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Arcturus Therapeutics Holdings Inc. (ARCT)

Q1 2014 Earnings Call· Thu, May 15, 2014

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Transcript

Operator

Operator

Good day, ladies and gentlemen and welcome to the Alcobra First Quarter 2014 Results Call. At this time, all participants are in a listen-only mode. Later we will conduct a question-and-answer session and instructions will be given at that time. (Operator Instructions) As a reminder, this conference call is being recorded. I’d now like to turn the call over to Michael Wood of LifeSci Advisors. Sir, you may begin.

Michael Wood

Management

Good morning and thank you. Before the market opened this morning, Alcobra announced financial results for the first quarter of 2014. If you've not yet received this news release or if you would like to be added to the company's distribution list, please call LifeSci Advisors in New York at 646-597-6979 and opt to speak with Veronica Molina. Before turning the call over to management, I’d like to make the following remarks concerning forward-looking statements. The conference call contains certain forward-looking statements within the meaning of the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Because such statements deal with future events and are based on Alcobra’s current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Alcobra could differ materially from those described and/or implied by the statements on this conference call. For example, forward-looking statements include statements that Alcobra is expecting to receive multiple data readouts, timing of receipt of such data readouts, statements that imply that will receive favorable results in clinical trials of MDX, and timing of completion of clinical trials and receipt of results. Statements regarding the design, timing initiation, and successful completion of enrollments in the company’s Phase 2 pediatric clinical trials, and Phase 2 Fragile X programs, if such trials or other trials are commenced at all. Statements regarding the sufficiency of the company’s financial resources to be at certain milestones and whether such milestones may be achieved at all. In addition, historical results or conclusions from scientific research do not guarantee that the results – future results would not suggest different conclusions or that historical results referred to on this call could be interpreted differently in light of additional research. The forward-looking statements contained or implied on this call are subject to other risks and uncertainties, including those described under the headline Risk Factors in Alcobra Ltd.’s Annual Report on Form 20-F for the fiscal year ended December 31, 2013 filed with the Securities Exchange Commission and in subsequent filings with the SEC. Except as otherwise required by law, Alcobra disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as of the result of new information, future events or circumstances or otherwise. At this time, it is now my pleasure to turn the call over to Dr. Yaron Daniely, President and Chief Executive Officer of Alcobra. Yaron, please go ahead.

Yaron Daniely

Management

Thank you, Michael. And to those of you who are on our call live thank you for joining us. Today I’ll review the status of our lead drug candidate, Metadoxine extended-release or MDX including an update on our Phase 3 trial. I’ll also provide an update on other clinical milestones and highlights on important recent and upcoming corporate events. Before I get to this report though let me introduce our new CFO, Dr. Tomer Berkovitz, who is sitting with me in the room. Tomer joined our team earlier this month and he is on our call today to provide Alcobra’s Q1 2014 financial report. By means of background Tomer previously was an Executive Director in JPMorgan’s New York investment banking division where he gained significant experience advising Senior Management and Board of Directors of numerous companies in the S&P 500. In this capacity, Tomer played a leading role in several capital market and M&A transitions in the Health Care sector. He also served in Citi Group’s investment banking division and prior to that served in the Israel Defense Forces as an Economist in the Financial Advisory Unit to the Chief of Staff. Turning to our lead drug candidate MDX, we were pleased in March that following FDA acceptance of our IND for MDX. We began enrolling patients in a Phase 3 clinical trial in the treatment of adults with ADHD. Our plan is to complete the study in the early second half of 2014 with a goal of reporting top-line results in the third quarter of the year as we previously indicated. With two placebo-controlled Phase 2 study behind us both of which show statistically significant changes from baseline in FDA approved and validated clinical endpoints. This Phase 3 study is designed to confirm MDX as a fast-acting highly effective…

Tomer Berkovitz

Management

Thank you, Yaron. And thank you to our shareholders for joining us. First of all on a personal note I would like say that I am delighted to join the Alcobra team and looking forward to meeting many of you soon. Now turning to our financials, total operating expenses for the first quarter of 2014 were $7.8 million of which $1.3 million was non-cash charges for stock-based compensation. This is compared to total operating expenses of $5.6 million in the previous quarter of which $0.5 million was stock based compensation. Research and development expenses were $5.5 million for the first quarter of 2014 compared to $4.6 million in the previous quarter. As we had indicated in the past R&D expenses have increased due to our aggressive efforts on executing and achieving our clinical development milestones. This quarter we also a separate pre-commercialization expenses in the financials, which were $0.5 million for the first quarter of 2014. These expenses are associated with our accelerated efforts related to market research and analysis as well as business development. This activity is led by our Chief Commercial Officer, David Baker, who joined Alcobra in the first quarter after 10 years at Shire, most recently the Vice President of Commercial Strategy and New Business in its Neuroscience Business Units. Finally our liquidity position as of March 31 was $44.4 million, which includes $10.4 million in cash and cash equivalents as well as $34 million in short-term deposits. This is compared to total liquidity of $50.1 million as of the end of 2013. I’ll now turn the call back to the operator for Q&A. I’ll now turn the call back to the operator for Q&A.

Operator

Operator

(Operator Instructions) Our first question comes from Annabel Samimy of Stifel. Your line is open. Annabel E. Samimy – Stifel, Nicolaus & Co., Inc.: Hi, thanks for taking my call. Just three questions with enrollment on Phase right now that either you are seeing or you able to gather whether the mix efficiency you are getting – is exactly which you expected in terms of inattentive versus the mix ADHD, and I’ve got some follow-up questions after that. Thanks.

Yaron Daniely

Management

Annabel E. Samimy – Stifel, Nicolaus & Co., Inc.: Okay, great. And then with the Fragile X study, we notice that obviously Novartis have dropped their study, as FDA learn anything new from some of the failures of other programs and the clinical protocol that you’ve submitted to them is it as you expect or there any changes based on some of the new knowledge that they’ve gained.

Yaron Daniely

Management

Well, I’d say that there are several distinct differences between the development programs previously from Seaside, and Roche, and Novartis, and Fragile X and Alcobra development plan and they do reflect some differences in the protocol that we’ve submitted. It’s important to note at least two key differences. Number one the target, the Novartis and Seaside, Roche compounds are all mGluR5 targeted this is based on an mGluR5 hypothesis involvement in Fragile X. As you know our compound although is also related to the GABA glutamate circuit, it is not an mGluR5 directed agent. And so just starting off on a very fundamental pharmacological level this is a different target. The second it has to do with clinical trial design, so the Roche, Seaside, Novartis compounds were developed specifically for Fragile X. And so the endpoint used in these various Phase 2 and Phase 3 studies where very general and broad endpoint attempting to cover a very wide range of behavioral and clinical and cognitive endpoints in Fragile X. We are coming in with I think what would have be an advantage in understanding a much better what our drug is capable of doing. And so in our clinical trial protocol we are focused particularly on the primary endpoint, but also in the selection of secondary endpoint on the cognitive deficits and the potential cognitive benefits in Fragile X. So we are not going to be focusing as a primary measure on general behavioral outcomes. We really believe that the best chance to success it capture a cognitive benefit in this population. The same way that we have seen the benefit come through in the ADHD trial and the pre-clinical models that we have done today. And we are very delighted that the FDA has cleared the protocol with its selection of primary and secondary endpoints. Annabel E. Samimy – Stifel, Nicolaus & Co., Inc.: Okay, great. And just on the financial side you had mentioned the cash is positioned for completion of clinical trials through this year and through next year, does that include additional Phase 3 trials for pediatric Fragile X, the second Phase 3 or is that going to be essentially difficult for all that?

Tomer Berkovitz

Management

Yes Annabel this is Tomer. So basically the cash we have right now is sufficient to fund all three programs, the two programs in ADHD for both the adult and pediatric and Fragile X basically to completion. Annabel E. Samimy – Stifel, Nicolaus & Co., Inc.: Okay, great. Thank you.

Operator

Operator

Thank you. Our next question comes from Jason Butler of JMP Securities. Your line is open. Jason N. Butler – JMP Securities LLC: Hey, thanks for taking the question. First one on the pediatric population, can you give us any more information on the efficacy assessments that you’ll be conducting in the first Phase 2 trial whether those any statistical power for those assessments or if you are really just looking for trends at this point?

Tomer Berkovitz

Management

Thanks, Jason. The Phase 2 trials follow the classical Phase 2 ADHD pediatric power design, it looks as a primary endpoint only on safety, tolerability, PK issues and really doesn’t evaluate as a primary endpoint in any efficacy outcomes. There are plenty of efficacy outcomes measured at secondary endpoint, but the power end of the study is really to evaluate the tolerability and the dose using in pediatric population. This is what the agency expects as a first in pediatric study, and this is what we complied with. I think it’s important to note that there is never been a compound that for ADHD that was effective in adults and ineffective in K. So our expectation is that there is really not going to be any efficacy issues in evaluating this product in children and the pediatric population, in fact that the other way around in pediatric populations the magnitude of the effect of all ADHD drugs today has been significantly higher then drug then their effect sizes seen in adult trial. So if anything, I expect the efficacy measures to be even more robust and even more significant in the pediatric trials than in the adult trials. But keeping inline with the conservative strategic regulatory pathway that Alcobra has taken with the agency have thus proven to be successful. We have designed our Phase 2 inline with the expectation of really evaluating PK, tolerability, and safety on primary and just collecting data on efficacy of secondary endpoints. Jason N. Butler – JMP Securities LLC:

Yaron Daniely

Management

Sure, so first I’d ask that you know that the poster that was presented at the SOBP meeting as well as the poster that was presented earlier at the same week at the APA meeting are both available for download on our website. And I hope people feel free to go in and download and look at that more intensely. There was a lot of data presented at SOBP on the pharmacological function of Metadoxine or MDX as well as some neurophysiological study is looking at conduction of electricity in nerve cells in brain, tissue and there really a wide array of data presented there on the GABA glutamate system modulation. We know that the GABA glutamate circuit, which is the kind of the accelerator and break circuit in the brain is closely linked to cognitive function. There are many cognitive impairment, schizophrenia, autism, ADHD just a name of few that have been linked to a dysregulated GABA glutamate balance were normally you have high levels of glutamate, the excitatory neurotransmitter and low levels of GABA, the inhibitory neurotransmitter. What we’re able to show both in vivo and ex-vivo, and in vitro is that treatment with Metadoxine in a very dose response manner increases the amount of GABA signaling and internal cause of the decrease or a better control over the glutamate excitatory signaling restoring a balance. And I think it’s important to note that because the deduction from that is that the drug seem to be working very effectively on impaired GABA glutamate circuit, but not wall tide or normal GABA glutamate circuits. This is truly a corrective action of MDX rather than a kind of a broad enhancing effect of cognition in general. And you can see that data very clearly on the SOBP poster, you will see…

Yaron Daniely

Management

Thanks, Jason.

Operator

Operator

Thank you. (Operator Instructions) Our next question comes from John Newman of Canaccord. Your line is open. John L. Newman – Canaccord Genuity, Inc.: Hi, guys, thanks for taking the questions and Yaron thanks for the update this morning is very thorough and informative. I just had a question about the design of Phase 2 ADHD study in pediatrics, could you talk about any sort of controls that you have built into that study to make sure that you have sufficient population of primary inattentive patients. And can you talk about the expectation for sort of looking forward commercial use of the drug as it’s approved in pediatrics given that the hyperactivity in pediatrics is a little bit different than it is in adults. Thanks.

Yaron Daniely

Management

Sure, the Phase 2 pediatric ADHD trial is planned as a placebo control trial with 82 subjects, 41 enrolled in the treatment arm and 41 enrolled in the placebo arm, and this is a multicenter of study. The population, John is entirely predominately inattentive ADHD. So for this study, because it’s a safety tolerability NPK study, we feel comfortable that for the regulatory pathway, the only patient population that should be enrolled is predominately inattentive ADHD. John L. Newman – Canaccord Genuity, Inc.: And could you also talk a little bit about the commercial launch maybe this question also for David, what was seen when Strattera was launched commercially and just how Strattera took market share from different areas of the market including a stimulant market that was maybe little bit unexpected at that time? Thanks.

Yaron Daniely

Management

Yeah, thanks for the question. So when Strattera launched in 2002 as many of you’ve heard me say it would be most successful CNS drug launch in history. It sold about $300 million the first year, $600 million the second year and at its peak had a 20% market share. And as many of you have heard from David Baker, our Chief Commercial Officer back in the early 2000 it was really a three horse rate between Adderall, Concerta and Strattera actually who’ll be the market leader. Now it’s important to note that market share was only marginally derived from the new diagnosis and new treatments. Some of that came from patients who were not on any kind of therapy. And Strattera was their first ADHD therapy, but the vast majority of that 20% market share came from prescriptions that were previously stimulant prescriptions, and physicians that were turning over patients from Adderall, from Concerta or Ritalin really methylphenidate to Strattera because of the perceived benefit of a non scheduled effective drug. So to borrow this question to explain our position on MDX penetration I think it’s important to note that although the low hanging fruit for MDX is certainly the $1 billion existing market for non-stimulants primarily lead by Strattera, and to some extend by Intuniv and Kapvay. If we believe that the dynamics of the market would be that large numbers from the stimulant treated adults that just don’t really have an alternative do not want to be on a non-stimulant it takes for ever to start working, and as a low effect side would be actually switched over given the availability of our rapidly acting effective non-schedule drug. And we believe the penetration to that “stimulant” market would also be significant together with the penetration to the non-stimulant market as well as to those who are diagnosed with ADHD. But actually are not on any kind of therapy because of lack of efficacy or concerns with tolerability et cetera. John L. Newman – Canaccord Genuity, Inc.: Excellent. Thank you very much.

Yaron Daniely

Management

Thank you.

Operator

Operator

Thank you. Our next question comes from Yi Chen of Aegis Capital. Your line is open. If you have your phone on mute, please take it off from mute.

Yaron Daniely

Management

Hello. Yi Chen – Aegis Capital Corp.: Hi, thank you for taking my questions. You mentioned Roche is candidate for Fragile X Syndrome and it seems they have recently completed a Phase 2 trail, have you heard anything on their status or results?

Tomer Berkovitz

Management

This is the October earnings call, we have not, I have not I’ve not referred anything. So Yaron. Yi Chen – Aegis Capital Corp.: Okay.

Yaron Daniely

Management

I know that the trial was completed and results have not been publicly announced as well as I think that the Phase 2 trial that proceeded this Phase 3 trial I’m not aware of the results being announced for that trial as well. Yi Chen – Aegis Capital Corp.: Okay, okay. And regarding the Phase 2 trial for pediatric ADHD I apologize if I missed that in your prepared remarks, as it already started?

Yaron Daniely

Management

No, it’s starting this quarter. Yi Chen – Aegis Capital Corp.: This quarter, okay. And when do you expect to complete the enrollment?

Yaron Daniely

Management

Before the end of the year, so we expect both the Fragile X adolescents and adult study and the pediatric ADHD study to be completed before the end of the year in Q4. Yi Chen – Aegis Capital Corp.: Shall we expect to see the data before the end of the year to?

Yaron Daniely

Management

I expect to be able to report top-line date before the end of the year. Yi Chen – Aegis Capital Corp.: Okay, great. Thank you. Yi Chen – Aegis Capital Corp.: Sure.

Operator

Operator

Thank you. And I’m not showing any further questions at this time. I’d like to turn the call back over to Yaron for closing remarks.

Yaron Daniely

Management

Well, again thank you everyone for joining us this quarter, a lot of exciting developments in the past quarter and looking forward to a very exciting second half of 2014. I look forward to seeing you in our Banking as well as R&D conferences this summer. Have a great day.

Operator

Operator

Ladies and gentlemen thank you for participating in today’s conference. This does conclude the program. You may all disconnect. Everyone have a great day.