Acadia $ACAD has decided to change the game for its drug Nuplazid (pimavanserin) going further into Phase III, after the FDA handed out its breakthrough drug designation for the therapy.
Approved already for Parkinson’s disease psychosis, the biotech had been setting the stage for a pivotal trial in Alzheimer’s disease psychosis for their 5-HT2A therapy. But in announcing the launch of their HARMONY study, execs say now that they will recruit a mix of patients with Alzheimer’s disease, dementia with Lewy bodies, Parkinson’s disease dementia, vascular dementia and frontotemporal dementia.
The move comes as regulators provided the BTD title to Nuplazid as a treatment for disease psychosis, an endorsement for easier access at the FDA that should help the biotech push through the next round of late-stage work.
At the same time, they’re scrapping a Phase II Alzheimer’s study after posting somewhat controversial mid-stage data with mixed results. The drug hit at week 6 but failed to on the psychosis score at week 12.
The biotech says it decided to stop the study because it overlapped with their Phase III sites. Perhaps not coincidentally, it removes any chance of negative data squirreling their work in Phase III.
Shares at Acadia surged about 6% this morning.
Leerink’s Paul Matteis added up the news and concluded that it provided a mixed picture for the company at this stage.
On the plus side, FDA feedback awarding breakthrough designation and allowing for a single pivotal study is encouraging, especially with respect to how the agency views pimavanserin safety. Moreover, if the study succeeds, the addressable population could be very large, rendering how we currently model the ADP revenue oppty conservative. However, ACAD’s “basket” approach to this new ph3 may introduce additional challenges that are simply difficult to handicap: while the trial works to enrich the placebo-controlled portion for pimavanserin responders, we see the inclusion of such a heterogeneous population in a single pivotal as a risky strategy.
“We are pleased the FDA has agreed to an efficient development path for pimavanserin in this broad indication and granted Breakthrough Therapy Designation in recognition of this serious unmet need,” said Acadia R&D chief Serge Stankovic.
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