Celsion's (CLSN) CEO Michael Tardugno on Q2 2014 Results - Earnings Call Transcript

Aug. 8.14 | About: Celsion Corporation (CLSN)

Celsion Corporation (NASDAQ:CLSN)

Q2 2014 Earnings Conference Call

August 7, 2014 5:00 PM ET


Jeffrey Church – SVP, CFO and Corporate Secretary

Michael Tardugno – President and CEO


Keith Markey – Griffin Securities

Reni Benjamin – H.C. Wainwright & Co

Bob Green [ph] – Private Investor


Good morning. My name is Tony, and I will be your conference operator today. At this time, I’d like to welcome everyone to the Celsion’s Second Quarter 2014 Financial Results Conference Call. All lines have been placed on mute to prevent any background noise. After the speakers’ remarks, there will be a question-and-answer session. (Operator instructions)

I would now like to turn the call over to Jeffrey Church, Senior Vice President and Chief Financial Officer of Celsion. Please proceed.

Jeffrey Church

Thank you. Good morning, everyone, and thank you for joining us today to discuss our second quarter 2014 financial results, which we announced this morning. Today’s call will be archived. The replay will be available beginning today at 2:00 P.M. Eastern, and will remain available by phone until Thursday, August 21, 2014. And will also be on Celsion’s website for 30 days.

Before we begin the call, we wish to inform participants that forward-looking statements are made pursuant to the Safe Harbor Provision of the Private Securities Litigation Reform Act of 1995. You are cautioned that such forward-looking statements involve risks and uncertainties including, without limitation, the risk of clinical failures, delays or increased costs, unforeseen changes in the cost of our research and development activities; possible acquisition of other technology assets or businesses; and possible adverse action by customers, suppliers, competitors, regulatory authorities and other risks detailed from time-to-time in the company’s periodic reports filed with the Securities and Exchange Commission. Following our formal remarks today, we will open the call for questions.

I’d like to turn the call over now to Michael Tardugno, President and CEO of Celsion. Mike?

Michael Tardugno

Thank you, Jeff. Good morning everyone. We have a lot to discuss today, so I’ll get right to it. Before doing so, I want to remind you, I’m joined today by Jeff Church, from whom you’ve just heard, our CFO and Senior Vice President. Dr. Nick Borys and Anwer, who normally would be with us for this call, are not today. They are attending on a business, but they will be on future calls, I can promise you that.

So welcome once again to our quarterly earnings call, and to the new Celsion, the company that all at once, with the acquisition of EGEN, this marvelous science-based company in Huntsville, Alabama, all at once is a fully integrated development company with R&D capability from feasibility through NDA, with platform technologies in chemotherapy, immunotherapy and RNA therapy and clinical trials in Phase III, Phase II and Phase I, in some of cancers globally most important forms, we are extraordinarily well positioned.

Celsion now has multiple opportunities to create value for our shareholders, and represents a development company. I’m sure that you will agree by the unique proportion.

On the development front, as our recent news fore points up, we’re having an extremely productive year thus far, and one that I suspect will continue to demonstrate significant progress, not only with ThermoDox for primary liver and recurrent chest wall breast cancers, but also with our equally important immunotherapy and RNA platforms.

If I can take the liberty now to quote one of our great presidents, “To Those, Whom Much Is Given, Much Is Expected” then I’d add, particularly this morning, with our EGEN acquisition, you should expect much from Celsion. So here is a shortlist. And it reminds the shortlist that what we will be reporting over the next 12 months.

From our first immunotherapy candidate EGEN-001, which I’m going to refer to as GEN-1. By the end of the year, we’ll be reporting translational data from our Phase Ib study of platinum-resistant ovarian cancer patients, demonstrating the expression of IL-12, and the activation of an associated potent multi-mechanistic anti-cancer immune response. Whilst from the same study will be available early next year.

We’re also fully expect to report regulatory support from our planned Phase II study in first-line ovarian patients in the first quarter of next year. In a second indication for EGEN-001, one that could very well become a priority for the company, we will be reporting substantial pre-clinical data in support of our planned IND submission for the GBM glioblastoma ovarian cancer indication.

Again, we fully expect that the data will be followed by FDA agreement for a Phase I study in post-resection GBM patients. For this, we are targeting the second quarter of 2015.

From our second product candidate, a PPC, that’s a polyethylenimine nanoparticle that has the unique capability for delivering Messenger RNA, as well as other long and double chain nucleotide sequences.

From this second product candidate, we plan to report data from a non-human primate study. The study is designed to collaborate in the second species, data which reliably demonstrates highly priced and clinically very relevant Messenger RNA lung expression. Lung expression of RNA is a characteristic uniquely associated with our proprietary PPC delivery technology, making it a very attractive delivery platform.

And from our ThermoDox studies, you should expect ongoing data from the DIGNITY study, which if it continues to show overwhelmingly positive tumor response results, we will approach FDA to discuss next steps. And if you recall from prior written agreements, we know that FDA will accept local tumor control as the registrational endpoint in this unfortunate highly refractory end-stage population.

And of course we will provide regular DMC updates from our Phase III OPTIMA study in primary liver cancer, a study that is currently recruiting patients.

Now for those of you who are new to our company, I’d like to spend a few minutes in our strategic asset acquisition purchase, the deal which by anyone’s definition is incredibly investor friendly. I’d ask Jeff to discuss the deal terms in a little bit more detail when he covers the financials.

In June, we announced and completed our acquisition of EGEN, a privately-held company whose primary focus is the development of nucleic acid-based therapeutics for the treatment of cancer among other therapeutic areas. This transformation will then establish Celsion, as I have said, as a fully integrated oncology company. It provides us with programs and the leading edge of cancer research and with R&D talent and competences end-to-end, from drug discovery through to commercialization.

With this deal, we have improved the depth of our technology and research base, and have created a new trajectory for Celsion in three distinct ways. First, we now have multiple technology platforms, three to be exact, from which to generate future growth. They include of course, our proprietary heat sensitive liposomal platform, exclusively we licensed – with exclusive licensee from Duke University, LTSL platform is the technology underpinning our ThermoDox product.

TheraPlas is the second platform. TheraPlas is a synthetic non-viral nanoparticle for the delivery of double stranded DNA pharmacologic immunotherapy products, and for single or double stranded therapeutic RNA.

And third, TheraSilence, a liposomal nanoparticle for the delivery of siRNA and miRNA or microRNA.

Secondly, we now go to the pipeline that includes a robust pre-clinical development program in earlier stage research that represent potential leading edge technology to treat difficult disease targets.

And third, we have multiple product candidates at the clinical level, which I will now review. Let’s start with ThermoDox.

Our Phase III OPTIMA study is designed to evaluate ThermoDox in combination with standardized radiofrequency ablation or sRFA in hepatocellular carcinoma or HCC, sRFA for HCC. The study will enroll approximately 550 patients globally and up to 100 sites in North America, Europe, China and Asia Pacific. It is a two-arm, double-blinded randomized study comparing ThermoDox in combination with standardized RFA or sRFA, which will be standardized across a minimum of 45 minutes across all investigators in the study versus standardized RFA alone.

The primary endpoint is overall survival. The study is pilot to show a 33% improvement in OS, and I want you to keep 33% in mind, we’re going to refer back to that in a minute.

The statistical plan calls for two interim efficacy analysis by an independent data monitoring committee. The office spend is de minimis for these two lines.

The support for OPTIMA is significant and comes from; one, an exhaustive retrospective analysis of the previous Phase III HEAT study; two, it’s based on convincing post-hoc OS data; and three, it’s further reinforced with prospective confirmatory pre-clinical data in computational models.

Post-hoc findings and hypothesis supporting OPTIMA do not represent our view alone. The data were shared along with our RFA learnings, in particular, with HCC research community during major international medical conferences.

In the final analysis, we have received full support, and I would say, share a great deal of optimism for the OPTIMA study with virtually all of the most important names in HCC research worldwide. I think with some modestly, I’d like to say that, we now know more about the use of RFA to treat the intermediate stage lesions in HCC than just about any company on the planet.

What we’ve learned from the study is this. For tumors greater than three centimeters, RFA device limitations have to be considered. To be effective, and that is to fully destroy the tumor and create a clean margin multiple overlapping ablations creating longer dual times are required.

When we say dual time, that is the achievement time greater than 45 minutes, also improves the heat radiant to a larger volume and allows for increased local ThermoDox profusion, and ultimately a high tissue concentration of doxorubicin. Longer times plus increased dox ensure a better outcome. The findings are convincing.

The most recent clinical evidence supports this thesis. It comes from our June 30 quarterly Overall Survival sweep of the HEAT study patients. In this subgroup of 285 patients, who received standardized RFA treatment, 285 represents 41% of the total study, we noted an 57% improvement in OS in the ThermoDox plus sRFA arm versus standardized RFA alone, 57% improvement.

I want you to reflect back in my earlier comment regarding the primary endpoint of Overall Survival that we’re planning for the OPTIMA study. In that case, we were looking for 33% improvement. So the evidence in our post-hoc analysis suggests that we should be able to meet that target quite easily, quite handily.

So 57% improvement versus sRFA alone, that’s over two years improvement in survival. That’s pretty astounding. Two years. A bullet for wedding and graduation. If you recall, Nexavar or sorafenib was approved on just 11 weeks OS benefit. And while there should be caution since this is a retrospective analysis, these findings are nonetheless striking in that they have one remain constant over each of the six quarterly dead sweeps.

They demonstrate impressive confidence with a p-value of 0.037. The magnitude effect is unequivocal. This is not a borderline improvement. Well wobble in the data could have an impact. These data are further supported by multivariate Cox Regression Analyses, which do not discount in anyway sRFA plus ThermoDox, the leading factor for the OS benefit.

The goal of OPTIMA is to support registration in key liver markets worldwide. To that end, we’re working with multiple agencies, hopeful regulatory agencies to advance our global registration strategy.

In addition to the U.S. FDA clearance that we received in the first quarter of this year, we have a received approval to commence the study in sites in Taiwan, Hong Kong, South Korea, Malaysia and Canada. And we have also submitted an application for accelerated approval to the China FDA, Food and Drug Administration. The CFDA has reviewed the HEAT study data, with Dr. Borys and myself, including the subgroup of patients treated in China and it’s committed to working with us to initiate the OPTIMA study in China as quickly as possible.

In Europe, we are taking advantage of the Harmonization Process, which allows for a central and parallel approval of the key countries including Germany, France, Italy and Spain. In addition, we plan to meet with the EMA later this year to discuss our ThermoDox MAA or marketing approval filing strategy.

Beyond HCC, we are evaluating ThermoDox in the Phase II DIGNITY study. A ThermoDox plus hyperthermia in recurrent chest wall breast cancer. As you know, we continue to report very impressive results in this, difficult to treat, refractory population, so patients post-hysterectomy, who have failed at least two lines of chemotherapy and have failed radiation before enrolling in our trial.

In July, we reported updated interim findings demonstrating that a local response rate of greater than 50% has been observed in the first patients with refractory disease, notably with three complete responses. I’d also point out that these findings are consistent and have been consistent with the objective responses that we’ve seen in two previous Phase I studies accounting for some 27 patients.

Additionally, we are supporting a broad range studies using HIFU to provide ThermoDox activation in multiple indications. The most advanced program is now recruiting patients in a Phase II liver met study in Britain, followed closely by a breast cancer study, which we hope to initiate in the Netherlands. Both of these programs are co-founded and co-funded in Europe by government grants. One grant of which is up to $10 million.

Now I’d like to turn our attention to EGEN-001. EGEN-001 is our Phase II ready immunotherapy candidate for the treatment of ovarian and brain cancer. It is an IL-12 DNA plasmid encased in a PPC nanoparticle delivery system. The system promotes transaction of the plasmid into cells, which then use the cellular mechanism to enable persistent and durable local secretion of the IL-12 protein, the interleukin-12 protein, which lasts for several days or weeks depending upon the target.

As many of you know, IL-12 is one of the most active cytokines for the induction of a potent anti-cancer immunity response. It acts through the induction of T-lymphocyte, a natural killer self-proliferation and has also shown an anti-angiogenic effect, and has demonstrated a potential in a variety of cancers as a recombinant protein and as a protein that’s manufactured ex vivo in fermentation plants [ph].

This ex vivo that’s outside of the body produced IL-12, and when used it locally systemically to treat cancers has poor pharmacokinetics and is associated with serious toxicities. Scientific evidence collected today have shown that EGEN-001’s potential to overcome these limitations by controlling the expression of IL-12 in vivo, we see very encouraging safety profile and similarly encouraging indications of efficacy.

Phase I clinical studies have established the safe therapeutic dose, and have demonstrated strong disease control rates in a tough to treat platinum-resistant ovarian cancer population. 001 has been safely combined with first-line chemotherapies, carboplatin and docetaxel in platinum-sensitive ovarian cancer patients. While the end was small, we noted a positive disease control in OS rates in this population.

We are currently completing a Phase Ib trial in platinum-resistant ovarian cancer patients, under direction of the GOG, that’s the Gynecologic Oncology Group. By year-end, we will be reporting translational data, demonstrating the expression of IL-12 and the activation of the associated potent multi-mechanistic anti-cancer immune response. In early next year, we should be able to assess an OS signal.

The data today strongly support a rationale for advancing EGEN-001 into a Phase II combination trial with the goal of targeting first-line plus the standard of care or activation of immune response is frequently more effective for subjects whose immune system is healthier and less compromised by previous chemotherapy. We’re on track to meet with the FDA regarding our Phase II plan.

We’re hoping to do so by year end, with FDA concurrence in the first quarter of next year, we could be treating patients in the second quarter. I’d also point out that applications for EGEN-001 extend beyond ovarian cancer. In parallel with our ovarian program, we are working on a second and perhaps the most exciting clinical research for the company and that is a development program in glioblastoma multiforme or GBM.

As I have said, this could well become a priority for Celsion. As you know, a small well-designed study may provide for registrational opportunity or approval, we’ll see. Our pre-clinical studies have demonstrated that administration of EGEN-001 in the brain can lead to an IL-12 expression that lasts for at least one month. So we are fast moving towards an IND in this indication, supported by follow-on pre-clinical proof of concept safety and bio-distribution studies which are now in the last planning stages.

Our goal is to submit an IND briefing book and protocol by year-end and plan for Phase I study in the first half of next year. The study design contemplates combining IL-12 immuno activation with the standard of care chemotherapy in post reception patients. OS of course would be an ideal endpoint, particularly in this population.

You may also know that the EGEN-001 is a non-viral approach, which could accelerate or should accelerate, regulatory acceptance over our other approaches under development.

Now I’d like to turn to EGEN-002, an RNA therapeutic based in our TheraSilence platform, which is at the concept stage. At this concept stage, setting the basis for partnering or collaboration. EGEN-002 is a combination of two unique molecular targets for cancer therapy and has the potential as a treatment candidate for lung cancer. The concept uses a two-pronged approach; a combination designed to inhibit tumor growth by anti-angiogenesis, and promote direct killing of the tumor with microRNA. We have demonstrated evidence of siRNA delivery and gene silencing into lung. And with this approach and look forward to advancing pre-clinical development of this program.

Beyond our own clinical development efforts, we are also seeking to leverage the TheraPlas and TheraSilence platforms externally. You should know that we have a current joint development program focused on lung application of messenger RNA, the next milestone of which is to collaborate urine [ph] data in a non-human primate study. Some very exciting news could come from this project near-term.

From our press releases, you know that we recently launched the commercialization of our reagent products based on these two validated platforms, that’s TheraSilence and TheraPlas, and believe we can generate most revenues from this business to help support our maturing oncology pipeline, but more importantly however, reagents sales to leading research and academic centers will serve to validate our proprietary non-viral vector delivery platform.

So you can see we have a very active clinical development effort focused on three promising candidates, with the leading edge of cancer research, and we look forward to updating you on our progress.

So now I’ll turn the call over to Jeff for a review of our financials. Jeff?

Jeffrey Church

Thank you, Mike. Before I review the financial results for the second quarter, I would like to discuss the economics around the EGEN acquisition. The acquisition broadens our technology platforms and product pipeline in two of the most exciting areas of drug development; immunotherapy and RNA therapy. The acquisition will help mitigate the risks associated with the single-product single-technology platform.

The negotiated deal terms as Mike stated are user-friendly and represent a deal structure that is one of shared competence and shared risk. The EGEN investors fully share development risk with us. The initial upfront payment consisting of $3 million in cash and $10.6 million in common stock represented 30% of the total considerable for the acquisition, and put no pressure on our balance sheet.

We completed the transaction on June 20 and ended the second quarter with over $15 million in cash. Our venture debt lender, Hercules Technology Growth Capital, after completing their due diligence and reviewing the transaction fully endorse the acquisition to a second $5 million drawer of our existing limit facility.

This cash infusion more than covers the upfront cash payment and our transaction-related expenses. In addition to the upfront payment, a total of $30.4 million in well-defined value-creating development milestones will triggers set [ph] base earn-out payable to EGEN’s shareholders based upon the completion of important clinical objective in our ovarian and GBM studies for EGEN-001 and joint development and clinical progress with TheraSilence, including partnering and out-license initiatives.

70% of the acquisition price is tied directly to contingent value-creating milestones. If these milestones are achieved, both, Celsion and EGEN shareholders win. This transaction is user-friendly, win-win, risk-mitigating and value-creating.

I would now like to turn to the second quarter financial results. Starting with cash, we reported total cash and investments at June 30, 2014 of $50.1 million, as compared to $43.8 million at the end of 2013. Our cash position reflects the proceeds from a registered direct common stock offering in January 2014, along with the second drawer in June of $5 million under our Hercules loan facility.

Our current cash balance puts us in a strong financial position to advance the development of our expanded product pipeline. Our projected cash usage is estimated to reach $2 million per month, with the full implementation of the OPTIMA study, and the integration and clinical development of the newly acquired assets from EGEN. Our current cash is projected to carry up into the second half of 2016, well pass the announcement of many key development and partnering activities.

Net cash used for operations was $9 million for this first six months of 2014, or approximately $1.5 million per month. This compares to $3.4 million for the same six month period in 2013. This increase was driven by a $5 million non-refundable cash payment, which we received from Hisun Pharmaceutical Company in the first quarter of last year, coupled with a $600,000 increase in operating expenditures in the current year, largely a result of startup costs for our Phase III OPTIMA study, which included site initiation expenses and the manufacture of clinical drug supply.

Research and development costs were $6.1 million in the first six months of 2014. That’s an increase of $833,000 compared to the prior year, due again to the above mentioned increase in costs associated with starting up the OPTIMA study.

Our G&A expenses were $4.7 million for the first half of 2014 versus $3.6 million for the same period in 2013, primarily as a result of higher insurance premiums and personnel costs, including an increase of $900,000 in non-cash stock-option expense.

The second quarter of 2014 also included one-time costs of $1.1 million associated with the June acquisition of EGEN. And this related to legal and banking fees, as well as due diligence expenses we incurred in getting the transaction completed.

For the first half of 2014, the company reported a net loss of $12.1 million, compared to a net loss of $230,000 in the first half of 2013. Let me just point out. Last year’s net loss was positively impacted by two non-cash non-operating items, which included a non-cash benefit of $8.7 million from the change in valuation in common stock warrant liability associated with equity financings in September 2009 and June 2013.

The net loss for 2013 also was impacted by a non-cash deemed dividend from the beneficial conversion feature of $4.6 million on the preferred stock equity financing announced in February 2013.

If you look at comparable periods in 2014 and 2013 without these items, our operating loss for the second quarter of 2014 was $2.6 million higher than the comparable period last year, and $3 million higher in the first six months of 2014, when compared to the first six months last year.

Both of these three and six month periods ended June 30, 2014 included the $1.1 million in transaction-related acquisition cost. Our corporate focus has always been and will continue to be aligned with advancing our product pipeline through value-creating clinical trials and outside collaborations.

I will now turn the call back to Mike.

Michael Tardugno

Well, thank you Jeff. As always, you have the talent to bring these financials to life for us to really enjoy your report. Thank you so much. And particularly your comments regarding the deal terms for the EGEN acquisition, I hope keep on the other end of the line. I appreciate the value of this deal, not only from a technology perspective, but also the economics and the financials.

Well, I hope you’ll agree with us. This is an exciting time for Celsion. With our newly expanded pipeline, we have a defined strategy on oncology that is devoted to evaluating innovative therapies and first-line setting. I can’t say enough for that, very important area of research in first-line. It provides us with access to patients, who otherwise would not be interested in joining trials.

With active development efforts in primarily liver cancer, ovarian cancer, recurrent chest wall cancer and glioblastoma, we have a broad range of activity in some of the most important cancers in the world. We are advancing our Phase III OPTIMA trial for ThermoDox, targeting registration in the significant markets around the world, and we’re moving with our plans for important clinical trials for EGEN-001 next year.

And we’ll be providing data, as I pointed out, and information as I pointed out, early in my comments to support our clinical program for EGEN-001 over the course of the next few quarters.

EGEN-001 next year, Phase II in ovarian cancer, Phase I in glioblastoma, and we’re establishing a footprint in the breakout RNAi or siRNA space through the EGEN-002 product and through our TheraSilence program.

So, I will conclude my remarks by saying that we have a strong balance sheet, with the funding to advance our key development efforts. We believe that we have the right mix of programs and technology in our pipeline to create value for our shareholders, and most importantly, to make a significant difference in the lives of cancer patients and their family.

As always, we greatly appreciate your interest in the company and we look forward to updating you on our progress, as we continue to move forward with these very important clinical programs.

Now we’ll go to questions operator, so I’d like to ask the audience to limit them to more than two, so that everyone can have an opportunity to get answer. So operator, if you’ll open up the line please?

Question-and-Answer Session


Thank you. (Operator Instructions) We’ll move first to Keith Markey with Griffin Securities. Please go ahead. Your line is open.

Michael Tardugno

Good morning, Keith.

Keith Markey – Griffin Securities

Good morning. Congratulations on making an excellent acquisition, and good report on the R&D programs. Quick question, could you tell us what the status of patients’ enrollment is or might be in the foreign countries and regions where you’ve gotten regulatory approval for the OPTIMA study?

Michael Tardugno

Good question, Keith. So we do not have a patient yet in the trial. I’m expecting that we will be reporting patient very soon. We have multiple sites that are actively recruiting. There has really been no – we’re right on track with initiating sites in the countries where we have approval and I’m actually very, very encouraged by that.

In terms to us, that China will meet their – we’ll meet our timelines in China for achieving a clinical trial agreement or CTA. Typically for most companies, the time period from application to approval is about 12 months. We’re following very closely – and by the way, the progress of any clinical trial application in China is a matter of public record. You could follow it yourself, of course you’d have to be able to read Chinese characters, but you can follow yourself.

We do follow it very carefully. So China is accounting for about 30% of our patients in the previous trial, and re-enroll very quickly. So bringing China on board efficiently is important. I want to go a little bit beyond your question, because I think the first patient in only begins the effort.

We have met in a couple of medical conferences and will be meeting again at the upcoming ILCA Conference in Kyoto, a number of our investigators. I think they report to everybody on the phone. As I mentioned earlier, the enthusiasm for this trial among our investigator group is remarkable.

I think it becomes very clear to the medical community and particularly to our investigators, that appropriately using RFA within its design limitations. I don’t want to say was inappropriately used during the conduct of the trial, but we now know more about the use of RFA in larger tumors than any group of researchers and sponsors.

The application of this new knowledge and the well-controlled clinical study is exciting. And I think what we’re going to find is that once the study sites are initiated, that enrollment will progress very nicely, but for the most part, we are on track as I said and as I continue to talk to investors, we believe that from the time the first patient has enrolled to the time they complete 550 patients, the time period of the above goes somewhere between 32 and 34 months. I think we have a good handle on that.

With any luck at all, we should be reporting at the first interim analysis some efficacy results, if in fact, the clinical benefit is sufficient to unwind the trial, we will be reporting the results, but we should be able to conduct the first interim analysis in about 36 months following the first enrolled patient.

So I know, I answered a lot more than the question that you asked, but thanks for the opportunity to get on my cell box [ph].

Keith Markey – Griffin Securities

Thank you. One question, I’ll throw it, Jeff, financial. Could you give us a sense as to what you expect the burn rate might be in 2015?

Jeffrey Church

Yes, we’ve done a very thorough analysis. Right now, we’re utilizing about $1.5 million per month. We expect that to increase to approximately $2 million a month over the next 18 months. That’s for 2015, about $2 million a month.

Keith Markey – Griffin Securities

Thank you very much.

Michael Tardugno

Keith, may I add to that, so that’s kind of like a straight – that’s like an average. You know that our peaks and valleys [ph] in spending particularly as it relates to making large payments to CROs, but Jeff’s earlier comment, I think is the important one. We do believe that – and we take – and he takes, he is like sliding whiplash when it comes to controlling expenses in the company. We’ll be taking a very sharp pencil and my sense here is that we can talk with confidence about 24 to 27 month runway with the cash that we have, which in the meantime which should allow us to put some very important points on the board.

Keith Markey – Griffin Securities

Thank you very much.

Michael Tardugno



Thank you. (Operator Instructions) Meanwhile we’ll move to Reni Benjamin with H.C. Wainwright. Please go ahead. Your line is open.

Reni Benjamin – H.C. Wainwright & Co

Hi good morning guys.

Michael Tardugno

Good morning, Ren.

Reni Benjamin – H.C. Wainwright & Co

Good morning. Thanks for taking the questions. I guess just a couple. One in regards to GEN-001. Can you give us a little bit more color as to what the translational data maybe? Are we going to be looking at biomarkers or what should we be looking for?

Michael Tardugno

Biomarkers for the most part. Those surrogates that represent the expression of IL-12. The activation of the immune system and all the appropriate components of the immune system, Ren.

Reni Benjamin – H.C. Wainwright & Co


Michael Tardugno

So yes, let me mark biomarkers. For the most part, the study has been accumulating tissue samples, as well as blood samples. And we think we have patients that’d be able to submit the samples from analysis. And I am hopeful to present that data sooner rather than later given what we know so far in earlier studies, it should suggest that the activation in the immune response in a very profound way, we will see.

Reni Benjamin – H.C. Wainwright & Co

And can you just remind us, how big is this study? How many patients have been enrolled and what’s the target enrollment?

Michael Tardugno

Yes. So this has been a dosing escalation study. It combines – again it’s platinum sensitive – I’m sorry, platinum-resistant patients. It’s in combination with Doxil. So it’s been dosing escalation for both, the chemotherapy as well as the EGEN-001. We’ve been through three cohorts. So up to this point, I’ve done only two, but the enrollment maybe very close. We have 9 or 12 patients, but we’re looking for three more to round out the last cohort.

So what we haven’t talked about is our interest in – since we have not seen any significant safety issues with the immunotherapy with the IL-12 plasmid, we have been discussing with investigators in the GOG trial, the strategy for an additional dose escalation, at least one additional dose escalation.

So there is a chance there that we could be putting as many as six more patients on the study, but that would not stop us from gathering data from the translational analysis that we’re looking for.

So above 15 patients on the study, Ren, it could be as many as 21 if we get an agreement to dose escalate the IL-12 plasma.

Reni Benjamin – H.C. Wainwright & Co

And this is the only study that’s ongoing right now with the 001, correct?

Michael Tardugno

That’s correct.

Reni Benjamin – H.C. Wainwright & Co

Okay. So as my second question, can you talk a talk little bit – it’s just a two part question. One, how many more sweeps of the HEAT study do you think might occur going forward, or we kind of looked at the final data and can you give us I guess your thoughts as to why – how we should be interpreting further data sweeps if that’s going to happen?

Michael Tardugno

Yes, for the overall study, we have reached a million. For the subgroup we have not. We’re very close. We’re within a few patients. This fairly efficient like to point to a stable dataset once you’ve reached the median, you probably know more about that than I do.

So we’re close enough I think technically to be able to call in clean success. On the other hand, we have been debating the value of this dataset in support of a registrational program. And it seems – in our most recent discussion, it seems to me one more data sweep will take us to the median for the subgroup that we’re following, the patients treated with radiofrequency ablation, greater than 45 minutes with single lesions, 41% of the study.

So we think that this important data could ameliorate any concerns, if there are any. I don’t think there are, but it could ameliorate any concerns for FDA’s need for some confirmation in the OPTIMA study data. So as I say with a little bit of caution, my first reaction to as close as we work to the median was just, this is probably our final study and reflection after talking with our experts and some of the statisticians who have been involved with these kinds of applications, we may choose to continue for one more data sweep, in which case, of course we will publish the results.

The results have been – as I said, have been consistent and the magnitude of factors and so strong that [indiscernible] take a major change, you can’t even imagine. Will take some kind of a major change in order to give us anything that would confound what we’ve seen today, Ren.

So the strategy here if we continue and the likelihood is 99% yes. If we continue with one more sweep, it would be for regulatory purposes, it would be to support the 33% improvement target, 8% power, 33% improvement target in the OPTIMA study.

Reni Benjamin – H.C. Wainwright & Co

Excellent. I have a couple more questions, but I’ll jump back in the queue.

Michael Tardugno

No, go ahead. You can take one more if you would like.

Reni Benjamin – H.C. Wainwright & Co

Okay, that would be great. DIGNITY, can you talk a little bit about the durable response rate about how many more patients do you think you need to be recruited to kind of make that go, no-go decision before you go to the FDA to talk about path forward?

Michael Tardugno

Yes, so let me – I maybe speculating with you a little bit [indiscernible] it’s for everybody on the line. So this is an ongoing dialog within the company. So we have recruited 27 now, almost 40 patients among three studies. The protocol in each of the three studies is essentially the same, and a little bit more data that we’re collecting in Phase II study, essentially you can consider it to be for sampling including pharmacokinetics that FDA I believe is interested in. The trial shows some kind of relationship between blood plasma concentration and response.

Although in our study, I don’t think they are going to find anything, because it’s just about every patient and every dose treated for an hour with ThermoDox for an hour or so around an hour and a systemic dose of ThermoDox starting at 20 actually, 20 milligrams per meter square, while currently dosing at the MCD [ph] in the study is 40 milligrams per meter squared dosed.

Virtually every patient is shown an objective response for the stable disease. And you know this disease progresses. I mean it’s failed and it’s moving, it’s an aggressive recurrence of cancer, so stable disease is an important item [ph].

But every patient, 100% of patients for the most part have shown – I may take that maybe one patient ignore it, but let me just check, but approximately 100% of patients have shown an objective response. Many patients showing a partial response for a remission and I think now with five or six patients among these three studies showing a complete response, pretty remarkable and it’s a gasp from some of our investigators.

While we like to complete the enrollment on the study, we think we have 13 kind of which are valuable for efficacy, seven more round off it 20. If the data continues, the results – if the results continue to show that this tumor response, you may ask the question of durability. Right now the median durability and with patients still on the studies, they haven’t progressed is about 88 days overall, that’s the question you asked me separately, and it took me a little bit of time to calculate it, make to calculate we had to go back in patient records.

So I mean it’s clear to us that this threshold for the definition of durability of 90 days, it’s typically that’s accepted according to resist criteria. It’s something that’s imminently achievable if we haven’t already achieved it.

So let’s assume that we have this durable response, with not this durable response definition. And we pick up seven more patients, maybe six of which are valuable, showing a 55% or 60% objective response rate, we’re going to go to the FDA with the – I think a crushing and maybe a proposal with regards to next step in the study.

And I have said, and again thanks for this [indiscernible] but I have said, I think a company like ours it has very little – very few opportunities like this. We are treating patients who have simply no other options, they are great, great women, they are facing the end of life with the disease that’s going to take the integument off their chest. In many cases a very debilitating infection and painful source. If we can prevent that during the course of the rest of their lives, I think we have an endpoint, FDA is agreeing with us that does deserves registrational consideration.

Do we have enough of an end to talk about some kind of a conditional filing? Probably not. Do we have enough of an end to consider with FDA and maybe some of our key investigators on alternative to continue clinical trial with holding limitations associated, maybe moving into a more humanitarian news or kind of a program, that maybe the next logical step here. We can’t give up on these patients, we won’t. As long as they are willing, we’re willing.

And I know it’s a long answer. I hope that covered your questions.

Reni Benjamin – H.C. Wainwright & Co

Yes, it did. Thank you very much. And good luck.


Thank you. Next we’ll move to Bob Green [ph], Investor. Please go ahead.

Michael Tardugno

Hi Bob. Good to hear your voice again.

Bob Green [ph] – Private Investor

Good morning. I just wanted to confirm something. I thought I heard you say, we haven’t reached a median Overall Survival after the HEAT study?

Michael Tardugno

So let me clarify that. So for the Overall Study we have, for the cohort that we are following to assess a survival benefit, the cohort has the minimum treatment time with radiofrequency ablation of 45 minutes. We are close, but we have not.

Bob Green [ph] – Private Investor

Okay. And talking to that same group, those numbers looks so good. Has there been any thought at all of going to the FDA and asking if you could put in NDA for that group?

Michael Tardugno

I am with you. I really am. I think there is a certain discipline and a generally accepted understanding about retrospective studies that – I mean it’s really tested. It’s test of time. Retrospective is now seen more often than not, and I don’t want to discount our result in anyway, are more often not – they are just not reliable. So what we have, Bob, is we have a magnitude effect and the improvement is substantial. It hasn’t wobbled. It’s in controverted mode [ph].

We have marks to high level of confidence. It’s difficult to call it statistical significance, because it’s not prospective, but had it been prospective we would have a p-value that is statistically significant. But the p-value that we calculate for this group is highly confident, we’re highly confident. 96.5% confident that we’re on the right track.

I think there may come a time when the FDA will accept to meeting with us to discuss this data, but it’s going to probably will be have to be in context with the OPTIMA study. I don’t believe it’s probably not scientifically or clinically responsible for us to try to conceive over the circumstance where we are the exception to FDA’s rule on considering these kinds of datasets.

Bob Green [ph] – Private Investor

Have you looked at applying for the Breakthrough Therapy Designations?

Michael Tardugno

So I think that’s a step that we will take particularly for the current chest wall breast cancer program, but at this point for the HCC study – the advantage at this point clearly is probably lost. We’re already in the Phase III study. If the data is as strong as we think it is, we’ll have – and we do of course have fast track understanding for ThermoDox in HCC. That’s a work in Organ Designations, all of which provides us with significant benefit in dealing with the agency, assuming we have a positive dataset.

Bob Green [ph] – Private Investor

Okay. Sir, you answered my questions. Thank you very much.

Michael Tardugno

Thank you for your interest in being a loyal shareholder of the company.


And thank you. At this time, I’ll turn the call back over for closing comments or remarks.

Michael Tardugno

Well, thank you all. I just want to repeat a couple of things that I said earlier in closing. It’s always a pleasure to first [indiscernible]. Particularly now this is an exciting time at Celsion. I hope you agree. With our newly expanded pipeline, we have a defined strategy in oncology and devoted to evaluating these innovative therapies. In cancer some of global – in cancers some of – cancers globally most important forms, primary liver cancer, ovarian cancer, recurrent chest wall breast cancer, glioblastoma could be a priority of the company. We’ll see soon.

I conclude by saying that we have a strong balance sheet, as Jeff points out. We are very careful and very mindful of our expenditures. With this balance sheet, we have the funding to advance our key development efforts, and we believe we have the right mix of programs, but more importantly, we have the support of loyal shareholders like you. So it’s our pleasure. We greatly appreciate your interest in the company and we do look forward to updating you on our continued progress. Thank you very much and have a nice day.


Thank you. This does conclude today’s conference. You may disconnect at any time, and have a great day.

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