Lumos Pharma Q1 2024 Earnings Call Transcript

Quartr
Provided by Quartr
May 15, 2024

There are 12 speakers on the call.

Operator

Greetings and welcome to Lumos Pharma First Quarter 20 24 Financial Results and Clinical Programs Update Call. At this time, all participants are in a listen only mode. A brief question and answer session will follow the formal presentation. As a reminder, this conference is being recorded. It is now my pleasure to introduce your host, Ms.

Operator

Lisa Miller, Vice President, Investor Relations. Thank you, Ms. Miller. You may begin.

Speaker 1

Uncertainties that could cause actual results to differ materially from historical experience or present expectations. 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 these forward looking statements. Information presented on this call is contained in the press release we issued yesterday afternoon and in our Form 8 ks, which may be accessed from the Investors page of the Lumos Pharma website. Speaking on today's call will be Rick Hawkins, CEO and Chairman John McHugh, President and Chief Scientific Officer and Lori Lalley, Chief Financial Officer.

Speaker 1

Doctor. Duke Paduktuanan, Chief Medical Officer, will also join the call for the question and answer session. It's now my pleasure to turn the call over to Rick for our opening remarks.

Speaker 2

Thank you, Lisa, and good morning, everyone. I'm pleased to be speaking with you today to provide an update on our progress advancing LUM-two zero one as the first oral therapeutic for moderate pediatric growth hormone deficiency or PGHD. Our recent months have been incredibly productive at Lumos, marked by significant milestones. We've had a constructive end of Phase 2 meeting with the FDA and conducted fresh analyses on updated data from our ORGrow-two twelve and 210 trials. I'll keep my opening remarks concise before handing over to my colleagues to review in greater detail the slides we've made so far this year.

Speaker 2

So let's begin. As detailed in our press release, we recently held a collaborative in the Phase 2 meeting with the FDA, where we reviewed the promising data from our Phase 2 ORROV-two ten and 212 trials and discussed an optimal Phase 3 design for a successful regulatory path forward. Both Phase 2 oral growth trials, as you may recall, successfully met their primary and secondary endpoints, providing robust evidence supporting the potential of oral LUM-two zero one in treating moderate PGHD. Additionally, the trials demonstrated the effectiveness of our predictive enrichment marker test in identifying patients likely to respond to LUM-two zero one. During our dialogue with the FDA, it was acknowledged that LUM-two zero one operates distinctly from growth hormone or its mimetics.

Speaker 2

The FDA recognized LENP-two zero one as a novel growth hormone promoter with a unique mechanism of action and consequently proposed that we consider a placebo controlled Phase 3 trial design. This trial design would not require an active comparator arm with a non inferiority margin, but would necessitate demonstrating clinically significant improvements in growth compared to placebo. Now we are delighted by these developments as we see them greatly enhancing the prospects for a successful Phase 3 trial of VLUT-two zero one. Pending final FDA approval of this trial design, we plan to initiate this trial by year end 2024. We firmly believe that this refined design significantly bolsters our chances of advancing LUM-two zero one toward approval as the first oral therapeutic for moderate PTHD.

Speaker 2

PGHD. Our confidence in LUM-two zero one was further strengthened through our examination of additional data from our Phase 2 oral growth trials. As detailed in our press release, we unveiled updated 12 month and 24 month data from these trials, which We are confident that the advancements made position Lumos Pharma as a late stage biopharmaceutical company with substantial growth prospects and continue to support the potential of oral LUM-two zero one to disrupt the $4,700,000,000 global market injectable growth hormone deficiency. Now I'll hand the call over to John McHugh for further insights into our interaction with the FDA and our strategic planning for this pivotal Phase 3 trial. John?

Speaker 3

Thanks, Rick, and good morning, everyone. As Rick mentioned, we recently concluded our end of Phase II meeting with the FDA to evaluate data from our oral growth trials and explore potential strategies for a Phase 3 pivotal trial. A key highlight from the meeting was its overwhelmingly positive and constructive nature. With approximately 30 FDA staff members present, including senior representatives from pertinent departments, the atmosphere remained collegial and centered on determining approach for the pivotal trial design of LIM-twenty one in treating moderate PGHD. The data package we presented to the FDA underscore the outcomes of our Phase 2 oral growth trials.

Speaker 3

As you may recall, these trials successfully met their primary and secondary endpoints demonstrating that LOOM-twenty one achieved annualized height velocity consistent with predetermined targets derived from historical benchmarks in a moderate PGHD patient population. Additionally, the growth rates observed at both 6 12 months on treatment with LOOP-two zero one at the 1.6 mgkgday dose aligned with historical recombinant human growth rates in similar patient cohorts with sustained efficacy observed at both 12 24 months. Notably, LOOM-twenty 1 also exhibited the ability to normalize IGF-one standard deviation scores within 6 months of treatment initiation. Furthermore, the trial data provided preliminary confirmation that our predictive merchant marker or PEM strategy accurately identified potential ILUM21 responders and showcased the PEM classification as 100% reproducible, surpassing the predefined statistical margin. Additionally, safety profile of investigational LUM-two zero one was further validated.

Speaker 3

Upon reviewing these data, FDA representatives indicated a shift in their perspective regarding LUM-two zero one's distinct mechanism. They acknowledge that LUM21 is not merely a growth hormone mimetic, but rather a distinct growth promoter. The FDA's understanding of these data prompted them to suggest that LUM21 should not necessarily be directly compared to traditional growth hormone products. As a reminder, LOOM-twenty one is a small molecule that binds to the growth hormone's cratogog receptor 1a in both the pituitary and the hypothalamus. LUM21 agonizes the receptor and that signals the release of stored growth hormone in the somatotropes.

Speaker 3

By also modulating somatostatin and growth hormone releasing hormone in the hypothalamus, LUM-two zero one restores the natural pulsatile release of growth hormone. The increase in the amplitude or peak of each growth hormone pulse boosts the circulating levels of growth hormone, subsequently elevating the levels of circulating IGF-one. Both growth hormone and IGF-one then act on the growth plates in the long bones of children with growth hormone deficiency stimulating growth. The FDA's acknowledgment that LUM21 operates as a novel growth promoter rather than a mimetic of injectable exogenous recombinant human growth hormone enables a more expansive view in considering design approaches for a Phase 3 trial. A significant portion of the meeting was dedicated to exploring different options, culminating in the suggestion by the FDA that we contemplate a placebo controlled design.

Speaker 3

Heading into the FDA meeting, we examined historical pivotal trial designs with growth promoting agents and proposed to the FDA a standard non inferiority design consisting of, in this case, a 12 month study evaluating BLU-two zero one against the lower approved dose of growth hormone. We chose this comparator dose because it more closely mirrors the physiological levels of growth hormone and IGF-one that LIM-two zero one restores. We engage in productive discussions with the FDA regarding this non inferiority approach, but during these conversations, the FDA suggested we could explore a placebo controlled trial. A placebo controlled trial would be required to demonstrate clinically significant growth compared to a placebo with 12 months of treatment. Following the FDA's recommendation and drawing from insights provided by our regulatory consultants, clinical and scientific advisory board, statisticians, we have designed a placebo controlled trial featuring a 2:one randomization of LIM-two zero one to placebo.

Speaker 3

One arm will receive LUM-two zero one for 12 months, while the other arm will initially receive placebo and then transition to LUM-two zero one after 6 months, remaining on treatment for the next 6 months. All subjects enrolled in the trial will be PEM positive. This Phase III trial design serves 2 strategic objectives. The first, providing the FDA with ample data to evaluate LUM-two zero one for approval and ensuring that all subjects receive treatment with LUM-two zero one, the active agent. For this trial, we envisage 2 co primary endpoints.

Speaker 3

First, the 12 month treatment arm must demonstrate clinically significant growth. 2nd, there will be a pairwise comparison within subject assessing growth on placebo versus growth on LUM-two zero one, which must also exhibit clinically meaningful growth. Following the 12 month trial period, all participants will have the option to transition into long term safety extension, which will provide LUMTA-one treatment for up to 3 years. We are confident that the trial size is more than adequate to meet the described co primary endpoints. We anticipate finalizing this cystin over the next couple of months and pending approval from the agency we aim to initiate the Phase 3 trial before the end of 2024.

Speaker 3

Our potential for success in a pivotal trial is bolstered by the evolving data and deeper analysis from our oral growth trials. In yesterday's press release, we unveiled preliminary updated 12 24 month growth data from our Org Growth 210 trial. The data snapshot from our trials, specifically the 6 and updated 12 month data in LUM-two zero one when measured against similar we are focusing on treating the moderate PTHD population while excluding the more severe growth hormone deficient cases. Our updated data consistently demonstrate that we are achieving growth rates similar to those observed in historical data for the moderate PGHD patient cohort. Further analyses compare the 6 12 month growth rates on LUM-two zero one and baseline growth rates.

Speaker 3

These findings highlight a noteworthy increase in AHV or annualized site velocity from baseline upon treatment of LUMTA-one. The baseline growth rate of 4.7 centimeters per year documented in our Phase 2 trial should serve as an indicator of the placebo arms annualized growth rate in our Phase 3 trial. The full 12 month data for all cohorts in the ORGOV-two ten trial reinforces our choice to advance the 1.6 mg per kg LUMETO1 dose into Phase 3. Finally, we combined the 1.63.2 mgs per kg per day cohorts from our 2 Phase 2 trials since their growth rates were not statistically different. These data include all subjects who are treated to 24 months, excluding those who transitioned from TENOR I to TENOR II to enable a comparison to historical Pfizer KIGS database.

Speaker 3

These updated data continue to the enduring efficacy of LUB-two zero one. When comparing year 1 AHV to year 2 AHV for LUB-two zero one, only a modest drop off of approximately ten percent is observed. This stands in contrast to the published data from the less severe GHD population in the KEGS database treated with recombinant hemagrowth hormone where the drop off is closer to 20%. These findings from our Phase 2 studies highlighted how LUM-two zero one can normalize growth hormone secretion and IGF-one levels, thus restoring normal physiological growth with sustained benefits over time. These data were presented at the FDA end of Phase 2 meeting and were compelling enough to convince the agency of LUMTA-one's novel mechanism of action, granting them the freedom to suggest innovative Phase 3 study designs.

Speaker 3

We believe that this placebo controlled design significantly mitigates our regulatory risk and enhances our potential to introduce the 1st oral therapeutic for growth hormone deficiency to the market. This innovative oral therapeutic is anticipated to be a welcome treatment option for pediatric endocrinologists seeking to address pediatric growth and deficiency in their patients. Earlier this month, analyses of top line oral growth trial results were presented at 3 major endocrinology conferences: at the Pediatric Endocrine Society meeting in Chicago by Doctor. Andrew Dawber, highlighting data showing LUM21 achieved comparable annualized to daily recombinant growth hormone in PGHD at the 1.6 mgkgday dose with a promising safety profile. And at 2 subsequent conferences in Stockholm, the Growth Harmonic Research Society and European Congress of Endocrinology, Doctor.

Speaker 3

Peter Clayton presented a comprehensive analysis of LOOP-two zero one restoration of growth hormone secretion and the increase in HV induced and moderate PTHD

Speaker 2

patients.

Speaker 3

We are pleased to announce that we have had 2 abstracts accepted for presentation at the upcoming Endocrine Society meeting or ENDO next month, where we also plan to release the full 12 month data from our ORGrowth-two twelve trial with additional analyses from the ORGrowth 210 trial. The maturing data from our ORGrowth trials continue to resonate with the global endocrinology community and we are encouraged by the growing interest among experts as we finalize our plans for pivotal trial. With that, I would like to turn it over to Lori for a review of our financial results for Q1.

Speaker 4

Thanks, John. We ended the quarter on March 31, 2024 with cash, cash equivalents and short term investments totaling $23,200,000 as compared to $36,000,000 on December 31, 2023. Cash on hand is expected to support operations through the Q3 of this year, inclusive of Phase 3 planning and preparatory activities. With the recent developments from the FDA, we are confident that we will be able to finance our Phase 3 trial for patients with pediatric growth hormone deficiency in the near term. Research and development expenses were $7,200,000 an increase of $2,900,000 for the quarter ended March 31, 2024 compared to the same period in 2023, primarily due to increases of $2,000,000 in licensing expense, dollars 800,000 in clinical trial expenses and $200,000 in consulting expenses, offset by a decrease of $100,000 in personnel related expenses.

Speaker 4

General and administrative expenses were $3,800,000 a decrease of $600,000 compared to the same period in 2023, primarily due to decreases of $400,000 in licensing expenses, dollars 100,000 in travel expenses, dollars 100,000 in consulting expenses and $100,000 in other expenses offset by an increase of $100,000 in personnel related expenses. The net loss for the quarter ended March 31, 2024 was 10,400,000 dollars compared to a net loss of $7,300,000 for the same period in 2023. Lumos Pharma ended Q1 2024 with 8,107,000 121 shares outstanding. And now I will turn it over to Rick for his closing remarks.

Speaker 2

Thank you, Lori and John. As mentioned earlier in the call, it's been an exciting and fruitful time for Lumos, and we are steadily progressing towards our objective of unlocking the potential of LUM-two zero one as a pioneering oral therapeutic poised to revolutionize the global growth hormone market, which has been dominated by injectable products for nearly 4 decades. Before opening the call to your questions, I'd like to take a moment to discuss the commercial potential we envision for oral LUM-two zero one. If approved, we believe LUM-two zero one presents several potential advantages over current injectable recombinant growth hormone products, including sustained growth benefits and the restoration of physiological pulsatile release of growth hormone within the natural endocrine feedback loop. As an oral therapy, LUM-two zero one represents an appealing alternative to daily and weekly injections and has the potential to broaden the pediatric growth hormone deficient treatment market, particularly among moderately growth hormone deficient patients.

Speaker 2

Additionally, it's worth noting that as a small molecule, the cost of goods for commercial scale production of LUM-two zero one would be significantly lower than for recombinant growth hormone. And as we consistently emphasize, we view LUM-two zero one not only as a singular product, but also as a robust pipeline. We see the potential for LIM-two zero one to address 10 additional indications currently treated with recombinant growth hormone, including Prader Willi syndrome and idiopathic short stature. With the FDA acknowledging LUM-two zero one as a novel growth promoter, we believe this paves the way for a streamlined clinical trial design for these other indications as well as for attractive commercial positioning. Now this is truly a pivotal moment in the company's trajectory.

Speaker 2

With the positive guidance from the FDA, we're propelling our late stage program for oral LUM-two zero one towards a regulatory pathway with a high likelihood of success. Additionally, we anticipate some very exciting developments in the coming year and eagerly anticipate sharing updates on our progress with you. Thank you all very much for listening today. And operator, we're ready to take questions. Will, please.

Operator

Thank you. We will now be conducting a question and answer session. The first question comes from the line of Yasmeen Rahimi with Piper Sandler. Please go ahead.

Speaker 5

Hi, team. This is Liam Hustler on for Yas. Thanks so much for the update this morning. Just to start, in regard to the upcoming Phase 3 study, I was wondering if you could provide some update on your expected bar for success in connection to growth? And then also any clarity on why you chose the specific dose to move forward with?

Speaker 5

Further, what is the overall safety database requirement for approval? And have you had any chance to engage with the EMA in regards to the Phase 3 design as well? And then also if you could comment at all on how long you expect it to take to enroll the Phase 3 trial as well as if there's any overlap between the Phase 2 and Phase 3 sites. Yes, and that's for now.

Speaker 2

Okay. All excellent questions. And I think the bar to success question probably should be answered by John. So go ahead, John.

Speaker 3

Yes. So as we mentioned, we have to show clinically meaningful growth. And so what we have agreed to in the past with the FDA to define clinically meaningful growth is that agreement with the FDA at the end of 12 months in our Phase 2 trial subjects have to achieve 6.7 centimeters per year growth. That was what we defined as the minimal clinically meaningful growth to continue on treatment with LOOP-two zero one. So one of the options that we have is to present that as an option for clinically meaningful growth in this Phase 3 study.

Speaker 3

I think just to tackle some of your other questions, the 1.6 mgkgday dose we've shown throughout the study to be both numerically the highest AHV across the 3 doses. We don't really see a difference between the 1.63.2 mgkg per day doses. So I think there's no point to go to that higher dose. We do believe that there's a pharmacodynamic plateau, so we're not getting increased growth hormone secretion at the higher dose. So it would make sense that we wouldn't see an increase in AHV.

Speaker 2

And John And John Two of your four questions.

Speaker 3

Yes, the interaction with EMA. So the interaction with EMA, we will start that interaction after we have finalized our protocol with the FDA.

Speaker 2

Yes. And Duke, why don't you talk about the enrollment, number of patients enrollment and so on?

Speaker 6

Yes. So thank you. So I think answer the first question, do we have overlap of a Phase II sign of Phase III? Yes. There we have some Phase II sites to continue to pass it in Phase III.

Speaker 6

And also we plan to initiate a new site for Phase 3 as well. As you know, the number of subjects that enroll in Phase 2 much smaller than Phase 3, which is nearly double. We want to increase the number of country and number of sites around the world. At this point, we have significant kind of like increase awareness of Phase 2 trial at the global scale. We do get significant increase in interest of the KOL around the world as well.

Speaker 6

So we do believe that with enrollment timeline as we planned initially 15 to 18 months just be intact if we start their enrollment towards the end of this year. So again, pretty much everything on track and we do believe that we'll be able to enroll all those patients with the new placebo controlled trial at the timely manner and especially when we increase the number of site and the number of country to participate in this trial.

Speaker 2

Okay. Go ahead.

Speaker 5

Awesome. And then just one more question, sorry. Just, how do you plan on funding the Phase 3 beyond year end 3Q 2024? Are there any details on that?

Speaker 2

Yes. Laurie, go ahead.

Speaker 4

Yes. We've been really busy since we've had our FDA meeting and planning for the upcoming Phase III trial design. Now that we have some of the details finalized, we are confident that we can go out to the community and finance the Phase 3 trial as is planned. Also, as we've said previously, we believe that there is opportunity to bring in potential strategic potential partners for regional licensing deals, which would also allow for non dilutive financing in some of those territories that we would not necessarily go out and commercialize on our own.

Speaker 5

Great. Thank you so much.

Speaker 2

All right.

Operator

Thank you. Next question comes from the line of Charles Duncan with Cantor Fitzgerald. Please go ahead.

Speaker 7

Yes. Good morning, Rick and team. Congrats on the progress I did have a couple of questions on the Phase 3 trial design and then one on your perspective regarding FDA engagement. So with regard to the Phase 3 trial design, I'm wondering if you could affirm that the enrollment criteria in Phase 3 will be the same or if not how different from the Phase 2? And then the second question I had with regard to that is if you could provide a little more color on the co primaries.

Speaker 7

Is it a true co primary or is there call it a step down from the 1st 12 month AHV to the 2nd? Thanks.

Speaker 2

Yes. Duke, why don't you answer the first part of the Phase 3 design question in terms of enrollment criteria? And then, John, if you'll talk about the co primary?

Speaker 6

Yes. Thank you. That's a very good question. So the majority of inclusion, exclusion criteria pretty much similar compared to Phase 3 and Phase 2. The only small changes that we want to implement is number 1, the upper limit of the age enrollment, we're going to lower 1 year.

Speaker 6

The reason behind that because we want to make sure since we compare to placebo, we want to make very clear that no single subject would get into the tender stage 2 during the 12 month trial period. And we have a bonus criteria that initially the bonus delay was greater equal to 6 months, but now we're going to have it greater than 12 months. And other than that, pretty much the same. And again, part of this inclusion criteria for the moderate PTSD, PTSD. We do believe that majority of physician who actually will participate in this trial will see significant when we have a placebo controlled trial, the different primary endpoint would be very clear.

Speaker 6

As you know, I think in majority of those patients, as you see the data John presented earlier, the baseline hyperlocity coming in this trial was 4.7 and our trial achieved 7.6 centimeter at 12 months. So basically, we see very clearly that we can achieve the clinical significance based on the API requirement at the end of this Phase III trial.

Speaker 3

Helpful.

Speaker 2

And John, the co primary?

Speaker 3

Yes. So Chas, we do see these as co primary endpoints, the 12 month growth endpoint and the 6 month comparison to placebo, both showing clinically meaningful growth. So that's the plan going in. Obviously, we'll release more details once we have a final sign off from the FDA on the full Phase 3 protocol.

Speaker 7

And that will include powering, John? Of the 2 do you power to or does it not matter?

Speaker 3

No, we'll pair it to both. They'll both be equal they'll both be powered. And we do believe right now that we have that the end that we have is more than sufficient to power both of those endpoints.

Speaker 7

Okay. Neat design, is the agency

Speaker 6

Duke? No, they did not. And actually, most of their, they tried to get approved, the focus on the efficacy in terms of cord velocity. And the bone density trial in general, you may not see segment change in the 1st 12 months. So that would not require for this 12 month study.

Speaker 7

Okay. And last question regarding the agency perspective and always hazard for that speculating on that. But what do you think was the key shift or driver to the shift in the view of this of LUM-two zero one being a stimulator, not a simple mimetic. And did you get a sense that they appreciated the compliance challenges with the current standard of care with the growth hormone injectables?

Speaker 2

Yes. Go ahead, John.

Speaker 3

So I think the turning point, Chas, just in their viewing of our mechanism was around the extensive data that we've shared publicly and with the FDA about restoration of pulsatile ultradian rhythm of growth hormone release. And I think that data package was pretty substantial. And as you know, we've shown that we can restore normal pulses and levels of growth hormone across a 24 hour period in growth hormone deficient subjects, which amounts to about 20% of the growth hormone that you need from an exogenous bolus dose. And we're achieving almost the same amount of AHV. And I think that level of understanding and realizing that our growth number delivery is significantly more efficient because we're doing it in a pulsatile 24 hour period.

Speaker 3

I think that was really the key to differentiate us away from exogenous growth. I'm going to really open up this whole discussion about creative ways to evaluate the growth potential of LUM-two zero one in Phase 3.

Speaker 7

Good deal. Great to hear. Appreciation of the pulse hotel mechanism always a key point of our thesis. Thanks for taking the questions.

Speaker 2

Thank you, Jay.

Operator

Thank you. Next question comes from the line of Leland Gershell with Oppenheimer and Co. Please go ahead.

Speaker 8

Hi, good morning. Thanks for taking our questions. I also wanted to comment on the positive surprise to the agency view on the Phase 2 assignment. Couple of questions from us, just for saying that we advocate at the same time that we're trying to hear about the placebo control versus inferiority versus the early growth of the infection. Maybe early days, but want to hear to what extent could those data or the lack thereof of comparability data to go from an impact endocrinologists view in 201 as they go to potentially use

Speaker 9

it in their patients? And 2, want

Speaker 8

to ask, given the ancillary but important metabolic benefits of adenomorin, will you be looking for those potential benefits apart from docetil from the Phase 3? Thank you.

Speaker 2

Yes. Duke, I only heard part of the question, but I think that was really for you. And so why don't you start?

Speaker 8

You know what?

Speaker 6

Honestly, I cannot hear the question quite well. So

Speaker 2

if anybody can be So yes, go ahead, John.

Speaker 3

So, I think Leland was asking about the impact of not having comparative treatment data in Phase 3 for pediatric endocrinologists as they think about how to prescribe this once we're on the market?

Speaker 6

Right. Okay. So Leland, that's a very good question, right. I think this is very important, right. As you know, first of all, FDA fully understood that mechanism of action of RUM21 totally different than growth hormone and they make it clear to us that you are not growth hormone.

Speaker 6

And as a pediatric endocrinologist, you know that if you participate in the trial, if the patient with growth hormone deficiency such as in moderate PTSD, the only impact with no treatments only height, no other segment detrimental outcome that could implicate the patient without treatment for 6 months for 1 year. So we do believe that most physicians fully aware of that, especially the patient who enrolled in this study is prepubertal. So again, pivotal impact with no treatment 6 months to 1 month is not going to have significant impact in final adult high. With that said, we do believe that when we conduct this trial, they will have no issue to enroll the subject. And not to mention that all the patients participate in this trial potentially will be able to enroll into long term extension study, which is we already have that in place and FDA approved that long term extension study for 3 years.

Speaker 6

So I do believe that we incentive to really help for physician to enroll their patient knowing that in the long term will be benefit to that patient especially when the drug is approved.

Speaker 8

Just the metabolic benefit, LEN-two zero one apart from growth itself may have other beneficial effects on the body, wondering if you're going to assess the patient safety.

Speaker 2

John, do you want to answer that question?

Speaker 3

So I think we won't be examining that question specifically in this Phase 3 study. We do have an investigator initiated study going on in NAFLD where some of that data is being collected in the context of examining the impact on liver fat. So it's being run at MGH. But for our Phase 3 trial, we're going to focus on the approval endpoints and the path forward in PGHD to get an approval.

Speaker 8

Thank you.

Speaker 2

But Leland, I think you were also referring to okay, go ahead.

Operator

Thank you. The next question comes from the line of Ed White with H. C. Wainwright. Please go ahead.

Speaker 10

Good morning. Thanks for the update this morning. So just a couple of questions from me. Can you comment on your manufacturing readiness for the Phase 3 trial and regulatory filings?

Speaker 2

John, go ahead. Manufacturing readiness.

Speaker 3

Yes. So thanks for the question, Ed. So yes, we as you know, we recently filed a patent on a new drug product form for Phase 3, which will give us very tight dose variance across the large weight range for children with pediatric growth hormone deficiency and also provide easier routes of administration. So we have a mini tablet in a capsule, so larger kids can take the capsule with the mini tablets, smaller kids can open the capsule and take the mini tablets by themselves or in soft food. So that design is in place.

Speaker 3

The bridging studies are complete and we're moving forward to get our Phase 3 material ready to go. So there are no hiccups in the road there. We're well prepared to provide that Phase 3commercial material to get us through Phase 3.

Speaker 10

Okay. Thanks, John. And just a question on the study. As you mentioned, it's going to have some overlap with the Phase 2. Can you just let us know what you're thinking of as far as geographical breakdown for the U.

Speaker 10

S. Versus outside the U. S. For sites and the number of patients enrolled?

Speaker 2

Duke, why don't you answer that question?

Speaker 6

Yes. So for DSR, we plan right now is that we plan to enroll about 150 subjects. So 2 to 1 randomization will be 100 subject in LUM201 and 50 subjects in placebo. We plan to include more country outside the U. S.

Speaker 6

We're in the process of sending out a site survey to multiple region around the world. We're waiting for those to receive those sites away back before make decision which country we go to. So around the site that we planned is about 90 site ish. So again, so potentially about 14 country. However, the final number cited in the country will be finalized when we received a survey back.

Speaker 2

And Leland, if you recall, you were Drop the chat. Okay. And Leland, if you go ahead.

Speaker 10

I'm sorry, Rick. Sorry for interrupting you, Rick. Operator? My last question, could I ask another question?

Speaker 2

Sure, absolutely.

Speaker 10

So the we've talked about this before, but out of the 60% to 62% of the patient population that's eligible, I just want to get your thoughts on why children who are eligible wouldn't want to be on the oral solution versus the injectable, especially since as you mentioned, the cost will be lower, but the ease of use is apparent. And so I'm just taking the other side of that and why wouldn't patients and children want to take your product?

Speaker 2

Well, we did some preliminary research, Leland. No, this is Ed, excuse me, Ed. And it's pretty clear, when we ask both the families, but also the peat endos, if they had a choice between a weekly injection or a once a day oral, they overwhelmingly said that they would prefer to take a daily oral. That's sort of a given. So and of course, we have a way to identify the patients who are drug will most likely be effective in and that is our predictive enrichment or PEM strategy.

Speaker 2

And that further enhances our ability to not only be successful in this trial, but also to easily identify those patients that will be effective.

Speaker 6

Yes. I would like to add on top of what Rick just said. As a pediatric endocrinologist, majority of patients with PTSD fall into PIM positive category. As you know, over 40 years, we don't have option, but just give injection to those children, right, just until about 4, 5 years and we get long acting approved, but does not get rid of injection, which is some of the children do not want it, especially not only treatment for a month or so, not a year or so, but much longer period of time. So physician in general, I do believe that their perception was if this drug get approved, they will offer this LUMTO-one to mature those patients by using PEM positive cut off.

Speaker 6

With the PEM positive, they potentially can be on the treatment. So I do believe that this is extremely important, right, to have this option for the treatment for patient moving forward. We never really have this option until LUM21 at this point.

Speaker 10

Okay. Thanks for taking my questions.

Operator

Thank you. Next question comes from the line of Kathleen Novak with Jones Research. Please go ahead.

Speaker 11

Hi, guys. Good morning. I just wanted to drill down on the cash position. You need to bring in additional funding before you start the Phase 3. Do you see any inflection points between now and the start that might be that you might be able to use to bring in new investors?

Speaker 11

And then just throwing out the possibility of pursuing strategic partnership, for example, do you think they'd want to see before stepping in? Are there more analyses that you think partners or investors would be interested in, or at this point are most of the relevant data in hand?

Speaker 2

Yes. Catherine, it's a good question. I don't think there's any question that investors have enough information at this stage to make a decision. I think we're as Laurie said, we're very confident since the FDA gave us this trial design with a placebo comparative arm that we are fully confident we're going to be able to raise the capital needed to get this drug across the finish line. And Laurie, I don't know if you want to add any more that or anyone else on the team.

Speaker 11

Yes. I think, Catherine, I think when we've talked

Speaker 4

with investors, I think a lot of investors have just been waiting to determine what the Phase 3 trial design would look like. And now that we have gotten feedback from the FDA, they have proposed a placebo controlled trial as an option. As we finalize that trial design and move forward with FDA approval of that trial design, we do believe that that will instill confidence in the investors and allow us to complete a financing in this near term. As far as looking for regional partners, we've talked previously about looking for ex U. S.

Speaker 4

Options for regional partners and retaining U. S. Rights, of course. And so that is something that we will continue to pursue. And I think what they also were waiting on in the partners that we've talked with have also been waiting on what the Phase 3 trial design will look like.

Speaker 4

So now that we have that information in hand and we have more of those details, I think that is why we are confident we will be able to move forward and complete the financing in the near term.

Speaker 11

Got it. And then just I guess clarification on the first co primary endpoint. If you can help me understand the statistical analysis. Since placebo is not followed for 12 months, what is the hypothesis as seen assumption for the 1st co primary endpoint? If you could clarify.

Speaker 2

John, go ahead if you would answer that question.

Speaker 3

Sure. So, as we mentioned, we just have to show clinically meaningful growth. And so we talked a little bit about one approach to show that, which is the agreement we already have with the FDA on 6.7 centimeters per year representing minimal clinically meaningful growth. So we're using that to transition essentially from our Phase 2 study into our long term safety extension. And that is an agreement we already have with the FDA.

Speaker 3

So that is the first option that we'll pursue.

Speaker 11

Okay. And then just one more, thinking about this down the road, right now you're focusing on treatment naive patients, but it seems like does this make sense to use after an initial year or 2 with growth hormone for people who aren't maybe interested in having daily injections indefinitely?

Speaker 2

Duke, let's go ahead, if you will.

Speaker 6

Yes. So I think that that's a very good question. I do believe that most patients will look into that potentially because with the patient room to 1. At this point, we don't have data to show yet. So what we think that moving forward, when we get these Phase III trial done, we put that in registry.

Speaker 6

So majority of those patients going to get into the long term extension, especially registry is someone who is not in Phase II. They can continue to have the Phase 4 study when the drug is approved. We'll be able to determine some of those, especially when the patient want to discontinue daily and go into room tour 1, we'll be able to evaluate dose efficacy in those trials.

Speaker 11

Okay, got it. That's helpful. Thanks.

Operator

Thank you. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation.

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Earnings Conference Call
Lumos Pharma Q1 2024
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