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spk15: Good afternoon and welcome to Lumos Pharma's third quarter 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.
spk23: 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. Additional information concerning factors that could cause actual results to differ is contained in our periodic reports filed with 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 8K, which may be accessed from the Investors page of the company's website. Speaking on today's call will be Rick Hawkins, CEO and Chairman, and Lori Lawley, Chief Financial Officer. Following their prepared remarks, Rick and Laurie will be joined by John McHugh, our President and Chief Scientific Officer, as well as Dr. David B. Karpf, our Chief Medical Officer for the question and answer session. I will now turn the call over to Rick.
spk05: Thank you, Lisa. Good afternoon, everyone, and thank you for joining us on today's call. After the market closed today, we issued a press release announcing our financial results for the 2021 third quarter and detailing our progress advancing Loom 201 for the treatment of pediatric growth hormone deficiency, or PGHD. On today's call, I'll provide the overview of our Loom 201 program and broader development strategy and give an update on our clinical trials, and Lori Lawley will review our financial results. Then we'll be happy to take your questions. So I'm pleased to report that since we last spoke with you, we've made steady progress on our Loom 201 program. The majority of sites for our Oral Growth 210 trial are now open for enrollment, and importantly, most of those that we opened recently or will open soon are historically high enrollment sites. With screening and enrollment for Oral Growth 210 progressing, we continue to expect the primary outcome data readout for this trial in the second half of 2023. Enrollment has picked up in our Oral Grow 212 trial. You'll recall that Oral Grow 212 is a single-site, open-label trial designed to provide pharmacodynamic data on LUM201. Primary endpoint is six-month data with additional 12-month data to be captured. Since the trial is open-label, an interim analysis may be conducted at the company's discretion. As a reminder, our Orgo 210 trial is a global clinical study evaluating oral LUM201 in approximately 80 patients diagnosed with PGHD. The primary clinical outcome is annualized height velocity in patients selected by our predictive enrichment marker, or PEM, strategy. Secondary outcome measures include comparison of annualized height velocity of LUM201 in three dose levels, 0.8, 1.6, and 3.2 mg per kg versus a control arm of patients treated with recombinant growth hormone at a daily dose of 0.24 mg per kg per week. Dose levels of LUM201 were selected to span the entire dose response curve elucidated in a prior PKPD study. The goals of our Oral Growth 210 trial are to identify the optimal dose of LUM201 to be used in a Phase III registration trial and validate the PEM strategy. During the last quarter, we made significant progress toward our goal of opening approximately 50 sites. Currently, we have over 40 sites open and screening patients. Additional high-volume sites expected to open shortly include those in Russia, Ukraine, New Zealand, and Israel. And based on the progress made in recent weeks, we feel confident that enrollment supports our six-month data readout for the Oral Growth 210 trial the second half of 2023, with additional 12-month data to be collected. Turning now to our Oral Growth 212 study. This is our single-site open-label trial evaluating the pharmacokinetic and pharmacodynamic effects of LUM201 in up to 24 PGAC patients at two dose levels, 1.6 and 3.2 mg per kg per day. This trial continues to enroll patients at an encouraging pace. The objective of the oral growth 212 trial is to confirm prior clinical data demonstrating the amplified pulsatile release of endogenous growth hormone unique to LUM201 and the potential for this mechanism of action to increase growth hormone secretion across the entire dose-response curve in the majority of PGSD patients. The primary endpoint is six months of PK, PD, and high-velocity data with additional 12-month data to be captured. And given the open-label design of this trial, we have the ability to perform an interim analysis at our discretion. Now, in addition to advancing our clinical trials for LUM201 and PGHD, during the quarter, we continued to explore expansion opportunities for LUM201 and to other therapeutic areas where injectable recombinant human growth hormone is a standard of care. As we've said previously, we believe that LUM201 is a pipeline and a product, and through its unique mechanism of action, may have the potential to be efficacious in indications such as Turner syndrome, Prader-Willi syndrome, idiopathic short stature, and children born small for gestational age. We are actively reviewing potential clinical development plans for LUM201 in several of these indications and are in advanced discussions with key opinion leaders and our clinical and scientific advisory board about the best next step for expanding our Loom 201 pipeline. So we look forward to providing a more detailed update on these plans soon. We also continue to be judicious in our pursuit of rare disease assets beyond Loom 201 with careful attention to shareholder value creation. That said, our priority remains Loom 201 and its development. So before I turn the call over to Lori, I just want to remind everyone of some context surrounding our LUM201 program and some of the reasons why we have confidence in our current clinical trials and the potential of LUM201 generally. Now, you may recall, unlike standard of care PGST therapies or the weekly injectables coming to market, LUM201 is an oral growth hormone secretagogue that acts within the body's natural endocrine pathway. And LUM201 acts selectively on receptors in the pituitary and hypothalamus to stimulate the body's ability to release growth hormone at the same intervals and subject to the same intricate feedback loops that occur naturally. This mechanism enables the naturally occurring IGF-1 feedback loop to be preserved to help regulate the balance of growth hormone and IGF-1 levels in the body. So less severe PGHD patients are those with an intact pituitary growth hormone axis, but who are secreting insufficient growth hormone to attain normal height, are those who should respond best to LUM201. Evidence suggests that this group represents approximately 60% of the total PGHD population, and our trials are designed using specific predictive enrichment markers, or PEMs, to identify this addressable population. And with that, I'll pass it over to Lori for a review of our third quarter financial results. Lori?
spk16: Thanks, Rick, and good afternoon, everyone. During our third quarter, research and development expenses were $4.1 million compared to $2.1 million in the same period in 2020. This increase of $2 million was primarily due to increases of $1.8 million in clinical trial and contract manufacturing expenses. $400,000 in personnel-related expenses and $100,000 in stock compensation expense, offset by a decrease of $300,000 in supplies and other expenses. General and administrative expenses in the third quarter of 2021 were $3.4 million compared to $5.2 million in the third quarter of 2020. This decrease of $1.8 million was primarily due to decreases of $1.3 million in personnel-related expenses and $500,000 in legal consulting and other operating expenditures. For the third quarter, we recorded a net loss of $7.5 million compared to net income of $1.8 million for the prior year quarter. In Q3 of 2020, the company recorded a gain of $6.5 million related to the sale of our priority review voucher and a tax benefit of $2.4 million during Q3 of 2020, which offset the operating expenses in that quarter of $7.2 million. We ended the third quarter of 2021 with cash and cash equivalents totaling $1.7 million compared to $98.7 million on December 31, 2020. We currently expect our cash on hand as of the end of third quarter to support operations for the six-month data readouts from both the ORA Growth 210 and ORA Growth 212 trials. I'll now turn it back to Rick for closing remarks.
spk05: Thank you, Lori, and before taking your questions, I just want to remind everyone that our immediate focus is on the execution of our current clinical plan for Loom 201 and exploration of the indications we will pursue next to expand this platform. While lifecycle management of Loom 201 is our priority, we will opportunistically consider other business development opportunities as they present, and then we'll remain highly selective though, and focus on long-term value creation for our shareholders. So we look forward to sharing more details with you soon. So, operator, we're now ready to take questions.
spk15: Thank you. And to ask a question, you will need to press star 1 on your telephone. To withdraw your question, press the pound key. Again, to ask a question, please press star 1. And our first question is from Charles Duncan of Cantor Fitzgerald. Your line is open.
spk11: Yeah, thank you. Good afternoon, Rick and team. Thanks for taking our questions and thanks for the quick update. Wanted to ask you a little bit about the 210 study. You mentioned high enrolling sites and I guess I'm wondering how you know what a high-enrolling site is. Did you do some feasibility study to be able to demonstrate that? And then with regard to timing, I know that you're deploying the predictive enrichment marker paradigm, and that makes sense, but could you remind us what your screen-to-enrollment assumptions are and give us a sense of color to the timelines for six-month readout in that study?
spk05: Thank you for the question, Charles. You know, in terms of the high enrollment sites, I think these investigators are incredibly well known, and as you know, growth hormone, recombinant growth hormone has been around for 35 years, and there have been a number of clinical trials that have been conducted over the years. I think mainly looking at data from those trials in terms of investigators and their enrollment patterns, they've become pretty clear. And as I said, some of those sites, those high-enrolling sites are now coming online, so our expectation of having an increased enrollment is certainly there. We haven't released any information on screen failures, or I'm not sure it's as relevant in a trial like this, especially when we're using a predictive enrichment marker or PEMs, but we haven't released any data there, Charles.
spk11: Okay, well, I guess I'm assuming you mentioned later on in your comments that patients who may respond you know, to growth hormone, but inadequately represent maybe 50% of the patient population. So that's probably a good place to start. But I guess I'm wondering if you think that patients who, you know, may present to a clinical trial may be enriched in any way in terms of, you know, more of them presenting that may respond to PEM or not.
spk05: John, do you want to answer that question? John, I think you're on hold or on mute.
spk08: Can you hear me now?
spk04: Yes.
spk08: I think you can hear me now. You know, the data indicates that upwards of 60% of patients diagnosed with PGHD should be responsive to our molecule just based on database analysis and kind of the general division of growth hormone deficiency spectrum. So I think with that said, you know, we... We've done a lot of work to educate all of our investigators on what type of patients are going to be most responsive to our molecule, and they've actually done quite a good job at finding patients who fit the needs just generally for a patient with what's called kind of idiopathic growth hormone deficiency, so not organic, not the most severely growth hormone deficient kids. And so I think the screening rates we're seeing right now don't align with screening studies that we've done in large databases of everyone diagnosed with growth hormone deficiency. So I think we're getting a fairly high return rate because the clinicians on our sites are pretty cognizant of the kind of patient who's going to respond to our therapeutic.
spk11: Okay, very helpful. Last question is back to or on to 212. This 212 study, Rick mentioned conducting an interim analysis that would be conducted at the company's discretion, and I guess I'm wondering what kind of factors you may consider in doing that interim analysis. Does it have something to do with the protocol that's specified or a certain number of patients? We would like to hear updates, so looking forward to that, but just kind of wondering what would drive that decision.
spk06: Yeah, go ahead, John.
spk08: So I think what we'd like to see out of that interim analysis is essentially a large enough N at each of the two doses that we're exploring, so 1.6 and 3.2 mg per kg per day, that we have a data set that I think is important to share. We've released data on the 0.8 mg per kg dose in three patients, two PEM positives and one PEM negative, and that's great kind of case report type information, but we'd like to have a larger cohort when we release the higher doses. And, you know, we have defined that in our protocol, and as soon as we reach that interim decision, we'll move forward with that.
spk11: Okay. Thanks, John. Thanks for taking the questions, Rick.
spk03: Thank you, Charles.
spk15: Our next question is from Yasmin Rahimi of Piper Sandler. Your line is open.
spk13: Hi, team. Can you hear me?
spk25: Yes.
spk14: Oh, cool. Thank you. This is Jessie on for Yas. Congrats on the quarter, and thank you for taking our questions and hosting the call. I wanted to kind of get into the Python expansion. I know that during the prepared remarks, you mentioned Again, talking about Ascension-Turner Syndrome and Prader-Willi, and I just wanted to know, can you please explain how LUM201 would work in these indications and which one would Lumos most prefer to go after?
spk05: So, John, why don't you go ahead with the mechanism of action in those patient populations?
spk08: Yeah, so I think the way we've looked at this is there are, you know, a group of patients who have an active access that can be stimulated with a growth hormone secretagogue like LUM201 to increase their growth hormone release, right, and then in turn increases IGF-1 and downstream modulators. And you know, for growth hormone deficiency, it's, for pediatric growth hormone deficiency, it aligns perfectly with our mechanism if we choose our patient populations correctly. And we spend a lot of time just aligning our thinking around many of the other indications, right, where there are kind of a constellation of things that might affect, you know, for many of them, an individual child's growth patterns, right? Some of those other indications are a little bit more complicated, and so what we're spending time doing right now is weighing the different contributions and looking at how successful doses of growth hormone are in stimulating growth. Now, not all these approved indications are growth-driven. There are indications for adults, but that's just an example of how we are trying to make sure that our mechanism you know, and the kind of patients that can respond to our molecules, those who have an active axis and can be stimulated with growth hormone secretion to increase their growth hormone production, right? So that's really the thinking we're doing as we go through the 11 approved indications for recombinant human growth hormone and try to prioritize them as we go forward.
spk14: Thank you. And then when you say the updates are soon, can we expect them on the 4Q call or 2022? Can you Can you let us know?
spk05: Absolutely.
spk14: Okay. Oh, sorry. I meant, like, will we have an update on which indication on the first year fall, or would it be in 2022 or beyond?
spk05: You know, we haven't talked about the time we're going to, the time period we're going to do, but we're actually going through our discussions with our KOLs and our clinical and scientific advisory board at the moment. And we're not quite there yet, but I think soon we'll be able to tell you which ones we're going to first.
spk14: All right, thank you so much, Tim. Those are my questions.
spk15: And our next question is from Elmer Peros of Roth Capital Partners. Your line is open.
spk02: Good afternoon, Vic. And I just had one question, please. I just wanted to verify whether the clinicaltrials.gov information is correct. I see that there are 42 sites are now open, and 40 out of the 42 is actually recruiting. And I think you plan to have 50 altogether, just trying to see how close to the final number you are.
spk05: Yeah, I'm not sure that the site keeps up with the total number, but to say the least, we have at least those 40 sites open and recruiting patients. And as we speak, we're activating more of these sites, and as I mentioned earlier, I think there are some traditional high-enrollment sites that are now just coming online, so we're optimistic that that's going to improve our enrollment considerably in the very near future. Thank you for confirming that, Rick. Thank you, Delmer.
spk15: And our next question is from Catherine Novak of Jones Research. Your line is open.
spk17: Hi, thanks so much for taking my question. I just wanted to ask kind of like a bigger picture question about the pediatric growth hormone market. You know, with the approval of Ascendas' TransCon GH, I guess, how do you see that shaping the market? And especially, what have you heard from KOLs about potential prescribing practices for weekly growth hormone, you know, given that this product is sometimes associated with higher than normal levels of IGF-1?
spk05: We haven't gotten a lot of feedback. We're really focused on our own programs. However, the market release price we're more than pleased with. I can imagine as a small molecule and a lower cost of manufacturing, that leaves us a great deal of flexibility in pricing, especially at that level. I think it's a little too early to tell what the response in the market is going to be, and from the pediatric endocrinologist, but maybe I can ask Dr. Karf to talk about that a little bit more.
spk09: Sure. Again, as Rick said, I work very closely on what became Skytrofa program, and it has been approved. I have not even heard yet that it's been actually launched. I would imagine it will take some time to get onto formularies, which is the real driver for sales, because it's really the outpatient. It's the cost of the patient that dictates sales on the market. And There is no question that the cost of goods for Loom 201 will be much less than the cost of goods for Skytrofa, which does provide quite good flexibility.
spk17: Got it. OK. Yeah, I think maybe what I was trying to get at is comfort in general with a weekly. Have you heard anybody who might be a little bit hesitant to prescribe weekly GH, or is IGF-1 levels not a concern for some KOLs?
spk05: David, if you want to address that at all?
spk10: Sure.
spk09: I think that most of the key opinion leaders that I talked with when I was came to the development to send us, did not have a big concern about IGF-1 levels because traditionally, because kids with growth hormone deficiency are rarely diagnosed right when they develop it, there's a lag time to diagnosis and treatment. You don't want to target kind of the average IGF-1 value in these kids, maybe if you've got five years to treat them to adult height. So you want to tend to be in the upper half of the normal range. And there are data showing that if you target a positive two, you get better growth than if you target an IGF-1 STS of zero. And by definition, if you're targeting an IGF-1 STS of two, you're going to have half above, half below. So I don't think that it's a big concern. And there's no question that on everything else being equal, most kids and parents would prefer one injection a week compared to seven injections per week. But by the same token, it's not at all unlikely that a lot of people would prefer a once daily oral compound to even a once weekly injection.
spk05: And Catherine, I think that as pediatric endocrinologists, many of them will wait and see, I believe, to see what the patient population reports or their colleagues report over time. I think there's going to be some early adopters, but I also think there will be some pediatric endocrinologists who will not jump in right away. perhaps because of the excursions of ITF1 levels that sometimes can be of concern to some clinicians.
spk17: Got it. Thanks very much.
spk04: Thank you, Catherine.
spk15: Thank you. I am showing no further questions in the queue at this time. Thank you for joining us and enjoy your afternoon.
spk05: Thank you very much. Thank you. Thank you. you music Thank you.
spk24: Thank you. music music
spk15: Good afternoon and welcome to Lumos Pharma's third quarter 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.
spk23: 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. Additional information concerning factors that could cause actual results to differ is contained in our periodic reports filed with 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 8K, which may be accessed from the Investors page of the company's website. Speaking on today's call will be Rick Hawkins, CEO and Chairman, and Lori Lawley, Chief Financial Officer. Following their prepared remarks, Rick and Lori will be joined by John McHugh, our President and Chief Scientific Officer, as well as Dr. David B. Karpf, our Chief Medical Officer for the question and answer session. I will now turn the call over to Rick.
spk05: Thank you, Lisa. Good afternoon, everyone, and thank you for joining us on today's call. After the market closed today, we issued a press release announcing our financial results for the 2021 third quarter and detailing our progress advancing Loom 201 for the treatment of pediatric growth hormone deficiency, or PGHD. On today's call, I'll provide the overview of our Loom 201 program and broader development strategy and give an update on our clinical trials, and Lori Lawley will review our financial results. Then we'll be happy to take your questions. So I'm pleased to report that since we last spoke with you, we've made steady progress on our Loom 201 program. The majority of sites for our Oral Growth 210 trial are now open for enrollment, and importantly, most of those that we opened recently or will open soon are historically high enrollment sites. With screening and enrollment for Oral Growth 210 progressing, we continue to expect the primary outcome data readout for this trial in the second half of 2023. Enrollment has picked up in our Oral Grow 212 trial. You'll recall that Oral Grow 212 is a single-site, open-label trial designed to provide pharmacodynamic data on LUM201. Primary endpoint is six-month data with additional 12-month data to be captured. Since the trial is open-label, an interim analysis may be conducted at the company's discretion. As a reminder, our Orgo 210 trial is a global clinical study evaluating oral LUM201 in approximately 80 patients diagnosed with PGHD. The primary clinical outcome is annualized height velocity in patients selected by our predictive enrichment marker, or PEM, strategy. Secondary outcome measures include comparison of annualized height velocity of LUM201 in three dose levels, 0.8, 1.6, and 3.2 mg per kg versus a control arm of patients treated with recombinant growth hormone at a daily dose of 0.24 mg per kg per week. Dose levels of LUM201 were selected to span the entire dose response curve elucidated in a prior PKPD study. The goals of our Oral Growth 210 trial are to identify the optimal dose of LUM201 to be used in a Phase III registration trial and validate the PEM strategy. During the last quarter, we made significant progress toward our goal of opening approximately 50 sites. Currently, we have over 40 sites open and screening patients. Additional high volume sites expected to open shortly include those in Russia, Ukraine, New Zealand, and Israel. And based on the progress made in recent weeks, We feel confident that enrollment supports our six-month data readout for the Oral Growth 210 trial in the second half of 2023 with additional 12-month data to be collected. Turning now to our Oral Growth 212 study. This is our single-site open-label trial evaluating the pharmacokinetic and pharmacodynamic effects of LUM201 in up to 24 PGAC patients at two dose levels, 1.6 and 3.2 mg per kg per day. This trial continues to enroll patients at an encouraging pace. The objective of the Oral Growth 212 trial is to confirm prior clinical data demonstrating the amplified pulsatile release of endogenous growth hormone unique to LUM201 and the potential for this mechanism of action to increase growth hormone secretion across the entire dose-response curve in the majority of PGSD patients. The primary endpoint is six months of PK, PD, and high-velocity data with additional 12-month data to be captured. And given the open-label design of this trial, we have the ability to perform an interim analysis at our discretion. Now, in addition to advancing our clinical trials for LUM201 and PGHD, during the quarter, we continued to explore expansion opportunities for LUM201 and to other therapeutic areas where injectable recombinant human growth hormone is a standard of care. As we've said previously, we believe that LUM201 is a pipeline and a product, and through its unique mechanism of action, may have the potential to be efficacious in indications such as Turner syndrome, Prader-Willi syndrome, idiopathic short stature, and children born small for gestational age. We are actively reviewing potential clinical development plans for Loom 201 and several of these indications and are in advanced discussions with key opinion leaders and our clinical and scientific advisory board about the best next step for expanding our Loom 201 pipeline. So we look forward to providing a more detailed update on these plans soon. We also continue to be judicious in our pursuit of rare disease assets beyond LUM201 with careful attention to shareholder value creation. That said, our priority remains LUM201 and its development. So before I turn the call over to Laurie, I just want to remind everyone of some context surrounding our LUM201 program and some of the reasons why we have confidence in our current clinical trials and the potential of LUM201 generally. Now, you may recall, unlike standard-of-care PGST therapies or the weekly injectables coming to market, LUM201 is an oral growth hormone secretagogue that acts within the body's natural endocrine pathway. And LUM201 acts selectively on receptors in the pituitary and hypothalamus to stimulate the body's ability to release growth hormone at the same intervals and subject to the same endocrine feedback loops that occur naturally. This mechanism enables the naturally occurring IGF-1 feedback loop to be preserved to help regulate the balance of growth hormone and IGF-1 levels in the body. So less severe PGHD patients are those with an intact pituitary growth hormone axis but who are secreting insufficient growth hormone to attain normal height are those who should respond best to LUM201. Evidence suggests that this group represents approximately 60% of the total PGAC population, and our trials are designed using specific predictive enrichment markers, or PEMs, to identify this addressable population. And with that, I'll pass it over to Lori for a review of our third quarter financial results. Lori?
spk16: Thanks, Rick, and good afternoon, everyone. During our third quarter, research and development expenses were $4.1 million compared to $2.1 million in the same period in 2020. This increase of $2 million was primarily due to increases of $1.8 million in clinical trial and contract manufacturing expenses, $400,000 in personnel-related expenses, and $100,000 in stock compensation expense, offset by a decrease of $300,000 in supplies and other expenses. General and administrative expenses in the third quarter of 2021 were $3.4 million compared to $5.2 million in the third quarter of 2020. This decrease of $1.8 million was primarily due to decreases of $1.3 million in personnel-related expenses and $500,000 in legal consulting and other operating expenditures. For the third quarter, we recorded a net loss of $7.5 million compared to net income of $1.8 million for the prior year quarter. In Q3 of 2020, the company recorded a gain of $6.5 million related to the sale of our priority review voucher and a tax benefit of $2.4 million during Q3 of 2020, which offset the operating expenses in that quarter of $7.2 million. We ended the third quarter of 2021 with cash and cash equivalents totaling $1.7 million compared to $98.7 million on December 31, 2020. We currently expect our cash on hand as of the end of third quarter to support operations for the six-month data readouts from both the ORA Growth 210 and ORA Growth 212 trials. I'll now turn it back to Rick for closing remarks.
spk05: Thank you, Lori. And before taking your questions, I just want to remind everyone that our immediate focus is on the execution of our current clinical plan for LUM201 and exploration of the indications we will pursue next to expand this platform. While lifecycle management of Loom 201 is our priority, we will opportunistically consider other business development opportunities as they present. And then we'll remain highly selective, though, and focus on long-term value creation for our shareholders. So we look forward to sharing more details with you soon. So, operator, we're now ready to take questions.
spk15: Thank you. And to ask a question, you will need to press star 1 on your telephone. To withdraw your question, press the pound key. Again, to ask a question, please press star 1. And our first question is from Charles Duncan of Cantor Fitzgerald. Your line is open.
spk11: Yeah, thank you. Good afternoon, Rick and team. Thanks for taking our questions, and thanks for the quick update. I wanted to ask you a little bit about the 210 study. You mentioned high-enrolling sites, and I guess I'm wondering how you know what a high-enrolling site is. Did you do some feasibility study to be able to demonstrate that? And then with regard to timing, I know that you're deploying the predictive enrichment marker paradigm, and that makes sense, but could you remind us what your screen-to-enrollment assumptions are and give us a sense of color to the timelines for a six-month readout in that study?
spk05: Thank you for the question, Charles. In terms of the high-enrollment sites, I think These investigators are incredibly well-known, and as you know, growth hormone, recombinant growth hormone has been around for 35 years, and there have been a number of clinical trials that have been conducted over the years. I think mainly looking at data from those trials in terms of investigators and their enrollment patterns, they've become pretty clear. And as I said, some of those sites, those high-enrolling sites, are now coming online. So our expectation of having an increased enrollment is certainly there. We haven't released any information on screen failures, or I'm not sure it's as relevant in a trial like this, especially when we're using a predictive enrichment marker or PEMs. but we haven't released any data there, Charles.
spk11: Okay, well, I guess I'm assuming you mentioned, you know, later on in your comments that, you know, patients who may respond, you know, to growth hormone but inadequately represent maybe 50% of the patient population, so that's probably a good place to start, but I guess I'm wondering if if you think that patients who may present to a clinical trial may be enriched in any way in terms of more of them presenting that may respond to PEM or not.
spk05: John, do you want to answer that question? John, I think you're on hold or on mute.
spk08: Can you hear me now?
spk04: Yes.
spk08: I think you can hear me now. So the data indicates that upwards of 60% of patients diagnosed with PGHD should be responsive to our molecule just based on database analysis and kind of the general division of growth hormone deficiency spectrum. So I think with that said, you know, we – We've done a lot of work to educate all of our investigators on what type of patients are going to be most responsive to our molecule, and they've actually done quite a good job at finding patients who fit the needs just generally for a patient with what's called kind of idiopathic growth hormone deficiency, so not organic, not the most severely growth hormone deficient kids. And so I think, you know, the screening rates we're seeing right now don't align with screening studies that we've done in large databases of everyone diagnosed with growth hormone deficiency. So I think we're getting a fairly high return rate because the clinicians on our sites are pretty cognizant of the kind of patient who's going to respond to our therapeutic.
spk11: Okay, very helpful. Last question is back to or on to 212. This 212 study, Rick mentioned conducting an interim analysis that would be conducted at the company's discretion, and I guess I'm wondering what kind of factors you may consider in doing that interim analysis. Does it have something to do with the protocol that's specified or a certain number of patients? We would like to hear updates, so looking forward to that, but just kind of wondering what would drive that decision.
spk06: Yeah, go ahead, John.
spk08: So I think what we'd like to see out of that interim analysis is essentially a large enough N at each of the two doses that we're exploring, so 1.6 and 3.2 mg per kg per day, that we have a data set that I think is important to share. We've released data on the 0.8 mg per kg dose in three patients, two PEM positives and one PEM negative, and that's great kind of case report type information, but we'd like to have a larger cohort when we release the higher doses. And, you know, we have defined that in our protocol, and as soon as we reach that interim decision, we'll move forward with that.
spk11: Okay. Thanks, John. Thanks for taking the questions, Rick.
spk03: Thank you, Charles.
spk15: Our next question is from Yasmin Rahimi of Piper Sandler. Your line is open.
spk13: Hi, Tim. Can you hear me?
spk25: Yes.
spk14: Oh, cool. Thank you. This is Jessie on for Yas. Congrats on the quarter, and thank you for taking our questions and hosting the call. I wanted to kind of get into the Python expansion. I know that during the prepared remarks, you mentioned Again, talking about Ascension-Turner Syndrome and Prader-Willi. And I just wanted to know, can you please explain how LUM201 would work in these indications and which one would Lumos most prefer to go after?
spk05: So, John, why don't you go ahead with the mechanism of action in those patient populations.
spk08: Yeah, so I think the way we've looked at this is there are, you know, a group of patients who have an active access that can be stimulated with a growth hormone secreted gog like LUM201 to increase their growth hormone release, right, and that in turn increases IGF-1 and downstream modulators. And, you know, for growth hormone deficiency, it's for pediatric growth hormone deficiency, it aligns perfectly with our mechanism if we choose our patient populations correctly and we spent a lot of time just aligning our thinking around many of the other indications right where there are kind of a constellation of things that might affect and you know for many of them an individual child's growth patterns right some of those other indications are a little bit more complicated and so what we're spending time doing right now is you know weighing the different contributions and looking at how successful you know, doses of growth hormone are in stimulating growth. Now, not all these approved indications are growth-driven. There are indications for adults. But that's just an example of how we are trying to make sure that our mechanism, you know, and the kind of patients who can respond to our molecules, those who have an active axis and can be stimulated with growth hormone secreticol to increase their growth hormone production, right? So that's really the thinking we're doing as we go through the 11 approved indications. for common human growth hormone and try to prioritize them as we go forward.
spk14: Thank you. And then when you say the updates are soon, can we expect them on the 4Q call or 2022? Can you let us know?
spk05: Absolutely.
spk14: Sorry, I meant like will we have an update on which indications on the first recall or would it be in 2022 or beyond?
spk05: You know, we haven't talked about the time we're going to, the time period we're going to do, but we're actually going through our discussions with our KOLs and our Clinical and Scientific Advisory Board at the moment. And we're not quite there yet, but I think soon we'll be able to tell you which ones we're going to first.
spk14: All right, thank you so much, team. Those are my questions.
spk15: And our next question is from Elmer Peros of Roth Capital Partners. Your line is open.
spk02: Good afternoon, Rick. And I just had one question, please. I just wanted to verify whether the clinicaltrials.gov information is correct. I see that there are 42 sites are now open. and 40 out of the 42 is actually recruiting, and I think you plan to have 50 altogether, just trying to see how close to the final number you are.
spk05: Yeah, I'm not sure that the site keeps up with the total number, but to say the least, we have at least 40 sites open. and recruiting patients. And as we speak, we're activating more of these sites. And as I mentioned earlier, I think there are some traditional high-enrollment sites that are now just coming online. So we're optimistic that that's going to improve our enrollment considerably in the very near future.
spk02: Thank you for confirming that, Rick.
spk05: Thank you, Delmer.
spk15: And our next question is from Catherine Novak of Jones Research. Your line is open.
spk17: Hi. Thanks so much for taking my question. I just wanted to ask kind of like a bigger picture question about the pediatric growth hormone market. You know, with the approval of Ascendus' TransCon GH, I guess, how do you see that shaping the market? And especially, what have you heard from KOLs about potential prescribing practices for weekly growth hormone, you know, given that this product is sometimes associated with higher than normal levels of IGF-1?
spk05: You know, we haven't gotten a lot of feedback. We're really focused on our own programs. However, the market release price we're, you know, more than pleased with. I can imagine it's a small molecule and a lower cost of manufacturing. that leaves us a great deal of flexibility in pricing, especially at that level. I think it's a little too early to tell what the response in the market is going to be, and from the pediatric endocrinologist, but maybe I can ask Dr. Karf to talk about that a little bit more.
spk09: Sure. Again, as Rick said, I work very closely on the What Became Skytropha program, and it has been approved. I have not even heard yet that it's been actually launched. I would imagine it will take some time to get onto formularies, which is the real driver for sales, because it's really the outpatient, it's the cost of the patient that dictates sales on the market. And there's no question that the cost of goods for Loom 201 will be much less than the cost of goods for Skytrofa, which does provide quite good flexibility.
spk17: Got it. Okay. Yeah, I think Maybe what I was trying to get at is comfort in general with a weekly. Have you heard anybody who might be a little bit hesitant to prescribe weekly GH? Or is IGF-1 levels not a concern for some KOLs?
spk05: David, if you want to address that at all?
spk10: Sure.
spk09: I think that most of the key opinion leaders that I talked with when I was heading the development of Ascendus did not have a big concern about IGF-1 levels because traditionally, because kids with growth hormone deficiency are rarely diagnosed right when they develop it, there's a lag time to diagnosis and treatment. You don't want to target kind of the average IGF-1 value in these kids, but if you've got five years to treat them to adult height. So you want to tend to be in the upper half of the normal range. And there are data showing that if you target a positive two, you get better growth than if you target an IGF-1 STS of zero. And by definition, if you're targeting an IGF-1 STS of two, you're going to have half above, half below. So I don't think that it's a big concern. And there's no question that on everything else being equal, most kids and parents would prefer one injection a week compared to seven injections per week. But by the same token, it's not at all unlikely that a lot of people would prefer a once daily oral compound to even a once weekly injection.
spk05: And, Catherine, I think that as pediatric endocrinologists, many of them will wait and see, I believe, to see what the patient population reports or their colleagues report over time. I think there's going to be some early adopters, but I also think there will be some pediatric endocrinologists who will not jump in right away, perhaps because of the excursions of IGF-1 levels that sometimes can be of concern to some clinicians.
spk17: Got it. Thanks very much.
spk04: Thank you, Catherine.
spk15: Thank you. I am showing no further questions in the queue at this time. Thank you for joining us and enjoy your afternoon.
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