Mandos team members gathered on October 15th to share in the Global Wave of Light initiative to shine hope, love, and recognition on the NPC community #LightForNiemannPick

New Analyses in NPC

Mandos Health presented new analyses at the 2025 American Neurological Association (ANA) Annual Meeting (September 13–16, Baltimore, MD) and Child Neurology Society (October 8-11, Charlotte, NC), highlighting our continued work to advance understanding of the use of adrabetadex in NPC.

As we promised the community, we released this data as soon as the analyses were complete. We recognized, beyond being the right thing to do, it was ethical to inform patients making clinical decisions related to EAP participation. Following through on our commitments to the NPC community is core to our mission.

The two posters presenting data on adrabetadex were:

• Survival: “Substantial survival benefit with intrathecal adrabetadex in individuals with infantile-onset Niemann-Pick Disease Type C1”
• Biomarkers: “Restoration of cholesterol trafficking results in decreased markers of neuronal damage in individuals with Niemann-Pick Disease Type C1”

ANA informed us that our survival abstract received the ‘Abstract of Distinction’ award. This prestigious recognition highlights the quality of our scientific work.

These findings reflect our commitment to rigorous data evaluation and provide critical new context for the drug's clinical profile. We have consistently explored innovative approaches and used every learning to inform the path forward. We are grateful to the clinicians, families, and researchers whose collaboration made this possible.

We are excited to continue publishing and providing additional data and analyses to the community moving forward.

Development Program Update

Mandos Health has completed the new analyses of adrabetadex and is working towards a New Drug Application (NDA) submission. We will continue to provide updates moving forward.

EAP

We know how important ongoing access is for families. We remain fully committed to the EAP and will continue providing drug supply.

New & Upcoming Events

October is Niemann-Pick Disease Awareness Month

Mandos Health, by Beren Therapeutics Public Benefit Corporation (PBC), is a proud supporter of Niemann-Pick Disease Awareness Month. We will continue to share community communications and posts with our team and connections.

#niemannpickawareness #NPC #raredisease

• Mandos Health team members participated in the recent INPDA Annual Meeting in Argentina, September 18–21. We were honored to present data, network with colleagues, and strategize about the future.

• We attended the Child Neurology Society (CNS) in Charlotte, NC from October 8–11 where we had our first ever Mandos Health medical team booth and presented the new analyses.

Please reach out if you would like to connect with us at any future events: patientadvocacy@mandoshealth.com

Thank You

We extend our deepest gratitude to the NPC community—patients, families, clinicians, researchers, and advocates. We know that every advance we make is built on the dedication and sacrifices of patients, caregivers, and their families. We cannot thank you enough. As our CEO Jason Camm noted at the recent INPDA Annual, “This program would never have happened if not for the patients and their families working together to uncover its potential.” We firmly believe this and carry that spirit in all we do.

Your dedication has made our work possible and continues to drive us forward.

Stay up to date with us at www.mandoshealth.com

* Adrabetadex is an investigational drug that has not been approved by the U.S. Food and Drug Administration and has not been found to be safe or effective to treat NPC or any other condition. All data referenced are exploratory and subject to further validation. For more information, please contact patientadvocacy@mandoshealth.com