Chiesi is officially a significant strategic player in Lysosomal Storage Disorders given its recent US and European approval for Fabry. It could have the emerging standard of care in the multi-billion Fabry market. This increases the strategic significance of xB3 to Chiesi given Chiesi's licensing xB3 deal for four Lysosomal Storage Disorders and that the rush to treat the brain is on as validated by Denali, etc. Fabry also involves the brain. BTI's lead xB3 pipeline program is for Hunter which DNLI presented data for today potentially strengthening the path forward. BTI also has Gaucher's in its internal pipeline which is the largest Lysosomal Storage Disorder. DNLI has also now shown the ability to lower NfL which is important for a number of neurodegenerative diseases.
DNLI's release today for Hunter:
“These are impressive data and the first report of a robust reduction in NfL observed with any treatment in MPS II,” said Henrik Zetterberg, M.D., Ph.D., Professor and Chief Physician at the University of Gothenburg. “NfL is an established marker of neuroaxonal damage, which has demonstrated utility as a biomarker of therapeutic response in other neurodegenerative diseases such as CLN2, MS, SMA, and ALS.”
“The robust reduction and normalization of CSF heparan sulfate, and now the downstream reduction in NfL after treatment, are consistent with positive changes in clinical outcomes measures we have observed from interim analyses of the ongoing Phase 1/2 study,” said Carole Ho, M.D., Chief Medical Officer at Denali. “As we advance DNL310 as a potential treatment option for individuals living with MPS II, we look forward to ongoing engagement with the FDA and the MPS community.”