Beren Therapeutics P.B.C. has secured $300 million in combined financing to support preparations for the potential commercial launch of adrabetadex and expand long-term care initiatives for families affected by infantile-onset Niemann-Pick disease, type C. The Thousand Oaks, California-based biotechnology company said the financing will strengthen its commercial readiness efforts ahead of the U.S. Food and Drug Administration’s Prescription Drug User Fee Act target action date of November 17, 2026.
Financing Combines Equity and Non-Dilutive Capital
The financing package includes $135 million in equity funding from specialist healthcare investors, corporate investors, and long-term growth partners. Participants include Wellington Partners, JIC Venture Growth Investments, Founders Fund, Narya Capital, Eisai Co., Ltd., and other institutional investors. Beren also entered into a financing arrangement with Hercules Capital that provides up to $165 million through a senior secured debt facility and royalty financing.
Proceeds to Support Adrabetadex Launch Readiness
Beren plans to use the proceeds to support activities tied to the potential U.S. launch of adrabetadex, an investigational therapy under FDA Priority Review for infantile-onset NPC. The company said the funding will help build commercial readiness capabilities, patient access programs, care infrastructure, and resources designed to assist families throughout diagnosis, treatment, reimbursement, and long-term disease management. If approved, adrabetadex would represent a first-in-class disease-modifying treatment for infantile-onset NPC and the only approved therapy designed to directly address the accumulated intracellular cholesterol that drives the disorder.
Addressing a Severe Pediatric Disease
Infantile-onset NPC is the most severe and rapidly progressive form of Niemann-Pick disease, type C, a rare and fatal pediatric neurodegenerative condition caused by impaired intracellular cholesterol trafficking. The disease is linked to pathogenic variants in the NPC1 or NPC2 genes and leads to progressive neurological decline, premature mortality, and extensive care needs for affected families. Earlier neurological onset is associated with faster disease progression, making timely diagnosis and intervention especially important for infants and young children.
Company Focuses on Family Support
Beren said its strategy extends beyond drug development to address the broader challenges families face while managing a complex, lifelong disease journey. The company intends to invest in programs that support earlier diagnosis, local access to sites of care, centralized family support, real-world evidence generation, and stronger community learning networks. Founder and Chief Executive Officer Jason Camm said families affected by infantile-onset NPC face burdens that go well beyond treatment availability, including specialist coordination, reimbursement barriers, and evolving care needs over many years.
Adrabetadex Targets Cholesterol Trafficking
Adrabetadex is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers being developed to treat Niemann-Pick disease, type C. Beren says the therapy is designed to re-establish intracellular cholesterol trafficking and directly address the underlying disease biology. Clinical trial and expanded access data cited by the company suggest adrabetadex has been generally well tolerated, with reported adverse events including hearing impairment, post-dose fatigue, and ataxia.
Strategic Financing Details
Under the Hercules Capital agreement, Beren may access up to $110 million in term loans tied to regulatory and revenue milestones, with $30 million already drawn. The package also includes $55 million in royalty financing to be funded upon FDA approval of adrabetadex, carrying royalties on U.S. and international net sales subject to a cap and redemption terms. Morgan Stanley & Co. LLC acted as sole structuring agent, while Wilson Sonsini Goodrich & Rosati advised Beren and Latham & Watkins advised Hercules.
Building Toward Potential Commercialization
Beren, the parent company of Mandos LLC, has supported the NPC community since 2021 through an Expanded Access Program and is preparing for potential commercialization if adrabetadex receives approval. The company’s public benefit purpose emphasizes integrating the needs of patients, caregivers, clinicians, and health systems into drug development and access planning. The new financing is intended to help Beren become financially self-sustaining through a potential commercial launch while preserving flexibility for long-term growth initiatives.
The $300 million financing gives Beren Therapeutics substantial resources as it approaches a key regulatory milestone for adrabetadex in infantile-onset NPC. By combining commercial launch preparation with patient access and family support programs, the company is seeking to address both the biological and practical challenges associated with the disease. With an FDA decision expected by November 17, 2026, Beren’s next phase will be closely watched by investors, clinicians, and families affected by this rare pediatric disorder.