ENTA - NASDAQ

Good afternoon and welcome to Enanta Pharmaceuticals' Fiscal First Quarter Financial Results Conference Call. At this time, all participants are on a listen-only mode. There will be a question-and-answer session at the end of the prepared remarks. Please be advised that this call is being recorded. I would now like to turn the call over to Jennifer Viera, Investor Relations. Please go ahead.

Thank you, operator, and thanks to everyone for joining us this afternoon. The news release with our fiscal first quarter financial results, was issued this afternoon and is available on our website. Making remarks on today's call are Dr. Jay Luly, President and Chief Executive Officer; and Paul Mellett, our Chief Financial Officer. Dr. Scott Rottinghaus, our Chief Medical Officer; and Dr. Tara Kieffer, our Chief Product Strategy Officer, will be available during the Q&A portion of this call. Before we begin with our formal remarks, we want to remind you that we will be making forward-looking statements, which may include our plans and expectations with respect to our research and development pipeline and financial projections, all of which involve certain assumptions and risks beyond our control that, could cause our actual developments and results to differ materially from those statements. A description of these risks is in our most recent Form 10-K, and our other periodic reports filed with the SEC. Enanta does not undertake any obligation to update any forward-looking statements made during this call. With that, I'd like to turn the call over to Dr. Jay Luly, President and CEO. Jay?

Thank you, Jennifer, and good afternoon, everyone. In the first quarter of 2024, Enanta began an important year, which has the potential to advance our programs, in both virology and immunology and drive value across the company. Through our recent expansion into immunology, our mission continues to center around the development of small molecule treatments for indications of high unmet need, and we are leveraging our drug discovery capabilities, to bolster our pipeline for near and long-term value creation. Today, I'll provide an overview of our progress during the first quarter, beginning with our respiratory syncytial virus, or RSV, program, and then segue into our new immunology program, targeting chronic spontaneous urticaria, or CSU. RSV is a severe respiratory infection associated with significant morbidity and mortality that, can cause serious disease in infants, children, and other high-risk populations, including the elderly and individuals with congestive heart failure, chronic obstructive pulmonary disease, or asthma. Despite the availability of vaccines and prophylactic monoclonal antibodies, the uptake has been low, and breakthrough infections will still occur. The current rate for adult RSV vaccine adoption, is estimated to be only 11% of the eligible population. Further, both strategies for pediatric prophylaxis, the maternal vaccine, and monoclonal antibodies, provide short-term passive immunity for infants, and only shift the infant's first infection to the next season. Because of this significant need for safe and effective treatments, our goal is to develop an oral, best-in-class treatment for RSV, through our broad development program, which includes zelicapavir, an N-protein inhibitor, formerly known as EDP-938, and EDP-323, an L-protein inhibitor. Both have fast-track designation from the FDA. Our conviction in our approach to RSV, is rooted in the core mechanism of our molecules, replication inhibition. We believe both zelicapavir and EDP-323 have robust potential as monotherapies, but we're also excited by the opportunity, to combine them and potentially broaden the treatment window for addressable patient populations.

Thank you, Jay. I'd like to remind everyone that Enanta reports on the September 30 fiscal year schedule. Today, we are reporting results for our fiscal first quarter ended December 31, 2023. For the quarter, total revenue was $18 million and consisted of royalty revenue, earned on AbbVie's global MAVYRET net product sales. This compares to total revenue of $23.6 million, for the same period in 2022. As a reminder, 54.5% of Enanta's ongoing royalties from AbbVie's net sales of MAVYRET that are included in our revenue are being paid over to OMERS, the royalty buyer, in our April 2023 royalty sale transaction. For financial reporting purposes, the sale transaction was treated as debt with the upfront purchase payment to us of $200 million recorded as a liability. As such, we continue to record 100% of the royalties earned as revenue, and will then amortize the debt liability proportionately as 54.5% of the cash royalty payments are paid to OMERS until a cap of 1.42 times the purchase payment is met, after which point 100% of the cash royalty payments will be retained by Enanta. Non-cash interest expense of the debt will be recorded in Enanta's consolidated statement of operations, based on an imputed interest rate. Interest expense was $3.4 million for the three months ended December 31, 2023. Moving on to our other expenses, the three months ended December 31, 2023. Research and development expenses totaled $36.4 million, compared to $40.9 million for the same period in 2022. The decrease was primarily due to a decrease in costs associated, with our COVID-19 program, as we previously announced that our plans, to pursue any future COVID-19 efforts, would be in the context of a collaboration. General and administrative expense for the quarter was $16.5 million, compared to $12.7 million for the same period in 2022. This increase was primarily due to an increase in stock compensation expense and an increase in legal expenses related to the company's patent infringement suit against Pfizer. Other income net totaled $0.9 million. Enanta recorded an income tax benefit of $0.6 million for the three months ended December 31, 2023 for interest earned on a pending $28 million federal income tax refund, compared to an income tax benefit of less than $0.1 million for the three months ended December 31, 2022. Net loss for the three months ended December 31, 2023 was $33.4 million, or a loss of $1.58 per diluted common share, compared to a net loss of $29 million, or a loss of $1.39 per diluted common share for the corresponding period in 2022. Enanta ended the quarter with approximately $337 million in cash and marketable securities. We expect that our current cash, cash equivalents and short-term marketable securities, as well as our ongoing retained portion of royalties, will continue to be sufficient to meet the anticipated cash requirements of our existing business and development programs, through fiscal 2027. Further financial details are included in our press release and will be available in our report on Form 10-Q when filed. I'd now like to turn the call back to the operator and open up the lines for questions. Operator?

And thank you. [Operator Instructions] And one moment for our first question. And our first question comes from Roy Buchanan from JMP. Your line is now open.

Hi, thanks for taking the questions. I just had a few on zelicapavir. First one on the RSVPEDs readout, presumably in 3Q. I guess under the negative scenario where you don't see any virological effects, is it conceivable that you just wind down the program, stop RSVHR, or would you still see RSVHR readout? And then in that same scenario, and if the challenge file for 323 is highly positive, would you potentially look to immediately combine the two agents? Then I have a follow-up? Thanks.

Thanks, Roy. This is Jay. So starting with the challenge study, 323 is also on track for Q3. It's a very potent L-inhibitors. As you know, I know you know a lot about the data. And given the potency, given the PK, given the huge multiples we drive over the protein-adjusted EC90, we're very hopeful that it should show, efficacy, comparable to zelicapavir. The question of getting into combination studies right away is an interesting one. But I think, we'll probably be most interested in fully characterizing single-agent efficacy for both zelicapavir and 323 in real world and then contemplate combinations. Of course, in parallel, you could be scouting out, some of the combinations in challenge studies, foreshadowing that. But I think we wouldn't want to slow down single-agent characterization of 323. The PEDs study, again, we're on track for Q3. You're asking an interesting question. If PEDs didn't show anything, what would you do with HR? Different patient populations, I think you've got to look at the facts and circumstances, around any clinical trial result in one patient population, done under one set of conditions. And then, make good judgments as to how it might, or might not relate to a different clinical trial in a different patient population, under a different set of circumstances. So, we just need to look at data. So that's my thought on that.

Okay. Great. Presumably, HR is not too far behind. Just a follow-up, I wondered for zelicapavir, if you can put a dollar value on the two markets, the PEDs and the HR, just in your view. What do you think is a rough dollar value? Thank you.

Well, there are no established therapeutics in this market. So, I think there's a great opportunity to build, the first opportunity for an RSV treatment ever in each of those patient populations and yet other high-risk patient populations. I'm not going to be able to give you an exact dollar amount, but - it's got to be $1 billion market opportunity. It has $1 billion on it, let's just say that. And PEDs directionally, is probably the bigger piece of that, although I think, there's more data and information on RSV in PEDs, than there is in adults. And so, we're also not going to underestimate what that adult market could ultimately look like. Clearly, there's been a lot of interest in that space, in promoting vaccine opportunities for elderly. And they're doing quite well, in prophylaxis, even though only a small portion of the eligible patient population is getting vaccinated and everybody else, which is the overwhelming majority of people, aren't getting vaccinated, and would still be susceptible to infections. And even some of the vaccinated people, could be getting breakthrough infections. So, give us more time to see how that market evolves a little bit, work up final bits on a potential product profile. But we're very encouraged by our position in the field, sort of the leadership position we have in RSV portfolio, and also the fact that there are no approved drugs on the market to each unmet need. So it's a good opportunity.

Okay. Thank you.

You're welcome.

And thank you. And one moment for our next question. And our next question comes from Roanna Ruiz from Leerink. Your line is now open.

Great. Afternoon, everyone. So a question on your CSU program. So could you walk us through some of the elements that excited you about, this indication over other similar immunology indications? And I was curious what additional optimization might you, try to be working on, to get to a final candidate that could be deemed, like best in disease? And I have a follow-up after that?

Okay. Well, I'll handle part of that question, and then I'll let Tara Kieffer, Heads our Product Strategy, talk about the other part. So with regards to the optimization, I think we showed some data at JPMorgan on a prototype molecule, which we think is far along in terms of our optimization profile. We're still making lots and lots of molecules, continuing to tweak bits and pieces. But obviously, among the things we're looking at, is just really honing down potency, selectivity, making sure we've got good safety. And of course, our old friend pharmacokinetics, and hopefully once daily dosing, all of those kinds of things that we like to build in to every one of our molecules. CSU is attractive, and maybe I'm already answering some of the questions, but we're not certainly limiting ourselves to that. I think it happened to be the first program that we've announced in the area. But we're working on a few other things. We're piloting other programs, getting involved. So you can expect that there will be a broader footprint, certainly, as our slide deck anticipates in the field. And we go about it in the way that, we've done in a lot of our, well, in pretty much all of our programs. We get the biology figured out and sorted, really important to do that. Try to figure out chemical matter that, we can get into. Make sure we've got strong commercial rationale in terms of competitive landscape, potential product profiles, we set all that stuff. Start making molecules, get on the boards, start filing intellectual property. And we tend to do all of that, before we really announce a program. So suffice it to say, that's ongoing in other areas, and as the year rolls out, we'll come out with more. Does that answer your question?

Yes, that helps. And I have a follow-up on RSVPEDs as well. So given the top line coming, what do you hope to see in terms of efficacy and safety results, and how might you use that data to inform a go or no-go decision for advancing to registrational trials?

Yes, maybe since I didn't give Tara the chance on the last question, I'll let her take this one.

Sure. Yes, of course. So, Roanna, as the RSVPEDs study, first in P, so doing some dose ranging, and then looking at that optimal dose at virology, so the primary endpoint in the second part of the study. We'll certainly look at other endpoints, clinical endpoints like symptoms, but with the size of the study, we'll primarily be looking at virology endpoints. And really what we're hoping to see is some directional data and numerical trends in the virology endpoints that give us the confidence to take the program forward into a larger, more robust Phase 3 program as we move forward. So that's really what we're looking to achieve in this kind of initial team study.

Got it. Thanks.

And thank you. And one moment for our next question. And our next question comes from Akash Tewari from Jefferies. Your line is now open.

Hi, this is Amy on for Akash. Thank you for taking our question. It doesn't seem like the Phase 2b RSVPEDs study is powered to hit on symptoms or viral load reduction. So what would be a strong enough signal for you to move it into Phase 3? And then additionally, how are you thinking about the oral from death severe failure for Gilead? Does that change strategic value for your protease inhibitor at all? Thank you.

You want to take this one like.

Yes, I can build on - for the RSVPEDs piece. Yes, we will again, primarily be looking at virology. And I think there's not a lot of benchmarks in this area that, we could point to, or compare to. There is one data set out of a company called [ArcBio] where in a Phase 3 trial, they showed about a 0.6 log drop. And that did translate into an improvement, specifically significant improvement on symptoms. So that is the one sort of benchmark that we have. But again, numerical trends and directional data showing that zelicapavir is showing an improved trend in virology, compared to placebo, would give us the confidence to move forward into a Phase 3 study. Again, we'll look at symptoms. And as you said, it is a small study and the likelihood of seeing something on that, certainly in a statistically significant way is probably not as high, but we'll look at that and see what we get.

Okay. And the second part, I guess, is relates to oral remdesivir for COVID. I mean, I guess our initial reaction, I mean, we, like everybody else, just got that news late yesterday afternoon. I think it simplifies the COVID landscape, which is - one of the things that I think everybody who's involved in COVID, including us, everybody who's interested in COVID, whether its strategics or the government. We're trying to figure out what the competitive landscape is, or what the arsenal of drugs is going to be available for COVID patients. And it seems like there's one fewer now. So that's going to help clarify things. I guess, Chianogi is another one that's due to turn over a card pretty soon here. I guess there's a question about what's Pfizer doing with their follow-on molecule. We haven't seen that they've advanced it, but so a question mark there that will hopefully get sorted here in the nearer term. And then, we'll have a more complete view of what that competitive landscape looks like, which is, again, important for anybody who would be making funding decisions going forward. Our plan, as we've stated a few times before, is to only pursue 235 in the context of a collaboration.

Okay. Understood. Thank you so much. That was very helpful.

You're welcome.

And thank you. And one moment for our next question. And our next question comes from Eric Joseph from JPMorgan. Your line is now open.

Oh, thanks. Just a quick...

Eric, I'm sorry. You're sounding a little low there.

I'm sorry. Can you hear me now?

Yes, sir.

Yes. Okay. Thanks for taking the question. Just your 3Q guidance for reading out RSVPEDs, does that anticipate full accrual of your target of 90 patients? If you sort of end up tracking under that goal after this season, do you go ahead with a readout, or do you perhaps sort of push out timelines a bit?

No, I think we're still targeting to, at least hit the target enrollment, that's the plan, that's plan A. And to report data on the full set in Q3.

Okay. Great. And sorry if I missed it earlier, but can you just comment a little bit on just how accrual is taking place with RSV high risk and sort of how much further behind it might be, from a full accrual, or how much further behind basically a readout from that study is tracking relative to RSVPEDs? Thank you.

Yes, so, quarter-after-quarter, I've been asked, which study do you think is going to read out? And I could never answer the question because, especially when we target as a goal of Q3 for data. But as I said, I think at the JPMorgan conference, as we get a little further into this season, we'll have a better sense of it and should be able to make a call. And clearly today we're making that call. It looks like PEDs, is the one that will. In terms of the HR, how far behind is it? I think we just got to continue the recruitment in that. Obviously, I think we're going to need to go to the Southern Hemisphere, continue on beyond the Northern Hemisphere season. And we'll just give updates as we go. Once we have a good, more, crisp target guidance to provide it, we will. But we did see it. And to remind you – since it's a larger study than PEDs, right? So it's roughly twice the size. And it started later than PEDs. But we did see a nice uptick in this Northern Hemisphere season. It was really gratifying to see that. So, we've got over 100 sites now. We're in over 15 countries. Yes, we got a pretty big catcher's mitt on now. And - it's just all about execution and hoping, the trends towards normalcy continue. But that's been again, something that we've seen this COVID season in terms of when it started and the shape of the season and everything else. This is the first season we've seen like this in years.

Okay. Great. That's helpful. Maybe one follow-up, if I could. If you have a clear, what seems to be clear evidence of, reducing viral load in RSVPEDs? Can you talk a little bit about gating steps running an efficacy study in the pediatric population? Is that dependent on results from RSV high risk? Thank you.

No, I mean there, I can let Scott comment on that. But they're isn't, they're just very different patient populations. Scott, I don't know.

Sorry, Scott Rottinghaus, it's different patient populations. And we'd feel comfortable moving forward in pediatrics with positive results from our pediatrics study.

Okay. Great. Thanks for taking the questions.

You're welcome.

And thank you. And one moment for our next question. And our next question is Jay Olson from OPCO. Your line is now open.

Hi, this is John [indiscernible] for Jay. Thanks for taking the question. I'm glad some progress. Maybe two questions for us. First on the RSV program. Just for zelicapavir, if you decide to move the program into a pivotal study, just wondering how are you thinking about the adoption of RSV vaccine in your target patient population? And would you maybe exclude patients who recently took the vaccine? Were there some other thoughts around that for the moment? And I have another question for the CSU program?

Hi, Scott, you want to take the RSV?

Yes, for sure. So from a vaccine perspective, obviously the coverage of vaccines is going to be far from 100%. And they're not 100% efficacious. So, we still see the market and the clinical opportunity persisting there. So that's the first important point. And then in terms of a putative Phase 3 study in pediatrics, we envision including patients broadly, including patients who break through on vaccine or [indiscernible]. So we again, envision broadly studying patients in pediatrics.

You had it. That's helpful. And for the CSU program, I'm just wondering your thoughts on how would you position the oral KIT inhibitor into the treatment landscape for CSU? And there's like recent BDK inhibitor that are positively. So just wondering if you think that's kind of a good benchmark, for efficacy you're shooting for? Thanks.

Hi, this is Tara. Yes, thanks for the question. I guess the way we look at the CSU landscape is broadly at all the different mechanisms. If you think about the standard of care, being antihistamines and only 50% of the patients really being controlled on that, very few of those go on to get the only indicated biologic, which is [Xolair]. The data coming through from the BDK inhibitors, which are an oral option in development. Those studies did read out positive. I would kind of put their efficacy to be somewhat similar to Xolair in that camp. What we're excited about from a KIT inhibitor perspective, is the data that was generated through an antibody program in Phase 2 where they have seen some of the best efficacy in this disease so far. And that's really the benchmark we're looking towards and hoping to replicate that data with an oral option. So that's sort of how we're seeing this evolve. Obviously, we'll continue to watch as the data comes out, hoping to provide additional efficacy over and above what a BDK inhibitor might provide.

Yes, that's maybe a quick follow-up on the last part. I think there are some like side effects for antibody approach, maybe including the hair color change or some change in blood cells. So do you think the oral KIT inhibitor, may have some advantage on safety as well? Thanks.

Yes, so there are certainly known side effects, on-target side effects for KIT inhibitors. Overall, the antibody has had a good safety profile. Most of the AEs were mild or moderate, and they resolved. The ones that are known on target that you mentioned in terms of your neutropenia, there were generally mild hematologic impacts, and the neutropenia sort of stabilized after a short time period of a week or two of dosing. And so, what we did find out is that it didn't get worse with longer dosing, and so it seems quite manageable, at least at the levels that they're observing so far in the clinic. They were not associated with infections, at least in the trials thus far, though. And so, we're obviously keeping an eye on it, but we don't think that will be a limitation.

Okay. Got it. Thank you so much.

And thank you. And one moment for our next question. And our next question comes from Brian Skorney from Baird. Your line is now open.

Hi, guys. Thanks for taking the question. This is [Charlie] on for Brian. Just a couple quick ones here. We were wondering if you could give us some more color on how severe symptoms are and how long RSV tends to last, more thinking about pediatric and high-risk patients relative both to each other and to low-risk adults, such as those that were enrolled in the RSVPEDs trial. And then secondly, just you spoke to antibodies and CSU. Just wondering, is this a main focus for you guys in terms of how you're designing the specificity of your lead candidate? And do you consider that a bar for you to reach? And how else are you thinking about that? Thank you.

So I'll jump in on the RSV question. The kids that we've been enrolling are typical kids, often with their first episode of RSV infection. And they have symptoms typically pushing two weeks, longer than you'd expect to see in young, healthy adults given their, immune naivete. So that's kind of the length of symptoms, 10 to 14 days. And again, severity varies in our study, the sorts of symptoms that can often get you hospitalized. So fairly severe. And with adults, again, the high-risk adults that we're enrolling in our study, patients with COPD, CHF, again, have a more severe symptomatic profile, again, a couple of weeks in most cases. So that's, I think, the general severity and length of that disease. If that helps.

And I'm sorry, could you repeat your question on CSU?

Yes. And that's very helpful. Thank you on RSV. But for CSU, we were just wondering how you're thinking about designing the specificity and thinking about the antibody that you mentioned earlier. Is that kind of your goal in terms of the specificity of your molecules?

Yes. So I mean, the goal of the program is to have something that's potent to KIT. And so we've looked at that preclinically in both binding and cellular function assays. And we see nanomolar activity there. And then to be highly selective against KIT versus other kinases. We shared some preliminary data for that a few weeks ago. And showing good selectivity, we continue to optimize the compounds that we have and study them for susceptivity. But that is the goal, yes.

Thank you.

You bet.

And thank you. [Operator Instructions] And one moment for our next question. Ed, our next question comes from Ed Arce from H.C. Wainwright. Your line is now open.

Hi, everyone. Thank you for taking my questions. This is Thomas, asking a couple of questions for Ed. So for the new immunology program that is to be unveiled later this year, just try to figure out, what are some expectations that you have for now, whether it be for a large disease or small disease, or something along the line of the new KIT inhibitor program in terms of large market. And then lastly, a large unmet need as well?

Yes. So we're primarily targeting chronic spontaneous urticaria with this molecule as a primary indication.

I'm sorry. Were you talking about a future to be announced program?

Yes. The second immunology program that's to be announced this year?

Yes, so we - yes, I mean, we're trying to be very thoughtful about it going after, good markets, right? Good markets in areas where we think a small molecule could make - an important impact. But beyond that, we'll announce the program when we announce it.

I mean, I think some of the things we think about in selecting programs. Obviously, having a good market opportunity, something that has a high unmet medical need, ideally programs where there's a clear clinical path and biomarkers, or early signs of efficacy that we can get early on in the program and really understanding the underlying disease, cause of the disease and having confidence in the target and mechanism are some of the criteria we look at.

Got it. Thanks. Thank you for the additional color. And the next question, one financial question, first of all, just trying to confirm last quarter for fiscal year 2024, OpEx guidance, R&D $100 million to $120 million and then, G&A $45 million to $50 million. Just trying to figure out whether that's still on target for this fiscal year?

Yes, that is still our targeted spend at this point, yes.

All right. Fantastic. Thank you again for taking my questions.

Thank you.

And thank you. And I am showing no further questions. I would now like to turn the call back over to Jennifer Viera for closing remarks.

Thank you, everyone, for joining us today. If you have additional questions, please feel free to contact us either by email or call the office. Thanks so much and have a good night.