Hello, and thank you for joining the H.C. Wainwright's twenty-sixth Annual Global Investment Conference. My name is Oren Livnat, Specialty Pharmaceuticals Analyst here at H.C. Wainwright, and it's my pleasure to welcome Prashant Kohli, CEO of Acasti Pharma. Acasti is a late clinical stage drug delivery company, with its lead asset aiming to be a new standard of care for the urgent treatment of life-threatening aneurysmal subarachnoid hemorrhage, or SAH. I do cover Acasti with a buy rating, and I see it as a very much under-the-radar story, and we look forward to the completion of enrollment in its pivotal trial around the end of the year, and NDA filing potentially first half of next year. So it's a very exciting time at Acasti, and with that, I'll let you take it away, Prashant.
Thank you, Oren. Appreciate it. Hello, everyone. Excited to join the H.C. Wainwright Conference this year as well and present the Acasti story. Forward-looking statements disclosure here. So to summarize, Acasti Pharma, we're late stage, as Oren mentioned, targeting a rare disease called subarachnoid hemorrhage. Our asset, GTX-104, is ideally positioned to disrupt the standard of care dosage form for SAH or subarachnoid hemorrhage condition. The current dosage form of nimodipine, which is a calcium channel blocker and the standard of care in subarachnoid hemorrhage, is only available as an oral capsule or a solution.
And it presents significant unmet medical needs, which we believe our GTX-104 novel intravenous formulation of nimodipine is very well positioned to address those issues, and potentially displace the oral formulation to become the standard of care in subarachnoid hemorrhage. So clinically, the molecule nimodipine is de-risked. It is written into the care guidelines of SAH by the American Heart Association and American Stroke Association. The Joint Commission monitors the usage of nimodipine at hospitals where these patients are treated. So it's not an optional therapy or one that requires physicians to be convinced to adopt nimodipine. It's already being used in the United States, predominantly in near 100% of the patient population that are treated in a certified stroke center.
The regulatory pathway for GTX-104 is relatively de-risked as well, requiring one pivotal safety trial called STRIVE-ON, and we are way past the 50% enrollment in that trial. We already have data in over 160 subjects, multiple phase 1 trial, very compelling safety data, so we have strong confidence going into our STRIVE-ON trial. We anticipate the trial wrapping up enrollment, fully enrolled, late this year, very early next year. We're on track to filing our potential NDA in the first half of next year. It's a compelling commercial market opportunity, $300 million serviceable addressable market, $500 million total addressable market.
We have an orphan drug designation for GTX-104, and we have a robust patent estate covering not only the United States, but also the major global markets ex-US. Our cash position is strong, fully funds the STRIVE-ON trial, inclusive of filing our NDA application middle of next year. Our cash runway as of our most recent guidance is into second calendar quarter of 2026. So we have a strong investment thesis, late-stage company, you know, with a very, very near-term catalyst event, wrapping up our trial in short order. With that, I will switch gear and talk a little bit about the condition SAH. Life-threatening condition, it literally strikes out of the blue, relatively younger age. Average age is about 59 years old.
For reasons not entirely well understood in the medical community, more women than men are afflicted with SAH. It's described as the worst headache in one's life. A number of patients are, you know, do get unconscious upon the insult. Ideally, these patients are taken to a certified stroke center. CT angiogram diagnosis is the gold standard, which detects SAH. That is a rupture of an aneurysm deep inside the brain. Annual cases 50,000 in the U.S. As you can imagine, surgical intervention is required to stop the bleed, either endovascularly. It used to be more craniotomy, but in the U.S., it's moved endovascular. The patient then is put on nimodipine therapy.
Nimodipine is the only FDA-approved drug to improve that has been demonstrated in multiple efficacy trials to improve outcomes in SAH. The patients are on nimodipine therapy for up to 21 days. About a third of the patients, rule of thumb, is ultimately do not survive, so it has very high, the disease has a very high burden. Another third end up living with long-term morbidity. Very, very tough, very challenging condition on patients and their family members. What are the challenges with oral nimodipine? As you can imagine, these patients are severely ill. Half of them, more than half of them are either unconscious or dysphagic, unable to swallow. Nimodipine has very high dosing burden. It has also very high first-pass metabolism, which requires the drug to be given very frequently, several times a day.
In fact, it's every four hours, six times a day for up to twenty-one days. These are two massive pills, 30 milligrams each. So it's 60 milligrams dosed six times a day for up to twenty-one days. In an ICU setting, especially in a critical ill patient population, physicians prefer to stick a needle in, and that's how they, you know, prefer to give the drug, most drugs. Historically, there have been few severe medication errors where the oral formulation, the contents were extracted, syringed out, and parenterally injected to the patients, which led to few deaths as well.
So there is a danger of fatal medication error, goes on to show how quote-unquote intuitive, natural, normal it is for clinicians in an ICU to deliver a drug by sticking a needle in. About half the patients, 45%, receive the drug through the nasogastric tube. There's published literature with PK analysis that has demonstrated when the drug is given through the feeding tube, the bioavailability is barely detectable in blood. And if it's not in the systemic circulation, it's not gonna get to the brain, where it's required to improve outcomes. In an ICU, very challenging with these patients being heavily monitored as well by the clinical staff, there's an issue of cognitive overload.
One of the side effects of the drug is hypotension, where the blood pressure drops. Oftentimes, physicians have to intervene by withholding the dose of nimodipine to stabilize the blood pressure. In about a third of the cases, patients have to be administered invasive rescue therapy, intra-arterial pressors, sometimes even angioplasty. So the only drug known to improve outcomes is routinely being withheld from patients due to this hypotension issue, and, you know, could lead to poor outcomes. There is an oral solution of nimodipine that also is administered through the nasogastric tube. That formulation was really designed to address the challenge of a capsule preventing the clinical staff to extract the contents of the capsule and squirting it down the nasogastric tube.
However, that liquid formulation does cause severe diarrhea. Published literature shows an average of four days of severe diarrhea in patient population. In our multiple phase I trial, we also identified significant PK variability that either is too low and if it is that, then might be subtherapeutic dose or too high, which a lot of clinicians believe could cause to hypotension. So a lot of challenges with oral and those are the issues we believe an intravenous formulation, GTX-104, the first-ever aqueous formulation of nimodipine, can help address. It's a lyophilized technology, room temperature stable, using GRAS excipients. Very elegant formulation that has been shown to have a lot of stability as well in our extensive CMC work that we have done with GTX-104.
In terms of the value proposition from a clinical standpoint, IV 100% bioavailable, predictable drug concentration, we believe, also offers better hypotension management. IV allows for easier titration of dose in real time to manage blood pressure, making sure that the patients are on nimodipine therapy for the full therapeutic dose, higher probability of ensuring that, as opposed to oral, which has to be withheld, and also issues with nimodipine around GI liabilities, gut motility issues, CYP liabilities , we believe, are adequately addressed with IV as well.
From a hospital standpoint, better managing hypotension is the strongest clinically relevant value proposition, reducing nursing burden associated with giving nimodipine therapy several times a day, could potentially lead to shorter ICU length of stay and compliance with the Joint Commission. All of those value propositions, we believe, should lead to better patient outcomes, faster recovery and safer as well. In terms of the phase I data that you know we have had multiple phase I studies, our last one was in 2022. When we met with the FDA pre-pandemic, and FDA has been very consistent in terms of regulatory pathway, what it would take for us to submit an NDA application.
This is step one of two, which was to ensure from a PK standpoint, we met day one Cmax and day three AUC, which this trial successfully accomplished, and step two is the STRIVE-ON trial leading to the potential NDA. I'll share some of the data with you. GTX-104, the blue box versus the green and nimodipine. We were not required to meet bioequivalence, however, we did 80%-125%, day one Cmax and day three AUC. As you can see on the first box, 104, 100% bioavailable, given that it's an IV formulation. Nimodipine capsule's only 7.2%. So what that means is at a fraction of the dose, in fact, 28 milligrams given via IV, is pharmacokinetically equivalent to 360 milligrams given orally.
Whenever you have such a strong, such a large reduction, obviously, is a better sign from a safety standpoint as well. Not only that, the pharmacokinetic variability with oral is all over the map, as seen in the green box and whisker. You're looking at exposure data here, and IV very well constant. Very, you know, very predictable, narrow band, highly, you know, predictable concentration of nimodipine. We believe this profile of IV relative to oral is where the hypotension issue can be adequately addressed. And we saw that in the data for hypotension in our phase 1 trial.
This was in healthy subjects, but very comparable hypotension, if not a slightly better hypotension profile relative to the oral capsule. The trial was not designed to tease out any differences in hypotension, but you know, we do see a signal here in our phase 1 trial. So on the body of the strong safety data, safety profile, we initiated the STRIVE-ON trial in the fall of 2023. The design of the trial is a 100-patient, open-label trial, randomized one-to-one, 50 on the IV arm, 50 oral, and it's a safety trial. Our primary endpoint is to demonstrate comparable hypotension between the two arms. That's what the FDA guided us to, along with other AE data that we, you know, we're capturing.
In addition, we are also capturing health, economic, and outcomes data. We believe these data points can really help us to commercialize and hopefully detect a signal that will allow us to market more effectively the drug. It's a 21-day treatment period, up to 21-day treatment period. The follow-up is day 30 and day 90. As I mentioned earlier, we are more than 50% enrolled in the trial, well on our way to hitting the 100-patient mark. The addressable market, we have done extensive legwork understanding the dynamics, selling into a hospital, the P&T Committee formulary. The work started several years ago, right at the cusp of entering the clinic with our first phase 1 trial.
What our primary research found is that there's a high concentration of patient volume in the hospital setting. About 400 hospitals in the country treat about 70% of the patient volume. So in terms of bringing the drug to market, highly experienced hospital sales force, 30-40 reps, we can bring the drug to market ourselves. Our team has experience bringing drugs to market, rare and orphan disease in a hospital setting as well. But opportunistically, we'll also be looking at partnership opportunities, both in the US as well as outside the US, where we believe that there's a significant upside potential in Europe and China in particular as well.
We had earlier this year done some more follow-up work with P&T Committee decision-makers in a subset of these hospitals, and strong likelihood, in fact, 80% indicated embracing GTX-104 on their hospital formularies. You know, assuming the target product profile that was outlined earlier and the strong value proposition. So we've had good receptivity from the market. Competitively, there is nothing on the horizon anywhere near the stage that GTX-104 is in. Nimodipine is a foundational therapy, standard of care in the US. It's gonna be there for a very long time, and we believe GTX-104 can be that foundational therapy with very long commercial legs and could be highly profitable upon potential approval.
I talked about our orphan drug designation, seven years market exclusivity in the U.S., with a strong patent, patent firewall. In fact, our first patent doesn't expire until 2037. The earliest one, the most latest one is 2042. So good protection, both from an orphan designation standpoint and patents. Strong team, highly experienced in the disease state. Dr. Loch Macdonald, he's our Chief Medical Officer, world's leading authority, neurosurgeon, physician, scientist in the field of subarachnoid hemorrhage, the most cited author in SAH. He's been instrumental in driving our strategy with the FDA as well as leading the STRIVE-ON trial. In addition, we have a strong clin ops and a product management team, rounding up our staff.
Our last reported cash position as of June thirtieth, about, you know, just shy of $20 million. As I mentioned earlier, we have strong cash position to fully fund our trial, take us past NDA filing, into, you know, into well into 2026, calendar 2026. So with that, I'll pause here. Thank you for your time, and I'll turn it over to Oren.
Thanks, Prashant. Very exciting. I had a couple follow-up questions. Can you talk a little bit more about what you need to show in the pivotal safety trial, perhaps versus what you hope to show in a best-case scenario to maximally differentiate your product? Is there any potential with the 30- and then 90-day follow-up to show, even if not powered for it, to show superiority on any sort of efficacy or outcome perspective?
So I'll talk about the regulatory first, and then address your second question. From a regulatory standpoint, what the FDA set the bar is to show, demonstrate comparable safety relative to oral. This is not a statistically powered trial, as you mentioned. So 50 IV, 50 oral, our primary endpoint is a number of patients that develop a clinically significant hypotensive event between those two arms. We have to be comparable from a hypotension standpoint between the two arms. On the heels of strong phase I data, we believe that there's good, you know, good evidence going into the trial should be relatively de-risked.
Now, in terms of addressing your second question, you know, we're casting a wider net with health economic outcomes data as well, and at day 30 follow-up and day 90 follow-up, you know, we hope to tease out a signal, some clinical benefit between the two arms, even though it's not really designed... The study is not designed to do so. But we believe that there's a possibility to show some benefit between those two arms with the IV, you know, ideally performing a lot better than oral. But from an FDA standpoint, you know, that's not the bar we need to hit.
I'm getting a lot of good anecdotal feedback from hospitals that are recruiting patients in STRIVE-ON. My team and I personally spent a lot of time visiting these hospitals, talking to PIs, talking to nurses and physician staff. Strong ease of convenience. The use, the benefit of IV. One physician talked about a stick-it-and-forget-it type, you know, analogy with an IV relative to all the choreography they have to jump through all these hoops to get the oral shoved down the feeding tube and manage food effects and drug interactions. So that clearly was really encouraging to get some anecdotal feedback around the experience of physicians and clinicians.
Yeah, you sort of segued right into my next question, which is gonna be along that early experience. I mean, of course, it's an ongoing trial, and you said it's anecdotal, of course, but this is open label, obviously. And I'm just curious, you know, has training gone as expected? Clearly, it should be easier, but any change in procedures is always something new. And I'm curious what the experience was there. And also, anecdotally, besides ease of use, the set-it-and-forget-it aspect, are people excited in these centers? I mean, this is a potentially life-changing therapy, right? Are there any early reports on people perhaps seeing better efficacy or just, you know, a smoother, safer experience with this product?
It's good. Now, imagine I can't really address, you know, the harder endpoints, you know, for the trial. But in terms of the feedback, has been very encouraging, very excited, very energized. Hospital staff, lead physician, you know, physician investigators, PIs, principal investigators, as well as the hospital staff. There competitively isn't any other trial for subarachnoid hemorrhage ongoing, nor anything on the horizon. And so, there is a... There was a lot of excitement. We had well over 100 sites that wanted to participate in the trial, and these are relatively, you know, high-volume sites, and we picked the top 25 to 30 sites to be part of our trial.
So that just goes on to show how much excitement there was to be part of our trial. Continue to see a lot of excitement. Anecdotally, you know, we had PIs share how you know, there's some patients that had to be switched over to oral for whatever the reason was, as they were being discharged into a step-down unit. And you know, they liked the IV ease of use and, you know, couldn't fathom swallowing these two massive pills now. So again, both from a patient standpoint as well as, you know, the clinical, the physician staff standpoint, you know, there is this intuitive you know, delivery with IV that just sticks.
And we'll wait for the data to see if we can tease out some more meaningful differences. We believe hypotension, better hypotension management will certainly be one of the strong value propositions, and making sure that the patient is on as close to the therapeutic dose you know as practical, relative to frequent missed doses on the oral side. And all of those you know will you know should lead to better you know better outcomes. But that's not how our trial is not designed to show superiority from an efficacy standpoint.
You know, assuming everything goes as planned, and this product can be approved, do you expect the AHA and ASA guidelines to be updated in due course to specifically include or perhaps, recommend as first line treatment, your product versus oral nimodipine?
So we have been thinking very, very long and hard about exactly, you know, what it would take for us to be written into the care guidelines for GTX-104. The last update to the guidelines was in 2023, first half of 2023 last year. Typically, most guidelines require 18 to 20-24 months of real world evidence data once a drug is on the market. Our strategy fully calls for making sure that we have surveillance, we are capturing data once the drug is on the market, build a strong body of evidence. We have an exceptional Scientific Advisory Board, key opinion leaders, who's who of this SAH space in the neuro ICU. The sites that we have picked for the trial, our trial, the PIs are leading voices in this field.
So triangulating all of those stakeholders and collecting RWE should allow us to really proposition for GTX-104 to be written into the care guidelines, and we believe it should be the standard of care for patients in the United States. Given the issues and challenges with oral, why would you take any risk and expose patient to suboptimal, you know, therapeutic dose of the only FDA-approved drug known to improve outcomes? There is nothing else in the US, so those are, you know, tailwinds that hopefully we, you know, we can put together a strong, robust case, not only upon launch, but also continue that sustained effort, you know, and really get written into the care guidelines.
That's certainly one of our commercial goals as well.
Sure. Well, Prashant, we are bumping up against our time. I greatly appreciate you presenting and joining us here at the conference, and I wish you luck. It's only a few months away. A lot of exciting times coming.
Thank you, Oren. Appreciate your time.
Thanks for joining.