Thousand Oaks, California-based Beren Therapeutics P.B.C. has secured USD 300 million in combined financing to support the potential commercial launch of adrabetadex, its lead candidate for infantile-onset Niemann-Pick disease type C (I-NPC), a rare and rapidly fatal pediatric neurodegenerative disease. The funding comprises a USD 135 million equity round alongside up to USD 165 million in non-dilutive capital from Hercules Capital, Inc. (NYSE: HTGC), structured as a senior secured debt facility and royalty financing.
The transaction is designed to position Beren as financially self-sustaining through a potential commercial launch, with the US FDA having accepted the company's New Drug Application (NDA) for adrabetadex under Priority Review. The FDA recently extended its review time for the molecule, issuing a PDUFA target action date of November 17, 2026.
The equity round brought together Wellington Partners, JIC Venture Growth Investments (JIC VGI), Founders Fund, Narya Capital, Eisai Co., Ltd., and other institutional investors. The non-dilutive component with Hercules Capital includes up to USD 110 million in term loans tied to regulatory and revenue milestones — of which USD 30 million has been drawn — and a USD 55 million royalty financing tranche to be funded upon FDA approval of adrabetadex. The royalty financing carries a 7.5% royalty on US net sales and 5% on net sales outside the US, capped at 1.75x through 2031, with an option to redeem at a lower multiple in the first two years.
Beren said proceeds will support commercial readiness activities including accelerating diagnosis to enable early intervention, establishing a single point of contact for families, ensuring local access to sites of care, and generating longitudinal real-world evidence. The company and its subsidiary Mandos LLC have supported the NPC community since 2021 through an Expanded Access Program.
No prior funding rounds were disclosed in the press release or available data for Beren Therapeutics.
Adrabetadex and the Case for I-NPC Treatment
I-NPC is the most severe form of Niemann-Pick disease type C, a rare autosomal-recessive disorder caused by pathogenic variants in the NPC1 or NPC2 genes. These mutations impair intracellular cholesterol trafficking, leading to progressive neurological decline. Patients with early infantile-onset — defined as neurological symptom onset before age two — have a mean age of death of approximately 5.6 years. For late-infantile onset, the mean age of death is approximately 13.4 years.
Adrabetadex is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers. Data suggest the compound re-establishes intracellular cholesterol trafficking, directly addressing the underlying pathology of NPC rather than managing downstream symptoms. The US FDA has granted adrabetadex Breakthrough Therapy Designation. If approved, it would represent the only therapy specifically authorized for I-NPC and the only agent designed to act on accumulated intracellular cholesterol driving the disease.