Q3 2021 Cellectis SA Earnings Call
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Thank you for joining US today. This program will begin momentarily. Please remain on the line. Thank you for joining US today. This program will begin momentarily. Please remain on the line.
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Greetings and welcome to the select US Q3, 2021 earnings call at.
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I will now turn the conference over to your host Eric to time, Chief Financial Officer. Thank you you may begin.
Thank you and welcome everyone to a selected third quarter 2021 corporate update and financial results Conference call.
Joining me on the call today with prepared remarks, yeah don't tell them basically.
Chief Executive Officer, and Cary bounds, our Chief Medical Officer.
Yesterday evening each.
Its interim report and press release.
Hey, Paul you know our financial results.
Third quarter and nine months ending September 2021.
These people and press release are available on our website at <unk> com.
As a reminder, we'll make forward looking statements regarding <unk> financial final thought I would do in addition to its manufacturing you figured out what they can do.
These statements are subject to risk and uncertainties that may cause actual results to differ from so let's talk about it.
A description of these.
You know it must be some form 20-F filed with atg four.
On December 31st 2020, and subsequent filings <unk> makes with AT&T from time to time.
I'd like now to handle the Cordova.
Thank you John Good morning, and thank you everyone for joining us today.
Over the course of the third quarter in the last nine months of 2021.
So like this has achieved theories of key milestones.
Credibly grateful and proud of all the hard work achieved by our team our partners and our stakeholders.
2021 has been a productive year, thus far for selective we have made significant progress on all fronts that we're thrilled to share with you over the next half hour.
Notably.
Yesterday.
Select has announced that the release of two abstracts accepted for presentation at the third.
60, <unk> American Society of Hematology annual meeting.
So like this will present additional preliminary clinical data from its bally's year, one trial, a few car T 22 in patients with relapsed or refractory b cell acute lymphoblastic leukemia.
And presentation of our first preclinical data.
<unk> zero, one for the treatment of sickle cell disease.
Additionally, with regards to our preclinical your car T pipeline focusing on solid tumors. We have made notable progress with your cart metal.
Our allogeneic car T cell product candidate targeting the document expressing solid tumors.
Excited to share the first preclinical data.
Demonstrating in vitro.
In vivo anti tumor activity will be presented at the society for immunotherapy of cancer annual meeting later this month.
In 2018, Blake this made transformative decision to internalize the manufacturing, it's a pretty prolonged this decision we feel that crucial competitive advantage in today's world.
This investment in our GMP manufacturing facility provides selective with independents and control over its gene and cell therapy processes from buffers to DNA messenger RNA vectors and of course, our cell therapy, such as car Ts.
We craved and all our processes, our departments and our production in the cell and gene therapy space whoever owns the process all of the product.
Due to the success of completion of both of our GMP manufacturing facilities, we can move swiftly into an innovative.
R&D stage, two clinical trials with potential to produce commercial level supplies in the future.
We're proud to be one of the only companies of our size that are capable of moving into innovating new Ids from RMB, two clinical trial to manufacturing and delivery directly to the patient all in house, we believe that bringing manufacturing in house.
Could contribute to eliminating some of the barriers competitor are facing.
Our goal is to provide consistency and safety on our production and short lead time are met and adaptability.
As each disease targeted by our advanced therapies may require cutting edge innovation at the level of our manufacturing capabilities.
Need to fully mastered the process.
Fortunately, having all manufacturing in house means that we can rapidly version.
Promising therapeutic candidate as we monitor clinical responses, leading to the best possible prolonged registrational filing.
Our GMP manufacturing facility is now fully operational for the production of starting material.
On the other side of the pond, a Raleigh GMP manufacturing facility qualification of the facility equipment and systems was completed successfully in Q3.
Qualification of the second new car production suite equipment remains on track to enable start engineering runs of the third new card product in early 2022.
I would like to turn the call over to Dr. Carrie Brownstein our.
Our chief Medical officer to give an update of our three sponsored clinical trials and preclinical product pipeline.
Please go ahead.
Thank you Andre select has continued to progress our phase one clinical trials evaluating our three proprietary allogeneic car T cell therapies in hematologic malignancies, Bally's zero, one evaluator in your cart 'twenty two in patients with relapsed or refractory D cell acute lymphoblastic.
EMEA Amylene zero, one evaluating your cart 123 in patients with relapsed or refractory acute myeloid leukemia, and Melanie zero, one evaluating your cart C. S. One in patients with relapsed or refractory multiple myeloma.
As Andre mentioned 'twenty 2021 has been a busy and productive year for selective with our proprietary clinical and preclinical program, making substantial progress and we are excited to share additional preliminary clinical data from our bodies zero one trial at the American Society of Hematology 63rd Avenue.
Meaning next month.
The abstract includes updated preliminary results from the phase one.
Label dose escalation Bali zero, one study in patients with relapsed refractory B a L. L from the first cohort of patients who received you cart 'twenty two after fludarabine cyclophosphamide and Allen choose an absence of depletion.
The addition of Alemtuzumab to F. C was well tolerated deepen host T cell depletion and promoted car T cell expansion and persistence the data presented support the safety and activity of your cart 'twenty two after FCA lymphoid depletions in patients with relapsed refractory B E L L and the additional.
Data will be presented at the Congress enrollment in the study is ongoing.
Additionally, preclinical data from the child Logan's here at one an autologous gene therapy product candidate designed to repair the mutated beta globin gene and subsequently restore production of hemoglobin <unk> in patients with sickle cell disease was awarded a poster presentation at ash. The data that will be presented are the first demonstration that tail.
And based engineering could be used to correct. The mutation in the beta globin gene of homozygous sickle cell anemia patient derived hematopoietic stem and progenitor cells.
The data showed high level of hemoglobin, a expression where version of cycling phenotype. The capacity of <unk> zero, one edited cells to engraft in vivo in a low level of off target cleavage.
Collectively the data demonstrate high efficiency and safety of tantalum treatment and they just P. CS and positioned it as the best in class Gene editing technology for gene therapy product development.
We're also pleased to share that our partner Allergan will present data from our licensed allogeneic car T programs at Ash data from both the phase one alpha to study evaluating allo five of <unk> in patients with relapsed refractory non Hodgkin's lymphoma, and the phase one Universal study evaluating allo 715 in patients with relapsed.
Multiple myeloma will be shared in oral presentations.
Allergy and will also present a poster on data from their phase one Alpha study evaluating allo 501 in patients with relapsed refractory non Hodgkin's lymphoma.
The second quarter of 2021 we presented preliminary translational data from the first group of patients enrolled in the Melanie zero one trial of your Cart C. S. One at the virtual 24th annual meeting of the American Society of gene and cell therapy. The early preliminary data presented validates the S. One as a target for allogeneic car T.
Cells in multiple myeloma you.
Your cart T S. One expansion and persistence was observed and correlated with changes in relevant serum cytokines and anti myeloma activity.
Select is advancing one of the most robust allogeneic car T pipeline and we anticipate filing two additional new drug applications for two novel you car T product candidates in 2022 you cart 'twenty by 'twenty, two and your cart Meso U.
Your cart 'twenty by 'twenty, two with the first allogeneic dwell car T cell product candidate, which is being developed for patients with b cell non Hodgkin's lymphoma.
New cart meso is an allogeneic car T cell product candidate targeting mesothelin a tumor associated antigen that is highly inconsistently expressed in mesothelioma and pancreatic cancer and it's also over expressed in a subset of other solid tumors.
In addition to expressing measles beyond directed car your cart Meso also leverages Talon gene editing to overcome immune suppression mediated by TGF beta.
You caught me, though is being developed for patients with mesothelioma expressing solid tumors selected will present initial preclinical data that support antitumor activity of your cart views, though in a poster presentation at the society for immunotherapy of cancers, 36th annual meeting in Washington, D C and virtually on November 10th to 14.
In 2021 with that I'd like to hand, the call back over to Eric to Tom selected <unk>, Chief Financial Officer for an overview of our financials for the quarter. Eric. Please go ahead.
Thank you Carrie.
We provide a brief overview of our financials for the third quarter and first nine months of 2021.
I would like to highlight that the cache caching UN grandfathering for etc.
Cash position of selected excluding Kelly.
September such as 2021 was 201 in $1 million.
Compared to $244 million as of December 31st 2020.
This difference mainly reflects $92 million of net cash flows used in operating investing and niche financing activities, which were partially offset by $45 million of net equity proceeds raised from the competitor ATM program in April 2021, and $10 million of proceeds from this.
Doug or extending it.
This cash position is expected to be sufficient to fund 60 standard and our bench strength.
Twenty-twenty sweep.
The consolidated cash cash equivalents, John spinoffs for states and restricted cash position of 17.
Including Kelly was $216 million.
September 2021, compared to $274 million as of December 31st 2020.
The net cash flows used in operating capital expenditure and lease financing activities were $92 million at citic teeth and $15 million in the first nine months of 2021.
The net loss attributable to shareholders of Citic piece, excluding Kelly was 75 million thereof.
First nine months of 2021 compared to $21 million in 2020, this $54 million decrease in the net loss between 2020, one and 'twenty 'twenty was primarily driven by unique venues and other income of $30 million and.
An increase in R&D expenses of $32 million, which was partially offset by an increase in net financial gain by $8 million.
The consolidated net loss attributable to shareholders of celebrities, including at least $189 million or $2 per share in the first nine months of 2021 compared to 42 million got out Oh 98 cents per share in 2020.
The consolidated adjusted net loss attributable to shareholders of <unk>, excluding noncash stock based compensation expenses was $80 million or $1.79 per share in the first nine months of 2021 compound to $30 million.
Oh 72 cents per share in 2020.
We are laser focused to spend our cash on developing Deepak I know what are your one product candidate in the Clinique and operating I'll set up manufacturing facilities in Paris and in writing on the order I am I will focus on maintaining an efficient corporate infrastructure should enable more limited cost.
In CIT bank.
With that I would like to hand, the call back over to Andre for concluding remarks Andre. Please go ahead.
Thank you Eric.
Our allogeneic car T platform position us at the forefront of developing novel <unk> car T therapeutics that.
And the next generation of cancer therapies.
We continue to leverage our expertise in gene editing and clinical development to transform the lives of patients with cancer and rare genetic diseases and we look forward to continuing this effort in Q4 2021 into 2022 and <unk>.
Beyond our selectors.
We continue to leverage our groundbreaking gene editing platform to dislodge novel corporate or in medicine to transform the lives of patients with serious diseases.
Our truck brokerage or a clinical stage programs are focused on patients with advanced Hematological malignancies, and we continue to advance our robust pipeline into the clinic.
Additional college settings, including solid tumors and to address the unmet medical needs of patients with severe genetic disease.
With that.
I'd like to open the call for question and answers.
Thank you.
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Our first question comes from the line of YOD gallon they'll come of it with Citigroup. Please proceed with your question.
Yeah, Hi, Andrew I understand thank you very much for taking the question.
Question on required 20, Chillemi ice data.
And so I have a bit more detail with respect to that.
David you will see in the presentation and what is your internal bar it would qualify.
Basketball dataset.
Okay.
Well. Thank you so much for this question I think the most appropriate person to answer. The question is probably carrying you carried like to take this question.
Sure absolutely Hi, how are you all nice to hear from you.
So what we're planning on showing is the first handful of patients that were treated with the therapy and cyclophosphamide and Alan to the map.
Lynn for depletion regimen. So in the abstract is the first group that we had ready to prepare for the abstract but we will have additional data at the meeting and just no whereas as you know we're still in the dose escalation phase of the study. So we're not quite where we need to be from what we'd consider a bar for them.
Approval so to speak for that as you know we are in a very relapsed refractory group of patients. We will have patients who have previous CD 19, directed therapy for acute leukemia, I'm not expecting as we move through our clinical development plan.
For our first shot on goal so to speak with this product I would not expect the bar to be tremendously high.
Got it okay.
And then I just had a question I don't know if you caught me, though for the data to be presented to.
To the extent that you can comment can you give us a preview of that dataset and what are the key features of that preclinical data set to support.
Clinical development.
Gary do you want to also described.
You said it well.
As you want.
Okay. So Andrew I think unless you're in a better position to do that yeah. Thanks, Yeah. So like me, though has been developed it's I think it's like the first a off the shelf car T.
I called the complication that you've seen in other type of sourcing for allogeneic car T targeting meso and one of the features is extremely interesting that would be showed at ash on this is like the knockout of the TGF beta receptor on the cell TGF beta receptor is.
It always is the potential down.
Later for T cells in the tumor micro environment and one of the big challenges select us is tackling through older car T. We're developing for solid tumors is trying to.
Tackled the tumor microenvironment, the TM E such.
Such as Fab for example that is something that supposed to blow the cooler seemed like an article but it definitely fab is the mall.
Program, there will poke holes into the solid tumor protection and.
Meso is essentially a card that's not going to be shut down by the negative feedback loop that you have with TGF beta two that is present in the microenvironment in this type of data that we have we show also that this type of car will probably work in combo.
And that would probably give very interesting features not only for mesothelioma bark potentially for a lot of different type.
Of cancers, the FCA V with selected also with all the attribute this car has which is like a CDP.
From all the other car would've loved the CD 52 knockout so it will work on the Alemtuzumab and.
<unk> and TCR Alpha plus it's a triple knockout. So I think it's the first of its kind triple knockout car T.
We're currently preparing this cart to go into clinic and I think the data are compelling and definitely encourage us in vitro as well as in vivo frequent data against like the compelling results for <unk>.
Tackling series of solid tumors.
Thank you.
Thank you. Our next question comes from the line of Gena Wang with Barclays. Please proceed with your question.
Thank you for taking my questions I have a few regarding amount of big picture questions.
So the first one maybe I'm all for Andre wondering you know the your partner Allergan had the clinical hold and I think that that their earnings call. They common you know what they did not disclose too much but did come and there's some concern about what translocation. So just wondering kind of how is the read through to you.
All programs and then also regarding your drop product how much do you test regarding each step you know with the chromosome.
Translocation.
And how do you detect that.
And then my second question is regarding the.
<unk> Allogeneic car T approach in general we did see several companies and also your partner Allergan will.
We will share some data on consolidation approach and if we did see improved a complete response letter D. A.
Complete response rate and we don't know about the durability, but any thoughts you know from this approach like apply to your programs.
Well, thank you gena somewhat further questions.
First of all I think I'm going to let the allergy management team command.
Uh huh.
The clinical hold because I think it's like we're desperately.
<unk> supported very confident.
On the handling of the situation by the allergy team, that's making fantastic.
Work on their side.
Extremely excited by the data, they're showing on your car T 19, our allo 501 actually.
I think this is definitely a transformative.
Uh huh.
The.
Data on the allogeneic car special with the consolidation I'll come back to this.
To speak about the impact on select US there is like of course, we're monitoring the situation very closely we're helping as much as we can do whatever we can we will definitely help allergy too.
Go through the spirit and we have strong confidence in the product and the outcome and we believe that this is like this trial is going to resume.
Currently.
Nevertheless.
Chromosomal aberration or quite frequent and among human population.
Would it be certain chromosomes more aberration, because there are subject to.
January combination due to the stay where they are so like a lot of.
Our immune cells and T cells undergo again every combination at this stage.
At a certain stage and sometimes these DNA recombination can lead to some crumbled normal changes in there.
However.
Few things that should be said first.
The prologue did not in danger.
Like do you see that theres like here's a patient that have been dosed.
Those with the same platform. So we're confident on selected.
Global platform and are not only gene editing, but also the global platform, where we can block our allogeneic car T to be very stable. The second thing is that T cell adult T cells. You don't there is no cell line of adult T cell <unk> gel and then finally.
The.
The.
Cancer is like <unk> cel term cancers. This is all the beauty about the allogeneic approaches of gastric cancer, especially T cell cancer T cell cancer from like an allogeneic donors. This is not going to happen actually never happened in the history and I'd say its something that could be.
So we're quite confident with this and.
We believe the platform under contract will come stronger from this space and.
Also with a lot of confidence about what do we have so we have no concerns today on that potential.
Risk on what selected us and our partners are doing and we believe that this is like on an intermediate phase in there on the country, we're very focused and very positive on the outcome of the data that not only our partners are having but also select us eclipse producing and these all are <unk>.
Product and target that selective has been accepting and I think for the size of the company and driving so many life saving products something that is definitely the transformative and will remain with the same type of technologies.
That we have now on the consolidation study, it's something I've been always very strong advocate, but I think it could be interesting to have carriers view because I've been speaking about lack of consolidation studies since years, and we'll discuss this together Gina and.
But carry a to have like a pressure item myself.
More of a medical Doctor.
So Carey if you want to say a few words about this.
Sure hygiene are nice to hear from you as well so I'm really excited about the presentation that includes all of the consolidation treatment I think that and I've been saying this since I joined select is now a year and a half ago. I mean, one of the main Dvds and key point that I think is going to make allogeneic car T cell therapies.
Successful is the fact that you can't give additional doses and that's one of the huge drawbacks of the autologous setting we all know that these <unk>.
Additional treatment and whether we're using chemotherapy.
Chemotherapy, whether using antibody therapies, whether using small molecules remember it is in the history of treating any of these diseases has to do with being able to get the cancer cells to a state that not only you don't see them anymore, but that you can't measure them anymore and the deeper into remission you couldn't bring a patient to the longer.
Their duration as well as their longer their survival and potential for a cure so giving additional treatment is always on the right way to go when you can and and therefore, it really excited to finally being able to see a larger data set of patients who received more than one therapy in the allogeneic setting and showing that it's able to.
Really make a difference.
In treatment for these patients.
Yeah.
Thank you very much.
Thank you. Our next question comes from the line of Michael Schmidt with Guggenheim. Please proceed with your question.
Yeah. This is kelsey on for Mike well, Thanks for taking our question and I've got two quick ones and where.
Could you just provide some color on where you're at with the other ongoing car T programs. So your cart 123 and C. S. One.
And then with respect to the Tau Globin program I guess could you just remind us.
Kind of the key areas of differentiation versus existing competitors in the field and how you kind of think about that thanks. So much.
Gary do you want to give an update on one to three anti S. One I can give an update for you Sean.
That's perfect sorry, I was on mute for a second yeah. So for your cart one twenty-three for relapsed refractory AML and for the I'll start with that one first so that study as.
We said, it's been moving through dose escalation I think as you know AML is a tough is a tough space with patients who are you know quite sick and tends to be a you know little more fragile than you would see with acute lymphoblastic leukemia or with lymphoma, though it is taking a bit longer but we're moving through dose escalation and I'm we're hoping.
To have additional data at.
At some point next year, we'll see two a share which with everyone as far as Hum. The myeloma study for your cart C. S. One that too as you know we had been on clinical hold we reopened doctors changing the protocol, adding some additional safety pieces for.
Our guidance for odd treatment adverse events as well as some other rules that the FDA was requiring to ensure safety as me move forward. So we've been moving through dose escalation and that too has been moving along nicely. There's lots of interest from all of our investigators and again. This is another program that we hope to be able to show additional data later next.
Here as you know.
Earlier this year, we did present the original translational data from that first dataset before the holes, which did show some interesting translational data that included the expansion of the cells that shows all the cytokines that we expect to see and we did see some response.
So we're you know we're super excited about this program continuing to move forward and are looking forward to share data when the time's right.
Thank you Carrie on the <unk> side.
The big differentiation that we have most of the current trials that you see for tackling Tao.
The beta globin or beta thalassemia or sickle cell disease.
Is based on the knockout.
One repressor to reactivate a different type of hemoglobin fetal hemoglobin.
So you keep difficult to model in the cells.
You will destroy unaudited another gene. So there is one gene that is disrupted with mutations and theyre selling the cells to destroy another gene that is a threat rhopressa and finally reactivated gene that it's not supposed to be expressed that congress toward kind of the the the phenotype, which works actually.
Lee.
There's like a result that has been produced so far.
What people imagine on what could be the gene editing is if there is a point mutation.
Induces signaling.
Hey, Moslem Jean.
People imagine figure out other than the mutation would be fixing back the mutation, which is not happening in most of these trials.
So the approach selected is taking is.
Repairing the mutation in the hemoglobin.
Jim and fixing this mutation in restoring the adult hemoglobin and the cells.
Without leaving any cars or new cars into DNA. So you pushed back the gene to the sequence.
It was supposed to be this is an approach where not.
Of course.
There are there is competition in this field, but we believe that selective approach with the technologies, we're developing such a talent and the approach we have with our personal technology, which the genome Jim and the sizable technology brings it to a very high level.
<unk>.
Repaired adult haemoglobin select cells that are totally fixed with a favorable reduced side.
Side effects around and very high on target with.
Very accurate repair of the hemoglobin again, so thats what differentiate us for most of the approach that you see currently in the clinic and we believe that this is what patients want.
There is some argument to be fixed.
Right. Okay. Thank you so much.
Thank you. Our next question comes from the line of Kelly <unk> with Jefferies. Please proceed with your question.
Hi.
Hi, This is Dave on for Kelly. Thank you for taking the question.
Quick question on <unk>.
I loaded through is it possible that.
Are there things like CD 52.
Lynn for depletion regimen could be the reason for this and expect that the outcome and.
I mean those option is the.
For Ash data.
Can you provide some detail there to include patients who had prior CD 19 car T treatment. Thank you.
Well, thank you very much for the question well.
Yeah.
You mean.
Just to ask a precision like you mean that like the CD 52, monoclonal antibody could induce a inversion or like a chromosomal aberration somewhere.
Yes that was the question.
Oh.
Sure.
Personally I might not have all the information about this but it seems to me.
Complex, especially for a non internalizing antibodies such as like Alemtuzumab.
And to the lab to or like.
By generic fell into the map to induce such such type of.
<unk>.
Of course, some aberration that are quite classical in general it's not something that has not been observed as observed quite often.
And in.
In a lot of not only patients, but potentially the global population is not low cost that is not separable too to cross channel changes in there so.
Trim, it's part of the whole book extremely unlikely on the mechanistic side excuse me. The second part of the question I just stepped out.
The second part of the question is if you can provide any color on ash update do you, but do you have any data on patient with CD 19 car T treatment.
I can't I can answer that so sure I mean, I I D.
To make a long story short the ash abstract will include the next group of patients treated on the study all of your prior therapies, whether it was C. D Tonight, CD 19, or otherwise will be included in there are in there past medical history past prior treatment.
Dataset, so that data will be included but I'm not going to speak to today, what proportion of those patients. We do allow patients in the trial, who have had prior season 19 directed therapy and I think the you know we have already disclosed in terms of our study design for the expansion, we're going to focus on those group of patients, but for the dose escalation where.
Not limiting to only those types of patients.
Okay that helps thank you.
Sure.
Thank you. Our next question comes from the line of her Taj Singh with Oppenheimer <unk> Co. Please proceed with your question.
Oh, great. Thank you and thanks for the update.
Got a couple of quick questions. One is a follow up on the on the CD 19 project Hum.
Some of those patients got better on CD 19, directed therapies.
An antigen loss.
I don't know if that is all of the patients that relapse.
COVID-19.
Directed therapies without the subset.
And if it is a subset you know could that be a biomarker for example in dose expansion for.
And accelerated sort of like approval assuming.
You see good responses. So that's number one number two.
You know in terms of your solid tumor as would be the appealing and it's really interesting.
We're looking forward to this data among the first I believe in solid tumors with the pre clinically with call Allogeneic car T, but Andre you've spoken to this earlier, but can you talk specifically.
How our clinical development plan in phase, one and phase two could work I mean, you know the PD ones and PD L. The initial PD ones were approved in three to four years.
Therapy in various cancers.
Solid tumors seem to be no.
Can move faster.
Any thoughts there just in terms of what a development program could look like assuming you see success you know early on about the mesothelioma.
Thank you.
Thanks hurt us for the great questions and I'm going to split it into like I said the first part of the question. Your question, maybe Terry can answer this because who.
Second part of the question. She can also answer it I guess the.
Question.
Or maybe I won't and one thing that we can.
It's both but.
I can work.
Go ahead, please sir.
Yeah sure so.
The first part of the question about CD 19, So I think that's a good point, but no not everyone, who relapses and or it doesn't respond to a CD 19, directed therapy has damage and loss, but I think the numbers if I'm not mistaken at somewhere between 20 and 30 per site.
Hmm.
That said I wouldn't necessarily call that a biomarker, but I'll get to that in a second so there's not a tremendous amount of data in leukemia. Obviously after a CD 19 directed therapy and when we have the plan assuming that data and there's somebody you cart 19 data from Servier, but there's no its not as fast of a day.
Dataset as you would as we've seen for lymphoma. So it's hard to compare what we know from one data set to another but that said in terms of the development plan and we've been very open about this from the get go you know my plan for this program is to try to get it into patients as quickly as possible and have the.
Therapy that can go into a b L. A and as you know and that is exactly the group of patients that while it's a super small and niche indication. It is a place where the bar would be extremely low for the amount of data as well as the the response rate to see something and so absolutely.
That is a group of patients that we're focused on it in our clinical trial design for our expansion to focus on that group, because we know that group really need therapy. It needs. It quickly and could help us bring a product to market quick that said I'm. The reason, it's such an interesting piece is that it gives you.
Depending on how good the data is so even if it's you know it works in patients who have failed and maybe they have CD 19 loss.
<unk>.
It also may show dramatic activity, even in patients who haven't failed. So it gives us an opportunity to kind of move into multiple spaces in the in the in the treatment paradigm for these patients and I think it's super Super important.
Yes, and it's yes, and yes, but also now.
That makes sense.
Yeah.
Thanks, and just on the solid tumor question because.
Broadly what are your thoughts there and then once you get into the clinic, you know any potential for accelerated approval in various means of doing over expressing cancers.
Yeah, I mean I.
And he's a feeling program. It is a really interesting and exciting I do think however on it it really will be the first program targeting use at the island from an allogeneic setting and while yes can we have a chance for an accelerated approval its all going to depend on the data, we see and until we get into the clinic.
See what we see it's gonna be hard to make that to make that calculation, but obviously in my plans and when I look into how do we do clinical development I'm always looking for multiple different avenues to take so we can have staggered approaches and whether there is an accelerated approval path or a specific.
Hi, Neil.
But for example, highly specific cancer, where there's clear consistent over expression.
Where you would likely see a proof of concept and where there isn't a ton of options I would be looking for one option to go quickly and fast and that could be one.
I would also be looking for broader ways of getting larger indications and a bigger market share and things like that so all of that will be looked into and we would be putting together a comprehensive programs that we can find ways of doing that quickly and efficiently.
Great Alright, guys works what is what is interesting.
You know like the mobile client your body like PD one way.
Enter as far PD, one blockers enter into the solid tumors. So they can get into there. The problem is that immune cells are protected by the cancer associated fibroblast and 70% of solid tumors do not respond because of the difficulty of doing.
In system to access the tumor itself that continue to can continue to grow protected by the accounts. So the concept of blowing up the cash with the SAP car can.
Can be transformative.
This type of combos and approach to this is I think was carried describing is a very.
Y type of approach that can definitely.
And the potential that is today not really.
We cannot really have a grasp on this it could be transformative.
Great. Thank you Andre backing it up and down too.
Sure. Thanks.
Thank you. Thank you.
Our next question comes from the line of Jack Allen with Baird. Please proceed with your question.
Alright. Thank you guys so much for taking the questions.
I just had one brief one and I know I'm really avoid commenting on an allo genes specific instances here, but I was wondering if you could provide any update with regard to ongoing regulatory interactions you have with the FDA and regulators had any requests for additional product specifications or anything of that nature.
Move past this hold with algae. Thank you so much for taking the question.
Exactly thank you so much for the question.
Yes.
It's a hard question to answer fully for the simple reason, it's a lot of these interactions we have on a regular basis with the FDA are confidential and under a.
I don't want to share, particularly because it's also a competitive advantage selective fashion that's fine.
So far we haven't seen any significant change into this I guess that things are going to so there is a.
Some of these exchanges are very.
Fluid today and it doesn't change anything so I think that it's important to.
To monitor the situation on a daily basis, but it's like this is so far on a very good.
<unk> was there with our own trials.
Yeah.
Excuse me, but like it's difficult to share more than this.
I totally understand thank you so much.
Sure.
Thank you. Our next question comes from the line of Rajiv Prasad with William Blair. Please proceed with your question.
Thanks for taking the question in the Ash abstract for Bali, I said that the three patients in the FCA T O to discontinue I'm, just wondering why and as we think about the 20 by 'twenty two.
Nextgen candidate can you maybe put that in the context of your car T 22 development.
Sure I'm I'm, sorry, it's all I can I can yeah I can take this one so or abstract concludes the person the patients. We will have the other patients are additional patients in the abstract presentation at the meeting and all of that data will be in there. So I don't want to get into you know reasons for discontinuation.
It'll be in the dataset I don't think I can share that to the embargo rules since it's part of the dataset, but.
Again, just suffice it to say that we're still in dose escalation and we haven't reached our recommended phase two dose yet so.
There'll be a different reasons for why patients came off study.
And as far as 2022, I mean, I think the big difference is and why this is important as you well know the your cart 22, which was our first you know one of our first programs has there are QR eat them.
CD 20, I mind, the choke on it so it would be potentially deactivated and killed with Rituximab onboard and as you know in lymphoma.
We can't really use that as well since most patients will have we're talking to map on board.
And Rituxan that has been shown to be floating around in patient bodies for up to six months or longer and therefore, it would potentially diminish the activity of ourselves, but we wouldn't want to do that for lymphoma, So where positioning your cart 'twenty two for at a L. L for leukemia, because it's because of that and you know.
We're talking about there's a rarely used and in a L L. But 'twenty by 'twenty, two which not only is fantastic because it has the dual card. It really has an opportunity to really differentiate itself from what's out there, but therefore would not cause any issue with them we're talking about.
Yeah.
Thank you.
Our next question comes from the line of David Dye with F. N. B C. Please proceed with your question.
Hey, Thank you so much for taking my questions I have two questions first I just wanted to get some clarification on the EU cart. She tweets you ash abstract data, where you said one patient had a blast reduction consistent with Cri could you provide some additional color on this reduction is that a confirmed our cri and then a second question is that.
You also put in some really encouraging preclinical data for til globe and at Ash.
In Europe, which is a very differentiating from competitors, but could you comment on how would you see a particular data compare to the competitor programs from CRISPR and Blues and also is the I N D. You still on track for next year.
Yeah. So I can start with the first the first part of the question about your cart 'twenty two.
And now I'm trying to remember exactly what the.
Oh, sorry.
So the question is just a reduction consistent with Crs.
M C I R.
Yeah, well the thing is with with leukemia. It all depends on it's not the same as there's no such thing as confirmed or not confirmed in terms of the response criteria. So it's sort of you know.
It depends on the which response criteria you're being used whether you can counted as the actual cri or not so and you'll see in the data set when we present the data on what the patient had and what their blast reduction was it was quite impressive.
Impressive, let's say and however, there depending on which a response criteria you use them in leukemia, and there's a fear that the one is the M. D. C N, which is what we are and we were using there was also that modified chest in which was what was used for you know choosing to have and it's just a pet each.
One of them has different dependent on how long you can't measure it for it but unfortunately unlike with.
Other with like recessed for example, we're not checking the leukemia status in the bone marrow every day. So it's really hard to know exactly whether or not it meets certain criteria. So that's why we were kind of careful occasionally with our answer there, but I think it's extremely interesting and then it's extremely encouraging data in a very you know at a lower dose level and.
And our first group of patients treated with FCA, which is it is.
It's pretty amazing in my opinion.
And then I don't know Andre if you want to speak to talk with them.
Yes, the <unk> is.
Paul Rubin is currently being manufactured.
Once we're finished in manufacturing we have to go through all the requirement to.
No.
With the GMP material to start <unk>.
Bearing the R&D package. This is something where it will start probably next year once the product will be released.
This is the old done internally, so it's something that's quite exciting and.
I'm not giving any guidance for the IND filing today, but it's definitely a prolonged I will go into clinic.
Thank you. Our next question comes from the line of Nick Abbott with Wells Fargo. Please proceed with your question.
Well thank you.
First question, just going back to the ash abstract.
Data will be presented so have you cleared at a 2.5 make per kg dose if not when do you think you'll do that and that's the next step 5 million.
<unk> I am sorry 10.
Per kilo and I have a follow on thanks.
Sure I mean, I again, I I really given the embargo rules for ash I don't want to get into exactly what we're gonna be presenting on which cohorts in which datasets unless it was already in the abstract that's been released.
But they are in that.
Pointed out that the dose level, two with the 1 million and the two why is the she.
Two and a half in the next stage would be five yes.
Thanks.
And then just on the.
Absolutely linked account profile.
I know that there are no range balls on the data that's in the abstract but it shows only a 50% reduction nine days after infusion of the current bottoms out at 11.
Is that the profile you want to.
I would've expected ALC to have bottomed out pretty before infusion or at the time of infusion.
I have to pull up exactly what youre looking at because maybe it's not accurate the way of thing.
Interpret it but what I can tell you is the patients all went to zero with the length of depletion on the island She's an app. So you may be looking at the other line, which is the new cart.
Expansion.
Looking at the Blue line, which says absolute lymphocyte counts.
Oh I have to pull it up and I know in one of the pieces.
There was shown yeah I have to look at it I don't have it open in front of me, but what I do know is that.
We can get back to an email to explain it but what I can tell you is it depends on which where it was being measured from so in some cases, we're measuring the actually your cart cells and the absolute lymphocyte count so that could be what you're looking at but I'll look at it and can send you an email with a with a clarification.
Alright. Thanks.
We lost one of them, obviously is a very heavily pretreated population as you've said I think median as five in the abstract versus three at Ash last year.
So as you think about expansion.
Is this the kind of population side medium prized with car T failure and if so.
Yeah.
Is that a population do you think you can successfully complete the trial I mean, we already have a patient here who that is.
Apparently no no benefit from the car T, but it still has no lymphocytes because they had the on Tuesday map and then for depletion.
It's that patients obviously in a very challenging place yeah.
Yeah. That's a really good question I think I I think the answer though is yes.
And I think it's in our if you go to the clinical trials that Guy that has the information on our expansion cohort I believe but I think that this goes to what I said before it's really important to have a comprehensive multiple shot uncle plan for development of any product that I think particularly given our size and why.
Going to be able to bring a product to BLA and approval. This is definitely something that we can do and I think that while in leukemia, even with you've had multiple multiple therapies. You know it is a disease that typically is a younger healthier people. So it's not like acute myeloid leukemia, where most people. The vast majority of patients are over 65, when you get over.
75 for that matter and are super fail at multiple therapies, they're Oregon's don't work well generally speaking leukemia patients are younger it's the most common cancer in children. It is the most common cancer for young adults and you know we're going to be one of our arm. So to speak of development for this program is to really focus on.
These patients who are otherwise and superb shape.
Ooh, maybe they've had multiple therapies, but theyre in super good shape and can keep taking more and bring them to hopefully to a cure or somewhere where they can continue to have a long life and I think that's it's super important so I think in a L. L. A in particular, it's a little bit of a different story than when you were talking about myeloid leukaemia or some of these other diseases that you see in older people.
So I think in our expansion and where we would want to potentially have a faster market strategy would be in these younger CD 19 directed failures.
I'll need a therapy and are still otherwise.
Otherwise with the exception of their leukemia, I really well it needs to be able it needs to be treated I need something that they can move forward and potentially have a life.
So that said, though there are it's a small niche indication and therefore other strategies and other development plans are in place for us in terms of where else. We can go and how we go about doing that so we can bring it to a larger group and to your point potentially not necessarily people who failed five therapies that are also up.
Failed car T already.
But that will all terrific, okay, obviously yep.
Thank you very much.
Thank you.
Our final question comes from the line of Ingrid <unk> with Kempen. Please proceed with your question.
Hi, Hi team. Thank you for taking my question I'll keep it brief then I'm just checking.
Check with you once again and what are you know pets is scaling and you're sure shareholder ship. I think you mentioned you are planning to maintain it but would you be can't you monetize this any let's say in the short to mid term.
Hi, <unk> excuse me.
I missed the beginning of your question actually was not Super clear.
It's about one yeah sure Allison.
I just wanted to check with you what is your current thinking on K list and catalysts and now your shareholder ship.
Oh, Yeah actually currently we think Kelly just announced.
Leadership change with the arrival of micro car.
And also changing.
Change in strategy that has been induced not only by the new leadership, but also by the board of director.
Under a chairmanship.
Every day and we believe that this change is about to transform the company in a very meaningful manner and select this has a significant stake into cat Calix type thing.
We are.
The positive on the fact that this is going to bring a lot of value and you see that since then it has been quite well received a bye bye.
By the market.
As I said like select us as a desk.
Definitely has a vested interest in the success of Kelly's and waiting up to this and it's something that we believe is going to be very valuable in the future for for us as shareholders of calix.
So we have to wait up to the time this change in strategy is implemented and.
We believe the company has all the pieces.
To be extremely successful in the implementation of the strategy very rapidly and very swiftly, which we should be to our advantage.
Right and I think that's clear and if I may I have just one more question for Eric I guess looking ahead into next year, considering that you are planning to move some programs I'm Andy forward I.
Are you providing any guidance on your cash burn.
We don't we don't provide any guidance.
Okay. What we said you know the cash runway is early 2023.
On selective.
And we don't disclose you know milestone payment.
Our partnership with Xiaomi wherever we can get.
From a $10 million from 19.
<unk> so the partnership with <unk>, where we can get up to three began got off onto their own 15 Tanya.
And also with <unk> and <unk>.
I told you a series of milestones to be paid but currently the cash runway into 2023, it's unchanged.
Alright, Thank you very much.
Thank you. Thank you.
Ladies and gentlemen, we have reached the end of the question answer session I will now turn the call over to Andre totally go for closing remarks.
Well, thank you very much everyone for that.
Q&A, we're extremely excited to have all these questions and excited by our trials and our partners trials. There is a lot of things ongoing at select this I think that selected firstly.
Leaping point, a turning point for the company was internalizing older manufacturing.
And what comes next in the coming months and years as the transformative selected that is becoming a true like modified.
Got a pharmaceutical company, making things from a to Z in like one stop shop with gene editing car T oncology gene therapy and of course with a powerful store.
Strategy in the clinic as we've been sharing with carry and I think that this is going to change. So please.
<unk> closely and come to our meetings and posters at ash for waiting for you.
Thank you very much.
This concludes today's conference and you may disconnect. Your lines at this time. Thank you for your participation and have a wonderful day.