Q1 2020 Earnings Call

May 13, 2020

[music].

Good afternoon, ladies and gentlemen, and welcome to the deals our fourth quarter 2020, earning conference call.

All.

Looking only about.

They don't conducted question answer session and instructions given at that time.

As a lot of this conference call is being recorded.

I would not uncommon Albertsons Mr. Mitchell Chang Chief Financial Officer ideal about.

Go ahead.

Thanks, and welcome everyone to our first quarter 2020 earnings call the pressure Leach supporting our financial results is available on the Investor and media page of the corporate website at Triple W got feel about dot com.

Joining me on this call. This afternoon, our CEO, our chairman and Chief Executive Officer.

Youre dropping our chief Medical officer, and build regulates vice president had to commercial.

That's pretty minor, we'll be making forward looking statements regarding our financial outlook. In addition to regulatory current development and commercialization plans and research activities.

These statements are subject to risks uncertainties that may cause actual results to materially differ from those forecasted.

Description of these risks can be found in our most recent form 10-Q on file with the FCC.

Her too many companies into both from a sector very closely monitoring to clean up endemic including D.. So cheated restrictions on travel and work that had been implemented.

Well its its potential impact on our business in clinical trials.

And to which the krona virus impact at school do depend on future development.

You are highly uncertain and cannot be predicted including new information, which me emerge concerning the security of the Corona buyers and the actions to continue to client of ours or to treat is in fact I'm offenders.

Now I'd like to turn the call over to bring down our CEO.

Hi, good each good afternoon, everyone. Thank you put you on yachts today I'm pleased to report that we have yet another productive quarter an offer to have very strong start twentytwenty.

Participate if I will lead product candidate I know he's a map approaching about a one month one year, we our fixed the company milestone to date, our first potential U.S. regulatory approval.

Why would this would of course be a pivotal moment of our company well importantly, you wouldn't be our victory for the thousands of patients affected by you and my likes optic a spectrum disorder <unk> am away Steve.

In parallel to launch readiness activities, we continue to make Saudi to progress across our entire pipeline.

Most recently that you'd be healthy this afternoon.

That was promising you know movies jobs from our ongoing piece will be trial of the I'd be 7734.

And it just a several months ago, we initiate a phase twob trial, we I'd be 49 20 for the treatment of patients with stripping single I'll comment rheumatic diseases for which there are currently no approved disease modifying therapies.

Energy and if we talked when it's all of our teams are incredible despite the Cogs 19, pandemic, which is affecting Autobots today.

I like to take a moment to address outcome because response to the pandemic My first and foremost our thought how would the thousands of patients and their families who have been directly impacted.

And to the healthcare workers, helping to keep us all safe what you'd immensely grateful.

To that extent that I'm kind of help contained a spread of the wireless and flattens occur we continue to be nimble allergy season, making.

I was set up the outbreak we moved quickly to protect and to support our we didn't go each added a patient that we serve implementing a work from home policy and it successfully transitioning into virtual external stakeholders engagements.

While it's still too early to tell how impactful just pandemic will ultimately be at present, we have been fortunate to you experienced minimally facts, albeit an operations.

We have sufficient supply of drug product to supply our launch and to continue our ongoing clinical trials.

However, we have wasn't terribly posting woman's allowing patients to some of our trials you know what are the prioritize patients health ended that I'll dig investigators and I'll clinical trial sites.

They are coming weeks and months, we'll continue to long into the penetration carefully and to public items from local and federal health authorities.

Despite some adjustments I would they be operations the fixed they did it by probably 19 pandemic well positioned for success in Twentyth, Wendy and to be joint.

Had a precedent Oh R&D is focused on three distinct particularly in pathways or anybody because military and it didn't make the fact gang pathways that I know I range off autoimmune disorders.

We believe our coach which pushes our pipeline within a product meet you need watching you left more precise candidates that have the ability to create would go disease. Additionally, our research group continues to study empty basmati acuity when do you pathways.

Yes, I will brief history, well build a robust pipeline and today, how five molecule, which give out image three of the wage in clinic.

Our leading near term I still use of course, the potential MTO approval of I know, there's about a on blasting.

I see we are anywhere you worry if I'm too disease that affects about 10000 patients in the United States I know.

We knew that about 80% of AOMT patients have already anybodys to water channel protein called are performing for this or anybodys produced by that's not been S and the plasma cells, thereby and primarily to astrocytes central nervous system. The bindi of all couple reported anybody still central nervous system sales it's been.

The two critical attacks, which it damage the optical Europe spinal cord and green.

<unk> as a health and according to up nice can declined significantly with just one will attack.

Hi, many patients who come to you before school blindness, and a paralyses any experienced progressive worsening because ability with each subsequent to attack.

You see that devastating disease prevention feature on tax and maybe team disability, a crucial to patient health.

As you may recall.

We designed I'd be some have to target and TB Cdnineteen directed T cells, which include that's not matched and some plasma cells.

Based on strong efficacy and safety readout from our pivotal in momentum Kyle we have demonstrated significant with heck reduction as well as reductions worsening disability.

It's actually a U.M. arden issue and a reduction in ammo as the related hospitalizations, we believe that itll be the map has the potential to be up front ninth monotherapy for animal safety patients.

I will open label extension period has came out the insight into how I look at the map might work long term.

Then the integration of equipment. It's two years, there's some patient on either the map opt to five years results are consistent with that of the primary analyses and over 80% of patients remain attack free.

So we are highly encouraged by the potential long term benefits of I know, there's a map.

With that I like to handle the call but to be a week. It's our head of commercial it will provide more details about our launch strategy and a readiness bill.

Thanks, Ben as you've just heard with our PDUFA date of June 11, now just weeks away. We are soon approaching a very exciting and momentous time at our company.

At the outset of our launch planning when thinking about the most important characteristics of our field teams reprioritized experience in rare and neurological diseases as well as relationships with key customers.

At this would be our potential first launch we had the unique opportunity to hand pick a team dedicated exclusively animal westie.

To support the launch Viola has assembled season teams, including myself market access and sales representatives, but each team averaging over 15 years of experience the breadth and depth of knowledge of these teams is really impressive.

The cobot 19 pandemic has forced us to face some unique challenges specifically travel restrictions and limited physical access to our customers.

However, this has not been a major impediment to our progress as we were able to quickly pivot to remote interactions and alternative ways of disseminated disease education.

Based on our conversations with positions, including key opinion leaders as well as managed care providers and patients. We believe that I nimble isn't that meets many of the needs that our customers have in treating animal west D. and we are confident in our ability to reach those who could benefit most from it.

Importantly, despite other approaches on the market or in development. Many physicians remain very interested and b cell depletion and we're continuing to expand our educational efforts.

We're also strongly focused on the patient journey and our patient advocacy team has been working closely with animalistic specific advocacy groups, particularly the guffey Jackson charitable Foundation, the Samira Foundation, and the Steagall rare neuro immune association to better understand patients' needs and provide high quality.

Disease education.

Through this then we have sponsored a series of podcast to help share knowledge around animal SD spanning from diagnosis and treatment the quality of life and what it feels like to live with a condition.

We have heard from patients that this has been immensely helpful. In addressing many of their key questions, especially around the management of animals, Steve in the face of cold in 19.

And looking ahead you find a blessing of is approved we'll be focusing on newly diagnosed patients and those experiencing inadequate response to their current maintenance regimen.

Our aim is to help prevent future animal west the attacks and prevent further worsening of visibility.

We believe that I know, but listen I will provide significant value to the animal rescue community and our goal is to ensure patients have access to this important medication.

While our immediate focus has been under development and potential commercialization in animal Westie, we continue to evaluate the potential broader rule of minimalism out in the auto antibody pathway.

With that I would like to hand, the call over to you weren't drop off our chief Medical officer to provide an overview of the current and future development plans for on it but listen mab as well as a summary of our progress in our other clinical programs.

Thank you Bill.

We'll get into I live in liver mobs, and just a moment, but I would like to start this part of the earnings call presentation with some data that we it's just on a reported today that involves yeah do something somebody to before.

The you'll be something Southernsun for is wonderful antibodies that was designed to target an antigen that as specific exposed on the surface of cousins subjected to do so.

You must also played a multi comfortable golden inflammation.

And that a basic level they are in Britain and activating themselves and also contributed to a problem sell throughs, they've been tissues with patients in patients with other new diseases.

For example in skin biopsies, we'll keep asking you can see large enrichment of Pdcs Similarly, often biopsies.

The old and diseases, such as myositis.

You can see health and towns in large numbers of clubs to produce a lot of cytokines and maintain inflammation.

Dishes.

For the hypothesis is that well depleting, so you could potentially interrupt the pro inflammatory cycle.

So as a reminder, our phase one trial involves three cohorts first cohort, including patients with the mix of different other news diseases, such as lupus sibling systemic sclerosis.

And the secondly, cohorts enrolled patients with cutaneous with does go up to contains 12 patients and call. It the 11 patients and the goal was to test whether.

We're not only be.

The circulation, which we already knew from our phase one trial, but also in inflamed tissues and to that and we took a skin biopsies from these patients with to 10 years Lucas before and after treatment to that because actually you Numerate Oh, the peak seasons before and after treatment.

So this afternoon would report that <unk> interim data from covert too as another 50 and for clinical outcome measures from a subset of circle with <unk>.

The primary outcome in the struggle safety or we saw that the rate of adverse events and serious adverse events was comparable between active and placebo patients there were no unexpected safety findings.

We also so that's in cohort two you ought to be subject Hadnt before was potency.

DP Pcs in the skin biopsies of patients with Sealy after three months of frequent.

Finally, we found the dose dependent improvement in the clinical outcome measure called the classic continues lupus area and severity index, which is basically measuring all the activity of skin rashes.

We felt that the proportion of patients with improvements in the coffee bar either at least four points or 50% both of which is considered clinically meaningful was substantially larger in subjects treated the southern seventh before the placebo control.

We look forward to reporting final results from disclose including both from cohort three which we anticipate towards the end of the third quarter.

We have now in the process of preparing for phase two studies in one or more detention sort of thought to be driven in part by PVC and the pro inflammatory mediators they produce.

[noise] going back to you know there's enough we have made progress on our are less likely put them in most geographies I'm glad you just over the disease.

So as you know we are preparing potentially pivotal trials. Both of these indications until we have submitted the ideas as well as a scientific advice and similar things.

Countries outside of United States.

Obtained feedback from regulatory agencies incorporated that into.

Until our clinical plans and protocols the funded less political or approach in the process of on study up sites with the goal of starting a pivotal trials and both of these indications towards the middle of this year.

With of course, so did you start to be whether clinical trial sites little at that time I'll be ready to enroll patients as a result of the cobot blinking endemic of currently we're still expecting well to start these trials on time.

What's that youre familiar with my senior gross.

There are inflammatory disorders and stuff. The just because has a number of similarities between animal spectrum disorder is also mediated by apologetic all the antibody and it results in a normal muscle weakness other electrical systems.

I just before related disease is.

Could you give you gotta sparked by two were like swelling in fibrosis or various affected organs, which has caused by infiltration of seasoned team explicitly plasma cells of a gentleman edgy for deepens.

Many different organs and nobody can be involved commonly involved or it could be a pancreas celebrate plans, which are prepared to me in them typically.

Well so both of these are good and indications and that's the impact so.

Relatively large numbers of patients and so we really looking forward to starting these trials in these two my second indications.

Finally up so I like to address the I'd be 49 20.

That is our fusion protein that's designed to buy and people can they get them activated T cells blocking the interaction with city for expressing cells.

Let's see what do you see 40 million pathway is a key co stimulatory effect that is required for the activation of T cells, but also plays other important biological goals.

As you know we have initiated seems to be trial in patients with children syndrome.

This is the second most common robotic disease. After <unk> why doesn't it was characterized by Muslim eye dryness, but can also result in fatigue chronic pain and other suspended institutions.

Additionally trials.

And your attention I focus on patients for the high systemic disease activity and we are also including these patients in our trial.

Also looking at them another population of patients who have less specific disease activity, but the house symptom burden relates to dry mouth fatigue I was just symptoms.

We dose the first pitching this plan at the end of 29 tea and enrolled additional patients in early 2020.

Almost who continued to be followed in all trial.

Voluntarily Paul enrollment of patients due to the Covance 19, a pandemic.

We are continuously evaluating the situation, that's a different participating sites and countries.

So I look forward to resuming enrollment as soon as tasted possible.

With that I would like to turn it over time as shown on our Chief Financial Officer.

Thanks, Sean I like to refer you to the press release issued earlier today for a summary of our financial results for the first quarter ended March 31st 2020, and take this opportunity to preclude review a few items.

On a quarter or R&D investment totaled $26.8 million, that's we continue to advance our pipeline.

For a screening we invested $15.3 million into first quarter, which includes our U.S. precommercialization investments or I never losing about animal and state.

And all our total operating expense for the first quarter was $42.1 million.

Together with our other income.

Operating loss for the quarter ended March 31st 2020 was $40.8 million, which translate to a GAAP net loss per share of 80 cents for Q1 2020.

For cash we ended the first quarter with approximately $335 million in cash cash equivalent and investment.

With that I'd like to ended over back to bank.

Thanks, Mitch. Thank you again for joining our call today as you have heard we had a strong quarter and are well positioned protective and a successful year ahead.

We especially look forward trial, because the near term milestones that potential FDA approval and commercialization of I know, there's a map which would it be a first our company.

Many thanks to our teams for their continued hard work your question. This forward.

In addition to the divestment up several other pipeline when kids.

Now I would like to open the call two questions operator.

Thank you.

Ladies and girls I got the question. Please press Star then one on you touched on telephone if your question and the answer you, which means that MCU leased breakfast how Q.

One moment please bar first question.

Our first question conference Seamus Fernandez of Guggenheim Your line is open.

Thanks very much for the question then congratulations on the.

Before I do that.

Yarn My one question there I I know the I'm a little bit at the data that you mentioned there in terms of the the differential between the arms on the quality scores. So well just hoping you could clarify that and then you know as you think about the or that the overall develop.

And approach or 773 for can you just give us your sensata yarn, you've you've studied a lot of products and movements in the past you feel that at least it's early data.

No sort of winds up to some of the other therapies that that you've seen in clinical development and then just a quick clarification, where there any was there any evidence of a have a meaningful increase in herpes related reinfection. Thank you.

Okay.

Thanks for your question was with respect to the cloud. We are we did observed dose dependent separation between active and placebo there was a little bit of a separation and cohort two there wasn't much clearer supervision and core three I'm not going to present, a numerical values today, but suffice it to say.

There was a very clear I'm separation between the active and under the fever.

You know we're currently in the process of prioritizing and designing on phase two trial for this molecule that lupus is clearly included in the in the list of is high on the list, Yes, you're correct. We have successfully experience and conducting lupus 12, you know I'm the challenges and a pit.

FFO, but we also think that there is substantial remaining unmet medical need and this indication. It's currently still only one biologic approved which has relatively modest efficacy and is still.

Plenty of unmet medical need in patients with severe manifestations nephritis and other I'm serious symptoms of lupus. So it's clearly on our radar screen. We doing the work currently of discussing what is the most efficient.

Design potentially in the lupus study, but also considering additional indications beyond lupus square Ptcs are known to play a role.

And with respect to your last question and herpes, we did not see any increase any increase in or because infections or reactivation.

Great Congratulations again I'll jump back into <unk>.

Thank you. Our next question comes on cap Honda Morgan Stanley. Your line is open.

Congrats on the progress and thanks for taking the question I get the person how many patients were in cohort three for the interim analysis.

And then take on the current data do you think you capture that therapeutic window or would you want to look at even higher dose getting that the safety was comparable to placebo even in cohort three and then I've a follow up.

So the of of the ends in the cohorts, where age for code 112, and cohorts to and 11 and over three I'm out of those 11.

There were I think.

Nine evaluable in that they had past the the time of the primary endpoint, but Beatty five I'm. So that's just the data that we've included I'm in the interim analysis.

<unk>.

Oh, yes for don't think so we still.

I haven't completed our modeling so we will inputs the data into or PK PD efficacy model.

We will wait until we have to complete data for the available to make a definitive sort of statement or decision on the on the on the right dose. It seems I'm from the preliminary data that the that the a higher dose or that was tested.

Had a bigger impact on the class B, but you know whether or not this is the the peak or whether or potentially a higher doses needs to be looked at I think we will be guided by the PK PD modeling.

Great. Thanks, and then you've indicated that upon approval.

I was talking about it wasn't that friend in though you're able to begin the commercial launch shortly after some companies have decided to push off launches to later in the year given the challenges in the current environment, including like lack of face to face interactions and and lower patient visits. So can you talk about your decision to lot. Shortly after approval and what gives you confidence that the current environment won't be significant headwind here long.

Thanks.

Great question, you're obviously this is a dynamic situation, we're dealing with nationwide. The cobot 19 pandemic has affected different parts of the country very differently. The good news is with our experienced sales force. We have some very good relationship that we think we can rely on for a different parts. In addition, we know.

That there are parts of the country that are opening already we've done some research recently don't understand little bit about some regional differences in the regional plans will be very different depending on if your hotspot or not so we think that we have a product that is moving along the needs of our target audience and we think that we have some relationships that we can count on.

You have meaningful interactions with physicians after approval and therefore, we think the best a path for us is to get the product to patients who need it as quickly as we can.

Great. Thanks, a lot.

Thank you. Our next question comes from Paul its way of Goldman Sachs. Your line is open.

Hi, This is kinda Jenkins on for Paul and I do you think about the heterogeneity in live at the disease can you just talk about if there's any settings, where are you seeing PDC activity. It's depletion make more my sense is that therapeutic approach.

Yeah, so you're perfectly correct and pointing out that what are the key issues in the in drug development into this is heterogeneity heterogeneity. Both in terms of political manifestations, but also potentially with respect to underlying biological pathways and.

Are we doing actually a lot of work to disentangle some of the heterogeneity. We're looking at several biomarkers that are associated with the Bdcs. We're looking at Oh, the interferon signature or we look at interferon regulated.

Means expressed in into skin as well and so we tried to correlates to the extent oftentimes is relatively small trial.

These biomarkers with the clinical outcomes that we have observed. So this would work is still ongoing and we hope to leverage to potentially be able to stratify or [laughter] are focused here.

The phase two populations to include those patients who were most likely to benefit.

Any thoughts on and then when you think about Sjogrens then got minute in particular, the population that maybe had high September it and that Leslie <unk> disease. How it can't allows then maybe patient. He didn't think about what conditions are met important or will it be clinic clinically meaningful there and then kind of at the follow up to that what's the size of that.

The election, how does that compare to the more severe grid.

Yeah. So if you ask patients I think you will consistently hear that fatigue is on the very much on the top of their list of concerns followed by by dryness over here. These patients are very.

Unpleasant symptoms caused by driving dry mouth dry eyes, and I'm you know absence of both decreases in other words as well. So I think these are the due to the key patient reported outcomes that need to be captured in the way, we recapturing patient reported outcomes is over the validated.

Instrument difficulty a S. Three and that includes many of these things.

The the but the number of patients and the high symptom a burden subgroup.

In in the trial is.

I catch I can't remember it off the top a bad but I think it's.

Uh huh.

No. It's it's.

Roughly equal size I think it's a little bit larger than.

The size entity.

The because with a with a high some with the hi disease activity as a roughly equal but they are they will be under my separately one with an objective outcome. The S died and one with a patient reported outcome. The S. Three.

And does that child kind of correlate well to the real world population differences like what percentage of patient Martin Universal died and my brain.

No. So in the in the real World depopulation, but there hasn't been burden is much larger than the one with a high disease activity, but you know do the the trial size is more driven by statistical considerations to go to buy power considerations and by input performance rather than to reflect the of the real world.

Any kind of and thank you so much.

Our next question comes a friendship Ricardo.

Cowen Your line is open.

Great. Thank you very much congratulations on all the progress.

Two categories. The question first on seven seven tree or when we see additional data later in the year can you give us a sense for what kind of having doses and what kind of hollaway. My follow up you might expect and also we could be sharing with us more specifics or can you can comment now about the patience I believe from an enrollment criteria.

Standpoint could have been though is with and without systemic lupus and of course that cutaneous can have various types of manifestations of the kids persist chronic any sort of.

You know characterization of the types of patients that we'll see would be helpful. As we all began to be slightly Mac out, but this could look out from a market opportunity standpoint. Thanks.

So the the trial included patients, both with and without systemic lupus, but they have to have two tickets, but at this stage. They they couldn't a systemic lupus or just your opinion and in the trial it was approximately.

Half half those patients with and without systemic lupus.

And then in terms of what will we see at the end I'm in the final analysis. So the final analysis will include the U.S.P.D. analysis, all skins, and biopsies and cohort three from these Bob will be processed when the last patient and cover three has had there.

85 clubs getting biopsy and then these sounds good buses will be process, and analyze which takes a little bit of type and then at the end of the trial, we will have a complete data with respect to safety.

Clinical outcome measures and a PK PD biomarkers for all three cohorts.

Patient the for the primary endpoint is assessed that after three month at 85, but there will be additional safety follow up at least from day 140, Oh for all patients and depending on the recovery kinetics of pdcs potentially be young but some patients.

Great well look forward to that and then I never lives a man just to make sure that were up to date, obviously very close to the PDUFA date I think the last time, you communicated publicly 10th felt comfortable and expressing that you do not expect a black box and as far as any last minute type labeling discussions I was there anything to share with us in that regard and then secondly.

If you do launch help us get a sense for what you think we'll be able to measure what kind of metrics will be out there in terms of another prescription data. Realizing that this is a specialty product, but you know have lost are we going to be as we begin to try and figure out how the initial launch is going in this environment. Thank you.

I'll take that one on label so there's no updates to share no status changes since our last.

Cool and then I'll pass it over to Bill for the second part of the question.

Good question, obviously as we've talked about before from from a focus perspective, we're focusing on both newly diagnosed patients in patients who are having inadequate response to their existing immunosuppressant therapies are on their roughly 10000 actionable patients we've seen in the marketplace and we'll be going after.

During the key institutions, along with some of the community doctors, who were seeing a high number of patients as well too as kind of our priority focus at the immediate launch period again as mentioned before in one of the last question. This will vary probably significantly across the nation, depending on the status of opening up a with the.

Different areas, but we expect to be able to have good relationships and good discussions with our key target doctors right after launch.

But in terms of third party prescription tracking data et cetera, we're gonna be.

Kind of navigating our way through a bit of a five is that fair or is there anything that you would comment in terms of.

You know how well the lens that we should be looking through any kind of.

Prescription progress are there.

It's going to be able to fogging sort out what for sure.

Okay. Thanks.

Thank you next question comes from Laura Chico Wedbush Securities. Your line is out there.

Hey, Thanks, very much for taking my questions I'm I guess, one on sometime before I I know you're saving detail for later, but could you just talk qualitatively, perhaps to the degree of PTC depletion in the peripheral blood versus scan I guess any distinctions that you might call out there and then you kind of mentioned the follow up.

But any insight thus far into the durability of action and then I have one.

Hello.

So the.

You see depletion is quite potent both in peripheral blood and in the tissue most patients depletes into peripheral blood to a nickel and she's near complete depletion in the peripheral blood and we see the same thing in the tissue as well or we see near a completes a reduction of pdcs industry.

Biopsies when compared to baseline.

And that is true even in a cohort to despite the fact that you'll get clinical outcomes seem to be better and call. It three that in cohort two but well the PD point of view that was already quite a quite good effect and core too.

Wonderful I guess, one follow up on four nine to zero.

Could you just talk to the level of steroids usage in the patients that have more symptomatic disease and I guess, if you could just remind us how are you looking at or are you evaluating steroids bearing effect.

Went forward I too, though is dose there.

So I'm sure there's really not part of 'em standard of care in patients with with children. So steroids will not be onboard and the trial and it's also got coming to use.

In the real world because it's just simply doesn't work very well. The same is true for many other immunosuppressive drugs that have been tested enough of it found not to work, particularly well I'm. So they're worth bearing is obviously very important in diseases, where there's a lot of 'em steroid use such as lupus and and other conditions.

It is less critical in disease like children syndrome.

Thanks very much.

Thank you. Our next question have around cells or where are you on H.C. Wainwright. Your line is open.

Thanks, very much for taking my questions. The first one is for Bill I was wondering if you could elaborate on what virtual event than four I you expect to highlight I never let Matt in post the approval decision.

Yes. Good question, we're still evaluating somebody options there as I mentioned before we are doing some a webcast of Facebook live events that had been very successful and while they were targeted initially to patients. We've also heard from physicians that they were very helpful with some basic understanding.

We're doing virtual detailing as many people are mid disease State perspective, we'll continue that afterwards, and we're looking at other options as well too that seem to be affected the kind of breaking through the noise given that many people are now in the virtual setting or trying to go after a similar position basis.

And can you comment on the rise of Tele medicine, and whether that's likely to have any potential near and or medium term impact for that matter on the I. Nevertheless, mad launch dynamic.

Great question, I mean, clearly we're seeing a lot more tell others, it's going on right now I'm talking with positions. They do feel that that is kind of an anomaly that that that will go down significantly once things start opening up.

But there may be some role for that for more follow up visits and anything else. However in the research. We've done a physicians are very concerned about there in a must be patients considering that any attack can be so severe and disabling long term firmly disabling. So they are more likely to try to to get their patients in front of them to do a full of.

Calculation, which is really more difficult to do in tele medicine that it wouldn't be face to face. Obviously, so we don't expect to have a material impact on our expectations right now with the telemedicine that's going on currently.

Great. Thanks, and then the next couple of questions are for Jordan I was wondering if you could maybe provide us with some additional.

Granular what steps youre, taking in the ongoing trials of I know, but listen up and 49 20 to ensure that patients are not lost a follow up and then if you have any sense of with what timing enrollment might resume in the phase two trial up I know it was not for kidney transplant decent ization and the trial for 49 20 in children's can go.

Where and I as I understood enrollment has.

So far been Paul.

Right.

So we really haven't had a much issue with loved what losing patients do follow up so the largest group of patients that we currently have active in an ongoing clinical trial is nemo patients in the open label trial, we have thus far enough really seen any drop off or.

And he missed visits as a result of Workover. There are two other 12. So were 49 21 in rheumatoid arthritis, and one in Sjogrens, where there's a small handful of patients were enrolled prior to pausing enrollment as due to the cobot crisis and Ah Similarly in those patients they.

I have continued to come to the 12 15 have completed their visits I'm on time, and Weve been able to conduct dosing visits as well as a follow up visit so that didn't seem to be much of an issue.

The the timeframe when we think trokendi resumed its difficult to predict and it's going to be difficult, it's gonna be different and by by region. So we are right now in contact with frequent contact with the investigator sites are talking to them to understand what their local situation is what.

Institutional policies are on whether or not clinical research is on hold and you know how the epidemiology is developing their various locations there clearly some locations where depends on it because on the Wayne.

These investigators of these regions are ready potentially to resume screening and enrollment in the reasonably near future, perhaps as early as as of June or July and then there are other regions, where via the Pacific or the epidemiology is still very much on the rise or remains.

At a very high level and so those regions on not anywhere close to close to ready to resume clinical research. So it's good the answer is going to be different and in different parts of the world of even in different parts of the country.

Okay. That's very helpful. And then just very quickly on 70 734, you mentioned that there could be multiple phase two setting, which this molecule might be explored I was just wondering if you could give us an idea of specifically what you consider to be the highest priority ones are within the phase two context specifically.

And whether or not you think any of those studies might potentially start before the end up this year or if we should be looking towards those as potentially 2021 stocked event.

Oh, So I think we're as I said earlier, we are still really in the process of a prioritizing indication. So I think at this stage, it's a little bit premature to gives you like to prioritize list with respect to two trial started I think next year, it's more likely.

Thank you.

Our next question comes from that team and yet it under the how your line is open.

Hey, guys I appreciate the opportunity for question Congrats on all the problem. That's just a couple of question on enabled as a map ops fund.

Could you guys comment on de levering up a nation Monday and pod would do you I below towards on the nibbling on is the best guess still I'm gonna before the C. the positive patients, which is about 80 or send them them onto <unk> can you just common what sort of thing to actually be handle.

There's no updates to share on this at this stage.

Okay, and then on the other European fund or any update in terms of European doesn't occur discussions.

Yeah.

Okay, that's exactly right on that partnership.

We are you actually discussions with potential partners.

Second Hawaii's on that fighting.

The funny.

Still.

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Turning to file a video this year.

No and then just on the launch down on a you know given that you have convenience advantage relative to the new drug that has been out there on the markets to live right can you talk about how you watch jumped up to leverage back is there any new market research that you have conducted.

And any new learnings now relative to the peak over they bought minimum thumps up convenient and then the other part of the of the question is talk about the willingness of patients to start page Oh physicians to start picking on a b cell depletion therapy in the covert Enlightenment, Wendy's, Ted it which tend to be.

A little bit more immunosuppressive I was just talk about these dynamics if you could thank you.

So a it's a very good point clearly one of the advantages that we have a against a the existing therapy. That's out there is a dosing advantage and I think that.

It is something that I think resonates well with physicians, given especially now that patients do not want to go to an institution to get infusions, but clearly that is one of several elements of our product profile, it's very attractive to physicians.

The safety the efficacy the multiple efficacy endpoints that we have along with the familiarity of the mechanism of action for all strong elements here that really come combined together to give us a very strong overall product profile and one that physicians have told US a is very attractive to them for their animosity patients both their new patients and for.

Patients, who would be thinking about switching so while convenience may play a little bit more of a role short term. We feel that that is you know one of many elements that we have going for us a in our positioning of this product.

You want to talk about B cell therapies and cobot, yeah. So I think in this disease, it's quite clear that's patient need to be on some immunosuppressive immunosuppressive regimens I'm, giving their risk of attacks with a potentially devastating consequences on the tax I think theres consensus in the community that's sub.

Type of a immunosuppressive regimens must be instituted and.

That's really not great data at the moment.

Whether I'm certain regimens are more immunosuppressive or are associated with a greater risk of covert than others.

It's actually a numerous efforts are ongoing currently to better understand as well, including an initiative for the Cathy Jackson Foundation I'm a survey of of experts in this field from our discussions with experts we did get the since that a lot of people think that Oh that does not appear to be an increased.

Risk of of Cobot in patients who are currently on our BLT Peter So the data still quite scanned and we're looking forward to getting better data on this but that is sort of the good stuff that we're receiving from I'm talking about this exact issue with the with experts in the field. So that does not seem to be a whole lot of.

Oh, I mean, there's certainly some concern, but there's not a whole lot of data to suggest that a b cell depletion is associated with any particular was there.

Got it and just one final question, but you want to pad upfront or specifically as it relates to enabling the ma'am I'm, giving that during the cold environment are you able to I'm sort of conduct all that required meetings will work one or are there any required meeting that needs to be conducting imports and I'm just from Paris.

Perspective, thank you.

Great question, clearly a lot of the means move to a more virtual environment or the nice thing is from a payer side of things a lot of the meetings already were virtual so we have not seen an impact a real impact on the frequency or the quality of the meetings you've had in discussions with payers.

Thank you very much.

Thank you Sir I'm showing no further questions like kind of call back of the management for any closing remarks.

Yeah I just wanted to I think you know again.

I would call today, we appreciate that quick questions you asked.

Have a good evening and I'm pleased to be sounds like you have any additional questions. Thank you.

Ladies and gentleman that that concludes today's topic. Thank you participating you may all disconnect have a great that.

[music].

Good afternoon, ladies and gentlemen, I want to they deal with our first quarter 2020, <unk> earnings Conference call.

So all that's on a listen only mode.

They don't get asked the question answer session and instructions given at that time.

As a lot of this call the call if they recorded.

I would not the helicopter Albertsons Mr. Mitchell trailer Chief Financial Officer, I'd be El Nio. Please go ahead.

Thanks, and welcome everyone to our first quarter 2020 earnings call.

That's really supporting our financial results, it's available on the Investor and media page on the web site at Triple W. Dot feel about dot com.

Joining me on this call. This afternoon, our CEO, our chairman and Chief Executive Officer.

Guard dropping our chief Medical officer, and build targets Vice President had to commercial.

It's pretty minor, we'll be making forward looking statements regarding our financial outlook. In addition to regulatory.

Development and commercialization plans at research activities.

These statements are subject to risks uncertainties that may cause actual results to materially differ.

Those forecasting a description of these risks can be found in our most recent form 10-Q on file with the FCC.

I'm not too many companies and about pharma sector very closely monitoring to growing up endemic including the associated restrictions on travel and work that had been implemented.

That's well its and its potential impact on our business and the trials.

Central which the krona virus impact.

Well just depend on future developments, which are highly uncertain I cannot be predicted including new information, which meet emerge that journey. The severity of the krona fires and the action to contain to current affairs work it treats its impact amongst others.

Now I'd like to turn the call over to bring down our CEO.

I think I mean, it's good afternoon, everyone. Thank you for joining US today I'm pleased to report that we had yet another productive quarter, an opt out very strong start 2020.

With that participate if I will lead product candidate I know he's a map approaching about a one by one year, we our fixed the company a milestone to date, our trust potential U.S. regulatory approval.

Why this would of course be a pivotal moments for our company well importantly, you or would it be our victory, but the thousands of patients affected by you all my likes I'll pick a spectrum disorder or a on the west the.

In parallel to launch readiness activities, we continue to make Saudi depart Wes across our entire pipeline.

Most recently, that's usually healthy this afternoon.

Well I was surprised me, saying you know movies jobs from our ongoing piece one be trial, we I'd be 7734.

And it just several months ago, we initiated a phase twob trial I'll be I'd be 49, 20, well that's getting to know patients with stripping single I'll comment rheumatic diseases for which there are currently no approved disease modifying therapies.

Energy and if we stopped when it's all of our teams are incredible despite the public 19 pandemic, which is affecting Autobots today.

I like to take a moment to address outcome because it was forced to the pandemic of course in the fall most our thoughts how would that thousands of patients and their families who had been directly impacted.

Thanks to healthcare workers happy to Kip <unk>, all safe what your monthly grateful.

To that extent that I'm kind of how contained a spread of the wireless and to flatten the curve. We continue to be nimble allergy season make you.

And I wish that up the outbreak we moved quickly to protect and to support our employees added a patient that we spoke implementing a work from home policy and successfully transitioning into virtual external stakeholders engagements.

While it's still too early to tell how impactful. This pandemic will ultimately be at present, we have been fortunate we experienced minimally facts, albeit an operations.

Have sufficient supply of drug product to supply, our orange and to continue our ongoing clinical trials.

However, we have voluntarily party almost all the patients the some of our trials you know what are the prioritize patients health added that I'll begin that theaters and I'll clinical trial sites.

How many weeks at him, but well continue to monitor that penetration carefully enter photo guidance from local and federal health authorities.

Despite some adjustments I would they be operations if they did it by a couple 19 pandemic well positioned for success in twentytwenty and to be joint.

Had a precedent Oh R&D is focused on three distinct signaling pathways or anybody because they literally and it makes that guy pathways that I know I range up autoimmune disorders.

We believe our approach, which puts she was how pipeline we think a product like you need watching a lot more precise candidates that have the ability to create what would you be.

Additionally, our research group continues to study and <unk> when do you pathways.

Yes, our brief history, well build a robust pipeline and today, how five like yours give out in but the reality the wage Nick.

Oh eating near term milestone we use of course, the potential FPL global of I know, there's about amble ASP.

<unk>, where do you worry if I'm through disease that affects about 10000 patients need that United States or no.

We know that about 80% off a M. A C patients have already anybodys toward our channel protein called I'll, probably a fall. This all the anybodys produced by that's not blast and the blast myself that abide primarily to astrocytes central nervous system.

Finally up or well order anybody's still central Europe systems House, it's believed to kriegel attacks, which it damage the optical Europe spinal cord.

Great.

Hello, I like what do you have nice has declined significantly with just one will attack.

Hi, many patients will come to you, but what's what blindness, and a paralysis and experienced progressive it wasn't able to disability with each subsequent to attack.

We see that devastating disease prevent your future attacks and limiting disability crucial to patients health.

As you may recall.

We designed I'd be some have to target <unk> 50 might be expected b cells, which include that's not matched and that's much else.

Based on strong efficacy and safety was out from our pivotal and momentum Kyle will demonstrate that ticky tack reduction as well as reductions worsening of visibility.

It's actually a U.M., our leadership and a reduction in ammo SP related hospitalizations, we believe that itll be them back as a potential <unk> monotherapy for the patients.

I will open label extension period, Heskey insights into how I look at the map might work long term.

Let me duration of treatment. These two years, there's some patient on the map opt to five years, we've talked a consistent with that of the primary analyses and over 80% of patients remain a tax free.

So we are highly encouraged by the potential long term benefits.

There's a map.

With that I like to handle the call but to be a week. It's our head of commercial what provides more details about our launch strategy and the readiness Bill.

Thanks, Ben as you've just heard with our PDUFA date of June 11, now just weeks away. We are soon approaching a very exciting and momentous time at our company.

As this would be our potential first launch we had the unique opportunity to hand pick a team dedicated exclusively animal westie.

To support the launch Viola has assembled season teams, including myself market access in sales representatives each team averaging over 15 years of experience the breadth and depth of knowledge of these teams is really impressive.

The cobot 19 pandemic has forced us to face some unique challenges specifically travel restrictions and limited physical access to our customers.

However, this has not been a major impediment to our progress as we were able to quickly pivot to remote interactions and alternative ways of disseminated disease education.

Based on our conversations with positions, including key opinion leaders as well as managed care providers and patients. We believe that I nimble isn't that meets many of the needs that our customers have in treating and I'll, let Steve and we are confident in our ability to reach those who could benefit most from it.

Importantly, despite other approaches on the market or in development. Many physicians remain very interested in b cell depletion and we're continuing to expand our educational efforts.

We're also strongly focused on the patient journey and our patient advocacy team has been working closely with animal once these specific advocacy groups, particularly the guffey Jackson charitable Foundation, the Samira Foundation, and the Steagall rare neuro immune association to better understand patients' needs and provide high quality.

<unk> disease education.

Sure. This then we have sponsored a series of podcast to help share knowledge around animal SD spanning from diagnosis and treatment the quality of life and what it feels like to live with a condition.

We have heard from patients that this has been immensely helpful. In addressing many of their key questions, especially around the management of animals, Steve in the face of cold in 19.

Looking ahead, you find a blessing is approved we'll be focusing on newly diagnosed patients and those experiencing inadequate response to their current maintenance regimen.

Our aim is to help prevent future animosity attacks and prevent further worsening of visibility.

We believe that I nimble as Mike will provide significant value to the animal rescue community and our goal is to ensure patients have access to this important medication.

While our immediate focus has been under development and potential commercialization and then I'll, let Steve we continue to evaluate the potential broader role of cannibalization mob in the auto antibody pathway.

With that I would like to hand, the call over to you weren't drop off our chief Medical officer to provide an overview of the current and future development plans for on it but listen that as well as a summary of our progress in our other clinical programs.

Thank you Bill.

We'll get into a little over them, all and just a moment, but I would like to start.

As part of the earnings call presentation.

Well some data that we have just oh recorded today. That's it involves yes 77 weeks before.

Let me something Southernsun for is one of total everybody's problem is designed to target an antigen that a specific exposed.

So it sounds are subject to split itself.

So the played a multifaceted rolling inflammation.

And that a basic level they are important and activating upsells and also contributed.

No problem settlements the initiatives with patients in patients with other diseases for.

For example in skin biopsies lupus can you can see large enrichment of Ptcs. Similarly Olson biopsies.

The other diseases such as myositis.

If you felt the need to sounds in large numbers of clubs to produce a lot of cytokines and maintained inflammation.

Dishes.

Well <unk> offices that are depleting cells potentially interrupt the pro inflammatory cycle.

So as a reminder, our phase one trial involves three cohorts first cohort, including patients with the mix of just with auto immune diseases, such as lupus juggling systemic sclerosis.

And the secular cohorts enrolled patients with continuous does go up to contain 12 patients.

The 11 patients. So the goal was to test whether.

We're not only be.

The circulation, which we already knew from Barclays. While they trial, but also inflamed tissues and to that and we took skin biopsies from these patients with 10 years Lukas before and after treatments is up because actually.

So you know more rate Oh, the peak seasons before and after treatment.

So this afternoon, we reported on PD interim data from cohort two as well 60 and clinical outcome measures from a subset of call. It.

Pardon me upcoming disposal safety.

We saw that so right now adverse events.

This was comparable between active and placebo patients there are no unexpected safety findings.

Also so that's in cohort two.

Yes, because it's having a free float was <unk> eight.

Do you see in the skin biopsies of patients with Sealy after treatment of treatment.

Finally, we found the dose dependent improvement and the clinical outcome measure.

It's called the classic continued scoop area and severity index, which is basically misery.

Activity, that's getting rashes.

The placebo control.

We look forward to report a final results from this trial, including.

Both from corporate greed, which we anticipate towards the end of the third quarter.

We have now in the process of preparing for phase two studies in one or more indications little thought to be driven in part by you and the pro inflammatory mediators they produce.

Okay, but there's a lot we have made progress on all of us cycle.

In most geographies I did you forward into disease.

So as you know we are very potentially pivotal trials both of these indications.

We have submitted.

As well as scientific advice.

Similar things in countries outside the United States.

Obtained feedback from regulatory agencies incorporated.

And so on clinical plans and protocols.

Finally, let's politically currently in the process of study up sites with the goal of starting a pivotal trials in both of these indications on towards the middle of this year.

Good luck course uncertainty second being leather clinical trial sites will at that time will be ready to enroll patients.

As a result of Colby let Jean.

Currently we are still expecting well to start these trials on time.

Lots of Youre familiar with my senior gross.

So there are inflammatory disorders who's not the differences on has a number of similarities with and it'll spectrum disorder is also mediated by apologetic all the antibody.

Results.

Well the muscle weakness other electrical systems.

I'd you before related disease is.

You got a sparked by two or like swelling in fibrosis or various affected organs, which has caused by infiltration, you'll see that you've expressed in plasma cells.

But generally edgy for antibodies.

Many different organs and nobody can be involved commonly involved or include.

Yes, I celebrate plans, which are prepared to dealers.

Well so both of these are important syndications bombed out the impact so.

Relatively large numbers of patients and so we really looking forward to starting these trials in these are too much second indications.

Finally hub, so I'd like to address these 49 20.

That is our fusion protein that's designed by people can they get them activated T cells blocking the interaction to see before expressing cells.

Let's see what do you see for the Lincoln pathway as a key co stimulatory.

That is required for the activation of T cells, but also plays other important biological wells.

As you know we have initiated seems to be trial in patients with certain syndrome.

This is the second most common robotic disease after a little light ensign is characterized by.

But with an eye dryness, that's can also results.

Fatigue, chronic pain and other suspended.

Patients.

Additionally trials.

And that focus on patients.

Hi, systemic disease activity and we are also included in these patients and knowledge file, but we're also looking at will be another population of patients who have less specific disease activity, but the house symptom burden. So.

The dry mouth fatigue I was just symptoms.

We dose the full stationed in displayed at the end of 2019.

Enrolled additional patients in early 2020.

Those who continued to be followed and I'll follow up we voluntarily Paul enrollment of patients due to the cold like.

Pandemic.

We are continuously evaluating the situation that's different participating sites and countries.

Look forward to resuming enrollment as soon as they see possible.

With that I would like to turn it over to the Shanghai <unk> Chief Financial Officer.

Thanks, Sean what I'd like to refer you to the press release issued earlier today for a summary of our financial results for the first quarter ended March 31st 2020, and take this opportunity typically review a few items.

On a quarter or R&D investment totaled $26.8 billion. That's we continue to advance our pipeline.

For a screen and eight we invested $15.3 million into first quarter, which includes our U.S. precommercialization investments or I never lose about animal at state.

And all our total operating expense for the first quarter was $42.1 million.

Letters with our other income.

Operating loss for the quarter ended March 31st 2020 was $40.8 million, which translate to a GAAP net loss per share of 80 cents for Q1 2020.

Our cash we ended the first quarter with approximately $335 million in cash cash equivalents and investment.

With that I'd like to ended over back to bank.

Thanks, Mitch. Thank you again for joining our call today as you have heard we had a strong quarter and how well positioned productive and so thats why you go ahead.

We especially look forward to all because the near term milestones that potential FDA approval and commercialization of I know, there's a map, which would it be up for us for our company.

Many thanks to our teams for their continued how to work Asha this forward.

Addition to that development up several other pipeline molecules.

Now I would like to open the call two questions operator.

Thank you.

Ladies and Gotta look like as the question. Please press Star then one on you touched on telecom. If your question at the answer which means that MCU. Please breakfast how Q.

One moment please for our first question.

Our first question comes from Seamus Fernandez of Guggenheim. Your line is open.

Thanks, very much for the questions and congratulations.

Well I data.

Yarn My one question there.

I missed the a little bit of the data that you mentioned there in terms of the.

The differential between the arms on the Causey score. So I was just hoping you could clarify that and then as you think about the that the overall development approach or 773 for can you give us your sense I know yarn, you've you've studied a lot of products and move that's in the path.

Yield it at least as early data.

No sort of winds up to some of the other therapies that that you've seen in clinical development and then just a quick clarification.

Were there any was there any evidence of a a meaningful increase in or be a related reinfection. Thank you.

Okay.

Thanks for your questions during the with respect to the cloud.

We didnt observe dose dependent separation between active and placebo there was a little bit of post separation in cohort two there wasn't much clear separation and core three I'm not going to present, a numerical values today, but suffice to say I'm. There was a very clear separation between the active and placebo.

We're currently in the process of prioritizing and designing phase two trials for this molecule in lupus is clearly included in the in the list of is high on the list, Yes, you're correct, we have sufficient experience and conducting lupus trial. So we know I'm the challenges and pitfalls, but we also.

Think that there is substantial remaining unmet medical need in this indication. It's currently still only one biologic approved which has relatively modest efficacy and some is still.

The square Ptcs.

To play a role.

And with respect to your last question on her because we did not see any increase any increase in or because infections or reactivation.

Great Congratulations again, I'll I'll jump back in.

Okay. Our next question comes from JP Morgan Stanley Your line is open.

Congrats on the progress thanks for taking my question.

I get the person how many patients where in cohort three for the interim analysis.

And then based on the current data do you think you capture that therapeutic window or would you want to look at even higher dose getting that the safety was comparable to placebo. Even then coordthree and then I've a follow up.

<unk>.

So the.

The the ends in the cohorts were age for code 112, and cohorts to and 11 in cohort three.

One of those 11, there were I think.

Nine evaluable in that they had a pass the the time of the primary endpoint, but Beatty five.

So that's just the data that we've included in the interim analysis.

<unk>.

Oh, yes for don't think so we still.

I haven't completed our modeling so we will inputs the data into our PK PD efficacy model.

We will wait until we have the complete data for the available to Vega.

Definitive sort of statement or decision on the on the on the right dose it seems I'm from the preliminary data that the the a higher dose or that was tested.

Had a bigger impact on the class.

But you know whether or not this is the the peak or whether or potentially a higher doses needs to be looked at I think we will be guided by the PK PD modeling.

Great. Thanks, and then you've indicated that upon approval.

And then goes in that front in though you're able to begin the commercial launch shortly after some companies have decided to push off launches later in the year given the challenges in the kind of environment, including like lack of face to face interactions and and lower patient visits. So can you talk about your decision to launch shortly after approval and what gives you confidence that the current environment won't be significant headwind your launch.

Thanks.

Great question you. Obviously this is a dynamic situation, we're dealing with nationwide. The cobot 19 pandemic has affected different parts of the country very differently. The good news is with our experienced sales force. We have some very good relationships that we think we can rely on for a different parts. In addition, we.

No that there are parts of the country that are opening already we've done some research recently to understand little bit about some regional differences in the regional plans will be very different depending on if your hotspot or not.

So we think that we have a product that is meeting a lot of the needs of our target audience and we think that we have some relationships that we can count on to have meaningful interactions with physicians after approval and therefore, we think the best a path for us is to get the product to patients who need it as quickly as we can.

Great. Thanks, a lot.

Thank you. Our next question comes from Paul Swinand Goldman Sachs. Your line is open.

Hi, this is changing hands on for Paul.

So as you think about the heterogeneity in lupus disease can you just talk about if there's any settings, where are you seeing PDP activity and its depletion make more must says that the therapeutic approach.

Yeah, so you're perfectly correct and pointing out the what are the key issues in the in drug development into this is heterogeneity heterogeneity, both in terms of clinical manifestations, but also potentially.

With respect to underlying biological pathways and are we doing actually a lot of work disentangle. Some of the heterogeneity. We're looking at several biomarkers that are associated with the Bdcs, we're looking at.

The interferon signature relocated a fair unregulated genes expressed in into skin as well and so we tried to correlates to the extent possible in is relatively small trial. These biomarkers with the clinical outcomes that we have observed. So this work is still ongoing and we hope to leverage to.

Potentially be able to stratify or [laughter] or focus the.

<unk> Jude populations to include those patients who were both elected to benefit.

Yes.

And then when you think about Sjogrens engagement in particular this population that maybe had high September and but less disease. However, kalo, then maybe patient I agree to think about what their most important or will it be clinic clinically meaningful there and then kind of at the follow up to that what's the size of that population how does that compare to the.

More severe grid.

Yeah. So if you ask patients I think you will consistently hear that fatigue is on the very much of the top of their list of concerns a follow up eye dryness over these patients are very.

Unpleasant symptoms caused by drive dry mouth dry eyes, and I'm, you know absence of both decreases in other words as well.

So I think these are the the key patient reported outcomes that need to be captured in the way. We recapturing patient reported outcomes is over the validated instrument difficult D. S. Three and that includes many of these things.

The the but the number of patients and the high symptom a burden subgroup.

In the trial is.

I catch I can't remember it off the top a bad but I think it's.

Uh huh.

No it's.

Roughly equal size I think it's a little bit larger than.

The size entity.

The the size with a with a high some with the high disease activity as a roughly equal but they will be under my separately on the one with an objective outcome the USDA and one with a patient reported outcome. The S. Three.

And does that child kind of correlate well to the real world population differences and what percentage of patients has more than the original died.

Okay.

No. So in the in the real world the population, but there hasn't been burden is much larger than the one with a high disease activity, but.

The the trial size is more driven by statistical consideration to go to buy power considerations and by input performance rather than to reflect the real world.

Then he thought it and thank you so much.

Our next question comes up question look panel.

Cowen Your line is open.

Great. Thank you very much congratulations on all the progress.

Two categories. The question first on seven seven tree or when we see additional data later in the year can you give us a sense for what kind of how many doctors and what kind of hollaway. My follow up you might expect and also we could be sharing with us more specifics or can even comment now about the patience I believe from an enrollment criteria.

Standpoint could have been though is with them without systemic lupus and of course that cutaneous can have various types of manifestations of the kids persist chronic any sort of.

You know characterization of the types of patients that we'll see would be helpful. As we all began to be slightly knock out but this could look out from a market opportunity standpoint. Thanks.

So the the trial included patients both with and without systemic lupus.

Lupus, but they have to have two tickets, but at the station they they could've systemic lupus or just your JV and in the trial it was approximately.

Our path those patients with and without systemic lupus.

And then in terms of what will we see at the end and the final analysis for the final analysis will include the P.D. analysis, all skins and biopsies and.

Cohort three.

So these Bob will be processed when the last patient and cover three has had there day 85 I was getting biopsy and then is as good buses will be process and analyze which takes a little bit of <unk> and then at the end of the trial, we will have a complete data with respect to safety.

Clinical outcome measure of and a PK PD biomarkers for all three cohorts patients. The for the primary endpoint is assessed that after three month at 85, but there will be additional safety follow up at least through day 140, Oh for all patients and depending on the recovery kinetic.

Oh, I'm pdcs potentially be young, but some patients.

Great well look forward to that and then on the Netherlands, a man just to make sure that were up to date, obviously very close to that they do have said date I think the last time you communicated publicly.

You felt comfortable and expressing that you do not expect a black box and as far as any last minute type labeling discussions I was there anything to share with us in that regard and then secondly, if you do launch help us get a sense for what you think we'll be able to measure what kind of metrics will be out there in terms of another prescription data.

And that this is a specialty product, but have lost are we going to be as we begin to try and figure out how the initial launch is going in this environment. Thank you.

I'll take that one on label so there's no updates to share no status changes since our last.

Call and then I'll pass it over to Bill for the second part of the question.

Good question, obviously as we've talked about before from from a focus perspective, we're focusing on both newly diagnosed patients in patients who are having inadequate response to their existing.

Immunosuppressant therapies are on their roughly 10000 actionable patients we've seen in the marketplace and we'll be going after the key institutions along with some of the community doctors, who were seeing a high number of patients as well too as kind of our priority focus at the immediate launch period again as mentioned before in one of the last.

A question this will vary probably significantly across the nation, depending on the status of opening up a with a different areas.

But we expect to be able to have good relationships and good discussions with our key targeted doctors right after launch.

But in terms of third party prescription tracking data et cetera, we're going to be.

Kind of navigating our way through a bit of a fog is that fair or is there anything that you would comment in terms of.

How well the lens that we should be looking through any kind of a prescription progress or data.

It's going to be able to foggy to sort out what for sure.

Okay. Thanks.

Thank you next question comes from Laura Chico Wedbush Securities. Your line is out there.

Hey, Thanks, very much for taking my questions I'm I guess, one on sounds entry for I. I know you're saving details for later, but could you just talk qualitatively, perhaps to the degree of PTC depletion in the peripheral blood versus the skin I guess any distinctions that you might call out there and then you kind of mentioned the follow up.

But any insight thus far into the durability of action and then I have one.

So.

So the.

You see depletion is quite potent both in peripheral blood and in the tissue.

Patients depletes into peripheral blood to a new <unk> achieved near complete the depletion and the growth of blood and we see the same thing in the tissue as well or we see near a completes a reduction of pdcs into skin biopsies when compared to baseline.

And that is true even in a cohort to despite the fact that you'll get clinical outcomes seem to be better in call. It three that uncover too, but well the PD point of view that was already quite a good effect and going to.

Wonderful and I guess, one follow up on four nine to zero.

So I'm sure there's really not part of 'em standard of care in patients with with children. So steroids will not be onboard and the trial and it's also got coming to use.

In the real world because it's just simply doesn't work very well. The same is true for many other immunosuppressive drugs that have been tested enough of it found not to work, particularly well.

This was a lot of from steroid use such as lupus and and other conditions. It is less critical in a disease like Sjogrens Andrew.

Thanks very much.

Thank you. Our next question comes around Salvador <unk> why are you on H.C. Wainwright. Your line is open.

Thanks, very much for taking my questions. The first one is for Bill I was wondering if you could elaborate on what virtual events and four I you expect to highlight I never let Matt in post the approval decision.

Yes. Good question, we're still evaluating somebody options there as I mentioned before we're doing some a webcast of Facebook live events that have been very successful and while they were targeted initially to patients. We've also heard from physicians that they were very helpful. With some basic understanding we're doing virtual detailing as many people.

While our mid disease State perspective, we'll continue that afterwards, and we're looking at other options as well too that seem to be affected the kind of breaking through the noise given that many people are now into virtual setting.

Turning to go after a similar physician basis.

And can you comment on the rise of Tele medicine, and whether that's likely to have any potential near and or medium term impact for that matter on the I. Nevertheless, mad launch dynamics.

Great question, I mean, clearly we're seeing a lot more tell others, it's going on right now I'm talking with physicians. They do feel that that is kind of an anomaly that that that will go down significantly once things start opening up.

But there may be some role for that for more follow up visits and anything else. However in the research. We've done physicians are very concerned about their enema, let's be patients considering that any attack can be so severe and disabling long term firmly disabling. So they are more likely to try to get their patients in front of them to do a full of.

Calculation, which is really more difficult to do in telemedicine, then it wouldn't be face to face. Obviously, so we don't expect to have a material impact on our expectations right now with the telemedicine thats going on currently.

Great. Thanks, and then the next couple of questions are for Jordan I was wondering if you could maybe provide us with some additional.

Granular what steps Youre, taking in the ongoing trials of I never lives Nab and 49 20 to ensure that patients are not lost a follow up and then if you have any sense of with what timing enrollment might resume in the phase two trial that I never listen that for kidney transplant defense privation and the trial for 49 20 in children syndrome.

Where and I as I understood enrollment has so far been Paul.

Right.

So.

We really haven't had a much issue with lost a was losing patients do follow up so the largest group of patients that we currently have active in an ongoing clinical trial. This.

Oh patients in the open label trial, we have thus far not really seen any drop off or.

Any missed visits as a result of Covance.

There are two other trials so we're putting on 21 in rheumatoid arthritis.

One of Sjogrens, where there's just a small handful of patients were enrolled prior to pausing enrollment of due to the cobot crisis and similarly in those patients. They have continued to come to the trial cooking have completed their visits I'm on time, and we have been able to conduct dosing visit.

As well as a follow up visits.

Visits so that didnt seem to be a much of an issue.

The the timeframe when we think trokendi resumed its difficult to predict and it's good to be difficult, it's gonna be different and by by region. So we are right now in contacted frequent contact with investigative sites are talking to them to understand what their local situation is what.

They are institutional policies are on whether or not clinical research is on hold and you know how the.

We all energy is developing their various locations there are clearly some locations where depends on it because on the Wayne. These investigators of these regions are ready potentially to resume screening and enrollment in the reasonably near future progress as early as.

So June or July and then there are other regions where via the Pacific over the epidemiology is still very much on the rise or remains at a very high level and so those regions on not anywhere close to close to ready to resume clinical research. It's always goes the answer is going to be different and in different parts of.

The world and even in different parts of the country.

Okay. That's very helpful. And then just very quickly on 70 734, you mentioned that there could be multiple phase two setting in which this molecule might be explored I was just wondering if you could give us an idea of specifically what you consider to be the highest priority ones.

Within the phase two context, specifically and whether or not you think any of those studies might potentially start before the end of this year or we should be looking towards though as potentially 2021 stock events.

Oh, So I think we're as I said earlier, we are still really in the process of prioritizing indication. So I think at this stage, it's a little bit premature to gives you like to prioritize list with respect to a trial start up like next year, it's more likely.

Thank you.

Our next question comes from that team.

Yes, I mean do they have your line is open.

Hey, guys appreciated the opportunity for question Congrats on all the clubs I was just a couple of question on enable doesn't match up slammed could you guys comment on the level up a nation. When do you have had that would be right below tours on the leveling fun.

The base can still I'm gonna before the seed a positive patients which is about 80.

Or send them to market can you just common what sort of interaction is idle.

There's no updates to share on this at this stage.

Okay, and then on the other European fund or any update in terms of European regulatory discussions.

Yeah.

To ask why.

Position.

We are you actually discussions with potential partners like for a second Hawaii's on fighting.

Sorry.

Steel.

Having to file a video this year.

No and then just on the launch dynamic.

Given that you have convenience advantage relative to the new drug that has been out there on the market. So Larry can you talk about how you watch jumped up to leverage that is there any new markedly such that you have conducted and any new learnings now relative to the people where they bought minimum thumps up.

The convenient and then the other part of the of the question is talk about the willingness outpatient to stop page Oh physicians to start patient on a beach out eating therapy in decode Enlightenment Wendy's Ted It was tend to be a little bit more immuno suppressive.

She just talk about these dynamics if you could thank you.

So it's a very good point clearly one of the advantages that we have a against a the existing therapy. That's out there is a dosing advantage and I think that it is something that I think resonates well with physicians, given especially now that patients do not want to go to an institution to get infusion.

But clearly that is one of several elements of our product profile, it's very attractive to positions the safety the efficacy the multiple efficacy endpoints that we have along with the familiarity of the mechanism of action are all strong elements here that really come combined together to give us a very strong overall product profile and one that.

Additions have told us is very attractive to them for their animal rescue patients both their new patients and for patients who would be thinking about switching so while convenience may play a little bit more of a role short term. We feel that that is you know one of many elements that we have going for us in our positioning of this product.

You want to talk about B cell therapies and cobot yeah.

So I think in this disease, it's quite clear that patients need to be on some immunosuppressive immunosuppressive regimens given their risk of attacks with the potentially devastating consequences on the Texas I think theres consensus in the community that subtype of immunosuppressive regimens must be instituted and.

That's really not great data at the moment.

Whether I'm certain regimens are more immunosuppressive or are associated with a greater risk of covert than others.

That's actually a numerous efforts ongoing karthik to better understand as well, including an initiative by the Gulf Injection Foundation, a survey of of experts in this field from our discussions with experts we did get the since that a lot of people think that Oh that does not appear to be an increased.

Risk of of Cobot in patients who are currently on a b cells you. Peter So the data is still quite scams and we're looking forward to getting better data on this but that is sort of a bigger stocked up we're receiving from I'm talking about this exact issue with the with experts in the field. So that does not seem to be a whole lot of.

Oh, I mean, that's certainly concerned, but there's not a whole lot of data to suggest that b cell depletion is associated with any particular was there.

Got it and just one final question, which is on the pad upfront or specifically as it relates to enable is a man given that you got in the cold weather environment are you able to I'm sort of come down all the acquired meetings that were born or are there any required anything that needs to be conducting enforce and I'm just from Paris.

Perspective, thank you.

Thank you very much.

Thank you Sir I'm showing no further questions like turn the call back over to management for any closing remark.

Yeah I just wanted to I think you all against launching our call today, we know accretion definitely since you asked.

Good evening and I'm pleased to be sounds like you have any additional questions. Thank you.

Ladies and gentlemen does that include pay conference. Thank you participating you may all disconnect have a great that.

Q1 2020 Earnings Call

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