Beren Therapeutics

Thank you, NATIONAL NIEMANN-PICK DISEASE FOUNDATION (NNPDF), and everyone in the NPC community. We are back home from Orlando and deeply grateful for every conversation we had at the Family Support & Medical Conference last week. Meaningful conversations, substantive science, and a great "playful puppies session" (which Beren was proud to sponsor) made this a truly special event. Until next time!

This week, our team is heading to the NNPDF Family Support & Medical Conference in Orlando. We are grateful for the opportunity to present some of our work in Niemann-Pick disease, type C. Most of all, we look forward to spending time with the families, caregivers, and clinicians who guide everything we do.  Join us at the Friday afternoon session to hear presentations from Beren's Global Head of Medical Affairs Irene von Hennigs, PharmD and Elizabeth Berry-Kravis of Rush University Medical Center. In addition, our team will be sharing six posters from our research on adrabetadex in NPC. Healthcare providers can find our posters, plain language summaries, and more resources at https://lnkd.in/e438GDqT Thank you, NATIONAL NIEMANN-PICK DISEASE FOUNDATION for all the work you do and for convening this community.  

Today Beren announced $300 million in new financing to build long-term support for families living with infantile-onset NPC. Read more from our founder and CEO Jason Camm on what it makes possible, and and on the long-term work of supporting families well beyond a potential approval. Adrabetadex is an investigational drug intended to treat infantile-onset Niemann-Pick disease, type C (I-NPC) and is under FDA Priority Review, with a PDUFA target action date of November 17, 2026. If approved, it would be one step on a much longer road. https://lnkd.in/eZQ2jYJj

Thank you to the Ara Parseghian Medical Research Fund for another extraordinary effort to bring all of us — families, clinicians, and researchers — to the annual Michael, Marcia, and Christa Parseghian scientific conference for Niemann-Pick Type C research this weekend.  Gatherings like these are critical for us to be able to share our work over the course of the year. Our work is also decidedly a collective effort, which means that we also gather to share and renew the insistence that underlies it.     We're grateful to everyone who took efforts to bring their work to Tucson, who attended our sessions and shared with us over the course of the weekend, and we look forward to having even more to share next year. 

At this year's APMRF conference, our team is sharing six posters from our research on adrabetadex, an investigational therapy for infantile-onset NPC, which is currently under review by the FDA.    The presentations help inform questions this community has been working through together:   - how the timing of treatment relates to long-term outcomes   - what we're learning about how to sequence use of different NPC therapies  - the underlying biology of cholesterol trafficking in the disease  We're grateful to the Ara Parseghian Medical Research Fund for the opportunity to share, as well as to the investigators leading this work. Above all, we thank the families whose participation makes this type of scientific progress possible to support all families living with NPC. More detail is available in our press release here: https://lnkd.in/gSNm6uV5

The Beren team will be at #SIMD2026 next week. We're excited to connect with the inherited metabolic disorders community and discuss advances in care. Come find us to say hello. Learn more at berentx.com #NiemannPick #Inheritedmetabolicdisorders 

Today, we welcome leaders from the rare disease patient, scientific, government, and biopharma communities to Washington, DC, for our 15th Scientific Workshop, "Making What Matters Count: Advancing Patient-Centered Rare Disease Therapy Development Methodologies, Tools, and Knowledge Management," for specific community partners. Expert panels will review case studies on topics such as innovative clinical trial designs, novel surrogate endpoint development, and patient-centered research clinical endpoints, discuss what enabled these achievements, and brainstorm ideas for creating more consistent and scalable progress across the rare disease community. From these discussions, we will produce an action report highlighting participants' suggestions for improving knowledge management systems, regulatory guidance, and legislative policy. To learn more about the Scientific Workshop Series and to view select recordings and the action report following the Workshop, please visit our website: https://lnkd.in/dxJF89b Thank you to all of our 2026 Sponsors! This year's event is presented by Alexion Pharmaceuticals, Inc. Our Priority Review sponsors are Otsuka Pharmaceutical Companies (U.S.), Sanofi, and Sarepta Therapeutics. Our Clinical Trial sponsors include Alliance for Regenerative Medicine , Astellas Pharma, Biotechnology Innovation Organization, BridgeBio, Burroughs Wellcome Fund, Chiesi USA, Inc., Harmony Biosciences , ITF Therapeutics LLC, IQVIA, The Rare Disease Company Coalition, Stealth BioTherapeutics, Takeda, Ultragenyx, and Vertex Pharmaceuticals. Our Discovery sponsors include AZAFAROS B.V., Beren Therapeutics, Fulcrum Therapeutics, Palvella Therapeutics, REGENXBIO, and Stoke Therapeutics. We couldn't have done this without you!

At Beren, everything we do begins with the needs of patients and families living with NPC. We are committed to supporting the clinicians who care for them by providing clear and up-to-date scientific and clinical information. Additional information is available at medical.berentx.com

Today, Dr. Liz Berry-Kravis is presenting survival, clinical, and biomarker data related to infantile-onset Niemann-Pick disease type C (NPC) at ACMG - American College of Medical Genetics and Genomics’ Annual Clinical Genetics.  Learn more by reading the press release: https://lnkd.in/epK4y7uk #ACMGMtg26 #ACMG2026 #NiemannPick #RareDisease

We’re looking forward to joining other industry leaders at this week’s #ACMG2026. Stop by booth 111 to connect with our Beren team. Learn more about Beren at berentx.com #NiemannPick #RareDisease #acmgmtg26