Expert Interview
Discussing Denali Therapeutics’ investigational brain-penetrant enzyme therapy, tividenofusp alfa, for mucopolysaccharidosis type II (Hunter syndrome)
Ticker(s): DNLI, TAKInstitution: University of Minnesota Medical School
- Professor of Pediatrics and Experimental & Clinical Pharmacology
- Manages hundreds of patients with Sanfilippo Syndrome Type B (MPS IIIB) and several patients with Hunter Sydrome.
- International leader in lysosomal and metabolic diseases, focusing on development of therapies for mucopolysaccharidoses and related lysosomal storage disorders, including pioneering work in bone marrow transplantation, enzyme replacement strategies, and early clinical trials of gene therapy; leads large collaborative research networks studying diagnosis and treatment of rare genetic diseases
How are you currently managing patients with Hunter syndrome, and what limitations do you see with existing therapies in addressing neurologic versus peripheral disease manifestations?
What is your interpretation of the clinical data for tividenofusp alfa, particularly the reported effects on CNS biomarkers, peripheral biomarkers, and clinical measures such as cognition, adaptive behavior, hearing, or organ involvement?
Added By: sara_adminHow clinically meaningful do you believe a brain-penetrant enzyme replacement therapy could be for patients with MPS II, especially if initiated before advanced neurologic impairment?
Added By: sara_adminHow do you expect to discuss the option of switching with your patients? Do you expect to have these discussions with stable patients? Right away? At annual check-in?
Added By: jima3Do you have any concerns about antibody development/Neutralizing antibodies that could impact the efficacy of Avlayah? How will that be assessed?
Added By: jima3What is on your "checklist" for evaluating patients for whether they should switch to Avlayah
Added By: jima3will you proactively call in patients for an appointment to switch them or wait for their annual visit - especially if they don't reach out to you immediately
Added By: jima3Do you expect any of your patients remaining on Elaprase? Which patients? Why?
Added By: jima3Do geneticists at small community hospitals have familiarity around new therapies? Do they treat Hunter patients or refer them out to COEs?
Added By: jima3How concerning are the precautions around membranous nephropathy and anemia listed on the label?
Added By: jima3What additional data do you wish/hope to have to support your decision to prescribe Avlayah?
Added By: jima3Can a patient return to Elaprase if they can not tolerate Avlayah? Will antibodies make that impossible?
Added By: jima3Are IRRs a concern with Avlayah?
Added By: jima3Are You Interested In These Questions?
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