Q4 2020 Gamida Cell Ltd Earnings Call
Ladies and gentlemen, thank you for standing by and welcome to Yoga meter sales conference call for the full year financial 2020 results. My name is brandy and I will be your operator for todays call. Please be advised that this call is being recorded at gamete of sales request now I would like to introduce your.
Our host for today's conference Mr. Josh Gamer mesh Chief business Officer. Please go ahead.
Thank you Brandy and good morning, everyone. Welcome to day today's call during which we will provide an update on the company and review our financial results for the full year of 'twenty 'twenty earlier.
Earlier. This morning, we issued a press release summarizing our financial results and progress across the company, which is available on our website at www Dot commuter cell dotcom here with me on our call today are Julian Adams, Chief Executive Officer, Rohit, Sim and Togs, Chief Medical Officer and Shiloh.
And create chief financial Officer.
And Michelle core fan, our Chief operating officer, and Chief Commercial Officer, and Tracy Lowly our Chief Scientific Officer are also on hand for the Q&A portion of the call following our prepared remarks.
During this call we may make forward looking statements about our future expectations and plans, including clinical development and commercial objectives, the therapeutic potential of our product candidates, our operational plans and strategies and projected operating expenses and cash runway.
Our actual results may differ materially from what we project today due to a number of important factors, including the considerations described and the risk factors section of our form 20-F, and and other filings that give me the cell makes with the SEC from time to time.
These forward looking statements represent our views only as of today and we caution you that we may not update them and the future whether as a result of new information future events or otherwise now I'd like to turn the call over to Julian.
Thank you, Josh and thanks to everyone for joining us this morning.
And can meet a cell we are at the forefront of developing and commercializing potentially curative medicines for patients.
By harnessing our proprietary Nam cell expansion and technology.
This platform provides us with an opportunity to create therapies that could redefine standards of care for patients with life threatening diseases.
2020 was an important year for gamete of cell as we made significant progress across our entire pipeline, including Oh, Madhu B cell, which is poised to become the first FDA approved cell therapy for bone marrow transplantation.
And GDA 201, and then Nate natural killer cell or NK cell therapy.
Today, we'll review both programs and summarize our progress around plans to bring ahmedou cell to patients and the commercial setting pending FDA review.
Starting with our lead program <unk> has completed a pivotal randomized phase III study.
This global trial evaluated <unk> cell versus standard cord blood and patients with hematologic malignancies, who needed a bone marrow transplant, but did not have a suitable matched donor.
The primary endpoint of the study was timed to neutrophil and grasp and or the time it took for the white blood cells to recover following transplantation.
In 2020, we reported the Ahmedou cell phase III data, including primary and secondary endpoints, demonstrating its clinical benefit and me.
Made important advances to be launch ready upon FDA approval.
Following our December type B meeting with FDA.
We now have a very clear understanding of the ahmedou cell commercial manufacturing requirements.
We are on track to fulfill these requirements to submit the full BLA by the end of 2021 and.
And meet the commercial supply needs.
As we continue to advance them and do yourself for a potential launch and prepare to become a commercial organization. We are establishing key commercial capabilities, including the creation of gamete of cell assist a program designed to support a positive patient and transplant center experience.
With over 13000 patients with hematologic malignancies.
Eligible for transplant annually and.
And approximately 200 transplant centers in the U S. We plan to focus on patient access and support of every individual and their caregivers at each step of the process.
In addition, we have conducted extensive market research and have been encouraged by the feedback from physicians and payers, indicating the opportunity for <unk> cell to improve safety and efficacy outcomes.
Following FDA approval the market research supports that Oh, Madhu, the cell will be and important therapy for all patients in need them and the allogeneic stem cell transplant, who do not have access to and appropriate match related donor.
Oh Madhu cell also has the opportunity to increase access for patients who are not currently able to find a match.
Moving to the rest of our pipeline. We are also pleased to report significant progress for GDA 201, our first NK cell based product candidates.
Natural killer cells are innate immune cells that hold tremendous promise for treating cancer.
The challenge and the field includes the ability to expand NK cells and culture, while preserving their functionality.
And our NAND based technology and <unk>.
Dresses this challenge and potentially improves their direct tumor, killing potential and antibody dependent cellular cytotoxicity known as ADC.
GDA 201 has demonstrated impressive proof of concept and our phase one study.
Which was designed to assess the safety of GDA 201 in combination with a monoclonal antibody and <unk>.
Non hodgkin lymphoma or multiple myeloma.
The data from our ongoing phase one study has demonstrated striking early signs of efficacy with multiple durable complete responses, while being very well tolerated and patients with advanced lymphoma.
We have now developed a cryopreserved formulation and support of our vision to support.
A multi centered off the shelf <unk>.
And the generic cell therapy, with durable deep responses and a favorable safety profile.
Furthermore, we are leveraging the Nam expansion platform to develop a pipeline of gene editing NK cell product candidates with enhanced function for both hematologic malignancies and solid tumors.
Based on the impressive clinical data generated by both of them and <unk> and GDA 201, we have significantly strengthened our financial position by raising approximately $220 million in the past year.
These proceeds will support the development of both programs and provide sufficient capital.
Through the potential.
And <unk> product approval and launch by mid 2020.
I want to conclude my introductory remarks by acknowledging the challenges of the past year as we navigated through the COVID-19 pandemic.
And the resilience and dedication.
Of the gamete of cell team as we work to bring <unk> to patients.
I'll now turn the call over to <unk> <unk>, our Chief Medical officer to provide further details on <unk> and GDA 201.
Right.
Thank you Julian and good morning, everyone.
This morning, I'll review, our clinical program and highlight the data we presented throughout 2020 and both of them and do the cell and GDA 201.
I'll start with the phase III study of I'm going to give yourself.
And May 2020, we were pleased to report that the study met its primary endpoint times and you're just fill and glass net.
In October we reported the phase III study also met all three prespecified secondary endpoint, platelet and Grafman infections and hospitalization.
These positive study outcomes as well as additional exploratory endpoints will presented in a peer reviewed setting for the first time at the TCT meeting a few weeks ago.
And there will be an encore presentation at the presidential session of the European bone marrow transplant annual meeting next week.
The trial was well conducted and rigorously designed study.
And alive and and intent to treat basis.
125 patients were randomized two to do the cell arm and 60 suite the standard cord blood.
And the graphics and baseline disease characteristics were well balanced and.
And reflected a diverse population of patients with hematologic malignancies in need and allogeneic bone marrow transplant.
Clinical outcomes supported the superior neutrophil and graph and demonstrated in the primary endpoint analysis.
Typically patients randomized to Oh, Madhu, Michelle had fewer serious bacterial fungal and viral infections and the comparator group.
This has also been significantly less time and the hospital and the first 100 days after transplant.
Importantly, and <unk> was generally well tolerated and in particular, the incidents of both acute and chronic graft versus host disease, which have been shown to be lower and patients transplanted with cord blood than and other modalities, what statistically similar and the two arms.
While the study was not designed to detect a difference and we lap one mortality endpoint. We reported the results of these analyses, including non relapse or transplant related mortality, which was 11% for patients randomized to <unk> cell and 24% per patients randomized to Paragon and.
And overall survival, which was 73% and the ahmedou cell arm and 52% per control.
The data we presented in 2020 continue to strengthen our confidence and the clinical potential on the day the cell.
I want to thank the investigators patients and caregivers that participated in our study we are grateful for their support as we move and the field forward.
With these data in hand, we anticipate submitting the BLA for him and DUBA cell in the fourth quarter of this year.
We believe that I'm gonna do B cell also has potential to treat patients with diseases beyond hematologic malignancy and are currently investigating and would you be felt and patients with severe aplastic anemia.
Life, threatening blood disorder, and and investigator sponsored phase one and two study being led by Dr. Richard child at the National Institutes of health.
At the recent American Society of Hematology Conference, we presented data from a total of eight patients.
And wanted to one stem cell transplant with Oh, Madhu itself plus high flow identical stem cell and five patients who received omidria the cell as a standalone graft.
The data showed that transplantation with Oh, Madhu cell filing and reduced intensity conditioning was generally well tolerated and led to a rapid and graph net.
With a median follow up of 10 months seven of the eight patients had early and sustained and Brac net and we're no longer dependent on transfusions.
Neutrophil and platelet recovery occurred at a median of 10 days and 31 days respectively.
We are encouraged by these data in patients who are at high risk of graft failure with conventional quite blood transplant and the study is still ongoing.
As Julian mentioned in addition to AUM and do the cell we are advancing our first and K cell therapy GDA 201.
We have demonstrated impressive proof of concept and our phase one study, which is designed to assess the safety of GDA 201 in combination with a monoclonal antibody and patients with non hodgkin lymphoma or multiple myeloma.
And the studies being conducted by Dr. Veronica back and Nova and the University of Minnesota.
At Ash, we presented safety data from 35 patients with relapsed or refractory disease 19 patients with non Hodgkin lymphoma, and 16 with myeloma.
GDA 201 was generally well tolerated with no dose limiting toxicity, no gvhd and importantly, no neurotoxicity observed.
We were also very impressed with the promising clinical activity and all doses evaluated and patients with lymphoma.
These patients were heavily pretreated with a median of three prior lines of chemotherapy.
Of the 19 patients with lymphoma, 13 had complete responses and one had a partial response from overall response rate of 74% and a complete response rate of 68%.
Responses were observed in patients with both Follicular and diffuse large b cell histology.
We're developing a cryopreserved formulation and will be submitting our IND and the second half of this year. This will allow us to conduct a multicenter multi dose study to explore the potential of GDA 201, as an off the shelf cell therapy and patients with lymphoma.
And very proud of our team and their dedication to advancing our clinical study during the global pandemic.
I'll now turn the call over to Shai to review our financial results.
Thank you Renee and good morning, everyone today, I will summarize our financial results for the full year 2020.
And as of December 31, 2020, we had total cash and cash equivalents of $127 $2 million compared to $55 $4 million as of December 31st 2019.
And as Julian mentioned during 2020 and were able to significantly and and sorry capital position, which raising total of $144 million and gross proceeds from our May and December public offerings.
In addition, and February 2021, we announced a 75 million and donuts financing with <unk> capital management.
Those financing transaction extends our cash runway beyond the potential BLA approved BLA approval for me to be cell expected by mid 2022.
Research and development expenses for the year.
41, one.
And $1 million compared to 31 point fighting and dogs from 2019.
The increase was mainly due to advancing day, GDA 201 clinical programs and clinical activities related to concluding our phase III trial as well as additional head count we think the R&D organization.
Commercial expenses from 2020 were $8 $7 million compared to $4 $7 million last year.
The increase was mainly attributed to Ami do B cell launch readiness activities, which include the addition to our.
Two our commercial leadership team.
General and administrative expenses were $12 2 million daus for 2020 compared to 12.1 and even the last for 2019. The increase was mainly attributed to $1 2 million dollar increase and professional services expenses, including legal and insurance.
And by a decrease of $1 $2 million and travel and noncash compensation expenses.
Finance expenses net were $10 $4 million for the year.
Compared to finance income net of $13 $8 million in 2019.
The increased expenses are primarily due to non cash expenses, resulting from revaluation of warrants owned by our shareholders and the revaluation of the Israeli innovation authority royalty bearing Guan <unk>.
Net loss per the year was $72 $7 million compared to a net loss of $34 $4 million last year.
We expect.
Cash used for ongoing operating activities. This year will range from $100 million to $120 million.
Anticipate our current total cash position will support our ongoing operating activities into the second half of next year.
This cash runway guidance based on our current operational plan and exclude any additional funding to be received or business development activities that may be undertaken.
I will turn the call back over to Julien.
Thanks Shai.
We are dedicated to finding cures for patients with hematologic malignancies and blood disorders.
And we are excited about the opportunities ahead.
<unk> cell, we expect to submit the BLA and the fourth quarter of this year and are committed to being launch ready at the time of approval.
With GDA 201, we have very compelling data and lymphoma and are planning to initiate the gamete of cell sponsored clinical study, which could transition into a registrational trial. If the data are consistent with our phase one results. So as you can see we expect 2021 to be a transformational year.
<unk> forgive me to cell and we look forward to updating you on our progress throughout the year.
And now we will open the call for questions Randy.
At this time, if you would like to ask a question. Please press Star then the number one on your telephone keypad again net of stars and the number one.
Your first question comes from the line of Ted <unk> with Piper Sandler.
Great. Thank you very much and congrats on the other progress Julian maybe you can just walk us through sort of what's being done both in terms of preparing for the BLA submission, but also concurrently with respect to commercial buildup.
And buildup as you prepare for Omidria vishal. Thank you.
Okay, Let me begin with.
The modules that are being prepared for the BLA submission.
One clinical module is complete and ready.
And.
The clinical module will be completed in the near term.
And lastly, the CMC module will be completed by the fourth quarter, all of which will be unnecessary for from.
For BLA submission.
Let me turn it over to Michele to talk about our commercial preparations.
Excellent. Thank you Julian and good morning, Ted and Reed.
Guards to commercial there there's really been three key aspects. We're focused on that the first is as leaders and shai alluded to this in his prepared remarks, we brought in some outstanding leaders on the commercial team Rocio Megami, leading market access with great experience and hematology and cell therapy, Linda Stamler was recently brought onto lead Mark.
Getting and account management and they join a couple of our other already established leaders. So we have a great leadership team and place. The next key area was to finalize the commercial strategy. Upon FDA approval will take great market insights that support the fact that upon FDA approval ahmedou cell will be a very important there.
European option for patients, who need and allogeneic stem cell transplant, who do not have access to and appropriate matched related toner, it's a very exciting market opportunity for them and do the cell and we are very clear in terms of what now needs to be done to execute on that strategy and that's really the third part. So we've got our launch readiness plans.
Underway really that all encompassing thought around making sure everything we're doing is leading to a positive experience for the patients and for the transplant centers. We're focused on four key aspects and making sure we educate the transplant centers, making sure that we educate the payers and the third is making sure we have that strong support system.
And through can meet a cell assist and finally, our commercial manufacturing readiness.
Julian alluded to this in his prepared remarks, where we're moving along very nicely with our new facility in Israel and care God. We're also partnering with Alonso for their readiness for the BLA. So we're very much looking forward to the opportunity to introduce ahmedou cell to patients upon FDA approval.
Okay, and so do you think you relative to you. Thank you Ted.
Your next question comes from the line of Jonathan Miller with Evercore ISI.
Hi, guys. Thanks for taking my question and I'll, just follow up a little bit on that commercial ramp question, obviously with the BLA and <unk> got some time to prep and I understand you've got a strategy in place now how do you think about spend ramping throughout the year as you hire folks and how biggest sales team will you need.
To achieve your commercial plans and then secondly, I'd love to ask about the GDA 201, one of the interesting factors.
About this product is naturalistic ex branch of the NK cells with NIM, but now you're introducing additional.
And later in the pipeline you are introducing additional engineering.
And gene editing to these products, how do you think about balancing adding that functionality Walter and maintain a healthy cell profile and maintain their biological per pilots.
Product sales.
So from Michelle please.
Address the first question and thank you Jonathan.
And I'll address the P and K questions.
Yeah, absolutely. Thank you Jonathan good morning.
So in regards to the ramp up and personnel obviously the personnel in the field will be key to our success.
The good news for US is this is a very efficient launch from the standpoint of personnel and and also truly spending and so when you look at the target. So let's start with the transplant centers. There's roughly 200 fact accredited transplant centers and the U S.
No. Many of these centers quite well Rohit and her team had direct experience with at least 20 during the clinical trials and then we certainly know more centers. So and we also know that 70 centers make up about 80% of the transplants in the U S. So we anticipate a very targeted and efficient launch in terms of field.
Force in terms of actual numbers. So our field personnel will fall into three main categories with obviously supporting groups. The first will be on the commercial side.
GAAP management team. If you look at benchmarks of other cell therapy launches and the U S, where you're probably looking at 20 to 25 personnel that would be needed to effectively launch the therapy and have that positive patient experience will also have individuals focused on that the payer space led by Rocio Mangano and that's also relative to.
Small footprint because the U S. Private payers are relatively consolidated so probably about 10 personnel. There and then we will have an outstanding group led by Rone eats medical affairs colleagues that or our medical science liaisons, who are supporting the educational initiatives with both the transplant centers and the payers. So we have.
Clear path going forward and you know again based on our experience and cell therapy. It will be a very efficient launch from a head count and spending standpoint.
Julian and I will turn it back to you John.
Turning to the NK cell. So our first product candidate GDA 201 is a non engineered.
Expanded.
Cell.
Therapy in combination with Rituximab for lymphoma, and let me invite Tracey to talk about.
Our plans for engineering, the NK cells, particularly as we consider addressing solid tumors.
Sure. Thanks, Thank you Julian and thank you for that question Jonathan.
And with regards to your first part and we're still as Julian said expanding up our NK cells with nicotinamide and because of this we have unique potential and our NK cells to maintain receptors that are on the cell surface such as T.
And the 62 L, which we believe uniquely causes ourselves to home into heme malignancies, and lymphoid, Oregon, and as well as a very important and CD 16 receptor for ADC, So additional editing effort.
And we'll maintain those properties and and and function of the sales that we see with Nam, but will further enhance what we think will allow ourselves to survive and suppressive tumor microenvironment by editing off.
Some negative receptors and negative co stimulatory receptors, just can make the cells potentially more active and persistent.
In the tumor microenvironment as we expand upon this research effort and the lab this year to make these engineered products.
Towards the end of it that makes sense.
Does that.
And we adequately answered your question Jonathan.
Well I look forward to seeing how that program and develops and exactly how their sales behavior.
Yes for now I think that yes.
And we havent named specific targets yet.
We will do so at an appropriate time.
And the future, but right now the the researches ongoing and Tracy supervision.
And we're very encouraged by what we're seeing.
And your next question comes from the line of Jason Butler JMP Securities.
Hi, it's Roy on for Jason and Thanks for taking our questions I'm, sorry, if I missed these and the prepared remarks, but I'm not.
Everything at the BMT meeting next week that we can expect that that's different from the TCT meeting and then for Omar <unk> cell I'm. Just wondering if you guys can disclose how many patients have been treated and the expanded access and I had a follow up on 201.
Roni.
This is for you.
Thanks, and so.
With respect to the E. BMT meeting we're excited at the opportunity.
And that the data will be presented and a European forum and its being highlighted in a high profile search and the presidential session. There will also be a.
Panel session in which the investigators the European P I, Dr. Guillermo sand.
Who will be.
Participating in the Q&A session lives later on that same day and in terms of the data themselves.
Basically the same data set that was presented at the TCT meeting.
But of course, the with and.
Some added nuance from Dr. Stan.
And his and his remarks and his panel discussion.
In terms of a standard assets we haven't.
And then.
Updating on the number of patients or the progress at this point. The study is open at six sites across the U S.
It's opening and a variety of sites and there is enthusiasm from investigators to enroll patients.
And the future, we do hope to provide some detail about the patients and their outcomes, but we havent been giving updates on an ongoing basis.
Okay fair enough, so and I've heard.
201.
Just what remains gating for the IND filing and can you remind us if the cryopreserved formulation has been finalized and.
And is the FDA going to sign off on the formulation prior to the formal full R&D filing and how does that work.
Tracy.
Would you address that please.
Sure sure. Thank you Julien.
And in terms of what what's left before we file the IND.
It really is CMC.
Section four.
GDA 201, and finalizing their cryo formulation. The cryo formulation has been finalized and the lab. It's now just a matter of <unk>.
And at scale for clinical scale, it's now just a matter of manufacturing.
And also of that before or clinical batches for stability and our GMP facility, which will be in Israel.
Four we're able to file the IND.
And yet presumably we're in active discussions with the FDA. So both the protocol and of and the final formulation of the off the shelf allo product that's cryo preserved will.
We will be discussed and agreed upon with them.
Great. Thank you.
Your next question comes from the line of Gregory <unk> with RBC capital markets.
Hey, Julien team congratulations on all the progress and thanks for taking my question Julian just in regards to the modules I just wanted to confirm and Boyd you mentioned that the clinical module will be.
Completed and submitted shortly as well as the manufacturing.
By end of year, just curious if you could maybe characterize or quantify just the level of analytical and clinical comparability that that is necessary and from a day to sell.
With the extension not disclosing the patients now, but just curious if there is a dataset.
Patient number that the FDA would like to see.
For their comfort there and then just just just in terms of the preparations great to hear all the all the color from between you and Michelle I'm. Just curious what has that the extended time line from Rolling forecast 2020 start being Rachel and this year and what does that afford and do as far as that preparation and getting that that launch.
And maybe just on the final question there.
From Michele really good where are the gaps or the skeptical areas that are most likely to get it and the way of realizing the amezquita cell vision ultimately thank you very much.
Yes, So let me just start at a high level and turn it to <unk> to talk about the clinical module.
And and the CMC requirements.
We had a very detailed conversation with FDA and December.
And we have very clear understanding of what they're looking for in.
In terms of CMC.
Both analytical comparability as well as clinical comparability from our commercial and manufacturing sites.
Really do you have anything more to add.
Yeah, I think I think we shared data with the FDA about our clinical results and we've told them.
Or share with them our plan in terms of the new commercial facilities.
We believe that.
Submitting a high level data on three to five patients will be sufficient.
For establishing and.
And the clinical results are adequate to support our production at those sites. So that that's where we're that's where we've landed in terms of and your production.
And Michele.
The second part of the question to you yeah.
Absolutely and and thank you and and interestingly there was a two part question there they're actually both addressed and the same way so what the extra time allowed us for commercial readiness and any concerns around potential gaps you know what we were able to do with the additional few bonds was identified where where there are potential challenges and.
How would we be able to overcome them. So now we have that ability to address them proactively.
Really came down to three key areas and one was hiring and so as I mentioned, we have been able to take the opportunity to hire some great individuals with commercial experience both on the commercial side and then on the operations side to the the two specific areas that we've also been able to proactively focus on just addressing.
<unk> would be around manufacturing for commercial readiness and our actual commercial launch so manufacturing for commercial readiness, we're making very very nice progress at our facility and incur a got you know just not only in terms of the FDA requirements for the BLA, but also for overall commercial supply.
And us upon FDA approval. So that is moving along nicely to plan and then the second area around commercial readiness. This gave us some additional time to understand the needs of the transplant centers upon potential approval of ahmedou, but cell and nuts and partnership with <unk> Medical Affairs team. We also have the.
Attunity to also partner with Rooney each team to focus on the education of the commercial payers.
And really making sure that they understand the great unmet need from a day per cell and understand the value proposition of armour DUBA cell. Both in terms of the clinical data and the health economic side. So we're excited about the progress on both the manufacturing and commercial side for launch readiness.
So Julian I'll turn it back to you.
Yeah.
And I.
Hopefully adequately answered your question any further follow up on your and that debt.
That's great. Thank you very much I appreciate the color.
<unk>.
Your next question comes from the line of Mark Breidenbach with Oppenheimer.
Hey, guys.
Guys. Thanks for taking the questions.
This is related to some of the others that have already come up but Julien I was wondering if you could give us a little more color on why the CMC module is the rate limiting step and the BLA application for BLA filing.
And.
Have you reached an agreement with the FDA regarding.
Sort of.
Criteria yet.
Yeah, I mean, it's not uncommon that CMC is usually the rate limiting.
Step four.
Any product, but in particular and cell therapy.
And where there's a lot more attention.
And just different criteria too.
To be adequate.
We're supplying.
Commercial.
Marketplace.
This is not unusual.
Michelle you can probably reflect on your experience at kite when you were launching just Carter.
Thank you Julien so that the we received very clear feedback from FDA at the December type B meeting and in terms of the requirements we needed to focus on for the CMC module and just just one point to recall is we manufacture our phase three and <unk> cell at a different.
And launch a facility and we knew this wasn't a surprise for US we knew we were going to be transition and commercial supply to launch another Lynch facility and also to cure and got so what the F. D. A had had discussed with us and December and what we're focused on now is the comparability requirements that are needed to for the commercial for.
<unk> as compared to the the phase three so these are analytical comparability the clinical data that we discussed some of the methods validation work all of that is mapped out and on track.
Our cure got facility at this point and time lines, the Netherlands had had already begun some of the work on that so.
The Big takeaway is FDA was very clear in December and where were now moving along nicely and partnership with our research colleagues and also our clinical colleagues to assure not only readiness or the BLA, but this also gives us the opportunity to assure readiness for the commercial supply from the manufacturing standpoint too.
Okay. That's super helpful and just a quick follow up on GDA 201, given that there are some key differences in.
And the types of donors that you plan to use for your trial and the fact that you are using cryopreservation versus fresh product that we've seen in the academic sponsored study.
Wondering if you have plans to present any.
Preclinical.
Either in vitro cell, killing assays or mouse data.
Demonstrating equivalents between these two versions of GDA 201 later this year. Thanks.
Thanks for your question.
Tracy I'll turn that over to you.
And as well yeah sure Dan and thank you, yes, and yes.
You point out.
The for our ion E filing GDA 201 will now be a completely allo off the shelf and cryo preserved product. It it's still from the same source from normal doughnut, a police as material, but just as a reminder, the clinic.
Clinical study that was done with collaboration with Minnesota.
What is from a related donor. So the majority of those were half blow down ex whereas this will be completely allo.
We do have plans.
To present, some mechanistic work that we continue to do understanding the furthest phenotype of our Nam expanded NK cell over this new manufacturing protocol, which is cryo preserved.
And with with our sites and presenting some of this work towards the end of this year and at a conference.
And maybe likely ash, but maybe another research conference and at that time, we can present and to work on both pre clinically and the comparability between the fresh and frozen that that work is ongoing and the and the lab and Israel and we have done quite extensive studies to show that we maintain both the function.
And and the phenotype with.
And with the expanded Nam NK cells, both at harvest and and both after thought of the cryo preserved formulation. So that when we will obviously presented data and.
And the IND filing as well.
Got it that would be Super Super helpful. Thanks for taking the questions.
Okay.
Your next question comes from the line of Gil Blum with Needham and company.
Hello, everyone and thank you for taking our questions.
So just a quick one we saw something of a trend with the overall survival and.
And the.
Pivotal study any thoughts and conducting an outcome study maybe post approval or.
Do you have any plans in that direction.
So indeed, we saw a strong trend for us.
Overall survival.
And would remind you that we were not powered for overall survival.
And let me invite Rooney to further comment on the follow up of our patients.
Absolutely, yes, so thank you.
In terms of following additional information on patients we will have additional follow up for the patients and our phase III study and overtime.
And and as well as patients who enroll and our expanded access study, which is basically a single arm.
Study of Oh, Madhu the cell.
And post approval, we don't have any.
Clear plans yet at this point on the type of follow up that will do for our patients and the real world, but all patients who are transplanted and the U S.
<unk> followed in the registry by CIB MTR, so there may be an opportunity to leverage that and.
And the post marketing setting.
Alright excellent and.
And maybe it kind of in and out there question. So so we share a lot of different benefits for the now platform and.
Any thoughts about using this for for <unk>.
Gene therapy purposes, and it allows the expansion of.
And amount of Florida stem cells, and now Youre looking at engineered amount of poetic stem cell. So the leap isn't.
And really large there.
It's a very good point and that is not lost on us.
And we are exploring quite a number of avenues.
And the laboratory setting.
And we have nothing to report at this time, but.
But stay tuned because we think this platform.
And it will be the gift that keeps on giving.
As we.
Entertain.
Other expansion.
And of <unk>.
Different cell types.
And again, we think this is a universal.
Expansion platform and.
Are doing a lot of preclinical work to us.
Understand.
Greater specificity the mechanism of action.
And how this can be applied across the board.
Excellent. Thank you for taking our questions and congratulations on all the progress.
Thank you.
There are no further questions at this time and I would.
Now I'd like to turn the call back over to Julien for any closing remarks.
Thank you everyone for joining us on today's call and.
And we look forward to and exciting 2021 and.
And future engagements with you all and thank you.
This concludes today's conference call you may now disconnect.
Okay.
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