Greetings and welcome to the Cellectis Full Year 2017 Financial Results Conference Call. At this time, all participants are in a listen-only mode. A brief question-and-answer session will follow the formal presentation. [Operator Instructions] As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Mr.
Simon Harnest, Vice President of Corporate Strategy and Finance. Thank you. You may begin..
Thank you and welcome, everyone, to Cellectis fiscal year and fourth quarter 2017 financial results conference call. Joining me on the call today with prepared remarks are André Choulika, our Chairman and Chief Executive Officer; and Eric Dutang, our Chief Financial Officer.
Yesterday evening, Cellectis issued a press release reporting our financial results for the fourth quarter and year ended December 31, 2017. This press release is available on our website at www.cellectis.com.
As a quick reminder, we will make forward-looking statements regarding financial outlook in addition to regulatory and product development plans. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted.
A description of these risks can be found in our more recent Form 20-F on file with the SEC. With that, I would like to turn over the call over to André. André, please go ahead..
gene-editing on the one side and CAR T-cells on the other side. By combining these two technologies, we had set the trajectory of the concept of gene-edited allogeneic CAR Ts to become a revolutionary therapy in the field of oncology and beyond.
At Cellectis we are combining the cutting-edge, our proprietary gene-editing technology with the latest generation of CAR T constructs, resulting in an unparalleled proprietary knowledge of CGMP-grade cell manufacturing. We are taking the first steps in transforming cell therapy into genuine pharmaceutical product.
Since 2014, we have been producing through series of manufacturing campaigns large-scale gene-edited allogeneic CAR T-cell batches under internationally recognized CGMP standards, which led to the first-ever clinical application of an allogeneic CAR T product candidate, UCART19, for compassionate use back in June and November 2015.
These first patients treated with UCART19 for an aggressive ALL are still in complete remission and cancer free today. And our partner, Servier, continues to invent this next generation product candidate through clinical trials.
The driving force behind allogeneic off-the-shelf CAR T platform is our desire to make this ground-breaking treatment modality more accessible to patients and clinicians worldwide; and to remove some of the uncertainties associated with CAR T regarding cell quality, yields or manufacturing uncertainties as well as the time-lag between Asraises [ph] and injection, and to enable a treatment below the cost of individualized autologous CAR T therapy.
In one word, to this revolutionary therapy, CAR T, we plan to bring market access.
At the recent American Society of Hematology annual meeting or ASH in December 2017, our corporate partner, Servier, along with our clinical partner, UCL and King’s College, London, presented the first set of adult and pediatric CD19-positive ALL patient treated with UCART19 from their ongoing Phase I dose escalation trial.
Only patient who have exhausted all available treatment option, including the ones that failed enrollment into an autologous CAR T therapy were eligible to be enrolled in the trial.
All in all, five out of five pediatric patients were completely tumor free defined as MRD negative at the day-28 post-UCART19 administration dosed at 4 million cells per kilo. Five out of seven adult patients were completely tumor free MRD negative at day-28 post-UCART19 administration dosed with a 100,000 cells per kilo.
We’re also pleased with the safety profile of UCART19 to date with no serious adverse events related to GvHD and only two cases of cytokine release syndrome gathered – greater than grade 3 in 14 patients treated to date with no neurotoxicity.
We’re looking forward to more presentations this year by our partner on UCART19 that will include longer-term follow-up of these initial patients, as well as data from higher doses cohorts. These data demonstrate that allogeneic CAR Ts are not a hypothetical future for patients.
At Cellectis, we made that happen and it’s happening right now today in hospitals in the United States and in Europe. Over the past three years, in addition to the production of UCART19, we have manufactured another wholly-owned allogeneic CAR T product candidate, UCART123, which is currently in Phase I trial in AML and BPDCN in United States.
After a hold by the FDA in September 2017, UCART123 is back in trial at a dose at 62,000 cells per kilo as the new start dose for the dose escalation – for the escalation phase. Currently, we are manufacturing our third product UCART22, which is set to enter clinical trial in the second half of this year in ALL and non-Hodgkin lymphoma.
In 2018, we will build up on the clinical proof-of-concept of the selected UCART platform started almost three years ago with UCART19, by advancing our wholly-owned product candidate UCART123 and UCART22 through clinical trials. Cellectis are currently running two dose escalation trials with UCART123, the AML trial, which is led by Dr.
Gail Roboz at Weill Cornell Medical School in New York City. And the BPDCN trial is by Dr. Naveen Pemmaraju at MD Anderson Cancer Center in Texas. While, we already have a series of patient waiting to be enrolled in these trials there is one significant aspect to be considered for patient safety.
It’s an FDA requirement for all allogeneic donor-derived CAR T-Cell program to go through a dose escalation phase, where the dose limiting toxicity and the maximum tolerated dose are defined. This is one of the differentiating factor to autologous CAR T-Cell program.
The requirement enabled us to empirically establish the relationship between CAR T-Cell dosing and the anti-tumor effect of our CAR T-Cell. We expect to complete these dose escalation phase for UCART123 within the next 12-months before moving into the expansion phase in 2019, where we plan to enroll-up up to 150 AML and BPDCN patients.
This is where the true advantage of allogeneic CAR T-Cell manufacturing process kicks in. As we have already produced enough vials of UCART123 upfront to treat patient simultaneously through the expansion phase of these studies. AML is a particularly difficult to treat disease. The current standard of care leaves a huge gap in unmet medical need.
We believe UCART123 has the potential to significantly impact the tumor burden in patient. Our goal is to move through the dose escalation phases of these trials through 2018 to enable us to open expansion cohorts in 2019.
We expect to report an initial clinical data by the end of this year that details the initial safety and anti-tumor activity observed in the dose escalation trial.
Following in the steps of 19 is UCART22, an interesting target for the treatment of ALL and NHL in patient who either expressed CD22 from the onset or have relapsed CD19 negative for CD19 directed CAR T treatment and still expresses CD22.
We are currently in the final stage of cGMP manufacturing and we plan to submit an IND for UCART22 in the second quarter of this year to treat ALL and non-Hodgkin lymphoma patient with clinical trials in the U.S. to start shortly after.
While, we are in the first stage of manufacturing final stage – we are in the final stage of manufacturing UCART22, we are already running multiple production runs and process development for UCARTCS1 in multiple myeloma. UCARTCS1 is the first time that CAR T-Cell therapy will go after this target.
The trick is you need to knock-out CS1 or SLAMF7 from the surface of CAR T-Cells first, using gene editing before integrating a CAR that targets CS1.
We believe that UCARTCS1 represent a promising therapy for multiple myeloma patients build on one hand on the validation of the target by – produced by the use of alemtuzumab as a monoclonal antibody, and on the other hand by the promising results producing by autologous BCMA CAR T approach for multiple myeloma.
CS1 has the potentially advantageous safety profile and CS1 is not expressed on healthy tissues as BCMA is. UCARTCS1 will follow UCART22 with an IND filing plan in one-year. As a platform behind our clinical progress, Cellectis is housing one of the strongest R&D department our space.
One highlight was our recent presentation of our proprietary TALEN gene editing technology at the Keystone Conference in February. We presented a poster on an engineering CAR T-Cell with the TCR alpha and TCR beta knock out, beta-2-microglobulin knock out, the CAR-T knock in and NK inhibitor knock in.
While our single gene knock out deficiencies are close to 100%, this multiplexed gene editing platform including the use of multi gene targeting by DNA break-induced homologous recombination with an overall efficiency of 68%, showcase the power of our technology and knowledge.
We have shown that TALEN are capable of achieving simultaneous double knock out and – double knock in and double knock out efficiency over 68%. This is the highest efficiency to date achieved in such a multiplex knock out and knock in, in T-Cell with any gene editing technology back by a factor close of two times.
And this is just the glimpse at our capacity. In the past, we have shown that we could achieve at the R&D level, could robustly translate into cGMP production for clinical supply. This is our strength.
Therefore, this represent the next step in our manufacturing platform starting in 2019, we are planning to manufacture CAR T product candidate on the basis of targeted gene insertion of CAR T replacing the traditional antiviral vector approach. Another very important key aspect is yields.
Achieving high performance gene editing is great, but selective goal is producing pharmaceuticals, therefore, yield through all the manufacturing process is key. In 2010, we have acquired an asset of CytoPulse – the assets of CytoPulse, an electroporation company based in Maryland.
And we have heavily invested to develop the technology until to date to achieve very high level of cell transduction and viability post T-Cell transduction. We have perfected our proprietary electroporation technology to handle large quantities of cells like gently transducing cells with TALEN.
This results in very high transduction rate of cells in cGMP conditions. But most excitingly the cell survival post electroporation is also huge. This translate into high yields and of course low COGS. This is key. A large distinguishing factor for Cellectis in the gene editing space is our IP.
Our proprietary patent portfolio that encompasses a large umbrella patent in gene editing, starting by basic license homologous recombination patent, no nucleus needed, to the nucleus-based gene editing space using homologous recombination as well as non-homologous end joining, together with a specific patent families as they related to TALEN or Magnetelasers [ph].
We have recently also announced the issuance of specific patent related to the editing of T-Cells using CRISPR. However, due to the superior efficiency and plasticity as well as safety, we’re using TALEN for the manufacturing of all of our therapeutic CAR T-Cell programs.
Turning now to our partner programs, we have been extremely impressed with the progress made by our partners. Servier recently announced the expansion of UCART19 in multiple centers in the U.S. and the EU, once the dose escalation phase is completed.
UCART19 will move into the expansion phase in 2019, which could be followed rapidly by registration trial. We are looking forward to hear more updates this year. Pfizer has shown very exciting results on the first off-the-shelf BCMA CAR T program for multiple myeloma, and also the first allogeneic solid tumor CAR T targeting EGFRvIII for glioblastoma.
We strongly believe that successes of CAR T in solid tumor will be through allogeneic CAR T and gene editing. We strongly believe in the superior potential of the CAR T programs of our partner, Pfizer and Servier, both in terms of potential and span. We form today the leading team of the allo-CART space by far.
With a competition walking up – waking up on the huge opportunity, our intention is to accelerate. With that, I would like to turn the call over to Eric Dutang, our Chief Financial Officer, for a discussion of our financial results. Eric, please..
$38 million proceeds received as part of the Calyxt IPO; $7 million proceeds from the sale and leaseback transaction at Calyxt; $15 million of positive ForEx impact on cash flows, which were partially offset by $52 million net cash flows used by operating activities. The team continues to execute the strategy with financial discipline.
We expect that the cash, cash equivalents and current financial assets position of $297 million as of December 31, 2017, will be sufficient to fund current operations into 2020. Revenues were $34 million for the 12-month period ended December 31, 2017, compared to $56 million in 2016.
Collaboration revenues decreased by $19 million, which was notably explained by $8 million one-time milestones revenue recorded in the second quarter of 2016 in connection with UCART19, $6 million decrease in revenue recognition of upfront payments, and $5 million decrease in revenue of research and development services and supply of UCART19 products.
Operating expenses were $126 million for the 12-month period ended December 31, 2017, compared to $124 million in 2016. Excluding non-cash stock-based compensation expenses of $49 million in 2017 and $59 million in 2016, adjusted total operating expenses increased by $12 million from $65 million in 2016 to $77 million in 2017.
For the 12-month period, R&D expenses remained stable at $79 million, excluding non-cash stock-based compensation expenses, adjusted R&D expenses increased by $9 million from $46 million in 2016 to $55 million in 2017.
This increase in adjusted R&D expenses was notably related to UCART123, UCARTCS1, UCART22 and other product candidate development, including payments to third parties and cost related to clinical trials, purchases of biological materials and expenses associated with the use of laboratories and other facilities.
For the 12-month period, we recorded $45 million and $43 million of SG&A expenses in 2017 and 2016 respectively. Excluding non-cash stock-based compensation expenses, adjusted SG&A expenses remained stable at $18 million between both periods.
Net loss attributable to shareholders of Cellectis was $99 million for the 12-month period in 2017 or $2.78 per share, compared to a net loss of $67 million in 2016 or $1.91 per share.
Excluding non-cash stock-based compensation expenses, adjusted loss attributable to shareholders of Cellectis for the 12-month period ended December 31, 2017 was $50 million or $1.41 per share compared to $9 million in 2016 or $0.24 per share. I now turn the presentation back over to André for closing remarks..
Well, thank you very much, Eric. I would like to highlight again what quite remarkable progress was made in 2017 by transforming the off-the-shelf CAR T-cell concept into reality. Today, everyone knows that the future CAR Ts is in gene-editing. It’s a fact. We made it a reality.
I believe, we can say without a doubt that we have only just scratched the surface of what a powerful treatment gene-edited CAR T-cell therapy represents.
In 2018, Cellectis will be building up on the foundation set during the past five years and will reach a turning point in 2019, reaching expansion phase and extending our leading into the allogeneic CAR T-cell field together with our partners.
By the end of this year, three of the off-the-shelf CAR T product candidates will be in the clinic with our wholly-owned UCART123 and UCART22 programs as well as partnered UCART19.
Looking ahead into 2019, we will have two different oncology programs, ALL, AML and an expansion phase, and expect to advance additional UCART product candidate into clinical development with UCARTCS1 and UCARTCLL1.
This year and next, we will continue the robust development of the best-in-class manufacturing process of our next-generation CAR T therapeutics, including implementing our proprietary gene-targeted gene insertion technology into our pipeline of product candidates.
To support this progress, we will significantly expand our clinical leadership team and footprint in the U.S. and will invest significantly in proprietary assets, such as in-house manufacturing of commercial supplies with the state-of-the-art industrial process and large-scale multiplexed gene-editing.
At Cellectis, we think big and with a proof-of-concept in hand, we are pushing forward full force with our partners to bring allogeneic CAR T-cell program into the clinic and to the patients worldwide.
We also plan to file an IND for a program built on our TALEN gene-editing platform outside of the field of oncology in the future, extending our lead in the gene-editing space. We look forward to sharing more about our exciting work in this area at the medical meetings throughout 2018 and 2019.
With that, I want to thank you very much for your attention. And I’d like to open the call for questions. Joining me for the Q&A will be Eric Dutang and Simon Harnest. Operator, please go ahead..
Thank you. [Operator Instructions] Our first question comes from the line of Christopher Marai with Nomura Instinet. Please proceed with your question..
Hi. Hi, congrats on the progress and thanks for taking my question. First, I was wondering if you could further elaborate on your UCART123 studies and the type of data we might see yearend.
You discussed obviously the requirement to have dose-escalation for the allogeneic CAR T approaches and I was just wondering is it your expectation to have the recommended Phase II dose sort of declared in the yearend update for that program.
And then secondarily with respect to the expansion phase, how should we think about that? It’s about 150 patients. That looks to be about the size of some of the registrational trial. In AML, obviously, I guess, you’re selecting for patients with CD123 or that’s relatively also heavily expressed in AML anyway.
Can you maybe walk us through that approach with respect to registration? Thank you..
Well, thank you, Chris for these very, very good questions and good morning by the way. I thought it should be quite early for you. Yes, actually the dose escalation, we of course plan by like within one year to have the right dose.
We think that the start dose, we started with last year at 6.25x105, which led to the clinical hold after the injection of the BPDCN patient with a grade 4 CRS was probably a good dose.
But we’re now like 62,000 cells per kg, like 62,500 cells per kg, which is a very low dose, so we still think we’re below the optimal dose and we have to move forward into the dose escalation. It’s a three-plus-three dose escalation. Potentially, we can maybe, just for confirmation, have three more patients.
There is a lot of enthusiasm around like the product and we would like to – you will probably see that Cellectis is trying to accelerate this in the future.
So we’d like to push forward into a speed-up process and we are working with our Chief Medical Officer, they’re trying to speed – and our Chief Regulatory Officer to try to speed up this process to try to reach the time where we have defined this best dose. But the dose finding is a critical step in oncology.
If you go too fast in there and you miss the right dose, it might also hamper the potential of the product. So we are doing this extremely seriously. Now, for the second part of doing this seriously and we expect probably to complete this within a year. On the expansion phase, I said, up to 150 patients for AML and BPDCN, it’s like both trials.
We will of course be extremely open and try to dialog with the FDA to try to see what is the threshold to go for registration. But whenever we have results and meeting the endpoints, we will of course like run into registration. This is why also we’re starting to think about what should be a commercial supply manufacturing plan.
So we should get started for now..
Great. Thank you. And then, with respect to the UCART22 program, maybe you can elaborate on some of the patient populations you may go into with those CAR T naïve patients or would you go into patients who have previously progressed, having previously received CD19-based CAR Ts? Thank you..
The trial is, like in the trial we’d like to be totally agnostic like concerning patient that relapse CD19 negative, CD22 positive. The only criteria we’re looking at is the presence of CD22 on the surface of the B-cell malignancy.
One of the things also is like the – CD22, the gene, then CD19, so the stability of expression on the cells under there, variegation cell expression from patient to patient could be quite different. So what we will be focusing on is, of course, the presence of the tumor-associated antigen on the tumor surface.
We expect potentially to have maybe less of potential compared to UCART19 to go directly in patient untreated with CAR T or maybe we will have the potential to go with patient that have not been treated with CAR T. But definitely you have series of patients that do relapse CD19-negative and are CD22 positive.
Or potentially, you could use this in combo. So it could be also definitely very interesting way to be able to treat patient like with a shot of 19, hold by shot of 22. So the potential of off-the-shelf CAR Ts and the potential of repeat dosing and combo therapy is unexplored for now and we definitely have the intention to do this.
So it’s like really start like we’re not like hampering ourself of any, like preventing ourselves to go in like any direction for now, very open..
Thank you. Our next question comes from Pete Lawson with SunTrust Robinson Humphrey. Please proceed with your question..
André, thanks for taking the questions and good morning to you or good afternoon. Just I apologize, I joined late so if I – if you already mentioned this.
Just on 123 reopening, how many patients have you enrolled since November?.
I did not said it, how many were enrolled, but like the trial have restarted like since end of 2017 and, but, it’s not the number we communicated on..
Okay.
And you don’t want to talk about like how many already in or just what the pipeline is like for patients?.
No, not in this call. We’ll probably talk about this later. But for an earnings call, I think that is pretty too early to speak about this, like the trial have really like resumed since like the end of like 2017. It’s maybe early to start like giving numbers on this. We’re – you’ll probably see some information concerning this in the coming months..
Got you. Thank you. But it sounds like there’s pent-up demand, and wonder if you could also talk through additional sites that you will be opening for the 123 trial? Thank you..
Yes. Currently, it’s essentially at MD Anderson and Cornell for both trials and like the expansion will come up in the future. There’s probably two new sites that will open in the U.S. and potentially more sites outside the U.S. during the year.
So the real intention of the company to try to speed-up the patient enrollment and make like an expanded potential different type of protocols and therapies, and trying to tune this up, but our intention is to put the pedal in the metal..
Perfect. Okay. Thanks for taking my questions..
Thank you. Our next question comes from Hartaj Singh with Oppenheimer & Co. Please proceed with your question..
My part, on UCART22, it’s getting really interesting that you’re also now mentioning B-cell NHL, André, as an area that you are focused on the relapse setting there. What’s the – can you just tell us a little bit about what percentage of the NHL population is U22 positive in this relapse setting? ALL, I imagine is a lot smaller than going to NHL.
And then I got a quick question on also GvHD. We were at the BMT Tandem Meeting out in Salt Lake City, and it’s interesting in bone marrow transplantation. They regularly see about 7% to 10% of GvHD docs that are pretty used to dealing with it, but sometimes we get pushback from investors on the low percentage point GvHD that you see.
Can you just put that in context? What you are seeing versus what the BMT community used to dealing with? Thank you..
Hi, Hartaj. Well, thank you very much for these questions. So concerning GvHD, I am starting – I will start by the question on GvHD. Concerning GvHD with being dosing series of patients for now, between UCART19 and UCART123, and the process of manufacturing is fully similar because there is two editing in 19 and one editing in 123.
We can consider today as the problem of GvHD as irrelevant in our process.
The worst case scenario that we had was I think the first patient and what’s like probably one of the first batch we ever made clinically – like for clinical supplies was for UCART19 was the first patient that we dose as the suspected grade 2 GvHD and even though we are not 100% sure that this was related to ourselves and more or like the other – like the analysis of the skin GvHD that was detected, there was a grade 2 that disappeared in 48 hours after using topical cream seem to be the result of the previous bone marrow transplant that happened that failed.
But currently, for example, for adults we don’t have – like for 123, we’ve seen no signs of GvHD for adults, for the 19 we see no signs of GvHD and few skin rashes that could be grade 1 GvHD in two cases I think. So I – it’s cannot be compared with like allo-bone-marrow transplant for now.
And with the performance of cell filtration and the process of gene editing that we have and I considered this problem as, of course, something that has to be a real concern during the manufacturing, of course, and like the safety of the product, but the QC and all the details that we are dealing with can definitely lead with product that are super high quality and then don’t lead to GvHD, so it’s not a problem anymore and I think that part of the past after now.
Now like the first part of the question was?.
What part of….
Sorry..
Yeah, what part of NHL is CD22 percentage [indiscernible]..
Yeah, okay, okay, okay, I remember the question. So like 22, the expression of 22 on B-cells, it’s present on all B-cells, unless there is a mutation that leads to the disappearance of 22. It’s not that much of a problem. It’s more the variation in the expression of like CD22 under surface of cells. So you see there’s a wobble in the expression.
So certain patients are high expressers and this is fine. Some other patients are low expressers, where you have an iatrogenic population, and we don’t know exactly how much you can wipe out down the threshold of suboptimal expression of CD22 under surface. Now I get that once you’re starting using the CD22 as a target.
You’d probably fall into maybe a category of like pressure over the tumor, where you might have CD22 negative. But we know that, 22 is always expressed on B-cells up to now.
It’s like CD19 is very like much copycat with this instability and the expression that makes it may be a target that could be very valuable and used in combo with 19 for all the B-cell leukemia in general. So we like this product, we think it’s a very good combo product for B-cell leukemia, it’s a kind of the copycat of CD19, UCART19.
But we believe that also the rationale beside the portfolio and the combination of CART or using like the same old CART brings huge potential.
Now in NHL or ALL or CLL, we always can like find these population and our intention is not to – without regard to any other option that I cited, is trying to push down UCART22 in all the potential indication, where it could be useful, always in trying to get the patient that have the best expresser – expression for CD22. Of course….
Great….
But Logistics [ph] are developing a portfolio..
Sure. Got it. I really appreciate the answers. Thank you..
Thank you. Our next question comes from Wangzhi Li with Ladenburg Thalmann. Please proceed with your question..
Hey, good morning. Thanks for taking my questions. So just a few, may be started with UCART19. You said, you guys are going to present the data at the upcoming EBMT meeting on March 21.
Can you maybe provide a little more color in terms of – what we should expect the data, you mentioned a longer follow-up, could we also could expect more patient or maybe how many more additional patient, any additional color there?.
Wangzhi, hi, how are you? Like, you want me to have like problem with Servier, if I say a word on this, they will not be very happy about me, speaking about what they’re going to present. So like I will not – I cannot say this, because I very much respect my partners and I am not allowed to speak of other programs.
And second, I think, it would be really pity to start to disclose the results before like the physicians have the occasion to present these data by themselves. So I’m sorry, I am not going to be able to answer this question. But it’s a good question. Thank you very much..
Okay. No problem. Okay, so maybe you cannot answer. The second question too. So I just wanted may be your thinking in terms, you mentioned that the UCART19, we have the expansion phase in 2019 followed with the registration trial, which I assume the ALL.
I just what’s your thinking or your partner thinking right now, on spending UCART19 with DLBCL?.
I think our partners have the plan to develop UCART19 in all indications, not with everything that – like every tumor that could be targeted with like UCART19 will have a potential development.
But of course, started with ALL pediatric and adult, but you will very soon to see how much effort we’re putting on this and how much acceleration our intention is in the future. Yes, DLBCL will be part of it, but non-Hodgkin lymphomas, in general, and all other tumors that have CD19 on the surface.
The results we have obtained with ALL are very convincing to try to expand this into a lot of different type of indications. And I think that there’s absolutely no restriction behind this. No, this product is not meant to become only bridge to transplant. No, this product is not to become only for ALL.
This product is going to become major product for all B-cell leukemia as much as the expressed CD19. And as I said it, it could also be potential synergies with other products targeting B-cell such as UCART22..
Got it. And I’m sure, you can answer this one.
I just want to, if any updates on the two compassionate use patients, because, right now, they should have been followed up for long-time, right? Are they still in remission?.
Yes. These two patients are still in complete remission, we’re close to three years for the first patient like in June, it will be the case and we are up to 2.5 years for the second patients, it was dosed in November 2015. This is spectacular, seriously, because like they receive one dose of UCART19 and this is it.
These patients failed all other therapies including bone marrow transplant, blinatumomab, everything, it’s like it was really the first demonstration. And, of course, everyone was expecting these patients to relapse, which is I think not good for patients in general. And we are extremely pleased to see that like we do have long-term remission..
Great. Great to learn. Maybe last question, as you mentioned earlier in your call, the gene editing space, they’re getting very active.
So just maybe share some color in terms of Cellectis thinking or plan and go on the business development front for this year or next year?.
Well, yeah, actually there is like everyone is becoming extremely agitated about using gene editing in CAR T-Cells these days. And suddenly, hey, like it’s becoming the main ID and we’ve been talking advocating this in several years, and I know that most of you have been following Cellectis on this lead.
There is a strong conversation between lot of gene-editing companies, including Cellectis and other players in this field.
The future of CAR T, the future of cell therapy, if cells are becoming a therapy, then the future lies in the ability to program the DNA of these cells to make them better products, either for the strong tissue such as CAR T so it’s like ablation of tissues, such as a cancer or reconstructing tissues, repairing tissues or healing, and all these things.
So gene-editing is part of the future of medicine and it’s becoming a reality since three years since Cellectis is pushing this forward. And are we pushing forward to make more deals in this field? Cellectis has the intention today to bring our portfolio of product and beyond the product you already know to commercialization. This is my goal.
This is the goal of Cellectis. It’s becoming a commercial company in the future and being able to commercialize very important products for the patients, and promote the high standard products.
It has been the development part of the strategy, potentially to bring in new technologies, but our goal as we are today is essentially to try to bring 123 in to commercial, to bring 22 to commercial, CS1, CLL1 et cetera and develop the wholly-owned portfolio of products either for CAR T, but also beyond CAR T.
Now, yes, there is a lot of like business development interest all around our space today. And I think that 2018 is going to be a huge year for CAR T and gene-editing in general.
When I look over the announcement of our partners around, because there potential lot of INDs and new patients that are going to be dosed, so I think it’s going to be a real turning point, in the industry in general.
Well, with the past deal from like Gilead with Sangamo BioSciences that shows the interest at – it’s exactly the same type of deal we signed with Pfizer in like 2014, four years ago, like almost four years ago.
So we’re excited to see that finally other companies are starting to say while the future of autologous CAR T, it’s allogeneic CAR T, we’re not alone anymore..
Got it. Okay, great. Thank you very much for taking my questions..
Thank you very much. Thank you..
Thank you our final question comes from Biren Amin with Jefferies. Please proceed with your question..
Yeah, hey, guys. Thanks for taking my questions.
Just for the 123 trial, given other AML studies allow for bridge-to-transplant in patients that achieved complete remission, is this something that you’re allowing for the UCART123 study in AML?.
Of course, Biren, this is – hi, Biren, this is André. How are you? The – making a single shot in the patient did make sense back five or six years ago, when CAR T started to spring, because whatever you can get from like T cells, from a patient and you can do with those.
Then with the use of like antiviral transaction, then finally you inject everything you had and like you see what’s happening. With the potential of having off-the-shelf products and with the potential of the ability to develop a therapy and not just like a therapeutic procedure, there is a span of potential that is limited.
Today, we still have to find a right dose, what is the number of cells, the potency they have injected to the patient confronting to this tumor mass would give that much of activity or tumor melting at the end.
We have to know exactly the robustness in manufacturing, what is the activity of cells to melt down tumor cells, how many tumor cells they can melt. And once you get all these data, then the potential of developing different type of administration mode is unlimited.
And we have a strong conviction at Cellectis, in order to reduce potentially the initial CRS that we have, for example for the AML and the BPDCN patient last year in the summer, it’s potentially to go with lower doses of starts and expand the number of CAR T cells with repeat dosing, so the potential issues.
But first of all, we have to go through the dose escalation with a potential to re-dose afterwards and you will see that the protocol that was decided by Cellectis will expand its potential, in the future in different type of protocol settings, but we’re strong believers by this type of approach.
Not only this, but what I said in – like during this call, you can go after tumor that have, for example, multiple type of tumor-associated antigen, tackle the tumor with CD123, then retackle this tumor with let’s say CLL1 et cetera to be sure that you cleaned up all the cells with a repeat dosing one after the other in trying to reduce slowly the tumor for the comfort of the patient and his safety, but also the ability to totally clean up on the long-term to patient even at a potential to re-dose maybe six months after, I think nothing can be ruled out at the stage of today with a potential of opening a freezer and pull out a vial, whatever this vial is, and analyzing the tumor and how the tumor reacts.
I think that this is the key component to the CAR T therapy in the next decade and probably in the 21st century..
Great. Thank you..
Thank you. There are no further questions at this time. I would like to turn the call back over to Mr. Harnest for any closing remarks..
Thank you, everyone, for the great questions, and for obviously tuning into our yearend financial call. We really appreciate the interest in the company and look forward to seeing you at future conferences. Thank you so much..
Thank you. This concludes today’s teleconference. You may disconnect your lines at this time. Thank you for your participation and have a wonderful day..