Imunon Inc (IMNN) 2017 Q2 法說會逐字稿

Good morning. My name is Emilia, and I will be your conference operator today. At this time, I would like to welcome you all to Celsion's Second Quarter 2017 Earnings Conference Call. (Operator Instructions) At this time, I'd like to turn the call over to Mr. Jeffrey Church, Celsion's Senior Vice President and Chief Financial Officer. Please proceed, Mr. Church.

Thank you. Good morning, everyone, and welcome to our second quarter 2017 investor conference call, which we announced this morning before the market opened. During our call today, Michael Tardugno will provide an operational update on our clinical programs. And I will summarize our financial results for the second quarter and first 6 months of 2017. Today's call will be archived and the replay will be available beginning tomorrow and will remain available by phone until August 29, 2017, as well as available on Celsion's website for 90 days.

Before we begin the call, we wish to inform participants that we will be making forward-looking statements regarding Celsion's current expectations and projections about future events. Generally, forward-looking statements can be identified by terminologies such as may, should, expects, anticipate, intends, plans, believes, estimates or similar expressions. These statements are based upon current beliefs, expectations and assumptions and are subject to a number of risks and uncertainties, including those set forth in the company's periodic reports filed with the Securities and Exchange Commission, many of which are difficult to predict. No forward-looking statements can be guaranteed, and actual results may differ materially from such statements.

The information on this call is provided only as of the date of this call. And Celsion undertakes no obligation to update any forward-looking statements contained in this conference call based upon new information, future events or otherwise, except as required by law. At the conclusion of today's formal remarks, we will open the call for questions. I'd now like to turn the call over to Mr. Michael Tardugno, Celsion's Chairman, President and CEO. Mike?

Thank you, Jeff. Good morning, everyone, and thanks for joining us for today's call. With me are Dr. Nicholas Borys, Celsion's Chief Medical Officer; and Jeffrey Church, of course, from whom you have just heard, our Chief Financial Officer. As always, we are delighted to have the opportunity to update you on our progress and answer your questions. And for those of you who know me, you can probably tell from my voice, I'm fighting a cold so please bear with me. We've got a lot to talk about, and I'll try to keep the volume up.

I'd like to start by saying once again that our fundamentals are sound, our progress significant and our excitement palpable as the achievement of several important milestones were reported during the second quarter, milestones that continue to drive momentum both in and outside of the clinic. Our corporate goals remain unchanged and that is to develop novel, innovative simple therapeutic solutions to address complex unmet medical needs, all the while, pursuing long-term value for our shareholders. We do so with a great deal of focus on 2 primary clinical programs. The first is the Phase III Optima Study, where we're working to rapidly advance the evaluation of our HEAT-sensitive liposomal formulation of doxorubicin, that's ThermoDox, for the treatment of newly diagnosed primary liver cancer patients. With over 850,000 incidents, primary liver cancer, or HCC or hepatocellular carcinoma as it's known, as you know is the world's largest unmet medical need remaining in oncology.

Our second and equally important work is in ovarian cancer. Leveraging our clinical development expertise, we have expanded our product pipeline to include the emerging science of immunotherapy. A novel IL-12, that's interleukin 12 gene-mediated product candidate GEN-1, is now being evaluated exclusively in the OVATION Study of newly diagnosed Stage III and IV of ovarian cancer patients. With both these programs treating patients in first line, success with either will establish both a significant advance in medicine and, as you can imagine, a substantial return to our shareholders.

It's worth repeating what I said just a moment ago, and what I have said repeatedly during our prior calls. The fundamentals of your company are sound, without question are sound. Let me give you 3 reasons to believe. First, based on the data from 285 patients in the prior HEAT study, our highly derisked, Phase III Optima Study of ThermoDox plus optimized RFA, that's RFA conducted for 45 minutes, in newly diagnosed HCC patients is on track and on budget. The study is being conducted, and I must say in a most professional and highly efficient manner with all of the major costs and heavy lifting behind us.

We have active support from some of the most respected researchers and important institutions in the liver cancer world with over 60 active clinical sites recruiting patients. 14 regulatory authorities from across the globe, including Europe, North America and Asia, in all the major HCC markets, they have reviewed and approved the OPTIMA Study vertical. We've engaged internationally recognized world-class CROs and data management teams to ensure good clinical practice, ICH compliance, protocol adherence and high-quality data analytics. We're enrolling patients in line with our budget projections and feel confident that a 550-patient enrollment requirement will be met mid next year. Bottom line, we are less than 1 year from completing enrollment and approximately 5 to 6 quarters from the first look at efficacy data.

We have a proven and highly reliable supply chain with 3 redundant contract manufacturing organizations, that CMOs, registered and capable of producing ThermoDox in all regions of the world providing a cost structure that ALBA guarantees high gross margins regardless of the country or the local economy. And finally, the study's independent Data Monitoring Committee just recently completed a preplanned review of the first 275 or 50% of the patients randomized in the trial as of April. Based on their assessment of safety, data quality, protocol compliance and trial risk, the DMC unanimously recommended that the study continue without revision. I'd note that the DMC also commended the company for the rate of enrollment in the study with the potential to significantly expand the use of RFA in HCC.

So with virtually no operational risk and all the major costs behind us, we now look forward to study completion and data readout. And if you believe the many analyses supporting OPTIMA conducted by the company and by others and including an independent analysis of the OS data conducted by the National Institutes of Health, that's the NIH, then you have to agree that our chances for success in this very important study are quite good.

Second reason to believe is GEN-1 in the OVATION Study has shown clear signs of biological activity and is showing early signs and -- but -- early but significant trends of clinical benefit. A Phase Ib dose-escalating study evaluating our innovative, gene-mediated IL-12, that's interleukin 12 immunotherapy, known as GEN-1 and newly diagnosed ovarian cancer patients has completed enrollment with 15 evaluable patients. Clinical findings from the study were presented by our lead investigator, Dr. Premal Thaker, Professor of Medicine at Washington University in St. Louis for the 2017 ASCO Conference.

Recently, translational data from the study was reviewed with leading immuno-oncology experts at the [Roberto] Roswell Park Cancer Institute. The translational data has shown that production of therapeutic IL-12 in this patient population is dose-dependent. There appears to be a clear relationship between the dose and activation of key elements of the immune system, particularly the cytokine interferon-gamma, the protein responsible for tumor specific T-cell activation and trafficking. Importantly, early clinical findings appear to tie this translational data with the margin-negative resections that are reported in all of the patients treated at the highest dose cohort.

Third reason to believe, while we operate with virtually no mistakes or surprises, as you know, our cost structure is lean and our spending is efficient. We continue to operate with a small staff supplemented with highly professional contract research organizations. Our future spending on average will be approximately $1.3 million per month, that's approximately $15 million per year to conduct 2 major clinical studies: a global Phase III study that we just talked about, and our advanced clinical trials in newly diagnosed ovarian cancer patients. I'd say It can't get much better than that.

So to summarize, Phase III OPTIMA Study is on track. The OVATION is complete and showing exciting potential for GEN-1. And we have a cost structure, the envy of our industry. It's our value that our fundamentals are not sound. We're going to work [if not of] significance because if we're right, you're looking at the potential for billion-dollar market opportunities in both indications.

Now I'd like to go on to a little bit more detail starting with ThermoDox. And just to repeat, the Phase III OPTIMA study is now over 60% enrolled, on track to complete enrollment of 550 patients during the second quarter of next year. Our target population is newly diagnosed HCC patients. The primary endpoint is overall survival. The study is 80% powered to show a 33% reduction in the risk of death. If achieved, ThermoDox will be -- will represent the only nonsurgical curative option for intermediate HCC patients. That's a single administration of ThermoDox plus RFA, in many cases, performed in an outpatient setting, which potentially could be a curative treatment. The hypothesis for the OPTIMA study is supported with statistically significant but not preplanned -- I'll repeat it, but not preplanned, statistically significant subgroup data from our prior HEAT study, 701 patient evaluation of ThermoDox in combination with RFA.

The OPTIMA study design was derived from 285 patients from the HEAT studies whose single lesion was treated with RFAs standardized for greater than 45 minutes. And this large, well-bounded, well-balanced subgroup represented over 40% of the HEAT studies patients. A group that we filed for over 3 years, we see that treatment with the combination of ThermoDox and standardized RFA, that's RFA for more than 45 minutes, provided an average 54% reduction -- risk improvement in overall survival. The Hazard Ratio in this analysis was 0.65 with a p equal to 0.02. Simply put, this translates into a greater than 2-year survival benefit for the ThermoDox arm or the optimized RFA-only group.

And after 80 months, 80 months, the median overall survival for the ThermoDox group still had not been reached. In this population, historically, median time of death in this population is about 36 months. In our study, it was 57 months in a -- for a comparable group treated with RFA for 45 minutes. Now these conclusions and observations are not ours alone. As I mentioned, the NIH conducted an independent analysis of all single lesion patients, more than 60% of the study population. They concluded that the longer RFA heating times when combined with our ThermoDox, resulted in a statistically significant improvement in overall survival. That was not true for the RFA alone, so RFA without ThermoDox.

So an important key quality measure also reviewed by the DMC was compliance with the minimum of 45-minute RFA treatment specified in the protocol. We were asked about this compliance rate, and so I am pleased to report to you that there has been 99% plus compliance rate with this study specific requirement. In addition to maintaining our focus on quality, execution of the OPTIMA Study we're also highly focused on the commercial opportunity for ThermoDox, which is led by our regulatory strategy. ThermoDox has received FDA Fast Track Designation, which provides, from among other things, priority review. ThermoDox has been granted orphan drug designation for primary liver cancer in both the U.S. and Europe which expands market exclusivity for 7 and 10 years, respectively, in these major revenue markets.

Based on our prior discussions and subject to a successful trial, we have designed a study to enroll sufficient number of patients from each race or country to support registrational filings in the U.S., Europe, China, South Korea, Taiwan and Vietnam among others. The China FDA, that's the CFDA informed Celsion in a face-to-face meeting that if the ongoing Phase III OPTIMA Study's success from the trial could serve as a basis for a direct regulatory filing in China without the need for a prior approval in the U.S. or Europe, which is typically required, this will allow, as I pointed out before, the company to accelerate its plans for regulatory filing in China. And if approved, provide for a significantly earlier launch in China than it was originally expected. Why is this important? Well, China represents the most -- perhaps, the most significant market opportunity for ThermoDox globally as 50% of the 850,000 new cases diagnosed each year originate in China, that's 425,000 approximately new incidents of HCC in this -- in their country in this market. The future largest market for pharmaceuticals in the world, I would say, safe to say that we are well positioned.

Now I'd like to turn to GEN-1 for a little bit more detail here also, our immuno-oncology candidate developed on our TheraPlas platform. GEN-1 is a gene-mediated immunotherapeutic which recruits the entirety of the immune system to fight malignancies. GEN-1's active agent is a DNA plasmid coded for the cytokine interleukin 12, as we've mentioned earlier, IL-12 plasmid is incorporated into a nonviral nanoparticle delivery system. That delivery system is known as TheraPlas. When administered locally into a body cavity like the peritoneum or the bladder or even a cavity that's been created by the surgical removal of a tumor mass, the nanoparticles invade surrounding cells and take over the metabolic machinery, then turning each cell into a mini factory for the sustained local production of IL-12.

Our first indication for GEN-1 is ovarian cancer. For patients newly diagnosed with this advanced malignancy, there's less than 45% chance of a 5-year survival, one of the major reasons this diagnosis is made when patients are symptomatic at Stages III and IV, when the cancer has metastasized. In previous clinical trials, this patient population -- within this patient population, GEN-1 has been used either as a monotherapy or in combination with chemotherapy with some promising results. As indicated earlier, Celsion's first trial on this indication, that's one that we sponsored after acquiring the GEN-1 asset we call the OVATION Study, is now fully enrolled. Clinical findings in translational data are being analyzed with the final review of all the data by our Scientific and Medical Advisory Committee in September.

The OVATION Study enrolled 15 evaluable newly diagnosed Stage III and Stage IV ovarian cancer patients, all patients presented with heavy disease burden. Prior to the debulking surgery, patients were treated with standard platinum and taxane. The goal of which is to improve surgical outcome by shrinking the tumor and reducing the accompanied fluids known as ascites. To this regimen, we added 8 weekly cycles of GEN-1. Following treatment with GEN-1, patients then received interval debulking surgery. So I'd like to re-summarize the early -- I'd say, early, but remarkable, nonetheless, results that we have seen so far. And we'll continue to update you with this information as it becomes available.

Of the 14 evaluable patients treated during the entire trial, we saw 100% disease control rate, 86% objective response rate as measured by research criteria, 2 patients demonstrated a complete response, 10 patients a partial response and 2 patients demonstrated stable disease of their cancer. Of the 5 patients treated in the highest dose cohort, there was 100% objective response rate with 1 complete response and 4 partial responses. All 14 patients had successful resections of their tumors, with 9 patients with 64% having an R0 resection, which indicates -- R0 indicates a microscopically margin-negative resection in which no growth or microscopic tumor remains in the tumor bed. Of the 5 patients treated at the highest dose cohort, all 5 patients, 100%, experienced an R0 surgical resection. 7 of the 8 patients, 87%, in the highest 2 dose cohorts experienced an R0 resection. All patients experienced a dramatic and clinically significant decrease that was over 90% in their cancer antigen 125 protein, that's CA-125 protein levels, as of their most recent follow-up. And with the average decline for all patients treated at 97% -- remarkably at 97%.

CA-125 is used to monitor certain cancers during and after treatment. CA-125 is present in greater concentrations in ovarian cancer cells and other cells. Now what's clinically significant here is a 50% reduction. So I'll just compare the 2. We had over a 97% average decline in CA-125. 50% reduction is considered clinically meaningful. Of the 7 patients who received GEN-1 treatment over 1 year ago and are being followed, only 1 patient's cancer has progressed after almost 12 months. It's 11.7 months. This compares favorably to the historical median progression-free survival of 12 months for newly diagnosed patients with Stage III and IV ovarian cancer. Of the remaining 6 patients who have been on this study for over a year, the PFS is over 16 months. That's the longest progression-free patient at 22.4 months.

So we also just reported the latest translational data for 12 of 15 patients which indicated convincing evidence of IL-12 for gene transfer and immune system activity. Data from the remaining 3 patients will be incorporated in September and reviewed by our Scientific Advisory Committee. A few weeks ago, we reported that translational data was -- this translational data was reviewed with leading immuno-oncology experts in Roswell Park Cancer Institute, and I will summarize as follows. The treatment-related changes in the immune system activating cytokines like interferon-gamma and pro-tumor VEGF levels, follow a dose-dependent trend and were predominantly in the peritoneal fluid compartment with little or no changes observed in the patient's systemic circulation. We saw pronounced decreases in the density of immunosuppressive Treg cell signals like -- Treg cells like the FOXP3 and PD-L1 and increases in CD8 cells in the tumor microenvironment. The ratio of CD8 cells to immunosuppressive cells was increased by approximately 75%. While it was increased in approximately 75% of patients suggesting an overall shift in the immune tumor environment from immunosuppressive to pro-immune stimulatory, an increase in the rate of CD8 to immunosuppressive T-cell populations is a leading indicator and believed to be a good predictor of improved overall survival. These translational research findings demonstrate that GEN-1 in ovarian cancer patients is biologically active and promotes a pro-immune T-cell population dynamic in the tumor microenvironment. More importantly, these distinct immunological changes in the local disease environment appear to translate into clinical benefit and one that continue the development of GEN-1 as we will do.

Now to summarize, GEN-1 and ovarian cancer patients is biologically active, appears to have an immuno-stimulatory effect in the peritoneal fluid in a dose-dependent manner and promotes a pro-immune T-cell activity in the tumor tissue itself. So our next steps in our clinical strategy following review by our Scientific Advisory Board will be to outline our Phase II program for GEN-1, which could likely continue in the current population of newly diagnosed patients while they were considering the alternative in platinum-resistant patients. But we'll make that decision following the review by the Scientific Advisory Group. Following that, we, of course, will submit our protocol for Phase II program with the NDA, hopefully, before the end of the fourth quarter.

So with that, those are my prepared remarks. I'll now turn the call over to Jeff to review our financial results. Jeff?

Thank you, Mike. During the second quarter, we continued to efficiently utilize our cash as we effectively execute our clinical development initiatives. We ended the second quarter with $3.6 million of total cash and investment. That included the exercise of outstanding warrants during the second quarter totaling over $5 million.

Subsequent to the end of the quarter, this is in early July, we announced the completion of a $5 million registered direct equity offering with several institutional investors. This capital infusion should cover our operating needs for the balance of 2017, at which time we will be less than 6 months from completing patient enrollment in our pivotal Phase III study in primary liver cancer and about 4 quarters from the first planned interim efficacy analysis. This significant value inflection for the company is now coming into view.

Celsion's 2017 second quarter financials were included in the press release, which we issued before the market opened this morning. Our Form 10-Q for the quarter ended June 30, 2017, was filed on Monday, August 14, after the market closed. We continue to monitor our cash expenditures to ensure the most efficient use of cash to create shareholder value. Our clinical development focus is squarely on the enrollment of our pivotal Phase III trial for ThermoDox in primary liver cancer and the earlier-phase studies for GEN-1 in ovarian cancer. Operating expenses were $9.6 million in the first half of 2017 compared to $10.2 million in the same period of 2016. Cash used for operation in the second quarter ended June 30, 2017, was $4.2 million compared to $4.3 million in the prior year. Cash used for operations in the 6 months ended June 30, 2017, was $7.3 million and that compared to $9 million in the prior year.

These results are in line with our earlier projections and are the result of our cost reduction efforts implemented during 2016, a tighter product development focus as mentioned above and prudent cash management. Also, during the second quarter, we paid off our venture debt facility with Hercules. The company currently has no debt on its balance sheet. We continue to operate with a lean organizational structure which now has less than 20 full-time equivalent employees. As spending is directed to research and development activities, we expect our current -- quarterly cash used for operations to be under $4 million per quarter for the balance of 2017 and 2018. As we look forward, we believe that maintaining a strong balance sheet is important to continue the strong development momentum we have built around our lead product programs.

For the second quarter ended June 30, 2017, we reported a net loss of $4.9 million compared to a net loss of $4.5 million in the same prior year period. For the 6 months ended June 30, 2017, our net loss was $10.1 million compared to a net loss of $10.2 million for the same 6-month period in 2016. We continue to evaluate our organizational structure and have aligned our resources and clinical programs with our near-term development objectives. R&D costs were $3 million in the second quarter compared to $3.3 million in the same period last year. R&D costs for the first 6 months of 2017 were $6.5 million compared to $6.8 million last year. Our research and development expenditures in the current year are focused on continuing the enrollment and treatment of patients in the Phase III OPTIMA Study and completion of enrollment and follow up of patients in the Phase I OVATION Study using GEN-1 to treat ovarian cancer.

General and administrative expenses for the second quarter were relatively constant when compared to the prior year about $1.6 million this year compared to $1.5 million last year. G&A expenses were down $300,000 in the first half of 2017 when compared to the same period in 2016. This 10% decrease was primarily the result of lower personnel and operating costs resulting from the reorganization and staff reductions announced in 2016, lower insurance premiums and professional services cost and a tighter clinical focus on those programs that will drive shareholder value in the near term through the readout of our pivotal Phase III OPTIMA Study.

Other nonoperating expenses increased by $530,000 in the second quarter and $330,000 in the 6-month period ended June 30, 2017. Primarily as a result of a noncash, I want to emphasize, noncash adjustment to our earn-out milestone liability, this was offset by lower interest expense on our venture debt facility with Hercules. As I mentioned earlier, this loan facility was fully paid off in June 2017. The second quarter of 2017 also included a deemed dividend charge related to the repricing of certain outstanding warrants during the month of June. As I mentioned earlier, the company received over $5 million in gross proceeds from the exercise of warrants during the quarter. Included in this total are approximately 1.3 million warrants which were repriced and then immediately exercised which yielded over $3 million in gross proceeds to the company. These warrants were previously registered and were included in our fully diluted capitalization.

I will now turn the call back to Mike.

Sorry, I was -- we were on mute. Okay, so I will just repeat just the concluding remarks. The Phase III OPTIMA Study is on track. OVATION is complete and showing exciting potential for GEN-1. As Jeff points out, our cost structure, well managed and the envy -- I would say, the envy of our industry. We are looking forward now to continuing our work. So with that, I'd like to open the line for questions. Operator, if you would. And I'd ask those on the line, if you'd limit your questions to no more than 2, so that we can provide everyone an opportunity to participate in the Q&A. Operator?

(Operator Instructions) And we'll go first to Hartaj Singh with Oppenheimer.

This is Emma on for Hartaj, actually. With regard to GEN-1, you mentioned in the past there was interest in evaluating its potential in a post-surgery setting to delay or prevent tumor recurrence. Could you just give us any update or color on the latest thinking there and how that might fit into the clinical development plan?

I'm sorry, I just missed the first part of your question. If you just repeat that, please?

Sure, so on GEN-1, you'd mentioned in the past that there was interest in evaluating its potential in a post-surgery setting to delay or prevent tumor recurrence. So just any update on the color or latest thinking there on how it might fit into the clinical development plan?

I see. So as a maintenance therapy, I mean, follow-up surgery continually to treat patients. Nick, do you want to...

As you know, there's plenty of roles for immunotherapeutic therapies. And we're looking for the ideal fit for GEN-1. So in this last set of clinical studies that we've done, we're now looking at -- in this neoadjuvant setting where we have the tumor at the time of enrollment of the patient. We get the biopsy of the tumor, and then we get follow-up on the patients once the tumor has been removed in that interim surgical setting. So I think at the end of this, we're going to have pretty a good idea where is going to be the strong points for GEN-1 and the effect it directly has on the tumor. So I think, again, the message here is watch the space. And I think there might be an interesting role there.

Yes, I think if I just could add something. I was reading in the recent [JCO] some -- an article regarding some of the stem cells, the pre-cancer -- cancer-related stem cells that go untreated like chemotherapy. And they seem to find a way to continue to evade the immune system -- come the source of metastases. I mean, there's some suggestion here that immunotherapy, maybe GEN-1, maybe one of those therapeutics that has the potential to address those cancer-related stem cells that go untreated when the primary disease has been addressed. So that's going to be part of our discussion with the Scientific Advisory Committee in September.

(Operator Instructions) And we'll go next to Jason McCarthy from Maxim Group .

Two questions. One -- so first off, Mike, you were saying you're about 60% enrolled of the 550 patients now and you're on target for second quarter '18 to complete enrollment. Can you just review with me the timing for completion of enrollment? And after that, when would you expect the first interim look at the data?

Yes, good question. Thank you. So over 60% enrollment, 60% of 550, you could do it with your pencil, it's 530 patients -- more than 530 patients, quite a few more actually. Our enrollment rate target has been exceeded. Actually in the last 3 months, we have been planning about 18 patients a month. We're over 20 patients a month. And you can kind of do the math yourself. If that continues, Jason, we should be in the middle of the second quarter of next year completing enrollment in the study. And it could even get better. Nick Borys has been very anxious. Dr. Borys has been very anxious to initiate a few more new sites. We've commissioned our CRO in Vietnam to add 2 more sites. And we're working with the Chinese CRO whose attached to our global CRO to add 5 more sites in China. So there's really every reason for us to believe that the enrollment rate can pick up. So we're actually quite pleased with where we're at after actually experiencing an over 1 year delay in getting study approval in China. Of course, we weren't the only ones. The entire world waited for China to reorganize the CFDA to accept the new protocol applications. So the death rate is really what -- and that's a horrible way to think about it, I guess. But the event rate is key to the first interim data look. On the first interim look, we'll be at 118 events, Jason. We're looking for about a hazard ratio of about 0.62, I believe, p equal to 0.05 to unblind the study. The data that supports that, of course, comes from the HEAT study in this very similar population. Not as well controlled as we are controlling in the -- I would say in the OPTIMA Study where the -- this patient population treated with more than 45 minutes saw a hazard ratio of 0.65 with p equal to 0.02. So there's an odds-on chance here that at 118 events, we would be courting a success. If not, we have an -- as you know we have a second interim analysis followed by the final analysis. So the question was when will we get that first look? Obviously, the DMC, the meeting was just recently completed. We know the number of events. I'm not going to share them in this call. The number of events are evaluated based on the enrollment rate. Our statistician gives us prediction of one that 118 number will be hit. So our thinking here -- our best thinking here is about the 2 quarters following full enrollment of the study, and that could change. And we will give you an update in the next quarter based on -- we'll have, actually, I'd think a firmer handle on the number of events in the next quarter. So that's our best thinking at this point, Jason.

Okay, great. And just the split between China and the rest of the sites. How many patients were Chinese-based patients? Do you need for China FDA to allow you to file there?

Yes, so the official line on that is 200 patients, 100 pairs of Chinese patients. And we're recruiting patients in Hong Kong, Mainland China and in Taiwan, although Taiwan has been the subject of some political discussion. Apparently, Taiwanese Chinese are not China Chinese in some halls of the CFDA. That being said, we had a very productive conversation with the Deputy Director of CDE of the CFDA interoffice, Dr. Borys, and I. Nick gave an overview of the study and what we were trying to achieve in those. And they actually are focused on the China population -- the Chinese population from the HEAT study and her comments to us were I think very -- not only surprising, very supportive. She indicated that our -- since this is such a health emergency in China that we will not have to wait for a -- if the study is successful and if we hit our end points, we will not have to wait for a CPP from either the U.S. or Europe which is typically required before filing an MDA. We could file immediately. That was I think a blockbuster comment from her. She also indicated that the 200 patient requirement was under review. And so we'll know more, I think, here in the next month or so, we intend to -- Nick and I, along with some others, will be in China in September to meet with our investigators as we do annually. And we want to take some time to try to sort this out a little bit more. But 200 patients is the official line. We understand that's evolving. And it may be -- we may be allowed to include Chinese patients in from other countries like Taiwan, certainly from Hong Kong.

And we'll go next to Keith Markey from Griffin Securities.

Two questions. One, I was wondering if you have -- if any of the GEN-1 patients that had the R0 resection have progressed with their disease? Or are those patients still disease-stable, I guess, or free?

I don't know the answer to that.

As far as we know, all the R0 patients are still -- have no evidence of disease. So we're following them very carefully on that.

And the progression in this population is really interesting. When we discussed PFS with our study investigators, they suggest to us that we should expect a normal distribution like a Gaussian curve. And that would mean, for example, some patients will progress after 2 months, some patients will progress after 6 months, some patients 9, some patients 12, 15, 18, 20. Then we get the median is 12. In our -- we have not seen any progressions at the -- in early marks. The only progression we have is at just about 12 months. And the rest of the patients now treated over 12 months as I pointed out. I mean, this whole thing is skewing to a positive PFS result. Now that -- certainly that could change. I mean, I don't want to get ahead of myself here. But -- and if it does, of course, we will be very fairly open about it. But PFS, the leading indicator of overall survival, is very encouraging at this point.

Very good. And then I was wondering if there might be any news from the NIH about the HIFU study they're doing?

[Within] the Children's Hospital?

Yes.

Yes, I think -- I'll report on that one. This has just been a pet project for Dr. Borys. We've been very excited about it. And frankly, it's unfortunate, that I have to report to you that we are not aware of any patients who have enrolled in the study.

(Operator Instructions)

Okay. Well, I want to thank everybody for your time this morning. I apologize again for the quality of my voice. Hopefully, I get over this cold pretty quickly. And just again to conclude, we remain excited -- not remain, we are very excited about the potential for both these programs in chemotherapy and this legacy of oncology with our innovative product, ThermoDox. Not only are we excited -- we're excited, but the NIH, our investigators, the world is looking forward to the results for this ThermoDox study in primary liver cancer. And in immunotherapy, where we're seeing some early and new trends. We don't want to overstate it early. Nonetheless, remarkable clinical findings so far in our Phase I program moving quickly to Phase II once our Scientific Advisory and Medical Advisory Group meet in September. On all of that, we value your continued support, and we thank you very much for joining us on the call. Our promise to you is that we will keep you updated. And our promise also is to continue to work to deliver innovative oncology therapeutics to address some of the most important prevalent cancers with the highest unmet need. Thank you again for joining us on today's call. And with that, we'll adjoin the telephone call. Thank you.

Ladies and gentlemen, this does conclude our conference for today. Thank you so much, for your participation. You may now disconnect.