Community Update — Building Long-Term Support for Families

Dear NPC Community Partners,

This is the first update you are receiving under the Beren name, and we want to take a moment to explain why. Much of the work you have come to know, including the Expanded Access Program and our submission to the FDA, has been carried out by Mandos LLC, a wholly owned subsidiary of Beren Therapeutics. Going forward, that work continues under the Beren name. The team, the commitment, and the people you have come to trust are the same. We are bringing it all together under one name, Beren, so it is clear who stands behind this work for the long term.

We are writing to share an update on how we are preparing to support families living with infantile-onset Niemann-Pick disease type C (I-NPC) in the months and years ahead.

First of all, thank you. Everything described below grew directly out of what this community has told us, over years of listening, about what living with NPC actually requires.

We have secured new long-term funding that lets us commit to this work for the long haul. What matters most to us is what that makes possible. We are building a set of programs designed to support families across the full NPC journey, from the first search for a diagnosis through the day-to-day of care, whatever the road ahead holds.

We believe adrabetadex, if approved, has the potential to become an important treatment option for children with I-NPC. Preparing for a potential approval is about more than manufacturing medicine. It also means investing now in the people, systems, and infrastructure needed to support families before, during, and after treatment.

Here is what we are building:

  • Faster, clearer diagnosis. Too many families spend years searching for answers. Every year of diagnostic delay matters because children are losing developmental time they cannot get back. We are investing in dedicated patient identification and in the most comprehensive genetic testing we can offer, so the path to a diagnosis is shorter and clearer.
  • A single point of contact. A diagnosis can turn a parent into a full-time coordinator of specialists, insurance, and logistics. We are building dedicated rare disease case management, so one call can take the place of many.
  • Care closer to home. We are developing a network of care sites intended to reduce travel and bring treatment nearer to where families live.
  • Shared learning that grows over time. We are gathering real-world evidence so families, clinicians, and treatment centers can keep learning from one another, and so care keeps improving year over year.
  • A connected community. We are strengthening peer-to-peer education and connection, so families and clinicians can learn from others who understand this disease firsthand.

Recently, our team had the privilege of joining the NPC community at the Ara Parseghian Medical Research Fund (APMRF) scientific conference in Tucson, one of the few gatherings that brings families, clinicians, and researchers together in the same place at the same time. I had the chance to sit down with APMRF director Sean Kassen for a fireside conversation, and we came to that conference to listen as much as to share.

At the APMRF conference, our team presented six scientific analyses focused on adrabetadex. Together, these data reinforced several important themes: the importance of early diagnosis and prompt treatment, a deeper understanding of how intrathecal adrabetadex reaches and acts within the central nervous system, and emerging experience with adrabetadex used alongside other approved NPC therapies. Most importantly, the findings continue to support the idea that earlier intervention may help preserve function and improve long-term outcomes for children living with I-NPC. We remain committed to sharing what we learn openly and promptly with the families and clinicians who have shaped this work from the beginning.

A note on where things stand with adrabetadex. Adrabetadex remains an investigational drug. The FDA accepted our New Drug Application for infantile-onset NPC and is reviewing it under Priority Review, with a target action date of November 17, 2026. Throughout the review, our Expanded Access Program continues and remains available to all eligible patients. Families who believe adrabetadex may be relevant to their child should continue discussions with their treating physician and care team.

We are sharing these plans now because the support families need cannot wait for a single date on the calendar. It has to be built in advance and built to last.  

From all of us at Beren, thank you for your trust, your partnership, and your patience. We recognize the daily realities that people living with NPC and their caregivers face, and your guidance will continue to shape this work with urgency and care.

With gratitude,

Jason Camm
Chief Executive Officer
Beren Therapeutics P.B.C.

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