Lisata Therapeutics, Inc. (LSTA) Q4 2024 Earnings Report, Transcript and Summary

Welcome to the Lisata Therapeutics' Full Year 2024 Financial Results and Business Update Conference Call. Currently, all participants are in a listen-only mode. Following management's prepared remarks, we will hold a Q&A session. [Operator Instructions]. As a reminder, this call is being recorded today, Thursday, February 27, 2025. I will now turn the call over to John Menditto, Vice President of Investor Relations and Corporate Communications at Lisata. Please go ahead, sir.

Thank you, operator, and good afternoon, everyone. Welcome to Lisata's full year 2024 conference call to discuss our financial results and to provide a business update. Joining me today from our management team are Dr. David Mazzo, President and Chief Executive Officer; Dr. Kristen Buck, Executive Vice President of Research and Development and Chief Medical Officer; and James Nisco, Senior Vice President of Finance and Treasury and Chief Accounting Officer. Shortly before this call, we issued a press release announcing our full year 2024 financial results, which is available under the Investors and News section of the company website, along with a webcast replay of this call. If you have not received this news release or if you'd like to be added to the company's e-mail distribution list, please subscribe to the e-mail alerts on the company website or e-mail me at jmenditto@lisata.com. Before we begin, I remind you that comments made by management during this conference call will contain forward-looking statements that involve risks and uncertainties regarding the operations and future results of Lisata. I encourage you to review the company's filings with the Securities and Exchange Commission, including, without limitation, its Forms 10-Q, 8-K, and 10-K, which identify specific risk factors that may cause actual results or events to differ materially from those described in the forward-looking statements. Furthermore, the content of this conference call contains time-sensitive information that is accurate only as of the date of this live broadcast, Thursday, February 27, 2025. Lisata Therapeutics undertakes no obligation to revise or update any statements to reflect events or circumstances after the date of this conference call. With that, I'll now turn the call over to Dr. Mazzo. Dave?

Thank you, John and good afternoon, everyone. It's a pleasure to be here again to provide an overview of Lisata's recent business highlights, discuss our full-year 2024 financial results, and give an update on the progress of our development programs. Over the course of 2024 and now into early 2025, we have advanced our development portfolio centered around our novel product candidate, certepetide, for the treatment of advanced solid tumors and other difficult to treat diseases. The ongoing accumulation of both preclinical data and early clinical data supports our belief that certepetide has the potential to become an integral part of a revised standard of care treatment regimen for advanced solid tumors, including pancreatic cancer, cholangiocarcinoma, glioblastoma, colon cancer, appendiceal cancer and melanoma. We were particularly encouraged by the preliminary results from Cohort A of the ASCEND trial and the iLSTA trials presented at the 2025 ASCO GI Symposium in January. These data, while early, provide further validation of certepetide's potential and reinforce our conviction that certepetide could become a critical component of future treatment regimens for these devastating cancers. We are working diligently to advance these studies and explore additional anticancer combinations and treatment strategies. Beyond oncology, we are also excited about the potential of certepetide in other therapeutic areas. We have initiated the preclinical investigation of certepetide as a potential therapeutic agent for the treatment of endometriosis, the disease which affects hundreds of millions of women worldwide and for which there remains a pronounced unmet medical need. Looking ahead, we anticipate 2025 will be a data rich year for Lisata. We have several key milestones on the horizon and will share our progress and key findings as they become available. Following the review of our financial results, Dr. Kristen Buck, our Chief Medical Officer and Head of Research and Development will provide a detailed update on our ongoing and planned clinical and preclinical programs, including timelines and key objectives. With that, I will now turn the call over to James Nisco, our Senior Vice President of Finance and Treasury and Chief Accounting Officer. James?

Thanks, Dave. Good afternoon, all. I'm pleased to join you today to present a summary of our full year 2024 financial results. Starting with operating expenses. For the year ended December 31, 2024, revenue totaled $1 million in connection with an upfront license fee related to the exclusive license and collaboration agreement with Kuva Labs, Inc. The company did not have any revenue for the year ended December 31, 2023. For the year ended December 31, 2024, operating expenses totaled $23.4 million compared to $25.7 million for the year ended December 31, 2023, representing a decrease of $2.3 million or 8.9%. Research and development expenses were approximately $11.3 million for the year ended December 31, 2024 compared to $12.7 million for the year ended December 31, 2023 representing a decrease of approximately $1.4 million or 11%. This was primarily due to a reduction in expenses associated with the Phase 2b ASCEND trial, which completed enrollment in the prior year, lower spend on chemistry manufacturing and controls and lower equity expense. General and administrative expenses were approximately $12.1 million for the year ended December 31, 2024 compared to $13 million for the year ended December 31, 2023 representing a decrease of approximately $0.9 million or 6.9%. This was primarily due to one-off related severance costs in the prior year associated with the elimination of the Chief Business Officer position on May 1, 2023. A reduction in equity expense, a decrease in Directors and officers insurance premiums and a reduction in spend on legal fees, partially offset by one-off settlement related costs and an increase in consulting expenses. Overall, net losses were $20 million and $20.8 million for the years ended December 31, 2024 and 2023 respectively. It is noteworthy that we continue to make progress according to our plans for our R&D and business activities, while continuing our legacy of prudent capital management and expense minimization. Turning now to our balance sheet and cash flow. As of December 31, 2024, we had cash, cash equivalents and marketable securities of approximately $31.2 million. Based on Lisata's existing and planned activities, the company believes available funds will support current operations into the second quarter of 2026. Lastly, an update regarding the net operating loss sale. Earlier this year, we received $0.9 million in non-dilutive funding as an approved participant of the Technology Business Tax Certificate Transfer Program, sponsored by the New Jersey Economic Development Authority. The program enables qualifying New Jersey based biotechnology or technology companies to sell a percentage of their New Jersey net operating losses and research and development tax credits to unrelated qualifying corporations with a lifetime cap on the tax benefit sales of $20 million. To date, under the program, we have sold $19.6 million in tax benefits for net proceeds of $18.4 million. With that, I will now turn the call over to Dr. Kristen Buck to provide an overview of the company's development programs. Kristen?

Thank you, James, and good afternoon, everyone. Before I review our development portfolio, allow me to summarize some important background information, especially for those who are listening to me for the first time. Despite advances in cancer therapy, many solid tumors are still associated with poor outcomes. Advanced solid tumors such as pancreatic cancer, gastric cancer and glioblastoma multiforme are surrounded by a dense fibrotic tissue known as the stroma, which limits access of most pharmacotherapies to the tumor. In addition, many solid tumors also harbor a hostile tumor microenvironment, which suppresses a patient's immune system and makes it less effective in fighting cancer cells. The combination of a dense stroma and a hostile tumor microenvironment prevents many chemotherapies and immunotherapies from being optimally effective in treating these cancers. This coupled with the fact that most anti-cancer therapies are not efficient in targeting only cancerous tissue defines the major challenges in treating solid tumors. To overcome these obstacles, our investigational products certepetide, leverages the naturally occurring CendR active transport system to selectively deliver anti-cancer drugs through the tumor stroma and into the tumor. Simultaneously, certepetide has been shown to modify the tumor microenvironment, making it less immunosuppressive and therefore increasing the tumor's susceptibility to immunotherapy and our body's own immune system, while also inhibiting the metastatic cascade. For more specifics regarding certepetide's mechanism of action, I invite you to visit our website and view the animated video pertaining thereto as well as the relevant slides in the corporate presentation. Beyond our strategic clinical development plan, we have focused on optimizing our regulatory strategy. To-date, our approach has yielded significant results for certepetide with special regulatory designations across multiple health authorities. These include several orphan drug designations, a fast track designation and a rare pediatric disease designation. One such achievement occurred this past September when certepetide was granted an orphan drug designation by the U.S. Food and Drug Administration for the treatment of cholangiocarcinoma. As mentioned, our regulatory and clinical development strategy for certepetide prioritizes rapid registration. We are actively evaluating certepetide's potential as a selective tumor targeting and penetrating enhancer and tumor microenvironment modifier in combination with a variety of standard of care therapies in advanced solid tumors. Now for an update on our individual development programs. The ASCEND trial is a 158 patient double blind, randomized, placebo controlled clinical trial evaluating certepetide in combination with standard of care gemcitabine and nab-paclitaxel chemotherapy in patients with metastatic pancreatic ductal adenocarcinoma. The trial is being conducted at 25 sites in Australia and New Zealand led by the Australasian Gastro-Intestinal Cancer Trials Group or AGITG in collaboration with the National Health and Medical Research Council Clinical Trial Centre at the University of Sydney. As mentioned on prior calls, the ASCEND trial is an investigator initiated trial that Lisata inherited upon our acquisition of Cend Therapeutics. The original trial was designed with more of an academic nature rather than one with commercial objectives and was statistically powered based on a six month progression free survival primary endpoint. After the acquisition, Lisata collaborated with the sponsor of the trial, AGITG, to modify the trial to ensure it provided clinical outcomes that would best support the next steps in development of certepetide from a regulatory perspective. As such, the ASCEND protocol was amended to include an additional cohort of patients, cohort B, not statistically powered to evaluate an additional certepetide dosing regimen. The ASCEND protocol was also amended to capture overall survival outcomes for both Cohort A and Cohort B, as overall survival is considered the gold standard endpoint in pancreatic cancer trials. Since the ASCEND protocol was amended following trial initiation, data from Cohort B is lagging Cohort A data by several months. Cohort A with 95 patients receiving a single IV dose of certepetide or placebo in combination with standard of care completed enrollment in the third quarter of 2023. As recently announced, preliminary Cohort A data was presented at the 2025 ASCO GI Symposium, which showed a positive trend in overall survival, including four complete responses in the certepetide treated group compared to none in the placebo treated group. Data from Cohort B with 63 patients receiving two IV doses of certepetide or placebo administered four hours apart in combination with standard of care are expected in the coming months with a final analysis of both cohorts available thereafter. The BOLSTER trial is our Phase 2a double-blind placebo controlled multi-center randomized trial in the United States evaluating certepetide in combination with standard of care in first and second line cholangiocarcinoma. Enrollment was completed in first line cholangiocarcinoma nearly six months ahead of plan, accelerating anticipated top line data readout to mid-2025. A second cohort has been added to the BOLSTER trial evaluating certepetide in subjects in second-line cholangiocarcinoma on top of standard of care. With more than 30% of the patients enrolled in the second-line cholangiocarcinoma cohort, we continue to target later this year for the completion of enrollment. CENDIFOX is a Phase 1b/2a open-label trial in the United States evaluating certepetide in combination with neoadjuvant FOLFIRINOX based therapies in pancreatic, colon, and appendiceal cancers. In December 2024, the company announced enrollment and completion in all three cohorts. This single center study being conducted at the University of Kansas Cancer Center was designed with a three cycle FOLFIRINOX run-in period to ensure patients met specific criteria before receiving treatment with certepetide. Of the 66 patients enrolled, 50 met these criteria and were treated with certepetide across three cohorts, including 24 with resectable or borderline resectable pancreatic cancer, 15 with high grade colon or appendiceal cancer and peritoneal metastasis, and 11 with oligometastatic colon cancer. The trial will provide Lisata with valuable pre- and post-treatment tumor tissue data for immuno profiling along with long-term patient outcome information. We anticipate data in the coming months, and we will share the key findings when available. Qilu Pharmaceutical, the licensee of certepetide in the Greater China territory, is also currently evaluating certepetide in combination with gemcitabine and nab-paclitaxel as a treatment for metastatic pancreatic ductal adenocarcinoma. Qilu is currently treating patients in their Phase 2 placebo controlled trial in pancreatic cancer. They recently reported that they completed enrollment with the revised target enrollment of 96 patients. The revised study is still sufficiently powered, and this change will expedite the study and allow for earlier data analysis in preparation for a Phase 3 trial. According to guidance from Qilu, data are expected in the next 12 to 18 months with a Phase 3 study planned start thereafter. In collaboration with AstraZeneca in Australia and the funding sponsor of the iLSTA trial, WARPNINE, we are evaluating certepetide in a Phase 1b/2a randomized placebo controlled three arm single blind, single center safety early efficacy and pharmacodynamic trial. The trial is being conducted on Australia evaluating certepetide in combination with the checkpoint inhibitor durvalumab plus standard of care gemcitabine and nab-paclitaxel chemotherapy versus certepetide in combination with standard of care no durvalumab versus standard of care alone in patients with locally advanced non-resectable pancreatic cancer. Preliminary results from the first 17 of 30 patients enrolled in the iLSTA trial were presented at the 2025 ASCO GI Symposium. The interim analysis suggests that certepetide, in combination with standard of care chemotherapy and immunotherapy, improves treatment outcomes for patients with locally advanced non-resectable pancreatic cancer, while also provoking tumor infiltrating lymphocytes in subjects who have RECIST responses. These findings also support preclinical data indicating that certepetide enhances the effectiveness of immunotherapy. With 27 of the 30 targeted patients enrolled, enrollment remains on track to be completed by the first half of 2025. A study of certepetide in combination with temozolomide in glioblastoma multiforme or brain cancer has been initiated with several patients already enrolled and treated. This study is designed as a Phase 2a double-blind placebo controlled randomized proof-of-concept study evaluating certepetide when added to standard of care temozolomide versus temozolomide alone and matching certepetide placebo in subjects with newly diagnosed glioblastoma. This actively enrolling study is being conducted across multiple sites in Estonia and Latvia and is planned to also include a site in Lithuania. The study is targeted to enroll 30 patients with a randomization of two to one, certepetide plus standard of care versus placebo plus standard of care. Enrollment completion is targeted for the second half of 2025. FORTIFIDE is currently planned to be a Phase 1b2a double blind, placebo controlled, three arm randomized study in the United States evaluating the safety, tolerability and efficacy of a four hour continuous infusion of certepetide in combination with standard of care in patients with first line metastatic pancreatic cancer who have progressed on FOLFIRINOX -- excuse me, second line metastatic pancreatic cancer who have progressed on FOLFIRINOX. As part of this study, we have engaged Haystack Oncology to use their MRD technology to measure circulating tumor DNA levels at multiple time points in patients throughout the study as an exploratory endpoint for analyzing the early therapeutic effect of certepetide. Lisata is currently evaluating alternative strategies to achieve the FORTIFIDE study objective with a focus on identifying options that are faster and or less expensive. We hope to have more to share in the coming months. Additionally, Lisata recently entered into several preclinical research collaborations to further investigate the therapeutic potential of certepetide. These collaborations encompass both oncology and other therapeutic areas and are intended to identify new strategic opportunities for the drug's development. These collaborations include a sponsored research agreement with the University of Cincinnati to assess certepetide in combination with bevacizumab, a VEGF inhibitor, and a preclinical murine model for the treatment of endometriosis. This trial is the first exploration of certepetide in a non-cancer related indication and aims to assess if certepetide can improve the delivery of bevacizumab to endometriosis lesions, potentially reducing lesion burden and abdominal pain. Data from this preclinical trial are currently under review. And while early signals are encouraging and suggest the potential for further investigation, the company will need to evaluate the financial resources required to determine the best path forward. Our partnership with Valo Therapeutics to investigate the benefits of combining certepetide with Valo's PeptiCRAd, a customizable oncolytic adenovirus platform technology, and a checkpoint inhibitor in a preclinical murine model for the treatment of melanoma. And as previously noted, following results from an earlier preclinical study, Lisata entered into a global license agreement with Kuva Labs to explore the synergistic potential of certepetide as a targeting and delivery agent for Kuva's NanoMark imaging technology in solid tumors. Kuva has communicated that it intends on commencing its imaging study in the first half of this year with results anticipated in early 2026. Relatedly, Lisata will provision certepetide to Kuva for its clinical study via clinical supply agreement. This collaboration reinforces our belief in the broad applicability of certepetide beyond a therapeutic setting. Beyond the clinical studies I've outlined, we are actively exploring additional opportunities to advance our development strategy, including progressing certepetide in combination with gemcitabine and nab-paclitaxel into a Phase 3 study in pancreatic cancer. However, we remain focused on only initiating trials that can be funded through data with existing or guaranteed capital and that can be executed within a reasonable period of time. As a reminder, several of the clinical studies I mentioned earlier are investigator initiated trials. And although we have great confidence in the investigators running these studies, Lisata has limited control and thus timelines and expectations may be subject to change. That said, we are extremely grateful to the investigators and especially to the patients participating in certepetide clinical trials around the world. For those who are interested, a more comprehensive description of each trial is available in the appendix section of the corporate presentation on our website. Additionally, in the body of the presentation, there are two slides that depict the anticipated timing and execution of key milestones and data readouts from our trial. As Dr. Mazzo previously mentioned, 2025 will be a data rich year for Lisata and we are looking forward to reporting those results. With that, I will now turn the call back to Dave.

Thanks, Kristen. Overall, we achieved our target milestones in 2024, positioning us well for continued success in 2025. We look forward to sharing further corporate and development updates throughout the year, including key information regarding our ongoing and planned trials as they become available. We believe that certepetide represents a significant opportunity to create both meaningful patient benefit and long-term shareholder value. With that overview, operator, we're now ready to take questions.

Thank you. [Operator Instructions]. Our first question comes from Sara Nik with H.C. Wainwright. You may proceed.

Hi, this is Sara on for Joe Pantginis. I had a question, regarding the ASCEND trial. I know you can't exactly speak to timing, but regarding the data cut that's expected from Cohort B, can you give any insight into what kind of data we could expect? Would it be along the similar lines of what we saw earlier this year from Cohort A? Thanks.

Hi, Sarh. Thanks very much for the question and giving me the opportunity to clarify. For the Cohort B data, we expect something that would be akin to what was reported for Cohort A. That is an analysis of both the progression free survival and at least the preliminary, if not final, overall survival of all patients in that cohort. So, we're looking forward to that because those data will be indicative of final trial results and also will give us a good head start into planning for Phase 3 assuming that they'll be supportive.

Okay. Thanks. That's helpful.

Thank you.

Thank you. Our next question comes from Pete Enderlin with MAZ Partners. You may proceed.

Hello, everybody. Thank you. So just following up on that with regard to the ASCEND Cohort B, you said in coming months, can you be a little more specific? Is that you hope by the end of the year or in the third quarter, fourth quarter? What does that look like?

So the best I can do Pete, because as I think we've explained at least in writing in the past and I can take a moment here to repeat it. We, under contract with AGITG and the CTC of the University of Sydney do not have any control over the release of the data. This is an investigator initiated set of trials and so they control the data release. As best as we know right now, the plans are to, to attempt to have all of the analyses done in time to submit an abstract so that the data could be presented at the American Society of Clinical Oncology or ASCO meeting, which is the last few days of May, first few days of June this year. If they get their analyses done on time and submit that abstract, then that would be the date of the public disclosure most likely. If they missed the deadline for the submission of the abstract, the next relevant major International Scientific Meeting is ESMO, the European Society of Medical Oncology, and that would be in I think actually October. So to narrow it down, it's most likely going to be sometime from second through third quarter of this year that the data will become available. Although we're working with them, them being the AGITG and CTC to see if they can get the analyses done faster and allow for release of the information under other circumstances. So that's the best we can say right now.

Well, thanks. That's great. I actually expected you to say ASCO in January of next year. So, that sounds very encouraging. Now I have asked a question that's a little bit abrupt or brusque or whatever you want to call it. And that is when you reported the Cohort A results preliminary data about a month ago, the stock had a severe reaction. I think it's fair to say. And so the question is, what if anything did the Street miss or misinterpret? What is really the import, but what if the street misunderstand or not quite realize when you report those results?

, : As we've said in our press release, the reason we added Cohort B was because we anticipated the type of results that we got out of Cohort A, which is results that trend in the right direction are actually consistent with what we've seen in every other trial preclinical and clinically, but perhaps not fully refined and that's because we were working on optimizing the dosing scheme and that's why we added Cohort B in the first place. And so, for us, it wasn't a big surprise. But I think for certain people who perhaps didn't understand the nuances of the trial design or simply were betting on, a big announcement, that's why they were disappointed. It's unfortunate because it's the full trial that counts, not any bits and pieces.

Yes. Thanks. That makes a lot of sense. Can I just ask one more quick one?

Sure. Go ahead.

And that is, will the Kuva deal lead to, let's say, future diagnostics that will enable better analysis in clinical trials say a Phase 3 clinical trial that we look at tumor sizes and all that sort of stuff? Or is it not really directly related to that?

Well, once again, I will give you my interpretation and conclusions based on the transaction with Kuva and discussions with their head. But I can't speak for Kuva. So I want to make that very, very clear. Kuva speaks for themselves. But I believe that the intention with Kuva is to do exactly what you just described, not only for clinical trials, but actually as a personalized medicine for treatment of actual patients once certepetide is approved. So the goal is to identify those patients and those tumors that are particularly susceptible to increased permeability in the presence of certepetide, and therefore, demonstrate or identify those patients who would respond better with certepetide in their anticancer treatment regime. So I think that's exactly what they intend to do and I think we'll be doing it collectively both clinically and hopefully, eventually, commercially.

Great. Thanks, Dave.

You're welcome. Be well, Pete.

Thank you. Our next question comes from Steve Brozak with WBB Securities. You may proceed.

Hey, guys. Thanks for taking the questions. I do have a question and it's not about timing or outcomes per se in terms of when the data comes out. But what are some of the expectations, not from yourselves, but what you've been talking to a number of pharmaceutical partners. What are they looking for in data that would have appealed to them? Obviously, pancreatic cancer is a critical disease and it's always found late and you obviously are looking at improving outcomes clinically in a significant fashion. What can you tell us in any granularity without talking about timing that you would be looking that pharmaceutical partners have told you that they're looking for? And I will hop back in the queue. Thank you.

Hey, Steve. Thanks very much for joining and for the question. So in general, what I'll say is not particularly profound, because it would apply almost to any oncology program at which a large pharma company would be looking and considering. But in our particular case, what they're looking for are the complete trial results from the ASCEND trial for the reasons I just explained to the prior caller, the complete trials what counts, and also a consistency of both therapeutic effect and safety. And so they want to know that the drug works consistently, that we have a good handle on the relative magnitude of that effect so that we can appropriately size and power and optimize Phase 3 registration protocol and that there have been no untoward safety signals that have come out of exposure to larger populations. And so up until, well, let's just say to-date, we've been able to do that and we'll be expecting to be able to do that hopefully with the results from Cohort B when we get them. And of course, this is all done in the context of in comparison with standard of care, right? The whole point of the matter is that the addition of certepetide is designed to improve standard of care. So I'm talking about therapeutic effect. I'm talking about the combination of the cytotoxic gemcitabine and nab-paclitaxel with certepetide and seeing that they have an improved effect over treatment without certepetide.

[Indiscernible] that legal term. For the purposes of clarity, when you're talking about improvement effect and obviously it isn't a lapping matter, we're talking about people who are predictably unfortunately going to die. And you are looking at being able to improve those outcomes. Is that without getting too granular, is that a fair statement?

That is exactly the case. Yes, sir.

Okay. Let me hop back in the queue. Thanks for taking the questions.

Thanks, Steve. You're welcome.

Thank you. Our next question comes from Kemp Dolliver with Brookline Capital Markets. You may proceed.

All right. Thank you. Most everything is pretty straightforward here. But you have an interesting comment about the Fortified trial and alternative approaches to achieving the study objective. Are you in a position to elaborate on any of that?

Kemp, thanks for joining and appreciate your question. Thank you. I can say what we're thinking. Originally, the Fortified trial was designed to elicit or elicit further information around the pharmacodynamics of certepetide in humans. And the original concept was that it would have to have its conclusions based upon both progression free survival and overall survival data in patients. And as we know, they've take a long time to get to and it's sort of a perverse situation. But the better the drug works, the longer it takes to get that information. And so we've been considering other ways and now that we have a strategic partnership with Kuva, it prompted the thought process around a possible means of eliciting the same information using imaging techniques and imaging endpoints for these purposes. Now we haven't yet refined that. We're certainly not in a position to discuss a protocol, but that's what we're talking about. So if it -- if the idea works, we should be able to derive the type of information that we were looking to get from Fortified in both a fraction of the time and at a fraction of the cost. And obviously, that's very appealing.

That's great. Thank you.

Thank you, Kemp.

Thank you. This concludes the question-and-answer session. I will now turn the call back to Dr. Mazzo for any closing remarks.

Well, again, thank you all for participating in today's call. We look forward to speaking with you again during our next quarterly conference call and to continuing to provide updates on our achievements and progress. We remain grateful for your continued interest and support. Stay well, and please have a good evening.

Thank you. This concludes the conference. Thank you for your participation. You may now disconnect.