Realty Income Q1 2023 Earnings Call Transcript

Quartr
Provided by Quartr
May 3, 2023

There are 9 speakers on the call.

Operator

Afternoon, and welcome to Lumos Pharma's Q1 2023 Financial Results Conference Call. Currently, all participants are in a listen only mode. Later, we will conduct a question and answer session and instructions will follow at that time. As a reminder, this conference call is being recorded. I will now turn the call over to Lisa Miller, Senior Director of Investor Relations.

Speaker 1

Thank you, operator. Before we proceed I would like to remind everyone that certain statements made during this call are forward looking statements under U. S. Federal Securities Laws. These statements are subject to risks and uncertainties that could cause actual results to differ materially from historical experience or present expectations.

Speaker 1

Additional information concerning factors that could cause actual results to differ is contained in our periodic reports filed with the SEC. The forward looking statements made during this call speak only as of the date hereof, and the company undertakes no obligation to update or revise the forward looking statements. Information presented on this call is contained in the press release we issued this afternoon and in our Form 10 Q, which may be accessed from the Investors page of the company's website. Speaking on today's call will be Rick Hawkins, CEO and Chairman and Lori Lalley, our CFO John McHugh, our President and Chief Science Officer and David B. Karp, our Chief Medical Officer will join for the question and answer section.

Speaker 1

I will now turn the call over to Rick.

Speaker 2

Thank you, Lisa, and good afternoon, everyone. And after the market closed today, we issued a press release Announcing our Q1 2023 financial results and providing an update on our clinical programs. As is our practice, we'll keep our prepared remarks on today's call brief, so we can maximize the time available for Q and A. I'll touch on some of the highlights from the quarter in recent weeks before turning it over to Laurie for a review of our financial results, And John McHugh and David Karp will join us to answer your questions. So let's begin.

Speaker 2

As we reported this afternoon, During our Q1 of 2023, we made significant progress in advancing our oral therapeutic candidate LUM-two zero one An idiopathic pediatric growth hormone deficiency led by the completion of patient enrollment in both our Phase 2 oral growth 210 and ORGrowth 212 trials. And we can confirm our expectation to report primary outcome battle On up to 82 subjects in the dose range finding ORGrow-two ten trial and up to 22 subjects in the PKPD ORGrow-two twelve trial in the Q4 of 2023. Importantly, as predicted, baseline characteristics for Orbro-two ten subjects converged across the 1.6 mg per kg LUM-two zero one and the growth hormone cohorts between the interim data we announced in November And full enrollment. You may recall that the control arm for the interim analysis included outliers with greater growth And prior data precedents, with the conversions of age and other key predictive baseline characteristics across these two cohorts At full enrollment, we now have better balance and baseline characteristics and therefore believe we should see more similar growth rates between these two arms at Final analysis. During the quarter, we were pleased to see an updated interim data from our trials presented at the 2023 International Meeting A pediatric endocrinology or MPE that was held in Buenos Aires, Argentina in March.

Speaker 2

The presented data demonstrate that LOOMCSA 1 possesses both a natural endogenous mechanism of action with potency to stimulate meaningful growth in a moderate idiopathic PJC patient population as well as a favorable safety profile. This included an oral presentation of oral growth 212 data by Doctor. Fernando Casorla that further supported the pulsatile The mechanism of action of LUM-two zero one and highlighted growth stimulated with LUM-two zero one in PEM positive idiopathic PGHD subjects and a poster presentation by Doctor. Allison Lumsford demonstrating That the 1.6 mg per kg LUM-two zero one dose produced 8.6 centimeters a year annualized height velocity, Again, in line with historical growth for moderate idiopathic BGSD patients treated with injectable standard of care Growth hormone. Again, data demonstrated that LOOM-two zero one possesses a favorable safety and tolerability profile.

Speaker 2

The presented data further reinforce our prediction from the interim analysis we announced last November that the 1.6 mg per kg LUNP-two zero one dose Is on track to meet growth expectations based on historical database averages. Importantly, we believe the MFAE Medical meeting Was critical for raising awareness and understanding of LUM-two zero one among key opinion leaders in global pediatric endocrine community. Among the pediatric endocrinologists in attendance at FA, the interest in LUKTIN-one and its potential as an oral therapeutic to treat PGHD was significant. There are those present who are familiar with Merck's original evaluation of LUM-two zero one in all comers PGHD trial And the lack of success there. Yet the trial yet that trial I should say enrolled both severe PGSD patients unable to secrete growth hormone As well as more moderate or idiopathic PTSD subjects.

Speaker 2

Once this audience was shown Lumos Pharma's data demonstrating that moderate idiopathic PCSK patients selected by our PEM strategy grew in line with historical averages. There was an acknowledgment that LUM-two zero one had the potential to become the 1st oral therapeutic to address this patient population created solely by injectable therapies for the last 40 years. And we are pleased that interim data from our both Oral growth trials were also accepted for presentation at the upcoming pediatric Indian Society meeting later this week, Including an oral presentation on oral growth 210 trial interim data given by Doctor. Andrew Dawber, Chief of Endocrinology at Children's National Hospital and a poster presentation on Oral Growth 212 trial interim data by Doctor. Kathorla and presented by our own Doctor.

Speaker 2

David Karp. We believe that presentations at MPE and PES and other medical meetings will continue to increase awareness of BLUM-two zero one among the pediatric Endocrinologists' community and Gartner even greater excitement for the potential for oral therapeutic candidate in pediatric growth hormone deficiency. Now turning to other developments now. We continue to support our clinical collaboration with Doctor. Laurel at Massachusetts General Hospital to explore the potential of LUM-two zero one in non alcoholic fatty liver disease or NAFLD.

Speaker 2

This investigator initiated pilot study was supported by data presented by Doctor. Dichtell at the ENDO 2022 conference. And at that medical meeting, Doctor. Dichteller reviewed positive data evaluating injectable growth hormone in NAFLD We supported the assessment of oral LUM-two zero one in the same indication. As NAFLD is a chronic condition Rembrandt and approximately 25% of adults worldwide, a daily oral therapeutic such as LUM-two zero one Could provide a welcome alternative to a lifetime of daily injections of gross hormone in this indication.

Speaker 2

Enrollment in this pilot study, LUM-two zero one in NAFLD is continuing. And as we mentioned on our last earnings call, LUMOS Pharma filed a novel formulation patent for LUM-two zero one last year, which could have sent IP protection to 2,040 2. Now currently, LUN-two zero one has patent protection through 2,036 for the detection and treatment of growth hormone deficiency As well as orphan drug designation, which offers extended protection of up to 7.5 12 years And the date of drug approval in the U. S. And Europe respectively.

Speaker 2

We expect to hear from the U. S. Patent Office later this year. As previously mentioned, we believe that LOOM-two zero one has the potential to address up to 10 other indications currently treated by injectable growth hormone. We've done a lot of work internally to assess the potential of BLINC-two zero one in other indications and geographic regions.

Speaker 2

As we mentioned on our last earnings call, we have narrowed our focus to idiopathic short stature or ISS and Prader Willi Syndrome where we see a sizable opportunity both in the U. S. And internationally. While we assess these opportunities, We remain committed to the prudent use of our cash and ensuring our capital allocation is focused on advancing our core program. With that, I'm going to turn it over to Lori for a review of our financial results.

Speaker 3

Thank you, Rick. Lumox Pharma ended the quarter on March 31, 2023 with cash, cash equivalents and short term investments totaling $58,000,000 compared to $67,400,000 on December 31, 2022. We reiterate our expectation for average cash use of approximately $9,500,000 to $10,500,000 per quarter through 2023. Cash, cash equivalents and short term investments as of March 31, 2023 are expected to support operations into the Q3 of 2024, well beyond our announcement of top line Phase 2 trial results in the Q4 of 2023. Research and development expenses were $4,400,000 for the quarter ended March 31, 2023, an increase of approximately $100,000 compared to the prior year period.

Speaker 3

The increase was primarily due to an increase of 0 point $5,000,000 in clinical trial expense and $100,000 in legal and consulting expenses offset by a decrease of $500,000 in contract manufacturing expenses. The increase was primarily due to increases of $400,000 in licensing expenses, dollars 200,000 in personnel related expenses and $200,000 In travel expenses, the net loss for the quarter ended March 31, 2023 was $7,300,000 compared to a net loss of $7,700,000 for the same period in 2022. We ended Q1 2023 with 8,183,296 shares outstanding. With that, I will turn the call back to Rick to conclude for us.

Speaker 2

Thank you, Laurie. To recap, our Phase 2 clinical trials for LUM-two zero one are now fully enrolled,

Speaker 4

And we're

Speaker 2

in a position to report top line data from both studies in the Q4 of 2023. Additionally, between our interim data announcement Last November, in full enrollment, age and other baseline characteristics for oral growth 210 subjects are converged across 1.6 mg per kg LUM-two zero one and growth hormone cohorts as predicted. Given the stratification of the trial by age and the balancing effect of the additional subjects included at full enrollment, Our confidence in these trials is reinforced by additional data presented at Empe in March and our data to be presented at PES later this week. We believe that presented data further demonstrate that LUM-two zero one possesses both a favorable safety profile and a natural endogenous mechanism of action with potency to stimulate meaningful growth in this idiopathic PGS D patient population. We continue to support the exploration of LUM-two zero one for the treatment of NAFLD through a pilot investigator initiated trial.

Speaker 2

We have narrowed our focus for future indications for LUMQ-one to 2 compelling opportunities in attractive markets. In addition, by prioritizing our PGHD program and being conservative with our cash usage, We expect our current capital to support operations into the Q3 of 2024. We also submitted patent application for Loom201, which if approved, extended IP protection for the commercialized version of Loom201 through November Of 2042. So 2023 is off to a good start for Lumos Pharma. We believe we're poised to demonstrate that LUKOIL1 has the potential to disrupt the worldwide growth hormone market This has been dominated for almost 40 years by injectable products.

Speaker 2

We're excited to continue to advance our programs and look forward to disclosing top line data the Q4 of 2023. Thank you very much. And operator, we're ready to take questions.

Operator

Thank you. At this time, we will conduct the question and answer session. Please standby while we compile the Q and A roster. Our first question comes from the line of Catherine Novak of Jones Research. Your line is now open.

Speaker 5

Hi, good afternoon. Congrats On the quarter, thanks so much for taking my questions. I'm just curious as you're attending these medical meetings, speaking with KOLs, How significant are 6 month AHV data for KOL's new space? We know that the important endpoint it's going to be 12 months and that's where you also start to see the attenuation of injectable growth hormone, AHV. So how important is it going to be to show 12 data in this Phase 2b as well?

Speaker 5

And can you give us a sense of how much follow-up you'll have for some patients beyond the initial 6 months?

Speaker 2

Thanks for the question, Catherine. I'm going to let our Chief Medical Officer, David Karp, begin to answer that question. I think that John You may have something to add. So go ahead, David.

Speaker 4

Sure. Thank you very much for the question. It's important to point out that the Phase 2 studies For all of the I'm going to

Speaker 6

ask you to go to

Speaker 4

the ones, our 6 months in duration. So 6 month HV is, to some So I should point out, the 12 month is the endpoint in the pivotal Phase 3 trials. And we were very encouraged that the 12 month data looked actually even better for us than the 6 month data did in the interim analysis. Let's see. We are committed As you know, we have fully enrolled all 82 subjects well, each subjects in the 210 trial, 11 subjects in each group in the 212 trial and we'll have the 6 month height velocity on all of those subjects in the 4Q presentation.

Speaker 4

So we also showed some 9 12 month data. In this kind of a data, we'll have even a greater sample size At 912 and later time points as well. So, we should have a reasonable amount of 12 month data in the Q4 to present. John, do I have anything?

Speaker 7

No, I mean, I think I'll just stress a little bit, Catherine, that we'll have data beyond 12 months as well. So the durability of the fact can be there And we can really understand what the slope is of an individual patients growth is.

Speaker 5

Got it. And then I'm wondering if you can give me a sense of next steps, potential next steps following the Phase 2b readout. Can we anticipate initiation of registration and directed Phase 3 program? How confident are you in taking this 1.6 mg per kg dose Forward into pivotal studies and are there any modifications you might make to enrollment or trial design for a pivotal study?

Speaker 2

Thanks, Catherine. I can tell you, we are fully committed to the whole team And doing everything it's going to take to start this Phase 3 study as quickly as possible, including the recruitment Of the study sites around the world. First, we know who the high responders have been in the Phase 2 study. But in addition to that, we're adding some other territories around the world that have been traditionally high in rollers in countries like China, Extremely hiring rollers in fact. But also I think we're pretty confident going forward given the fact That when all the long acting growth hormone studies were being conducted, especially the later stage, There was a great deal of competition between 3 separate companies who are competing for the same patient population.

Speaker 2

I think we have a sort of a clean slate And we don't have this as much of a let's say that the competition has been diminished. No one else as far as we know doing trials in PTSD out there at this time or by the time we start our Phase 3. John, do you want to add in terms of the other part of that question?

Speaker 7

Sure. So I heard 3 questions, Catherine, so I'll start with the first one, which is kind of timing of moving from Phase 2. So we are incredibly methodical in how we're approaching this and Thinking through this large data package that we're going to get, obviously, we'll analyze it Quickly and get it out in top line form to investors and the public. We'll also dig very deeply into this data and prepare our Request for an end of Phase 2 meeting with the FDA, right? And that'll be a very important step towards moving to our pivotal.

Speaker 7

We'll negotiate kind of the outline for the Phase 3 trial, the non inferiority margin, all the pieces that are going to be required for us to move forward. We'll also have a Discussion with the FDA about our Phase 3 commercial products. So I think that'll be a very important next step. And as Rick said, we'll be working very, very closely with Our CRO to do as much upfront work as we can in the U. S.

Speaker 7

And globally, right? And so when we get both the U. S. And ex U. S.

Speaker 7

Regulatory authorities, okay, that we'll be ready to really quickly move into enrolling subjects. So you also asked about the 1.6 being the dose to move forward. And I think that's right now, that's What we feel comfortable with after the interim analysis, but obviously we're going to wait until we have the full data set of Q4 to make a final decision there. The data right now points to 1.6 as being optimal. And the third part of your question was about Patient selection in Phase 3.

Speaker 7

And I think the way I would answer that is that there's we've learned an enormous amount About the kind of patients who respond to our ILUM-two zero one from our Phase 2 study, right? So we Not only figured out what those patients are like, we've applied this And strategy to those subjects to really isolate them prospectively and bring them into the trial. We understand In terms of enrollment, we found a number of KOLs who are really good at ICE We're finding these patients among their treated population. So I think we have a lot of knowledge to apply to the Phase 3 kind of final designs to help us very quickly isolate the right population and look

Speaker 2

forward.

Speaker 7

So I think I've checked off all three of the questions.

Speaker 2

Yes, Cath, I might add, with meetings like MPA and other connections with the investigator community, I think there's a and we as we share the results with these investigators and KOLs, I think there's almost a palpable excitement in the community Because this is the first time that they've been able to work with an oral therapeutic in this space. So I think that bodes well for us And getting enrollment going well for this Phase 3 trial.

Speaker 4

Can I add a little bit?

Speaker 2

Sure, sure. Go ahead, David.

Speaker 4

Okay. You also seem to ask about the design of the Phase 3. And there what I'd like to do is say that the key factor Relief for successful Phase 3 is having a good homogeneity of the patient population and having good balance between the groups. I'd like to point out that the you can see the impact of heterogeneity if you look at the Phase 3 ASCENDU's HEIGHT trial versus the Phase 3 REAL4 Segroiter trial. Both of those trials are inherently more heterogeneous than our trials because they rule the full spectrum of disease, severe organic To moderate, less severe idiopathic.

Speaker 4

But the REAL-four SUGRAY trial had a higher percentage of severe patients than did the HIDE trial. As a result, the growth hormone response in the control group of that trial was 11.7 centimeters per year in 12 months. In contrast in the HEIGHT trial with severe but not as many as the real, the response was 10.3, 1.4 centimeters lower. Because our Phase 3 will be much more homogeneous trial because we're only selecting the less severe idiopathic who are further also Pemchosen, it will be inherently more homogeneous than either of those two trials. And because it's less severe, we would expect to see A high velocity similar to that in the EDUFAF population in the high trial, which is concordant with the databases, Right, 8.3 to 8.6 centimeters per year.

Speaker 4

And then, there are 3 factors which will predict the Phase 3 trial will be even more homogeneous, Certainly, it's not our Phase II trial. Most importantly, and have a better balance, important to the Phase II trial. As far as balance, The Phase 3 will be much, much larger than Phase 2. So the Phase 3 will be one dose of LUM-two zero one in 120 to 140 subjects Versus 60 to 70 subjects on growth hormone. So the much larger trial inherently would get much more A better much better balance than either our 10 subject per group interim analysis or even the 20 subjects we're reporting In Q4.

Speaker 4

The greater size of the trial also allows us to stratify by 3 factors instead of 2 factors, which will also improve the balance. And then what really speaks to the homogeneity is that based upon our learnings from Phase 2, we're actually adding 2 inclusion criteria, Which will make it important very similar to that in the high trial, which should further improve the homogeneity of the trial. Does that answer your question about the Phase 3 design?

Speaker 5

That does answer the question. Thanks so much for taking the questions. Looking forward to data coming up at the end of this year. Thank you very much.

Speaker 2

Thank you, Catherine.

Operator

One moment for our next question. Our next question comes from Charles Duncan of Cantor Fitzgerald. Your line is now open.

Speaker 6

Hi. Yes, good Rick and team, thanks for taking our question and congrats on progress in the quarter. I had a couple of questions. First about the NPE meeting, it sounds like, well, as you characterize it was enthusiasm was Helpful. And I guess I'm wondering when you talk to KOLs about the severe versus moderate Patient population.

Speaker 6

I guess I'm wondering if that is a paradigm that is going to prove to be A challenge in the commercial setting or do you think that the use of the PEMS system is really going to help out there? And then in terms of the implications for the Phase 3 program, could we almost assume that the PEMs and I guess the enthusiasm or confidence that a patient may respond is going to enhance enrollment rates perhaps over the past experience in this field?

Speaker 2

Thanks for the question, Charles. And John, I'm going to let start with your answer.

Speaker 7

Sure. Thanks, Charles, we appreciate the question. So I think we had, as Rick mentioned, a presentation and a poster At Inpay and Booth as well. And so we got lots of interactions with, I think, Some of the key European, South American and U. S.

Speaker 7

Pediatric endocrinologists. And They really got an understanding of a broad understanding of the mechanism of action of our molecule and how it is different than growth hormone. And I think that connection with the type of patient population that is going to be treated is very clear to most of the that we chat with and they really do see the impact this molecule could have in that population, right, the more moderate PGHD population. And I'll just mention broadly in this population that there are kids who are needle phobic and The kids who are probably most needle phobic tend to be in that moderate tGHD population, right. The kids are really severely birth and deficient Are going to take the shots or take the treatment however it comes.

Speaker 7

And I think that there is an opportunity there for This group of kids who is more moderate and maybe a little needle averse, I think clinicians really like the opportunity to be able to offer them That's oral. So I think there's a good understanding as we start to present more and more of this clinical data that we've been releasing among the Pediatric Endocrinology Community about the advantages that this molecule offers to the moderate population. Very

Speaker 2

good. Anything you want to add there?

Speaker 4

I'll just add that Even in the current time, pediatric endocrinologists are dividing their Population into idiopathic versus severe organic. And they're really seeing a lot more idiopathic Right now, they just can offer them 1 therapy, but I think that this will play into the enrollment in our trial because they're already making The separation between the organic and the idiopathic population in their current Kids, I think also that the so just having idiopathic GHD is pretty predictive for response to M21 and that's really augmented I also have been PEM positive. I will also point out that the PEM test based upon the Phase 2 data will be much simpler in Phase 3 and at launch With simply the single dose of LUM-two zero one and a single blood test an hour later, which I think will be very, very acceptable, Because it's far less burdensome than the 2 stim tests these kids undergo with all of our pediatric endocrinologists.

Speaker 6

Helpful, John and David. I appreciate that added color. Had a question about the commercial form. I'm not sure if I Missed heard or misheard this, but do you have a commercial form to be able to move into Phase 3? Or is that something you're still developing?

Speaker 6

And would you start a Phase 3 without a commercial form if it is the case that you're still developing?

Speaker 2

Good question, Charles, and I'm going to let John answer that.

Speaker 7

Yes. I mean, obviously, we want to Interact with the FDA and get agreement with them about how we're going to move forward with a commercial product. And the best Possible way to do that is to fully explore that product in Phase 3. And I think We've talked about the patent we recently submitted, which is tied into formulation and Aspects of the molecule that let new opportunities in formulation. So I think we're going to move down that path with agreement with the FDA about the best way to utilize the molecule and make this A really easy molecule to administer.

Speaker 7

So our plan is to move forward And get agreement with the FDA, as I mentioned, as we move towards a Phase 3 study.

Speaker 2

And to answer your question further, Charles, Of course, that product will be used in our Phase III trial.

Speaker 6

Okay. That clarifies it. Then last question is more strategic. John or Rick, excuse me, we've talked in the past about Potential for other geographies in which PGHD is really a big Yes, Xu. And I think you alluded to China earlier this call.

Speaker 6

And I guess I'm wondering, do you think that the data that you could In the Q4, could even further enhance opportunities To pursue development either with a collaborator or not in other geographies.

Speaker 2

Yes. I don't think there's any question that when a company gets at this stage in the development of their product, There's typically what happens is a number of players show up. And as you know, in this, this is a mature space. There are major players. And there's no question that we'll be fully engaged And choosing the right partners at the right time, the right territories is a nice problem to have.

Speaker 2

And our business development is led by almost a 30 year veteran, Aaron Stewart. And Aaron is a busy man these days, let's put it that way.

Speaker 6

Okay. Thanks for taking my questions.

Speaker 2

Thank you, Charles.

Operator

One moment for the next question. Our next question comes from Leland Gershell of Oppenheimer. Your line is now open.

Speaker 8

Thanks for the update and for taking my questions. Rick, wanted to know in addition to the baseline characteristics update for the 1.6 milligrams, I know that's the dose of focus for FORWARD study, But wondering if you may give us the opportunity to hear on what may be a narrowing of the baseline differences in the other Two doses that you're looking at in the study? Thanks.

Speaker 2

Yes. John, I'm going to let you I answered that question, but thanks for the question, Leland.

Speaker 7

Yes. We just focus on 1.6% growth mark Those are kind of the 2 most important cohort doses or the 2 important cohorts to compare based on our interim analysis, But we obviously have a fine data. And I think the trends are similar across all the cohorts. There's a little moving around here and there Among the different cohorts, but they all are moving generally in the same direction.

Speaker 8

Okay. Thank you. And also want to ask with respect to starting the Phase 3, this may have been asked earlier, but with respect to the New oral formulation, assuming that the USPTO grants those claims, would you would it simply be a matter of a bridging study, bioequivalence Type study to get into the Phase 3 or any other clinical work that you have to do? Thanks.

Speaker 2

Yes. Go ahead, John.

Speaker 7

Yes. I mean, in general, when you introduce new formulation, you have to do a bridging study, right, to compare back to the data that you have in Phase 2. So that is correct.

Speaker 8

And presumably that could be done inside of 2024. How should we think about The timing of that with respect to then starting the next pivotal study?

Speaker 2

John?

Speaker 7

Yes. I mean, we're obviously keen to keep that up as As soon as possible, right? As soon as we can, because I think it is it would be key to moving forward. So yes, it is definitely on our to do list.

Speaker 2

Yes. Okay. As much as any work that we can do, Leland, on a parallel basis, Without too much risk, we're going to do. We want to start this Phase 3 study as quickly as we possibly can.

Speaker 8

Right. Okay. So Rick, so it sounds like you may be generating those data in parallel with completing the 210 and 212 studies Or at least starting to and then we you could be a bit accelerated as we get into starting the Phase 3?

Speaker 2

Well, There's a lot of work to be done between the completion of the trial, the reporting of the data, everything that needs to be done leading up to the In the Phase 2 meeting with the FDA. So yes, so in that time period, obviously, there's a lot of parallel work that's going to be done.

Speaker 8

Got it. Very good. Okay. Thanks very much for taking our questions.

Operator

One moment for our next question. Our next question comes from Ed White of H. C. Wainwright. Your line is now open.

Speaker 7

Good afternoon. Thanks for taking my questions. Actually, I just have one. You had mentioned that you'd narrowed the indication targets To ISS and PWS, just wondering how to think of the timing to the start of The next indication, do you think that it's possible to decide which target you will go after this year And then initiate the study for the new indication next year, can you run that study alongside of the Phase 3 That you're planning?

Speaker 2

Yes. Yes. Good question, Ed. And obviously, As a company at this stage in development and the market conditions as they are, we're really fortunate to have The cash runway that we have in terms of getting the readout of these studies, we're not going to we're going to focus all of our resources On the PGHD indication, obviously, but as the market evolves and we're able to Access more capital, then we'll make those decisions along the way. But obviously, these are we spend a lot of time with the KOL community and ISS PWS, know that there would be excellent directions for us to take and we'll probably start those studies as

Speaker 7

Okay, great. Thanks Rick.

Operator

One moment for the next question. Our next question comes from Yasmeen Rahimi of Piper Sandler. Your line is now open.

Speaker 5

Hi, team. Thank you so much for taking the questions. 2 for you. Maybe, if you could just allude to what the cost of the Phase 3 would be based on some of the analysis that you have run, so you could provide some color there. And then also maybe some color around when you do the math in terms of like enrollment timeline and 12 52 week treatment duration like assuming you start your Phase 3 in early 2024, like How soon could you be able to bring it to the finish line?

Speaker 5

If you could just provide some timing around that would be helpful. And then the 3rd Component of my question is, like, what are the various data scenarios That you are expecting to see and what action would you take as we're headed into the top line data from 210 and 211. And I'll jump back into the queue. Thank you.

Speaker 2

Okay. Thanks, Yasmeen. Good to hear from you. Appreciate the question. I think there's a great deal of precedence in terms of how long it's taken most companies To enter about a study of about this size.

Speaker 2

And I want to go back to, I think my discussion a little bit earlier and the fact is that there was a great deal of competition between 3 companies Concurrently conducting 3 large Phase 3 studies globally. That doesn't exist for us. That's one positive thing. And Also what John said earlier and the fact is that we I think we've taught the KOL community quite well in terms Of the type of patient we'd be looking for in this clinical trial, in addition to the fact that obviously Derisking all the trials by using our PEM strategy. In terms of time to enroll, I think once again, I think the ASCENDIS trial, the Phase III took about 18 months.

Speaker 2

I think the trial was perhaps a little bit larger than we're thinking of or roughly so. But as I said, We don't have the restraints of a great deal of competition from a number of areas. In addition, by Adding a geographic area like China that has super enrollers, I think we're going to do everything we can to even Beat that strategy. And of course, all patients will be treated for a year. In terms of the Data scenarios and we're expecting to see John, would you comment please?

Speaker 7

Sure. Maybe I'll just comment a little bit first on the Timing, yes, I think I'll just reiterate that when our data package does read out in Q4 of this year, We have to put together a package and request a meeting for end of Phase 2 with the agency. And from there, we have to agree on our Phase 3 protocol, the non inferiority margin. And then after that meeting, we submit the protocol and then we get the okay from the We also have to reach out to all of the other regulatory agencies ex U. S.

Speaker 7

Get their buy in and then we can officially start the Phase 3. We'll do as much upfront work as we can As we talked about in terms of lining sites of getting everything ready as quickly as we can while we wait for all those regulatory approvals. So I just wanted to Jeff, through the timeline of starting Phase 3. And then, what was the other piece you wanted to

Speaker 2

comment on? The last one was the cost of the Phase 3, and I'm going to let Laurie jump in and answer that question.

Speaker 3

Sure. Yes, I think at this point in time, we are not giving any guidance around the cost The Phase 3, we continue, as John mentioned, to refine and work on the planning and there's still quite a bit To be done, to be prepared for a Phase 3. And so at the point in time that

Speaker 4

we have those specifics of the costs available, we will definitely put out that guidance. Yes. And yes, I think that in spite of

Speaker 2

what the current market conditions are, I think there have been Really good examples of several companies or multiple companies who have been successful in raising capital with positive results. And so that's heartening to us. I think there seem to be Even more and more examples as time goes on. So we hope that the market conditions are also improved by the time we get this readout.

Speaker 5

Great. Thank you so much, team.

Speaker 2

Sure.

Operator

Thank you for your participation in today's conference. This does conclude the program. You may now

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