Q1 2021 ChemoCentryx Inc Earnings Call
Good day, and thank you for standing by welcome to the Chemo centric first quarter 2021 of the natural results conference call at the.
This time, all participants are in a listen only mode. After the speaker's presentation. There will be a question and answer the question to ask the question. During the session you will need to press star one on the telephone. Please be advised that today's conference is being recorded if you require for any further assistance. Please press star zero and.
I'd now like you had the conference over to Lee Roth of Burns Mcclellan.
Go ahead Sir.
Thank you Leigh good afternoon, and welcome to the Chemo Centrex first quarter 2021 financial results Conference call.
Earlier this afternoon the company issued a press release, providing an overview of its financial results for the first quarter ended March 31, 2021. This release along with the few slides that you may find helpful. While you listen to the call are available on the Investor Relations section of the Companys website at Www Dot chemo Centrex dotcom.
Joining me on the call today is Dr. Thomas Schall, President and Chief Executive Officer of Chemo Centrex, who will review of the company's recent business and the clinical progress. Following his comments, Susan <unk> Executive Vice President and Chief financial and administrative officer of Chemo Centrex will provide an overview of the company's financial highlights for the quarter before turning the.
Call back over to Tom for closing remarks.
During today's call, we will be making certain forward looking statements as explained on slide two.
These forward looking statements are based on current information assumptions and expectations that are subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in such forward looking statements. These risks are described in the company's filings made with the Securities and Exchange Commission, including our annual report on form 10-K.
And filed on March one 2021, you are cautioned not to place any undue reliance on these forward looking statements and chemo centrex disclaims any obligation to update such statements.
In addition, this conference call contains time sensitive information and accurate only as of the date of the live broadcast April 2009 for 2021.
<unk> undertakes no obligation to revise or update any forward looking statements to reflect events or circumstances. After the date of this live conference call.
With that it's now my pleasure to turn the call over to Tom Tom.
Thank you Lee and good afternoon to everyone listening.
And for joining us on our first quarter 2021 conference call.
It's been just 59 days since our Q4 and full year 2020 call So and my remarks today.
We'll briefly review three main areas first debacle of pin and anchor associated vasculitis, as we move ever closer to potential launch.
And update on our progress and making a bottle of pills on pipeline and the drug and third our plans for CTX.
And our oral immune checkpoint inhibitor.
Moving on to slide three I'll start with highlights from our virtual R&D day, which was held on April 14th.
This was held as we look forward to the potential launch of our first medication soon after the <unk> day on July seven, which we believed deeply offers new hope to patients.
Speaking of patients those of you who attended our R&D day on April 14, we will have heard and powerful and moving on account of <unk>.
And just like to live within the basket of items.
The person who is enduring the associated vasculitis, Mr. Glenn Massey described as long and winding and journey towards diagnosis, and then with the severe consequences consequences not only of the devastating disease itself, but also the consequences of the treatments currently available.
Let expressed the hope for and alternative having to take as inputs and.
Medicine on top of medicine.
Glen story is a vivid reminder of the desperate need for new therapy.
And as for Glenn is family and all the others and must endure the scourge of a disease, who have only current treatments that often trade one bad illness for another it is for them and we have.
Team of centric go to work each day and each night.
Make no mistake, we will not stop.
Non rest easy and we will not give up until the hope of abacus and is delivered to the debt.
And our R&D day, we discussed some new data as shown on slide for from the successful at the could trial. The information covers and eight week period immediately following the 52 week endpoint determination.
And which time patient stops taking about the plan.
During that period for weeks 52 week 60, again once people and stopped taking them off of and there were six relapses out of 158 subjects and the bottle and group and seven relapses out of the 157 subjects and the prednisone and group.
This suggests a waning of efficacy once about the pantry stock.
It was notable to that of key indicator of renal function and the estimated glomerular filtration rate for Egfr, which has steadily increased and the abacus and route from the startup studies per week 52, and then starting to modestly decline after a bunch of stock.
And stop.
These observations may support the idea of continued benefit of therapy, with the backup and and anchor and associated vasculitis.
Our R&D day also featured two world renowned and clinicians, who treat and kind of associated vasculitis Doctor and Peter Merkel share Rheumatology and director of the Pen Vasculitis Center at University of Pennsylvania.
And Dr. David Chang director of the Vasculitis, and Lupus service and added Brooks Hospital, and Cambridge, England.
Doctor Merkle laid out the unmet needs of business UBS disease. He pointed to the many different areas of the body, which anchor associated vasculitis and strike and patients.
And developing into and Oregon or life threatening disease.
Blaine and at the current therapy includes two months use of glucocorticoid and for too long and leading to patients.
And quite as Dr vertical put it isn't part of.
Plays are both the best drugs, we currently have and also the worst.
Dr. Jane summarized the potential for a box of pad to reduce the number of relapses, which would reduce the risk of permanent organ damage and reduce the cost of care.
Doctor Jane also emphasize the debacle of pens to improve quality of life is a remarkable and rare finding and anchor associated vasculitis studies.
Dr. Jane referred to the New England Journal of Medicine publication of February 18th see slide five.
The article on the advocate trial findings and received then nearly 35 hours and page views and putting it at the 92nd percentile of all New England Journal articles on.
Bad for an orphan disease during the pandemic.
Finally, and Dr. Gene described the market and some potential monotherapy for non severe disease, helping to prevent longer term relapse.
I would remind you that approximately two thirds of the total patients and advocate balanced across both the backup and on placebo arms did not receive rituximab or another immuno suppressant during the last 26 weeks of the advocate trial.
Why is this important.
Because and in fact, the last 26 weeks and advocate.
Allows for a glimpse of of Aqua and activity and a placebo controlled blinded fashion for some 200, plus patients and that period of the trial.
It showed apocalypse and efficacy and sustaining our emission and without the help of and Immunosuppressants such as additional writeups of Matt for Cyclophosphamide.
This may be particularly.
Really useful in times like the COVID-19.
During the Q&A session of the Doctor Merkle and Dr. Jane indicated that they saw debacle of pain as a potential maintenance therapy of viewpoint that is supported by the strong data to the 52 week blind the dosing period of the trial and also by the observations for weeks 52 to 61 of the drug was stopped as I mentioned earlier.
The New England Journal of Medicine article and the editorial that accompany the the new data on what happens to patients and the eight weeks after they stopped.
The strength of the case made like two of the world's top experts and above.
Above all the patients testimony.
All of these elements and suggest that the need for a novel therapy is urgent the.
Burden of disease is great.
And debacle per day will transform care.
Now we look forward to next week's may six FDA arthritis Advisory Committee meeting.
From the filing of our NDA, and we have consistently anticipated and ebb and preparing for such a meeting.
And the fact that of Aqua and its a new molecular entity and and orphan disease with only one previous and approved drug.
Our approach to the AD comm meeting is to deliver a high degree of granularity and response to the topics raised by the FDA over the course of the review in order to tell of the committee with its deliberations and the agency and the subsequent decision.
It is also an excellent opportunity to share knowledge of the significant complexities of this rare disease.
And one hopes to hear from patients whose lives have been turned upside down.
And <unk> associated vasculitis.
It is of forum and our view to underscore the strength of the African clinical data, which we believe provide a favorable benefit risk profile as a potential paradigm shifting treatment alternatives.
For anchor associated vasculitis patients.
Okay.
Well, we have now received the Fda's briefing book for the Advisory Committee and the document it's not arms of the Fda's and is confidential and.
Therefore, we will not be commenting commenting on it today.
However, we do expect the FDA to make the outcome of recent materials available publicly before the AD Comm meeting day.
We expected the F D a to pressure test our NDA as would be normal for any AD comm meeting.
As we said during R&D day, we believe based on historical discussions with the FDA that the AD comm topics may include the phase III trial design and understanding how income vasculitis is treated the ad.
And could trials statistics on assumptions safety reviews and benefit risk profile.
In our presentation, and we look forward to expanding our perspectives and our information to address the FDA questions. We on.
And our confidence and this approach.
We believe the clinically meaningful primary and secondary endpoints signals and consistently favor of Aqua and Eric.
Accordingly, we have confidence and the anticipated outcome of the Yadkin on me.
In light of this and pending meeting we will be entering into a quiet period. Following the conclusion of today's earning call activities.
While we appreciate that you will likely have questions ahead of next week's meeting and we'll likely have additional questions. Upon availability of the briefing materials. We hope you understand and can appreciate that we will not be commenting further on the income until after its conclusion on may six.
Turning now to our preparations for potential launch.
During R&D day, our Chief operating officer, Josh, but summarized our commercial plans.
If you look at slide six Tosh reported on primary market research and illustrated the potential market interest and debacle of fans.
With 97% of 125, Rheumatologists and Nephrologists survey, indicating that they would prescribe avago and if approved by the FDA.
And 100 percentage of them all of them, finding the abacus and data compelling either extremely seller monitor for yourself.
About the greatest unmet need and Inc of escalators. It is interesting to see the better safety fewer side effects were seen as the top priority for 47 per cent of these experts compared to 31% who choose better efficacy.
As you can see from slide seven and.
The market research study of 138 physicians, comprising mainly rheumatologists and nephrologists with a handful of other specialists, who treat inc. The vasculitis.
Which was conducted in April of 2021 that is somewhat after we released the data you can see 65% had heard Dan of a backup plan and.
And 89% and a positive impression of interest profile.
We are now raising the profile of anchor and associated vasculitis through our rethink income disease awareness campaign running on line and we think anchor dot com and print journals and at relevant the Congresses.
We have also held pre approval information exchange with payers covering roughly 85 per cent of the Yo.
And states and kind of associated vasculitis patient population.
The process, which is still ongoing.
Payers are receptive to a bump of pants and value proposition.
And we are building a high support capability to help individual patients obtain access to of Aqua and after lunch.
Since it typically takes six to 12 months for new medications to be listed on the electronic for Clarence.
Turning to slide eight.
We estimate that the eligible patient population for of Aqua and the United States is up to about 20000 patients of whom 8000 and form our initially addressable segments that is those who need it most namely patients who are newly diagnosed or relapsed and with <unk>.
Oregon or life threatening disease.
And sure.
We are well on our way to building a world class commercial capability.
We will be ready for a rapid launch if approved after the Paducah date of July seven.
Our partner <unk> for pharma is making commercialization plants also ahead of the expected regulatory decisions and Europe, and Japan, and the second half of 2020 one.
Bye for wouldn't pay us royalties from teams and the mid 20% on <unk>.
Potential net sales of one aggregate sales of long.
As you can see from slide nine.
Anchor associated vasculitis is just the start for a box okay.
We are moving forward on other fronts as we seek to turn a backup and into our pipeline and a drug with multiple orphan disease indications.
First slide 10 shows our planned debilitating skin disorder hidradenitis Suppurativa for Hs.
We are on track to launch by the end of this year of pivotal phase III trial of a bottle of pin and patients with severe Hs.
Our phase II of Aurora clinical trial demonstrated that and the most severe form of Hs. The so called early stage three patients of Aqua Pan could provide a significant improvement compared to placebo. After 12 weeks of there were.
We are following these phase II patients for an additional 24 weeks and we will report out those data when we had.
We expect the phase III trial to enroll of somewhere between 304 hundred patients divided into two arms. The primary endpoint will be the hidradenitis superb team of clinical response score for high score measured against placebo at 12 weeks consistent with the phase <unk> study.
And with the randomized blinded period and for a total of six months with an open label follow up.
H S. It's another indication with blockbuster potential for them on okay since and estimated to the 30 to 50000 patients and the U S have early stage III disease.
And filed for orphan drug designation for about the pen and early stage III patients.
Note too that were scheduled for a post recession and the society for investigative Dermatology and me.
Moving back the kidney disease, the results of our accolade trial of a backup and and the very rare disease of Siem reap luminary, Allopathy, which we plan to publish and at the top of Nephrology Journal included and an important finding shown on slide 11 of our.
Okay, and demonstrated significant improvement and renal function as measured by estimated preliminary of their filtration rate in Stark contrast of the background medication on placebo arm.
T F. R is what most nephrologist would regard as the gold standard for measuring kidney function and of finding like this it's never previously been seen and a randomized controlled trial patients received three dream.
We are preparing for a meeting with the FDA to discuss with the agency the evidence of clinical benefit of of backup and and the treatment of CPG.
At our R&D day, we reported and some new observations from week 26 to 52, and the accolade trial using the new C. III disease chronic city index as the graphic on slide 12 shows the Chronicity score and patients with the placebo Roes and the first 26 weeks of the study but the.
And kind of after these placebo patients and crossed over to a lack of it.
This suggests and effect of about the pan and arrest and fibrosis or scarring in and around the kidneys filtration units the preliminary of life.
This is the second clinical trial, which of Aqua Panis achieves statistical significance and improving the old function as assessed by Egfr.
The other being a neck of ESCO items.
This is a promising sign two for patients with other kidney diseases, such as lupus nephritis, and we can which can also lead to kidney destruction as depicted on slide 13.
Of Aqua Pan might offer of lupus nephritis patients the chance of a more targeted therapy and the broad immunosuppression and is currently used in an effort to control that disease.
Current therapy typically only provides limited benefits. So the patient need is great and we hope to introduce the box of pan into clinical development for Ela and during the second hands free.
Mainly as we turn to slide 14, a few words about plans for our next generation oral checkpoint inhibitor Ccs five five.
As noted on slide 15.
The <unk> 559 was featured in a poster session at the American Association for Cancer Research meeting earlier this month.
Current antibody based Immunotherapies have contributed significantly the cancer treatment, but not always without safety consequences.
Pneumonitis for example can be profound and the.
Non small cell lung cancer patients treated with antibody immunotherapy.
As we discussed during our R&D day on April 14th.
Lab data on our orally active PD L. One PD one pathway inhibitor of Ccs five five net suggest that is a small molecule and may penetrate the tumor environment better than and antibody and preclinical data showed excellent results and tumor shrinking and tumor remission and veeva.
Slide 16 outlines our plan to take <unk> of five nine to the next stage of clinical development and then how to evaluate its value proposition.
We expect to initiate the phase one b trial and cancer patients later this quarter.
I will now turn the call over to Susan to outline the strength of our financial position.
Thank you Tom.
Our first quarter 2021 financial results were included in our press release today and are summarized on slide 17.
Revenue was $10 4 million for the first quarter of 2020, one compared to 6 million for the same period in 2020.
The increase in 2020, one and the first quarter revenue was primarily due to the $10 million of milestone payments and buy for for this.
February of 'twenty, 'twenty, one and symptoms of Japan, NDA and for everybody and and the treatment of income tax guidance.
Research and development expenses were $23 4 million for the first quarter of 2021 compared to $19 3 million and the same period last year and.
The increase was primarily attributable to the manufacturer of commercial drug supply and anticipation of the launch of a thoughtful of patents and treatment of bank basket items and costs associated with the advancement of CCA and five five times, our orally available small molecule pinpoint and compare.
These increases were partially offset by loans things accumulated expenses.
The completion of patient enrollment of the of Funko Pan alone.
And patients with the jazz and the gist.
Continuation of the clinical development of CCA and split for CRM, and <unk> and 2020.
General and administrative expenses were $16 3 million for the first quarter of 2020, one compared to the point.
The same period last year and so.
Increase was primarily due to higher employee related expenses, including those associated with the commercialization planning efforts and higher professional fees.
He's real results produced a first quarter 2021 net losses of $29 7 million compared to a net loss of 21 7 million and the same period and 2020.
Total shares outstanding at March 31, 20, and 21 for approximately $69 7 million shares.
Closed the first quarter of 2020, one with 424 months.
And cash cash equivalents and investments and for 2021, we continue expect to utilize the cash and investments in the range of $145 million to $165 million.
Tom.
Thank you Susan.
To summarize the for.
First quarter of 2021 as.
<unk> positions us strongly for the rest of the year as you can see from slide 18.
We are well prepared for the FDA Advisory Committee on May six and.
And the potential commercial launch soon after the Purdue if the date of July seven.
We aim to meet with the MTA to discuss the clinical benefit of a box of patent for the treatment of CCG and.
We plan to initiate our next cycle of three clinical trials over the next few quarters first and then.
Human studies in cancer patients for our small molecule of checkpoint inhibitors and <unk> $5 nine.
A phase two to be adaptive study of the backroom Han and L N.
And the pivotal phase III trial of a block of pen and hidradenitis Suppurativa early stage three patients.
By the time I reported to you on our next quarter. The results. We may have taken the final step to becoming a forward integrated novel Therapeutics company.
And our first commercially available medications.
Again.
Make no mistake, we will continue to be implacable and our pursuit of new science.
And the interest of the patients the clinicians.
Clinicians.
And the investors that we serve.
I will now turn the call over the operator for your questions on.
Operator.
Thank you.
And if he would like to ask the question. Please press star one on your telephone keypad.
If you wish to ask the question simply press Star one on your telephone keypad and and the interest of time. Please limit your questions to one question and one follow up question. Your first question comes from Michelle Gilson from Canaccord. Your line is now open.
Hi, Tom Hi, Suzanne and thank you for taking my question.
Hum.
The the first one.
Out of the sort of anticipated and con topics that you outlined at the R&D day is there one that you anticipate will be.
And of greater focus and the others.
And I guess from a higher level.
Michel and I think that's it and it's.
Very good question of course, I think we've outlined the spectrum of items that we think will be.
And important there will probably be others as well other than that I don't think I'm going to speculate too much about the AD com at this point, because we're very close to it and it's really up to the FDA about what kind of questions are going to put to the committee. So we'll be ready for any and all the questions that they might pose.
Alright, and then you know just as a follow up what one of the areas that you.
Uh huh.
The debt you know we've heard some feedback on is the trial design.
And and obviously for for advocate the standard of care was what's different than it is on today.
When advocate was initiated so including the the steri regimen as well as maintenance therapy could.
Could you maybe walk us through how you think about the box here sitting in with the current standard of care and how it compares to the current standard of care on.
And you know it.
Our topic comes up and the AD Com and you know how and how you.
And to support I think.
Of occupants position.
And.
Okay.
Within the current standard of care.
Michelle I think Theres a lot of misconceptions about what is current standard of care of there and there are some facts that have changed since we started the trial, but let me put it the let me be very clear we tried to make the advocate trial as close to real world practice as possible.
And I did that include steroid regimen and tapering.
And then best practice and in fact, that's now of best practices.
We tried to make the background medication of either cyclophosphamide or rituximab as close to real world practice and in fact, we were completely.
Aligning ourselves with the label of all of those medications at the time, including where I touched on it.
So what we got was and excellent trial design and that reflected then real world practice and still to a large extent now the.
The one thing that has changed is right tux of Mab label was expanded and towards the very end of the advocate trial by the way to allow for increased frequency of dosing. So before and it was essentially one regimen at the start of the treatment phase, which is for and infusion space by one week.
And now you can top up of according to the label as frequently as every six months or so now while that is becoming the emerging paradigm, especially for patients that have a history of anchor associated vasculitis relapse the.
The latest figures I saw by the way for newly diagnosed disease I don't know if that's yet been adopted as the majority of paradigm. So I think while Rituximab slaybaugh has expanded and fairly recently.
I'm not sure I would call it exactly of change standard of care, but it is a very fair point to say well look bright tech some of the Jews ever more frequently.
And all of the better.
The features of the advocate trial actually allow us to leapfrog to what I think is the gold standard.
Which would be better for of maintenance therapy. Once people are quite the asset how would you do that trial you do a double blind randomized placebo controlled trial.
And as I referred to in my remarks de facto you can get some of that information from weeks 26 to 52 and the advocate trial for.
From the 65% of the people and that trial, who had of Aqua Pan plus rituximab at the start.
And then went on to have no further medication and as the trial and we're on so they didn't get tapped up with Rituxan and then what did we see we saw about a 71% sustained remission and the of Aqua Penn group versus a 50 middle of 50% remission and the pregnancy.
And on group and this is the right types of Mab strategy alone and it's not a tiny number of people its 200 plus people.
Bigger than the whole rave trial.
So what that tells US is that we are of very good way of maintaining remission.
After people get to acquire and state and kept that first 26 weeks and there isn't a need to give them the necessarily more of a touchdown.
And <unk> is not without its consequences.
It's at least and observation is it just the trial designed to answer that question no but is it a feature of this trial.
That does shed light on that question could of Aqua can be used as a monotherapy and the maintenance revenue and the answer as well and he's these data suggest that it could and that's what doctor Merkle and Dr. Jane alluded to in their remarks, and both international Congresses and Dr. Jain at the on the R&D day. So I think that's how best I would answer the question we did an excellent.
And we reflect standard real world practices at the time and still and the fact that we didn't add additional writeups and that actually gives the feature to our trial, which I think is very valuable.
And the again right text of Mab is a changing landscape it's not used.
Routinely I think even at this point for newly diagnosed people on the second six months most of the reserve for people with the history of relapse. So that's I think that's what I would put to the community at this point.
It's again the set of observations based on data.
Thank you so much for taking my question.
Thank you.
This week.
Thank you.
Your next question comes from Steve <unk> from Raymond James Your line is now open.
Hi, This is more of on a couple of on for Steve.
And thank you for taking our questions and.
And in terms of the close of the week 52 relapsed rates you indicated I'm not sure whether it's fair to annualize those rates from 4% over eight weeks to 26 per cent per year, but with that rate the comparable to what you would expect outside of the trial setting and can you also remind us whether you share this data with the FDA.
I don't think it's very at the analyzed the data because it's such a short period and and it's still fairly small so I don't want to over interpret that data.
So that's an important point.
And by the way I would say that when we talk about numbers and comparisons.
We're all trying to compare and number from study as the study be and we're looking at the numeral, but the definitions can be different and that really is of critical importance. So I would have to say be cautious about what we talked about with relapsed from study to study how is it defined.
So certainly and our study what was interesting is there was a moderate decline.
In the rate of sustained remission once of the people stopped taking of Aqua pen and it started to be similar to what we see and the prednisone.
And so that was an interesting observation, it's again and a weak periods still a fairly small number of people, but it was an interesting observation I probably wouldn't have made that much of it. If we hadn't also seen again a modest decline in egfr after a steady increase week by week from very early on and the <unk>.
While continuing to increase out to week 52, so again the suggestion is that.
And there might be neat on.
The benefit I, keeping people on tobacco and and I think it's really I'll limit my interpretation to that at this point and again without going into what would be and are you now on our briefing book per se, but at least that's the that's a document that we own I think it's fair to say that those data are the FTAA or aware of those data.
Yeah.
Okay. Thank you very much.
Thank you.
Thank you and your next question comes from data and haul from Stifel. Your line is now open.
Yeah, good afternoon, and thanks for taking our questions and very much looking for to the outcome next week.
But Tom I just wanted to go back to some of the additional data that you are that you presented at the R&D day, specifically and just wondering.
The highlight of the Rituximab cohort data.
And looking at sort of the debt.
<unk> monotherapy and as you just mentioned to the prior question, but.
Just wondering if you had done any additional subgroup analyses for example patients that do not have the renal involvement and I'm, just wondering coming from them on rheumatology and focus.
What about those patients that did not necessarily have the renal involvement what to do there.
And I guess the follow up question to that is.
In terms of flares.
Given that the trial was non inferior, but superior and the last 26 weeks. It wasn't 100%. So I would presume there can be some players that happened in the interim and any color you can provide in terms of what types of players where severity that you saw during that period.
Yeah.
Flares were defined I think fairly carefully in the new England Journal paper, So I, probably would refer you to that.
Rather than speak off the cuff, but to be clear you kind of not had.
The flare certainly over a certain set of criteria that would that would constitute.
The major flare you could not have had a flair between weeks 26, and 52 and be considered and sustained remission at week 52. So the fact that some 66% of the patient population and the abacus and group.
And nearly 67% and I recall on <unk>.
Thinking of coating on the top of my head versus the 55% and the Prednisone group. We're still in remission is considerable and as you said says statistically superior and the comparison.
Mind you mind also this is all based on intent to treat numbers. So that the we know that 10% of the people did drop out of the trial.
Or had.
Had a basically.
And.
And so they were not available for that analysis that takes us down from a 100 day 90 available as the top number it and get the ITT analysis and in fact, if you look at the numbers and additional 10% while still in the study had top Ted stopped taking study medication basically and both groups. So again, if you really had for whatever.
The reason, we wanted to say well if you looked at the number of people still on both study drugs over that period of time.
Again, the top number would be about 80, so it's kind of an interesting way of thinking about it but we have a considerable sustained remission rate clearly better than the pet prednisone group the.
Without getting into the subgroup analysis too much I will say this debt we look at all of the subgroups.
And especially when we look at the.
Some of the of the factors that you're talking about but essentially subgroups do consistently as well numerically or better than the prednisone group. They do that by not having to take the prednisone and know the daily oral prednisone and learn along the way and typically they also have and.
<unk> in the clinically relevant secondary endpoints when theyre on the box okay.
T F R.
Relapse rates lower et cetera, So we hope to be able to publish a lot more of this data as we get through the next phase of our development program, but I would be hard pressed to point to any subgroup of at this point that doesn't do at least as well typically better than prednisone based therapy and even if the <unk>.
<unk> were exactly the same which is atypical by the way for any subgroup, but even if they were exactly the same remember they've got there without taking the prednisone and load.
So they get the benefit of reduced prednisone induced toxicities and as I mentioned, the other clinically relevant and secondaries and also fall in line with that so the signals.
<unk>.
Show of the locker pin and benefit across most of these parameters.
Okay awesome, thanks, very much looking forward to next week.
Yeah.
Thank you and your next question comes from Ken.
Tim Hoff from Piper Sandler Your line is now open.
And much and.
I'll start by saying break the leg or good luck or whatever you believe and for next week, but I.
I think the day there are so compelling here.
The scratching my head to see what the FDA could really kind of find the fault with and I guess, the one question I keep coming back to us.
The FDA recognized and we got them out of asking you to put words in their mouth, but do they recognize how bad of the doctor of the steroids are.
And do you think there.
There needs to be further advocates and there or do they pretty much understand and accept that.
Well Ted if it is a really good question.
I know and my own case, I mean, I've been studying this disorder.
For a long time, but the first couple of years it was studying it from afar.
And I could read the charts of the side effects and I could conceptualize somewhat of an abstract what this might mean and painted a pretty bad picture for me and sort of my intellect.
And at Washington until I started.
Talking and see individuals who are on the steroid regimens and truly got just helpful.
Yes.
And on the steroids, they're just poison.
I've made the statement publicly and people tell me and provocative when I make it but I think it's backed up by science of steroids name and kill for.
Full stop and until you see that.
Until you live with it.
You just don't you can't grant.
I can't speak for the FDA I'm sure. There are many find professionals, who have had clinical experience maybe even seen this disease.
But I can say that most of us even those knowledgeable and this disorder and <unk>.
Just don't get it until you see it firsthand.
So I'll just leave the question of <unk>.
That's the case all across the board for all of Us and.
And then I have to agree I mean, I I don't think I appreciate the visit until I was talking to the something here, but so I do think you're you're working for the patients here and that's very clear and every time, we hear more testimony from patients and so clear to me and so we're seeing and the best of luck next week and.
And certainly be paying close attention.
And Q2.
Thank you and your next question comes from Ed White from H C. Wain right. Your line is now open.
Hi, Tom and Susan Thanks for taking my questions.
Just a couple of maybe one for Susan first.
Can you give us an update on where you are with the sales force or.
Are they in place now including the.
The the related costs.
And.
Okay.
And prepare to launch prior to July 7th if in fact, the approval comes before the <unk> date.
Sure and thanks, and thanks for the question and actually we have the benefit of having cash spent on the language and today, our chief operating income So I think all of our.
Hello, Tom Stanton and question time.
Susan Thank you very much of the punting the question of a happy to take it.
Yes, so regarding the field force what I can share is.
We have been and the process of building up our field force that comprises both medical science liaisons.
Well as sales professionals.
The.
The medical science liaisons, all enroll and they've been interacting with key opinion leaders.
And having appropriate disease education conversations about anchor associated vasculitis.
We have hired our sales managers and the all in the process of hiring and sales professionals and what I.
Can share with you is that those sales professionals will be on board with adequate time to train them on the disease state to train them on the backup on and to train them on the customers and the relative geography.
That work is about to take place very shortly and in terms of your question around should the approval come a few days.
Bulk of the seven and seven Paducah date, we expect to be ready to move very very quickly if it does come early.
Great. Thanks, Paul.
And Tom maybe a question on for you rigs.
Regarding the lupus nephritis study.
Maybe you can give us a little bit more detail on what that study will look like.
And any thoughts you can give us on the size of the study protocols et cetera.
Thanks.
And thank you.
We would love to begin and advocate type trial of lupus nephritis immediately and we found it a little bit more complicated than that.
Ultimately, that's what we aspire to it will take a step or two to get to that level of trial, which we would aspire to be the registration step of the study for a box of Pan and lupus nephritis and it turns out of the way of medicine is practiced and lupus nephritis, though is a little bit different.
They are very used to just add on therapies and looking for incremental benefit I'm, not saying incremental and small way certainly significant benefit.
And that's how the medicine has been practiced so even with all of the latest therapies and there had been some really nice advances recently as you know.
It's still probably these are added on to glucocorticoids. For example, there's still a lot of daily prednisone and the the squirrel routinely and.
We're still only getting true benefit for about 44 zero per cent of the patient population, even with all of the latest care.
And that's on top of non insignificant amounts of data and Fred So I think we're gonna have to go through of step as we head to some time ago as well, but we can do it at a very efficient and sort of accelerated way to first showing that yes, we can safely stepped down GC and the presence of of Alco pan to virtually.
And nothing on a daily basis, and then expand the trial are quite rapidly and vigorously to get to a larger and and something that looks a bit like advocate and go forward and using that as the registration step so beyond that I'm going to not comment too much further on the details.
Because it's a it's an active process right now and but I think we're getting we're getting to a very powerful plan here that will be very effective for the squirrel.
Yeah.
Yes.
Okay. Thanks, Tom and good luck next week.
Thank you Anne.
Your next question comes from the line of Joseph Schwartz from SBB Leerink. Your line is now open.
Alright, Thanks, very much I was wondering if you could elaborate on your commercial preparedness in terms of your interactions with treating physicians and maybe even groups of patients and advocacy.
Folks themselves for example, I heard you mentioned Youre rethinking of the initiative is there any way that you could quantify the reception you've seen so far and.
Terms of registrations for more information.
And maybe how many gay the patients are treated at the centers that have accepted your outreach.
Well, the tosh, perhaps you'd like to speak to that topic.
Yeah, well I can talk about that yes. So the question is about the reception to our product so far and about education and so what I can share with you is that we have been interacting well, let me step back. So when you look at this marketplace. The top 400, physicians, who treat and corresponding sketch of vasculitis.
Comprising of Rheumatologists and Nephrologists, they right about 30% of the prescriptions. So initially at launch that cohort of customers will be a big big source of the tension for us because they see they think the bulk of the patients and they can have the biggest positive impact on these patients and their lives and the subsequent.
Health care ecosystem costs. So we've been interacting with those physicians on a very appropriate places and go.
And and disease awareness with them many of them have seen the ad.
Ed for publication and the New England Journal of Medicine published in February.
<unk> seen the wholly independent and the company to pay your editorial.
And also in that same edition and.
And the you you've seen the download just think of that Tom talked about over 35000 downloads, making it one of the most popular new England Journal articles of recent history and so there's a tremendous amount of interest you also saw the slide the Tom shared with some brief the market research that we've done pretty recently and found that <unk>.
Search is currently ongoing and.
And that research speaks to the awareness and familiarity for.
<unk>, primarily rheumatologists and Nephrologists have a round of that compounded and 65% of physicians have heard of of that coupon now and.
And 18, 9% of of positive impression of the of that profile.
And we expect this awareness of of that coupon and the positive impression to.
Elevate after the AD comm and as we get closer to the FDA approval.
Mhm, Okay, and I know.
And then.
Go ahead no question around patients as well so we have been conducting patient advisory board really to understand the disease a lot, but that's what we can really get on to the skin to understand all of these patients feeling what are they hoping pool from a new treatment for on Tom's alluded to many of those factors.
We've also been engaging appropriately again with patient advocacy advocacy groups because they have very close relationship with these patients lay on.
Understand where these patients go for treatment they understand what these patients are going through and the most importantly understand what these patients one for one for future treatments and any organization the typing to introduce the new medicine and or product service for these patients. So we can book and working very very closely with.
The patient advocacy groups as well.
Hopefully the quest.
<unk>.
Yeah, It's super helpful. Thank you.
So I guess my follow up question would be you know of you come across any intriguing analogs for VAALCO, PA and that might be and instructive for how the AAV launch could go based on the ability to replace steroids, which has happened and other areas of medicine.
As well as offer other important clinical benefits.
And any dialogues that we should keep in mind as we're modeling the lunch.
Oh look I wouldn't want to speculate on the launch uptake at the stage.
Well I'm comfortable sharing is that look if you look at the actual patient population of that Tom alluded to we believe there were 20000 and potential patients.
And the initially the focus will be on those 8000 patients who mimic what we see and the advocate study, which is anchor associates of patients either with life threatening organ threatening disease. We believe those patients all of potentially ideal for meditation locker, Pakistan and.
That'll be the focus certainly in the short term should we get FDA approval, but we do believe there is potential for Iraq, Qatar and youth and the other two and the other 12000 patients who have what we call grumbling disease, whereby yes. The condition is not life threatening it doesn't have an immediate organ threatening disease, but they've got.
Lumping disease, there's still on corn, it glucocorticoid steroids and quite frankly, they're not well.
So that's what I would tell you that.
Thanks for taking my arms.
And in terms of uptake in terms of uptake.
But look.
And reiterate the point of the told Knight as the this.
This is a new drug and it's going to take US six to 12 months. If you look at various players various hospital systems across the U S and their processes and the timelines for new drug reviews. It can be anywhere from six to 12 months before these medicines the added to the electronics formularies and once it's added.
And so on and electronic formulary the.
Prior authorizations medical necessity of pills they tend to go through that process as the low.
Loser and shorter so for the first year, we expect that to be a little bit of a challenge because we worked through paper based systems with physicians has to do individual patient appeals to get the drug approved based on medical necessity.
But that's normal for a new drug, particularly and rare disease or the specialty space.
Makes sense makes sense, thanks for all of the color.
Thank you.
And your next question comes from the lab.
Line of other Palm Rama from J P. Morgan Your line is now open.
Hey, guys. Thanks, so much for taking the question I was looking at the FDA Advisory Committee page for May 6th.
For later today and it was noted that some of the presentations will be pre recorded on those pre recording would actually be available May force do you do you have a sense of which portions are being prerecorded and any of the chemo centric the portion being pre recorded just the.
And get a sense of what we what the totality of what we might see on sort of may for thanks. So much.
Thank you and good to hear from you and yes, you know the world of COVID-19 is still with us and it just created although we do of world of all of these advisory Committee meetings are done.
So that's correct, but I think.
There will be prerecorded the sessions from us the sponsor and things they get posted on May for it that's my understanding.
It's the so called.
That's the so called long or 60 minute presentation, 60 minute of plus or minus by the way.
I think FDA also has the presentation and and I believe that that might get publicly posted of I'm, a little bit confused myself to be honest with you, but that's my current understanding and then the committee here's the prerecorded publications as well.
On the day of the meeting there is an additional short presentation I believe both by US So that the committee can ask us additional questions the sponsor and buy the F D. A.
And so that's additional there may be some overlap between those two or there may be some new information and I.
I can't really speak to content.
But yes, it's a little bit different and COVID-19 then of course agency I'm sorry, The committee will ask questions both to us and I'd say.
And so having another question period with the agency and then in the afternoon. The open public forum occurs as well. So it's a it's a much different format and this era as we try to be virtual there are constraints, because it's all slides and recording and it's not in person and Theres not video allowed and all of that other stuff.
And so it's not it's not perfect, but it still can be a very effective for them. So yes, I anticipate based on my current knowledge.
The presentation from us will be posted publicly on may 4th but that will be a somewhat different presentation that will actually occur on may six so that would be an additional one.
Got it thanks, so much for taking the question.
Thank you.
Okay.
And your next question comes from the yen on Xu from Wells Fargo Securities. Your line is now open.
Hi, Thanks for taking my questions.
<unk>, you elaborate and it really well the rituximab for use in the advocate. The study I'm wondering if you could also elaborate on the steroid use and as well could you talk about the speed of tapering and the control arm and whether that could be considered standard of care.
Physicians and also equally importantly, the fact that the steroids tapered down to zero over 21 weeks.
And instead of you know a low lower those are kind of a maintenance dose whether that can be considered standard of care by physicians and thanks.
Good day and is the very very important questions.
When we started the design of the trial.
Some years ago in fact, we and panels.
The body of experts, both academic and practicing clinicians and.
And we asked them look we read the literature, we've done our interviews.
We can't come up with what the steroid taper really looks like and this field.
And rheumatologist tell us that they taper steroids within six months and yet.
The patients are still on five or 10 makes a day.
Nephrologist will say they taper steroids within six months of their they're essentially down to zero by you know for weeks.
What is standard so the fact is we took a range of.
Of what the standard was we appeal to all of the clinical trials at the time, including text of asked which was ongoing at the time.
And what we got as of consensus from the expert panel.
As a emerging modern standard of steroid taper at least and aspired standard which is let's get people off of daily schedule glucocorticoid within six months and.
So that was that's what we put into our trial is sort of a model.
Standard of care best practice, and the real World, there's probably a lot more.
Persistent glucocorticoid use which is fine all of the good and just speaks again to the need for a substitute or alternative accordingly.
With a highly targeted therapy by the way so I believe that if you talk to any individual status.
The physician.
Just like of statistics, they might say well, that's not how I would taper I would actually keep them on five big for another six months or something like that this does represent modern and best practice trying to minimize both daily steroid load has a fairly rapid taper and get them off of the daily reported quite.
Certainly by by six months.
And so the I think we reflect that and our trial design the steroids.
And.
And I think we're right and again within the range of total glucocorticoid load.
Over the six months and even the 52 week period, where we have of you know essentially of AR.
Seven fold media and higher glucocorticoid exposure and the prednisone group over the entire period of the trial.
Nine for media and higher of oral glucocorticoid during the period of the trial.
And so there's a massive reduction and total glucocorticoid and again our goal is to eliminate.
As much as possible the need for daily scheduled oral prednisone. So.
I think we I think we can say where standard of care, although any given the physician she or he will have their own opinion on that and their practice and you are quite correct rheumatologists tend to keep people on daily prednisone and once they get down to five biggs the kind of dismiss that.
I Wonder why that is though because the the data really shows that theres, probably no states daily steroid dose right down the two and a half migs.
The very recent paper of meta analysis published across six auto immune diseases, including vasculitis undefined kind of vasculitis really showed that even at two and a half mix of oral prep per day, there is a threefold.
Increase and severe cardiovascular disease year on year and.
And as you get up towards.
Higher doses 25, 30 Megs per day.
There is a sixfold increase and cardiovascular disease and that's what's the parameter was measured and that paper. So again, it's really.
Got it and used to steroids and their effects, but it doesn't mean that they're not really dangerous even at low doses. So sorry about the long winded answer, but we did try to standardize best modern practice with glucocorticoid admission administration, particularly the daily oral prednisone and I think we were successful on that.
Great Great. Thank you for it for that detail with the answer and then just a follow up quick follow up for just to anticipate and anticipate what happens next week.
No you prepared your briefing document without knowledge of the Fda's briefing document and so when when when the brick and documents are both made available and next week do you think we could find answers to fda's questions and your document or.
With better a wait for the actual clinical.
And the competing to find out thanks.
I think with and without speaking to the Fda's briefing book.
And I fundamentally believe that the answer to our questions about how outside experts will think about this data.
Need to wait for the and comedy Committee meeting meeting and we'll know at the end of the day on May six what outside experts think about the the data package I wouldn't.
And I think that's what this is all about both we and the FDA are trying to get an idea of what informed outside people thing not necessarily vasculitis experts by the way, but informed rheumatologists and Nephrologists what do they think of this data on balance and so.
My advisers and wait for May six and we'll see what the past the answer is.
Got it got it thank you and best of luck in the next week.
Thank you very much.
Thank you there and no further question at this time I'd like to hand, the call over to the Doctor Shaw for any closing remarks.
Well. Thank you very much for everyone for their attendance today I really appreciate your time and the energies. We look forward to talking to you again in the near future and again. Thanks for your time and you may now disconnect. Good day.
Ladies and gentlemen. This concludes today's conference call you may now.
And I disconnect. Thank you for my patients.
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