Hmmmm. Is Shire's failure to meet its primary endpoint of slowing cognitive decline in its Sanfilippo syndrome type A (MPS-IIIA) lysosomal storage disease (LSD) trial of SHP610 due to inefficient crossing of the blood brain barrier? If so, they know who to call.

From the Fierce Biotech article:

"Now toShire, which quietly got rid of its midstage candidate SHP610, a med that was being studied in patients with Sanfilippo syndrome type A (MPS-IIIA). “The Phase IIb study of SHP610 in pediatric patients with early stage Sanfilippo A did not meet its primary endpoint of slowing the cognitive decline in patients,” Shire said in a brief update amid its financials.

The Big Biotech said it will now “terminate all clinical trials of SHP610” and plans to publish the results of the SHP610 program “for the benefit of the Sanfilippo community.”

Shire is however not the only co that was testing for this disease area, with Alexion ($ALXN) recently touting its progress on its experimental Sanfilippo B drug, with gene therapy biotech Lysogene and Abeona Therapeutics ($ABEO) also working on Type A forms of the disease."