Q3 2024 BioRestorative Therapies Inc Earnings Call and Business Update
Speaker Change: Greetings. Welcome to the Biorestorative Preliminary BRTX 100 Phase 2 Study Data Review Conference Call. At this time, all participants are in a listen-only mode.
Speaker Change: A question and answer session will follow the formal presentation. If anyone should require operator assistance during the conference, please press star zero on your telephone keypad. Please note this conference is being recorded. I will now turn the conference over to your host, Stephen Kilmer, Investor Relations. You may begin.
Stephen Kilmer: Thank you. Good afternoon everyone. Let me start by pointing out that this conference call will include forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.
Stephen Kilmer: All forward-looking statements are based on biorestorative therapies' current beliefs, assumptions, and expectations, and such statements involve known and unknown risks, uncertainties, and other factors that may cause actual results, performance, or achievements to be materially different from those implied by such statements.
No forward-looking statement can be guaranteed.
Stephen Kilmer: For details and factors among others that could affect expectations, see part one, item 1A of our annual report on Form 10-K for the year ended December 31st, 2023 as amended.
Stephen Kilmer: And part two, item 1A of our quarterly report on form 10-Q for the period ended September 30th, 2024, filed with the Securities and Exchange Commission.
Stephen Kilmer: Listeners are cautioned not to place undue reliance on these supporting statements which speak only as of the date of this conference call.
Stephen Kilmer: BioRestore.gov undertakes no obligation to publicly update or revise any forfeiture statement, whether as a result of new information, future events, or otherwise, other than if required by law.
Speaker Change: On the call representing the company are Lance Alstott, BioResources Chairman and Chief Executive Officer, Francisco Silva, its Vice President of Research and Development, and Robert Kristol, the company's Chief Financial Officer. With that said, I'll now turn the call over to Lance.
Lance Alstott: Thanks Steve and good afternoon everyone welcome to the call on behalf of the management team and everyone here at Biorestorative. I'd like to thank you for your interest in our company and for those of you who are shareholders we obviously appreciate your support.
Lance Alstott: As you can see from the press release that we issued,
earlier today. We continue.
Lance Alstott: to execute very well across all areas of our business in the third quarter. As excited as we are about our financial and operating performance this quarter and the progress that we're making in our Thermostem program,
Lance Alstott: It was really the preliminary Phase 2 BRTX-100 data that we announced this morning that is most exciting, with very encouraging data for 10 patients at 26 weeks and 4 patients at a year. Accordingly, we're going to break with tradition a little here, and rather than review our financial results,
Lance Alstott: I'm going to ask Francisco to jump right into delivering the same BRTX-100 preliminary data slides that he gave today at the ORS-PSRS 7th International Spine Research Symposium. Of course, if you have any questions about the third quarter financial results, I'm going to be happy to answer them.
Lance Alstott: Our Thermostem brown fat program or our commercial biocosmeceuticals business would be happy to answer them during the call's Q&A session as well. I'll turn it over to Francisco now.
Great. Thank you, Lance, and good afternoon, everyone.
Francisco Silva: So, our lead cell therapy candidate is BRTX100. It's a novel cell-based therapeutic engineered to target areas of the body that have little blood flow.
Francisco Silva: and oxygen content. The product is formulated using autologous or your own cultured mesenchymal stem cells collected from the patient's bone marrow.
Francisco Silva: as well as platelet lysate that's collected from the patient's blood.
Francisco Silva: We intend that the product will be used for the non-surgical treatment of painful lumbar sacral disc disorders or the complementary therapeutic to a surgical procedure.
Francisco Silva: The safety and efficacy data of BRTX-100 in treating chronic lumbar disc disease, or CLDD, is being evaluated in an ongoing Phase 2 prospective randomized double-blinded and sham-controlled study.
Francisco Silva: A total of up to 99 eligible subjects will be enrolled at up to 16 clinical sites across the United States, and subjects included in the trial will be randomized 2-to-1 to receive either BRTX or placebo with the sham controlled.
Speaker Change: As Lance mentioned, I'm pleased to review the same slides that I discussed earlier today at a podium presentation at the ORS SPINE meeting.
Speaker Change: And so, to go through the slide deck, I'm just going to quickly jump through.
Speaker Change: Some of the slides, obviously, we have our border plate forward-looking statements.
Speaker Change: And on slide three, I just want to quickly review this microenvironment, which is really important in understanding the impact.
Speaker Change: of the product, the way it's been designed and engineered because it's really targeting the harsh microenvironment that's found within the disc. It has a very high osmolarity. It's low glucose. It has very low oxygen tension of about 2%.
Speaker Change: Lactate levels are also low, and pH, which is extremely detrimental to cells. So, this is important because...
Speaker Change: When you're developing a biologic and you're trying to remodel the disc, especially in within the nucleus pulposus,
Speaker Change: If you put cells in there that are not acclimated to this harsh environment, you're going to have a lot of cell death that could potentially cause the patient to experience worsening symptoms.
Speaker Change: of the treatment or the treatment might not work at all because the cells need to be alive for them to actually.
Speaker Change: perform their anti-inflammatory activities. So really understanding the microenvironment is critical when developing a product and that's what we have done over the last couple of years in developing our BRTX100 product. So in the microenvironment,
As a healthy disc degenerates, let me go back slowly.
As a healthy disc degenerates,
Sorry, guys, but this is moving a little bit different.
Bye.
Lance Alstott: Hey Francisco, I'm sorry, it's Lance. I think based on the investor profile,
Lance Alstott: the environment as well as the product candidate. Let's talk about the results. That's really the most exciting part of it.
Speaker Change: Sure, okay. Yeah, no problem. Yeah, so let's go to slide number 17 and in slide number 17 is the
total of N10 patients that are treated using the product.
Speaker Change: BRTX100, and in this particular group of patients that have been treated, you can see a direct correlation that at 26 weeks.
Speaker Change: 70% of the patients, again, this is blinded, but very, very encouraging, 70% of the patients had an improvement in both pain as well as function measured by the VAS and the ODI.
Speaker Change: So, 7 of the 10 improved and 3 of the patients did not.
Speaker Change: hit the benchmark of 30% improvement, which is dictated by the FDA as being efficacious.
Speaker Change: So that's very encouraging from the point of view of seeing that the data is falling in line within the randomization.
Speaker Change: of the patients treated. And we expect that this trend will continue to grow as the patients move on from the 26 weekend point to the 52 weekend point.
Thank you.
And again, this is true as you
Speaker Change: review some of the other scales that we use for patient-reported outcomes, which is
Another one is the RMDQ.
Speaker Change: which is also showing a decrease in time in patients that have been treated or treated with the clinical protocol in terms of either being in the sham or the treatment group.
as well as the FRI, which is another scale that...
Speaker Change: is used to determine efficacy. You see very similar trends in terms of the patient profile decreasing once the patients are enrolled in the trial.
So, to summarize, what we found is that
Speaker Change: Very early, we're seeing very strong safety signals, so we have not
Speaker Change: experienced to date any severe adverse event or dose limiting toxicity or current dose of 40 million cells injected intradiscally.
Speaker Change: So this is very, very encouraging, and this is our primary endpoint in terms of safety. So we feel very strong that this is gonna continue throughout the trial.
More importantly is the efficacy, which is...
Speaker Change: We're starting to see very strong trends emerge in both the VAAS and the ODI.
showing significance compared to baseline.
Speaker Change: And there's a responder rate that is also beginning to form now with the additional data that we're seeing that 7 of the 10 patients met the preliminary efficacy endpoints of improving by both 30% in pain and function.
Speaker Change: Another item that we're very encouraged about because we're starting to see a lot of this positive data emerge is that we're contemplating a 26-week
Speaker Change: Preliminary Efficacy Endpoint Interim Analysis. So that's something that's on the horizon for us. In addition to that, we're looking to expand the product profile and indication of BRTX100 to include cervical indications.
Thank you.
Speaker Change: Thank you, Francisco. So, as you can see from what Francisco just reviewed, the blinded preliminary clinical data of safety and efficacy endpoints from the ongoing Phase II clinical trial for the treatment of chronic lumbar disc disease are very encouraging with patient-reported pain and function outcomes.
demonstrating a very clear and positive trend.
Speaker Change: So, to summarize, at 26 weeks, like Francisco mentioned, 70 percent.
of the first 10 patients in the study reported.
Speaker Change: a greater than 30% increase in function and a more than 30% decrease in pain.
Speaker Change: Now, I want to highlight that while the 30% increase in function and decrease in pain
have been set forth by the FDA as...
Speaker Change: as preliminary endpoints for efficacy, we find those to be somewhat low, and we have demonstrated the ability to do far greater than those hurdles of 30%. So, if you look at each of these patients...
Individually, those that responded, they responded.
far better than the 30% some going as high as.
close to 90% improvement in pain and function.
Speaker Change: and others circling around that sort of geography. So we're very pleased, we're very optimistic about these results.
Speaker Change: It really puts us in a position to continue with our enrollment, which we continue to progress well, and we expect to complete before the end of 2024.
Speaker Change: We know that if the data continues with this trend, we're confident that we will hit our efficacy endpoints for the Phase 2 trial.
Speaker Change: And we will intend to present more of this data from the trial with an even larger patient population.
Speaker Change: in the coming months. I expect to be somewhere early next year, January and February, with a larger group both out to 52 weeks and 26 weeks.
Speaker Change: Finally, from a financial perspective, I'd be remiss by not mentioning that we ended the third quarter in a very strong position with cash, cash equivalents, and investments held in marketable securities, $13.1 million with no outstanding debt.
That, combined with our steadily improving financial performance,
Speaker Change: provides us with additional financial flexibility and puts us on the path.
to profitability.
Speaker Change: So, thank you very much for listening in. Those are our concluding remarks as it pertains to our prepared script, and we're happy to take any questions that you may have.
Speaker Change: Okay. So when was that data cut the two weeks because you know when you show two weeks it kind of makes some would think that there is not that many more than 10, because you know it's just two week old data. So when was that cut.
This data is several months old.
Speaker Change: In terms of.
Speaker Change: The two week time point.
Speaker Change: Where some of the patients hit the two week point. So yeah, we have more than 10 patient data most of the.
Speaker Change: Patient data, where it'll be a lot more cumulative will be presented at <unk> in February.
Speaker Change: Okay. So now I can see why you're still sticking with 99 by year end 'twenty four thanks, thanks for that clarity.
Speaker Change: When we see you know.
Speaker Change: When will we see the first unblinded data.
Do you intend to come out with additional 26, and 52 week data still on a blinded basis or will we see unblinded. The next time.
Speaker Change: Oh, no it will still be blinded at that you know again this is the academic.
Speaker Change: Conference, where we're presenting that to be very careful how we present the data as well to maintain the blind. So that's.
Speaker Change: Something even though the results are.
Speaker Change: Impressive and very encouraging from from the point of view of the emerging responder rate.
Speaker Change: But this is where we are so encouraged by the positive result is where we're looking at the possibility of an interim analysis at 26 weeks.
Speaker Change: For the patients and I think that would be inappropriate time to have unblinded data, where you know we have the sham versus the treatment group with beer Tx.
Speaker Change: And you can't postulate when that Unblinded 26 week look interim look might might occur Kenya.
Speaker Change: I mean.
Again, I mean, it's it's all you know we need to make sure. We don't compromise the integrity of the trial. There was a lot of logistics in terms of releasing but if we were to do an interim analysis. It would be early next year.
Speaker Change: Okay.
Speaker Change: Yes, I would I would weigh in and commit to you know sometime in the first half of 'twenty five.
Speaker Change: But you know that's subject to further updates.
Speaker Change: That's definitely helpful. So do you think the fourth patient that behaves like the other three that back in February of this year.
Speaker Change: Three versus one and that was the split you were talking about do you think that fourth patient the sailing that got just essentially started to work and that's why you have you know four for 52 weeks versus the clear line between three and four last year and I think yeah, and I think and I think that's a that's a great comment and I mean, that's the beauty.
Speaker Change: All of our clinical trial is that if it's well designed and the product is efficacious you'd begin to see trends emerge in.
Speaker Change: That one subject.
Speaker Change: As part of that group.
Speaker Change: We have that was a ceiling treated and it's very well known that's saving does have therapeutic effects that are not long term maintained but they do impact. The trials. So you know we're very happy that we were able to amend our protocol.
Speaker Change: To have now a sham group and so at the 26 weeks. This is where the sham the sham patients are or are being recorded and so we expect that you know presenting 52 week data of these particular patients you'll have a better delta and a better view of it in terms of.
Speaker Change: What's happening.
Speaker Change: But the 20th theaters.
Speaker Change: Go ahead.
Speaker Change: Can we can we can we be confident that the two placebos to be represented you know two of the six extra patients that that turned four into 10 did they both get champs or perhaps the biggest failings as well.
Speaker Change: Only one patient with you.
Alien.
Speaker Change: Injection, great everyone, everyone else Sham.
Speaker Change: And and then and just just to further your two to answer that as well as you look at the 52 week data with the four patients now we don't know, which one have received the sham injection versus the beer T X 100, but there's considerable separation in the results while all of them 100.
Speaker Change: Percent achieved the 30% it it appears that one only achieved 30% while the others achieved.
Speaker Change: Results far more significant than 30%. So we still see a separation there and it's very possible that you know the placebo effect to take hold.
Speaker Change: And you know the desk was further hydrated and lay out lowered its P. H and kind of maintained a healthier environment. So we can expect that I guess from time to time, but the Francisco's point. The 26 week data, where we have the sham injection those are a little bit more.
Speaker Change: Or bifurcated between those that responded and those that did not and it kind of matches up with the randomization of two to one.
That's that's very helpful. Rob how long do you think 13 million less.
Speaker Change: Well.
Speaker Change: I'll speak for Rob here. He is in the background, but while we don't commit to timing, we don't anticipate having to do a refinancing.
Speaker Change: Anytime soon so we we have all the cash that we need to achieve our strategic goals and get to an answer within this trial.
Speaker Change: Thank you very much guys congrats.
Speaker Change: Thanks, Jonathan.
Speaker Change: The next question comes from Michael <unk> of which with Maxim Group. Please proceed.
Speaker Change: Hey, guys. Thank you so much for taking my question and congrats on a really interesting data.
Speaker Change: Yeah.
Speaker Change: Thanks, Mike I guess to kick things off and just follow up a little bit on the point you were.
Speaker Change: Talking about just before on the 52 week data, where you saw all core patients respond.
Could you just give an idea of the magnitude of response for patients one through three versus patient four because I believe that's how it broke down at the prior interim blinded look where you add patients one two and three responding significantly more than patient four.
Yes, and that that trend is continuing again, we have to be careful in terms of how we present. This data we don't want to.
Speaker Change: And blind.
Speaker Change: But the trend is continuing.
Speaker Change: With one patient having.
Speaker Change: Some effect based on the.
Speaker Change: The randomization.
Speaker Change: But the other three patients are significantly lower which is.
Again, the numbers the numbers range, but there are greater than 60% improvement compared to baseline.
Speaker Change: Yeah.
Speaker Change: Alright, Thank you and then.
Speaker Change: Just I guess more broadly right given what we have seen from the USDA seeming to be a bit more supportive of the others in the cell therapy space like we've seen with Capricorn meso blast.
Speaker Change: Would it be possible that if you are able to achieve both pain and function in the study because that would be something that you could file for accelerated approval.
Speaker Change: Well, we believe so I mean, it's something that that it's if again I mean, this is 10 patients but the path there.
Speaker Change: The data is very encouraging considering what's been out there you know what.
Speaker Change: With either clinical other clinical trials in the disc space or investigation investigator initiated studies.
Speaker Change: So we're very encouraged that this data as it continues to show these effects that we could potentially use this you know the theater for pivotal.
Speaker Change: Which would really help us and accelerate the commercialization of the product, which is I think really needed in terms of the overall market in the U S and getting cell based therapies approved.
Speaker Change: Basically when you're you know you're looking.
Speaker Change: If these musculoskeletal disorders, which are tremendous so there's a huge market for it and we're very confident with our product.
Speaker Change: Alright. Thank you and then one last one for me and I'll hop back into queue, Michael Michael I'll also Oh My God also mentioned that because of what you just mentioned and the temperature at the FDA in terms of its more pro cell therapy. Our view, we have we've changed some of our strategy to consider.
Speaker Change: Looking at this data as pivotal data perhaps.
Speaker Change: And I'm trying to accelerate that that study as appropriate. So we do appreciate that comment and we do believe that the FDA has taken a.
Speaker Change: I'm much more positive view of the sector in general.
Yeah. Thank you and actually the that doesn't mean someone into my next question I just wanted to ask you a little bit about some of the secondary that you'd be looking at in the.
Speaker Change: And you know the final unblinded data and in particular, whether you will be looking at how the percent change from baseline varies between groups.
Speaker Change: Can you can you repeat the question I'm, sorry, I didn't understand it.
Speaker Change: Oh just for the.
Or the actual final unblinded data in terms of secondary endpoints as something that you'll be looking at the percent change from baseline on both the pain and function scales for the different groups.
Speaker Change: In addition to the responder analysis.
Speaker Change: Right. So the the FDA has guided that it's a 30% change.
Speaker Change: Change.
Is there they're M C I D that they've they've put into our protocol.
Speaker Change: We feel that we can exceed that based on the trending date.
Speaker Change: Data points that are coming out.
Speaker Change: So we feel very strongly that we're going to not only hit the the the M. C. R. M C. I D in terms of efficacy.
But we will be able to exceed that.
Speaker Change: And we're also looking at radiographic. We're also looking at radiographic endpoints and a whole host of other measures that we think would be just good general information for people to have to demonstrate that it's not just you know a subjective issue, but if we can demonstrate that there's radiographic improve.
Speaker Change: And the desk, that's a very powerful that's a very powerful set of data.
Speaker Change: Yeah, and just to add to that that's really important here is that you know keep in mind that we have not disclosed or release any MRI.
Speaker Change: Images of any of these patients.
Speaker Change: So that's something that's something to look forward to in terms of additional.
Speaker Change: Evidence of the the remodeling that's happening within the disk.
Speaker Change: Alright, well, thank you very much and once again congratulations on the compelling data here.
Speaker Change: Thanks, So much Mike I appreciate it.
Speaker Change: Once again, if there are any remaining questions or comments. Please indicate so by pressing star one. The next question comes from Alima Gyros with Rodman. Please proceed.
Speaker Change: Yes, good afternoon, everyone, what I'd like to box plants, if you could remind us where the pain improvement in a functional improvement are these co primary endpoints.
Speaker Change: And.
Speaker Change: If that is so would.
Speaker Change: Would you have to demonstrate it at the group level or would you have to demonstrate it within the same patient that they will both with tax to improve at least 30% on the vas and at least 30% on the ODI for them to be called the response here. Thank you for.
Speaker Change: Verifying that.
Speaker Change: Yes, they have to both respond in both the bonds as well as the ODI to be labeled a responder.
So in the current study you see jacko that'd be seven out of 10.
Speaker Change: Is the seven that respond yes, Craig.
Speaker Change: Six weeks ago.
Speaker Change: Yep.
Speaker Change: And all of them are thank you for your question I think I know, where you're going because there has been a bit of a change in thought within the agency.
Speaker Change: And people are now looking at just pain as opposed to function as well.
Speaker Change: It leaves US you know open strategically as we think about amending proto.
Speaker Change: Protocols are as we go through this to you know really increase our probability of success by you know limiting it perhaps to just one factor such as pain, because we do believe that there is precedent for that out there now.
So it's something that we're we are discussing but as of now the protocol is written for both pain and function and.
Speaker Change: The good news for US is the responder group.
Speaker Change: Hum does clear that what we think is the minimal bar of 30% in both both disciplines.
Speaker Change: Yes, I mean at the end of the day, if you were.
Speaker Change: Successful they both that would make our story that's for a stronger argument towards rather well while also be show showing you know some some changes radio graphically and an improvement in the you know the.
Speaker Change: The physics of the desk would be also very interesting and compelling, which we intend to do yes.
Yes.
Speaker Change: That was all thank you so much.
Speaker Change: Thanks, Alan I appreciate it.
Speaker Change: We have no further questions in queue I'd like to turn the floor back to management for any closing remarks.
Lance Alstott: I would just say this is lance on behalf of our team we really appreciate it.
Lance Alstott: You are following us and spending the time to get further clarity on our quarterly accomplishments and certainly your interest in our clinical data. We will continue to update everyone as appropriate on new developments within our company and we intend to have more data.
Lance Alstott: Available in the January February timeframe, so stay tuned, but we're really excited to share with what we're working on so have a great night and thanks again for your interest.
Speaker Change: Thank you. This concludes today's conference and you may disconnect. Your lines at this time. Thank you for your participation.