Q3 2021 Lineage Cell Therapeutics Inc Earnings Call
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Thank you.
[music].
Yeah.
Welcome to the lineage cell therapeutics third quarter 2021 conference call.
At this time all participants are in a listen only mode.
An audio webcast of this call is available on the investors section of lineage website at <unk>.
He told me he told me he thought lineage so dot com.
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I would now like to introduce your host for today's conference.
Got it home.
Rector of Investor relations at lineage.
Mr. Cohen. Please go ahead.
Thank you Chris.
Good afternoon, and thank you for joining US a press release reporting our third quarter 2021 financial results was issued earlier today November 10th 2021 and can be found on the investors section of our website. Please note that today's discussion will contain forward looking statements within the meaning of federal.
Securities laws, including statements regarding our strategy goals product candidates clinical trials data announcements and updates anticipated regulatory meetings and interactions planned manufacturing improvement financing cash management and runway anticipated collaboration opportunities and benefits.
And commercial potential statements made during this discussion that are not statements of historical fact should be considered forward looking statements, which are subject to significant risks and uncertainties actual results or performance may differ materially from the expectations indicated by our forward looking statements due to known and.
Unknown risks and uncertainties.
We caution you not to place undue reliance on any forward looking statements, which speak only as of today and are qualified by the cautionary statements and risk factors in our filings with the SEC, including in our quarterly report on Form 10-Q filed today November 10, and our annual report on Form 10-K for the year ended December.
31, 2020 with US today are Brian Kelley, our Chief Executive Officer, Kevin Cook, Our Chief Financial Officer, and Gary Ho, Our senior Vice President of clinical and Medical Affairs, Brian and Kevin will provide some prepared remarks, and then all of the executives will be available for questions from analysts.
That I would like to turn the call over to Brian.
Thank you Ana and good afternoon, everyone. We appreciate you joining us on the call today.
As you will have seen in today's press release, the lineage team had another productive quarter, most notably we delivered additional positive clinical data from our lead asset off Virginia, which is intended to treat dry age related macular degeneration with geographic atrophy.
We also took important strides to advance our obesity, one program to treat spinal cord injury.
And we continue to grow our executive team with the appointment of Mr. George Samuel as our General Counsel.
Lastly, we continue to generate awareness for the company, our data and our mission through various investor and medical events presentations in other engagements.
As I say each call lineage is pioneering a new branch of medicine.
Our approach for each of our product candidates is to manufacture differentiated cell types with all batches for ever produced from a single cell line.
And transplant those differentiate itself into the body in order to restore or improve function, which has been lost due to aging injury or disease.
Simply put.
We believe that a certain type of cell in your body is dying or dysfunctional you need to replace that specific type of cell.
And as a reminder, our approach does not require any gene editing or reprogramming of the cells original DNA.
It's impossible to perfectly control those edits and there are unanswered questions about how often they will lead to clinical complications.
And these safety concerns are not theoretical as you've probably seen recently and reports from companies which employ them.
We are involved in the debate over which editing approach or platform is best because we avoid these risks entirely.
When we manufacture ourselves we harnessed the natural developmental programming contained within each of our single source cell lines.
This regenerative medicine platform, which we own as broad and powerful.
Not only for the value of our three current indications, but also because our cell transplants approach might someday be applicable to a large number of additional cell types and address many different unmet needs.
We view our approach is the path to reaching our long term goal of becoming the leading cell therapy company.
With that I will turn to a brief review of our recent accomplishments and set some expectations for the remainder of this year and early 2022.
I will as usual I'll begin with <unk>, our lead product candidate intended to treat dry AMD with G. A a disease with no FDA approved treatments.
Our approach to treating this disease transplant healthy new retina cells to replace or support the ones that have died or dysfunctional.
Replacing the entire cell avoids risks and limitations inherent in approaches that target just a single pathway, which may be insufficient to drive a clear clinical benefit.
Or gene therapy approaches which targets.
Certain mutation, which might only be relevant to a small subset of patients.
Importantly, we now have evidence that our approach can not only slow the disease process, which is the objective of the leading companies in this space, but also even halt or reverse and area of atrophy.
Which obviously would have a far greater impact on disease progression.
We feel evidence of this reversal of G E in three patients so far.
Particularly in those who received a fulsome covering up our oxygen sells across their atrophic area.
This phenomenon is something we refer to as retinal restoration and is identifiable by the new appearance of critical layers of retina, specifically the presence of new areas of RPE Montclair with overlying ellipsoid zone external limiting membrane in the outer loop nuclear layer all of which were not present in previously.
Firmed atrophic areas at the time of baseline assessment.
This is a remarkable clinical observation, which is achievable with just a one time administration of RPE cells performed at about 30 minutes under local anesthesia.
We believe our approach may have an enormous advantage in terms of compliance convenience and benefit over traditional drugs being developed by competitors they required monthly or semi monthly injections.
We also have seen phase III data from one of the leading complement inhibition companies.
It is unclear whether that data will support approval, but if we assume that it does given the debt products critical benefit was somewhat modest it clearly leaves abundant room for improvement and opportunity.
But in the restoration data, which we have previously presented is something which has never been shown with any complement inhibitors or oxidative stress producers, but in addition to the anatomical observation we have reported.
We also have seen functional improvement in the majority of our patients treated eyes.
In contrast, visual acuity decline and the majority of our patients untreated eyes.
Seeing these benefits, even though a small population is exciting but visual acuity can be a difficult assessment to rely on in clinical trials because of the size and location of atrophy only broadly correlates with vision.
For this reason, we expect our primary efficacy endpoint in a registrational trial will be the change in the rate of growth of asking overtime, which is similar to what is being pursued by companies like propel us and it's Eric.
Overall the data set we are collecting includes both functional and anatomical evidence, which further reinforces our belief that the suspension of operation RPE cells can generate clinically meaningful outcomes in patients with dry AMD with ta and particularly in those with earlier stage neutral effect disease.
Looking ahead.
We will be providing an important clinical updates at the 2021 Academy of Ophthalmology annual meeting, which takes place this week and through this coming weekend.
Dr. Michael <unk>, who is a retinal imaging experts from UCLA is there any eye Institute will present, new imaging data, which he and Dr. Castaldo are generated from our cohort four patients.
You're already aware that we have recorded rapid restoration using high resolution <unk> imaging, which is necessary because of the inability of older SaaS technology to detect our healthy RPE cells.
But more recently have performed a sub study analysis of that same data using a separate technology known as <unk> October.
<unk> is a validated part 11 compliant image grading software, which can provide quantitative information on the thickness in the area of acute layers of the retina.
Using this approach we can interrogate not just the boundary of G E as defined by the formation of new Lebron layers, but now we're able to report on the individual measurements of thickness and volume of areas such as the outer nuclear layer or the RPE drew some complex.
Dr. <unk> also will provide a general clinical study update but what we're most excited about in the coming days is being able to provide a second separate method of measuring retinal restoration.
And for that data to again come from an independent and highly respected group of experts.
And because of this presentation will be made at the largest ophthalmology meeting in the world. We are hopeful that these data will foster greater professional awareness of our program.
In addition.
We also are working with our advisors to prepare for multiple engagements with FDA.
First aspects of comprehensive designation, our manufacturing plans and the design of a late stage clinical trial.
We anticipate the first of these events will occur in this quarter and will continue into the first quarter of 2022.
Throughout this year, we have provided a cascade of positive updates from the use of origin with increasing evidence of benefit in.
And the durability of those benefits has risen as more and more time has lapsed from the date of treatment.
With our minimum 12 months of data on all patients available later this quarter.
We are excited to move this program forward and unlock the potential of this approach overall.
We believe we are seeing the validation of our cell transplants approach with each passing day and that the manufacture and delivery of a replacement RPE cells eventually will be proven to be the most powerful and effective methods of treating this degenerative condition.
And next we'll move to OTC, one hour cell transplant for treating spinal cord injury patients.
Oh P. C. One is a one time administration of oligodendrocyte progenitor cells, which are delivered directly into the area of spinal cord injury.
As with dry AMD. There currently are no FDA approved treatments for spinal cord injury. Despite many attempts.
Our view of this indication is it is very similar to dry AMD in so far as we may be able to demonstrate that transplanting a whole functional cell into damaged area can accomplish things, which are beyond the reach of traditional small molecules or other biologic approaches.
Those efforts have not yet led to any product approvals. So it's clearly time for some new approaches.
Because of clinical effect alone is insufficient for commercial success. We spent the past two years working on a new manufacturing process for OTC one.
Our attention to this area led to tremendous advances in the purity scale and reproducibility of our OTC one batches.
This new process was recently moved into our in house GMP facility at a batch size, which can support a registrational sized clinical trial.
And we expect to complete GMP production via this new process and with unusual and inject formulation in the first quarter of next year.
Ensuring ample supplies clinical material for the controlled study we wish to conduct in this indication.
At the same time that we're completing steps to introduce our newly manufactured lots into clinical development.
We also plan to initiate an interim clinical safety study of a new delivery device through a collaboration with near gained technologies.
This new device was developed to help avoid complications and risks which could occur with an earlier delivery system.
Obviously delivering materials spinal cord is a delicate procedure and anything we can do to reduce patient risk is important to us.
We also have learned from the option program that careful placement of cells can be correlated with better outcomes. So it made sense for us to invest in superior delivery options and.
And two of the notable advantages of this new device easier manipulation of the X Y Z axis and the ability to deliver sales much more slowly because of the patient can continue to be connected to a respirator during delivery.
This is a huge advancement over the prior method and we are excited to be pioneering this approach.
Over the past quarter, we completed the manufacture of clinical device prototypes and continue the required verification and validation work based on FDA input, which we received.
We expect to complete that evaluation this quarter.
Ideally thereafter, we intend to initiate human clinical performance and safety testing with the new delivery device and plan to submit an amendment to our open IND in the first quarter of 2022.
We also are preparing for an FDA interaction discuss our cell manufacturing improvements, which we similarly anticipate to occur in the first quarter of 2022.
Interestingly.
Because this performance trial is focused on safety and delivery, we are able to expand the eligible patient criteria beyond the sub acute population treated to date.
And we have feedback from FDA, indicating their openness to us including patients with chronic injuries on this trial.
This was notable not only because it's generally easier to identify and enroll patients who have chronic injury.
But also because any hints of improvement in patients who have functionally plateaued for years would be an incredible fight it.
To be clear showing an improvement in mobility in chronic injury patients is not the intent of the study. It's just something we want to keep aware, we didn't expect to see retinal restoration in the auction program. So it's important to collect the data across a large number of parameters.
Sometimes you find unexpected surprises.
Overall.
I'm pleased with the progress we have made to get all of these pieces fall into place in a coordinated manner.
We have the GMP clinical material, new delivery device testing and some new ideas on regulatory strategy. All converging next year to support further testing of OTC one.
That will conclude the OTC one section by adding that the work that we've done on this program reflects our commitment to advancing nascent cell therapy technologies into commercially viable product candidates.
I have said many times that cell therapy is on the cusp of a breakout but that breakout will require a maturation from early or academic programs through manufacturing and delivery enhancements and ultimately to the generation of controlled clinical data.
The lineage model is to serve as that bridge and facilitate the adoption of this new branch of medicine.
Now thirdly, I'll move to back to our investigational off the shelf dendritic cell cancer vaccine.
Back to as many of you will recall is comprised of mature dendritic cells, which we manufacture from.
Brian Carey established cell banks, which are then loaded with a tumor specific target or antigen to instruct them deploy the body's immune system to attack and eliminate cancer cells.
Our partner cancer Research UK continues to be responsible for this study, but enrollment has been impacted by COVID-19 restrictions across the U K.
C. R. U K is still working on enrolling the final patient into the study and following completion of enrollment and we anticipate additional clinical data will become available and recorded.
Because the trial operationally is out of our hands were unable to accurately predict when that final patient will be treated.
But I will share that I am increasingly frustrated by the pace of enrollment in this trial.
And we have initiated steps to address this situation so that the vac to program can advance any more predictable fashion under our direction and control.
Meanwhile, our focus is on making improvements and modernization to the fact manufacturing process, which will help prepare back to for future trials and provide competitive advantages for any future about programs. We made design in the dance.
This manufacturing focused investment is similar to what we did successfully for both operation and O P. C. One and its core so when you're just competitive advantage in cell and gene therapy.
As part of the manufacturing enhancement process, we aim to enhance the flexibility of the BACS platform, because one could theoretically insert any antigen into our dendritic cells and we believe that that platform is therefore capable of producing a tremendous number of product candidates with each one being distinguished by the specific.
Jim, which our dendritic cells are presenting to the patient's immune system.
This opens up a large number of potential corporate partnerships by allowing us to use our dendritic cells as carriers for other companies antigens, while simultaneously retaining the option to advance our own programs and these partnerships can diversify our oncology pipeline across more programs and provide new opportunities for success.
Without the financial burden of independent development and as Youre aware, we already have the first instance of this with our Glioblastoma program, which we have partner.
So overall.
I believe we have continued to execute on our plans and generate valuable progress advancing all three of our clinical programs and further strengthening the company's capabilities.
With that I'm happy to turn the financial update over to Kevin.
Thank you, Brian and good afternoon, everyone. Let's briefly review our recent financial results.
Total revenues for the third quarter were approximately $2 3 million a $1 $7 million increase from the same period in 2020.
This increase was due primarily to increased royalty revenue from a certain partner based on an updated communication to us regarding the royalties owed.
Total operating expenses for the third quarter of 2021 or approximately $8 $1 million, an increase of approximately $900000 as compared to the same period in 2020.
That increase was primarily related to increased litigation expenses related to the hysteria steel as well as share based compensation expense.
As a result of these items our loss from operations for the third quarter was approximately $6 $8 million, an increase of $100000 as compared to the same period in 2020.
The net loss attributable to lineage for the third quarter of 2021 was $7 $8 million or <unk> <unk> per share as compared to $7 $8 million net loss last year or <unk> <unk> per share for the same period.
Turning to the balance sheet. The company ended the quarter with approximately $65 million in cash cash equivalents and marketable securities.
That wraps up the financial section. So I. Thank you for your time, and we'll now turn the call back over to Brian.
Alright, Thanks, Kevin.
I want to spend the last minute or two here just being really clear about some of the events and milestones that we anticipate so first just three days from now additional interim data from the opportune clinical study will be featured at the 2021 American Academy of Ophthalmology annual meeting in that presentation I described by Dr. Michael.
Before year end, we plan to provide an additional data update which will be able to include a minimum of 12 months on all treated patients in the <unk> study and as a reminder that was that was that is the primary endpoint for that study at 12 month observation period.
We also have multiple winter our interactions with the FDA planned where we will discuss origin product designation, our manufacturing plans and later stage clinical development.
Those steps are anticipated to begin this quarter and continue into the first quarter of next year.
So D O P. C. One program, we aim to complete the evaluation of the mirror gained parenchymal spinal delivery system or the U S. P D system in non clinical testing.
That is anticipated in this quarter.
We also expect to complete GMP production of OTC, one via our improved and larger scale manufacturing process. Using also our new thought and inject formulation that is anticipated to occur in the first quarter of next year.
We have an FDA interaction planned to discuss some recent manufacturing improvements we made to OTC. One that's anticipated in the first quarter of next year and we're looking forward to initiating clinical performance and safety testing of the new delivery device with an IND amendment and that submission is planned for the first quarter of next year.
With respect to the Vac programs.
Completion of enrollment by cancer Research U K and the ongoing back to phase one in non small cell lung cancer is something we hope will occur in the first quarter of 2022.
Continued development of the new back based therapeutic with a strategic partner will be ongoing throughout next year and thirdly, we will be evaluating opportunities for new back product candidates based on internally identified or partner tumor antigens, which also will be ongoing throughout next year.
For lineage <unk>, we believe the field of cell therapy is poised for explosive growth is it scalable allogeneic off the shelf approaches.
Which are prepared in formulations that are optimal for clinical settings have the ability to provide significant commercial advantages over our autologous or patient derived sources and methods and our objective.
As to Usher in a new branch of medicine, using our proprietary differentiation protocols and I believe the data we have been generating are supportive of that goal.
With that I would like to thank you very much for joining us. This afternoon and operator, we are ready to respond to any analyst questions that they may have.
Ladies and gentlemen, if you have a question at this time. Please press the star and then the number one key on your Touchtone telephone.
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Your first question comes from the line of my Young one Titan.
From B Riley.
Your line is open.
Hi, This is actually William on for my I really appreciate you, taking our questions today and great overview.
Third quarter.
Really a lot of good events that went on.
Just curious in regards to your upcoming FDA interactions for your offer Jen.
I was curious if you'd be discussing the and I might have this wrong.
After that you said that you'll be highlighting coming.
Coming up this weekend.
And then the possibility of about incorporating this into later studies and also just kind of at a high level.
See about potential designation changes or incorporation into a subsequent clinical study just show again that there's treatment effect not only on the area of G gotcha atrophy, but throughout the layers of the rental structure of interest.
And then I I guess get high level then.
With with those.
You said there would be multiple F D a.
Will you be communicating those finding an ongoing process.
And yeah, so you're willing.
Yeah, Yeah, Yeah, actually I jumped bird part two so for competitive reasons, we obviously won't be previewing, our regulatory strategy your discussions, but after we hold the N B F. D. A meeting so we have the specific feedback to the plan that we will pursue that is something that we absolutely would be.
<unk> would be sharing typically for how lineage those things we would share that an abundant detail. So.
Have said several times that there are many designs, which might fit well for our particular situation, including various adaptive or or continuous phase two three designs and the work is currently ongoing to determine the clinical plan that we will present to the F. D. A is are you know for.
And best choice, but as a reminder, we we only completed enrollment on November 10th of last year literally one year ago. So those patients home now just finally reached literally today. The primary endpoint of one year post treatment. So I think we're on track and moving quickly and any discussions on the next studied.
Designer going to need to include those data so they're being collected and analyzed and that's why I will will expect that interaction in particular I'm study design would be something that would occur in two one of next year rather than two four this year.
Right that makes sense and then one about O. P. C. One you mentioned that you have a new thought inject formulation I was wondering if you'd get extra any information on how that might be improved over your current formation formulation, sorry, maybe a high level, but also curious maybe like cell viability or just an extra.
[noise] benefit to the patient you might expect.
And the key advantages here, what we're really trying to accomplish with a formulation that was ready to use so one of the headaches of most cell therapy approaches is the dose preparation and in particular for the O P. C. One.
The prior study required the preparation of the cells and had to wash out the medium and and placed them and count them. In this work had to begin the day before so you had tissue culture activities and then eventually you could you could get that material into the patient. The next day that's.
Big headache, and we have the technology.
The experienced the success of doing that with oxygen. So we deployed some of the same approaches in order to have a thought and inject formulation, which will go from a frozen state right into the delivery needle and be administered to the patient without at long delay. So we are we are where.
Putting those kinds of commercial planning and utility anticipation into practice, while we're going through and working on things like scale or you know and all of our programs whether we're following up on on clinical data et cetera. So those are some of the key advantages and I I have an S.
Submit a about a 90% reduction.
In the in the handling of manipulation costs and complexity and by the way. It also will help vastly reduce the possibility of a dosing era, which did occur in the hands of a prior sponsor at one of the sites. So there are a lot of advantages to having a thorn inject formulation and as you.
You did hear me say on the in the body of the call that one of the things that we're really excited about is it will be able to administer ourselves more slowly by.
By using this formulation compatible <unk> with the new device, because we will not be we will not have to take dictation off the respirator. During the administration of the cells and just as an F Y I I was watching some preclinical animal work very recently and the.
One of the cells is you know is essentially automated you know everyone's pencils down for five minutes, while the cells are being administered it it used to be a manual process, where you were you know pushing it and quickly and you have to hurry because there was a clock running because the respirator was not what's not functioning at that time. So we think this is a huge and you know really under.
Appreciated benefit to utilizing this new formulation with this new manufacturing process and this new device and we think it will facilitate the clinical activity and even open up additional sites that maybe didn't have the capabilities to handle these cells, but they certainly have the ability to just saw them at the bed.
Side in the surgical suite and administer them.
Excellent I really appreciate that extra color and thank you for answering our questions today and record everything. Thanks I appreciate that thank you.
He went next question comes from the line of account Kenneth from each seat top with U L. I N E.
Okay.
Everybody. Good afternoon. Thanks for taking the question O'brien I Wanna ask a specific question and then I'm going to get a little more macro. So during your prepared comments you gave us a little bit of a tease with regard to O. P. C. One, saying that your toying with new ideas with regard to regulatory strategy I'm. Just wondering if you could provide a.
Little more off color on that comment.
Oh boy, you're really putting me on the spot.
<unk> [laughter], let's get not yet.
So here's what I can say here, here's what I'm comfortable saying is that they.
But there are there are clinical settings or their disease settings, where b N points are are difficult, let me dry M. D. As one of them you know, we'd all love to think that visual acuity is is easy to rely on but it's it's challenging which is why the the world shifted to more of an <unk>.
Uncle and point in N G. A growth. So similar set you a similar situation exists in the setting of spinal cord injury, it's very difficult to demonstrate improvements in mobility.
Because the tools and assessments that are generally collected are fairly crude you know three point scales and so forth and there are there are other assessments that have been in development and other companies are in an academic centers for many years.
That are maturing and are probably more useful in the way that I would try to put a finer point on this is that I talked to a patient who was on this study who received O P. C. One cells can.
Can he told me.
You know I didn't score is high on one of the the scales because the particular mobility that particular movement, which I had gained didn't registered.
And so he said, but it was useful for me I could sort of curl, a finger and hold on to a mug or a cup and so it was useful for him for independence and his ability to keep himself hydrated, but it never showed up as a benefit clinically because there was a disconnect between his gain a function.
And the score the scale that was being used so without going into specific details. There are other tools that are available and I'm very interested in how we might re approach some of that in order to increase our probability of success by narrowing the gap between.
<unk>, what the cells and the therapy can provide and what is being measured.
And I have some experience with this previously in my career and and I think that there are you know.
I'm looking forward to hopefully getting some more information and being able to share more of a conceptually that's what I'm talking about trying to make sure that what we're measuring is not just clinically important but it's also more closely tied to the benefits that we believe this therapy can provide.
No. That's really helpful. Thanks, and I look forward to I F D. A visibility on their feedback. So you know going to the more macro front as I alluded to you have a lot of logistical things going on at the company you know manufacturing of yourselves, you know first and foremost.
As well as you know your your device initiatives with O. P. C. One so I guess I'll ask the question. This way you know based on all those logistics that you have to deal with in our dealing with do you have any impact or have you had to you know change your plans with regard to all of the global so.
Apply chain issues that everyone's experiencing right now.
That is a very clever question I'm proud to the the manufacturing folks in particular were concerned about supply chain issues and brought it to our attention. So we have done a lot of pre purchasing of critical materials and we have had the bed.
The fifth of being an international company right. We have approximately half of our staff located in Israel and half of our staff located in the United States. There have been times, where we have.
Acquired or purchased equipment or materials in one country and shipped it to the other and by the way sometimes you can find cost savings, but it's more important for us to have availability of those materials. So I have heard stories about.
Lack of availability of.
Things as simple as pipette tips, and plastic I'm, a little bit more concerned with Regents key reagents, because if you are running a two month long manufacturing campaign, and you'll get to day 40, and you're supposed to add you know secret ingredient number seven and it's not available you done.
So the manufacturing team, which is the same team working on all three programs right. That's one of our efficiencies intentional efficiencies about how we run the business has been very good about advanced purchasing and basically stockpiling in particular things materials of reagents that don't have expiration dates and so forth. So.
Yeah, I I hope that every company is able to avoid those challenges, but one easy way to solve them is to just you know order from multiple suppliers and and know that you'll eventually be using that stuff.
That that's really helpful. Thanks for that and it's obviously an ongoing issue and it's it's nice to hear that you planned ahead and are dealing with it. So I guess my other my last question is also somewhat macro focused but on the back program beyond the Nonsmall Dr. Two in the lung cancer program I guess, how would you.
Characterize your ongoing B D discussions for additional antigens that people might be interested in.
How would I characterize those discussions Gulf up discussions and how advanced or immature they might be.
[noise] you know almost as a rule jokes are very good questions are very fair question, but almost as a rule I try not to comment on business development activity or tried to put any sort of.
Uh-huh potential or anticipation behind it because I have experienced.
Huge global partnerships that fell apart you know 24 hours before they were signed for you know almost unrelated reasons and so what I would say is we maintain a a high level of interest in engaging with different entities actually a very broad array of entities.
Form and type of those entities with respect to partnering but I'm far more comfortable letting something get signed and announced then to say for example, I Wanna have you know three of these this year or one big one this year or any kind of guidance at all on partnering I I I think we'll just we'll just let let the surprises happen when they happen during the.
Year on any of our programs.
Yeah, and you know what Brian that's a very fair answer and you know I had to ask it anyway because of the you know the potential around you know beyond lung cancer, you know of the platform potential off the backs sell so but thank you for all the color. Thanks a lot.
Great stuff Joe Thank you.
You weren't next question is from the line of Kristen Blue Sky from Cantor Fitzgerald, Caroline you something.
Good afternoon. This is Rick on for Kristen. Thank you for taking our questions.
Follow up on an earlier question on the multiple guided F D. A interactions in coming quarters around opera <unk>. How are you thinking about priorities and the potential cadence of regulatory discussions as you're thinking about moving the program forward.
Yeah, I'm gonna invite Gary to to respond to that and provide a little bit more detailed than we have in the past sure.
Sure. So <unk> it is <unk> <unk> announced Tuesday, so we've already got bounced direct designation, which affords a certain interactions with the agency. Some some ability to have them help US guide. The program. There are other designations that we can seek that we think we could fulfill the obligations to one of them is our match. So that's a regenerative medicine.
Dance therapy designation.
And you know as the F. D. A review data. They can also suggest if they think that your day to meet their expectations. You can also be consider submitting for breaking therapy. So in addition to our fast tracked we're considering those two ultra options as well.
Taken with our data, which again, we just didn't <unk> one of your posts last patient being treated.
Those days are being analyzed collected right now.
The study reports of being written and we will review in the study meeting with the agency.
So I just got the profile and point size and scope of the next study.
So that would occur following the designation meeting.
[noise]. Thank you for that answer and one more perhaps four O. P. C. One you guided that you could move from a non clinical testing with the PSD system and four Q. This year, two potential clinical and safety testing with O P. C. One with potential IMT Amendment and.
First quarter of next year could you. Please discuss how rapidly you believe you could move from the I N D ammendment stage into clinical testing. Thanks.
Gary do you have some thoughts on the the the gap between I mean, I know, there's a 30 day waiting period, but any any further color on that's our readiness there yeah.
Yeah. So the the agency will review and a number of our are supporting documents behind that will work on the protocol is Brian speaks to that there's the 30 day period of no comment.
But.
Hope would be to to you know obviously you could get the first Batman as quickly as possible. So you know we.
We haven't yet set a date on that get the as quickly as the agency would allow us basically.
Okay. Thank you very much.
Alright next question comes from the line of cheese Mcconkey prompt Maxine.
Caroline or something.
Hey, Brian This is Michael Aquino, which on the line for Jason and Thanks for taking my question.
Hi, Michael.
So I'd like to look.
Could you talk a bit about the importance of now having a cheese typical significance. After the nine month data and offer them study and it just gives you a clear idea of the expected effect size and what kind of trial size you need for a later stage studies and are there any come power.
We'll trials, we can look at to get an idea.
So I think it's probably worse.
<unk> cleared that the the statistical difference exists, which is wonderful, but we're talking about a difference.
Treated and untreated I sort of population.
Datapoints overtime.
The.
[noise] [noise] [noise] [noise] the size of the study [noise].
Details around the end point, all the things that everyone, including us would like to know more about really are a part of the output from R. F D a engagement.
But I think I can add that there are some precedents out there that are informative alright, I don't think that if if you. If you keep in mind that the size of the study is a function C. N required for a statistical plan to give your evidence based upon.
On some benefit or some delta.
That they're sort of a zone of reasonableness.
So if you for example ask on one and where where do you start getting sort of ridiculous as far as your benefit.
And what I mean by that is.
If the bar that exists out there is slowing growth.
Of G E and we're reversing it obviously, we have a whopping clinical excuse me a whopping effect that can be observed in a very small number of patients.
However, when you're doing Registrational studies, there's also sort of a you know a realistic war.
As to how many patients the agency is going to I'm exposures patient is going into the F. D. A is going to want to see in order to feel comfortable with granting a marketing authorization right. So we have we have a scenario where they're sort of you know we can't we can't become overly optimistic or overly aggressive.
Is because we run into the reality of needing to have enough exposures. They were satisfying agency.
So that's one side the other side is that if you make a study so large.
You start to detect with statistical significance, even a small effect right that'd becomes very silly and that's where you know a thousand patient study or something like that that's where you're showing statistical significance of some tiny little benefit and that's not our objective and in fact, we benefited tremendously from the palace Donna.
Which showed a across different studies, what 12 to 29 27, I don't remember per cent range right. So that's kind of a floor right. That's the comparison setting aside frequency of treatment safety profile, just saying clinical benefit that's sort of the comparable floor that we're working with and I think that that's well supported.
By the future user.
User community of saying, we'd like to see you know 20, 25% slow reduction in the growth of G. A so all of that is to say that there is some reasonable boundary conditions, but I could shorten it to be like.
You know look I think it's going to be fewer than 300, I don't know, we haven't gone in but if you're looking for.
Give me an order of magnitude reality check the precedents, which are out there and the size of the clinical benefit and the reference points from complement inhibition and the need to have enough exposures you start closing down the range of what's possible. So you know somewhere between 203.
Irritation seems most likely to me, but we won't have the final number and we will you know make any further clarity on that until we actually have the output from that meeting with empty here.
Alright. Thank you and then can you just provide a bit more granularity on when you're expecting to hear the output from those upcoming meeting both of one four opera done as well for O P. C. One.
Yeah, there's a whole series of.
Events right in a in a covered them in the in the call. The the most notable one around design of an offer Jim clinical study. That's a meeting that we would expect to have in Q1 next year off Virgin has several engagements.
We've got completion of certain activities and then at least two engagements with F. D. A in Q1, so there's actually a very heavy amount of F. D. A engagement that we anticipate in Q1, but I will be clear that those engagements can bury uhm.
Don't always have an in person sit down meeting sometimes big questions can be addressed through written correspondence, sometimes it's a call. You know we don't we don't have everything determined you know we leave it to the agency to direct US is there what they are looking for in terms of the form of meeting and et cetera. So.
You know Q1 is going to be somewhat of an incredible corner for us because the number and substance of those regulatory interactions is so high and that's going to inform them. So much around how long it'll take them only studies whatever needs studies probability of success that people ascribe.
To these studies when they can initiate enrollment et cetera et cetera. So it it's a really important start to the year for us.
Alright. Thank you very much and then one more if you don't mind just like a touch on you you were talking earlier about the ability to do three D measurement of the geographic atrophy and is there a possibility that you could detect additional side.
<unk> a retinal restoration beyond those original three patients where you've already determined it because he would just didn't previously have the tools to measure it.
Yeah, I mean, I'm going to say, yes, because I didn't expect to see retinal restoration to begin with.
So.
Is it possible absolutely, but perhaps Gary you could add something to have that technology works and what we might be able to learn from it.
Sure Yeah. So there's the thing about it is this is being done independently and make blinded passion and I think everyone knows they were treated but they don't any group. This doing the announced don't know in advance who is or is not original restoration nation.
And so independently they identify deserves of ivor or incomplete retinal atrophy as well as sira repair.
And so yes, there's a good possibility there there may be additional patients that they will identify that that are reginald restoration. So we look to them to to validate.
And I wish they'd done so already the three that we've identified eventually identify additional patients that show a clear benefit.
Alright, Thank you very much.
Well.
Ladies and gentlemen, if you have a question at this time police practice Alright, then the number one key on your attached tone telephone.
I'm thinking <unk>.
And the number one key on your attached Tom Palestine.
Alright next question comes from the line of Teen Leon from Cleveland.
Airline your cell phone.
Hi, Thanks for taking my questions just one for me when you meet with F. D. A what is the.
[noise] primary end point that you would hope to establish for running a pivotal study with opera John Thank you.
Yeah, we're gonna have to erase what.
All the leading companies are doing which is the the the the same form of of change in G. A area overtime with three separate assessments I think there's been some misconception in the past and some places are not your place, but I think there's been some misconception because we've had this visual occur.
<unk> data, which is so compelling that that would be what we would elect to pursue.
I think that we view that as subordinate to being able to reverse the the size of the G. A so we would be monitoring the change of G. A over time, we we presume we would be proposing that just like the leading companies in this space the key diff.
Prince is it we'd like to rely on O C T as the assessment tool because F. A F.
Is incapable of seeing ourselves. So the deck is rather stacked against us with F. A F. Because ourselves are invisible using up a off so it would be kind of ridiculous it would be.
Inappropriate it's not it's not a matter of futility idiots why would you use the wrong tool to try to assess it. So I don't think there's a particular.
A particular level of risk I don't think that the F. D. A is going to say no F. A F. As the only imaging tool, we would ever accept that seems kind of ridiculous when the field. The experts in the field I think are very well aligned O C. T is a more powerful and informative imaging tool and I think.
People are going to be delighted that we would be embracing a better camera better imaging technology to measure essentially the same sort of anatomical changes over time.
Gary is there anything I left out there that I should add about about that.
No I think we've covered it.
Again, everyone acknowledges that O C. T is a superior way and taken together you know.
But the Micropro tree and visual acuity the other functional data. In addition to the structural data. The agency is already considered as an approval endpoint. We thinks it makes it the billing story for for origin to utilize that in point and shows true benefit to the patients.
[noise] got it thanks.
Sure.
Great.
I am showing a Friday questions at this time I would now like to turn the conference back to Mister, Brian Cooley right concluding remarks.
Well. Thank you everyone is always we appreciate your support as we position lineage to become a leader in cell therapy and cell transplant medicine. We obviously still have more we expect to accomplish this year, but I think 2022, and even 2023 are gonna be even better as we continue to deliver and his awareness of our accomplishments continue.
<unk>. So thank you very much and have a great afternoon or evening.
This concludes today's conference call. Thank you for your participation and have a wonderful day, you mean now disconnect.
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