Ann: Phase 3 trial of trofinetide in Rett syndrome commences, page-8

Some relevant mentions from the transcript of today’s ACADIA Earnings Call particularly in regard Phase 3 timeline and end points.

MENTION 1

Roy Buchanan

Hi.It's Roy for Jason. Thanks for taking our question. Just a quick on the PhaseIII for trofinetide, how many sites are you guys planning and how quickly doyou think you might enroll that trial? Thanks.

Serge Stankovic

Yes.In that trial, we plan to enroll about 180 girls. So the number of sites iscorrespondent to that and it's not much bigger than what was done in the PhaseII trial. We plan initially to have about 16 sites or thereabout, and we willbe evaluating as we move forward in terms of the number of sites. But that'swhere we see it.

Interms of the recruitment period, obviously we will be assessing the base ofrecruitment as we progress in the trial. We just initiated the trial. Butinitially, we anticipated it may take between 12 and 18 months to completeenrollment in the trial.

MENTION 2

Beau Miller

Okay.Yes. Thanks for the helpful color there. And then, I guess, quickly ontrofinetide. Can you just remind us of the co-primary endpoints, and whether ornot both of those are required for approval?

Orhow the statistical hierarchy works there? And I guess, overall how do you kindof plan to manage the patient heterogeneity in this population, just given itencompasses, five-year-olds to 20-year-olds. And it can be quite variableinter-patient? Thanks.

Serge Stankovic

Yes.The first part, we have a, co-primary endpoints. One is Rett Syndrome BehaviorQuestionnaire, which is a caregiver assessment. And then the second is ClinicalGlobal Impression of Improvement, which is a physician assessment.

So,we need to win on both, in order for our trial to be positive, as being aco-primary measure. To your second part of your question in terms ofheterogeneity both measures.

Imean, Rett Syndrome Behavioral Questionnaire is a fairly broad assessment ofthe variety of symptoms that occur within the Rett syndrome. So, it's a -- itcapture -- because there are certainly a number of individual differences ineach particular patient.

Thisscale capture a fairly wide range of these symptoms, and it will be able toassess patients on the individual basis, vis-à-vis their baseline. Similarly,with Clinical Global Impression obviously, it's a physician assessment of thetotality of symptoms.

Sowe believe that, with those two scales we will be able to account for thevariability of symptom inter-subject variability so to speak, measuringindividual changes on the patients.

MENTION 3

Jay Olson

Hey,congrats on the quarter and thanks for taking my questions. I had two quickones. …. And then, my second question is a financial question, with regards tocapital allocation priorities and whether or not you're actively consideringany new business development opportunities. Thank you.

Steve Davis

Great.Thanks Michael. And in terms of the second part of your question, businessdevelopment is one of our three strategic pillars and we've publicly discussedfor some time our focus on building our pipeline through focused transactions.An example of that is our deal to acquire to North American rights to trofinetide,and we're beginning to see some of the fruits of that as we commence our PhaseIII program there. That kind of deal is an ideal strategic fit for us. Itleverages both our development and commercial capabilities. So, we'll continueto pursue those kinds of opportunities as we progress.

https://seekingalpha.com/article/4300699-acadia-pharmaceuticals-inc-acad-ceo-steve-davis-q3-2019-results-earnings-call-transcript?page=1