Good morning and welcome to the first company presentation, ANI Pharmaceuticals, at RBC Capital Markets' initial virtual ophthalmology conference. My name is Doug Miehm, and I may need to correct my thoughts on whether today is actually going to be a good day. I expect the conference to have a good day, but the broader market is obviously another question. Today, it's our pleasure to have Nikhil Lalwani, President and CEO of ANI. For those not as familiar with ANI, I would describe it as a hybrid pharmaceutical company with a high-quality focus on branded, rare disease, and generic products. The company operates in several verticals, but I believe Nikhil is in the best position to provide an overview of his company. With that, Nikhil, why don't I turn it over to you, and you can talk about the business more broadly before we start talking about the ophthalmology aspects of ANI. Over to you.
Thank you, Doug, and good morning, everybody, and thank you for joining us. We really appreciate it. As Doug said, this is an important day, so thank you for taking time to be with us. ANI Pharmaceuticals operates in three segments: driving robust, profitable growth, rare disease, generics, and brands. In aggregate, the total company guidance—just to tell you a little bit about the company in numbers first—the total company guidance for 2025 revenues was $756 million–$776 million. Adjusted non-GAAP EBITDA guidance for 2025 was $190 million–$200 million, and the non-GAAP EPS guidance was $6.12–$6.49. ANI's rare disease business is our key area of focus and the primary driver of growth going forward. Over the past four years, we've built our rare disease business, beginning with the launch of our lead asset, Purified Cortrophin Gel, in January of 2022.
In 2024, we expanded the scope and scale of our rare disease business with the acquisition of Alimera and marking our foray, or I guess deeper foray, into ophthalmology. Rare disease will account for 48%–49% of our revenues in 2025 at $362–$377 million. As I said before, we have two franchises: Cortrophin Gel, which grew north of 75% to almost $200 million in just the third year since launch—this is the 2024 number—and our guidance for 2025 for Cortrophin is $265–$274 million, growing approximately 35%. Our ophthalmology franchises, ILUVIEN and YUTIQ, came to us through the Alimera acquisition that closed in late Q3 of 2024, and our guidance for 2025 is $97–$103 million. Our generics business is fueled by a strong and high-performing R&D engine, operational excellence, and a U.S.-based manufacturing footprint.
Generics accounted for $301 million in revenues in 2024, and we expect our generics business to be a low double-digit grower in 2025. Finally, our brands business is smaller but has very healthy margins and cash flow that we can use to help fund our future growth. While rare disease remains the key area of focus, you know, and within that, ophthalmology is a critical therapeutic area, we're also proud of our track record of success and performance in our two other segments, which we expect to continue generating strong financial returns for our company and creating a virtuous cycle of growth.
Excellent. Great overview. You know, I was contemplating including this question in today's overview, but I think it's very appropriate that perhaps we touch on how tariffs could affect your company and perhaps the broader pharmaceutical market to start with, and then we can get into the other aspects of the business.
Sure. Thank you, Doug. Yes, it is clearly a very topical question and fair for you to bring it up first thing in the morning. Look, you know, as I was saying in my overview, all our generics business, our rare disease business, and our brands business has products sourced from the U.S. You know, in fact, for our rare disease and brands business, it's entirely from the U.S., from U.S.-based manufacturing. For our generics business, you know, I would say more than three-quarters of our products are sold, are made at manufacturing plants in the U.S., which are owned by us, two in Baudette, Minnesota, and one in East Windsor, New Jersey. When you think about the tariffs, if you, you know, in our world or from a pharmaceutical perspective, I would divide it into two buckets.
One is tariffs on finished goods, which, you know, in different pockets of business, it could be a tailwind for us, right? Generics, there is a significant amount of generics that comes from products in India for the U.S. market. If there are tariffs imposed there, that could be a potential tailwind for us. Again, it depends on, you know, and look, this is an evolving situation, but it also depends on who is absorbing the impact of the tariffs, right? That is a market dynamic that, you know, we'll have to see how that will play out. I think the second part is really the input materials, right? I think the administration does want to support U.S. manufacturing.
I believe that they will be very thoughtful about imposing tariffs on input materials, you know, that are, you know, in excess of what is already in place, right? There is already some tariffs in place for, you know, active pharmaceutical ingredients or API from China and from other countries. I think the escalation of those could, you know, have an impact on, you know, folks that are manufacturing in the U.S. That is, I think, I am sure that the administration will be thoughtful about implementing tariffs in those areas. Obviously, this is an evolving situation, and we continue to closely monitor the same.
Okay. That's a great overview. Thanks so much. Where I want to start is right at the very beginning. You have this rare disease focus, and I'm really curious as to why ANI originally set up the sales force of, call it, 10, 11 people in ophthalmology prior to the acquisition of Alimera. What was it about that market that you identified as attractive?
Got it. Yeah, look, maybe a little bit of background on Cortrophin. So Purified Cortrophin Gel, which is our lead asset, is a porcine-derived purified corticotropin. It's a late-line treatment option for patients struggling with certain chronic autoimmune and inflammatory disorders. And Cortrophin is approved for multiple indications across therapeutic areas. Now, we initially launched, which is in 2022, January 2022, we launched into three areas, right? Neurology, nephrology, and rheumatology with a combined sales force. So one sales force detailing into these three different therapeutic areas. As we look to expand Cortrophin Gel, we prioritize ophthalmology as a strategic area of focus for a couple of different reasons. Number one, in ophthalmology, Cortrophin is indicated for severe, acute, and chronic allergic and inflammatory conditions affecting the eye, and its adnexa, such as keratitis.
Ophthalmology, as a percentage of total ACTH prescribers, has almost doubled to more than 10% over four years. As we looked at, you know, ophthalmology, we saw that ACTH in itself, you know, was a significant opportunity and therefore synergistic with Cortrophin. We also obviously did a scan of what was available, you know, in the market in terms of assets, because, you know, we do not have a development engine at this time, an R&D engine of our own. We looked at across assets in phase 3 and beyond and saw that, you know, this was an area that we could find assets and would be attractive for M&A.
For those two reasons, we, you know, first, again, because your question is the initial 11, we did a targeted sales force to explore the area of ophthalmology and launch our presence for Cortrophin into ophthalmology. And then obviously, we did the acquisition of Alimera to expand it beyond just selling Cortrophin. Yeah.
Okay. Maybe we can continue on a little bit about that. With the Alimera acquisition, we're starting to see the background as to the strategic rationale behind the acquisition. Perhaps you can expand on that a little bit more and the two products that came along with it and what they're indicated to treat, et cetera, et cetera.
Sure. The acquisition of Alimera was aligned with the, you know, M&A strategy we were pursuing, and I started speaking a little bit about. Specifically, it expanded the scope and scale of our rare disease business. It strengthened one of our priority therapeutic areas, ophthalmology, which is a key area for our lead asset, you know, Cortrophin and the overall ACTH market. Very importantly, the deal gave ANI two durable assets with growth potential. Importantly, we expect ILUVIEN and Yutiq to be durable given their patent protection and high barriers to genericization. You know, as far as what are these products indicated for, they are both intravitreal implants, long-acting intravitreal implants, where the active ingredient is fluocinolone acetonide. They are in two different strengths: 0.19 milligram of fluocinolone and 0.18 milligrams.
They are indicated for Illuvien is indicated for both diabetic macular edema and non-infectious uveitis affecting the posterior segment of the eye. That is Illuvien. Then Yutiq is indicated for non-infectious uveitis affecting the posterior segment of the eye.
Okay. Perfect. Great introduction. Just before we get into the specifics of DME and uveitis, I wanted to touch on the fact that you're likely going to transition almost completely over to Illuvien over time from Yutiq. Could you expand on that? And if it, you know, what are the implications of that for the company and perhaps why?
Sure, sure. Our decision to merge the labels of Illuvien and Yutiq was driven by the intent to increase the supply security for our ophthalmology franchises. Note, both Illuvien and Yutiq are substantially similar ophthalmic intravitreal implants with the same active ingredient, fluocinolone acetonide, and almost identical strengths, right? Again, a quick reminder, Illuvien having 0.19 milligrams of fluocinolone and Yutiq having 0.18 milligrams of fluocinolone. In fact, the clinical trials for both NIUPS, that's non-infectious uveitis affecting the posterior segment of the eye, and DME, diabetic macular edema, were run on implants with 0.18 milligrams of fluocinolone acetonide. The newer manufacturing process used exclusively for Illuvien results in the strength of 0.19 milligrams per implant.
What we did is we submitted a prior approval supplement or PAS to the FDA and successfully received approval to add Yutiq's indication of chronic NIUPS affecting chronic NIUPS to the Illuvien label. We plan to transition commercialization efforts later this year for both DME and chronic NIUPS to a single product, Illuvien. As a reminder, Illuvien is already approved and marketed for both DME and NIUPS outside the U.S., including in 17 European countries and the Middle East. In addition, we extended our partnership with our contract manufacturer, Siegfried, for five years until 2029. Siegfried has been a reliable partner for Illuvien for over 10 years. As a part of this extension, we agreed to partner with Siegfried to upgrade the equipment on the existing manufacturing line in Irvine and significantly expand capacity through the addition of a second manufacturing line. Both the equipment and equipment upgrade and capacity expansion initiatives are on track.
Okay. Fantastic. Let's spend a minute on DME. Maybe you can describe the disease state, epidemiology, and commentary on the current treatment paradigm just so that people can understand where Illuvien fits in that whole construct.
Absolutely. The market opportunity for DME and for Illuvien and DME represents more than 50,000 underserved patients. You know, one in 15 people with diabetes develops DME. Anti-VEGF, which stands for vascular endothelial growth factor, as many of you would be aware, therapies are considered the mainstay DME treatment, and steroids are usually reserved for patients who are not well served by anti-VEGF therapy. Providers typically cycle through anti-VEGF treatments, which, you know, does not always end up in success. Given the multifactorial nature of inflammation, an estimated 75,000 patients have a suboptimal response even after treatment with multiple anti-VEGF therapies. 70% of those patients respond positively to a steroid trial. Illuvien is an ideal treatment option for these patients. Illuvien is differentiated from other intravitreal steroids by its duration, durability of therapy.
Illuvien is the only approved three-year option, delivering a stable dose of steroids for a long period of time. Most other steroid injections and implants have durability lasting weeks or months. This makes it a good option for patients for whom a prior steroid was demonstrated to be safe and effective. Of those 50,000 patients that I just referred to, less than 5,000 patients received Illuvien in 2024. This opportunity supports our confidence in growth for Illuvien in the U.S. market.
Okay. That's very helpful. You're talking about less than 5,000 patient starts with the drug with a target opportunity that is 10 times that amount. Are there any practical aspects that have to do with the treatment of this patient population that could materially prevent the company from making inroads relative to that 50,000? Over time, could we see reasonable penetration of that market?
Yeah. We believe this is the total addressable market as our analysis takes into account prior anti-VEGF and prior steroid experience. Making 53,000 patients a very good estimate of the current Illuvien opportunity in DME. We also have a New Day Trial and a positive readout from the New Day Trial, which, you know, I can describe a little bit for you, the New Day Trial. That has the opportunity to expand that market as it could move Illuvien, in fact, up in the treatment algorithm, right? There are about 900,000 patients in the country that have DME, you know, and I was talking about a small subset, 53,000 being the addressable market. You know, a positive readout from the New Day Trial has the opportunity to expand that market as it could move Illuvien up in the treatment algorithm, potentially in combination with rather than post-anti-VEGF therapy.
Now, the obvious next question is, what is the New Day study? I'm happy to give an overview if you'd like. Yeah?
Of course.
Yeah. Thank you, Doug. New Day is investigating the use of Illuvien in combination with the current standard of care anti-VEGFs for the treatment of DME. If the results are positive, it could demonstrate a benefit of moving Illuvien to early line therapy in combination with anti-VEGF therapy to provide better outcomes for patients. New Day was fully enrolled with 306 patients in approximately 42 sites. The last patient last visit occurred in early January. We expect top line results in this quarter, meaning the second quarter of 2025.
Okay. Just a couple of wrap-up questions on that trial then. How large was it? Can you describe the entry criteria for the patients? Were they naive to treatment, or had they had previous treatments? Just to give us a better idea of the patient population.
Yeah. Your first question was the.
Oh, just the size. I gather it was around 300.
306 patients. Yeah. I just, yeah, I just said that. Three hundred and six patients. It was conducted across 42 sites in the U.S. The second question was the inclusion criteria. On that, essentially, it's patients early in the treatment, you know, early in the DME treatment. Either they're naive to anti-VEGFs or they haven't received an anti-VEGF for at least the last 12 months. Less than four or not received an anti-VEGF in the last 12 months.
Okay. Fantastic. One question I did have is when you think about that time of 50,000 or 53,000 patients, are those patients typically treated bilaterally? What would be the implications from a pricing perspective and ultimate revenue perspective for the company taking that into consideration?
Sure. Look, DME can present bilaterally or unilaterally. Even when presenting bilaterally, it may progress at different rates and require different treatment options for each eye. Some studies have shown that over 50% of patients with DME have it bilaterally. Our pricing is based on per implant. Yeah. I think that's how we, you know, for each eye, that's the cost of an implant. It's obviously for a three-year period.
Okay. Okay. Excellent. What I wanted to do before I move on to uveitis is just talk about the competitive environment, any competitive threats that you may see specific to your product within the DME targeted group.
Sure. Look, the common theme amongst new products in the DME market, as we see it, has been to improve durability in hopes to reduce the need for more injections. To this day, Illuvien remains the only product indicated for DME with efficacy of up to three years. That is how we think about the competitive landscape evolving going forward. Yeah.
Okay. Perfect. Let's switch to chronic non-infectious uveitis for the posterior segment. Maybe you can talk about that market like you did for DME and where would Illuvien fit in it.
Sure. Non-infectious uveitis affecting the posterior segment of the eye frequently runs a relapsing and sometimes smoldering disease course that can cause damage to photoreceptors and may result in cumulative damage to the retina, leading to a significant deterioration of vision. The indication of Illuvien and Yutiq for NIUPS can include chronic intermediate, posterior, or panuveitis. It can also include chronic non-infectious uveitis affecting the posterior segment of the eye that develops following ocular surgical procedures. Corticosteroids are the mainstay of treatment in chronic NIUPS, right? Unlike DME, where anti-VEGF is the mainstay. Treatment options include local treatments like Yutiq and Illuvien, other intravitreal corticosteroid implants such as dexamethasone, and ocular corticosteroid injections and systemic steroids and systemic immunomodulatory therapy. There is no requirement for a steroid challenge in our NIUPS indication, so it can be used early or later in the course of treatment.
You know, there's about 100,000 patients with, you know, uveitis in the posterior segment. That is the addressable market for us. You know, very similar to Illuvien, I mean, sorry, to DME, there are less than 5,000 patients that are on therapy today with or were put on therapy for NIUPS with Yutiq last year. Obviously, going forward, as we had described, you know, we'll be promoting Illuvien now that the FDA has approved the label for NIUPS with Illuvien.
Yeah. Okay. One of the things, other trials that you have ongoing right now is SYNCHRONICITY. But it utilizes Yutiq, and I don't think it's going to be a big issue to transition. However, could you talk about the potential implications of that trial like you did for DME?
Sure. SYNCHRONICITY is designed to provide retina and uveitis specialists with a broader sense of the utility of Yutiq across, and now Illuvien, across a variety of patients with chronic NIUPS. The trial had enrolled—it's an open- label study—and the trial had enrolled 110 patients in approximately 25 sites around the U.S. We expect preliminary top line results also in the second quarter of 2025. Then, you know, final results at the end of the year, early next year.
Okay. Finally, maybe we can wrap up on this space just with respect to the competitive threats question again, products like Ozurdex and Retisert and where they may fit in this whole thing relative to your product.
Sure. Look, corticosteroids are the mainstay treatment in chronic NIUPS due to the inflammatory nature of the disease. Illuvien and Yutiq are differentiated by its durability, right, being the longest-acting corticosteroid on the market, right, even in comparison to the two you mentioned, which are in weeks and months versus—I'm sorry—Ozurdex, which is in, you know, so Illuvien is the longest-acting corticosteroid on the market. There are other agents in development, and, you know, we continue to monitor the competitive landscape.
Okay. Excellent. One of the things I did want to spend some time on was the US Ophthalmology Force, where you've gone from 30– 46 now across the whole company. What KPIs are you watching and why as you look at the three products?
Yeah. Primary KPI for sales would be the sales of Illuvien, Yutiq, and Cortrophin Gel. We are also monitoring input parameters such as mix of calls, target lists to refine and improve the effectiveness of our newly combined sales force selling three products.
Okay. Good. I know that for Q1 2025, you had indicated some headwinds for the combination of Illuvien and Yutiq due to what you described as evolving market access dynamics. Perhaps you could describe that and how long that might last before we start to see a continuation of the growth.
Yeah. In the past, patients with Medicare and Medicare Advantage Plans had access to programs that assisted with offsetting the cost of the patient responsibility based on financial need. Some of these programs have not received adequate funding yet in 2025. What we are doing about it is, first and foremost, ANI has in place a patient assistance program that provides access for patients in financial need with free Illuvien and Yutiq as needed. Secondly, we are spending time with our HCPs to understand their response to these market access changes and refining our commercial approach accordingly. You know, as we have shared in the prepared remarks, there are currently fewer than 5,000 patients on therapy for each of Illuvien and Yutiq.
We estimate that the addressable patient population for each drug is approximately 6-10 times that based on the epidemiological data that we had shared earlier. While this is a near-term issue to work through, we remain confident of the growth prospects for our products in both DME and NIUPS.
Okay. You obviously think there's a great opportunity with these drugs. I just underscore the fact that you went out recently and bought the royalties back, that 3% royalty from SWK. Maybe you can just expand on that quickly as we start to wrap up, and maybe there'll be one more question before I thank you.
Sure. We did a financial assessment, and it made sense for us to buy the royalty back given the confidence we have in our Illuvien and Yutiq franchises. As simple as that.
Okay. Okay. Maybe to wrap things up, people might be curious about the IP status of the key drug, Illuvien, and how that fits into how you're operating the business.
Sure, Doug. Sustainability of Illuvien is driven really in the long term by the barriers to genericization. ANI has a strong generics R&D capability and is able to assess the risk-effort-reward combination for Illuvien. We believe that that makes it unviable. You know, a generic development program would require a multi-year clinical program with significant risk and require significant investment. We believe that, you know, again, the risk-effort-reward combination for an Illuvien generic makes it unviable. It will take, you know, a long period of time, you know, to get done. We continue to obviously monitor the competitive landscape and really remain confident in the barriers to genericization.
Excellent. I think we're basically out of time. I want to truly thank you for taking the opportunity to sit down with us today. It sounds like a really interesting story. Thanks again for being the first company at our inaugural ophthalmology conference. Appreciate it. Thank you, Nikhil.
Yeah. Thank you, Doug. We'll talk to you soon. Thank you, Investors. Take care.
Okay, then. Bye-bye.