Q2 2023 Editas Medicine Inc Earnings Call
Good morning, and welcome to editors Medicine second quarter Conference call. All participants are now in a listen only mode there'll 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 relation.
At Edison Medicine.
Thank you Maria good morning, everyone and welcome to our second quarter 2023 conference call.
Earlier. This morning, we issued a press release, providing our financial results and recent corporate updates.
A replay of today's call will be available in the investors section of our website approximately two hours after its completion.
After our prepared remarks, we will open the call for Q&A.
As a reminder, various remarks that we make during this call our company's future expectations plans and prospects constitute forward looking statements for the purposes of the safe Harbor provisions under the plan.
Securities Litigation Reform Act of 1995.
Actual results may differ materially from those indicated by these forward looking statements as a result of various important factors.
<unk> that was 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 filings.
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 substance.
Hey.
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.
Thanks, Christy and good morning, everyone. Thank you for joining us today for Eddie past, the second quarter earnings call I'm joined today by three other members of the <unk> Executive team, our Chief Medical Officer based on by our New Chief Financial Officer, Eric Seara, and our new Chief Scientific Officer, Linda Berkeley.
I joined <unk>, one year ago tasked with guiding its evolution from platform development company to a commercial therapeutics company.
In January of this year, we shared Eddie passive strategy and a set of objectives for 2023.
The strategy just as a reminder comprises three pillars first to accelerate the clinical development of edit 301, our autologous ex vivo cell therapy for sickle cell disease, and beta thalassemia and drive toward approval and launch.
Second to strengthen our discovery organization by dividing it into an advanced technology group had a translational therapeutic discovery group and so sharpen our discovery focused to in vivo editing therapies and third expand our business development activities to park, a complementary technological capabilities that can advance our in people.
Pipeline development. In addition to out licensing our robust IP and know how to maximize the use of CRISPR based medicines.
Our 'twenty to 'twenty three objectives are providing a clinical update from the edit 301, Ruby trial by the end of 2023, providing a clinical update from edit 301 edits all trial for <unk>.
Transfusion dependent thalassemia by the end of 2023.
Dosing 20 total patients in our edit 301, Ruby trial by year end hiring a new chief scientific officer with specific expertise aligned to our vision, which we have just done.
Advancing discovery of in vivo editing hematopoietic stem cells, and other tissues, and leveraging our robust IP portfolio and business development to drive value and complement or gene editing technology capabilities.
So how we executed against this strategy as these objectives in the second quarter.
The leadership side, we made two important hires to drive our new strategy.
Just last week, we announced that Linda Berkeley, a respected scientist and highly experienced and successful therapeutic discovery leader has joined <unk> as our new Chief Scientific Officer Linda.
Linda has an outstanding track record of inventing or contributing to the foundations of multiple approved medicines and late stage clinical candidates.
We're looking forward to that and his leadership as we build on the current in vivo editing work in hematopoietic stem cells and other tissues.
Also eight weeks ago, we announced that Eric Lucia.
Really experienced former biotech buy side Investor Chief Financial Officer, and strategic corporate Finance executive who joined <unk> as our new Chief Financial Officer, you will hear from him later on this call.
As a reminder, under our new target selection criteria, we would select therapeutic targets that allow our genome editing approach to differentiate maximally from the current standard of care for serious diseases. The target selection criteria will work to identify targets and maximize the probability of technical regulatory and commercial success Linda.
Linda will tell you about these developments at a future date.
We remain on track to dose 20, Ruby patients by the end of 2023 and we commenced parallel dosing of patients in our edit found trial in the second quarter of this year.
In June of this year, we presented clinical data from our Ruby trial at the European Hematology Association annual scientific meeting and data from both our Ruby and that it's L trials at a company sponsored webinar.
We remain on track to provide another clinical trial update by year end for both trials.
Share further updates on enrollment progress in the coming months.
Based on will share further details regarding the developer to edit 301 in his remarks as well as recapitulate, the Ruby and edits all takeaways and clinical data that we provided in June .
And as we have previously stated we plan to engage with the FDA in the second half of the year.
After Ehow and our company sponsored webinar, we raised approximately $117 billion in net proceeds from our follow on issuance of common stock extending our cash runway into the third quarter of 2025 as we continue our advancement of edit 301 towards BLA prepare for commercial manufacturing of it at three one and build our pipeline to transform.
The treatment of serious diseases.
As we progress towards our goal of delivering life changing medicines to patients. We also continue to expand our footprint to support our manufacturing and quality management for clinical supply and for preparation of commercial launch. We recently increased our teen room capacity and are moving to a new easier Devin facility. This new facility and increased capacity was.
First the scaling of editorial one program manufacturing clinical supply for our Ruby and NFL trials and prepare us commercial readiness.
Turning to our intellectual property position as a reminder, Eddie Chasse holds a large portfolio our foundational U S and international patents and is the exclusive licensee of Harvard University and broad institutes past nine patented states covering past nine used in making human medicines.
A small fraction of these patents are invalid and the ongoing U S. PTO interference proceedings, we remain confident that our IP portfolio provides meaningful value now and in the future.
We are energized by the promising efficacy and safety data that we shared in June . So you think that edit 301, maybe a clinically differentiated onetime durable medicines that can provide life changing clinical medicine or benefits to patients with sickle cell disease, and beta thalassemia longterm, specifically driving early and robust correction of <unk>.
EMEA and sustained increases in fetal hemoglobin.
With our sharpened our strategic focus are world class scientists and employees in our keen drive and execution, we continue to build momentum to progress our strategy to deliver differentiated editing medicines to patients with serious genetic diseases.
Look forward to update you on our progress in executing our new strategy throughout the year now I would turn the call over to based on our Chief Medical Officer.
Thank you Kevin good morning, everyone.
Let's start with the editorial wider Ruby trial for severe sickle cell disease.
As Guillermo sat in the East we stated in his remarks, we continue to dose and in locations and will be trial.
As we have previously shared we began parallel dosing a patient b trial earlier this year and.
And we remain on track to dose totaled 20 patient trial by year end and to provide additional clinical data by yet.
We will share further updates on enrollment progress in the coming months.
As Jim also mentioned, we plan to engage with FDA in the second half of the year.
In the edit they'll try up 83, one for transfusion dependent beta thalassemia or PDP.
We continue to dose then enrolled patient.
We command the parallel dosing in this trial in the second quarter, we remain on track to pop out and additional clinical updates from anything I've tried but yet.
As we have done all the movie trial. We're also taking multiple measures to accelerate the development of Eddy Street, one for T T and have a strong positive momentum.
We have even more multi location.
Those all have been competed a preset cycle and having a 30 34 cells edited or in the process of prices.
Turning to the edit 301 couldn't go data in June we shared a promising clinical data in an oral presentation at the European Hematology Association Congress Oh, yeah.
Followed by our company sponsored Webinar, where were also presented positive initial data from the first patient treated in anything that trial.
The Ruby data from the <unk> cover the safety and efficacy data from the first four patients treated.
Quoting 10 day 10 months data from the first patient and six month data from the second patient.
The data, including total hemoglobin and feed them.
Excitingly the data were consistent with the clinical results we shared in last December .
I would like to take a few minutes to recap some of the Ruby and NFL key takeaways from our presentation in June .
This included the following.
It is really a one drive early robust correction of anemia to a normal logical range of total hemoglobin in as early as four months after Edison confusion.
And if we weren't drives robust sustained increase in fetal hemoglobin in excess of 40%.
All four dosed Ruby secrecy location.
Pain free Batzel acoustic events.
Since Eddie when treatment.
All dosed patients for Ruby patients and one NFL patient showed a successful mutual feeling Griffin within one month of dosing and has stopped with red blood cell transfusion.
And it's really one was ground tolerated by patients and the safety profile was consistent with my black it yourself and conditioning and a topic that came up with stem cell transplant.
And to the trajectory of collection of anemia, and increase the expression of fetal hemoglobin was consistent across Alex we want to just stick with relocations and better communication and the same for all the time points.
Looking at the data in more detail.
It will be steady patient one total hemoglobin, which is normal peak logical level by five months after 83, when infusion and these normal levels persisted during the 10 months fallout.
Patient <unk> fetal hemoglobin fraction increased to 45, 5%.
By myself.
Pacific during the 10 months follow up.
The increase of total hemoglobin fetal globin fetal hemoglobin, although there will be patient to stream all follow the same trajectory application what.
Or how do you fill the first patient experience with <unk> hundred one we assembled a backup for Ruby patient.
The first NFL patient achieved a fetal hemoglobin fraction of 34, 9% over four Gram per deciliter, and you just went and a half months.
<unk> Street on treatment.
We continue to believe that a editorial one can potentially provide robust clinical benefit to patients potentially provide clinical differentiation and long term.
As we have previously stated.
The choice of Christopher enzyme and the gene editing target to switch on freedom of expression matters.
And if one uses our proprietary ace got swap a enzyme to edit etch be Q1 to promote it.
<unk> got 12, eight increases editing efficiency and significantly reduce off target editing when compared to other CRISPR nuclease, including has not.
H P Q1, two editing in humans, CD 34 partial warehouse without in greater wet blackout protection normal prevent to capacity and improved grade plus out health when compared to anything up at PCR 11 eight.
Based on clinical data so far we believe that sustained normal level up totally hemoglobin could be a potential point of differentiation for Eddie for you what.
As a reminder.
And normal total hemoglobin level is an important clinical outcome for patients.
And the correction of anemia has significantly improved quality of life and ameliorate and organ damage.
As I shared last quarter.
I've been visiting our Ruby and edit their trial clinical trial site.
And continuously speaking with investigators I appreciate the enthusiasm and support from the investigators and study site.
Pleased with the momentum of anti PD, one in patient recruitment, a precise editing and dosing in both studies.
I'm excited to hear from the investigators the patients.
Patient dosing with Ed if we won already see positive changes in their lives.
We look forward to sharing additional updates as the year proved methods, including additional Ruby and edit their clinical trial data by year end.
Now I will turn the call over to Eric Our Chief Financial Officer to review our financials.
Thank you Barry and good morning to everyone. Let me first say that I'm excited to be speaking to you today.
Came to editors because I was impressed by the quality of the company's science its leadership in the gene editing field, it's strong IP portfolio and it is highly differentiated from other players in the field.
I was eager to take this opportunity and joined <unk> at this pivotal time, we just shared exciting human data for our lead asset and we're preparing our regulatory and commercialization strategy.
I look forward to leveraging my experience to help the company execute our vision to bring innovative medicines to patients and drive shareholder returns.
With that I'd like to refer you to our press release issued earlier today for a summary of our financial results for the second quarter 2023.
In future quarters, I will give more guidance and for now I'll take this opportunity to briefly review a few items.
Our cash cash equivalents and marketable securities as of June 30th or.
480 million compared to $402 million as of March 31, 2023 <unk>.
We're expecting we expect our existing cash cash equivalents and marketable securities to fund, our operating expenses and capital expenditures into the third quarter of 2025.
Revenue for the second quarter, 2023 was $2 9 million compared to $6 4 million in the same period last year.
The decrease is related to a program license opt in from our collaborator Bristol Myers Squibb that occurred in the second quarter of 2022 that did not occur in the second quarter of 2023.
<unk> expenses for the quarter was $17 2 million, which remains light, which remained relatively flat from $16 9 million for the second quarter of 2022.
The slight increase in expense is primarily attributable to increased professional services expenses to support BD activities, partially offset by a decrease in stock compensation expense.
R&D expenses this quarter were $29 8 million, representing a decrease from $43 7 million from the second quarter 2022.
The decrease in expenses reflects the focus on execution following the strategic re prioritization of our portfolio offset by increased activities to accelerate the development of edit 301.
Overall at a test remains in a strong financial position and our sharpened discovery focus coupled with our recent capital raise allow us to concentrate our talent and extend our cash runway further into 2025, which provides ample resources to support our continued progress and there will be an edit out clinical trials that are added.
Oh, one continue commercial manufacturing preparation and advance our discovery efforts.
As a former investor I know the value of buy side and sell side knowledge and I look forward to working with the company and hearing from our shareholders as we work to advance our gene editing medicines.
With that I will hand, the call back to Gilmore.
Thank you Eric.
It has been an exciting year for edit has thus far and fulfilling first full year for me at a test we look forward to continuing our transformation and sharing our progress with you as we as a reminder, our strategic objectives for the year include providing a clinical update from the edit 301, Ruby study by end of this year, providing a clinical data update from <unk>.
101, NFL trial for transfusion dependent thalassemia by the end of 2023 dosing 20 total patients in our edit 301, Ruby study by year end advancing discovery of in vivo editing hematopoietic stem cells other tissues and leveraging our robust IP portfolio.
Development to drive value with complement or gene editing technology capabilities as always it must be set that we could not achieve our objectives with the support of our patients investigators employees and you. Thanks very much for your interest in Eddy tests, and we're happy to answer questions now. Thank you.
At this time, we'll be conducting a question and answer session. If you 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. We ask that you limit your questions to wanted to follow up so that others may have an opportunity to ask questions.
Participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys.
One moment, please I'll we poll for questions.
Our first question comes from Terence Flynn with Morgan Stanley . Please proceed with your question.
Great. Good morning, Thanks for taking the questions.
Two for me I was just wondering if you could provide any update on the timelines for your first in vivo program and then secondly on the ease or facility, what's going to be required there from an FDA perspective, and do you have to make any changes or do any bridging work to use product from the two.
Cities and the expanded trials. Thank you.
Thanks, very much for your questions.
Apropos as in vivo.
As I've said, our focus is on maximizing the poverty of our technical and commercial success with care positive differentiation against the standard of care. The work is continuing to progress and I'm, particularly delighted to welcome Linda at Berkeley here, whose leadership is really <unk>.
Going to double down and really help us drive this forward and we'll update you on timelines after the appropriate time, when Linda has had a chance to really settle in and make our mark.
With regard to the Asia or a facility we anticipate from a regulatory point of view, obviously, we are engaging our planning to engage with the FDA in this half of the year and are on track at to have those conversations and we don't anticipate.
A significant or major work in the transition it essentially.
The processes, we have are largely locked down and as I say, we are engaging with the agencies just to ensure that we are aligned on our readiness for a commercialization there.
Yeah.
Our next question comes from Samantha Macau with Citi. Please proceed with your question.
Hi, good morning, and thank you for taking the question.
Now that you have wonder on the team could you discuss some of the challenges that and be able to elaborate yes, hematopoietic stem cells and how youre thinking about mitigating these challenges going forward. Thank.
Okay.
Yeah, Thanks pretty much Samantha for your question I'm going to say Linda as you say has just joined so I'm going to talk about how she.
She and I and the Blender if you want to add anything please feel free.
One of the things is that Linda and I are really highly aligned philosophically on our approach to this we have made we actually have some ongoing work and work is progressing on there that the challenge is obviously our to deliver.
The ex executive functional enzyme and guides to the hematopoietic stem cells. The good news is that you you know we feel that we have certainly solved two of the three challenges the first being that we have human validation of our cask 12 day enzyme Ah thanks to our three.
Oh, one program and also we have validated in man in our clinical trials and the use of our targeting of the gamma globin promoter specific targeting and delivering all of those are mechanisms to the humana stem cell is something that we're working on currently and will be happy to update you more.
On specifics on progress there at an appropriate time.
Yeah.
Our next question comes from Greg Harrison with Bank of America. Please proceed with your question.
Hey, good morning, and thanks for taking the question.
What feedback have you had from physician you know since the recent update.
The 301 program regarding a clinically meaningful dataset and sickle cell, especially with respect to hemoglobin <unk>.
Response and.
What are they looking for.
From the next update.
Thanks, very much Greg I'm going to ask it based song.
To take that question.
Thanks, Greg My question.
Absolutely we received very positive feedback from all.
Our investigators as well as patient community.
Investigators there really to see gradually see the consistent CLO readout and the normalization of their total hemoglobin and I actually ask a specific question to one of our investigators.
And you know how do you feel that normalization or towards mobile and with Nevada, and he will stay that's app with ebay important and he has his own patient that.
Already told him about the energy that the patient has a different they have and their life change in there too. So that's supposed to pone clinical observation and the patient likewise effective and relate to the telephone patient community perspective.
After the June presentation, our volume of patient inquiries increased 10 times over that period of time after the after the presentation. So we're very pleased on that too and we continue to see receive inquiries I know what I mean.
But also Canada, and we are helping them to two moving through and understand the trial and finding visibility whether they join us.
Hey, Rick.
Great. Thanks for taking the question great to hear the progress.
Thank you Craig.
Our next question comes from Joon Lee with true Securities. Please proceed with your question.
Hi, Congrats on the progress and thanks for taking our questions your cash guidance into the third quarter of 25 is a pretty specific what's baked into that guidance. Specifically does that include preclinical and IND, enabling studies of in vivo programs.
<unk> or BLA and pre commercial activities for edit 301. In fact is that your intention to commercialize edit 301 yourself or would you be looking to a partner to help me with the heavy lifting on the commercial effort. Thank you.
So I think juvenile I, let Eric start Ah yeah with respect to the.
Specific.
Dollars that are included into the cash runway guidance.
Putting all of the BLA activities and everything we need to do to prepare.
For that within the guidance with respect to I N D.
Enabling studies, we do have some early.
Preclinical research amounts in that and we'll see what Linda comes up with in the future as to whether or not.
We reallocate some some dollars from other programs or not but.
That's where we are at this point with respect to guidance in terms of commercialization partner, Yes June we actually see commercialization as you have a nice upside, particularly for it makes you sure we expand our global footprint.
And with previous stages that we certainly want to partner ex U S. Obviously as are.
We talk to partners or potential partners. If the terms really line up with where we are to ensure that we can maximize value for patients and shareholders. We could consider co. Commercialization addition, too.
Global co development and commercialization in the United States.
Thank you.
Thanks.
Our next question comes from Gena Wang with Barclays. Please proceed with your question.
Thank you two very quick questions regarding the in vivo target is it fair to say it would be a liver targeted and it was a lipid nanoparticle delivery and then regarding the manufacturing just follow up so you'll have a easier facility.
What is your plan for commercial manufacturing capacity in the future.
Holly.
Yes tried to do a wholly in house or would you plan to also collaborate with the C. D M O regarding the manufacturing commercial supply.
Thanks, very very much gena with regards to the in vivo targets at all.
Okay and would talk about in the future.
Okay.
Thank you.
Our next question comes from <unk>, Let's depot. Please proceed with your <unk>.
Hey, good morning, Thanks for taking my question two from me as Wild one with regards to <unk> remark on total hemoglobin is being a differentiation I guess at what point do you kind of draw the line in the sand and look at that as a differentiator from potential competitors and then as a follow up to that.
Vertex last night announced that there will be an AD Tom for X S. L. I guess, what are you kind of thinking about in terms of a point of contention or debate or discussion, whereas we approach that and how would you see the <expletive> cast 12, a targeting H b G promoter as potentially raising another AD com Ah when you.
Guys go down that line. Thanks, so much.
Thanks to my question.
Regarding the total hoot moving and related differentiation. We are in the current political but already actively look into that and as a online previously we're looking for free category with things when he is coming from the logical and other lab values beyond the one in the second.
It is the end all can function that thought it would be patient what outcome unplug it life and maybe a few it on the the last two we in a kind of protocol, we monitor a key organ functions, but such as cardiovascular pulmonary and renal function, we have multiple measure to measure the function.
So we believe that the hold of him and there will be increased what it impacted the quality of life and in Oregon Health. So that's why we focus on the end organ function.
And then then regarding reported patient or the outcome there could be a very important part of that.
Initiation for example, fatigue is the main one of the main complain bicycles locations and that and already data to show that fatigue can really related to the anemia on that too and similarly for pain. That's also another another Ah Ah Ah Ah the complaint that the patient.
Half and we also have a clear closer monitoring of those issue report outcome. Among other things we can share more.
Yep and then yeah.
Yeah.
You want to add for the technician is no place in English then the second question is about AD come and yes, we we heard the news about the icon, which of course. This is a novel area that M E Com, which is I'm not a surprise and we feel that will help us to learn more about how their data looked like.
And how the the the expert committee view about all Additionally, how old are your feelings about.
And then relate to.
Oh magazines about action, we do not believe that target always kept 12, a cost additional consent from mechanism action must back here you know as I mentioned before we actually our targeting of H B 212 promoter is mimicking the nature magnetism of the game.
The participants Ah.
Ah Ah Ah Ah Ah Ah the the Ah [laughter] I really sweet, but now I'm thinking blogging on that so that's kind of the nature of mutation without targeting too and so we're looking forward to see the outcome.
The inflammation from that come from the from the Ah the accident.
Great. Thanks, so much.
Our next question comes from <unk>, and I didn't with Cowen and company. Please proceed with your questions.
Alright, Thanks for taking my questions to from US as well first in terms of the year and update on Reuben NFL. What is your most recent thinking about the number of patients. So it will be disclosed Ah for both trials and and the follow up for the patience and their disclosures.
Yeah. Thanks for your question, what's everyone's jumping yes. We are we are on track Joseph totaled 20 patient by you and we are we are not disclosing the specific data to be released at the end, but we are on track to provide clean update it for both studies.
Yet.
Great and then certain questions on the interference proceedings that you're referencing the prepared remarks can you remind us what are the next steps are in those proceedings and when they could conclude.
Yes that thanks, Bill the oral really it's the scheduling of oral presentations is the next step those are a presentation of yet to be scheduled and then what we'd anticipate the judgments in early to mid 2024 Ah. It's worth stating that we are confident that we prevail as we have two four at both in front of the.
Federal circuits Appeals court as well as with the U S. A P T O.
That's very helpful.
Thanks for technical or something.
Our next question comes from <unk> do with Wells Fargo. Please proceed with your questions.
Hi, Thanks for taking the questions I wanted to ask about your F. D. A interaction in the second half of the year what will be the focus there do you think you have enough data to inquire about pivotal path do you think the kind of treatment.
Forwarded to the competitor in terms of 17 patients forming the basis for a payment a cohort and the length of follow up could be similarity afforded to your to add it to 301 as well and also lastly, do you think.
You have enough differentiation on the total hemoglobin to request a breakthrough designation. Thanks.
Thanks, very much N N for that detailed question I'll start with regards to the F. D. A interactions that we are planning for this year or this half of the year, we will be talking about multiple elements, including our a C. M C. As I've said.
As well as having a cynical interaction with regards to the clinical data I'm glad that you highlighted you know, where we see bar, Texas BLA and the acceptance of an efficacy pool verses up 17 patients Ah with about 18 months I'll follow up data, which I think is a helpful precedent.
Uncertainty and I'm element of guidance that we can actually use and then as based on has said the the add that will be scheduled towards the end of this year by the F. D. A I think will actually also at the very illuminating as we continue in our continuous.
S of aligning with the agency and understanding we can come to an agreement on bottom there'll be ultimately are pivotal part and B L. A filing a strategy with regards to differentiation based on that I Wonder if you want to add anything yeah yeah.
Yeah, and I I think to your question about differentiation is related to the <unk> to the eligibility fall back through explanation, we have carefully looking into into the regulatory vaccinations and it might've been keeping bees in there and it's something that we are excited by the data we have seen so far and evaluate the day carefully and.
Ah Ah Ah Ah on these past.
And very helpful.
Related to your earlier question about you know 17 patients bomb from like the X F. L. F. Gilmore mansion that we want no more especially after the L. F calm, but it's a good reference boss and definitely still require a final alignment with F. D. On it but it's got a package has to be quiet.
Yeah, and I think the good thing that I should of course highlights. It was at that we are on track to dose 20 patients by the end of this year and so that number.
Is it a helpful number for lining up with what we see with the B L. A acceptance for exercise.
Thank you.
Great very helpful. Thank you.
Our next question comes from Rick and Koski with Cantor Fitzgerald. Please proceed with your question.
Hi, everyone. Good morning, and welcome telling Dineric, thanks for taking my questions.
So I wonder if we get some clarification on manufacturing.
Expanded agreement with the aspirin group seems to be focused on clinical and commercial radio readiness for U S markets given that the footprint in Massachusetts.
Does the company have any manufacturing capabilities and European markets or would any European based manufacturing have to come from another potential partner.
Mm, thanks, very much Ah Rick.
You're a bank branch in in in in reminding everyone that we have this exciting new agreement with Asia as we get ready for a b L. A and and commercial launch with regard to X U S. We'd be consistent in our messaging that we actually would prefer actually expect.
A partner to help us with the ex you asked for the point of view expanding our our footprint and obviously when we talk about that we talked about in terms of co development co commercialization at all the activities necessary for that.
Alright got it and I do have one quick follow up sure and what are the areas of development that was highlighted back in January of this year was exploring the use of mild air conditioning recommends for stem cell transplant.
So my question is once a milder conditioning regimen is developed I was curious how this could potentially be folded into clinical trials and what the what the approval password. It looks right here for you Smith added three one.
Thanks, very much Rick for that and reminding us that important because obviously bye bye.
My craving a four by the development of a minder conditioning, we see a path to increasing eligibility Ah for sickle cell disease and T. D patients of which are very serious diseases, but it against backed by the condition you can extend or expands the the mind or or I'm, sorry, the eligible population.
We continue to evaluate that and it's a question really of balancing the reduction of toxicity is within grasp efficacy in in in in general terms, we see might've conditioning as something that the transplant sites will use and as they grow comfortable with it they will expand it.
Cross their use protocols and that's how we actually would see is enabling Ah Autolycus X people therapeutics.
Got it thanks for taking my question. Thank.
Thank you.
Our next question comes from Rich law with credit. Please. Please proceed with your question.
Hi, guys. Good morning, my congrats to both Eric and Linda for joining the company. So the question I have is like can you provide insight to how the partner programs are progressing so the 11th Alpha beta to sell programs with the M. S and Yankees, what's your mind about biosciences and when do you expect to you come to be disclosed in more detail.
And then I have a second follow up question as well sure. Thanks, very much rich with regard to the B M. S House debated T cells.
We're actually happy with the progress there have been 11th opt ins you know most recently five in 2022 two of those options have passed D. C. In the most advanced as an I N D. Enabling studies beyond that is B M. S that is in the driving seat I would actually provide.
More specifics on the program and with regard to shoreline. We're really delighted to have had the opportunity to divest R. A N K a.
<unk> Ah programs at two shoreline shiny shoreline is actually happy with where they're going and I think really any more specific updates would come from shoreline.
Great. Thanks for that and then the second question I have is following up on the milder conditioning with that unable to use up therapy.
An outpatient setting versus inpatient setting.
So that is actually I I I think at the very key of optimize conditioning, because it's <unk>, it's really a combination of reducing the risk to patients, while obviously, enabling them and still.
Maintaining a optimal.
Transplanted engraftment outcome, but yes that is one key element certainly either to do it as an outpatient or substantially reduced as a need for inpatient monitoring of the patients and you know we continue our evaluation of that because we see it as an important.
Elephant for expanding the eligible patient population.
Great. Thank you so much.
Very much Rick.
Our next question comes from Jack Allen with Bird. Please proceed with your question.
Great. Thank you so much for taking my questions and congratulations to the team on the progress.
Maybe first to start on the on the clinical side. It seems like you've made a lot of progress dosing patients with sickle cell disease that are adults, but I was wondering if you had any comments as it relates to your plans to move in a younger patients I know somebody of your competitors I'm moving the patient sorry, 12 to 18 years old how did you hear any thoughts there and then I have one quick follow up.
Thanks, Jack I go to ask based on to take that question. Yeah. Jack. Thanks ballpark question absolute Okay. We are intended to expand these Ah Ah Ah study to all all all age groups. So we are actually actively working on that and with more.
Data, we are much more comfortable to go beyond the patient population.
Great. Thank you so much and then maybe on the I P. Font, you mentioned that the hearings have not been schedule minutes interference case quite yet, but I'd love to hear any thoughts you have as it relates to leveraging our I T and <unk>.
One of your competitors advanced or potential approval I guess should we should we think about essentially a settlement coming before approval occur to come after or how do you think about conversations disarming that I P and how you were looking to leverage your strong position there.
I I think I'll keep physician or I P is that we really want to ensure that which is used to enable therapeutics for multiple patients with multiple franklin multiple terrafugia errors and indeed, there's a large number of cash nine products that are in development across both <unk> and.
Vivo and across multiple disease areas as we think about leveraging that from an economic are monetization point of view, we see that that that will vary depending on the on the stage of developing the disease area beyond that we're not going to discuss you know individual companies or negotiations.
Alright, <unk>, sorry, <unk> follow up on the I T. Can you just remind us as to who has the duty to litigate glad for you that you are your license or from my perspective.
So I I I think I'm not going to talk about the details of our our legal strategy, but you you know the key thing about protection of I P. As that that we will we will actually protect your I P. Obviously, you want to insure and we are open for business and very willing to give licenses, but we will protect her eyes.
P.
Great. Thank you so much for the color and congratulations on the progress and.
Thank you so much.
Our next question comes from J, All Sandwich Oppenheimer. Please proceed with your question.
Oh, Hey, congrats on the new additions to your team and thank you for taking the questions can you provide an update on the number of patients enrolled in the sickle cell thalassemia trials to date and since you're on track to dose 20 patients.
<unk> <unk> do you have a similar dosing go for thou Asemia and if so how many and then separately can you talk about the X U S opportunity for circle silence I was female.
So I'd ask based on to address your first question on enrollment.
Yeah.
Thank you. So we are have great momentum have been wanting patient dosing patients as I. Just mentioned we're on track that that was 20 patients yeah, and and we previous month, we'd actually to have 20 patient already involved in the in the in the last quarter.
East So we hope we will I'll provide an update on the woman in the coming months and so that we can share more information on that and and regarding the goal of the of the number of patient doses. The NFL, we have not Ah Ah Ah Ah disclose the ethical.
Oh, so you're not pregnant.
And then with regard to the X U S opportunity you know, we see actually a.
A substantial <unk> opportunity there and we also see that and believes that the best way to maximize the value for patients and frankly for us and our shareholders is to seek a partner with a large footprint and there are a number of parker's in that space.
That are potentially very interesting to us, but as I say the the opportunity is large and we want to make sure that we optimize the delivery to that opportunity, which is why we're seeking a large parker.
Great. Thanks for taking the questions. Thanks, very much J.
Our next question comes from many <unk> with Leerink partners. Please proceed with your question.
I guess, that's taking a question obviously a lot of questions on this call been around for clinical trials execution enrollment et cetera, I Wanna dig into N points. If we could you've discussed earlier on this call pain and other sort of fatigue. Some quality of life issues is important areas of potential the French.
Nation Uhm as clinical trial important these are pretty notoriously variable and have some element of subjectivity. What efforts have you made if any to ensure comparability between your data on these end points and that your competitors, who are likely to have an AD com approval. Prior so that we can <unk>.
Really differentiate as I clearly demonstrated differentiation if any thank you.
Thank you very much I'm gonna ask based on to address that yeah. Thanks. Thanks for a Christian many beg a question about the endpoints and maybe at the next step back a little bit on that so after I joined the joined the editors and the mid up last year and one thing.
Doing it was actually two men at a protocol, we actually changed the primary unemployment.
As the the.
The complete the relation of the C. B, a b O eat that is consistent with other face we couldn't try it in a few minutes.
[noise] about 30, so that's kind of the primary endpoint perspective.
As I mentioned that there are many an appointment we are looking to including that and organ functions and.
<unk> and you have to you have absolutely make it a point about possible variations, especially the pianos in there too. So we're looking to into that direction that will be we have more data on that and the method understanding but we feel that this is the area that we.
We have you know palpitations and the disease by itself. We have we will have more information about the <unk> and the other data and allow us to have a understanding where we stand for those end points.
Great I guess, one quick follow up you type all that how to understand where you guys are on those end points.
Did you do you have any evidence of good point is two and the literature are based on experience and <unk> data from other companies lay around around what duration of follow up we would need to be able to compare cross trial.
Statistical caveats, notwithstanding and kind of provide clear evidence of where you guys are versus where your competitors are like how long follow up would we need.
<unk> with your friendship on paid on fatigue on quality of life. Yeah. Yeah. They may get a question. That's something we are looking to like to first of all I would say yeah. There are some inflammation already published if like downplaying the allogeneic transplant, that's back to your policy courtyard disease.
Vacation in mind about your plans on that to see how patient improve after the b.
The the the.
<unk>, a stem cell transplant, while those patient and then.
And some of the specific end points someone will take a longer and the other with a shorter time for example, we already see the total will be increases beige or the time of period. So that's kind of a puppy taking showed a time on boys and.
And so except for some quality of life you may six six months or 12 months ago and fall at the end organ damage and that's actually space that will continue to learn they are actually publication for example to say from from central nervous system and after the the allogeneic transplant Misfeed improvement.
The dynamic improvement in the blood vessel in the brain. So there's a lot of studies in this space, but again, we asked you don't need a new character. We we are looking into that we're confident that it will walk me see something over the period of time to see some differentiation.
Great that's really helpful. Thanks, guys.
I think it's also worth Reemphasizing of course that you know the biochemical physiological differences that were already describing our circuit generating significant excitement with prescribing a treatment physicians I'd actually also within the patient community is based on alluded to in the feedback on the follow up.
That we've had since presented you know very exciting data around the correction of anemia. In addition to robust enjoyed those people came with open expression at the.
How many things in our webinar insurance.
Our next question comes from Luca inside with RBC. Please proceed with your question.
Oh, great Uhm. Thanks, so much for taking my question Congrats on all the progress maybe one more manufacturing since you're moving your manufacturer processed your new facility.
To assume that the F. D. A will ask you to run a clinical Richie study similar to what <unk>. What do you think the just analytical comparable is studying be sufficient so again any call their much appreciated and then maybe on R&D pretty material declining you already spend this quarter versus prior corner. So I'm wondering if you could provide any additional color what role.
That it actually thinks about modeling deadline going forward and finally, one last one on D D any update on partnering <unk> excellent.
Yeah, Thanks, very much Luka with regards to the amount of facts xraying.
I think a couple of key points. One is that we are manufacturing within at the age of space. We're already running our clinic is fly in a separate Asia space with regards to the details and specifics of what would be required. This is a would be part of our alignment at meetings with the F. D. A.
As we have outlined with regard to understand I I will start and then ask Eric if he wants to actually add any additional color to the spent the key thing about the already spent obviously was that following the rollout of our new strategy, where we sharpened our focus on in vivo and really <unk>.
<unk>, an accelerating our trio on asset we did actually.
Sharpen and narrow or a pipeline and so that had an impact on spend with with.
The day of divestiture of R. N K assets to at shoreline as well as the cessation of R. A V inherits rather dystrophy programs, Eric I don't want to add anything more to that no I think you've covered that pretty well I would just add from a philosophical basis that I think we're all on line.
We Wanna be focused on high conviction R&D spend high conviction assets.
We're not really into a quote unquote, Brian Indeed pipelines and quote unquote lots of shots uncle now, we really want to focus our resources on a few assets that we have I conviction on and I think that's a philosophy that the team here chairs across the board.
Think that's an important piece of guidance and it actually a key reason that's Linda.
Linda has joined US it's not just about you know at continuing that Ah to advance the work that we have in progress are in people, but Linda is very aligned philosophically at with myself and leadership team when it comes to approach to selecting targets and maximizing the poverty of technical and commercial success like clearly select.
<unk> targets, where we can with our technology can differentiate from the standard of care in a meaningful manner with regard to the update on L. C 10 I.
I will give you a more specific update you know when if we have a it take or or a deal I will tell you. There has been at some interest and obviously we are we believe that we saw something a signal.
In the patient they set that we analyzed and hope that that's a another firm or sponsored with especially interest in depth disorder would actually take it forward.
Alright, thanks, so much thanks again.
Our next question comes from Lisa Peco with Evercore ISI. Please proceed with your question.
Hi, Thanks for taking my question I, just had I wanted to drill down a little bit more on the central air conditioning regimen program. So.
What are you working on in terms of approaching that what do you think are the most.
I guess promising approach it out there you know.
What is your thinking on when something like that to come to market and I guess if someone else.
Develop something like that is that something you think you could fold into your program or how do you think about.
Instead of owning something like that versus someone else and your access to it. Thanks.
Thank you very much for the question well at first I just wanted to say that I'm Super excited to them joined anytime I'm I'm. So impressed with that foundation I'll technology as well as it really truly excellent of.
Of this team coming to your question of course might have air conditioning is such a crucial parameter for many therapeutic indications that apology. So many therapy indications as well as of course, the sickle cell disease and so it's been sell it after.
To have my other conditioning regimens for for many years and we are of course, uhm monitoring the landscape and keeping very active mm mm I and all of the various uhm approaches that are out there as well as I've only been here for seven days, so I'm getting up to speed.
What the team is doing internally I can.
So if I can get more for additional comments, but of course, we are going to be very active in terms of.
Perfect. Thanks, very much Linda and you know with regard to how we would fold as in I think you're I I <unk> I think I've actually touched on us, which is that might've conditioning as Linda said is generalizable across multiple elements or or or I say therapeutic areas. When it comes to the use of stem cell transplantation and so we.
C at any advances certainly in in stomach therapeutics, that's impact and can result in might've conditioning, having an impact cross expanding the eligibility or the eligible patient population and obviously that would have an impact an hour therapeutic use obviously looking beyond might've cause.
<unk> you know the in vivo target as a matter of would extend ourselves as an additional step which could eliminate the need for any conditioning as well as actually further reduce the burden on patients and health care systems by eliminating the need for harvesting Ah freezing and collecting C. D 34 poster stem cells.
And the way we received rolling in as we would see the development of a minder conditioning therapeutic like whatever sponsor adopted a transplant centers in there at transplant protocols and then generalizing across.
The therapeutics basis that use stem cell transplantation.
Thanks.
Our next question comes from Bryan King with J P. Morgan. Please proceed with your questions.
Hey, guys. Thanks for taking my question. This morning, just one quick one from me on manufacturing on the ancient facility do you have any insight on when this facility could come online how does that timing potentially fit into the pivotal portion of the ongoing studies and S. U will discuss.
What the F D. A on C. M C. In the second half. This year are there any specific important items that you need to get online with the agency. Thank you.
Thanks, very much Brian with regard to online the timing of this deal and this expansion is really tied to our at DLA readiness planning and so we are happy.
Happy with the progress with Asia, and the readiness to be online.
At the time, that's appropriate to support at our BLA with regard to F. T alignment I'm not going to go into the specifics here, but essentially we just want to confirm and agree with our understanding around the guidance and their.
<unk> the guidance and our program as it pertains to a a so therapeutic.
Thank you ma'am.
Thanks, Brian .
Our next question is from Steve Seedhouse with Raymond James. Please proceed with your question.
Hi, Good morning. This is Nick out for Steve We were wondering if and how you're measuring off target or translocations for editorial wanted Rudy R. N F L and and whether or not you're quite a show instead of thank you.
Thanks, very much Nick for the question, we actually have a a comprehensive we have a comprehensive set up assays for looking at off target edits were actually very happy with the data we have seen interestingly, we actually saw a following.
<unk> proposed essentials assays Ah controls to the agency that many of those remarks were reflected in the guidance that was published after our submissions, but we were actually very happy we're seeing off at target edits, we believe that'd really supports.
Validates our approach to choose and ask has 12, a crisper enzyme which is you know is differentiation both for its efficiency as well as its specificity and very low.
Indeed on detectable off target editing. So all is actually moving consistently with the selection of that enzyme in his prioritization in our app portfolio.
Alright, thank you thanks.
Thanks, very much Nick.
Ladies and gentlemen, we have reached the end of our question to answer session, which concludes today's call. Thank you. Once again for your participation you may know.
Mmm.
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