Q2 2023 Lineage Cell Therapeutics Inc Earnings Call
Walking through the lineage cell Therapeutics second quarter 2023 conference call. At this time, all participants are in a listen only mode and the audio webcast of this call is available on the investors section of lineage is web site at Www dot lineage cell dot com.
This call is subject to copyright property of lineage and recordings reproduction or transmission of this call without the express written consent of lineage are strictly prohibited as a reminder, today's call's being recorded I would now like to introduce your host for today's call.
Wanna home head of Investor Relations at lineage Mrs. Hulme. Please go ahead.
Thank you mind deep good afternoon, and thank you for joining US a press release reporting our second quarter 2023 financial results was issued earlier today August 10, 2023 and can be found on the investors section of our website. Please note that today's remarks and responses to your questions reflect.
Management's views as of today, only and will contain forward looking statements within the meaning of federal Securities laws 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 the company's actual results or performance.
<unk> may differ materially from the expectations indicated by such forward looking statements for a discussion of certain factors that could cause the company's results or performance to differ we refer you to the forward looking statements section in today's press release and in the company's SEC filings, including its most recent annual ROE.
Pork on Form 10-K, and its subsequent quarterly reports on Form 10-Q, 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 described in our SEC filings.
With us today are Brian Culley, our Chief Executive Officer, Joe, How our Chief Financial Officer, and Garry Hoak, Our senior Vice President of clinical and medical affairs with that I'd like to turn the call over to Brian . Thank you Ana and good afternoon, everyone. We appreciate you taking the time to join us today.
I'm happy to share that I'm feeling very good about how things are going a lot of the items that I will comment on today reflect a business that continues to be on track with its development plans and has many reasons to be excited about the future.
I'm going to kick off as usual with opportunities in particular I want to comment on recent developments in the dry AMD space and how I see those events positively affecting our lead program.
As you no doubt are aware there've been safety issues disclosed recently related to the use of sites over the first FDA approved agent available to treat geographic atrophy secondary to dry AMD.
I'm not going to speculate on the root cause of these safety issues because the story is still evolving, but I am going to highlight that a rare but serious side effect is exactly the sort of thing that can derail a product when it only offers a small clinical benefit.
As we all know the clinical experience to date with complement inhibitors shows that at best if you actually receive all of your monthly or monthly shots in a given year you may see about a 20% reduction in G. A growth compared to your expected growth.
And in terms of the patient experience, you're unlikely to feel any differently when taking it and you'll continue to lose your vision, which is why there was so much debate about whether the product would have enough clinical benefit to be approved.
But it did received marketing authorization and the early signs whether it was going to be a very successful launch due of course to the absence of other choices.
High unmet need in this condition.
But drugs with small clinical benefits can be highly susceptible to safety issues.
Whether you're the F D a prescriber or an individual patient the utility of a given drug stems from its benefits and its risks.
When the rewards are small and perhaps require years to observe even rare side effects can be devastating to our product.
So while the initial launch of <unk> was compelling and highlighted the extraordinary enthusiasm which exists on the demand side.
Its achilles' heel may end up being its limited clinical benefit which is unable to overcome the risks of catastrophic vision loss.
While this risk appear small on an absolute basis they.
The use case for our product is not limited to its risk, but rather the benefits, which must outweighed at risk.
I think the key takeaway is that both patients and prescribers care incredibly deeply about vision.
The hope for preserving even though a small bit of vision, even if that takes several years to be achieved is capable of driving enormous demand.
But the risk of losing vision is so unacceptable that the market opportunity may remain unfulfilled until the product is approved for which the gains are more clearly worth the risks.
For investors.
It means finding a therapy, which is safer more effective or ideally both.
Overall I think this most recent chapter in the dry AMD A&D story highlights three things first it is provided prescriber level evidence of an enormous market opportunity.
Before the risk of vision loss due to vasculitis was disclosed the early sales of site full re were impressive and help to validate widely held commercial projections in dry AMD.
Second <unk>.
Despite the approvals of <unk> and now also is we've heard from prescribers and thought leaders that there continues to be a need for more effective agents to treat dry AMD.
These complement inhibitors appear to be temporary and incomplete solutions.
More exciting effective and infrequently dosed candidates are needed.
And if you're a regular on these calls you've heard me talk about the unprecedented clinical benefits, which were achieved by patients in our phase Iia trial, who received offers and sells across substantially all of the area of G E and with a single surgical administration.
As investors begin to look beyond the recent approval of first generation agents and until the pipeline of future dry AMD programs. We believe lineage is ideally positioned with a potential best in class and first in class product candidate with a proposed treatment profile, which in such patients doesn't merely slow.
Progression, but can in some cases stop or even reverse gea.
And do so with an increase in visual acuity, starting in weeks and lasting for years.
While every product has its benefits and risks we believe a commercially approved agent with a significantly larger clinical benefit with more easily tolerate a low frequency risk of adverse events.
The third highlight is that this makes us feel even stronger about our partnership with Roche and Genentech.
Roche has extensive clinical experience with complement inhibitors, but the development of those assets were discontinued.
Meanwhile, they added a completely new approach to treating gea via the licensing deal we signed for origin.
When it comes to treating G. A I believe Roche was right to move on from the complement pathway and embrace agents with larger potential clinical benefits.
Okay.
For reasons I've shared previously I'm, not yet able to provide details about the ongoing phase Iia trial of oxygen, but Roche continues to enroll patients that a reminder, the primary endpoint occurs just 90 days following each transplant.
Roche has nearly two quarters of enrollment experience behind them and additional sites are expected to come online. This year, which you can follow the clinical trials dot Gov.
I'm not able to guide to when top line data will be available, but we continue to be hopeful that the data will show that Roche is able to reproduce the clinical benefits, which lineage reported in the phase Iia trial.
And that they also will build upon our early success with additional insight into the safest and easiest way to deliver operation.
Moving next to obesity, one our spinal cord program as we expected. We recently received a response from FDA to our type B meeting submission.
The submission was conducted to discuss and clarify the use of our new spinal cord cell delivery device.
I am pleased to share with you today that based on the content of that response, we do not need to conduct additional back and forth discussions with the agency on this topic our.
Our next step will be to submit the IND amendment for this new system and through that amendment, we will be permitted to provide some final content and clarifications that were requested in the Fda's response.
The agency also said that the clinical design seems acceptable and tentatively agreed that no additional non clinical in vivo studies would be needed.
In light of this feedback on our proposed trial, we remain on track to submit the <unk> IND amendment in the fourth quarter.
Assuming no further comments arrived in the 30 days following that submission that will permit us to proudly bring <unk> back into clinical testing by initiating the dose study in sub acute and chronic patients.
Related to obesity, one a briefly want to provide a follow up on the first annual spinal cord injury, Investor Symposium, which we created and hosted in June .
The event brought together therapeutic area experts researchers corporate representatives individuals' would lift experienced caregivers advocacy organizations investors analysts and members of the public and the media and alongside presentations and panel discussions.
We heard from people, who have participated in Sci clinical trials and what they would like to see in future trials.
The response to this event has been far beyond our expectations and we already have begun thinking about how we can improve it next year, including by inviting representatives from regulatory agencies.
Moving next to back to we've recently received data on the eight patients with advanced non small cell lung cancer, who were enrolled in the U K based phase one trial conducted by cancer Research UK.
The most notable points from those data were that the vac to product candidate appeared to be well tolerated in all treated patients.
The adverse events were observed were modest and ones, which we expect from a therapy designed to generate a robust and durable immune response.
Five of eight patients demonstrated a best response of immune related stable disease, and three demonstrated immune related progressive disease.
Early spot assayed indicated that two patients had durable responses and two others had transient responses against segments of the telomerase tumor antigen, which had been loaded onto the allogeneic dendritic cells.
As a whole these data provide an important connection between the proposed mechanism and the clinical observations in this trial and while this was a small sample three of the eight patients all of whom had refractory disease reached the two year survival endpoint.
This was an important trial to assess the tolerability and mechanism of back too and the overall safety and efficacy data, which was collected affirms our belief in the potential for allogeneic cell therapy to address certain types of cancer.
We appreciate the patience and the team at cancer research UK for their efforts to complete this study.
In terms of our path forward, because many different antigens could be employed in our allogeneic dendritic cell system, we believe strategic alliances offer us the best way to advance the Vac platform and our BD team continues to be engaged in exploratory discussions for the development of back assets.
While there is no assurance that any partnerships, we're exploring will come to fruition. We know encouraging clinical results have recently been reported in the neo antigen vaccine space.
In addition to the BD talks we intend to continue monitoring this landscape to help inform our corporate strategy and determine the best development path for back to or any other bank platform programs, which we may pursue.
Lastly, we recently received information, which indicates that vac to development could be eligible for certain funding and have begun to evaluate that avenue as well as the others I just mentioned.
For A&P, one which is our cell transplant program for hearing loss.
Preclinical testing is ongoing through a collaboration with the University of Michigan.
Our initial objectives from this collaboration are to evaluate and craftsman of ourselves and certain anatomical destinations.
That's how long sales can survive after transplantation to those locations.
I don't wish to get ahead of myself on this today, but the initial findings from this study have been encouraging and we plan to provide an update on this program later this year.
As some of you may have seen just a few days ago. Another hearing loss company was acquired making that two early stage gene therapy hearing loss acquisitions in the past year.
We believe these recent acquisitions in the hearing loss space served to validate our decision to expand the lineage platform into hearing loss and will provide competitive competitors for the A&P one program.
And as I previously said.
I think cell therapy can have advantages over certain kinds of gene therapy, because replacing the entire cell means you don't have to select for patients who carry a specific genetic defect.
We think this offer cell therapy larger addressable markets, while matching the advantages of the one and done treatment schedule of gene therapy.
So to wrap up this part of the call I want to mention a few of the ways in which we will be working to create near term value for lineage shareholders.
I believe one of the questions for investors at the moment is whether Roche will independently reproduce our findings of improved retinal structure.
So we will be doing everything we can to help support their efforts to enroll and conduct that study.
In parallel we will be working closely with Roche and genentech employees to transfer our production process to them, which will enable them to manufacture <unk> in house, which continues to be part of the overall plan for <unk> development.
And thirdly, we expect some additional data updates from the phase <unk> study of opportune to be presented at medical meetings. This year, which is always helpful for increasing awareness of our program and to demonstrate our partners' commitment to it.
With respect to our pipeline programs. The most notable item will be getting the IND amendment for <unk>, one submitted before the end of the year.
We also expect to have updates from other areas of our business as we always aspire to reach newsworthy milestones from across our portfolio.
And while we do try to make a lot of progress each quarter and reach those milestones. We also try very hard to keep our spending under control.
So with that serving as a transition I will now hand, the call over to Jill for a discussion of our financials.
Thanks, Brian and good afternoon, everyone, beginning with our balance sheet I'm pleased to report that we have continued to be smart with our spending and are well capitalized to conduct the near term activities with which Brian just outlined.
Our reported cash cash equivalents and marketable securities as of June 32023 totaled $45 9 million, which is expected to support our current planned operations into the fourth quarter of 2024. Please note. This cash amount does not account for any of the Roche genentech milestone payments for any.
Business development, our grant revenues, which we may receive during the same period.
Next we will review our second quarter operating results.
Our revenue was generated primarily from licensing fees royalties collaboration revenues and research grants.
Total revenues were $3 2 million, a net decrease of $1 4 million as compared to approximately $4 6 million for the same period in 2022.
The decrease was primarily driven by lower collaboration and licensing revenue recognized from deferred revenues from the Roche agreement.
Operating expenses are comprised of research and development expenses and general and administrative expenses total.
Total operating expenses were $8 1 million a decrease of <unk> 5 million as compared to $8 6 million for the same period in 2022.
R&D expenses were $3 9 million, a net increase of <unk> 6 million as compared to $3 3 million for the same period in 2022.
And this increase was primarily driven by <unk> 4 million and higher operation program related expenses, and <unk> 3 million in non clinical related expenses to support the <unk> program.
G&A expenses were $4 2 million a net decrease of approximately $1 1 million compared to the $5 3 million for the same period. In 2022. This decrease was primarily driven by $5 million and lower litigation and legal expenses and overall reduction in cost incurred for services by third parties consulting.
Costs and stock based related compensation expenses.
Loss from operations was $5 million, an increase of $1 8 million as compared to $4 2 million for the same period in 2022.
Other income and expenses included other expenses of <unk> 2 million compared to other expenses of $2 5 million for the same period. In 2022. This change was primarily driven by exchange rate fluctuations related to our international subsidiaries fair market value changes in marketable equity securities and interest income from our marketed.
<unk> debt securities.
The net loss was $5 2 million or <unk> <unk> per share compared to a net loss of $6 8 million or <unk> <unk> per share for the same period in 2022.
Overall, we continue to maintain our same spending discipline as we have adhered to for years, and which has served us well in the past as the biotech markets continue to face uncertainty, we believe that maintaining disciplined with our spending will continue to allow us to maintain our plan to reach meaningful milestones.
Important progress and create value for shareholders from our investments in our programs.
Now, let me hand, the call back to Brian .
To wrap up we believe lineage is pioneering approach, which has large commercial opportunities, but few analogous competitors.
While stem cells were once thought to represent a powerful new kind of therapeutic in and of themselves. The reality may be that the true value from stem cells isn't using them as medicine, but using them as a starting material to generate other cell types, including cell types, which can and in certain cases already have shown the ability to <unk>.
<unk> clinical efficacy outcomes that do not occur by chance and vastly exceed the best available alternatives.
Most of the success stories to date in cell therapy can be found in oncology or transplant medicine, but data reported recently in non cancer indications such as type one diabetes and parkinson disease as well as our own achievements in dry AMD with <unk> suggest that advancements in the tools understanding and best.
<unk> flotation of differentiated cells in a replace and restore approach may be on the cusp of an exciting era.
I continue to believe lineage is making good decisions and the challenging biotech environment, we've been conservative and disciplined with our spending and we're advancing our programs in a responsible way.
Our collaboration with Genentech, and Roche is progressing extremely well and one of the things we will be particularly excited to work on this year, we'll be continuing to support them and the further clinical development of opportune.
As always we sincerely appreciate your support of the company as we look to position lineage to become the leader in cell therapy and cell transplant medicine.
And with that Monday, Jill Gary and I are ready to take analyst questions.
The floor is now open for your questions to ask a question at this time. Please press star one on your telephone keypad, if any point you'd like to withdraw from the queue. Please press star one again.
We will now take a moment to compile our roster.
Okay.
Our first question comes from the line of Jack Allen from Baird. Please go ahead.
Great. Thanks for taking the questions and congratulations to the team on all the progress made over the course of the quarter.
Maybe to start I know, Brian you talked a lot about the Gi opportunity with offer gen.
Could you maybe speak a little bit more finally about what you can do to help your partner enroll this phase Iia study as expeditiously as possible I know, it's hard to talk about timelines, but any specific activities you can do to help aid in their execution of this trial.
Okay.
It's limited when I'm able to share specifically, but what I can note is that to date Genentech has relied on the same clinical centers, which we had used in the phase Iia study. So there's certain familiarity relationships knowhow understanding.
That could be beneficial, but with respect to the specific activities that we perform.
I would characterize those as more contribute Terry.
Our contribution will in nature and not as much the frontline decision, making and as you already know Roche is paying for the clinical development of <unk> in this study and any future studies, which they conduct so we don't have any of the burden, but we actually look for opportunities to.
Beyond what is contractually expected of us.
In any creative way to try to be helpful. In the further <unk> of the origin program.
Great great. Thanks for that color and it's great to hear Youre, taking an active active.
<unk> as you can in helping congrats that asset forward.
Maybe obviously one very quickly that's great to hear that you've got in the feedback from the FDA and you're planning to implement the device with the IMD Amendment here I guess, maybe can you step back and talk a little bit about your longer term aspirations for obesity one.
Previously the my understanding that you're going to dose at a few patients on this new device and then maybe moving to a larger study can you talk about a little bit more about the.
Maybe.
Three to five year plan here.
The most important and outstanding question about RPC, one, which we want to answer and intend to answer is what is the magnitude of benefit which patients can enjoy if they receive the oligodendrocyte cells, which we manufacture.
In order to answer that question, we will need to conduct a larger and controlled study.
The study design has not been determined it is premature of us to determine it.
However, there are a couple of steps that we want to complete before we are able and prepared to conduct that larger study one of those you're well aware of which is deploying and demonstrating the utility of the new delivery system. It's a lot smaller many fewer components.
In part it's easier to administer and it has advantages that we think will be beneficial on safety and perhaps even advocacy.
The second thing that we need to do is to introduce the new cells, which we manufacture here at lineage as I believe you and others are aware I've I've talked extensively about the achievements of the manufacturing team that they have made on the manufacturer of OTC one in terms of the reproducibility the <unk>.
Gail the control and the purity.
When those two elements are complete then we would be able to.
Have the back and forth with FDA to design and propose a larger comparative study and so thats really where we want to go with this we would need to clear a couple of what I think are relatively straightforward hurdles and then when we get past those we want to conduct that larger study and answered that the critical question, which is.
Is this a therapy that can help individuals gain mobility after a spinal cord injury that they would not otherwise experience, but for the receipt of these cells to their spinal cord.
Great. Thank you so much for taking the questions and all the color and congratulations again on the progress.
Thank you Jack.
Okay.
Our next question comes from the line of Joe Panther Guinness from H C. Wainwright. Please go ahead.
Hey, everybody. Good afternoon, thanks for taking the questions. So Brian My first question I promise there is no pun intended but.
Do you envision or any potential complementarity between <unk> and I survey or a <unk>.
I've heard a lot of vision jokes in my five years here and there.
That is certainly one of them I'm.
I'm going to refer the question to Gary.
To talk about the compatibility and how our therapy could be used in addition to or instead of.
Our complement inhibitor.
Yes, so Joe I think obviously based on the initial launches I celebrate clearly theres a huge unmet need.
So the big claim space.
There is no reason that opportunity can be used in conjunction with a complement inhibitor that you approved agents.
Or allow a couple of minute ever to be used first and then follow up with our general vice versa.
There is a potential synergistic effect of the two products three drive secondly.
And that in effect that they both might benefit from co administration. So obviously, we will need to conduct those co administration study at some point, but we don't think it will impact.
If anything it will benefit both both.
Approaches to treating dry AMD.
No. That's helpful. Thank you and then wanted to switch over to the black.
Please me back.
Platform.
I guess, how should we view or what are you doing to percolate sort of the platform concept you know behind the scenes other than sort of having in bounds in marketing. It as a platform are you doing any particular, you know scientific work.
You know to be able to help marketed.
Do you have the potential to be able to hand sells out in an NDA.
Stay.
Situation, where a third party can sort of do their own experiments hassle, how is the platform being percolated.
The answer is that it is being viewed through a number of different prongs. So one of those would be thinking about working with the other party to use a different antigen or combination of antigens, which could be presented on the surface of the dendritic cell.
<unk>.
And you could imagine that that idea can range from <unk>.
Work that has been done at academic centers for more of the the Pan cancer antigen like Turkey, or you could imagine harnessing some of the discoveries that are coming out of the AI field and machine learning to rationally select.
The antigen that you might use either on an individual patient basis or again more collectively for antigens that could serve this purpose more broadly.
We have other prongs that we're considering which include manufacturing we think that there is still a lot of room to improve the production and drive down the costs of the dendritic cell system.
And we think that from a clinical perspective, theres, a prong that can be viewed with the dendritic cell as a tool because it essentially goes beyond the first step of the immune system, which is we are pre packaging the antigen into the dendritic cell so rather than using lipid nanoparticles or if you.
You are concerned that your antigen is not.
Tickling the immune system quite in the right way utilizing natures previously designed system for presenting those antigens could be beneficial it is extremely difficult to tackle all of those prongs simultaneously with all of their individual permutations, but when I say that the BD people.
<unk> have exploratory conversations these are the kinds of prongs that they are thinking about and talking about with various parties each of which in isolation could be beneficial the vac program and help elevate the visibility and importance of that program.
And which altogether it could provide us with some de risking by having multiple approaches.
Partly supported by partnership and then perhaps partly supported by lineage.
I appreciate the color Bryan Thanks, a lot.
Thank you Joe and thank you also for participating as a moderator at that spinal cord first annual spinal cord conference. We appreciate you and Jack and others.
Our next question comes from the line of Kristen <unk> from Cantor Fitzgerald. Please go ahead.
Hi, Good afternoon. This is Jason Bouvier on for Christian Thanks for taking our questions I have two questions.
Just going back to the phase Iia trial being managed by Genentech.
Do you have any information on the number of patients or subjects had been enrolled.
Sort of the rate of enrollment and how many are left and the second question is.
How are you thinking about prioritizing earlier stage programs and any potential projects better.
Not yet out in the pipeline.
We were not permitted to disclose or share that level of granularity with respect to enrollment.
And you and everyone. Unfortunately will need to just kind of make their own estimates and projections.
With respect to what I'll call the balance between our lead program and other programs.
We are trying to strike a balance within that balance which is to say that we are certainly planning for success. If one isn't planning for success. One presumably is planning for failure. So that that is pretty straightforward that we are looking to build a company here that in part will be elevated by the success the future success.
<unk> offer gen in dry AMD.
It is also abundantly clear that this environment is not one of the best that has ever been for the biotech industry. So we can't be irresponsible in how we invest in our pipeline program. So we have today multiple assets. They are staged with respect to where they are clinically or pre clinically.
How far they are along in development and that staging can be intentionally adjusted to reflect an appropriate level of financial commitment. So there are some areas that we think that the return on our investment and the value to our business is quite high and the Best example, I would point to there was.
Our decision to spend approximately $1 million to initiate the A&P, one program and to get that into preclinical testing with that relatively small amount of R&D spending and I shared previously some exciting early stage acquisitions that were done for what I believe were.
<unk> single asset hearing loss program. So we use that sort of discipline and that critical ROI evaluation as a way to manage our spending across the platform and we're always asking ourselves if it's something that we should look at grant funding is it something.
We should be thinking about partnering or is it something we just want to carefully and stepwise move forward all the while while we wait with others for what we hope will be exciting future data from the opportune program.
Great. Thanks, a lot for that answer.
Thank you Jason.
Our next question comes from line of May Yang Man Tammy from B Riley Securities. Please go ahead.
Hey, good Madison the onshore.
Thanks for taking my call and congrats on the progress.
Just a quick question.
Once you submit your R&D amendment and for Q.
And the 30 days.
Yes.
How long will it be.
Before you actually.
Then move into the clinic, there and then.
Secondly.
It sounds like you guys will no longer be responsible for production.
Production as Youre transferring.
Capacity to your partners.
Regarding.
Upper Jim.
And I'm, just wondering how long that transfer process.
What take thanks.
Thank you for the questions Madison with respect to OTC, one we always look to minimize the amount of time between when we are cleared to open a site in getting through the contracting process and actually opening a site a lot of the time is regrettably on the <unk>.
<unk> side, the legal and the contracting side I think generally speaking sponsors are good about turning around their documentation, but each site is its own animal and poses its own challenges. So we do as much as we can to start. These trials quickly I have no reason to think that we would.
Benefit in any way by having a delay so while we know that this will be a study at a small number of sites. We are going to do everything we can to be able to start very quickly.
After the 38 clearing period has occurred but there are some centers out there that won't engage fully until you have an IND open and thats very frustrating, but many others allow you to parallel paths with an expectation of when you'll be ready. So it's a blend when you have a multicenter trial youre going to find different.
Hurdles, but for US the best answer is for us to pre plan and be ready to hit the ground running to the extent possible with redundancy in parallel paths work being done in advance of that 30 day period.
With respect to the second question.
I have no idea how long it takes to transfer a cell therapy production process to a big pharma partner, because we've never done it before.
However, we have abundant faith in our manufacturing production team and their ability to train and teach skilled collaborators to achieve the work as probably everyone on the call knows manufacturing sales consistently and <unk>.
<unk> and controlling the process is very difficult and there have been many failures over the years in laboratories around the world, but we have seen many examples of extraordinary success by our in house manufacturing team.
One of those examples I spoke to with A&P, one being able to create a differentiation protocol very quickly. Another example would be the enormous scale of production, which they achieved with OTC won a third example would be how well they were able to improve the purity and the scale of the <unk> one program.
Excuse me I misspoke I meant opportunity previously and <unk> in that case. So those are three examples from three different programs, which illustrate the capabilities of the manufacturing team and I have no reason to think that Roche and Genentech has a paucity of resources or capabilities quite the opposite I expected.
They have abundant capabilities in this regard so while it's not a straightforward and simple task you can mail it in to somebody you don't just send them a document you really need to be sitting next to them going through repeating it several times I really am unable to tell you when I think that process would be completed.
But we will pay great attention to it and work very closely with our partner to give them the best possible chance of success.
Got it I appreciate the color.
Thank you Madison.
Our next question comes from the line of Michael are keen which for Maxim Group. Please go ahead.
Hey, Brian . Thank you for taking my questions and congrats on the progress this quarter.
Thank you Michael.
Okay.
I think to start off I'd, just like to ask a little bit about them back to program.
<unk>.
To see if you could kind of prime us for.
Full data from that study with the additional analyses ongoing.
At your UK partner.
Which of those which of those particular analyses do you consider to be the most important or.
Both validating the platform as a whole as well as the vac to.
Program itself.
Thank you Michael for the question I will refer to Dr. <unk> to respond to you.
So they're ongoing investigations or looking at biopsies from the tumors to look for tumor infiltrating lymphocytes, we're keenly interested in those data.
Looking at skin biopsies at the site of administration to see what type of cell influx occurred.
First semesters, all the way up to six administrations.
W are interested that profile changes over time.
Additionally, we are looking at.
Current Elisa panels look to see if we have a th one rotation to cytokine profile shift.
Wasn't there a baseline and all of this would be indicative of potential immune response to the antigens of interest and May show that there was at least some anti tumor effect as well.
Certainly interested in looking at so those data are being analyzed and we await the reports from cancer Research UK.
Alright, thank you for that.
And then I would just like to ask about.
Specifically in the auditory disease space, you have gene therapy.
Going in.
There are some pretty significant.
Significant attention.
Do you see having additional one and done therapies out there paving the way as making it more attractive for development for A&P. One and then do you have any additional color on specific indications you might look to go into what this gets towards the clinic.
Thanks, Michael I have a two part answer.
<unk>.
The refinement of the indication and the intended patient population will be driven by the data, which we collect initially pre clinically and then evolving into the clinic.
Difficult for us with such a.
New approach to be definitive to be credibly definitive about how we see the best use of this intervention.
And we're quite aware that there are many types of hearing impairment some of them chemical some of them physical some of the more reflecting aging in.
<unk>.
Degenerative.
<unk>.
The.
The.
Notable aspect of other approaches and let's call. It success that we're seeing in approaches for hearing loss I think is very beneficial to our earlier program because it's beginning to establish the existence and refine some of those questions.
About what are the right clinical endpoints to us what are the economics of our program look like because ultimately if we're going to get credit for what today is a preclinical program that.
It's going to come through some sort of evaluation exercise, which is going to need to have some sort of an addressable market, which is going to need to have some refined patient population. So while it is exciting to go into a new area with an incredible paucity or dearth of other.
<unk> threats.
The trade off for that is we don't have a mature and established commercial.
A market that we can point to with certainty and say, we know exactly what patients we're going after so in light of the options of a mature and crowded space compared to this new area I'm delighted to be going into this new area, because we can partly define it for ourselves rather than.
The forced into following others, but I think theres a lot to learn in the hearing loss space from some of the early forays that we're seeing in particular in gene therapy and as I described earlier, even if a gene therapy is able to wonderfully address one specific.
Deficiency in the genome through some sort of repair or replacement of that genetic information that is going to lead. Many other kinds of hearing loss available to an approach that is replacing the entire genome through the transplant of a cell.
Thank you I appreciate your insight.
Thank you for the question.
Our next question comes from the line of Jack Allen from Baird. Please go ahead.
Great. Thanks, again for taking the questions and a follow up here.
Jogger thought as one of my peers was asking a question about <unk>.
Particularly on enrollment of the offer Gen Phase Iia study.
You mentioned in response to my question do you have a lot of experience with the sites that are being utilized by.
The collaborator I guess any historical context, you could speak to as it relates to the ability of those sites to enroll patients in your phase II <unk> study.
One of the things that I like to highlight.
Some time ago was that the early forays into this study.
Enrolled very slowly.
In part because there were very few sites open and in part because these were the very first an incredibly courageous patients to receive this therapy.
By the end of the study this team at lineage had had.
Taking control of enrollment we had opened additional sites and we were able to enroll I think the last four patients were all within a very crowded period of maybe six or eight weeks.
That is reflecting tremendous variability of pace of enrollment which occurs not just at the study level, but also at the individual site level.
So I think there are some factors that go in our favor in particular I think the conversation between a prospective patient and.
In the center and the surgeon is different today, because youre talking about a program that has pharma credibility and 24 well.
Our highly accessible sets of data to be able to refer to rather than being the first of its kind.
But at the same time this is an optimization study.
And I believe that Roche would like to minimize the number of variables to the extent it's possible. So that they can gain as much information as possible from this study and in doing so it's not surprising to us that they have elected to start out with just the sites that we had used previously.
I expect that that will expand over time I don't have specific information about that but I think it's a general sentiment that I am encouraged that there is more.
Horsepower and there is more experienced available for this trial being run by Roche and Genentech than what was done in the hands of lineage. Although obviously, we were quite delighted with the finding some success that we had in our phase one.
Great. That's great color. Thank you again for taking the follow up.
I appreciate that Jack Thank you.
Our next question comes from the line of Joe Pat <unk> from H C. Wainwright. Please go ahead.
Hey, guys. Thanks for taking the follow up as well so when youre looking at the A&P One program I wanted to dive in a little bit here.
Can you describe sort of a little more of the models you are looking at right now and what's planned and I sort of want to correlate that with the Takeouts that you referred to of a COO Olson decibel now they are using gene therapy as you alluded to there.
They were very focused on targeting of their avs and in very specific mutations, which you don't necessarily need to do so they were looking at animal models in particular like auto Ferland models or what have you what kind of models would you.
Look to do to be able to address a broader hearing loss concept. Thanks.
Thank you Joe.
Great question I understand the question, we do plan to provide an update on A&P. One later this year and it probably the question is even more suitable at that time because at this point our focus is and our emphasis is primarily on the delivery of ourself.
<unk> and the durability of ourselves as everyone knows if your if your sales aren't present, theyre not going to be functional and thats, where we need to go. So we're not at the level, where we are designing our own functional tests or making comparisons to studies that have been done.
One in the space, we really are just focused on stepwise progress here, which means ensuring that we can get the cells, where we want them to go and ensuring that they are still present after a clinically relevant amount of time and using the models that are quite conventional in the space.
Which are various forms of rodent models.
But I think when we do a more fulsome update it'll it'll be easier to be able to begin to draw parallels across what we're doing and what some of the other folks are doing but I do think that.
To the extent, you're thinking about gene therapy needing to repair genetic defects and models having to have a specific defect. So that gene therapy companies can show that they can fix that DNA.
And then have a clinical outcome from that.
Our approach would be that we do not need to have such a difficulty that we can use what I would characterize as crude or forms of deafness, because we arent trapped by or forced into the narrow segment of a single gene being.
<unk> for the absence of effective hearing.
That that makes great sense I appreciate that and I'm just going to just focus on one of your D word's and Thats the delivery.
Obviously, you need the experiments to look at durability. So.
AAV is much smaller than cells and you're you're dealing with a very limited volume environment are you looking at what's essentially been relatively standardized surgical techniques. Now I know this is pretty forward looking or is there something more unique to cells that we'd have to consider or is it too early to really.
Go down that alley.
Youre asking an excellent question because of course cells are much larger than viral vectors.
And one place, where we think that Thats extremely interesting is in the setting of the <unk>, we know that.
Viral vectors can travel across the optic chiasm and appear on the other contralateral untreated eyes, so that makes control arms in that space a bit challenging.
With respect to delivery to the ear.
It is extraordinary.
To learn what is capable with teeny tiny needles and a steady hand.
So we do have some challenges in the delivery as we do have challenges with the delivery to the spinal cord or to the eye or to everywhere because as you correctly note if the stuff doesn't get where it needs to be it's not going to work. So I think what we will do is we'll share some of our find.
<unk> and some of our methods in particular at that update later this year, but I think that generally speaking we have not I can.
I can say that we have not needed to invent anything new in order to perform these preclinical studies if that answer is at least helpful. In the interim.
It certainly does Brian Thanks, a lot I can't wait to hear about it.
Thank you Jeff.
Yes.
That.
Okay.
Good evening.
The analyst call I would now like to turn the call over to Brian Culley for closing remarks.
Just like to say thank you everyone. It's exciting to have such great interest and all the things that we're doing and we will continue to work hard to make this company interesting and exciting and successful. Thank you and have a great afternoon.
Thank you ladies and gentlemen, this does conclude today's call. Thank you for your participation you may now disconnect.
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