Beren Therapeutics has secured $300m in combined funding to support the potential commercial launch of its infantile-onset Niemann-Pick disease, type C (I-NPC) therapy, adrabetadex.
Split into an Eisai-backed equity funding round worth $135m, as well as up to $165m worth of strategic financing from Hercules Capital, the total sum will allow Beren to execute adrabetadex’s potential US launch in I-NPC, provided the drug secures the regulatory go-ahead.
Designed to directly address the buildup of cholesterol in various cells around the body, the underlying cause of NPC, adrabetadex contains a mixture of molecules that act as cholesterol shuttles, transporting trapped molecules out of the cell and re-establishing normal lipid trafficking. Beren says that this mechanism can help to slow the neurodegeneration associated with this toxic cholesterol buildup,
Upon its potential US approval, Beren is also taking steps to ensure that patients can receive treatment with the drug, which it will achieve by pouring funds into building an access infrastructure to provide treatment at local care sites. Beren will also set aside some capital to provide support to patients’ families, with efforts like speeding up diagnosis to facilitate early treatment and establishing a single point of contact for families planned by the public benefit corporation.
Extended review time creates uncertainty
However, the therapy’s future on the NPC market currently hangs in the balance, as the US Food and Drug Administration (FDA) recently extended the review time for adrabetadex in I-NPC by three months. This came after the agency requested further updates and clarifications to the existing data within Beren’s application under a “major amendment”, which pushes the FDA’s new target review date for the drug back to 17 November 2026.
If adrabetadex were to secure approval, it would join Zevra Therapeutics’ Miplyffa (arimoclomol) and IntraBio’s Aqneursa (levacetylleucine) on the I-NPC market, the former of which recently secured an exclusivity extension through to 19 November 2041 – more than doubling the remaining exclusivity on the asset.
Treating I-NPC
I-NPC is a rare disease that impacts the cellular waste disposal system’s ability to remove harmful cholesterol, which can cause buildup and subsequent progressive neurological decline and impacts on both quality of life and life expectancy. The impacts of this disease are particularly prevalent in patients who first have symptoms before the age of six, as early onset is commonly associated with rapid progression and poor outcomes.
According to the International Niemann-Pick Disease Registry (INPDR), around one in every 100,000 people globally have NPC, and those who have the disease often face challenges in obtaining a diagnosis, as well as barriers to quality care.
Currently, there are several companies looking to improve outcomes in NPC through their therapeutics. This includes Azafaros, which is currently exploring the potential of its oral therapy, nizubaglustat, in the Phase III NAVIGATE study (NCT07054515).
Cyclo Therapeutics is also looking into the efficacy and safety of its NPC drug, trappsol cyclo, in the Phase III TransportNPC study (NCT04860960), which logged an efficacy win in 2025 after the drug triggered a clinical improvement in seven of eight children treated for the disease.
