This conference call transcript was computer generated and almost certianly contains errors. This transcript is provided for information purposes only.EarningsCall, LLC makes no representation about the accuracy of the aforementioned transcript, and you are cautioned not to place undue reliance on the information provided by the transcript.
spk08: Good afternoon, and welcome to Lomas Parma 2023 fourth quarter and year-end conference call. Currently, all participants are on a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow that time. As a reminder, this conference call is being recorded. I will now turn the call over to Lisa Miller, Vice President of Investor Relations. Please go ahead.
spk09: Thank you, Operator. Before we proceed with this call, I'd 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 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-K, 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, Dr. Duke Patukchwanan, Chief Medical Officer, and Lori Lawley, our Chief Financial Officer. Following our prepared remarks, the management team will be available for a question and answer session. I will now turn the call over to Rick.
spk04: Thank you, Lisa, and good afternoon, everyone. After the market closed today, we issued a press release announcing our results for 2023. I'm pleased to report that 2023 was a successful year for Lumos, led by our announcement of positive top-line results from our Oral Growth 210 and Oral Growth 212 trials of LUM201 in pediatric growth hormone deficiency. On today's call, we'll briefly summarize our trial results and other progress made in 2020 to recap highlights for recent weeks and layout plans for 2024. Then we'll be happy to answer your questions. Before I begin, I'd like to take a moment to congratulate Dr. Peset Duke Pichakchewana on his recent promotion to the role of Chief Medical Officer for Lumos. Dr. Duke's exceptional credentials are widely acknowledged, given his renowned expertise in growth disorders and his current leadership at the Human Growth Foundation, among various other distinguished roles in his esteemed career. Since joining us in 2022, Dr. Duke played a pivotal role in steering our oral growth trials to fruition, providing invaluable guidance, leveraging his influential network for enrollment, and significantly contributing to analysis of trial data. As our new CMO, Dr. Duke will continue to advocate for exploration of LUM201 within the PGSD clinical community, a mission that has already garnered remarkable interest from pediatric endocrinologists globally, thanks to his advocacy efforts. Furthermore, Dr. Duke will be an integral part of shaping the trial design for a Pivotal Loom 201 trial in PGHD, details of which we're going to delve into later during this call. Dr. Duke will be joining the call today for our Q&A session. So please join me in congratulating him on his well-deserved promotion. Let's now revisit the key highlights of the results we disclosed last November. As many of you are aware, Loom 201 has successfully met all primary and secondary endpoints in both Wargrove trials, demonstrating clear proof of concept for its efficacy as an oral alternative to daily and weekly injectables for patients with PGHD. Results from Wargrove 210 demonstrated that the 1.6 mg per kg dose of Loom 201 achieved annualized high velocities, or AHVs, of 8.2 centimeters a year at six months, and 8 centimeters a year at 12 months, aligning consistently with historical growth rates for the moderate PGSD population. The difference in AHV at 6 and 12 months between the optimal 1.6 mg per kg LUM201 dose and the recombinant growth hormone comparator arm fell within the non-inferiority margin of less than 2 centimeters a year, and that's a criterion recently used for FDA approvals. Although oral growth 210 was not specifically designed to establish non-inferiority, we are pleased to note that the growth outcomes observed in this study are in line with the non-inferiority thresholds seen in recent successful FDA applications. These findings enable us to design a successful phase 3 trial to show non-inferiority against the daily injectable growth hormone comparator arms. Now, the preliminary 24 months of the 201 data we presented in November were gathered from a subset of the ORAGO 210 patient or participants who met the protocol requirements for an extension beyond 12 months combined with ORAGO 212 subjects. These data revealed a sustained effect showing a minimal decrease of approximately 6% in annualized high velocity from year one to year two. as opposed to the significant decline of approximately 20% reported for daily recombinant growth hormone in moderate PGHD patients. Now, OrgGrowth210 successfully achieved its pre-specified primary endpoint, validating our predictive enrichment marker or PEM test, while also meeting the secondary endpoint by demonstrating 100% reproducibility of PEM-positive classifications. Additional data from the oral growth 212 trial demonstrated that only 20% growth hormone concentration was needed to achieve comparable annualized height velocity. That suggests that LUM201 is more efficient than exogenous growth hormone to promote growth. These data confirm the importance of LUM201's unique mechanism of action to restore the natural physiology of pulsatile growth hormone secretion. Our data from both trials provided a clear safety profile for investigation on LUM201. Now, following the release of top-line results in November, ongoing data collection and analysis have continued. We anticipate presenting comprehensive 12-month annualized high-velocity data from the oral growth 210 trial in the second quarter of this year, most likely at a major medical conference. This data set will include 12-month AHV data on all enrolled patients from the Oral Growth 210 trial. Additional 24-month data beyond the data set we announced in November will also be available. We expect to present these data in additional analysis at several meetings throughout 2024 to capitalize on the growing interest and excitement in the pediatric endocrine community about LUM201 as potentially the first oral therapeutic in this population. And following the announcement of our top line results, we've been diligently conducting additional analyses in preparation for end of phase two meeting with the FDA, and that's scheduled for the second quarter. Our data package for this meeting is comprehensive, featuring a larger data set compared to other growth hormone counterparts at this developmental stage. These data show LUM201 induces annualized height velocities consistent with historical levels for moderate PGHD on recombinant growth hormone. Data also demonstrate that oral LUM201 produces a robust and enduring response at the 1.6 mg per kg dose, making use of our predictive enrichment marker or PEM strategy for selecting suitable moderate PGHD patients, thereby further de-risking our Phase III program As a result, we approached this end-of-Phase II meeting with a high level of confidence. Now, long before we announced top-line results from our Argrove trials, we've been actively involved in advanced planning for a pivotal Phase III trial. Following our Phase II meeting with the FDA, we will finalize the ultimate design of the trial, which we anticipate initiating in the fourth quarter of 2024. We believe that this trial's results will support a new drug application for Loom 201 and the PGSD indication. With that, I'm going to turn it over to Lori Lawley, our CFO, for a brief overview of our financial results.
spk11: Thanks, Rick. Loom on Pharma ended the year of December 31, 2023, with cash, cash equivalents, and short-term investments totaling $36.1 million, compared to $67.4 million on December 31, 2022. Cash on hand as of December 31, 2023, is expected to support operations through the third quarter of 2024, inclusive of our Phase III preparations and other operational activities. Initiation of our Phase III clinical trial by end of 2024 is subject to securing financing in the near term. Research and development expenses were $22.1 million, an increase of $4.2 for the year ended December 31st, 2023, compared to the same period in 2022, primarily due to increases of 3.3 million in clinical trial expenses, 0.9 million in contract manufacturing expenses, 0.2 million in consulting expenses, and 0.2 million in other expenses, partially offset by a 0.4 million dollar decrease in personnel related expenses. General and administrative expenses were 16.6 million, An increase of $0.9 million for the year ended December 31, 2023, compared to the same period in 2022, primarily due to increases of $0.5 million in personnel-related expenses, $0.4 million in royalty expenses paid to the Public Health Agency of Canada, and $0.1 million in travel expenses, partially offset by a $0.1 million decrease in other expenses. The net loss for the year ended December 31, 2023, with $34 million, compared to a net loss of $31.1 million for the same period in 2022. We end at Q4 2023 with 8,102,555 shares outstanding. And with that, I will turn it back to Rick for his closing remarks.
spk04: Thanks, Lori. And to conclude, we're absolutely thrilled by the progress of our program to date. and by the excitement among the endocrine community regarding the potential for the first oral therapy for PGHD. We've been on a podium presenting data on numerous endocrine conferences over the past two years and have seen the overwhelming acceptance of and the excitement for oral LUM201. We're confident in the distinct advantages offered by LUM201 for patients with moderate PGHD and enthusiastic about advancing to the next developmental stage for this asset. We firmly believe that the dataset we are submitting to the FDA for upcoming end-of-Phase II meeting provides ample support for a pivotal trial of LUM201 at the 1.6 mixed-pricate dose, affirming its potential as a viable alternative to injectable products for appropriately selected moderate PGHD patients. And we eagerly anticipate sharing further insights from our oral growth trials throughout this year and keeping you abreast of our progress. So thank you for your attention, everyone. And operator, we can now open this up for questions.
spk08: Thank you. Ladies and gentlemen, to ask a question, you will need to press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, simply press star 1-1 again. Please stand by while we compile the Q&A roster. Now, first question, coming from the lineup, Charles Duncan with Cantor. Your line is open.
spk06: Hey, good afternoon, Rick and team. Congrats on the progress last year. Looking forward to hearing more updates here in the near term. Thanks for taking our question. I had a question about the end of Phase 2 meeting with agency. Is that actually scheduled, and can you provide us some insight on what the key question is. Is it really something that needs to be answered or do you feel like this is more a point of execution in terms of going forward? And then when would you anticipate being able to share the outcome? Would it be post the meeting minutes or do you think it'll be pretty straightforward and you'll share it shortly after the meeting?
spk04: I'm going to ask a good question, Charles, and thank you for it. I'm going to turn that question over to John McHugh.
spk07: Yeah, thanks, Charles. I appreciate the question. So, you know, before we start our Phase 3 study, we really would like to use this end of Phase 2 meeting to take everything we've learned, you know, from Phase 2 data that we've read out We put that into our phase three protocol and we want to walk away from that meeting with agreement on all the specifics of that protocol. So I think it's a very important time for us to talk through any questions the agency has on the data as well as come to agreement on the path to move forward. So it is, it will be a very, it'll be a good step forward for us when we get through that and we have agreement on the phase three protocol. So we expect to communicate that information, the outcomes of that when we hear back from the FDA, probably in the meeting minutes, right? So we've said that that timeframe is in Q2 of this year.
spk04: Okay. And, you know, Charles, I might add is that, you know, there are a lot of historical precedents here. We feel confident about this meeting and really an outstanding briefing book I think historically these studies have been 12-month non-inferiority trials against an active comparator. The non-inferiority margin historically has been 1.8 to 2 centimeters, and we also expect to have a randomization to the 1.6 mg per kg a day, a 2 to 1 randomization to growth hormone. I think most of these studies have been about 200 subjects. We expect a similar design. And also, of course, we validated our PEMS strategy. I think we de-risk our program pretty considerably, given the fact that we can select patients we believe our drug will be effective in, and that is the moderate growth hormone deficient patient. So, we're pretty confident about our meeting with the FDA.
spk06: It's helpful if I could just ask a follow-on question to that, those two observations. Rick, would you anticipate the PEM strategy to help you design and conduct a smaller sample size or conduct one about that size with, you know, perhaps greater confidence in the signal-to-noise?
spk15: John, do you want to go there? Yeah, so I think the...
spk07: you know, how we view the pen test is it's going to kind of raise our efficiency, right? We're going to be able to bring in subjects that are likely responders to our molecule. So I don't think it's going to reduce that size. It'll make it a more efficient trial.
spk06: Okay. Last question in terms of business development activity. At one time you had talked about possibly XUS partnering. I guess I'm wondering if you have any further thoughts on that, and then I'll hop back in the queue. Thanks.
spk04: Yeah, thanks for that question, Charles. And sure, I think that you can imagine as the first oral product in this very large global market, there are certain ex-U.S. regional markets and companies that have completed an outreach to us or vice versa. And we continue with those discussions and at the appropriate time. I think that we will partner with anyone who we believe is going to be a good partner in those markets. Obviously, this will allow us to bring in some non-dilutive money, and I think that's one of the goals that we would have for ourselves.
spk06: Okay. Thanks for taking the questions, Rick.
spk08: Thank you. And our next question coming from the line of Leland Groeschel with Oppenheimer. Leland, it's open.
spk03: Thank you, Rick and team, for the update. Just a question from me with respect to additional indication potential for 201 as we think about, you know, use of growth hormone products today and indications such as turners and other such disorders. Just wondering if you could comment on any plans down the road to explore 201's potential to be approved for those additional label indications. Thank you.
spk04: Yeah, and John, I'm going to let you start with that as an answer, and thank you for the question, Leland.
spk07: So, we've spent a lot of time, Leland, you know, thinking through the best opportunities to advance loom 201 and pass PGHD. PGHD is a great opportunity, I think, for us, to show the effect of the molecule on restoring natural pulsatility. And we can take that same mechanism and philosophy and apply it to many of the other 11 indications that are approved for injectable growth hormone. So I think we're getting to the point where we have the data set where we can start to think about how to move forward, but we have not yet made any decisions on the exact next step that we're going to take. But we have, I think, the data and have done the background research on each one of those indications to make some good decisions shortly.
spk04: And Leland also recalled we've got a pilot program underway in a broader cardiometabolic opportunity of non-alcoholic fatty liver disease at MGH. Now, it's an investigator-initiated trial. But this investigator did demonstrate in a recombinant growth hormone trial in this indication some pretty significant reductions in liver fat in this population. So we're pretty excited to work with this investigator. I think it's a little premature right now to talk about anything beyond that, but we're always looking for ways to increase shareholder value with additional indications.
spk03: Got it. Great. So at this point, we don't have a view on when she may have data on her study of 201, correct?
spk04: No. She doesn't have the trial fully enrolled yet, and it's a six-month trial. So we haven't really talked about that yet in the marketplace.
spk03: Okay. Well, stay tuned and look forward to hearing updates following your FDA meeting. Thank you. Okay.
spk08: Thank you. And our next question, coming from the lineup, Ed White with HC1. Radio line is open.
spk16: Hi. Thanks for taking my questions. So how should we be thinking about the phase three start in the fourth quarter? Is that just when you'll be opening sites, or do you intend or do you think that you can actually treat patients in during the fourth quarter.
spk04: Okay. Thank you for that question. Ed and Duke, will you answer that for us?
spk15: I think you're on mute.
spk10: Thank you. Yeah. Sorry about that. Thank you. That was a very good question, right? As of now, we do diligently, like as John mentioned, right? We already have a plan for the phase three plan. We get ready to really discuss with FDA what we have planned. In the meantime, we do a prep work, what we need to get done in regards to have a timeline that will be proposed, right? So we think that by the end of this year, we'll be able to screen the subject and get, you know, the timeline enrolled like what we proposed in the past. And I feel very optimistic because as Rick mentioned, earlier that we have overwhelming requests from the KOL around the world because they learn about other Phase II results and they do believe that this can be a great, potentially, the new drug therapy for their patient, the first oral therapy. So we do believe that, you know, a lot of investigators who actually, some of those participate in other Phase II trials and some new to LUM201. However, you know, with the request of participation, We do believe the timeline, when we get this survey up and running and we have a final, you know, site identified, I think that the rolling can go very quickly and as we proposed in the plan in the past.
spk04: And, you know, obviously we have to finalize the phase three protocol. We're obviously working with our vendor partners very actively to We've been active on the podium at all these endocrine meetings around the world, and we've contacted and direct contact with really experienced investigators as a result. There's a lot more work that has to be done in terms of qualifying and activating the sites. And of course, we have our phase three drug product manufactured and ready to go, so we will have it ready to go. So, all the preparation that needs to be done, we're doing right now, and we hope to have, you know, plenty of patients screened in the study before the end of the year.
spk02: Okay. Thanks, Rick, for taking my question. Sure.
spk08: Thank you. And our next question, coming from the lineup, Katherine Novak with Jones Research. Your line is open.
spk13: Oh, hi. Good afternoon, guys. Just one question. Can you remind us of what percent of the oral growth patients enrolled were boys versus girls? And there is a difference between how these two populations might be managed by pediatric endocrinologists, you know, a sense that there could potentially encourage patients who might not choose to have daily injections try an oral growth hormone therapy instead.
spk04: Duke, why don't you answer that question? Thanks. Appreciate the question, Catherine.
spk10: Yeah, thank you, Catherine. That's a very good question. Our trial is very, you know, kind of like unique, right? As you know, when you look at most of phase three trial, especially long acting, as in this novel Pfizer, majority of those subject that enroll in a trial are male predominant, right? In our study, it's pretty much that, you know, male a little bit more than female, but not that huge difference, which is very, very interesting. I think when we go back to the first global phase three trial, you can see that the number of countries are going to increase, the number of sites are going to increase, because the timeline we want to enroll, we have a goal that we want to complete enrollment within 15 to 18 months, which is, again, this goal is actually better than the goal that those previous phase three trials have been running. Part of it is that that's still in COVID. And now we don't have COVID. And not to mention that we do not expect a lot of competitor to run the phase three trial, right? And in regards to, you know, the oral, I think that to enroll these subjects, I heard loud and clear. When they start the phase two, try to increase enrollment and autonomy manner. We realized that a lot of subjects really interested to participate. in this oral room 12-1 trial. Again, you know, I don't think that we expect any issue of, you know, get the patient enrolled, especially most of them would like to be able, you know, to be on this drug, and especially during the clinical trial, because it would take years for us to really get the drug approved. I hope that I answered all the questions you had.
spk13: Yeah, that's very helpful. And actually, just one more on the end of phase two meeting. You know, we've heard over and over from care wells that growth beyond 12 months is really the most important aspect to them. You know, are these data going to be a part of your discussion with FDA? And how important is that to the phase three child design?
spk04: Boy, that is a very good question, Catherine. And I'm going to ask John to answer it.
spk07: Yeah, thank you, Catherine. So I think the data that we've shown about the small subset of kids that we have, you know, at 24 months on growth has been very telling. You know, the restoration of normal growth hormone pulsatility, normal IGF-1 levels, and then, you know, more natural growth that results from that has led to very consistent and durable growth comparing year two to year one, right? We lose only a small, tiny bit, 6% of our HV compared to a much larger drop-off that you see with daily injectable growth hormone. So that is a really important piece of data that we certainly will chat with the FDA about. We do anticipate, you know, much as Rick said earlier, a standard phase three trial design of 12 months. But because we have a number of subjects already that have gone past that 12-month timeframe, we're moving subjects from our 24-month-long ORAGROW-210 study into a long-term safety extension. We will have quite a lot of longer-term data by the time we get to phase three and get to an NDA. So I think that data will be very helpful in addition to what we see in the phase three 12-month study that Rick described. So it is very important, and it will be a very nice package of durable data that we can bring.
spk13: Great. That's very helpful.
spk14: Thanks a lot, guys.
spk08: Thank you. And I'm showing no further questions in queue at this time. And ladies and gentlemen, this concludes the LUMOS Farm Offboard Quarter 2023 earnings call. Thank you and have a good day. you you Thank you. Thank you. Thank you. Good afternoon, and welcome to Lomas Parma 2023 fourth quarter and year-end conference call. Currently, all participants are on a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow that time. As a reminder, this conference call is being recorded. I will now turn the call over to Lisa Miller, Vice President of Investor Relations. Please go ahead.
spk09: Thank you, Operator. Before we proceed with this call, I'd 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 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-K, 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, Dr. Duke Patukchwanan, Chief Medical Officer, and Lori Lawley, our Chief Financial Officer. Following our prepared remarks, the management team will be available for a question and answer session. I will now turn the call over to Rick.
spk04: Thank you, Lisa, and good afternoon, everyone. After the market closed today, we issued a press release announcing our results for 2023. I'm pleased to report that 2023 was a successful year for Lumos, led by our announcement of positive top-line results from our Oral Growth 210 and Oral Growth 212 trials of LUM201 in pediatric growth hormone deficiency. On today's call, we'll briefly summarize our trial results and other progress made in 2020 to recap highlights for recent weeks and layout plans for 2024. Then we'll be happy to answer your questions. Before I begin, I'd like to take a moment to congratulate Dr. Paset Duke Pichakchewanan on his recent promotion to the role of Chief Medical Officer for Lumos. Dr. Duke's exceptional credentials are widely acknowledged, given his renowned expertise in growth disorders and his current leadership at the Human Growth Foundation, among various other distinguished roles in his esteemed career. Since joining us in 2022, Dr. Duke played a pivotal role in steering our oral growth trials to fruition, providing invaluable guidance, leveraging his influential network for enrollment, and significantly contributing to analysis of trial data. As our new CMO, Dr. Duke will continue to advocate for exploration of LUM201 within the PGSD clinical community, a mission that has already garnered remarkable interest from pediatric endocrinologists globally, thanks to his advocacy efforts. Furthermore, Dr. Duke will be an integral part of shaping the trial design for a Pivotal Loom 201 trial in PGHD, details of which we're going to delve into later during this call. Dr. Duke will be joining the call today for our Q&A session. So please join me in congratulating him on his well-deserved promotion. Let's now revisit the key highlights of the results we disclosed last November. As many of you are aware, Loom 201 has successfully met all primary and secondary endpoints in both Wargrove trials, demonstrating clear proof of concept for its efficacy as an oral alternative to daily and weekly injectables for patients with PGHD. Results from Wargrove 210 demonstrated that the 1.6 mg per kg dose of Loom 201 achieved annualized high velocities, or AHVs, of 8.2 centimeters a year at six months, and 8 centimeters a year at 12 months, aligning consistently with historical growth rates for the moderate PGSD population. The difference in AHV at 6 and 12 months between the optimal 1.6 mg per kg LUM201 dose and the recombinant growth hormone comparator arm fell within the non-inferiority margin of less than 2 centimeters a year, and that's a criterion recently used for FDA approvals. Although oral growth 210 was not specifically designed to establish non-inferiority, we are pleased to note that the growth outcomes observed in this study are in line with the non-inferiority thresholds seen in recent successful FDA applications. These findings enable us to design a successful phase 3 trial to show non-inferiority against the daily injectable growth hormone comparator arms. Now, the preliminary 24 months of the 201 data we presented in November were gathered from a subset of the ORAGRO 210 participants who met the protocol requirements for an extension beyond 12 months combined with ORAGRO 212 subjects. These data revealed a sustained effect showing a minimal decrease of approximately 6% in annualized height velocity from year one to year two. as opposed to the significant decline of approximately 20 percent reported for daily recombinant growth hormone in moderate PGHD patients. Now, OrgGrowth210 successfully achieved its pre-specified primary endpoint, validating our predictive enrichment marker, or PEM test, while also meeting the secondary endpoint by demonstrating 100 percent reproducibility of PEM-positive classification. Additional data from the oral growth 212 trial demonstrated that only 20% growth hormone concentration was needed to achieve comparable annualized height velocity. That suggests that LUM201 is more efficient than exogenous growth hormone to promote growth. These data confirm the importance of LUM201's unique mechanism of action to restore the natural physiology of pulsatile growth hormone secretion. Our data from both trials provided a clear safety profile for investigational LUM201. Now, following the release of top-line results in November, ongoing data collection and analysis have continued. We anticipate presenting comprehensive 12-month annualized high-velocity data from the Oral Growth 210 trial in the second quarter of this year, most likely at a major medical conference. This data set will include 12-month AHV data on all enrolled patients from the Oral Growth 210 trial. Additional 24-month data beyond the data set we announced in November will also be available. We expect to present these data in additional analysis at several meetings throughout 2024 to capitalize on the growing interest and excitement in the pediatric endocrine community about LUM201 as potentially the first oral therapeutic in this population. And following the announcement of our top line results, we've been diligently conducting additional analyses in preparation for end of phase two meeting with the FDA, and that's scheduled for the second quarter. Our data package for this meeting is comprehensive featuring a larger data set compared to other growth hormone counterparts at this developmental stage. These data show LUM201 induces annualized high velocities consistent with historical levels for moderate PGHD on recombinant growth hormone. Data also demonstrate that oral LUM201 produces a robust and enduring response at the 1.6 mg per kg dose making use of our predictive enrichment marker or PIM strategy for selecting suitable moderate PGHD patients, thereby further de-risking our Phase III program. As a result, we approached this end-of-Phase II meeting with a high level of confidence. Now, long before we announced top-line results from our oral growth trials, we've been actively involved in advanced planning for a pivotal Phase III trial. Following our Phase II meeting with the FDA, we will finalize the ultimate design of the trial, which we anticipate initiating in the fourth quarter of 2024. We believe that this trial's results will support a new drug application for LUM201 in the PGSD indication. With that, I'm going to turn it over to Lori Lawley, our CFO, for a brief overview of our financial results.
spk11: Thanks, Rick. Lumos Pharma ended the year of December 31, 2023, with cash, cash equivalents, and short-term investments totaling $36.1 million compared to $67.4 million on December 31, 2022. Cash on hand as of December 31, 2023, is expected to support operations through the third quarter of 2024, inclusive of our Phase III preparations and other operational activities. Initiation of our Phase III clinical trial by end of 2024 is subject to securing financing in the near term. Research and development expenses were $22.1 million, an increase of $4.2 million for the year ended December 31, 2023, compared to the same period in 2022, primarily due to increases of $3.3 million in clinical trial expenses, $0.9 million in contract manufacturing expenses, 0.2 million in consulting expenses, and 0.2 million in other expenses, partially offset by a 0.4 million dollar decrease in personnel related expenses. General and administrative expenses were 16.6 million, an increase of 0.9 million for the year ended December 31st, 2023, compared to the same period in 2022, primarily due to increases of 0.5 million in personnel related expenses, $0.4 million in royalty expenses paid to the Public Health Agency of Canada, and $0.1 million in travel expenses, partially offset by a $0.1 million decrease in other expenses. The net loss for the year ended December 31, 2023, with $34 million, compared to a net loss of $31.1 million for the same period in 2022. We end at Q4 2023 with 8,102,555 shares outstanding. And with that, I will turn it back to Rick for his closing remarks.
spk04: Thanks, Lori. And to conclude, we're absolutely thrilled by the progress of our program to date and by the excitement among the endocrine community regarding the potential for the first oral therapy for PGHD. We've been on a podium presenting data on numerous endocrine conferences over the past two years and have seen the overwhelming acceptance of and the excitement for oral LUM201. confident in the distinct advantages offered by Loom 201 for patients with moderate PGHD and enthusiastic about advancing to the next developmental stage for this asset. We firmly believe that the data set we are submitting to the FDA for upcoming end-of-Phase II meeting provides ample support for a pivotal trial of Loom 201 at the 1.6 mixed-pricate dose, affirming its potential as a viable alternative to injectable products for appropriately selected moderate PGSD patients. And we eagerly anticipate sharing further insights from our oral growth trials throughout this year and keeping you abreast of our progress. So thank you for your attention, everyone. And operator, we can now open this up for questions.
spk08: Thank you. Ladies and gentlemen, to ask a question, you will need to press star 1-1 on your telephone and wait for your name to be announced. to enjoy a question, simply press star one, one again. Please stand by while we compile the Q&A roster. Now, first question coming from the lineup, Charles Duncan with Kantor. Your line is open.
spk06: Hey, good afternoon, Rick and team. Congrats on the progress last year. Looking forward to hearing more updates here in the near term. Thanks for taking our questions. I had a key question about the end of Phase 2 meeting with agency. Is that actually scheduled and can you provide us some insight on what the key question is? Is it really something that needs to be answered or do you feel like this is more a point of execution in terms of going forward? And then when would you anticipate being able to share the outcome? Would it be post the meeting minutes, or do you think it'll be pretty straightforward and you'll share it shortly after the meeting?
spk04: I'm going to ask a good question, Charles, and thank you for it. I'm going to turn that question over to John McHugh.
spk07: Yeah, thanks, Charles. I appreciate the question. So, you know, before we start our phase three study, we really would like to use this end of phase two meeting to take everything we've learned from phase two data that we've read out. We put that into our phase three protocol and we want to walk away from that meeting with agreement on all the specifics of that protocol. So I think it's a very important time for us to talk through any questions the agency has on the data. as well as come to agreement on the path to move forward. So it will be a good step forward for us when we get through that and we have agreement on the phase three protocol. So we expect to communicate that information, the outcomes of that when we hear back from the FDA, probably in the meeting minutes, right? So we've said that that timeframe is in Q2 of this year.
spk04: Okay. And, you know, Charles, I might add is that, you know, there are a lot of historical precedents here. We feel confident about this meeting and really an outstanding briefing book. I think that historically these studies have been, you know, 12-month non-inferiority trials against an active, you know, comparator. You know, the non-inferiority margin historically has been 1.8 to 2 centimeters in And, you know, we also, you know, expect to have a randomization to the 1.6 mg per kg a day, you know, a two-to-one randomization to growth hormone. I think most of these studies have been about 200 subjects. We expect, you know, a similar design. And also, of course, you know, we validated our PEM strategy. I think we de-risk our program pretty considerably, given the fact that we can select patients we believe will be, our drug will be effective in, and that is the moderate growth hormone deficient patient. So, we're pretty confident about our meeting with the, our community meeting with the FDA.
spk06: It's helpful if I could just ask a follow-on question to that, those two observations. Rick, would you anticipate the PEM strategy to help you design and conduct a smaller sample size or conduct one about that size with, you know, perhaps greater confidence in the signal-to-noise?
spk15: John, you want to go there? Yeah.
spk07: So I think the, you know, how we view the pen test is it's going to kind of raise our efficiency, right? We're going to be able to bring in subjects that are likely responders to our molecule. So I don't think it's going to reduce that size. It'll make it a more efficient trial.
spk06: Okay. Last question in terms of business development activity. At one time you had talked about possibly XUS, partnering. I guess I'm wondering if you have any further thoughts on that, and then I'll hop back in the queue.
spk04: Thanks. Yeah, thanks for that question, Charles, and Sure. I think that you can imagine as the first oral product in this very large global market, there are certain U.S. regional markets and companies that have completed an outreach to us or vice versa. And we continue with those discussions and at the appropriate time. I think that we will partner with anyone who we believe is going to be a good partner in those markets. Obviously, this will allow us to bring in some non-dilutive money, and I think that's one of the goals that we would have for ourselves.
spk06: Okay. Thanks for taking the questions, Rick.
spk08: Thank you. And our next question coming from the line of Leland Groeschel with Oppenheimer. Leland, it's open.
spk03: Thank you, Rick, and team for the update. Just a question from me with respect to additional indication potential for 201 as we think about, you know, use of growth hormone products today and indications such as turners and other such disorders. Just wondering if you could comment on any plans down the road to explore 201's potential to be approved for those additional label indications. Thank you.
spk04: Yeah, and John, I'm going to let you start with that as an answer, and thank you for the question, Leland.
spk07: We've spent a lot of time, Leland, you know, thinking through the best opportunities to advance loom 201 and pass PGHD. PGHD is a great opportunity, I think, for us to to show the effect of the molecule on restoring natural pulsatility. And we can take that same mechanism and philosophy and apply it to many of the other 11 indications that are approved for injectable growth hormone. So I think we're getting to the point where we have the data set where we can start to think about how to move forward, but we have not yet made any decisions on the exact next step that we're going to take. But we have, I think, the data and have done the background research on each one of those indications to make some good decisions shortly.
spk04: And Leland also recalled we've got a pilot program underway in a broader cardiometabolic opportunity of non-alcoholic fatty liver disease at MGH. Now, it's an investigator-initiated trial. But this investigator did demonstrate in a recombinant growth hormone trial in this indication some pretty significant reductions in liver fat in this population. So we're pretty excited to work with this investigator. I think it's a little premature right now to talk about anything beyond that, but we're always looking for ways to increase shareholder value with additional indications.
spk03: Got it. Great. So at this point, we don't have a view on when she may have data on her study of 201, correct?
spk04: No. She doesn't have the trial fully enrolled yet, and it's a six-month trial. So we haven't really talked about that yet in the marketplace.
spk03: Okay. Well, stay tuned and look forward to hearing updates following your FDA meeting. Thank you. Okay.
spk08: Thank you. And our next question, coming from the lineup, Ed White with HC, when is open?
spk16: Hi, thanks for taking my questions. So how should we be thinking about the phase three start in the fourth quarter? Is that just when you'll be opening sites, or do you intend or do you think that you can actually treat patients in during the fourth quarter.
spk04: Okay. Thank you for that question, Ed. And, Duke, will you answer that for us?
spk15: I think you're on mute.
spk10: Thank you. Yeah. Sorry about that. Thank you. That was a very good question, right. As of now, we do diligence, like as John mentioned, right. We already have a plan for the phase three plan. We get ready to really discuss with FDA what we have planned. In the meantime, we do a prep work, what we need to get done in regards to have a timeline that will be proposed, right? So we think that by the end of this year, we'll be able to screen the subject and get, you know, the timeline enrolled like what we proposed in the past. And I feel very optimistic because as Rick mentioned, earlier that we have overwhelming requests from the KOL around the world because they learn about other phase two results and they do believe that this can be a great potentially the new drug therapy for their patient the first oral therapy so we do believe that you know a lot investigator who actually some of those participate in our phase two trial and some news to loom 201 however you know with the request of participation We do believe the timeline, when we get this survey up and running and we have a final site identified, I think that the rolling can go very quickly and as we proposed in the plan in the past.
spk04: And obviously, we have to finalize the phase three protocol. We're obviously working with our vendor partners very actively. We've been active on the podium at all these endocrine meetings around the world, and we've contacted and direct contact with really experienced investigators as a result. There's a lot more work that has to be done in terms of qualifying and activating the sites. And of course, we have our phase three drug product manufactured and ready to go, so we will have it ready to go. So, all the preparation that needs to be done, we're doing right now, and we hope to have, you know, plenty of patients screened in the study before the end of the year.
spk02: Okay. Thanks, Rick, for taking my question. Sure.
spk08: Thank you. And our next question, coming from the lineup, Katherine Novak with Jones Research. Your line is open.
spk13: Oh, hi. Good afternoon, guys. Just one question. Can you remind us of what percent of the oral growth patients enrolled were boys versus girls? And there's a difference between how these two populations might be managed by pediatric endocrinologists, you know, a sense that there could potentially encourage patients who might not choose to have a daily injection try an oral growth hormone therapy instead.
spk04: Duke, why don't you answer that question? Thanks. Appreciate the question, Catherine.
spk10: Yeah, thank you, Catherine. That's a very good question. All the trials vary, you know, kind of like unique, right? As you know, when you look at most of phase three trials, especially long-acting, as in this novel Pfizer, majority of those subjects that enroll in a trial are male predominant, right? In our study, it's pretty much that, you know, male a little bit more than female, but not that huge difference, which is very, very interesting. I think when we go back to the first global phase three trial, you can see that, you know, the number of countries going to increase, the number of sites going to increase because, you know, the timeline we want to enroll, we have a goal that, you know, we want to complete enrollment within 15 to 18 months, which is, again, this goal is actually, you know, better than the goal that those previous phase three trials have been running. Part of it is that, you know, that's dealing COVID. And now we don't have COVID. And not to mention that we do not expect a lot of competitor to run the phase three trial, right? And in regards to, you know, the oral, I think that to enroll these subjects, I heard loud and clear. When they start the phase two, try to increase enrollment and autonomy manner. We realized that a lot of subjects really interested to participate. in this oral room to one trial. Again, you know, I don't think that we expect any issue of, you know, get the patient enrolled, especially most of them would like to be able, you know, to be on this drug, and especially during the clinical trial, because it would take years for us to really get the drug approved. I hope that I answered all the questions you had.
spk13: Yeah, that's very helpful. And actually, just one more on the end of phase two meeting. You know, we've heard over and over from care wells that growth beyond 12 months is really the most important aspect to them. You know, are these data going to be a part of your discussion with FDA? And how important is that to the phase three child design?
spk04: Boy, that is a very good question, Catherine. And I'm going to ask John to answer it.
spk07: Yeah, thank you, Catherine. So I think the data that we've shown about the small subset of kids that we have, you know, at 24 months on growth has been very telling. You know, the restoration of normal growth, normal pulsatility, normal IGF-1 levels, and then, you know, more natural growth that results from that has led to very consistent and durable growth comparing year two to year one right we lose only a small tiny bit six percent of our hv compared to a much larger drop-off that you see with daily injectable growth hormone so that is a really important piece of data that we certainly will chat with the fda about we do anticipate you know much as rick said earlier a standard phase three trial design of 12 months. But because we have a number of subjects already that have gone past that 12-month timeframe, we're moving subjects from our 24-month-long ORAGRA 210 study into a long-term safety extension. We will have quite a lot of longer-term data by the time we get to phase three and get to an NDA. So I think that data will be very helpful in addition to what we see in the phase three 12-month study that Rick described. So it is very important, and it will be a very nice package of durable data that we can bring.
spk13: Great. That's very helpful.
spk14: Thanks a lot, guys.
spk08: Thank you. And I'm showing no further questions in queue at this time. And ladies and gentlemen, this concludes the LUMOS Farm Offboard Quarter 2023 Earnings Call. Thank you and have a good day.
Disclaimer