Novo Nordisk A/S Q3 2023 Earnings Call Transcript

Quartr
Provided by Quartr
November 13, 2023

There are 7 speakers on the call.

Operator

Greetings and welcome to the Molecular and Biotech Third Quarter 2023 Conference Call and Webcast. At this time, all participants are in a listen only mode. A question and answer session will follow the formal presentation. As a reminder, this conference is being recorded. At this time, I'd like to turn the call over to Jeanine Thomas, Investor Relations.

Operator

Thank you. You may begin.

Speaker 1

Thank you, Daryl. Good morning and welcome everyone. At this time, I would like to remind our listeners that remarks made during this webcast may state management's intentions, beliefs, Expectations or future projections. These are forward looking statements and involve risks and uncertainties. Forward looking statements on this call are made pursuant to the Safe Harbor provisions As a result, you should not place undue reliance on any forward looking statements.

Speaker 1

Some of the factors that could cause actual results to differ materially from these contemplated Such forward looking statements are discussed in the periodic reports Molekulin files with the Securities and Exchange Commission. These documents are available In the Investors section of the company's website and on the Securities and Exchange Commission's website, we encourage you to review these documents Additionally, certain information contained in this webcast relates to or is based on studies, publications, surveys and other data obtained from 3rd party sources and the company's own estimates and research. While the company believes these 3rd party sources to be reliable as of the date of this Presentation, it does not independently verify and makes no representation as to the adequacy or completeness of or that any independent source of verified any information obtained from 3rd party source. Any data discussed regarding clinical trials and progress are considered preliminary and subject to change. So joining us on today's call from Loma's leadership I would now like to turn the call over to Walter Klimt, Chairman and CEO.

Speaker 1

Wally, please proceed.

Speaker 2

Thanks, Janine, and hello, everyone. I'm Wally Klempe, Founder, CEO and Chairman of Molecular and Biotech. We are really excited to welcome you to this Q3 earnings call. We've been saying for some time now that this would be our year of data And we are finally able to deliver on the Phase 2 data we've been hoping for. To be clear, this is preliminary and subject And there's a lot more data to come in the next few quarters, but we have a start and it's a good one We're going to share it with you on this call.

Speaker 2

As we walk through the data today, I'd ask you to keep in mind one of our core beliefs That is the most important therapeutic tool for both AML and advanced STS has been and continues to be And anthracycline. Despite the fact that there had been more than a dozen new drug approvals in these indications over the last 5 or so years, The first line therapy for both indications and the best hope for a positive outcome remains with the use of anthracyclines. Unfortunately, today's antrocyclines have major limitations that prevent most patients from sharing in this benefit. But those days are about to be over. Molekulin intends on making this indispensable tool available to those patients who have until now then excluded from their use.

Speaker 2

And of course, the drug we intend to do this with is anamycin, our lead program in a pipeline that is As many of you know, we're doing this with a highly efficient capital structure by exploiting a global network of preeminent collaborators. Now our pipeline is robust and too complicated to capture in just one slide. After all, between The trials that we've completed are currently running and are approved to begin. We're talking about 11 clinical trials for our technologies. Today, we'll focus on just 2 of them, our European trial in acute myeloid leukemia and our U.

Speaker 2

S. Trial in advanced Soft tissue sarcoma. As a reminder, we have orphan drug designation and fast track status in both indications And we have a strong patent position for anamycin and a range of potential indications. For those of you who are new to the story, It's critical to understand why the use of anthracyclines has been limited for so long. Far and away, The greatest limitation to current antrocyclines is cardiotoxicity.

Speaker 2

This is the primary reason Patients either can't receive antrocyclines or have to terminate treatment before receiving the desired benefit. As well, there are also limitations resulting from tissue organ distribution and multidrug resistance mechanisms. As a result, There are many patients who simply don't get to benefit from today's antarcyclines. Now, when it comes to cardiotoxicity, the data are quite surprising. Anthrocyclines are so cardiotoxic in fact That the FDA has established a lifetime maximum allowable dose of 5 50 milligrams per square meter.

Speaker 2

As you can see from the chart on the left, if you only accumulate 100 milligrams per square meter, your risk of cardiac impairment is negligible. But if you go up to that maximum, there's a 65% chance you will experience some form of cardiac impairment. And if you go up to 8 50 milligrams, it's 100% certain you will have some kind of impairment. Now to make this even more graphic, the chart on the right shows that 600 milligrams per square meter, there's an 8% chance You'll have full on heart failure during treatment. When you translate this reality to our lead indications, You see that more than half of all patients diagnosed with AML cannot receive currently prescribed anticyclines because they're deemed unfit Likewise, in advanced soft tissue sarcoma, even though the standard of care therapy Is always anchored around an anthracycline usually doxorubicin.

Speaker 2

Only about 30% of patients will respond And all of those will relapse and once they're at the lifetime maximum allowable dose, they're relegated to a range The best answers currently available are simply not enough. In AML, that is a drug called venetoclax, most often used in combination with azacitidine. This Zenasa combination is capable of generating a complete response in 37% of patients, which is good for that 37%. And this drug generates a $500,000,000 a year in revenue for AbbVie, but there's a strong consensus among clinicians that Venaza is just too Hard on patients. It's very difficult for patients to tolerate and it's clear there needs to be a better answer.

Speaker 2

In advanced STS, the best fallback treatments appear to be dicarbazine and trabectin, but the best they can offer is about a 3 month PFS To around half the patients with no appreciable improvement in overall survival, there simply must be a better answer. And now there is. Anamycin is what we call a next generation anthracycline that's designed to be non cardiotoxic And appears to be improving outcomes in both AML and STS. In AML, we've completed Phase 1 testing and are now in Phase 2. And in STS, we've not only completed Phase 1, we've also completed enrollment in our Phase 2 study and are preparing for a pivotal approval trial.

Speaker 2

Antamycin does have orphan drug and fast track status in both indications and importantly strong patent protection through 2,040. So when we say anamycin is non cardiotoxic, we mean 100% non Cardiotoxic. It's important to stress this as there are other players out there with anthracycline technologies that they claim are less cardiotoxic than to Rubicin, but to our knowledge, no one is capable of making the claim we make because we're the only ones testing every aspect of cardio toxicity and then submitting our data to an independent cardiology expert at the Cleveland Clinic. And in addition to being completely non cardiotoxic, we appear to be easier on patients than currently approved anthracyclines. As a small example, 65% or more patients treated with doxorubicin will lose their hair.

Speaker 2

We're seeing less than 10% of patients having any hair loss with anamycin. But despite the fact that anamycin is easier on patients, it is actually more potent than doxorubicin in most tumor models and it's able to avoid the multidrug resistance mechanisms that limit the efficacy of doxorubicin in many patients. And that means even though we're concentrating on AML and STS, anamycin should be relevant to 10 times as many patients once expanded to additional indications. As it relates to expanding our pipeline by We are very effectively utilizing non dilutive institutional funding for clinical development. In addition to our sponsored research at And we're expecting several clinical trials to benefit from outside funding yet this year this coming year.

Speaker 2

And our sponsored research has paid some big dividends thus far. In addition to demonstrating the synergy between anamycin and cytarabine that's now playing out In our Phase 2 AML trial, this ongoing research also eliminated the fact that anamycin is 30 times better at accumulating in the lungs Bendoxorubicin, making it an ideal candidate for advanced STS, which is most commonly metastatic what is the Paul Womack to give you the specifics on the new data.

Speaker 3

Thanks, Wally. It has taken a long time to get to the point of generating Phase 2 clinical data for AML, But we are finally there. Part of the challenge from a regulatory standpoint was needing to demonstrate the safety and the efficacy of anamycin as a single agent before combining it with cytarabine for AML patients. Given that we also had to demonstrate that patients could be safely dosed at well above The FDA's maximum allowable anthracycline dose. This also meant we had to work with patients who were receiving 3rd, 4th, 5th line or worse therapies, which are the most difficult patients to treat and expect any impact.

Speaker 3

Notwithstanding all these challenges, we are now finally treating AML patients in a Phase 2 study with anamycin In combination with cytarabine, a combination that our preclinical testing suggested would be even more effective for AML patients. And this appears to be the case. We now have data from the first eight patients Who incidentally entered our trial with a median of 4 prior therapies, we are very pleased to report that among patients We have completed their anamycin dosing. We have 3 complete responses. This represents a 38% response rate, Something would not be expected in such heavily pretreated patients.

Speaker 3

For comparison, You may recall that Wally mentioned the approval of VENAZA was based on a 37% complete response rate. So we are Already performing at a level that we believe will support marketing approval of anamycin. And what's more, We are doing this in heavily pretreated patients, whereas the VENAZA trial For approval within first line therapy patients. When you look at the 1st, one of them responded to VENAZA, but then relapsed. 1 was refractory to VENAZA And the other had no prior exposure to VENAZA.

Speaker 3

The first two responders are now confirmed as durable With 1 at 8 months of remission and climbing and the other having just received a bone marrow transplant 3 months after treatment with anamycin. We can't yet declare durability for the 3rd patient Simply because not enough time has passed since their treatment and remission. It's also worth pointing out that all three of these patients were Treated well above the lifetime maximum allowable anthracycline dose of 5 50 milligrams per meter square. Again, this is what we were hoping to see in AML. And if we continue to see this level of activity, We think we'll be in a very good position to establishing a marketing approval pathway.

Speaker 3

Turning to our soft tissue sarcoma, we now have complete enrollment of the Phase 2 arm of our U. S. Clinical trial and are able to present some preliminary data from the trial as a whole. There's a lot to process here, but in total, We believe this trial's data bodes well for an ultimate approval in advanced soft tissue sarcoma. The unmet need here is so great that most of the recent approvals have been based on the very modest improvements In progression free survival or PFS, it took us a while in this trial to 0 in on the optimal dosing regimen.

Speaker 3

And it turns out that the most productive dosing regimen is not the highest dose we tested. When you isolate for this optimum dose, Which turned out to be around 3 30 milligrams per meter square. The column on the far right Joseph, we had a 78% response rate with 56% of the patients Reaching 3 months or higher progression free survival among patients who had received 1 or 2 prior therapies. That compares favorably with other approvals for the indication, but it's only half the story. One of the core falls of second line therapies is delivering extended overall survival.

Speaker 3

Although we haven't had enough elapsed time in the Phase 2 group to reach the median overall survival, since 12 of the 17 patients receiving antamycin are Still alive and the median overall survival won't be reached until the number still alive falls all the way down to 8. We can look to the Phase I cohorts data where we see overall survival at 11 months and climbing. Considering that the median number of months from diagnosis for patients entering the Phase II portion of our trial was 20, The overall survival that we appear to be headed for could be an even stronger basis for a marketing approval. We had the pleasure of sharing these data with a group of soft tissue sarcoma key opinion leaders At the recent CTOS, that's Connected Tissue Oncology Society Conference in Dublin, and the enthusiasm was high. As a result, we now have 2 different groups proposing to run their own versions of a pivotal approval trial, which we expect to be kicking off next year.

Speaker 3

I'll now hand it off to our Executive Vice President and Chief Financial Officer, John Foster, to wrap up the call. John? Thanks, Paul. We ended the quarter with roughly $25,000,000 in cash on hand, And our balance sheet remains clean with no debt and little overhang with warrants. With our current burn rate, This cash will get us into the Q3 of 2024, a runway that we've been consistent in our discussion since our last major equity raise 2021, we have just short of 30,000,000 shares outstanding.

Speaker 3

This runway allows us, We believe to deliver these milestones into 2023, early 2024 and set the table for delivering the milestones later In 2024, regarding anamycin for the treatment of AML with MB-one hundred and six being an open label trial, We will continue our quarterly clinical trial updates and also concurrently with other events such as scientific conferences or other public presentations, thus delivering the efficacy discussed. And the EMA will end the Phase 2 meeting. From that, we expect to identify the next steps for the next pivotal clinical trial and begin that at Regarding treatment of advanced soft tissue sarcoma, We will continue to monitor subjects for OS and PFS and we expect to report in the Q2 of 2024 that final readout. That's not stopping us and moving forward with discussions with investigators for a possible investigator funded trial, Either partly or fully in first line treatment of AVANCE STS, just as Paul discussed. The response to our meetings with investigators at CTOTS leads us to believe that the next program could be identified in either the U.

Speaker 3

S. Or the EU in the first half 2024 and initiated in the second half. All of these milestones are building upon the efficacy data that we just discussed.

Speaker 2

Wally? Thanks, John. For quite some time now, We've been touting the non cardiotoxic nature of anamycin, but there is just no substitute for data. We've now treated 66 patients, most of whom were taken well above the lifetime maximum allowable dose and not a single one has exhibited any signs of cardiotoxicity. But the absence of cardiotoxicity is meaningless without efficacy.

Speaker 2

And up until now, that's been the big unanswered question. Could anamycin Well, that question is now being answered. Yes, the data are preliminary and we need to substantiate what we've shown you with a few more patients. But with those caveats in mind, anamycin is delivering and it has everything it needs In order to become what we believe will be a multi $1,000,000,000 drug. And it all comes back to our core belief.

Speaker 2

The most important tool for AML and advanced STS as well as a host of other hard to treat cancers Antamycin finally represents a real improvement in anthracyclines and we're going to make this tool available to those patients Who until now have been denied this opportunity. So that wraps up our prepared presentation for the quarter. Jeanine, we'd be happy to handle any questions folks have.

Operator

Thank you. We will now be conducting a question and answer session. Our first question has come from the line of Jonathan Ashcroft with ROTH MKM, please proceed with your questions.

Speaker 4

Thanks. Good morning. Congrats on the data. But what I wanted to ask about Can you really exclude from the percent CR rate analysis in AML, the cytarabine SAE as well as

Speaker 2

Paul, I think you're probably best to address the statistical basis for establishing CR rates. So you want to tackle this one?

Speaker 3

Yes. Certainly in a pivotal trial With the intent to treat analysis, you would include them all. And as a Phase 2 study, we do things differently. The pivotal trial, because the person had an allergic reaction to cytarabine, we would have just continued Treating with anamycin alone since in the 105 monotherapy trial, we were getting similar results of a CR rate around 40%. So we just wanted to present here patients who had finished.

Speaker 3

I would point out we've got another patient In this trial, who finished their therapy, we have verbal confirmation from the site that they had a CR, But we don't put that in the slide deck until we have the written documentation. So we are trying to be very specific in a Phase 2 study with patients who have documented data and who exactly followed the protocol, whereas in the pivotal Phase 3 trial, It would be more broadly in the intent to treat analysis.

Speaker 4

Okay. Is it fair to say that for the STS LMM dataset in today's press release that it is no new data compared to the update a week ago?

Speaker 2

Yes. John, you're most familiar with what's been publicly Shared, so do you want to dial that in?

Speaker 3

No, I would disagree, Jonathan. If you look at the chart That we presented on Slide 19 and also in the Q and also believe the press release, when you start Remember, we took all comers into this trial. We took up to people with up to 11 prior therapies. And if you look at the right hand side of that chart, You get down to where we're looking at second and third line therapies at a dose that we zeroed in on and we're getting substantially better data. I'd look at that waterfall data.

Speaker 4

But it's the same end of patients that was prior reported on, it's just greater duration data, correct?

Speaker 3

No. Well, we reported the Phase 1b, the 67% was Phase 1b and then we moved on and now we're presenting the Phase 2 data. And so what we did was we added 17 patients and you can see the waterfall data how it goes across that chart. And so we end up once we start looking at the 330 milligrams and below patients, we found out that 360 and 390 was just too much And people with less prior therapies. We got better results.

Speaker 3

We got results comparative to dicarzabine and trabectathy. And worse than why And worse than

Speaker 2

why That's fair to

Speaker 4

me though, I mean, I've written up notes in the past of 3 Phase 1b patients at the recommended Phase 2 dose and 14 Phase 2 patients, all of whom must be taking the recommended Phase 2 dose and that adds up to 17.

Speaker 2

Yes. I think the point here, Jonathan, is since that last report, we now have updated PFS, updated OS, As well as segment data that just hadn't been elucidated. So I take your point, but I think there's important new information that we're sharing for the first time in this release.

Speaker 4

All right. Lastly, how many patients do you expect to have to enroll in a pivotal trial for both AML and STSLM.

Speaker 2

So Paul, you spent more time than anybody focusing on the proposed Pivotal trial protocols and I know you've had lengthy discussions both with investigators and statisticians. So you want to tackle that one?

Speaker 3

The ultimate answer is whatever the FDA demands. For soft tissue sarcoma, We have not finalized the plans for the pivotal trial yet because As I mentioned, patients are still going on and we have not reached median overall survival and the like. We would anticipate it will be in the few 100, but we can't give you an exact Number yet, because that trial, the efficacy data are still coming in. For the AML, As we have mentioned before, we would like to go for first line therapy. The 100 and 6 trial we're doing now, We are now enrolling first line therapy patients to get data to make sure that the results are similar, if not better than what we have seen to date.

Speaker 3

Based on FDA guidance documents, we would propose going to the FDA and getting approval based on a single trial. There is a pathway for a single trial with single arm therapy through accelerated approval with subsequent confirmatory trial Through the accelerated approval process, we would propose that a single arm trial would take about 100 patients. If we're required to do a randomized Trial would be about 300. Okay. Paul, let me add on to that.

Speaker 3

You mean, elderly and unfit first line, correct? Yes, sorry. For elderly for our first line therapy in elderly and unfit patients, we would do a single arm study, which is not that far off from what Venaza was doing and get approval on a single arm study accelerated approval. And then again, if you do accelerated approval, you need a Infirmatory trial, more of a traditional randomized trial, where we would go broader indication, randomized versus an active control arm.

Speaker 4

Okay. Well, just to make it clear that it's somewhere in the lower triple digits of patients per trial is helpful. Thanks. Can you lastly And sir, have you what kind of progress have you made in finding funding or getting An investigator sponsored trial going with the other two products 1066 and 1112.

Speaker 2

So, let me start there. But John, you may have some nuance that you want to add to this. Probably one of the most prolific areas of outside funding has been with our STAT3 inhibitor 1066. And just a quick recap, MD Anderson sponsored a trial there, then Emory University in pediatric brain tumors And then the investigator that was at MD Anderson moved to Northwestern University. And we've been in ongoing discussions with that It's very likely that that investigator will come through with another externally funded trial for brain tumors in 1066.

Speaker 2

And we know that the folks at Emory are awaiting some Additional data to come from the adult progress before they kick off. They've already indicated they want to kick off another pediatric brain tumor trial. So They're symbiotic there. They're comparing information. There's essentially a consortium of folks Across the U.

Speaker 2

S. That are have now recognized there's clearly some potential for activity of 1066 in brain tumors, especially when it comes to pediatrics. And then finally, we are we continue to grind away on an IV formulation for 10 And the investigators I've just described have all made it clear the instant that we have an IV formulation, They intend to switch to that, because we all recognize that the PK characteristics are probably going to be better with an IV delivery On the 11/22 side, we continue to right now just be moving at what I call grant speed On the basis of U. S. Government grant funding of virology research as it relates to 11.22, so that's our primary But I let's face it, that stuff is highly speculative And it's slow moving.

Speaker 2

So I would say that the slowest program we have in the portfolio right now is 11.22.

Speaker 4

All right. Thanks.

Speaker 2

And then I would add to that,

Speaker 3

I would add that the Northwestern trial is already listed on clinicaltrials.gov. It's 1066 in combination with radiation against GBM.

Speaker 4

Okay. Thank you. And the Q will come out later today?

Speaker 3

The queue should have already been filed.

Speaker 4

Okay. Thank you, guys.

Speaker 3

Thanks, Jonathan.

Operator

Thank you. Our next question has come from the line of Jason McCarthy with Maxim Group. Please proceed with your questions.

Speaker 5

Hi, guys. Thanks for taking the questions. In the STS data, with the median PFS, looks like it's 3.4 months for patients who are less than 330 mgs per meter squared. You Mentioned that the overall survival median so far has reached 11 months 15 patients from Phase 1b. What was the travectinib OS before it was approved.

Speaker 5

I know it didn't move the needle much on OS, but it did it must have had an OS. That's the first. Yes, okay.

Speaker 3

I'll jump in there because I've looked at the paper that we're referencing, they really don't go into PFS, I mean OS data, they really focus on PFS data. That's why we're so excited about the meeting.

Speaker 5

And mechanistically From for Anna Mayatsen, what do you think the differences are where you're having To go 330 mg per meter squared or less in the STS trial, but you're going higher in the AML trial? Let me start that off and Paul fill in wherever you think I maybe haven't covered it adequately. But

Speaker 2

I think there are 2 dynamics here at work. One of them is The specter of thrombocytopenia in patients. And so we know that even though we can technically Cross the hurdle of not reaching a DLT and keep we in fact, we never did establish an MTD in that trial. But as we bumped up against 390, it was the handwriting was on the wall that the thrombocytopenia issue with patients It was going to become a barrier to ongoing treatment. And so we felt like, okay, we're seeing Similar activity at lower dose levels, so let's not push the envelope here.

Speaker 2

Let's work In the center of the therapeutic window, if you will. More specifically to your because I understand your question, Why do we think that we might not get any more benefit out of higher dosing because classically speaking, higher is better with an anthracycline. You go to the maximum. There is a scientific analysis that the inventor has gone through Whereby there are some mechanistic reasons when you get to a certain level, as you know, is a topoisomerized to poison, There appears to be the potential for a point of diminishing returns where you simply Can't get any more cell kill benefit out of increasing the dosage, but you can actually start to have Some sort of counter effective results and that's we don't have the specific Mechanism data to support that argument, but it's a theory. But there is a theory out there that says there's a point where You can go too far with the topo II poison and not get any additional benefit out of it.

Speaker 2

But Paul, do you want to add anything to that?

Speaker 3

Yes. Let me just add just the indications. You are correct, that's a good point. The 330 we're doing for Soft tissue sarcoma is actually lower than the 230 we're doing for AML because the 230 is for 3 straight days, which becomes 690. If you look at the soft tissue sarcoma, our number one by far adverse event is Low blood clamps, especially platelets.

Speaker 3

And that forces us at times to stop. We could go up to 390. We didn't have a DLT, but we had to delay therapy because of the low platelet counts, because it was hitting the bone marrow. Well, with AML, that's where you got to hit. That's where the cancer is.

Speaker 3

It is the blood cells in the bone marrow. So you've got to blast the hell out of them essentially And wipe them out for a while. So the toxicity that is forcing us to hold and delay dosing in soft tissue sarcoma if we go too high, That's not an issue with AML, because in AML, here the safety location is also the efficacy location. So it's just With an adverse event for one indication for the other, that's efficacy. So I think that's the primary reason for the difference and why We're keeping it a little lower than soft tissue sarcoma than an AML, if that makes sense to you.

Speaker 5

Got it. Last question, you had mentioned Potentially selecting another indication next year. Can you provide a little bit more Detail as to what you may be thinking perhaps along the lines of where an antarcycline may be a key therapy or a go to therapy Kind of what the PFS OS might look like in whatever said indication?

Speaker 2

Yes. We can't speak to Speculating on what the human PFS OS implications of this would be, but we've got very compelling data in a number of indications. For example, triple negative breast cancer met to the lungs. Also renal cell carcinoma But the one that probably gets the most excitement out there is pancreatic cancer And specifically pancreatic cancer mets to the liver. And as you as I'm sure you know, Jason, most pancreatic cancer patients present with liver mets.

Speaker 2

And so, it's the most common site of metastasis. And We've talked for now a long time about the fact that we can hyperaccumulate in the lungs. It turns out we also hyperaccumulate In the liver as compared to doxorubicin, I think the ratio is like 6 to 9 times more than doxorubicin. So it might be the first opportunity to actually bring an antracycline to the table in pancreatic cancer. So if we were to kind of pick and choose and say who what investigator funded study would we be most enthusiastic It's probably that one.

Speaker 5

Okay. Just a follow-up to that then. Because you're accumulating in the liver, does that reduce the potential for Healthy tissue talks in the liver because a lot of chemotherapies are just not used in the liver because the liver is already weakened because it has Either liver, METs or primary tumors there?

Speaker 2

Well, I mean, we what we know from the animal data Is that we don't see any disproportionate increase in liver toxicity in animals vis a vis doxorubicin, even though we appear to accumulate it 9 times the level. Paul, you've been monitoring Liver toxicities in human patients, what's your perspective there?

Speaker 3

To date, we have seen no indication of liver toxicity. You look for what's liver toxicity based on what's called Pye's Criteria, that's Hy, he's a famous physician in liver disease. We've seen none. We have not seen bilirubin go up. We Have seen liver enzymes fluctuate a little, but they never go up to a clinically significant degree.

Speaker 3

So as of Now, I guess, we're approaching 70 patients being treated in all of our studies. We don't have evidence of liver toxicity.

Speaker 5

Great. Thank you, guys.

Speaker 2

Thanks, Jason.

Operator

Thank you. Our next questions come from the line of Jeff Jones with Oppenheimer. Please proceed with your question.

Speaker 2

A

Speaker 6

couple of questions Following on from what's been discussed already, my understanding of your path Forward in STS LomaMet is that you're looking at similar to trabectin A PFS endpoint for approval, is that correct?

Speaker 2

Well, I think we would modify that to say we really think that we've got window of opportunity here to be the combination of both PFS and for the first time ever PFS that actually translated into increased OS.

Speaker 3

Again, from a regulatory standpoint, we would in any clinical trial, we'll be measuring both variables, PFS and OS. We would prefer to have PFS since it would be quicker approval, obviously. But as Wally said, the data are becoming very impressive for OS. So we would probably at an end of Phase 2 meeting proposed to The FDA initial approval on PFS and then add OS into the product labeling Just for better marketing opportunities.

Speaker 6

Okay. That makes sense. And then I guess as I look at the OS State or at the PFS data across the subgroups that you've provided, Your while it's 3.4 in the 3.30 mg per meter squared with less than 2 prior lines of therapy, It's in the 2 to 2.5 range when you look at the more intent to treat Populations for both the Phase 1b and 2 intravectin in the PFS was somewhere on the order of 2.5 months. So even taking the 3.4 months, you're adding A month, a month

Operator

and a

Speaker 6

half to PFS. And so it goes to the question of clinical meaningfulness. And as you probably know, there was a company that just dropped a trial or Frocter program with the PFS of 3.6 months. So They just abandoned the product with the 3.6 month PFS. So that overall is critically important.

Speaker 2

Jeff, one thing I would point out that I think is a point of difference First of all, it's not greater than a first line therapy. It's 2 or fewer prior therapies in that right hand column. But It's important to note that we chose the most difficult subset of patients In our inclusion, exclusion criteria here. So we're not taking all advanced. This was not an all comers trial.

Speaker 2

This is not all advanced soft tissue sarcoma. This is only lung mets. And in every trial we've ever seen published, while no one actually gives specific data for the Lung NETs subgroup underperformed visavis the overall population. So we Because we accumulate in the lungs the way we do, we deliberately chose this worst possible selection criteria. Now having said that, We think the more likely approval trial design will be an all comers trial and it's very likely to be first line patients.

Speaker 2

So, we realize that the data we're talking about today It's not precisely the target that we're setting for a likely approval trial, but everything gets easier for us, we think, As we design this approval trial structure.

Speaker 6

Okay. That's helpful. Thank you. On the AML side, you're in sort of your timelines, you're talking about an end of Phase To the first half of 'twenty four and a potential pivotal start, second half of the year. And as you were talking about Z days, depending on Single arm or otherwise in a randomized study 100 to 300 patients, What do the costs look like for pivotal study, both frankly in AML and SDS Alumets or SDSs as you were just implying?

Speaker 2

John, that's clearly in your ballpark. You want to handle that one?

Speaker 3

Sure. So you're talking about a Phase twothree Sorry, you're talking roughly $18,000,000

Speaker 6

drug included full year. In which indication?

Speaker 2

Hey, But it looks like there's enough in Paul's segment, he mentioned this, there's enough enthusiasm on the STS side among Institutions that are specializing in this, it looks very likely that we're going to have External funding for a pivotal trial on STS.

Speaker 6

Okay. What would the total funding be required for the STS pivotal study.

Speaker 3

Well, I would say From a stand well, it really depends how much we get from the externally funded trial. You're talking about

Speaker 6

Exclusive of external funding. So all in and then external funding would Obviously, reduce your investment requirement.

Speaker 3

Yes. Phase 2b, not a pivotal would probably just to get it off the ground It would be fairly cheap. We'd estimate less than 5,000,000. That does not include the pivotal population.

Speaker 2

I think practically speaking, it's frank, it's not a lot different than the pivotal AML trial In terms of cost.

Speaker 3

Okay, that's helpful. Thanks guys. Thanks, Jeff.

Operator

Thank you. We have reached the end of our question and answer session. I would now like to turn the floor back over to Mr. Klimt for closing remarks.

Speaker 2

Thanks, Daryl. Look, we're clearly excited about this data. Having it The first time, something real to talk about. I think we're finally at that point where we're able to Stop talking about what might happen in the future and start talking about what is happening right now. Look, Our loyal supporters have been patient for a very long time.

Speaker 2

So we couldn't be happier to be finally delivering this kind of data. And for those of you who are new to the story, frankly, your timing couldn't be better. You should expect more positive updates in the next Few quarters because this data is building. And so we're going to establish a pivotal pathway for ranamycin and we'll communicate

Operator

Thank you. This does conclude today's teleconference. We appreciate your participation. You may disconnect your lines at this time. Enjoy the rest of your day.

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Earnings Conference Call
Novo Nordisk A/S Q3 2023
00:00 / 00:00