Calliditas Therapeutics AB (publ) Q1 2023 Earnings Call
Speaker 1: This call is being recorded. Your line is muted.
Speaker 2: Welcome to Caleditaster of Youth Dixq1 Report 2023. For the first part of the conference call, the participants will be in listen only mode. During the question-answer session, participants are able to ask questions by dialing star five on the telephone keypad.
Speaker 2: Now I will hand the conference over to CEO , Dania Aguar, Lucander. Please go ahead, Dania.
Speaker 3: Thank you very much and welcome everybody to this Q1 2023 report. With me today, I have Frederick Johansson, Chief Financial Officer, Richard Phillipson, our Chief Medical Officer, and Andrew Udall, President of North America.
Speaker 3: Next slide, please. I'd like to just draw your attention to the disclaimer page. It relates to any forward-looking statements, and I would like to refer you to the company's reports and other filings, including those which contain risk factors and other relevant information.
Speaker 3: I'd like to just draw your attention to the disclaimer page. It relates to any forward-looking statements, and I would like to refer to the company's reports and other filings, including those which contain risk factors and other relevant information. Next page, please.
Speaker 3: So some highlights for Q1. In February , received conditional marketing authorization from the MA JRA for Kim Pego, which thus became the first ever approved treatment for IJ Nefropathy in the UK.
Speaker 3: In March, we obviously had our main event for the quarter. We read out top line data from the global placebo control randomized phase. We studied known as Mesa Guard, which made its primary endpoint of kidney function as expressed as EGFR. The trial was very successful.
Speaker 3: and met the primary endpoint with a highly significant statistical p value of 0.001.
Speaker 3: With regards to Tarpeo, Q1 saw a record number of new enrollments, reflecting growth of over 30% over Q4. And in addition, the quarter also saw the largest ever increasing unique subscribers, resulting in a total of 918 unique entrologists.
Speaker 3: after scribing at Tarpeo's and launch.
Revenues for the quarter mounted to $191.4 million Swedish trams with net sales from Tarpeo, representing $185.7 million of that or just under $18 million for the quarter.
Our outlook for the year remains unchanged, and we're very excited about the initial reactions we've had from KOLs, and we've selected nephrologists, with whom we have had the opportunity to share the phase three data with, and yeah, we really look forward to being able to share that data bit broadly with the nephrology community going forward. Next page please.
So about the Nesogore trial. So actually Richard will shortly provide you with the details from our top line reader from the Phase III trial. As I mentioned, though we're extremely excited about the results as really this focus on the top of the disease test gate.
targeting this presumed origin of the disease seem to generate not only an immediate kidney protective benefit, but really actually also have long term durability.
And these strong results, we believe, will further support our thesis that Tarpayo is indeed disease modifying in hygiene and property. And it's even more exciting, obviously, this effect has been seen in respect of a baseline UTCR levels.
We do believe that this data once it does become more familiar and the astrology community is going to be have a very significant impact on treatment paradigm of IJ in the property.
In addition to the EGFR data, we also saw a durable UPCR response, where UPCR levels remain below 30% reduction for the, also for the entire observational period of 15 months, post the initial treatment period of 9 months.
On the basis of these data, we plan to file a full approval with the FDA for the entire study population of NEPA Guard in July with a regulatory decision to be expected in the first half of 2024 and the exact timing of that decision.
is dependent on whether that regulatory process will be conducted under priority or standard review.
process will be conducted under priority or standard review. Next slide, please.
Quick pipeline update. We are on track to report out on biomarker data for the set of next head and neck cancer trial around mid-year, this year, as we previously disclosed.
We are still experiencing some challenges in terms of recruitment of the transform study in PDC. The IPF, the Immusculate IPF trial is still recruiting on plan.
In terms of the biomarker data, we're super excited about reading that app. And we hope obviously that this proof of concept study will provide us with information that will be very helpful across all of the kind of different rare disease trials which are presently running.
In addition, we've decided to expand our clinical pipeline with a study in all parts of the room. That's an orphan renal disease. Whereas of today, there are no FDA or EMA approved drugs.
and we will on the basis of extensive preclinical work launch a clinical trial in all ports involving approximately 20 patients and we hope to start then the second half of this year.
And we also continue to explore the whole NOX and HIDER platform for other renal diseases, which we may be in a position to pursue later on.
Next slide please. So with that, I'm going to hand over to Richard Phillips, then who will take you through the top line data.
Thanks very much for any. Next slide please.
So I'll start by briefly reviewing the phase three study trial design. The NETEGARD study enrolled patients with biopsy proven IgA nephropathy, proteinuria of one gram per day or higher and an EGFR of 35 to 90 ml per minute.
and with well controlled blood pressure whilst on optimized RAS inhibition.
Innocent press therapy was not permitted during the study and changes to anti-hypertensive medications were discouraged.
Patients were randomized to receive top KO at a dose of 16 milligrams per day or placebo for a nine month treatment period. An interim analysis of change from baseline and protein era in the first 199 patients enrolled and treated for nine months formed the basis for soluteage.
and conditional approval in the US and Europe respectively.
The final analysis of the medical study is based on 364 patients full analysis set to efficacy. Treaties for nine months and followed up for further 15 months without investigational treatment. With a primary endpoint of average change from baseline in EGFR over the entire 24 month period of treatment and observation.
Next slide please.
In total, 305 patients were randomized into the study.
This includes an additional 29 Chinese patients required a local Chinese regulatory purposes only.
The faith analysis set of 389 patients includes all randomized patients who received at least one dose of study medication.
The full analysis set comprising 364 patients is the dataset used for efficacy analyses performed for the GLOBE study.
It's noteworthy that only just over 10% of patients were the group from the study at any time, indicating it was good rate of potential of patients in the study. And early discontinuations were broadly similar in the Nefacolum placebo treatment groups.
And flight! Please!
Moving on to demographics, overall the enrolled patient population is clearly represented at the intended primary hygiene of property population.
Disease characteristics describe a clinically relevant high-risk population. Treatment groups are balanced with regards to baseline characteristics and note blood pressure was well controlled for study entry.
has been an increase in the proportion of Asian patients in the study population since the use of data from the intraminalysis reflected of the active increase of patients in China.
has been an increase in the proportion of Asian patients in the study population since the use of data from the intraminalysis reflected of the active recruits of patients in China. Next slide.
Primary endpoint of time-weighted average change from baseline in UGFR during nine months of treatment and 15 months of observation was done. On average, during two years of treatment and observation, the loss of UGFR was 2.47 miles per second.
the nephekon 16 milligrams versus a loss of 7.52 mL per minute to the placebo. There was therefore an average treatment difference of 5.05 mL per minute favoring nephekon, a result that was highly statistically significant.
versus a loss of 7.52 mL per minute for placebo. There was, therefore, an average treatment difference of 5.05 mL per minute favoring methicon, a result that was highly statistically significant. Next slide, please.
Several different supportive analyses of EGFR Total 2-Year Slope were performed. These are all statistically significant with improvements in slope estimated to fall as arranged but approximately 1.8 and through the last minute per year.
The differences in two-year total slope observed between NETHICON and placebo are considered clinically relevant since all of the estimates are well in excess of the difference per year required to predict clinical treatment effects on the composite endpoint of end-stage kidney disease, eGFR less than 15 ml per minute or a sustained doubling of serum crapsin.
published by Ingrid Allen 2019. Next slide please.
When we look at the EGFR outcomes in placebo-treated patients, at 9 months there was a decline in EGFR of approximately 8%, corresponding to a loss of approximately 4.6 mL per minute, which increased to a decline of 21.5%, or 12 mL per minute by 24 months.
In contrast, in patients' treatment with Neficomy EGFR, we could potentially stay able compared to baseline at nine months and about 24 months that being a decline in EGFR of 11% corresponding to a loss of approximately six bills per minute.
So in summary, nine months of dosing with 16 milligrams of Netacon in 364 patients resulted in 50% less loss of kidney function.
compared to placebo at 24 months.
Next slide please.
So, turning to proteinuria, we've seen a cumulative improvement in proteinuria in patients treated with nephicon versus placebo during the 9 month treatment period, which continued to to improve at 12 months.
At month 24, proteinuria levels in patients who had received methacon were still at a reduced level, similar to that observed at the 9-month time point. Next slide. So, in summary, for
The primary endpoint of average change from baseline in EGFR over the two-year treatment and observation period was MAC and was highly statistically significant.
supportive analyses of two-year EGFAR slope were also statistically significant and are clinically relevant.
All estimates are well in excess of the threshold required to predict clinically meaningful treatment effects.
A treatment benefit on EGFR was apparent across baseline UPCR subgroups.
And a sustained proteinuria effect and long-lasting EGFR treatment benefit was observed even after 15 months of treatment supporting disease modification.
So now next slide to safety. When we look specifically here at the nine month treatment period overall we saw a pattern of adverse events in the safety analysis set that was similar to the interim analysis and which has previously been described in our publication in Kidney International and which is also described in product information.
The most frequently occurring adverse events occurring in 5% or more nethicontry to patients.
and 2% or higher than placebo or peripheral edema, hypertension, muscle spasms, acne, upper respiratory tract infection, face edema, increased weight, dyspepsia, arthralgia and increase in white cell count.
It's important to note that any ongoing adverse events typically resolve at the end of treatment and common adverse events occurring during the 15 month follow-up period are generally unremarkable with a similar frequency of reporting in metacombs versus placebo treatment groups.
So next slide.
So in summary, the broadly adverse event profile was similar to that reported in the interim analysis.
The most commonly reported headless events
observed with an increased frequency compared to placebo, what peripheral edema, hypertension, muscle spasms, or technique, the majority of these events were mild or moderate in disparity and adverse events led to discontinuation of study drug in fuel and 10% metaconcrete of patients.
And when we looked at objective measures of mean weight and blood pressure, these showed unclenically relevant, fully reversible changes.
I'll now pass over to Hendy Eudel.
Thank you, Richard. Next slide.
While our $17.8 million in net sales were impacted by the typical end of year, early patient refills, and patient insurance changes at the beginning of the year, the first quarter of the year saw substantial enrollment and new prescriber growth, which are both.
key leading indicators for future net sales growth. March was a record month in sales and enrollments, pushing the quarter total to 408 patient enrollments, representing a 30% growth over Q4. In addition, we had 276 new nephrology prescribers
are receiving TARPEO. During the quarter, 85% of patients enrolled in TARPEO touchpoints, excluding those still waiting for an insurance decision, received TARPEO. This conversion rate is consistent with the rate reported for full year 2022.
As time goes on, we have more and more patient success stories and patients that have reached nine months of treatment with TARPAO. While treatment length can be variable, as we see more patients completing nine months on treatment, we observe that the majority of those that completed nine months remained on therapy beyond this. We believe this reflects the risk, reward, profile, of product as more and more
this month we have several late-breaking oral presentations and posters accepted for the European Reenal Association Congress coming up in June next month.
These presentations and posters will provide further information and build upon the impressive results that Richard just reviewed with us.
As you would suspect, the peer-to-peer conversations and reactions to the data by advisors and investigators is extremely positive. The next three quarters have additional key nephrology meetings and conferences providing opportunities for further data exchange.
and presentations as we continue to assess the results of the Nefagard Phase 3 study. And with that, I will turn it over to Frederick to provide our financial results. Thank you, Andy. And good afternoon and good morning, everyone. I will now present to you a differential overview for the first quarter of 2023. And as always,
All numbers present to you are a median set unless otherwise stated. To start, we reported 109 to 14 million net revenues for the quarter. For the same period last year, we reported net revenues of 49.7 million.
So pay on that product sales for the first quarter, amount to $185.7 million or $17.8 million.
In addition, we also recorded 5.7 million for the paired and redness related to partners, primarily from Kim Pagarois, from Stada.
For total operating expenses for the first quarter amounted to 3.62.4 million compared to 2567.5 million for the same period last year.
Compared to poor quarter loss here, or up extra crease by 26.3 million in G1.
And in comparison, if we remove the effect in the quarter from a weakened SDK and increase sources of security rules from auction programs due to increase in or shall rise in March, we would have decreased by the up-exp additional 21 million for a total of, so for the seven million between Q4 and Q1.
The cost for research and development increased by 13.4 million in the first quarter to 126.7 million compared with 113.3 million for the year previous year.
Increase and R&D expenses, redness, primarily from don't go in, operations for the set on the feed trots, and the preparations for the start and the output drive.
The cost for sales and marketing increased by 73.3 million to 167 million compared to 93.9 million in front of the state period previous year.
If you want, we now have the cost for the full extended commercial team, which should be compared to the same for the last year, which we were in the first quarter of commercialization.
The above led to an operating loss of 100 when the input of the first quarter compared to 208.4 million for the same tier loss.
The first quarter cash used in operating activities was $230.9 million compared to $199.1 million for the same year previous year.
This leads us with a net decrease in cash in the quarter of 237.8 million. And we have a very health cash position at the end of the quarter.
1 billion and 13 million, which we believe is sufficient to take us to profit-building.
That was all for me. Thank you, and now back to you, Renan.
Thank you, Frederick. So just some key kind of summary takeaways to finish off the presentation after which we will take questions.
Thank you, Frederick. So just some key kind of summary takeaways to finish off the presentation after which we will take questions. So as you heard from Richard.
We had very strong age-uapar data, really showing a kidney protective effect, positive reader from phase three, Nefrog or trial, which we believe supports disease modification from the treatment of Nefcon, which are branded as tarpeo in the US and kinpago in Europe .
We will be filing for full approval for the entire study population, which is planned for July this year, and STOTA is expected to file with EMA for full approval also in the second half of this year.
As you're from Andy, we had record numbers of enrollments for the quarter and the highest ever growth today of new prescribers in Q1. We also, which resulted in revenues of 191 million Swedish crowns in total and tarpeo sales of 185.7 million Swedish crowns. We also got MHRA, M-HRA conditional approval of Mephagon, which provided the first and only approved medication for hygiene and property in the UK.
So with that, I'm going to hand over for any questions.
If you wish to ask a question please dial star 5 on your telephone keypad 20Q If you wish to withdraw your question please dial star 5 again on your telephone keypad Our first question comes from the line of Yigal Nohomovitch from Siddhi
Please go ahead, your line is open.
Hi team, this is Oshik Mubarak on FreeGal. Thanks for taking my questions. Just the first one on the Tarpeo guidance. It feels like you're assuming some significant acceleration of growth in the remainder of 2023. Can you talk about some of the dynamics behind that and what you think the key drivers will be?
It seems to hit the midpoint. You're going to need to achieve some pretty significant growth rates. So just wondering what your thoughts on how that might appear quarter over quarter for next two or three quarters. Thanks. Sure. And I'll have a start and then Andy can fill in with any other additional details. So in terms of what we've seen obviously this corridor already is really high level.
recommendations amongst nephrologists. This obviously provides leverage into the entire system, and we also believe that in addition to that, we will, by actually kind of getting this kind of phase three data out into the nephrology community, not been a kind of commercial fashion, but just merely from clean up.
abstracts and oral presentations of conferences or manuscripts, et cetera. We believe that this data truly is groundbreaking and we've certainly had the feedback that we've had from KWALS who have we been able to share some of this data with really support the fact that this is truly disease modifying.
I think that you're going to start to see beginning with ERA, EDTA, the full trial results, hit the podium. And I think that people will enjoy and see the reaction to the data as we have with advisors and people that have been brought in.
to assess the data with us. So I think that that's certainly one catalyst as well as more and more successes of patients that were started late in 2022. Our stuff will start to see a lot more of those success stories with new prescribers you heard about the...
large number of new prescribers at the beginning of this year in the first quarter. So I think that these things will build on itself during the launch growth here. God of the super helpful. And if I can ask one more, you alluded to patients at this point.
The majority of the patients on Torpeo staying on treatment longer than nine months are able to give us any more detail on that maybe, maybe how much longer than nine months they're staying short. And if there are any challenges with reimbursement associated with that. Next.
Sure. So just to be clear, length of treatment can be variable, okay? But what we are seeing is, and it is still early for us to talk about long and length of treatment here, but what we are seeing is those that have completed nine months seem to stay on a little longer and the majority of those patients, okay?
But we are there are success stories of patients that have that are more mild. Let's just say that may stop after seven, eight months. And those are deemed as successes. So, so that's we just want to provide that feedback in the sense that it's panning out almost exactly how
and they're going to treat if it's patient starts and there's a much more severe patient and they're doing well and they're avoiding dialysis. They're going to stay on treatment if they're tolerating the medication. And if not, if they are more mild patient or earlier in the disease,
and they're doing well and their protein area is well below the threshold for them considering them at risk. They would stop, but they continue to see these patients and should the patients start to progress again, they would treat again. And the last part of your question just as far as payers, no, we have not seen any issues or concerns as far as coverage with that.
And if there are requirements, we would certainly make sure a patient didn't discontinue or have any interruptions of treatment. So I think that that provides it. Your last question actually you had a point about how long. That's way too early for us to tell how long they go on for these patients.
there are requirements, we would certainly make sure a patient didn't discontinue or have any interruptions of treatment. So I think that that provides it. Your last question actually you had a point about how long. It's way too early for us to tell how long they go on for these patients. Very helpful. Thanks very much.
The next question comes from the line of Morrie Raycroft from Jeffries. Your line is open now. Hi, thanks for taking my questions. As a follow-up to the earlier guidance question,
I'm wondering how does your latest commercial data shape your assumptions for upper or lower ends of revenue guidance for this year? Are you relying more on keeping patients on drug or getting new patients on drug?
I mean, I think that in terms of what we're kind of constant, I mean, obviously it's both. I mean, obviously, I think that there's going to be a, as Andy mentioned, is going to be a combination of, you know, shorter duration treatments, where actually the physicians have achieved their treatment goal. And there also seems to be
I think a higher number than what we were initially expecting, who actually are staying on drug longer, longer than the nine months initial treatment cycle would indicate. So I think that there will be clearly there's a combination there. I think in terms of
of truly kind of penetrating the community. So that there is sufficient information about the drug out there. So I think that's really what we are kind of focused on. So in terms of the guidance, I mean, I think it's certainly kind of easier from this particular quarter to say that it's clear that hitting the upper end of that guidance might be
or answer to that question. That's really helpful. And one other question, just wondering if you can elaborate on next steps for pursuing full approval. Do you need to have another round of a pre-NDA regulatory feedback in order to file? And what are your expectations for the review timeline? So obviously I think that with this data, which is extreme.
the entire study population. And as I said, a very kind of clear and crisp data. So we would file and the timing for filing really that we're targeting in July .
In terms of the we will request priority review as this is kind of a supplemental filing. But obviously it's always up to the regulators to, based on the workload that they have and other considerations, to decide whether they will do the priority review or the standard review. And the difference there is obviously six months.
Review time for priority in 10 months, review time for standard procedure.
Got it. Makes sense. Thanks for taking my question. The next question comes from Susila Hernandez from CLK. Please go ahead, your line is open.
Thank you. This is Cecilia for this end. Thank you for taking our questions. Do you already see an uptake in prescriptions after the offline phase three results? And also could you elaborate on how you expect your operating expenses to develop over the quarter and what will be the drivers? Thank you.
Sure, I mean, I think that obviously this is really between, so the first quarter, you know, would be, would be way too early to see any impact from that. Really, I mean, the, the, the real impact really is going to be in 2024 because we are not really able to commercially promote on the basis of the new data until there's been a regulatory.
approval and an updated level. I think the only kind of benefit that I think we will have is just as I mentioned before that through kind of oral presentations and conferences and abstract at other kind of manuscripts etc. I think that will start making its way into the kind of nephrology community.
really from the in the second half of this year because that will really start with the era EDTA which takes place in the middle of June .
Frederick, I will hand over the second question to you. Yes, we don't give any guidance on the operating expenses, but looking at.
At the start of the last year's optics of 1.25 billion, we of course also see an inflation effect in our operations and this year also we do have the extended Salesforce on board for the full year. We also start gaming programming now So I think that that is the drivers what we we will see
in addition to last year. Okay, thank you. The next question comes from the line of Rami Kaktouda from LifeSci Capital.
Please go ahead, your line is open. Hey guys, thanks for taking my questions as well. Two quick ones for me. First, has the expanded Salesforce been fully deployed and when do you expect to see the impact of that on Tarpeo sales? And then, do you plan on running any post-marketing studies with NEPCON and iGAN to evaluate longer-term dosing, retreatment, combination with SGLT2?
with most of them come with nephrology experience, you're gonna see more and more uptake as the year wears on. So they were in the field deployed in November , all of them, and I think you're gonna start to see them hit their stride soon.
As far as the other stuff, I'll let Renee comment. Sure, Richard. So with regards to additional studies, that is something that we are looking into. And so we are having kind of internal discussions with regards to what type of studies may be most useful from the kind of nephrologist and patient perspective.
we haven't made any decision with the Guard's Tony additional studies, but it's certainly something that we're looking into on the basis of having read out the full phase three data.
Thanks so much. The next question comes from Dan Aksioti from Parito Securities. Please go ahead. One question would be regarding the mentioning that you saw records enrollment in December .
How did that translate into sales? Were some of that maybe not the book them in January ? And could you comment if you see the same growth as CIO Cino in Marshalls in April and May? Thank you. I want to clarify the first piece, and then I'll take the second. Yeah, no, just we did not see a record enrollments in.
takes a little bit to realize the income from an enrollment. But the record enrollments, just to clarify, was during Q1 and March was in particularly strong.
And in terms of continuing, I think we're very encouraged by the level of enrollments that we are seeing post Q1.
Okay, thank you. And another question, how is Everest Medicine planning for launch assuming approval in the second half? Are they ready to launch directly or do we need to assume a few months until they launch after approval? Thank you. I think that it'll be a similar situation as the one we had in Europe .
a delay between kind of the actual approval and the first kind of shipment of product.
Okay, thank you. On the last question, you reach around 2000 subscribers now. There are around 10 times as many around there. And the US, what are your plans to accelerate the pace of reaching them and educating them? And do you see a change now with the full data since March?
Do you want to take that, Andy? Yeah, sure. So, just to be clear, while there may be over 10,000 nephrologists, there's really not—many of them don't treat IgA nephropathy patients, and they're not in the clinics that see these patients. So, we believe that that number is in the—
4,000 range to cover the vast majority of of nephrologists treating these patients. So over a thousand, you can do the math and these typical markets are 80, 20, and 80% of the load comes from about 20% of the target. So that's...
that gives you a better sense of the size of the market as far as prescribers. And as far as the data and those kind of things, as we keep saying, I mean...
you're not going to see the impact of the full data from Nefigar trial. You guys are more in tune, those on this call, listening to it than many of the nephrologists because it hasn't hit their inbox yet. And as Renee said, we're not permitted to promote on this. These will come out.
peer-to-peer meetings, congresses, and the first one of those is next month in the middle of the month in Milan at ERA EDTA. Thank you very much.
The next question comes from Ingrid Gaffenau from Brian , Cornier and company. Please go ahead, your line is open.
Direct it.
Do you have a question? Then we'll move to Vamil De van from Guggenheim. Your line is open. You are now muted.
Hi, this is Arseniy on for VAML. Thanks for taking our questions. Maybe expanding on some of the prior questions about these new prescribers translating.
Are there any shifts this quarter in the gross to net or the Medicaid channel contribution or any other factor besides this delay from enrollment to shipment, the kind of impact of sales this quarter? No. I would say no.
There's no change on gross to net or anything like that this quarter. And then maybe one more. So there is dramatic growth in the number of new prescribers. How should we think about the rate at which the old prescribers are enrolling new patients?
Because based on the numbers, this quarter it looks like the majority of new enrollments obviously came from the new prescribers.
Yeah, I think it's variable depending on their patient load, you know, and some see more patients than others and some are faster adopters than others, and some nephrologists may try a product on a patient or two and do their own kind of...
clinical trial meaning to see the success of sometimes it takes a few months before they start to write their next patient or the patient after that So so there's no real set answer We may have a new prescriber now that becomes one of our you know big fans that writes a lot of it It's just there's no there's no set answer to that. Sorry
I think it's just worth pointing out obviously that there are, this is a rare disease. And a lot of these physicians don't have a huge number of physicians necessarily. And so that's obviously why it is important.
to kind of you know continue to penetrate the actual prescriber universe as well as obviously, you know, continuing to support those prescribers who have already written prescriptions and who have patients on drugs. And obviously we do that by, you know, having our hub services.
and providing a lot of these additional support to the community as well as education. Understood. Thank you. The next question comes from Arthur Hieh from HC Wainwright. Please go ahead. Your line is open. Hi everyone. This is Arthur from HC Wainwright and thanks for taking my question. So I guess my first question is for...
Neficom in the Japanese market, when could we hear more regarding the development strategy and timeline for Neficom in Japan? So I don't have an exact time to provide to you. There are quite a lot of ongoing discussions and collaborations with regards to...
kind of establishing the most effective way to kind of design that kind of program. And so I'll have to get back to you once I actually, that's been a little bit better established, but it's certainly being worked on very actively. But obviously it'll have to kind of be after we hear back from the regulators.
in terms of their acceptance of the proposed development program and we have not yet done that. So I'm going to have to come back to you about that when we have more information. Sure, no, no, no, no, no issues. Thanks for the color. And my second question is, could you give us a little update on the enrollment for...
CBC study and and remind us what's the data set away can expect for the In-show update for intramarine out. Thank you
Sure. So in terms of the interim readout, so that's that interim readout is a biomarker readout to subset of the patients. This is in the head and neck cancer trial. So subset of those patients, we will have matched biopsy.
And we will actually then be able to, you know, have a biomarker related readout, which really looks at the microenvironment of the tumor. And we're doing this obviously to really do this as a translational study from what we observed in the preclinical models that we have, obviously with the view to seeing a similar effect in the clinic.
So that's really something that is still expected to be around mid-year, kind of July-ish, probably. So that's still on track, and I think this will be something that we really look forward to. We think it'll be very exciting to see if we can actually kind of show that translational effect, which we believe will have an impact and be very helpful.
across kind of the different rare disease programs that we're presently running. In terms of PBC, this is obviously a larger study. And at the moment, we're running the phase 2b portion of that adaptive design. And so we are recruiting patients and the view is that the target is for the first half of next year to actually then select the dose which would be the kind of the dose level for the phase 3 portion of that trial. Thanks for the cutter. Thank you.
The final question comes from the line of Johan Ondros from RedEye. Please go ahead, your line is open.
Thank you for taking our questions. I hope you can hear me. It's a follow-up. Of course, unique subscriber is a prerequisite and very important, but the ratio between enrolled patients and unique subscribers seems to be fairly steady, slightly north of one and a half. We should expect that to keep.
to pick up going forward. Andy, you wanna take that? I think it's hard to answer that question in the sense that,
As I said before there's going to be, like any market, there are people that see a lot more patients and treat a lot more patients than some others. So there's value obviously in someone that even writes a few prescriptions, has a few patients, but obviously there's more value, you know, in a prescriber that has a larger patient load. So it's...
Yes, I would expect it to grow, but at some point, you know, when the denominator keeps growing with ones at the beginning, it's hard to, you know, change that ratio. But we have plenty of prescribers that write for several, several patients.
I would expect it to grow, but at some point, you know, when the denominator keeps growing with ones at the beginning, it's hard to, you know, change that ratio. But we have plenty of prescribers that write for several, several patients. Yes. Yes, you.
pointed out that the sort of 80, 20 rule applies in this area as well. And do you recognize an element that some of the specialists sort of go ahead with the first patient and then wait for experience and clinical real-life experience before moving ahead? Absolutely, absolutely. You see that, you know, everyone's different as far as their level of adoption.
And if you jump on the cogs it's like they're higher and the quarter should be expected to come down a bit Not that it's a major point, but it's an important point Frederick, do you want to take that?
Yeah, I can take that. I think in the first quarter of last year we had a
Extremely low cost of goods sold and also in the remainder of 2022. So I think we are at a normal level now.
And what about the break even you loaded to that earlier? I think earlier you mentioned perhaps mid year 23 is that still feasible? I mean I think we kind of guided to cover.
what the curve and from the revenue perspective actually looks like. And so I think that we will probably, I think it would be highly unlikely, and that we will do that in Q2. And so I think it's definitely something that the core focus for us, and that we continue to drive towards.
And we certainly think that that's still possible as we sit here today. To expect to week break even on run rate during 23.
Yeah. Yeah. There are no more questions from the Telquat this time, so I hand the word back to you, Renee.
Well, thank you very much for listening to our Q1 2022 report and we look forward to catching up again when we report our Q2 numbers. Thank you.