RedHill Biopharma | Benzinga Healthcare Small Cap Conference

I will be making forward-looking statements. Please see our filings for a full disclosure of the risks. So, RedHill, for those of you not familiar with us, we're a publicly listed U.S. specialty biopharmaceutical company. Our ticker is RDHL. Our primary focus is commercialization and development of drugs for gastrointestinal disease and infectious disease. I will be talking about different aspects of our company today, with a major focus on our R&D, specifically opaganib. This is the product we announced top-line results for in our global Phase 2/3 study. And as we have announced, while we were disappointed not to meet our primary endpoint, opaganib did show a consistent benefit in the per-protocol population versus placebo, and we're currently analyzing the study results, especially with an eye to earlier-stage patients. And there will be an upcoming webcast with new data from that study soon. Our second program that we'll be talking about is RHB-107, upamostat. That's our other COVID program. We have an ongoing Phase 2/3 study with this drug in an outpatient setting, which is the largest subgroup in COVID. This is a novel, once-daily oral pill, and we have a lot to talk about with this one as well. And then finally, we'll be talking about these three drugs you see at the bottom: Movantik, Talicia, and Aemcolo, which comprise our three FDA-approved products we promote in the United States out of our headquarters in Raleigh, North Carolina, with 100 sales reps. And we recently announced our financial results for the second quarter, and I'll discuss some of those highlights also.

This is our full pipeline. As I mentioned, our three commercial products at the top: Talicia for H. pylori infection, Movantik for opioid-induced constipation, and then Aemcolo for traveler's diarrhea. And then at the bottom part of the slide, our full R&D pipeline. The COVID programs that I mentioned in the beginning at the bottom here are opaganib and RHB-107. I'll also talk about RHB-204 for NTM disease. This is a Phase 3 study that we have ongoing. Very exciting indication as there is nothing approved first-line by FDA to treat NTM disease.

This is a snapshot of the company, of where we want to grow to and what we want to become. Right now, we promote these three products. We have a full pipeline of products that are tomorrow's commercial products, we hope, if we get approved. And with the commercial platform that we've built, with 100 sales reps, we hope to grow and to become a leading U.S. specialty pharmaceutical company.

Our financial highlights as of the second quarter: we have $71 million in cash, and the market cap, which of course changes on a regular basis, but that is as of the last time we updated the presentation.

So, turning to our commercial operations, we have a strong U.S. presence. Our commercial headquarters and our U.S.-wide operations have in-house all the key skill sets you want to see in commercial operations, not just on the sales side of things, but also marketing, managed markets, market access, which are very, very important, as well as supply chain, medical affairs, and compliance. The team is very seasoned. A lot of executives out of Salix, which was the number one GI company in the world before it was bought out by Valeant. And we've shown to be a very resilient organization despite the challenging conditions of the pandemic.

Here you see our quarterly net revenue numbers, and you see that they stay pretty consistent, even though nationwide pharmaceutical Rx numbers are down significantly and have been since the beginning of the pandemic. So we've been able to show that we're resilient, and we hope that with the pandemic achieving new normalcy all the time, that number will continue to grow. And we hope that I'll be able to report a very good second half of the year through Q3 and Q4.

Now I'll talk about the commercial products specifically. Movantik first. This is a drug for the treatment of opioid-induced constipation in adults with chronic non-cancer pain. Oral naloxegol tablets. It was approved in the U.S. in 2014, launched by AstraZeneca in 2015. They did a great job making this the brand leader. It was the first drug of its class approved, and it stayed as the brand leader since launch. This is the market as a whole. There are about 50 million Americans with chronic pain. A significant number of them are prescribed opioids, and a significant number of those opioid users have OIC. And of those, a significant number have little to no benefit from constipation treatments. So it's a fairly large market and room to grow because of the significant unmet need that we still see with these patients. Again, constipation is the number one side effect of opioids. Movantik is part of the PAMORA class of drugs. It's a class of drugs designed specifically to address this issue, and because it's designed specifically for this indication, it's a very effective drug and very well received by doctors and physicians. In fact, there's been over two million prescriptions written for it since launch. And as I mentioned, Movantik is the number one prescribed drug in its class, in the PAMORA class, with great coverage. Nine out of 10 lives are covered, both on the commercial and the government side, which means we have a good ability for patients to access this drug.

I'll now switch gears and talk about Talicia. So, Talicia is addressing a big unmet medical need in H. pylori. And H. pylori infection is a serious infection. It's a carcinogen. It's typically an infection you get as a child, and it stays with you throughout your adult life. In many people, it can create clinical issues such as gastric cancer or peptic ulcer disease, among others. That's why when it's diagnosed by a physician, it will be treated immediately. And the way it's treated is with a triple drug combination, typically clarithromycin or metronidazole-based combination. And the problem is that over time, that approach is shown to be less and less effective as the bacteria has grown to be more and more resistant to that standard of care. So there's a big medical need here for a drug to address the growing resistance to clarithromycin. In fact, both the WHO and the FDA have identified H. pylori as a high priority for new treatments. We have developed Talicia, which is a triple drug combination taking out clarithromycin, which is the product that has the resistance issue, and putting in rifabutin. And what we saw in the clinical studies is very nice effectiveness in this patient population. We got approved by FDA, and we launched the product in March 2020. Right now, promoting to about 25,000 healthcare professionals, and you can see here some of the marketing pieces that we have that have been developed by our commercial team. Our growth trajectory still since launch has been something we're proud of. We see the growth even with the challenging environment of COVID, which for a new product launch is definitely a challenge. Yet we've been able to show some very nice growth, including in Q2, which is what we recently announced in our earnings call, which we showed over 10% quarter-over-quarter growth. We continue to get better and better coverage, which is also very important in this drug category. Right now, 8 out of 10 commercial lives, 6 out of 10 government lives. We continue to get wins, and we continue to hope to improve that coverage over time.

To summarize Talicia, this is a product that we've launched for H. pylori. It's a relatively large indication with an unmet medical need. We've developed a drug with great efficacy, addressing the concerns of resistance, which is a top priority from a public health perspective and something that's very concerning to physicians as well. And we're aiming for Talicia to become the new gold standard, the new preferred first-line treatment for H. pylori infection.

Our third product is Aemcolo. This is a product for the treatment of traveler's diarrhea by non-invasive strains of E. coli, a serious problem for travelers, especially from the United States, who are going to high-risk countries, typically developing countries in the southern hemisphere. This is a very large number of people, at least pre-COVID. Now, of course, travel has stopped, so this market has dried up somewhat. But we are ready with this product. We continue to do our marketing efforts and other efforts on the commercial fronts, and we anticipate this will be a significant product when travel returns.

I'll now switch gears and talk about opaganib, our SK2 inhibitor for COVID-19, among other indications. So, to give a little bit of background on this product, we had generated very nice data in vitro to show that opaganib completely inhibits viral replication in coronavirus among the various strains that you hear about, from the Beta strain or the South African strain, the Gamma strain, the Brazilian strain, and very importantly, the Delta strain or the Indian strain. And because of that consistent results that we've seen in viral replication, as well as the anti-inflammatory effect and the antiviral effect we've seen in other virus strains, we decided to move forward in a full development program for this drug, which consisted of a Phase 2 study, which you see on the right here, which we've announced positive data, and the recently announced global Phase 2/3 study and the data that we reported recently. And I'll talk through that data in more detail. First, the Phase 2 study. So this was a small, non-powered study of 40 patients done in the United States. We found opaganib to be safe and a consistent trend of greater improvement across multiple key primary and secondary endpoints, very importantly including patients being discharged from hospital by day 14, which is an important endpoint, and patients getting off oxygen. So we were very happy with the study. And then we did this global Phase 2/3 study also in patients with hospitalized severe COVID pneumonia. This was a very large study, global study, 475 patients. And what we saw here is, first and disappointingly, the study did not meet its primary endpoint, and we were frustrated from that, of course. But we did see in the analysis, at least this very initial analysis that we've done, is a potential effect in the subset of patients requiring less oxygen. So these are patients who are earlier in the disease, where antiviral activity is more important. And we believe the antiviral effect of opaganib is very important to its mechanism of action. So what we're doing now is we're analyzing the data, and we will have a webcast soon with additional information from the study, and we hope that that will provide guidance regarding future development work that we're doing with opaganib in COVID.

Our second product is upamostat, or RHB-107, which we're also developing in a number of indications, including COVID. So this is a serine protease inhibitor that has also been through a lot of pre-clinical work, similar to opaganib. And where we developed this drug is in the non-hospitalized patient population with symptomatic COVID. So this is a less severe population, and we have an ongoing U.S. Phase 2/3 study going right now. And we've also had the study approved in South Africa and additional sites in the United States in order to accelerate recruitment. This is a little bit about our study design: 310 patients to be enrolled in a two-part, multi-center, randomized, double-blind, placebo-controlled study. We've brought in a lot of innovative methods in order to make it work from home, given COVID and all the sensitivity around doing a COVID study. And very importantly, the primary endpoint is time to sustained recovery, which is a very good, well-established endpoint. We're testing patients for various viral strains, and as I mentioned, we're taking a lot of steps to accelerate recruitment of the study. As a reminder about RHB-107, it's a once-daily, orally administered treatment, and it's given earlier in the course of disease. And we think that because it targets the host cell, it could be effective against the various variants that we hear and are very concerned about. So the profile of this drug is very attractive, and if it's successful in the study, will be a very important tool in the battle against COVID.

In the last few minutes that I have, I'll talk about RHB-204 for NTM disease. A little bit of background in NTM disease for the listeners who may not be familiar: this is a very difficult-to-treat infection. There's no FDA-approved first-line standard of care. These bacteria are ubiquitous. It's a pulmonary disease, and when you get it, it pretty much stays with you your entire life and has to be treated with long-term dosing with three or more antibiotics. Nothing approved first-line, so there's a very big unmet medical need here. And even though it's an orphan disease, there's a significant number of patients, about 110,000 patients, probably more, since these numbers are from 2017. So what we've done is we've developed RHB-204, which is an all-in-one combination of three antibiotic drugs, each known to be active against NTM from the literature that's been seen with them and this disease. And what we're doing is we're developing this drug as a first-line treatment. Again, there's nothing approved first-line. And this study that you see here is a 125-subject study, multi-center, randomized, double-blind, placebo-controlled study, and 40 clinical sites in the United States. And this is a two-part study. So this is to keep in mind with some of the feedback we got from FDA about how they would like to see this clinical study designed. Very importantly, primary endpoints in line with recent FDA feedback is first sputum culture conversion, which is a more traditional endpoint in NTM disease, month six of treatment, and a quality of life questionnaire, which we're also doing from baseline to month six. So a very exciting study for us. This study is ongoing and recruiting, and we're also looking for ways to improve recruitment with this study as well. Certainly, pulmonary clinical studies have also been affected by the pandemic and the concern about doing studies in this climate, but we are moving forward with this, and we're taking all the precautions so we can do the study safely and effectively.

So with that, I'll just summarize in a couple minutes who we are, what we have, and then I'll turn it back over to Spencer. So, RedHill is a NASDAQ-listed company, U.S.-focused in GI and infectious disease. We recently announced top-line data in opaganib, which showed some promising signals even though we did not meet our primary endpoint, and we'll be having an upcoming webcast with some of the data from that study soon. We have a second COVID-19 program, RHB-107 or upamostat, that's in an ongoing Phase 2/3 study in an outpatient setting. We think this drug has a lot of promise. So there are a lot of big pharma in this race. We think that we are the David versus Goliath here, and we have a lot of promise with our drug and a very attractive profile in being a novel, once-daily oral pill. In addition to our other R&D products, we also have a commercial operation promoting three FDA-approved products: Movantik, which we acquired from AstraZeneca and have now made this a RedHill product, and this is for opioid-induced constipation; Talicia for H. pylori infection, a very important indication we developed this product in-house and are now launching it with our proprietary commercial team; and then Aemcolo, which we acquired from Cosmo along with a strategic investment, and this will be an important product once international travel comes back. Our commercial operation is led by a very experienced Salix team. We've had record quarterly revenues in Q2, showing increases in scripts both for Talicia and Movantik, and we're very excited about what the rest of the year holds, both for our commercial operation and on R&D. So with that, I'd like to thank Benzinga for inviting us to participate, and I'll turn it over to Spencer for concluding remarks. Thank you.