Lumos Pharma Q3 2023 & Study Update Earnings Call Transcript

Quartr
Provided by Quartr
November 8, 2023

There are 11 speakers on the call.

Operator

Good morning, and welcome to Lumos Pharma's Top Line 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 with the call, 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 yesterday and in our Form 8 ks, 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 John McHugh, our President and Chief Scientific Officer Doctor. Duke Tuuk Chuanan, Senior Vice President of Global Clinical and Medical Affairs and Luke Lalley, Chief Financial Officer.

Speaker 1

I will now turn the call over to Rick.

Speaker 2

Thank you, Lisa, and good morning, everyone. After the market closed yesterday, we issued a press release Announcing top line results from our Phase II oral Growth 210 and oral Growth 212 trials of oral LUM-two zero one In pediatric growth hormone deficiency or PGSV, I'm pleased to report that all primary and secondary endpoints

Operator

Ladies and gentlemen, please stand by. We are experiencing technical difficulties. We'll be right back. Ladies and gentlemen, thank you for standing by, and we apologize for the technical difficulty. We will now return to the conference.

Operator

Please go ahead.

Speaker 2

So after the market closed yesterday, we issued a press release announcing top line results from our Phase 2 ORGOV-two ten and ORGOV-two twelve Trials of oral LUM-two zero one in pediatric ophthalmoid deficiency or PGHZ. I'm pleased to report that all primary End secondary endpoints were met in both trials and data from these trials provide supportive evidence to advance LUM-two zero one to A Phase 3 clinical trial. I'm going to commence by offering a summary outlining how we believe these data convincingly establish The validity of ILU-two zero one has a potential oral replacement for moderate PJC patients, eliminating the need for daily and weekly injections. Subsequently, I'll hand over to the discussion John McHugh, our President and Chief Scientific Officer, who will provide a thorough examination of these results. So to begin, findings from our oral GROW-two ten study indicate That the LOOM-two zero one dosage of 1.6 mg per kg resulted in annualized height velocities or AHVs Of 8.2 centimeters a year at the 6 month mark and 8 centimeters a year at the 12 month mark.

Speaker 2

These figures align with the historical growth rates observed in the moderate PTSD population. The difference in annualized high velocities at both the 6 12 month time periods between the ideal 1.6 mgkg LUM-two zero one dose Andorakarma Human Growth Hormone Comparative Group falls within the non inferiority threshold of less than 2 centimeters a year, which aligns with the criteria recently employed by FDA approvals. While it's important to note that this study was not designed With the power to establish non inferiority, we're delighted to observe that the growth outcomes in this study are on par With the recombinant human growth hormone comparator arm falling within the recent Phase 3 non inferiority thresholds utilized for FDA approval. Furthermore, the preliminary 24 month 201 data compiled from 2 sources, and that is a subset of the Orgov 210 participants who met the protocol requirements for an extension Beyond 12 months and the Orgov 212 subjects revealed a sustained effect With no material decrease in annualized height velocity between the 12 month and the 24 month time frames. Moreover, in the Orgov-two ten study, we successfully attained Our predefined primary endpoint confirming the validity of our predictive enrichment marker or pen test.

Speaker 2

We also met the secondary endpoint by demonstrating 100% reproducibility of the pem positive classification. Additionally, data from the ORGOV-two twelve trial highlighted that LUM-two thousand and one Achieve growth comparable to Exizantin's injectable recombinant growth hormone while utilizing only 20% of the growth hormone concentration levels. This discovery underscores the significance of LUM-two zero one's unique Pulsatile mechanism of action and its capacity to restore the natural pulsatile release of growth hormone. And lastly, regarding safety data gathered from both our Phase 2 trials, we obtained a well defined safety profile for LUM-two zero one. And at present, there have been no safety concerns associated with its use.

Speaker 2

And considering that LUM-two-one induces annualized high velocities Alignment with historical norms for moderate PJC patients on recombinant human growth hormone that demonstrates a robust and enduring response validates our PEM strategy for the selection of suitable moderate PGSV patients and continues to maintain a clean safety record Without any signals of concern, we are confident in the strength of our data set for presentation during our end of Phase 2 meeting With the FDA and a finalization of our Phase III trial plans. So with that, I'm going to now hand it over to John McHugh For a more comprehensive review of our data. John?

Speaker 3

Thank you, Rick, and good morning, everyone. Before I review I would first like to remind everyone of LUM-two zero one's unique mechanism of action. LUM-two zero one is a small molecule that agonizes a Specific receptor on the pituitary and hypothalamus to stimulate pulsatile growth hormone release over 24 hours, which thereby increases IGF-one in circulation and both the growth hormone and IGF-one then act Also unique to LOOM-two zero one is that this molecule intervenes above the release of growth hormone, so the naturally evolved feedback mechanisms that define Physiological levels of growth hormone and IGF-one are still intact. This prevents LUM-two zero one from over stimulating the pituitary and producing too much This unique mechanism has implications regarding who might respond to our molecule. For analysis of LUM-two zero one's mechanism and previous clinical studies in this patient population, we're able to establish cutoff levels For both baseline IGF-one and LUM-two zero one stimulated growth hormone to determine likely responders to LUM-two zero one.

Speaker 3

We found that patients with a baseline IGF-one of greater than 30 nanograms per ml and a peak growth hormone stimulation value greater than or equal to 5 These cutoffs represent our predictive enrichment markers, Now I'd like to provide a brief overview of the Oragrove-two ten trial results. As many of you may recall, this trial involves a dose finding study with 82 subjects with the Primary goal of assessing the annualized height velocity or AHV at 6 months on treatment. Additional objectives included the confirmation of the pre specified outcomes assessing the effectiveness of our Penn strategy and the ability to choose an optimal dose for our Phase 3 trial. It's worth noting that as a Phase 2 trial, the ORGrowth 210 study is not designed to establish statistical non inferiority in AHV between LING-two zero one and the control arm. To begin, I will discuss the baseline characteristics That predict growth for the subjects participating in our dose finding ERGrowth 210 trial.

Speaker 3

During our interim analysis, We observed imbalances among the various treatment arms, particularly in terms of age, which seemed to augment the differences in growth rates during treatment. However, as we continue to enroll subjects in the trial with a focus on the 1.6 mg per kg LUM-two zero one arm and the comparator We have observed the expected narrowing of these imbalances. Consequently, the growth rates in these specific Covert has started to align. We anticipate that in a fully powered Phase 3 trial, we will likely achieve better balance We conducted a standard ANCOVA analysis Determine the least square mean of AHVs at both the 6 month 12 month intervals for the study. For the 1.6 Make per kg dosage of Loom-two zero one.

Speaker 3

Loom-two zero one achieved an AHV of 8.2 centimeters per year and 8.0 centimeters per year at 6 12 months, respectively. It's worth highlighting that the disparity of 1.8 centimeters per year at 6 months and 1.7 centimeters per year at 12 months between the 1.6 mg per kg dose And the recombinant human growth hormone comparator arm are within the historical non impurity margins commonly agreed to in recent Phase 3 growth trials. Furthermore, when examining the 12 month AHV data available for 50 out of the 81 subjects, it is clear that the growth Observed remain consistent and enduring at the 12 month milestone. Allow me to direct your attention to the unexpected increase This outcome deviated from the expectations set by numerous historical trials, which anticipated growth rates in the range of 8.3 to 8.6 6 centimeters per year for moderate PGH speed. We suspect that this atypical growth in the daily growth hormone of this study was likely influenced The higher recombinant growth hormone dosage administered and the presence of specific growth outliers.

Speaker 3

LOOP-two zero one PHV aligns with growth rates historically observed in similar patient populations from the Phase 4 Eli Lilly Genasys database, The Pfizer KIGS database and a Spanish study of moderate PGHD patients. Furthermore, at the 12 month time point, IM-two zero one exhibited an annualized height velocity within 1.3 centimeters per year of the daily injectable recombinant human growth hormone arm of Pfizer Phase 3 somatrogon trial for the subset of subjects that are similar to our moderate PGHD population. Now to shift our focus to the predefined primary and secondary statistical objectives of evaluating our PENN strategy. We met our preliminary validation of the PEM strategy as being effective in enriching our trial for LUB-two zero one responders. Additionally, we achieved 100% reproducibility in the PEM positive classification of subjects randomized in the ORGrowth 210 trial.

Speaker 3

These two outcomes reinforce our confidence in using this PEM strategy for our Phase 3 program. IGF-one working in conjunction with growth hormone plays a role in stimulating growth by acting on the open growth plates of the long bones. Data from the ORGrowth 210 trial revealed that LUM-two zero one successfully restored IGF-one levels to a normalized state within 6 months of treatment and this effect persisted at the 12 month time point. Importantly, we believe that this capacity to normalize IGF-one is a direct consequence LUM-two zero one's pulsatile mechanism of action, which operates within the natural endocrine feedback loop, effectively regulating growth hormone and IGF-one levels to mimic the experience of normally grown children. In summary, the ERGOT-two ten trial successfully achieved all of its primary and secondary end LUM21 demonstrated annualized site velocity results that are in line with historical benchmarks for a moderate PTHD population meeting our growth expectations.

Speaker 3

Additionally, ILUM-two zero one was shown to restore IGF-one standard deviation scores to a normalized within 6 months of treatment and this effect was durable for the 12 month time point. Furthermore, the continuing safety profile of BLU-two zero one was reaffirmed. Now I will turn the call over to Duke to discuss our mechanistic PKPD or growth 212 trial. Duke?

Speaker 4

Thank you, John. Transitioning to the outcome from our PKPD oral GROW-two twelve study, It is very important to note that this was a single site trial involving a more uniform patient population Compared to our multinational oral growth 210 trial, the 212 study was designed To investigate the impact of LUN-two zero one on endogenous growth hormone positivity and analyte high velocity, while also assessing the pharmacokinetics and pharmacodynamics of LUM-two zero one in a pediatric growth hormone deficiency population. The demographic of those enrolled in ORAGR012 highlight a greater degree of deformity within this trial in contrast to what we observed in ORACON-two ten. However, the best line characteristics across both trials were comparable enough To permit combining them for further analysis, the annualized high velocity finding At the month 6 mark of treatment, we view a substantial growth improvement from pretreatment growth rates These findings will corroborate by the analyte high velocity data from the 12 month treatment population The IGF-one standard deviation score data reaffirmed the outcome of the ORACLE-two ten study, Illustrating that lutro1 effectively restores IGF-one level with the sustained impact lasting up to 12 months of treatment and no excursion beyond 2 standard deviations were observed in this time period.

Speaker 4

PKPD data demonstrated That LUM-twelve-one promotes an elevation in pulsatile secretion of growth hormone, Restoring the levels in normal physiology. For examining the 24 hour pulsatile secretion, The data reveal that LUM-two zero one achieved growth comparable to exogenous injectable recombinant human growth hormone, while utilizing only 20% of the growth hormone concentration levels. These results imply growth hormone secretion generated by LUM-two zero one is We want to emphasize that LUMTRO1's ability to restore the endogenous pulsatized secretion of growth hormone enables This remarkable increase in growth with significantly less circulating growth hormone, All while maintaining IL-one level within normal range. In summary, AUTO GROT-two twelve achieved all primary and secondary endpoints with the data providing strong support Now shifting our focus to the combined data from these two studies, early findings for The 24 month period suggests that LUM-twelve-one exhibit a more enduring 1, compared to recombinant human growth hormone, we hypothesized that The small decline in year 2 annual high velocity compared to recombinant human growth hormone can be attributed To the LUMTO-one's ability to bring growth hormone and IGF-one levels back to normal Through its unique pulsatile secretion mechanism, the investigational safety data Combined from these two trials indicate the absent of significant serious adverse events.

Speaker 4

No participant withdrawal due to serious adverse events or adverse events And no noteworthy safety concern observed to date. With that, I will pass the floor over to Laurie for a brief summary of our Q3 financial results, Which we also reported yesterday.

Speaker 5

Thank you, Duke. Lumo Pharma ended the quarter on September 30, With cash, cash equivalents and short term investments totaling $42,700,000 compared to $67,400,000 on December 31, 2022. The company expects use of approximately $9,000,000 to $10,000,000 in the Q4 of 2023. Cash on hand as of September 30, 2023 is expected to support operations through the Q3 of 2024, inclusive of the activities related to planning and initiation of a pivotal Phase 3 clinical trial. Research and development expenses were $5,000,000 for the quarter ended September 30, 2023, compared to $4,100,000 for the same period in 2022, primarily due to an increase of $900,000 in clinical trial expenses and $200,000 in consulting expenses, offset by a decrease of $200,000 in personnel related expenses.

Speaker 5

General and administrative expenses were $3,900,000 for the quarter ended September 30, 2023, unchanged as compared to the same period in 2022. The net loss for the quarter ended September 30, 2023 was $8,300,000 compared to a net loss of $7,300,000 for the same period in 2022. Lumos Pharma ended the Q3 of 2023 with 7,914,582 shares outstanding. Now, I'll turn the call over to Rick for some final remarks and forthcoming activities.

Speaker 1

Rick, I believe you're on mute.

Speaker 2

Yes. Thank you, Laurie and John and Duke. As is evident, we're very excited and impressed by the data that we shared today. Both oral drug trials have successfully achieved their primary and secondary objectives. The annualized high velocity data from the LUM-two-one into 1.6 mgs PKPD data suggests unique advantages associated with the mechanism of action of LUMTRU-one for patients with moderate PTAC.

Speaker 2

In the following stages of this program, we're currently immersed in a careful preparation for Phase 3 trial. With the data now available, the initial step involves seeking an end of Phase 2 meeting with the FDA to evaluate the Phase 2 outcomes And establish the final design of the pivotal trial. We plan to make the request for the end of Phase 2 meeting with the FDA Within the first half of twenty twenty four, we expect to kick off the Phase 3 program in the latter half of twenty twenty four. So in conclusion, we're confident that our findings suggest that LUM-two zero one has the capacity to revolutionize the global growth hormone market, Which has been dominated by injectable products for nearly 4 decades. We're enthusiastic about progressing with this significant program And we want to thank all of our clinical investigators, their staff, their patients, families who participated in these trials, And we owe each and every one of them our gratitude for the successful realization of these trials.

Speaker 2

We believe this sets the stage Thank you for your attention. And operator, we can now open things up for questions.

Operator

Thank you. At this time, we will be conducting a question and answer Our first question is from Charles Duncan with Cantor Fitzgerald.

Speaker 6

Hey, good morning, Rick and team. First of all, thank you for taking the question. And secondly, yes, congratulations, very nice results, good for patients, Potentially transformative. I had a couple of questions for Duke and I'm trying to have him answer this from the perspective of A doctor who used to treat patients or maybe he still does with pediatric growth hormone deficiency, I guess, I'm wondering if he could provide a little bit more color on the lack of considerable decline Over time, not only in terms of, yes, the effect size in 1 year, but the effect size Over a couple of years. And then I also had a question on the safety observations.

Speaker 6

Thank you.

Speaker 2

Yes. So, go ahead, Duke.

Speaker 4

Yes. So thank you for the question. This is very good, right? As you know that most of the growth hormone therapy, the dose of the growth hormone that provides for the patient with DIL injection is supra physiology. LUM-two zero one have a unique mechanism that basically provide 24 hour growth hormone secretion.

Speaker 4

And we do believe that with these, drugs, the adenomic physiology, you can enhance the cordless secretion Throughout the growth period. As you know, children, they're growing depend on when you start the treatment. The boy will continue to grow until 18 and girl continue to grow about 15. So basically, with the normal physiology, it's going to match up With the normal growth pattern age appropriate. And that is signaling not just only the safety profile itself, But basically, the continued progress of the growth and we do believe that this type of process, dealing puberty, The GLUM-twelve-one can help to promote growth, align with the pivotal growth spirit as well.

Speaker 6

That's helpful. I had a question on safety and I'd like you to, yes, answer this in the context of Potential safety or lack thereof of super physiological growth hormone. I guess I'm wondering, in terms of the 40% Treatment related AEs in the 1.6 arm, Can you provide some color, any observations that were maybe greater than 5% of control? And then there was, I think, one patient with Transaminase increase, how do you see that, especially relative to the current standard of care?

Speaker 4

Yes. So thank you. So let me separate into 2 words, right. So the safety for LUMTRO1 versus supra physiology Those treated by growth hormone, right? So in our study, as you can see, the percentage of RUNE-two-one, percentage of the adverse event may be a little bit higher, But part of that due to 2 things.

Speaker 4

If you can see that part of the PIM strategy, every single patient Who came into the trial regardless they get enrolled or not. If they're exposed to LUNTO-one even one dose, we need to document that adverse event. Majority of those adverse events, even though you see the presenters seem on the high side, but none of them related to LUM-two zero one. Come to the treatment adverse event that have been demonstrated LEN-two zero one, especially One subject would increase transaminates. So as you can see that that specific subject when you look into the details, They only my elevation of liver enzyme and resolve all the time when to continue to follow dose patient.

Speaker 4

Now unlike The supra featured dose that treated with daily growth hormone and long acting. As you know, especially recently approved long acting growth hormone, You can see significant higher IGF-one SDS and growth hormone concentration. In 1 week Treated with growth hormone, long acting growth hormone, you can see that the growth hormone in circulation at least the 1st 3 to 4 days After the injection, you can see a very high suprafysiology. However, it come down towards The optimal or baseline towards 7 days. And IGF-one excursion higher because When you keep exogenous daily growth hormone, you shut down a negative feedback loop, which is basically you cannot control how high level IGF-one will That's one of the unique mechanism of LUM-two zero one.

Speaker 4

That differentiates itself From the daily growth hormone therapy, because we would not be able to generate IGR1 high norm normal because We have intact feedback that control the IGR1 within normal range. And one thing I want to point out as I presented earlier, It's very interesting that we only need approximately 1 fifth of the growth hormone concentration In order to achieve the comparable growth velocity, this is a very breakthrough information that potentially could be a unique Treatment for the patient in the future.

Speaker 6

Got it. One last multipart question For Rick or John Morris, call it strategic, know that you haven't met with the FDA, so don't expect you to be all that granular on this. But If you contemplate Phase 3 design, can you give us a sense of rough size And does the PEM strategy help you to, at least make that more capital efficient, if you will, if not in terms of Size, at least in terms of patient population. And then, maybe for Rick, are you contemplating partnering this And would that be a geographic partnership or perhaps a broader? Thanks.

Speaker 2

John, why don't you start with that question?

Speaker 3

Thanks, Charles. So I think, as you said, we haven't finalized our Phase 3 plans, but we have been looking towards what the other long acting Molecules in this space have used for their Phase 3 year pivotal designs. And we anticipate a similar size trial, so something on the order of 200 subjects. In many of those studies, they did a 2 to 1 randomization between the test article and the comparator arm. So That is more that is the direction that we're planning towards.

Speaker 3

But obviously, we have to do more work right now Finalizing our Phase 3 plans based on the data we just turned over and then negotiate with the FDA to agree on that. So that's the vision we have right now. And I'll turn it over to Rick to answer the rest.

Speaker 2

Thanks, John. Charles, I think we're really fortunate Be at this stage in development as a company as we're looking forward to a global Phase 3 trial, any company that's at late stage in development, Usually, a lot of good things happen in terms of potential partners that may show up. I think we've guided in the past That, yes, we've been active in talking to a number of people around the world. We want to keep the major markets for ourselves. And I think many companies at this stage do consider regional partnerships where they can bring in a substantial amount of non dilutive capital.

Speaker 2

And that combined with a typical raise at this stage, would provide a healthy outlook for the company financially.

Speaker 6

Thank you for taking my questions. Congrats on the results.

Operator

Our next question is from Yasmeen Rahimi with Piper Sandler.

Speaker 7

Good morning, team, and congrats on the amazing data. Quick questions for you. I guess the first one is really great to see the 12 months data. Could you comment on if moving forward you have any additional Time points beyond 12 months to see at the growth chart as patients continue on treatment. If we don't have it on hand, when do we expect to have that?

Speaker 7

And then, I have a second question as well.

Speaker 2

Okay. Good question, Yas, and I'm going to let John address that.

Speaker 3

So this study is still ongoing. The 210 and 212 studies are both continuing to go forward. So The 12 month data that we've presented as of today is just how many subjects made it to 12 months when we read out the full 6 month data package. So as time goes on and the rest of the cohort makes it to 12 months, that's another Very important data set that we'll be sharing when we get to that point.

Speaker 7

So could we envision Multiple readouts into 2024 from those data cuts and major conferences. If you could just maybe talk about the cadence of data in the next 12 months, that could be helpful.

Speaker 3

Yes. So we will fully enroll The full cohort at 12 months will absolutely come out next year, in the first half of next year. And I think we have several key Endocrine conferences that happen in the springtime with Pediatric Endocrine Society and the ENDO meeting. And then later in the year, we'll have the SB meeting Outside the U. S.

Speaker 3

So I think those are going to be the 3 key time points for scientific presentations on our full data set. We are also hosting more currently a KOL session on December 6, where we'll This current data set in much more detail.

Speaker 7

Wonderful. And then maybe a last question is, Could you maybe talk about what are areas of discussion in regards to Phase 3 design? You talked a little bit about the potential size, but maybe what are areas where maybe you may have negotiation with the agency in regards to Pivotal 4. And I'll jump back into the queue. And thanks again and congrats.

Speaker 2

Yes. Thank you for that question, Yaz. And I'm going to turn that back over to John too.

Speaker 3

Yes. I mean, I think The data package we just turned over has the pieces that we need to have this discussion with the FDA, right? We have to look at the effect size, the variability in each And we have to take that and plan for the size trial that I think is going to be appropriate to run For Phase 3. So there are lots of pieces of data that are still kind of slowly coming out. One of those is The PK data from our 212 study, we're still waiting for that full data package to come out and those are all some of the pieces that we'd like to have in hand before we finalize our Phase

Speaker 7

Thank you very much.

Speaker 2

Thank you, Yigas.

Operator

Our next question is from Leland Gershell with Oppenheimer.

Speaker 2

Hey, good morning, Rick

Speaker 8

and team and adding my congratulations as well on this terrific readout. Also great to See the 100% reproducibility in the PEM testing element. And to that end, I wanted to ask Maybe early days, but wanted to ask with respect to the criteria for the PEM test. Did you see In terms of the IGF-one baseline or the growth hormone response and what a particular the degree to which a particular patient increase their height velocity?

Speaker 2

Excellent question. And John, would you please address it?

Speaker 3

Sure. So Leon, that's something we're interested in. We're digging into that individual kind of subject by We don't have a we haven't done enough work on that to draw firm conclusions yet, but it is obviously something of interest that we're excited about and Yes, just exploring, how much information we can get out of the current data set. That is one of the pieces that we're still working through.

Speaker 8

Okay. And also with respect to the AHV data, it looks like from the data that you show here that the error bars are tight. But I want to ask With respect to AHV across the 1.6 milligram per kg cohort, could you kind of the range of AHVs that you saw and particularly with respect to those who were responders, The seventy-thirty breakdown responder rate, what the age fees look like in those who

Speaker 2

Yes, John, why don't you address that?

Speaker 3

So we don't we haven't broken it down on an individual subject by subject basis yet. But I think

Speaker 6

Clearly,

Speaker 3

the standard error of the mean that we have in the graphs that we released is very tight, right. So this is Very tight for the 212 study. So that's a very homogeneous population at baseline and their growth response is very homogeneous to RINCE-two zero one. And I think we see a very similar thing in the 210 study, very tight error bars. So all of those Subjects are growing very well and at a very similar rate.

Speaker 8

Great. And then just lastly, with respect Seeing a fuller breakout of these data, would we have to wait for the ENDO conference next June or could we see these presented maybe at an earlier conference?

Speaker 3

John? So there will be, this KOL presentation that I spoke about earlier on December 6th, We will have 3, Graybar clinicians in the trial discussing the results, Hopefully, in a more fulsome manner as we continue to dig through the data. But, PES, Pediatric Endocrine Society, is in May, and that's The first big meeting that we'll have the opportunity to present that next year.

Speaker 8

All right, terrific. Well, again, congratulations and thank you.

Speaker 2

Thank you, Leland.

Operator

Our next question is from Ed White with H. C. Wainwright.

Speaker 9

Good morning. Congratulations on the data and thanks for taking my questions. So I just wanted to get your thoughts and perhaps more information on the growth hormone control That reached the 10 centimeters per year in growth. You had said that the higher dosage in the presence of outliers Was the reason that it was above the range. I just want to get, if you can, the numbers of Patients that you consider the outliers, what was their recorded growth and what was the increased dose And why the higher doses were seen in the study?

Speaker 2

Thanks for your question, Ed. And John, if you'd begin to answer that.

Speaker 3

First, so Ed, at the interim analysis Data presentation last November, we talked about the 2 outliers that we had in the growth hormone arm. These were the 2 of the 3 youngest subjects that enrolled in our trial and we had some really strong growth. One of them grew at about 16 centimeters per year annualized at the 6 month time point. The other one was I think 13 or 14. So these subjects are growing very, very well for their age.

Speaker 3

And they are They were 20% of the data set at interim with 10 subjects per cohort and now they're Down to 10% with 20 subjects, but they're still there, right? And so I think the good news is We didn't have any other very high growing subjects enroll between interim and now. So those are those 2 subjects are still the largest outliers. They still exist. And the comment around the Growth hormone dosage is really that there is a wide range of growth hormone dosages that are approved on the label, right?

Speaker 3

And The 34 microns per kilogram per day is at the upper end of the doses that are approved for nordotrophin and genotrophin. So We're just pointing out that that has been the convention to use that dose going forward and it is at the higher end of the approved dose I think it's a combination of both those 2 very young subjects We're given the higher end of the dose response and responded enormously well. That is the outliers we're speaking to.

Speaker 9

Okay. Thanks, John. And another question was just If you can, break out the number of patients treated in the U. S. Versus international, any thoughts you have on what Phase 3 enrollment will look like geographically.

Speaker 9

And were there any differences in the Different geographic areas between the U. S. And outside the U. S?

Speaker 2

Thanks for the question, Ed. And Duke, if you would begin to answer that and John jump in. I think I can't hear you on the question.

Speaker 4

Yes. Thank you for the question. That's very good. As you know, the global Phase 3 trial, right, what we plan moving forward, it can be global Phase 3, not only in the U. S.

Speaker 4

However, The U. S. Will be the number one enrollment site, right? We can have majority of the site in the U. S.

Speaker 4

And we have some outside the U. S. As well. In the meantime, we try to do feasibility survey So looking at because all of this is not only about enrolled subject. We need to think about CMC, the drug supply of each of the country.

Speaker 4

So how we can provide the drug to each different country because each country requires regulatory requirements. So with that said, we're still

Speaker 2

Okay. Thank you.

Operator

Our next question is from Catherine Novak with Jones Trading.

Speaker 5

Hi, good morning. Congrats on the data. Thanks so much for taking my questions. First question is for Greg and team.

Speaker 10

I just wanted to ask

Speaker 5

a little bit more about the enrollment dynamics. Given the time required to fully enroll

Speaker 7

the Phase 2b study, do

Speaker 10

you think there's anything you can do to

Speaker 5

accelerate enrollment of And then as a follow-up,

Speaker 7

Do you see competition for

Speaker 5

treatment naive patients based on the recent approval of long acting injectable growth hormone? Thanks.

Speaker 2

Thanks for the question, Catherine. I think it's really a good one. We've really thought quite a bit about this. I think as we are in the planning process here, historically, you look at the competition for patients that was in the marketplace for Quite some time with 3 companies conducting large global Phase III trials, there was a great deal of competition for those patients. There's a lot less currently.

Speaker 2

So I think that's going to help us. But I also believe that we chose some very interesting and connected, But, investigators are really keenly interested in what we're doing because these are mainly very highly experienced investigators, but they've worked with All injectable products for their entire career. So having the first chance to work with a daily oral certainly got their interest. And I believe the results from these two trials is going to pique their interest that much more and it's going to go directly to, I think you did a really good enrollment in Phase 3. I think I was that did I answer all of your questions there?

Speaker 5

No. Yes, thanks. That was helpful. And I have one additional question thinking about this from prescribers' perspective. You showed that patients on LUM-two zero one, they maintained IGF-one levels within one stage deviation, which as you mentioned is not the case with injectable growth hormone.

Speaker 5

Thinking about the Scriber's perspective, what are some of the negative outcomes of having consistently elevated IGF-one? How compelling is this from a commercial standpoint when prescribers are making these decisions in a more moderate population?

Speaker 2

Yes. Maybe I can start with an answer and Duke, if you would jump in right afterwards. But I think we've got some additional insight Very recently, we had a KOL event. These were a set of 5 clinicians not Attached to our as investigators to our development program, but independent of us. And I think the consensus Of the concern that they had about especially long acting growth hormone and excursions of IGF-one over different periods of time, especially for a patient who's going to be on drug for perhaps 6 or 7 years of their lives, Definitely came out as a concern.

Speaker 2

And a couple of investigators said, essentially, our goal is Normalize growth hormone production and normalize their growth. And as they put it, this is exactly what this drug does. So that part to us and hearing that from independent investigators was really exciting to hear. And Duke, if you want to add anything to that, please?

Speaker 4

Yes. So that's a very good question, right? I am a pediatric endocrinologist my So I treat a lot of patients. And we know that since the glaucoma recombinant glaucoma had been approved since 1985, We are somewhat concerned about the long term effect of the growth hormone, especially if it's IGF-one very high. As you know, high H1 theoretically could increase the cancer risk.

Speaker 4

But so far more than 30 year, we have a large Multiple company follow those patients with growth hormone. It's very clear that IGF-one is Growth hormone dose related. If you increase the growth hormone, the higher the dose, IGF-one go up higher. The concept of DALI, at this point, we feel quite comfortable after 30 years. However, the new drug therapy, which is long acting, The mechanism of action is different because the half life is longer.

Speaker 4

So the patient, during 1 week period, you exposed higher growth hormone as a daily basis and higher IGF-one in general. But again, each drug of the long acting have different mechanism of action, but in general, That's what you can see. However, if the drug get approved, we don't see a significant safety signal at 12 month period, But I think as a practitioner, we want to see a long term data, a safety profile. As you know, gross amount, we're not treating only a few months or a few year. It could be 5 to 10 year, depending where the patient received the treatment.

Speaker 4

With that said, I think long term follow-up should be continue to see is that any issue with the high excursion H1. As a physician ourselves, if we have the drop that we can provide efficiency, promote growth With the clear safety profile, IGO-1b in normal range, that would be ideal, especially if it's oral therapy versus injection.

Speaker 5

Got it. No, that's very helpful on that. Congrats again on the data. Thanks for taking my questions.

Speaker 2

Thank you, Catherine.

Operator

Ladies and gentlemen, we are showing no further questions. Thank you for participating.

Speaker 1

Apologies, operator. I believe, there is a follow-up from Cantor. They're trying to get into the Q.

Operator

Okay. And then what is the name? Is there

Speaker 1

any way you could assist them on the other end?

Operator

Yes. Standby, please. Mr. Duncan, are you there?

Speaker 6

Yes. Can you hear me?

Operator

Yes. Please proceed, sir.

Speaker 6

Okay. Thank you for taking the follow-up. I had two brief questions. Wondering, since we did this diligence a while back, Can you remind us of the dosing paradigm, given that sometimes children have trouble swallowing pills? I guess I'm wondering, I think these are micro tablets.

Speaker 6

Did you have any issues with regard to compliance? And then second question is regarding intellectual property. Again, I apologize, but I Have forgotten this. Can you give us a sense of the IP behind Loom201? And should it be approved, what do you think the kind of the life of the IP is with regard to that approval?

Speaker 6

Thanks.

Speaker 2

Okay. The first question, Duke, why don't you answer that, if you will, on compliance?

Speaker 4

So far, the compliance that we see some of the subject compliant, but not related to They're taking pill, right? Because I think that some of those issues could be drug shipping delay, etcetera. But so far, as you may or may not know, right, the pill is very small. And even the youngest children in these trials, 4 years We'll have no problem with swallowing the pills. So I think that the size of the pill is not an issue With the compliant in terms in regards to this trial.

Speaker 3

Yes. Okay. And if I may add to that, we do have, As we spoke about, we do have a new patent where we're focusing on even smaller mini tablets for our Phase 3 and Commercial plans moving forward. So we expect that to make it even easier for children to take our

Speaker 2

And Charles, that was a patent that was filed earlier this year. We hope to hear something back very soon From the Bureau and that patent will go out to 2,040 2. We believe that, that will be an Orange Book listed patent and has to be around composition of matter of the formulation of unique properties of the formulation itself. We found in the process a novelty around the compatibility of the chemical. So It's really an important patent for us that will go out to 2,040 2.

Speaker 2

And remember, our method of use patent is around The diagnosis and treatment of growth hormone across the board, not just PTSD, is by the use of our PEM strategy. And that has been issued in the U. S. And many other parts of the world, and it continues to be so in new countries almost Every few months. That goes up to 2,036.

Speaker 2

And then, of course, we have orphan drug designation in the U. S, Europe, And we'll file for that in other parts of the world as time goes on.

Speaker 6

Very good. Thanks for taking the follow-up, Larry.

Speaker 2

Thank you.

Operator

Thank you. Ladies and gentlemen, there are no further questions at this time. Thank you for participating. This ends our call for today.

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Earnings Conference Call
Lumos Pharma Q3 2023 & Study Update
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