Call Start: 10:00
Call End: 10:26
ACADIA Pharmaceuticals (NASDAQ:ACAD)
Jefferies Global Healthcare Conference
June 2, 2014 10:00 AM ET
Uli Hacksell - CEO
So the next presenter is Uli Hacksell, he is the CEO of ACADIA Pharmaceutical.
Thank you and good morning to you all, thanks for joining us. Let me first say that I’m going to make some forward looking statements today and therefore I do encourage you to read the risk factors that we have filed with the SEC. Now, ACADIA is seen as focused company we have a pipeline that we think is quite exiting led by a compound called pimavanserin which is Phase 3 for Parkinson’s disease Psychoses a condition developed any acceptable therapy today. pimavanserin is a new type of drug to treat psychoses with, it doesn’t the kind of side effects that you see with traditional antipsychotics, we believe that it has great commercial potential and we are excited about trying to build a strong neurology franchise based on the success with pimavanserin in the United States which we think is, represents a perfect opportunity for ACADIA to really address the commercial opportunities with pimavanserin.
Importantly we have worldwide rights to this molecule and we see great opportunities also outside of the US overtime. Behind pimavanserin we have two clinical collaborations with Allergan which emanate from discovery collaborations. One Phase 2 program for chronic pain, one Phase 1 program for glaucoma, both of these programs are conducted, run and paid for by Allergan, what we hope to be able to do over time is to receive addition milestones royalties from Allergan. We also have earlier stage programs that digress, to see small defining principles for conditions like Parkinson’s disease, Alzheimer’s disease etc. and those are funded predominantly by external forces including Michael J. Fox foundation and the NIH.
But the focus of ACADIA is very much on pimavanserin and we think this is a commercial exciting opportunity because not only of the first indication that they’re moving forward with Parkinson’s disease psychosis but also about related to the great expansion opportunities into the neurological space into areas like Alzheimer’s disease psychosis, Lewy body diseases, psychosis etc. and also into the psychiatry space, exemplified by schizophrenia, what we also think is important is that the first step for us is to build a neurology focused sales force in the US and that can be done relatively, in a relatively straightforward manner and then expand from there. So we think that pimavanserin represents unique perfect opportunity for ACADIA to move into the commercial phase. Pimavanserin itself is a selective molecule that selectively blocks one of the serotonin receptors called the 5HT-2A receptor.
It is a molecule that has demonstrated a very attractive effectiveness in treating psychosis; it has also demonstrated very attractive safety and tolerability profile. The side effect profile of pimavanserin is placebo like which is something that we haven’t heard about when it comes to other anti-psychotic therapies today. We have a great IP related to pimavanserin that takes us into mid 2028 in the United States, so we will have lots of time to fully explore the commercial potential of the drug. The fact that we gave again kept all the rights to pimavanserin is another very important aspect of course for the potential of pimavanserin for ACADIA.
So why did we take Parkinson’s disease psychosis as a lead indication for pimavanserin. Well, this is an area with a large unmet medical need, Parkinson’s psychosis consists of hallucinations and delusions that very negatively affect not only the patient but also the caregiver in fact Parkinson’s psychosis is the major reason why Parkinson’s patients have to go to nursing homes. It becomes too difficult for the caregiver to deal with the patient and by the way the caregiver is frequently responsive. And there’s nothing to treat these patients with that is [Indiscernible], there’s no approved drug to treat PDP, and the reason for that is that the anti-psychotic drugs, they counter act the therapy for Parkinson’s disease itself. It’s not only a serious condition it’s also a common condition. In fact about 40% of Parkinson’s’ patients suffer from psychosis. And this psychosis may be divided into mild, moderate and severe psychosis, so about one third if you look at the number of patients. In the US alone there are about 1 million Parkinson’s patients. This is an increasing number, the same goes for the rest of the world, Parkinson’s disease becomes from frequent and therefore Parkinson’s psychoses become more frequent with an aging population that we see everywhere. So it’s a severe and common condition without a good therapy. And this line really addresses the problem with the existing anti-psychotic drugs and the reason why they cannot be used in Parkinson’s psychosis. So the medications that are used to treat Parkinson’s disease, address the fact that in Parkinson’s disease you have a death of the cells that produce dopamine, so the therapy for Parkinson’s disease is to try to supplement that with dopamine stimulating drugs. Drugs that stimulate the dopamine d2 receptor, and as you can see on this slide all the anti-psychotic drugs they block the dopamine d2 receptor so they are pharmacologically counter indicated for use for this indication. At the same time they also address large number of additional receptors which are, which really add to the unpleasant side effect profile of these drugs. For example, they powerfully block the H1 receptor which causes severe sedation and they interact with the alpha receptors which causes cardiovascular problems. Pimavanserin doesn’t have these problems; it’s only blocks the 5-HT2A receptors. It doesn’t touch the dopamine receptors or the histamine or the alpha receptors. Therefore it’s safe. It’s well tolerated by the Parkinson’s patients and it doesn’t interfere with the therapy for the motor symptoms in Parkinson’s disease.
So we have conducted a pivotal Phase 3 study that we call the -020 study. We reported that in the end of 2012, and it showed that pimavanserin has a great profile, it’s effective in treating the psychosis in Parkinson’s patients, it also showed that pimavanserin can be effective when it comes to dealing with sleep problems in Parkinson’s patients, it reduced the caregiver burden, importantly and in addition to that pimavanserin was safe and well tolerated. So this was a six week study which was conducted in 200 patients in North America, randomized 1:1 between placebo and 40 milligrams of pimavanserin. The study was designed so that we really tried to minimize placebo effects. So, for example, we used a brief psychosocial therapy run in before we randomized patients, and this essentially consisted of teaching the patient and the caregiver to interact on a subject of common interest to relieve some of the stress in their relationship and also to have both caregiver and patient interact with the site ahead of randomization. So we believe that this was important aspect of the low placebo response in this study. We also used centralized rating which means that we interviewed -- we had a small group of raters who interviewed patient and caregiver independently of the investigator, through a video link, that way we could minimize the number of raters, we could ensure that we used the same type of rating throughout and we believe that that really produced the variability and the study.
Also, we, by doing that we could remove any kind of investigatory bias in the study which is another important factor. So the outcome of the study at six weeks was in fact spectacular. You can see in blue what happens over time in 40 mg pimavanserin arm and in yellow what happened in the placebo arm. That six weeks we had a highly significant effect of pimavanserin on the psychosis which was measured using the SAPS-PD scale, a scale which reflects the symptoms which are typical for Parkinson’s disease psychosis. We also saw a significant improvement after four weeks. This improvement that we saw was also clinically significant which is of course back and forth. We also have the investigators themselves looking at the psychosis in the patients using a different scale, the CDI scale and that was done independently over the centralized raters. And what you can see in the table here is that the significance based on the independent investigator assessment of the psychosis improvement was at high as when we host these centralized independent raters. So regardless of what rating scale we used, regardless of who rated the patients, we observed highly significant improvement of these patients when they were on pimavanserin.
We also -- when they started at the improvement or the reduction in psychosis was driven not only by improvement in hallucinations or improvement in delusions but by both show SAPS-H and SAPS-D reflect hallucinations and delusions respectively; and both came out significantly improved in the pimavanserin treated patients. We had the UPDRS scale as the key secondary endpoint in this study. And what we really wanted to demonstrate here was that the patients given pimavanserin did not have any versioning of motor symptoms compared to placebo. And that’s what we showed and that is not surprising because we do not touch the dopamine receptors with pimavanserin. If we had used atypical antipsychotic drug instead, you’d have seen a significant versioning of motor symptoms in these patients if the drug would have been used at effective doses.
This study, the O20 study did not only demonstrate motor rate tolerability and psychotic efficacy but also improvements in sleep. This was not really surprising because we know that pimavanserin has the ability to make you sleep deeper if not wake up less. It is not a sedating molecule. It doesn’t help you to go to sleep. It only makes you sleep deeper. It changes the sleep architecture in such a way that you spend less time in superficial sleep and more time in deep sleep. So you sleep more like a baby essentially, and that is reflected here in the highly significant improvement in the quality of night time sleep on pimavanserin. What we also saw was an improvement in the daytime wakefulness in the patients on pimavanserin. This is also important because for some patients do not only have sleep problems at night, they tend to suffer from excessive sleepiness during the day and this is of course an important additional benefit from pimavanserin’s clinical profile.
This study also demonstrated that caregivers had sort of positive effect on pimavanserin. So in the study we also -- caregivers to fill out a form that related to how difficult it was to interact with the patients. And you can see six week’s -- we saw a significant, a highly significant production in the caregiver burden as assessed by the caregivers themselves. And I think this is a very-very important background because we can’t, we believe translates these effects into health economical benefits into a delayed time to nursing home placements and we are very excited about this. And I think it is important to note that caregivers, in this study they spent 24 hours a day with the patients and they are the -- the fact that they really saw a meaningful improvement in the patients and that was reflected in caregivers having reached less problems in dealing with the patient is I think very-very important.
When patients have completed our studies, like the -020study, they have had the opportunity to move into over internal open label expansion study. And this has provided us with a large safety database which we are still building on. We currently have more than 800 years of PDP exposure in this safety database. We have patients on track for more than eight years and throughout when we analyze the study we see a very favorable long-term safety profile of the drug which is completely differentiated from what you would expect to see with one of the atypical antipsychotic drug which in fact increased the mortality of patients of this type, and also have a number of side effect problems. With increased differentiation from the antipsychotics which are currently used off label for PDP and related indications is the key advantage of pimavanserin.
So when we saw the data from the -020 study we assembled a briefing package that not only consisted of this particular study outcome but also had all the long-term data in it. It also reflect -- essentially describe the whole program for pimavanserin. And we asked the FDA in that briefing package if they would be willing to file an NDA based on only one pivotal study. So this question was asked to the psychiatric division which has been the division of the FDA that we are interacting with. And to our knowledge they have not previously given anyone the opportunity to move ahead with just one pivotal study.
They came back to us with a very-very clear answer that you see in bold here first in writing and then repeated at the face-to-face meeting that we have with them saying that the -020 study data together with the supportive data that we have previously put together also [Indiscernible] to support NDA filing for Parkinson’s psychosis. So that meant that, we could stop working on a second pivotal, that we could just focus on getting the remainder of the program done. So that’s say that time so save us money but we still have some additional things that we have to do before we could file. We had a CMC program that we had to put in place we have to prepare regulatory batches we put those batches on stability testing in October last year so we will have the required 12-month stability in October this year and that is what we have on the critical timeline for submission which puts out near the end of 2014 this year for NDA submission.
Parkinson’s disease psychosis is not only interesting because it provides us with a relatively fast entry and quick entry into the neurology market it’s also be believe it perfect entry to provide us with the opportunity to over time broaden into related indications. Initially into Alzheimer’s disease psychosis which we think is three to five times larger market than Parkinson’s disease psychosis eventually into other related neurological indications at Lewy body dementia psychosis et cetera in fact all neurological indications have related psychosis problems. So we think that pimavanserin because of its safety tolerability and efficacy will be an ideal substitute for the off label antipsychotic drugs for these indications. I want to remind you that all antipsychotic drugs today both the typical antipsychotics and the atypical antipsychotic have black box warning for use in elderly demented patients with psychosis and we think this is where pimavanserin can out compete the antipsychotics very strongly.
So Alzheimer’s disease psychosis is the next focus area for pimavanserin, it’s a big indication 25 to 50% of Alzheimer’s patients suffered from psychosis and there are over 5 million such patients in the U.S. and this is of course rapidly increasing number. There is not FDA approved drug for this indication again because they are all the antipsychotic drugs have the black box warning for use in these types of patients. And we think that the increasing symptoms both in Alzheimer’s psychosis and in Parkinson’s psychosis so despite the fact that the pathophysiology is different in Alzheimer’s and Parkinson’s disease we think that we will be very successful to treat the psychosis for both indications. We started an ADP study late last year and we expect that it will take about years from initiation to top line data which would position us near the end of 2015 for data of this study.
The study itself is conducted in the London area where we have the ability to work with the center that is high skilled in the area of clinical trials of Alzheimer’s patient and but there is a large number of nursing homes associated with this center. Those nursing homes are so called research nursing homes, which means that there is lot of data on the patients available, that the caregivers of the nurses are well educated. The close proximity besides also means that we can cover the rating of the patients with the small [indiscernible], we think that’s very important. The key efficacy endpoint here if of course psychosis improvements we measure that with the psychosis part of the MPI the nursing home version but we also look at the number of additional effects of this drug in a broader sense we look at irritability, aggression, daily activities of daily living, cognition, et cetera, et cetera, and sleep.
So we will get the broad kind of understanding from this study that will teach us how to optimally design a pivotal Phase III study. We also see a great opportunities with pimavanserin and schizophrenia, there is a tremendous unmet medical need here as well. The current antipsychotic drugs have very unpleasant side effect profile and we think that pimavanserin can compete highly effectively with these drugs.
We’re planning for another schizophrenia study. We have already shown that pimavanserin when it’s used that’s a co-therapy with low doses of respiridone has similar effectiveness as a high dose of respiridone but much after side effect profile. In the study that we’re planning we will look at the standalone ability of standalone therapy with pimavanserin to improve the maintenance therapy of schizophrenia.
So just to summarize about pimavanserin we have the new chemical entity with a very interesting clinical profile with the unique pharmacology that completed differentiated from the off label use of anti-psychotics and anti-neurology space. We have a great IP with the drug and we believe that it represents very major commercial opportunity.
Few words about ACADIA, we’re still small, a little bit more than 50 people and we’re located in San Diego. We have very strong cash position, we have guided that the cash at hand will take us into 2017. So that means that we have cash that takes us through the first year of launch with pimavanserin and beyond.
So our key priorities for this year is obviously first to complete the NDA and submit it. We are doing a lot of pre-commercial activities because we want to make sure that we have done everything possible to ensure a very successful launch with pimavanserin. At the same time we’re working on the lifecycle management, I told you about the ADP study which we read out in 2015. But we have also started to plan for additional studies for a schizophrenia study, for a sleep study in Parkinson’s patients and for an observational study in Parkinson’s patients. And we are this year determining how to move forward in Europe as well.
So with that I thank you for your attention and we have a breakout session in Carnegie where we can have a further discussion. Thank you.
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