Q1 2025 Longeveron Inc Earnings Call
Greetings and welcome to the longevity or on first quarter 'twenty 25 earnings call.
Speaker Change: At this time all participants are in a listen only mode. A brief question and answer session will follow the formal presentation should anyone require operator assistance. During the conference. Please press star zero on your telephone keypad.
As a reminder, this conference is being recorded.
Speaker Change: It is now my pleasure to introduce your host Derek call of an Investor Relations Advisory solutions. Thank you you may begin.
Speaker Change: Thank you operator, good afternoon, everyone and thank you for joining us today to review along Jeb Ron's 2025 first quarter financial results and business update.
Speaker Change: After the U S markets closed today, we issued a press release with financial results for Q1, 2025, which can be found under the investors section of the long jump on the website.
Speaker Change: On the call today are while his Shah Chief Executive Officer, Joshua Here Co founder Chief Science Officer, and Chairman of the board.
Speaker Change: AGA Pheno about Chief Medical Officer, Lisa Luckily, our Chief Financial Officer, and Deb and blast Chief Technology Officer.
Speaker Change: As a reminder, during this call we will be making forward looking statements. These statements are subject to certain risks and uncertainties that could cause actual results to differ materially from these statements any such statements should be considered in conjunction with cautionary statements in our press release and risk factors discussed in the company's filings with Securities and Exchange Commission, which we encourage you to review.
Speaker Change: Following the company's prepared remarks, we will open the call to questions from covering analysts with that let me hand, the call over to Dr. Joshua here founder and chairman of the Board Josh.
Dr. Joshua: Thank you Derek.
Dr. Joshua: Good afternoon, everyone and thank you very much for joining us today, we greatly appreciate your interest and longevity.
Dr. Joshua: We are very pleased to update you on our continued successful progress advancing stem cell therapy with our lead product lateral immerse yourself for multiple chronic indications.
Dr. Joshua: Simply put this period.
Dr. Joshua: Over the past two years through next year is the most exciting time in the company's history.
Speaker Change: 10 years ago, when I Cofounded the company a stem cell therapy vision was an academic idea.
Speaker Change: Today, we could see the possibility of our first BLA submission.
Speaker Change: Getting approval for lateral master cell for HLA chess.
Speaker Change: This directly reflects our mission speaking to help patients and families through the application of stem cell research.
Speaker Change: And all timers disease, we've generated incredibly important clinical data.
Speaker Change: Porting the therapeutic potential of lateral master cell in this devastating neurodegenerative disorder.
Speaker Change: It's a progressive memory loss and death and currently has very limited therapeutic options.
Speaker Change: This data has been accepted for presentation at multiple leading industry conferences and was published in the prestigious peer reviewed journal nature Medicine, and the April 'twenty twenty-five issue.
Speaker Change: We were honored to also have the opportunity to discuss these phase II results in a recent article in neurology today.
Speaker Change: Our motivation and dedication to advancing stem cell therapy research has only been further invigorated by the reception to this data from the thought leader community.
Speaker Change: The next 12 to 18 months or potentially transformational period for longevity on with multiple critical milestones and we are incredibly excited about the opportunity for this progress and to share the developments with you.
Speaker Change: I will now hand, the call over to Y O y L.
Speaker Change: Thank you Josh.
Speaker Change: Josh stated so well that's a very exciting time for the company will continue to focus on diligent efficient execution of our strategic plan for launching their own and lateral not yourself.
Speaker Change: In February of this year, we achieved an important milestone when their international non proprietary name expert committee of the World Health organization.
Speaker Change: Lateral master cells has the non proprietary name for la myself be this.
Speaker Change: As an important step in the development and potential future commercialization of our cell therapy lateral mass yourself.
Speaker Change: It will be using their name lateral maths yourself going forward when graph dressing our cell therapy and reduce their usage of laminates I'll be.
Speaker Change: As a reminder for those of you in your tower story long Jupiter one is a regenerative medicine company developing cutting edge cellular therapy.
Speaker Change: Our stem cell therapy lateral master itself has delivered several positive initial results across five clinical trial in three indications.
Speaker Change: Phase one and two in Alzheimer disease.
Speaker Change: One and two and aging related frailty and phase, one and hyperplastic left heart syndrome or edge L at chess.
Speaker Change: A rare pediatric orphan disease.
Speaker Change: The company's development program for these three initial indications addressed your market U S market opportunity of approximately five plus billion or plus billion and up to 1 billion drifts back at me.
Speaker Change: Our strategic plan is built on the strength of the science underlying our stem cell therapy lateral master itself.
Speaker Change: Our goal for 2020 five center on Christian.
Speaker Change: The execution of that strategic plan with emphasis on three primary operational goals.
Speaker Change: Opus, two which is the phase two be a study for HLA chats completion.
Speaker Change: And so let's just be L. A preparedness and commercialization now rather than us.
Speaker Change: Assuming a strategic collaboration for the Alzheimer disease program.
Speaker Change: Actually Chad is a key strategic priority for us.
Speaker Change: We believe the Agila chest program has high probability of success.
Speaker Change: And the shortest path to potential regulatory approval and future commercialization across our pipeline.
Speaker Change: We are currently nearing completion of enrollment of the Lps to phase II B study.
Speaker Change: Evaluating lateral match yourself as a potential adjunctive treatment.
Speaker Change: Standard of care or etch alert shop space yet.
Speaker Change: I'll pause to have achieved approximately 95% single woman.
Speaker Change: And we expect to complete enrollment in the second quarter of this year, which is the current existing corridor.
Speaker Change: Yeah.
Speaker Change: The determination by the U S food and drug administration at our meeting in August of last year that Opus two is pivotal significantly accelerated the potential regulatory path for lateral mask herself.
Speaker Change: And if supported by clinical data from the clinical trial would allow us to initiate a rolling submission of a BLA with the food and drug administration in 2026.
Speaker Change: With the potential for BLA submission on the horizon, we hired Devin blast as our Chief Technology Officer.
Speaker Change: He has over 15 years of distinguished experience and the development and manufacturing of advanced therapy.
Speaker Change: <unk> is leading the company's technological and manufacturing strategies.
Speaker Change: He will be providing a brief commentary and our etch I'll, let Chad BLA preparedness activities.
Speaker Change: The lateral match yourself Alzheimer disease program continues to garner important recognition and support.
Speaker Change: Doctor hair mentioned results from clear mind phase Iia clinical trial evaluating lateral master yourself.
Speaker Change: In mild Alzheimer's disease were published in nature Medicine in March of this year.
Speaker Change: And featured in an interview with Dr. Hare in neurology today in April.
Data from this trial were previously presented as a featured research oral presentation at the 'twenty 'twenty four Alzheimers agitation International conference held at the end of July of 'twenty 'twenty four.
Speaker Change: The late breaking and also presented as a late breaking poster presentation at the 17th edition of the clinical trials on Alzheimer's disease conference seat that 24 in October.
Speaker Change: In March of this year, we held a type b meeting with the FDA.
Speaker Change: Discuss the regulatory pathway for a potential BLA or lateral mass without and miles outside my Dizzy.
Speaker Change: We reach foundational alignment on the overall study design or the proposed single pivotal seamless adaptive.
Speaker Change: Phase two three clinical trial.
Speaker Change: So support.
Speaker Change: And accelerated path to potential approval.
Speaker Change: The FDA agreed to consider our BLA submission based on our positive interim results from this plant single study.
Speaker Change: With this regulatory clarity in hand, we are focused on seeking partnership opportunity.
Speaker Change: And or non dilutive funding for the Alzheimer disease program.
Speaker Change: I'm incredibly pleased with the progress we are making to advancing lateral master yourself at the cellular therapy for multiple indications with important unmet medical need.
Speaker Change: We are now approaching multiple potential transformational milestones over the next 12 to 18 months and I am thoroughly excited by the opportunity for lateral Nashville, so larger grown patients and shareholders.
Speaker Change: With that I will turn the call to Dr. Laura I guess, the Nova to provide update on our clinical development program.
Speaker Change: Italia.
Speaker Change: Thank you Joe and good afternoon, everyone.
Speaker Change: He didn't distributional code that is why our master cell stem cell derived.
Speaker Change: Alright culture expanded meeting chemo stem cells and we see.
Speaker Change: That's a source from the bone marrow of young healthy adult dawned on us.
Speaker Change: The number of functional M. A CS in the body declines as the H <unk> Zealand interest in day in day use for aging related conditions.
Speaker Change: <unk> unique properties, including the ability to reduce inflammation.
Tissue repair and regeneration modulate immune response and improve vascularization also support their application in summary of cardiovascular disease condition with high unmet medical need.
Speaker Change: We believe that by using the same cells that promote tissue repair, Oregon maintenance and immune system function, we can develop safe and effective therapy for some of the most difficult diseases and conditions.
Speaker Change: Based on positive when you show the a lot of them, yes for cell development programs have received five M D designation.
Speaker Change: Well the HLA chip program orphan drug designation fast track designation and rare pediatric disease designation.
Speaker Change: And for the Alzheimers disease program with Zenith Madison Advanced Cherokee Ahmad designation and fast track designation each of niche offer me feed for their program development and regulatory costs.
Speaker Change: That's why you all mentioned that our <unk> program is a primary focus for the near term pathway to potential approval in an area or a clear unmet medical need.
Speaker Change: <unk> is a rare pediatric disease in each the last PMT call one of the pumping chamber of the heart.
Speaker Change: Either severely underdeveloped omission.
Speaker Change: Cause the left ventricle is the chamber that normally pump the blood to the body infants born get this condition have a profound reduction in blood flow and thus cannot get the normal supply of oxygen to their Oregon.
Speaker Change: In order for the children to survive they undergo a complicated T stage car T. Construction, so journey over the course of the five years, so they're light.
Speaker Change: Despite this surgical reconstruction only 50% of the affected children survive two <unk> without her heart transplantation.
Speaker Change: Our program is designed to both improve their heart function in these children with the goal of potentially enhance.
Speaker Change: Their survival.
Speaker Change: Our phase one clinical study altice one.
Speaker Change: He might have eaten Laramie is just so in four months old in from Chile.
Speaker Change: Chile Chats E Z.
Speaker Change: <unk> hundred percent transplant free survival after five years old and treatment.
Speaker Change: These contracts have been approximately 10% mortality rate.
Speaker Change: In the historical control data.
Speaker Change: Five years post treatment long term survival data from the L. P. Swan Phase one clinical trial was presented and the congenital heart surgeon societies cause. He's just shows annual meeting in October Cleantech means you for them.
Speaker Change: We are currently conducting the phase two b clinical trial is to evaluate the potential of a lot of them you have to sell to improve right means equals a function and look them outcomes in pediatric patients. It's a challenge.
Speaker Change: This tool is being conducted in collaboration with the National Heart lung and Blood Institute through grants from the National Institute of Health.
Speaker Change: We expect to complete enrollment of the trial before the end of the second quarter.
Speaker Change: He will then focus on supporting our investigative sites through completion of 12 month primary endpoint cool off period in preparation for the data collection and analysis at the end of this target to support potential BLA submission.
Speaker Change: If results from LTE to a body camera.
Speaker Change: Wouldn't be positioned to initiate a rolling BLA submission with the M E in 'twenty six.
Speaker Change: I now hand, the call over to David and block, our Chief Technology Officer Damian.
Speaker Change: Thank you and Italia good afternoon, it's a pleasure to join the call for the first time with everyone. That's why I mentioned my background is in the manufacturer and development of advanced therapies, which is why I'm delighted to have joined longevity at this transformational period in the company's development of cellular therapies.
Speaker Change: This year, we don't see a significant focus on organizational readiness the potential BLA filing for HLA Jess in 2026.
Speaker Change: The biological license application is a formal request for marketing approval submitted to the FDA for a biologic somewhat analogous to the process of submitting a new drug application for small molecule therapy BLA.
Speaker Change: You always are evaluated by the center for Biologics evaluation and research or fever, and entity distinct from the center for drug evaluation and research or Peter.
Speaker Change: In a relatively short period of time, we have developed a strategic plan to ensure BLA readiness should help us to generate positive results, our CMC or chemistry manufacturing and controls plan is focused on meeting our BLA timeline without delays at the lowest cost.
Speaker Change: We are evaluating all strategic options to achieve that outcome.
Speaker Change: This includes exploring commercial manufacturing options for the Mr. So either by enhancing our facilities and internal capabilities to meet our commercial readiness or alternatively contracting the commercial manufacture of the lira, Mr. Salt for HLA test to a CMO.
Speaker Change: Our P&C playing identifies the necessary tasks and outlines the substantial amount of work required to achieve BLA readiness, ensuring we are well prepared to submit all of the necessary CMC components of the application in a timely manner.
Speaker Change: While there's obviously a lot of work to accomplish ahead of any potential filings, we're off to a strong start and making consistent progress every day.
Our goal is to substantially advanced BLA readiness. This year ahead of data readout potentially shortening the timeline from clinical trial data read out to BLA submission.
Speaker Change: I hand, the call over to Lisa Luckily, our Chief financial Officer to discuss our financial results for the first quarter Lisa.
Lisa: Thank you Devin and good afternoon, everyone.
Lisa: Afternoon, we issued a press release and filed our quarterly report on Form 10-Q, both of which present our financial results in detail. So I will touch on some highlights.
Lisa: Revenue for the first quarter of 2025, and 'twenty 'twenty $440.4 million and zero point $5 million, respectively. This represents a decrease of zero point $1 million or 30% in 2025 compared to 2024, driven primarily by decreased purchasing demand for our Bahamas registry trial part.
Lisa: Offset by an increase of our manufacturing services contract revenue.
Lisa: Clinical trial revenue from the Bahamas registry trial in the first quarter of 2025 zero point $3 million, a decrease of zero point $2 million from the first quarter of 2024.
Lisa: As I said data decreased persistent demand.
Lisa: Contract manufacturing revenue for the first three months ended March 31, 2025 and 2024.
Lisa: Zero point $1 million and $33000, respectively, reflecting an increase of approximately zero point $1 million due to increased activity from our manufacturing services contract.
Lisa: General and administrative expenses for the three months ended March 31, 2025 increased to approximately $2 $9 million compared to $2.2 million for the same period in 2024.
Lisa: The increase of approximately zero point $7 million or 34% was primarily related to an increase in personnel related costs, including equity based compensation.
Lisa: Research and development expenses for the three months ended March 31st 2025 increased 13% to approximately $2 $5 million from approximately $2 $2 million for the same period in 2024.
Lisa: Our net loss for the quarter increased to approximately $5 million from $4 million for the same period in 2024.
Lisa: Our cash and cash equivalents as of March 31st 2025 were $14 $3 million.
Lisa: We believe our existing cash and cash equivalents will fund our operating expenses and capital expenditure requirements late into the third quarter of 2025 based on our current operating budget and cash flow forecast.
Lisa: Following our successful type C meeting with the FDA in August of 'twenty 'twenty four regarding the H L. H S regulatory pathway, we have to.
Lisa: Again ramping up our BLA, enabling activities.
Lisa: We currently anticipate a potential BLA filing with the FDA in 2020 six if the current opus two trial initially just as successful.
Lisa: The company's operating expenses and capital expenditure requirements will increase throughout calendar 2025, as a result of these BLA enabling activities.
Lisa: We expect that our current operating plan will require increased spending and additional capital investments to support these initiatives and we intend to seek additional financing capital raises and our non dilutive funding options to support them.
Lisa: Additionally.
Lisa: A positive type B meeting with the FDA in March 'twenty 'twenty five with respect to the Alzheimers disease regulatory pathway. We are focused on seeking partnership opportunities and are non dilutive funding for the Alzheimers disease.
Lisa: Disease program, including a proposed single pivotal seamless adaptive phase two slash three clinical trial.
Lisa: There are a lot of important things happening right now for lunch Evraz and we're excited for these opportunities and to keep you updated on our progress.
Lisa: I will now hand, the call back to IL.
Speaker Change: Thank you Lisa.
Speaker Change: Launch of Rone has made tremendous progress in two important stem cell development program.
Chad: Actually Chad.
Chad: Alzheimer's disease.
Chad: We are now approaching multiple potentially transformational milestones, including completion of pivotal phase two b clinical trial, an edge on their chest.
Chad: Our first potential BLA submission for HLA chat and based on the strength of a phase Iia clinical data potential partnering for Alzheimer's disease program.
Chad: Our team's expertise and industry experience enable the organization to accomplish so much with a small team and fewer resources and I am incredibly proud of their efforts and accomplishments on behalf of patients and shareholders.
Chad: We deeply appreciate the support of all the stakeholders and look forward to continued collaboration and progress in the future.
Chad: Operator.
Speaker Change: We would now like to open the call for questions from our covering analysts.
Speaker Change: Thank you we will now be conducting a question and answer session. If you'd like to ask a question. Please press star one on your telephone keypad, a confirmation tone will indicate your line is in the question queue. You May Press Star two if you would like to remove your question from the queue for participants using speaker equipment and may be necessary to pick up your handset before.
Speaker Change: Pressing the star Keys, one moment, please while we poll for questions.
Speaker Change: Okay.
Speaker Change: The first question is from Blah, Blah, Blah and Apache's Alpine from Roth Capital. Please go ahead.
Apache's Alpine: Good afternoon, everyone and congrats on the progress. So we have three questions. So firstly I wanted to talk a little bit about intelligence market.
Apache's Alpine: So this is primarily you know for me to get some clarity the way I look at the market. So you'll see it in the past you know roughly 1000 babies are born every year.
Apache's Alpine: U S intelligence, so even if you assume 15% mortality, we still have 850 babies will need to undergo the stage two surgery every year within four to six months. After the Norwood procedure right. So my question is do all stage one survivors will ultimately qualify to receive lateral Mr. Schoen.
Apache's Alpine: Is there a reason why certain stage one surviving patients would not want lateral Mr. So as you can imagine the reason I'm asking this question because I wanted to understand whether some of the enrollment challenges you're facing would ultimately impact your commercial adoption.
Apache's Alpine: Alright, so well balanced that's a great question and I may have several members of the team to answer. This one I will I'll answer the last part which is regarding the enrollment challenges.
Apache's Alpine: I I really believe that.
Apache's Alpine: Well, everybody see it and everybody loves to see more prediction I think we have actually done a very good job as a company and enrolling in an orphan disease compared to other products. I mean are we are almost 95% enrolled.
Apache's Alpine: If you do the math that means that we have one patient left.
Apache's Alpine: <unk>, which we are confident we're going to get this quarter and we finished the enrollment enrolling 44% of that annual population. In this study that's a sizeable population for an orphan disease and the fact that we were able to enroll it in a matter of less than three years actually it's a it's a very good achievement so in that front.
Apache's Alpine: I know it has been challenging and I know everybody would like to see more clarity I really believe that we have done a very good job and they're going to ask for the market. The market opportunity is very big because we have our exclusion criteria does not exclude a lot of patients, but I will have dr or hair or Natal.
Apache's Alpine: I am sorry, exactly who are the patients are at school, then I will come back and talk about the market opportunity as well.
Apache's Alpine: Okay.
Apache's Alpine: Oh gosh.
Apache's Alpine: Josh.
Josh: Oh, Hi, Hi, while yes, I just wanted to make a comment on.
Apache's Alpine: The trial as well.
Apache's Alpine: I think that I want to emphasize that not only is this trial.
Apache's Alpine: Excellently conducted but it's it is going to be the most rigorous trial conducted in babies with congenital heart disease for cell therapy.
Apache's Alpine: Because of the rigorous design and size of the patient population with regard to the market I I think that.
Apache's Alpine: The majority of children, who go to the states to surgery, the Glen procedure.
Apache's Alpine: We will be considered for for inclusion in an in this treatment once the once approved I think there are very few reasons too.
Apache's Alpine: Not consider.
Apache's Alpine: Treating a child once they make it to stage two if the if the.
Apache's Alpine: If the product is approved the B L. A.
Apache's Alpine: Yeah. So so.
Apache's Alpine: The balance to be to be more specific and tell you how have you been forecasting or market or that market on the commercial opportunity.
Apache's Alpine: Dissipated 1000 babies that are born with this condition every year.
Apache's Alpine: Many of them actually end up going direct aid because as you know that the first stage surgery happened within the first week of birth.
Apache's Alpine: And.
Apache's Alpine: Even if 90% of them survived the second stage surgery that that leaves about 900 patients every year within those 900 patients. We assumed is that following the only exclusion criteria. If these patients have a comorbidity, which was a valve.
Apache's Alpine: <unk> moist tricuspid valve issues that population that had that comorbidity is less than 10%. So here is another out of that 900, you can take out about 10% of the population that leave you with the 800.
Apache's Alpine: Those 800 patients we assume that some of them for whatever reason they choose elective lead that they would not want to have that therapy. So our our penetration that assume about 65% two thirds of the market will happen, which we believe that represents a very good sizable opportunity.
Apache's Alpine: Two are in the market place and better than the most conservative pricing estimates will be north of about half a billion dollars just in the U S and that's not including any patient from outside the U S.
Speaker Change: Alright, great. Thanks for the detailed answer I just wanted to move to the second question and I also have a follow up so my second question about the F. T I know it.
Speaker Change: It doesn't mean, if there has been in the news many times in the last few months for many reasons and one of which is leadership changes at the CBER Division and this is the division you'll be ultimately dealing with when you approach your BLA filing and potentially for the P. Army. So my question is have you seen any indication or.
Speaker Change: What sort of any signals whatsoever.
Speaker Change: Basically makes you believe you'll be able it will be impacted by either leadership changes or by policy changes at the FDA.
Speaker Change: I'm, so sorry to be direct we have not seen anything I mean, the most recent interaction with the FDA was in March 13th of course before that naming of the new Siebert director.
Speaker Change: But the interaction has been very supportive.
Speaker Change: The FDA has been very collaborative the team remain very supportive of our plans and we have minutes a game that we have agreed so there is nothing here to.
Speaker Change: Allow us to assume otherwise from an information that we have I also want to tell you that regardless of any leadership change my our philosophy at launch of her own. It we have to be buttoned up in our application and that's why we're sorry, we're having a head start.
Speaker Change: We want us to absolutely submit the best possible BLA ready.
Speaker Change: Ready with all the information that is needed for the two absolutely pasture with the drug approval and get that approval on the first one.
Speaker Change: Alright, great and one last question on this is for Devon, David Nice to meet you. So as Youre contemplating you know manufacturing in house. This is contracting in Seattle. So I was wondering if you could provide some commentary on your current capacity and potentially you know your plans to scale our operations, assuming you know you're ultimately going to be.
Speaker Change: For manufacturing as you think about you know transitioning to a pre commercial company. So if you can lay out some of your you know the current plans and maybe potentially the investments that might be necessary to rapidly advance towards commercialization stage. There would be helpful. And also in this regard if you can tell us if there is any bottlenecks that would limit you.
Speaker Change: Our freedom to operate because when you go to commercial I wanted to better understand you know, whether youre hinging or someone else's patents. So any commentary would be helpful.
Speaker Change: Of course.
Speaker Change: So regarding our our capacity so.
Speaker Change: One lots of layer Mr. So we believe is enough to.
Speaker Change: So news at least.
Speaker Change: <unk> hundred doses for this product so what's the commercial markets and the number of babies that are born this year, we believe that the you know the.
Speaker Change: Current yield sizes are.
Speaker Change: One that will be able to supply the drug commercially.
Speaker Change: And that's what our current manufacturing process.
Speaker Change: The investments that we're making is primarily and ramping up our G&P systems looking at facilities, which could include a CMO that has commercial capabilities.
Speaker Change: And capable of receiving a BLA.
Speaker Change: Additionally, we are ramping up.
Speaker Change: Internal staff is all experts who have gone through this process before.
Speaker Change: Two.
Speaker Change: Ensure timely BLA.
Speaker Change: Alright, that's it from me. Thank you team and our advanced congrats to wrap up Lp's two enrollment.
Speaker Change: Thank you Paul violin.
Speaker Change: The next question is from rents Elvira Zhou from H C. Wainwright. Please go ahead.
Speaker Change: Firstly, I was wondering if you could provide us with some feedback, some color on the applicability of value-based pricing to determine the best possible pricing paradigm for Laramester Cell in each L.H.S.
and in particular, if value-based pricing is applicable.
Speaker Change: If it is applicable in what context, how might this factor into how pricing is ultimately determined? And if you look at recent drug launches in the rare and ultra rare disease space,
Speaker Change: Are there any that are currently ongoing or that we expect later this year that might provide appropriate precedent benchmarks?
Speaker Change: for the pricing of Laramespacel in HLHF, if ultimately approved. And then the second one is a very minor one, but I was wondering if you could comment on the timing with which
Speaker Change: Laramester Sel would need to secure regulatory approval in order to be eligible for a PRV, assuming that the PRV program overall is not reauthorized by Congress.
Thank you.
Speaker Change: All right, so Ram, let me answer the two questions. They're regarding the PRV. Right now the current PRV expired in September of next year. And I don't think that if it doesn't get renewed
Speaker Change: This means that it will expire before we get a market authorization for Lanermaster Selvaraju.
Speaker Change: Two sides of the aisle, and I can tell you there is a big support on both sides of the aisle to support the program or the initiative is called Give a Kid the Chance Act.
Speaker Change: and I believe once the Congress start to put all their priorities and budgets and things like that, it will get reauthorized and for an extension, and in this case we will be eligible to get it. So that's regarding the PRV and what is the likelihood?
Speaker Change: and Full Transparency. And we are working, and again, as I said, this is the very top priority for us.
Speaker Change: and we are part of Bio and CLS and by Florida and many other organizations and they know how
Regarding the value-based pricing and the reference pricing.
Speaker Change: So, the only product that is currently the closest product to our product in the inter-air disease is the product
Speaker Change: that was approved for me the BLAAD for GBA today, Graphless Associates Disease, which is also [inaudible]
Speaker Change: and Orphan disease as well, the cost of therapy I believe as they have announced and again I'm just speaking out of their own disclosure is about 1.5 million for the course of treatment.
Speaker Change: So, if you use this as a benchmark at the closest, you can throw your pricing. As I said, we have not made a final pricing decision on any of these things.
Speaker Change: But we are one time use only so there is no chronic utilization and we believe that we can command a good
Premium, especially that we're looking for.
Speaker Change: So, overall we believe that we will be able to have a very good story around value based pricing if we need it.
Speaker Change: I believe that we will be able to generate a substantial revenue for the company.
Thank you very much.
Speaker Change: The next question is from Michael Okunewitch from Maxim Group. Please go ahead.
Michael Okuniewicz: Hey guys, thanks so much for taking my questions today, and we're asking all the progress you've made.
Thank you, Michael.
Dr. Raghuram Selvaraju, Raghuram Selvaraju,
Speaker Change: I guess to start things off, I'd like to see if you could talk a little bit about what sort of a sales force you would need in ATLHS, and in particular among those 12 centers that are running the Alpha2 study, how much of the volume of the Glenn surgery actually passes through these centers.
Speaker Change: Alright, that's a great question and I'm honestly, I'm so excited.
Speaker Change: In this call, to start to see that we're getting commercial question because that's what we're excited about here also in Longeveron. So to answer your question is I will tell you Michael I came from the commercial world and we used to see. Bye.
Speaker Change: There is a rule called 80-20, so 20% of our customer generate 80% of our business and I would tell you that 12 centers that we have used in our clinical trials, they actually conducted the majority, almost 80% of these surgeries.
Speaker Change: So, that's a big plus for us is that we are actually conducting a trial in high volume centers.
Speaker Change: Surgery, or set of surgeries as you know, and having those relationship established, then the experience with the drug will give us a huge benefit. The number of...
Speaker Change: Exertions who are or Pediatric Cardiac Surgeon who perform these surgeries in the US, they are about less than 50 on my last count. And we anticipate a need for more than four or five cells for us across the United States.
Speaker Change: 12 to 15 people and that's assume manage market support, marketing support, patient services support and all of that. So it's a very focused organization and
Speaker Change: That's the beauty of operating an orphaned disease. You really don't need larger resources to be able to service the patients who suffered from these diseases.
Yes, thank you. That's certainly a good answer to hear.
Thank you. Bye-bye.
Speaker Change: and then just talking a little bit about the plans for scaling out your manufacturing. I'd like to get a sense of it.
Speaker Change: How much of the stage of partnering discussions in all timers might impact the decision to go for a CDMO or to build out in-house, or is that not really a factor here?
Speaker Change: The so definitely I can absolutely then I Devon can add this we absolutely know for fact that we cannot manufacture
Thank you for joining us.
Speaker Change: for Alzheimer's within our existing facility, because the plan that we aligned with the FDA on.
Speaker Change: is that we're going to do a pivotal phase two, three, as you hear then, our call today, which is a great plan, because that is probably the fastest and most accelerated path that they have agreed to in that area, which we are really excited about.
Speaker Change: But that also means that clinical trial material, even for that study, will have to be produced in a commercial level facility, which in this case, we know it cannot be our existing facility and either CDMO or we're going to have to...
Speaker Change: I see if the partnering discussion lead to a partner with a facility that they can support that, but definitely not going to happen in our facility in Miami.
Thank you.
Speaker Change: Do the decisions of the CDMO for HLHS and Alzheimer are tied together? To a very remote, I think we're looking at them as two distinct criteria, but definitely one of, if we're going to move that CDMO route for HLHS.
Speaker Change: We definitely, that's one of the questions that we have it on the table. Can this in the future also help us at a larger scale with Alzheimer's disease?
Devon, do you want to add any comments on this?
Dr. Raghuram Selvaraju, Raghuram Selvaraju
I'm just just to reiterate that you know the
Speaker Change: While we're looking at multiple different options, the CDMO option is one of them and the CDMO that we have been looking at do have the capability for a larger scale manufacturing for an indication like Alzheimer's.
Speaker Change: and many more. Thank you for watching. We hope you enjoyed this video. If you did, please give us a thumbs up and subscribe to our channel. See you next time.
Speaker Change: Alright, thank you for that. The one last one for me and I'll hop back in the queue.
Speaker Change: Just I'd like to see if you can help me understand a little bit more what the adaptive protocol might look like for the Alzheimer's study. Would that be something like a phase two portion that confirms powering and then a phase three portion for approval or would it just be one single straight through protocol?
Speaker Change: Yeah, so I'll give you a high level and that's how I can get into the details. So the agreement for the FDA is that we're going to do one single study.
This study will have about 1600 patients or 1650.
Speaker Change: and the first portion of the study, which we call a phase two adaptive, will have 600 patients. It will be a three-arm study, one placebo, and two different dosing regimen for Latin
Speaker Change: And at the end of the period of the 600, we're going to do an interim analysis.
Speaker Change: Looking at the same primary endpoints that we would use for the end of the study. If that end-from-analysis at the 600 is positive,
Speaker Change: The FDA agrees that they will give us an approval based on the 600 with the principle to continue the study for the 1650 to get larger patient set after the approval is complete.
Speaker Change: So that's the plan that we agreed to with the FDA and Nataya, feel free to give more details or answer any additional questions from Michael.
Natalia: Absolutely, Raya. Just to add what Raya mentioned already, their idea of...
Speaker Change: Adaptive Design, yes, is to do seamless, so they are not interrupted face to.
We are moving directly to phase three.
O 72 weeks of treatment.
Speaker Change: and they are talking about as I will mention 600 patients, 200 patients are armed.
Speaker Change: and now decisions will be made based on the conditional power. At that time we have opportunities either to go for approval, traditional approval, if there is evidence of efficacy or we can choose another dose for the complete trial.
Speaker Change: Patients on the study complete 72 weeks of treatment with the full analysis at the end of the trial, so...
Speaker Change: This is the opportunity to have, first of all, to have one trial operationally seamless. We don't have to do, you know, all this separational behavior, it's that there is going to be continuous
Cheatman, and also to have this opportunity to have a traditional approval sooner.
Oh yeah.
Dr. Raghuram Selvaraju, Raghuram Selvaraju,
Speaker Change: All right, thank you very much for the additional color and once again from grass and all of the progress you've made over the past several quarters.
Dr. Raghuram Selvaraju, Raghuram Selvaraju
Thanks Michael.
Operator: There are no further questions at this time. I would like to turn the floor back over to while Hashad for our closing comments.
All right.
Operator: Well, thank you, operator, and thank you all for attending our call today. We greatly appreciate your interest and support and look forward to updating you on our progress. Thank you.
Operator: This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation.
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