Q4 2020 Cellectis SA Earnings Call
Greetings and welcome to the select as the fourth quarter and full year, 2020 earnings call.
This time, all participants are in a listen only mode and <unk>.
<unk> and answer session will follow the formal presentation.
If anyone should require operator assistance during todays conference. Please press star zero from your telephone keypad. Please note. This conference is being recorded.
At this time I'll turn the conference over to Simon Harnessed, Chief Investment Officer, Simon you may begin.
Thank you and welcome everyone and selected the fourth quarter and full year corporate update and financial results conference call for the 2020 fiscal year.
Joining me on the call today with prepared remarks, our Doctor of Andre School, they kind of our Chief Executive Officer, Dr. Carrie Brownstein, our Chief Medical Officer, and Erik do Tom Our Chief Financial Officer.
Yesterday evening select the file its annual report and issued a press release reporting our financial results for the fourth quarter and year, ending December 31, 2020.
These reports and press releases are available on our website at select the dot com.
As a reminder, we will make forward looking statements regarding select the financial outlook and addition to its 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 and our most recent form 20-F filed with the SEC and the financial report, including the management report for the year ending on December 31, 2020 and subsequent filings and select it makes with the SEC from time to time.
I would now like to turn the call over to Andre Andre. Please go ahead.
Thank you Simon.
Good morning, and thank you everyone for joining us today.
The 20 has been focused on advancing our company objectives.
And the challenges the world is facing so like the says achieved key milestones and we are incredibly grateful.
And I'm proud of all the hard work, we achieved by our team our partners and our stakeholders during this challenging year.
Some of the progress we've made in 'twenty, and 'twenty, where I'm moving into 2021 determine.
And then to a cure.
We're thrilled to share with you.
Over the next half an hour this progress and all of these vision and an exciting future for select us.
And 'twenty 'twenty, we fully entered into clinical development of.
The first three wholly controlled clinical programs.
With our pioneering allogeneic <unk> car T cell programs in and out AML and multiple myeloma.
The lumpy alternative targets to the overcrowded.
The Knights and became a landscape.
We have already shared early data on our L and O program Bally zero one at Ash despite.
December.
All three programs are currently enrolling patient and phase one dose escalation trials and.
We are investigating differently from the policing depletion regimens.
While the 'twenty 'twenty was an extremely challenging here with the significant impact on logistics. So.
This successfully completed the construction of our in house manufacturing facilities on time, and Raleigh, North Carolina, and and Paris, France.
Both facilities are now up and running with the tech transfer between Paris and rally happening in the first half of this year in order for a rally to start manufacturing JMP <unk> car T cell batches and the second half of this year.
Successful product of Walkman is highly dependent upon manufacturing from stable critical supplies to drive the execution of our clinical plans.
And shrink consistent quality and meeting regulatory expectations.
And our facilities and rally and Paris will allow us to achieve a large degree of spending fact from independents.
Which is one of the most important value driver for any cell and gene therapy company.
We expect to achieve this.
By year end and believe this will propel selected further ahead as we continue to lead this class of felt and.
The therapy copies.
Our clinical execution strongly accelerated during 2020 as we established a world class team of clinical experts from the car T cell and the hematology and oncology space.
Brown, Chief our Chief Medical Officer, Doctor, Carrie Brownstein, who joins us from Celgene.
Terry and her team have been instrumental and establishing a broad clinical presence for selected within seven of the top U S heart hospitals, giving us and unprecedented access to a large patient population from which the enroll into our clinical trials and.
In addition to expanding our general management and global manufacturing team with top talent.
We appointed Mr jump here Jeremy.
And the chairman of the board of Director and November 'twenty 'twenty.
<unk> is the season leader with deep AIDS of the experience within the global Biopharma industry, most notably and the previous CEO of Black So smithkline.
From gene editing to car T cells like this all has always been the strong scientific leader and the.
In the space.
As an example.
Sure of magazine recently ranked top of organizations in term of number of patent filed and owned and the car T cell space.
And this shrinking selected speaking it become at the fourth institution worldwide sharp behind Japan.
P M and.
Novartis.
And the same publication and among the top 20 car T cell inventors six so close to one third of our from select.
This is of Great achievement.
We're also lifted at the number one biotech company and the study.
And this is just for car T and I'm not talking about gene editing.
Yes.
And our leader and this field and we have plenty of followers.
And the business development front, we're pleased about our recent agreement with site told you of Therapeutics, which we announced last month.
This partnership lead us to expand our town and gene editing platform into I P. S T J.
The rise natural care of cells.
And chimeric antigen receptor so car NK cells for a series of different tumor types and.
We will be responsible to the custom tailoring, which will be used by site Tovia two added ice tea of cheese to be differentiated into NK cells addressing multiple gene target for fear of pretty cute and several cancer indications.
I told you will be you will conduct the development of the mutually agreed upon.
There are pretty candidates and we will be eligible for up to 716 minutes and $716 million of development regulatory and sales milestones as well as the single digit royalty payment of net sale.
We will also received an equity stake of.
$15 million and I told you of stock as well as an option to invest and future financing round, which will allow us to be part of I told you as the growing value driver.
Together with the research collaboration and exclusive worldwide license agreement with idle time on gene edited tumor infiltrating lymphocytes.
We announced last year. This partnership shows the growing attraction and relevance of our talent platform as the gene editing solution of choice for cell therapy.
We're looking forward to see these next generation gene edited cell therapy programs become a reality for cancer patients.
Our partnerships with allergy and of Turkey are also gaining momentum and further establish establishing our growing allogeneic car T platform value.
Allergy presented initial data from the Allo 715 program and relapsed refractory multiple myeloma at the Ash in December of 'twenty 'twenty.
The first dataset ever presented on the knowledge and ex cell therapy targeting B C and a.
And this initial data readout 31 patient treated with Allo 715.
And what were Evaluable for safety and 26 patients were evaluable for initial efficacy.
Allergy and is now moving into the next steps and the study.
Namely optimizing cell doses and meant for depletion.
And plan to provide and update on allo seven one and five later this year.
Allergy and also initiated of cohort exploring allo 715, and combination with the gamma Secretase inhibitor.
The euro gas state.
With their partner Spring works therapeutics.
And is on track to submit the 90 for Allo 605, the first true burkhart targeting b Sammy for multiple myeloma and the first half of this year.
Regarding C D and 19 allergy and presented initial data on 22, and then shall patient treated with allo 501 of which 19 were valuable for efficacy.
And I ask <unk> 'twenty 'twenty and.
Hello, two alpha allergy and allo five of one eight outside of two trial is designed to enroll the mall genus patient population forecast and relapsed refractory L. D C L.
And was recently granted fast track designation for the treatment of this population.
The Alpha two trial is designed to potentially move into pivotal phase III.
The trial.
The data supported which would make us eligible for milestone payments.
How low will be and assessing the best course of action for people, who trial and second half of this year.
And this product into the trajectory to potentially become the first ever approved allogeneic car T cell candidate and the world.
Finally.
We're excited about the third target license to allergy and see.
Saturday.
Entering the clinic this year with the traverse trial evaluating allo 316, and clear cell renal cell carcinoma. This.
This is our first license allogeneic car T targeting solid tumors and we're eligible for milestone payments for the first patient dosed.
In addition, further derisking, our all of our Allogeneic car T platform.
Our alliance with Servier, and allergy and our major value drivers and selected.
The agreement on CD 19 was amended last year now makes us eligible to up to $410 million and the Bachmann and sales milestone.
Plus low double digit royalty on sales and the licensing agreement with the allergy and 15 additional allogeneic car T targets makes us eligible to over $2.8 billion and true potential future milestone and pretty much.
Plus royalty on sales.
With that I would like to hand, the call over to Doctor Carrie Brownstein our.
Our chief Medical officer for an overview of FERC proprietary clinical programs Kerry. Please go ahead.
Thank you Andre I would like to start with our first proprietary product you cart 22 of our seating and 22 directed Talon gene edited allogeneic off the shelf car T cell product candidates currently being evaluated and patients with relapsed and refractory b cell acute mental.
Plastic leukemia or B a L L.
We presented preliminary data from patients enrolled and the first two dose levels of our phase one dose escalation trial Bali the euro wine at the American Society of Hematology annual meeting in December. This is the first publicly released data from select the fifth Bali zero, one clinical trial.
And.
As of the November 2nd 'twenty, and 'twenty data cut off seven patients were enrolled and five patients received new car 22, following the Olympic completion, with the diabetes and cyclophosphamide or ex seat pre conditioning.
And one patient failed screening and one patient with discontinued prior to the administration of <unk> 22, due to an adverse event related to the lift the depletion regimen.
Importantly, no patient experienced the fund and he can talk.
The city of immune cell associated neurotoxicity syndrome graft versus host disease.
Adverse events of special interest nor of grade three or higher adverse events or serious adverse events related to your card and 22.
No patients discontinued treatment due to a new car of 22 related treatment emergent adverse event.
Two patients in dose blah, blah blah and achieved an objective response of the complete remission with incomplete count recovery or Cri at day 28, one of which attains to attain the complete remission or CR at day, 42, and proceeded to hematopoietic stem cell transplant. After subsequent therapy with either to the NAV.
One patient and dose level to achieve the noteworthy reduction and bone marrow blasts of 60 per cent that screening to 13% at day 28 after treatment with <unk> 22, and subsequently progressed.
Who wants to take the competition was observed in all patients within the DLT period and core element of the analysis of your car T cell expansion and persistence remains ongoing.
We are encouraged that <unk> 22 demonstrated preliminary signs of activity at low dose level with F. Sealant, the depletion without unexpected nor significant treatment related toxicity.
Our next step is to evaluate and optimize the cell dose and once the depletion and order to augment these early responses.
We've added and arms of the trial and which we explored. The addition of Alan to the lab and anti C. D and 52 antibody added to the ex the length of depletion regimen, which we call FCA.
Which is expected to result, and a deeper and more sustained T cell depletion and thereby promote expansion and persistence of the car twenty-two yourself.
We are currently enrolling patients and dose level, two with FCA and we plan to give an interim clinical data update on this cohort and the second half of this year.
Coming to your card and the S. One R. C S. One directed.
And gene edited Allogeneic car T cell product candidate being evaluated and patients with relapsed or refractory multiple myeloma.
We collect and preliminary translational data from the first group of patients enrolled last year, and we plan to share and early look at this data and the first half of this year.
Similar to the CMA and C. S. One the widely and consistently expressed target on the plasma cells of multiple myeloma.
Interestingly the S. One has also expressed and other immune cells, including T cells and NK cells.
<unk> is uniquely positioned to leverage this important myeloma target because through the use of our gene editing technology, we are able to knock out the S. One from the T cells prior to inserting the C. S. One directed car, which avoids the T cell fratricide during manufacturing.
And the additional differentiating factor is that our U car T. S oneself self generate lots of depletion and we're targeting C. S. One expressing lymphocytes and thus did not require additional pre conditioning, where the C D and 52 of directed antibody.
While the FDA had placed a clinical hold on this program last year following the patient death and dose level, two which occurred towards the end of the 28 day observation period. This whole of the has been inflicted with updates of the protocol and the study has resumed.
Next is new car at 123 of our C. D 120th of redirected talent gene edited allogeneic car T cell product candidate being evaluated and patients with relapsed and refractory acute myelogenous leukemia.
This program is the dressing one of the highest unmet medical need of population and the successful car T cell program and relapse and refractory AML could be of significant paradigm shift per patient.
Our current clinical product with an enhanced version of your cart 123, which includes updates of the manufacturing process and incorporate the C. D 32 knockout to allow the use of and F. C. Eight months of depletion regimen.
We are currently enrolling in our phase one dose escalation trial with two study arms, one with Etsy and one with FCA and once the depletion enrollment in both arms is ongoing and the data obtained will give us great insight into the car T cell kinetics and host lymphocyte recovery and using both strategies. We are looking forward to sharing the data from the program line.
Available.
Building on the technology platform of our current proprietary clinical program I would like to add that we plan to submit our IND and initiate new clinical trials. This year for novel proprietary product candidates targeting additional oncology indications as well the genetic disease setting we look forward to sharing additional information and the near future.
Our licensed Allogeneic car T cell development programs are making great progress as well as Ondrej mentioned before this year, we are expecting rich clinical data flow from allergies and survey from our licensed C. D and 19 programs and relapsed refractory NHL and from allergy and for the CMA program and multiple myeloma and various medical meetings.
And further validating the allogeneic car T cell approach, which we invented and pioneered with that I would like to hand, the call over to Eric Our Chief Financial Officer for a more detailed overview of our business development activity. Eric. Please go ahead.
Thank you Carrie and good morning.
Before I provide a brief overview of kind.
And short for the quarter and year end.
And to spend a few minutes on some of our business development activities in 2020 and need.
Early 2021.
In particular.
The beginning of 2021, where notes all of our alliance with type of young which includes up to seven out of it and six.
$60 million of development and figure out jewelry and SaaS and my students and we will also achieve single digit way I'd keep payments on the net sales of all products commercialized by sector.
We also received and equity stake of $15 million inside of Yahoo took all of the nooks and cash payment of $15 million yourself and conditions are not met by December 31st 2021, as well and auction to invest in future financing round.
<unk>.
In 2020, we announced our collaboration with <unk> and gene EBIT Tls with significant undisclosed development and sales milestones plus royalties on sales along with an amendment of our.
And now sheep agreement with Celgene.
The two phones by the $10 million in development and SaaS my students per dose.
Low double digit royalties on sales.
For the later.
We will potentially received in 2020, one milestone payment from the first people to try out of.
The five one and in large b cell lots of them.
Finding.
The remainder.
We are also eligible to receive up to two $8 billion in development and sales milestone.
High single digit royalties on sales from low gene and where do you get to 15 targets.
We will potentially we see in 2020, one milestone payment from the launch of the Travers trial of <unk>.
Low suite 16, and renal cell carcinoma.
Now onto our financials and the cash cash equivalents current financial assets and of which the cash position of selected excluding Kelly as of December after the first two.
2020, what two of them put and $44 million compared to query on the items and $4 million as of December 31st 2019.
This difference many reflects $28 million of Coty, we see from <unk> in the first quarter of 2020 and the E T.
Of 21 million thereof, the guarantee loan.
Which were offset by one of the and $2 million of or the net cash flows used in operating investing and lease financing activities.
And this cash position is expected to be sufficient to sponsored of key's standalone of Berry into late 2022.
This one with discount any future milestone payments.
The consolidated cash cash you keep items Q1 final sort of said and the rest of your cash position of celebrities including Kelly.
One 201, and 74 million the loss I spoke.
December 31st 2020, compared to 364 million that out as of December 31st 2019.
The net cash flow used in operating and capital expenditure and neither of these were at $86 million 60, and $44 million Kelly in the full year of 2020.
The net loss attributable to share our dose of facility. Excluding cash used was $54 million in the full year of 2020 compared to a net loss of $75 million in 2000 22019.
The $21 million increase in the net results between 2020 and 'twenty and 19 was primarily driven by a significant increase in order of and use that and the other income of $44 million, which was partially offset by you and increased.
And the expenses of $5 million, and a decrease and financial gains of 19 million and at all.
The consolidated net loss attributable to shareholders of <unk>, including Kt.
$81 million or one day, and 91 cents per share and the full year of 2020 compared to one of the one and $2 million of $2.41 per share in 'twenty and Nike.
The consolidated adjusted net loss attributable to shareholders of Citic piece, excluding noncash stock based compensation expenses.
$67 million or one dawn and 77 cents per share and the full year of 2020 compared to 17 9 million data out of <unk>.
And 86 cents per share in 2019.
Well the laser focused to spin out of cash on developing the pipeline of 41 product candidate in the clinic and operating hours and set up the of manufacturing facilities in price and in writing.
On the other and I'll focus on maintaining and nutrition corporate infrastructure and enable more of limited growth in the G&A spend.
With that I would like to and the quarterback of up to one of the April and feeding remarks Alright. Please go ahead.
Thank you Eric.
The game.
Very proud of the organization and team that we have built over the past years.
We started treating the first patient and pediatric allo and 2015 under compassionate use protocol.
With the first ever gene edited allogeneic car T cell therapy.
Those babies.
And now he's young kids in school and continue to be tumor free.
This is what motivates us.
And party to get to work every day with the mission to save the lives and offer of treatment option to those cancer patients.
That have exhausted all other treatment options.
Fast forward.
From 2015 to today.
We now are fully integrated gene editing biotech company.
Seven therapeutic development.
Graham and clinical trials, including three wholly controlled targets.
Based on our appropriate repayment and gene editing technology.
To further expand our current clinical pipeline.
In the middle of finalize and preclinical work on the series of New Allogeneic car T cell targets and other product candidates.
We strongly believe.
And our preferred jury cell and gene therapy platform combined with our in house manufacturing facility and our <unk>.
The clinical pipeline and will lead to paradigm shift and the cancer therapeutics and cell therapies and general positioning us at the forefront of this promising scientific field.
With that.
And to open the call for Q&A.
Thank you at the sign will now be conducting the question and answer session.
The way to ask a question. Please press star one from your telephone keypad and of confirmation tone will indicate your line is and the question queue do.
Do you mean first start to feel a true most of your question from the queue from.
And for participants using speaker equipment and may be necessary to pick up your handset before pressing the star keys.
To allow as many as possible to ask the question today. Please limit yourself to one question and one follow up.
Thank you and our first question is from the line of Michael Schmidt with Guggenheim. Please proceed with your question.
Oh, Hey, guys. Good morning, Thanks for taking my question I actually had a question around your investment into the car NK space as part of the site to Olivia collaboration I was just curious if you could share maybe how the site until the technology and the.
Roche together with with the Europe child and approach how all of that.
My deferral of compared to what others are doing and the car NK space and the longer term, how you think car NK cell products might be position and lots of two car T products within the oncology indications. Thank you so much.
Thanks, Michael I appreciate the question and thanks for joining US this morning and at the time and very good question. You know, we make very selective and business development deals with very high quality technology partners, we want to be very selective as I said, because there is just such a well.
Both of the new technologies coming into the market and we think as I told you. It's one of the best partners and the Ips derived NK cell space.
It's obviously a space that catches a lot of momentum currently and we can make a significant difference here by adding our talent and gene edits and the collaborations and also.
Positioned more of an investment for selective geared to the fact that we actually have an equity stake and the company rather than an upfront cash payment and so we are very encouraged by the progress of the company. So far and we're continuing to be very excited about the growth that can come out of the collaborations.
You know as we said, we have obviously over $700 million and development milestones and regulatory milestone and some sales milestones.
But for US it's much more interesting to actually make a significant change to improve with gene editing what is a very of wonderful the scientific platform and on the Ips derived NK cell phone and so for us. It's a foray into this phase. This will include some solid tumor targets as well.
But without taking the focus and resources away from us to focus on our off the shelf car T cell treatments, which we think have.
And the best shots on golf are the targets that we're pursuing and.
Andrea feel free to add anything.
Well.
In fact, something that is important for us because there is a lot of collection of among all of the gene editing technologies, and we see more and more people the.
Net are focusing on talent and interest.
Because of the performance of the technology and the <unk>.
Quality of the film that comes out plus like this is the one stop shop and the field of gene editing.
Means we have the electroporation technologies, we have the the.
The talent and technologies, we have or other gene editing technologies, and we have a huge amount of experience and handling cells and editing them.
On the fact of between NK and T cells et cetera, and select this is focusing on T cells. I told you I was focusing on N. K, we cannot be on all fronts and we believe very strongly that the the focus that we have on primary T cells is very promising and very straightforward.
Currently because of the successes that we are starting to see and the field. The NK, we believe and them. We think cytokine of the best partner to do it but we don't think that select the strict spread itself into all directions y and the T cell space, we'd like primary cells for the simple reason the T cells are more.
More of a cocktail then NK cells and ours of course like different type of empty itself, but in the space of T cells and see the ACD force Gamma Delta T sand from memory et cetera. So it's the cocktail that is part of the know how of select this and how to extract it from a local back of the healthy donor and that's also.
No not within the reach of our immediate reach of filing of product on something that would be of sophisticated product.
And they're now we believe and the Ips he and the stem cell differentiation and something that we are very excited about the technology of five Toby on the come proud of from very prestigious labs and the way on the West Coast and we think the it's going to deliver very strong result of the.
Again.
Yes.
Okay. Thank you.
Our next question comes from the line of Gena Wang with Barclays. Please proceed with your questions.
Q.
I also have one question regarding alcohol per game partnership.
I'm just wondering on the if.
Any color you can share with the initial target and all.
Also we'll set tobi or select us be responsible and co manufacturing and.
The second question is if they go out of the first one no question that's why volume.
The clinical hold letter was much faster or earlier than expected can you give a little more color of what it was.
Looking forward and and what was the modification of the protocol.
And forward for all of the program.
Yes, Hey, it's the and I. Thank you so much for the wonderful questions.
And I can take the first part of the question and and then I'll direct it over to Terry who is the Chief Medical Officer, who can answer a little bit of the background on the C. S. One program.
So first I told me a day.
He will be responsible for the manufacturing up there in case of our programs.
Provide the technology for them needed to make the gene editing part, which is a pretty straightforward system that we have perfected and house and which we have already provided for for example, and <unk> programs for the gene editing approaches there.
The similar setup and we.
And we haven't given too much color around the target obviously of the company instead of a private company and you now have the benefit to move forward without disclosing the targets too early but the mix of solid and liquid tumor targets and.
And Carrie and maybe you could talk a little bit about the S. One. Thank you so much.
Yeah.
Yeah, Hi, Gena and nice to hear you and so yeah. We were really pleased with the lifting of the hold back in the fall and.
And and you know as we had previously disclosed to everyone that the hold initially was due to a patient death at dose level two during the dose limiting toxicity window and we.
We are planning on sharing data and hopefully very soon.
From the first group of patients from last year prior to the hold and with that we will be providing additional details on what we were.
And what we've done to reopen and the study.
Our next question is from the line of the Celgene and Richter with Goldman Sachs. Please proceed with your questions.
Good morning, Thanks for taking my questions and just wondering if you could provide us with some timelines for the.
And the the the program the new program is moving into the clinic be it genetic diseases that you mentioned, where you're looking to fall I M DS or these natural killer Ips C cell programs.
Yes, I used the word salvia and thank you so much.
So first of all of.
And the timelines for our new and ease that Carey mentioned briefly on the call and this is something we will talk much more about this year as we're progressing towards and E filing and what we do want to show is that we have been quiet for the last couple of years on our preclinical development and all of gene editing platform why.
At the same time actually making a lot of progress on that front and we would really like to show the street what is under the surface at select us because we are a world leading gene editing company with a platform and T cells and internally, we have partnerships with leading companies and NK cells and tilt.
And we have a gene therapy program that we would like to really put front and center as the new pillar of value for the company, but for US the biggest focus and time line. This year is it's getting very exciting with our clinical development of our current proprietary car T program, we are advancing to the dose escalate.
And phases that we mentioned so we expect some very interesting data flow of out of our current clinical programs and then we expect new and I N D filings within 12 months, So bear with US just a little longer I think we'll give more information and the second and third quarter of this year and as we're good.
And really close to the filing.
Yeah.
Thank you.
Our next question is from the line of hard Taj Singh with Oppenheimer. Please proceed with your question.
Great. Thank you for the question and and all of the progress.
Just one question.
Few companies.
And out in the field.
All of them.
The problems locating things as simple as pipettes also plasmids.
And et cetera.
It seems to be vacuum and vacuum the up into the COVID-19 space and how.
Andre how are you positioned with your Paris, and urology facility and this regards.
And so being an economist by the end of this year and then I just have a quick follow up off of that thank you.
Well. Thank you very much heart touch for the question and I think it's an important question and we.
We took the decision in 2018 to start constructing our manufacturing plan and we would take a took a decision.
Worth to split and order to go faster on the first phase and the because the second phase would be longer. So the first phase was essentially to built up and manufacturing plant fully GMP to manufacture DNA.
<unk>.
<unk> for the Palin and vectors to bring to the the cars and five itself and that's operational since 2020 and start producing imagine.
And in 2020 with the Covid crisis trying to order and the mrna with what's happening and there, even if plasma and et cetera, it's becoming a real complication and.
And when I'm, saying that select is full integration of kind of one stop shop growth like from the idea of the bench and so we can manufacture everything all the raw materials.
Make of car T and <unk>.
And so internally we can immediately have done we have all of the quality system around as we have all of the vectors or the mrna everything gets shipped to rally round is going to go operational this year and the cell and gene therapy is becoming a more and more tense market and the fact that we can't build everything internally, including sometimes the <unk>.
But the electroporation technologies our hours. So we can tune up all of the little details around this.
And I believe two day and 2021 and it's one of the biggest strength and the differentiate the company very strongly.
Great Great. Thank you Andre.
And then just a quick question on your 1% to three I think last year with the new on the you had mentioned you know the U.
You'd be looking at patients with maybe a better performance status, maybe a slightly younger group of patients.
And there might be.
Little bit more of a window between the core between doses.
Has that changed or are you still sort of going through those initial phases of that Oh that escalation protocol and thank you for all of the question.
Hey, guys, maybe I can take it and I can hand, it over to carry them. So we're currently dosing patients at dose level two and.
And the arm of addition of Alan to the map to the pre conditioning cycle.
And so we actually do two arms of the study and parallel one with Blue Sky and one of his flu type of plus Alemtuzumab and Terry maybe you could take over here for the patient population with trading and.
You know and and the dose escalation plan forward.
Yeah. So thanks for the question. So you know as Simon pointed out we have two arms now opened and that trial and we're evaluating using alemtuzumab or not I'd like to use the attitudes of not been the patient population and I think it's important to point out and make that clear of patients with AML are.
And somewhat.
And our unstable at the right word, but it's the it's a it's a tough group of patients to crack they tend to be older. They tend to have poor performance status et cetera, and so it's really critically important to look at.
Precondition and using alemtuzumab or not because of ounces and that can sometimes add its own issues. So we wanted to be sure that said in terms of their performance and that's the protocol and it is.
How's the people with relapsed refractory disease, we have and.
And I went to ability built and to ensure that patients and.
We're enrolling patients that should be able to tolerate and and side effects that we would expect like by the kind of at least etcetera, but that said that the patients with and that will tend to be unpredictable.
Unpredictable.
What I'm very comfortable with and the proud of is made of a really strong medical team, particularly and the leukemia space that had been built and since I joined.
And I'm early last year, and a and we're very close with our investigators were very close and.
Speak with them regularly and discuss the patients and go over all of their criteria to ensure that we all think that this is the appropriate study for those patients.
So I feel confident that we're enrolling patients that can tolerate our products. So we can get to the answers and it's very difficult to treat patient population.
Great. Thank you.
Our next question is from the line of Hugo North of US with Citigroup. Please proceed with your question.
Hey, Great Hi, Andrew the assignment and Carrie. Thank you very much for taking the questions I just had one of them new card and 22 can you quantify what you mean by the correlated the analysis of the cell expansion and persistence do you mean correlation of the cell expansion and persistence with response and will this analysis determine whether you need to adjust the length of depletion regimen and.
And then a separate financial question you just could you just comment where you stand with respect to your stake and Calix is that something you would consider selling the further financed the company. Thank you.
Yeah. Thank you. So maybe first question for Kerry and and I can start with the second question and really quick.
So for Calix.
And we're over 50% shareholder and the company.
And we loved what the company is doing and they actually have what we believe is the very strong period of growth ahead of them. So.
The police monitor their earnings call as well that just happened last night or yesterday.
It's the wonderful company with scars to the value because it's the only publicly traded gene editing company and the agricultural space currently with.
And with a full focus on sustainability and improving crops and the AG space for the consumer consumption.
It's again something that we don't expect anytime soon.
Terry if you want to answer the question from 'twenty two thank you.
Yeah sure. Thanks, so much so correlate of analysis or basically all of our translational work, where we're going to be looking at and the lymphocyte recovery the subsets of T and K B cell subsets as they come back and correlating that with our expansion data omnicell.
And the cartels as well as the efficacy and where I'm looking at that very closely to help us understand what our best foot forward will be whether or not and we need to make any additional adjustments to the pre conditioning that trust and we can give the piece of your cart cells of the best chance of success.
Okay. Thank you very much.
Youre welcome. Thank you.
Our next question is from the line of Wang Qi Li with Ladenburg. Please proceed with your question.
Hi, Thanks for taking my question.
Two question two of the first one is regarding the new programs you would expect to R&D the sphere and the carrier mentioned, Oh, So theres a new direction for genetic disease. So my question is can you provide more color.
In terms of the number of new R&D. This year is just the one program and motive of programs and the for the genetic disease can you provide any more color on what kind of direction or space.
And Oh excuse me the ex vivo approach for genetic disease and any color would be helpful.
And then the last question is the on the car NK partnership.
Can you provide some high level color on the target and it's going to do it.
And I'd be.
And we'll let them with your current the cardio targets or is it is allowed to vote for some sand targets.
Thanks, Lindsey very good questions I will have to say short answer for the first question. We will provide more color on our programs that are of previously undisclosed and the second quarter of this year. So we.
We will give you all of the details then and I'm just the.
The rest assured it's the very exciting program, where we think we have a huge differentiating technology for them. So it's something that currently cannot be done with any other technology.
The second question on NK cells, and Rick do you want to give a little more color on the targets.
And believe we have given that kind of.
Well.
Hi, I'm on the well.
Well, we haven't disclosed the targets because it's more on the site of your outside to say this and like where are we.
We.
Everyone's coughing and everyone in the space and more specially the everyone coughing and.
And the space.
So if you want to hear about what we're doing there like it could fuel and even though the market the new ideas and we will definitely do this but like to prepare ourselves as much as possible to try to like move forward very rapidly, but then the site totally outside of I think that nothing of this was the close we're super excited by the Ids.
We've been growing together and.
It's no there is no information and we can unfortunately are on this outside the.
Outside of the fact of maybe waiting for the second half sort of like second quarter into Q.
To reflect the update on the pipeline the preclinical pipeline we have.
Okay fair enough. Thank you very much thereabout.
Yeah.
Our next question is from the line of beer and I mean with Jefferies. Please proceed with your question.
Yeah, Hi, guys. Thanks for taking my questions maybe on the.
And the myeloma program you know given the clinical hold was lifted and mid November when do you expect to start dosing patients.
And in that trial.
Carrie would you like to take that one thank you Barry and good question.
Sure absolutely. So once the hold was lifted we started all of the.
Downstream activities that would allow us to start you can tell the thing and you.
We have already begun the opening of our sites and should you know shouldn't be planning on the first patient with me the only thing hopefully very soon.
Okay, and then I guess of.
With any of these three programs do you anticipate that you'll have sufficient clinical data by end of this year, where you can make a decision to go into a pivotal cohort next year.
So our plans as of now our as as we have the data available to give a good package of data as I've said before that and meaningful.
And I think we previously stated I think and your cart 22 of them is moving forward on the most quickly and and that could be something that potentially and.
We could consider but we will have the it depends on what we see with our data and how things out and move forward.
Great. Thank you.
Sure.
Our next question is from the line of Nick Abbott with Wells Fargo. Please proceed with your questions.
Hi, good morning, and thanks for taking our questions and just.
And as you call. It 22 have you demonstrated direct evidence of how is the immunity tugs and you call. It 22, there's the correlation.
Between them to.
Tusa of reconstitution and.
D and disappearance of your kind of 'twenty, two but have you actually identified which host cells targeting the Yukon 20 T cells.
Yeah, no and disappointing we had Oh go ahead sorry.
No no.
Yeah sure I'm, sorry, I was sort of weighted yeah. So you know what we're doing all of that work, we're really looking to see.
To me and more important well both both things are very important and any of the solid recovery as well as the which sells are actually targeting.
The allogeneic car T two of them.
To be rejected and so we are looking at all of that data. We don't have that data available to share and that said I do think the key is really ensuring that we are able to keep the count recovery long enough for the cart cells to expand and do their business and I think that the addition of.
Alan She's the map and ensuring that we find the best way of including it for both safety and efficacy.
And it's really key for this year in terms of our goals and we will be looking at all of that data to help inform the best plan from moving forward, whether it would be in a.
Pivotal situation of our or otherwise.
And that's of Great segue to my second question, which is all of them to the med currently looking at 20 milligrams.
Consecutively for three days, so how long do you expect that to the effect of a dampening down the supposed to me and recovery how much variability do you expect to see and how long do you want to keep that Oh, it's the media recovery down.
Yeah. That's of Great question again, I think this all goes to the data.
We know from earlier data with you know with allogeneic transplant.
And at the sit out on the cheese and AD can and you know it.
Used and pre conditioning for transplant and typically.
And you would expect.
The and limit the depletion to be six weeks or so it can be for quite a long time, because it's the monoclonal antibody, obviously with the very long half life.
And so you know finding that balance where we're <unk>.
The thing the host immune system that day.
Long enough for the cells to ablate.
The leukemia, but not too long that you end up with other issues. It is a critically important piece of this puzzle and as we gather data I wanna be making data driven decisions on how we do that so to answer the question I can't exactly answer how long.
Because it's going to depend on what we see with the data.
Alright, thanks for the questions sure. Thank you.
The next question is from the line of Rajiv Prasad with William Blair. Please proceed with your questions.
Hi, there this is Sami anther Raj thanks for taking our questions and congrats on the progress.
And based on your data and the data from Allergan today. How are you guys thinking about re dosing of cross your trials and then also could you remind us if you plan on enrolling patients refractory to BT, and EE therapies and air and sea.
C S. One trial for multiple myeloma.
Absolutely carry you go ahead.
I said the second the the second part is on the second part of the question faster. So I'll start with that so in all of our trials, whether it's the CMA or see the 19 or other auto or allo car T and for that matter, we don't exclude them. So our programs, which I think makes them unique and exciting is that we're able to you and the.
All patients and potentially see of signal and patient who's already failed one of the therapy and would not be able to go on so let's say, if they had and adobe and see them and they may not go onto and alopecia and they for example, so it does give us more shots on goal because they are alternative targets and we're really the only people pursuing them and the allo space and Ah.
Large way so I think.
And that's really important.
The unique.
Property of our program. So the short answer is yes, they can come on and the study.
In regards to re dosing and absolutely you know one of the key most important and exciting pieces of allogeneic car Ts is the fact that we can read of it and so we are exploring that and these studies.
And we will be you know what do we have a date of the share would be exciting to share that with you all.
Thanks.
Thank you.
Our next question is from the line of Ingrid and singer with Kimpton. Please proceed with your question.
Okay.
Hello, and good morning. Thank you for taking my question acknowledged the it made the difficult for you to speak from your partners and the.
The the best of her knowledge when would you be expecting to hear something more from Servier analogy about their plans for your COVID-19, a O L.
GAAP and great. Thank you so much for the question. It's the very important point, because it's obviously a strong value driver for us not just from an economics perspective, and milestone revenue, but also from the platform validation perspective.
So you've heard 19 was the first product that selective brought into the clinic and 2015 and that has since been renamed Allo 501, and allo five of one that's equal to your card and 19. However, there's been a new version of yogurt and 19 that omits are built and off switch which is activated by rituxan.
And that program is called Allo, 501, and so and short, it's just and easier to produce product and that allows us to use the <unk> 19 of allo five of one with patients that have been treated with Rituximab, which is part of the standard of care approach and NH allocations, So allergy and and serve you are now moving.
And forwards and a unified fashion the allo five one a program and that Allo five one a program as the.
Allergy and has recently mentioned is slated for an update and the second quarter of this year at a medical meeting and so this is just a few months down. The line now wherever you will have an update on the patients treated with allo five of one and the allo 518 and.
And both of the company is upset that they would like to join forces to initiate a phase two study by the end of this year for this program, which could be potentially the the pivotal registration trial for this program. So it's a very exciting time for us and select us because we think the first program that we brought into the clinic as of Trailblazer.
Of the Allogeneic gene edited off the shelf car T cell space is now actually on track towards potentially the first product approval over the next 18 months or of 24 months. So.
Look out for news on that front from Serbia, and allergy and there will be data on this program mid this year and potentially and initiation of the phase two trial at the end of this year.
Alright. Thank you some of it was clear.
Our next question is from the line of Jack Allen with Baird. Please proceed with your questions.
Alright. Thank you so much for taking the questions I wanted to ask about your thoughts with regards to top of the timing of getting the Tau and based products and into the clinic and solid tumors. I know you mentioned that allo of 316 is expected to enter the clinic in the coming months, but was also wondering if you could touch on the timing with respect to advancing clinical products and with respect to the eye.
And since I Soviet partnerships as well.
Thank you so much.
Thanks, Jack Yeah. It's the wonderful question, we think solid tumor approaches are really the next frontier for cell therapy as this pertains to our partnership as well as to our proprietary programs that we're aiming to move into the clinic. So we mentioned growth in the past the new <unk> will also address solid.
Of targets from selective so proprietary targets to select us I think will make and interesting development case here, but as you said also on the tills and NK cell platform. So you know we wanted to Derisk and technology first and liquid tumors, where we've seen really great responses with our car T cell approaches and now we're expanding.
And that beyond liquid tumors and just solid tumors.
Yeah.
Awesome. Thanks, so much.
Thank you. The next question is from the line of Sumit Roy with Jones trading. Please proceed with your questions Hi.
Hi, Thank you for taking the question sticking with the.
In line with the prior question.
Could you give us some broad stroke, a thought process on Tac and probably seem like especially looking through the length of the Tovia deal are you picking winning strategies here with Ibs C and kisses line or how do you pick between gamma Delta of Alpha beta and.
And are you looking to you know tissue penetration of cell type or piece of the modification what what do you think we've cracked the solid tumor and not.
Terry maybe you want to provide some comment on that first.
Yeah.
And thank you so much for four of that correct Terry sorry, I was I was on the.
I'm not 100 per cent clear on the question to be honest with you. So just in terms of which.
Targets are not no.
So the cell type like.
Or you think still thinking Alpha beta T cell is the right set of type two to tackle the solid tumor or with title V of deal Youre, indicating maybe Ips the NK cells out of a better candidate for.
Oh, the tissue penetration and the solid tumor versus liquid yeah.
So I think that's a good question that we just don't know we don't know the answer to.
And you know and I.
I also would turn to and.
Andreas and for more color on that but I do think of but I, but I think it's critical here for us is selected.
We have the gene editing platform. So we can do all of these different.
Approaches to whether it would be of liquid tumor solid tumor or other diseases and I think that.
Applying this platform two different.
All of these different platforms, whether it be Ips CS Gamma Delta Alpha beta and what have you are.
Our important clinical and research questions that need to be answered because I don't think there's any answer and so I think it's what's important that we're doing here at select it says the word we have our feet our toes their fingers and all of these different potential options that will help patients in need and that's why I think being here, so exciting and because we can.
And make a difference and all the different ways and we will figure out what is actually the best strategy, what whether it be per a liquid tumors and solid tumor or other diseases.
I cannot agree more with carriers and the limitation that select this has is not technical we can do.
Everything technically our gene editing technology is extremely powerful and there is more and more papers that are coming showing that the access of palin to any kind of gain a and the precision of the technology and its accuracy and its specificity and the ability to.
To promote Danny recombination of replacement and correction of et cetera is unprecedented and this in this.
Arena.
And plus we have like duration et cetera, the limitation are not technical force like.
The problem is more of a focus of the company.
And also was for the past few years on the manufacturing, but we just unplugged the blockage that we have the manufacturing.
Internalized the buyback and this year 2021 will have no limit in term of what we can manufacture for clinical supplies, but also for commercial supplies and.
And also carry is building and a huge and awesome clinical team around the her from clean off the translational et cetera, and that's like the limit as more people and what we can do and of course, the finances of the company that have to fall longer.
We cannot spread ourselves on the thin layer of the problem is certainly not technology wise, because we own it all the problem is on the the focus of the company and I think we're really laser focused on what we're doing currently.
Alright, Thank you and good luck with the upcoming catalysts.
Thanks.
Thank you at the same we've reached the end of our question and answer session and I'll hand, the floor back over to Simon harnessed for closing remarks.
Thank you so much and again. Thank you all for joining US I'm you know, we'll have a couple of follow up calls off the deaths. So afraid of always reach out to me and again I'm very excited about this year, because I think theres going to be a lot of clinical data coming and we're all very much eagerly waiting. So thank you again for joining us and we'll speak to you soon.
Thank you and this will conclude today's conference you may disconnect. Your lines of at this time, we thank you for your participation.
And.