosomal-recessive, severe, heterogeneous, neurodegenerative disorder caused by pathogenic variants in the NPC1 (~95% of cases) or NPC2 genes, leading to impaired intracellular cholesterol trafficking resulting in progressive neurological decline and premature mortality. Infantile-onset NPC (I-NPC) refers to NPC in infants and children who first experience neurological symptoms <6 years of age. Earlier neurological onset is associated with more rapid progression and poorer prognosis, with mean ages of death 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).

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 November 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 is committed to the development of adrabetadex for individuals living with Niemann-Pick disease, type C (NPC) and has 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 website at https://www.BerenTx.com or visit Beren’s LinkedIn channel.