Beren Therapeutics Announces Six Presentations at the 2026 Ara Parseghian Medical Research Fund (APMRF) Conference

THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Beren Therapeutics P.B.C., the parent company of Mandos LLC and a leader in cholesterol trafficking biology and cyclodextrin-based therapeutics, today announced that the company will present six posters at the 2026 Michael, Marcia, and Christa Parseghian Scientific Conference for Niemann-Pick Type C Research, sponsored by the family of former Notre Dame head football coach Ara Parseghian, taking place May 30 through June 2 in Tucson, Arizona. The posters present clinical and preclinical data on adrabetadex, an investigational cyclodextrin-based therapy under review by the U.S. Food and Drug Administration (FDA) for infantile-onset Niemann-Pick disease, type C (I-NPC). Beren’s Chief Executive Officer Jason Camm will also participate in a fireside chat at the conference with APMRF director Sean Kassen on Sunday, May 31 at 4 p.m. MST.

Poster Presentations

The following posters will be presented during the conference poster sessions on Sunday, May 31 and Monday, June 1.

Earlier treatment and long-term outcomes

• Poster #25: Outcomes in participants with infantile-onset Niemann-Pick disease type C receiving prompt and sustained treatment with adrabetadex
  • Data suggest adrabetadex administration was associated with slower disease progression relative to published natural history data. Observations also suggest differences based on timing of treatment initiation.

• Poster #26: Survival outcomes by baseline disease burden in participants with infantile-onset Niemann-Pick disease type C treated with adrabetadex
  • Retrospective survival analyses of individuals with I-NPC treated with adrabetadex – in clinical studies or the Expanded Access Program – support the importance of early diagnosis and prompt initiation of treatment before irreversible neurodegeneration occurs.

Adrabetadex use alongside approved NPC therapies

• Poster #6: Early evidence of an emerging NPC treatment paradigm: adrabetadex use alongside arimoclomol and/or N-acetyl-L-leucine
  • This represents the first systematic characterization of intrathecal adrabetadex use alongside approved NPC oral therapies. The treatment patterns observed in the Expanded Access Program may provide early insight into how adrabetadex can serve as a foundational therapy while complementary therapies are layered based on patient need.

• Poster #2: Adrabetadex alone or in combination with arimoclomol and levacetylleucine promotes oligodendrocyte proliferation, synaptic network growth, and myelination in an in vitro mouse model of Niemann-Pick disease type C
  • Preclinical data suggest adrabetadex demonstrated greater potency than approved NPC therapies across key measures of neuronal and oligodendrocyte health. Combination of adrabetadex with arimoclomol or NALL achieved comparable effects at substantially lower concentrations than monotherapy, supporting mechanistic compatibility between adrabetadex and therapies with complementary mechanisms.

Mechanism of action

• Poster #18.1: Mechanism of action of adrabetadex for treatment of Niemann-Pick disease type C
  • Preclinical data for centrally administered adrabetadex are consistent with broad CNS distribution and sustained target engagement of intracellular cholesterol, supporting the mechanistic basis for restoration of intracellular cholesterol trafficking in superficial and deep brain regions.

• Poster #18.2: Disease modifying effects of centrally administered adrabetadex for treatment of Niemann-Pick disease type C
  • Preclinical NPC mouse model data offer support for the effects of adrabetadex on myelination, neuronal integrity, and neurodegenerative biomarkers, with findings offering mechanistic support for improved clinical outcomes.

“We appreciate the opportunity to present the most current data in support of our adrabetadex program. These data deepen our understanding of adrabetadex in infantile-onset NPC and reinforce the importance of recognizing and treating this disease as early as possible,” said Irene von Hennigs, PharmD, SVP of Medical Affairs of Beren Therapeutics P.B.C. “We will keep sharing what we learn, openly and promptly, alongside the families and clinicians who have shaped this work from the beginning.”

Fireside chat with Beren Therapeutics CEO Jason Camm

Jason Camm, Chief Executive Officer of Beren Therapeutics P.B.C., will also participate in a fireside chat at the conference with Sean Kassen, Director of the Ara Parseghian Medical Research Fund at the University of Notre Dame at 4 p.m. MST on Sunday, May 31.

The full posters are available to healthcare professionals on Beren’s website at https://medical.berentx.com/.

The FDA granted adrabetadex Breakthrough Therapy Designation in 2025, and in February 2026, the NDA for adrabetadex in infantile-onset NPC was accepted by the FDA for Priority Review with a PDUFA target action date of November 17, 2026. Adrabetadex has not been approved by the FDA or any other health authority at this time.

About Infantile-Onset Niemann-Pick Disease, Type C

Niemann-Pick disease, type C (NPC) is a rare, autosomal-recessive, severe, heterogeneous, neurodegenerative disorder caused by pathogenic variants in the NPC1 (~95% of cases) or NPC2 genes, leading to impaired intracellular cholesterol trafficking resulting in progressive neurological decline and premature mortality. Infantile-onset NPC (I-NPC) refers to NPC in infants and children who first experience neurological symptoms <6 years of age. Earlier neurological onset is associated with more rapid progression and poorer prognosis, with mean ages of death of ~5.6 years for early infantile-onset (age of neurological onset <2 years) and ~13.4 years for late-infantile onset (2 to <6 years).

About Adrabetadex

Adrabetadex is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers suitable for intrathecal delivery, under investigation as a treatment for Niemann-Pick disease, type C (NPC). Clinical and nonclinical data demonstrate that adrabetadex directly targets the underlying pathophysiology of NPC by re-establishing intracellular cholesterol trafficking. Adrabetadex is generally well tolerated, with a well-characterized safety profile established over more than a decade of clinical development. The most common adverse events are hearing impairment (manageable with hearing aids when necessary) and post-dose fatigue and ataxia. Adrabetadex has not been approved by the FDA or any other health authority at this time.

About Beren Therapeutics P.B.C.

Beren Therapeutics P.B.C.^® is a founder-led, clinical-stage biotechnology company pioneering the discovery, development, and commercialization of cyclodextrin-based therapeutics for conditions characterized by defective cholesterol trafficking. Beren and its subsidiary Mandos LLC^® are committed to the development of adrabetadex for individuals living with Niemann-Pick disease, type C (NPC) and have supported the NPC community by providing access to adrabetadex through an Expanded Access Program (EAP).

Beren’s public benefit purpose is to discover, develop, and deliver novel therapies that provide optimal benefit for patients, and to do so by integrating the needs of patients, caregivers, clinicians, and health systems from the beginning of the development process and maintaining a long-term focus on delivering meaningful therapies and access.

Beren is headquartered in Thousand Oaks, Calif. To learn more about Beren, the adrabetadex program, and Beren’s cholesterol-trafficking focused therapeutic strategy, visit the company’s website or visit Beren’s LinkedIn channel.