Beren Therapeutics Announces Six Presentations at the 2026 Ara Parseghian Medical Research Fund (APMRF) Conference
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 the company will present six posters at the 2026 Michael, Marcia, and Christa Parseghian Scientific Conference for Niemann-Pick Type C Research, sponsored by the family of former Notre Dame head football coach Ara Parseghian, taking place May 30 through June 2 in Tucson, Arizona. The posters present clinical and preclinical data on adrabetadex, an investigational cyclodextrin-based therapy under review by the U.S. Food and Drug Administration (FDA) for infantile-onset Niemann-Pick disease, type C (I-NPC). Beren’s Chief Executive Officer Jason Camm will also participate in a fireside chat at the conference with APMRF director Sean Kassen on Sunday, May 31 at 4 p.m. MST.
Beren Therapeutics Announces FDA Extension of Review Period for New Drug Application (NDA) for Adrabetadex for the Treatment of Infantile-Onset Niemann-Pick Disease, Type C
Beren Therapeutics P.B.C.®, the parent company of Mandos LLC® and a leader in cholesterol trafficking biology and cyclodextrin-based therapeutics, announced that the U.S. Food and Drug Administration (FDA) has extended by three months the review timeline of its New Drug Application (NDA) for adrabetadex for the treatment of infantile-onset Niemann-Pick disease, type C (I-NPC). The new Prescription Drug User Fee Act (PDUFA) target action date is November 17, 2026.
Beren Therapeutics Presents Data Supporting the Efficacy of Adrabetadex in Infantile-Onset NPC at 2026 ACMG Clinical Genetics Meeting
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.
Beren Therapeutics Announces FDA Acceptance of its New Drug Application for Adrabetadex in Infantile-Onset Niemann-Pick Disease Type C
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). If approved, adrabetadex would represent a first-in-class, disease-modifying approach to treat infantile-onset NPC, a rare and rapidly fatal pediatric neurodegenerative disorder. The FDA assigned adrabetadex a Prescription Drug User Fee Act (PDUFA) target action date of August 17, 2026.
