Mesoblast Ltd
ASX:MSB
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Mesoblast Ltd
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Thank you all for standing by. Hello and welcome to the Mesoblast Financial Results for the period ended March 31, 2023. An announcement and presentation have been lodged with the ASX and will also be available on the home and investor pages at www.mesoblast.com. At this time all participants are in a listen-only mode. Later we will conduct a question-and-answer session and instructions will follow at that time. As a reminder, this conference is being recorded. Before we begin, let me remind you that during today’s conference call, the company will be making forward-looking statements that represent the company’s intentions, expectations or beliefs concerning future events.
These forward-looking statements are qualified by important factors set forth in today’s announcement and the company’s filing with the SEC, which could cause actual results to differ materially from those in such forward-looking statements. In addition, any forward-looking statements represent the company’s views only as of the date of this webcast and should not be relied upon as representing the company’s views of any subsequent dates. The company specifically disclaims any obligations to update such statements.
With that, I would now like to turn the call over to Dr. Silviu Itescu, Chief Executive Officer of Mesoblast. Please go ahead.
Thank you, operator. Good afternoon, good morning to everybody on the call to our operational highlights and financial results for the quarter ended March 31, 2023. If we could, go to Slide 4 please. On the call with me today is our Chief Medical Officer, Dr. Eric Rose; and our Interim Chief Financial Officer, Andrew Chaponnel. Slide 4 shows a snapshot of the investment highlights. We have a novel allogeneic cell therapy platform with two lead candidates both being platforms for multiple products and indications. Remestemcel-L, rexlemestrocel-L, each have got their own specific indications and I’ll be talking in detail in this presentation on both of those platforms. Financially, we have substantial cash on hand and there’ll be a detailed presentation by Andrew Chaponnel on our finances for the quarter.
Next slide please, Slide 5. This slide is a summary of our late-stage clinical pipeline based on our mesenchymal stromal cell platforms. As you can see, remestemcel-L for pediatric steroid refractory GVHD graft-versus-host disease is the most advanced, and it’s currently before the FDA with the review of the BLA with a PDUFA goal date of August 2, 2023. The manufacturing inspection has been conducted and I’ll talk more about that shortly. Remestemcel-L is also being developed for adult steroid refractory graft-versus-host disease, acute respiratory distress syndrome, and inflammatory bowel disease. Rexlemestrocel-L second generation stromal cell platform is being developed for chronic inflammatory low back pain, discogenic low back pain and rexlemestrocel-L is also being developed for inflammatory heart failure. And for both these indications, the FDA has granted Regenerative Medicine Advanced Therapy designations.
We can now go to the next slide and I’ll hand over to Andrew to talk about our financial results, please.
Thanks, Silviu. If you could, please turn to the financial highlights for the third quarter of FY 2023 on Slide 7. For the quarter, revenue from royalties on the sales of TEMCELL in Japan by our licensee were $1.8 million. On a constant currency basis, royalties grew 4% in the quarter to $2 million. For the 12-months we recognized revenue of $7.6 million from royalties on product sales. Net cash usage for operating activities in the third quarter was $16.2 million; this represented a 4% increase on the third quarter of FY 2022, and a 34% reduction of $8.3 million on the third quarter of FY 2021.
In April, we successfully completed global private placement of $40.0 million through existing major U.S., UK, and Australian shareholders. As of March 31, 2023, cash-on-hand was $48.8 million. We have a pro-forma cash of $88.8 million after adjusting for proceeds of $40 million raised in the April private placement. We also have up to an additional $40 million available to be drawn down from existing financing facilities subject to achieving certain milestones.
Turning to Slide 8, you’ll see we are reporting an improved loss before tax for the quarter. Revenue is predominately from royalties on sales of TEMCELL in Japan and despite an increase in underlying sales volume of 4% in the third quarter of FY 2023 compared to the third quarter of FY 2022, reported revenue decreased slightly given that Japanese yen has depreciated against the U.S. dollar. Our R&D expenditure was reduced by 14% for the quarter. Our R&D spend in the quarter was primarily to support the remestemcel-L BLA re-submission and preparation for pivotal studies of rexlemestrocel-L. We continued our investment in manufacturing activities to support the potential launch of remestemcel-L for the treatment of steroid refractory acute GVHD. On FDA approval, $31 million of remestemcel-L relaunch inventory will be recognized on the balance sheet. Finance costs for the quarter includes $3.8 million of non-cash expenditure comprising accruing interest and borrowing costs.
Now, hand the call back to Silviu for the end of the presentation.
Thanks, Andrew. Slide 10, please. This slide summarizes the mechanistic process that this acute graft versus host disease, a fatal complication of an allogeneic bone marrow transplant, essentially following chemotherapy, there is extensive tissue damage in the patient who subsequently receives an unrelated donor bone marrow transplant. The transplant then attacks the body through defined pathways that are driven by T cells. T cells are activated, they’re the orchestrator of multiple cytokines produced by other cell types resulting in a cytokine storm, which is destructive for the gut, the liver, the skin, and other organs. And in those patients who have the most severe organ disease, mortality approaches 90%.
If we could go to Slide 11, please. So the market opportunity for Mesoblast is large, more than 30,000 allogeneic bone marrow transplants are performed globally. Of these, about 20% of pediatric, we are targeting the most severe cases, we initially targeting pediatric GVHD and intent to target the more severe adult cases as well.
Next slide, please. Slide 12 summarizes the survival data across three different studies involving more than 300 children treated with remestemcel-L. These studies were designed to demonstrate whether remestemcel-L provided an early survival benefit. Three studies outlined here include a randomized controlled subset of 27 children within an earlier study called Protocol 280. The second study was a pivotal Phase 3 trial called Study 001, and the third study was an expanded access protocol of 241 children who received remestemcel-L having failed other available therapies.
What you can see here is that in each of the three studies there is a substantial early survival benefit from 66% in the sickest children were remestemcel-L was used as salvage therapy to 79% in the randomized controlled subgroup study. And the comparators for each of these outcomes showed substantially lower survival outcomes at day 100 from 54% to 57%. In the EAP 275 study, a subset analysis comparing outcomes in Grade D disease, the most severe form of the disease against survival in the database at the International Bone Marrow Transplant Registry showed a significant survival benefit of 51% in those who received remestemcel-L versus just 31% in those who received best available therapy.
So across all of these studies, there’s evidence of early day 100 survival benefit using remestemcel-L. If we go to the next slide, in newer data, that’s now before the FDA analysis of survival through day 100 through day 180, six months in the pivotal Phase 3 trial in patients with the highest risk for mortality. Those with biomarkers, the MAP biomarkers scores above 0.29 demonstrated a significant survival benefit compared to a propensity matched control cohort, the so-called magic cohort. And you can see that in the control children treated with best available therapy, only 10% were alive beyond three months compared to 67% alive in those who received remestemcel-L. Next slide, please.
Slide 14, so what is the status of our BLA for remestemcel-L in pediatric patients with steroid refractory acute GVHD. The new data under reviewed by the FDA are outlined on this slide. The BLA was resubmitted in January 30. The BLA file was considered by the FDA to be a complete response, was accepted for review, and we were given a PDUFA goal date of August 2, 2023. The new data under review shows durable long-term survival of patients in the Phase 3 trial, increased survival in high-risk patients compared with propensity matched controls and a positive correlation between in vitro potency assay and survival. Additional data that the FDA is reviewing shows that the valid data potency assay has low variability and can adequately demonstrate manufactured consistency and reproducibility. Next slide, please.
In addition today, we announced that the manufacturing inspection of the remestemcel-L process has been conducted by the FDA. The FDA inspection did not result in the issuance of any Form 483. Form 483 is always provided at the conclusion of an inspection if investigators have observed any conditions that in their judgment may constitute violations of the FDA Cosmetic Act and related Acts.
So we were very pleased that no issuance of a Form 483 was provided. The Establishment Inspection Report or the EIR is expected to be issued by the FDA in the coming weeks, which will provide a detailed summary and a final assessment of the inspection.
Next slide, please, Slide 16. Mesoblast provided new results from a full year observational survival study performed by the Center for International Blood and Bone Marrow Transplant Research, CIBMTR on 51 valuable patients with acute GVHD who are enrolled in our Phase 3 clinical trial remestemcel-L.
These data provide a cornerstone of our BLA resubmission. Overall survival in the remestemcel-L cohort was 63% of one year, 51% of two years, and remained roughly at that level through at least four years and beyond. In other words, approximately 50% of children with this very fatal disease of whom almost 90% were Grade C/D disease are alive not just at two years, but for as long as the follow-up has allowed us to evaluate the outcomes.
These data provide assurance that the short-term responses and early survival through 180 days previously shown to the FDA in the BLA submitted previously are unlikely to have risen by chance. And these long-term survival outcomes really are a cornerstone of our BLA resubmission.
Next slide, Slide 17. This slide shows a comparison between two-year outcomes on the right with remestemcel-L from our Phase 3 trial, 51% survival against the largest pediatric cohort study published to date which is the MacMillan study on the left from University of Minnesota showing just 35% survival in children with steroid refractory disease. And it’s important to point out that in the MacMillan study on the left children with treated with best available therapy only 22% had Grade 3/4 disease, whereas 89% of our study, on the right had Grade C/D disease at same level of severity. So very different patient populations much more severe in the remestemcel-L treated than yet, survival for 24 months substantially improved.
We can go to the next slide, please, Slide 18. This summarizes again the long-term survival data in the column with dark blue over four years against four studies that have been recently published between 2020 and 2022. The MacMillan study I’ve already described, but in addition, there are four other studies that relate to adults with steroid refractory acute graft versus host disease. And whilst, they all have lower levels of severity in terms of patients with Grade 3/4 disease in comparison to our Phase 3 trial, the relative survival in grade – the relative two-year survival in each of these studies is of the order of between 25% and 38%, comparing to 51% of two-year outcomes in our Phase 3 trial.
So we believe that the long-term survival that we’re seeing in GVHD001 treated patients with remestemcel-L provides a durable long-term outcome that is not comparable with any of the recently performed studies including those with ruxolitinib.
Next slide, please, Slide 19. And moving from new clinical data to new data around potency, which is part of the BLA submission, it’s important to note that the primary mechanism by which GVHD progresses is due to alloreactive T-cell activation, T-cell proliferation, and orchestrations I mentioned earlier of inflammatory cytokine production by these activated T-cells. In vitro assay measuring inhibition of T-cell activation was established during development of remestemcel-L prior to the Phase 3 trial as a potential measure of product potency.
This assay was used to measure the ability of individual remestemcel-L lots to inhibit T-cell activation prior to their use in both the expanded access Protocol 275 and prior to the Phase 3 trial GVHD001. We, therefore, evaluated and established correlations between survival outcomes in these two pediatric studies with potency of the lots that the children received as measured by inhibition of T-cell activation. And these correlative analyses have been presented to the FDA.
Slide 20. Next slide is representation of some of these potency data, which were presented earlier this year at the tandem meetings of the Bone Marrow Transplant Society. Firstly, as you can be seen in the Kaplan-Meier slide on the right, there was an association between higher levels of inhibition of T-cell activation byproduct locks received in day 180 survival, such that those patients who got product above the median had 85% survival versus those who got product below the median. This establishes that the greater the potency of products received in the Phase 3, the greater the survival outcome. In further subgroup analyses, looking at patients by higher risk severity including the Minnesota high risk category, the magic algorithm score above 0.29 and grade D disease in all of these subgroup analyses product lots with potency above the median were associated with higher survival than product lots with potency below the median.
I think it’s important, however, to also note that particularly with grade D disease, even products with lower levels of potency gave survival outcomes of 50%, which is substantially higher than the expected survival for grade D disease in the CIBMTR registry using best available therapy where that survival outcome is only 30%. So even a lower potency product have substantially high survival than best available therapy, and of course, the relationship between potency and survival is now established through the Phase 3 trial.
If we go to Slide 21, the next slide, we then reevaluated survival outcomes in the EAP study, one single study by the presence of our newly established improved manufacturing product that is now called Ryoncil. With the product produced prior to 2008 that was called Prochymal, that product had a lower potency as measured by our current potency assay than the new product Ryoncil.
So what you see here on this slide, on the left hand side, is survival for a 100 days in those children who had failed all other therapies in our EAP program by Prochymal versus Ryoncil treatment. And as you can see, a significantly higher survival, 74% was achieved with Ryoncil than was achieved with Prochymal 56%. Even more important, if you can look on the figure on the right, this survival benefit with the improved manufacture product Ryoncil is even more significant in those patients at highest risk of mortality those with grade D disease. And 106 patients with grade D disease, Ryoncil achieved a 72% survival, whereas Prochymal, the lower potency product achieved only 44% survival.
So these studies, these analyses within a single protocol, the expanded access protocol is able to establish not only that we have a potency assay that predicts survival at the level mechanistically of T-cell activation, the primary mechanism of action, the GVHD, but it is able to also delineate higher potency products from lower potency products and their relationship to improve survival.
Next slide, please, Slide 22. So those – having summarized those data, the new data that formed part of the BLA. Let’s talk now about our go-to-market strategy with the assumption hopefully that PDUFA after August is gives us a positive outcome. The pre-launch activities that are active and ongoing are engagement of the highest transplant volume centers with experience using Ryoncil.
Non-promotional activities include profiling of the centers, education on disease awareness, unmet needs, and support for payer engagement. We are currently hiring select positions to build out a small commercial team. The key activities of this team are market access to initiate payer outreach, medical, providing education to payers, corporate leadership initiating engagement with the top 15 centers and regional sales directors leading center profiling. Importantly, manufacturing preparation has been ongoing throughout with $31 million of remestemcel-L pre-launch inventory in hand.
Slide 23. The post-approval launch activities are intended to be a staged approach initially targeting the highest transplant volume centers. Those centers are those not only with highest volume, but also with experience with our product. The plan is to build out an efficient targeted sales force with understanding that 15 – or the 15 highest volume transplant centers account for about 50% of all patients. The key activities post-approval will be to initiate commercial onboarding and logistics, MSLs engaging the centers around medical and scientific needs, logistical and reimbursement support offered as needed and center certification on a center by center basis for remestemcel-L administration.
Having now covered where we are with remestemcel-L, let’s move to Rexlemestrocel-L, our next-generation product for chronic low back pain and for inflammatory heart disease. Slide 25. We’ve said previously, of course, that discogenic back pain impacts large numbers of patients both in the U.S. and in the EU. In the U.S. alone, it impacts 7 million patients in each of the U.S. and EU5, and that’s our target market, very well-defined patients who have underlying degenerative disc disease as the cause of their chronic pain. It’s important that 50% of opioid prescriptions are for chronic lower back pain due to this condition. So we expect that if we have an successful treatment here, we might have an important impact on the opioid epidemic.
Let’s go to Slide 26. The summary of the program is outlined on this slide. We’ve gained alignment with the FDA on the appropriate pivotal Phase 3 study, which seeks to replicate the significant reduction in pain that was seen as early as 12 months and through up to 36 months in our first Phase 3 trial. FDA has agreed with our plans for pain reduction through 12 months as the primary endpoint of the pivotal trial and with functional improvement and reduction in opioid use as the key secondary endpoints.
The planned Phase 3 program will include additional 20% of patients recruited across the EU to support regulatory submissions simultaneously or in close sequence to both FDA and EMA. We have an RMAT designation for discogenic back pain received from the FDA earlier this year in February, and we expect to commence patient enrollment in the third quarter of this year.
Next slide, please 27. The RMAT designation that we received from FDA provides all the benefits of both breakthrough and fast track designations, including a rolling review and eligibility for priority review on filing of a BLA. The objective of the pivotal trial is to replicate as closely as possible the data that we obtained from the first Phase 3 trial, which showed that a single injection of rexlemestrocel-L into the lumbar disc resulted in significant reduction in pain relative to saline controls as early as 12 months throughout 24 months and throughout 36 months in a 404 patients study.
The pain reduction through 36 months was also seen in a subset of patients who were using opioids at baseline. They were not insignificant, 168 patients. In this group there was substantially greater reduction at all-time points in pain compared with saline controls. And very importantly, at the end of the 36 month of follow-up, 28% of patients who had received rexlemestrocel-L together with its carrier HA were not taking opioids compared with only 8% of saline treated controls. This is a very important observation that we think is critical to evaluate again, prospectively in the pivotal trial.
Slide 28, is a snapshot of the reduction in pain that we’re seeing in the first study. And it was maximal in those patients who had pain with duration below the median for the whole trial, which was approximately 68 months. So therefore, this group of patients is likely to have had the highest levels of inflammation and so the target in the pivotal trial will be this group of patients with pain for up to five years as the objective is to address as early as possible this very large unmet medical need where we think that likelihood of treatment response is greatest.
Finally, let me move to the last couple of slides that address our cardiac program, inflammatory heart disease. Again, cardiovascular disease remains a leading cause of death in the U.S., particularly those patients with the more advanced forms of heart failure, now and heart damage who finally progress either to mortality or an LVAD or a transplant.
We have an existing, if we’ve got a Slide 31 please, we have an existing regenerative medicine advanced therapy designation to cover the use of our cells in the most severe patients, those who have an LVAD in place. But we really believe that there’s a continuum of disease that rexlemestrocel-L is likely to be beneficial in, and that the continuum is from patients in Class II, Class III and Class IV defined by very high levels of biomarkers of severity such as NTproBNP and markers of inflammation such as CRP and as well as those patients who then finally move into end-stage disease who have an LVAD in place. That’s our target market.
Slide 32, is a summary of new data that were published this quarter in the Premier Journal, Cardiovascular Journal, Journal of American College of Cardiology data from the 537 patient Phase 3 trial called DREAM Heart Failure, which followed severely advanced patients with heart failure for up to 30 months of follow up. And the key outcomes that were published in this important publication was that, left ventricular ejection fraction was significantly improved from baseline through 12 months. In those patients, particularly in those with evidence of inflammation, the major complications of heart attacks or stroke were reduced by 57% in all treated patients, and by 75% in those with inflammation. And the so-called three point MACE Major Adverse Cardiac Event rate in patients, which includes the endpoint of cardiovascular death reduction was reduced in those with inflammation by 37%. These results were spectacular, and we’re very excited about these results and the implication for patients with severe disease.
If we go to Slide 33, the overall totality of data, 159 patients in LVAD study number two and 537 patients in DREAM Heart Failure are now being evaluated in totality for a continuum of mechanistic data that may allow us to structure a pivotal trial as we talk to the FDA about pathways towards approval.
And on that note, I think I’ll stop and open this to questions, please.
Thank you. [Operator Instructions] Your first question comes from Edward Tenthoff from Piper Sandler. Please go ahead.
Great. Thank you guys, and thanks for the detail on the presentation. So my friend Mike, was wondering what still has to be done for the ahead of the NDA? Are there any remaining pieces that need to be filed or any interactions with the FDA or at this point, are we really just waiting for this to do today? Thanks.
Well, having completed the manufacturing inspection which was the biggest item still outstanding, we continue to interact with FDA on a variety of questions that backwards and forwards in regard to the review. But other than that, really the major item that remains outstanding is a discussion around the specifics of the label. And that’s really – that still remains ahead of us and we’re very excited as we proceed towards the PDUFA date.
Absolutely. Well, excited for upcoming approval. And then when it comes to lower back pain, what are the steps in order to initiate that base rate? Is that such an exciting opportunity today?
So we have alignment with the FDA on the protocol itself. We’ve already manufactured the product for the trial. We will be having a discussion with the FDA under the RMAT designation to ensure that we have our approach aligned in terms of manufacturing potency and the like. Those we will be proceeding in parallel with commencement of the study. We expect to initiate enrollment across about 40 sites in the U.S. in the third quarter, calendar quarter. So we’re on track to get things going.
Awesome. Great news. Thanks, Silviu.
Thank you.
Thank you. Your next question comes from Louise Chen from Cantor. Please go ahead.
Hello, everyone. This is Carvey [ph] on for Louise Chen from Cantor. Thank you for taking our questions. First, your PDUFA date is fast approaching. If approved, how quickly can you commercialize the product? And secondly, how are you thinking about OpEx for the rest of 2023 and 2024 fiscal years? Thank you.
We have the commercial team that is currently in place and building. It will not need a very large footprint. We expect to have about 15 or so sales force in place at the time of launch. The launch is going to be a targeted launch initially focusing on the highest volume transplant centers, but we should be ready to launch as soon as we’re approved. We have already got inventory in place. The discussions are ongoing now with payers. Reimbursement will take some time, of course. But we should be in a position to initiate launch as soon as we’re approved, although the pace of the launch is going to be staged.
In terms of CapEx, I think you asked or OpEx rather. We don’t see in the short-term changes in quarterly spend. Other than as we have product approved, we will be seeking – we will be looking towards label extension, particularly in adult GVHD as the first cab off the rank. And so how we fund that program is really a flexible possibility. We have access to additional capital throughout financial structures that are currently in place through debt. We have – are in discussions with various strategic financial partners around royalty based structures, et cetera. But in general, I think our plan at the moment is to maintain the current OpEx spent on a quarterly basis.
Got it. Thank you. Looking forward to the PDUFA date. Congrats on progress. Thank you.
Thank you. [Operator Instructions] Your next question comes from Sami Corwin from William Blair. Please go ahead.
Hi there. Thanks for taking my questions. Hi, thanks for taking my questions. I was curious if there are any other possible alternatives in terms of the outcome for the EIR other than the FDA, just kind of giving you guys a thumbs up. And then with regards to the study in steroid-refractory aGVHD in adults, how exactly could that trial differ from study 001 and study 280 in terms of design and potential endpoints? Thank you.
Okay, thank you. So the first question was about the expected EIR in the next couple of weeks on the manufacturing inspection. Look, we – it’s hard to anticipate what the observations and the final determinations of the FDA on manufacturing inspection. But I will say that a 483 Form can only be provided at the end of the inspection when the FDA and the facility, and the company are face-to-face. And the 483 Form was not provided, so that cannot be provided later. So we’re very pleased by that. In other words there are no – there are no official outcomes that would’ve been required for improvements under 483 Form. There may be other small issues that that come up in the EIR that’s the final document, but the kind of major concerns that that are in 483 will not be there.
With respect to an adult program, I might just ask Eric, Eric, would you like to comment on how you see an adult program going forward?
Can you hear me?
Yes, we can.
Good. We have had 10th interest in our product on the part of adults oncologists that face the same problem as the pediatric oncologist. Whether or not it will – what type of trial we’re discussing with members of the CIBMTR now there is a clinical trials group that is supported by the NIH that we’re in discussion with about doing a trial on adults that presumably will extension the label to that age group as well, but the design of such a trial is still outstanding.
Yes. And I think we have flexibility in thinking about the degree of severity in the adults whether we use a randomized control design or whether we use a propensity matched control design. I think all of those are on the table, and they will form part of our ongoing discussions with the FDA.
Great. And then just one more if I can. Considering you plan on running two Phase 3 trials and potentially launching the aGVHD product. Do you think you have enough cash on hand to accomplish all near-term milestones? Thank you.
Yes. As I said earlier, there are multiple alternative ways by which we may fund these various trials. And they include collaborations as Eric just mentioned with academic groups like the – like NIH or registries like CIBMTR, for example, and that that would have a substantial impact on our resource allocation. Secondly, there are financial groups that – that we are in discussions with who are interested in potentially royalty based structures that would again, defer our spend requirements. And thirdly, we have access to up to an additional $40 million in pre-arranged debt structures with our existing facilities that become available post FDA approval, should we want to access them.
Great. Thank you and congrats on the progress.
Thank you.
Thank you. [Operator Instructions] Your next question comes from John Hester from Bell Potter. Please go ahead.
Yes. Good morning, Silviu. Just want to take you back to Slide 21 on the right hand side of that chart, which talks about the Grade D banded access program a 100 survival rate and in the Ryoncil line that suggested that the survival rate there was 72%, and that’s in Grade D patients, who are the sickest of the sick. So just confirm for me, is that Ryoncil product, that’s – that was the product that was used, in your clinical trial, correct. So what is…
That is correct.
Yes. So what’s the difference between that survival rate, 72% and the low 53%? I think it was, or 51% in the data on Page 18.
Yes. That 50 – yes, I’m sorry. On Slide 18…
Survival.
Yes, that’s 51% at two years. The data that we’re talking about here in on Slide 21 is 72% at three months, the 100.
Okay.
That’s right. So it’s early survival data in the EAP, which only followed up children through 100 days. And then the longer term survival of our Phase 3 trial is now out to four years. Even a 51% two-year survival rate is substantially greater than the survival in the mid-20s to mid-30s with best available therapy, which includes ruxolitinib in other studies.
And is there any confusion now as to the level of potency that the product that’s been manufactured in recent years will deliver? Because this analysis indicates there was a significant difference between the earlier Prochymal product and the – products…
That’s precisely the point. That’s exactly the point. The clarification has come through the analysis that demonstrates that improvement in manufacturing, changes in manufacturing, which were implemented in, at a particular point in time in the development history of the product, delineates a more potent product currently in use in our studies from the less potent product that was called Prochymal that failed in other earlier studies. And so we believe that the, the reason that we’re seeing greater clinical outcomes with our newly improved product is because of its greater potency as measured by our validated potency assay. And that is very encouraging both in terms of the pediatric data and the data that we expect to generate in adults.
So to what extent has that – new data on the potency been available in over the last two years since the complete response letter? Or has that always been available?
No, these are data that are newly generated as part of the BLA resubmission in alignment with optimization of the potency assay. But the assay itself has been in place and was in place when products were being used in both the EAP and the Phase 3 trial. And so what this provides us now is an understanding of how changes in manufacturing have resulted in a more potent product, which correlates with better survival.
Okay. And just finally during the inspection of the plant in Singapore, was the inspectors able to see product being manufactured at that time? Assume that they were.
Yes. The reason for the inspection is that four inspectors were on site and observing the entire manufacturing process from beginning to end over a 10-day period, every detail of the manufacturing process was observed and inspected.
Excellent. That’s all for me. Thank you.
Thank you.
Thank you. As there are no further questions at this time that brings us to an end of today’s call. I’ll now hand back to Dr. Itescu for any closing remarks.
Great. Thank you everybody for joining us today. We’re very excited about how the process is evolving on the BLA resubmission and our interactions, very collaborative interactions with the FDA. And we look forward to the upcoming PDUFA decision date. As we build out our plans for hopefully a successful outcome and a post-approval launch. Thank you everybody.
That does conclude our conference for today. Thank you for participating. You may now disconnect.