EIGR (2021 - Q2)

Good day, ladies and gentlemen. And welcome to Eiger BioPharmaceutical Second 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. [Operator Instructions] 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. You may begin. Sri Ryal

Good afternoon and thank you for joining us today. Welcome to our quarterly financial results and business update call. We issued a press release earlier this afternoon with our Q2 financial results, which is available on our website at eigerbio.com. For today's call, we will have prepared remarks from the management team followed by Q&A. We will be using slides for the webcast. We will have a replay available on the Investor section of our website. Joining me on today's call are David Cory, our President and CEO; Eldon Mayer, our Chief Commercial Officer; and Dr. Ingrid Choong, Senior Vice President, Clinical Development. Dr. Colin Hislop, Senior Vice President, Clinical and Development Operations, will be available for the Q&A portion of the call. We'd like to remind investors that this call will include forward-looking statements within the meaning of the safe harbor provision of the Private Securities Litigation Reform Act of 1995, including but not limited to the types of statements identified as forward-looking in our 2020 annual report on Form 10-K, our quarterly reports on Form 10-Q, and our subsequent periodic reports filed with the SEC, which will all be available on our website in the Investors section. For a further description of the risks and uncertainties that could cause actual results to differ materially from those expressed in the forward-looking statements as well as the risks related to our business, please see our periodic reports filed with the SEC. These forward-looking statements represent our views only as of the date of this call and involves substantial risks and uncertainties including many that are beyond our control and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligations to update such statements. I'll now turn the call over to David.

Thanks, Sri. Good afternoon, everyone, and thank you for joining us today. We are making great progress across our pipeline, which includes multiple late-stage therapies for rare diseases. We are very proud in Eiger that every one of our rare disease programs now has FDA breakthrough therapy designation. And these include lonafarnib, peginterferon lambda for hepatitis delta virus infection, Avexitide for post-bariatric hypoglycemia and now congenital hyperinsulinism, and Zokinvy for progeria, which is now commercially approved. Eiger is well positioned to advance several catalysts for value creation in 2021 and beyond. We look forward to updating you today on our progress and future plans. Our lead clinical development programs are focused on hepatitis delta virus, the most severe form of human viral hepatitis. Hepatitis delta virus is always a co-infection with HBV and is found in approximately 6% of HBV infected patients. However, hepatitis delta virus causes a much more rapid progression of liver disease than HBV alone. Sadly, 60% of HDV patients die within 10 years after diagnosis. This is a large orphan disease in the US and Europe with an urgent unmet medical need. There is no FDA approved therapy for hepatitis delta virus. Eiger is pioneering the development of treatments in this space. We are often asked, what would a win look like for HDV patients? HDV is a devastating rapidly progressive disease. The first goal of therapy is reduction in HDV viral load and reduction in liver inflammation to slow progression of disease, which has been shown to lead to improved outcomes. Importantly, this is consistent with FDA guidance for development of HDV therapies. Additionally, HDV therapies will require chronic dosing. Convenient administration for long-term treatment could improve compliance and outcomes which are critical to both patients and physicians. We believe that lonafarnib, a daily oral medication, and peginterferon lambda, a weekly subcutaneous injection, offer the most convenient dosing compared to other late stage HDV programs in development. At Eiger, we are advancing an HDV platform strategy that provides multiple pass to win four patients. We have two well-characterized promising late-stage product candidates in lonafarnib and peginterferon lambda. Both have different mechanisms of action, are conveniently administered, and should benefit HDV patients alone and in combinations. Lonafarnib, the only oral treatment in development for HDV, is currently dosing in the Phase 3 D-LIVR study. D-LIVR is the largest global Phase 3 study to be conducted in HDV with over 100 sites across 20 countries. The D-LIVR study design opens the door to deliver a win for HDV patients with the all oral lonafarnib-based regimen and in combination peginterferon alfa. We are pleased to seek continued momentum in D-LIVR, which is now over 90% enrolled. This includes patients randomized to date and patients in screening that are expected to be randomized. We are on track to complete enrollment before end of 2021, setting up for pivotal topline data release in late 2022. Our second therapy in development for HDV is peginterferon lambda, a well-tolerated interferon, which is beginning Phase 3. We have concurrence with FDA and EMA for a single pivotal study called LIMT-2 and we expect to enroll our first patient in this study before the end of the year. LIMT-2 represents an efficient pathway for peginterferon lambda approval in HDV and we are excited to begin this study. For over a decade, Eiger has been engaged in the clinical development of therapies for viral hepatitis. We have gained a deep understanding of the need of HDV patients and a position to care for them. We believe that lonafarnib and peginterferon lambda will become foundational therapies to be used alone and in combination for the treatment of HDV. Eiger is well positioned to be a leader in HDV, a billion-dollar plus commercial market opportunity. Beyond HDV, we continue to advance the rest of our rare disease pipeline. Avexitide is a novel, first in class targeted therapy for two very different orphan metabolic disorders, post-bariatric hypoglycemia, which has been granted FDA breakthrough therapy designation, and congenital hyperinsulinism, which has been granted FDA breakthrough therapy designation and rare pediatric disease designation. We plan to complete manufacturing and device development as well as regulatory activities this year to support registration enabling clinical trials as early as 2022 for both indications. Avexitide represents a significant commercial opportunity and we will provide guidance in the future on how we plan to advance this program. While re remain focused on our mission to developing commercialized targeted therapies for serious rare and ultra rare diseases, we have opportunistically and in a capital efficient manner explored the potential of peginterferon lambda as a convenient outpatient treatment for COVID-19. The placebo-controlled Phase 3 TOGETHER Study is enrolling and dosing patients across multiple clinical sites in Brazil. Positive results from this study could support a submission for Emergency Use Authorization. We look forward to reporting interim utility data analysis potentially by year end. Turning to our first approved product. We were pleased that Zokinvy was the recipient of the 2021 National Organization of Rare Disorders Industry Innovation Award and is now a nominee for the 2021 Prix Galien USA Best Pharmaceutical Agent Award. The US launch of Zokinvy continues to progress very well and we expect EMA approval later this year. Zokinvy has demonstrated our ability to go from I&D to NDA to approval and launch and to successfully navigate complex global regulatory landscapes. Importantly, we have established infrastructure along with commercial and medical affairs functions that can scale and grow for additional commercial launches across larger orphan disease indications in the future. Before turning the call over to Ingrid to discuss our clinical development programs in more detail, I'd like to note that we entered the quarter with approximately 140 million in cash and investments, enough to fund our planned Phase 3 HDV studies and sufficient runway to fund planned operations in the Q4 2023. Ingrid?

Thanks, David. We're making great progress across our clinical development programs. We have deliberately designed and are executing an HDV platform strategy with potential to generate approvals from multiple HDV treatment regimens. Taking a moment to dive deeper into our HDV strategy, as David mentioned, we believe a win for HDV patients are approved therapies that suppress HDV virus, which has been shown to lead to improved hepatic function, improved liver histology, and improved survival. We also believe that for chronic HDV therapies, convenient administration as well as antiviral activity are important considerations for patients and their physicians. Lonafarnib and peginterferon lambda potentially offer both. In Phase 2, the all oral regimen of lonafarnib 50 milligrams b.i.d. with ritonavir achieved a composite endpoint of a two log decline in HDV-RNA and normalization of liver enzymes or ALT in 29% of patients at week 24 of treatment. When lonafarnib was combined with peginterferon alfa, the response rate more than doubled to 63%. Importantly, the combination of lonafarnib and peginterferon alfa was synergistic on the composite endpoint and proving both reduction in HDV viral load as well as ALT normalization. These data was the basis for our Phase 3 D-LIVR study design. D-LIVR is a foundational global Phase 3 trial that we believe positions Eiger to be a leader in HDV. The D-LIVR primary endpoint is a composite of a two log decline in HDV-RNA plus ALT normalization as was demonstrated in Phase 2. The D-LIVR study design creates two opportunities for regulatory approval of lonafarnib-based regimens, an all oral and a combination with peginterferon alfa. In addition, D-LIVR is the only HDV study requiring baseline and end of treatment biopsies, which will support key secondary endpoints examining histologic improvement. Biopsies have been shown to be significantly more reliable than fibroscans in the assessing liver inflammation. As David mentioned, the Phase 3 D-LIVR study is now over 90% enrolled. This puts us on track to report topline data in late 2022. When complete, D-LIVR will provide the largest global safety database from a single HDV study and a wealth of data to inform and drive Eiger's HDV platform strategy. We are equally excited to advance our second therapy in development for HDV. Peginterferon lambda is a first in class Type 3 well-tolerated interferon, now in Phase 3 in LIMT-2 study. In Phase 2, peginterferon lambda was dosed once weekly in HDV-infected patients for 48 weeks with 24-week follow up. Thirty six percent of patients who received peginterferon lambda achieved a durable virologic response or DVR, which is HDV-RNA below limit of quantification or undetectable at 24-weeks post treatment. This post-treatment DVR endpoint is most meaningful for both regulatory agencies and physicians as it demonstrates durability of response to treatment for HDV patients. LIMT-2 is a randomized two-arm study. Arm one is 48 weeks of peginterferon lambda once weekly treatment followed by 24 weeks off treatment. Arm two is four weeks of no treatment. The primary endpoint is a proportion of patients with a durable virologic response at 24-weeks post-treatment in arm one compared to 12-weeks of no treatment in arm two. This is a very straightforward study of 150 patients that we anticipate will enroll across 40 to 50 global sites. LIMT-2 sites will be primarily selected from the best performing sites of the D-LIVR study, which should allow us to enroll quickly and efficiently. We have agreement with EMA and FDA on LIMT-2 of a single pivotal study of peginterferon lambda for HDV and on track to enroll our first patient by end of year. We believe LIMT-2 is the most expeditious pathway for peginterferon lambda approval in HDV. A successful outcome in LIMT-2 could lead to approval of peginterferon lambda as a monotherapy for HDV and open the door for it to become the interferon of choice in combination therapies for the treatment of HDV. We believe that peginterferon lambda tolerability profile will be preferred by physicians and patients leading to better compliance and improved outcomes. Lonafarnib and peginterferon lambda have distinct and complimentary mechanism that can be used alone, in combination with each other, and in combination with other HDV regimens to suppress virus, reduce liver inflammation, and improve outcomes. Ultimately, our goal will be complete suppression of HDV virus and HDV cure. To that end, the combination of peginterferon lambda and lonafarnib with ritonavir achieved the most robust antiviral effect further demonstrating the opportunity for this combination in the future. I'll now turn the call over to Eldon for our commercial update.

Thanks, Ingrid. I'm pleased to provide an update on our commercial progress and the Zokinvy launch. Our initial efforts have been focused on the approximately 20 identified patients in the U.S. where we launched in January. In anticipation of EMA approval later this year, we're actively preparing for launch in Europe where there are also approximately 20 identified patients. As David noted, the U.S. Zokinvy launch continues to progress very well. We reported 2.1 million in Zokinvy net sales in Q2, bringing our year-to-date net sales to 5.7 million. Importantly, we've converted the vast majority of the 20 identified U.S. patients to commercial supply. The patient support center we established, known as Eiger OneCare, has facilitated an efficient U.S. launch. The primary goal of Eiger OneCare is unencumbered patient access to Zokinvy, and this program has delivered continuous access with zero out-of-pocket cost for all patients. In Eiger, our commitment is to provide Zokinvy access for every child and young adult with progeria and processing-deficient progeroid laminopathies. Ultimately, our goal is to deliver global commercial launches across our late-stage rare disease programs. Our execution is the Zokinvy launch has enabled us to serve the progeria community while establishing a commercial distribution network, patient support services, and payor engagement that will be scalable for future HDV and other larger orphan indications. And with that, I'll hand the call over to Sri for our financial update.

Thanks, Eldon. The press release we've issued this afternoon included a financial update. I'll call out a few of the highlights. As Eldon noted, Q2 Zokinvy net sales were $2.1 million, in comparison to $3.6 million reported in the first quarter, which included an initial channel inventory at our specialty pharmacy after we commercially launched in the U.S. in January this year. The contribution from Zokinvy will help offset expenses as we advance the rest of our pipeline. Turning to our GAAP operating expenses, cost of goods sold is approximately $0.3 million in the quarter. The second quarter R&D expenses were $14.3 million and SG&A expenses total $5.9 million. We did report a net loss of $19.2 million or $0.57 on a per share basis. Finally, we continue to operate with a strong cash position and ended the quarter with $139.8 million in cash, cash equivalent, and investments. This provides us with the runway into Q4 2023 fully funds are ongoing Phase 3 HDV studies, as well as the activities required to enable Phase 3 Avexitide studies as early as 2022. I will now hand the call back to David for closing comments.

Thank you, Sri. As you've head in our prepared remarks today, Eiger continues to successfully execute our strategy and deliver on our pipeline commitments across the board. Our HDV platform is advancing with multiple opportunities to win for patients chronically infected with hepatitis delta virus infection. The Phase 3 D-LIVR study is over 90% enrolled. Full enrollment is expected before the end of 2021 and release of topline data is expected in late 2022. The Phase 3 LIMT-2 study is on track for first patient enrolled by yearend. We're planning for Avexitide to be Phase 3 ready for post-bariatric hypoglyecemia and congenital hyperinsulinism as early as 2022. We expect EMA approval of Zokinvy before the end of the year, building on a successful U.S. commercial launch. Importantly, we have the cash necessary to support the execution and achievement of these milestones. With our pipeline of multiple breakthrough therapy designated programs rapidly advancing, we look forward to several investor updates this year, including participation in six upcoming banking and investor conferences, attendance at AASLV [ph] in November including a planned Eiger-sponsored HDV focused KOL investor event, future earnings calls, and continuous communication efforts on our progress. At this point, I'd like to acknowledge the Eiger team for their relentless efforts across our programs and thank all of you for joining us on our Q2 financial results and business update call. Operator, at this time, please provide instructions for the Q&A portion of the call.

Thank you. [Operator Instructions] Our first question comes from the line of Maury Raycroft of Jefferies. Your line is now open. You may ask your question. Again, Ms. Raycroft?

Hi. This is Kenny Chan on for Maury Raycroft. So, I have one question regarding the HDV program. What are your thoughts on Gilead's bulevirtide 301 data from IZO [ph] and how does it compared to lona monotherapy in terms of HDV and the composite HDV plus ALT normalization endpoints? And are there any implications for bulevirtide combo therapy? Thanks.

Thanks so much for your question. I'll ask Ingrid Choong to address those.

Thanks, Kenny. Yes, our Phase 2 lonafarnib-ritonavir all oral achieved a 30% composite endpoint after 24 weeks of therapy. At 24 weeks, bulevirtide monotherapy, which is a daily subcutaneous injection, achieved a 30% composite endpoint as well. When lonafarnib-ritonavir was combined with peginterferon alfa 60% of patients achieved the composite endpoint after 24 weeks of therapy. Bulevirtide combined with peginterferon alfa showed a composite response rate of 30%. So, we believe that our Phase 2 data suggest that lonafarnib with ritonavir in combination with peginterferon alfa had a synergistic impact on the FDA guided composite endpoint on the reduction in both the viral load and the liver inflammation. And we look forward to deliver top link 48 weeks data in late 2022.

I'll just add to answer the second part of your question. Given the fact that lonafarnib and pegylated interferon lambda work by different mechanisms to fight HVD viral infection. We believe that there is an opportunity for combination therapies in the future as Ingrid outlined in her prepared remarks. To answer your question with regard to potential combinations with other agents with different mechanisms, investigators and key opinion leaders are already talking about cocktail options for the future to drive toward a cure and we look forward to providing more updates as those studies actually are initiated and data readout.

Thanks.

Thank you. We have next question comes from the line of Yigal Nochomovitz from Citigroup. Your line is now open. You may ask your question.

Hi, team. This is Ashiq Mubarack on for Yigal and thanks for taking my question. I guess the first one will be on the D-LIVR readout next year. Can you talk a little bit about how or to what extent you will release the data? I just want to confirm there isn't going to be an interim look ahead of the sort end 48-week data point. And maybe at what point after the D-LIVR study is over will you start engaging with the FDA about a potential filing? Thanks.

Sure. I'll turn that one over to Ingrid as well. Ingrid?

Sure. So, as we said in our prepared remarks, the timing of the D-LIVR readout is at 48 weeks after the last patient has received last dose. So, this is planned for the end of 2022. We are not planning for any interim readouts, so the topline data will be at the end of next year. And with that, once we've cleaned and locked the data -- reported out the data, we will discuss next steps as to when we would go to FDA.

Okay. Got it. And maybe a quick one on Avexitide. Sounds like you've been engaging with the FDA on both post-bariatric hypoglycemia and congenital hyperinsulinism. I guess, when can we expect some details on what those studies might look like and maybe how long it will take to run, et cetera?

Sure. So, I'll provide thoughts and if Sri would like to add any additional thoughts I'll turn it over to him. We believe that post-bariatric hypoglycemia indication as well as congenital hyperinsulinism are both significant unmet medical needs. And more and more, we view Avexitide as a significant commercial opportunity at Eiger for both of these indications. We were obviously very pleased to announce that congenital hyperinsulinism as an indication for Avexitide has been granted breakthrough therapy designation and that pairs with the Avexitide post-bariatric hypoglycemia indication already having obtained breakthrough therapy designation. This year, we are focused on completing manufacturing, doing all the right modification. And for the BTD or breakthrough announcement today, we are in active discussions with regulators about next steps for Phase 3 study design for both PDH and congenital hyperinsulinism and we'll definitely provide additional guidance as it becomes available.

Okay. Thank you very much.

Next question we have from the line of Brian Skorney of Baird. Your line is now open. You may ask your question.

Hi. This is Jack Allen dialing in for Brian. Congrats on the progress this quarter and thank you for taking our questions. Our question is mainly focused on Avexitide as well being so timely with the breakthrough therapy designation being granted this morning. We are hoping that you'll provide a little bit more context around the congenital hyperinsulinism disorder and the current treatment landscape in the space. And then -- I know you just touched on this -- but any other additional color you could provide with respect to the timing of [indiscernible] would be greatly appreciated. Thank you.

Sure. Ingrid, would you like to comment?

Yes. So, congenital hyperinsulinism is a ultra rare disease, 1 in 5,000 patients, and it's a disorder that if left untreated results in brain damage in 50% of patients. And so, what Avexitide offers is a top -- targeted therapy to block GLP1 as a GLP1 antagonist to alleviate these hyperinsulinemic episodes.

Yes. And I would just add to that as you're probably aware that congenital hyperinsulinism space in terms of therapeutics targeting this indication has grown in the last 12 to 24 months, heightening not only our awareness of this disorder and that the unmet medical need. But I think of the medical community at large, which makes us even more excited today to announce breakthrough therapy designation having been granted from FDA. And as you probably know, again, there are now multiple therapeutics in clinical phases of development importantly, but none of them have the targeted mechanism of blocking GLP1, which we believe is a necessary approach and mechanism towards treating this very, very difficult disorder. And so, we'll provide update as we move forward obviously with regulatory announcements in terms of opportunities moving forward.

Thank you so much for the color.

Thank you. Next question comes from the line of Michael Higgins from Ladenburg Thalmann. Your line is now open. You may ask your question.

Thanks, operator, and congrats, guys on the continued successes including the breakthrough progress [ph] and Avexitide. A couple of questions if I could start off here with HDV, your primary programs. If you can confirm for us any material changes in the design and or execution of those sites globally, including any differences in the pre-treatment or prior treatment differences across the globe. And then as you're getting ready to get through the start of LIMT-2, any potential to be cannibalizing D-LIVR with the enrollments of the two trials? Thanks

Sure. I'll turn that one over to Ingrid.

Michael, thanks for your question. For your first question regarding study design for D-LIVR, there's been no material changes from what we've discussed in the past. We've been very consistent as far. And I think your second - your - the follow-on to your first question was about patient demographics across all sites. And so far, our understanding is that patient demographics is balance pretty evenly across all sites.

And I'll just add to the third part of your question, Michael, which was are we concerned about LIMT-2 patient cannibalizing D-LIVR patients. There's -- the reality is we have staged the nature of the D-LIVR study competing enrollment and the wrap up of the LIMT-2 study really well, such that we plan to complete enrollment of 400 patients within 2021 literally as targeted sites are ramping up to begin enrolling patients in the lambda LIMT-2 study. And so, we do not see cannibalism of patients out of D-LIVR by the LIMT-2 study. Quite the opposite, we are opportunistically able to select the best performing D-LIVR sites that will be a part of the LIMT-2 study, which we think will allow us to quickly and efficiently enroll 150 patients in the lambda LIMT-2 trial to get that study completed as quickly as possible.

Thanks, David. That's really helpful. Just a follow up to that then. Typically, there's a hockey stick pattern with prior enrollment. Is there a way for that to bleed over into LIMT-2s enrollment such that the sites are up and going as identified patients and whether the design could be different such that it's kind of a restart for LIMT-2?

Yes. That is a great question. And I will respond and the group can add color if they like. The good news with hepatitis delta virus infection is that there are multiple treatments now in the clinic and awareness is growing day by day. And with that, interest in presenting studies to patients who are diagnosed is increasing. And that is allowing forth increasing opportunities to enroll patients in clinical trials, which as you appreciate, are critical for us to move both lonafarnib and lambda towards registration. And so, we're very excited actually to report that awareness of hepatitis delta is growing. And this is good for patients because more is being identified and diagnosed that ultimately we believe will get into clinical trials that we can then treat with commercial products in the future.

And I'll just add to that, Michael, that physicians and patients are very excited about a well-tolerated interferon for the treatment of HDV. And so, there's a lot of excitement and enthusiasm ahead of us starting to enroll the LIMT-2 study.

Makes sense. Just one last follow up on that. Are you comfortable with roughly a year and half to enroll LIMT-2 considering what you just said about patients coming in and kind of having that base available? Any comments on that, time in general, will be super helpful. Thanks.

Hey, Michael. It's Sri. We haven't provided specific guidance yet on enrollment limits [ph]. As Ingrid said, it's 150 patients, so it's been half the size of D-LIVR and we'll be selecting the best performing site to D-LIVR. So, we think we've got some good line of sight as if where we're going to get to these patients, so we're not providing specific guidance yet in terms of the timing of enrollment.

I appreciate all of the time and the answers. Thanks, guys. And congrats to you.

Thank you.

Thank you. Next question comes from the line of Bert Hazlett of BTIG. Your line is now open. You may ask your question.

Thanks. Congratulations on the progress and the breakthrough designation. Just two questions. One, with regard to EMA and the approval, should we be thinking about the same type of label that we got in the U.S. kind of with broader progeria-like disorders? And then when should we actually be thinking about revenue with regard to that? And then secondly, I have a follow on congenital hyperinsulinism.

Sure. I'll turn your question Zokinvy progress in Europe to Eldon.

Yes. Hi, there. So, we have not stated anything about the label that we expect at this point. And I think for that question, it's really uncertain until we have the final labels. I think we'd like to provide an update when we do get that later on - later this year.

But we are - as mentioned in the prepared comments, we are tracking, we believe, toward approval for Zokinvy in Europe by year end. And so, we're obviously anxiously anticipating and looking forward to providing an update as soon as possible.

Okay. Terrific. And then with regard to congenital hyperinsulinism, could you just provide a little bit more color as to what really - what types of the data that you accrued and based to probe the breakthrough designation? Was it a particular patient group, the neonates or others? Just a little more color on your interaction that really drove the breakthrough designation for that program.

Sure. I'll let Ingrid provide some commentary and then maybe I'd color after.

Yes. So, for the breakthrough designation, we shared with the agency all available data that had been generated in congenital hyperinsulinism with Avexitide and that included three studies, one in neonates, one in children, and one in adolescence for total of 39 patients dose with Avexitide.

Yes. I'll just, Bert, and thank you for your question. The Phase 2 studies, as Ingrid mentioned, provided, we believe, very solid proof of concepts. And when I say we, I speak to the investigators at CHOP or Children's Hospital of Philadelphia who demonstrated POC across 39 patients in three different Phase 2 studies. And to further answer your question, those included neonates, children and adolescence with congenital hyperinsulinism. And that data was the basis for Avexitide being granted breakthrough therapy designation by FDA. We think that this is an unmet medical need that the metabolic group at FDA is very much focused on moving forward with therapeutics to help this patient population. And we're very excited to have such a positive dialogue with the agency and look forward to providing updates as we move forward.

So, then the - just - I know this question was asked, maybe in slightly different terms earlier, but in terms of the scale of next steps and the interaction with agency given the breakthrough designation, how large do you think can a confirmatory work might need to be for licensure in the US?

Yes. That's a fair question, Bert. And while we're continuing to have active and ongoing discussions with the agency, what we have commented publicly on is that the size for the neonate and child studies in congenital hyperinsulinism for other therapeutics have been in the range of 40 to 50 patients. So a very focused program with - especially with an active and effective therapy we believe may lead in a similar direction. But obviously, we'll provide guidance as we have that from FDA.

Okay. Thank you. I'll get back in the queue. Thanks.

Thank you.

Thank you. [Operator Instructions] I'm not showing any further questions. I would now like to turn the call over to Sri Ryali for any further remarks.

Well, thank you, Mel. This concludes our call. If you have additional questions, please contact us at info@eigerbio.com or you can reach out to a member of the management team. Thanks everyone for joining us today.

Thank you. Ladies and gentlemen, this concludes today's conference call. Thank you all for participating. You may now disconnect.