BioCardia Inc. Q1 2023 Earnings Call
Speaker 2: Good day, ladies and gentlemen. Thank you for standing by. Good afternoon and welcome to the Biocardia 2023 first-quarter conference call. At this time, all participants are in the listen-only mode.
Speaker 2: Should you need assistance, please signal a conference specialist by pressing the star key followed by zero. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press star, then one on your telephone keypad. To withdraw your question, please press star, then two.
Speaker 2: Participants of this call are advised that the audio of this conference call is being broadcast live over the internet and is also being recorded for playback purposes. A webcast replay of the call will be available approximately one hour after the end of the call through August 10, 2023.
Speaker 2: I would now like to then call over to Miranda Peto of Firecardia Investor Relations. Please go ahead, Miranda.
Speaker 3: Good afternoon and thank you for participating in today's conference call. Joining me from BioCard is leadership team R Peter Omen, PhD, President and Chief Executive Officer and Deputy Crown, the Communist Chief Financial Officer.
Speaker 3: During this call, management will be making forward-looking statements, including statements that address bio-carriers expectations for future performance and operational results.
Speaker 3: Reference to management's intentions, belief, projections, outlook, analyses, and current expectations.
Speaker 3: such factors include, among others, the inherent uncertainties associated with developing new products, technologies, and obtaining regulatory approvals.
Speaker 3: We're looking statements involve risks and other factors that may cause actual results to differ materially from these statements. For more information about these risks, please refer to the risk factors and cautionary statements described in Biocardial Reports on Form.
Speaker 3: 10K files on March 29, 2020.
Speaker 3: The content of this call contains time-sensitive information that is accurate only as of today, May 10, 2023.
Speaker 3: Except those require our law, the company disclaims any obligation to publicly update or revise any information to reflect events or circumstances that occur after this call.
Speaker 3: It is now my pleasure to turn the call over to Dr. Peter Almond, P.H.C. by our Cardius President and CEO . Peter, please go ahead. Thank you, Miranda, and good afternoon to everybody on the call.
Speaker 4: It has only been six weeks since our last call at the end of March, and BioCardi is steady as she goes.
Speaker 4: We have some near-term catalysts that we can detail progress towards as well as the broader opportunities for success ahead.
Speaker 4: Bio-Carty's current efforts are focused on advancing its autologous and its allogeneic cell therapy platforms to treat significant unmet cardiovascular impulmonary diseases.
Speaker 4: Specifically, Schema cart failure, chronic malchaloschemia, and acute respiratory distress syndrome.
Speaker 4: All of our cell-based therapies involve local delivery of the therapeutic to the heart or lungs where we intend them to act locally.
Speaker 4: Part failure is an enormous unmet need that affects more than 26 million people worldwide.
Speaker 4: The latest blockbuster drugs in the Schema Card failure of reduced ejection fraction provide great benefits to patients.
Speaker 4: but don't appear to have much of an impact on mortality.
Speaker 4: Patients in the published results of the pivotal trials for these new drugs have a cardiac mortality of roughly 7% and an all-caused mortality of 10% per year regardless of whether they were treated or controlled patients.
Speaker 4: This makes clear that a heart failure is still a problem in great need of new therapeutic solutions. Our autologous mononuclear self-therapy platform, which we call Cardi-Empself therapy, is being advanced in two cardiac clinical indications.
Speaker 4: In preclinical studies, cardiac mononuclear cell therapy has been shown to release proteins locally within the tissues to facilitate cardiac recovery after heart damage with improvements in heart perfusion and contractile function.
Speaker 4: All known previous clinical studies, similar to the approach we are taking in our two lead cardiac and self therapy programs, have shown patient benefits on average.
Speaker 4: In some of these studies, including our own, the benefits have been remarkable.
Speaker 4: The FDA has supported this promise by granting breakthrough device designation to cardiac cell therapy in the indication of a Schema card failure of reduced ejection fraction.
Speaker 4: Advancing this and our other three therapeutic candidates is what we are all about.
Speaker 4: Our efforts to complete the Cardiamp autologous cell therapy pivotal clinical trials for the indications of heart failure or BCDA01 and chronic myocardial ischemia or BCDA02 are furthest along clinically.
Speaker 4: The Cardi Amp South Therapy Heart Failure Trial, or BC-01, is a Phase 3-260 patient randomized controlled clinical study intended to provide the primary data to support safety and efficacy in pursuit of marketed clearance.
Speaker 4: Clinical investigators at 20 active partner sites across the United States and Canada have enrolled 120 patients to date with 10 additional control patients having crossed over to receive therapy.
Speaker 4: We feel there is clearly increased momentum here, potentially driven by the clinical data presented at the American College of Cardiology in March, showing 100 percent survival and patient benefits across many endpoints at two years, including a 35 percent increase in the heart, left, and trickler ejection fraction.
Speaker 4: As many know, we have been working on implementing an adaptive statistical analysis plan to the trial with distinguished consultants, including former FDA leaders and a respected statistical consulting group.
Speaker 4: In our last March 29 call, we were headed into a meeting scheduled to discuss the FDA's comments on March 31st.
Speaker 4: In our last March 29 call, we were headed into a meeting scheduled to discuss the FDA's comments on March 31st. That meeting went well.
Speaker 4: The discussion of the Adaptive Statistical Analysis Plan as provided was well received by the agency's statisticians.
Speaker 4: The FDA's primary concern was whether we would have enough safety data to support approval in an indication as large as a scheme of cart failure with reduced ejection fraction if the trial was stopped early for efficacy.
Speaker 4: The agency's primary concern with safety.
Speaker 4: was with respect to the safety of the delivery of the cells. We shared with the FDA that we feel we do have sufficient data already with 353 interventions with our delivery system in the clinical indication of ischemic heart failure reduced ejection fraction to date.
Speaker 4: and 129 patients treated so far, with the cells being advanced in the CARDI-AMP trial. The FDA was unaware of this more expensive data set, and we agreed to detail it for them.
Speaker 4: We also work with them on creative ideas to further enhance the safety experience ahead.
Speaker 4: After summarizing minutes from our perspective and acceptance of the FDA minutes provided, we submitted a revised supplement for the Adaptive Statistical Analysis Plan incorporating all of the comments from the agency on April 26.
Speaker 4: We now anticipate a response from the agency on May 26th.
Speaker 4: The next pre-specified formal data safety monitoring board review is anticipated at the end of June 2023. We still believe it is likely we will be able to have the adaptive statistical analysis plan in place for the meeting. The specific details of any potential adaptive statistical analysis plan are available. Thank you. Thank you. Thank you. Thank you. Thank you. Thank you. Thank you. Thank you. Thank you.
Speaker 4: In combination with any modifications to the data safety monitoring board charter, we'll dictate what happens at this next and subsequent data safety monitoring board reviews. As the CardiM South Therapy Hard Failure Trial was over 90% powered for success with a range of 86 to 126 patients. There is potential that the trial could be its primary efficacy endpoint on the patients.
Speaker 4: that have been enrolled to date. Although the next data review event has potential to be a great success if the trial has stopped early for efficacy.
Speaker 4: in a role to date. Although the next data review event has potential to be a great success at the trial stopped early for efficacy, the review will also be a success.
Speaker 4: should the trial continue as planned. Our second therapeutic program is with the same autologous self every the treatment of chronic malchaloschemia with refractory angina or BCDO2.
Speaker 4: The CARDI-AMP chronic myocardioschemia trial is a phase 3 multicenter, randomized, double-blinded, whole study of up to 343 patients at up to 40 clinical sites.
Speaker 4: A sufficient number of patients to complete the open labeled rolling cohorts have already been consented.
Speaker 4: It has anticipated this trial report out, the Openly will roll in cohort results in 2023.
Speaker 4: As we have shared previously, in July , we had our second consultation with Japan's pharmaceutical and medical device agency regarding registration of cardi-amp cell therapy for ischemic heart failure.
Speaker 4: Biocardia still expects to complete a formal submission towards Japanese approval in the second quarter of 2023.
Speaker 4: In Japan, another autologous South Airbeez has received conditional approval for heart failure.
Speaker 4: In Japan, another autologous cell therapy has received conditional approval for heart failure based on seven patients treated.
Speaker 4: and these treatments require open heart surgery to access the heart.
Speaker 4: Although our clinical data is from overseas,
Speaker 4: rigorous control trials and have a minimally invasive procedure with our Helix biotherapeutic delivery system. Our cell processing platform is approved in Japan already for therapeutic applications for non-cardiac indications by our partner Zimmer Biomed and our minimally invasive Helix biotherapeutic delivery system
Speaker 4: is approved in the European Union. Our feeling is that there are many reasons that Japan PMDA should be inclined to approve Cardiampsal Therapy for the benefit of patients and to continue to advance Japan's leadership in regenerative medicine therapies.
Speaker 4: Now, I'd like to move to our two allogeneic cell therapy product candidates based on our allogeneic neuro-kind-of-one receptor positive mesenchymal stem cell platform. These are off-the-shelf cells from young healthy donors intended to be expanded to produce many doses for many patients.
Speaker 4: The neurokinin-1 receptor positive mesenchymal stem cells are particularly interesting as neurokinin-1 is the primary receptor for substance P, an important neuropeptide mediator of inflammation, which plays a central role in both heart failure and regenerative processes following myocardial injury.
Speaker 4: I encourage listeners to Google Substance P to understand why advancing the messing-kindle stem cells that bind to this neural peptide is exciting.
Speaker 4: Our allogeneic mesenchymal stem cell program in ischemic etiology, heart failure of reduced ejection fraction is designated as BCD03.
Speaker 4: This is a Phase 1-2 multi-center randomized double-blind and controlled study of up to 69 patients designed to assess the safety and efficacy of this therapeutic candidate. The investigational new drug application was approved by the FDA in December 2022.
Speaker 4: And the trial is designed for patients ineligible for the company's Phase III Carding at Part Failure trial, studying autologous center.
Speaker 4: Clinical grade, allogeneic cells with a manufacturing or sunny-vailed facility and are ready for use. These cells will be delivered under the protocol without a proprietary minimally-invasive biotherapeutic delivery system.
Speaker 4: We still expect to begin enrolling patients in the second quarter of 2023, which has seven weeks remaining.
Speaker 4: our allogeneic mesenchymal stem cell program in patients recovering from acute respiratory distress syndrome
Speaker 4: which we have designated BC-04 was approved by the FDA in April 2022 to treat patients. The trial is a Phase I multi-center open-level study of up to nine patients.
Speaker 4: While the number of patients with COVID-induced ARDS has decreased, ARDS unrelated to COVID is still significantly impacting patients.
Speaker 4: The company intends to work with the FDA to modify the study eligibility criteria to include these patients.
Speaker 4: In this trial, increasing dosages of the cells will be initially evaluated, and then the optimal dose will be taken to phase two in a randomized study in adult patients recovering from ARDS. This therapy is intended to address the enormous unmet need of sustained local and systemic inflammation after a patient is taken off respiratory support. The goals of accelerating recovery, enhancing survival, and reducing both relapse and re-hospitalization.
Speaker 4: Clinical grade cells are also ready for use in this study. The ARCH trial is expected to commence following the initiation of BC-03, studying these allogeneic mesenchymal stem cells for heart failure.
Speaker 4: In summary, we are advancing four clinical stage therapeutic product candidates that address important unmet cardiac and pulmonary diseases.
Speaker 4: based on our Autologous and our Allergen-AXL therapy platforms.
Speaker 4: From these therapeutic development efforts, we now have four active business development initiatives.
Speaker 4: First, is partnering our CardiM South therapy platform internationally.
Speaker 4: Second, is licensing out our clinical stage neurocine-in-one receptor positive mesenchymal stem cell platform. For other clinical indications, it's shown promise with other mesenchymal stem cell preparations.
Speaker 4: Third is licensing our catheter based bio therapeutic delivery systems for cell gene and protein therapy candidates to the heart, such as in the blue lock relationship we began last year.
Speaker 4: And fourth is monetizing our Avance Transceptile Introducer Sheet product.
Speaker 4: We are looking forward to announcing an additional patent issuance related to our Helix Bio-Theor Product Delivery Platform and feel very good about our broader intellectual property estate.
Speaker 4: I will now pass the call to David McCleum, our CFO , who review our Q1 2023 financial results. David?
Speaker 5: Thank you, Peter, and good afternoon, everyone. Revenues were approximately $64,000 for the three months and it March 31st, 2023, comparable to the $60,000 for the three months and it March 31st, 2022.
Speaker 5: Research and development expenses increased to approximately $2.4 million in the first quarter of 2023 compared to approximately $2.2 million in the first quarter of 2022. Primarily due to expected increases in support of the Cardiant Heart failure trial. S.G.A. expenses remained at approximately $1.2 million in the first quarter.
Speaker 5: was approximately $2.6 million as compared to approximately $2.9 million in the first quarter of 2022.
Speaker 5: Biocardia ended the quarter with approximately 4.9 million in cash and cash equivalence, providing runway in the Q3 without additional capital or funding for business development activities that Peter touched on in his remarks.
Speaker 5: This concludes management's prepared comments. We're ready to take questions.
Speaker 2: Thank you. We will now begin the question and answer session. To ask a question you may press star then one on your touchtone phone. If you are using the speaker phone please pick up your handset before pressing the keys. If at any time your question has been addressed and you would like to withdraw your question please press star then two. At the time we will pause momentarily to assemble our roster.
Speaker 2: Our first question comes from Joe Pan-Genees with HC Vaynewright. Please go ahead.
Speaker 5: Good afternoon. Thanks for taking the question. Two primary questions, Peter. So first, with regard to BCDA01, nice to hear you're making progress with your FDA discussions. So for the current patients enrolled, you said you had 10 control patients that had crossed over.
Speaker 6: So can you remind us the criteria for crossover and then following crossover, what could be the potential impact of new or concomitant meds?
Speaker 4: Okay, so that's, that's, well, thank you, Joe. I was waiting for a second question there. I think I've got the breakdown. So first on the, so appreciate the question and I really appreciate you being on the call. So on BC01, the 10 crossover patients.
Speaker 4: crossed over after they met their two-year follow-up endpoint in the trial and essentially exited the trial. The requirement for those patients is substantially what it is to be included in the trial, though I think the workup is a little bit less.
Speaker 4: because some of them may have deteriorated. There is actually a patient that you can see who has crossed over on our patient facing website, which is CardiAMP.com. And so yeah, we've had 10 patients crossed over. The FDA approved this last year, I think, or maybe it was the year before, because of the safety profil they're seeing in the trial.
Speaker 4: in the sense that these patients enrolled in the trial should be given the option if they and their physicians think it makes sense for them to proceed.
Speaker 4: Got it. Got it. The patients. Sorry, sorry. The last piece is these patients are then followed for another year. And so for the trial, they provide additional safety data for us, which, you know, as you heard on my FDA comments, is valuable for the FDA.
Speaker 4: And I actually may use that to note that if you consider the FDA comments I shared, those comments were all around stopping the trial early, they want to have sufficient safety, but that's fundamentally to provide support for approving the therapy.
Speaker 6: So that's actually a really nice conversation to be having. No, that's good. Thanks. And then the second question really is around those FDA discussions. Obviously, I know you can't be too specific right now since it's ongoing and it's iterative, but, you know, just curious if you could take some broad strokes about, I guess, your prepared comment surrounding ideas to enhance the safety package.
Speaker 6: Since you can't increase patient knowledge.
Speaker 4: Well, I guess, yes, I can share that. Well, actually, it's probably best to wait until we have the final details from the agency. It is only a few weeks out, and we can share it then. But the key is patient numbers.
Speaker 4: for approving the therapy. And I'll share with folks when we design this trial. You know, that was one of the primary considerations because of the power we had going into it is that we wanted to have enough patients to enroll in the trial, to enable the agency to have comfort, to approve
Speaker 4: Again, our goal here at BioCard is not just to deliver a positive trial. Our goal is to deliver a product for these patients and the physicians who care for them. And at that point in time, the dialogue was around what number would be acceptable for the agency. And the...
Speaker 4: they wouldn't provide us with a specific number. But as we've gone downstream, it's our safety profile in this program and in our second program that also help us. So I guess let me detail the results of the Adaptive Susicic Analysis Plan when we have the FDA's blessing for it.
Speaker 4: And I'm hopeful that that will be in just a few short weeks here. There enough. Appreciate feedback.
Speaker 2: Thank you, Joe. Appreciate the questions. The next question comes from Kumar Rajavit. A rod capital please go ahead.
Speaker 7: Thank you for taking my questions and congratulations Peter from all the progress.
Speaker 7: So with respect to the feedback that is expected from the FDA at the end of the June , the expectation is that you have taken care of whatever the questions they had in the feedback they have given before and we are pretty much close to the final stage in terms of, you know, getting agreement with FDA.
Speaker 7: with regard to the adaptive statistical plan.
Speaker 4: So thank you, Kumar, for the question and also for being on the call. That is our sense. If for those who have been involved with conversations with the FDA, they ask more questions than they provide real clear guidance on where to go next. But...
Speaker 4: We have a pretty sophisticated team that's come into this conversation. We are working with literally a world-class regulatory group. Our own internal regulatory team, which is...
Speaker 4: nothing short of phenomenal, and perhaps the most respective statistical analysis plan developers with respect to an adaptive design that from the agency's perspective. So my sense is we went into this
Speaker 4: very seriously and I think the agency recognized that. And so they, everything, every single question that they raised and that which we discussed, I think was extremely well addressed. So that said, you know, we can never predict the future with the agency.
Speaker 4: they're trying to do their best by all the folks in the United States. And if they do come back to us with follow on questions, we will address them. But our hope is that they will come back to us and bless the statistical analysis plan. And then we will roll it into
Speaker 7: the next Data Safety Monitoring Board review, which is currently being scheduled for the end of June . Okay, so the expectation is that by that meeting, you will have more clarity. And also, with regard to the PMDA submission, maybe you can just provide us a little bit with regard to, once you submit it, what is the...
Speaker 7: expectation and the timeline there and also in that context you mentioned about two out licensing or product distribution opportunities maybe you can talk a little bit about that. Thanks so much. Appreciate the questions Kumar. So on PMDA submission so we've had two separate consultations with
Speaker 4: This is the pharmaceutical medical device agency in Japan. And this is around securing an approval for the Cardiamps cell therapy platform for the treatment of a Schemeck etiology heart failure of reduced ejection fraction, which is our lead program. And the conversations we've had with the agency.
Speaker 4: you know, so far have been all focused on really the same question.
Speaker 4: is we are keen on securing approval based on the data that we already have in hand.
Speaker 4: not waiting to complete the pivotal trial in the United States, but rather based on the quite significant data sets and experience and the number of approvals we already have. And so all of the questions from PMDA and our two consultations have been...
Speaker 4: focused around clarity of certain issues, certain procedural elements. We've been impressed by them and they've been rather sophisticated. But, you know, the submission process where in the midst of, you know, does have some challenges with respect to translation delays.
Speaker 4: because a rather large document will be provided to them containing substantially the information which they've already seen in different formats in Japanese for them to consider. Our expectation is after we submit this to them that we will have them come back to us with
Speaker 4: a series of questions, which we'll have to respond to in short order. And our hope is that at the end of the day, the key issue is, will they approve this without pre-approval data in Japan? And...
Speaker 4: You know, the four words we are, we are any four, are your proposal is acceptable.
Speaker 4: And so that's really where we're headed for on this. And we think we've got all of the support we need, but again, we're working with regulators that complicates it.
Speaker 4: So, and that's about all I can share at this point in time. Again, as this bio-cardi is approach, we have multiple different experience consultants we're working with. We have incredibly distinguished, conational principal investigators in Japan that we're hoping to be
Speaker 4: working with on a post-marketing study after we have approval, in addition to just incredibly distinguished folks on the regulatory and processes in Japan. So, it's been a delight. On outlicensing, I detailed the various categories of outlicensing. We have a lot of activity currently.
Speaker 4: And our senses we have capital to get through a couple of deals as well as all of these milestones we're detailing for BC-01, 2, and 3. So I'd say stay tuned on the out licensing. Unfortunately, they're similar to the FDA that we can't-
Speaker 4: you know, be guaranteed what their timelines are, what their response is going to be. But by being buttoned up, you know, it helps.
Speaker 4: And I think we are a buttoned up organization. So again, I'll have to say stay tuned for those discussions. They do involve out licensing products and distribution deals around product candidates that we have that don't really reduce So you don't think we are nice but, I think the?? of all times. And we want to see that there is left enough noise running on the phone. So you don't think we are nice but, I think the?? of all times.
Speaker 4: the value of anything we're currently doing in the United States. They're all additional value propositions.
Speaker 2: We really help those thanks so much. I appreciate the questions, Kuhnwaha. Have a great afternoon. Again, as a reminder, if you have a question, please press star then want to be joined into the queue.
Speaker 8: Our next question comes from Laura Surreal with Alliance Global Partners. Please go ahead. Hello, this is Laura calling in for Jim Moloy from AGP. Thank you for taking our questions. So for the DC-D-A02 trial, it was mentioned in the previous call that TROB design modifications were being planned in order to speed up enrollment.
Speaker 8: So, what's the overall status on any of these changes that are being proposed to the FDA? And then also for the BCDA 04 trial, when do you expect to meet with the FDA as well to modify the study eligibility?
Speaker 4: Good questions, Laura. Appreciate you being on the call. So the answers, I have easy answers, but I'll give you more calls. So BC-02, we're not going to engage the FDA in changing the trial design until we complete the initial role in cohort, which is imminent.
Speaker 4: And that trial design is going to involve substantial structural changes in the inclusion, exclusion as well as the primary endpoint. This trial, I guess I would say for folks on the call, is substantially equivalent to the trial that Baxter Healthcare advanced in this indication focused solely on CD34 cells.
Speaker 4: and everybody criticized Baxter HealthCare for how long the trial took for them to enroll 90 patients, and then they ultimately just stopped the trial and walked away from it, even though they had great efficacy signals that ultimately were published. We now understand the difficulties Baxter was having. And so, thank you for information on the information Alright.
Speaker 4: working with our KOLs and physicians and pursuing the realm of what's possible. And my sense today is we're going to go after...
Speaker 4: an endpoint that's primarily image and self-assessment driven.
Speaker 4: IE probably using PET and using Seattle, and you're not a questionnaire versus using the Cardio Pulmonary Exercise Time. And the good thing for investors is eliminating the Cardio Pulmonary Exercise Time criteria, which we may still measure at baseline and follow up,
Speaker 4: making it less important will greatly reduce the cost of this trial. And that's another nice advantage. So the trial will go faster. We'll have data that's more objective for physicians. And you know, we expect that on every front it will be advantageous to us.
Speaker 4: So that's on the BCDO2. On BCDO4, the dynamic there is really easy. That's a quick supplement to the agency and I do not expect any agency pushback whatsoever. Essentially, when we launched that program, we were going after patients.
Speaker 4: who had acute respiratory distress syndrome secondary to having COVID. Since then, as we all know, there's not a whole lot of patients with COVID who are winding up on ventilator with acute respiratory distress syndrome. And so by eliminating the requirement to have had COVID before,
Speaker 4: We'll be going after a classic arts population. We're also awaiting this a very large NIH study coming out shortly in this space, being led by the University of California San Francisco. And so as we make that supplement, we're also awaiting that data. So just because of bandwidth issues, we...
Speaker 4: as after the BCDO 3 trial getting started.
Speaker 4: no further questions I would now like to turn the conference back over to Peter Altman for any closing remarks. I want to thank all of you for participating in today's call and for your interest in biocardia.
Speaker 4: for their questions, I would now like to turn the conference back over to Peter Altman for any serving remarks. I want to thank all of you for participating in today's call and for your interest in bio-cardia. We do look forward to sharing our continued progress. Thank you.
Speaker 2: Stay healthy, be kind, and have a wonderful afternoon. The conference has now concluded. Thank you for attending today's presentation. You may all now disconnect.