Breakthrough Therapy Designation is based on FDA’s review of survival analyses comparing adrabetadex-treated patients with external controls and marks a significant regulatory milestone ahead of an New Drug Apllication submission. THOUSAND OAKS, Calif.– December 9, 2025 – Beren Therapeutics P.B.C.®, a Public Benefit Corporation and parent company of Mandos LLC®, today announced that Mandos received Breakthrough Therapy Designation …
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Research News
Great Data On Adrabetadex/Cyclodextrin Released By Mandos Health
Wednesday, 9/10/25 – In advance of the 2025 American Neurological Association (ANA) meeting, Mandos Health releases two scientific abstracts with promising data for the treatment of NPC patients with Adrabetadex. The lead abstract, designated by ANA as an “Abstract of Distinction,” presents data showing a statistically significant survival benefit in patients with infantile-onset NPC who …
Azafaros Secures €132M in Financing to advance Phase 3 clinical programs, including NPC
13 May 2025 Leiden, Netherlands, 13 May 2025 – Azafaros, a clinical-stage company focused on developing disease-modifying therapeutics to offer new treatment options to patients with rare lysosomal storage disorders, announces the completion of an oversubscribed €132M Series B financing led by Jeito Capital, co-led by Forbion Growth and with additional participation from Seroba, Pictet Group …