image
Healthcare - Biotechnology - NASDAQ - US
$ 2.02
-2.88 %
$ 230 M
Market Cap
-1.23
P/E
EARNINGS CALL TRANSCRIPT
EARNINGS CALL TRANSCRIPT 2016 - Q1
image
Executives

Scott Wolchko – President and Chief Executive Officer Dan Shoemaker – Chief Scientific Officer.

Analysts

Jeffrey Lin – Leerink Nick Abbott – Wells Fargo Alex Xenakis – BMO Capital Ren Benjamin – Raymond James Ted Tenthoff – Piper Jaffray Jim Birchenough – Wells Fargo.

Operator

Welcome to Fate Therapeutics’ First Quarter 2016 Financial Results Conference Call. At this time, all participants are in a listen-only mode. This call is being webcast live on the Investor and Media section of Fate’s website at fatetherapeutics.com.

This call is the property of Fate Therapeutics and recordings, reproduction or transmission of this call without the expressed written consent of Fate is strictly prohibited. As a reminder, today’s call is being recorded. I’d now like to introduce Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Thank you. Good afternoon and thanks everyone for joining us for the Fate Therapeutics first quarter 2016 earnings call. Shortly after 4:00 PM Eastern Time today, we issued a press release with our first quarter 2016 financial results, which can be found on the Investors and Media section of our website under press releases.

In addition, our first quarter 2016 10-Q was filed shortly thereafter and can be found on the Investors and Media section of our website under Financial Information.

Before we begin, I’d like to remind everyone that except for statements of historical facts, the statements made by management and responses to questions on this conference call are forward-looking statements under the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995.

These statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward-looking statements.

Please see the forward-looking statement disclaimer on the Company’s earnings press release issued after the close of market today, as well as the risk factors in the Company’s SEC filings, included in our Form 10-Q for the quarter ended March 31, 2016 that was filed with the SEC today.

Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made, as the facts and circumstances underlying these forward-looking statements may change, except as required by law, Fate Therapeutics disclaims any obligation to update these forward-looking statements to reflect future information, events, or circumstances.

Joining me on the call today is Dr. Dan Shoemaker, our Chief Scientific Officer. I will begin the call with an update on the progress of our Phase 1/2 clinical trial of ProTmune, we are poised to begin subject enrollment. Dan will then highlight our expected participation in multiple industry leading scientific conferences over the next several weeks.

Where we will be releasing new and encouraging data across each of our preclinical programs. And finally, I’ll review our financial results for the first quarter of 2016 before opening the call up for questions and further discussion.

Over the past several months, we’ve had the pleasure of engaging leaders across the hematopoietic cell transplantation community. This dialogue has reinforced our view of the challenges that compromised the curative potential of allogeneic hematopoietic cell transplantation for patients with hematologic malignancies and rare genetic disorders.

We are confident that ProTmune, our ex vivo small molecule programmed cellular immunotherapy is innovative and highly differentiated. And we believe the ProTmune has the potential to address these challenges and revolutionize allogeneic HCT. In January 2016, our investigational new drug application for ProTmune was cleared by the FDA.

We are currently initiating a Phase 1/2 clinical trial in allogeneic HCT using mobilized peripheral blood. The donor cell source used in approximately 70% of the 30,000 allogeneic HCT procedures performed each year.

Our objective in developing ProTmune is to prevent acute graft-versus-host disease and cytomegalovirus infection, both of which are leading causes of morbidity and mortality in transplant recipients.

It is important to note that there are currently no FDA approved therapies for the prevention of GvHD or CMV infection in patients undergoing allogeneic HCT. In February 2016, the urgent need for new therapies to attenuate GvHD and CMV infection was further underscored by multiple oral and poster presentations at the BMT Tandem Meetings.

The combined annual meetings of the Center for International Blood and Marrow Transplant Research and the American Society of Blood and Marrow Transplantation.

The sentiment from key opinion leaders and investigators in the community with whom we met, was that acute GvHD and CMV infection remains significant unmet medical needs for patients undergoing HCT.

With incident rates of 40% to 50% for acute GvHD and 50% to 55% for CMV infection and over 90% of these incidences occurring during the first 100 days following transplant.

We are gratified by the strong interest and collaborative involvement with ProTmune that we have received from the allogeneic hematopoietic cell transplant community, which we believe reflects the urgent need for new therapies as well as the groundbreaking nature of our therapeutic strategy.

Interested principle investigators have continued to extol the simplicity and elegance of our ex-vivo small molecule programming approach, and our compelling preclinical data package.

We have received feedback that our ex-vivo small molecule programming approach integrates easily into the current HCT treatment paradigm avoiding costly and cumbersome processes such as the depletion or genetic engineering of donor T cells.

Additionally, our most recent preclinical data shared at the 2016 BMT Tandem Meetings continue to impress investigators that we are targeting for participation in our Phase 1/2 study. This new preclinical data demonstrated that programmed donor T cells retained potent anti-tumor activity in vivo.

These data complement previously presented preclinical data demonstrating that the adoptive transfer programmed mobilized peripheral blood cells results in a statistically significant reduction in GvHD score and an improvement in survival.

Since filing the IND in January, key opinion leaders and interested principal investigators have reviewed the Phase 1/2 study protocol and their feedback reinforces our belief that the study is well designed to robustly assess the potential of ProTmune to prevent GvHD and CMV infection.

Under the protocol, we have limited the inclusion criteria to transplant recipients that are at high risk for acute GvHD and CMV infection. Where subjects are being administered mobilized peripheral blood cells from a matched unrelated donor and have antibody evidence of prior CMV infection.

Additionally, we are assessing the cumulative incidences of acute GvHD and clinically significant CMV infection at day 100 [indiscernible]. We’re also exploring numerous other endpoints including cumulative incidence of bacterial, fungal, viral and parasitic infections at day 100, cumulative incidence of relapse and cumulative incidence of survival.

Over the past several months ProTmune’s highly differentiated value proposition has clearly struck a chord and taken hold. And preparation for patient enrollment, we have now completed the formation of our independent data monitoring committee as well as our two endpoint adjudication committees.

Esteemed members of the hematopoietic cell transplant community have agreed to join our assessment committees. These include on the CMV committee Dr.

Francisco Marty Associate Professor Harvard Medical School, who presented the findings of the Phase 3 clinical trial of brincidofovir for the prevention of CMV after allogeneic HCT at the 2006 Tandem meetings. And on the GvHD committee, Dr.

Nelson Chao, Professor of Medicine and Chief of the Division of Cellular Therapy, Duke University, who was a renowned investigator of immunological events that occur with GvHD and of protocols for its prevention.

Additionally, given the receptive enthusiasm from the HCT community, we have elected to increase the number of sites eligible for participation in the Phase 1 stage of our study. We are now actively engaged with seven U.S. centers. That we believe are well positioned to potentially contribute to Phase 1 subject enrollment.

We have also initiated discussions with a number of additional centers including centers outside of the United States that have expressed interest in participating in the Phase 2 stage of our study. At this time, we are poised to begin subject enrollment. In our multiply center randomized controlled Phase 1/2 clinical trial of ProTmune.

We believe we remain on track to complete subject enrollment in 2016, for the open label Phase 1 stage of the study under which 10 subjects are expected to undergo mobilized peripheral blood HCT with ProTmune. In the fourth quarter of 2016, we expect to report safety and efficacy data from this Phase 1 stage.

We also expect to initiate subject enrollment in the fourth quarter of 2016 for the randomized controlled open label Phase 2 stage of the study, which is intended to enroll 30 subjects, undergoing mobilized peripheral blood HCT with ProTmune. And 30 subjects undergoing standard of care mobilized peripheral blood HCT.

I will now turn the call over to Dan, who will highlight our expected participation in multiple industry leading scientific conferences over the next several weeks. Where we will be releasing new and encouraging data across each of our preclinical stage programs..

Dan Shoemaker

Thanks, Scott. We’ve continue to make exciting progress on each of our preclinical programs. And we plan to share highlights of our progress at industry-leading scientific conferences in the coming weeks.

We believe our progress and results will demonstrate the unique therapeutic value that can be delivered through ex-vivo cell programming and the highly differentiated profile of our programmed adoptive immunotherapy pipeline.

First, in 2015 we launched collaboration with the University of Minnesota to develop an NK cancer immunotherapy with Jeffrey Miller, Professor of Medicine and Deputy Director at the University of Minnesota Cancer Center, who is a renowned NK clinical investigator.

We believe NK-cells offer several unique advantages over T cells for using cancer immunotherapy. The anti-tumor activity of NK-cells is independent of tumor antigen exposure.

The inhibitory mechanisms of NK-cells prevent the killing of normal cells and tissue, and finally the synergistic interaction between NK-cells and antibodies enables efficient and a highly-targeted killing of cancer cells. On May 16, at The Innate Killer Summit in San Diego, California Dr.

Miller plans to present preclinical data from our small molecule programmed adaptive NK-cell cancer immunotherapy program, which we are currently advancing through clinical translation. Dr.

Miller plans to highlight the enhanced direct anti-tumor and antibody-dependent cell-mediated cytotoxicity of our adaptive NK-cell immunotherapy as compared to other NK-cell populations. Additionally in a several presentation Dr.

Bob Valamehr, Executive Director at Fate Therapeutics plan to present the small molecule discovery platform that we used to identify FT1238, the modulator that we applied to program donor source NK-cells to generate a unique biological properties and the therapeutic potential of our adaptive NK-cell cancer immunotherapy.

We also launched collaboration in 2015 with Boston Children’s Hospital to develop an immunoregulatory CD34 cell therapy with Dr. Paolo Fiorina, Assistant Professor of Pediatrics at Harvard Medical School and a renowned type one diabetes investigator.

We believe CD34 cells hold the unique potential to immunologically check T cell autoreactivity, which is a hallmark in certain autoimmune and inflammatory disorders. CD34 cells have several defining characteristics which make them ideal immunoregulatory cells.

First CD34 cells are naturally trafficking cells with the potential to rapidly migrate the cells of – to sites of T cell activation CD34 cells also possess potent immunoregulatory capabilities that are independent of specific antigen recognition.

Finally CD34 cells are easily sourced and manufactured and have been safely administered across HLA barriers to millions of patients in the clinical setting.

While we have previously disclosed our identification of a set of small molecule modulators that enhanced the potential of CD34 cell to traffic to sites of T-cell proliferation and expressed powerful immunosuppressive factors including PD-L1 an abstract of closing new preclinical data will selected for presentation at the American Diabetes Association 76th Scientific Sessions, in New Orleans, Louisiana from June 10 to June 14.

During this conference, we plan to provide a detailed preclinical update on our program CD34 immunoregulatory cell therapy and discuss its potential for development in autoimmune and inflammatory diseases. Finally, we continue to make great strides in advancing the use of pluripotent cells for off-the-shelf engineered cancer immunotherapies.

We believe our off-the-shelf platform which combines genetic engineering of pluripotent cells with rapid and efficient generation of the immune cells has the potential to overcome key limitations of patient-source cell therapies including the requirement to source, isolate, engineer and expand cells for each individual patient.

Using a proprietary cell line based approach, we have demonstrated the potential to officially insert genetic constructs to promote multi-dimensional functionality including cell persistence, tumor targeting and checkpoint inhibition and to differentiate engineered pluripotent cells in the functional immune cells.

At the International Society for Stem Cell Research Meeting in San Francisco from June 22 to June 25, three aspects have been accepted for presentation covering our off-the-shelf engineered cancer immunotherapy platform.

Additionally, we plan to host a three hour focus lesson entitled, Fighting Cancer with Off-the-Shelf iPSC Immunotherapies, a session which will include leading scientific and medical investigators in the field of cancer immunotherapy as guest speakers to discuss the future for off-the-self cancer immunotherapy.

I will now turn the call back over to Scott, for review of our first quarter financial results..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Thanks Dan. For the first quarter ended March 31, 2016, Fate Therapeutics reported a net loss of $8.4 million or $0.29 per share as compared to a net loss of $7.9 million or $0.38 per share for the same period last year.

Revenue was $1.3 million for the first quarter of 2016, which was generated from our strategic research collaboration with Juno Therapeutics. Research and development expenses for the first quarter of 2016, were $6.6 million compared to $4.6 million for the same period last year.

The increase was primarily related to an increase in expenditures for laboratory equipment and supplies relating to the conduct of our research activities, including under our collaboration with Juno.

As well as an increase in personal expenses including stock-based compensation expense, resulting from additional headcount to support the conduct of our research activities including under our collaboration with Juno.

General and administrative expenses for the first quarter of 2016 were $2.6 million compared to $2.8 million for the same period last year. This decrease was primarily related to a decrease in corporate stock registration fees.

After adjusting for research funding proceeds from Juno of $500,000 and for stock-based compensation expense of approximately $800,000 total operating expenses for the first quarter of 2016 were $7.9 million.

At the end of the first quarter of 2016 cash, cash equivalents and short-term investments were $55.6 million debt outstanding under our facility with Silicon Valley Bank was $16.6 million and common stock outstanding was approximately 28.9 million shares.

In closing we are energized by the excitement and momentum that has emerged to support the advancement of our programmed adoptive immunotherapies. We believe ProTmune has the potential to prevent acute GvHD and CMV infection and to become standard of care across allogeneic transplantation.

We’re poised to begin subject enrollment in our Phase 1/2 study of ProTmune and we look forward to getting our first glimpses of its therapeutic impact in the coming weeks and months. Additionally, we have successfully leverage collaboration’s with pioneering investigators at leading medical institutions including Dr.

Jeffrey Miller at the University of Minnesota, and Dr. Paolo Fiorina, at Boston Children’s Hospital to build a deep unencumbered pipeline of programmed adoptive immunotherapies. We look forward to announcing preclinical data and our development plans for these programs had multiple industry-leading scientific conferences over the next month.

Finally, I would like to thank the members of the investment community that have continued to support our mission at Fate Therapeutics. We believe that the company is strategically well-positioned and that our programs are prime to begin demonstrating their novel therapeutic potential.

We look forward to sharing our programs developments with our supporters during the coming weeks and months. And with that, I’d like to turn the call over to the operator for any questions..

Operator

[Operator Instructions] Our first question comes from Jeffrey Lin from Leerink. Your line is now open..

Jeffrey Lin

Hi, thanks for taking my question. I’m substituting in for Paul. So I guess my first question, I’m just trying to get a little understanding on the independent CMV and GvHD monitoring committee.

Will this be sort of like a central review or could you probably give some little color on that?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Sure.

It’s a – there are two separate committees, one committee has been set up specifically with GvHD experts, the second committee has been set up specifically with CMV experts and these committees are – have been established to essentially adjudicate in a blinded fashion the primary endpoints that we’re looking at in the study which are cumulative incidence of GvHD at day 100 and cumulative incidence of clinically significant CMV infection at day 100..

Jeffrey Lin

All right. Great, thank you..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

So really their intent is in an unbiased basis to review the clinical efficacy of the product..

Jeffrey Lin

Okay, great.

My next question is could you perhaps give us a little bit of color on the Juno collaboration revenue like what does the collaboration revenue entail and is there could you provide any kind of like future guidance on the future revenues?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Sure. The Juno collaboration revenue on a quarterly basis is essentially comprised of two elements. The first element is the recognition of deferred revenue that comes from the upfront payment that Juno paid Fate when we entered into the collaboration. As well as the premium on the stock price that Juno paid Fate in buying its shares at $8 a share.

And so we recognized that premium and that upfront on a pro rata basis over the period of the research collaboration which is four years. That comes out to about $500,000 per quarter. In addition to that, we receive on a quarterly basis research and development funding from Juno.

That totals about $500,000 per quarter and presuming we perform those research services in that quarter we recognized that revenue also. In this – and that’s $500,000. In this particular quarter, we’ve recognized some additional revenue at the initial period of the collaboration back in May of 2015 when the collaboration was initiated.

Juno paid us research funding, where we did not perform services. In this quarter we essentially caught up with respect to the performance of those services and recognized additional revenue..

Jeffrey Lin

Okay.

And my last question is, for the NK-cells and the data present, do you happen to know if you have preclinical data suggesting that your NK-cells may retain – memory of like phenotype?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Yes. So absolutely we look forward to presenting that information. It was one of the original observations that Jeff Miller saw when he identified this particular phenotype of NK-cells which he calls and refers to as the adaptive phenotype.

One of the unique properties of this specific subset of NK-cells is the long lived persistence or memory like features of this particular cell type. And we will be discussing more – more specifics around that at next week’s conference..

Jeffrey Lin

All right, great. Thanks for taking my question..

Operator

Our next question comes from Jim Birchenough from Wells Fargo. Your line is now open..

Nick Abbott

This is Nick in for Jim this afternoon. Thanks for taking my question. Scott, you gave some fairly alarming statistics for acute GvHD and CMV.

Are those from the matched unrelated donor population or do those include higher degrees of mismatch?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

So it’s a great question. So the 40% to 50% range that we referred to with respect to acute GvHD is typically what is reported in the matched unrelated donor setting. That does not include for instance the mismatch setting where rates of acute GvHD can actually exceed 50% and approach 60%..

Nick Abbott

Okay, thanks..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

So that is the range – the range that we quoted is in line or matches the patient population, which we are targeting in our Phase 1/2 study..

Nick Abbott

So given that, I’m not saying patient [indiscernible] study but you’re just being conservative in timelines I mean with seven centers participating assume they all doing multiple transplants a month I would have thought unless you have other quite stroke exclusion criteria that you would be able to enroll this very quickly..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

I mean I think look, ProTmune is a new experience for us with mobilized peripheral blood, we’ve seen terrific enthusiasm to-date with respect to investigators interest in the study. I like to believe that we are being conservative with respect to our enrollment projection here.

But obviously we will know the answer with respect to have a better view on rates of enrollment in the weeks and months ahead. We feel very comfortable with the timelines that we’ve disclosed though with respect to the completion of enrollment in 2016 as well as the reporting of data and efficacy from the Phase 1 stage..

Nick Abbott

And maybe as a follow-up then obviously that – Phase 2 portion is small I’m assuming they could be supportive of registration but not registration enabling what do you think of Phase 3 looks like, do you think is placebo controlled or do you think you might have to go against an active comparator if one is approved?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

It’s a good question, it’s not a question. It’s not something we’ve discussed specifically with the FDA at this point in time, I think our assumption has been especially if we are going to run a or try to proceed with a single study as part of our registration strategy that we will include a control arm.

Others in this setting have included control arms at least in the setting of hematologic malignancies. To your point with respect to some of the more striking statistics with respect to incidence as well as the potential rates of enrollment. We do think we can run an efficient registration study even if it would include a control arm..

Nick Abbott

Okay. Thanks. I will jump back in the queue..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Sure..

Operator

Our next question comes from Do Kim from BMO Capital. Your line is now open..

Alex Xenakis

Hi, this is Alex Xenakis on for Do. Congratulations, everyone. Thanks for taking the question..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Sure..

Alex Xenakis

For the ProTmune clinical trial, can you talk a little bit more about what success of trial look like and would ProTmune still be success. If only there is an improvement on one of the endpoints.

Then which one would be more important GvHD or CMV?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Yes. So that’s a great question.

And I think from our perspective, when we’ve looked at the findings success criteria, we’ve actually heard given sort of the morbidity and mortality associated with GvHD and CMV infections, that really only a modest improvement is required to get transplant physicians excited about a potential product like ProTmune, especially given its simplicity, its elegance and its ability to fit within.

Cut the current paradigm of transplantation. GvHD if you talking to transplant physicians as sort of public enemy number one it occurs very early, when a patient experiences GvHD the immediate reaction is to put on immunosuppressive agents to try and deal with the GvHD when immunosuppressive agents are added, infections flare up.

So it is this sort of continuous cycle that builds when GvHD occurs. In thinking about what is more significant versus not there are really no preventive strategies for either GvHD more infections, so I think both endpoints are very viable with respect to a strategy in seeking approval.

I think both endpoints have a strong value proposition associated with them. Both are a leading cause of morbidity and mortality infections for instance, certainly are expensive, they extend hospital stays by as much as 20 days.

So from our standpoint when we really looked at this and we thought about whether or not we should promote one endpoint over the other in design the study, in talking to transplant physicians we ultimately felt like we should explore both endpoints equally as part of the Phase 1/2 design..

Alex Xenakis

Make sense, thanks. And one more question.

How do you see the transplant market growing with the new cancer therapies that are in development particularly in immuno-oncology?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Yes, another great question. I think the jury’s still out on that I mean we really need to understand obviously where the field of CAR T is – how that’s going to play out in the setting of AML. And certainly we need to understand the durability with respect to those CAR T therapies.

I mean it’s not talked about publicly or in significant circles but many patients that do receive CAR T therapy today still do go on to transplant.

So I do think in talking to this – in talking to transplant physicians that transplant is absolutely still going to have a role in the treatment of patients with hematologic malignancies and rare genetic disorders..

Alex Xenakis

Great, thanks..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Sure..

Operator

[Operator Instructions] Our next question comes from Ren Benjamin from Raymond James. Your line is now open..

Ren Benjamin

Hey, good afternoon, thanks for taking the questions. Maybe we can just start off with ProTmune. Scott, I think you mentioned that you are actively engaged with seven centers are these seven centers sort of on board and there are – approve the protocol, they’re just screening patients or are you still in discussions with them.

Can you just kind of give us some color as to….

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Yes, sure. Absolutely, we’re at the active stage of trial launch with these centers. And so to give you a little bit of color what’s involved with that – with the trial launch. Obviously we go through qualification visits and this is on the manufacturing side. As a reminder ProTmune is manufactured onsite at these clinical transplant centers.

So we have qualification visits, we have training visits, and then in addition to those interactions we also have site initiation visits. So with these seven centers we’re sort of it various stages of that continuum.

In the process of engaging centers across those different types of visits, we are beginning to look and in engage with the principal investigators with respect to potential subjects that could be enrolled in our study when after site initiation visits have been completed and we are able to enroll patients..

Ren Benjamin

Kind of based on that analysis do you have a sense roughly of how many patients let’s say, per month would be coming in and I’m just trying to get an idea as to when that Phase 2 starts and all these centers are onboard how quickly it may enroll?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Yes. At this point in time I don’t think we can give you guidance on that I mean the best sort of reference that we’ve referred to is other studies that have been run in the hematopoietic cell transplantation setting – for instance there was recently completed a large Phase 3 study. That was run to look specifically at prevention of CMV infection.

And that study I believe enrolled approximately 400 patients over two years..

Ren Benjamin

Got it. And then just kind of following on ProTmune. The Phase 1 portion for which we expect to see data at the end of this year. Is it essentially just a prelude to the Phase 2 or are we trying to optimize the dose.

What is the Phase 1 exactly trying to determine?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

So the Phase 1 portion of the study is – we have no just take a step back I mean ProTmune is a new product. It does not have any human experience. So the Phase 1 portion of the study is really to establish safety in those 10 patients. That said, in those 10 patients we are absolutely tracking rates of acute GvHD and rates of CMV infection.

The initiation of the Phase 2 study or portion of the study is not dependent on efficacy but is simply dependent on demonstrating safety in those 10 patients were safety is measured by rates of engraftment, secondary graft failure and death within the first 30 days..

Ren Benjamin

And will you look at different doses as well or….

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

No, it’s essentially a single dose. So in the allogeneic transplant setting patients get the dose that is received from the donor or available from the donor..

Ren Benjamin

Got it, okay. Sort of switching gears to the NK platform.

Can you talk a little bit about the difference between let’s say your franchise and other NK franchise that are out there? And maybe a little bit longer-term when do you think this could translate into the clinic?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Sure. I think, I would rather hold off on answering any of the details around that question for another week we are going to be presenting the program including Jeff Miller presentation of that program next week here in San Diego at The Innate Killer Cell Summit.

But generally speaking, we believe we are developing a very unique subset of NK-cells referred to as the adaptive NK-cell. We believe that that cell has long lived or persistent properties with memory like features, we will be discussing in detail some of these cytotoxic properties that we’ve seen with that particular cell both in vitro and in vivo.

And we will talk a little bit about our development plan for that specific cell therapy..

Ren Benjamin

Okay. And just one final one for me. The iPSC platform should we be viewing this – I don’t know maybe primarily as sort of the next generation of CAR T technologies or potentially the next generation of NK-cell technologies.

And looking at it from a benefit of manufacturing or how should we be looking at the future of this program?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

My personal opinion is I tend to think of this program as iPS cell technology in the field of cancer immunotherapy is disruptive. I think iPS cell technology and using pluripotent cells allows for the creation of therapeutics that could not otherwise be created with patient sourced cells.

And so from our perspective whether it’s an NK-cell or a T cell, I think using pluripotent cells allows us and using cell lines allows us to introduce multiple degrees of functionality that based on today’s technology just cannot be generated with patient sourced cells.

So for instance, I think as Dan alluded to, we do have the ability to insert constructs that can express or enable the certain therapeutic features like persistence, like targeting, like checkpoint. And so I do think that using cell lines you can create therapeutics that are not otherwise able to be created at least using patients for cells today.

I do think it also provides a significant platform on the manufacturing side where substantial degrees of efficiency can be achieved essentially being able to use engineer one-time created cell line and then deliver that therapeutic on-demand and off-the-shelf fashion..

Ren Benjamin

Got it. Thanks very much..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Sure..

Operator

Our next question comes from Ted Tenthoff from Piper Jaffray. Your line is now open..

Ted Tenthoff

Great. Thank you very much for the update. And I apologize if this question was asked already, I’m bouncing between a couple of calls.

When I say that the ProTmune study is ready to begin enrollment, how long do you think enrollment will take when you start kind of in the summer and when do you think we could actually get data from our Phase 1/2?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Yes, so the Phase 1 stage. Hi, Ted. So the Phase 1 stage of this study we disclosed that we believe we will have data on that Phase 1 stage in the fourth quarter of 2016. And we expect to complete subject enrollment this year. And we expect to readout data and efficacy of the Phase 1 stage in the fourth quarter of 2016.

With respect to ultimate enrollment rates of the study and its totality including the Phase 2 stage. I think it’s too early for us to gauge what the exact enrollment rates are going to look like, we’ve been pleasantly surprised by the level of engagement that we’ve received from transplant centers and potential investigators.

And so you may have missed this but we absolutely increased the number of centers that we’re targeting for participation in the Phase 1 stage of the study. And if that type of momentum continues we may elect to do that in the Phase 2 stage as well..

Ted Tenthoff

And then kind of this a factor of limited competition in terms of product in these days to be kind of offer to these patients in clinical trials is it excitement for the mechanism of action in the biology what’s really driving that interest?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

I think it’s a combination of factors I mean, I think as sort of we alluded to in our prepared comments, GvHD and CMV are leading causes of morbidity and mortality and essentially one of every two patients undergoing allogeneic transplantation is stricken with one of these two events.

And so given that I think there is a tremendous desire to search and explore for new therapeutics. That can address GvHD and CMV head on. Additionally, I think there is strong interest and a very attractive nature of our therapeutic itself.

We’re using small molecules within a transplant center to essentially improve the therapeutic potential of these cells.

It fits very neatly into standard of care and I think from a transplant physician center’s perspective, if we are able to have a profound effect on GvHD or CMV infection given the particular nature of our product and its ease of implementation. I think there’s just a lot of excitement around that..

Ted Tenthoff

Awesome. That’s very good to hear..

Operator

And we have a follow-up question from Jim Birchenough from Wells Fargo. Your line is now open..

Jim Birchenough

Hi, thanks for taking the follow-up. I just want to go back to the CD34 program.

What are the barriers stopping you from going into the clinic in fact given some of the impressive data that you’ve seen?.

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Again this program was accepted for an abstract presentation at the American Diabetes Association in – I think the middle of June. So we’re four weeks away from presenting that program. I’d like to hold off on any particular remarks about that program given the embargo in our directionality on that program until that time.

I’m generally speaking I will say CD34, you well aware have been explored absolutely in clinical studies have been given to probably hundreds of thousands of patients either in clinical trials or in the setting of hematopoietic cell transplantation.

The cells are safe, they are profound traffickers and we discovered that they have pretty interesting immunosuppressive potential. So I think – I think there is potentially a very clear path forward for us moving an immunoregulatory CD34 cell into the clinical setting..

Jim Birchenough

Okay, thanks. And then I just following-up on Ted’s question I guess it’s certainly you never as you said why you increase the number of centers..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Simply the reason we increased the number of centers which is based on our interactions with principal investigators. Over the past couple months obviously we had the opportunity to sit down with potential investigators at ASH.

We also have the opportunity to sit down with investigators at the Tandem meetings and while we were initially targeting participation of only two or three sites in the Phase 1 stage of the study. There has just been interest in demand from more investigators than we originally expected I would say.

And so we have decided to engage more centers and enroll and potentially have those centers enroll patients. There’s just been a lot of interest in what we do – in what we’re doing here with ProTmune..

Jim Birchenough

That’s good. Thank you..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Yes..

Operator

And I’m showing no further questions. I would now like to turn the call back over to Scott Wolchko for any further remarks..

Scott Wolchko Founder, Chief Executive Officer, Chief Financial Officer, President, Principal Financial & Accounting Officer and Director

Thank you. I’d really like to thank everyone for their participation in today’s call. A lot of great questions and we certainly look forward to speaking with you again in the near future as we move into the enrollment stage of our patients in the ProTmune study.

And disclose more about our NK-cell and CD34 programs at upcoming scientific conferences here in the next couple of weeks. Thank you very much..

Operator

Ladies and gentlemen, thank you for participating in today’s conference. You may all disconnect. Everyone have a great day..

ALL TRANSCRIPTS
2024 Q-1
2023 Q-4 Q-3 Q-2 Q-1
2022 Q-4 Q-3 Q-2 Q-1
2021 Q-4 Q-3 Q-2 Q-1
2020 Q-4 Q-3 Q-2 Q-1
2019 Q-4 Q-3 Q-2 Q-1
2018 Q-4 Q-3 Q-2 Q-1
2017 Q-4 Q-3 Q-2 Q-1
2016 Q-4 Q-3 Q-2 Q-1
2015 Q-4 Q-3 Q-2 Q-1
2014 Q-4 Q-3 Q-2