ACADIA Pharmaceuticals Inc.
Q3 2012 Earnings Call Transcript

Published: 6 Nov 2012

[Operator:]

Good day ladies and gentlemen, and welcome to ACADIA Pharmaceuticals’ Q3 2012 Financial Results Conference Call. My name is Keith and I’ll be your operator for today. (Operator instructions.) And I would now like to turn the presentation over to Tom Aasen, Executive Vice President and Chief Financial Officer at ACADIA. Please proceed.

[Tom Aasen:]

Thank you. Good afternoon and welcome to ACADIA’s Q3 2012 conference call. This call is being recorded and an archived copy will be available on our website at www.acadia-pharm.com through November 19th. Joining me on the call today from ACADIA are Dr. Uli Hacksell, our Chief Executive Officer, and Dr. Roger Mills, our Executive Vice President of Development. We will begin the call today with some introductory remarks by Uli and I will briefly comment on our Q3 financial results. After this, Roger and Uli will provide you with an update on our development programs and we will then open the floor to your questions. Before we proceed I would first like to remind you that during our call today we will be making a number of forward-looking statements including statements regarding our and our partners’ research and development programs and plans, including the timing, design and results of clinical trials; the benefits to be derived from and the commercial potential for our product candidates in each case including Pimavanserin; benefits to be derived from changes to clinical trial designs; plans regarding the development of and future commercialization of Pimavanserin; the value of Pimavanserin; and our future expenses, collaboration and grant payments, cash position, stock performance and growth potential. These forward-looking statements are based on current information, assumptions and expectations that are inherently subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in the forward-looking statements. These factors and other risks associated with our business can be found in our filings with the SEC, including our Annual Report on Form 10(k) for the year ended December 31, 2011, and on other filings. You are cautioned not to place undue reliance on these forward-looking statements which are made only as of today’s date. ACADIA disclaims any obligation to update these forward-looking statements. I’ll now turn the call over to Uli, our Chief Executive Officer.

[Uli Hacksell:]

Thank you, Tom, and good afternoon. Let me first take this opportunity to thank all of you for joining us on today’s conference call. As many of you know, this is an exciting time at ACADIA as we await top line results from the pivotal -020 study in our Phase III program for Pimavanserin for Parkinson’s disease psychosis or PDP. During Q3 we completed patient enrollment in the [Phase III] study and I am pleased to report today that we remain on track to report top line results this month. We believe that a successful study should significantly increase the value of Pimavanserin. Currently there is no FDA-approved therapy for PDP. We believe that Pimavanserin has the potential to be the first safe and effective drug that will treat PDP without compromising motor control, thereby significantly improving the quality of life for patients with Parkinson’s disease. PDP is a large unmet medical need and we are focused on advancing our Phase III program [with forward] registration for this indication. As Roger will share with you later, while we are finalizing the -020 study we’re also busy with preparations for the second planned pivotal trial in our PDP program, which we call the -021 study. Beyond PDP we believe that Pimavanserin has broad potential to address a range of other neurological and psychiatric disorders that are poorly served by existing antipsychotics. Importantly we hold worldwide commercialization rights to Pimavanserin. While Pimavanserin provides the foundation to our product pipeline we have several additional programs in our R&D portfolio. Our pipeline also includes two clinical stage programs in the areas of chronic pain and glaucoma in collaboration with Allergan; and two programs in the advanced preclinical stages directed at Parkinson’s disease and other neurological disorders. All of our programs have been generated from internal discoveries and offer what we believe are innovative approaches that may address large potential commercial market opportunities. Overall, our pipeline of product candidates led by our Phase III PDP program with Pimavanserin positions ACADIA with multiple products and commercial opportunities, and with significant growth potential. Before we review our programs in a bit more detail let me ask Tom to briefly comment on our Q3 results.

[Tom Aasen:]

Thank you, Uli. As expected, we reported a smaller net loss for Q3 following the termination of our collaboration with Meiji Seika Pharma and the resulting recognition of all remaining revenue from that agreement. Our revenues totaled $3.5 million for Q3 of which $3.0 million related to the Meiji collaboration with the remainder generated from our ongoing collaborations with Allergan as well as other agreements. This compared to total revenues of $584,000 for Q3 2011. Turning to our operating expenses, our R&D expenses were slightly higher in Q3 relative to the comparable quarter of 2011, reflecting increased Pimavanserin clinical costs while our G&A expenses were consistent quarter-over-quarter. Finally, let’s turn to our cash position and guidance. We closed Q3 2012 with $23.1 million in cash and investment securities. During Q3 we used approximately $5.3 million in cash to fund our operating activities. At the same time we raised $7.0 million in gross proceeds during Q3 from the sale of 3.49 million shares of common stock under our previously established at the market agreement. Following the Q3 raise we have not used the ATM further. We feel this amount of capital raise was ideal to provide ACADIA with additional financial flexibility. These added resources will enable us to accelerate our preparations for the second planned pivotal trial in our Pimavanserin PDP program that Uli mentioned and to extend our cash runway. We now expect that our existing cash and anticipated payments from our collaborations will be sufficient to fund our operations into the second half of 2013. Let me turn the call over to Roger who will provide you an update on our Phase III PDP program with Pimavanserin.

[Roger Mills:]

Thank you, Tom, and good afternoon. As Uli mentioned, this is an especially exciting time for our team as we approach top line results from the -020 study in our Phase III PDP program. -020 was a randomized, multi-center, double-blind placebo-controlled study designed to evaluate the efficacy, tolerability, and safety of Pimavanserin in patients with PDP. The study incorporates several design enhancements that were guided by previous data in our PDP program and designed to mitigate placebo response and reduce data variability. This study has been conducted exclusively in North America. Importantly, this geographic focus enabled us to use a small centralized group of highly-trained independent raters to conduct blinded assessments at a primary endpoint at all study sites. In a two-week period between screening and randomization, patients participated in a brief psychosocial therapy program. This was designed to help patients adapt to a clinical trial setting and to pull initial placebo responses ahead of the baseline assessment. To participate in the study, patients were required to have moderate to severe psychosis as measured by the Neuropsychiatric Inventory Scale or NPI at screening; and the Scale for the Assessment of Positive Screenings, or SAPS, at the time of the baseline assessment. Criteria for study entry were tightened based on our observation in previous studies of a larger placebo response in patients with milder psychotic symptoms. Importantly, the baseline SAPS score was assessed independently from the NPI which provided an important check and balance. We believe the strengthened criteria for enrollment together with the brief psychosocial therapy were effective in filtering out patients with milder psychotic symptoms who we believe are more likely to respond to placebo. A total of 199 patients were randomized in the study on a 1

[Uli Hacksell:]

Thank you, Roger. We are excited about the upcoming -020 study results and believe that a successful trial should provide the opportunity to drive significant value for our stockholders. PDP is a serious disorder that develops in up to 60% of patients with Parkinson’ s disease. It contributes substantially to the burden of Parkinson’s disease and is the major cause of nursing home placements among Parkinson’s patients. We believe Pimavanserin, with its innovative and well-tolerated non-dopaminergic profile has the opportunity to be a first-in-class therapy that will effectively treat psychosis in Parkinson’s patients without compromising motor control. While our strategy currently focuses on advancing our Phase III PDP program towards registration, we also intend to use this program as a foundation to develop and commercialize Pimavanserin for other neurological and psychiatric disorders that are underserved by currently available antipsychotics. One such neurological disorder is Alzheimer’s disease psychosis, or ADP, which affects an estimated 25% to 50% of Alzheimer’s patients. Similar to PDP there is currently no therapy approved to treat ADP in the United States. During Q3, we published results of studies with Pimavanserin in a preclinical model of Alzheimer’s disease which suggests that Pimavanserin may be effective in the treatment of ADP. We believe that these findings, together with the favorable safety profile observed to date in elderly patients with Parkinson’s disease, suggests that Pimavanserin may be ideally suited to address the need for a novel ADP treatment that is safe, effective, and well-tolerated. We believe that Pimavanserin also has considerable commercial potential in schizophrenia. During Q3 data from our earlier Phase II co-therapy trial was published in the journal Schizophrenia Research. This trial demonstrated that co-therapy with Pimavanserin and a sub-therapeutic dose of Risperdal provided an attractive clinical profile – the treatment equally as effective as a higher dose of Risperdal but with a much improved side effect profile and with a faster onset of action. As we continue to advance our Phase III PDP program we will consider potential opportunities that may allow us to both accelerate development in ex-US regions and to broaden the program to drive increased value for our stockholders. Let me now touch briefly on the other programs in our pipeline. First, to our longstanding alliance with Allergan

[Operator:]

(Operator instructions.) Your first question is from the line of Bert Hazlett with ROTH Capital Partners. Please go ahead.

[Bert Hazlett:]

Yes, thank you for taking the questions and we’re all eagerly anticipating the Phase III data release of -020. Just if you could remind us, anyone of you folks I guess, with regard to the tolerability of the agent and what you might expect coming out of -020 based on the prior results or on the long-term extension studies. I’ll leave it there and I’ll follow up in a second.

[Roger Mills:]

Yeah, hi Bert, thanks for the question. Obviously as we will be announcing the data this month we’re not actually covering specifics from the -020 study. But we do have as mentioned earlier on the call, we’ve got fairly extensive experience of using Pimavanserin in a range of patients with PDP across a number of studies; and the tolerability, both in the previous blinded studies, the comparator studies versus placebo – the -012 and -014, and even the earlier Phase II study – the tolerability and safety has been really similar between the active groups and placebo. It’s been very placebo-like in those comparator studies. Obviously the largest amount of safety data that we have generated have been in the long-term open label studies where all patients have received Pimavanserin and we’ve really seen that the drug has been very well-tolerated in that population. And I think it has the potential to very much be an improvement on the atypical agents in this population where there are very severe safety concerns regarding the off-label use of the existing atypicals in these elderly and neurodegenerative conditions.

[Bert Hazlett:]

Thank you very much for that. And again, if we can just fast forward, assuming there is a potential positive outcome – and I know that’s an assumption on my part – in terms of the acceleration of additional indications with the molecule or as a result of potential licensing, can you talk about what a successful outcome might mean in terms of your decision matrix? Would the licensing be a part of the next set of decisions or would you potentially look to accelerate other indications yourself in the absence of licensing? Just a little bit of additional thought assuming successful outcomes here in -020.

[Uli Hacksell:]

Let me take this. First of all, currently we are purely focused on finalizing the -020 study which we believe provides great potential to significantly increase the value of Pimavanserin. Our next step would be to move on quickly with the -021 study and remaining MDA studies to ensure then that we are moving the whole Phase III program towards registration, and so that we can build additional value that way. Now, when it comes to partners we have continued to keep potential partners updated on the progress of the Pimavanserin program. And while we are continuing to move forward with the program we certainly see the opportunity to broaden the specific PDP program into other neurology and psychiatric applications. As you know, we have mentioned Alzheimer’s disease psychosis and schizophrenia as very tempting kind of avenues to explore Pimavanserin. We think those indications provide great opportunities to further expand the value of Pimavanserin. Also, which is very important for us, is to accelerate development in other geographic regions outside of the US; and the most important thing perhaps is that we have worldwide rights for Pimavanserin and that means that all options are open to us.

[Bert Hazlett:]

I guess my question is more along the lines of would you expect to pursue those yourself or would you expect those to pursue either other geographies or indications with partners? Any sense of that at this point?

[Uli Hacksell:]

Well, you know we see the options to do either in the future and we will look at the opportunities and take them [when they appear]. The important thing is that we can move forward with Pimavanserin on our own but we also have opportunities for other scenarios since we have kept the worldwide rights for Pimavanserin.

[Bert Hazlett:]

I appreciate that, thank you very much.

[Operator:]

Your next question is from the line of Brian Lian with SunTrust. Please go ahead.

[Brian Lian:]

Hi, thanks for taking the questions. I have a question on minimizing the placebo response. Can you give us any sort of a historical perspective on how the brief psychosocial therapy has impacted placebo response rates in other trials? Has this been an effective sort of validated approach?

[Roger Mills:]

Yes, it has actually been used. Primarily it came from the Alzheimer’s field where there was a very similar, or remains a very similar challenge with regards to placebo response. It was developed for use in Alzheimer’s patients and therefore it was quite a nice transition to be able to move it through into this similar population with psychosis in PD. And it clearly showed therapeutic utility in that Alzheimer’s population, and it’s really from our advisors who’ve used it effectively in that field that we adopted it for purpose with the -020 study. The aim is essentially to try and draw the placebo response ahead of the randomized portion of the study. You can, in terms of looking at that you may think that you can continue the study out for a long time but that’s very difficult to manage these patients for long periods in a placebo-controlled study, and it wouldn’t be very attractive for patients that come in nor to investigators. By using the brief psychosocial therapy it really offers a couple of things. It offers us the ability to pull that placebo response as I say ahead of the randomized portion; but it also offers patients, and we’re looking for patients with more severe disease – it offers patients, caregivers, and the investigators something to be able to use with patients during the screening period when they’re not in the study and don’t have access to any therapeutic.

[Brian Lian:]

Okay, thanks very much – that’s really helpful. And with the -021 study, can you talk about any key structural differences that might be incorporated in that trial? You talked about a core group of sites that will be used. Will the overall number of sites be the same and will there be any changes planned?

[Roger Mills:]

We’re not giving specifics at the moment but we really do intend that the study will be essentially the same as the -020 study. So we intend the study to be North American-based. We intend to use the same centralized rating that we’ve used in the -020. We’ll continue to use the upfront brief psychosocial therapy so it’ll be very, very similar if not exactly the same to the -020 study.

[Brian Lian:]

Okay, thanks very much.

[Operator:]

Your next question is from the line of Jason Butler with JMP Securities. Please go ahead.

[Jason Butler:]

Hi guys, thanks for taking my questions. The first one

[Roger Mills:]

Yeah, so there are actually three elements to enrollment. There are two structural elements and then there is relying on the investigator to really determine which patients would be ideal for the study. The two structural hurdles to get into the study really are at screening and at baseline. So the screening takes place initially; two weeks later the patient comes in for their baseline visit, and prior to them being randomized they actually have a further assessment. The assessment at screening is the NPI, the Neuropsychiatric Index, and the assessment at the baseline is using the SAPS. Now, the NPI is performed by the investigator; the SAPS is performed by the independent raters. So not only do we have two hurdles but in fact those two assessments are done by different groups, so that provides an important check and balance in terms of bringing patients in. What we did based on the data that we saw from the previous study, the -012 where we saw that patients with milder disease tended to respond to placebo – we actually looked at the NPI and we increased the score for patients to come into the study; and also we did a very similar increase with the SAPS at the baseline. So both at screening and at baseline we increased the hurdle of severity of patients coming into the study.

[Jason Butler:]

Okay great, thanks. And then can you give us an idea of, if any, what additional efficacy information you’ll be able to get from the open label safety study?

[Roger Mills:]

So the open label safety study I think in its definition is based around safety. We don’t have specific measures of efficacy in that study, save the obvious surrogate of patients who remain on the study because the criteria for that is that patients remain on the study in the opinion of their investigator who feels that the patients are deriving benefits from being on the study. But it is primarily a safety study – that’s what we are looking at in the trial.

[Jason Butler:]

Okay, great. Thank you very much.

[Operator:]

Your next question is from the line of Juan Sanchez with Ladenburg Thalmann. Please go ahead.

[Juan Sanchez:]

Good evening guys, how are you? Just a couple of reminders

[Roger Mills:]

Thanks, and just in terms of the first question…

[Uli Hacksell:]

The first one was the dropout rate.

[Roger Mills:]

[We won’t] give specifics on the dropouts here, but what is very interesting is that in terms of patients going through with PDP, traditionally in the psychiatric area dropouts have been very high. In schizophrenia, sometimes over 50% of the patients will drop. In PDP it’s actually the opposite – very few patients drop out of the study. And even if they do drop out, the analysis is able to take that into account unless the patient is completely un-evaluable, so if they have one reading at baseline with no follow up reading. This is an incredibly compliant population. It’s just an elderly population who take their drugs on time, they go through the study and complete. So we don’t actually have, we’ve not seen in previous studies and we don’t expect in this study that there is any form of excessive dropouts, and the power takes all that into account. It’s based on a standard Phase III 90% powering. With respect to the margin of improvement, I think it’s premature to discuss what the point difference will be, and obviously we’re very close to actually being able to look at the data. So I think we’ll look at it in the light of actually having the data in front of us, but I think this study is very well-powered to show a clinical effect of the drug.

[Juan Sanchez:]

Thank you, guys, and good luck.

[Uli Hacksell:]

Thanks so much.

[Operator:]

Your next question is from the line of George Zavoico of MLV & Co. Please go ahead.

[George Zavoico:]

Hi Tom, Uli, and Roger; I’m looking forward to the results later this month as everyone else is. A couple questions

[Roger Mills:]

I think the study, the nice thing about this study in many respects is it’s a very real world study. Given the duration that we are interacting with these patients, there’s a lot of things going on in their lives – people move, there’s all sorts of things which is what you’d expect because, as I say, it’s a very real world study. We have not seen any safety signals in this population which would suggest there is any safety concern, nor any safety reasons that patients would drop out because of safety signals. And as I said, there are many, many events occurring in these patients’ lives during the period on the study but we’ve not seen any signal which causes us concerns.

[George Zavoico:]

And then on the -015 study, there’s no end to it? I mean there’s no ten-year limit or seven-year limit? It’s just going on until patients decide to stop?

[Uli Hacksell:]

Until we are on the market.

[George Zavoico:]

Until you’re on the market, of course! [laughing] Great. A question about the Nurr-1 and ER Beta programs

[Uli Hacksell:]

So let me [preface] that. What I tried to say earlier on, that was the (inaudible) in trying to bring one of these drugs into development next year – I did not say that it would be [moving into the clinical] necessarily. So but currently these drugs are essentially completely supported by (inaudible) grants, something that we’re very proud of because we have been able to compete with academic and other commercial applications for money and done so very successfully. We cannot really speculate on how grants may be possibly able to cover clinical expenses. We will see in the future. We do not… It’s too early to speculate on that simply I think. One thing is certainly on that, it is more difficult to get grant support for clinical studies than for preclinical studies.

[George Zavoico:]

Yeah, that’s what I was wondering about, that’s what I thought. Okay, we’ll look forward to some of the preclinical data coming out hopefully that you’ve worked on with a grant. I imagine those are going to be submitted to the usual medical or research conferences sometime in the next year or two?

[Uli Hacksell:]

Yes, absolutely. In fact we do intend to present posters. We have done that before as well and we will most likely have applications coming out as well.

[George Zavoico:]

Thank you very much.

[Uli Hacksell:]

Thank you.

[Operator:]

(Operator instructions.) And your next question is from the line of Alan Carr with Needham. Please go ahead.

[Alan Carr:]

Hi, thanks for taking my questions. I was wondering if you can give us a couple things

[Uli Hacksell:]

You’re up, Roger.

[Roger Mills:]

Thanks, Uli. So Alan, I think as we’ve, on previous calls we’ve indicated that the baseline scores have been really demonstrative, and I think we’ve seen that the structural design that we put into the -020 study has resulted in patients with milder disease being screened out of the study. And in the complete enrollment of the study I think that has been consistent, so we’ve not seen any changes in the later stages of the study. So really the design changes that we made really do seem to have been effective in screening out patients with milder symptoms, and of course that’s important because those are the ones who were most likely to respond to placebo in the previous study.

[Uli Hacksell:]

So when it comes to the Allergan collaboration, I can take that. As you know, we have two clinical stage programs in collaboration with Allergan and one discovery collaboration. When it comes to the Adrenergic pain program, Allergan has conducted several Phase II studies and have communicated previously and they are still doing that, that they are seeking a partner for further development. That (inaudible), they look to find a partner that can also help them to commercialize such a product in an area that is predominantly served by general practitioners. They have indicated also that this particular strategy relates to their focus on specialty pharmaceutical markets. The second program, which is the Muscarinic glaucoma program, that is in Phase I clinical development; and then the third program as I indicated previously, that’s a discovery collaboration which is focused on ophthalmological applications. And that’s what we can say today about the Allergan collaboration.

[Alan Carr:]

Alright, thanks very much.

[Tom Aasen:]

Thanks.

[Operator:]

And ladies and gentlemen, we have no other questions at this time. Dr. Hacksell, please proceed with your closing remarks.

[Uli Hacksell:]

So thanks again to everyone for joining us on today’s call and for your continued support. We look forward to updating you in the near future. Thank you.

[Operator:]

Thank you for your participation in today’s conference call. This concludes the presentation and you may now disconnect. Have a good day.