Q1 2021 Calliditas Therapeutics AB Earnings Call
Per months and patients, who received and Mexico and 16 milligram of such as the 7% reduction in Egfr and patients who received placebo that equates to approximately of loss of full mil per minute and Egfr and for the nine months treatment period and again the comparison between Mexico and on the placebos.
Highly statistically significant for nowadays also noteworthy that we continue.
And to see the decline in proteinuria at the.
12 month time point when patients have been of treatment the cream of them.
In terms of Tolerability of the treatment was generally well tolerated the safety profile was in keeping with day to be.
Particularly the notice of the most severe infections and contrast to off label use of systemic corticosteroid and they're still in the life of the clinical effects on the cardiovascular and metabolic system.
Moving on to slide five.
I'd like to just briefly summarize the outcome of ups and biomarker analyses that have been performed.
Just on the sample taken from the phase two be Mexican and trial.
And taken together these imports and the number so you can share modification the circulating biomarkers relevant to the pathogenesis of Iga nephropathy. So.
So specifically and so all in association with Mexico, and the treatment reduction sit and galactose deficient Iga one.
And and Ikea immune complexes and real.
Also showed a reduction in past T cell activating factor, which puts and activating the actual piece, though so April.
And so trading revenue impact from sites like the pathogen and by market and all I can.
The moving to slide six and a summary, then in terms of the phase three clinical trial, and we saw robust demonstration of efficacy and tons of reduction in proteinuria and stabilization of Egfr patients who received in Mexico on the Tolerability side.
The profile in line with the active ingredient at the site.
And with the high first pass metabolism of basketball and low systemic availability.
Overall, and the clinical development program and seeing high consistency.
And I could think of age of course, the things to be in the phase III clinical trials enrolling a broad range of patients with all of the chain.
And the.
The J, Paul stabilization that we've seen across and the two large randomized placebo controlled study provides per supports for the cause of the multi client and Mexico.
And now hand back to revenue.
Thank you.
And if we are turning the page chain on slide number seven.
So in addition to the defining event of a regulatory filing and other events and the quarter included the full enrollment into the metric our trial and.
Which took place in early January.
And we're very proud of as an organization to achieve the enrollment goal and during the pandemic and and we look forward to the full readout and in early 2020 three.
The quarter also saw the first patient enrolled and the early studies. So this is the extension study in which patients can enroll and once they have completed the two years and the net the guard trial and and we are excited about being able to offer and epic onto all qualifying participants and the net to guard study and the passion.
And we recently decided not to pursue the and ethics and study and the second half of this year due to the combination of the significant delay of the study start and due to the ongoing pandemic and and recent interaction and supporting alternative formats for cheating and decided the treatment paradigms post the potential approval and this was not the regulatory studies has no bearing from a regulatory per.
Got it.
Finally in Q1, and we saw the successful readout of the stuff and active phase one study, which supports higher dosing going forward and Richard will shortly give you some more details around the pivotal trial, which we plan to start and the second half of this year.
And we continue to build out and the U S and as part of that and Andrew You're down was recently promoted to president of the North America and he's been with us for almost three years and has strong ties the cross the entire European organization, which I believe at the key component of building a successful transatlantic business.
I will turn over to Andy with no further Ado and who will take you through some of our recent progress and the U S.
Thanks, Renee and.
For on page eight now.
Over the last quarter, we've continued to grow our team, bringing on key members of our U S leadership team, which included the head of marketing head of U S sales and the VP of Medical Affairs. Our team will continue to grow significantly over the next several quarters as we prepare for commercialization and the fourth.
Quarter this year.
This growth comes the need to expand our office space and we were on the process of finalizing or at least on the substantially larger office space and New York City.
Next page please.
As the slide shows we have continued to meet our timelines and with an FDA action date of September 15th we were preparing for commercialization and the fourth quarter of this year.
Next page 10 please.
And our preparations we continue to conduct market research and get further entrenched and the Iga nephropathy market and the trends continue to be encouraging.
Over the next few slides I'd like the highlight some recently completed syndicated research conducted by Spirit and global insights. This research includes 188, and Nephrologist and chart audits of 468, Iga nephropathy patients.
Slide 11 please.
It is clear this is and unsatisfied market.
46 per cent of the Nephrologist rate Iga nephropathy is extremely challenging to manage and non dialysis patients.
The 2% believe there are few or no effective treatment options currently available.
They did nephrologist anticipate the 65 per cent of their current patients progressed to dialysis and end stage renal disease.
53 per cent of the Nephrologist would liked to replace the systemic steroids high doses and they're using for trim and option.
And 80% believed that early intervention is critical to successful outcomes and this number has been growing.
And you go to slide 12 please.
When asked about Napa comp when asked about the pipeline that for Com had the greatest unaided awareness by Nephrologist with almost half of them reading themselves is very familiar with and African and the phase III results.
This wariness of continue to increase from the last reporting period and all of these debt rated themselves as very familiar they indicated they are extremely likely to prescribe and ethicon for 70% of their current patients.
Next slide.
We continue to work to entrench ourselves in this market and educate and work on the disease awareness campaign and as you can see here, which launched earlier this quarter and it can be found on the Iga nephropathy culprit dotcom.
And with that I think will go to the next slide in the past of Richard to review of the preclinical activities.
Yeah.
Thank you very much on me so now I'm.
I'm on slide 14, and I'll summarize the clinical at the activities.
The activity.
On going.
And at present and so in terms of let the quote the.
The net the GOG study the phase three clinical trial is fully enrolled as of January this year.
And we anticipate for the last patient and will complete two years overall commodity price of 23.
In terms of set the Max it.
We remain on track to start the pace Creek phase two B III clinical trial and primary biliary cholangitis and the second half of the here and similarly.
We remain on track to start the phase II proof of concept study and squamous cell carcinoma of the head and neck and the <unk>.
And second half of this year.
We have ongoing and active.
The sponsor.
Duffy.
And official pulmonary fibrosis and diabetic kidney disease.
And they met the con.
Open label extension.
And is open and active and enrolling patients.
Yeah.
Moving on to slide 15, and just to give some background about primary biliary cholangitis the talcott hold on.
The next to the phase two phase three clinical trial of this is a rare auto immune disease for the height female preponderance of the female and male ratios and nine to one of the rare disease and it affects the round, one and creates courthouse and people.
It's kind of Suraj pathologic PD.
So of the structure of the small intrahepatic bile ducts.
Clinical features which include debilitating the.
Tag and itchy skin and event.
And actually the congestion progressing to cirrhosis with all of of the attendant complications, but the interest me and many patients are the initial diagnosis, it's night and day asymptomatic.
And based on and then total liver enzyme and the miles.
The tools.
Moving on to slide 16.
Day rates and remains the clear on the need in PBC, despite existing therapies in particular.
With respect to the critical features that I've mentioned, such as the Chi and.
And it changes both of which can be highly debilitating.
<unk> 45 per cent patients show and they'd have inadequate response or are intolerant and so you do see a the first line therapy.
Kind of the second line therapy.
Okay.
And it kind of in fact works and the corrected.
So that's and axis as well positions and the potential treatments and P. D. C has the unique mechanism of action.
Wei and inflammatory and anti fibrotic proxy.
And it demonstrates the benefits and the phase Iia clinical trial.
With respect to some of the.
Peak and liver stiffness and it's clearly from correct on that.
The logic reacted, but with also an unremarkable safety profile.
Cousins of the clinical trial that we have planned on slide seven and team. This will be a study in PBC.
The 52 week randomized placebo controlled double blind adaptive they used to be three clinical trial. The primary end point the based on the reduction in the alkaline phosphatase and we will be studying two doses of <unk> and ask it.
Daily doses of 216 milligrams per day administers the add on therapy and patients with PBC.
Elevated liver stiffness and intolerant sort of an adequate response and you can see it.
Hello.
So approximately.
318 patients and pumps.
260, investigational and cause the North America, Europe, Israel, Australia and using them.
We will be performing in the interim analysis.
The comprising approximately 30% of the planned sample size, one and 19 nine randomized patients completed the week 24 visit.
And this is expected.
And the first half of tons of 'twenty three.
And what is the current thinking on the possible outcome for well Netcom and I have a second question on the on the net com commercialization.
You have made from key appointment and the U S and the team has been doing the quick work.
The prepare for the U S.
Come on from elevation, but wondering if you could share with us your car in the U S. The strategy, including sales and the marketing also importantly, any new thoughts and feedback on the U S pricing.
Yeah.
Right. So let me, let's come back on and all of these things are done and I will try to address that the regulatory side and which of them.
Feel free to.
And so obviously.
Thanks in terms of I believe the in terms of of the obviously the decent eight of the target day for the 55 is the 15th of September.
And then we went and formed at the end of April obviously that we've been granted a priority review.
And and I actually I'm, sorry, I can't remember I mean, I don't remember off the top of my head in terms of them you know the mid cycle.
Date, and put them, but obviously, it's a fairly short review period and total.
From kind of from May to September and obviously, they're also needs to be label negotiation and theyre somewhere and Ah the gods and ought to come on with the if we had been informed that there was the plan for non coffee would obviously have communicated that and sort of assuming that there was no such communication from the from the FDA at the time of <unk>.
And of the granting of the priority of review.
And I guess, you know considering the very very short time period.
And that we're talking about here between kind of you know may and September I guess that it and.
And our view and it's not it's not particularly likely and that there would be but they're suddenly would be and are coming down and they think the S. T. A generally tends to announce that and you know well in advance in order to allow the company also to be prepared for such and ask Com and obviously the FDA has some work in order to.
Organize these outcomes as well.
So not to say that it's possible, but I guess the matter of view it would be highly unlikely at this stage based on the communications that we have so far.
From the regulators and.
In terms of the.
The I think that was on the regulatory side I think in terms of the U S preparation of as I said I will hand over to Andy to to answer those questions.
Sure I think your first question was on the sales force.
And as we've indicated we continue to build two about the sales force of 40, we brought on head of few of sales bring on the sales management are right now we're in the process of looking for those folks and Oh as I said, we're building to a sales force of about 40.
As far as pricing.
We continue to where our work on market access area.
And as the spirits research.
As discussed earlier indicated a large percentage of these patients from the chart audits of 63 to 73 per cent of the Iga nephropathy patients of commercial insurance, which is encouraging.
And we continue to hear from the payers that they recognize that the product and.
And they're pinning of towards the high cost of end stage renal disease and dialysis.
Of the 50 per cent of patients that progressed and stage renal disease.
And 2019, we conducted some work with IQ, the Oh, using our phase II data and and as we've previously indicated.
We took the price range is that excuse the hundred thousand there, but price range between 55 and 85000 per of course of therapy are seem to be appropriate are managed appropriately without any barriers to access for patients, meaning it would be required to be written by the prologist and the patient would have to have.
And Iga nephropathy confirmed indications of a kidney biopsy.
To be encourage continued to draw of work.
The value based pricing strategy and the recent launches also in this area and the space. There was a product that was recently launched or product targeting the Friday on nephritis provided good clinical value that has a price of about $12000 per month. So we continue to be income.
<unk> and the opportunities and as we've worked to do our work here to finalize the.
Yeah.
And Keith and that's.
Question comes from the line of kind of bounce from Amy of Stifel. Please go ahead to them on the site.
Sorry, everyone on this is Nick Rubino on for Annabel. Thanks for taking the question.
First of all.
Well, we see the 12 month per scenario of data and.
Good day, just a better of extrapolate and progression in the future and then on the Max of the.
And the P B and C trial, and it looks like the.
Primary endpoint is just going to be and a L. P. Rather than G. G T and E. L. P levels, we've seen before and why would the G T drop.
And in head and neck and can you give us the sense of how the clinical trial and is gonna be designed thank you.
Okay.
And.
In terms of the assets in terms of kind of the the D. G and I guess that was something that was the endpoint that was eased in and kind of the phase Iia trial and.
And what St tie attacks and designed and ran earlier, obviously at the kind of the approvable endpoint and P. D C and.
And not G. G T and it's actually a piece of that obviously the endpoint that we have chosen at the kind of the approvable endpoint and in P. D. C. S interest eight and a pivotal trial of okay Registrational and.
And that's why the endpoint has been chosen.
In terms of the head and neck cancer, I guess not something that you know we are still working on and there's quite a lot of preparations going on there I think it's something that we would hope to share with you and me.
More detail on.
The next kind of quarterly.
The call really.
And and and which I'm happy friendship and kind of give you know not lineup of kind of what we're thinking about are there. If you want to say before we address the out months sure.
And you know.
I already mentioned the.
And then the hypothesis is based on some very strong preclinical data the life.
And so it kind of associated fibroblasts and shameless.
And Nemo and head and neck cancer.
Population of patients.
And would that to the Uh huh.
Sorry, I was having that comes of that tumors with high numbers of caps.
And so clearly we want to focus on the population of patients with type of net come with high cost level.
And Ah again and from preclinical data and know there are some interesting effects relate to.
Two of them.
The benefits of immunotherapy.
And having that kind of a true.
And actually.
How modular the chain caps and.
And can improve because of almost two immunotherapy.
I think those of the kind of areas, we're looking out to and greater detail now.
And so hopefully we can share publicly.
And so all of the nice update.
And so in terms of any and it kind of the data beyond and above what's been publicly released and we are very constrained since this is a and ongoing.
Trial, which is continues to be blinded and and I think that it's very clear that you know the regulators are and you know.
Very keen that Ah, Yeah limited data and.
Its share yeah.
Externally at this point in time and.
And I think they were actually you know we have provided a earlier and I believe and our R&D day and that'll be see we do not have the 12 month data for the entire population.
And that's obviously day was the only a subset of patients who had reached 12 months at the time of the database lock.
And but what we have been saying in terms of proteinuria, obviously is that based on the trends that we saw from that.
Group of patients, who had reached that time and we would expect the cohort two and.
And basically you have a result in terms of overall treatment effect.
The somewhere and the kind of mid Forty's and so somewhere between 42 to 48 per cent.
As of kind of treatment effect at 12 months, but obviously this is just a you know and estimate based on the trend seen and a partial population and but.
But I think that that's really kind of all of that we can disclose at this point and time and I think it's been very clear that any further disclosure of any of them or any other information and it's not something that we're going to be able to day.
Okay, great. Thank you very much.
Thank you.
Next question comes from the line or from and comfortable first of month, sorry of counsel and please go ahead of two of them on and Sir.
Okay.
Hey, guys congrats on the update and thanks for taking my questions.
While it's early are there any key learnings from the launch of the product and he was mentioning that made a quite snuck on.
Okay.
I'm sorry can you repeat that are there any.
[noise] key learnings from kind of the launch that Andy was talking about that May also applies and ethicon.
All right and D do you want to take that.
I think it's kind of of early to really project or make any conclusions.
Sorry, and.
And of your disappearing we can't hear you you must if for some reason are going in and out I can't hear you and I think it's difficult to make any conclusions at this early time and their launch for us the only.
The thing you know we continue to monitor the whole market and how things are going on and these and this interesting time of the pandemic and and the marketing mix those of the kind of things that we are looking at right now, but it's hard to draw any conclusions from a from the recent launch.
Oh gosh.
Got it and then quickly can you remind us where you stand in terms of manufacturing capacity for the potential launch at the end of the year.
Yeah. So in terms of the so I guess I mean in terms of the kind of manufacturing situation. We do have and you know with the same kind of formulation and the commercial kind of commercially that we're going to use if we used for the phase III.
And so we're not kind of expecting any waves or changes or other kind of complication and.
And and you know, but otherwise I mean, yeah. We're kind of just you know working as normal in order to kind of try to no again.
Execute on the plan that we put in place for a while that we will be in a position to launch in Q4, a year, which is the earliest possible upon the fact it happened so.
It sounds great. Thank you.
Thank you now and next question comes from the line of countless extra on Who's a private investor. Please go ahead two of them on the site.
And thanks for taking my question and congratulation on the priority.
And redo designation.
Was wondering if somebody could elaborate on the risk for us.
The other multi quite of really doesn't it doesn't imply the medicines already approved for other indications such as the dozen called core of CT demand be used off label for Iga nephropathy, depending on approval of mythical neither of the U S. A.
The E U.
Sure. So obviously this is an orphan indication to start with obviously and an orphan indication you do have a market exclusivity and and which obviously is.
And it's relevant relative to this in terms of not being able to have any kind of competition from other and you know potential.
Potential parties with the similar substance of the same types of the mode of action.
And so that gives the seven year and Martha passivity and the U S and tenure of market exclusivity in Europe subject to approval.
And secondly, obviously.
These are these kind of formulations have been formulated to target completely different diseases and this is the crohn's disease and the crohn's disease actually exist and your entire got and so these formulations are different in terms of how they've been a kind of why they had been kind of formulated the way that the.
They are and they are basically you know of proof for nine milligrams for use of core three months.
And so I think Theres also has never really been any clinical data are ever kind of produced and it and finally it is not something that we hear or see a bad or you now see anyone actually China, and we don't share that from the market or from Nephrologists are the it has been used and obviously if you are true.
And to experiment with some of these formulations, which are formulated unfortunately of different diseases, and <unk> and is much lower of concentrations.
And you will most likely end up having true yep.
Gave you the type of doses of multiples multiples of what they have been of train four and.
And there's obviously then we'll give a most likely it will be significant and severe side effects, depending on how much higher than the approved a few of you are forced to go in order to see any clinical effect since the formulation of Mexico on it so highly targeted and focused.
Really on the kind of area of the of payers patches.
And so I think and you know it would be.
And I'm not sure what the purpose would be to kind of again go backwards and start kind of and its exposing patients to you know exploratory off label are you know drugs of which you know up until now have clearly China have not been not the kind of been proven in terms of efficacy or safety.
Okay. Thank God of just kind of a quick follow up on that the this.
And it's kind of in the year market because of it in the U S and tenure and the EU and that's also for that's for the for the indication so.
Even if somebody house and approved agents that could be used they would not be able to do it and for.
For that particular and enable.
That's correct. So it is for that indication that you have of market exclusivity for that indication for the period of time and and this is a this is why no and no one actually can promote or market.
And that and also therefore cannot be reimbursed.
And for that indication.
Perfect. Thank you that answers all my questions. Thank you.
Thank you. We currently have one for the question of in the queue. So interest as a reminder, participants if you do wish to ask a question. Please go on and see where we want them on your telephone keypad smell.
And I'm not the next question comes from the line of Johan Universe, or where it on please go ahead of two of them on this item.
Thank you for taking my question and congratulations again.
Corey.
And as Paul to keep out the cool briefly anyway, and if I understand it.
And you seem to be on.
All of the Vulcan.
And the securing a U P and corporate and stuff, Greg and B the pulse.
The question.
Yeah.
Yes, I mean, we aren't we are running and we are running a process in order to to kind of select them some of them to be our commercial partner. So yes. We are and now we are in the middle of of running that process.
No.
And the old could you or the.
Stepping back from the planned to initiate the metric than the.
Can you.
Explore the give a bit more color on that the fish from.
Sure no. So I think in terms of the again. The study was meant to be initiated a quite some time ago, but obviously due to the pandemic, there's been kind of continuous delay and obviously, where we are now in terms of and I'll get priority review with accelerated assessment.
We are positioned to be the first ever free drug to be on the market and.
We have obviously been and quite a lot of of interactions and a variety of forums and and I think it's become quite clear that there are other alternatives, which probably will be better.
The better for the patients may be significantly foster and it might be a better way of China of achieving some insight into the treatment paradigms and what's really what's the purpose of an ethics and if it was not of regulatory study and so we believe that there you know there are other options, which would be preferable.
And again, you know better alternatives to achieve the same outcomes.
Rather than that and kind of the yeah. The method.
And then trial.
And we can expect more.
The information on the pod.
Perhaps when you get more.
The backing and experience from the clinics.
And next year.
Yes, and we will I'm sure, we're going to be providing additional information.
Later on and the year in terms of some of them being some of the kind of complementary activities that we maybe maybe on undertaking.
Yep.
Could you and <unk>.
Sure.
The Korean market the market study or.
No.
The two.
And to the earlier from six.
63% to 75 per cent or thereabout seems to be on the future.
True.
And patients.
Seems to be groceries and sure how should the.
Hum.
How much of that segment is and played before actually securing.
Both of them.
I am not sure.
And what do you mean by the are they and play I mean, the there everybody's and play both commercial and government subsidized.
It's just a matter of you know.
They determined and on their formularies and.
The out of pocket of course, and those things for the patients.
Yeah, we're not the segmenting of removing anybody from a market. That's just indicated the commercial the large commercial population is favorable.
When you're looking to commercialize.
And what the.
Hum and frame it.
For the.
And Bruce Mann.
Albeit.
And do you approach of large part of that segment.
We're going to approach all of the largest pbms and now we have people that are gonna begin, calling on payers and and that market as early as the summer.
Yeah.
And the column on the you'd be able to approach a one or two the mam.
True.
Coast.
The formal approval.
Well the most of them don't provide an approval on the first right off the bat. So they'll cover you until they make a decision on familiar and that could take three months six months. It really does the planned dependent and will.
We'll be calling on all of them as much as moving on and off of it and but they have their own pace on certain things, but that's typical for any product into the market.
Excellent. Thank you so much.
Yeah.
Thank you and that's the most of all the questions coming through at this time all of how about small speakers for closing comments.
Thank you very much for participating and thank you for the questions and we look forward to catching up with you again at the next quarterly report. Thank you.
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