Matinas BioPharma - Q2 2024

Executive Summary

• Signed a non-binding term sheet for global licensing of oral MAT2203, advancing toward a definitive agreement and Phase 3 ORALTO initiation; management highlighted “dramatic increase” in physician requests for access as a key catalyst.
• Q2 2024 financials: no revenue; total costs and expenses decreased to $5.8M; net loss improved to $5.7M ($0.02 per share) vs. $6.1M ($0.03) YoY; cash and marketable securities were $14.3M at quarter-end, supported by April’s $10M raise.
• ORALTO timeline: company expects trial initiation in Q4 2024, with first-patient-first-visit more likely in 2025; ~24 months enrollment anticipated—timeline clarity is a near-term stock narrative driver.
• LNC platform: continued progress with oncology and inflammation programs, but recent small oligonucleotide efficacy has been less consistent and oncology models showed weight loss at higher doses—optimization underway.
• Street consensus (S&P Global) not available in current environment; comparative analysis vs. estimates cannot be provided—note unavailability explicitly.

What Went Well and What Went Wrong

What Went Well

• Signed a non-binding term sheet with a single partner for global rights to develop and commercialize MAT2203; management is “very encouraged by this progress” and preparing for rapid ORALTO start post-deal.
• Compassionate/Expanded Use Program momentum and efficacy: 31 patients enrolled (6 under evaluation), with 15 completed courses showing 8 complete responses and 7 improved; 7 aspergillosis patients treated with positive results.
• Operating discipline: YoY OpEx down ($5.8M vs. $6.2M), net loss narrowed ($5.7M vs. $6.1M), and liquidity improved with $14.3M cash and marketable securities as of 6/30/24 following a $10M financing in April.

What Went Wrong

• No revenue this quarter (and in the prior-year quarter), underscoring reliance on external partnerships and financing until commercialization.
• LNC small oligonucleotide program showed “less consistent” therapeutic efficacy in follow-up studies—requires additional optimization before product candidate identification.
• Oncology models (LNC-docetaxel and LNC-miriplatin) demonstrated tumor inhibition but also weight loss at higher doses and combinations; management is working to improve therapeutic index and attributes weight loss in part to cargo toxicity and dosing approach.

Transcript

Operator (participant)

Welcome to the Matinas BioPharma Second Quarter 2024 Financial Results conference call. Currently, all participants are in a listen-only mode. Following management's prepared remarks, we will hold a Q&A session. To ask a question at that time, please press the star key, followed by the number one on your touchtone telephone. If anyone has difficulty hearing the conference, please press star zero for operator assistance. As a reminder, this conference is being recorded. I would now like to turn the conference over to Jody Cain. Please go ahead.

Jody Cain (SVP)

This is Jody Cain with LHA. Thank you for participating in today's call. Joining me from Matinas BioPharma are Jerry Jabbour, Chief Executive Officer, and Keith Kucinski, Chief Financial Officer. Dr. Terry Matkovits, Chief Development Officer, and Dr. Terry Ferguson, Chief Medical Officer, will be available during the question and answer session. I'd like to remind listeners that remarks made during this call may state management's future intentions, hopes, beliefs, expectations, or projections. These are forward-looking statements and involve risks and uncertainties. Forward-looking statements made pursuant to the safe harbor provisions of federal securities laws. These forward-looking statements are based on current expectations, and actual results could differ materially. As a result, you should not place undue reliance on forward-looking statements.

Some of the factors that could cause actual results to differ materially from those contemplated by such forward-looking statements are discussed in the periodic reports Matinas BioPharma files with the Securities and Exchange Commission. These documents are available in the investors section of the company's website and on sec.gov. Furthermore, the content of this conference call contains information that is accurate only as of the date of the live broadcast, August 14th, 2024. Matinas BioPharma undertakes no obligation to revise or update any statements to reflect events or circumstances, except as required by law. Now I'd like to turn the call over to Jerry Jabbour. Jerry?

Jerry Jabbour (CEO)

Thank you, Jody. Good afternoon, everyone, and thank you for joining us. We're pleased to begin today's call by reporting that we have signed a non-binding term sheet for the licensing of global rights to develop, manufacture, and commercialize MAT2203. Our ongoing constructive dialogues to partner this oral formulation of the potent yet toxic antifungal amphotericin B cover not only the initial indication of patients with invasive aspergillosis with limited or no treatment options, but potentially all future treatment indications. We believe we have identified the right partner to maximize MAT2203's value in multiple territories. Our discussions have been quite productive and have covered a wide range of topics, from development, strategy, and commercial manufacturing to regulatory and commercial positioning.

We should state that this is a non-binding term sheet and there is no guarantee that it will result in a definitive agreement, but we are very encouraged by this progress. Partnering this life-changing asset has been our top priority, and we've now taken a significant step forward in that process. We have confidence that we are on a path to placing this asset in highly capable hands, which is in the best interest of our company and our shareholders. Preparations are ongoing to enable the ORALTO phase III registration trial with MAT2203 to initiate as soon as possible following the consummation of a partnership agreement. In the meantime, we continue to build real-world clinical evidence supporting MAT2203 in a variety of challenging and life-threatening fungal infections through our Compassionate Expanded Use Access Program.

In this program, MAT2203 has continuously demonstrated its potential to achieve positive clinical outcomes, even in critically ill patients, and to do so safely. Since our last public update on June 24, seven additional patients, ranging in age from 15-71, have gained access to oral MAT2203 through this program. This brings total enrollment to 31, with an additional six patients currently under evaluation. These latest patients were afflicted with a variety of serious and life-threatening invasive fungal infections. To date, 15 patients in the Compassionate Expanded Use Access Program have completed a full course of treatment with MAT2203. The median treatment time was 16 weeks, with a range of two to 49 weeks. Of these 15 patients, eight had a complete response and the other seven were dramatically improved. Response to treatment for all patients was assessed by the treating physician.

Nine additional patients are continuing to receive longer-term treatment with positive ongoing effects, and five have just recently initiated treatment. As of now, only two patients have discontinued MAT2203 under this program, both during the first week. One discontinued due to intolerance secondary to underlying and unrelated GI conditions, and the other due to a terminal condition unrelated to their fungal infection. Notably, seven patients with invasive aspergillosis have been or are being treated, and all of them have shown positive clinical results. The positive outcomes in this program highlight the significant value of MAT2203 and bolster our confidence in the success of the ORALTO phase III trial. It's no surprise that given MAT2203's consistently positive clinical impact and highly favorable safety profile in patients with a variety of deadly invasive fungal infections, we're experiencing a dramatic increase in requests by physicians seeking access for their patients.

Turning now to updates with our LNC platform. Recent studies have increased our understanding of its potential in delivering both small oligonucleotides and small molecule oncology drugs. This includes LNC cellular uptake and cargo delivery. We also continue to build our knowledge base with this technology in the areas of oncology and inflammation. We believe that there are significant unmet needs and a potential opportunity for the LNC mechanism of action to play a meaningful role in combination with effective treatments plagued by inefficient or non-specific delivery and/or significant safety and toxicity concerns. Regarding our LNC formulation of the widely used chemotherapeutic agent docetaxel, following the success in melanoma that we've discussed on past calls, recent studies in animal models of breast, prostate, and lung cancer have all demonstrated varying degrees of tumor growth inhibition with daily oral dosing of LNC-docetaxel.

We have also expanded our in vivo studies to include daily oral LNC-docetaxel in combination with IV docetaxel. These studies have demonstrated an even greater degree of tumor inhibition, but also resulted in added weight loss. We are continuing to evaluate several strategies intended to further improve the therapeutic index of this widely used chemotherapeutic agent. We have also begun evaluating the capabilities of our LNC platform with other chemotherapeutic agents. In vitro testing of an LNC formulation of Miriplatin, which is a highly toxic agent, only approved outside the U.S., for intra-arterial use, demonstrated strong cellular uptake and tumor cell killing capabilities. More recent in vivo testing indicated that while the oral LNC formulation of miriplatin was very effective in inhibiting tumor growth, it was also associated with weight loss, especially at higher doses, which may relate to the overall toxicity of the agent itself.

We continue to evaluate the potential causes of this weight loss. In today's update announcement, we also highlighted a recently completed series of in vitro studies that investigated the potential relationship between the expression of surface phosphatidylserine or PS, and the extent of LNC uptake into certain tumor cells. We previously observed a relationship between tumor cell surface PS and our LNC platform in preferential tumor targeting. Based upon these studies, surface PS expression appears to be one, but not the only driving factor for cellular uptake. Additional work is ongoing to better understand and predict the efficacy of LNC-delivered chemotherapeutics.

Lastly, in reviewing our parallel work in inflammation, following encouraging in vivo data demonstrating the successful oral delivery, biological activity, and potential therapeutic efficacy of two different LNC-formulated small oligonucleotides targeting the inflammatory cytokines IL-17A and TNF alpha, the findings from more recent follow-up in vivo studies have been less consistent. As such, additional optimization is required prior to identifying a potential product candidate. Overall, we believe good progress has been made in better understanding the potential for the LNC platform, but there is significant time and additional work ahead for us to be able to identify additional potential product candidates. We expect to be in a better position to provide additional guidance following the consummation of a MAT2203 partnership. With those comments, I'll turn the call over to our CFO, Keith Kucinski. Keith?

Keith Kucinski (CFO)

Thank you, Jerry, and good afternoon. Today, the company reported its financial results for the second quarter of 2024, which reflected a net loss of $5.7 million or $0.02 per share. This compares to a net loss of $6.1 million or $0.03 per share for the second quarter of 2023. The company did not report any revenue during either of these periods. Total costs and expenses for the second quarter of 2024 were $5.8 million, compared with $6.2 million for the second quarter of 2023. The year-over-year decrease was primarily due to lower clinical development expenses, personnel costs, and administrative expenses. Turning to our six-month results.

Our net loss for the six months of 2024 were $11.5 million, or $0.05 per share, compared with a net loss for the first six months of 2023, of $11.6 million, also $0.05 per share. We reported no revenue for the first six months, I'm sorry, for the six months ended June 30, 2024. This compares with $1.1 million in revenue for the prior year period, which was generated from research collaborations with BioNTech and Genentech. Total costs and expenses for the first six months of 2024 were $11.7 million, compared with $12.8 million for the first six months of 2023. This decrease primarily reflects a decline in R&D expenses, as well as lower general and administrative expenses.

Cash, cash equivalents, and marketable securities as of June 30th, 2024, were $14.3 million, compared with $13.8 million as of December 31st, 2023. This increase reflects $10 million in gross proceeds raised through a registered direct offering in April, more than offsetting the company's negative cash flow from operations over the six-month period. With that, I'll turn the call back to Jerry.

Jerry Jabbour (CEO)

Thanks, Keith. In closing, we are reporting positive, real progress in consummating a partnership transaction with MAT2203, that we believe will be in the best interest of our company and bring value to our shareholders. We have signed a non-binding term sheet with a potential global partner and remain engaged in ongoing constructive discussions with our prospective licensee, with the goal of finalizing this transaction as soon as possible. Our Compassionate Expanded Use Access Program continues to highlight the ability of MAT2203 to safely target and effectively eradicate a variety of severe, potentially deadly invasive fungal infections, even in the most challenging clinical circumstances. This program is gaining awareness within the medical community, with requests for access to MAT2203 significantly increasing in recent months.

In fact, just yesterday, we received two additional requests, bringing us close to 40 total enrollees, should all current evaluations result in patient admittance and emergency IND approvals. This essential program is functioning like a basket study of invasive fungal infections and could position us and or our partner for the support of potential expanded label discussions at the time of NDA filing. We continue to expand our knowledge base with our LNC platform. We're evaluating the best next steps for this technology as we determine how to maximize return to shareholders, and look forward to providing additional guidance on next steps once we have secured our MAT2203 partnership, which remains the key focus of the company. With that review, I'll turn the call over to the operator for Q&A. Morgan?

Operator (participant)

As a reminder, if you wish to register to ask a question in today's Q&A session, you will need to press star, then the number one on your telephone. If your question has been answered and you wish to withdraw your request, you may do so by pressing the pound key. If you are using a speakerphone, please pick up your handset before entering your request. One moment, please, for the first question. Your first question comes from Scott Henry with Alliance Global Partners. Your line is open.

Scott Henry (Managing Director and Senior Research Analyst)

Thank you, and good afternoon. Just a couple quick questions. First, good news on the non-binding term sheet. Obviously, you can't give out specifics, but generally speaking, should we be thinking about an upfront payment and as well, reimbursement for the ORALTO trials? Are those kind of key components of what you're looking for in this type of deal through this agreement or another one?

Jerry Jabbour (CEO)

Hi, Scott. Thanks, thanks for the question. You know, actually, without commenting on it specifically, I do think it's appropriate to say that we would expect, you know, not just a non-binding term sheet, but a definitive agreement to be structured similar to what you would typically see and have seen in this area, with some combination, you know, of an upfront payment, certainly development and commercial milestones, royalties, and the additional potential elements of, of how you're going to treat clinical development costs, and manufacturing, which we have outlined for investors before, specifically referencing, our intended tech transfer of manufacture, from here, at Matinas to Thermo Fisher.

So there's a lot of moving parts there, but I think, you know, as investors try to get their arms around what is the meaning of a non-binding term sheet, it's just that, it's the framework for a deal. But all of those elements have been discussed, and I think it's safe to say that any definitive agreement that we arrive at with this intended partner on a global basis would include, you know, some combination of those essential elements.

Scott Henry (Managing Director and Senior Research Analyst)

Okay, thank you for that color. And then, you know, given the timeline for a partnership is taking a little longer than expected, would you still expect the ORALTO trial to start in fourth quarter of 2024, or perhaps we should think about early 2025? How would you think about that?

Jerry Jabbour (CEO)

Yeah, it's a fair question, and I think it's appropriate for us to address that this has taken, I think, longer than any of us have expected, and I think that speaks to the complexity of these sorts of dialogues. But it also speaks to how important it is to have complete alignment across the board and no surprises. I mean, if you look in this category in the recent past, you know, you had a global big pharma that was surprised by CMC issues, which arose after a definitive agreement was signed. And I think that has caused certainly everyone in this category to make sure that all of those boxes are checked.

So the diligence process around this transaction has been pretty thorough, and I think that has explained, you know, the passage of time as much as anything else. And why it's taken a little bit longer, I do think it eliminates surprises, you know, post a partnership announcement and does put you in position to get things started as quickly as possible once a transaction is consummated, and if a transaction is consummated. In terms of timing, we're not sitting on our laurels, so the team here is actively working with a global CRO to sort of put pieces in place. You know, it's a sliding scale, Scott, in terms of, you know, are you going to have product ready?

Any time, you know, that you're getting ready to start a phase III trial, there are a number of steps that need to be done and boxes checked before you can have first patient, first dose. But we do think that we have confidence that the study can initiate in the fourth quarter of this year. But whether or not that results in a first patient, first visit in 2024, that's probably safer for 2025. But taking that into account, there are a number of steps that we've taken in discussions with the global CRO to take sort of this delay into account, make some adjustments to how the study is set up, the number of sites, so that you're not losing sort of enrollment time on the back end.

So we've tried to be proactive in addressing that issue so that at the end of the day, not only are we putting ourselves in position to initiate the trial or our partner to initiate the trial as soon as we can, but also sort of maximize the time for phase III and sort of get through it as quickly as we can. So eventually we can have this product be filed under an NDA and be ready for commercialization, you know, as soon as possible. We still project that enrollment's gonna take about 24 months in this study. So things have lined up. Yes, the calendar has slid a little bit, but we're working hard to make sure that doesn't have too much of an impact on the back end.

Scott Henry (Managing Director and Senior Research Analyst)

Okay, great. Final question, with regards to the LNC platform, in the oncology indications, yeah, how should we interpret the additional weight loss we're seeing? Obviously, you know, typically a good thing, but less so in an oncology trial. Would you think that is molecule related, or would you think it's a lack of thriving? Just curious. I saw it in there a couple of times, how you would interpret that.

Jerry Jabbour (CEO)

Yeah, and this is like, Scott, it's a great question, and this is sort of the interesting and challenging part of early-stage drug development, right? Because at the same time, you know, you're trying to drive and show that you have biological activity and therapeutic efficacy, particularly, I mean, and biological activity would apply more to the oligonucleotide. But if we're talking about LNC-docetaxel, for example, some of what we're doing is cranking the dose really high. And so that, to some degree, when you're talking about docetaxel, we think could explain some of the weight loss.

But we also think, remember, this is an oral therapy that's traditionally been given IV, and when you're giving an oral therapy in animal studies, you could have gavage issues, which could have all sorts of ramifications on how these animals feed or eat, which could be contributing to the weight loss. And so that's in the case of docetaxel, and that's why we're looking for ways to sort of optimize that formulation to not only increase the therapeutic index, but be able to give as much as we can and even split the dose into two doses, and we've even begun to see a dose response there, which is positive. When you look at Miriplatin, you're dealing with a different animal altogether.

It's a highly, much more highly toxic agent, and different cargo, sometimes, you know, create different characteristics of the LNCs. In the case of Miriplatin, I think without drawing too many conclusions, because a lot of work is still ongoing internally, we would attribute more of the weight loss we saw on the Miriplatin side to the toxicity of the agent, but we're doing some additional work to verify that that is in fact the case. And at the end of the day, what we want to be able to do is encapsulate whatever chemotherapeutic agent, you know, we're targeting in a way that we can create that oral availability, that we can still have that, specific tumor targeting and lower toxicity. And we're just in these early stages, you're optimizing, you're cranking the dose sometimes, you're splitting the dose sometimes.

So it's not surprising that in some contexts you're going to see weight loss, but the team is working hard to better understand those, and at least in the case of Miriplatin, we think it's agent specific and not related to the platform.

Scott Henry (Managing Director and Senior Research Analyst)

Okay, great. Thank you for taking the question.

Jerry Jabbour (CEO)

Scott, thanks very much.

Operator (participant)

Your next question comes from Jason McCarthy with Maxim Group. Your line is open.

Joanne Lee (Biotech Equity Research Associate)

Hi, this is Joanne Lee on the call for Jason McCarthy. Thanks for taking the question, and congratulations on securing the partnership for ORALTO. My question on the, you know, anticipated timeline for the phase III launch was answered, but just, you know, as a follow-up, with the recent milestone achieved, could you just remind us on some of the key design elements and, you know, objective measures of the upcoming and non-inferiority study?

Jerry Jabbour (CEO)

Sure. Joanne, thanks very much for the question, and I'm going to turn it over to Dr. Matkovits to talk about the design. But I would say we're really proud of our progress. I wouldn't quite say milestone achieved yet. You know, we have not secured the definitive agreement, and obviously, we're working very hard to deliver that as soon as we can, and that's when we'll pop the champagne. But we do think that this is an important, you know, progress toward that milestone, narrowing it down to a single party, working with them to make sure that all of those questions are answered and that we're setting the program up for success as soon as we pass that baton.

We don't want to have any drops, but that we'll consider the milestone achieved when we have a definitive agreement, so just want to be fair about that. But Terry, for a second, why don't you just remind everyone what that design element of the ORALTO study looks like, number of patients, what we're going up against, and what's the key primary and secondary endpoints?

Terry Matkovits (Chief Development Officer)

Sure. Thanks, Joanne, for the question. So to remind everyone, the ORALTO trial is a non-inferiority trial, a 2-arm trial, where we are targeting assessing the efficacy of MAT2203 after a 2-day IV ampho step down to MAT2203 with treatment out to 12 weeks. And this non-inferiority trial will be targeting patients with invasive aspergillosis who have no other treatment options. So they either have a toxicity or a lack of tolerance to the azole class, or they have some significant clinical drug-drug interactions that preclude them from the ability of being treated with an azole. So the comparator arm will be an arm where patients will receive IV liposomal amphotericin for as long as they're able to tolerate the treatment, with the primary endpoint of mortality at 6 weeks.

Now, we've added some key secondary endpoints to the trial because we believe that one of the key attributes of MAT2203 is the ability to receive the drug longer term without any of the safety sequelae that you see with IV-administered ampho. So a key secondary endpoint will be safety, looking at the proportion of patients who have to switch from IV amphotericin in the comparator arm to another treatment regimen, whether that be a drug holiday from IV ampho or a reduction of dose, and comparing those toxicity-related dosing changes, if you will, and testing for superiority. And that test would be done at the 12-week endpoint.

We'll also be looking at global response to treatment, so looking at clinical, radiological, and mycological response at 12 weeks, and also importantly, looking at the pharmacoeconomic impact of oral MAT2203 because the value proposition, in addition to the safety benefit, is the ability to transition patients as early as possible to the outpatient setting, where they have a lower use of healthcare resources and a higher quality of life. And so we're tracking this in our ongoing compassionate use program, where we're seeing that patients are very quickly transitioned to home care after they're able to switch from IV amphotericin to our oral MAT2203. And of course, throughout the course of the study, we'll be evaluating mortality through the 12-week endpoint.

Joanne Lee (Biotech Equity Research Associate)

Great. I appreciate all those details. Looking forward to more updates on the program. Thank you.

Terry Matkovits (Chief Development Officer)

Thank you.

Jerry Jabbour (CEO)

Thanks, Joanne.

Operator (participant)

At this time, there are no further questions in queue. I'd like to turn the call back over to Jerry Jabbour for any further remarks.

Jerry Jabbour (CEO)

Thanks, Morgan, and thank you for joining us today and for your interest in Matinas. We remain excited and very optimistic about our company's future and look forward to reporting further progress during our third quarter conference call in November. Thank you again, and have a nice evening.

Operator (participant)

This concludes the Matinas BioPharma Second Quarter 2024 conference call. Thank you for attending, and have a wonderful rest of your day.