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.

Beren Therapeutics P.B.C.®, the parent company of Mandos LLC® and leader in cholesterol trafficking biology and cyclodextrin-based therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review its New Drug Application (NDA) for adrabetadex, an investigational cyclodextrin therapy designed to increase intracellular cholesterol trafficking in Niemann-Pick disease type C (NPC).

During the webinar, Jason Camm, Chief Executive Officer, Beren Therapeutics P.B.C., shared an important milestone.

We are pleased to share that on December 17, 2025, Beren Therapeutics P.B.C.®, through its subsidiary Mandos LLC®, submitted a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for adrabetadex. This application seeks approval for the treatment of patients with infantile-onset Niemann-Pick disease type C (NPC).

Beren Therapeutics P.B.C.® (“Beren”) today announced that its subsidiary Mandos LLC® (“Mandos”) has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for adrabetadex, an investigational therapy for the treatment of infantile-onset Niemann-Pick disease type C (NPC).

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).

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 54th Child Neurology Society (CNS) Annual Meeting, taking place on October 8-11, 2025 in Charlotte, North Carolina.

Our entire team of over 60 dedicated professionals at Mandos Health® is thrilled to share a new update as we continue our commitment to transparency, collaboration, and progress in Niemann-Pick disease type C (NPC) for the global NPC community.

Mandos Health® by Beren Therapeutics Public Benefit Corporation (P.B.C.) announced today that new analyses detailing the clinical and biological effects of its investigational drug, adrabetadex, in Niemann-Pick Disease Type C (NPC) will be presented at the 150th annual meeting of the American Neurological Association (ANA) September 13–16 in Baltimore, Maryland.

We are excited to share important updates on our progress as we continue to build a science-driven organization dedicated to NPC.