Acurx Pharmaceuticals, Inc. (ACXP) Earnings Call Transcript & Summary

Good afternoon, everyone. I'm Colin Minicus, I'm an associate here at JPMorgan's Healthcare Group. It's my pleasure to welcome Acurx to the 42nd Annual JPMorgan Healthcare Conference. As a reminder, we'll plan to hold Q&A at the end of today's session. Without further ado, please welcome CEO, Dave Luci.

Thank you, Colin, and thank you to our host at JPMorgan for this opportunity. We're delighted to be here today. We'll have a Q&A after a brief presentation, and we look forward to any questions. Today, we will have forward-looking statements, and we do refer folks to our SEC filings for our 10-Qs and 10-Ks and historical information. Our executive summary, Acurx Pharmaceuticals, NASDAQ ticker ACXP, is developing a new class of antibiotics to treat difficult-to-treat Gram-positive bacterial infections with a lead antibiotic candidate targeting the treatment of C. difficile infection and moving into Phase III. It's a novel mechanism of action. It's -- what we do is we target the polymerase IIIC enzyme, which is the primary catalyst for production of new DNA in low guanine and low cytosine Gram-positive bacterial cells, which include C. difficile and with our second antibiotic candidate, MRSA. C. difficile is an area of unmet medical need according to the CDC. There are 2 antibiotics currently recommended to treat C. difficile in the U.S. The first is oral vancomycin, which has a 20% to 40% recurrence rate and the second of which is fidaxomicin, which is used last line, which is unduly expensive in terms of cost and also has a 14% recurrence rate. We feel with our antibiotic candidate going into Phase III, cumulative data thus far supports clinically comparable data to both of these antibiotics currently approved with a better safety profile and with no reinfections, 100% sustained cure for all patients that use our drug with mild-to-moderate C. difficile infection. We also have a cost and a price point in mind that could significantly undercut either of these drugs. So we are Phase III ready with successful Phase II data, 96% cures at end of treatment, 100% sustained cures, meaning no reinfections up against vancomycin. We need to replicate that trial in Phase III. From a financing point of view, we have the funds we need to start enrolling patients in Phase III, $9.2 million at the end of the third quarter of '23, and we put up a $17 million ATM, which we're drawing down upon to pay for the first of the Phase III trials. And CMC is in process for Phase III drug supply. And our cost of goods is anticipated at $300 or less for a full course of 10-day treatment. So the takeaway from the executive summary slide is we're going into Phase III with a drug that has got clinical efficacy, comparable to any other antibiotic recommended to treat C. difficile, a great safety profile. We're restoring the microbiome in the process, and we have sustained cures across the board with no reinfections. So let's dig into the details. This chart shows the data for the 2 antibiotics approved to treat C. difficile infection in the U.S. that are recommended, vancomycin and fidaxomicin. It also includes ridinilazole from Summit Therapeutics, which now failed as of the fourth quarter of last year in Phase III. So we're next up. From a regulatory point of view with the FDA, we're next in line going into Phase III with data that, thus far, hasn't been seen in C. difficile over the past decades, and it's first in class of a new class of antibiotics. Antibiotics are still the gold standard to treat patients with mild-to-moderate C. difficile infection. There are other companies like Seres Therapeutics with VOWST, and there's Rebiotix in rCDI market, recurrent C. difficile infection, but only used after frontline antibiotic. Now if ibezapolstat consistently -- is consistent in Phase III with the data that we've generated to date and gets approved, one could reasonably ask if we've solved for rCDI, because the patients that take ibezapolstat frontline thus far are not experiencing any reinfections. There are also fecal transplants in severe cases of C. difficile infection. They have FDA alerts on their labels, so they're unsafe, and they're only used in extreme situations. Our pipeline does include a second pol IIIC inhibitor, which is ACX-375C. It's preclinical at the lead optimization stage of development. And we're excavating 2 to 4 more molecules behind that all pol IIIC inhibitors that we hope to get into preclinical activities in 2024. The mechanism of action of ibezapolstat, it's active against the pol IIIC enzyme, which only exists in low guanine and low cytosine Gram-positive bacterial cells, which include importantly, MRSA and C. difficile bacteria. And we're able to lyse the cell and stop the pol IIIC enzyme from building new DNA for new bacterial cells. C. difficile is a large market that we're solving for, $1.5 billion currently. And in 8 years' time, it will be up to $2.3 billion. So it's a big market and growing. So if we were to be used frontline in this market, we think the recurrent C. diff portion of the market would be significantly reduced. So our Phase II success includes patients in mild-to-moderate C. difficile infection, 25 out of 26 patients in Phase II had a cure at the end of treatment at day 10 as measured on day 12, and 25 out of 25 of those cures on 100% were sustained cures. From an IP and regulatory perspective, we have 10 years of market exclusivity for ibezapolstat, 5 from being a new molecular entity and 5 from our status as qualified infectious disease product designated by the FDA. So that's more than enough that would be needed to maximize our commercial potential of ibezapolstat. We will be publishing the Phase IIb data that we recently completed in 2024 at scientific presentations. So how is it that we're so successful compared to the standard of care in terms of 100% sustained cures? Well, the answer is in the fact that ibezapolstat is a narrow-spectrum antibiotic. It only has activity against C. difficile infection. And it doesn't have any activity against the other 4 classes -- 5 classes of bacteria that constitute the healthy microbiome. Whereas if you look at the vancomycin column here, vancomycin is a broad-spectrum antibiotic. It was first used and approved to treat patients with C-difficile infection way back in 1986, not because it was the most pristine, perfect drug for C. diff, because there was nothing available. Fidaxomicin, of course, was not approved at that time, was not even developed. So C. diff was the only show in town with a drug at the time called metronidazole. But you can see from the vancomycin data here that vancomycin is destroying your healthy microbiome, while it's curing the direct infection. And that dysbiosis is what causes the high reinfection rates, that's what makes it a vulnerable frontline therapy. That's why Pfizer and Sanofi both try to get vaccines approved to treat C. diff to take that frontline banner and were unsuccessful. So having cleared Phase II, we're going to the FDA to design our Phase III trial. That will occur in the second quarter of 2024. We're expecting 2 Phase III trials. We're going to run them sequentially and use the data off of the first smaller Phase II as we hope to design it. And with the price increase that we expect off of that, we would raise money to do the second Phase III, not having to worry about patents because we have 10 years of regulatory exclusivity from the date of the FDA approval. So why would we be successful in Phase III? Well, here's a list of reasons. And they are the same reasons as why we were already successful in Phase IIa and Phase IIb, which is because we're getting 100x the concentration of ibezapolstat to the infection site, the colon that is needed to kill the bacteria. We're also -- we also have a very favorable bile acid ratio. And we expect to continue this in Phase III as we move forward. And we think this is why there are no reinfections that we can report to date. So our second program, ACX-375C, we won't highlight much of this other than to say that it's a lead optimization in preclinical. It targets a very large market, MRSA infections, which continue to be a majority of the hospital-based infections in the U.S.A. today. MRSA is still an area of unmet medical need. The patents do go out to December 2039, and we do expect new molecular entity status as well as QIDP for this program. So this, too, it's a couple of years behind ibezapolstat, but this could even be a larger drug than ibezapolstat when it gets into the clinic. So we're managed by myself and Bob DeLuccia and Rob Shawah. Bob DeLuccia, our Executive Chairman, started his career at Pfizer, took a few antibiotics out during that time, rose to the level of President of Sanofi U.S. Rob Shawah has been in many public companies as a CFO and Chief Accounting Officer. And I started my career in Ernst & Young and Paul Hastings, New York before going in as the first employee of a company called Bioenvision, which we sold to Genzyme back in 2007, and that really gave me the itch to leave the world a better place than where I found it by continuing to try to develop life-saving drugs. Our management team includes a great list of R&D folks with vast experience and success. They include [ Larry, Morton ] and Xiang Yu, who are senior executives at Cubist Pharma, and took out Cubicin for Cubist Pharma, which has a very similar MIC profile as our ACX-375, and they are now running that program. The Board of Directors is spearheaded by Joe Scodari, the former Head of the Pharmaceutical Division, Chairman of the Pharmaceutical division of Johnson & Johnson; and Jack Dean, the former Head of Preclinical Development at Sanofi. Our scientific and corporate advisers include Fred Hassan, who helps us with business development activities as well as the 3 authors of the IDSA treatment guidelines for the treatment of C. difficile infection. Our upcoming milestones are many and close in. As you can see in January, we will come out with all of the remaining data from our Phase IIb clinical trial. That includes extended cure data, microbiome head-to-head data and microbiology head-to-head data. In the second quarter, we have the FDA meeting, and we will begin to prepare for a third quarter European Medicines Agency meeting. In the third quarter, we'll also continue by finalizing the CMC required to enroll patients in Phase III, and we'll embark on our made in the USA manufacturing practice. So that's great. What's our exit strategy? Well, with Phase IIb behind us and being Phase III ready, we will move to Plan A, which is M&A for us. And on a parallel track, we will prepare to enroll patients in Phase III. So there's been a lot of recent M&A transactions. I would just highlight Tillotts Pharmaceuticals bought from Astellas, the European rights to fidaxomicin, which is owned by Merck in the U.S. for $125 million in November 2020. That's just for European rights and CIS states, so that should give you a direct comparison for what our rights are worth in the U.S., Europe and abroad. So our pathway to commercialization, take what we have, add international Phase III trials and then seek regulatory approvals in the U.S., Europe, the U.K., Canada and Japan. So we plan to leverage success. And that's Acurx Pharmaceuticals. I'd love to take questions.

Thank you, Dave. Maybe one question on my end. Could you just talk about the cash runway Acurx has at the moment?

Certainly. So our runway should take us through the first of the 2 Phase III trials. We reported $9.2 million at the end of the third quarter. And we have -- we put up a $17 million ATM that we're now drawing down. We have a very high average daily trading volume, so it's been very easy for us to do that in a very measured way. Our plan is to have the entire ATM drawn down before we enroll the first patient. And on the current pace, we can do that with 3 months to spare.

Maybe one more from my end. You mentioned plans for ibezapolstat in ex U.S. Could you maybe update us on the current filings and what your plans are going forward?

Sure. So we don't have filings yet in Europe. So our plan is to continue to work with the FDA, have a post Phase II meeting. And when we have full transparency with the FDA, full agreement, we'll then be able to take all of our data, including the structure and design of the international Phase III trials to the European Medicines Agency, so they'll have the advantage of seeing what the FDA has agreed to. And then we would anticipate developing a more fulsome plan in Europe like we have in the U.S. The nice thing is, in Europe, you also have 8 years of market exclusivity in our case that we would have to work with the FDA to insure. But again, we're not -- we would not be relying upon patent life to exploit the commercial value.

And then maybe one more question. You mentioned that Sanofi and Pfizer were unsuccessful in C. diff. Can you talk about how you plan to differentiate yourselves from these 2 previous trials? And why do you think those were unsuccessful?

Well, Sanofi and Pfizer had vaccines. So I don't have experience working with vaccines. I understand they involve very much higher number of patients and sometimes they're used prophylactically and Summit Therapeutics, which had ridinilazole, another antibiotic like ours, they had a superiority study. And our Phase III will be a non-inferiority study, like the Phase II trial design. So we think they set their bar too high on themselves. They may have had regulatory reasons in Europe to do that. But we would just do a non-inferiority study. And if the data is like the Phase II data was, we would have a win, and we would be going to the FDA for approval.

Well, if there are no further questions, I'd like to thank Dave for giving a great presentation today, and thank you all for making this week such a special week. Thank you.

Thank you, Colin.