Ovid Therapeutics Inc

NASDAQ:OVID

Earnings Call Transcript 2017-Q4

[Operator]

Good afternoon, ladies and gentlemen, and thank you for standing by, and welcome to the Ovid Therapeutics Fourth Quarter and Year-end 2017 Financial Results and Business Update Conference Call. [Operator Instructions].

It is now my pleasure to turn the call over to Laura Pike, Senior Director of Investor and Public Relations of Ovid Therapeutics. Please go ahead.

[Unknown Executive]

Thank you, operator. Good evening, and welcome to Ovid Therapeutics Fourth Quarter and Year-end 2017 Conference Call. We issued a press release a short while ago, providing a review of our financial results for this period and an overview of our recent corporate highlights. This press release can be accessed on the investors' portion of our website at investors.ovidrx.com.

Joining me on the call today are Dr. Jeremy Levin, our Chairman and Chief Executive Officer; Dr. Amit Rakhit, our Chief Medical and Portfolio Officer; and Dr. Yaron Werber, our Chief Business and Financial Officer.

During our prepared remarks, Jeremy will provide a summary of our recent corporate developments in the context of Ovid's overall mission and business strategy. Amit will review this data of our current development program and Yaron will provide a brief overview of our financial highlights and business development strategy, before we open the call up for questions.

Dr. Matthew During, our Chief Scientific Officer and founder of Ovid, will join us for Q&A.

As a reminder, during today's call, we'll be making forward-looking statements. Various remarks that we make during this call about the company's future expectations, plans and prospects constitute forward-looking statements for the purposes of the Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995. Forward-looking statements contained on this call include but are not limited to, statements about the development of new therapies for previously unidentified disorders, the age groups of patients being addressed in our programs, the development of new programs using data sets from preclinical models, the progress, timing, scope, design and results of clinical trials for Ovid's drug candidates, the reporting of clinical data regarding Ovid's drug candidates and the company's future financial results. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors. Please refer to Ovid's filings with the SEC, which are available from the SEC on Ovid's website for information concerning the risk factors that could affect the company. In addition, all information we provide on this conference call represents our views only as of today and should not be relied upon as representing our views as of any other date. While we may elect to update these forward-looking statements at some point in the future, we undertake no obligation to update or revise any forward-looking statements, we may make on this call on account of new information, future events or otherwise.

I will now turn the call over to Jeremy.

[Jeremy Levin, executive]

Thank you, Laura. Good evening, everyone, and thank you for joining us. It's a pleasure to welcome you to our first financial results and business update conference call. 2017 was a year of significant progress for Ovid. I'm very pleased and excited to share with you a review of this progress, and opportunities we have in front of us in 2018. As many of you know, we dedicate our efforts to developing medicines, which we hope will have a profound impact on patients and their families who live with rare neurological disorders. We believe that there is a substantial and unfulfilled opportunity for us in this area because it's estimated, there are approximately 1 million people in the U.S. affected by such rare neurological disorders. This is an area of drug development, with a significant need, that has become a compelling new area of focus for the biotechnology industry, and one in which we have taken significant steps.

In addition, this area is changing rapidly. In recent years, the size and understanding of these disorders has dramatically accelerated and is unlocking exciting new approaches to treat these important disorders. Advances are being made on many fronts, and new therapies are being developed, including those for epilepsies and disorders of movement and behavior.

Our knowledge on how best to address these disorders is expanding, both industry-wide and at Ovid. And as we build our understanding of these rare neurological conditions, we gain an appreciation for the way the different molecular pathways underlying these disorders, helped drive the symptoms patients suffer. This in turn allows us to transfer the knowledge gained about relevant molecular pathways and clinical endpoints for one disorder to another, which we believe will enable us to build a scalable business model.

Ovid has set out to be a leader in this field, and by keeping our focus on neurology, it is our belief that our model offers us a potential to produce multiple medicines in the future and therefore, succeed in our mission.

We at Ovid are not just at the forefront of the science and understanding of the tremendous progress in neurology, but we also translate our expertise into a consistent trajectory of disciplined execution. We believe we have demonstrated that in 2017, showing solid progress across many areas, including execution across our clinical development programs, which have significantly advanced our product candidates. Specifically, in neurodevelopmental disorders, we initiated the Phase II STARS trial with OV101 in Angelman syndrome in adults and also in adolescence. We completed our Phase I PK study in adolescents with Angelman and Fragile X syndrome as well as juvenile toxicology studies for OV101. These steps prepare the way for trials in younger patients.

Added to this, on the clinical front, together with our partner Takeda, we initiated a Phase Ib/IIa clinical trial of OV935 in adults with rare developmental and epileptic encephalopathies. We also moved expeditiously on the regulatory front and secured FDA orphan drug designations for both OV101 and 935, and Fast Track designations for OV101. While we focus on a nimble, efficient and disciplined approach to drug development, we also built the company in other areas. Amongst the steps we took to build value, we continue to focus on strengthening our intellectual property position. As a result, we were issued multiple new patents extending the marketing exclusivity of our compounds, well into the 2030s. Most recently, we were granted a new method of use patent for OV101 in the treatment of Fragile X syndrome.

Additionally, we executed on a high-value novel business development transaction and secured an innovative corporate alliance with an outstanding collaborator, Takeda. And finally, we completed our IPO in transition to a NASDAQ-listed company, positioning ourselves for what we hope will be a robust future as a public company with a diverse group of stockholders and a capital base that allows us to take advantage of the opportunities in front of us. All of these accomplishments were made possible by the strength, diversity and professionalism, we built into the team at Ovid and its Board of Directors. This in turn has established the groundwork for a deep and lasting relationship with those who matter the most for our future, the patients and their families who are touched by these disorders and for whom we are working to develop new medicines.

For a young company, these are significant steps and we're proud of our accomplishments. But we anticipate these steps are only the beginning. As we look to 2018, among some of the items I want to bring to your attention, is the focus and commitment we have to all age groups of those afflicted by these lifelong disorders. In our programs in rare epilepsies or neurodevelopmental disorders, based on our understanding of the science and medicine, we believe patients may see significant benefits, the earlier that interventions take place in the course of their disease. Based on this belief, we generated datasets and relative preclinical models, some of which we've already published. And we now are ready to act on that information and move from adults to younger patients. Acting on that understanding, today, we announced our intention to initiate the next stage of development with OV101, bringing it into adolescence and young adults with Fragile X syndrome. This is just 1 example of how we're executing on our vision to make an impact on rare neurological disorders. We believe 2018 is poised to be an important year for Ovid. We're deeply energized and working tremendously hard. We have recently completed enrollment of the adult and adolescent Phase II STARS study in Angelman syndrome, and are expecting top line data in the third quarter of 2018.

We also expect topline results from the Phase Ib/IIa trial for OV935 in developmental epileptic encephalopathies in the second half of the year. Beyond this, we expect to expand the clinical development of both our lead programs with several studies planned. In addition, reflecting the fact that we have an ongoing and active scientific and clinical research program, we expect data presentations at medical meetings throughout the year, starting with the American Association of Neurology Annual meeting next month, where we will have a total of 11 poster presentations on OV101 and 935.

I am pleased with the company's progress. We've built an outstanding team, a strong business strategy and a focused pipeline of highly innovative medicines. This is coupled with the intent to expand our pipeline, not just with additional molecules, but also to test our medicines in adults, adolescents and children as early as possible in these lifelong disorders, allowing us the potential to bring benefits to different age groups and creating the possibility for us to impact the lives of millions over many years.

Together, we believe these steps present Ovid with the ability to achieve a high growth of sustainable growth.

I will now turn the call over to Amit, who will walk through the status of each of our developmental programs in detail.

[Amit Rakhit, executive]

Thanks, Jeremy. To start, I'm pleased to announce that we recently completed enrollment in our Phase II STARS clinical trial, investigating the safety and efficacy of OV101 for the treatment of adults and adolescents, ages 13 to 49 years, for the confirmed diagnosis of Angelman syndrome. Interest in the study has been strong, and we're pleased to announce that the study was overenrolled. This milestone follows our successful completion of our Phase I clinical trial last November, which evaluated the safety, pharmacokinetics and tolerability of OV101 in adolescents diagnosed with, either Angelman syndrome or Fragile X syndrome. While OV101 already has an extensive safety database of over 4,000 adults, the Phase I clinical trial represented the first time adolescents were given OV101. The Phase I results show that OV101 has a similar safety and tolerability profile in adolescents and in young adults alike.

And so, based on these results, we amended the STARS trial protocol to include adolescent participants.

Now turning to the STARS trial, this study is a 12-week randomized, double-blind placebo controlled Phase II clinical trial, in which participants are randomized to 1 of 3 treatment arms. Arm 1 is 15 milligrams of OV101, once daily at night. Arm 2 is 15 milligrams at night and 10 milligrams in the morning, and arm 3 is placebo. Recall, that the STARS trial was designed to establish safety and tolerability in people with Angelman syndrome.

In addition, we're exploring the clinical utility of potentially normalizing GABAA levels that are low in this disorder by restoring the tonic inhibitory signal that is disrupted. As such, the study includes prospectively defined secondary efficacy endpoints that reflect the clinical symptoms that are manifested, due to disruption of GABAA signaling in Angelman syndrome. Overall, we believe this study will provide us with the strategic opportunity to have an early look into signals or trends of efficacy with 2 different dose regimens of OV101, across 2 different age groups.

Importantly, we believe the study will inform the next stage of development with OV101 as we intend to develop it for all patient age groups. The primary endpoint of the STARS trial will assess the safety and tolerability of OV101. The trial also has several exploratory endpoints to evaluate measures, related to clinical global impression, maladaptive behavior, sleep, gross and fine motor skills and health-related quality of life. The trial was designed in consultation with the Angelman syndrome community, and we're proud of our continuing collaboration with the families, caregivers and the entire Angelman syndrome community. Top-line results from this trial are expected in the third quarter of 2018.

With a prevalence of around 1 in 12,000 to 1 in 20,000 patients in the U.S., Angelman syndrome represents a significant unmet need in the medical community, and there are no FDA approved therapies available for these patients. In recognition of this unmet need, the FDA granted Fast Track designation for OV101 in Angelman syndrome, last December. The FDA's Fast Track process is designed to expedite the development and the review of drugs, used to treat serious conditions with inadequate treatment options. Current treatment options for Angelman syndrome are limited to symptomatic agents and antiseizure medications.

Based on our strong preclinical dataset in Angelman syndrome models, we believe OV101 has the potential to become the first therapy to address the underlying path of physiology, by a restoration of tonic inhibition in Angelman syndrome patients. Later this year, we plan to announce details on our plans to further our clinical program for children with Angelman syndrome.

Now let's move to Fragile X, which is our second developmental area with OV101. As with Angelman syndrome, loss of tonic inhibition is also implicated in the symptomatology of Fragile X syndrome, a genetic condition that causes intellectual disability, behavioral and learning challenges and various physical characteristics. Fragile X syndrome has an estimated prevalence of around 68,000 to 85,000 patients in the U.S. And like Angelman syndrome, there are no FDA approved therapies available.

Following the completion of the OV101 PK study late last year and a recent receipt of FDA Fast Track designation for Fragile X, announced earlier this month, we are pleased to provide more details today around our next steps for the development of OV101 in adolescents with Fragile X syndrome. We plan to initiate a Phase II multidose, 3-arm clinical trial this year, which is expected to randomize 30-male participants, 13 to 22 years old, diagnosed with Fragile X syndrome. The name of the study is the Rocket Study. The primary endpoint of Rocket will be the safety and tolerability of OV101 in people with Fragile X syndrome. Secondary endpoints include an evaluation of changes in behavior during 12 weeks of treatment with OV101. We expect to initiate this study in 2018.

Turning to OV935, which is our 50-50 global co-development program with Takeda, we continue to enroll participants in a randomized, double-blind, placebo controlled, dose escalation Phase Ib/IIa clinical trial in adults with rare developmental and epileptic encephalopathies or dEE. dEE, in this study includes conditions such as Dravet syndrome, Lennox-Gastaut syndrome and Tuberous Sclerosis Complex. dEE is a classification of a heterogeneous group of epilepsy syndromes associated with severe, cognitive and behavioral disturbances, and was recently established by the ILAE, or International League Against Epilepsy Task Force. While there are many treatments available for epilepsy, few therapeutic options exist for patients with dEE which is often marked by intractable seizures that frequently result in cognitive, neurologic and behavioral deficits. OV935 is a novel cholesterol 24-hydroxylase (CH24H) inhibitor that reduces production of 24 hydroxycholesterol or 24HC. We believe that 24HC is a potent activator of the N-Methyl-D-Aspartate or NMDA receptor pathway, which is involved in excitatory neuronal signaling and plays an important role in initiation and propagation of epileptic activity. We believe that OV935 has a potential to target the increased pro-epileptic signaling that occurs in dEEs and provide a treatment for people with rare epilepsies who currently do not have sufficient options. Our Phase Ib/IIa clinical trial with OV935 is one of the first clinical trials to employ this classification of dEE. And like our STARS trial, the study is primarily designed to assess safety, tolerability and enable us to evaluate dosing, pharmacokinetics and 24HC biomarker levels in the blood. The ongoing multicenter Phase Ib/IIa dEE trial, includes an initial 1-month baseline period, followed by a 1-month double-blind dose escalation phase of therapy.

Thereafter, all participants will continue for an additional 2-month open-label treatment phase. The primary endpoint of the study is to characterize the safety and tolerability of OV935. Secondary and exploratory endpoints include evaluation of PK parameters, change in 24-hydroxycholesterol levels and change in seizure frequency from baseline to the end of the study. We anticipate sharing top-line data in the second half of 2018.

Importantly, this trial is the beginning of a planned broader development program for OV935, as we believe that NMDA over-activation, plays a central role in the pathophysiology of dEE's as well as other rare epilepsies. We look forward to keeping you apprised of our progress. In December, at the American Epilepsy Society annual meeting, we and Takeda presented new data from previous clinical and preclinical studies demonstrating the potential of OV935, as a novel treatment for dEE. Phase I clinical trials of OV935 showed a strong safety profile and well-characterized PK profile. The results of further clinical and preclinical studies demonstrated that plasma 24HC concentration decreased by 47% to 63% from baseline in a dose-dependent fashion and achieved steady-state levels by day 7. This is important because as part of our broader development program, plasma 24HC is being assessed as a potential biomarker for OV935, which may then inform future clinical trial designs and help clinicians individualize the use of OV935.

Also in December, we were pleased the FDA granted Orphan drug designations for OV935 for, both Dravet syndrome and Lennox-Gastaut syndrome, 2 types of dEE.

In closing, at the American Academy of Neurology annual meeting, taking place April 21 through 27, 2018, in Los Angeles California, as Jeremy already mentioned, we will have 11 poster presentations, highlighting data from our programs in Angelman syndrome, Fragile X and epilepsy. As many of you know, this is a prominent neurology conference in the U.S. We're pleased the meeting organizers have recognized the growing importance of rare neurological disorders and have included this category in the meeting's agenda. We're looking forward to interacting with the broader neurology community next month at the meeting.

With that, I'll turn the call over to Yaron, who will provide an overview of our financial highlights and business development strategy.

[Yaron Werber, executive]

Thanks, Amit. Now let me turn to review our financial results for the fourth quarter and full year of 2017. For the fourth quarter and fiscal year 2017, we reported net losses of $11 million and $64.8 million respectively, as compared to $7.6 million and $22.4 million for the same period in 2016. The increase was primarily due to higher expenses relating to the clinical studies of OV101, cost-related to the Takeda collaboration for OV935, which included a one-time noncash equity charge of $25.9 million, higher preclinical development expenses and an increase in headcount to support our expanded operations. We closed the fourth quarter of 2017 with approximately $87 million in cash and cash equivalents. We remain committed to a disciplined and strategic approach regarding the use of our capital. We believe we are well financed, with at least 12 months of cash on hand, and importantly, that our current cash position is sufficient to deliver on our 2 upcoming proof-of-concepts that are readouts anticipated later this year.

Now turning briefly to discuss our business development strategy. Recall, Ovid is focused exclusively on developing impactful medicines for patients and families, living with rare neurological disorders. We remain committed to leveraging transactions strategically to grow our pipeline within our current focused areas. While the timing of any potential deal is hard to predict, it is an active process for us as we are continuously evaluating new opportunities.

We would like to turn the call back to the operator, so we can go ahead and take your questions. Operator?

[Operator]

[Operator Instructions] Our first question comes from the line of Eric Schmidt of Cowen and Company.

[Eric Schmidt, analyst]

Maybe first from Amit on the STARS study. I know, you got the go-ahead to enroll adolescents late last year, kind of late in the enrollment of the trial. Were you able to get some adolescent patients into the study? Can you comment on, if so, how many?

[Amit Rakhit, executive]

Yes. So at this point, we're not commenting on the actual number of patients in each age group, but recall that the time in which we amended the STARS study to include adolescents, we were already about 2/3 enrolled. But since that time, we did see strong demand from the adolescents with Angelman syndrome to enroll in the study. And so given the demand that we had and in order to accommodate this interest from the patient community, we actually kept the study open to enrollment a little longer than we anticipated and, therefore, we actually over-enrolled the study. And during that time, we continued to enroll both adults and adolescents, and did meet the number of the adolescents that we intended to include in this study. So we're going to share more information at AAN, American Academy of Neurology, in -- next month. And there'll be a poster that highlights the baseline demographics. And then, we'll release the top line results in Q3.

[Eric Schmidt, analyst]

Okay. In terms of that Q3 top line results, you think that the press release itself will have enough data so that we can get a good flavor of any and all safety and efficacy endpoints? I'm just thinking about what you could say in a release versus a medical meeting?

[Amit Rakhit, executive]

Yes, that's a great question also, Eric. So we'll definitely have enough information to give a sense of what the top line information is from the study. Recall that the primary endpoint for STARS is safety and tolerability. So we'll be in a position to comment on that. And then will just depend on at the time of the data release, what else we include in that statement.

[Eric Schmidt, analyst]

Okay. Maybe one last question maybe for either Jeremy or Yaron for the business development side. I know, you can't predict the timing of any new bus/dev activity. But sounds like things are still quite active across number fronts. I don't want to put words in your mouth, is it a gem of a goal to get some new compounds in 2018?

[Jeremy Levin, executive]

Eric, it's Jeremy. I'll hand that to Yaron and Yaron can you just respond, thanks.

[Yaron Werber, executive]

Yes Hi, Eric. So this is something that is certainly an active process for us that really the whole senior team and many levels within the company is consistently -- are consistently engaged in. There's not a lot that we can comment on other than we do believe there is compelling opportunities. We do offer a fairly differentiated model and flexibility as to how we can do deals. And as you know, at times we do bring a potentially preclinical asset that we don't necessarily discuss publicly until they become more relevant. But yes, this is an active process and stay tuned as we -- we'll share more information as soon as we can.

[Operator]

Our next question comes from the line of Tim Lugo from William Blair.

[Tim Lugo, analyst]

For the Rockets study, are you using the same dosing regimen that's used in the STARS with the 15 mg arm, 15 plus 10, and a placebo?

[Jeremy Levin, executive]

I think, why don't you grab that, Amit.

[Amit Rakhit, executive]

Hi Tim. So we're not prepared to release information about dosing at this point. We are going to communicate that at a future point when the study starts. So right now, we're preparing to open sites and are pretty well advanced in our efforts on this study, but we'll release further details as we open those sites.

[Tim Lugo, analyst]

Understood, and I guess ahead of the STARS readout, can you remind me if the natural history suggests any acceleration of disease progression between the adolescent patients versus the adults? I assume for some of the behavior endpoints, there will be a difference there. But is there anything for motor function or some of the global assessments that -- where you see those 2 patient pools probably differing?

[Amit Rakhit, executive]

Yes, so -- that's a great question also. So we'll have more information from our own study once the study is complete, and we actually analyze the data between adolescents and adults. We have probably one of the larger databases now because we've conducted this study. And I think the other studies that have been out there are single center in the past, probably don't get to the answer of the question you're getting too. So we'll have more data because we have the most robust dataset right now in this community.

[Jeremy Levin, executive]

And just to address the question, I think Matt will make a quick comment on that as well.

[Matthew During, executive]

Just to emphasize the -- all the data that exists on adults in Angelman, previously came from questionnaires. There is actually no direct measurement or engagement with that community outside of questionnaires. So I think the data that we've collected and that we will release with the STARS readout will actually be the first data on natural history in adults from the actual patients themselves with direct measurements.

[Tim Lugo, analyst]

Understood. And maybe before the 935 readout, can you remind me how you're capturing your seizure data? I know it might be a little different than some of the other top-line results we've seen from CBD or FinPharma just so there's not a confusion in the market?

[Jeremy Levin, executive]

You bet, again Amit, why don't you take that?

[Amit Rakhit, executive]

So we're capturing seizure information, we are doing EEGs, we are doing seizure diaries to capture. So 2 different ways of capturing that information.

[Tim Lugo, analyst]

And convulsive versus drop seizures, would that be teased out?

[Amit Rakhit, executive]

We will be looking at the different types of seizures in this population, and we also -- we will have that as one of the analysis that we'll be conducting.

[Operator]

And our next question comes from the line of Jason Butler with JMP Securities .

[Jason Butler, analyst]

First of all, can you talk about any of the efficacy measures you'll be looking at in the Fragile X study? Are you looking at things like the CGI, hyperactivity, cognition, anxiety and any specific tools that you're using that you can talk to us about now?

[Jeremy Levin, executive]

Hi Jason, again Amit it's getting heavy-duty here, so why don't I pass it back to him as well?

[Amit Rakhit, executive]

That's a great question. So we're actually going to release more information about the overall design of the Fragile X program at a future date. But I will let you know that we're planning on using some typical scales that you've seen in other Fragile X studies in the academic community. So things like the ABC-C, which is aberrant behavior checklist community scale, the ADAMS, the anxiety, depression and mood scale, and we are actually are using some of the adaptive scales that are specific to Fragile X as well. The primary endpoint will be safety tolerability, and this will be done at baseline and at week 12, but we'll also be capturing those specific evaluations up with those scales as well.

[Jeremy Levin, executive]

And Matt wants to add a comment to that, if that's okay.

[Matthew During, executive]

I just want to state that we're emphasizing those endpoints and measurements that speak to the preclinical data and the methods of drug -- that we have of the drug. And so that's why there's emphasis primarily also where we have existing tools and assessment instruments that we think evaluates this and that we believe we can interpret. So that's why, I think this emphasis is largely behavioral but as we've said, we will measure other things as well.

[Jeremy Levin, executive]

Thanks, Matt.

[Jason Butler, analyst]

And then maybe one more for Matt just on the Angelman syndrome. Can you talk about any preclinical data that you have or are aware of that supports that treating earlier in the course of a disease could impact progression of the disease or the course of disease?

[Matthew During, executive]

Yes, I can -- I'll refer to the data that is public that is in the public literature, which I think a lot of people aren't aware, which relates to relatively recent data on gene restoration in the mouse model which clearly demonstrates that unlike several other disorders like Rett syndrome, we can go into late and even to other [indiscernible]panels and get full penetrative[indiscernible]correction for most of the symptoms, in Angelman, that's not the case. And what's clear is that from a gene restoration, we've actually normalized UV3 function, you really require going in [innately ] or even embryonically to reverse most of the benefit[indiscernible] features. And what we see is that there is a progression of symptoms, some symptoms clearly will be more tractable as you go in earlier, I think that's almost entirely within most of neuro-development disorders, earlier translates to better. And I think there's nothing different in terms of Angelman compared to other similar syndromes.

[Jason Butler, analyst]

Great, and then one quick last one from me. You mentioned before that you will be presenting some baseline data from STARS at AAN. Any other posters that you can highlight at this point or information that will be at the conference you'd highlight at this point?

[Amit Rakhit, executive]

Hi, it's Amit, again. Yes, great question. So we have 11 posters across both OV101 and OV935 being presented. So one of them I already mentioned, is a STARS' baseline information, which will be a poster presented there. We also have an OV935 baseline of the adult study that's ongoing. We will also be presenting that as well, those will be new information. Some of the others are encores, but those are 2 that I would highlight as new releases of information next month.

[Operator]

Our next question is from the line of Yigal Nochomovitz of Citigroup.

[Yigal Nochomovitz, analyst]

On the STARS trial, I think I counted around 17 secondary endpoints on various aspects of efficacy, and I recognize it's a Phase II study and then also recognize that it's a signal finding study with respect to the efficacy parameters. But it would be helpful if you could sort of just outline which of those 17 you sort of see as more relevant versus less relevant? Which ones are must haves, if you can say that and which ones would be nice to have? If you could just provide a little bit more context on that given that it is a lot of secondary endpoints.

[Jeremy Levin, executive]

Hi, Yigal. Again, I'm going to pitch that to Amit.

[Amit Rakhit, executive]

Okay. Here you go. Yes, that's -- so when we think about the STARS trial, there are multiple secondary endpoints to that you just mentioned. So it's a bit premature to speculate as to which endpoints may be more important than others. But from our interactions with the Thought Leader Community, with families, we really believe that the disruption of tonic inhibition has an effect on a combination of symptoms in this disorder. And so what we're evaluating are areas like behavior, motor function and sleep, all of which we believe are important in the disorder. And this will actually -- the way we actually are looking at these specific domains, really track to the different questionnaires or assessments that are listed on pintrials.gov. They map to either behavior, motor functional and sleep. And overall, this study will then give us an opportunity to look at some early signals of efficacy in these areas, with 2 different dose regimens across 2 different age groups.

[Yigal Nochomovitz, analyst]

And with respect to behavioral, sleep and motor function, are you going sort of aggregate the results when you discuss them in the data release? Or will we get detail pretty much across the board sort of -- kind of what Eric was asking?

[Amit Rakhit, executive]

Yes, so I'm not quite commenting on how we're going to release the data. I think we will definitely give you a robust information set when we do release the information in Q3.

[Yigal Nochomovitz, analyst]

Okay. And then, Yaron, I think you just -- I couldn't hear quite clearly, did you say you have 12 months of cash on hand? Is that right?

[Yaron Werber, executive]

Hi Yigal. So we said, we have more than 12 months of cash. We finished the quarter and the year with $87 million, and the use of cash if you look at last quarter was about just over $9 million.

[Operator]

Our next question is from the line of Sarah Weber of Piper Jaffray.

[Sarah Weber, analyst]

So for dEE, I realize you're still enrolling, but is there anything you can say about the safety and tolerability you've seen to date on a blinded basis? And then also maybe the average dose that patients have been able to reach in the protocol?

[Jeremy Levin, executive]

Good evening, Sarah. Yaron -- sorry, it's going to be Amit to comment. Amit?

[Matthew During, executive]

Yes. I mean, I think it's a good -- it's Matt here. It's a good question. We can comment that we've been able to enroll and these patients and titrate up to their target dose. And we believe, so far that this has been extremely well tolerated. So we're very happy, so far with the tolerability that we've observed. Of course it's still preliminary but so far the drug has been well tolerated and we have been able to titrate up to the maximum dose.

[Jeremy Levin, executive]

Amit, any comments you'd like to add?

[Amit Rakhit, executive]

Yes. So, Sarah, yes we definitely have remained on track with enrollment, and we plan to report top-line data second half of this year. We are currently enrolling -- the data is still blinded, but we don't have further information at this point. But as Matt said, we continue to enroll as planned with those studies.

[Sarah Weber, analyst]

Okay, great. And then for Fragile X for the Rocket study. Any color on the number of planned sites and will they all be U.S. sites?

[Jeremy Levin, executive]

Amit, again.

[Amit Rakhit, executive]

So -- no that's -- we're looking at -- so currently definitely the U.S. is involved with those sites with Fragile X program. We're exploring some international sites but primarily it would likely be U.S.

[Sarah Weber, analyst]

Okay, awesome. And then just one last question on the patent protection. So in addition to the new method of use patent for OV101, I guess, what's your strategy for strengthening this portfolio, I mean, moving forward? Or do you feel good about where the protections are right now?

[Jeremy Levin, executive]

It's Jeremy here. We feel very good about what we've accomplished. From the inception of the company, you will recall that the first -- one of the first employees was actually one of the top patent lawyers from a very, very well-founded background. So we've been examining different types of approaches to our patent strategy. But the overall intent behind this is to secure as long a patent life as possible, as we can for all of our products. And as we have stated to date, we've received a significant amount -- number of patents, and they are issued and giving us extensions through well into the 2030s.

[Operator]

And at this time, I'm showing no further questions. I'd like to turn the call back over to Ovid's Chairman and CEO, Dr. Jeremy Levin for closing remarks.

[Jeremy Levin, executive]

Well hi, thank you so much everybody for taking the time this evening to join us on the call. As we discussed today, we feel we are executing our strategic plan and as we are doing so, we are accomplishing many of our goals. I want to thank particularly the employees for all their hard work and dedication, which has been tremendous to observe and to participate with. And on behalf of everybody at Ovid, I want to thank you, those of you who are on the phone today, and those others who will listen to this for your support. We really look forward to updating you on our progress during what we think is a very exciting year ahead of us, and we're very enthusiastic about this year. So thank you very much, everybody, and good evening to you.

[Operator]

Ladies and gentlemen, thank you for your participation in today's conference. This does conclude the program. You may now disconnect. Everyone, have a great day.