Increasingly Favorable Regulatory Environment Bodes Well for ALXN1840. Increase PT to $117 from $104.

研报英文原文证据摘录

Increasingly Favorable Regulatory Environment Bodes Well for ALXN1840. Increase PT to $117 from $104.

■ Supportive Phase 2 Trials Should Meet the Bar for Confirmatory Evidence: The updated guidance notes that if a single trial

is adequately designed to provide highly persuasive results, it is expected that the available early phase information may

provide sufficient confirmatory evidence. We believe the three ph 2 trials (WTX-101-201, ALXN1840-WD-205, and the copper

balance study) from ~65 pts showing a stat sig benefit on maintaining normalized copper levels, reductions in hepatic copper

levels, and improvement in copper balance/increase in fecal copper excretion with the updated analysis meet the bar for

confirmatory evidence.

■ Benefit-Risk Trade Off Aligns in MNPR's Favor: The latest guidance affirms that any sponsor development program must be

sufficient to determine benefit/risk. This will be the most critical component of the ALXN1840 application, as any lingering

uncertainty surrounding the drug's mechanism of action and signals of liver enzyme elevations in select studies will be weighedBIOTECHNOLOGY

against the greater copper mobilization, positive trends on neurologic improvement, renal and liver related SAEs <5% out to

six years across 266 pts, and an easier dosing format. Net-net, the benefits outweigh the risks in a pt population such as WD

which has high unmet need in our view.

■ Application Status and Path from Here: Earlier in June, mgmt noted to us that they are actively engaged in the NDA submissionEQUITY

process, with the FDA having made an information request for which the co submitted data (MNPR is working with hepatology

and neurology divisions of agency). Constructive discussions remain ongoing, which mgmt believes reduces the risk of a