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spk10: Good afternoon and welcome to Lumos Pharma's fourth quarter 2021 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. Ma'am, please go ahead.
spk00: 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 filing 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 8-K, which may be accessed from the investors' page of the company's website. Joining today's call will be Rick Hawkins, CEO and Chairman, John McHugh, President and Chief Scientific Officer, Dr. David B. Karp, our Chief Medical Officer, and Lori Lawley, Chief Financial Officer. Following prepared remarks, we will open the call to questions. I will now turn the call over to Rick.
spk04: Thank you, Lisa, and 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 new plan for an interim analysis of two of our ongoing oral growth trials and our financial results for the full year 2021. On today's call, I will provide a clinical update before turning it over to Lori, who will review our financial results. Then John and David will join us for a Q&A session. I'll begin with the exciting news we announced today for our Oral Growth 210 and 212 trials. We're very pleased to report that screening and enrollment trends in both trials are sufficient for us to announce interim analyses of clinical and safety data, the results of which we expect to report by year-end 2022. This early look at the data will evaluate the safety and annualized height velocity on three dose levels of LUM201 against a standard dose of recombinant human growth hormone in 40 patients at six months on therapy. We look forward to providing this preliminary assessment of LUM201's potential to treat idiopathic PGHD patients who would otherwise face years of burdensome injections as their only course of treatment. Now, as a reminder, our Oral Growth 210 trial is our global clinical study evaluating oral LUM201 in approximately 80 patients diagnosed with idiopathic PGHD. The primary clinical outcome is annualized height velocity in patients selected by our predictive enrichment marker, or PEM, strategy. The key clinical endpoint is the comparison of the annualized height velocity of LUM201 at three dose levels, 0.8, 1.6, and 3.2 milligrams per kilogram per versus a control arm of patients treated with injectable recombinant growth hormone at a daily dose of 0.24 milligrams per kilogram per week. Dose levels of LUM201 were selected to span the entire dose response curve elucidated in a prior PK PD 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, as well as validate the PEM strategy. The Oral Growth 210 trial is nearing the 50% enrollment milestone, and essentially all of our trial sites, excluding Ukraine and Russia, are open for enrollment. Given the recent events in Ukraine and Russia, we have suspended all clinical activity at trial locations in Russia and are unable to enroll patients in Ukraine due to the ongoing conflict. We are, therefore, now targeting approximately 40 sites for our Oral Growth 210 trial. The enrollment and screening at these approximately 40 sites have maintained an encouraging trajectory, and, therefore, we continue to anticipate primary outcome data on 80 patients from oil growth 210 in the second half of 2023. The ongoing conflict may, however, adversely impact our business in the future, and it remains too early to evaluate all the potential effects of this crisis. Before moving on, I'd like to express our concerns and our support for our research colleagues and the people of Ukraine, as well as our sincere hope for their safety and rapid restoration of peace. So enrollment continues to meet our expectations in our Oral Growth 212 trial, 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 milligrams per kilogram per day. We're pleased to report that the minimum subject enrollment we set for the interim analyses will soon be met with 10 subjects, and we anticipate announcing the results of this interim analyses, evaluating safety and high-velocity data by the end of 2022. You'll recall that the objective of our 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 PGHD patients. The primary endpoint is six months of PKPD and high-velocity data, with a total of 12 months of high-velocity data to be captured in this trial. Turning now to our ORGO213 trial, or the exploratory SWITCH study. We have initiated an open-label, multi-center, Phase II study evaluating the growth effects and safety of LUM201 following 12 months of daily recombinant growth hormone in up to 20 PGHD patients who have completed the Oral Growth 210 trial. Subjects will be administered LUM201 at a dose level of 3.2 milligrams per kilogram per day for up to 12 months. Primary outcome for the 213 trial is annualized height velocity, and secondary outcomes include safety and other PD measures. In addition to advancing our clinical trials for LUM201 and PGHD, we continue to explore expansion opportunities for LUM201 and two other therapeutic areas where injectable recombinant human growth hormone is the standard of care. We believe that LUM201 is a pipeline and a product and through its unique mechanism of action, may have the potential to treat many of the indications comprising the $3.4 billion growth hormone market. I'll pass over to Lori for a review of our four-year 2021 financial results. Lori?
spk02: Thank you, Rick, and good afternoon, everyone. We ended our fiscal year on December 31, 2021, with cash and cash equivalent totaling $94.8 million. compared to our prior year cash balance of 98.7 million. We expect an average cash use of approximately eight and a half to nine and a half million per quarter through 2022. Cash on hand as of year end 2021 is expected to support our operations through the primary outcome data readout for both ORA Growth 210 and ORA Growth 212 trials. Research and development expenses were 16.2 million for the full year ending December 31st, 2021, This is an increase of $7 million over the R&D expenses of $9.2 million for the full year ending December 31, 2020. This increase was primarily due to increases of $5.5 million in clinical trial and contract manufacturing expenses and $2 million in personnel-related expenses. Our G&A expenses were $15.3 million for the full year ending December 31, 2021. This is a decrease of $1.9 million over the G&A expenses compared to the same period in 2020. A decrease is primarily due to decreases in legal and consulting expenses, which were higher in 2020 due to merger-related expenses. The net loss for year-ended December 31, 2021, was $30.4 million, compared to a net loss of $5.7 million for the same period in 2020. I will now turn it back to Rick for closing remarks.
spk04: Thank you, Lori. In closing, our fourth quarter and the start of 2022 have been productive for Lumos, and we are cautiously optimistic for the year ahead. Enrollment in our oral growth trials is progressing despite numerous challenges over the course of the prior year. We're now able to provide an early look at LUM201 efficacy in patients with idiopathic PGHD with interim data for both our 210 and 212 trials by the end of 2022. Based upon prior trials of growth hormone and PGHD, we believe these data from both Oral Growth 210 and Oral Growth 212 trials should be adequate to provide an initial indication of LUM201's impact on height velocity compared to growth hormone. Our Christ position is strong and sufficient to carry us through the primary outcome data readouts for both of these trials. So we look forward to updating you on our progress as the year unfolds. And operator, we're now ready to take questions.
spk10: Thank you. As a reminder, to ask a question, you will need to press star and then the number one on your telephone keypad. Again, just press star and then the number one on your telephone keypad. And to withdraw your question, just press the pound key. Please stand by while we compile the Q&A roster. Your first question comes from the line of Charles Duncan with Cantor Fitzgerald. Sir, you may proceed with your question.
spk08: Thank you. Good afternoon, Rick and team. Congratulations on a good year of progress, and thanks for the update on the clinical trials. So I had a couple of questions, specifically with regard to the aneurysms. I guess I'm wondering if those were discussed with the FDA or if that wasn't necessary since you're going to be looking on a blinded basis. And secondly, I'm wondering if you could provide us some additional color on the predictive enrichment markers in terms of the screen failure rate.
spk04: Okay, I'm going to let the first question, John McHugh, if you'd answer that, I'd appreciate it.
spk06: Sure, happy to, Rick, and nice to hear your voice again, Charles. So the 212 study is, as we said all along, is an open-label study, and we knew we were going to look at that data as time went on. For the 210 study, we've simply reached a time where it's an appropriate number of patients per cohort that we feel very comfortable taking a peek at that data and seeing how it may may play out when we compare to that same group of patients in the recombinant human growth hormone arm.
spk08: And will there be any kind of stopping rules, say, for futility or some certain amount of efficacy that you'd like to see to continue, or are you just taking a look and will be continuing to enroll? I guess you will have already enrolled quite a few more patients at that time anyway, right?
spk06: Yeah, that is correct. Obviously, the data are what the data are. We'll respond to them when we get to see them. But our plan is to fully enroll this trial and look at the data set when it rolls out. But I will say that the cohort size for 210 of about 10 patients for each cohort, and then the top two doses will have an additional four or five patients from the 212 study that we could combine. It's a fairly realistic-sized dataset for us to take a look at and really just get an initial look at how the efficacy is progressing within 201.
spk04: David, would you address the projected enrichment marker and the screen failure question that Charles had?
spk07: I'd be happy to. Charles, nice hearing you again. I'm glad you asked about that because we've worked very hard to educate the investigators on the optimal patients to screen for the study, and they are really coming through. As a result, we have a far, far lower screen failure rate based upon the PEM screening. We anticipate 40%, and I can't give you a precise number, but it's at least a fourfold lower screen failure rate based upon the PEM. So that's going very, very well. If I could just add a little bit extra to your first question, a more explicit answer would be that the interim analysis in the 210 study actually is in the current protocol that was submitted to the FDA. But we're only announcing it now because of the enrollment, we have confidence that we will have interim analysis by the end of the year.
spk08: Okay, that's helpful. Added color. I appreciate that, David. And then one quick follow-up, and then I'll hop back in the queue, and that is relative to the SWITCH study. I guess I'm not sure I fully understand it. Maybe I spaced something when you were talking about it. But in terms of the patients that will switch, those are patients who are on the 210 study and on the control arm, and then they will switch over to SWITCH. to the agent, or how will it exactly work? And then how come you're working with the highest dose?
spk04: That's right, Charles. But, David, if you go ahead and answer the rest of the question, go ahead.
spk07: Sure. Yeah, that's precisely right, Charles. So the eventual 20 subjects who will be in the 210 study on nortotropin for common human growth hormone, we'll have the opportunity to transfer into this early switch study so they can get 12 months treatment with LUM201. And we chose that dose because at the present time, seeing good tolerability across all the doses and knowing the dose response curve from the prior PKD-PD study, we thought it made sense to put them on the top dose. So that's why the design is what it is.
spk08: Okay, thank you for taking my questions.
spk10: Thank you. Your next question comes from the line of Yasmin Rahimi with Piper Sandler. Ma'am, please proceed with your question. Hi, team. Can you guys hear me?
spk04: Yes, we can.
spk11: Great. This is Jessie on for Yaz. I had a question about the interim analysis for 210. So what are you guys expecting to see in terms of height, velocity, and safety at interim? And I guess same question goes for 212 as well, because that's the same interim endpoint.
spk04: John, why don't you go ahead and start with that question, okay? Absolutely.
spk06: I think almost every Phase II study that's been run in this space with either growth hormone or long acting has generally run a comparator arm or recombinant human growth hormone alongside their exploratory cohorts. And we've done the same. And we've subset the population by using our enrichment marker to enrich in patients that can be responsive. And really the key thing here is now that we have a clinical cohort, study that's looking only at patients that we selected with our markers is really just to compare how our arms are doing compared to the concurrent or common human growth hormone arm. So that's really going to be a key piece of data that comes out of this. You know, and we'll work, well, that's one of the key things that we'll be looking at, right? We do have three doses of our molecule. We'd be, you know, very excited to see a dose response. We would predict there'd be a dose response somewhere across those three doses, and we'd like to see that as well. But really, the key is comparing and trying to get, trying to identify the dose that gives us, you know, comparable growth to the recombinant human growth hormone arm that's run concurrently.
spk11: Great. Thank you. And is that the same reason for the 212? Or what do you want to do with the 212?
spk06: Yeah, so for 212, you know, there's a lot of data that we're generating. The initial data that we're going to have the opportunity to look at is really just annualized height velocity. And since these kids are essentially the same patient population as the 210, it does enable us to, as a postdoc, kind of mix the two sets of kids together and increase the N of the top two doses. So I think, you know, Longer term, we have a lot of pharmacodynamic data that's going to come out of that trial. It takes a little bit longer to pull that out and analyze it than it does annualized site velocity. So right now, we're just focusing on the annualized site velocity for the 212. Great.
spk10: Thank you.
spk06: Thank you.
spk10: Thank you. Your next question comes from the line of Ed White with HC Wainwright. Sir, please go ahead.
spk05: Good afternoon. Thanks for taking my questions. So I think most of them have been asked already, maybe just on the number of sites open. With the war in Ukraine and Russia, you had mentioned that you are now planning for targeting 40 sites. And I think in November, you had mentioned that you had more than 40 sites open. Where are you now after removing the sites for Russia and the Ukraine?
spk04: Ed, thanks for that good question. And I think it's a question that everyone in our industry who has study sites in that part of the world are facing. We had nine sites, five in Russia, four in Ukraine. As we told you, everything is on pause. No patients had been randomized from those two sites. And so even without those locations or patients coming from those locations, we're really encouraged about our enrollments. We're also adding a few sites here and there, but things are going well as we speak, and we think that we'll – we certainly believe that we'll reach the goal that we just mentioned in our prior discussion, you know, before the end of 2023. But things are going well in spite of that.
spk05: Okay, thanks, Rick. And I guess just the last question that we all ask each quarter is just you always mention other indications, and I was just wondering if you're making any progress in any other indications or, you know, has there been any change to the strategy or anything that any one indication that you're prioritizing now over the others? Thank you.
spk04: Yeah, John, you want to answer that question?
spk06: Sure. As we said, you know, we've dug in and looked at a lot of these different secondary indications, thought about how our unique mechanism of action of LUM201 would fit into those settings. And we've always wanted to get a sense of what the dose responsiveness of LUM201 would be in the PGHD population first. And that really does help us kind of find do the final refinement of where we plan to go for those secondary indications. So we're getting to a point now where I think once we have that data, we'll be able to, you know, finalize our discussions and our decisions on those secondary indications.
spk05: So, John, just thinking ahead, since you're planning on announcing interim data by year end, is that when we should expect to hear more?
spk06: Well, yeah, I mean, we're expecting by the very end of the year. We do want to look at that data and then try to apply it across the secondary indications. We've done much of the footwork, but it really, you know, when the data comes in, we'll be able to make some decisions about how it applies to other indications. So I won't promise right then, but probably shortly thereafter.
spk05: Okay. Thanks for taking my questions. Thank you.
spk10: Your next question. Thank you. Your next question comes from the line of Catherine Novak with Jones Research. Ma'am, please go ahead.
spk03: Hi. Congrats on the update. Hi, Catherine. Thanks so much for taking my question. I have a question on the interim analysis for 2012. You know, in addition to the annualized height velocity, I guess I'm kind of curious about what's key to understanding the 24-hour pulsatility in these positive enrichment marker – patients, you know, can you remind me, you know, how many 12-hour pulsatility assessments, you know, we're going to get during the 12-month dosing period? And what do we expect to see in terms of change from baseline as far as how that looks?
spk04: John, I think you're closer to the data from the prior study, but also knowledgeable about the details of the blood draws. Why don't you start, and David, you could add.
spk06: Great. Nice to chat with you again, Catherine. I think, you know, the data we've showed previously is on the 0.8 mg per kg per day cohort from the O2O study. And, you know, we've shown kind of two PEM positive patients and how they, you know, how their pulsatility changed. Generally, looking at it in the context of just a visual representation, but also looking in terms of AUC. And, you know, that's kind of the baseline data that got us excited about repeating the study on a larger scale. So in that study, we're not doing any 0.8 mcg per kg per day kids, just the top two doses, 1.6 and 3.2. And at baseline and at six months on treatment, we will do the same pulsatility assessment. It would be every 10-minute sampling for 12 hours after they take their morning dose of LUM201. So we'll get a very nice assessment of how their pulsatility changes with respect to their baseline. And I think that will be a very interesting piece of data at those top two doses. It gives us an opportunity to potentially look at dose responsiveness on the PD marker. And it also gives us a little bit of a sense of trying to understand when we modulate that growth hormone levels in a pulsatile fashion by amplifying each of those individual peaks, what impact does it have really on the 12 or 24-hour growth hormone levels, and how does that relate to growth at six months? Those PD analyses are pretty complex, and they're going to take a little bit more time than just calculating the analyzed height velocity. So that full PD analysis, which I think ties very nicely in with the mechanism of action of LUM201, will take a little bit longer than the annualized site velocity that we expect to be able to show you folks at the end of the year. Does that help?
spk03: Yeah, that's helpful. I think it's just an interesting measurement, so kind of curious to get more information about how to, you know, be prepared to analyze those data when they come out. My second question was around the 213 study. You know, was this already factored into your cash runway and you still expect to be, you know, have cash sufficiency until the, after the ORA growth 210 six-month data? And then another question was, you know, I wasn't sure how many patients FDA wanted 12-month safety data on. So, you know, can the 213 study be used to accelerate this 12-month safety data requirement that was previously announced?
spk04: Let's answer the financial question first. And Lori, go ahead.
spk02: Sure. Hi, Katherine. So the cash at the end of 2021, as we've said, is sufficient to get us through both the readouts for 210 and 212 on the primary data, which is the six-month treatment, and then also continue to support operations during those times. Does that answer your question? Yes, that answers that question. Thanks.
spk04: Yeah. And then, John, the 12-month safety.
spk06: So I think overall what we've tried to do with our Phase II program is build a really comprehensive and deep data set, right? So we have the 210 study where we're going to have all of those patients extended for 12 months who have taken 12 additional to 12 months for those patients who are on lumen 201. And then this new study basically takes the patients who were on or the subjects who were on the recombinant human growth hormone arm, many who entered this trial because they thought the odds were good they would get an oral and not a daily injectable, but they drew the short straw and took an injectable every day. We did want to make sure that they had an opportunity to take an oral growth hormone secretagogue for a year and see how they responded to that. I think all of the safety data, collecting 12 months of data in our 210 study, in our 212 study, in addition, this SWITCH study is really going to build a very nice data set, I think, both in terms of efficacy. This little SWITCH study will give us some interesting data about kids who could switch from common human growth hormone onto LUM201. It's not large enough to really be very robust, but I think with 20 patients, we'll get a very good insight into which patients would respond. And they're all PEM-positive kids because they were PEM-positive to get into the 210 study. So I think I just look at it in terms of how robust the data set is going to be when we finish our Phase II studies, and I think it's going to be pretty impressive.
spk03: Okay, thanks. Thanks for that. All right, congrats on the quarter.
spk04: Thanks, Catherine. Appreciate it very much.
spk10: Thank you. Your next question comes from the line of Elmer Piros with Ross Capital Partner. Sir, please go ahead.
spk01: Yes, hi. What I'd like to clarify, Rick, I think Charles alluded to the interim analysis being blinded. I wasn't sure if that is the case. If you could please clarify that.
spk04: Good question, Elmer. And John, go ahead, if you will.
spk06: So I think we have obviously one arm getting a daily injectable and the other arm's getting an oral, right? So I think this isn't double-blinded by any sense. So I think we have the opportunity to look at this data and analyze it. Obviously, we're going to blind the folks making medical decisions to the data, right?
spk01: And so in what way it may compromise continued enrollment if it's widely successful?
spk06: I didn't understand that. In what way?
spk01: So if you announce that the oral LUM201 is equivalent at the highest dose, to daily growth hormone injections, is there a risk that patients would want to actually or wouldn't want to continue to enroll or be treated? This may be very hypothetical.
spk04: David, why don't you answer that question as a clinician?
spk07: Sure. Based on when the interim analysis data will be announced, I don't know that we would have any more enrollment to do in the trial, quite honestly. So that would be a theoretical concern, but I think it will not have any impact.
spk01: Okay, okay. Thank you. Thank you for clarifying that. And just to make sure that I also understood correctly, so there hasn't been any patients enrolled from the Russian and the Ukrainian sites?
spk07: No, there is not. No patients randomized in either of those two countries.
spk01: Okay, okay. And that was all. Thank you so much, and we'll see you next week at our conference.
spk04: Yeah, looking forward to it.
spk01: In person. Yes. Thank you.
spk10: Thank you, and your... Last question would be a follow-up from Charles Duncan with Kanpur Fitzgerald. Sir, please go ahead.
spk08: Sorry about that. I was on mute. Thanks for taking the follow-up. Appreciate Elamir pointing out the blinded thing. But I do wonder whether or not there will be any opportunity to make this an adaptive design trial. So you feel like at that time you'll have the trial completely enrolled and therefore patients randomized to the dose and no opportunity to make it adaptive?
spk04: John, why don't you answer Chaz's question?
spk06: So our current predictions are that we're not going to be quite fully enrolled by the time we read that out, but the majority of folks will be enrolled in the study. So I think there's not that much opportunity to make a difference with an adaptive design.
spk08: Okay. And then kind of as a follow-up, I'm just wondering about guidance for second half of the year full data then you know let's say that you know sometime in December you're fully enrolled you do the interim would seem to me that certainly by June you may have the data and I understand it takes a while to work through the data set etc but is that what's really governing kind of timelines maybe a little conservatism for you know data analysis
spk04: Chas, thanks for asking that excellent question. I mean, I think you know us by now. We're trying to be conservative in our approach to the market and making sure that we follow through and we do exactly as we tell you we're going to do. You know, I think these are still uncertain times. We certainly hope that enrollment is going to continue at the pace that we're currently seeing. We were counting on enrollment from Ukraine and Russia, and other parts of the world are going to have to pick up. So I think it's a little too early to announce anything that will happen before the end of 2023. But, you know, as usual, you'll be one of the first to know.
spk08: Yeah, I'm just hoping to know the same time everyone else knows. Of course. Yeah, so let me ask you about 213. You talked about there being 20 patients that you intend to enroll in that study, and it seems like a really interesting study. So I'm kind of wondering... why you wouldn't enroll all 40 patients who are on the control arm from 210?
spk04: David, why don't you answer that question?
spk07: Sure. The full 210 study is enrolling 80 subjects, 20 subjects in each group. So the 20 subjects is the intended enrollment of the control arm.
spk08: Got it.
spk07: Charles?
spk08: Thanks for clarifying. Got it. Got it.
spk07: Thanks for clarifying my bad on that. Okay.
spk08: But you do have an OLE that you intend to switch people or patients over, and you'll give us an update on that later on this year?
spk06: Absolutely. Yes, we will.
spk08: Yeah. Okay. Thanks for taking my follow-up.
spk05: Okay.
spk10: Thank you. I'm showing no further questions in the queue at this time. Thank you for joining us and enjoy your afternoon.
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