Hello, and welcome to the Aurinia Pharmaceuticals Audry Update. At this time, all participants are in a listen only mode. A question and answer session will follow the formal presentation. As a reminder, this conference is being recorded. It's now my pleasure to turn the call over to Glenn Schulman.
Please go ahead, sir.
Thank you, Kevin, and good afternoon, everyone. Welcome to this afternoon's conference call as we review the top line results of the AUDREY Phase twothree clinical study of VOS for the potential treatment of dry eye syndrome. Joining me from the Urania team on the call this afternoon are Mr. Peter Greenleaf, President and CEO Doctor. Neil Solomons, Chief Medical Officer Mr.
Mike Martin, Chief Business Officer and Mr. Joe Miller, our Chief Financial Officer. This afternoon, we issued a press release with the top line results from the AUDREY trial, which should be available on our website at ww.irineafarma.com. I'd like to remind everyone that today's call is being webcast live on Aurinia's Investor Relations website, and a replay will be available approximately two hours after the completion of today's call. Please also note that the content of today's call is the property of Aurinia and may not be recorded, reproduced or transcribed without prior written consent obtained from Aurinia.
For approval, please feel free to reach out to me, Glenn Schulman, via e mail at iruriniapharma dot com. During the course of this call, we may make forward looking statements based on our current expectations. These forward looking statements are subject to a number of significant risks and uncertainties, and our actual results may differ materially. For a discussion of factors that could affect our future financial results and business, please refer to the disclosure in today's press release and our most recent filings with Canadian securities authorities and reports that we file on Form six ks with the U. S.
Securities and Exchange Commission. Also, please note that all these statements made during today's call are occurring as of today, Monday, 11/02/2020, and are based upon information currently available to us. Except as required by law, we assume no obligation to update any such statements as of this date. With that, let me now turn the call over to Peter Greenleaf, President and CEO of Aurinia. Peter?
Thanks, Glenn, and I want to thank you all for taking the time to join us as we provide an update on the top line results from our Phase twothree AUDREY clinical trial that evaluated ophthalmic formulation of voclosporin, or most of you know as VOS, as a potential treatment for dry eye syndrome. So without belaboring the point, folks, we're obviously disappointed with the results that we observed in this dose ranging trial. As many of you recall, in 2018, we conducted an exploratory Phase II trial evaluating VOS in a head to head against Restasis, which has a cyclosporine emulsion formulation for dry eye, we had a lot of confidence going into that study. In January, we reported results from this trial of that year showing that VOS led to robust and significant treatment effects compared to Restasis. While VOS showed no different from RESTASIS on the primary endpoint improvements in drop discomfort at one minute, data from the secondary endpoint showed the potential for VOS to become a best in class therapy as measured by established approval pathways from existing treatments.
We were encouraged to observe improvements across subjective and objective measures. So with that, we embarked upon a dose ranging Phase IIIII trial against vehicle to further establish the potential of VOS for this disease. As noted in today's press release, we were surprised to observe a less than robust level of activity when compared to results in the Phase IIa study. Doctor. Neal Solomons will walk through the top line results momentarily.
But in summary, we failed to observe a statistically superior outcome in the dry eye patient population enrolled in this Phase twothree study. While we observed a dose dependent activity, initial analysis highlights that this study enrolled a very severe dry eye population. This population showed encouraging odds ratios versus vehicle, but failed to show statistically significant improvements compared to vehicle. As many of you know, dry eye is a difficult indication to study, highlighted by the number of pivotal trials that have been required by other therapies to receive FDA approval. We will continue to dig into the data and conduct additional post hoc analyses.
We have made the strategic decision though at this point to suspend the VOS development program for the near term. At Aurinia, we believe in responsible drug development for the benefits of patients in need. Obviously, this is a tough decision, but one guided by data and of course, the strategic considerations for the dry eye patient community and our key stakeholders. We believe that the resources previously committed to advancing VOS for dry eye could potentially provide better outcomes by investing in our core capabilities of Aurinia and other areas, namely developing autoimmune therapies for severe and less common conditions for underserved populations. We will be reporting out third quarter financial results next week.
But recall that as of the July 2020, Aurinia had a robust cash position of approximately $442,000,000 This obviously provides ample resources to fully fund the launch of voclosporin following the FDA approval as well as funding for the rest of our development programs, which of course included VOS. With the suspension of this program, this obviously extends our cash runway and our focus can now double down on our passion, our heightened intensity and our desire to prepare for the potential approval of voclosporin and the LN launch opportunity. In parallel, we will continue to build our scientific expertise by expanding and diversifying our development pipeline over time. I'd like to once again take this opportunity to thank all the investigators and especially the patients for trusting in us and participating in our efforts to advance our understanding of VOS in this indication. And with that, I'll turn the call over now to Doctor.
Solomons to add additional color regarding the AUDREY trial. Neil?
Thanks, Peter. As Peter mentioned, this is a tough data set to receive, especially after seeing the robust results we previously generated in the VOS exploratory trial. AUDIENCE was a randomized, double masked, vehicle controlled, dose ranging study evaluating the efficacy and safety of VOS in subjects with dry eye syndrome. Initiated back in October 2019, we enrolled a total of five zero eight subjects. The study consisted of four arms with a one:one:one:one randomization schedule, in which patients received either zero point two percent, zero point one percent, zero point zero five percent VOS or vehicle dosed twice daily for twelve weeks.
The primary outcome measure for the trial was the proportion of subjects with a 10 millimeter improvement in Schirmer TESS at four weeks or better. Secondary outcome measures evaluated in the trial included Schirmer tear test at other time points, fluorescein corneal staining at multiple time points, change in eye dryness, burning, stinging, itching, photophobia, eye pain and foreign body sensation at multiple time points and additional safety endpoints. As detailed in today's press release, we did not observe statistically significant improvements in STT at four weeks when comparing the dose group against vehicle. Since receiving the data, we've been digging into results and have made several observations.
First, there did appear to be both a dose dependent effect on tolerability as well as efficacy. However, the data are somewhat inconsistent
and didn't reach the threshold of activity that we had designed the study to observe. Subjects enrolled into the trial did seem to be a bit older than those subjects enrolled in our initial EXPLORERITY trial. Further, the subjects came into the trial with what appears to be a more severe dry eye disease. On average, baseline STT for all subjects was substantially lower than those result enrolled in either other DES studies or our exploratory study with VOS. As Peter mentioned, the team continues to interrogate the data set and is doing a number of post hoc analyses.
These analyses will take some time, but given the totality of data set, I agree that it is appropriate to suspend the dry eye program at this time. Aurinia has always aimed to fulfill the medical needs of patients in a meaningful and substantial way. And on first pass, the order results do not meet our internal goals at this time. With that, I'll pass it back to Peter for some closing remarks. Peter?
Thanks, Neil. With a strong balance sheet and cash, cash equivalents and short term investments of approximately USD $442,000,000 at the July, we remain fully funded to support the launch of voclosporin in lupus nephritis and execute on our broader pipeline ambitions in the future. We will be reporting our third quarter results next week on November 10, so we look forward to speaking with you further then. Thank you for your attention today, and the team is here to take your questions. So with that, operator, can you please open us up to a Q and A session?
Thank
you. Certainly. We'll now be conducting a question and answer session. Our first question today is coming from Alethia Young from Cantor Fitzgerald. Your line is now live.
Hey guys, thanks for taking my questions and sorry about the readout here. But I guess sort of surprising after the Phase two and maybe a setback for patients, but it does sort of kind of clean up the story and focus you on autoimmune. So with that, can you I just want you to kind of level to everyone to there's no read to make to the ongoing review of lupus nephritis? And will these data be shared with the FDA? And then second, what other indications might you think about strategically?
Or do you think it's kind of better to deploy more of the capital for the launch here? And then just while we have you, can you just talk a little bit about what you've been doing on the launch prep potential panel side as well? Thanks.
Yes. So why don't I start off and then I'd ask Neil to join me in with his commentary. I guess the first thing I would say that maybe didn't echo as loud as it should on the conference call is, I think dry eye was a unique opportunity for us because of the compound that we had and what we saw in the Phase II exploratory study. We've always said along the way that strategically, it wasn't aligned with where we were looking to take the company and where the company's core capabilities have been developed from, and that's in the autoimmune disease area and rare kidney sort of at the start, but more broadly, the autoimmune inflammatory space. Second, listen, if the FDA asks for the data, we'll be able to disclose the data to them and we'd be more than happy to do that.
But obviously, we're dealing with different divisions here and we've not historically had that question come from the agency on the previous studies done, but I'll ask Neil to maybe expand upon that. And then the last thing I'll say before I ask Neil to dig a little deeper maybe into the FDA question is on the deployment of capital. I think first and foremost in priority order, this just gives us an ability to heighten our focus on the lupus the potential lupus nephritis launch ahead of us. It extends our runway in terms of the investment that we can make in making that launch both in The U. S.
And globally immensely successful. And it also gives us the ability to look at other potential pipeline decisions. Today, we're not really ready to disclose what our decisions will be there. But I do think for the longevity of the company, diversification of the pipeline is going to be a key priority for us. So while I wouldn't exclude any further development for voclosporin and other indications as long as the IP holds up, I would say that diversifying the science in our pipeline probably makes a lot of sense at this stage of the game, too.
Neil, what would you add about the FDA engagement?
Yes. So I mean, I think that the obviously, when the study is wrapped up, the study report will be submitted to the IND, but that is the ophthalmological division. We don't anticipate this being shared with the reviewing division nor it being in fact relevant as a topical version of the drug in a sort of unique vehicle compared to the oral systemic administration. So we're not so we don't expect this to any impact there. So you asked there was a yes, you were also asking about PrEP's currently ongoing PrEP's for the with the NDA.
The review is ongoing. Again, we've had word from the FDA that they're also anticipating an advisory panel. And we're going according to our previously stated guidance.
Great. Thank you.
Thank you. Our next question is coming from Maury Raycroft from Jefferies. Your line is now live.
Hi. This is Farthin on for Maury. So a couple of questions. Is it possible that VACIL patients who are on different background medications that probably used to manage the symptoms and this led to the placebo effect, whereas in the head to head study versus the status, this was actually a more controlled study?
It's a good question. I'll have Neil take that, talking about the Phase II comparison to how we or excuse me, the IIa to the IIb and changes we may have made on background patient population or meds, etcetera. So Neil, you want to try that?
Yes, sure. I mean the background or certainly entry criteria were very similar. And if we look at the placebo effect or the vehicle effect, it was pretty much what we expected and it was in line with the vehicle effect that's been observed in other studies. So we don't think that that's an issue at all to do with that. Certainly, were some patients that appear to on the face of it have a higher and more severe disease.
But again, that wouldn't account for the entire totality of results that we saw.
I see. And for the secondary outcomes, were any of them statistically significant?
We've not reported those yet. There were some that were various time points. But as we run through the data, that will be reported in due course. But for the most part, the primary and key segment were not statistically significant, but there were some observations at certain time points that were.
I see. And last question is you mentioned that the more severe patients basically in the Phase twothree, were there any patients that were basically not as severe? Any distinction between the subset?
We have there were patients, and we've certainly in part of our post hoc analysis looking to try and apply the severity that we saw in the exploratory study to the patients in the AURODY trial. But this is ongoing post hoc work at the moment. But also just be aware this is post hoc, so this is more for the team to actually understand the reasons for what we saw and see whether there's any explanation for the slightly different results that we saw in this study compared to the exploratory trial.
Thank you. Got it.
Thanks, Maury.
Thank you. Our next question today is coming from Ed Arce from H. C. Wainwright. Your line is now live.
Great. Thanks.
Yes.
So this result obviously is a bit of a disappointment, especially given the prior results. But I agree that in the big picture, this does kind of clean up the profile and the story as you get ready for the potential launch in LN. So just a couple of questions for me. One, with the study itself, I know Neil, you had discussed in your prepared remarks the assessment that there was a dose dependent effect on both safety and efficacy. But I'm just wondering, given the three doses and the results seem so close, especially the low dose versus the high dose, which is 4x multiple on the concentration and was very similar.
I'm just wondering if there's anything further you could share there.
Yes, Neil. Neil, go ahead on that.
Yes, sure. So I think your question is more around the fact that there wasn't a profound dose effect given the sort of four times concentration difference between the high dose and low dose. Is that your question? Right.
Yes.
Yes. I mean, we observed that too, Ed. And I think, as we said before, we don't fully understand all the reasons that certainly some of it could be related to the severity of some of the patients perhaps. They were not able to respond to any therapy no matter how concentrated. And like I said to the caller before, to the question before, we are exploring a lot of this in our post hoc analyses, trying to apply some different criteria to the results to better understand them.
I suppose the other thing as well is that these dry eye studies, there is inherent variability if you look across the network of dry eye trials, and maybe that's also we're seeing an element of that as well. But I anything else I'll say at this point will be speculation.
Sure. Okay. So the other question is around the idea of expanding your pipeline. Obviously, that has been of some interest. And you mentioned previously in your prepared remarks, Peter, the focus generally in autoimmune and more specifically, at least in the near term, continuing around rare kidney diseases and perhaps even proteinuria diseases.
You had mentioned you're not prepared really to discuss potential indications that you might pursue. But perhaps if you could discuss what you believe are some of the key selection criteria as you work through that process.
Phil, I'll ask Neil to double down on this question with me. The one thing, I guess, I would just like to make sure we reinforce right upfront is that this is as much a strategic decision for us. The team could continue to run down the data and find us a potential path to bring a drug and the trial forward in dry eye that could potentially win based upon what we had set out in terms best of what we found out in this study. But there are sort of three components. One, we set a very high bar in terms of or very high hurdle, high bar in terms of what we expect in terms of innovation and patient impact.
And we didn't see what we wanted to see here. So the rest falls into sort of a strategic fit decision, the best use of investor capital and how it all sort of equates into time, risk, the strategic fit and IP. And that's why we're coming forward and saying, listen, this looks like one we want to suspend and potentially search elsewhere. As it pertains to voclosporin, specifically in other rare proteinuric kidney diseases, Neil and his team are interrogating that right now. And between I think where the balance we have to try to strike is doing really solid differentiation work on the product that continues to develop to differentiate the compound from other first generation CNIs, we're definitely going to do.
Those are not necessarily indications or approval type work. The second is what we can fund through scientific exploration through the normal post market Phase IV type of work. There will definitely be things we do there. What we are trying to do now is figure out based on scientific rationale, commercial possibility and IP runway, what the best potential areas could be to go after a labeled approach. And I think what I just did is give you what those criteria are, but but not in any the right order.
I think we have to find something that would have true scientific rationale. If that's there, then there has to be real strong potential commercial rationale and wrapped around both of those as intellectual property. So Neil, what did I miss there?
No. I mean, obviously, I think you covered most of it, but the other thing is operational feasibility, whether you can actually get the patients with the patients around that. And of course, that feeds into commercial case of these indications as well. So that's something that we're looking at very, very carefully, whether you can actually whether the patients are actually indeed out there. But other than that, I think you covered it, Peter.
Okay, great. And then just lastly, given, as you said, this the continuation of VOS had already been considered in part of your cash runway. And so now suspending the study does free up some capital and extend the runway. Would you be prepared today to say by how much that would extend?
Not specifically, Ed, because we really haven't guided to that at this stage of the game, but let us take that back. We've got a quarter we have to report coming up and see if we can give you any clearer breed on that. But I think it's fair to say that we had a Phase III program in the mix scheduled for the next, let's call it, eighteen to twenty four months. And if that goes away and you can do your estimates on what the dry eye study in Phase III might cost with how many patients, then that becomes capital to reallocate. And as we said in priority order right now, the reallocation would be to extend our runway for a successful potential successful commercial launch in The U.
S. Once we have FDA approval.
Fair enough. Thank you so much.
Thanks, Ed.
Thank you. Our next question is coming from Justin Kim from Oppenheimer. Your line is now live.
Hi, good afternoon. Thanks for taking the question. I know it's a little it's still a little early days with the data, but do you have any color on how well the study was executed, things like safety and tolerability and compliance during the study as well as maybe any observed impact from COVID-nineteen?
Good questions. Neil?
Yes. I mean, yes, they are good questions, And yes, we have looked at that. And the compliance was exceptionally good. I'll take you back a step and say that we used a highly reputable CRO that really pretty much only does studies in eyes. We were very pleased with the process.
We were very pleased with their conduct and the way they actually executed the trial. And so in terms of the impact of COVID, we're conducting sub analysis to see whether there's any impacts on the data. Our preliminary analysis suggests that there's absolutely no impact whatsoever on the patients who came on after the start of the pandemic to those that were recruited beforehand. And like I say, that with the combined with the exceptional compliance means we had very few, if any, concerns about the actual running of the study.
Got it. That makes a lot of sense. I mean the one thing I also wanted to maybe touch on was just in terms of the decision to suspend development, were those decisions made having the twelve week data available? And if not, to what extent might those data change any decisions with how you think about discontinuing the program or finding a partner, those types of options for VOS at this stage?
I think the data in total is why we're basing our decision. Neil, it's clear that there's more we need to uncover. But and what we have uncovered to date, it's clear to us that the development pathway would obviously be longer. You'd have to repeat studies. And the effect that we saw in and Neil, if I misstep in terms of my articulation of that, please correct me.
That sort of coupled with the impact that we saw and the best use of the funds that we have, we made the decision that we're better served moving in a different direction with those resources.
And yes, we did, in fact, have all the we're in possession of all the twelve week data, and there's not it doesn't really add much, to be honest.
Thanks Justin.
Thank you. Our next question is coming from David Martin from Bloom Burton. Your line is now live.
Hi there guys. Thanks for taking my questions. The first one is just a confirmation. Neil, you kind of broke up when you talked about the AdCom. I just want to confirm, you said there's still no AdCom plant?
Correct.
Okay. Second question is, you talked mainly about the objective endpoints on the call and in the press release. Was there any more separation on the more subjective, the symptom based scores?
We haven't disclosed that, but no, there wasn't. The symptoms improved in all arms, and there was no appreciable separation from a statistical perspective.
Okay. And then the last question. Peter, you mentioned that you're good part of the work you're going to do now going forward is further differentiation work on voclosporin. I know that the clinical trial extension will get some long term safety out of that, I guess, probably in about a year. What other differentiation work is planned?
And when might we see those results?
It's a good question, David. And obviously, some of the work we're doing, we can't disclose yet because it's internal. I think you can point to, as I'm sure many saw, we announced some work over in Europe on some interesting, I think, differentiation work for voclosporin in terms of how it could impact transplant patients. That type of work is always ongoing in terms of us looking with investigators to find areas where compound and its difference and how it could impact patients is something we look at pretty regularly. And I think folks should look at that as one example of an area that we're supporting because of its potential to differentiate.
But at this stage, the work that Rob and his group are doing on the early research side or sort of PKPD type work and to further differentiate the product is just stuff we were working on and too early to disclose at this stage.
Thank you. We've reached the end of our question and answer session. I would turn the floor back over to management for any further or closing comments.
I just want to thank everybody for their time. Also want to thank again, thank our investigators and patients for working with us towards this outcome. We look forward to updating investors in just about a week or so on our quarterly call. Thanks for taking the time today.
Thank you. That does conclude today's teleconference. You may disconnect your line at this time and have a wonderful day. We thank you for your participation.