Beren Raises $300 Million to Prepare for Potential Commercial Launch of NPC Therapy

Rare Daily Staff

Beren Therapeutics said it raised $300 million to support the potential U.S. commercial launch of its investigational drug adrabetadex for infantile-onset Niemann-Pick disease, type C, a rare, rapidly progressive, and always fatal pediatric neurodegenerative disorder.

The capital will also fund long-term patient access and family support programs aimed at easing the complex care burden families face over what can be a multi-decade disease journey.

The financing package combines a $135 million equity round with up to $165 million in flexible, non-dilutive capital from Hercules Capital. The equity raise brings together a group of specialist healthcare and strategic investors, including Wellington Partners, Japan Investment Corporation Venture Growth Investments, Founders Fund, Narya Capital, Eisai, and other institutional backers. In parallel, Beren entered into a $165 million facility with Hercules, consisting of up to $110 million in senior secured term loans tied to regulatory and revenue milestones and a $55 million royalty financing that will be funded upon potential U.S. Food and Drug Administration approval of adrabetadex.

Infantile-onset NPC affects infants and children who first show neurological symptoms before age 6 and is caused by genetic defects that disrupt intracellular cholesterol trafficking, leading to progressive neurological decline and early death. Earlier onset is associated with faster progression, with mean ages of death around 5.6 years for early infantile-onset cases and 13.4 years for late-infantile onset.

Adrabetadex, a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers, is designed to restore cholesterol trafficking inside cells and directly target the accumulated cholesterol believed to drive disease pathology. Clinical trials and expanded access programs suggest the drug is generally well tolerated, with the main adverse events reported as hearing impairment, which can often be managed with hearing aids, and post-dose fatigue or ataxia.

Beren said the transaction is designed to position the company to become financially self-sustaining if adrabetadex reaches the market, while retaining the flexibility to invest in longer-term growth initiatives. The FDA has accepted Beren’s New Drug Application for adrabetadex in infantile-onset NPC and is reviewing it under Priority Review. The therapy has also received Breakthrough Therapy Designation but is not yet approved in any country. The agency set a target date of November 17, 2026, to act on Beren’s application to market the drug.

Under the terms of the Hercules agreement, Beren can access up to $110 million in term loans as it hits predefined regulatory and commercial milestones; $30 million has already been drawn. The $55 million royalty financing, which would be funded at the time of a potential FDA approval, is structured with a 7.5 percent royalty on U.S. net sales of adrabetadex and a 5 percent royalty on sales outside the United States, capped at 1.75 times the invested amount through 2031. The company also has the option to redeem the royalty stream at a lower multiple within the first two years, a feature aimed at lowering its long-term cost of capital.

“Families have consistently told us that their burden extends far beyond the availability of therapies, and that there remains a substantial unmet need for long-term support as families navigate diagnosis, treatment decisions, specialist care, reimbursement, and changing needs over a multi-decade journey with this disease,” said Jason Camm, founder and CEO of Beren. “Our vision is not only to develop new treatment options, but also to help address the broader challenges families face throughout their journey.”

Photo: Jason Camm, founder and CEO of Beren Therapeutics