EIGR (2021 - Q1)

Good afternoon, ladies and gentlemen, and welcome to the Eiger First Quarter 2021 Financial Results and Business Update Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. As a reminder, this call will be recorded. I would now like to introduce your host for today's conference, Mr. Sri Ryali, Chief Financial Officer of Eiger. Sir, you may begin. Sri Ryal

Great. Good afternoon and thank you for joining us today. Welcome to our inaugural quarterly earnings call. We issued a press release this afternoon with our Q1 financial results, which is available on our website at eigerbio.com. Joining me on today's call are David Cory, President and CEO; Eldon Mayer, Chief Commercial Officer; Dr. Ingrid Choong, Senior Vice President, Clinical Development; and Dr. Colin Hislop, Senior Vice President, Clinical and Development Operations.

Thanks, Sri, and good afternoon, everyone. Thank you for joining us today. We're making great progress across our pipeline this year advancing multiple potential catalysts for value creation in 2021 and beyond, and we look forward to updating you today on our progress to date and our future plans. At Eiger, we're developing and now commercializing first-in-class, well characterized therapies for life-threatening rare and ultra-rare diseases with high unmet medical needs. Our late-stage clinical development pipeline includes 3 promising FDA breakthrough designated therapies; lonafarnib, pegylated interferon lambda, and Avexitide. The FDA approval of our first New Drug Application late last year for Zokinvy to treat progeria and processing-deficient progeroid laminopathies is a victory for patients with these ultra-rare conditions and demonstrates our ability to take a program from IND submission through NDA filing, FDA approval, and commercialization. Eiger is well positioned to make significant progress across our late-stage pipeline this year, towards additional product approvals. We continue to grow our clinical development capabilities and with the Zokinvy launch, we've established commercial and medical affairs functions that can scale and grow as we advance to additional commercial launches across larger orphan disease indications in the future.

Thanks, David. Let me begin by sharing our team's continued excitement in bringing this important therapy to children and young adults with progeria and processing-deficient progeroid laminopathies. There are about 180 children in young adults identified with progeria and progeroid laminopathies around the world. Our initial commercial efforts are focused on the approximately 20 identified patients in the U.S., where we launched in January and then the 23 identified patients in Europe, where we expect approval later this year. Eiger has provided uninterrupted supply of Zokinvy to children and young adults in over 40 countries through clinical trials and through our expanded access program and will continue to do so in countries with regulatory approval and commercialization may be impractical. As we shared in our call following FDA approval of Zokinvy, with our pre-launch planning, and the ultra-rare nature of these diseases, we've been able to execute successfully a launch with a focus and efficient effort that's providing access to the approximately 20 identified children and young adults in the U.S. who are eligible for Zokinvy.

Thanks, Eldon. As David mentioned, we are making great progress across our clinical development portfolio. Our MAA for Zokinvy is currently under review by the EMA and we are expecting approval in the fourth quarter of 2021. Our Phase 3 HDV D-LIVR study which is 75% enrolled, is on track for completion of enrollment of 400 patients this year. And assuming current conditions hold, this would put us on track to announce top-line data in 2022. The D-LIVR primary endpoint is a composite of a 2 log decline in HDV RNA plus ALT normalization. The D-LIVR study includes 2 lonafarnib-based regimens and all oral arm of Lonafarnib ritonavir and a combination arm of lonafarnib ritonavir and peginterferon alpha, with each being compared to placebo. This study design creates 2 opportunities for regulatory approval of a lonafarnib-based regimen. In parallel, we are preparing a Phase 3 study of Lambda for HDV called LIMT-2 for initiation in the second half of this year. In Phase 2, we previously demonstrated 1 study that 36% of patients who received Lambda for 48 weeks were able to achieve a durable virologic response or HDV RNA below limit of quantification or undetectable at 24 weeks post-treatment. We're excited about these results which form the basis of our discussion with FDA and EMA regarding registration. We have agreement with both agencies on LIMT-2 as a single pivotal study of Lambda for HDV.

Thanks, Ingrid. The press release we issued this afternoon included a financial update which I will summarize. As Eldon noted, we reported $3.6 million in Zokinvy net sales in Q1 after launching commercially in the U.S. in January. Turning to our GAAP operating expenses, cost to goods sold is $53,000 in the quarter, as we had previously spent Zokinvy supply cost R&D. We expect low COGS as the previously expensed inventory is consumed. For first quarter R&D expenses, we're $13.8 million and SG&A expenses totaled $5.6 million. We did report GAAP earnings this quarter of $29.2 million or $0.85 on a fully diluted per share basis as a result of the one-time gain from the sale of our Zokinvy Priority Review Voucher which closed in January. Eiger sold a PRV for $95 million and retained half of the net proceeds, which was recorded to other income on our income statement for this quarter. Finally, we began the year with a strong cash position and ended the quarter with $160.5 million in cash, cash equivalents, and investments. This provides us with runway into Q4 2023, and importantly allows us to complete our Phase 3 HDV studies, D-LIVR and LIMT-2. I'll now hand the call back over to David.

Thanks, Sri. And you heard in our prepared remarks today, Eiger has a diverse and very promising late-stage pipeline targeting rare and ultra-rare diseases with high unmet medical need. We have multiple shots on goal across these programs with potential to deliver a number of value-creating catalysts in 2021 and beyond. This year, we plan to complete enrollment of 400 patients in our HDV Phase 3 D-LIVR study, which is currently 75% enrolled setting the stage for week 48 in the treatment results in 2022. We plan to initiate the Phase 3 LIMT-2 study to advance Lambda as our second therapy toward potential approval for HDV. We will continue the successful U.S. commercial launch of Zokinvy and prepare for EMA approval of Zokinvy for progeria and processing-deficient progeroid laminopathies. We will advance regulatory, manufacturing, and device development activities for avexitide and preserve options to advance clinical development for PBH and CHI. We will advance Lambda for COVID-19 in the ongoing Phase 3 TOGETHER platform study. And importantly, we have the cash needed to support the execution and achievement of all these milestones. At this point, I'd like to thank everyone for joining us today on our first of many quarterly financial results and business update calls, and turn it over to the operator to please provide instructions for the Q&A portion of the call.

Thank you, sir. And, speakers, our first question is from Maury Raycroft of Jefferies. Ma'am your line is open.

Hi, this is Kenny Chan on for Maury Raycroft. For the Phase 3 D-LIVR trial, can you provide some patient background on the enrollment?

Yeah, so a good question and thanks for joining us. The D-LIVR study, as we mentioned, is the largest and only global study of HDV active in over 20 countries now across 100 sites and as we communicated today in our prepared remarks, the study is currently 75% enrolled including patients enrolled and randomized to date as well as patients in screening that are expected to randomize. We haven't provided additional detailed guidance; however, we expect full enrollment of 400 patients by the end of this year, 2021, assuming current conditions hold.

Thanks.

And, speakers, our next question is from Yigal Nochomovitz of Citigroup. Sir, your line is open.

Hi, team, this is Ashiq Mubarack for Yigal. Thanks for taking my question and congrats on the first earnings call. I guess, can you briefly give us a reminder on the choice of primary endpoints between the D-LIVR study and LIMT-2? And maybe on D-LIVR, why you chose the 2 log decline versus some other metric? And then, maybe how you're thinking about the rate of decline with patients especially with the ALT normalization? Thanks.

Yeah, sure, all, and thanks for joining us and for your questions. I'll provide some preliminary comments and then ask Ingrid and also Colin to add any additional color. Importantly, we spent significant time with regulators negotiating endpoints for both the Phase 3 D-LIVR study and the Phase 3 LIMT-2 study for lonafarnib-based regimens and for Lambda, respectively. We believe that the endpoints line quite well or align quite well with delivering patient benefit in terms of reducing viral load and normalizing ALT, a liver enzyme related to liver inflammation, and that's 48-week end of treatment endpoint, which we've been able to demonstrate successfully in our Phase 2 program for lonafarnib. With Lambda, we actually demonstrated in our Phase 2 program a 24-week post-treatment endpoint that is a bit more traditional and something more aligned with previous HCV studies where an off-treatment endpoint by regulators was desired. And so, we were actually very pleased to offer this Phase 3 study design to FDA and EMA, and gain concurrence relatively quickly for a 48-week treatment course with a 24-week post-treatment endpoint of a DVR or Durable Virologic Response, defined as patients being below limit of quantification at 24 weeks post-treatment. And feel very confident that that as well will convert to improved patient outcomes. And I'll make sure I check with Ingrid, and make sure that I've covered it all related to this. Ingrid?

Yeah. And I'll just add that the LIMT-2 study will investigate 48 weeks of weekly Lambda treatment as a finite therapy with a 24-week post-treatment follow-up.

Okay. Okay. Great. And will you also be measuring surface antigen? I don't know if I saw that on the slide.

Yes. In both studies with the D-LIVR study and the LIMT-2 study, we'll be looking at HDV surface antigen.

I'll just add that importantly that these endpoints agreed upon by regulators are a part of the draft guidance for the development of therapeutics for HDV. So, we feel very, very good about the next steps on both the D-LIVR and LIMT-2 studies.

Okay. Awesome. Thank you very much.

And speakers, our next question from Robert Hazlett of BTIG. Sir, your line is open.

Yeah. Thanks. One, and then maybe another one on a second topic, I'll jump in there. Could you be just a little bit more clear, I think, I got the understanding of the - this talking through bottles. But could you just be a little bit more clear of like the exact number of the - the amount of stocking that you think actually occurred in the quarter? That would be helpful. And then I have a follow-up.

Yes, Bert. Good to hear your voice, and thanks for calling in and for your question. I'll turn that directly over to Eldon Mayer, our Chief Commercial Officer. Eldon?

Yes. Hi. So I gave an approximate number of 110, but let me walk you through We shipped 165 bottles to - from our 3PL to our dedicated specialty pharmacy. 53 of those bottles were patient demand. So, that leaves 112 bottles exactly that resided in the channel and I rounded that off to 110, as I mentioned earlier. So, those are the exact numbers. We expect, as I mentioned, roughly 6 to 8 weeks going forward, which translates to approximately 80 to 100 bottles going forward. Hope that answer your question.

Okay. Thanks. And just a quick one on CHI, could you just describe briefly about how the condition presents and when the patients are actually identified, just a little more of the disease itself? Thanks.

Sure, Bert. And thanks for your question. Congenital hyperinsulinism, as you're referring to, is a disorder of newborns, neonates and children, that presents with patients who are hypoglycemic and this is due to hyperinsulinemia. And we strongly believe based on the Phase 2 data generated at CHOP or Children's Hospital of Philadelphia that avexitide represents a targeted mechanism and a potential therapy for neonates and also children with this devastating disease. Importantly, FDA has granted avexitide in congenital hyperinsulinism rare pediatric disease designation, and we look forward to making additional announcements in the future as we advance.

Okay. Thank you. Congratulations on the quarter. Thanks.

And speakers, your next question from Michael Higgins of Ladenburg Thalmann. Sir, your line is open.

Thank you. Thanks, guys. Good to hear your voices again. Congrats on the continued execution despite COVID's challenges. Hoping to get an update from you on the enrollment for HDV, the D-LIVR. You've given us some updates on the patient counts. If you could give us an update on the sites, location, et cetera? I know in 20, you were trying to expand and obviously, COVID hit. Just trying to get some feedback as to how many sites are out there and if you're planning to expand that number? Thanks.

Sure. And good to hear your voice, Michael. Thanks for calling in and for your question. I'll turn that over to Ingrid to address number of sites and how things are progressing. Ingrid?

Hey, Michael, good to hear your voice. Yeah, as you know, the D-LIVR study is a global study. So, we are currently in 22 countries and over 100 sites. The most recent addition in countries is Russia and Ukraine and those have been great contributors to the enrollment.

Yeah. I would just add that we're very pleased with management of the D-LIVR study beginning early in 2020 with the emerging pandemic. Our first goal was patient safety and ensuring drug supply as well as global monitoring. And we did not lose the patients that had already been randomized. And importantly now, with study ramp progressing nicely such that we have provided guidance that we plan to complete enrollment of 400 patients end of 2021.

Yeah. That's fantastic. And then to have data next year as well is great progress and not losing anybody. That's interesting to hear. Follow-up on avexitide was touched on a bit in the Q&A here. I'm just trying to get a better understanding of the gating factors before staring a pivotal study. Is it as mentioned in the press release of more of a manufacturing hurdle or what's next with that? And I guess kind of a quick follow-up would be your partnering plans there. It looks like you're set to run this by yourself, but curious to see or hear your feedback of essential partner for that one. Thanks.

Sure. Thank you for that question. As we guided publicly at the beginning of 2020, we announced that we would seek potential strategic partnering option for avexitide. And during the course of that process, we developed greater conviction around the opportunity for avexitide in 2 different metabolic indications; in post-bariatric hypoglycemia and congenital hyperinsulinism. Importantly, we plan this year to complete manufacturing as well as device development and additional regulatory activities that will put us in a position that both programs; PBH and CHI, could move into registration as early as 2022. And so, that's our plan for 2021. And certainly at that point, we will be able to provide more guidance on the strategic direction for these programs and look forward to it.

One quick follow-up to that if I could and I'll jump back in the queue would be - what can you give us for a potential frame of the cost for something like this? This strikes me as a relatively small study that obviously pretty intensive with the sites and the patients.

Sure. I'll let Sri Ryali to speak to the financial.

Importantly, manufacturing and device development activities that we have initiated are within our cash runway guidance that was funded into Q4 . Any additional studies that would be conducted for CHI or PBH would be smaller studies and we haven't provided financial guidance yet in terms of what the cost of those would be or how we would proceed with that program, we will be able to do so after we get past some of these gating items that David talked about.

I appreciate that. Thanks, guys.

Thank you.

And speakers, we have our next questions from Shveta Dighe of Wedbush Securities. Ma'am your line is open.

Hi, this is Shveta on for Liana Moussatos. Thank you for taking my question. Can you provide some color on the European launch plan for the Zokinvy given that EMA approval is anticipated by year end? And how are you thinking about rest of the world given that about more than 100 patients are in the rest of the world?

Sure. And thank you for joining us today and for question. And I'll begin and then turn it over to Eldon. We - as announced, planned for approval of Zokinvy in EMA later this year. Obviously, the approval in November of 2020 for Zokinvy here in the U.S. for Progeria and progeroid laminopathies allowed us to begin to build infrastructure that will be scalable. And we are looking forward to EMA approval. And with that, I'll ask Eldon to comment a bit on next steps.

Hi, there. So, as been stated, our MAA is under review and with approval expected by year end. And, of course, we have not provided guidance on that yet, but it is - and typically negotiated on a country-by-country basis following approval. And one example, although not in Europe for outside the U.S., is where we have a partnership with Neopharm in Israel and we submitted regulatory application, which has been accepted and approval could come as early as Q4 this year. But setting that aside, we would expect in Europe that in some countries like Germany, reimbursement could be secured upon EMA approval and we're preparing for that. And in other countries though, it could take, as you may know, sometimes up to 1 to 2 years for reimbursement. However, we are exploring opportunities for reimbursement on a country-by-country basis that where there is potential to generate revenues earlier than full reimbursement approval through the various programs that are available. And we've taken a number of steps to prepare for that, conducted payer research. We're establishing our distribution and patient support services engaging with key doctors, and identify other infrastructure such as marketing agency and working with consultants as needed. So, that would be a quick summary. Anything, gentlemen, you want to add?

Yeah. I'll just add to Eldon's comments, to make sure that we address your question about territories and regions beyond the U.S. and Western Europe. There are obviously as we've guided, over 180 patients identified in over 30 countries around the world. And many of these patients lie outside of the Western world. And so, we have announced a partnership with Neopharm to distribute in Israel. And we'll update on that in the near future. And as well, many other countries that we are exploring appropriate pathways to ensure availability and also commercial revenue opportunities. And we'll guide on those region by region, as those become publicly announced and hopefully that helps.

Great, thank you.

Your next question, speakers, from Robert Hazlett of BTIG. Sir, please go ahead.

Hi, Bert, are you still with us?

Catherine, why don't you re-prompt for Q&A. And if there are no more questions, we can move to closing remarks.

Okay, sure, sir. And, sir, we have again, Robert Hazlett of BTIG. Sir, your line is open.

Let me try this and see if it works. Can you hear me?

Yes, very good to hear your voice.

My apologies. Just regarding Avexitide, a brief follow-up, maybe you touched on this, maybe you didn't, but with regard to PBH or the CHI, is there a priority in terms of indications? How should we think about the urgency with which you're going to consider either or both indication there?

Yeah, thanks, Bert. So with regard to the Avexitide program, our primary focus this year is to ensure that we manufacture drug products sufficient for Phase 3 enabling studies, as well as device work for both PBH and CHI indications, as well as any continued regulatory discussions that needs to take place to enable registration studies as early as 2022. Importantly, we view both post bariatric hypoglycemia and congenital hyperinsulinism as major unmet medical needs. And we believe that Avexitide represents a targeted therapeutic for both of these indications. And so, we view them as equally commercially valuable. And importantly, the unmet need within the patient populations is high. And so, we look forward to finding the most appropriate pathway to move Avexitide forward and we'll continue to provide guidance and updates as we have them.

Terrific. We look forward to those. Thank you.

And, speakers, I am not showing any further questions. I would now like to turn the call over back to Mr. Sri Ryali for any further remarks.

Right. Thank you, Catherine. This concludes our call. If you have additional questions, you can contact myself or the info@eigerbio e-mail address to reach out members of management team who are around. Thank you, everyone, for joining us today. I appreciate your time.

Ladies and gentlemen, thank you for participating in today's conference. This concludes today program. You may all disconnect. Everyone, have a great day.