Beren Therapeutics P.B.C. has raised $300 million in combined financing to support the development and potential commercialization of its investigational therapy adrabetadex, a treatment candidate for Infantile Niemann-Pick disease Type C (I-NPC), a rare and fatal pediatric neurodegenerative disorder.
The financing package consists of a $135 million equity investment and up to $165 million through a strategic financing facility provided by Hercules Capital, Inc. The facility includes a combination of senior secured debt and royalty-based financing. The new capital is expected to strengthen the company’s commercial readiness efforts, expand patient access programs, and enhance support services for families affected by the disease.
The funding comes at a pivotal time for Beren as the company advances adrabetadex toward a key regulatory milestone in the United States. The U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for adrabetadex and granted the investigational therapy Priority Review status. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of November 17, 2026.
If approved, adrabetadex would become the first disease-modifying therapy specifically approved for Infantile Niemann-Pick disease Type C, commonly known as I-NPC. The therapy would also be the first approved treatment designed to directly target the intracellular cholesterol accumulation that drives the progression of the disease.
I-NPC is considered the most severe and rapidly progressive form of Niemann-Pick disease Type C, a rare genetic disorder caused by impaired intracellular cholesterol trafficking. The condition leads to the buildup of cholesterol and other lipids inside cells, resulting in progressive neurological deterioration, developmental regression, and ultimately premature death. Currently, treatment options remain extremely limited, leaving significant unmet medical needs for affected children and their families.
According to Beren founder and Chief Executive Officer Jason Camm, the company’s mission extends beyond developing a treatment. He emphasized that families affected by I-NPC face ongoing challenges related to diagnosis, specialist care, treatment decisions, reimbursement processes, and long-term disease management. The new financing will help the company invest not only in commercialization efforts but also in patient access infrastructure and family support resources designed to address these broader challenges.
“Our vision is not only to develop new treatment options, but also to help address the broader challenges families face throughout their journey,” Camm said in a statement announcing the financing.
The participation of both equity investors and Hercules Capital reflects growing confidence in Beren’s scientific approach and the commercial potential of adrabetadex. The financing also positions the company to rapidly scale operations should the therapy receive FDA approval later this year.
For the rare disease community, the investment represents a significant step forward in advancing a potential new treatment for a devastating childhood disorder. With regulatory review underway and additional resources secured, Beren is now focused on preparing for a possible U.S. launch while continuing to support families affected by I-NPC.
The outcome of the FDA’s review later this year could mark a major milestone for both the company and the broader rare disease field, potentially introducing a first-in-class therapy that addresses the underlying cause of one of the most severe forms of Niemann-Pick disease.