CEO, RedHill Biopharma
RedHill Biopharma Ltd. @ Sachs_ELSF
RedHill Biopharma Ltd. presentation by Guy Goldberg, Chief Business Officer at the 18th Annual European Life Sciences CEO Forum #Sachs_ELSF.
About Guy Goldberg
Guy Goldberg, Chief Business Officer of RedHill Biopharma, has presented the company's pipeline and commercial operations at several investor conferences between 2020 and 2025. At the 18th Annual European Life Sciences CEO Forum in May 2025, Goldberg discussed the company's development of opaganib for acute radiation syndrome (ARS) in collaboration with BARDA, noting that the study is underway and has generated positive data. He also highlighted the company's commercial product Talicia for H. pylori infection, stating that updated guidelines could drive revenue growth in coming quarters, and mentioned plans for an additional study of RHB-104 for Crohn's disease using endoscopic data. In earlier appearances, Goldberg described RedHill as a fully integrated specialty biopharmaceutical company with three FDA-approved commercial products and a late-stage pipeline. He stated that the company's annualized revenues exceeded $80 million as of Q1 2021 and that it had a cash position of over $90 million at that time. Goldberg characterized opaganib as a host-directed SK2 inhibitor with a dual mechanism of action being investigated for COVID-19, ARS, and oncology, and noted that the company was pursuing regulatory pathways including potential emergency use authorization applications. He also emphasized the public health importance of H. pylori eradication, citing its classification by the WHO as a carcinogen.
Transcript (24 segments)
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Guy Goldberg0:12
Thank you, thanks everybody for attending today. Thanks for coming to my presentation. I will be making forward-looking statements. Please see our filings for a full disclosure of the risks. RedHill is a fully integrated specialty pharmaceutical company listed on NASDAQ, ticker RDHL. We're fully integrated in that we commercialize Talicia, which you see at the bottom here, and I'll be speaking about our product for H. pylori infection. We also have a full range of development products in late stage of development, five clinical assets. This is our full pipeline, and in interest of time, I won't be able to speak about all of them, but I will speak mostly about opaganib at the top and RHB-104 at the bottom for Crohn's. I'll walk through them now quickly just so you get a sense for what we're working on and where everything is in development.
So opaganib is in development for GI, for radioprotection. This is a program we have underway right now with the US government in collaboration with BARDA. The study is underway. We've already generated positive data and we're on our way forward there in in vivo models. It's also being developed in ARDS, including COVID-19, and I'll speak about that program. Also in oncology, we just very recently announced that we initiated a phase two study with opaganib in combination with Bayer's darolutamide that we're very excited about in advanced prostate cancer in Australia. I'll talk about that study design and how that will look going forward. And then finally, in Ebola virus. So opaganib has broad-acting antiviral effects. We've studied it in several antiviral indications including Ebola. This is actually funded through a contract with the US government, and I'll speak about that contract.
In addition, we have RHB-107 for opatamib, which is being developed in an ongoing phase two study right now in COVID-19 in an outpatient setting, and also in Ebola in collaboration with the US Army. RHB-102 is our long-acting granisetron that we're developing in oncology support, in gastritis where we've completed a phase three study, and in IBS-D where we've successfully completed a phase two study. RHB-204 is in development for NTM disease, and RHB-104 is our Crohn's disease program where we have positive phase three results and are looking to move that forward. So there's a lot going on. A company of our size obviously can't move everything along at the same time and do expensive clinical studies. So what we've done is almost exclusively fund our R&D through government collaborations, collaborations with Bayer, with industry, and that's how we've been able to move things forward despite budgetary restrictions.
So Talicia, this is our commercial product. This was developed in-house. We did two successful phase three studies, got it approved in the US. We licensed it also in the UAE where it was approved and launched. And I'll speak as well, very importantly, we had the guidelines updated by ACG so that now it's listed as a first-line option. That was very recently, and we hope that that will be an important catalyst to drive prescriptions up. Talicia is a combination of omeprazole, amoxicillin, and rifabutin, and it's promoted by our commercial team. So we have a real commercial team. It's not a contract organization. All the key executives are in-house and come from seasoned roles in other pharmaceutical organizations. So we think it's a very quality organization. We've promoted Talicia to thousands of GI specialists, PCPs, HCPs. We're at the conferences, and we have an effective commercial organization.
So as I mentioned, it's a combination of a PPI and two antibiotics, and that really is the special sauce here. The standard of care is clarithromycin-based treatment, and clarithromycin-based treatment faces increasing levels of resistance. So what we've done is take out the clarithromycin, put in rifabutin, which in a lab setting shows zero resistance, 0% resistance. And indeed, we've seen that in a clinical setting where we showed over 90% eradication rates in a confirmed adherent population, and it could be on the higher end of that as well because of the way that we define that population. The eradication rate remains high regardless of BMI or diabetes status, and it has a very favorable tolerability and safety profile.
Importantly, it's also an all-in-one formulation, which is a benefit over the standard of care which comes as separate pills that each one has its own dosing regimen. Here, in our case, it's an all-in-one formulation which provides enormous ease of adherence, which is very well liked by physicians' offices and also by patients. We have a lot of runway still with the IP going out to 2042, and it's a large market with over two million US patients treated annually. Probably a third of the US population is infected with H. pylori.
So importantly, in the United States, when you're talking about commercial, you can't really talk about success without talking about managed care coverage. Here, we've put a lot of effort. We have over 100 million lives covered that cover Talicia, and that includes seven out of 10 on the commercial side, six out of 10 on the government side. And that's supported by additional programs that we run, such as our warranty program, which basically says if you take Talicia and it does not eradicate H. pylori, we'll give you a full refund. Unfortunately, that has been utilized very, very rarely because we really think it's an effective drug that works when given and used correctly.
A couple words on H. pylori. So this is a chronic infection that is in the gastric mucosa. It's caused by this gram-negative bacteria. It causes peptic ulcers. It used to be thought that was caused by stress until it was proven in fact that it's caused by bacteria, which led to a Nobel Prize. And it's also, importantly, the strongest risk factor for gastric cancer. So gastric cancer is the fourth most common cause of cancer-related deaths worldwide, and it's been shown that if you can eradicate H. pylori, you can reduce gastric cancer death by 75%. And that is very important because it really is a public health concern.
You see here at the bottom, the FDA has recognized H. pylori pathogen as a potential serious threat to public health, which is also how we were able to get certain regulatory exclusivities. And both the WHO and FDA have identified clarithromycin-resistant H. pylori specifically as a priority for new development. As I mentioned, very broad infection rate in the US. You see it particularly high in the West Coast and East Coast, and also it's very high in Europe. In Europe, it tends to be higher in southern Europe and a little bit lower infection rates the more north you go. The resistance rates have been seen to 25-40%, but up to 50%.
And that's why the ACG guidelines were just updated a few months ago, saying that clarithromycin should not be a component of any therapy without prior resistance testing to confirm susceptibility. And that's very important because in practice, physicians really don't test susceptibility. A patient comes in, they take a breath test, they test positive, and a physician just wants to give a course of therapy and be done with it. And these ACG guidelines essentially promote Talicia to be the most attractive product available because we don't require susceptibility testing, and we really are the best alternative for a physician who's looking at the science and trying to find the best solution to a patient who's tested positive for H. pylori.
So to summarize, this market is large, two million treatments per year. There's diminished efficacy of the standard of care. The current brand medications lack any differentiation because they're all either clarithromycin or metronidazole-based. And we've brought in a new treatment into the market which we think will be a first-line, has a very attractive profile, and especially with the recent guidelines, we hope that will be a catalyst to future prescription and revenue growth.
I'll now talk about our R&D pipeline, the second half of my comments. This is a very busy slide. I don't want you to ruin your eyes on this, so I'll just mention that I'll be talking about opaganib and RHB-104 as our two key components, but we have other products in our pipeline that we're excited about and that we're actively developing. So opaganib, this is a first-in-class, broad-acting, host-directed or administered S1P inhibitor. We're developing it in a broad number of indications because of its unique mechanism of action. That includes viral, oncology, nuclear radioprotection, and other inflammatory diseases. Now, this is another busy slide that I don't want you to ruin your eyes on, so I will skip it. We'll just go and I'll fill in all that information in the upcoming slides.
So a little bit on the science. S1P is a target for sphingolipids, which have pathological roles in various diseases. Sphingolipids regulate several important signal and transduction processes and also intracellular targets, which is very important. So GSRD is a disease for which there's no approved treatment. It's a high priority for the US government and for governments in Europe given the political situations and the nuclear accidents that we've seen. It's basically an acute illness caused by the irradiation of the body at high penetrating radiation in a short period of time. So there's two major illnesses that come as a result of GSRD. One is hematological, the other is GI. And as I mentioned, there's no approved GI treatment. That's why our drug has received significant interest from the US government.
We've done eight US government-funded in vivo studies, both in radiation protection, also as it relates to cancer treatment and radiotherapy. And the results have been pretty consistent and very good, showing protection of normal tissue, and also in the case of oncology, improvement of anti-tumor activity, enhancement of tolerability and survival. So good basis of support there. What we've done is we've been accepted, we've applied and we're accepted into the NIAID's radiation nuclear countermeasure program. As I mentioned, this has become a top priority for the US government. The whole idea, the whole field of countermeasures, they've purchased hundreds of millions of dollars worth of drug and stockpiling contracts, and also put a lot of funding available for development, which we hope to benefit from in the future as well. We've also received an SBIR grant of $1.7 million from the US government through our partner Apache.
So this is just a summary, I think, of what I've said. We've shown in vivo that opaganib protects against anti-tumor activities, protects against radiation toxicity, and importantly, in the previous work that we've done, there's a pretty good safety database of over 470 patients. The reason I mention that is we hope that we'll be able to go through the animal rule pathway on the regulatory front here, and that means no large clinical studies for efficacy being required, which means a more efficient and quicker path to submission.
This is our COVID program. This area has fallen out of favor somewhat in the pharmaceutical industry. We think unwisely so. The reason is that Pfizer's Paxlovid still sells hundreds of millions of dollars per year. It's an imperfect drug. There's a strong need, especially for at-risk patients, and we think we may have one here. We've shown in a clinical study improved viral RNA clearance in patients who were hospitalized with severe COVID-19 pneumonia. That includes a 70% mortality benefit when given on top of standard of care, and also a 62% mortality reduction in the subgroup of moderately severe hospitalized patients in analysis that we did that was post-hoc and it was published in Microorganisms.
We also tested it in a number of different variants of COVID-19, and we've shown pretty consistently inhibition of the virus. This is on top of a phase two study that we did in hospitalized COVID patients, and as I mentioned, a very large safety database. As I mentioned also, it's a broad-acting antiviral because it's host-directed, which means we work in not just one pathogen but many. We've been doing testing with NIH on influenza, Chikungunya virus, RSV, and Ebola. This is one example of an in vitro study that was done in flu where we showed pretty potent in vitro inhibition, sorry, in vitro, not in vivo, in vitro inhibition with no evidence of toxicity.
We're also developing this as an anti-cancer agent. Running a little bit short on time, so I'll skip the mechanism of action. I'll just mention that we just announced that we started a phase two study in combination with Bayer's darolutamide. This is being done with Professor Lisa Horvath in Australia. It's an 80-patient study, randomized one-to-one, either with darolutamide placebo or darolutamide plus opaganib. The primary endpoint is 12-month progression-free survival. We've started the study, and we'll give updates as soon as we have a good clip of enrollment and we can give better guidance on when we may have some results.
Last five minutes, I'll talk about RHB-104. This is a drug that we've been developing for many years. It's very revolutionary in the field of Crohn's disease, which is primarily treated through immune suppression. What we do is rely on a completely different approach to Crohn's disease, which is looking at the underlying role that mycobacteria play in causing Crohn's disease. So this is based on a couple pieces of very good evidence. One is there's a causative agent in Johne's disease, which is a parallel disease in cattle, which acts and looks like Crohn's disease. You can see pictures here on the right. And it's known that if you have a healthy cow and you infect him with MAP, then he will develop symptoms that look like Crohn's. And then if you treat the cow with antibiotics that are effective against Crohn's, you can reduce and eliminate those symptoms. So there's a good animal model that shows that MAP is a harmful bacteria.
In addition, there's been many, many studies, these are just two examples but many others, that have shown a very high consistency of MAP infection in Crohn's patients. It's been shown consistently over many different investigators geographically, pretty consistently. And you don't see it in IBD, so you don't see it in UC, but you do see it in Crohn's. So it's a very interesting marker for Crohn's disease that's been shown consistently in the research. So what we've done is put together a triple combination of three antibiotics, all of them known to be prone intracellular because MAP exists inside of the cell, and all of them antimycobacterial with different mechanisms of action.
We tested this in a phase three study, in a global study. It was in over 100 sites. I don't have the details in this presentation. I can say it was 331 patients. We looked at the primary endpoint being a remission at 26 weeks, which was hit with statistical significance. We also looked at early induction of remission at 16 weeks, which hit statistical significance. We looked at response rate at 26 weeks, hit statistical significance as well, as duration and maintenance at 52 weeks, both of them hit statistical significance. So all the key endpoints and secondary endpoints that we were looking at all hit with dramatic fashion.
In addition, we looked for the benefit of RHB-104 on top of immunomodulators and corticosteroids, which are the two primary treatments given. And in a subanalysis that we did, we found that in both cases we hit statistical significance, which was exciting to us. And we also looked at endoscopy. So endoscopy has become kind of the favored way of evaluating Crohn's disease, not the CDAI score which was historically used. So here was voluntary, so I think we had 35 patients volunteered to do endoscopies, and the results were also very dramatic, including hitting statistical significance, although we weren't looking or powering for that endo as a prior to the study. So very positive results.
What we're looking now to do is an additional study in the future, which we'll be using, this was an all-comer study, so we'll be using MAP as a gating criteria and possibly looking at endoscopy as a primary or co-primary endpoint. So we're looking at doing something like that. That's in the works, and we hope to have an update on that in the future.
So in my last minute, I'll just provide a brief summary. So we are a fully integrated company. We have a lot going on for a company of our size. We find creative ways to get resources to move our products forward. We have a commercial operation which is break-even, and with a recent interesting catalyst in the updated guidelines, we hope to drive revenue up in the coming quarters. And then we're always looking for investments to help move forward some of these indications that we don't have US government support or partner support from Big Pharma to move them forward. Thank you very much for listening, and I think we're right on time. Thank you.