OPKO Health, Inc. (OPK) Earnings Call Transcript

Good afternoon, ladies and gentlemen. My name is James Curtis, and I'm delighted to be introducing Dr. Elias Zerhouni, the President and Vice Chairman of the Board for OPKO Health. Over to you.

Thank you. Well, thank you. Good afternoon, everyone. I'm really not going to read these forward-looking statements. I think you've seen that across the meeting. What I want to talk to you about is really the year that we've had at OPKO Health, which, to me, has been another transformative year, the year before we presented and we told you what we were going to do. This year, we're really accomplishing a tremendous amount of advances in the various portions of the company. So what I'm going to do is, first, I'm going to talk to you about the therapeutics part of the house, which is ModeX Therapeutics which is a biotech company based on a very unique platform that has advanced its pipeline. There are now 2 products in the clinic and 2 more coming this year: the novel long-acting biologics development that we've had with obviously developing the long-acting growth hormone product; but this year also developing a GLP-1/glucagon, oxyntomodulin analog that is going into the late preclinical phase. And then obviously, talked about the in-line pharmaceutical business, which is continuing to grow and being very promising, I will talk to you about the diagnostics side, the clinical diagnostics side of the house with the BioReference lab and what we've done there to restore profitability to that large component of our company. We'll show you how the financial strength of the company has been greatly enhanced in 2024 and give you, essentially, a perspective about 2025 and how we're going to think through going forward. So ModeX Therapeutics is a company that was acquired by OPKO Health in May 2022. It was created in 2020, and it was a spin-off from the breakthrough lab of Sanofi that was co-created by Gary Nabel, who's here, and myself. And we really developed the company to have 2 platforms: one, based on the idea that monoclonal antibodies are really 1 target, 1 antibody. But really, when you treat diseases, you treat diseases with combination of targets. And so we found a way to build antibodies that are multi-specific, up to 4 targets can be addressed at once and multifunctional. And so when you look at ModeX, there is a multi-specific antibodies target -- I mean, technology, which is designed to bind to 2 cell antigens and 2 tumor antigens in cancer, but we can also bind to 4 different sites in any virus that we would want to attack. And then there is a second platform that is developed on the basis of nanoparticles of ferritins that can carry multiple antigens. And this is how we developed a vaccine protocol -- vaccine, a model for Epstein-Barr virus. And those 2 platforms have attracted a lot of interest. And when you look at the applications of these platforms, it's immuno-oncology, immunology and viral diseases. And so when you look at the portfolio that ModeX has entered into, you see an enormous attractiveness for partners, actually, to partner with us. And so for example, if you look at the red, you can see that we've already completed a first Phase I with NIH. We have had the luck of having Merck be interested in our Epstein-Barr virus vaccine and working with us. We had, essentially, a collaboration where we took over the completeness -- the completion of the IND qualifying studies. And last month, Merck announced that they really entered into Phase I, and we're hoping to get results of that Phase I in the course of 2025. At the same time, BARDA, which is the federal agency that looks at infectious disease risk, funded us for a total of about $205 million to advance our platform in antiviral diseases starting with COVID. Now people would say, well, why COVID? I mean, COVID is over. It's not over. Today, if you look at the epidemiology of COVID, there are 3x more COVID cases than flu cases. And if you look at the deadly risk of the disease, it's still several hundred patients a week who pass away in the U.S. from COVID. More importantly is the immune-deficient population that really needs protection through multi-specific antibodies. And so that's why we received, first, a $59 million grant and then extended this year by $51 million supplemental to accelerate the development of COVID antibodies, but also flu antibodies, because there is a fear of a pandemic flu, again, with BARDA support. And we have an HIV multispecific, which is continuing its development with support from NIH in particular. Now having said that, the part of the portfolio that applies in my view, to the oncology therapeutic area is also advancing because we've been able to manufacture quadri-specific antibodies that can attack solid tumors with the c-MET, TROP2 targeting where many of the solid tumors have c-MET and TROP2 and then the CD3/CD28, which we have tested in the clinic when we were at Sanofi in a different format, different strengths, but it's entered the clinic and so far, we are halfway through the escalation, and we're really happy to say that it's advancing properly. We have a CD19/CD20 targeting 2 of the targets that you can think -- you can find on B-cell and B-cell lymphomas and liquid tumors with, again, a CD3/CD28 T cell engager, but the thing to really understand is that we're not just doing T cell engagement. We're engaging T cells and enhancing their activities because CD3 is an engager, but CD28 is an enhancer. And that's why there's been a tremendous amount of interest at this conference. We've had this particular molecule attract a lot of interest from multiple parties. And you've known, if you looked at the transactions that occurred in the past 3 months, there's been a lot of T cell engagement transactions, but none with CD3/CD28 dual signaling capable of being given. This molecule is also in pre-IND stage, should be in the clinic this year or the beginning of next year. And then we have an immune modulator. What we realized is that our technology can actually change the setting of the immune system, and it can reset the immune system, rejuvenate, restrengthen the immune system in patients who are deficient or in patients who are aged. As you know, aging is a state where the immune system is generally more deficient than it is in other parts of the life. And then we've also been asked to help with antibody radionuclide conjugate because we can actually define and design antibodies that will have a very specific targeting by being able to target not just 1 target, but 2 that you know are coexisting in any 1 particular tumor, so you actually avoid the off-target effects that you could have before -- because your antibody will attach to another tissue. So these are the pipeline advances that I wanted to share with you. It's transformative. We've never been a clinical company. Now we're a clinical company with 2 products in the market -- in development and 2 more coming over the next year. Now you say, okay, how much does it cost? So Merck, essentially, funded the development of the EBV vaccine for us. There was a $50 million upfront. So far, they've also funded the development that we continued all the way to Phase I and milestones is $87.5 million with $872 million that are coming for further milestones, entering into Phase II, Phase III and so on and including royalties in global sales if that happens. Now in the COVID area, we received $51 million additional funding this year for both COVID and flu, and we're eligible for up to $205 million committed funding by BARDA. And obviously, to date, we've had $110 million committed because, not only are they interested in the multi-specific antibodies, but on the platform. So they wanted to increase the yield of the platforms, and we're achieving very high titers right now with a continuous flow technology, but they also want us to use gene delivery, mRNA that would generate the antibodies in patients rather than producing the antibody to be given to patients. So active business development right now with a lot of incoming, a lot of interest in our platforms, hoping that this year we'll have, again, a year of success in creating new nondilutive partnerships. And these collaborations will accelerate further the development and the opportunity of expanding our pipeline further. So in long-acting biologics, we've been present for a long time. One of our subsidiary, which is OPKO Biologics in Israel is an expert company in lengthening the half-life of hormones. And so they achieved that with growth hormone, which became the product called NGENLA, which is now marketed by Pfizer. But in the portfolio, we've had other molecules, the GLP-2 molecules for short bowel syndrome, but also a co-agonist called GLP-1/glucagon, which is an analog of oxyntomodulin. Now we had developed this product with a PEGylated extension for -- and reached Phase II until we realized that the PEGylation led to very, very high doses. And we changed that with a fatty acid configuration, which now has a much better half-life and much better efficacy in preclinical models. We're pushing this molecule. But the nice advantage of this molecule is that you can actually deliver it in an oral form. And we are doing this with a company called Entera Bio, where they found a way of taking our molecule and formulating it in a way that can be actually absorbed orally rather than injected. We still have a once-a-week injectable form that we are working on as well for other associations that we are thinking about. So this is something that is in the preclinical stages, GMP stages of the IND process and hopefully, will be in testing towards the end of this year or beginning of next year. So product sales and the in-line products. I think in terms of global health sales from our Iberoamerica or Latin America and Spain, France division, there's been a 5% local currency growth with a 19% EBITDA margin. This business was about $20 million in 2018. It's now $120 million and growing at that rate. We have now begun to see revenues from the marketing of the long-acting growth hormone product called NGENLA, marketed by Pfizer in over 44 countries now. We got the approval last year in August for the U.S. market. Rayaldee, sustained sales for CKD therapy, pretty stable, really requires an understanding of the recent publications, which seem to indicate that the use of Rayaldee reduces parathyroid hormone, secondary hyperparathyroidism and then enhances the ability of the patient to sustain the changes in calcium and then prolong, actually, the interval between CKD 4 and dialysis. So that is a product that, obviously, we're trying to make everything possible for, changing the guidelines, which have been changed last year in terms of secondary hyperparathyroidism, in terms of being used for that patient population in CKD 4 and late 3, and we're expecting to see more adoption, obviously, in a market where there is quite a bit of optionality that is taken into account today. But none of these optionalities provide a true reduction in parathyroid hormone by increasing the level of vitamin D in the blood, which is the counter signal to PTH. EirGen is our manufacturing arm in Ireland and has continued to grow, provides now most of the products marketed by OPKO Health worldwide, but also has taken CDMO roles for other clients and has seen a 57% sales growth this year. Now, if you look at the other issue that we were facing, remember that we were very concerned about the performance of BioReference and its impact on the bottom line of the company. And so we have worked on restructuring BioReference. We reduced the head count, and we divested a part of the company that was really providing a tremendous amount of losses because it was so far away from our main lab. So we had laboratories in California, in Texas, in Florida. And when we looked at the cost of testing, we realized that it didn't cover the reimbursements that we were getting in those regions. So we decided to sell that. We had a competitive process and Labcorp bought the -- about $100 million of revenues that were concerned for $237 million in sales -- in asset sale. And so that really allowed us, in fact, to improve the performance of BioReference by closing the facilities, rightsizing the workforce from 3,300 to 2,000 and then focusing on our areas of strength. I mean our, oncology line is extremely appreciated by clients. It's very comprehensive and urology with a 4K test as well, well known nationally. And then we focused our clinical diagnostics laboratory work in New York, New Jersey because the proximity reduces your overall cost of testing. And with -- we're left with about $400 million in annual revenues for the -- for BioReference. So we've continued to restructure to achieve a stronger financial position and that strategic restructuring will continue. We'll look for opportunities. We want to maintain our best-in-class oncology and urology testing, grow the shares in attractive New York and New Jersey markets. They're still very, very active market and achieve sustainable profitability in 2025. Now all of this obviously required us to realign our capital structure to our capital allocation strategy. I mean, we have, essentially, a need to have a balance sheet reset, and we did effectively execute that this year. We talked about it at our last meeting here in J.P. Morgan. And so the year started with a convertible note conversion. We had a note that was due in 2025, about now. And so we lengthened the maturity of that note to 2029. So it was a $300 million convertible note, which gave us about $230 million of cash. Then the royalties from NGENLA attracted interest also. And we basically had a royalty monetization event with retention of about 2/3 of the value and milestone rights of $100 million for $250 million. So it was about a good way of capitalizing the company while retaining the upside of the product itself over time. And then I just told you about the sale of the clinical diagnostics and women's health assets outside New York, New Jersey to Labcorp, $231 million -- $238 million, sorry. And one of our companies that we spun out called GeneDx has really turned around in the rare genomic field -- genomic diagnostics and we've received proceeds of $189 million for that. At the same time, we try to improve shareholder value by buying back shares. And to date, we have repurchased about $90 million worth of shares and retired 81 million shares. So there's been a [ dilution ], if you will, of the float with a $30 million to repurchase convertible notes. So I would say that it has been a transformative year from the financial point of view with an enhanced financial position. We've maintained our R&D investments at a level that was equivalent to what was there before OPKO joined -- before ModeX joined, because we've been able to attract non-dilutive financing, which finances 50% of our R&D efforts at ModeX, and we've been able to return capital to shareholders. So that's why I call 2024 a successful year, a year where we really focus on the priorities of OPKO Health in terms of enhancing its financial structure, getting to profitability at the BioReference laboratory, which was a big drain on our financial structure and advancing our portfolios, both in line and up-and-coming portfolios in the clinical -- early clinical development phase. So the priorities remain the same for 2025. We need to generate long-term value. The pipeline progression at ModeX will continue for -- getting the data for our Phase I assets by midyear, 2 additional assets to enter clinical developments and continue to grow in nondilutive partnerships. Biologics, we will advance 2 molecules to IND stages and explore partnerships as well, maintain and accelerate revenue growth in our global pharmaceutical business. By reference, continue the strategic restructuring, grow the share in New York, New Jersey and achieve profitability in 2025, and then recapitalize financials achieved in 2024, really gives us a good runway and sustained funding for R&D and commercial operations. So with that, I'll be happy to take any questions.

If there are any questions from the floor, just raise your hand, and we'll get you a microphone. But just looking down here at the iPad, we've got a few. Could you tell us a bit about the news flow to be expected from ModeX now that you're entering the clinical stages?

Good question. I appreciate the question. So for me, news flow is important. As you know, in biotech today, entering a Phase I is not as important as having real data, clinical data at some point. So what is the clinical data we can expect, right? For the cancer molecule, we expect to see that it is tolerated in patients in fourth line. So we're talking about patients who've had 1, 2, 3 lines of therapies. They're not in the best of shape. If they can tolerate the escalation of dose, then we have the ability to expand that as a basket trial for many solid tumors. There are 14 solid tumors that could be addressed by this molecule. So it's a pipeline in a drug. And so what you would want to watch for is whether or not we've been able to escalate the dose to a therapeutic area without having a clinical hold or major problems. So that's one. In terms of the vaccine, it's really 200 patients that Merck is recruiting to evaluate, not only the safety of the vaccine, but also having indirect measures of efficacy. So biomarkers about how many antibodies were created by the injection and so on and so forth, what's the difference between the 2 groups. And that will then determine the quality of that data that we will have in Q2, Q3 will determine the future of that vaccine. So that's a major, major news flow. I think people don't appreciate that in vaccines, it's that news flow that really determines the value of the total program. Because if you go into Phase III, you're not going to stop. So that's the 2, the tangible one. I think the other ones is we're getting a lot of interest in our platform. Our platform has more capacity than what we're using. We have 70 employees. We're spending around $45 million a year for internal monies and then another $45 million from external sources. I don't think you want to grow without having other partners using the platform appropriately. And we've had a lot of interest. So news flow would be new partnerships, new validation if you will, of our platform. I think the other thing is BioReference, we're trying to restructure that business even faster. So there may be announces there -- announcements there. I think the other fact is we are working very hard to enhance the clinical portfolio because even though entry in Phase I may not be a big inflection point, while you didn't have it, you wouldn't have an inflection point, so you better have it. And so we want to enter these new molecules. I think the one that's supported by BARDA is very promising. We have a tremendous amount of enthusiasm from the government for us to advance that. But it's not just a government product. People don't appreciate that if we do that, we'll be able to access the public -- I mean, the public market, not just the government market, the regular market for the 30 million patients who have a diminished immune system. And that would be a major change because the revenue potential is real and -- for that and repeatable because it's become an endemic disease. It's no longer a pandemic disease. So those are some of the news I'm expecting, and crossing fingers, we'll get them.

Yes, we look forward to any updates. The second question here is with regards to BioReference. What's your forward strategy to achieve profitability there?