Good day, ladies and gentlemen, and welcome to the ACADIA Pharmaceuticals’ Second Quarter 2014, Financial Results Conference Call. My name is Lacy, and I will be your coordinator for today. (Operator Instructions).
I would now like to turn the presentation over to Lisa Barthelemy, Director of Investor Relations at ACADIA. Please proceed.
Good afternoon, and welcome to ACADIA’s second quarter financial results conference call. This call is being recorded and an archived copy will be available on our website at www.acadia-pharm.com through August 19, 2014.
Joining me on the call today from ACADIA are Dr. Uli Hacksell, our Chief Executive Officer; Steve Davis, Executive Vice President, Chief Financial Officer and Chief Business Officer, Dr. Roger Mills, our Executive Vice President of Development and Chief Medical Officer; and Terry Moore, our Executive Vice President and Chief Commercial Officer.
We will begin our call today with some introductory remarks, and brief comments regarding our recently announced financial results. Following this, we will provide you with an update on our development programs and business, 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 research and development programs and plans and our strategy, including the timing, results or implications of clinical trials or manufacturing development; the benefits to be derived from, future approval of and the commercial potential for product candidates in each case, including pimavanserin, the timing, content or likelihood of regulatory meetings, filings or approvals, future development and commercialization of pimavanserin, the expansion of the pimavanserin into additional indication and our future expenses, cash position and usage and growth potential.
During our call today, we may use words such as anticipate, believe, could, expect, intend, may, plan, potential, predict, project, should or the negative of those terms and similar expressions that convey future events or outcomes to identify these forward-looking statements.
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 made with the SEC, including our annual report on Form 10-K for the year ended December 31, 2013, and other filings including a report on Form 10-Q filed earlier today. 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 Hacksell, our Chief Executive Officer.
Thank you, Lisa, good afternoon. Let me first take this opportunity to thank all of you for joining us on today’s conference call. The first half of 2014 was a productive and exciting period for Acadia. We continued to make important progress in advancing our lead program with pimavanserin for Parkinson’s disease psychosis or PDP toward penetration.
Currently no drug is approved in the United States to treat PDP, debilitating this order and that can have devastating effects on patients as well as their careers and families.
Pimavanserin offers some innovative non- dopaminergic therapy and has the potential to be the first safe and effective medicine to treat PDP without compromising more to control.
During the second quarter, we completed our pre-NDA meetings with the FDA and we’re pleased by the positive introductions with the agency. Our focus remains on completing NDA enabling activities for pimavanserin. And I’m delighted to report that we remain on track for a planned submission of a new drug application or NDA near the end of this year.
On the commercial front, Terry and his highly experienced team have been working diligently on building the commercial infrastructure necessary to support the planned launch of pimavanserin for PDP in the United States.
In parallel with the advancement of our PDP program in the United States, we also have our planned path to registration for pimavanserin in Europe. Following informative interactions with regulatory agencies for multiple EU member States are being consultation with our European regulatory advisors. I’m pleased to report that we plan on submitting that case into EMEA next year, around six to nine months following NDA submission.
In addition to PDP, we believe pimavanserin has broad application and may transform the treatment of psychosis in a range of other neurological and neuropsychiatry disorders that are poly served by existing psychotic drugs.
Given pimavanserin demonstrated a psychotic effect benefits on sleep and favorable safety profile that we have observed in clinical trials to date. We’re excited about the opportunity to evaluate pimavanserin in other indications.
To that then we have continued advance enrollment in our official study of pimavanserin in Alzheimer’s psychosis or ADP. As is the case with PDP, no drug has been improved in the United States to treat ADP. And the off-label use of existing antipsychotics is linked to increased mortality, serious adverse events and cognitive decline in patients with dementia related psychosis.
In addition to our ongoing ADP study, we’re planning other studies in our lifecycle management program to further characterize potential aspects of pimavanserin’s clinical profile and to position it for other indications.
During the first half of this year, we also successfully completed a public offering and to strengthen our financial position. Importantly, we’re well capitalized to be able to experiment the breadth of – to build additional value in our pimavanserin franchise and invest in our commercial activities.
We have also continued to bring in accomplished professionals to Acadia and most recently announced the appointment of Steve Davis, as our Executive Vice President, Chief Financial Officer and Chief Business Officer. It’s my pleasure to introduce Steve on the call today.
Steve is a highly accomplished industry executive with extensive financial and operational experience and a proven track record building and driven confidence. We are delighted to have Steve on board and look forward to his contributions as we pursue our strategy of building a leading U.S. specialty neurology franchise using pimavanserin as the foundation.
On that note, I’ll now pass the call over to Steve, who will comment on our second quarter results.
Thank you very much Uli. Let me first start off by saying that I’m delighted to be a part of the Acadia team. I’ve had to pleasure to interact with many of you in other venues and I’m happy to reconnect here.
It is indeed a very exciting time to be joining the company. Our second quarter financial results continue to reflect the strategy we described previously. That is simply to increase our R&D investment in order to aggressively advance and build value in our pimavanserin franchise.
Our R&D expenses increased to $13.8 million for the second quarter from $7.1 million for the comparable quarter of 2013, this is due primarily to increase costs associated with NDA-enabling activities in our pimavanserin development program, R&D expenses for the just completed quarter also include $1.1 million in stock-based compensation expense.
G&A expenses increased to $8 million for the second quarter from $2.5 million for the comparable quarter of 2013, this reflects our continued investment in commercial pre-launch activities. I should point out the G&A expenses for the just completed quarter includes – compensation expense.
Finally turning to our cash position. In March, we raised net proceeds of approximately $197 million which significantly bolstered our financial position. We continue to maintain a strong cash position ending the second quarter with $354.5 million in cash and investment securities. We used approximately $14.8 million in cash during the second quarter to fund our operating activities.
In future periods, we expect our cash used in operations to increase as we continue to advance our PDP program to an NDA conduct commercial activities and execute on our pimavanserin livestock management program.
Importantly we have a strong cash run rate that really positions us well in two very important fronts. It enables us to expand and build additional value in our pimavanserin franchise through work we’re doing as Uli mentioned in ADP and planning and sleeping schizophrenia.
And it also equips us to find important commercial activities. These include pre-launch activities designed to optimize positioning for the plan PDP launch as well as subsequent sales and marketing efforts through and beyond the projected timeframe of our market launch.
As previously guided, we currently anticipate that our current plan, our cash resources will be sufficient to fund our operations, at least in the 2017.
With that I’ve concluded my remarks. Now turning the call over to Roger who will take you through an update on our pimavanserin program
Thank you, Steve and good afternoon. As mentioned, we continue to make important strides in advancing our PDA program towards registration. During the second quarter, we held routine pre-NDA meetings with FDA. These interactions are positive and enable us to outline and discuss our planned submissions and how the NDA would be organized.
Based on these positive interactions, we remain on track to the planned NDA submission to the end of 2014.
Importantly, our team has remained focused on completing the remaining activities in our pimavanserin PDP development program that are needed for the submission of our NDA.
This includes aspects of the CMC development including stability testing of the pimavanserin registration batches and supported studies including drug, drug interactions studies.
I’m pleased to report that our CMC program has continued to advance as planned. You may recall that our three require and registration batches of pimavanserin, the successfully manufactured that the commercial launch scale. And registration stability testing on these drug product batches was initiated last October.
During the second quarter we completed an assessment on the first six months stability from these registration blocks. And importantly, confirmed that these data consistent with historical data observed with our clinical trial formulation.
We are continuing to accumulate data in our registration stability program which is designed to comply with the ICA guidelines of 12-month stability, regulatory batches and to meet the regulatory filing requirements for major markets world-wide.
In addition, we continue to make very good progress with supported studies which include drug-drug interaction or DDI studies. The profile of pimavanserin appears consistent with what we’ve observed to date in our long-term PDP safety extension studies.
Our Phase III PDP open label safety extension trial referred to the 015 study, it is designed to continue until pimavanserin is commercially available. This ongoing study has allowed us to generate a large amount of valuable long-term safety data regarding the use of pimavanserin in patients with PDP.
We have already far exceeded the ICH guidelines for a quite one-year exposures with well over 250 patients have been treated for one year or longer. In fact, through our 015 study and the similar extensions tied to our earlier phase II trial, we have well over 100 patients who have been treated with pimavanserin for at least two years, and our longest single-patient exposure exceeds 8 years.
Our long-term safety data provides support for the potential of pimavanserin to offer significant advantages relative to current anti-psychotic frequent use off-label to the treatment of PDP.
Whilst our main priority is our plan then the A-submissions with the FDA, we have also outlined the path to registration in Europe. As Uli mentioned, we plan to submit our application to the EMEA next year around 6 to 9 months following the submission of the NDA.
As you may recall, we had been completed a series of interactions with regulatory agencies for multiple EU member states. These initial interactions are very informative we believe it is clear that our pivotal Phase III, (inaudible) study was seen as a strong study.
In addition, as is the case in the United States, there are concerns surrounding the current use of a typical in this patient population.
Following consultation with our European regulatory advisors and as we’ve completed our pivotal clinical program, we have decided not to apply for scientific advisory to the EMEA. We believe the optimal and most efficient path forward in the EU is to submit a marketing application to the EMEA.
Let me now turn to our life-cycle management programs of Pimavanserin, an Alzheimer’s disease psychosis or ADP. As you may recall, we initiate to the Phase II trial for ADP late last year.
We continued to advance enrollment in this randomized double-blind placebo controlled trial, referred to the 019 study, which is designed examine the efficacy and safety of pimavanserin in about 200 patients with ADP.
We are excited about the design of the study, which believe provides an excellent opportunity to explore the potential benefits of Pimavanserin in treating with psychosis a well as it relates to behavioral disturbances associated with Alzheimer’s disease. Importantly, we have incorporated many study design concepts for our successful, pivotal PDP trial into the 019 ADP study design.
Beyond ADP, we are planning additional studies on our lifecycle management program designed to further characterize and highlight aspects of pimavanserin’s clinical profile in other neurological and neuropsychiatric indication areas.
One area of keen interest for us is sleep disturbance, which represents a frequent, a major problem for patients with neurological disorders, including Parkinson’s and Alzheimer’s diseases.
Benefits from sleep in this population are of particular importance because sleep disturbances in PD can exacerbate psychosis worsen cognition and lead to more falls and increased care-giver burden.
Poor night time sleep quality, and excessive day time sleeping are associated to be the lower quality of line PB subjects and the caregivers. We have observed significant non-sedating sleep related benefits that pimavanserin in clinical studies, including our pivotal phase III O2O PDP study.
In the O2O study, pimavanserin demonstrated significant improvement in night time sleep. And this improvement was not accompanied by any sedation or hang-over effect. Pimavanserin also produced a significant improvement in day-time wakefulness in PDP patients.
As you may recall, the positive effects of pimavanserin are night time sleep and daytime wakefulness, did not correlate with psychosis measures thus indicating the sleep and wakefulness improvements of pimavanserin may represent independent treatment benefits.
We believe that an additional study in the sleep area may be allows to further, characterize and highlight these potentially important clinical benefits.
Another area that represents a large unmet medical need and a tremendous commercial opportunity for pimavanserin is schizophrenia. Today compliance is a major issue in patients with schizophrenia. In fact 74% of patients discontinued the anti-psychotic medications because of lack of efficacy or in total side-effects according to our Landmark government study, financed by the national institute of mental health.
Furthermore, the consequences of poor compliances of schizophrenia can be severe, lead to hospitalization and add to the high cost of treating the patient. We’re very excited about the potential of pimavanserin to be a mono-therapy in the maintenance phase of schizophrenia.
In this application, we believe that pimavanserin selected 5x-T2A blockade could allow for effective symptom control whilst avoiding interactions with dopamine and other receptors that are linked to many of the side-effects caused by existing antipsychotics.
We believe a noble, well tolerated maintenance therapy could result in better compliance of patients with schizophrenia and are planning a study in this area.
I’ll now turn the call back over to Uli.
Thank you, Roger. We continue to be excited about the prospect of pimavanserin to be the first drug approved in the United States to treat PDP.
PDP represents what we believe is an ideal lead indication of specialty market opportunity for pimavanserin. Our strategy is to commercialize pimavanserin for this indication in the United States, by establishing a specialty sales force focused primarily on neurologists and small group of high prescribing psychiatrist’s long-term care decisions.
During the second quarter our commercial team continued to conduct pre-commercial activities and make progress in recurring our supply chain distribution, preparing the product in terms of optimizing product positioning and preparing the organization in terms of structure, size and timing.
These ongoing efforts are deciding to ensure that the robust commercialization process and optimal strategy are in place to position Acadia for success in the planned U.S. market launch.
Our most recent market research efforts have focused on our understanding how pharmacy and medical director decision makers in managed care settings, view care available treatments for PDP.
Our findings indicate that payers recognize that there are no treatment options on the market with an approved indication to treat PDP and that’s safe and effective treatment of PDP is a significant under commitment to need and its’ value driver for a product treating PDP.
As was found in our market with physicians, payers also indicated that the ability to treat the PDP symptoms without impacting multi-control is one of the most important attributes in a drug for PDP.
As we have said before, because no drugs are approved to treat PDP in the United States, today the decision is frequently resort to off-label use of existing and psychotic drugs, even though these drugs carry a black-box warning for using elderly patient with dementia-related psychosis, to increased mortality and morbidity.
Many physicians have indicated that they will consider initiating treatment with current therapies when the patient symptoms become disruptive to the caregiver and/or to the patient’s family.
In addition, physicians face a dilemma because current antipsychotics conversion multi-symptoms in PDP by counteracting the customary dopamine replacement therapy for the multi-symptoms in Parkinson’s disease.
Because of this innovative non-dopaminergic 2HT2-A inverse agonist mechanist more of action, in advancement avoids this dopamine dilemma and has the potential to be the first safe and effective medicine to treat PDP without compromising motor control thereby significantly improving the quality of life per patient with Parkinson’s disease.
Consequently, with pimavanserin, physicians could have a treatment option that could be initiated at the onset of PDP symptoms without to say concerns, an off-label use of currency there available C-typical and psychotics.
The more we learn about various stakeholders in view PDP and its unmet needs, the more excited we are regarding the potential for pimavanserin to improve the lives of people impacted by their disease. We believe that if approved, pimavanserin has the profile to become the gold standard in the management paradigm for PDP.
Let me now touch briefly on several other programs we have in our R&D portfolio behind pimavanserin. Together with Allergan we have clinical stage programs in the areas of chronic pain and glaucoma. Allergan is also pursuing preclinical development of an additional compound which was advanced last year from our collaborative research as potential new treatment for glaucoma.
In addition we have two advanced un-partnered preclinical programs, our ER-beta and Nurr1 programs which may another approach as to treating Parkinson’s disease and other neurological diseases.
Pre-clinical studies in our ER-beta program has a novel pain treatment are supported by ground from the National Institute on neurological disorders has started. And we are also conducting pre-clinical studies with our ER-beta compound as an innovative approach to targeting neuro-degeneration associated with multiple-sclerosis.
Preclinical studies in our Nurr1 program directed at Parkinson’s disease had been supported by grant from the Michael J Fox Foundation.
Finally, before I close, I would like to add that the excitement here at Acadia, it’s palpable. We are growing as a company and establishing the foundation to transform Acadia into successful development and commercial organization. Our priorities remain tiered. We’re focused on our plan submission of our NDA for pimavanserin in PDP, nearly under this year.
They are conducting commercial activities in preparation for a planned successful launch in the U.S., and we are executing on our life-cycle management of pimavanserin in other areas of significant unmet medical need.
We believe that our pipeline of product candidates led by our Phase III pimavanserin program, positions Acadia with multiple attractive commercial opportunities and significant growth potential.
And most importantly, all of us at Acadia remain committed to bringing innovative medicines like pimavanserin to the market to improve the lives of patients, the neurological related central nervous system disorders.
Operator, you may now proceed with the Q&A session.
Thank you. (Operator Instructions). Our first question comes from the line of Cory Kasimov with JPMorgan. Please proceed.
Whitney – JPMorgan
Hi guys, this is actually Whitney on for Cory, apologies if I missed this but can you just review the rationale or not applying for scientific advice and EMEA?
Hi Whitney, this is roger. Yes, we’ve actually worked extensively with a leading group of regulatory advisors in Europe. These are ex-leading regulators from major countries in the EU.
And under their guidance they advised that really for central advice, it is designed for an area program and it is not the roll of active guide on the approvability of an MAA package, we’ve reviewed the package or the program that we’ve done to date.
As you said with various individual member states as well as having a critical review by these regulatory exports of the program and based on that we feel that the appropriate way forward is to go directly with the MAA.
Whitney – JPMorgan
Got it. And then, my second question was in – you planned in sleep you mentioned you are designing a trial there. Can you give any details on that trail or say whether or not it’s in PDP or ADP or you’re sort of exploring something else?
The actual first study that we would look at in fleet, we haven’t – with four relating of crystallizing our ideas now we would intend it being in PD patients.
Whitney – JPMorgan
Got it. Thanks for taking the questions.
Your next question comes from the line of Alan Carr with Needham & Company. Please proceed.
Alan Carr – Needham & Company
Hi, thanks for taking my questions. I guess to follow-up on the previous one can you give us any sense on timing for either the schizophrenia or sleep disturbance trials whether or not those might happen late this year or 2015 events?
And also with respect to getting events for the NDA submission in the U.S. are you done with all of the necessary clinical trials, the support of clinical trials? Thanks.
Hi Alan, so just in terms of the timing. Schizophrenia study will clearly be next year in terms of start. We are really looking at how the design of that study would best be achieved, the particular patient population that we would want to access and the duration of that study.
So, the real meat of the study design and when we pull that together and we’ll see expert advice through the process we’d be able to get a better picture as to when – not just when we’re going to start but to – that you have patient population that we are looking at.
In terms of sleep, that will be – it should be able to get a study in the sleep area started earlier whether it would be at the end of this year or early next year, we haven’t finalized yet.
And then, I think your third question was regarding the – where we are in the NDA process, yes. So, we’re actually well underway in terms of pulling everything obviously, the program involves the – on the supported studies it includes large amount of pre-clinical work looking at enzymes.
And then this clinical work and then obviously importantly it’s pulling to get the reports. And we’re well underway in getting the reports from those – from that program together and putting it in the format for the NDA.
Alan Carr – Needham & Company
So, the only remaining gaining event is finishing up the one-year with the stability testing?
That has been the case, sort of the key event through this program. So, and obviously 12-month stability, it takes 12 months.
Alan Carr – Needham & Company
Great. Okay. Thanks for taking my questions.
Our next question comes from the line of Jason Butler with JMP Securities. Please proceed.
Jason Butler – JMP Securities
Hi, guys thanks for taking the questions, just a follow-up on the European regulatory strategy. Have you actually had any conversations with specifically with members of CHMP that gives you the – gives you confidence to the single-trial specifically as going to be sufficient for approval?
I think as mentioned, we’ve not actually spoken to the CHMP individuals but obviously the key thing is that the experts that we’ve used they’ve been the very leading people until recently in the agency. Within that committee and we sought advice from them. And it takes another advice that we are moving along the path that we are.
Perhaps I can add to what you said Roger that, we believe that we have a strong data package. And our intention is obviously to use the same data package that is submitted to the FDA also for the MAA applications.
Our opinion is that this data package should be good enough for (inaudible) but we still need to have the discussions with MAA. And we thought we’d learn over time if they agree with us, if they don’t we will argue because we think that we have very strong arguments why the current data package should be sufficient.
I think it’s important to remember that the challenges facing patients and doctors in the United States are just the same as those facing doctors and patients in the EU. And the concerns obviously are the same both sides.
Jason Butler – JMP Securities
Okay. And then, can you just walk us through the next steps and the European MAA process is the first interaction you’ll have with MAA and CHMP submission of the file or are there any pre-submission discussions that you’ll be able to have?
In terms of process, we actually notified them that we are intending to submit the MAA and some procedural things that follow that. But the key thing would be the submission of MAA.
Jason Butler – JMP Securities
Okay, great. Thanks for taking the questions.
(Operator Instructions). And our next question comes from the line of Burt Hazlett with Ladenburg. Please proceed.
Burt Hazlett – Ladenburg
Thank you. I’ve got a follow-up or two again on EUROPE. So, just to be a little more – to drill down a little bit more specifically, what are the gating items then for the particular submission, I mean if it’s roughly the same package? Is the interaction, the gating item is the additional data to be generated, that’s the gating item, could you just describe in a little bit more granularity what is required that’s different between the two jurisdictions?
So, our gating item is the NDA itself, that’s what we’re focused on. We want to put a successful submission into the agency and make sure when that goes in that we are fully focused on supporting that to ensure smooth process through the NDA review. There are – the essence, the scientific essence of the NDA is exactly the same for the MAA but there are obviously European styles of slightly different documents that go into it. They’re not significantly different from the NDA, obviously so ICH compliant.
So, clearly, I think, as I say, we want to focus on the NDA make that successful and then be able to focus on the MAA to make sure that that too is successful.
Burt Hazlett – Ladenburg
Okay. Thank you for the color. And then, in terms of your commercialization strategy in the EU, how do you think about potential partners interaction with potential partners if you are considering that NM, lastly could you describe, are you prepared to share any of the data at all with regards to drug interaction studies or the work you’ve done there at this point?
So, just let me take it to our commercial strategy. We have been very clear that we want to build U.S. specialty neurology and franchise into U.S. from that cabinet intention of offering pimavanserin therefore in the U.S.
In the rest of the world, we have all the options of open because we have worldwide rise to pimavanserin. What we’re doing currently is we are building additional value in pimavanserin to various actions through the NDA that we will submit the approval through the API program – sorry the Alzheimer’s disease psychosis program, this additional program that we will move forward with pimavanserin.
So we’re not in a rush to partnering the rest of the world. Obviously we can move forward and achieve the registration without a partner. And we can even commercialize pimavanserin in the U.S., and Europe we have to partner. But coming back, we will come back to this at a later stage. We’re not in a rush currently.
Burt Hazlett – Ladenburg
Okay. And then, any of the drug interaction data, is there any data that you might be able to share with us at this particular point with regard to those studies?
We’re not giving specifics on that program right now. But we’ve not seen anything that causes of concern today.
Burt Hazlett – Ladenburg
Okay, thank you very much.
Sir, we have no questions at this time. Dr. Hacksell, please proceed to closing remarks.
So, thanks again to everyone for joining us on today’s call and for your continued support. We look forward talking to you in the future on our ongoing progress. Thank you so much.
Thank you for your participation in today’s conference. This concludes your presentation. You may all disconnect. Good day everyone.
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