Welcome to today's press conference with Camurus. For the first part of the conference call, the participants will be in listen-only mode. During the question and answer session, participants are able to ask questions by dialing star five on the telephone keypad. Now, I will hand the conference over to CEO and President, Fredrik Tiberg. Please go ahead.
Thank you so much, Einar, hi, everyone, and welcome to today's conference call, where we will present the top-line results from our pivotal acromegaly Phase 3 trial, ACROINNOVA 1. We're very pleased to do that today. Before going into the presentation, please note our forward-looking statements as usual. We will start this presentation with a short summary of the results, then our Chief Medical Officer, Alberto M. Pedroncelli, will give a brief introduction to acromegaly, octreotide SC depot, and also to himself here. We will have a more detailed presentation of the top-line results, followed by next steps and Q&A. With me in the call, I have Alberto, of course, here, and then also our VP, Clinical Development and Pharmacovigilance, Agneta Svedberg.
Just starting up with a quick overview of the results we received this morning. We are pleased to report that we achieved a high level of disease control and positive PRO outcomes with our product, octreotide SC depot, CAM2029. We displayed the primary endpoint showed superiority with 72.2% of patients in the CAM2029 group, showing IGF-1 response below 1 times upper limit of normal, versus 37.5% in the placebo group. Same thing for the key secondary endpoint. This was met with a 70% response rate for both IGF-1 and GH in the CAM2029 group versus 37.5% in the placebo.
Importantly, in this study, all sensitivity and supportive analysis confirmed the conclusion of the main analysis. We were also able to show well-maintained, well-controlled IGF-1, GH, and symptom scores over time with the treatment. Improved quality of life was demonstrated for CAM2029 versus standard of care at baseline. We also showed very positive patient satisfaction results and a well-tolerated safety profile. Overall, it was a very strong result. With this, I will just hand over to Alberto M. Pedroncelli for a disease overview and an introduction to CAM2029.
Thank you, Fredrik. Good afternoon, good morning. Just to give you a brief overview, you know that CAM2029 or octreotide SC depot, is being currently developed in acromegaly, in GEP-NET, in neuroendocrine tumors, and also in polycystic liver disease. All the three conditions are characterized by still being rare and also by still representing some unmet medical need. By going into more details. Go to the next slide, please. On the acromegaly, describing giving a few hints on the disease. This is a rare pituitary disorder, which is characterized by excess GH, which is in most cases, up to 95%, 97% of the patient, derived by a pituitary tumor that produce excess GH, leading to excess IGF-1 production at the liver level. It's a slow progressive disease.
It's a slow developing, slow progressive disease, and one of the features the patients experience is that from the time of an onset of a symptom to diagnosis, there is a long lag of time. Patients are diagnosed after about five, seven years after the onset of the first symptoms. What is important to note is that when patients are not controlled from a GH and IGF-1 perspective, this leads to an increased presence of mortality, and patient may have reduced life expectancy. What is common in all patients with acromegaly is that they have a reduced quality of life and still represent an unmet medical need.
Moving to the classical signs and symptoms that patients present with, they are on the top left part of the slide, patients tend to present with fatigue, headache, paresthesia, subcutaneous swelling, excess sweating, and joint pains. As you can see from this slide, it is a systemic disease because the excess production of GH and IGF-1 has an impact on all different body conditions, from cardiovascular disease to pulmonary disease, and also from endocrine and metabolic component. Moving to how patients with acromegaly are treated. The first treatment approach is usually a transsphenoidal surgery, is the excision of the pituitary tumor. Patients may present with micro or macroadenoma, which is dependent on the size.
We have to acknowledge that the vast majority of patients, about 70%-75% of the patients, present with a macroadenoma that has repercussion on the outcome of surgery. About 60% of the patients treated with surgery still are not controlled after the intervention, and these patients require medical treatment. The first-line medical treatment in these patients is represented by the first-generation somatostatin receptor ligand, and there are two of these. One is octreotide, and the other one is lanreotide. Octreotide is given as intramuscular injections every month, and Autogel is given deep subcutaneously every month. Sometimes, when patients have a very mild disease with slightly elevated IGF-I, dopamine agonist may be considered. Patients who are not controlled by either octreotide or lanreotide can have a few options.
Either pegvisomant, a growth hormone receptor antagonist, can be added to the first generation SRL, or can be patients up switch to pasireotide, which is a second-generation somatostatin receptor ligand. Now let's look at the available SRLs for the management of acromegaly. Let's look at octreotide. Octreotide, as you well know, and we have a long, long-standing experience with octreotide earlier, requires a complex reconstitution. Many patients and many experience during the administration, they experience needle clogging. There are several reports about this issue. Octreotide is given intramuscular and lanreotide, as I mentioned before, by deep subcutaneous injection. All of them, they require a healthcare professional to administer. Although lanreotide, in some countries, can be self-administered or by the patient himself or herself or by a partner.
However, in practice, they are always administered by a healthcare professional. From a, looking at the efficacy of these compounds, octreotide LAR is characterized by low octreotide bioavailability, which is less than 20%. Lanreotide has a lower receptor binding profile compared with octreotide. When we look at the long-term results, you can see, and all the data published in the literature are very much consistent, show that about 50% of the patients treated with the first-generation somatostatin receptor ligand are biochemical uncontrolled, indicating that GH or IGF-I, or both, are still above the upper limit of normal. You can go to it. Now let's move to this new compound, CAM2029 or octreotide SC depot, which has a convenient dosing that allows patients to be self-administered.
It has two presentations, as you can see on the right side of the slide. There is adding pre-filled syringe, which is being used in the current study that Fredrik is going to present immediately, soon after me, and also in the long-term extension. It's also available in a pen that is being currently used in the second study for acromegaly. One of the key points for CAM2029 is that it's characterized by high octreotide exposure because it has a very high bioavailability. It has a rapid onset, and this can provide immediate control of the signs and symptoms and biochemical control. With this, I would like to pass the podium to Fredrik.
Thank you so much, Alberto. Let's move into a more detailed presentation of the top-line results of this randomized, double-blind, placebo-controlled trial with octreotide SC depot. First, we will move into demographics, and you can see here from the patient population that it's a very balanced population. We have an average age of between 50 and 60, which is quite typical. As Alberto said before, you know, typically this disease is diagnosed in patients that are around 40 or about 40 years of age. We'll come back to this in a little bit later. We also have a balanced study in terms of sex, with around 50% in both groups being females, respective to males. From a weight perspective, you can see here that patients are in a normal level.
BMI is 30 for all groups, quite high in this patient population. From a regional perspective, we have EU countries, Europe, non-EU, which is Turkey, Russia, and the U.K., and then United States of America, and reasonably well distributed between the two treatment arms. Looking at the acromegaly history here, you can see that, and this fits very well with the fact that we had around 50 to 55, 58 years of age in these patients. Most of them were diagnosed after 10 years of had a time of about 10 years or more since their diagnosis. You can see also that almost all patients had pituitary surgery, as Alba commented before, in both groups.
What was very nice in this study is that we had approximately equal amounts of patients on treatment at baseline with octreotide LAR and lanreotide Autogel, which were also stratification factors in this study. That was another important component. When we had this discussion with the FDA about the study, one of the important components for them was that we should have a well-controlled population here, and that we do certainly have. Moving over to the design of the study, I've already mentioned that it's a randomized controlled double-blind trial to assess efficacy and safety of octreotide SC depot, CAM2029, in patients with acromegaly. The primary objective was here to establish superiority over placebo.
You can see from a primary endpoint perspective, we looked at the response rate in terms of having IGF-1 levels below the upper limit of normal ULN at the mean value at week 22 and week 24. Secondary endpoints, we had a proportion of patients again, with the same characteristics, but here we included those who had a dose decrease in the study. I can say already now that we didn't have any patients that needed to decrease the dose in the study, this came out in the same way as the primary endpoint. This last key secondary endpoint was a combination of GH and IGF-1 response at week 24. Other secondary endpoints in the study was biochemical response over time.
Importantly, we had multiple patient-reported outcomes in terms of patient satisfaction and quality of life. We looked at clinical signs and symptoms of acromegaly. We also had self and partner administration questionnaires and plasma concentration of octreotide. Today, we will only present top-line results, and we're still waiting for the full results coming out. I will focus on those observations that are part of our top-line findings. The patient population was 72 patients with confirmed acromegaly, and they should also be stable on treatment for the last 3 months. The assumption for the study, for the primary endpoint, a priori, was that we determined that we would have a 90% power to show treatment difference if we had an 80% response in the CAM2029 group versus a 40% response rate for placebo.
Quite in line with what we observed in practice, and we'll go to in the next slide, or the slide after that, I should say. Before going into the actual results, let's look at the patient disposition. As we already said, we randomized 72 patients, 2 to 1. The reason we had 2 to 1 was that we, of course, had placebo in the study, and we wanted to avoid to overexpose patients to the placebo treatment. We had 48 patients starting out in the CAM2029 group. One patient withdrew from the trial or withdrew consent after randomization, but before treatment, and a further patient withdrew consent after the first dose. The reason for this was not clarified or pointed out.
We can see that we had 42 patients in the active arm that completed the treatment and the trial, and we had 4 patients discontinuing, but completing the study. They discontinued treatment but completed the study, and these were due to AEs of mild or moderate nature. In the placebo arm, you can see that 24 people patients received placebo. Here, it's important that this is the FluidCrystal system without the octreotide component. It's a full double-blind design. They, we had 22 patients completing treatment, 2 discontinuing, 1 due to AE, and the second one due to rescue medication need. Overall, we had 24 patients in this group that completed treatment, or completed the study, and 22 completed treatment.
With that, let's move over to the top line efficacy results. Here you can see them. On the right-hand side is a forest plot showing the point estimate, either as favorable or if this would not be the case, as unfavorable or favorable to placebo. You can see here that we have a very positive outcome, with a point estimate of about 34%, 34.6% improvement or in favor of CAM2029 for the primary endpoint, and very similar results then for the two key secondary endpoints. The p-values for these were 0.018, and then for the combination, endpoint, 0.035. Very positive results overall.
We performed a number of sensitivity analysis on the primary endpoint, you can see out of the in this illustration that we had very little impact with the different assumptions that we applied in to the predetermined sensitivity impact, this predetermined sensitivity analysis. P-values are very consistent and so is the treatment difference. We also performed a tipping point analysis for patients with missing values, and there was, fortunately in this study, only one patient with missing values. Here we are, of course, imputed them as responders or non-responders. You can see that it had a minor impact on the treatment results and the treatment outcomes in terms of the p-value. Very consistent results throughout the different sensitivity analysis performed in the study.
We also did a number of supportive analysis. Here you see in the top, the primary endpoint and the different endpoint and the different outcomes with the supportive analysis. This was, for instance, in all patients who received the last dose of treatment, the week 20 dose. Here you can see the treatment difference increases to 81% for CAM2029 versus 41%, approximately for placebo. Looking at the per protocol analysis set, which includes all patients that received treatment consistent over the trial, as well as excluding 1 patient who got a forbidden treatment. We had in the last dosing sequence, we had 81% responders in the CAM2029 trial and 38.1% in the responder in the placebo group. Again, very consistent results and overall positive outcomes.
Moving over to a different statistical analysis here, we're looking at the statistical difference in time to loss of biochemical response in terms of IGF-I going above the upper limit of normal. We use the Cox regression analysis on the ITT population here. You can see that there's a very clear difference in the two treatment arms. The placebo group had a median time to loss of response of approximately 8.4 weeks, and you can see this by just looking at the 0.5 mark there and moving over to the curve. 8.4 weeks, approximately, in this patient group versus, well, we couldn't establish a time to loss of response in the active group.
The hazard ratio, as I said, was 0.1, so it means that it's about 10x more probable that the patient in the placebo group will lose control compared to a patient in the active treatment group. The p-value, again, 0.0001 below that. Looking over then at the stability of the IGF-I values in these groups, here we have to acknowledge that starting out the study, we had a number of screen failures, so I would say that the baseline and screening values are very compressed from a statistical standpoint. That is, of course, due to the selection process in the study.
You can see that in the active arm here, we have very good stability over time, virtually no change in the IGF-I levels over time, and that the week 24 value on the right-hand side, you see also the treatment difference and the p-value here. The active group, of course, you see a significant increase in the IGF-I divided by the upper limit of normal value, so it's normalized values, up to week 20, approximately. It looks like it's going down slightly there, but that is due to the fact that 1 patient is with high IGF-I values, has then been transferred over to rescue medication, and then is not part of the analysis for the last 2 time points.
I think the most important message here is that, you know, we have well-controlled IGF-I values over time, and these are all taken at the end of the treatment period, so at the end of each dosing period. I should also say that we had a ± 7 days in terms of dosing window. Many of these patients have up to 35 days, and actually in some cases, up to over 40 days between the administration and actually read out of the IGF-I value. It's really worst case data here and still maintaining extremely good control in terms of the IGF-I values. I already referred to the fact that we have a number of patient-reported outcomes in this study, I thought it was interesting to look at this.
We start out with the quality of life measure that we have included in this study. This is the AcroQoL, Acromegaly Quality of Life instrument. It's a validated instrument, here we are looking at the treatment difference between the two groups and the standard of care at baseline. We're looking first at the top here at the total score, you can see that despite the fact that these patients are extremely well controlled at baseline, we still see a significant improvement in the treatment arms. For CAM2029, this is an improvement of almost 5 in the score, and you can see that it's highly statistically significant on the right-hand side, a significant improvement over standard of care baseline.
For placebo, you also see a slight improvement. I should say that we come back to that later, that this is very much to do with the improved presentation of CAM2029 versus previous treatments, and I will come back to that. Importantly, for the physical domain score, which combines various physical components with function, you can see that the CAM2029 group shows a significant increase, whereas there is a slight decrease and very large variability in the placebo group here. Then the psychological domain, you have again a significant treatment difference for the CAM2029 group versus baseline, and slight or an improvement also for placebo. Again, we refer this mainly to the new treatment presentation, and we'll come back to that in the next slide. I think this is a very important result.
I have not seen or rarely seen changes in an already treated patient group. I think in that setting, this is quite unique results. We also have a number of patient satisfaction measures. The first scale is what we call the patient satisfaction scale, where patients were asked whether the treatment, that is CAM2029 or placebo, was better or worse than their previous treatment, that is standard of care. Here you can see that very few patients think that the treatment was worse. There is one patient in the placebo group that considered the placebo treatment to be much worse. Of course, also more patients that considered the placebo to be slightly worse.
Around 30% found the treatment to be about the same as their previous treatment. I think most impressive here is that we have almost 30% of patients, or actually 30% of patients that found CAM2029 to be much better than their previous treatment, and also quite a large number of patients that found it to be slightly better. I think overall, this is very encouraging results, and you can see also on the mean scores that they are relatively high or quite high, I would say, for the CAM2029 group here, up around average of almost 4 there on this scale.
You can also see that on the Treatment Satisfaction Questionnaire for Medication convenience score, which was measured in this study, you can see that it's a very large treatment difference. This is a scale from 0 - 100, but you have an improvement of 13.85 units in the CAM2029 score, reflecting the improvement in the administration of the product versus standard of care. You can see this against both of the treatments. I think this result is very strong. We should also consider that we are still looking at the prefilled syringe here, and in the next study we're performing, we'll also include the prefilled pen device, injection device, which has further benefits, we believe.
You can see that, of course, this is mainly relating to administration and also the placebo group. Of course, they received the same dose without the octreotide, also perceived this as a much more convenient treatment, consistent with, expectations. With those, important points, we're moving to safety and adverse events. I think it's comforting to see that adverse events in the study were mostly mild. None of the serious or severe AEs were related to octreotide SC depot, so all of the related adverse events for CAM2029 were either mild or moderate. You can see that, from a relatedness, it's approximately half of the patients experience any related AEs, and as I already pointed out, the majority of these are mild.
there are a number of moderate AEs, but very few severe and serious adverse events. The only serious adverse event that had any relatedness was a response in placebo group of colitis. No AEs leading to withdrawal from the trial, withdrawal from treatment we had, as you saw earlier. We had no AEs leading to dose reduction, and we didn't have any fatal reactions in the study. From a preferred term perspective, you can see here, actually, a majority of these AEs were related to mild injection site reactions. We did have
Self-assessment.
Yeah, yeah.
It's a little-
We did have solicited assessments here. All of these reactions were, a very vast majority of these reactions were mild, none were severe. We had very few systemic reactions, as you can see, only three patients experiencing headache. Overall, a very well-controlled population and few adverse events. Going over to summary and conclusions, we are extremely pleased with the results from the Phase 3 trial. Primary and key secondary endpoints were met with robust statistical significance. All sensitivity and supportive analysis confirmed efficacy. The trial met multiple additional secondary endpoints on biochemical control of IGF-1 and growth hormone with the octreotide SC depot.
We will, of course, come back to many of these and also additional endpoints in publication and further communication. Patients were generally well controlled, both regarding biochemical parameters and symptoms. We saw improvements in patient-reported outcomes compared to standard of care at baseline, both in terms of treatment satisfaction and Acromegaly Quality of Life scores. The safety profile was well tolerated, comparable to approved first-generation somatostatin receptor ligands. No new or unexpected safety findings. Interestingly, of course, we have also, in parallel with this, we have a long-term safety and extension trial that is under, or should I say, partial completion, because we will, we're expecting the first readouts in the third quarter, and we believe this will point further to the efficacy and the patient-reported outcomes.
We provide additional information about the safety profile, of course, of CAM2029. Looking at next steps now, and upcoming milestones, we're of course, also waiting for the full results from the trial, that is coming shortly, and we will, of course, write that up into a full clinical study report. We are expecting in the third quarter, I think in the early part of the third quarter, results from the second trial, ACROINNOVA 2. We are also preparing for a pre-NDA meeting with the agency, with the FDA in Q3, Q4, 2023, and also preparing for an NDA submission around the turn of the year. Of course, further regulatory submissions in Europe and the rest of the world. This is the first step.
I think it's a very transformative step for us as a company, finally, to have these results in place and a positive outcome of our first randomized control trial in acromegaly. We are moving forward, also, as I said, with the long-term safety trial. In parallel, our development in the gastroenteropancreatic neuroendocrine tumor area, the SORENTO study is progressing well, the randomized control trial to establish superiority versus standard of care in neuroendocrine tumors, and also our POSITANO polycystic liver disease study is continuing and progressing well. With that said, I think it's time to leave over to you to questions, and I'll hand over to the operator for some general information, and then let's go into the question and answer section. Thank you, everybody, for listening.
If you wish to ask a question, please dial star five on your telephone keypad to enter the queue. If you wish to withdraw your question, please dial star five again on the telephone keypad. Our first questions come from the line of Erik Hultgård. Your line is open. Please go ahead.
Hi, thanks a lot, and congratulations to the whole team for very encouraging headline results. I have three questions, if I may. First, if maybe it's too early, you don't have all the data, but. Could you comment on the secondary endpoint of clinical signs and symptoms, whether that, whether you expect that to reach statistical significance as well? Then, my second question relates to that. It was, I think, less than 10% of the patients in the active arm that came from the U.S. Do you expect that to trigger some pushback from the FDA or any concerns regarding that? Finally, I noted the four discontinuations due to adverse events, so 8%, 9% discontinuation in the active arm.
Maybe you could help us to put that in perspective to not only to placebo in the study, but also to standard of care, how that compares. Thank you.
Thank you. Thank you, Erik. We don't have the results yet for the outcomes of clinical signs and symptoms. What I can say already at this stage, just so, is that, and you can see that also from the... Is that symptoms are very well controlled in the study. In terms of analysis, we are waiting for the full analysis, and it's coming shortly. I will comment that later on here. Your second question was regarding?
Location.
That was-
Oh, yes. I'll leave that over to Alberto.
Yeah. Basically, we know that U.S. patients tend to be low represented in those different studies, and this is very much in line with other studies in this disease area. I have experiences when we have now about 10% of the patients coming from U.S., and I have experience with pasireotide and octreotide, where more or less we had the same proportion of patients. There was no pushback by FDA.
I think I can also comment on another thing there, is that we do have, you know, also patients from the U.S. in our long-term safety study, so that is also adding to this. I think Alberto’s experience here is very important. There was one question.
The last one on discontinuation.
Oh, yes, yeah.
Compared to standard of care.
I think there are two things. First of all, we should say that these were mild or moderate reactions. You know, these patients don't know if they are in the placebo group or in the active arm. I think there is certain sensitivity in this patient population group in terms of study performance. It also takes a little while before, you know, you're adjusted to a new treatment. I mean, this is approximately the same rate as what we have seen in other studies. They have been around, I would say, around 8% in most of these trials, so I think it's quite representative. I don't see that this is a matter of concern.
We actually had a little bit more, totally injection site reactions, for instance, in the placebo group, but that's not reflected in the. I think it's more chance findings, but it's nothing that concerns us.
What type of adverse events was in these four patients? I'm just trying to see if there is any connection to sort of the higher exposure that you get from.
No, they were not exposure related. I think 3 were mild or moderate injection site reactions. 4 of them.
Mm.
Yeah, one was.
Mild migraine.
One was a mild migraine. Overall.
Good
... you know, they were not what I would say, you know, strong indicators.
Not indicators at all.
Not indicators at all. Yes.
Thank you so much. Congratulations again for excellent results.
Thank you, Erik.
The next question comes from the line of Suzanna Queckbörner from Handelsbanken. Please go ahead.
Hello, thank you for taking my questions. I have three as well. We can see that the IGF-I control seems to be better than in the placebo, but I'm wondering in terms of the clinical relevance, how are you going to leverage this data when marketing it to patients, especially those that have been sort of steady on treatment for a number of years? Also, how are you going to use this data when approaching payers? Then my second question is regarding both Teva and Tipler products have been disrupting the octreotide and lanreotide markets in Europe and the USA, and showing quite a substantial decline in sales for both Novartis and Ipsen products. and doing so by pricing pressure, as well as flooding the market with their products.
How are you planning on placing CAM2029 in this space, and how are you thinking about pricing? Finally, I'm curious about around the manufacturing of CAM2029. There have been difficulties for other manufacturers, particularly biosimilar manufacturers. Perhaps you can talk about how CAM2029 differentiates itself from those?
Yes, so, I think, I leave the first question about, I mean, we have done, of course, extensive research on the product, both in terms of payer response and patient response. If you, if you speak to patient representatives, for instance, we did that last week, with one in the Pituitary Society meeting in Chicago. The big medical need in acromegaly is really around symptom control and quality of life of patients. Quality of life is just really a core component in the medical need of patients with acromegaly. I think, you know, we have, and we will, have further data strengthening our arguments in this area. When it comes to pricing, there are many different components that we are addressing.
Maybe, Alberto, would you like to co-comment that, what I said first? Yes.
Yes, I think it's the advantage of the data show that we are having good efficacy, as shown by Fredrik, IGF-I remains controlled and completely flat throughout the period of treatment. We have an add-on, and the add-on of the study shows the improved quality of life. This is a key component, especially as Fredrik was mentioning, now within both the scientific community and the treating physicians, but also especially among the patients, there is a more and more demand about quality of life, how to focus more on compounds that can enhance the quality of life during the long-term treatment. We needed to take into consideration that this is a lifelong treatment. It's not 5 or 6 months treatment, and then treatment can be discontinued.
Showing that we have efficacy on one side, controlling IGF-I and controlling GH, we maintain a basis going through biochemical control. At the same time, the improvement in quality of life, which is I would like to remind the audience, that is improvement from baseline. These patients have already been treated, they have shown improvement, and with octreotide SC depot, we are showing further improvement in their quality of life. I think this is the combination of efficacy and improvement in quality of life, are the good combination for enhanced patient satisfaction.
When it comes to, you know, the question about pricing and so forth, I think this is something that we are working in. We can comment more on that as we are going forward. I think there are many arguments for this product in terms of also reducing costs to payers and also to treatment institutions by not having to have a hospital administration, for instance. So that is one component in our, let's say, offering. Manufacturing-wise...
Fredrik, did we lose the line? Do you hear me, Suzanna?
Yes, I hear you.
Yeah. It seems like we lost the line of Fredrik. We'll see. See if we get them back in here. Sorry for the inconvenience. Is this Camilla on the line?
Hello?
Yes, we are on.
Yes.
We.
We are on.
introduce this time.
We don't know what happened here.
No. You're back on again. Perfect.
Okay. Please continue then.
Suzanna.
Yes?
Do you have any more questions?
No, that's great. Thank you very much.
Okay. The next question comes from the line of Peter Östling, from Pareto Securities. Please go ahead. Your line is open.
Yes, thank you, and also, congratulations for very good results. Maybe you could repeat what you said about manufacturing, because we lost you after you started to talk about manufacturing, one of the previous questions. Maybe you could answer that question again before I ask my questions, please.
Okay. I'm very sorry to say no, but, I mean, I think there's a big difference between a biosimilar and CAM2029. That's the first point, of course. I think we are well, you know, we are well put in place here in terms of our validated manufacturing process, and we are working with a partner, a manufacturing partner that has multiple FDA approvals and recent positive pre-approval inspection. We feel that we are in a good place when it comes to manufacturing. I'm sorry for the break up of the answer session before, Peter, but hopefully that is an answer that it has about as much as I can say.
Okay, great.
On the manufacturing topic.
Just two quick ones from me then. Most of the ones that I had on my list have already been answered or asked. Just a quick one. The study included both the syringe and the pen. I guess that the pen is the one that you will go and when you launch the product, or will it be both available in the syringe and the pen?
That's a good question. First of all, I have to make a correction there, that this study only included the syringe.
Okay.
I think that, yeah. Both the pen as we said before, last year, we performed a bridging PK study with the pen to the syringe. In the 647, the long-term safety study, we have both devices included. We have, you know, a very concise bridging approach here. The results that we will present next time will include the pen. In terms of the presentation that we'll bring to market, I mean, obviously, the pen is the lead presentation because it facilitates administration even more for the patients. We haven't excluded the possibility of having both presentations available. This is something You know, we are keeping our options to either having both or only the prefilled pen.
Okay, great. Finally, I was a little bit curious. One of the previous questions referred to the relatively low number of patients included from the U.S. I was just wondering. The answer was that this is the normal case. I was just wondering why that is, why it is so.
Yeah
... only a few patients included from the U.S. in these studies.
I wish I could say, but, I mean, we have experiences across indications, that, you know, recruitment in the U.S. is challenging, and it has become much, much more challenging since COVID, I should say. You know, U.S. healthcare institutions are really under pressure. They have lost a lot of staff. They are understaffed in terms of nurses and clinical personnel overall. It was challenging, and it is even more challenging today. I think that's the general, you know, feeling among people who are performing clinical studies in the United States.
Okay. Given that this fact with the staffing problem, I guess that you will use the possibility of self-administration as one advantage of CAM2029 when you talk with payers and so forth.
Absolutely. We have already, and we have seen that it resonates very well, you know, not with payers across the board. That is definitely a positive.
Okay. Yep. Thank you, Fredrik, and the team.
Thank you so much, Peter.
As a reminder, if you wish to ask a question, please dial star five on your telephone keypad. The next question. It's a follow-up from Erik Hultgård. Please go ahead. Your line is open.
Yes, hi, again. A couple of follow-ups, if I may. Maybe first on the quite high response rate in the placebo arm, and I was just wondering, it seems like the relatively shorter study duration compared to some previous studies sort of led to quite high response rates. My question is basically, will you continue to follow these patients to collect response rate data so the delta or the gap between the control arm and the active arm could increase over time? Secondly, maybe it's too early again, but have you at all looked at any predefined subgroup analysis and whether those are sort of echoing the overall results of the study, or if there is any outliers there? Thank you.
Yeah, going over to the response rates, of course, there are two components here. We have a very stable population, that is one, and the second one is, you know, it's correct that we had 6 months study, whereas most, if you take, for instance, the Chiasma study was a 9-month placebo-controlled trial. The reason we had 6 months was that we didn't want to expose patients, and this was also discussed with the agency, too long placebo periods. When it comes to follow-ups, actually, we have a very nice feature in this study, and that is that placebo patients are followed when they are transferred back to CAM2029, so they are kind of pseudo-naive, you could say, in a, in a sense.
We are not following them in a placebo state, but they are, you know, continuing, and they have the opportunity to continue the trial. You know, vast majority have continued into the extension part of the study, so the next six months. We will be able to follow them there, but in a treated state. In terms of the subgroup analysis, of course, these are top-line data, very recent data, so we have performed very little, but there is nothing that sticks out to us. We will, of course, dig deeper into this as we go forward, but so far, if you, for instance, look at things like the difference between the patients that come from one treatment or the other, octreotide LAR or lanreotide Autogel.
We haven't seen anything so far that has sticked out or been, you know, worth commenting. It looks overall very robust, the results from this trial, and that's very encouraging.
All right, great. Thank you so much. That's all from me.
There are no more questions at this time, so I hand the word back to you, Fredrik.
Did we have any questions from the line?
I think we have two, but I think they are more or less addressed. There's one question here from Didier on Afira Fund, if we think that the 24 weeks treatment period is enough for approval?
Well, yes. I mean, we have discussed this with the agency. We've got, you know, positive feedback from the FDA on the 24-week period. That was on an agreed protocol. We have had extensive discussions about the protocol and the duration of the trial.
One long question on the theme of the high placebo response rate from Cory Jubinville at LifeSci Capital . I think he also wanted to understand whether we saw different responses in certain geographies here, or if we or if the high responses was related to any residual octreotide or lanreotide activity at the end of the treatment period.
Yeah. I think it's too early for us to come. We will definitely come back to that question, you know, once we have... Again, I think, you know, there is a clear explanation for this in terms of having a very stable population, and also, the treatment period. I think... We will definitely come back to that as we are wrapping up this into publications and also completing the clinical trial report. Thank you for that question. No more questions from there?
No.
With that, then, if there is nothing else from the audience here?
No more from the telco.
I would just like to thank everybody for attending this telephone conference, and it's always a pleasure to report the results and especially good results. I wish you all a nice week, and this is Midsummer week in Sweden, so that's also an important finding or an important happening. Thank you, everybody, for listening in, and I'm sure we will be back shortly for more from Camurus and our clinical trial work. Thank you.
This concludes today's call. Have a nice day.