All right, good afternoon. Let's get started. David Amsellem from the biopharma team here at Piper Sandler. Welcome to day two of the 36th annual Piper Sandler Healthcare Conference. And with us is ANI Pharmaceuticals. We have Nikhil Lalwani, CEO, and Steve Carey, CFO. Thanks, gentlemen, for joining us. And maybe I'll just start off with kind of a bigger picture question, if you will. So you've really transformed the business with the ramp of Cortrophin. You acquired Alimera. Now almost half the business is rare disease-focused brands. So I guess looking forward, how are you thinking about where you want to take the business in terms of that additional asset acquisitions, bolting on additional commercial stage assets where you can leverage your rare disease capabilities, and just give us a sort of a lay of the land in terms of what comes next?
Yeah. Thank you for having us, David. And good afternoon, everyone. And thank you for joining us. We appreciate your kind words as you talk about the transformation of ANI and what the team has been able to accomplish. Over the past four-plus years, we've transitioned ANI from the legacy businesses that we had in generics and established brands to a rare disease business where rare disease, as you correctly pointed out, David, will account for more than 50% of ANI revenues in 2025. And we have three commercial and durable assets in Cortrophin Gel, ILUVIEN, and YUTIQ. ILUVIEN and YUTIQ coming to us through the acquisition of Alimera. As we look forward, we look to continue expanding the scope and scale of our rare disease business. And where do we look? How will we go about doing that?
Our lead asset, Cortrophin, covers multiple therapeutic areas: neurology, nephrology, rheumatology, pulmonology, and ophthalmology. We've already done one synergistic acquisition in ophthalmology where we now have a 46-person sales force that's out there detailing all three products. The next move from a BD-M&A perspective would be to expand scope and scale by looking at one of these synergistic similar M&A strategy, right? Look at an asset that is synergistic with Cortrophin, so in one of these priority therapeutic areas, is a commercial asset with durability and growth, as well as of a deal size that is appropriate for the company size that we currently are. So that's how we think about growth. Again, from a company perspective and capital allocation prioritization perspective, rare disease is where we will focus capital allocation for BD and M&A and growth.
Then for our Generics platform, which is growing at high single digits, low double digits, we'll continue to invest a percentage of sales in R&D to drive new product launches. We've had 16 new product launches this year. To continue delivering high single digits, low double digit growth on the back of that, along with our operational excellence, our U.S. manufacturing footprint, and strong compliance track record. That's sort of the overall picture in terms of where we're taking the company.
Okay. So let's talk about the Alimera acquisition, which was recently closed and the addition of ILUVIEN and YUTIQ. I guess just help us understand your thinking on the growth runway for both assets.
Sure. So Alimera, the two assets, ILUVIEN and YUTIQ, are durable assets because there are high barriers to genericization. It takes a significant investment and multiple years and a clinical study to genericize these assets. So we believe there's durability in addition to the IP landscape that is in place. Both these assets have significant growth potential. And let me talk about that. So ILUVIEN is a long-acting intravitreal implant that is indicated for diabetic macular edema, which is a leading cause of vision loss in patients. ILUVIEN is used. The current standard of care for diabetic macular edema is anti-VEGFs. And after patients are treated with multiple rounds of anti-VEGFs, when the response to anti-VEGFs is suboptimal, at that point, they're tried for steroid response. And when the response to steroids is positive, that's when they are considered for ILUVIEN.
So just in terms of the patient funnel, there are, according to ANI research, there's about 900,000 patients that have DME. Of those 900,000 patients, about 77,000 patients have suboptimal response to multiple anti-VEGFs. And 53,000 of those show positive response to steroid trial. Of those 53,000, less than 5,000 new patients starts for ILUVIEN currently in 2024. So what that means is that there's a significant runway for growth in ILUVIEN. Let me now turn my attention to YUTIQ. YUTIQ is indicated for the treatment of chronic non-infectious uveitis in the posterior segment of the eye. YUTIQ, so NIU-PS, has about 100,000 patients in the U.S. And current YUTIQ new patient starts is less than 5,000 also. So again, a significant runway for YUTIQ too. Unlike DME, for NIU-PS, steroids are the standard of care. And YUTIQ is the longest acting or longer acting intravitreal implant.
It has, again, a significant growth runway for YUTIQ.
Now, there's the NEW DAY trial.
Correct.
Looking at ILUVIEN in combination with anti-VEGF treatment. So how do you envision the impact of that trial in terms of whether it's opening, upgrade, or repair access, more adoption in terms of physicians wanting to use it? How do you think about the importance of that trial?
Sure, David. So for those that are not familiar, NEW DAY is a randomized single-masked controlled clinical trial, which is across multiple sites. And what it's doing is it's investigating the earlier utilization of ILUVIEN in the treatment of DME. There are 600 treatment naive sorry, anti-VEGF treatment naive patients that are in two different arms. In one arm, you have ILUVIEN in combination with the anti-VEGFs. And in the other arm, you have ILUVIEN sorry, anti-VEGFs, the current standard of care, which is anti-VEGFs alone. And what this is exploring is, does the combination of ILUVIEN in combination with anti-VEGFs, can that be used earlier in the treatment of DME? If positive, we believe that this can expand the utilization of ILUVIEN in the earlier treatment of DME.
As we were describing across the patient funnel a few minutes ago, currently, ILUVIEN is only considered after suboptimal response to multiple rounds of anti-VEGFs. This study, if positive, will support an earlier utilization of ILUVIEN in combination with anti-VEGFs for the treatment of DME. So the patient funnel, it just takes us sort of to a much broader patient pool.
Okay. And in terms of the increased availability of anti-VEGF biosimilars and associated lower cost of these agents, how do you think that could impact ILUVIEN? In other words, I mean, could it actually be a good thing if the anti-VEGFs are cheaper due to biosimilar availability, particularly to the extent that NEW DAY is successful?
Sure. So let me just complete one thing about NEW DAY that I wasn't able to complete, which was the last patient last visit for NEW DAY is in this month, in December 2024. And the top line results will come towards the end of the quarter or end of first quarter 2025 or second quarter of 2025. So it's almost imminent, coming soon. To your question on biosimilars, I think the introduction of biosimilars for anti-VEGFs is good for patients because it gives them another treatment option. Because ILUVIEN is currently used later in the treatment paradigm, right after multiple rounds of anti-VEGFs, the introduction of biosimilars, we do not believe will have a material impact on the use of ILUVIEN.
Having said that, to your point on if New Day is successful, the use of ILUVIEN in combination with anti-VEGFs could see a tailwind with the biosimilars. I mean, to be honest, with the three-year or with the longer-acting intravitreal implant that ILUVIEN is, we do not see treatment costs as a barrier to utilization of ILUVIEN. Yeah.
Okay. And then YUTIQ, there is the readout of the SYNCHRONICITY study. So I wanted to touch on that. That's a study in chronic non-infectious uveitis in the posterior segment. But talk about the overall importance commercially of that study as it relates to YUTIQ.
Yeah. So the Synchronicity study is an open-label study. We have about 106 patients enrolled across 25 different sites. The last patient, last visit for this study is in the end of 2025, in Q4 2025, and the readout in the first half of 2026. The Synchronicity study is exploring the use of YUTIQ in the treatment of chronic non-infectious uveitis in the posterior segment of the eye. And a positive readout on this study will help sort of further reinforce for retina specialists the use of YUTIQ in non-infectious uveitis in the posterior segment of the eye. As I said, there's about 100+ patients enrolled across 25 different sites. And last patient, last visit is towards the end of 2025.
Can you provide some color on the number of new starts annually on ILUVIEN and YUTIQ and also talk to the sales mix between the two products?
Yeah. As I said, there's less than 5,000 new patient starts on ILUVIEN and less than 5,000 new patient starts on YUTIQ. I think that's the granularity that we've given so far. And again, the opportunity is significant, right? 53,000 patients with suboptimal response to anti-VEGFs that show positive response to steroid trials from an ILUVIEN perspective and 100,000 patients with chronic NIU-PS. So again, the runway for growth for both assets is significant.
Okay. And the sales mix between the two? Or you're not.
Yeah, we don't, and this is also what Alimera, the part of.
Okay, so you've talked to the commercial synergies in ophthalmology between ILUVIEN, YUTIQ, and Cortrophin Gel. Can you talk about prescriber overlap, particularly in terms of how it benefits Cortrophin Gel?
Sure, so we now have in place a 46-person sales force across the nation. There is approximately 50% overlap between high prescribing potential HCPs for Cortrophin as well as for ILUVIEN and YUTIQ, and with this 46-person sales force, we believe we can reach over 3,600 ophthalmologists, and that helps us drive growth both for ILUVIEN and YUTIQ as well as for Cortrophin in ophthalmology.
Okay. Now moving over to Cortrophin Gel. So you're focusing on a number of therapeutic specialties. Just remind us of the core therapeutic specialties you're focusing on and where you see the biggest growth opportunities for the product.
Great. Yeah. No, thank you, David. I think Cortrophin has the benefit of being indicated across multiple autoimmune indications. In terms of therapeutic areas, when we launched, we launched in three therapeutic areas: nephrology, neurology, and rheumatology. We had one sales force targeting all three therapeutic areas. As we grew Cortrophin, we launched a pilot sales force in pulmonology and then expanded that pilot sales force in pulmonology, adding a second pilot sales force. Then obviously, we launched the ophthalmology pilot, which was successful, and we did the ophthalmology acquisition. When you think of Cortrophin today, there's a core sales force that details across nephrology, neurology, and rheumatology. There's a smaller sales force that's specialized in pulmonology. Now there's an ophthalmology sales force, which is detailing Cortrophin, ILUVIEN, and YUTIQ.
Look, when it comes to growth, I think that we're a little bit spoiled because we're seeing growth across therapeutic areas. In the core, the indications that we launched with nephrology, neurology, and rheumatology, we see a significant amount of growth coming from there. We also see growth in pulmonology and then ophthalmology. The early signs from the combined sales force is also very positive. So we're seeing growth across the therapeutic areas. And when you think of Cortrophin, I think two other things to highlight. One is that the category, which is the ACTH category, that had been declining over the years, first, the decline slowed after we launched. And then most importantly, in 2024, based on our guidance and the competitor's guidance, the only other player, the category will see 23%-24% growth.
And even with those added together, the category will be $650+ million, which is still a fair distance away from the peak where ACTH used to be, which was about $1.2 billion in 2017. Now, that $1.2 billion didn't include an indication that we have that the competitor does not have, which is acute gouty arthritis flares. That's another growth factor for us. We've launched a 1 mL vial for that. The standard presentation or the initial presentation we launched Cortrophin with was a 5 mL vial. Now we've launched a 1 mL vial for acute gouty arthritis flares. We did that late last year and are seeing traction in that area too.
So across the board for Cortrophin, we see a strong multi-year growth trajectory across therapeutic areas, both the ones we launched with, which is nephrology, neurology, rheumatology, as well as the newer indications of ophthalmology, pulmonology, and acute gouty arthritis flares.
Yeah. So I wanted to come back to the overall ACTH space and how it's returning to growth. I mean, Acthar Gel had been continuing to decline even when you were in the market. And maybe it's starting to show evidence of stabilization. But your entry, you were growing, you're obviously growing the Cortrophin footprint. So it sort of begs the question, is the growth for your product coming from doctors who had been using Acthar Gel previously and have moved over to Cortrophin Gel, or has there been some growth coming from doctors who are just new to the category altogether? Maybe help us understand in terms of the two buckets where the growth is where more of the growth is coming from.
Sure. So when we launched initially, the growth came from prescribers who had used because obviously, we had the benefit of seeing who were ACTH believers. And so initially, the growth came from prescribers that were not naive to ACTH but had used ACTH and maybe reduced their usage or were continuing to use ACTH. So that's where initially the growth came from. But today, the growth comes from both, from folks that use from prescribers that have used ACTH as well as naive to ACTH or new prescribers. And we're seeing growth across both segments.
Okay. What does the payer landscape look like for Cortrophin Gel? And can you also talk to net pricing, particularly relative to Acthar ?
Yeah, so since we launched, we tried to bring another treatment option for patients. We priced our WAC at a discount. Today, the discount is 23.5% lower than the competitor's product, and since we've launched, we've been working actively with payers to expand the access for the appropriate patients for Cortrophin Gel. Competitively, our competition has also been engaged with payers, and part of the growth has come from now we have a broader access to this therapy to patients for both products, and for the most part, there's parity access for both products. Yeah.
Are there any specific therapeutic specialties where access is more challenging than others? In other words, is MS harder than, say, rare autoimmune diseases of the adrenal gland?
Yeah, I mean, for sure. No, for sure. There is variance in the coverage levels of access. But again, I think that the team is working through our patient support team. Our field reimbursement team works to support the benefits investigation and to ensure the prior authorization conditions are met and that the patients in need get the appropriate patients get the therapeutic that they need.
Okay. You did allude to the 1 mL vial for acute gouty arthritis. I wanted to come back to that and just pick your brain on how big of an opportunity you think that could be here. That is a unique indication for you.
That's right.
It's not in the Acthar label. So how do you think about the potential impact of that?
Yeah. We think that there is a significant potential for acute gouty arthritis flares, which is an indication we have and the competitor doesn't. We haven't dimensionalized that yet, and it's early days. It's obvious it works slightly differently. It's buy and bill, unlike the self-administration, which is what for a number of the other indications Cortrophin is used for. But we think that there is significant potential to that, and we look forward to updating investors on the progress that we make for this indication.
Okay. And then I think you have a prefilled syringe for Cortrophin Gel. So maybe talk about the kind of impact that presentation could have.
Yeah. So just backing up, Cortrophin, we believe, has a strong multi-year growth trajectory. And to support that strong multi-year growth trajectory, we've introduced different presentations. So we introduced the 1 mL vial to support the acute gouty arthritis flares indication. And then for patients that have dexterity issues, what the prefilled syringe does is it reduces one step in the administration process for Cortrophin gel and is helpful for patients with dexterity issues. What we are also doing is continuing to look at other ways to reduce the pain points and enhance the convenience both for prescribers as well as for patients. And we're also working on generating further scientific evidence to support HCPs as they think about the usage of Cortrophin gel for their appropriate patients.
Okay. One of the things that has been sort of well documented is the unique process of making ACTH. Certainly, this is something that's animal sourced. And there's real complexity here. So I guess with that in mind, how are you thinking about the picket fences around Cortrophin Gel as we think about long-term exclusivity?
Yeah. No, I think that's a great question. I think one of the benefits that Cortrophin has is that it is porcine derived. And there have been multiple efforts made to genericize this drug that have been unsuccessful by leading players. And so we think the path to genericization is very complex and very tough. And obviously, to the best of our understanding, we're monitoring this on an ongoing basis. And to the best of our understanding, there's no substantial ongoing effort to genericize Cortrophin gel or the competing ACTH product. Equally important, since we've launched, we've added IP that has runway till 2041, 2043 to further sort of, to your point, protect the durability of our product. And the competitor also has got patents approved in that sort of time frame, which again gives them IP protection until a longer period of time too.
So to be clear, you think this is going to be a duopoly for the foreseeable future?
Yeah. We do not see indication of competition in either the ACTH category.
Okay. I didn't ask about the generics business. I probably should in the 55 seconds we have left. So it has been growing. It is an oral solids business. So usually, growth in oral solid generics don't usually get repeated often in the same sentence. But it's happening here. So maybe talk about the growth drivers here in the business and how you envision the impact of new launches in 2025.
Yeah, so first of all, the dosage forms we compete in are tablets, capsules, solutions, suspensions, gels. We have some controlled substances and hormonal products too. From our perspective, the growth and the success of our generics business is driven really by the strong R&D organization that we have. We've got 16 new products that we've launched this year. We're second in competitive generic therapy approvals. And what that does is it just gives us the anchor of driving high single-digit, low double-digit growth. That in combination with the U.S.-based manufacturing footprint, operational excellence, and a strong FDA compliance track record helps us continue to deliver high single-digit, low double-digit growth in our generics platform.
Okay, well, I'll leave it there. We're out of time. Thanks, Nikhil. Thanks, Steve. Thanks, everyone in the audience.
Thank you.