Onconova Therapeutics, Inc.
Q2 2019 Earnings Call Transcript

Published: 14 Aug 2019

[Operator:]

Good morning and welcome to Onconova Therapeutics' Corporate Update and Second Quarter 2019 Financial Results Conference Call.At this time, I would like to turn the call over to Avi Oler, Vice President of Corporate Development and General Counsel.

[Avi Oler:]

Thank you, Operator. Good morning everybody, and welcome to Onconova's second quarter 2019 corporate update and financial results conference call. Earlier this morning, we issued a press release with second quarter 2019 financial results and business updates. If you have not yet seen this morning's press release it is available on the Investor Relations page our Web site, at www.onconova.com.On today's call, Dr. Steve Fruchtman, President and CEO, will discuss the company's recent highlights and anticipated clinical and business progress. After Steve completes his opening remarks, Mark Guerin, our Chief Financial Officer, will review second quarter 2019 financial results. Following Mark's report, we will move to the Q&A portion of the call. Dr. Ric Woodman, our Chief Medical Officer, will also be available to respond to questions. Lastly, Steve will come back with some final brief remarks and a review of upcoming milestones.Before we begin, I would like to remind everyone that statements made during this conference call will include forward-looking statements under the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties that can cause actual results to differ materially. Forward-looking statements speak only as of the date they are made, as the underlying facts and circumstances may change. Except as required by law, Onconova disclaims any obligation to update these forward-looking statements to reflect future information, events, or circumstances. Please see the forward-looking statements disclaimer in the press release issued this morning and the risk factors in the company's current and future filings with the SEC.With that, I'll turn the call over to Steve.

[Steve Fruchtman:]

Thank you so much, Avi. Good morning everybody, and thank you for joining today's call. The first-half of 2019 was a strong period for Onconova, highlighted by our continued progress towards completing enrollment of our global pivotal Phase 3 INSPIRE trial. Upon completion of enrollment and 288 death events, we look forward with great anticipation to reporting top line data in the first-half of 2020. As a reminder to all, the INSPIRE trial is an open-label randomized controlled, international study designed to determine the efficacy, safety, and tolerability of single-agent IV rigosertib in the treatment of patients with second line high-risk MDS. Patients in this study are less than 82 years of age, and have progressed on, relapsed, or failed to respond to the previous standard of care with hypomethylating agent therapy.The study randomized patients to receive either IV rigosertib with best supportive care or the physician's choice of therapy with best supportive care. The primary endpoint of this study is overall survival of all randomized patients in the intent to treat population. There is also a second opportunity for an FDA approval, which is the sequential analysis of the overall survival of the very-high-risk subgroup as defined by the revised International Prognostics Scoring System. In terms of enrollment on the trial, more than 140 trial sites in 24 countries across four continents are open, including 21 sites in Japan. The company opened new clinical trial sites in countries already participating on the INSPIRE trial.Additional geographies, most importantly Brazil, because of its huge population and thus anticipated high number of patients with MDS, are being opened during the coming months with the goal of adding approximately 25 more sites there. This strategy is designed to support the goal of achieving full accrual to the INSPIRE trial by the end of 2019. Should rigosertib significantly prolong survival on the INSPIRE study, we believe rigosertib will be the first new treatment for higher risk MDS in more than 15 years.In addition to the INSPIRE trial, we are advancing toward the start of a new pivotal Phase 3 combination trial of oral rigosertib and azacitidine for the treatment of adult patients with first line higher risk MDS. In December of 2018, we applied to the U.S. Food and Drug Administration for a special protocol assessment, also known as a SPA, for this trial. We anticipate completion of the SPA process and the initiation of the pivotal Phase 3 trial toward the end of 2019. A number of important abstracts that present progress on the INSPIRE trial and the combination trial of azacitidine with oral rigosertib have already been submitted to the American Society of Hematology Annual Meeting, which is scheduled for December, 2019, in Orlando, Florida.While our focus remains on MDS and completing enrollment in the INSPIRE trial, we have made important progress with our pipeline program, ON 123300, our investigational first-in-class dual inhibitor of CDK4/6 and also ARK5, which we believe has the potential to treat numerous cancers, including refractory metastatic breast cancer. As a reminder, we entered into a license agreement with HanX Pharmaceuticals for ON 123300 in December of 2017, under which HanX will provide all funding required for Chinese IND-enabling studies performed for Chinese food and drug IND approval. The company also intend for these studies to be fully compliant with U.S. Food and Drug Administration standards. Onconova and HanX completed a pre-IND consultation with the U.S. FDA, and we have received the FDA's guidance for the manufacture of ON 123300 and the preclinical development plan.For those of you who are not familiar with the field, CDK inhibitors have emerged as promising compounds targeting very large cancer indications such as hormone receptor positive, metastatic breast cancer. The current generations of CDK inhibitors have their limitations. Due to its unique targeting of ARK5 as well as CDK 4/6, we believe ON 123300 has the potential to overcome many of these limitations making our drug candidate potentially suitable for certain cancers that may not be responsive to the current generation of CDK 4/6 inhibitors.If successful we believe all ON 123300 could address this very large market opportunity with a potentially better therapeutic. In addition to the INSPIRE trial with IV rigosertib and the planned Phase 3 trial of oral rigosertib combination with azacitidine in first-line MDS, we are also developing rigosertib for the treatment of pediatric RASopathies, a group of rare syndromes that together are the most common genetic conditions globally. These include Juvenile Myelomonocytic Leukemia, also known as JMML, a rare and aggressive childhood leukemia caused by excessive proliferation of non-acidic and granular acidic cells that unfortunately infiltrate various organs.The prognosis for children with JMML is very poor. It's invariably lethal and the only curative therapy is a hematopoietic stem cell transplant. Thus there is a significant unmet medical need. The JMML program may advance into clinical trials this year and we are excited about the potential of rigosertib due to its unique mechanism of action in the treatment of these devastating childhood illnesses.The NCI Pediatric Oncology branch has also studied patient derived Xenograph models from their patients with rash mutated cancers. In addition to these programs, we have plans to pursue solid tumor cancers driven by mutated rash genes or the rash pathway which together represent a third of all human cancers. This plan includes an investor, investigator initiated Phase 1 study of rigosertib in combination with a commercially available PD-1 inhibitor to be supplied to patients with progressive KRAS mutated non-small cell lung cancer.SymBio Pharmaceuticals, our collaborator for development and commercialization of rigosertib in Japan and Korea is planning to conduct a similar study along with us. Under the terms of the agreement signed in 2011, SymBio has an exclusive license for Japan and Korea and will develop and commercialize rigosertib in these countries. Onconova received an upfront payment and will receive milestone payments tied to the progress of rigosertib development as well as sales milestone payments plus royalties on net sales. Onconova supplies drug product for clinical trials conducted by SymBio in Japan. Our partner continues to work with us to conduct a KRAS mutated non-small cell lung cancer trial in Japan and we expect the trial will start this year.With respect to business development, we recently announced collaboration with Mission Bio, a San Francisco based company that studies single-cell populations to uncover mutations with their unique tapestry platform. The tapestry platform is the industry's first single-cell DNA sequencing platform, enabling detection of possible genetic heterogeneity in disease progression and treatment response. Mission Bio and Onconova with neutralized tapestry platform to study rigosertib as part of planned clinical trials. We believe single-cell genomics numbers may identify mutations with better resolution than that of traditional sequencing genomic message, allowing a view into each patient's disease at the cellular level.In addition, the tapestry platform to our research and development program by that addition, we are including the opportunity to set the study, single-cell clones in MDS and determine the sequence of required genetic abnormalities and the influence of rigosertib regarding clinical outcomes. These studies have the potential to make a meaningful difference in the lives of patients with MDS and greater understanding in the pathophysiology of MDS and rigosertib influences that pathophysiology.We also recently entered into an additional license agreement with our partner HanX, focused on the development, registration and commercialization of rigosertib, along with other novel oncology products in China. We see this second license agreement with HanX as important to the advancement of this study of rigosertib in high risk MDS and solid tumor indications in the future.Under the agreement, Onconova granted to HanX an exclusive license to develop and commercialize rigosertib in greater China, and a non-exclusive license to manufacture rigosertib in greater China as well. In exchange for these rights, HanX committed to upfront payments totaling $4 million. This includes a $2 million fee for an investment totaling $2 million in Onconova common stock purchased at a premium to-market. In addition, HanX agreed to place $2 million in escrow in local currency to cover rigosertib clinical development expenses in greater China.HanX also plans to participate in our INSPIRE Trial depending, on their regulatory approval and their timelines. Onconova could receive up to $45.5 million in regulatory, development and sales based milestone payments, plus tiered royalties up to double-digits by net sales in greater China. We will supply the finished product for sale in the licensed territories and HanX will also support our clinical trial initiatives in greater China.Now, I'd like to turn the call over to Mark Guerin, our Chief Financial Officer, for a discussion of our financial results for second quarter 2019. Mark?

[Mark Guerin:]

Thanks, Steve, and good morning, everyone. Cash and cash equivalents as of June 30, 2019 totaled $5.9 million compared to $17 million as of December 31, 2018. Based on our current projections and receipt of funds due from HanX, we believe that our cash will be sufficient to fund ongoing trials and operations late into the fourth quarter of 2019.The company was notified by NASDAQ on July 26 that NASDAQ has accepted the company's plan to regain compliance with stockholders' equity listing requirement by November 18, 2019. Net loss was $3.6 million for the second quarter ended June 30, 2019 compared to $4.3 million for the comparable 2018 quarter.Research and development expenses were $3.9 million for the second quarter ended June 30, 2019, and $4.1 million for the comparable period in 2018. General and administrative expenses were $1.8 million for the second quarter ended June 30, 2019, and $2.1 million for the comparable period in 2018. We continue to manage our resources carefully while maintaining a primary focus on completing the INSPIRE trial.This completes my financial review. I will now turn the call back to Steve.

[Steve Fruchtman:]

Thank you so much, Mark. With that, we would like to open the call for questions. After the question-and-answer period, I will finish with some closing remarks. Operator, please go ahead with questions, and thank you.

[Operator:]

Thank you. [Operator instructions] Our first question comes from Dr. Joe Pantginis with HC Wainwright. You may proceed with your question.

[Joe Pantginis:]

Good morning, gentlemen. Thanks for taking the questions. Guys my questions are little bit new one, so hopefully you can bear with me. With regard to INSPIRE, obviously this is a fluid -- the ongoing enrollment and timelines are very fluid. So I am just curious if you could discuss your prior guidance saying that the top line data might occur at the same time as enrollment. And how we might be able to read into the additional sites coming from Brazil and other sites as well while events continue to occur with patients that have been on study for quite some time now?

[Steve Fruchtman:]

So thank you, Joe. I will ask Ric to address your question.

[Ric Woodman:]

Thank you, Steve. There are several components to your question, Joe. And I will try to address each of them. First of all, as I think many of you may know, in March we announced that we achieved a critical milestone enrollment of 75%. We are certainly well above that. Enrollment continues to proceed as planned. We still plan to complete enrollment by the end of 2019.We look forward very much to the Brazilian contribution. In my own personal experiences numerous global studies, Brazil has always been strong contributor to hematology studies not only because of their population but there hematologic expertise and network of hematology centers. So I think that will certainly allow us every opportunity to complete enrollment this year. The change in VHR, the worst prognostic group, in the study varies over the course of the study.Currently, it's running around approximately 70%. And this is particularly important for your question about enrollment and survival events. Obviously, this population when untreated has the worst prognosis. Median overall survival is generally well below four months. Because this population is the largest sub-group in this study, it has two particular implications. It is possible that there will be a close relationship between enrollment of 360 patients and the required number of survival events per protocol which is 288 events.The greater the proportion of the VHR, the more likely the median overall survival in the control arm will be reduced. And this is certainly something we are anticipating and tracking carefully. One of the things that I think is particularly fortunate is that the number of patients who are lost to follow-up in the study is very small. And so, we will continue to track both recruitment and survival events closely.I think and also as Steve pointed out, there is a primary endpoint for the VHR subset. And this may be particularly important when we go to the primary endpoints analysis. With that, I hope I have addressed each of your questions, Joe. And thank you for them.

[Joe Pantginis:]

No, you certainly have, Ric. Thank you very much for that. And then the next question is sort of shifting over to the oral rigosertib and the SPA. My assumption is you guys are in hot and heavy discussions with the FDA with regard to getting to the finalization of the SPA. I was just curious at this point as things continue to progress, what the gating factors are? I know previously you were talking about maybe the statistical analysis plan. But, I was also curious does INSPIRE impact your discussions at all and would the FDA be waiting on anything from INSPIRE?

[Ric Woodman:]

Thank you again for that question. There is multiple parts to it. First let me clarify, I don't think we are in any hot and heavy discussions with the FDA. We are definitely in discussions and communication with the FDA. We are at the point where we finalized our responses through the initial review of the protocol. Many of which we agreed with the requested changes.Much of the study design is agreed upon. There are one or two final remaining points that we need clarification on. I think that the Phase 2 study of oral rigosertib and azacitidine has become particularly valuable and informative for both us and the FDA. And certainly that is a very positive thing when you go to develop a registrational phase 3 and you have a informative phase 2.I think in terms of INSPIRE, there's really -- there has been no linkage with INSPIRE and this phase 3 study of oral rigosertib combination. The only thing that might have an impact is if we were to have a positive survival endpoint with INSPIRE. The impact that would have on future regulatory discussion could be important.

[Steve Fruchtman:]

And Joe, just to add what Ric just said, we don't really know FDA thinking, but as you know it is unusual in oncology to have receptor response file. So in fact, the new phase three double trial, we understand through our communication with the FDA that it will be a response trial. And perhaps that is because INSPIRE is a survival trial, so the FDA views this as we will have two trials. One a survival trial to be completed, the data expected in first-half 2020. And therefore, the second in a way confirmatory trial in first line, they have accepted it to be a response trial with a composite response or complete response and partial response both implying the peripheral blood count has normalized.

[Joe Pantginis:]

Got it. No, that's very helpful also. And if you would indulge me one another question please, maybe for Mark and towards your current cash resources. You mentioned that that also includes anticipated milestones from HanX. I was curious if you do anticipate milestones, which I am sure you do based on your current collaborations and partnerships, but that you are not including just based on the timing of these milestones. And I will sort of link that to a leading question of how your potential partnering discussions are going that could lead to a significant non-dilutive inflow of cash? Thanks.

[Mark Guerin:]

Thanks, Joe. If I understood your question, we -- our cash runway that we -- that I mentioned in the earlier part of the call assume the upfront money from HanX. It does not assume any milestones. The milestones are a little bit further out. And, so, I think you are asking about milestone, it's not actually milestones. It's just the upfront.

[Joe Pantginis:]

No, understood. I am just saying like maybe there were milestone payments or collaborative payments that you might be expecting, but the timing is a little more unsure in relative near term say as we slip into 2020, but you are not ready to put that into your current cash guidance?

[Mark Guerin:]

That's correct, Joe.

[Joe Pantginis:]

Okay, got it.

[Mark Guerin:]

Well put. That's right.

[Joe Pantginis:]

Yes. And then the how this might link to how your ongoing partnering discussions are going?

[Steve Fruchtman:]

Avi will take that, Joe.

[Avi Oler:]

Hi, Joe. Thanks. So, in terms of your questions, and thank for it. Ric and Steve mentioning out our anticipated timing for top line data first after for 2020 and this relates very much to our business development discussions as we near that catalysts matter and company their ongoing process, there's nothing to announce at this time but we are seeing interest and those will heat up as we approach that event.

[Joe Pantginis:]

Thanks, Avi. Thanks a lot, guys.

[Steve Fruchtman:]

Thank you, Joe.

[Operator:]

Thank you. And our next question comes from Dr. Jason McCarthy with Maxim Group. You may proceed with your question.

[Naureen Quibria:]

Hi, good morning. This is actually Naureen on for Jason. So I sort of have a broad question within this as regards to RAS space, it's been heating up quite a bit particularly with regard to KRAS and some of the positive data that's been reported at ASCO by Amgen. And in that context would you know where you have Onconova which has the most advanced RAS program but has been sort of flying under the radar. Could you perhaps put into perspective Onconova is an oncology company and not just as a player in the MDS space but where rigosertib may also fit in, in this space?

[Steve Fruchtman:]

So thank you, Naureen for that insightful question. So we actually think the data presented by Amgen has reinvigorated the RAS space. As you know there's been a -- there's been a important question for decades is RAS targetable, we believe based on the paper published by Dr. Prem Reddy in 2016 that the mechanism of action of rigosertib which is unique targets the RAS pathway and RAS proteins that interact with RAS and thus the RAS pathways modulated. Amgen has presented data I believe in only 10 patients, we've treated over a thousand patients with rigosertib, the Amgen mechanism of action is somewhat different.It targets a specific mutation, rigosertib theoretically should modulate any gene abnormality involving the RAS pathway. So it's a broader mechanism of action but we actually thank Amgen for reinvigorating this space and to show the research scientists to confirm our data that RAS is targetable and to add to that, there is I believe for the first time a RAS based conference in Boston at sometime in September of 2019 and Onconova along with Amgen and a number of other companies will be presenting research and clinical data on targeting labs and clinical implications for patients with cancer and I mentioned the word cancer to answer the second part of your question.We are focused on MDS and we have been focused on MDS since I came to Onconova and join me to develop the clinical pathways but we believe rigosertib has the potential to target other diseases and thus we are moving as we mentioned in the earlier part of the call into a very important space and that is KRAS mutated non-small cell lung cancer or patients with lung cancer these days should have genetic, full genetic genotyping which is done 30% of the patients are having mutation of KRAS, the first-line therapy it typically involves an immune-oncology approach which has changed the standard of care but all of these patients will progress.So we believe there will be a role for targeting KRAS with rigosertib using an alternative IO approach and thus we will open as part of investigator initiated trial as part of a Phase 1 to determine the dose of an alternative immune-oncology agent provided commercially to the site plus oral rigosertib with patients with KRAS mutated non-small cell lung cancer. So I hope that answered your question.

[Naureen Quibria:]

It does, thank you. And actually leading into my second set of questions with regards to the KRAS studies that you are going to have, you mentioned that it'll be all the KRAS mutations. Correct. So in terms of the ISP as well the SymBio I believe the ISP you'll have a combination with a PD-1 is the SymBio going to be a monotherapy study or will it be in combination as well?

[Steve Fruchtman:]

No, you're right. We will target also patients with all KRAS mutated non-small cell lung cancer will be eligible for the trial and the trial in the U.S. and the trial in Japan is going to be a combination trial within immune-oncology. By the way, I'm sorry Naureen providing for free the immune-oncology agent will be provided for free for patients participating on the trial.

[Naureen Quibria:]

Great. And the last thing, so these are both going to be, if you can frame our expectations with regard to the timing and potential, I know it's a bit far away initial data readouts for any of these. Do you have any idea?

[Steve Fruchtman:]

No the reality. No, we anticipate the trial to be opening in the U.S. by the end of the year. It's a Phase 1, we hope the first number of patients to be entered onto the trial in 2019 and depending on we sees the MTD, we will determine how long the trial will take but we estimate maybe 20 to 30 patients but that's really not known. So it's a little bit unknown when we'll have the data but we're hoping to begin the trial, initiated trial this year.

[Naureen Quibria:]

Okay, wonderful. Thank you for taking my questions. That's it for me.

[Steve Fruchtman:]

Thank you, Naureen.

[Operator:]

Thank you. And our next question comes from Dr. Ahu Demir with Noble Capital Markets. You may proceed with your question.

[Ahu Demir:]

Hello team, thank you very much for taking my questions. My first question will be through published partnership with Mission Bio. I was wondering how this precision medicine approach will be added to your clinical trials, which clinical trials would have included for frontline high risk MDS, CDK programs. Also I was curious how patient samples are collected, it's genome sequencing and potentially the timeline when this approach will be implemented in the trials and when should we expect results from that?

[Steve Fruchtman:]

Well, thank you for your questions. I think that the clear primary focus with the Mission Bio tapestry platform is MDS. This is certainly a disease that for many reasons makes this platform of single-cell genomic analysis particularly valuable especially in patients who have failed HMAs. It's estimated that 80% of patients in that setting have a mutation. The vast majority have more than one mutation and the significance of each mutation is not entirely known appreciated particularly when it comes either to progression or in terms of response. And so this is particularly valuable in our understanding of the value and mechanism of action of rigosertib in these patients. We look forward to seeing this analysis. It does require somewhat of a different collection than the standard conventional novel gene sequencing approach which requires DNA, Mission Bio's platform requires cells, live cells collected from the aspirate.So this will require some different logistical aspects in the study. Certainly, I think that we would look at both INSPIRE even with the limited number of patients that are required to complete enrollment as well as interest in the upfront Phase 3 study with the combination of oral rigosertib and azacitidine. I think this cutting-edge science is anticipated to be particularly valuable. I think as we gain more experience, we'll move into other therapeutic areas. It always becomes a little more of a negotiation when you are discussing investigator sponsored trials but certainly I think the value of this platform is appreciated by everyone and those in the field are extremely interested in us incorporating this. I will make mention that one of the ASH abstracts that is submitted is looking at genomics in patients who have HMA failure. And that's one of the abstracts we did submit to ASH and it indicates our interest in understanding genomics.Thank you for your question. I hope I've covered all of your interests.

[Ahu Demir:]

You certainly did. Thank you very much, Avi. My next question will be on the CDK program. So, the main catalyst is of course the IND. However, I was curious if you plan to publish any additional data for differentiating factors of ON 123300 similar to your previous presentations where you actually showed favorable reduced neutropenia [ph] compared to other CDK programs. So do we have any results that we should keep an eye on?

[Ric Woodman:]

Well, thank you for the interest in ON 123300, abbreviated ON 123.

[Ahu Demir:]

That is helpful, Avi.

[Steve Fruchtman:]

This is Ric.

[Ric Woodman:]

Yes, it's Ric Woodman speaking.

[Ahu Demir:]

Oh, hi, Ric.

[Steve Fruchtman:]

Better off with Ric.

[Ric Woodman:]

So you have to keep in mind that we have no clinical human data on ON 123. What we do have is preclinical animal model data. And in that setting we see very similar safety profile to what has been published with the other CDK4/6 inhibitors, mainly the occurrence of underlying gastrointestinals effects. But having said that, the other organ systems are relatively spared, which makes our hope that when we get into the first inhuman study that that we will continue to see a promising safety profile. But at this time that remains unknown. The one thing that I can say in the animal models with ON 123, the effects in the bone marrow were limited and reversible.

[Steve Fruchtman:]

And to add to Rick's comments that in breast cancer cell lines that are refractory to the standard CDK4/6 inhibitors, they - these refractory breast cancer cell lines will respond to ON 123 and that's why we're excited about our compound and look forward in the Phase 1 to entering unfortunate women with hormone receptor positive metastatic breast cancer who progressed on commercially available CDK4/6 inhibitors, to see if there'll be response to ON 123. And obviously we're hoping to see that.

[Ahu Demir:]

Thank you, Steve. Thank you, Ric. I have two additional questions if I'm not taking too much of time. Regarding the last conference in Boston, end of September; you mentioned you will be presenting clinical data. Will it be NVS or do you plan to prevent data in probably like lung cancer or any other indications?

[Steve Fruchtman:]

Well, that's a great question. I haven't put the slide deck together yet, Ahu. But clearly, we'll be focusing on the mechanism of action of rigosertib, emphasizing the studies that prove it was a RAAS inhibitor, explaining that to the audience. We will present clinical data as well based on both the INSPIRE trial and the W Trial with all rigosertib. Since you asked the question and we're thinking about it, we probably will and should show our data in patient derived xenograft models with KRAS mutated non-small cell lung cancer. So we probably and should show that data as well. And thank you for that question, because it helps us think about what we should show at that upcoming conference.

[Ahu Demir:]

Sure, thank you, Steve. My last question will be to Mark. So I covered the whole theme. So, Mark, I know that R&D was reduced in this quarter. What was the driver for that?

[Mark Guerin:]

Well, I think it was in part because of lower expenses on the combination program in 2019 as compared to 2018. But that was partially offset by slightly higher spending in the 2019 period on INSPIRE, when compared to 2018. So really that's mostly it on the development cost side. Personnel costs are a little bit lower, but the big driver was the lower spend in clinical driven mostly by less spend on combination.

[Ahu Demir:]

I see. Thank you very much. Nice to hear all your voices, I appreciate you taking my questions.

[Avi Oler:]

Thanks, Ahu.

[Operator:]

Thank you. And our next question comes from Dr. Yale Jen with Laidlaw & Company. You may proceed with your question.

[Yale Jen:]

Good morning, and thanks for taking the questions, and many have been answered, so I have two here. The first one is - correct me if I'm wrong, that what I heard was that you might start the oral rigosertib's trial, Phase 3 trial toward the end of the year. Is that correct? If so, could you give us a little bit more color of that development?

[Steve Fruchtman:]

Sure, this is Steve. And thank you, Jen. So we would very much want to open up the DUBLIC [ph] 0915 trial in a small number of select sites. It depends on our ability to have financing to support that, because, you know, our goal is to [technical difficulty] resources for INSPIRE, but our other goal would be to open a number of sites, let's say, two in the U.S. And now with our collaboration with HanX, perhaps open a site in China as well and a site in Japan, because both China and Japan as well as internationally, all eager to begin the DUBLIC trial. So we'd like to start small in 2019, and then after additional financing is obtained, to be able to open up a global trial with all the sites required for that trial, but it would be premature to do that at this point due to financing. But we believe it would be beneficial to these patients at the development to rigosertib to open the DUBLIC trial small number of sites in the U.S., a site in China, a site in Japan and then grow from there.

[Yale Jen:]

And would that be depending on the getting the spot designation or that's just the two separate events?

[Steve Fruchtman:]

Well, in reality, as you know, we don't need the spot to open the sites. But we would prefer since we go in through the efforts of obtaining it. We have already received from the FDA the key feedback. What is that? They've accepted a response trial. We also understand from the FDA what the delta is anticipated for approval compared to the control arm. And those are the key, two key principles of the protocol. So the remaining discussions with the FDA, I recall some things, some technical factors about protocol. So the answer to your question is

[Yale Jen:]

Understood and appreciate that. Maybe one more question here, which is the collaboration with the mission or with tapestry you mentioned earlier that you were, I guess, apprised also to the spot patients in the INSPIRE study. First of all, would you give us some send, would that be all the patients still alive today? And second - or when you're going to actually conduct a study - the testing? And secondly, is there little bit more detail in terms of what aspect to be measured to attract the treatment efficiency or response? Thanks.

[Ric Woodman:]

Thank you for the questions. I think that patients on study will be of limited value for the tapestry single cell genomics analysis, in of the fact that we won't have baseline by which to understand the changes in the dynamics of the mutational profile. I think it's potentially possible that for patients who're coming into the study, it may be possible to collect samples. However, keep in mind that this is not DNA which can be obtained and stored and shipped anywhere in the world. These are single cells that are required. And when in discussions with Mission Bio, they told us that there's a limitation on what sites around the world they can get samples from in time to do the appropriate analysis. So, I think that the logistical challenges are greater with INSPIRE. It would obviously be easier to include genomic analysis in the upcoming new Phase 3 study, and we certainly have included that in the protocol exploratory endpoint with some form of genomic analysis. So I think right now a lot will be influenced by logistics.

[Yale Jen:]

Okay, great. Thanks a lot, and congrats on the progress so far.

[Steve Fruchtman:]

Thank you, Yale.

[Operator:]

Thank you. And I am not showing any further questions at this time. I would now like to turn the call back over to Steve Fruchtman for any further remarks.

[Steve Fruchtman:]

Well, thank you all for participating on today's update call, and your excellent questions which we hope we've addressed. We're very excited about the progress we're making in the INSPIRE trial and other pipeline programs. And I hope you share our enthusiasm. In addition to executing our NASDAQ-approved plan to regain our compliance with listing standards. Important milestones we look for in the near and medium-term will include, one, complete enrollment of the required 360 patients in the Phase 3 INSPIRE trial by the end of the year; two, finalization of the special protocol assessment agreement with the FDA for a pivotal Phase 3 combination trial of oral rigosertib and azacitidine in first line high risk MDS patients this year; three, initiation of a Phase 1 study with rigosertib in refractory KRAS-mutated non-small cell lung cancer; four, submission of an IND filing for ON 123300; and five and most importantly, the release of top line data from the INSPIRE trial in the first-half of 2020, following the accrual of 360 patients and 288 death events.This concludes my closing remarks. As always, we appreciate your continued support. We have some very important milestones coming up. Continue to follow us, and if you have any questions, please feel free to reach out and contact us, and thank you again.Operator?

[Operator:]

Ladies and gentlemen, thank you for participating in today's conference call. This concludes today's event. You may now disconnect.