Q1 2023 Editas Medicine Inc. Earnings Call
[music].
Good morning, and welcome to Eddie tough Medicine first quarter conference call.
All participants are now in a listen only mode.
There will be a question and answer session at the end of this call.
Please be advised that this call is being recorded at the company's request.
I would now like to turn the call over to Kristy Barnett corporate communications and Investor Relations.
And any tough medicine.
Thank you.
Good morning, everyone and welcome to our first quarter 2023.
Earlier. This morning, we issued a press release, providing our financial results.
<unk> corporate update.
A replay of today's call will be available at the investors section of our website approximately two hours.
Yeah.
After our prepared remarks, we will open the call for Q&A.
As a reminder, various remarks, we make during this call about the company's future expectations plans and prospects.
Such forward looking statements.
The safe Harbor provisions under the private Securities Litigation Reform Act of 1995.
Actual results may differ materially from those indicated.
These forward looking statements as a result of various important factors, including those discussed in the risk factors section of our most recent annual report on Form 10-K, which is on file with the SEC as updated by our subsequent flat in.
In addition, any forward looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date.
Except as required by law, we specifically disclaim any obligation to update or revise any forward looking statements, even if our views change.
Now I will turn the call over to our CEO Gilmore O'neill.
Thank you Christy and good morning, everyone. Thank you for joining us today on Eddie passes first quarter earnings call.
I'm joined today by two other members of the edits as executive team based on my our Chief Medical Officer, and Michelle Robertson, Our Chief Financial Officer as many of you know in early January we shared our strategy positions Eddie task as a leader in in vivo program with the gene editing and Haemoglobinopathy.
During the first quarter, we successfully executed this strategy driving to our goal of delivering life changing medicines to patients with previously untreatable or undertreated diseases, we are increasing our momentum and driving our ex vivo edit 201 program as we pursue a leadership position in hematopoietic stem cell medicines for hemoglobin.
Let's see.
As a quick recap there are three underlying pinterest to our new strategy first.
<unk> developed edit 301 for severe sickle cell disease, and transfusion dependent beta thalassemia or T. D. T. We have sharpened our discovery focused to in vivo administered genome editing medicines.
As part of that refocusing efforts, we previously announced that we had divested our eye NK cell franchise to shoreline Biosciences in January .
Second we are strengthening our discovery engine and technological capabilities, we have divided our research division into separate technology and drug discovery groups enhancing the capabilities of each and implementing our new target selection criteria.
Finally, our third strategic pillar is an increase an expanded approach to business development in tandem we will continue to deleverage our IP portfolio to drive out licensing and partnership discussions.
So how have we executed against our new strategy in the first quarter, we have increased our investment in our edit 301 program. After reviewing promising initial Ruby phase one two study data that indicated that we have a competitive and potentially differentiated program to treat sickle cell anemia and TDP. Additionally, we are investing.
To develop an in vivo approach for editing hematopoietic stem cells for the treatment of sickle cell disease, and TVT leveraging the unique and differentiated approach of edit 301.
That we have already seen a POC.
POC for insurance, we continue to ramp up enrollment and dosing of patients in the rupee trial for sickle cell disease and are on track to have dose 20 total patients by the end of 2023.
We are also excited to share that the FDA recently granted orphan drug designation to edit three one for the treatment of sickle cell disease, and we are pleased to announce that in June we will provide a ruby clinical data update in an oral presentation at the European Hematology Association or <unk> Congress and in our company sponsored webinar.
Based on with share further details regarding our June data readout, Andrew enrollment progress in his remarks.
On edit 301 for GTT, we are pleased to share that we dosed the first patient in our edits all phase one two trial in the first quarter and that the patient has successfully engrafted neutrophils and platelets.
Enrollment continues to progress and we remain on track to provide initial clinical data from the NFL trial by year end.
Moving to in vivo earlier this year, our drug discovery group began lead discovery work on in vivo therapeutic targets and Hfcs are hematopoietic stem cells and other tissues. As a reminder, under our new target selection criteria, we will select therapeutic targets that will allow our genome editing approach to differentiate maximally from the current standard of care for Sirius.
Diseases the.
The target selection criteria, we will work to ensure targets are selected that maximize the probability of technical regulatory and commercial success.
Our search for a new CSO to lead this drug discovery group continues to progress and I look forward to updating you on this search and our in vivo work in the future.
Turning to our intellectual property position since the founding of edit tests, we have placed substantial importance in securing robust intellectual property protections covering our cutting edge scientific discoveries and gene editing advancements to enable the development of novel transformative medicines for patients in need.
It is important to note that we have a large portfolio of foundational U S and international patents covering CRISPR Cas nine in human therapeutics, only some of which are subject to interference proceedings and we are confident that our IP portfolio will provide meaningful value in the future.
We are the exclusive licensee of Harvard University's and broad institutes cast nine patent to states and Eddie test is uniquely positioned to issue exclusive and nonexclusive licenses forecast nine to any company seeking to use these enzymes to make human medicines, including an in vivo and ex vivo therapeutic applications.
Our unique position as the exclusive licensee of this happened to states ensures that we are the party responsible for any licensing discussions as CRISPR Cas nine products enter the market, which given the size of the U S patient monitor the number of companies buying to develop CRISPR Cas nine medicines is a substantial position.
With our newly sharpened our strategic focus are world class scientists unemployed and our keen attention to execution. We continue to build upon the momentum from our clinical readouts milestones during the fourth quarter of 2022, we look forward to updating you on our progress and on the execution of our new strategy throughout the year now I will turn the call.
Over to pay song, our Chief Medical Officer, Lisa Thank you Jim.
Good morning, everyone.
Let's start with edit 301 study for severe sickle cell disease.
As Guillermo mentioned, we continue to enroll and dose patients in the <unk> study.
We have activated 20 study sites.
In all 19 patients almost doubling the number of patients enrolled from three months ago.
As we previously shared with you can parallel dosing a patient school year this year.
We're on track to provide an update on the Ruby clinical data post next month and the year end.
That's why I had to dose 22 locations by year end.
Turning to clinical data.
I'm excited that we will present, the Ruby clinical data as an oral presentation at the European Oncology Association or <unk> How Congress.
And at our company sponsored webinar in June .
The deaths that well, including safety and efficacy data from multiple patients equally.
Equally 10 months data from the first patient treated and six months data from the second patient treated equally.
Including total hemoglobin fetal hemoglobin.
We also shared data on safety neutrophil battling bachmann and basketball Qsymia depends on V O E filing the first small patient.
As a reminder, last December we presented initial data from the first two patients.
We will be fine.
The first patient who had five months of follow up after treatment with <unk>. One showed a clinically significant improvements across all hematological parameters and then nobody oes.
Specifically the patient had an increase of fetal hemoglobin fraction to 45, 4% five months after entry weren't infusion.
And the correction of anemia, with total hemoglobin level well into the normal range at $16 four grams per deciliter.
Yeah.
These initial clinical data indicated that edit 301 provides patients with high and sustained level of fetal hemoglobin and normal level of total hemoglobin.
These clinical observation is consistent with preclinical data, which has demonstrated that targeting chemical will be promoter enables increases of fetal hemoglobin independent of Erith stroke rehab Express.
Given the unique gene editing approach and in magna's back action by at least 301.
Courted by preclinical data and initial clinical data. We continue to believe that edit 301 can potentially provide robust clinical benefit to patients with severe sequels, ftes and potentially provide clinical differentiation in the long term.
As a reminder.
Stan normal total hemoglobin level is an important clinical outcome for patients.
As the correction of anemia can significantly improve quality of life and ameliorate and organ damage.
We believe <unk> normal level off told them, we wont be could be a potential point of differentiation for us for a while.
Turning to Eddie Eddie sale Phase one two trial for <unk>.
Transfusion dependent anemia.
As Guillermo mentioned earlier, we've dosed, our first patient in Q1, and the patient has successful neutrophil and platelet and grabbing it.
We remain on track to provide initial clinical data from the <unk> trial by year end.
As we have done for the rupee study. We're also taking multiple measures to accelerate the development of Eddy Street 140, P T and have strong market momentum.
We haven't enrolled multiple patients who have completed a free space and have their CD 34 positive cells edited.
All are in the process up phases.
Recently I've been traveling around the country visiting our movie and Alistair how clinical trial sites I.
I very much appreciate the enthusiasm and the support from the investigators and study sites.
I am pleased with the momentum of 83 to one.
Asian recruitment freezes editing and dosing in both studies.
I'm excited to hear from investigators that patient dosing with 81 have already seen costume changes in their lives.
We look forward to sharing additional updates as the year progresses.
Adding movie study data next month and at year end and sharing initial clinical data from anything else study by year end.
Now I'll turn the call over to Michel our Chief Financial Officer to review our financials.
Thank you Beth and good morning, everyone.
Like to refer you to our press release issued earlier today for a summary of our premiums result, the first quarter of 2020.
Take this opportunity to briefly review a few items.
Our cash cash equivalents and marketable securities as of March 31 were 402 million compared to 437 million as of December 31, 2022.
We expect our existing cash cash equivalence and marketable securities to fund, our operating expenses and capital expenditures into 2025.
Revenue for the first quarter of 2003 with $9 99.
Three 8 million.
Yes.
The increase is related to the previously announced sale of our oncology at the shoreline buyer frequency.
Related license, which was completed in January 2023.
G&A expenses for the quarter was one 3 billion compared with $19 5 million for the first quarter 2022.
$3 5 million increase is primarily attributable to increased professional services.
Support business development.
Partially offset by a decrease in stock.
Yeah.
R&D expenses this quarter were 38 million, which is flat compared to the first quarter of 2022. This reflects a decrease in expenses following the strategic prioritization of our portfolio.
Sat Fi increased investment to accelerate the development of edit 301.
Reallocation of capital in line with our strategic priority.
Overall, it is concentrated in a strong financial position and our shopping discovery allowed us to concentrate our count and extend our cash runway into 2025, which provide ample resources for our continued progress in both.
301 trial as well as advancing our research efforts.
Uh huh.
Other vivo discovery.
With that I will hand, the call back to Joe Moore.
Thank you Michelle.
It has been almost one year since I joined edit has in this time the company has demonstrated to clinical proof of concept.
Including a proof of concept for edit 301, which has the potential to be a competitive and differentiated product for the treatment of sickle cell disease and transfusion dependent beta thalassemia. In addition, as I stated in my opening remarks, we've taken a number of tangible steps to reshape the company around our new strategy, which we shared in early January and have begun executing on that strategy.
And this is just the beginning.
We look forward to continuing our transformation on sharing our progress with you as a reminder, our strategic objectives for the year include providing clinical updates from the edit three one Ruby study in June and end of 2023, providing clinical data from edits. We went edits Hal trial for TD T. By the end of 2023 dosing 20 total patients in our EDA.
Three on Ruby study about a year and hiring a new CSO with specific expertise aligned to our vision of advancing discovery of in vivo editing of hematopoietic stem cells and other tissues, and finally, leveraging our robust IP portfolio and business development activities to drive value and complement or gene editing technology capabilities.
Thank all the patients investigators and our employees are helping to drive our strategy for it. Thank you very much for your interest that it has and we're happy to answer questions. Thank you.
Thank you.
At this time, we'll be conducting a question and answer session.
I would like to ask a question. Please press star one on your telephone keypad.
A confirmation tone will indicate that your line is in the question queue.
You May press Star two if you would like to remove your question from the queue.
For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys one.
One moment, please while we poll for questions.
Thank you.
And our first question comes from Joon Lee with Troy Securities. Please proceed with your question.
Alright, thanks for taking our questions.
No Novartis recently terminated their sickle cell disease program after taking a couple of patients who said more benefits.
Given you're editing strategy is similar to what Novartis.
Just some differences why your promoter income strategies to continue to work with.
Novartis fail and I have a policy.
Here, we actually saw a very robust data with our increase in total hemoglobin are already increasing total hemoglobin as well as a robust fetal hemoglobin expression completely consistent with the preclinical data.
That were generated.
In testing our preclinical in our clinical hypotheses that our unique approach of combining an S cast 12, a effector CRISPR enzyme with at the targeting of a different region.
Of the H P. G want to promote her which was much closer in fact actually encompasses the area in which we see deletions are mutations association with hereditary persistence of fetal hemoglobin and I think we believe that all of those factors.
Points towards a key difference and differentiation and indeed, our preclinical data and our clinical data have actually support that hypothesis.
Great.
Can you remind me if you have any data quickly.
Having.
Comparing the entity.
Thank you, Jim you've been cast nine versus cats, as well that they're using.
What what the differences maybe.
You're starting to you and I'm seeing this amount of yeah. So I mean, comparing cats nine this is what I think.
Novartis and <unk>.
And cast 12 days, which is what you're using you get a different outcome.
Part of the same region in terms of getting deletions or emails.
Yeah.
You added bit breakdown, but let me try this is the base I'll, let me try to see whether I understand correctly.
And just echoing noise. So I think your question to say compared to Novartis and do we have a comparison, beating F&I and K 12.
And Duke and also the region just a kill more mentioned is that your question.
Yeah.
Target the same region, either with cash or.
Our cash 12 Amy.
What's the differences in outcome.
Yeah, Let me let me let me, let me just kind of in it too.
We did as a comparison we did.
Clinical perspective, we scanned a larger region.
Promoter of agent to identify wish everybody had to do the editing.
So without too many specifics by we recover the larger region the promoter.
S B G when attitude and we find out of that.
Wanted to the region, we still al-qaeda with Shaquille more just mention is consistent to the clean call great observation fault.
The U S B M H.
And then we also compare the cat nine we just catch toile and we found that the difference between Kathline catwalk.
That answer your question.
Yeah no.
Absolutely here you're in parakeet asphalt.
I was just curious what was driving that correctly. Thank you so much I'll hop back on.
Thank you.
Yeah.
Yeah.
Thank you. Our next question is from Samantha Simon Cowell with Citi. Please proceed with your question.
Hi, good morning, and thanks for taking the question just a couple for me first a presentation that yeah, well that is that a late breaking presentation I just wanted to clarify.
Ah Thanks, Samantha I just have based song.
She'll give you detail there.
It is a normal oral presentation.
Back to be accepted.
Okay got it. Thank you for that and then I also wanted to clarify I heard you mentioned, obviously, we will have updated data for the first and second patients and then <unk> I think so far patients. So what would be six patients in total that we will get data on V. O E. R is that Ibs D oar patients tunnel.
Total will be for patients at the <unk> presentation.
Got it Okay, and then you know when youre, making that cut off.
Incremental additional two patients.
Level.
Follow up it was cut off so I'm just curious is it a couple of months alright that one month.
Any other information you can provide would be helpful.
Yeah, we are where we are for the next two patient will have two months or more.
Perfect. Thank you so much for taking the questions.
Okay.
Just thought of one other thing I think it's important to understand that obviously, we will because the abstract which will be published later was based on data cut earlier, we will actually be presenting more data than in the abstract apps.
Perhaps he had congress.
Yeah. Thanks, Campbell and just kind of also follow up on that the abstract will be available on may 11th.
Yeah.
Thank you next question is from Dae Gon Ha with Stifel. Please proceed.
Okay, great. Thanks, so much for taking that question and I look forward to the data update next next month. So I guess I was just kind of.
I'm wondering about your strategy going forward. So maybe if you can kind of walk us through how you're thinking about next programs or priorities beyond at a three O on I think Gilmore you mentioned in vivo HSC editing, but curious is delivery tech where less burdensome conditioning, a stronger emphasis in your lineup or is it advancing new programs.
And if it's the latter I guess would you continue to do other ex vivo HSC or is it more of an in vivo and in that case would you also think about other Oregon that I've got a follow up.
Yeah. Thanks, very much Jay gone, we are a large part of our discovery focus is actually on in vivo I think that was a very important part and pivot of our strategy because we believe that it maximizes the heart it maximizes our ability to exploit the powerful technology.
That we have available to us.
From a bunch of the H S sees.
If you reduce the problem of in vivo to sort of three elements.
Selecting a robust effect, our molecule AR or enzyme CRISPR enzyme selecting a good targets and then delivery. We believe that we have solved two of those problems are with very robust human data in the use of our cash 12, a CRISPR enzymes and the target for that.
Pacific H B G. One to promote her.
And so that reduces it to a in vivo delivery problem as I said in my earlier remarks, our discovery group is actually working on that and we look forward to updating more at an appropriate time in the future I will say that we are looking actually also be on a H S. Six two.
Tissues and again, we'll give further updates in the future.
Got it. Thanks, so much and then second question I just wanted to follow up on a based on his commentary during prepared remarks. So as you were going into the field and kind of gauging physicians take on edit 301, you expressed or you commented on their high enthusiasm wondering if you could comment I guess what proportion of those.
The docs you visited are also looking to administer a C check still below one and I guess has there been any kind of gauge or ascertainment from your part as to what their sort of motivation would be in taking C. Checks don't blow on like are they lining up patients right now for CTX.
Below one what kind of sentiment do you have are there any reservations on that approach any thoughts on that would be helpful. Thanks. So much.
Okay. Thanks for that question, Yes, I visited quite a few number of study sites.
Actually many of them are.
Being participating the previous gene editing trials. So they are very enthusiastic about the approach we're taking.
Quoting the the different targeting a region for editing and the different enzyme to do and so actually the benefit of losses paid you'll see them as you get into a lot of experience in this field.
Yeah.
I think you know just the build.
Building on based songs remarks, you you know as you're obviously looking out towards the evolution of the market against the background of your enthusiasm for our investigators and indeed, the patients are with the increase in our acceleration enrollment.
You know, we anticipate that in the future that the vast majority of patients will be a wasting dosing.
At the time of our launch and I can go into more details on that but I think a very important point I think something that has really resonated with the investigators.
Is that are our initial clinical data were very encouraging as presenters in December consistent with our preclinical data and we're actually very confident that we will see replication in a subsequent patients as we continue to monitor them through the execution of the 301 studies.
Great. Thanks for taking our questions.
Thank you. Our next question comes from Steve <unk> with Raymond James. Please proceed with your question.
Good morning. Thanks, So much my question it actually requires a bit of a pre lubes I hope everyone can bear it for a moment.
Made a comment on globin locus editing, increasing fetal hemoglobin independent of erythropoietin stress and as you know the ice or recent Isa report, an extra selling covered and ongoing phlebotomy at least one in sickle cell and some of the earlier data releases for that program in thalassemia indicated phlebotomy is there as well.
But that just.
Just stop being reported at some moment, so it's not clear how prevalent lobotomy uses for.
Extra sell and this is all important because there was data at ash years ago as I'm sure. You also know, indicating the Bcl 11, eight added in cooperates with phlebotomy in primates to accentuate F cells and ultimately H b.
F levels, probably because of the stress you are with the polices that causes so.
All of that said I'm curious if you agree with.
With phlebotomy is potentially confounding fetal hemoglobin data for Bcl M&A approaches and if you know what is the impact specifically, where you're editing approach at the H B G want to locus.
It has provided me he's been like in your study and if you think this is all potentially a competitive advantage for you. Thanks.
Steve I think very much for your question.
So from our own clinical data or preclinical data and also public spend going up you're probably referring to for the <unk>.
11, a targeting approach.
Requires some stimulation for the.
Uh huh.
Quite a stress to increase their feed all of being a sufficient fetal globin expression and so that's actually the reason, we choosing that target and we are treating now and we actually just takes a longer time to get all of the target from clinical and preclinical.
The combination of a very robust.
And that was associated with a robust fetal hemoglobin expression, suggesting that indeed stressed roeske leases as we hypothesized at based on the known biology, and our non clinical data that our approach is not dependent on stressors cruises.
Thank you. Our next question is from Yanan, Zhu with Wells Fargo. Please proceed with your question.
Hi, Thanks a.
For taking our questions maybe to continue the discussion a little bit from the prior question Appeal Mall you mentioned.
That the total hemoglobin for the first patient.
It is quite robust and in a normal range.
The percent of fetal hemoglobin appears to be a very much in line with our competitor gene editing products.
So I was wondering is the great. Her total hemoglobin reported for that patient.
Is that due to the total number of red blood cells that could that potentially be a reason or could it be related to the baseline level of hemoglobin in that patient and along that line to continue a little further and to.
Perhaps looking into what we could expect from patient number two at <unk>. I was just wondering could you remind us are the baseline total him a baseline hemoglobin for patient number two and what is the normal range for the female patient, which obviously is the.
Second patient.
Thank you.
Thanks, Yeah, and so thanks for that question and then Hum.
I'll start with the with the answer to your first part of the question regarding the <unk> the feed.
Critical hemoglobin <unk> versus the number of red blood cells and all of these.
We what we see is actually robust arris rope hoist is for these patients we observed so they actually a more global level, it's country by both the the hemoglobin a sale as well as the number of Red blood cells and you can see that we actually do see that the the inquiry.
Also on both end of that and then also wanted to mention that.
Even though we also have like around 45% of fetal hemoglobin and because of the.
Total hemoglobin level is high and the total amount of fetal hemoglobin is also high so that's kind of on that and then regarding the second patient and we will present the data very soon.
I'll now comment on the specifics of the patient data, but I can.
Mentioned that of course, the male and female normal hemoglobin level of different usually if a male is around $13 five to up to 80 Gram per deciliter for female is around 12 to 14 and depending on the reference lab. So that's a difference in that too I think one of the thing you are.
Ask the question about does what was the baseline.
Hemoglobin of Ah patient, one and I think what we can say is that the hemoglobin or the total hemoglobin increase that we saw a car very rapidly just within the first few months of dosing are comprised.
Comprised of three and a half a gram.
Her deciliter increase yeah.
Yeah, just add another nuance on that baseline for sickle cell patients is basically for gene editing trial and because the patient when they prepare for a free seas and conditioning and they usually have blood transfusion. So the baseline and actually we have if not the lowest level rewrite cornea, we just set up a time of the visit.
As a baseline so actually has compounded it could be many different reason for the baseline on that too.
Just a nuance of lower yes, actually you know is lower than it is around the living over 10 Gram per deciliter, when we actually on our record for these patient. Mr. For example are about the baseline maybe live in about computing.
A very very nice thank you for all the explanations and maybe a quick follow up do.
Do you expect this to be also a differentiator for our T D T and perhaps maybe at a greater level of significance because of anemia is the major manifestation of that disease. Thank you.
Thanks, very much going on.
We designed our discovery group.
Scientists designed and selected the combination of cast 12, eight with the specific targeting of the H B G. One to promote her using a set of empirical experiments.
To determine what was the best approach to driving not just feature his wound to expression, but robust erythroid output that would be our red cell output from the bone marrow and that would be independent of stressors squeezes or anemia.
And those empirical experiments really determined that are approaching or directly targeting the H B G. One two promoter would be better and that was the original.
The original design hypothesis, the non clinical data preclinical data actually supported that showing.
Robust erythroid output in comparison to other approaches.
As well as robust fetal hemoglobin.
Expression and indeed that is what we have seen a you know as we disclosed in our first.
Sharing of the data of cuts of the data from our at Ruby study.
So obviously it is we haven't seen enough data in our edit filed patient, but what I can say is that RUBI has certainly demonstrated data that are consistent with both the preclinical data, which were supportive of the original biological and therapeutic hypotheses.
Great. Thanks for all the answers.
Thank you. Our next question is from Phil Nadeau with.
P D. Cowen. Please proceed with your question.
Okay.
Good morning, this is happening because when he goes for for sales. Thanks for taking my question.
On the C. D on the sickle cell program have you met with the FDA to being bedroom disagree with you on the regulatory path.
And then a separate question on the PPP program for the year, an update would that include only the Sentinel Ah patients that you initially built or would that or would you disclose additional patients.
We'll be disclosing any additional patients are you planning on reporting when you switch from central on dosing to borrow both of them. Thank you.
So I think what I'll do is ask based on two I've actually I'll keep track of the questions. They song if if I'd ask you just to ask the.
The question, the FCA and the regulatory interactions that yeah.
Yeah. Thanks, Thanks for your question.
So we we certainly Uh huh.
A lot of engagement and F D and you see that recently, we have the orphan drug designation and from the registration perspective, we previously announced that we actually.
Have the alignment on the potency assay with FDA, which where FDA will consider this efficacy data can be supporting registration and we will have further engagement with the agency to align on the total registration package for that BLA submission.
Which is also a plant.
And your.
Second part of the question is about the they pay let's say all a mouse data so.
So we are moving we along with our.
The address they will study and we expect that we will have a theater by yen at year end more than Sentinel patient and.
And so we're looking forward.
Yeah.
Yeah.
Okay. So at.
Well, we are actually patents do as you know I think the key thing is we're on track to.
Yes, the data for readout at the end of the year for that initial readout at the end of the year and we haven't determined if we're actually going to share that but I think the important point is that we are well on track to disclose a good initial data for the end of the year.
Got it thank you.
Yeah.
Thank you our next question.
She comes from Rick.
Thank housekeeping with Cantor Fitzgerald. Please proceed with your.
Hey, good morning, and congrats on all the progress.
I guess I'll expand a bit on the last question on our path towards registration for edit 301.
20 patients is a pretty substantial cohort size in sickle cell disease. So do you have any sense of how many patients worth of data you will need for a registrational filing.
Yeah. Thank you my question. So we certainly bad thing come back.
With a gene editing approach that to Oh, we have we wont be able to generate a substantial amount of data and any specifics on the number of patients to be able to use for registration we need to align with their major.
Regulatory agencies that we're planning to discuss with FDA.
Okay got it and I just have another quick one I.
I was hoping for a little bit more granularity on the collateral collaborative revenues for the quarter, where all of the $9 9 million in revenue attributable to the shoreline transaction or are there. Some other revenues attributable to other partnerships in there.
It's a combination of both the shoreline and then some other.
More like sub license revenue.
Okay got it thanks for taking the questions.
Thank you.
Thank you. Our next question is from Richard <unk> with Credit Suisse. Please proceed with your question.
Good morning, and thanks for taking my question I have a couple of questions for you guys. So what the appeals litigation pending what does it mean for companies such as CRISPR for texts that already filed a BLA for yourself that utilize CRISPR Cas night from your perspective are they don't have a license from you or the bro and could potentially launch the product before.
We know the outcome of the appeal.
Insight here would be helpful.
And then I have a follow up question. Okay. Thanks, very much rich Ah I think the key thing is is that we sort of anticipate the judgment in the early to mid 'twenty 'twenty. Four we are confident that we prevail as we have before largely because while they are under discussion as the application.
The law and not about new facts and applications at all by at P. Tab Ah that's setting aside that interference I think the important thing is to say that we have a portfolio of IP not subject to engineer.
And the interference that actually covers.
In development at using cast nine <unk> for the application of human Therapeutics.
No.
Looking forward, we are happy to grant licenses to enable delivery of this technology to patients.
And I believe that we should recognize significant value around that.
Oh, okay, great. Thanks, So in terms of getting license. So we're not going to know what the appeal decision likely before the.
The B L. A.
And also potentially the launch like how do you sort of think about that.
Well I think that there are a number of important points to make I think the first again just to remind that the appeal applies to some of our at cast nine Ah in humans therapeutics IP of states, but not all I think it's important to emphasize that we have a cast nine or IP around cost nine.
Use in human therapeutics that is not subject to any interference and therefore is not subject to that appeals case, and we actually believe that it actually covers a product in development.
And so.
What I think I want people to really understand is that that appeals case is around interference on some of our IP estate, but not all.
Yeah.
Okay got it and then just one more question for me so youre seeing some nextgen a C D therapies already in development with new conditioning agents. So you don't think feet. It doesn't seem like the shelf life for the first Gen therapies last too long.
Thoughts about this.
So just to be clear I understand your question.
You, you're actually questioning if the evolution of new conditioning would actually change the landscape for the products that are either or close to approval.
It's really much very much depends on the nature of the conditioning.
As we look.
It talks to Cushing, obviously, something we've looked at closely I one of the important things is to balance both the reduction of toxicities with and grasp and efficacy and I think we all see that it's a very important path to increasing our eligibility for.
For patients because more patients will be able to tolerate a non genotoxic less toxic conditioning regime. Many of the regimes or are some of the approaches are not actually editing dependent are they are actually and so we actually believe is that b.
The evolution of mild conditioning could actually expand and grow the size of the eligible patient population for all.
I think the important thing obviously also says that we are looking beyond a not just a.
Conditioning, but looking to in vivo editing as part of our strategy.
For the very simple reason that we believe that in vivo editing will further increase the eligibility of the patient population for treatment.
Got it very helpful. Thanks.
Thank you. Our next question is from Daiichi Chatto touches with Guggenheim. Please proceed with your question.
Good morning, everyone. This is Ryan for Samsung for Deb, Jay I, just wanted to build off of the conditioning discussion and sort of get the outline of your strategy.
Is it sort of bifurcated kind of exploring both in vivo editing and the opportunity to in license assets that would be alternative to your cell phone can you sort of map that out for us and just wanted to get your thoughts too on the ESG C. T abstracts I'm instead of what you've seen in the competitive landscape.
Inscape around conditioning, and especially given that a competitor is kind of moving forward with the C. D 117 approach.
Yeah. Thanks.
Thanks, very much Ed cheat I think I'll start and then I might have a based on comments from a strategic point of view, we are using a two pronged strategy we.
We have.
Directed investments significant investments.
Internally to our discovery of in vivo editing for.
Hematopoietic stem cells and I think as I said earlier, we believe that this is a problem that.
That we can focus on where we can focus on delivery. We're certainly in humans. We believe we have solved the two of the three at challenges around the selection of a crisper enzyme as well as a target.
In parallel we actually are continuing I have ongoing evaluations of.
Of milder conditioning approaches and I think you asked them more.
Importantly, all of our and follow up a question about the for example, the C. D 117, I'll ask based songs just to talk about that yeah. Yeah. Thanks. Thank you Mike Thanks for your question.
I can say that we have looking into these modern conditioning they deep.
Looking at the space as well as the internal efforts mice.
And they were generally probably two approaches one is that a C. D woman seven antibody and in that direction and the other one is actually doing the doing the cell modification together with gene editing. So the latter approach is still in.
The infancy, if I may say.
And the previous posted with antibody have many different exercise on that so I think we are very closely monitoring the space and understand these and I also wanted to mention that the module conditioning.
Excess fall is not going to be only successful for sickle cell transplant is gonna be successful fall little EMEA and many different genes or gene B. So peak area. So we are actually very much looking to the space.
Thanks for the insight.
Thank you. Our next question is from Madhu Kumar with Goldman Sachs. Please proceed with your question.
Hi, This is Rob on for Jade. Thanks for taking our question. We were just wondering how should we think about Florida, opex, given hum, our opex, particularly R&D given our robust recruitment engine, Ruby and how lumpy will spend being around cell editing and transplants versus bottle.
I'm, sorry, Rob I actually had great difficulty hearing your question could you just repeat that please.
Okay.
Sure. We're just wondering how we should.
Thinking about forward Opex, particularly spending in regard to.
Transfusions versus follow up.
Okay.
So I think you're asking me if I might make sure you're asking about forward looking opex around the execution of the Ruby study.
Is that correct okay.
Michele yeah. So.
So Rob I mean, we do disclose our annual Opex or quarterly Opex, but I can tell you that about half of our spend.
It is.
Both the Ruby trial, and the TVT trials so.
We don't expect an enormous increase quarter over quarter.
But as we do.
Do you feel it's more patient obviously, our R&D spend will go up but not substantially.
Thank you.
Our current I'll, just say when my current cash runway.
Sure.
Moving trial.
Thank you. Our next question is from Greg Harrison with Bank of America. Please proceed with your.
Good morning. This is Mary Kate on for Greg. Thanks for taking our question. So with 19 patients enrolled in plans for 'twenty two we dosed by year ends maybe how many sickle cell patient had been currently treated with edit 301, and maybe how can we expect to see that's represented and.
See read out by the year end update thank you.
Thanks for that question Mary. So we are we have 19 patient enrolled and among those.
Just mentioned of four have been dosed and we actually have more patients have been completely as leases have a CD 34 cells edited and read into scheduled dosing and then we have other patients who are in the process of free space and so we are very confident that we will be able to dose 20 patients by year end.
Great. Thank you.
Thank you. Our next question is from Luca <unk> with RBC capital markets. Please proceed with your question.
Oh, great. Thanks, so much for taking my question maybe on <unk>. Obviously, most patients are in southern Europe . So wondering if you could comment on what's the plan to capture that market and maybe how you're thinking about some new key muscles learns from the unsuccessful launch of Bluebird bio there and then maybe I'll speak to some of these wondering if you can comment on pricing, obviously, you're active or.
Poor suggests $1 $9 million. So wondering if that is actually aligned with your thinking and then maybe lastly on LCA 10, any update on partnering discussions there. Thanks so much.
Thanks, very much Luca so obviously beta thalassemia is a disease that dominates our parts of the world, particularly in Europe .
Southeast Asia, South Asia amongst others.
From our point of view of our forward looking we are actually focusing our efforts on North America. Currently we have shared in the past that from an upside point of view. We are looking are open to partnering.
Ideally targeting as a partner with a large global footprint.
Who would actually collaborate certainly and sort of X V or ex U S development and commercialization. So that's what I would say.
I can talk about a when we look to as you pointed out based on how this EMEA outside at North America I will say that we are happy very happy with the progress that we're making with execution here within the United States and North America.
With regard to pricing I think it's very early days yet for us to be talking about pricing. This is something that we would be very happy to discuss when we're actually closer.
To approval and launching and we look forward to future conversations around there obviously.
Look to the market evolution over that time, but we're going to talk about that at closer to launch and approval.
And then finally with regard to LCA 10.
We have we really have a practice of not really going into details until we have a deal signed and executed.
And maybe just ask one multipart.
Sorry, just one more point.
Actually Omar mentioned about pricing right. So certainly we are very early stage and we're happy to see the community is looking to the value of these gene editing therapy, and we are happy to see that to the OXXO reported recently in the space. So we as Guillermo mentioned that this will be evolving by the weekend. We are pleased to see that the entire <unk>.
Many T record against the value of the medicine in these field. Thank you.
Thanks, so much.
Thank you. Our next question is from Jay Olson with Oppenheimer. Please proceed with your question.
Hey, good morning. This is true around the life of Jade. Thanks for taking the question maybe two from US. So I'm just wondering if there's a chance where you have the capacity to do more than 20 patients for the sickle cell disease program this year and the.
Second question.
Ex U S strategy, what's your current thinking and if you are planning to partner that program.
Are you going to do that thank you.
Thanks very much.
So do we have capacity to dose more than 20 patients, yes, one of the important Ah.
Points.
Wrote out our strategy.
Was to again sharpen our focus on developing and accelerating 301, and indeed, we have deployed capital to enable not just the acceleration on the clinical side, but actually also to ensure that we have a capacity to address our.
Our other CMC capacity to edit and support those that that clinical acceleration. So indeed, we do have that capacity to dose more than 20 patients.
And then I think your second question was around ex U S and the time and partnership.
I think as I said before we are at we are interested in partnering we're looking to a partner with a global footprint that would actually certainly support ex U S.
Particularly on the development and commercialization and with regard to timing, we wouldn't really discuss the timing on this.
So we actually have a deal.
Signs of execution.
Okay.
Thank you. Our next question is from Joel Beatty with Baird. Please proceed with your question.
Hello. This is Benjamin polluter on for Joel Thanks for taking our question.
Looking across other late stage products in sickle cell and T. D. T. It appears that datasets are 30 patients could support approval. So with the auto task being on track to dose 20 patients by year end, how quickly do you think you'd be able to secure the necessary data to support regulatory approvals. Thank you.
Yeah. Thanks, Thanks, very much been based on.
Yeah, Yeah. Thanks for the question.
I mentioned earlier in terms of total number of patients required to support registration and then you'd have a requirement that would require the alignment with the regulatory agency and in terms of the progression of the.
So I think the key thing is is that you.
You've actually identified sort of a benchmark, but obviously, what we need to do is.
Planned assist with the regulators and come to an agreement on what the data set they would like to see for our programs.
Great. Thank you.
Okay.
Yeah.
Thank you.
Our next question is from Joon Lee with choice Securities. Please proceed with your question.
Hey, Thanks for taking the follow up question and sorry for the background noise about the translation.
So I had the same question I think questions, but maybe a different way of asking you know what percentage.
Super cell patients with hereditary persistence.
Hemoglobin have mutations along to the globin locus.
You're 11 8 million.
Oh I see.
A question so.
We we do not have all the specifics.
On your question on that but we we know is the mutation the promoter region directly impacted the fetal globin expression, but a P. C. L. A is a transcription factor, which impacted multiple different cell lumi.
And that the mutation of the BCA 11, a we'll have a much different impact.
From the.
Our physical wellbeing.
Only will have other impacts to <unk>.
Mutations it's much more complicated issue and then the SPF H with the promoter region and mutation for the chemical being promoters.
I think another way to June to actually characterize this.
Some of the prevalence is a little harder to quantify but another way of pointing it is really the strength of the signal.
The hereditary persistence of fetal hemoglobin and capacity to substantially mitigate the.
The effect of sickle cell disease and thalassemia.
Determined actually quite some time ago, because this phenotypic change.
This phenotype to genotype correlation was actually quite robust and identified actually a few decades ago, whereas the BC 11, eight was identified really had through a G was.
Assessment of genome wide.
Thank you.
Okay.
Yeah.
Thank you.
We have reached the end of the Q&A session and with that ladies and gentlemen. This concludes today's call.
Thank you once again for your participation you may now disconnect your lines.
Okay.
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