Medicinova Inc (MNOV) September 15, 2026 Conference Transcript

That sounds great. So good afternoon. As our moderator mentioned, my name is David Kreen, Chief Business Officer of Medicinova. So let's take a little reason to look at Medicinova. And I believe it's very straightforward. We have a fully enrolled late-stage ALS program, which is approaching a year-end readout on the double-blind portion of the data. We also have a second Phase II catalyst, which is expected in the next two weeks, and a capital structure with no debt and no warrants. So our objective at Medicinova is to reach these value-defining events this year without building the cost structure that is typically associated with a multi-program biotechnology company. So today's remarks include some forward-looking statements and are subject to the risks described in our SEC filings. You can see here MediSnow is led by an experienced and deliberately lean management team. It's headed up by Dr. Yuichi Iwaki, who's president, founder, and CEO of the company. He's been with the company since 2000, so 26 years of institutional knowledge. The relevant point here is that it's not the number of executives, but it's that we have the clinical, financial, regulatory, business development, and public company capabilities of a management team that's going to be needed to manage the upcoming catalysts that we have this year without carrying an oversized organization. Our board certainly adds nicely to that. We bring additional experience across company governance, investment management, private equity, and transactions. That combination is going to be very, very critical and very relevant now that Medicinova approaches these important clinical results and evaluates the regulatory financing and strategic alternatives that may potentially follow. You can see here our investment highlights on this slide, and I'll go over several of these. Our principal value driver is MN166, or known as IBUDALAS, and it's being applied in ALS. Enrollment in that late-stage program, which is a Phase 2B slash 3 clinical trials called Combat ALS, that enrollment is complete. It's 234 patients randomized, and top-line results are expected by year-end of this year. We have regulatory tailwinds with this program. We have orphan drug designation and fast-track designation from the FDA. We also have European orphan drug designation. Ibutilas, importantly, has more than 30 years of commercial experience outside of the United States. So that provides an established body of human exposure information around the molecule. This is not like a new chemical entity. So that's where it really distinguishes from a new chemical entity, where new chemical entities are entering with limited prior human exposure. The second near-term catalyst is MN001. This is being tested right now in a randomized Phase II study in patients with hypertriglyceridemia, type 2 diabetes, and fatty liver disease. Enrollment in that study is complete, and top-line data are expected in the next two weeks. So that's the nearest term catalyst. Now, beyond these two programs, we have additional opportunities, several of which are being advanced with external funding or academic leadership. Our capital position is very, very central to our thesis. We've reported $25.4 million on the balance sheet as of the second quarter. our historical annual operating cash burn has about 12 to 13 million. We've been doing that for about five or six years. We have no debt, no warrants, and our cash runway, importantly, is expected to take us through the key ALS and core program milestones that we're discussing today. You can see here, this really explains our unique model and how we allocate capital. On the left-hand side, you see ALS. That's our core focus. It's our principal value driver and the program receiving direct company investment. On the right are programs in dyslipidemia, fatty liver, oncology, and acute lung injury. Now, these programs create additional potential value, but we're not attempting to finance all these ourselves, every indication through the Medicineova balance sheet. Our core programs receive our focus company capital. Other programs move through the external funding, whether academic sponsorship, government support, or potential commercial partnerships. Now, this structure gives our shareholders exposure to multiple clinical programs without requiring the expense that's typically associated with a broad internal pipeline. So when we refer to our business model as operationally de-risked, this is what we mean. It's important from an execution and a financing standpoint that they've been reduced. We still need to do the clinical testing, and that's the outcome of our combat ALS in ALS, where we believe it's de-risk. So ALS remains a very difficult indication, and the trial that we have ongoing must establish that clinical benefit. See here, our Medicineova's pipeline, it contains two immediate valuation events. As I mentioned, the first is MN001. That Phase II readout is coming in the next couple weeks. The second one and more important one is the combat ALS top-line readout on the double-blind portion of that trial. And that's expected sometime in December. The other programs demonstrate the leverage within our portfolio. Several have certainly reached clinical development through external academic and government support. So the immediate investment case is principally around ALS. That's the approaching of the combat ALS readout at the end of the year. Our financial position gets us through that readout, and MN001 is a second and clinically independent catalyst. Our academic and government relationships are an important part of the business model. The partner programs shown here are funded by government agencies and run by academic researchers at institutions in the United States. Canada, Australia and the United Kingdom. Now these relationships are very important because it provides two forms of leverage. First, independent investigators and government agencies have chosen to commit resources to evaluating our compounds. That's very important. That provides a third-party validation. Secondly, Medicinova does not carry the full cost of conducting these studies so upon completion we retain full rights from a regulatory standpoint and commercialization standpoint these are not substitutes however for our core programs they are externally supported opportunities and that may create additional value without requiring proportional company capital so with that operating model established in everyone's mind let me turn to the program that that will principally determine Medicinova's next stage of value creation, and that's MN166 in ALS. You can see here MN166, as I mentioned, Ibutilis, small molecule, CNS penetrant compound in combat ALS. We're using it or administrating it orally. The molecule affects several pathways associated with inflammation and cellular stress, including macrophage migration inhibitory factor, which is MIF, multiple phosphodiesterases, and TLR4. Now, for the generalist investor, the essential point is not the number of individual targets that we're going after. ALS is biologically complex. MN166 is being evaluated as a multi-pathway approach to neuroinflammation and to neuroprotection, rather than it being a therapy focused just on one mechanism of action or a very narrowly defined genetic mutation. So that provides the biologic rationale for testing MN166 across a broad ALS population. Our mechanistic hypothesis really has three components you see here on the slide. First, inhibition of MIF, phosphodiesterase is in TLR4, and that may reduce the pro-inflammatory signaling and glial cell activation, which is associated with neurodegeneration, especially in ALS. Second, reducing that neuroinflammation component may help protect motor neurons from further injury. And then third, and we get this from preclinical work, which suggests that MN166 may enhance autophagy and support the clearance of TDP-43 protein aggregates, which are associated with ALS pathology. So these findings support the rationale for the MN166 program in ALS. They're not proof of clinical efficacy, and that's why we do clinical trials. The critical question is whether these biologic effects, which I just talked about, translate into a measurable benefit in function and survival. And that's what Combat ALS is designed to answer that particular question. So the decision to advance MN166 into the Combat ALS was really informed by and part by this Phase 2A study that's in front of you here. It was a randomized, placebo-controlled, double-blind study followed by an open-label extension. In this particular study, 51 patients were enrolled, 33 received MN166, plus Riluzal, so it was on top of standard of care, and then 16 received placebo plus standard of care Riluzal during the controlled period. These patients had ALS history of approximately five years, so this was a relatively advanced population in ALS. The responder analysis showed numerically more patients in the MN166 group who were stable or improved on the ALS, FRS, quality of life, and manual muscle testing. So let's need to be precise about this, what this study really established. It was small. The treatment groups were imbalanced. Certainly we recognize that. The responder analysis was not truly definitive evidence of efficacy, but we view these findings as hypothesis-generating. The study itself provided a clinical signal, and when considered alongside the mechanistic rationale, the human exposure experience that we've had with this molecule, it supported moving into a larger multi-center controlled trial, and that's what Combat ALS is intending to do to test whether the signal is reproducible and clinically meaningful. If you're not aware of the ALS market opportunity, approximately 30,000 in the United States are living with ALS and an average life expectancy is approximately two to five years from diagnosis. Existing treatments have not eliminated the need for therapies capable of producing meaningful and durable clinical benefit. In fact, the withdrawal of Relvio, of Amalix, is certainly a reminder to all of us developing drugs in this area that promising early results must be confirmed in a rigorous late-stage study, and it demonstrates why credibility and disciplined interpretation matter in ALS. As you know, ALS is a severe orphan drug disease with high unmet medical need, and MN-166 is not restricted to a small genetic subgroup. We're targeting sporadic ALS at broad populations. So a successful combat ALS result could position MN-166 as a meaningful therapeutic option. And the word could is very, very important to hold on to. The data must establish that, and that's why we're running our late stage clinical trials this is the study that matters most to investors and to and to medicinova combat it's a multi-center study conducted in the united states and canada patients were randomized one-to-one to receive either mn166 at 100 mgs per day or placebo the double blind treatment portion is 12 months followed by a six-month open-label extension. The study itself was designed for approximately 230 randomized patients and enrollment was achieved at 234. We completed that enrollment in September of 2025, so removing the significant clinical trial risk and finishing up the enrollment. The trial enrolled patients within 18 months of onset of ALS symptoms and that is materially earlier in the disease course and the population that we evaluated in our prior Phase IIa study. The primary endpoint here you see is the Combined Assessment of Functionality and Survival, or CAFS, and that combines change in the ALS FRS score with survival. Now this allows the study to assess two outcomes that matter directly to patients. One, how well they continue to function and whether they survive. The secondary endpoints you see there, muscle strength, quality of life, ALS, FRS, responder analysis, survival, safety, and tolerability. The design itself gives us a defined clinical question. Does MN166 produce a meaningful effect on function and survival over 12 months in a broad, early-stage ALS patient population? As I mentioned, enrollment is complete on this study, and our expectation is to report top-line data on the double-blind portion of the trial by the end of the year. You see here, this provides additional detail on those 234 patients at baseline and the study's eligibility criteria. I don't plan to walk you through every baseline measure here, but the data itself provides the context that investors will need when interpreting the top-line results. The protocol sought patients early enough in the disease course to allow study to measure progression while requiring sufficient baseline function and pulmonary capacity for participation. Patients were required to have symptom onset within 18 months and with a defined historical progression rate, and pulmonary function of at least 70% of predicted. The ALS landscape itself includes established therapies, genetically targeted treatments, and several programs in development. I'm not going to go over all this, but I would say that MN166 is differentiated by its oral administration, its broad intended ALS population, and a multi-targeted approach, and its history of commercial use outside of the United States. The commercial opportunity itself exists because currently available disease modifying options remain very limited. Gene-directed treatments apply only to a very small population or small percentage of those patients, and the need for a broadly applicable oral therapy remains substantial. Now, separate from Combat ALS, we also have an expanded access program. you can see here was funded by the nih and sponsoring a 22 million dollar expanded access program known as senobi the program was led by dr oskerson at mayo clinic down in jacksonville and it involved approximately 20 u.s sites it targeted 200 later stage als patients who did not qualify for combat als because of the disease duration and respiratory function and target enrollment on this was achieved in june of uh 2026 a few few months ago the program itself evaluates nfl als frs quality of life inflammatory cytokines safety and tolerability and this is an open label program in a different patient population than the combat als and it should so it's um it's not a substitute for the randomized combat als and it should not be interpreted as such but it's important is that it allows us to expand access to patients may generate additional clinical and biomarker information to represent substantial third-party validation and non-dilutive financial leverage from the nih and from the mayo clinic now let me just go over quickly to our second asset and that's mn166 you can see here it's an oral small molecule anti-inflammatory antifibrotic and lipid-lowering properties. Its mechanisms of action include inhibition of leukotriene and 5-lipooxygenase pathway, phosphodiesterase inhibition, and leukotriene receptor antagonism. And we're trying this molecule out in a very defined patient population, those having hypertriglyceridemia, type 2 diabetes, and fatty liver disease. The hypothesis is that MN166 may affect both circulating triglycerides and liver fat. The prior study that we chose it, you can see it here, we enrolled 19 patients with fatty liver or NASH and elevated triglycerides. It was multi-center, open-label. But from this study, what I can tell you is that we're treating these findings as a signal to be tested, not as a definitive evidence of efficacy. And that's the purpose of the currently randomized Phase II clinical trial. You can see here the ongoing trial is randomized, placebo-controlled, double-blind. It enrolled 40 patients at two centers in the United States. As I mentioned, target patient population, hypertriglyceridemia, type 2 diabetes, and fatty liver. patients received 500 mgs per day of MN001 or placebo for 24 weeks. You can see the primary and the secondary measures, but importantly on the primary, we're looking at change in liver fat as measured by FibroScan and change in fasting serum triglycerides. So it's significant in that But this study provides an independent clinical catalyst, which is a different indication, different mechanism, and potential partnering path, other than what we're doing in the ALS SRS store. So a positive result could support further development and or business development decisions. A negative result would not determine the outcome of what we're doing in the combat ALS study because the two programs are clinically independent. so just to remind you just given the interest of time let me go over to the clinical i'm sorry the financial overview we reported 25.4 million in cash as of the second quarter of 2026 our historical annual operating cash burn has been approximately 12 to 13 million zero debt zero no warrants our cash position is expected to carry the company through the als and the program milestones that i presented today we're not claiming that the current balance sheet however funds full commercialization capital requirements after the readouts will depend on the data on regulatory strategy that we're just discussing with the agencies development priorities and potential partnerships so the investment case is very very focused we offer exposure to a fully enrolled late-stage ALS readout, a separate phase two cardiometabolic catalyst, additional externally funded optionality programs, and a clean capital structure expected to take us through the key milestones. So that is why Medicinova and why now. And at this point, what I'd like to do is turn it over to the audience for any questions that they may have. Thank you.