Lumos Pharma Q2 2023 Earnings Call Transcript

Quartr
Provided by Quartr
August 9, 2023

There are 11 speakers on the call.

Operator

Afternoon, and welcome to Lumos Pharma's Q2 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 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 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 on the Investors page of the company's Web Speaking on today's call will be Rick Hakan, CEO and Chairman and Lori Lalley, our CFO John McHugh, President and Chief Scientific Officer as well as Doctor. Duke Petukchiwana, our Senior Vice President of Global Clinical Development and Medical Affairs will join for the question and answer section. I will now turn the call over to Rick.

Speaker 2

Well, thank you, Lisa, and good afternoon, everyone. After the market closed today, we issued a press release announcing our Q2 2023 financial results and provided 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 the highlights from the quarter and recent weeks before turning it over to Laurie for a review of our financial results. Then John McHugh and Doctor.

Speaker 2

Ptep Tewana or Doctor. Duke as we call him will join us to answer your questions. Doctor. Duke joined us about a year and a half ago from Ascendis, where he worked on their long acting injectable growth hormone therapy or therapeutic to approval. He is also President of the Human Growth Foundation and with his contacts and understanding of the potential of an oral therapeutic in this space, Duke has been instrumental in advancing the enrollment in our EROGROW-two ten trial and in preparation for our Phase 3 trial in pediatric growth hormone deficiency or PTHD.

Speaker 2

So let's begin. As reported this afternoon, during our Q2 of 2023, we made continued progress in advancing our oral therapeutic candidate LUT-two zero one and moderate idiopathic PGAC. We can confirm our expectation to report primary outcome data on up to 82 subjects in the dose range finding or a GROW-two ten trial and up to 22 subjects in a mechanistic PKPD oral growth 212 trial in the Q4 of 2023. And at this point, I'd like to remind everyone about our expectations for the primary readout. The primary endpoint for these trials is annualized height velocity or AHV at 6 months on treatment.

Speaker 2

And based on historical data, the predicted growth rate for LUM-two zero one is between 8.3 centimeters 8.6 centimeters per year for this moderate idiopathic BGSD population according to observed growth in several large historical databases. The other objectives of the ORG0210 trial are to confirm the utility of the predictive enrichment marker or PEM strategy And to determine the optimal dose for our Phase 3 trial. We also expect our primary outcome readout include AHP data at 12 months on treatment for up to 12 subjects for oral growth 2 10 cohort and up to 7 subjects for oral growth 212 cohort for a total of up to 62 subjects from both trials. Additional AHP data at 18 24 months on treatment are also expected for a small number of subjects. In addition, the Phase 2 trial Should demonstrate a safety profile comparable to the daily growth hormone control arm.

Speaker 2

And also as is the case for all Phase II trials, the Oral Growth 210 trial does not power to show non inferiority of annualized high velocity between LUM-two zero one and the control arm, but should inform the design of and dose selection for a successful registration Phase 3 trial. And as a reminder, the non inferiority margin between the treatment arm and the control growth hormone arm for a pivotal Phase 3 trial and this indication has historically been from 1.8 to 2 centimeters a year at 12 months on treatment. This has held true for recent approvals of long acting growth hormone products as well. So the next steps in the program, We're obviously engaging in meticulous planning for a Phase 3 trial. And after a thorough review of the data package, the first step will be to request an end of Phase 2 meeting with the FDA to review the Phase 2 results we agree upon the ultimate design of the Phase 3 trial.

Speaker 2

And based on regulatory precedence, we expect that this registrational trial will include approximately 180 to 200 PEM positive subjects, randomized 2:one versus growth hormone with a likely dose of 1.6 mgs per kg of LUM-two zero one to daily growth hormone, stratified by age and other factors to ensure balanced cohorts. Our proposed primary endpoint we'll be AHV at 12 months on therapy and the trial will utilize the new LUB-two zero one formulation For which we filed a novel formulation patent application last November enabled by unique properties of our compound, we could extend our IP protection to 2,040 2. This formulation of BLU-two zero one consists of many tablets in capsules, Which we believe will provide an easier oral dosage form for the wide age range of our target population. We expect to hear from the U. S.

Speaker 2

Patent Office later this year. Currently, LUM-two zero one has patent protection through 2,036 Plus applicable extensions for the detection and treatment of growth hormone deficiency as well as orphan drug designation, Which offers extended protection of up to 7.5 years and 12 years from the date of drug approval in the U. S. And Europe respectively. During the quarter, we were pleased to see further data and analysis from these two trials presented at the 2023 ENDO meeting.

Speaker 2

Data from these two abstracts were presented, including new data from the ORBOT-two twelve trial that showed an increase in IGF-one levels on LUB-two zero one at 6 months that remained within normal range, An increase in IGF-one SDS greater than 0 and a durable growth response up to 12 months of LUM-two zero one administration. There's clear evidence of potential drug effect for LOOP-two zero one was also observed in consistent improvement in AHV over baseline. Also new analysis of combined Orgro-two ten and Orgro-two twelve trial data at the 1.6 and the 3.2 mg per tick a day dose levels in 15 subjects from the Orbro-two twelve, 20 subjects from the Orbro-two ten. These combined results continue to demonstrate that there is a durable response to LUM-two zero one from 6 to 12 months at the 1.63.2 mgs perkgaday doses are comparable in the growth each stimulated. As many of you know, these data were highlighted in a key opinion leader webinar we hosted on June 21st, where Doctors Fernando Tesorla and Michael Tanzi shared their insights on the data and their continued conviction And the potential of LUMTRU-one to become the 1st oral therapeutic to address this patient population treated solely by injectable valve therapies for the last 4 years.

Speaker 2

If you've not seen the webinar, we encourage you to review the replay, Which is still available on our website. Additional analysis of data from the ORGOT-two twelve trial was accepted as a late breaking abstract for oral presentation at the upcoming Annual Meeting of the European Society of Pediatric Endocrinology or ESPY, Which was held in The Hague in the Netherlands September 21 to 23. This abstract by Fernando Casorla included deconvolution analysis of growth hormone secretion with LUM-two zero one administration in the moderate PJC population. And turning to other developments. We continue to support our clinical collaboration with Doctor.

Speaker 2

Positive results from this investigator's prior trial evaluating injectable growth hormone in NAFLD We're recently published in the Journal of Clinical Endocrinology and Metabolism. It was these compelling data that encouraged Doctor. Dikkel And Mass General to initiate the collaboration with Lumos Pharma to assess oral LUB-two zero one in the same indication. In the prior study, investigators hypothesized that growth hormone might reduce hepatic steatosis or fat buildup in the liver These patients with NAFLD, subjects were randomly assigned to a treatment group, 27 growth hormone and 26 the placebo group, with 41 completers overall, 20 on gross hormone, 21 on placebo at 6 months. Reduction in absolute percent of intrahepatic lipid content by proton magnetic resonance spectroscopy Was significantly greater in the growth hormone versus the placebo cohorts.

Speaker 2

Investigators concluded that growth hormone reduces liver fat without commensurate weight loss. These data are supportive of evaluation of oral LUM-two zero one in the NAFLD indication, LUM-two zero one pilot trial in NAFLD continues to enroll. As a reminder, the company's primary near term focus remains on advancing LUM-two zero one in PGHD. Now as previously mentioned, we believe that LIMB-two zero one has the potential to address about 10 other indications Currently treated by injectable growth hormone. We've done a lot of work internally to assess the potential of LUM-two zero one and other indications in different geographic regions worldwide.

Speaker 2

And as we've said before, we 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. And 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. So with that, I'm going to turn it over to Lori for a review of our financial results.

Speaker 2

Lori?

Speaker 3

Thank you, Rick. Lumos Pharma ended the quarter on June 30, 2023 with cash, cash equivalents and short term investments totaling $50,900,000 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 June 30, 2023 are expected to support operations through at least the next 12 months from the date of the filing of our Q2 2023 financial statement, which is well beyond our we expect to estimate a top line Phase 2 trial results in the Q4 of 2023. This guidance is inclusive of estimated cost should be incurred in preparation for a Phase 3 trial, including estimated costs for clinical trial site initiation, contract manufacturing expenses and regulatory expenses. These costs may change depending on the primary data from the Oragrost-two ten trial and subsequent feedback from regulatory agencies.

Speaker 3

Research and development expenses increased by $1,400,000 for the 3 months ended June 30, 2023 compared to the same period in 2022, primarily due to increases of $1,100,000 in contract manufacturing expenses, dollars 400,000 in clinical trial expenses $100,000 in personnel related expenses, offset by $200,000 decrease in consulting expenses. General and administrative expenses increased by $500,000 for the 3 months ended June 30, 2023, compared to the same period in 2022, Primarily due to increases of $200,000 in personnel related expenses, dollars 100,000 in stock compensation expenses, dollars 100,000 in travel expenses $10,100,000 in royalty expenses. The net loss for the quarter ended June 30, 2023 was $8,900,000 compared to a net loss of $7,800,000 for the same period in 2022. We ended Q3 with $8,000,000 of 41,345 shares with that, I will turn the call back to Rick to conclude for us.

Speaker 2

Thank you, Lori. So to recap, we can confirm our plan to report top line data from both ORGOT-two ten and ORGOT-two twelve trials in the Q4 of 2023. New interim data analysis presented at ENDO and other medical meetings this year further reinforce our confidence that primary outcome data from these 2 oral growth trials should achieve the predicted annualized high velocity at 6 months on treatment in line with historical averages of 8.3 to 8.6 centimeters a year for this moderate idiopathic BGSD population. Though our Phase II trials are not powered to show non inferiority, primary outcome data from these trials should support selection of ILUM-two zero one dose for a registrational Phase 3 trial where non inferiority to growth hormone of approximately 1.8 to 2 centimeters semiters should determine success based on historical approvals. Our novel formulation of oral GROW201 should also facilitate adherence to treatment protocols in both this pivotal trial and in the commercial setting and if a new patent is granted we extend IP protection beyond our current 2,036 expiration date.

Speaker 2

We believe oral LUM-two zero one Represents a platform therapeutic with the potential not only to disrupt the current worldwide $3,500,000,000 growth hormone market created by injectable growth hormone products. Now that excludes China, which is also another $1,000,000,000 But also to expand the market by increasing the historically low compliance and treatment rates due to the high burden of current injectable therapies. Additionally, we'll continue to explore utility in broader indications Such as NAFLD, our initial clinical studies has shown growth hormone treatment to be beneficial. And in the near term, we're focused on advancing our clinical programs in PGSG and we have the capital to support that effort well beyond our primary outcome readout later this year. And finally, in September, we plan to host both a KOL panel webinar and a KOL dinner where noted pediatric endocrinologists we'll discuss their experience with PJC patients, the therapeutic landscape and the potential for an early administered therapeutic to address other indications currently treated by injectable growth hormone.

Speaker 2

We're excited to continue to advance our clinical programs And look forward to disclosing top line data in the Q4 of 2023. So thank you very much for your time today. And operator, we're ready to take questions.

Operator

Thank you. The lines are now open for questions. Please standby while we compile the Q and A roster. Our first question is from Yasmeen Rahimi of Piper Sandler. Your line is now live.

Speaker 4

Good afternoon, team. Thank you so much for all the updates. I guess, could you maybe comment on how soon post the oral growth readout you could be in a position to kick off the Phase 3 studies. Maybe also some color on if you've started warehousing patients for the Phase 3 and help us around enrollment timeline? And then 3rd, when do you hope to get Color in regards to patents, the patents that are filed with the new formulation.

Speaker 4

I appreciate color on those topics. Thank you again.

Speaker 2

John, I'm going to let you answer that the first question that you guys asked.

Speaker 5

Yes. Right now, we're focused on thinking through all the steps to get from the data readout in the fall all the way out to the end of Phase 2 meeting, an agreement with the FDA on our Phase 3 protocol, right. So there's a lot of work involved there in integrating the data from these two studies, making any necessary adjustments to our Phase 3 clinical plans, Putting together that briefing book, waiting the 60 days to get the meeting and then hopefully quickly coming to agreement with FDA on what that Phase III protocol is going to look like and really from then is when we'll start to kick off really The extent of our focus on bringing trial sites up And getting ready to go. We've done a lot of the Phase 3 prep work as much as we can. We're working on that right now.

Speaker 5

But we really need to get that final agreement on the protocol really to start kicking things off and getting sites to be thinking about patients. Remember, the patients that we need are naive to treatment And so it's a little bit early right now to you can be watching patients, but I think going any further than that, we want to hold off on. In terms of the patent, so we filed this patent in November of last year, Right. So a 12 month decision time is not it's kind of an expected time line for when we might hear Back on that patent.

Speaker 2

Yes. And yes, let me just add. We have And obviously, a really experienced clinical development team. We've really performed well in this, the 210 and 212 trials and the in terms of time to enrollment. And I think that is due to 2 things.

Speaker 2

Number 1, highly experienced motivated staff has really managed these trials quite well. And the second thing is that there is a lot of interest. Most of these experienced investigators have been working their entire With injectable products, this is the first time that an oral product for PTSD has shown up. So they're pretty excited to work with us and so we get A lot of attention. I might add one other point and that is, you think about the typical time it takes to enroll a full 180, 200 patients and historically, you look at the companies who have done that in the long acting space.

Speaker 2

And they were all enrolling in a Phase 3 in competition with each other. They're Finished with those studies, obviously, those products are approved. While we certainly expect there's going to be some competition for these patients, We believe there's going to be a lot less than there was before. And therefore, we think enrollment is going to go well. But I maybe I'll just ask Duke to comment here because he's really been instrumental in his connection with all these are highly experienced sites.

Speaker 6

Thank you, Rick. So let me touch base with the timeline and enrollment, which is again most of their Phase 3 trial, that's one of the big hurdle that most company have experienced, right? With my connection with the KOL around the world, with experience they have done in the past, we hope that if we actually have well prepared site initiation and potentially could start up site enrollment across the world as pretty much as the same timeline, we'll be able to cut down the number of recruitment and timeline or complete enrollment. But again, a lot of factor involved, but we will do our best on strategic planning, as John had mentioned.

Speaker 2

Got it. Thank you. Yes. Did I answer your question?

Speaker 4

Yes. That was very helpful. Thank you, team.

Speaker 2

Thank you, Yaz.

Operator

Thank you. One moment please for the next question. Our next question is from Leland Gershell of Oppenheimer. Your line is now live.

Speaker 7

Hi, guys. Thanks for taking my question and for the comprehensive framing of how to think about Thanks as we head into the readout later this year. Couple of questions from me. First, with respect to Obviously, as we look forward to the data from ORGrowth-two ten next quarter, wondering if you'd be able to share the PEM screening results or give any color to whether that's the rates of success you've been seeing with the TEN screening have aligned with your expectations. And then number 2, as we look forward to the SP conference, you mentioned there'd be some additional analyses of the prior study, just wondering if you could share what the nature of those additional analyses might be.

Speaker 7

Thank you.

Speaker 2

John, do you want to start With the PM and the SP analysis?

Speaker 5

Sure. Thanks for the question, Leland. The PM test has been very effective as an inclusion criteria in the 210 study, we spent a lot of time educating the clinicians in that study as to which Subject to screen to bring into the trial and our success rate at inclusion is actually quite high, Right. We've got we had originally said in the entire population about 2 thirds of the kids would be PEM positive. We're actually seeing a much higher number of tests returning a positive value simply because of How the clinicians are focusing their efforts and looking for patients, right, looking towards the more moderate end of the spectrum And kids who kind of fit the criteria of that slice of the patient population.

Speaker 5

So we're getting A very high number of kids who are pem positive versus what you would expect from just looking at the epidemiology data of the whole spread of Birthworm deficiency. The second question about the endo or I'm sorry, the SB meeting. We've given out the title about the deconvolution analysis of the 212 study. So essentially, it's focused on the 212 study and the convolution of the PD data that is there. Those abstracts Haven't been published yet, but the program has been announced.

Speaker 5

So I think we'll have to wait until they publish the abstract we get a little bit more information about what's going to be released there, but from the title, that should give you a good sense of what's in there.

Speaker 7

Great. Thanks so much for taking the questions and I look forward to the updates.

Speaker 2

Thanks for the question, Leland.

Operator

Thank you. One moment please for the next question. Our next question is from Ed White of H. C. Wainwright.

Operator

Your line is now live.

Speaker 8

Hi, thanks for taking my questions. Just going back to the Phase 3 study and this might be difficult to answer, but how are you thinking about the number of sites that you're going to need for the Phase 3 to enroll that 180 to 200 patients. And how is the split going to go between U. S. Sites And ex U.

Speaker 8

S. Sites and also how are you thinking about the patient population, same question, U. S. Versus outside the U. S?

Speaker 2

John, you want to start?

Speaker 5

We haven't released the total number of sites. We're going to deepen discussions with CROs and trying to get their input on what they've seen in the past. Obviously, we're reviewing what some of the folks in these successful long acting programs have done. So we haven't really we haven't decided on a firm decision on the number of sites. Obviously, we are going to do a global Kyle, as we've talked about, I think it's important.

Speaker 5

We do expect to get different recruitment rates in different regions depending on access to growth hormone. And so I think we have to put all those together. In terms of what the FDA wants, obviously, I think we can certainly use almost all the U. S. And European sites are quite acceptable.

Speaker 5

And I think some of the Asian countries have requirements for which patients have to be or what number of patients have to be enrolled. So I think we're working through all those spots right now. We don't have a final plan yet, but they are all things that we're thinking through.

Speaker 2

Yes. Let me just continue with that. Ed, the benefit of being on the podium at all of these InterContinental meetings around the world is that all these experienced investigators have really attended those meetings With a great deal of interest because of us being the first oral. The result is that I think more and more people are We are becoming real believers in what we're doing and they've been reaching out to us and offering to be clinical sites. I don't think there's going to be a problem in choosing the sites.

Speaker 2

In fact, we have the benefit of more recent historical entry rates From recently just completed trials, by site and Duke of course has a great deal of experience in that. So I think that's going to be much to our benefit.

Speaker 8

Thanks, Rick. And perhaps a question on what's next for the company. You had mentioned that next indications are How should we be thinking about that? Are you prepared to perhaps a Phase 1 study concurrently with a Phase 3 study that we've just been discussing or will this be something that you're thinking about post Phase 3 data.

Speaker 2

Ed, I think that the answer to that question is, If we were a large company, we'd probably have a different plan. Capital markets are what they are. As we go out And do all the advanced planning for our Phase 3, that's where we're going to be focused. If the capital markets are going to permit us to Raise the appropriate amount of money over time, then we're going to start programs other programs perhaps Sooner than we normally would plan them. In addition to that, typically what happens when companies And their development plans get to the stage at the end of Phase 2, end of Phase 3, regional partners show up or at least that's what we're partners of all times.

Speaker 2

And as I think our Head of Business Development is a pretty busy guy at this stage and We're going to, I think, have some choices here, to look at some partners who could help us advance the program. And that could be in other indications too, although, we certainly haven't had those discussions with anyone yet.

Speaker 8

Great. Thanks, Rick. And perhaps a question for Laurie, just when thinking about R and D moving into the back half of the year, you mentioned that $1,100,000 in commercial contracting expenses hitting in the quarter. That's, I take it a one time event and we should be thinking about the run rate more like the Q1?

Speaker 3

I think our research and development expenses for Q2 are Pretty on par with what we should expect, Leland, going forward, not just in contract manufacturing expenses, but also in clinical. And recall that we are comparing this to 2022 and a lot of the efforts in 2023 are going towards Working to prepare for a Phase 3 clinical trial. So R and D, we expect overall R and D and G and A operating expenses to be between

Speaker 8

Okay. Thanks, Laurie. Thank you, everyone, for taking my questions.

Speaker 2

Thank you, Ed.

Operator

One moment please for next question. Our next question is from Catherine Novak of Jones Trading. Your line is now live.

Speaker 9

Hi, good afternoon everyone. Just a question on some KOL issues. What we've heard time and again That their real focus in PHD is 12, 24 months and beyond. And here we tend to see injectable growth hormone Attenuate after about the 1st year. So given the mechanism of action, is it safe to say you wouldn't expect this to be So dramatic with LUM-two zero one?

Speaker 9

And then how should this change our expectations for what we should see in the data in Phase 2 and Phase 3?

Speaker 2

Well, thanks for that excellent question, Catherine. And I John, will you answer that?

Speaker 5

Sure. So, 2 things to keep in mind. The Phase 3 study will be a 12 month And so kind of the key decision point for that Phase 3 will be non inferiority at 12 months. Obviously, we have Several ongoing trials now that are longer than 12 months, right, and we'll hopefully keep as many kids as we Again, in that trial and transition kids from the Phase 3 into an extension trial as well. So we will have a body of data On treatment past 12 months, but it won't be as comprehensive as the data we have at 6 12 months.

Speaker 5

And that's the same data package that all the long acting have brought in. That said, you can look at the difference in growth or look at the difference in annualized site velocity for individual patients At 6 months, at 12 months, at 18 months and look at the slope of that decline. And as you said, it's well documented that growth hormone has its kind of biggest peak in the 3 to 6 month range. And Every year after that you get a decline, you don't get the same growth every year, right? You get a decline in return.

Speaker 5

Loom-two zero one has been shown in adults, it's been published in adults that you can elevate IGF-one hundred growth hormone levels out to 24 months, right? So in adults that change body composition, we get that same level of continued elevation in kids, we would expect to see a continued growth, right? And I think some of the early data that we're going to reveal at the end of this Here on the handful of kids past 12 months will really start to help put those pieces together for us. But there are certainly scenarios where you could expect We're going to modulate kids to a more natural growth pattern that could be a much flatter growth slow than what you might see with kind of the pharmacological or supraphysiological levels of Injectable Growth Homa. Thank you for that question.

Speaker 5

Yes,

Speaker 3

of course.

Speaker 9

And then just one more. Now that we've got a couple weekly injectables in the market for CGHD, what kind of feedback are you getting From KOLs are what are you hearing? How excited are they to use them? And does this factor into your LUM-two zero one market expectations at all?

Speaker 2

Catherine, look, there's no question that this could be a highly competitive space. And you look at the price on a monthly basis between the 3 growth hormone, long acting growth hormones, there's something like $7,000 $8,000 $9,000 per month, I'll let to yearly cost of over $100,000 for a 35 kilo kid. That's expensive treatment. I think there are certain parts of the world where the standard of care is We continue to be a daily because of restriction in price or longer time to approvals in different markets around the world. We believe I mean, we've done our market research.

Speaker 2

We've asked the question both to Pete Endo's and the parents if you had a choice between a weekly injection or daily oral, they overwhelmingly say they would choose a daily oral. And on top of that, I want to remind you, we're a small molecule, our cost of goods and our flexibility and pricing and margins is It looks very favorable to us long term in this market.

Speaker 6

Well, Kathleen, may I add was the feedback from the KOL industry guard. Rick mentioned about the pricing. That's very important, right? It's more expensive than daily. The other issue that the KRL has some skeptical is the safety data profile.

Speaker 6

As you know that in order to get approved FDA, you only have 12 months safety data. However, long term safety, especially long acting, not only look at efficacy itself, the long term side effect, which is again, it's very unclear at the short term, right? When you look at profile, the growth hormone exposure for weekly growth hormone is much higher than daily growth hormone And also higher IGF-one. With that said, long term post Phase 4 study or post marking study need to continue in order to show off the safety profile in the long term fashion. So that's something that some KOLs still quite skeptical to really put all subjects in weekly cohort wound therapy in regard to efficacy itself, right?

Speaker 6

So not to mention it's very expensive.

Speaker 2

Yes. And Catherine recall, our mechanism of action is very different from growth hormones. We stay within the natural endocrine feedback loop. Those experiences you get with higher levels of growth hormone with especially the long acting is not Possible with our drug. Once again, that feedback loop stays within the natural physiology.

Speaker 9

Okay. That's answered my question. Thanks very much.

Speaker 2

Thanks.

Operator

One moment please for our next question. Our next question is from Pete Stavropoulos of Cantor Fitzgerald. Your line is now live.

Speaker 10

Hi, Rick and team. Nice to see all the progress made this quarter and movement towards data readout by year end. First question is, when you look at the data that you've generated to date using the PAMA, the predictive enrichment marker and the growth that you've observed in these patients, just curious to know as you move into a later larger study, If you expect to keep the cutoffs where they currently are or do you think you can increase the probability of success by focusing or enriching patients with a greater response? Or is the current algorithm sufficient?

Speaker 2

Pete, thank you for that good question. And John, I'm going to let you start.

Speaker 5

I think, Pete, we're comfortable with the enrichment that we're getting from using our PEM markers right now, but obviously we haven't looked at all the data yet, right? So we need to look at the AHV data for the other half of the patients that have come in. But I think we feel pretty confident based on the analysis that we originally did and as well as how things are rolling on time.

Speaker 10

Okay. And as you think about a Phase 3 design, I understand that you need to end the Phase 2 meeting And to come to an agreement with the agency. However, are there any modifications you might make to enrollments or trial design For a pivotal study based on the accumulating data from 210 and 212?

Speaker 2

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

Speaker 5

No. I think the Phase 3 design and basic kind of inclusion characteristics are pretty common across all the 3 long acting products that have been successful and Are pretty much how we would go forward. Obviously, our patient population has to be pem positive. That's a distinction. But other than that, I think that the timeline and the approximate ends, I think, are Pretty close to what we would expect after we do the analysis at the end of the study.

Speaker 5

So we don't see any radical departures From those that have come before us here.

Speaker 10

Okay. And sorry, go ahead. Do you have Something to add?

Speaker 2

No, go ahead. Please go ahead.

Speaker 10

And last question directed towards Duke, but Rick, John, more than welcome. So you've presented Luma has presented a number of times at different endocrinology meetings most recently at end of 2023. So just wondering when you speak to KOLs, some of your former colleagues and even practicing clinicians about LUM-two zero one, it's a mechanism of action and how it may be Just curious to hear what the reception of this candidate and approaches and the data presented today?

Speaker 2

Yes. And so, Dhiv, why don't you go ahead?

Speaker 6

Yes. So actually, it's very, very positive. As you may know, right, this is oral growth hormone security clock have been proposed for the treatment for years, But none of those trials have been successful. So here is the LUM-two zero one, which is mechanism of action of the drug cell is somewhat differentiated itself from the oral growth hormone secreting peptide or secrete the go out there, right. We have long acting, you generate IGF-one that sustain.

Speaker 6

And based on the presentation at PS and at ENDO, one represent only 210 data alone in regards to growth velocity and is very clearly shown that compared to historical data, moderate idiopathic population, the growth velocity somewhat match. And we look at the combined data, Trying to see that what the response look like. It seemed like the dose that have been selected, especially 1 point 63.2 is pretty comparable and you can see those data in 6 months 12 months result. But again, it's very small number, But most of physicians really feel intrigued about the data. They did not know that this growth hormone oral secret drug will work as Good, right.

Speaker 6

And again, because it was failed in the past. Not to mention that if this drug get approved, will be alternative for their patient and especially the impact of compliance should be less. And again, we expect that the burden injection with the majority of those patients suffering from daily or long acting could be somewhat overcome by this new therapy. But again, overall, it's very, very positive. They all just need to looking forward for the top line data and especially the new Phase 3 pivotal trial in the future.

Speaker 5

The only thing I would add to that, Pete, is that when we do these presentations at Impey in March, at PES and at Endo, Room is packed, right? And there are lots of good questions, lots of excitement, lots of people wanting to talk to the presenters about this product, so 1 on 1 and then in the lecture halls, we feel a lot of excitement and a lot of interest.

Speaker 2

Yes. And I might add, Pete, that we always have a booth at each one of these meetings and right alongside all the competitors in the growth hormone space. And I am absolutely amazed at the level of interest and The number of really great questions and just overall excitement that everybody feels, oh, the fact that there's a possibility of an oral Alternative for their patients. I mean, it's a very important and exciting space to be in right now just based on the feedback from these clinicians.

Speaker 10

That's great to hear. So thank you for taking our questions and congratulations on the progress.

Speaker 2

All right. Thank you, Pete.

Operator

Thank you. I'm showing no further questions on the line. This concludes the Lumos Pharma Second Quarter Earnings Call. Thank you and have a great

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Earnings Conference Call
Lumos Pharma Q2 2023
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