Plus Therapeutics, Inc. (NASDAQ:PSTV) announced its first quarter 2024 financial results and provided updates on its clinical programs and strategic developments. The company highlighted the closing of a private placement financing, raising $19.25 million, and the receipt of a $3 million grant from the US Department of Defense to support its pediatric cancer trials.
Plus Therapeutics also reported on the promising survival rates from its radioembolic therapy trials for glioblastoma (GBM) and leptomeningeal metastases (LM), showing a significant improvement over the standard of care. The company is preparing for further trials, including a pivotal trial strategy, and is working towards commercializing its CNSide diagnostic tests.
Key Takeaways
- Plus Therapeutics closed a private placement financing on May 9th, 2024, raising $19.25 million.
- A $3 million grant was received from the US Department of Defense for a Phase 1/2 trial in pediatric brain cancer.
- The company added neurooncologists Dr. Andrew Brenner and Dr. Barbara Blouw to its management team.
- Three patients were enrolled in Cohort 5 of the ReSPECT-LM Phase 1 trial, with five new sites added.
- The CNSide diagnostic platform was acquired, with a commercial launch planned for Q4 2024.
- Plus Therapeutics is continuing patient enrollment for the ReSPECT-GBM trial and is engaging with the FDA regarding Phase 2 data and pivotal trial design.
- Promising results from radioembolic therapy trials indicate a 63% improvement in overall survival for GBM and LM patients over standard care.
- The company has a cash balance of $3 million and recognized $1.7 million in grant revenue in Q1 2024.
- Anticipated IND approval for a pediatric trial in 2024 and plans for a Phase 2/3 pivotal trial in breast cancer.
Company Outlook
- Plus Therapeutics expects to receive grant advances totaling $6.9 million in 2024.
- The development timeline suggests an LM product approval may occur before GBM.
- The company is preparing for an aggressive approval timeline for a single-dose trial in breast cancer.
Bearish Highlights
- The company is waiting for funding before submitting a formal IND for the pediatric study.
- Plus Therapeutics has a cash balance of only $3 million at the end of Q1 2024.
Bullish Highlights
- Plus Therapeutics is optimistic about potential funding beyond Phase 1 for pediatric brain cancer.
- The company is excited about the collaboration with Lurie neuro-oncologists and neurosurgical experts.
Misses
- No specific misses were discussed during the earnings call.
Q&A Highlights
- The company will present at the Society of Nuclear Medicine Meeting in June and the SNO/ASCO presentation in November.
- Updates on the ReSPECT-LM trial will be shared, focusing on enrollment and safety.
- FDA approval in principle has been received for the pediatric study dosing regimen.
Throughout the call, Plus Therapeutics reiterated its commitment to advancing its clinical programs and expressed gratitude to its stakeholders, including doctors, patients, and employees. With strategic developments in place and a clear focus on its clinical trial advancements, the company is poised to make significant progress in the treatment of GBM, LM, and pediatric brain cancers.
InvestingPro Insights
Plus Therapeutics, Inc. (PSTV) has shown a dynamic presence in the biopharmaceutical industry with its recent financial results and clinical developments. As investors consider the potential of PSTV, it is important to look at key metrics and insights provided by InvestingPro.
InvestingPro Data shows a notable revenue growth in the last twelve months as of Q4 2023, with an increase of 2093.3%. This substantial growth is indicative of the company's expanding operations and may signal future potential. However, it is crucial to note that PSTV holds a negative Gross Profit Margin of -97.23% for the same period, reflecting costs that vastly exceed revenues—a situation that is not sustainable in the long term.
The company's Market Cap stands at $9.92M, which is relatively small in the biopharmaceutical industry, suggesting that PSTV is a more speculative investment with higher volatility and risk. The Price, Previous Close was recorded at $2.32, and while the company has experienced a strong return over the last month of 32.57%, it is essential to consider the broader performance and financial health.
InvestingPro Tips highlight that PSTV is quickly burning through cash and suffers from weak gross profit margins. This is a critical consideration for investors, as it may impact the company's ability to sustain operations without additional financing. Additionally, PSTV does not pay a dividend to shareholders, which is common for companies focused on growth and clinical trials but may be a factor for income-focused investors.
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Full transcript - Cytori Therapeutics (PSTV) Q1 2024:
Operator: Good afternoon, ladies and gentlemen. Welcome to the Plus Therapeutics' First Quarter 2024 Results Conference Call. Before we begin, we want to advise you that over the course of the call, any question-and-answer session, forward-looking statements will be made regarding events, trends, business prospects and financial performance, which may affect Plus Therapeutics' future operating results and financial position. All such statements are subject to risks and uncertainties, including the risks and uncertainties described under the Risk Factors section included in Plus Therapeutics' Annual Report on form 10-K and quarterly reports on form 10-Q, filed with the Securities and Exchange Commission from time to time. Plus Therapeutics advises you to review these risk factors in considering such statements. Plus therapeutics assumes no responsibility to update or revise any forward-looking statements to reflect events, trends or circumstances after the date they are made. It is now my pleasure to turn the floor over to Dr. Marc Hedrick, Plus Therapeutics' President and Chief Executive Officer. Sir, you may begin.
Marc Hedrick: Thank you, Victor. Good afternoon, everyone and thank you once again, for taking the time to join us today as we provide an overview of recent business highlights and discuss our first quarter 2024 financial results. Joining me for the call today are Mr. Andrew Sims, our Chief Financial Officer and Dr. Norman LaFrance, our Chief Medical Officer. I'll begin the call by reviewing our recent corporate and clinical progress in the first quarter and then turn the call over to Andrew to review our financials. Dr. LaFrance then will be joining us for Q&A. Let me begin by highlighting some recent corporate updates before discussing our specific therapeutic and diagnostic programs. So, first of all, on May 9th, 2024, we closed a private placement financing for an aggregate proceeds of up to $19.25 million, which consisted of an initial upfront funding of approximately $7.25 million and up to an additional approximately $12 million upon cash exercise of accompanying warrants at the election of the investors. The financing included participation from AIGH Capital Management, LLC with additional participation from healthcare-focused institutional investors and insiders. Second, we received a notice of award for $3 million grant from the United States Department of Defense, congressionally-directed medical research programs. The award will be used to fund a Phase 1/2 trial for pediatric patients with high-grade glioma and ependymoma following IND approval from the FDA. As a reminder, this grant illustrates our ongoing strategy to use external grant funding to support the initial preclinical and clinical development of new technology or indications, while minimizing the impact to the balance sheet and shareholder dilution. More specifically, our GBM program had just been acquired when we found out that we had a five-year grant funding from the NCI to fund that program through Phase 1/2. Second, our LM program received nearly $18 million of funding over three years from CPRIT. And today, that strategy continues with this DoD award for pediatric brain cancer therapeutic development. We're going to continue to leverage our novel technology, subject matter and grant management expertise to drive this strategy forward for the foreseeable future. Finally, to strengthen our team for planning for forthcoming pivotal trials, we added neurooncologist, Dr. Andrew Brenner and Dr. Barbara Blouw to our management team. Dr. Brenner is an MD Ph.D. and has been instrumental in developing rhenium obisbemeda and the related clinical strategy. And Dr. Blouw is a Ph.D. with extensive experience in clinical operations, CNS tumor biology biomarker development and neoplasms of the cerebral spinal fluid. Now, in terms of our clinical programs, I'll begin with updates on our ReSPECT-LM Phase 1 dose escalation basket trial for single administration of rhenium obisbemeda for leptomeningeal metastases. This trial is substantially funded by three year nearly $18 million product development award from CPRIT. During Q1, we announced the enrollment of the three required patients for dosing in Cohort 5 at a single radiation dose of 66.1 millicuries. As of the most recent data update in March 2024, 12 of 18 patients dosed remained alive. Multiple compassionate use doses have now also been made available to clinicians for patient survivors that have exceeded the trial follow-up period and continue to do well. We expect to present updated enrollment and safety data at the SNO/ASCO meeting in August 2024 in Denver. Furthermore, investigator and KOL enthusiasm for the trial, and the investigational therapy remains high and continues to grow. Recently, we added an additional five sites to the trial that will participate in patient recruitment for both the Phase 1 and subsequent follow-on trials. Our goal is to complete enrollment in the Phase 1 basket trial for a single dose this year and then to present the full dataset from all cohorts of the Phase 1 trial as they exist at that time at the SNO Annual Meeting in November 2024 in Houston. In parallel, we are presently in dialogue with the FDA regarding a multi-dose expansion arm of the Phase 1 trial. Ultimately, we believe multiple doses of rhenium obisbemeda will be the optimal therapeutic approach to suppress and potentially cure LM. We will provide an update once the plan is finalized and FDA approval is obtained. Additionally, as we move forward towards a recommended Phase 2 dose of rhenium obisbemeda for LM. we intend to expand our communications with the FDA regarding a pivotal Phase 2/3 trial design, which is currently in development with our team and KOL advisors. The initial planned pivotal trial will be focused on LM and metastatic breast cancer, for which we have orphan designation from the FDA. Last week, we hosted an investor call focused on the acquisition of the synergistic LM diagnostic platform called CNSide. Rather than fully revisiting the details of that call today, I would like to provide the highlights and encourage you to investigate the recording or [Technical Difficulty] when you -- which can be found on our website to learn more about this tremendous new opportunity for the company. So, here are the key highlights. And I'll begin with the four key points outlining the rationale for the acquisition. First of all, leptomeningeal cancer is a tough diagnosis to make and the current state-of-the-art diagnostics, which include MRI and cytology, lack diagnostic sensitivity. Second, we developed experience with the CNSide assay in our ReSPECT-LM trial and saw firsthand its value. Additionally, we have the opportunity to engage with multiple KOL physicians, who have come to see ReSPECT-LM assay as a gamechanger in the practice. Third, our toxi studies indicate LM is likely significantly under diagnosed. As such, an enhanced diagnostics such as Cnside means more patients may be able to take advantage of rhenium obisbemeda therapy for LM. Finally, we recognize that there is a substantial standalone commercial opportunity to develop, commercialize and then monetize the CNSide technology in the near term. So, based on that rationale and our supporting analysis, we exclusively acquired all essential CNSide-related assets to provide CNSide testing commercially, and this includes three types of tests. First of all, the cancer cell enumeration, or CTC, circulating tumor cell testing, which we call CSF-01. This quantifies adenocarcinoma and melanoma tumor cells for the CSF. Second, we obtained the FISH or fluorescence in situ hybridization testing, we call CSF-02 that determines cancer-specific gene expression for therapeutic guidance. And third, we acquired the next generation sequencing of cell-free DNA called CSF-03 that analyzes DNA to identify genetic mutations related to LM. In Q1 2024, prior to the acquisition, we validated and clinically implemented the CSF-01 test into our ReSPECT-LM trial as an exploratory endpoint and is currently in use today. As part of the acquisition, we acquired the FORESEE clinical trial data, which evaluates CNSide and its clinical utility in addressing therapeutic decision-making at LM. And last week, we disclosed that the trial met its primary endpoint and the presentation of the full FORESEE trial data is planned for the August 8th to 10th SNO/ASCO meeting in Denver. So, now looking forward, in terms of next steps, we will be expanding the availability of the test in our ReSPECT trial to longer time points in conjunction with our planned multiple dosing regime. We are also finalizing a complete business evaluation, including a reimbursement optimization strategy with the attention of commercially launching the test as soon as Q4 2024. As discussed in last week's call, we anticipate a total addressable market of over a half million tests annually with the potential for additional growth. We plan to further discuss the commercial opportunity and the plan at a later date. And finally, in parallel, we are talking to potential diagnostic business development partners that have expressed interest in CNSide. And then finally, just a reminder, you can learn more about the CNSide asset, our acquisition of that by visiting our website and look at the related press release and investor call details. So, now shift gears and regarding our ReSPECT-GBM trial evaluating rhenium obisbemeda in patients with recurrent glioblastoma. We continue to enroll both Phase 2 patients with GBM tumors less than or equal to 20 milliliters and also Phase 1 patients with GBM tumors that are greater than that. And that's in our dose escalation Phase 1 that's still ongoing and now in Cohort 8. As the NIH NCI funding for this trial that I mentioned earlier, which has been the primary financial supporter for the trial is winding down in 2024. And as we work to finish enrollment of the Phase 2 study, we are able to add new sites that will help speed trial completion and set us up for the Phase 3 study. So, besides our Texas sites in Dallas, Houston and San Antonio, we are now adding three new sites to the trial focused on key population centers: in the upper Midwest, the Ohio State University in the New York area, North Shore and Lenox Hill Hospitals, which are part of the Northwell network and in Florida, AdventHealth. These three [Technical Difficulty] to enroll in the second half of 2024 and will substantially contribute to a pivotal trial. We have set the goal for ourselves to complete enrollment in 2024 or early 2025, and enrollment timing will be influenced to a significant degree by participation of these new sites. We plan to provide a complete update on the Phase 2 data this fall at one of the key neurosurgery or neuro-oncology meetings, and that final meeting designation is to be determined. As a reminder, the data seen and reviewed to date in the Phase 2 trial has been -- excuse me highly promising in terms of both safety and efficacy, demonstrating thus far a 13-month overall survival as of November 2023, compared to approximately eight months for the standard of care or 63% improvement in OS over standard of care. As I mentioned last quarter, given the safety profile and the unprecedented amount of radiation, we can deliver to the CNS using our technology, we believe there's a tremendous potential for improving upon the standard of care in GBM. And also, there's a broader opportunity to leapfrog the primary means of radiation delivery for all CNS neoplasms, which is essentially external beam radiation therapy at this point. We continue to work behind the scenes with existing and potential partners that share our view of this opportunity and want to collaborate to explore the potential in a focused manner. In parallel to our plans to finalize the Phase 1 and Phase 2 trials, we are also planning our pivotal trial strategy and plan to meet with the FDA later this year to discuss the Phase 2 data and align on the pivotal trial design in GBM. Finally, I would like to briefly update you on our pediatric brain cancer program. As I also noted at the beginning of the call, with U.S. Department of Defense funding support, we will be finalizing our IND with the FDA and initiating a Phase 1 trial for children with pediatric high-grade glioma and ependymoma. We anticipate, based on several previous rounds of discussion with the FDA, that we can obtain IND approval in 2024 and perhaps be ready to begin enrollment early next year at our initial site, Lurie Children's Hospital of Chicago. Once the final trial details are agreed upon, we will provide an update on those. Now, with respect to our next generation radioembolic device called Rhenium-188 or Rhenium-186 NanoLiposome Biodegradable Alginate Microsphere or RNL-BAM. As previously mentioned, feedback from the FDA is that BAM will be regulated as a device, not a drug. With that decision in hand, we are now working on the device-related quality system, finalizing the device design requirements and expanding the related IP portfolio. And I anticipate providing more updates when warranted in the near future. Finally, although our current supply chain is both reliable and sufficient for our near-term needs, as we contemplate pivotal trials beginning in 2025, we are devoting significant energy to build out a supply chain that is ready for both pivotal trials and ultimately commercialization. Two key focus areas are as follows: first is to have a redundant and high-capacity GMP manufacturing capability. So, to complement our two existing manufacturers of the final drug product, we are in the process of selecting a third partner that meets GMP standards. This addition serving as an alternative and redundant source will ensure that collectively, we could fulfill our demand projections for rhenium obisbemeda through 2028. Second focus area is for additional radiation services capability. So, to support the commercial radioisotope supply, a key part of the overall manufacturing process, we will need to expand our network of providers. We are currently well into that process today and I'll report more as we are able to update. Moreover, we're enhancing both exclusive and non-exclusive supply agreements for key starting our intermediate drug products. We are also refining inventory-based strategies to guarantee reliable drug availability under any foreseeable demand scenario. And with that, I'll now turn the call over to our Chief Financial Officer, Andrew Sims, who will review the financials. Andrew?
Andrew Sims: Thank you, Marc. Good afternoon, everyone. Please refer to our press release issued earlier today for a summary of our financial results for the first quarter ended March 31, 2024. The cash balance was $3 million at March 31, 2024, compared to $8.6 million at December 31, 2023. The company recognized $1.7 million in grant revenue in the first quarter of 2024, compared to $0.5 million in the first quarter of 2023. This represents CPRIT's share of the cost incurred for our rhenium obisbemeda development for the treatment of patients with LM. We expect 2024 grant revenue to be in the range of $6 million to $7 million i.e. tracking [Technical Difficulty] Total operating loss for the first quarter of 2024 was $3.3 million, compared to $4.8 million in the same period of 2023. The decrease was primarily due to increased grant revenue. Net loss for the first quarter of 2024 was $0.75 per share, compared to $2.07 per share for the same period the prior year. I would also like to provide a more detailed update on our runway and cash position based on the recently-announced private placement and provide guidance on our grant funding for the remainder of 2024. There are two additional sources of cash that Plus has access to beyond the balance disclosed in cash on hand and liquid investments on our Q1 2024 balance sheet. The first, as we announced last week on May 6, we closed a private placement financing of up to $18 million from new healthcare-focused institutional investors and company insiders. In addition, it should be noted that as reported aftermarket on Form 8-K on May 9, this financing was subsequently upsized to $19.25 million with a total of $7.25 million received at closing. This $7.25 million amount represents approximately 12 months of incremental runway at our current burn. The second source of cash remains our continued funding through now three announced grants. Firstly, the CPRIT grant to support the ReSPECT-LM trial. With respect to expected grant advances from CPRIT in 2024 and to be clear, cash advances from CPRIT, we are on track to receive advances totaling $6.9 million in 2024. $3.4 million will be received in late Q2 or early Q3 and an additional $3.5 million will be received in late Q4. An additional $3.5 million is then due from CPRIT in 2025. Secondly, as reported on April 22nd, Plus has received an award recommendation from the United States Department of Defense for $3 million to support the upcoming ReSPECT pediatric brain cancer trial. This funding is expected to commence in late Q3 or early Q4 of 2024, and materially cover the costs of the planned Phase 1 trial. Funding is generally received annually in advance and covers a three-year period, i.e. approximately $1 million will be received under this grant in 2024. Plus also continues to benefit from the NIH grant to support the ReSPECT-GBM Phase 1/2 trial. Although expected to be complete in 2024, it currently covers approximately 90% of the overall trial costs. We also continue to source other non-dilutive sources of grant capital with a target of applying for at least $10 million per year. We will continue to only report on individual grants when they are awarded. Taken in total, this cash-on-hand placement financing, warrants if fully exercised and committed and contractual grant revenue is in excess of $35 million. I'll now turn it back to you, Marc.
Marc Hedrick: Thank you, Andrew. Before we move on to Q&A, I'll take a moment to provide guidance on anticipated key events and milestones taking us through the remainder of 2024. First, in terms of presentations, the company will be making. we will attend the Society of Nuclear Medicine & Molecular Imaging Annual Meeting in June, that's the 8th through 11th in 2024. We have two accepted abstracts. The first will be the ReSPECT-LM trial, and an update of initial safety and feasibility through cohorts 1 through 4. We also have a dosimetry presentation on the radiation absorbed dose to the spinal cord using beta emission radiopharmaceuticals in leptomeningeal metastases. We also intend to attend the SNO/ASCO or Society For Neuro-oncology and American Society of Clinical Oncology combined CNS Metastases Conference on August 8 through 10 in 2024. We have three anticipated abstracts. The first is the ReSPECT-LM trial and an update of enrollment and safety, as mentioned earlier in the presentation. Also, we will be presenting the full FORESEE clinical trial dataset on CSF tumor cell detection and data on its ability to help in clinical management of breast cancer and non-small cell lung cancer with patients with leptomeningeal disease. We'll also have a third presentation, which is based on the feasibility and relevance of CNSide as a scalable platform for disease management for patients with leptomeningeal disease. Later in the year, we anticipate a comprehensive update on safety and efficacy data from the Phase 1 ReSPECT-LM trial at the SNO Annual Meeting in November 2024. Also, later in 2024, we anticipate a meaningful update on the ReSPECT-GBM trial at either a neurosurgical or neuro-oncology meeting and that exact meeting is to be determined. In terms of FDA updates, we plan to provide feedback when available on two specific work streams. The first is the ReSPECT-LM Type C meeting for a multi-dose Phase 1 dose escalation trial that was created by the FDA and scheduled for meeting on June 10, 2024. We also anticipate FDA feedback in the second half of 2024 for the ReSPECT [Technical Difficulty] new drug application for pediatric ependymoma and high-grade glioma with the aim of securing regulatory approval for the trial. Additionally, we anticipate completing the ReSPECT-LM Phase 1 dose escalation trial enrollment this year, and determining the maximum tolerated and recommended Phase 2 doses. Also, we will report results of the preclinical combination studies of rhenium-186 obisbemeda with PD-1 and PD-L1 checkpoint inhibitors when that data is completed. We also will contract with a second GMP manufacturing supplier to better support the rhenium-186 obisbemeda supplied for pivotal trials and commercial readiness. And then finally, as Andrew mentioned, we are on track to file at least $10 million of new grant applications in 2024 and we'll announce those upon receipt of the notification of award. So, with that, Victor, I'll turn it back over to you. and let's have our Q&A session.
Operator: [Operator Instructions] One moment for our first question. Our first question will come from the line of Justin Walsh from Jones Trading. Your line is open.
Justin Walsh: Hi, congrats on the progress. Thanks for taking the questions. I know there's a lot of potential variability here. but I was wondering what your current thoughts are on the overall development timelines for rhenium obisbemeda in GBM versus LM?
Marc Hedrick: Hey, Justin, it's Marc. So, I think that actually, the LM development timeline, on the whole, could actually mean an approved product prior to GBM. And if you'd asked me that a year or two ago, I might have said something different, because we were more advanced than GBM. GBM, as you know, has there are multiple competitive trials. It's a much smaller number of patients. And the work required to enroll patients, do the case planning and so forth is materially different than with LM. Furthermore, there's and then in comparison, if you look at Lm, there's no approved products. We think likely that the FDA will accept the Phase 2/3 pivotal versus the requirement for a pivotal trial at GBM with overall survival as the endpoint. So, to your question, as it relates to LM, if we sort of say that is likely first to market, if you will, with the rhenium obisbemeda product. As we think about a potential Phase 2/3 pivotal single-dose trial for breast cancer beginning early in next year, we're thinking about 100 to 150 patients, perhaps a year or less to enroll in about a half a year in terms of follow-up, then you're kind of looking at an approval timeline that's pretty potentially aggressive. So that's kind of where we are on the whole and I'll just stop there and Dr. LaFrance wants to weigh in as well.
Norman LaFrance: It's a great question, Justin. This is Norman LaFrance. Everything that Marc says, I think is spot on in terms of explaining, how LM is going. I think a key point I want to emphasize is, LM has pleasantly surprised us in the Phase 1 dose escalation by showing similar to GBM and efficacy signal. We really didn't expect to get that kind of data until the Phase 2, which Marc had mentioned during his remarks is fully funded through Phase 2. Given our success in the Phase 1 dose escalation, the leptomeningeal development is accelerating much more quickly than we anticipated. And of course, we have to go to FDA in some of the aspects that Marc mentioned in terms of the study design. But instead of a standalone Phase 2, there's a reasonable likelihood that we could discuss with FDA at Phase 2/3 pivotal trial, perhaps a Phase 2 with a lead into Phase 3, I won't go into the details now. But the key point is in the last year, the LM data results have been so positive that it's really allowed us to really gain on the GBM trial in addition to the points Marc made. Thanks.
Marc Hedrick: Thank you, Doctor.
Justin Walsh: Got it. Thanks. Yes. So, my next question, you laid out the, I think, pretty well the different data catalysts and conferences we can expect things. I think you mentioned different cohorts. I wonder if there's any color you can provide on kind of expectations on how many additional patients' worth of data we can expect at some of these? I know some, I'm sure it will change by the time we actually get to the presentation.
Norman LaFrance: Yes. So, the way I would guide you, Justin, is, I think as we've said in the past, with respect to GBM, we're looking for another couple of patients in cohort 8 and we think cohort 8 is likely the last cohort just as a maximum feasible dose. What?
Marc Hedrick: It's Phase 1?
Norman LaFrance: In Phase 1, exactly. And then in terms of the Phase 2, we said that's a total of 34 patients. And we think that depending on what meeting we're at, we think we'll have a meaningful update. Our goal, as I said, to get all of those patients enrolled this year. I think that'll be a [Technical Difficulty] pathway to do that and adding three additional sites should help us. So that should give you an idea of kind of what we're looking at from GBM. From LM, so kind of reverting back to previous guidance. So, the FDA, we originally did cohorts 1 through 3. and then we had to go back and do a Part B, which they approved, which was cohorts 4 to 7. We think cohort 7 is probably at the upper end of what is likely to be a safe dose with a single administration. So, as I mentioned today, we're a dose of first three patients and are the three patients required in cohort 5. So, we have a couple of stopping points that are required as part of the trial with the FDA. But I think there's a good chance we'll get through all of those cohorts, whether we get a DLT and one of those cohorts or multiple DLTs that cause us to take that as the recommended Phase 2 dose. That sort of still remains to be seen. I will say that we are at [Technical Difficulty] some of the previous cohorts, where we've dose-escalated, we've done our three patients, not had a safety issue move forward. We've now gone back and backfilled some of those cohorts to maintain momentum in the trial and because of the demand for the product for these patients is so enormous. So, we have an obligation, I think, to try to treat as many patients as we can, the FDA will allow. And as I mentioned also, the demand for additional compassionate use doses as continues to escalate and it's actually stretching our resources a bit across the board. But by going back and rolling additional cohorts and finishing them out to six patients each, it allows us to collect additional safety data that we think will be overall beneficial for the overall program. So, that's a lot. I'll stop there and see if you have any follow-up.
Justin Walsh: Great. Yes, no, that's perfect. One more from me. It's pretty obvious that you guys are quite confident in rhenium obisbemeda's potential in GBM and LM. I'm kind of wondering what you believe the key clinical questions are for the asset at this point. Of course, beyond having to, of course, meet the endpoints in any of your current and upcoming trials?
Norman LaFrance: That's a great question. And it's probably different for each of those two indications, and you could probably expand that to whether you're treating a solid tumor in the brain or spinal cord that could be primary GBM, secondary GBM or brain mets or whether you're treating something in the CSF. So, as it relates to a solid mass in the brain or spinal cord ultimately, the key issue is delivery and absorbed dose. And I alluded to this in my remarks, but there is interest amongst partners and potential partners to expand that to other indications in the CNS and compete, where EBRT is sort of established anchor. And the key to that is case planning and delivery. And I think the drug is there developmentally, and we've shown it to be safe even in large volumes and high-radiation doses. The question is how do we optimize delivery. I think we know how to do that. So, now there, the question of building out case planning and software tools that can make that happen. So, that's how I would characterize that answer as it relates to those indications. The other is CSF. and I think that's a different issue. Delivery is easy. It's a 30-second procedure in the clinic. The question is what's the dose and what's the dosing profile? How many times can these patients tolerate that? And I don't think that's a mystery, just like with GBM. I think we've solved the mystery as it relates to case planning. We just need to implement it. I think we've solved the mystery as to whether the drug stays in the CSF and works. The question is, how do you do you use kind of archaic language that Dr. LaFrance loves, how to fractionate that? How do we take that dose and fractionate it over time, so it could either suppress or cure LM patients? I think that's the key clinical question there. Long answer, sorry.
Justin Walsh: Got it. Yes. No, no, no problem. Very enlightening. Thanks for taking the question.
Norman LaFrance: Thank you.
Operator: Thank you. One moment for our next question. Our next question is from the line of Edward Woo from Ascendiant Capital. Your line is open.
Edward Woo: Yes. Congratulations on all the progress. My question specifically is on that $3 million grant from the U.S. Department of Defense. You said that it's only to cover a Phase 1 in pediatric brain cancer. Is there any opportunities to expand that beyond the Phase 1 funding?
Norman LaFrance: Hi. this is Norman LaFrance. Good question. And yes, the short answer is yes. And we're not at liberty to comment about that. but we are cautiously optimistic there will be other funding capabilities to either accelerate the Phase 1 beyond a single site and to get additional funding for our own financing once those preliminary data are known. Given the adult data and given what we're hearing from the pediatric neuro-oncologists and pediatric neurosurgeons, they're pretty optimistic that we'll get the same platform of benefit that we're seeing in adults and be able to move forward in children. So, your point of what's after the Phase 1 in terms of our interest, and our potential resource and funding capability. We already have some funding options in play. I don't mean to be coy, but we're not at liberty to go over those now. But again, we're cautiously optimistic as Marc has mentioned that those are developing, and we already have a very good platform for the Phase 1 and one of the premier sites in this area. And I would see expanding the number of sites to accelerate completion of Phase 1 and going into a Phase 2 as quickly as possible. Thanks.
Marc Hedrick: Thank you, Ed.
Edward Woo: Great. Thanks for taking my questions and I wish you guys good luck. Thank you.
Marc Hedrick: Thank you.
Norman LaFrance: Thank you.
Andrew Sims: Thanks, Edward.
Operator: Thank you. One moment for our next question. Our next question will come from the line of Sean Lee from H.C. Wainwright. Your line is open.
Sean Lee: Hi. good afternoon, guys and thanks for taking my questions. My first one is on the upcoming ReSPECT-LM updates. So, in the prepared remarks, you mentioned that you are expected to present those updates at quite a few conferences this year. So, I was wondering whether there are any qualitative differences to the type of data that you're looking to present? For example, what can we expect to see at Society of Nuclear Medicine Meeting in June versus what we can expect to see at the last call meeting in August?
Marc Hedrick: Hey, Sean, it's Marc. Yes. thanks for prompting us to clarify that. So, the SNMMI presentation will largely be a re presentation of data already presented. And the rationale for doing that there is that, Norman reminded me, it's one additional cohort. The rationale for that is we've really got three audiences here. We've got the nuclear medicine doctors. we've got the neuro-oncologist and we've got the neurosurgeons, who put the Ommaya reservoirs in. So, it's really important we think long term for getting that data in front of those key constituencies. So, it allow us to go back and present that data to the new med physicians. The SNO/ASCO presentation on LM will be an update of enrollment and also on safety, and that will be as of that time. But we won't get into points of efficacy cell count and so forth. SNO will really be, I think, a more definitive presentation. That will be in November. By that time, I think there's a chance that the Phase 1 will be completely enrolled and we'll have some more meaningful data to discuss at that point. So, that's the data plan. And then I think just as I think, I mentioned this in my remarks. Also, at SNMMI, we'll be talking about the dosimetry data and that's a group that really appreciates that data. and we'll be able to provide that and get academic feedback at that meeting.
Sean Lee: Great. That makes it a lot more clear. Thanks. And my second question is on the pediatric study. So, have you decided on the dosing regimen for use for that? Because I know FDA tend to be pretty strict on these trials.
Norman LaFrance: Yes. This is Norman. I'll take that one, Sean. And very appropriate question. We've -- and I think Marc made this clear that we've already had a couple of iterations with FDA. And I think it's fair to say, we basically have the protocol approved in principle right down to the dosing. We have it basically broken down and it's comparable to how we did LM that we had the initial cohorts with lower [Technical Difficulty] in the pediatric situation, because ependymomas can be large, we're starting off with small [Technical Difficulty] tumors and there'll be both a similar to the GBMA volume and administered dose definition that the FDA has accepted. I very clearly state the FDA very much like the idea of our breaking up the pediatric Phase 1 into 2 segments, the small and medium-sized tumors, reviewing those [Technical Difficulty] that we did with LM with the first initial cohorts to give FDA comfort on what we're seeing, the safety profile, how well it's tolerated. And once they saw that in LM, they were gangbusters for us proceeding for cohorts 4 through 7. We took that success and applied it to the pediatric interactions with FDA and got a similar unanimous acceptance of the pediatric trial and we essentially are waiting we're waiting for this DoD grant submission and now funding, which we're very [Technical Difficulty] now send in the formal IND. We didn't want to get that in and then have it languish waiting for funding and having to work around that and FDA knew that. And there's some final, I'll call it, housekeeping questions we'll take care of with that. But I want to emphasize the dosing, which will be a function and beyond the scope of this call and we can deal with it offline if you really want to get into the study design. but it'll be a volume and administered dose escalation, and with very strong collaboration with the Lurie neuro-oncologists and neurosurgical folks. We're very excited about it. Thank you.
Marc Hedrick: And Sean, I agree. And I think it'll be scaled to cranial volume and to the uniqueness of those particular kinds of tumors, high-grade glioma, which tend to be very infiltrative and ependymoma, which needs to be highly recurrent.
Sean Lee: I see. Thanks for the clarity on that. That's all the questions I have.
Marc Hedrick: Great. Thank you.
Operator: Thank you. And I'm not showing any further questions in the queue. I'd like to turn the call back over to Dr. Hedrick for any closing remarks.
Marc Hedrick: Thank you, everybody for joining the call. We really appreciate the questions and appreciate your interest in the company. And we are thankful also to our doctors, who work with us, to the patients that trust us and our employees that help make it happen. So, look forward to talking to you again, soon. Thank you.
Operator: Thank you for your participation in today's conference. This does conclude the program. You may now disconnect. Everyone, have a great day.
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