Celldex Therapeutics Inc (CLDX) 2011 Q4 法說會逐字稿

Good morning and welcome to the Celldex Therapeutics' fourth quarter and year end 2011 update conference call.

My name is Diana, and I will be the operator on today's call.

Before we begin our discussion, I have been asked to caution listeners that today's speakers will be making forward-looking statements.

Such statements reflect on current views with respect to future events and are based on assumptions and subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such forward-looking statements.

Certain of the factors that might cause Celldex's actual results to differ materially from those in the forward-looking statements include those set forth under the headings Risk Factors, and Management Discussions and Analysis of Financial Condition and Results of Operations in Celldex's Annual Report on Form 10-K, quarterly reports on Form 10-Q, and its current reports on Form 8-K, as well as those described in Celldex's press releases and filings with the Securities and Exchange Commission.

All forward-looking statements are expressly qualified in their entirety by this cautionary notice.

You should carefully review all of these factors and be aware that there may be other factors that could cause these differences.

These forward-looking statements are based on information, plans, and estimates as of this call, and Celldex does not promise to update any forward-looking statements to reflect changes in underlying assumptions or factors, new information, future events, or other changes.

Please be advised that the question-and-answer period will be held at the close of the call.

I would now like to turn the call over to Mr.

Anthony Marucci, President and CEO of Celldex Therapeutics.

You may proceed

Good morning and thank you for joining us.

I am Anthony Marucci, President and CEO of Celldex.

Joining me on the call today are Chip Catlin, our Senior Vice President and Chief Financial Officer, Dr.

Tom Davis, our Senior Vice President and Chief Medical Officer, Dr.

Tibor Keler, Senior Vice President and Chief Scientific Officer, and Dr.

Ron Pepin, our Senior Vice President and Chief Business Officer.

2011 was a year of significant progress for Celldex and in 2012 we will continue this momentum.

I would like to take the opportunity this morning to recap the key accomplishments of 2011 and what we have accomplished to date in 2012, and then turn it over to Tom to provide a status report on our clinical programs.

Chip will then walk you through the fourth quarter and year end 2011 financial results which, as you know, we reported this morning.

I will then close the call by outlining the major milestones we intend to accomplish for the remainder of 2012 and open the call to questions.

In 2011, we made distinct progress in the development of several Celldex product candidates.

As you will see on slide 3, one of the major highlights of 2011 was the initiation of our Phase 3 trial for rindopepimut in glioblastoma called ACT IV.

In previous Phase 2 studies, rindo has demonstrated significant potential to help patients suffering from glioblastoma whose tumors express the molecule EGFRvIII, a disease with particularly poor prognosis and few treatment options.

We had detailed discussions with both the US and European regulatory authorities to design the ACT IV trial with rigorous registration standards to determine the benefit of the addition of rindo to the standard of care.

I am pleased to report the study is now actively enrolling patients.

We have also initiated a Phase 2 trial for rindo in patients with recurrent and refractory glioblastoma that express EGFRvIII.

This trial, called ReACT, will run in parallel with the ACT IV study.

It will also include patients with first and second relapse following receipt of standard of care in the setting.

On the business side of the rindo program, we secured Orphan Drug Designation in the EU, and we previously obtained Orphan Drug Designation in the US as well as Fast Track designation, and continue to build an expanded patent portfolio, so we feel rindo is well positioned as we work towards potential commercialization.

We also recently brought a new candidate into the clinic CDX-1127, which entered Phase 1 clinical development in solid tumors and hematological malignancies in November.

In addition to initiating these new studies, we completed enrollment of the EMERGE study, a Phase 2b trial of CDX-011 in patients with metastatic breast cancer that express the molecule GPNMB, and we expect to report topline data from this program at ASCO later this year.

We have made significant progress in the initiation of a Phase 2 pilot study of a program called CDX-1135 later this year in dense deposit disease.

Dense deposit disease is a complement mediated kidney disease in children and young adults.

It often leads to chronic kidney failure and kidney dialysis.

While it is a rare disease affecting fewer than 1,000 patients annually in the US, there is a significant unmet medical need in this space, as the current treatment options are very limited with no chance for long term control.

We have continued to strengthen what is already a very strong management team.

In July of 2011 we welcomed Dr.

Ron Pepin to our team as Senior VP and Chief Business Officer.

Ron most recently served as Vice President at Shire Pharmaceuticals, but we know him best from our firsthand experience working together when he served as Senior Vice President of Business Development at Medarex, and we are very pleased to have the opportunity to work with Ron once again.

As we begin 2012, we have continued this momentum initiating a Phase 1 trial in healthy volunteers of CDX-301.

Tom will go into more detail later in the call, but our initial development plans for this molecule are focused on the treatment of cancer patients requiring hematopoietic stem cell transplantation.

We are also making good progress in our CDX-1401 program as we completed enrollment of our Phase 1 study this quarter and will determine next steps with this program later in the year.

To complete our first quarter operating initiatives, we completed a financing that will provide financial runway into 2014 that will directly support further development of our programs and pipeline, which brings me to slide 4, which gives a quick snapshot of our current clinical pipeline.

We have been successful in building a robust stepped pipeline that spans all phases across multiple indications.

We are very excited about these programs and the potential we believe they hold and are eager to drive them forward over the remaining of the year.

With this, I am going to ask Tom to provide an update for each of our clinical programs.

Tom.

Thanks, Anthony.

On slide 5, you can see the trial design for ACT IV, our Phase 3 study evaluating the efficacy of rindopepimut in glioblastoma, Rindopepimut is a therapeutic cancer vaccine candidate that targets the tumor specific oncogene EGFRvIII, which provides a constant growth stimulus and confers an enhanced capacity for unregulated tumor growth.

The ACT IV study is a randomized, double blind, controlled study of rindopepimut plus GM-CSF added to the standard of care, temozolomide, in patients with newly diagnosed surgically resected EGFRvIII positive glioblastoma.

The primary objective of the study is to determine Overall Survival in these patients with newly diagnosed EGFRvIII positive glioblastoma after gross total resection compared to treatment with temozolomide alone.

Additional secondary end points include progression free survival, immune response, quality of life and safety.

All patients will receive the standard of care, temozolomide.

Half of the patients will be randomly assigned to receive combination with rindopepimut, given along with GM-CSF as a vaccine adjuvant, and half of the patients will be randomly assigned to receive the low dose of KLH, which will produce a local site reaction similar to what you would see with rindopepimut, to ensure study blinding.

Up to 450 patients will be enrolled at over 150 centers worldwide.

In order to maximize the useful data generated from this study, and to assure efficient accrual, the trial is divided into two cohorts.

We previously focused on patients with glioblastoma who have very little residual tumor after surgery, a Gross Total Resection.

The ACT IV study is designed to randomize 374 of these patients into an intention to treat population, and the primary analysis will be limited to this population, assuring that we are building upon our promising Phase 2 data.

However, some patients are only able to undergo a partial resection.

It is not known what benefit patients with a partial resection may gain from vaccination with rindopepimut, but anecdotal data in patients with bulky disease treated under compassionate use protocols suggest significant activity may be possible.

Including patients with a partial resection in this study could ultimately expand our indication if Overall Survival efficacy is seen.

Inclusion of the partial resection group also adds the potential for a clinical response endpoint and will likely provide faster data on PFS and OS.

Efficacy of the partially resected patients in the total population will be determined only in secondary analyses and will not compromise the primary end point.

The final accrual numbers to the study will depend on the outcome of interim analyses performed for the independent data monitoring committee.

These analyses will look for early efficacy and futility, and will also assess accrual and event rates.

They may either increase or decrease sample size dependent on survival rates or events, and will be performed at 50% and 75% of events.

The primary accrual of the study is based solely on the gross total resected patients.

We are very pleased with the eagerness that investigators have shown to participate in the study and screening of patients for enrollment is going well.

We hope to complete accrual in 2014, which would position us for a data readout 18 to 24 months later.

We are also exploring rindopepimut's potential in additional studies.

On slide 6, you will see a schematic outlining the ReACT study, a Phase 2 trial of rindopepimut in combination with Avastin in patients with recurrent EGFRvIII positive glioblastoma.

The study will be conducted at approximately 20 sites across the US and will enroll 95 patients in the first or second relapse following receipt of standard therapy.

Approximately 70 Avastin naive patients will be randomized to receive Avastin along with either rindopepimut or a control injection of KLH in a blinded fashion.

Additionally 25 patients whose glioblastoma has progressed during treatment with Avastin, will receive rindopepimut plus continued Avastin in a single treatment arm.

Patients in all three arms will be evaluated for the progression rate at six months, objective response rate, overall survival and progression free survival.

Dependent on preliminary results, which should be available in the first half of next year, we could identify a registration path with possible accelerated approval.

We are also working with Doctors Paul Fisher and Albert Wong of Stanford University on a pilot study of rindopepimut in pediatric pontine glioma.

Although radiation has some minimal benefit in this disease, these young patients tend to progress very quickly and life expectancy is extremely short, oftentimes measured only in months.

The investigators have shown that EGFRvIII mutation is present in a significant portion of these tumors.

It is our hope that if we can vaccinate children with pontine gliomas with rindopepimut, we may be get tumor shrinkage or growth delay, which would be a major step forward for these children and their families.

Moving on to slide 7, you can see the status of our CDX-11 program.

We recently announced that we completed enrollment in the EMERGE study, a randomized Phase 2b study evaluating CDX-11 in patients with previously treated metastatic or locally advanced breast cancer, including triple negative disease.

This patient population is heavily pretreated and their prognosis is poor.

CDX-11 is an antibody drug conjugate targeting glycoprotein NMB, or GPNMB for short, which is overexpressed in several cancers including breast cancer.

Patients in this study were specifically selected for GPNMB expression.

The study enrolled 120 patients who were randomized two to one to receive either CDX-11 or single agent Investigator's Choice.

Patients randomized to receive Investigator's Choice are allowed to cross over to CDX-11 following disease progression.

Study end points include overall response rate, duration of response, progression free survival, overall survival, and pharmacokinetics pharmacodynamics.

We intend to present topline results from this study at ASCO in June, at which point we will be able to more fully discuss potential next steps for CDX-11 in breast cancer and the potential for expansion into other cancers.

On slide 8, we present CDX-1127, a fully human monoclonal antibody that binds CD-27 an important co-stimulatory molecule on T cells.

CDX-1127 is designed to activate patient's immune cells against their cancer or other immune targets.

It has shown potent efficacy in several preclinical models.

In addition, CD-27 is overexpressed in certain lymphomas and leukemias, and can be directly targeted by CDX-1127.

To that end, we have initiated a Phase 1 study designed to test five escalating doses of CDX-1127 to determine a Phase 2 dose for further development.

The study will accrue approximately 30 patients with advanced refractory disease in each of the two arms, either solid tumors or hematologic malignancies.

The trial design incorporates both single dosing and multiple dosing regimens at each dose level.

The study is being conducted at multiple clinical sites across the United States and it is anticipated to complete accrual by year end.

Depending on the data, we may seek to initiate Phase 2 expansion cohorts in selected solid tumors and hematologic cancers, and can also consider development in combination with vaccines and other immune modulators.

Now on slide 9, in January this year we announced the initiation of a Phase 1 dose escalation study for CDX-301 in approximately 30 healthy volunteers in collaboration with Rockefeller University.

CDX-301 is Flt3 ligand, a hematopoietic growth factor that serves as a potent stem cell mobilizer and dendritic cell growth factor.

Celldex's first priority is to develop CDX-301 for hematopoietic stem cell mobilization, where it has shown activity in earlier clinical studies and has demonstrated improved blood cell reconstitution in in-vivo models.

We also believe that CDX-301 has significant potential to be developed in a number of additional indications including cancer, transplantation, autoimmune diseases, and infectious diseases.

To that end, we are planning expanded Phase 2 development, which is expected for 2013.

Slide 10 brings us to CDX-1401, a program licensed from Rockefeller University that builds upon the research of Nobel Laureate Ralph Steinman.

It is a fusion protein consisting of fully human monoclonal antibody, with specificity for the dendritic cell receptor, DEC-205, linked to the NY-ESO-1 tumor antigen.

CDX-1401 is the second product candidate using Celldex's proprietary dendritic cell targeting technology to selectively deliver the NY-ESO-1 antigen to Antigen Presenting Cells, including dendritic cells, to generate robust immune responses against cancer cells expressing NY-ESO-1.

NY-ESO-1 represents an important target for developing therapeutics against multiple cancers.

The antigen, which is expressed in a wide variety of cancer cells but not at significant levels in normal tissues, has been extensively characterized in preclinical and clinical studies and has been found to be highly immunogenic.

A Phase 1 study in patients with advanced and refractory melanoma and other cancers that are known to express NY-ESO-1 has completed accrual in six different cohorts, to enable to us optimize the vaccination regimen.

The study evaluated both dose and combination with Toll like receptor agonists.

To date, CDX-1401 has been well tolerated and has demonstrated no dose limiting toxicities.

We have also observed strong antibody and cellular immune responses against NY-ESO-1.

The next step for CDX-1401 is a potential Phase 2 study in combination with immune modulators in a select patient population.

We are establishing collaborative relationships with academic and research institutions to drive this program forward.

Slide 11 addresses a new program and a new indication with significant unmet medical need.

As Anthony outlined earlier, dense deposit disease, or membranoproliferative glomerulonephritis type 2, is a renal disease seen in children and young adults.

The disease leads to chronic kidney failure and renal dialysis.

Current treatment options are limited and have only modest ability to manage the disease, but offer no chance for long term control.

The disease is caused by uncontrolled activation of the alternate pathway in the complement cascade at the C3 step.

Byproducts of complement C3 are deposited in the kidney and block kidney function.

CDX-1135 is a potent inhibitor of complement C3 step, making it a potentially ideal candidate to control this disease.

Working with leading experts in the field, we have demonstrated CDX-1135's potential in both human in vitro studies and mouse models of dense deposit disease.

CDX-1135 has also consistently demonstrated a strong safety profile in both preclinical and clinical studies.

We have manufactured CDX-1135 and plan to initiate a pilot study in the second half of 2012 to determine the appropriate dosing regimen required to normalize C3 levels and to assess the potential for CDX-1135 to improve renal function in these young patients.

I will now turn the call over to Chip to review the financials.

Thank you, Tom.

I am now on slide 12.

For the fourth quarter of 2011, Celldex reported a net loss of $12.7 million, or $0.29 per share, compared to net income of $22.7 million, or $0.71 per share, for the fourth quarter of 2010.

Net income for the fourth quarter of 2010 included one-time items totaling $30.5 million for rindopepimut-related revenue recorded as a result of the termination of the Pfizer license agreement and a charge to royalty expense related to costs originally capitalized in connection with the Pfizer license agreement.

Celldex regained rights to rindopepimut during the fourth quarter of 2010.

Excluding these one-time items on a non-GAAP basis, Celldex would have reported a net loss of $7.8 million, or $0.24 per share for the fourth quarter of 2010.

The change in net loss between the three-month periods was primarily due to these one-time items as well as higher R&D expenses as a result of initiation of the rindopepimut ACT IV and ReACT studies and lower investment and other income in 2011 versus 2010.

For the twelve months ended December 31, 2011, Celldex reported a net loss of $44.8 million, or $1.13 per share, compared to a net loss of $2.5 million, or $0.08 per share for the twleve months ended December 31, 2010.

The increase in net loss of $42.3 million between years is primarily due to the one-time items discussed above.

Excluding these items, the non-GAAP net loss for 2010 was $33 million, or $1.04 per share.

R&D expenses in 2011 increased by $4.8 million compared to 2010 and were primarily a result of increased clinical trials costs of $4.6 million in 2011.

G&A expenses decreased by $1.2 million in 2011 compared to 2010, primarily due to decreased professional service related fees in 2011.

At December 31, 2011, Celldex reported cash, cash equivalents, and marketable securities of $53.3 million.

The decrease in cash, cash equivalents, and marketable securities of $9.5 million from September 30th, 2011, primarily reflects our fourth quarter operations-related cash burn of approximately $9.4 million.

To date in 2012, we have raised net proceeds of $8.5 million through the sale of 2.5 million shares of common stock under a controlled equity offering facility with Cantor Fitzgerald, and have issued 10.5 million shares of our common stock in an underwritten public offering resulting in net proceeds of $37.7 million, which provides a financial runway into 2014.

We have granted the underwriters a 30-day option to purchase up to an aggregate of 1,575,000 additional shares of common stock to cover overallotments, if any.

As of December 31, 2011, Celldex had 44.2 million shares outstanding.

As a result of our financing transactions in January and February of 2012, we now have 57.2 million shares outstanding.

I will now turn the call back over to Anthony for closing comments.

Thank you, Chip.

So as you heard from Tom and Chip, 2011 was a significant year for Celldex and 2012 is shaping up to be even better.

I want to recap and summarize the major milestones that you should be watching for outlined on slide 13 through the remainder of the year.

For rindopepimut, we will continue to screen and enroll patients in the ACT IV Phase 3 study in glioblastoma.

We also plan to complete accrual of the Phase 2 combination study in recurrent and refractory glioblastoma and will provide ongoing support to pontine glioma study sponsored by Stanford University.

As I mentioned, we will also present the results of the Phase 2b trial of CDX-011 in metastatic breast cancer at ASCO in June.

Potential future development of this compound includes additional advanced studies in breast cancer, and also expansion into such indications as lymphoma, melanoma, and squamous cell lung cancer.

We intend to complete the dose escalation of the Phase 1 trial of CDX-1127 in solid tumors and hematologic cancers by the end of the year, and based on those results we will seek to initiate Phase 2 expansion cohorts in the appropriate indications in 2013.

We will also complete accrual of the Phase 1 study of CDX-301 in healthy volunteers, which will provide important safety and dosage information to inform the next steps for this program.

We expect to focus on patients undergoing hematopoietic stem cell transplantation.

For CDX-1401, we will continue to work on establishing collaborative relationships with academic and research institutions to drive this program forward.

And finally, we intend to initiate a Phase 2 pilot study of CDX-1135 in dense deposit disease in the second half of the year.

As you can see, we've had a busy and exciting year ahead of us.

We begin 2012 with a strong financial backbone and strong position in the clinic.

As of the end of February, we had over $92 million in cash and investments on our balance sheet and we expect each of our clinical programs to achieve meaningful milestones over the next 12 to 24 months, and have also continued to update our shareholders on the progress of our overall strategic initiatives.

This concludes our prepared remarks and we are now ready for questions.

Operator.

(Operator Instructions).

And the first question will come from the line of Jonathan Aschoff, Brean Murray.

Thanks.

Good morning, guys.

Good morning Jonathan

a question, I guess, for Tom.

In any of the three prior GBM trials, have you ever correlated the tumors baseline level of immune cell infiltration with PFS or OS?

We have looked at that Jonathan and of course it is a very compelling question.

Is it just P3 expression levels or evidence of tumor infiltration that is going to predict the best responders?

While we have done that, we have not been able to see any clear correlations.

The bottom line though is in glioblastoma, almost all of the tumors do have high levels of lymphocytic infiltrates.

It is an immunogenic tumor from that perspective anyway.

So we are not sure that we can select better responders that way.

So clearly there is no effort in that direction in the ongoing trial?

Well, we will be looking at the tissues but we don't expect to see anything.

And how many partial resection patients are you going to add?

So when you look at the institutions across North America and certainly around the world, it is very hard to get a solid number how many patients have partial versus gross total, and it varies quite dramatically.

So the reason we emphasize 374 patients with gross total resection is that we know we can control that.

But the number of partials could vary depending on really what that rate is in different countries.

Generally looking at maximum accrual of 440 patients on the whole study, so you can see it is about 60 patients that we expect to come on, but that number could be different.

Okay.

Could you give us your best guess as to when the 1401 data might be available?

Well, so it completed accrual to the final cohort, and would expect to have data about the middle of the year.

We do want to follow longer term and get immune response data, so we are expecting to be able to present that at the end of the year at an appropriate immunology based conference.

Thanks a lot.

Thank you, Jonathan.

The next question will come from the line of Joe Pantginis, ROTH Capital Partners

Good morning.

Thanks guys.

A couple of questions if you don't mind.

On the ACT IV study, I am just curious,will you be providing any sense of enrollment trends going forward, or maybe some initial comments on how the excitement of the clinical investigators have been translating into increased screening, and then I just have a couple follow-ups if you don't mind?

Well, we won't be giving any updates during the year as to the enrollment per se, but we may give comments as to our expectations as to how it is accruing.

As far as investigator enthusiasm, we do believe it is very, very high and I can have Tom expand on that some more.

That is true.

We just had an investigator's meeting this last weekend and pretty much every site that is coming on board in North America was eager to join.

We thought it was a tremendous success.

I will point out, Joe, that because we are going to many countries with varying different timelines, it is going to take us six to 12 months to get the study fully up and running, so we wouldn't expect accrual to really kick in for another six months, hence it is just not really something we can talk about.

Sure that is helpful.

Thank you.

And then switching over to 011.

Obviously ASCO is a big event for you guys.

I know you did mention after the data you would talk about potential plans going forward.

I was just wondering if you could share any initial thoughts with regard to thoughts around BD, and also moving in as you said, potential other indications or taking it into Phase 3 with breast?

We will discuss those more at ASCO, Joe.

As far as BD goes, we are having conversations as we have said in the past.

We just can't put any timelines on any BD discussions.

Sure that is helpful, and a last question if you don't mind.

Obviously, 1127 had the ability to be a very exciting product especially with its immune properties and I think a lot of people will then look to draw potential parallels with Ipilimumab, so I was just wondering if you could highlight a key differentiating factor which I think also would be considered in the safety realm.

Maybe you could just highlight some of the key differentiations?

Very good question.

Of course, we were very involved with the development of Ipilimumab and think it is a tremendous product, but it is somewhat less specific in what kind of immune responses it activates.

So any ongoing immune response anti-CTLA4 can potentially unleash that, and of course, you do see the immune breakthrough events that are essentially forms of autoimmunity.

CD-27 on the other hand appears to be integral to a new or activating immune event, so that we will specifically be augmenting any new exposure to tumor antigens outside of the oncology realm.

It would work very well in combination with specific vaccines that are designed to generate something that the body does not already have ongoing.

Based on that we like to think that we are not going to see those autoimmune events, but at the end of the day it is really the antitumor activity that will be most critical for us, and the fact that we can both activate ongoing effects against solid tumors, but also directly target lymphomas, I think is very exciting.

As you can imagine, if we do have an effect akin to rituximab where we can induce cell death to lymphoma cells, we then can generate antigen stimulation, we can create that new vaccine effect I am describing, and the immune activation though CD-27 on the T cells can then potentiate an effect.

It can sort of be a circular pathway that can build over time.

Obviously, the Phase 1 data will be critical, and we are very excited to complete the study this year.

Great.

Thanks a lot, guys.

Thank you, Joe.

Next question comes from the line of Steve Brozak, WBB Securities.

Thanks for taking the questions.

First off, I am going to go to the macro level because there is a significant question that people have asked about, the fact that you guys just fund raised and frankly, obviously it is now self-evident that the importance of it is it gives you runway room through 2014.

But the other part is that you are probably the only unencumbered player out there, and want to know what your thoughts are and potential strategy.

ASCO is just around the corner in June.

Now that you are unencumbered and have the ability to basically control the information without having to consult a partner, how do you think that positions you vis-a-vis other potential presenters at ASCO?

I am not talking about in terms of what they are going to present, but in terms of how your approach is going to be and I have a follow-up after that.

Well, thanks for the question, Steve.

This is Anthony.

The fact that the assets are unencumbered does give us more flexibility to speak freely about the data from our clinical programs, so that is always a plus.

Having said that, in the past when we did have a partner we were somewhat collaborative in our collective thinking, but as it is our program now, we can certainly speak to the programs and to our plans going forward more freely.

So we think that is a real plus.

Now that also then leads me to the next question, because you have a wealth of different programs.

The idea is that now you can re-enter into collaboration, but now you obviously know what some of the potential problems might be, I shouldn't say problems but disadvantages of different programs, so that you can basically bake that into the next step.

Would that be an accurate statement?

Again, we have always said that the programs that we believe we can take over the goal line ourselves are the programs that we will do so with and those that we can't we will always seek partners and we are sticking to that strategy.

That hasn't changed at all.

So we will continue looking to partner those programs that require global partnerships and a bigger firm to do that, and those programs that we can take over ourselves, because you need a small sales force, a small infrastructure we will do that.

So the strategy hasn't changed.

Well again, congratulations gentlemen.

And by the way, thank you for a really great Investors Day.

It was really well received and I look forward to the data at ASCO.

Thank you Steve.

Appreciate it.

The next question comes from the line of Boris Peaker, Oppenheimer.

Hello.

Can you hear me?

Boris, we can hear you.

Great, a lot of my questions have been answered but I want to quickly follow up on rindo, the ACT IV study.

Can you mention how many sites you have open or how many countries are presently involved, or you already have online?

Well, we are operating in the North America now, and we are certainly working through the other countries, such as Australia, New Zealand, India, the EU, so we think our goal by the end of the year is to have at least 75% of all those sites up and running so we are on track.

Okay.

And now on 011, you mentioned that there is going to be data at ASCO.

Specifically what kind of data will you have?

Will you have just response rates, durability of response, some early survival results?

Hi, Boris.

This is Tom.

We completed the study in December, so at ASCO we will have over three months follow-up on all of the patients.

The study is designed to take a look for response at about that timeframe.

So we should have response data on the primary treatment for all 120 patients.

We will also have early PFS data, meaning we have fairly mature PFS data from the patients who were enrolled at the beginning of last year, but the more recent information won't be there so it is not going to be any kind of final numbers.

But it will give us a sense of what we can expect.

The overall survival data will be very immature at ASCO.

From our perspective, that is adequate information to have a good sense of what the product can do in GPNMB expressers, and to start making plans so that we can get to the next steps very quickly.

But the final data set, the final clean data, certainly won't be available until the end of this year, beginning of next year.

Do you think you will have, based on this response data, enough information to potentially make decisions about going into other indications with also GPNMB expression, or do you think you would need more in response data to actually make such decisions for other tumors?

Well, we know that CDX-11 is an active molecule.

We know that in both melanoma and breast cancer we see significant responses, that those responses can be durable, and that the toxicity profile is very well received.

And I think that was emphasized at the R&D Day, which I believe is still on our website.

The information in breast cancer coming out at ASCO will be integral to our plans in breast cancer.

It will answer questions along the lines of, do we need a single arm study, do we need a randomized trial, should we go after specifically triple negative patients, or a broader population?

When it comes to other indications, again, we already know the drug works in melanoma.

We are very interested in lymphoma and leukemia just because of the obvious success with Seattle Genetics' Adcetris.

So, expanding into other indications is less dependent on the results of the EMERGE study and more dependent on resourcing and timeframes.

Okay.

Thank you.

And my last question is on dense deposit disease.

Have other people tried to develop drugs in this indication, or if you can give us a little bit of a history of pharmaceutical development in that indication?

Well, to date development has been very limited.

Obviously it is a small indication and a very difficult disease that the pediatric characteristics have been somewhat prohibitive in the past as well.

Certainly some drugs have been tested in this setting, including Soliris, which is a C5 inhibitor.

Again, we don't think that there are any data suggesting that those interventions work significantly.

So right now, at least from the prospective of clinical development, we don't believe there is any other activity in this space.

Great, thank you for taking my questions.

Thanks, Boris.

And the next question comes from the line of Mara Goldstein, Cantor Fitzgerald

Thanks.

I have two questions one is on the ACT IV study and partial resection, and I am wondering if you might be able to talk a little bit more about the enrollment criteria for patients that have partial resection and the disease burden, and where you are willing to accept with that in terms of how it affects your overall numbers in the trial?

And then just a question on CDX-11.

Data is going to be at ASCO.

Do you know if that is going to be an oral presentation or an abstract presentation yet, and will you also be able to have analysis that correlates GPNMB at that time?

Sure Mara.

Good questions.

The bulkier patients in ACT IV, we haven't spoken a great deal about them because they are a secondary component to the study, but all along patients, doctors, even the FDA have told us that they would love to see what we could do in patients with more tumor.

As I mentioned earlier, it is going to be hard to predict exactly how many of those patients will come on study, there is no specified number, although it is something that our data monitoring committee will follow on an ongoing basis.

If we appear to be getting too few or too many of those patients, we will need to adjust the plans for that secondary analysis.

But as we always said, the primary analysis is based on the gross total resection patients, the same population that we have tested in the past, and all of the statistics that we have quoted are built upon that specific analysis.

As far as CDX-11 at ASCO goes, it is too early to have been assigned any specific type of presentation, so no we do not know what we will be given.

As you can imagine from what I said our abstract submission was a placeholder abstract essentially.

We didn't have full data.

We expect with a biologically active program in a randomized Phase 2 study, we should at least get a poster presentation, but we will at least wait a couple months before we know

Right.

Okay.

And I think you have talked before about CDX-11 in terms of trying to perform [out] correlation analysis with the response and outcome, just given the difficulty of measuring surface levels of GPNMB, and might you have elements of that at ASCO?

Absolutely, we have all of the tissues in hand, and in doing that analysis we will be able to correlate the expression patterns with the data that we can present, so response rate by expression pattern I think will be a key component to that presentation.

Okay.

And if I could just circle back on ACT IV, do you have a sense about screening and enrollment, and about how many patients it takes from a screening perspective to enroll one patient in the trial?

Well, we have a 31% expression rate for EGFRvIII and, now that we have expanded accrual to include those with bulkier tumors, we are expecting to get very close to that, about 30% of patients who are screened should end up on study.

Okay.

Thanks.

And the next question comes from the line of Biren Amin, Jefferies.

Hey, thanks guys for taking my question.

On 011, can you just remind us what your response rate assumptions are for both arms in the Phase 2 study?

So with the single agent chemotherapy we are predicting approximately a 5% response rate, and the study is designed to look for a 20% response rate, as well as all of the secondary end points.

This is the first time that we will be generating data for GPNMB expressers receiving single agent chemotherapy, so it could well be lower than that.

Okay.

And on 1135 with regards to the pilot study, do you expect that to be a similar design to the Soliris Phase 1 that is currently ongoing in six patients in dense deposit disease, and also maybe if you could cite any differences between 1135 and Soliris preclinically, that you have observed so far?

So the pilot study will be very similar.

We will be testing a small number of patients to see what impact we can have on the chemical endpoints, C3 levels, et cetera, and of course, ultimately on kidney function and then kinesiology.

There is not a lot of information around the Soliris effects, but their effects have been modest to date in patients.

And in the preclinical models, as we understand it, Soliris is essentially ineffective.

And that is not surprising, since Soliris really does not tackle C3, which is the key component to this disease.

Great.

Thanks for taking my questions.

Thanks.

(Operator Instructions).

The next question will come from the line of Christian Glennie, Edison Investment Research.

Hi.

Good morning.

Just one more follow-up on CDX-11.

I know you are keeping your (inaudible) of partnering of CDX-11 options open.

Are there any comments you could make in general about what impact, if any, the FDA approval last year for Seattle's Adcetris has had on general mood, and around antibody drug conjugates, and how that plays out?

So Tom again, clearly with that approval, we know the Seattle Genetics' technology works.

We know that it can get through the regulatory pathways.

So I think that has broken down a huge hurdle from our perspective and there is a lot of enthusiasm specifically around that technology which, of course, we use in CDX-11.

I'll ask Ron Pepin just to comment about the broad environment, since he is much more involved in that partnering aspect.

Sure.

I think that there has always been a lot of enthusiasm for antibody drug conjugates, but not a lot of proof in the clinics.

And I think that based on some of the very old drug, there was only one that was approved, that there was a long time waiting for improvements in the technology, and I think that Seattle Genetics did a wonderful job of refining their technology, and making it available to companies like Celldex, so that we can now build upon the underpinnings that they have put together in getting this technology through the clinic, and there is a lot of enthusiasm that is building that I can see among pharmaceutical companies, like biotech companies, to adopt this technology in combination with antibodies.

Okay.

Thank you.

Thank you.

And this concludes the question and answer portion for today.

I would now like to turn the call back to Mr.

Anthony Marucci, President and CEO, for closing remarks.

Thank you, Operator and thank you everyone for joining us this morning, and the great questions that you posed to us.

We look forward to keeping you updated throughout the year, with what promises to be a very busy year for us, and we look forward to our mid-year earnings call in August.

Thank you very much and have a good day.

Ladies and gentlemen, thank you for your participation.

You may now disconnect and have a great day.