Acadia, page-551

Hi Everyone,

I just finished listening to Acadia present at the Cantor Fitzgerald Global Healthcare Conference 2023 and below are my notes relating to Daybue:

• Acadia was represented by Mark Schneyer (CFO) and Dr Doug Williamson (Head of R&D). Steve Davis was not in attendance.
• In his opening remarks, MS stated that the Daybue launch “continues to go exactly as planned and exceed expectations”.
• Sales guidance for Q3 remains unchanged at US$45-55 million and no guidance given for Q4.
• Preliminary caregiver feedback is encouraging and reporting early signs of efficacy which is just as important as sales figures.
• ROW Deal - Acadia is very excited to bring Trofinetide to Retts patients worldwide (the brand name might not be Daybue outside of the US - TBC).
• The Moderator commented that Daybue’s US launch was “pretty impressive” and he was surprised at both the (high) adoption rate and Acadia’s confidence in providing sales guidance just two quarters in. MS replied that they could either give the market lots of KPI’s (which may be confusing) or sales guidance which maybe easier for investors to make sense of at this early stage of the launch.
• Acadia will continue to give one quarter forward guidance until, possibly, Q1, 2024 when they will switch to annual guidance.
• The Moderator mentioned 3 components of Daybue revenue: weight based dosing (a function of patient age); titration; and persistence. MS mentioned a fourth component: ultimate compliance to dose versus recommended dose which flows into product utilisation rates.
• The question was asked as to whether Daybue is being taken up by younger or older patients thus far. MS replied that the patient population broadly reflects the demographic of Retts patients but uptake is perhaps skewed slightly towards younger patients. DW chimed in to state that as Retts is a neurodevelopmental disorder, neurons don’t actually degenerate or die in patients and that’s why improvements can be seen at any age and this is what they are are seeing now.
• The “safety challenges” of Retts/Daybue was queried. DW was quick to point out that there were no safety issues but there were tolerability issues (gastrointestinal). In the clinical trial 40% of participants stayed in the one year study, which was considered normal.
• There were no diarrhoea management measures in the trial (perhaps a few protocols were implemented towards the end). In the real world rollout of Daybue so far, there has been a far lower rate of discontinuation due to GI issues. This was attributed to commonsense diarrhoea management protocols included in the prescribing label.
• The Prescription Refill Rate is an important metric for Acadia but is obscured by variables such as up and down titration by caregivers/doctors, “drug holidays” and true discontinuance.
• LOTUS Study - 2 year real world, observational study of Daybue will offer a more realistic profile of efficacy. Acadia will look at the study data every 3 months.
• ROW rollout - Acadia is excited to serve Retts patients worldwide and there is a high level of interest and awareness from around the world.
• Canada rollout will take 18 months, Europe will take a couple of years and a bridging study will be required in Japan.

• Acadia is not looking to build a “global empire” but do have the ability and global experience in-house to become more globally focussed.
• The Moderator said they would invite Acadia back to participate in Cantor Fitzgerald’s Brain Week Conference in December.

Unless another presentation comes in from left field, Acadia’s next event of interest will be their Q3 Earnings Call in early November (last year’s was held on the 2nd).

Night all