Q3 2021 Eiger BioPharmaceuticals Inc Earnings Call
Later, we will conduct a question and answer session and instructions will follow at that time, if anyone should require operator assistance. Please press Star then zero on your telephone.
This call will be recorded I would now like to introduce your host for today Mr.
Mr Qi, Li Chief Financial Officer you.
You may begin sir.
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 Q3 financial results.
On our website at IR bio dot 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 and we'll have a replay available on the investors section of our website.
Joining me on the call are David Cory President and CEO, Eldon Mayer, our Chief commercial officer, and Doctor Ingrid Chung Senior Vice President of clinical development, Dr. Colin Hislop Senior Vice President of clinical and development operations will join us for the Q&A portion.
I would like to remind investors that this call will include forward looking statements, including expectations concerning financial performance commercial products and potential future products in different therapeutic areas and stages of development.
The forward looking statements rely on certain assumptions and involve risks and uncertainties beyond <unk> control, which could cause our actual results to differ materially.
Description of these risks and uncertainties and uncertainties is contained in <unk> filings with the SEC, including our latest 10-K and 10-Q reports available on the IR website in the investors section.
All forward looking statements are based on information currently available to either and we assume no obligation to update these statements I'll now turn the call over to David.
Thanks, Sri and good afternoon, and thank you for joining us today at <unk>, we're focused on the development of innovative therapies to treat and cure hepatitis Delta virus infection and other serious rare diseases.
We continue to make great progress across our pipeline of multiple late stage therapies.
All five of our rare disease programs have FDA breakthrough therapy designation low enough on it and peg interferon Lambda for hepatitis Delta virus of exit tied for post bariatric hypoglycemia, and congenital hyperinsulinism and Zoe can be port progeria. This demonstrates a core strength of <unk>.
Our proven ability to advanced targeted therapies for serious rare and ultra rare diseases with urgent unmet medical needs Iger is well positioned to advance several near term value creating catalysts.
Our lead clinical programs are focused on hepatitis Delta virus infection. The most severe form of human viral hepatitis.
Hepatitis Delta virus is always at co infection with HBV and has found an approximately 6% of HBV infected patients. However, hepatitis delta virus causes a much more rapid progression of liver disease, and HBV alone, 60% of HDD patients die within 10 years after diagnosis.
This is a large orphan disease in the U S and Europe with an urgent unmet medical need.
There is no FDA approved therapy for HDD Iger is pioneering the development of treatments in this space as we discussed before we're often asked what would a win looked like for HDD patients HDD is a devastating rapidly progressive disease. The first goal of therapy.
Is reduction in HDD viral load and reduction in liver inflammation to slow disease progression, which has been shown to lead to improved outcomes. Importantly, this is consistent with FDA guidance for the development of HDD therapies.
And iger, we're advancing in HDD platform strategy that provides multiple pathways to win for patients. We have two well characterized promising late stage product candidates in loan of farnell and peg interferon Lambda both have different mechanisms of action are conveniently administered and should benefit.
Fit HDD patients alone and in combinations.
<unk> is the only oral treatment in development for HDD is currently dosing in the phase III deliver study with over 100 sites across 20 countries deliver is the largest phase III study to be conducted in HDD. The deliver study design opens the door to deliver a win for HDD.
Patients with the all oral lunar farnan based regimen and in combination with peg interferon Alpha.
We were very pleased to announce earlier this week that deliver is now fully enrolled with over 400 patients exceeding our target. This is an important milestone for iger and for HDD patients complete enrollment of deliver now sets up pivotal topline data released by the end of 2022.
Like to thank the patients and investigators and their teams for their ongoing participation and deliver as well as the <unk> clinical team and their steadfast efforts to fully enroll deliver we look forward to reporting data from this landmark study.
Our second therapy in development for HDD as peg interferon Lambda, a well tolerated interferon, which is now in a pivotal phase III study called limit to limit two represents an efficient pathway for peg interferon Lambda approval in HDD and we are now activating clinical trial sites and screening page.
<unk>.
For well over a decade iger has been leading the way in the clinical development of therapies for HDD. We have gained a deep understanding of the needs of HDD patients and the physicians who care for them, we believe that <unk> and peg interferon Lambda will have the potential to become foundational therapies for the treatment of HDD.
<unk> is well positioned to be a leader in HDD, a commercial opportunity projected to be in excess of $1 billion.
Beyond HDD, we're advancing the rest of our rare disease pipeline.
<unk> is a novel first in class targeted therapy for two very different orphan metabolic disorders post bariatric hypoglycemia and congenital hyperinsulinism, we're on track to complete regulatory activities this year and advanced manufacturing necessary to support registration, enabling clinical trials for both <unk>.
Indications, we view of Exenatide as a significant commercial opportunity and we will provide guidance in the future on how we plan to advance this program.
While we remain focused on our mission to develop innovative therapies to treat and cure HDD and other serious rare diseases, we have opportunistically and in a capital efficient manner explored the potential of peg interferon Lambda as a convenient outpatient treatment for COVID-19 the.
The placebo controlled phase III together study is enrolling and dosing patients across multiple clinical sites in Brazil. We are we're pleased to announce in September that the independent data safety monitoring board recommended continuation of Peg interferon Lambda and we look forward to reporting the next interim futility analysis before the.
End of this year.
Interferon Lambda is now the only investigational agent and that together study and positive results from this study could support a submission for emergency use authorization.
Resistance due to variance or new strains of Sars Covid two continue to be an ongoing concern with approved vaccines and monoclonal antibody treatments as well as oral that are currently in development Peg interferon Lambda is mechanism of action of stimulating the host immune response is agnostic to a rising variance and.
As such we believe may be ideally suited to treat newly diagnosed COVID-19, outpatient as a convenient single subcutaneous injection.
Now turning to <unk>.
We are proud at iger that our first approved product is for the treatment of progeria, a devastating universally fatal ultra rare pediatric disease, a premature aging.
The U S launch of Zoe can be has progressed well we're in discussions with the EMA regarding our MAA and expect an opinion around the end of this year. We've also received approval of a cohort <unk> program in France. This is an important achievement and will help bring Zoe can be to French patients with progeria.
Ingrid and the Eldon will provide additional details on our progress in Europe and with the commercial launch.
<unk> has demonstrated our ability to go from IND to NDA to approval and launch and to successfully navigate complex global regulatory landscapes, we've established important infrastructure, along with commercial and medical affairs functions that can scale and grow for additional commercial launches across larger orphan disease indications.
Patients in the future <unk>.
Finally, we ended the quarter with $120 million in cash and investments, which should fund our planned operations into Q4 2023, I'll now turn the call over to Ingrid to discuss our clinical development programs in more detail Ingrid.
Thanks, David we're making great progress across our clinical development program.
Liberally designed and are executing an HDD platform strategy with potential to generate approval from multiple HDD treatment regimens.
But deliver now fully enrolled and limit two underway iqos poised for important upcoming value creating milestone.
We've spoken in the past at length about our HDD strategy and given the scope and breadth of our program. It is worth recapping. We believe a win for HDD patients are approved therapies that suppressed HDD virus, which has been shown to lead to improved hepatic function improved liver histology and improved survival.
We also believe that for chronic HDD therapies convenient administration as well as anti viral activity are important considerations for patients and their physicians <unk>.
<unk> antagonist that lambda potentially offer both.
And phase II, the all oral regimen of loaner foreigners 50 milligrams PID with recon of ear achieved a composite endpoint. They two log decline in HDD, RNA and normalization of liver enzyme or a L. P and 29% of patients at week 24 of treatment.
<unk> partnered with combined with Peg interferon Alpha the response rate more than doubled to 63%.
Importantly, the combination of <unk> and peg interferon alpha with synergistic on the composite endpoint improving both reduction in HDD violeta as well as L. P. A normalization.
Upon a boosted with recon of here is the only therapy in development for HDD that has reported synergy on the composite endpoint when combined with peg interferon Alpha.
Our phase two data were the basis for our phase III deliver study deliver they foundational global phase III trial that we believe positions either it could be a leader in HDD the.
To deliver our primary endpoint is a composite of a too long decline in HDD, Arnie and Alps, and normalization as was demonstrated in phase III the.
But deliver study design create two opportunities for regulatory approval of <unk> based regimens and all oral in a combination with peg interferon alpha.
With that lever now fully enrolled we anticipate topline data by the end of 2022, which if positive will support global regulatory filings for lona foreigners seeking approval of two loan of Barnett based regimens for HDD.
When complete deliver will generate the single largest source of HDD patient data from a well controlled global phase III study to better characterize and understanding this devastating disease.
We're also excited to advance our second therapy for HDD Peg interferon Lambda is a first in class type three well tolerated interferon now into phase III limit to study.
And phase two peg interferon Lambda was dosed once weekly in HDD infective patients for 48 weeks of 24 week follow up.
36% of patients could receive peg interferon Lambda achieved a durable virologic response or D. V are defined as HDD RNA below the limit of quantitation or undetectable at 24 weeks post treatment.
Post treatment DVR endpoint is most meaningful for both regulatory agencies and physicians as it demonstrates the durability of response to treatment and potential for an HDD cure.
The phase III limit to study is randomized two arm study arm. One is 48 weeks of peg interferon Lambda Once weekly followed by 24 weeks of treatment arm to start with 12 weeks of no treatment followed by 48 weeks of treatment.
The primary endpoint is the proportion of patients with a durable Virologic response at 24 weeks post treatment in arm, one compared to 12 weeks with no treatment in arm two.
This is a very straightforward study of 150 patients where all patients will receive treatment.
We have started screening patients and are in process of activating approximately 50 sites across 13 countries.
These sites have been primarily selected from the best performing sites and deliver study, which should allow us to enroll quickly and efficiently.
A successful outcome and limit two could lead to approval of peg interferon Lambda as a mono therapy for HDD and opened the door for it to become the interferon of choice and combination therapies for the treatment of HDD.
We believe that peg interferon Lambda tolerability profile will be preferred by physicians and patients leading to better compliance and improve outcomes.
One up on up on Peg interferon Lambda have distinct and complementary mechanism that can be used alone in combination with each other and in combination with other HDD regimens to suppress virus reduced liver inflammation and improve outcomes.
Ultimately our goal is complete suppression of HDD virus and HDD chore.
That and the combination of peg interferon Lambda in Buena final per ton of beer has achieved the most robust antiviral effect. After 24 weeks of therapy further demonstrating the opportunity for this combination.
Turning to COVID-19, and the phase III together study we were pleased to report in September that the independent data safety monitoring board recommended continuing the peg interferon Lambda arm without any changes to study.
The per protocol interim futility analysis completed in mid September was based on a sample size of 453 patients randomized one to one active treatment to placebo.
As David noted Peg interferon Lambda is now the only remaining investigational agent and together the peg interferon Lambda arm targets enrollment of up to 800 patients at high risk for developing complications from progression of COVID-19.
The primary endpoint is a clinical outcome, comparing an emergency setting visits and hospitalizations and peg interferon lambda treatment versus placebo.
Look forward to reporting additional results in the future.
Finally, turning to our European marketing authorization application forms that can be for progeria, we've been engaged with the EMA over the past 18 months on our MAA.
Discussions with EMA have been focused on additional statistical analysis of our data to support the survival benefits demonstrated across a combined analysis of two different clinical trials.
Patients treated with some can be achieved a 60% reduction in mortality and a statistically significant survival benefit of at least two and a half years.
We believe the data are robust as indicated by our FDA approval last year.
Our discussions with EMA are ongoing and will likely continue through the end of the year around which time, we expect a C. H M P opinion.
I'll now turn the call over to Eldon for a commercial update.
I am pleased to provide an update on our commercial progress and that can be launch. Our initial efforts have been focused on the approximately 20 identified patients in the U S where we launched in January as David noted the U S. It can be launch has progressed well, we reported $3 million as it can be net sales in Q3, bringing our year to date.
Net sales to $8 8 million importantly, we've converted approximately 80% of the identified U S patients to commercially reimbursed supply.
The patient support center, we establish known as either one care has facilitated inefficient U S launch the primary goal of either one carrier's unencumbered patient access to the Kennedy and this program has delivered continuous access with zero out of pocket costs for all patients.
In anticipation of an approval in Europe, where there are also approximately 20 identified patients we're actively planning for launch.
We are engaged with a distributor and patient support services provider as well as local regulatory and reimbursement authorities.
We recently received approval for a cohort <unk> program or temporary use authorization program from French regulatory authorities and it just made our first shipments of the Kinsey under this program.
As a reserve for products, whose FC efficacy and safety are strongly presumed based on clinical trial data and whose therapeutic indication targets, a serious rare or disabling disease locking appropriate treatment in France pharmaceutical products, not yet granted marketing authorization and not recruiting for a clinical trial.
Can be used if the national agencies agency for medicines and health products safety for ASN authorizes an HBU.
Our commitment is to provide that can be accessed for every child and young adults with progeria and processing deficient progeria monopolies ultimately our goal is to deliver global commercial launches across our late stage rare disease programs. Our execution of this it can be launched has enabled us to serve the community.
While establishing a commercial distribution network patient support services and payer engagement there'll be scalable for future HDD and other larger orphan indications.
With that I'll hand, the call to <unk> for a financial update.
Thanks, Sheldon the press release, we issued this afternoon included a financial update and I'll call out a few highlights as Eldon noted Q3 13 net sales were $3 million. This.
Compares to $2 $1 million reported in the second quarter reflects a modest increase in inventory on hand at our specialty pharmacy. The contribution from there can be helped to offset expenses as we advance the rest of our pipeline.
Turning to our GAAP operating expenses cost of goods sold was approximately <unk> 3 million in the quarter for third quarter R&D expenses were $18 1 million and SG&A expenses totaled $6 $5 million.
Reported net loss of $22 2 million or <unk> 65 on a per share basis.
Finally, we continue to operate with a strong cash position and ended the quarter with $124 million in cash cash equivalents and investments is provides us with runway into Q4 2023 and funds our ongoing phase III HDD studies I'll hand, the call back to David now for closing comments.
Thanks, Sri we're executing on our goals across all of our programs and we are well positioned to finish 2021 strong setting up a pivotal 2022, our HDD platform is advancing with multiple opportunities to win for patients chronically infected with hepatitis Delta virus infection complete enrollment.
Of deliver puts us on course for topline data by the end of 2022, the phase III limit to study is screening patients and activating sites. We look forward to participating at <unk> next week and plan to host an investor key opinion leader event focused on HDD.
We are planning for <unk> to be phase III ready for post bariatric hypoglycemia and congenital hyperinsulinism as early as 2022, we expect <unk> opinion on our MAA for Zoe can be around the end of the year and finally, we look forward to additional interim futility analysis and final results from the.
Peg interferon Lambda COVID-19, together study, which if positive could support an emergency use authorization application.
At this point I'd like to acknowledge the <unk> team for their relentless efforts across our programs and thank all of you for joining us today on our Q3 call. Operator, please provide instructions for the QA portion of the call.
Thank you, Sir ladies and gentlemen, if you have a question at this time. Please press Star then the number one key on your thoughts stone telephone.
<unk> has been answered or you wish they moved ourselves from the queue. Please press the pound key.
We ask that you please limit yourself to one question and one follow up.
Our first question comes from the line.
<unk> has dropped from <unk>. Your line is open Healy asked your question.
Thank you. Thank you for holding the call. Thank you for taking my question and congratulations on all the progress you've made.
Two.
One with the two component with regard to the.
The study's upcoming in ft.
What is your expectation for the placebo.
<unk> rate for the loan of foreign or deliver trial and then really the same question for the peg interferon Lambda trial the limit to trial there.
And then I have a follow up on another topic.
Sure. So just to end thanks, Barry good to hear your voice. So just to restate. The question Youre looking at what we've contemplated or publicly communicated regarding expectations for placebo rate on both the deliver phase III study as well as the limit to phase III study and ill, let Ingrid cake.
I'll take those questions yeah, So what I'll say Bart nice to speak to you again as both deliver delivering the limits to study our sites for approval.
Pivotal trials.
And.
Untreated ACB patients with no treatment are not expected to spontaneously clear virus indicate the deliberate and they are not anticipated to achieve the two log decline in A&P normalization and certainly with no treatments are not expected to <unk> undetectable.
Yes, so I would just add to that bird that our expectation, although we havent communicated any specific numbers.
Is that the.
<unk>.
Two log decline in HDD RNA combined with ALG normalization again, our primary endpoint and deliver as a composite.
You don't see that very often happen without treatment and then limit to a very different endpoint with a DVR a durable virologic response, and you don't see HDD virus spontaneously clearing most key opinion leaders would tell you ever.
Without treatment.
Terrific look forward to more conversation about that.
And a week or so and then just one with regard to Zoe can be nice to see the progress there in France. When do you actually think we might be able to see revenue coming from the European.
Geographies.
Sure sure when do we expect to see revenue coming from European geographies and I'll, let our chief commercial officer handle that Eldon Mayer yes.
And as you probably heard on the call. We have just completed our first shipment of drug to Europe for the French Preapproval program the cohort to use so.
That would be the beginning of that commercialization beyond that.
Have not yet provided guidance on European revenue or reimbursement, which.
As you probably know it's typically negotiated on a country by country basis after regulatory approval.
But we do expect EMA opinion by the end of this year and we will be able to provide further information at that point.
Okay very much looking forward to more information there. Thank you.
Thank you Sir we have another question from the line of he got a national mortgage from Citi. Your line is open.
Alright. This is Oscar Mavarach on for Yigal. Thanks for taking my question I just wanted to go back to the point.
You can launch in Europe.
Are you expecting from the CHP.
Might be of interest.
And.
I know you've mentioned that there are 23 identified patients in Europe, just wondering how many are in France.
Maybe any other details on some of the other <unk>.
Specific numbers thanks.
Sure and thanks, good to hear from you and we will answer both of those questions first with regard to expectations.
<unk> CH MP interaction I'll, let Dr. Colin Hislop addressed that question Colin.
Yeah. Thanks, David.
We're in the process of.
Completing some sensitivity analyses that the.
<unk> requested all of us.
Eldon has.
Mentioned just recently in his remarks, we are expecting to complete that process.
And have to see HMP render their opinion by the end of this calendar year.
Yes, definitely and thanks for that just one other one other quick question, which is.
When are you expecting that the one was the first patient at the moment to be dosed or are you still expecting by year end or do you expect the screening process would take a little longer.
Sure. The question is when will <unk>, our first patient dosed in the <unk> interferon Lambda limit to registration trial in grid.
And start up activity for limits are going really well when the process of activating sites across 13 countries and we're planning for first patient to be randomized by the end of the year.
Okay, great. Thank you very much.
Thank you.
Thank you. Our next question comes from the line of Maury Raycroft from Jefferies. Your line is open you may ask your question.
Hi, everyone congrats on the progress.
Finishing enrollment with deliver and thanks for taking my questions.
I was just going to ask about the timeline for limit to enrollment.
You mentioned that you are going to be using the best performing sites from phase III deliver and so just wondering if.
I am guessing we should expect that study to enroll faster with the 150 patients based on the experience with deliver but are you seeing anything additional on how we should think about that.
Sure that's pretty self explanatory Maury, thank you and.
Good to hear from you I'll turn that question to Ingrid, Yes, Hi, Maury I think.
A good handle on the study yes, the limit to study is less than half the size of the deliver study.
Given that and the fact that we are choosing the best sites from deliver for limit two we do anticipate that enrollment will be much more efficient.
Haven't given guidance on how long it will take enrollment to complete but we'll provide that at a future date, we're definitely excited more about the limit to study.
Admittedly no that deliver was and is a complex study to enroll.
Reason, we were excited to announce over 400 patients.
And the deliver phase III program.
And we're excited about that opportunity pegged weighted interferon Lambda is a well tolerated interferon and to your point, we do believe that physicians will.
Desire and patients will desire, a well tolerated interferon to be used in the treatment of HDD and so our expectation is that while pay weighted interferon Lambda will initially be approved as a monotherapy.
The limit to study completion that there will be opportunities, where physicians will seek to prescribe <unk>.
Regulated interferon Lambda with loan of foreign <unk>, and maybe I'll turn it over to Ingrid to comment a little bit further and also add color around whats been done at the NIH as you mentioned Maury and and what we are looking forward to from the NIH and grid sure. So hi, Morey Sandler aware NIH reported out at.
Last year, the completed phase II lift one study and Thats a combination of peg interferon Lambda with Luna foreign at photon appear and that.
Indeed did show the most robust antiviral effect to date further demonstrating the opportunity for this combination of our proprietary compounds based on the promising terrible Virologic response of lift one of which was a 24 week treatment study. We're planning a follow on study with the NIH called lift two and this is a 48.
Weeks dosing of the combination of peg interferon Lambda with low enough on a per ton of year with a 24 week follow up and so hopefully the thought is that by dosing longer you will allow for consolidation of antiviral response and result in higher durable Virologic response rates and we look forward to reporting that out in the future.
Great. Okay. Thanks for taking my questions.
Thank you Laurie.
Over a placebo and we haven't given specifics as to the powering them, except to say that it is powered to show that statistical significance and as I had mentioned in in early two earlier question for placebo for patients who are not treated them. They are not expected to achieve that two log decline in H D D.
And they used to be RNA and L.
L T normalization, so the responders in the placebo arm should should be very low.
We feel very good in short about the design of the phase III deliver study based on our phase two data generated with <unk> and Ritonavir and then loan upon every ton of <unk> in combination with Pegylated interferon Alpha and definitely look forward to reporting.
Information as it becomes available and Bryan I want to make sure that we address the second question related to patient populations I think in the deliver study and the limit to study and I believe you were asking about background therapy were you, referring to whether or not patients were on nucleoside background therapy.
In both studies.
Well, yes, I guess my question was.
Why is deliver.
HBV nucleoside background therapy.
Wanted to allows for cessation of all therapy I don't think they are on therapy.
So actually both.
Both studies are.
Requiring patients to be on background nuc synthesis, an FDA requirement and FDA guidelines Yep Yep.
Okay. Thanks.
You bet.
Thank you again, if you have a question. Please press Star then the number one on your Dutch stone telephone.
Have another question from the line of Michael Higgins from Ladenburg Thalmann. Your line is open.
Thanks, operator, thanks, guys for taking the questions. Congratulations on continued progress with your development milestones.
One of the questions in HDD, let me switch it up a bit to <unk>.
If you could shed some light on us for the next steps.
What needs to be accomplished here.
You've got it in the presentation.
If you can give some feedback as to what's happening in Mexico.
Yeah.
I'll take the program forward and look forward to providing additional guidance in the future.
Okay, well look forward to that just one other question on another program of Progeria are you able to give us the average milligram per day of these patients that are on can take thanks.
Yes, I believe we've we've communicated that publicly and so I'm gonna turn that over to Ellen.
Yes, it's approximately 175 milligrams a day on average that's what we see in the U S for the patients who are currently on drug.
Just one quick quality, if I may how do you see the European adoption versus the pattern of adoption in the U S. You mentioned the patients are spread out quite a bit but.
In terms of the time, England enrollment or transition rather.
How would you gauge the too thanks.
Oh, let eldon tackle that one as well.
In the U S. We had a higher percentage of patients that were on our expanded access program than we see in Europe. So it's possible that could take a little bit more time to convert them to commercial supply.
But we are in the process of continuing to enroll them on maps and we will certainly have an update in the future for you on that and as mentioned the the HEU program.
Allowing us to provide drugs to patients.
And seek reimbursement from French authorities and now having actually made our first shipment of Zoe can be into France.
We think is definitely a great step in that direction, given how tight the progeria community is.
We definitely are confident about our ability to execute moving forward.
Perfect. Thanks, guys.
Thank you.
[noise]. Thank you I'm, just telling any further questions I would now like to take the call back.
Cheering alley for any Friday night, Okay.
Okay. Thank you. This concludes our call. If you have any additional questions. Please contact us that info about your buyer dot com or you can reach out to remember of the anger management team. Thanks to everyone again for joining us on our two three call.
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