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.
spk24: Good afternoon, ladies and gentlemen, and welcome to Loomis Pharma's second quarter results conference call. Currently, all participants are in the 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.
spk25: Thank you. 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 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 these forward-looking statements. Information presented on this call is contained in the press release we issued this morning, which may be accessed from the investor's page of the company's website. Joining me on today's call are Rick Hawkins, CEO and Chairman, John McHugh, President and Chief Scientific Officer, Dr. David B. Karp, Loomis' new Chief Medical Officer, and Lori Lawley, Chief Financial Officer and Corporate Controller. Following management's prepared remarks, we will open the call to your questions. I will now turn the call over to Rick.
spk17: Thank you, Lisa, and good morning, everyone, and thank you for joining us on today's call. This morning, we issued a press release detailing our results for the second quarter 2021. Since we held a call on July 21st to provide updated guidance on our clinical programs, I will keep my remarks brief before turning over the call to my colleagues for additional updates on our clinical trials and our financial results. Then we'll open the call for your questions. I'll begin with the recent steps we've taken to enhance our advisory board and strengthen our management team. Last week, we were pleased to announce the appointment of Dr. Mark Bock to our Clinical and Scientific Advisory Board, or CSAB. Dr. Bach joins our distinguished panel comprised of noted pediatric endocrinologists, Drs. Peter Clayton, Reiko Hirakawa, George Werther, and CSAB Chairman, Dr. Ron Rosenfeld. Dr. Bach is currently Chief Medical Officer for SHOTI and previously was Senior Vice President, Endocrine Medical Sciences at Ascendus Pharma. He is a pediatric endocrinologist with 30 years of clinical research and pharmaceutical development experience. including extensive global experience building and leading clinical teams that have successfully launched innovative pharmaceutical products into worldwide markets. Prior to the CEMDIS, Dr. Bach spent nine years at Janssen, a subsidiary of Johnson & Johnson, concluding his time there as head of Asia-Pacific Medical Sciences and head of China R&D. And prior to Janssen, Dr. Bach held positions of increasing responsibility in clinical research at Merck & Company, ending his tenure there as Vice President of Clinical Research Operations Worldwide. Now, early in his career, Dr. Bach conducted extensive clinical and preclinical research on gross hormone, IGF-1, and LUM-201, formerly known as MK-677, with data from his work published in scientific and medical journals. He brings to our advisory board a wealth of knowledge and experience with growth hormone-related disorders and a significant understanding of the unique advantages of LUM201 could offer to the growth hormone-deficient population. We are obviously thrilled to have him join our esteemed advisory board and look forward to his contributions to Lumos Pharma's clinical and commercial strategy. And earlier this week, we also announced the promotion of my respected colleague and friend, John McHugh, to the position of President of Lumos Pharma. John has served as our Chief Operating Officer since April of 2020 and our Chief Scientific Officer since he joined Lumos Pharma in 2016. John has nearly 30 years of experience of developing novel therapeutics, primarily for rare diseases, and has successfully advanced multiple therapies through preclinical and clinical development. And prior to joining Lumos Pharma, he served as vice president of research at Atar Pharma. We led a research team discovering and advancing protein-based therapeutics for rare diseases. He's also served as acting scientific director for the National Center for Advancing Translational Science, or NCATS, intramural group, a part of the National Institute of Health. At NCATS, his lab's work on rare and neglected tropical diseases and public-private partnerships led to the collaborative advancement of several therapeutic candidates currently being commercialized by pharmaceutical companies. John's promotion to president of Loomless Pharma is in recognition of his vital contributions in advancing Loom 201 for the treatment of PGHD as part of the succession planning conducted by the company and our board of directors. John's extensive clinical and management experience at the NIH and in the pharmaceutical industry, including his contributions during his tenure at Loomless Pharma, position him well to serve in this new role. And on Tuesday, we announced the expansion of our management team with the addition of Dr. David Karp as our new chief medical officer. David is an endocrinologist, an academic, an accomplished biopharmaceutical executive, and is currently an adjunct clinical professor in the Division of Endocrinology at Stanford University School of Medicine. David has 35 years of experience in the field of endocrinology, And as a biopharma executive, he has 30 years of experience in all facets of biopharmaceutical clinical development, from preclinical through phase four, regulatory interactions with both the FDA and international agencies, and marketing and launch activities. Most recently, he was vice president, clinical development and clinical operations for Ascendus Pharma, where he was responsible for several compounds and clinical development including Transcon GH, long-acting growth hormone for once-weekly treatment of growth hormone deficiency. He previously served as chief medical officer for several biotechnology companies, and he has also held leadership positions in clinical research and endocrinology at both Roche and previously at Merck, where he gained familiarity with our therapeutic candidate, LUM201. And we look forward to David's guidance and assistance in advancing Loom 201 toward commercial approval. So welcome, David.
spk04: Thank you, Rick, for your kind introduction. And good afternoon, everyone. I only wanted to add how excited I am to join the Lumos team. As Rick mentioned, I'm very familiar with Loom 201 Asset from my time at Merck. Based on my experience treating growth hormone deficiency and my experience developing a long-acting growth hormone for patients with GHD, I believe that Loom 201 as an oral therapy is potentially a breakthrough advance in this therapeutic area. Loomis has a well-developed clinical plan to advance this asset, and I look forward to working with Rick, John, and the rest of the team to execute on our clinical strategy.
spk17: Thank you, David, and again, welcome. And before turning the call over to John for an update on our clinical programs, I just want to highlight a common theme in these appointments, and that is the view held by many experts in endocrinology of the potential of LUMA 201 to be a breakthrough oral therapy for the treatment of PGHD. On the strength of that potential, we believe LUMA has put in place the talent and expertise needed to advance this asset and realize its full commercial expectations. So with that, I'll turn the call over to John.
spk13: Thank you, Rick, and welcome, David. We're all looking forward to working with you. As Rick mentioned earlier, we provided updated guidance on our clinical programs on our call on July 21st. As a reminder, the six-month primary outcome data readout for our Oral Growth 210 trial is now anticipated in the second half of 2023. The extension was prompted earlier by an initially slower pace of site initiation due to ongoing COVID-19 restrictions at certain international sites, and a disruption of the typical referral patterns for this patient population. We further expect referral patterns to normalize as children return to school and communities adapt to the persistent presence of COVID-19. As we continue to open new clinical trial sites for this trial, we are beginning to see the pace of screening and enrollment increase as compared to the first half of this year. Separately, we also announced the extension of the protocol for the OrgGrowth 210 trial for an additional six months on therapy in response to the FDA's request for 12 months data prior to the initiation of our previously planned three-year long-term extension study, the OrgGrowth 211 trial. Again, the primary outcome data readout of the OrgGrowth 210 trial remains at six months on therapy. This OrgGrowth 210 protocol extension mirrors the previous plan for the first six months of the Oral Growth 211 long-term extension study. We believe the extension of this trial will provide a more robust data package for future regulatory filings. The Oral Growth 211 trial was proposed to capture long-term treatment effects for patients who responded to LUM201 in previous trials. However, that study was not on a critical path for the initiation of a phase three trial. We announced the initiation of our Oral Growth 212 trial in Q2. We subsequently announced the proposed extension of the treatment period of this trial from six months to 12 months. Bar Growth 212 is a small, single-site, open-label trial to illustrate the pharmacokinetic and pharmacodynamic effects of LUM201 in PGHD and replicate the pulsability data that currently exists for LUM201 in adults and in a small subset of children from the Phase 2 O2O trial. We also decided to extend this trial to capture additional PK, PD, and height velocity data. The PD pulsatility assessment will continue to be assessed at six months on therapy. The trial was initiated in June and is currently enrolling patients. To recap, both the ORA Growth 210 and ORA Growth 212 trials will provide data readouts at six months on therapy as was originally planned. The extensions to 12 months of therapy will enhance the supporting data package for our overall LUM201 program. Furthermore, we do not anticipate these protocol changes on a standalone basis to extend our clinical development timeline. With that, I will turn the call over to Lori, who is joining us for the first time in her new role as CFO for a review of our second quarter financial results. Lori?
spk20: Thanks, John, and good afternoon, everyone. We ended the second quarter on June 30, 2021, with cash and cash equivalents totaling $107.7 million, compared to $98.7 million on December 31, 2020. Cash on hand as of the end of Q2 2021 is expected to support operations through the six-month data readout from both the ORA Growth 210 and ORA Growth 212 trials. We had research and development expenses for the second quarter of $4.1 million. an increase of $1.4 million over the same period in 2020. The change is primarily due to an increase of $1.8 million in clinical trial and contract manufacturing expenses, offset by a decrease of $.4 million in personnel and operational expenses. Our general and administrative expenses for the second quarter were $4.6 million, an increase of $.4 million compared to the same period in 2020. This increase is due to an increase of $1.1 million in personnel-related expenses, primarily due to the recording of severance expenses for a former CFO, offset by decreases in legal consulting and operational spend of $0.7 million. The net loss for the second quarter was $8.7 million, compared to a net loss of $5.4 million for the same period in 2020. We end at Q2 2021 with 8,357,391 shares outstanding. And with that, I will turn it back to Rick for closing remarks.
spk17: Thank you, Lori. So, before taking your questions, I just want to say that we have advanced our discussions with key opinion leaders in our Clinical Scientific Advisory Board to expand our LUM201 platform. We are actively reviewing the pathway for LUM201 and a certain subset of affected patients' and other potential indications, including Turner syndrome, Prader-Willi syndrome, idiopathic short stature, and for children born small for gestational age. We're confident in our belief that LUM201 represents a pipeline and a product, and look forward to advancing the next phase of the LUM201 life cycle. And we'll provide further updates when we're ready to discuss comprehensive clinical plans. In addition, We continue to evaluate select rare disease assets under consideration to add to our product portfolio. And as I stated before, we're being highly selective and judicious in our process and we'll focus on our decisions on what provides best long-term value creation for our shareholders. The enhancement of Loomis Pharma's leadership team and encouraging recent trends in screening and enrollment for both OroGrowth 210 and OroGrowth 212 trials are positive developments for the company. John, David, and Mark each bring significant clinical development and management expertise to their new roles with the company and strengthen our ability to execute on our clinical and corporate strategies. We believe that as schools reopen and pediatrician visits resume, we should see increased referrals and accelerated enrollment in our trials this fall. So with that, operator, we're now ready to take questions.
spk24: Ladies and gentlemen, if you have a question at this time, simply press a star, then the number one on your telephone keypad. If your question has been answered or you wish to remove yourself from the queue, Please press the pound key. Again, please press the star one to ask a question. One moment, please, for our first question. Your first question comes from the line of Yasmin Rahimi of Piper Sandler. Your line is open.
spk23: Hey, this is Felicia on for Yaz. Thank you so much for taking your questions. So we have two for you this morning. First, do you guys think that you'll be opening additional sites to maintain enrollment timelines? And if yes or no, could you please just provide a little bit of color on that? And next, we were wondering if your current cash balance is sufficient to build up your pipeline while also getting to the finish line for ORA Growth 210 and 212 studies. And should we be expecting pipeline expansion of Loom 201 in 2021 or 2022? Thank you. Well, the first question,
spk17: I believe we've anticipated for quite some time the concern over COVID-19. So we did a site expansion to about 52 sites quite some time ago. And as I mentioned earlier, the majority of those sites are open, and we're rapidly advancing and trying to open the rest of those sites. And also, I think there's a very broad geographic distribution. So, once again, I think that's going to help us too. But beyond to about 50 or 52 sites we have planned right now, I think that's going to be sufficient to meet our enrollments. And in terms of cash, we're in a very strong position. And as Lori said, we can get a readout on our 210 and 212 studies. It also means we're going to have to be that much more judicious in our review of anything that we want to bring into the company. This may open up us to looking at assets that are more in the preclinical or actually close to the clinic. But at the same time, I want to emphasize we have a robust process in place and we're being very careful and judicious in our review of anything that we want to bring in as an asset.
spk24: Thank you. Your next question comes from the line of Charles Duncan of . Your line is open.
spk09: Yes. Good morning. Thanks, Rick and team, for the update as well as taking my call. Congrats on all the new joiners, if you will, on the management team. I had a couple of questions. First of all, perhaps for John, in terms of the 210 study, I guess I'm wondering if you could remind us of the screen versus enrollment rates that you're looking for, the screen failure rates given the PEMM. And it seems like, and just maybe help me understand the second half of 23 data, it just seems like a year and a half to enroll this study is long. So is it perhaps that you're being overly conservative?
spk06: John, go ahead.
spk13: So we can talk first about the screen failure rate. So our our assessment of the screen failure rate or the percentage of patients with a PGHD diagnosis who are PEM positive, which I think is the important part here. We, you know, as we've published recently in the Journal of Endocrine Science, we did an analysis of a very large 20 year long multinational phase four study called the Genesis data set, which is based on the least experience with their recombinant growth hormone product. And we were able to apply our PEM cutoffs to that data set and generate a much broader and a large data set, a very nice assessment that, you know, about 60% of the patients would be PEM positive. Obviously, we're working on a much smaller scale than that data set as we do screens for our study. So we don't, you know, we don't expect to have data that – that is going to be as telling as the Genesis data set. So I think that's the screen failure set. And then for the second, for the re-guiding, really, it isn't about screen failures. It really isn't about anything other than just a slow recruitment that is based primarily, well, a slow process that's based primarily on delays that were primarily attributed to COVID-19. I would say that I think we are being conservative. We want to guide the market on a timeline and a pace of patient recruitment that we think is achievable, and we will work very hard to not only achieve that, but do all we can to beat that.
spk14: Okay. Go ahead.
spk17: Go ahead. I might add, Chas, I mean, there's no question there's a concern in everybody's mind about COVID-19 and its current status. We want to be conservative in our prediction at this stage, but certainly believe that with the number of new screen patients, it's encouraging.
spk09: Okay, and then if I may, and I apologize up front, but Dr. Karf, I'm going to put you on the spot, and that is that, you know, basically a question of why join LUMO, and really what I'm interested in, I'm particularly intrigued with the secretagogue and pulsatile release of growth hormone and how that may be different than, say, a simple replacement therapy. And I'm not being dismissive of the convenience and compliance benefit, but I'm just kind of wondering mechanistically if you could provide your thoughts on those different mechanisms.
spk04: Well, I could not agree with you more, actually. So what I have been attracted to with the molecule I knew as 677 is is precisely that. I mean, talk about a physiological replacement. It's been known for a long time that once weekly, once daily, neither one of those modalities actually recapitulates the normal growth hormone access, which is basically a very low basal with spurts of growth hormone mostly at night, multiple peaks. I was struck when I was at Merck with, I mean, here was an oral agent you could take in the morning, you could take it in the evening, and what it did was simply enhance the normal growth hormone peaks. I mean, talk about a marvelous design of a drug. Now, when I was at Merck, Merck was all about large populations. They were the antithesis of a company that was interested in a rare orphan indication. So this was a beautifully designed drug, and they wanted to try to search out a big population and they chose sarcopenia in the elderly, which unfortunately lacked and I think continues to lack a registration endpoint. So that was their attempt, I think, to make a large market like, you know, hypertension, hyperlipidemia, osteoporosis out of this molecule. But they had enough faith in this that they did license it to a colleague of mine, Michael Thorner, for development in PGHD. And I'm very excited about this. Now I'm, I'm bullish about trans con growth hormone. I'm a little bit less bullish, but reasonably bullish about topacitin. But what I see, I mean, when I retired from Ascendus last September, I was very happy seeing my 600 patients at Stanford and doing some consulting. And I really did not have any intentions at that time of getting back into a full-time position. When Rick called me and I realized what this molecule was, I mean, what excites me is that I've been able so far to bring three effective, well, I've committed three substantial NDAs and brought two effective drugs to patients and very soon a third drug to patients for a medical need. And I view this opportunity with Lumos as my last great chance of bringing a really effective drug to a significant population. unmet need in patients. I think that for the appropriate patients, this will be a very, very good treatment of their growth hormone deficiency. Did I answer your question?
spk09: Yeah, absolutely, and really appreciate all the color. I'm looking forward to seeing it move forward as well. Last question is for Rick, perhaps on strategy issues. I guess you mentioned the pipeline, and it would seem to me that you have kind of your hands full with Loom 201, and it's a promising candidate. And I guess I'm wondering how you look at it in terms of being a pipeline and a product with other indications relative to in-licensing opportunities. It would seem to me that the capital markets have kind of, competed against you with regard to the latter aspect of that or route in terms of that strategy. And I'm wondering if there's a favorite leading additional indication that you would want to highlight for Loom 201.
spk17: That's a good question, Chas. And I want to say that, you know, first and foremost, our priority remains to stay focused on executing on our Wargrove 210 study and 212 study plan. I think that also evaluating other indications of our compound because of its unique mechanism of action would likely be beneficial and provide, and we're going to provide updates at the appropriate time when those plans are more fully cooked. But obviously, this is a unique opportunity with this compound, and we are engaging all the important KOLs out there, and as we lay out our plans, you'll be the first to know. I think it's prudent for us, though, to continue based on our extensive backgrounds in the rare disease space. to continue to look for opportunities or other assets out there. Now, as you know, we've been doing this for quite some time, and we haven't pulled the trigger on anything, and that's because we have been as careful and as judicious as we possibly can because anything that we do, we want to make sure that it's a strong value proposition for our shareholders, and that's all there is to it. But I think it would be imprudent for us not to have a process in place to find a unique asset, just like the unique assets that we're developing in Loom 201.
spk09: Very good. Thanks for taking my questions and appreciate the update. Okay.
spk24: Your next question comes from the line of Ed White of HT Wingwright. Your line is open.
spk11: Good morning. Thanks for taking my questions. So maybe start with a question for Laurie. I'm curious as to R&D spend going forward. You know, we would expect to see the trend going up as the trials are initiated. Sequentially, R&D expenses were down. I'm just wondering if you can give us some thoughts on how we should be thinking about the next two quarters for R&D expense.
spk20: Sure, and thank you for the question. So, sequentially, compared to Q1, we had some severance costs that were recorded in Q1 for the departure of our former CMO of approximately $1.4 million. I would expect and anticipate that our Q2 spend is reflective of what we will expect going forward with increases as we advance our clinical trials and path forward towards a Phase III trial.
spk08: Great, thank you.
spk11: Does that answer your question? John, congratulations on your new role and title. I'm just wondering how your role is changing within the company, if you can give us any thoughts to that.
spk13: Well, thank you for the congratulations, Ed. And really, I think, you know, the focus will be will continue to be similar to the role I have been playing. I think it's a promotion where I'll be able to focus on both the operational and scientific aspects of the company and making sure that we deliver on all the aspects of our guidance.
spk11: Okay. And maybe just the last question that I have, we've been talking about enrollment in the 210 study and I'm just curious on two things. Now that we're seeing, you had mentioned that as children go back to school, there could be perhaps more pediatrician visits. We could see more interest in the studies. I'm just wondering how you're thinking about the Delta variant and is that going into your thought process on enrollment trends And then when could we see you give us an update on what the actual enrollment are in these two studies? Will you be telling us when you get halfway there or maybe perhaps over the next few quarters, do you plan on giving us updates on how the enrollment is going? Thank you.
spk17: Yeah, I'll start with that, Ed. You know, we haven't stated at any time in guidance that we're going to address when we're, you know, quarter or a half or three quarters of the way through. We just have our noses to the grindstone trying to make sure that we're doing everything we can to make sure this study goes as well as possible. Perhaps we would address the market at a more appropriate time, but right now it's a little bit too early on.
spk25: And John, could you take his first question regarding the Delta virus impact?
spk13: Yeah. So, you know, I think the Delta variant is much like the Alpha variant was last year, right? So the way we plan around that is, you know, right now, as Rick has mentioned, we have more than 50% of our sites up and running. Part of our delay in the past was as much about getting sites up through delays of getting through IRBs and, you know, interacting with lawyers and contract people at clinical sites who are working from home, which seemed to add an enormous amount of time. Uh, and I think we have those sites up and running. And, uh, and as we had mentioned in the past, we have, uh, sites spread across various geographies in the U S but also internationally in Eastern Europe and in, um, and in Australia and New Zealand. And I think the combination of those two things are really going to help us, um, kind of juggle, you know, move from area to area where, uh, you know, the pandemic waxes and wanes. But I also think regardless, at this point, communities are starting to learn how to live with COVID-19. And I don't, it's hard to imagine that we will go through another school year with the, you know, with remote learning for the entire year. I think we will find ways to be successful in educating kids which will stimulate trips to the pediatrician and referrals to the pediatric endocrinologist.
spk04: If I could just endorse what John just said, it absolutely mirrors my experience getting the transcom PTH studies enrolled under COVID, which is exactly what he said. The delay is getting sites initiated. But even the initial epidemic delay did not have a meaningful impact because once the kid is diagnosed, I mean, the parents really want this kid treated. So there was really minimal impact in a subject coming into the site. All of the delay was in really getting the site initiated and all the hoops you've got to go through to get a site initiated. So I just want to endorse what John said about that. I expect that to happen with the LUM201 trial as well.
spk11: Great. Thanks for taking my question. Thank you, Ed.
spk24: Your next question comes from the line of Yun Yang. Jeffrey, your line is open.
spk03: Hi. Good morning. Thanks for taking my question. This is Suji calling in for Yun. So for Dr. Cao, I just have a quick one question. So do you think that before starting phase three, the regulatory agencies might ask ask for additional study where you would have to compare the efficacy of LUM201 in PM positive versus PM negative to show that the LUM201 has selective advantageous activity in the positive population. Thank you.
spk17: I'll tell you this. Since Dr. Carpenter just arrived, I think I'm going to let John start with that question and perhaps have David finish it.
spk04: I'm careful making a stab at that, but go ahead.
spk13: Yeah, so I think, you know, we have Merck's previous data set that we've analyzed, and we've separated out PEM positive and PEM negative, and there's a dramatic difference, and obviously we've published on that. So I do think, actually, that the PEM positive data prospectively selecting those patients and bringing them into our ongoing trial is really going to be the proof that the FDA and I think the market will appreciate as well to show that this enrichment strategy really does work effectively. And remember, this enrichment strategy is based on physiology, right? So the kids who are PEM negative, they simply don't have an active axis. They don't have enough somatotropes. They can't store growth hormone. They're not going to be able to respond to our drug, which is just going to stimulate the release of endogenous growth hormone. So it seems mathematical when you look at how we generated that data, but it is tied to physiology. And I think we can make that case very effectively to regulators as well as in a scientific setting to show that as long as we can show the PM positive kids are growing and and we're able to enrich that population of responders with our cutoffs. I think that will be sufficient evidence.
spk17: And David, do you want to add anything?
spk04: Yeah, I'll just say that on the one hand, this is my second day at the company, but on the other hand, based upon my 30 years of experience dealing with the Endocrine Metabolic Group, I can say with, I think, some authority the following. First of all, I think the agencies, like third-party payers, would be absolutely thrilled at the selection process to identify the best patients for the drug. I think that the phase three trial, if I had to guesstimate, would basically be about a two-to-one randomization between PEM-selected patients for the LUM201 group versus unselected patients for daily growth hormone. And it'd be a 12-month study, and the likelihood of having to go for PEM-positive versus PEM-negative I would handicap as close to zero as to be negligible. There's no rationale for that. I would be willing to bet a lot of money it'll be PEM selected versus unselected for daily growth hormone. That'll be the phase three study. You shall see.
spk02: Great. Thank you.
spk24: Your last question comes from the line of Catherine Novak of Jones Trading. Your line is open.
spk19: Hi. Thanks very much for taking my questions. I had a couple on pipeline expansion possibilities. First, as you think about potential BD opportunities, what are some specifications that you're looking at in terms of assets that you think make sense in your portfolio?
spk17: Well, Catherine, thanks for your question. And, you know, our criteria has really always included starting with a good strategic fit, you know, a strong scientific rationale, and really a strong value proposition for our shareholders. We have been willing to look at the earlier stage opportunities and are going to continue to do so on a very selective basis. But, you know, and above all, you know, we'd like to stay in the endocrine space. I mean, we have a lot of experience, and especially with David arriving, I think the opportunities are going to expand for us to look at. And our process, I think, is a very good one. But as you know, we haven't pulled the trigger on anything because it hasn't met our really careful and judicious criteria.
spk19: Yeah, thanks. That makes sense. And then when you're thinking about expanding LUM201 into these other indications where growth hormone is approved, do you have an idea of what subset of patients might benefit from the LUM201 unique mechanism of action?
spk16: And, John, why don't you start with that?
spk13: So the only – growth number deficiency population that LUM2-1 has been tested in right now is in the PGHD population, right? So that's the population where we have a correlation of their PEM tests back to growth, right, the outcome. So each of the other indications that growth number is approved in has, you know, unique facets to the etiology of the disease, right? Sometimes there are things There is some growth hormone deficiency and there is some downstream modulation of receptor activity or IGF-1 activity. So that's really where we've been spending our time is looking through all those facets and trying to understand really the overall potential of LUM201 in that area and also thinking through how would pulsatility affect LUM201. each of those individual disease states versus, you know, the bolus, right? So we have good ideas of how that might have an impact in PGHD, and we want to understand that for some of the other indications. And then, you know, once we get past that, we do try to assess how frank is the growth hormone deficiency itself in each one of these indications, and that really can be an indicator of where our mechanism of action of stimulating the release of endogenous growth hormone is going to play in each one of these indications. And that's really what we're building, that data set through, you know, mining information after history data about each one of these diseases right now to put together our prioritized list of where we're going to go forward.
spk19: Thank you very much.
spk07: Thank you, Catherine.
spk24: Thank you. I'm showing no further questions in the queue at this time. I'll hand the call back to Mr. Hawkins for closing remarks.
spk17: Well, we thank you for joining us today, and we look forward to keeping everyone apprised of our program over the course of the year. Thank you very much.
spk24: Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect. Thank you. Thank you. you Thank you. music music Thank you.
spk22: Thank you.
spk24: Good afternoon, ladies and gentlemen, and welcome to Loomis Pharma's second quarter results conference call. Currently, all participants are in the 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.
spk25: Thank you. 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 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 these forward-looking statements. Information presented on this call is contained in the press release we issued this morning, which may be accessed from the investor's page of the company's website. Joining me on today's call are Rick Hawkins, CEO and Chairman, John McHugh, President and Chief Scientific Officer, Dr. David B. Karp, Loomis' new Chief Medical Officer, and Lori Lawley, Chief Financial Officer and Corporate Controller. Following management's prepared remarks, we will open the call to your questions. I will now turn the call over to Rick.
spk17: Thank you, Lisa, and good morning, everyone, and thank you for joining us on today's call. This morning, we issued a press release detailing our results for the second quarter 2021. Since we held a call on July 21st to provide updated guidance on our clinical programs, I will keep my remarks brief before turning over the call to my colleagues for additional updates on our clinical trials and our financial results. Then we'll open the call for your questions. I'll begin with the recent steps we've taken to enhance our advisory board and strengthen our management team. Last week, we were pleased to announce the appointment of Dr. Mark Bock to our Clinical and Scientific Advisory Board, or CSAB. Dr. Bach joins our distinguished panel comprised of noted pediatric endocrinologists, Drs. Peter Clayton, Reiko Harakawa, George Werther, and CSAB Chairman, Dr. Ron Rosenfeld. Dr. Bach is currently Chief Medical Officer for SHOTI and previously was Senior Vice President, Endocrine Medical Sciences at Ascendus Pharma. He is a pediatric endocrinologist with 30 years of clinical research and pharmaceutical development experience. including extensive global experience building and leading clinical teams that have successfully launched innovative pharmaceutical products into worldwide markets. Prior to Ascendus, Dr. Bach spent nine years at Janssen, a subsidiary of Johnson & Johnson, concluding his time there as head of Asia-Pacific Medical Sciences and head of China R&D. And prior to Janssen, Dr. Bach held positions of increasing responsibility in clinical research at Merck & Company, ending his tenure there as Vice President of Clinical Research Operations Worldwide. Now, early in his career, Dr. Block conducted extensive clinical and preclinical research on growth hormone, IGF-1, and LUM-201, formerly known as MK-677, with data from his work published in scientific and medical journals. He brings to our advisory board a wealth of knowledge and experience with growth hormone-related disorders and a significant understanding of the unique advantages of LUM201 could offer to the growth hormone-deficient population. We are obviously thrilled to have him join our esteemed advisory board and look forward to his contributions to Lumos Pharma's clinical and commercial strategy. And earlier this week, we also announced the promotion of my respected colleague and friend John McHugh to the position of President of Lumos Pharma. John has served as our Chief Operating Officer since April of 2020 and our Chief Scientific Officer since he joined Loomis Pharma in 2016. John has nearly 30 years of experience of developing novel therapeutics, primarily for rare diseases, and has successfully advanced multiple therapies through preclinical and clinical development. And prior to joining Lumos Pharma, he served as vice president of research at Atar Pharma, where he led a research team discovering and advancing protein-based therapeutics for rare diseases. He's also served as acting scientific director for the National Center for Advancing Translational Science, or NCATS, intramural group, a part of the National Institute of Health, At NCATS, his lab's work on rare and neglected tropical diseases and public-private partnerships led to the collaborative advancement of several therapeutic candidates currently being commercialized by pharmaceutical companies. John's promotion to president of Loomless Pharma is in recognition of his vital contributions in advancing Loom 201 for the treatment of PGHD as part of the succession planning conducted by the company and our board of directors. John's extensive clinical and management experience at the NIH and in the pharmaceutical industry, including his contributions during his tenure at Loomis Pharma, positioned him well to serve in this new role. And on Tuesday, we announced the expansion of our management team with the addition of Dr. David Karp as our new chief medical officer. David is an endocrinologist, an academic, an accomplished biopharmaceutical executive, and is currently an adjunct clinical professor in the Division of Endocrinology at Stanford University School of Medicine. David has 35 years of experience in the field of endocrinology, and as a biopharma executive, he has 30 years of experience in all facets of biopharmaceutical clinical development, from preclinical through phase four. regulatory interactions with both the FDA and international agencies, and marketing and launch activities. Most recently, he was Vice President, Clinical Development and Clinical Operations for Ascendus Pharma, where he was responsible for several compounds in clinical development, including Transcon GH, long-acting growth hormone, for once-weekly treatment of growth hormone deficiency. He previously served as chief medical officer for several biotechnology companies, and he has also held leadership positions in clinical research and endocrinology at both Roche and previously at Merck, where he gained familiarity with our therapeutic candidate, LUM201. And we look forward to David's guidance and assistance in advancing LUM201 toward commercial approval. So welcome, David.
spk04: Thank you, Rick, for your kind introduction. And good afternoon, everyone. I only wanted to add how excited I am to join the Lumos team. As Rick mentioned, I'm very familiar with Loom 201 asset from my time at Merck. Based on my experience treating growth hormone deficiency and my experience developing a long-acting growth hormone for patients with GHD, I believe that Loom 201 as an oral therapy is potentially a breakthrough advance in this therapeutic area. Loomis has a well-developed clinical plan to advance this asset, and I look forward to working with Rick, John, and the rest of the team to execute on our clinical strategy.
spk17: Thank you, David, and again, welcome. Before turning the call over to John for an update on our clinical programs, I just want to highlight common themes in these appointments. and that is the view held by many experts in endocrinology of the potential of LUM201 to be a breakthrough oral therapy for the treatment of PGHD. On the strength of that potential, we believe LUMUS has put in place the talent and expertise needed to advance this asset and realize its full commercial expectations. So with that, I'll turn the call over to John.
spk13: Thank you, Rick, and welcome, David. We're all looking forward to working with you. As Rick mentioned earlier, we provided updated guidance on our clinical programs on our call on July 21st. As a reminder, the six-month primary outcome data readout for our Oral Growth 210 trial is now anticipated in the second half of 2023. The extension was prompted by an initially slower pace of site initiation due to ongoing COVID-19 restrictions at certain international sites. and a disruption of the typical referral patterns for this patient population. We further expect referral patterns to normalize as children return to school and communities adapt to the persistent presence of COVID-19. As we continue to open new clinical trial sites for this trial, we are beginning to see the pace of screening and enrollment increase as compared to the first half of this year. Separately, we also announced the extension of the protocol for the OrgGrowth 210 trial for an additional six months on therapy in response to the FDA's request for 12 months data prior to the initiation of our previously planned three-year long-term extension study, the OrgGrowth 211 trial. Again, the primary outcome data readout of the OrgGrowth 210 trial remains at six months on therapy. This OrgGrowth 210 protocol extension mirrors the previous plan for the first six months of the Oral Growth 211 long-term extension study. We believe the extension of this trial will provide a more robust data package for future regulatory violence. The Oral Growth 211 trial was proposed to capture long-term treatment effects for patients who responded to LUM201 in previous trials. However, that study was not on a critical path for the initiation of a phase three trial. We announced the initiation of our Oral Growth 212 trial in Q2. We subsequently announced the proposed extension of the treatment period of this trial from six months to 12 months. Bar Growth 212 is a small, single-site, open-label trial to illustrate the pharmacokinetic and pharmacodynamic effects of LUM201 in PGHD and replicate the pulsability data that currently exists for LUM201 in adults and in a small subset of children from the Phase 2 O2O trial. We also decided to extend this trial to capture additional PK, PD, and height velocity data. The PD pulsatility assessment will continue to be assessed at six months on therapy. The trial was initiated in June and is currently enrolling patients. To recap, both the ORA Growth 210 and ORA Growth 212 trials will provide data readouts at six months on therapy as was originally planned. The extensions to 12 months of therapy will enhance the supporting data package for our overall LUME 201 program. Furthermore, we do not anticipate these protocol changes on a standalone basis to extend our clinical development timeline. With that, I will turn the call over to Lori, who is joining us for the first time in her new role as CFO for a review of our second quarter financial results. Lori?
spk20: Thanks, John, and good afternoon, everyone. We ended the second quarter on June 30, 2021, with cash and cash equivalents totaling $107.7 million, compared to $98.7 million on December 31, 2020. Cash on hand as of the end of Q2 2021 is expected to support operations through the six-month data readout from both the ORA Growth 210 and ORA Growth 212 trials. We had research and development expenses for the second quarter of $4.1 million. an increase of $1.4 million over the same period in 2020. The change is primarily due to an increase of $1.8 million in clinical trial and contract manufacturing expenses, offset by a decrease of $.4 million in personnel and operational expenses. Our general and administrative expenses for the second quarter were $4.6 million, an increase of $.4 million compared to the same period in 2020. This increase is due to an increase of $1.1 million in personnel-related expenses, primarily due to the recording of severance expenses for a former CFO, offset by decreases in legal consulting and operational spend of $0.7 million. The net loss for the second quarter was $8.7 million, compared to net loss of $5.4 million for the same period in 2020. We end at Q2 2021 with 8,357,391 shares outstanding. And with that, I will turn it back to Rick for closing remarks.
spk17: Thank you, Lori. So before taking your questions, I just want to say that we have advanced our discussions with key opinion leaders in our Clinical Scientific Advisory Board to expand our LUM201 platform. We are actively reviewing the pathway for LUM201 and a certain subset of effective patient's and other potential indications, including Turner syndrome, Prader-Willi syndrome, idiopathic short stature, and for children born small for gestational age. We're confident in our belief that Loom 201 represents a pipeline and a product, and look forward to advancing the next phase of the Loom 201 life cycle. And we'll provide further updates when we're ready to discuss comprehensive clinical plans. In addition, We continue to evaluate select rare disease assets under consideration to add to our product portfolio. And as I stated before, we're being highly selective and judicious in our process and we'll focus on our decisions on what provides best long-term value creation for our shareholders. The enhancement of Loomis Pharma's leadership team and encouraging recent trends in screening and enrollment for both Oribro 210 and Oribro 212 trials are positive developments for the company. John, David, and Mark each bring significant clinical development and management expertise to their new roles with the company and strengthen our ability to execute on our clinical and corporate strategy. We believe that as schools reopen and pediatrician visits resume, we should see increased referrals and accelerated enrollment in our trials this fall. So with that, operator, we're now ready to take questions.
spk24: Ladies and gentlemen, if you have a question at this time, simply press a star, then the number one on your telephone keypad. If your question has been answered or you wish to remove yourself from the queue, Please press the pound key. Again, please press the star one to ask a question. One moment, please, for our first question. Your first question comes from the line of Yasmin Rahimi of Piper Sandler. Your line is open.
spk23: Hey, this is Felicia on for Yaz. Thank you so much for taking your questions. So we have two for you this morning. First, do you guys think that you'll be opening additional sites to maintain enrollment timelines? And if yes or no, could you please just provide a little bit of color on that? And next, we were wondering if your current cash balance is sufficient to build up your pipeline while also getting to the finish line for ORA Group 210 and 212 studies. And should we be expecting pipeline expansion of Loom 201 in 2021 or 2022? Thank you.
spk17: Well, the first question, I believe we've anticipated for quite some time the concern over COVID-19. So we did a site expansion to about 52 sites quite some time ago. And as I mentioned earlier, the majority of those sites are open, and we're rapidly advancing and trying to open the rest of those sites. And also, I think there's a very broad geographic distribution. So, once again, I think that's going to help us too. But beyond to about 50 or 52 sites we have planned right now, I think that's going to be sufficient to meet our enrollments. And in terms of cash, we're in a very strong position. And as Lori said, we can get a readout on our 210 and 212 studies. It also means we're going to have to be that much more judicious in our review of anything that we want to bring into the company. This may open up us to looking at assets that are more in the preclinical or actually close to the clinic. But at the same time, I want to emphasize we have a robust process in place and we're being very careful and judicious in our review of anything that we want to bring in as an asset.
spk24: Thank you. Your next question comes from the line of Charles Duncan of . Your line is open.
spk09: Yes, good morning. Thanks, Rick and team, for the update as well as taking my call. Congrats on all the new joiners, if you will, on the management team. I had a couple of questions. First of all, perhaps for John, in terms of the 210 study, I guess I'm wondering if you could remind us of the screen versus enrollment rates that you're looking for, the screen failure rates given the PEMM. And it seems like, and just maybe help me understand the second half of 23 data, it just seems like a year and a half to enroll this study is long. So is it perhaps that you're being overly conservative?
spk06: John, go ahead.
spk13: So we can talk first about the screen failure rate. So our our assessment of the screen failure rate or the percentage of patients with a PGHD diagnosis who are PEM positive, which I think is the important part here. We, you know, as we've published recently in the Journal of Endocrine Science, we did an analysis of a very large 20-year-long multinational phase four study called the Genesis Data Set, which is based on Billy's experience with their recombinant growth hormone product. And we were able to apply our PEM cutoffs to that data set and generate a much broader and a large data set, a very nice assessment that, you know, about 60% of the patients would be PEM positive. Obviously, we're working on a much smaller scale than that data set as we do screens for our study. So we don't, you know, we don't expect to have data that – that is going to be as telling as the Genesis data set. So I think that's the screen failure set. And then for the second, for the re-guiding, really, it isn't about screen failures. It really isn't about anything other than just a slow recruitment that is based primarily, well, a slow process that's based primarily on delays that were primarily attributed to COVID-19. I would say that I think we are being conservative. We want to guide the market on a timeline and a pace of patient recruitment that we think is achievable, and we will work very hard to not only achieve that, but do all we can to beat that.
spk14: Okay.
spk17: And, Chas, I might add, Chas, I mean, there's no question, there's a concern in everybody's mind about COVID-19 and its current status. We want to be conservative in our prediction at this stage, but certainly believe that with the number of new screen patients, it's encouraging.
spk09: Okay, and then if I may, and I apologize up front, but Dr. Karf, I'm going to put you on the spot, and that is that, you know, basically a question of why join LUMO, and really what I'm interested in, I'm particularly intrigued with the secretagogue and pulsatile release of growth hormone and how that may be different than, say, a simple replacement therapy. And I'm not being dismissive of the convenience and compliance benefit, but I'm just kind of wondering mechanistically if you could provide your thoughts on those different mechanisms.
spk04: Well, I could not agree with you more, actually. So what I have been attracted to with the molecule I knew as 677 is precisely that. I mean, talk about a physiological replacement. It's been known for a long time that once weekly, once daily, neither one of those modalities actually recapitulates the normal growth hormone access, which is basically a very low basal with spurts of growth hormone, mostly at night, multiple peaks. And I was struck when I was at Merck with, I mean, here was an oral agent you could take in the morning, you could take it in the evening, and what it did was simply enhance the normal growth hormone peaks. I mean, talk about a marvelous design of a drug. Now, when I was at Merck, Merck was all about large populations. They were the antithesis of a company that was interested in a rare orphan indication. So this was a beautifully designed drug, and they wanted to try to search out a big population and they chose sarcopenia in the elderly, which unfortunately lacked and I think continues to lack a registration endpoint. So that was their attempt, I think, to make a large market like, you know, hypertension, hyperlipidemia, osteoporosis out of this molecule. But they had enough faith in this that they did license it to a colleague of mine, Michael Thorner, for development in PGHD. And I'm very excited about this. Now I'm, I'm bullish about trans con growth hormone. I'm a little bit less bullish, but reasonably bullish about topacitin. But what I see, I mean, when I retired from Ascendus last September, I was very happy seeing my 600 patients at Stanford and doing some consulting. And I really did not have any intentions at that time of getting back into a full-time position. When Rick called me and I realized what this molecule was, I mean, what excites me is that I've been able so far to bring three effective, well, I've committed three substantial NDAs and brought two effective drugs to patients and very soon a third drug to patients for a medical need. And I view this opportunity with Lumos as my last great chance of bringing a really effective drug to a significant population. unmet need in patients. I think that for the appropriate patients, this will be a very, very good treatment of their growth hormone deficiency. Did I answer your question?
spk09: Yeah, absolutely, and really appreciate all the color. I'm looking forward to seeing it move forward as well. Last question is for Rick, perhaps on strategy issues. I guess you mentioned the pipeline, and it would seem to me that you have kind of your hands full with Loom 201, and it's a promising candidate. And I guess I'm wondering how you look at it in terms of being a pipeline and a product with other indications relative to in-licensing opportunities. It would seem to me that the capital markets have kind of, competed against you with regard to the latter aspect of that or route in terms of that strategy. And I'm wondering if there's a favorite leading additional indication that you would want to highlight for Loom 201.
spk17: That's a good question, Chas. And I want to say that, you know, first and foremost, our priority remains to stay focused on executing on our Wargrove 210 study and 212 study plan. I think that also evaluating other indications of our compound because of its unique mechanism of action would likely be beneficial and provide, and we're going to provide updates at the appropriate time when those plans are more fully cooked. But, obviously, this is a unique opportunity with this compound, and we are engaging all the important KOLs out there, and as we lay out our plans, you'll be the first to know. I think it's prudent for us, though, to continue based on our extensive backgrounds in the rare disease space. to continue to look for opportunities. Are there other assets out there? Now, as you know, we've been doing this for quite some time, and we haven't pulled the trigger on anything, and that's because we have been as careful and as judicious as we possibly can because anything that we do, we want to make sure that it's a strong value proposition for our shareholders, and that's all there is to it. But I think it would be imprudent for us not to have a process in place to find a unique asset, just like the unique assets that we're developing in Loon 201.
spk09: Very good. Thanks for taking my questions and appreciate the update. Okay.
spk24: Your next question comes from the line of Ed White of HT Wingright. Your line is open.
spk11: Good morning. Thanks for taking my questions. So maybe start with a question for Laurie. I'm curious as to R&D spend going forward. You know, we would expect to see the trend going up as the trials are initiated. Sequentially, R&D expenses were down. I'm just wondering if you can give us some thoughts on how we should be thinking about the next two quarters for R&D expense.
spk20: Sure, and thank you for the question. So, sequentially, compared to Q1, we had some severance costs that were recorded in Q1 for the departure of our former CMO of approximately $1.4 million. I would expect and anticipate that our Q2 spend is reflective of what we will expect going forward with increases as we advance our clinical trials and path forward towards a Phase III trial.
spk08: Great. Thank you.
spk11: Does that answer your question? John, congratulations on your new role in Tidal. I'm just wondering how your role is changing within the company, if you can give us any thoughts to that.
spk13: Well, thank you for the congratulations, Ed. And really, I think, you know, the focus will be will continue to be similar to the role I have been playing. I think it's a promotion where I'll be able to, you know, to focus on both the operational and scientific aspects of the company and making sure that we deliver on all the aspects of our guidance.
spk11: Okay. And maybe just the last question that I have, we've been talking about enrollment in the 210 study and I'm just curious on two things. Now that we're seeing, you had mentioned that as children go back to school, there could be perhaps more pediatrician visits. We could see more interest in the studies. I'm just wondering how you're thinking about the Delta variant and is that going into your thought process on enrollment trends And then when could we see you give us an update on what the actual enrollment are in these two studies? Will you be telling us when you get halfway there or maybe perhaps over the next few quarters, do you plan on giving us updates on how the enrollment is going? Thank you.
spk17: Yeah, I'll start with that, Ed. You know, we haven't stated at any time in guidance that we're going to address when we're, you know, quarter or a half or three quarters of the way through. We just have our noses to the grindstone trying to make sure that we're doing everything we can to make sure this study goes as well as possible. Perhaps we would address the market at a more appropriate time, but right now it's a little bit too early on.
spk25: And John, could you take his first question regarding the Delta virus impact?
spk13: Yeah. So, you know, I think the Delta variant is much like the Alpha variant was last year, right? So the way we plan around that is, you know, right now, as Rick has mentioned, we have more than 50% of our sites up and running. Part of our delay in the past was as much about getting sites up through delays of getting through IRBs and, you know, interacting with lawyers and contract people at clinical sites who are working from home, which seemed to add an enormous amount of time. Uh, and I think we have those sites up and running. And, uh, and as we had mentioned in the past, we have, uh, sites spread across various geographies in the U S but also internationally in Eastern Europe and in, um, and in Australia and New Zealand. And I think the combination of those two things are really going to help us, uh, kind of juggle, you know, move from area to area where, uh, you know, the pandemic waxes and wanes. But I also think regardless, at this point, communities are starting to learn how to live with COVID-19. And I don't, it's hard to imagine that we will go through another school year with the, you know, with remote learning for the entire year. I think we will find ways to be successful in educating kids which will stimulate trips to the pediatrician and referrals to the pediatric endocrinologist.
spk04: If I could just endorse what John just said, it absolutely mirrors my experience getting the Transcom PTH studies enrolled under COVID, which is exactly what he said. The delay is getting sites initiated. But even the initial epidemic delay did not have a meaningful impact because once the kid is diagnosed, I mean, the parents really want this kid treated. So there was really minimal impact in a subject coming into the site. All of the delay was in really getting the site initiated and all the hoops you've got to go through to get a site initiated. So I just want to endorse what John said about that. I expect that to happen with the LUM201 trial as well.
spk11: Great. Thanks for taking my questions. Thank you, Ed.
spk24: Your next question comes from the line of Yun Yang of Jefferies. Your line is open.
spk03: Hi. Good morning. Thanks for taking my question. This is Suji calling in for Yun. So for Dr. Cao, I just have a quick one question. So do you think that before starting Bay Street, the regulatory agencies might ask ask for additional study where you would have to compare the efficacy of LUM201 in PM positive versus PM negative to show that the LUM201 has selective advantageous activity in the positive population. Thank you.
spk17: I'll tell you this. Since Dr. Carpenter just arrived, I think I'm going to let John start with that question and perhaps have David finish it.
spk04: I'm comfortable making a stab at that, but go ahead.
spk13: Yeah, so I think we have Merck's previous data set that we've analyzed, and we've separated out PEM positive and PEM negative, and there's a dramatic difference, and obviously we've published on that. So I do think, actually, that the PEM positive data prospectively selecting those patients and bringing them into our ongoing trial is really going to be the proof that the FDA and I think the market will appreciate as well to show that this enrichment strategy really does work effectively. And remember, this enrichment strategy is based on physiology, right? So the kids who are PEM negative, they simply don't have an active axis. They don't have enough somatotropes. They can't store growth hormone. They're not going to be able to respond to our drug, which is just going to stimulate the release of endogenous growth hormone. So it seems mathematical when you look at how we generated that data, but it is tied to physiology. And I think we can make that case very effectively to regulators as well as in a scientific setting to show that as long as we can show the PM positive kids are growing and and we're able to enrich that population of responders with our cutoffs. I think that will be sufficient evidence.
spk17: And David, do you want to add anything?
spk04: Yeah, I'll just say that on the one hand, this is my second day at the company, but on the other hand, based upon my 30 years of experience dealing with the Endocrine Metabolic Group, I can say with, I think, some authority the following. First of all, I think the agencies, like third-party payers, would be absolutely thrilled at the selection process to identify the best patients for the drug. I think that the phase three trial, if I had to guesstimate, would basically be about a two to one randomization between PEM selected patients for the LUM201 group versus unselected patients for daily growth hormone. And it'd be a 12 month study and the likelihood of having to go for PEM positive versus PEM negative I would handicap as close to zero as to be negligible. There's no rationale for that. I would be willing to bet a lot of money it'll be PEM selected versus unselected for daily growth hormone. That'll be the phase three study. You shall see.
spk02: Great. Thank you.
spk24: Your last question comes from the line of Katherine Novak of Jones Trading. Your line is open.
spk19: Hi. Thanks very much for taking my questions. I had a couple on pipeline expansion possibilities. First, as you think about potential BD opportunities, what are some specifications that you're looking at in terms of assets that you think make sense in your portfolio?
spk17: Catherine, thanks for your question. And, you know, our criteria is really always included, starting with a good strategic fit, you know, a strong scientific rationale, and really a strong value proposition for our shareholders. We have been willing to look at the earlier stage opportunities and are going to continue to do so on a very selective basis. But, you know, and above all, you know, we'd like to stay in the endocrine space. I mean, we have a lot of experience, and especially with David arriving, I think the opportunities are going to expand for us to look at. And our process, I think, is a very good one. But as you know, we haven't pulled the trigger on anything because it hasn't met our really careful and judicious criteria.
spk19: Yeah. Thanks. That makes sense. And then when you're thinking about expanding LUM201 into these other indications where growth hormone is approved, do you have an idea of what subset of patients might benefit from the LUM201 unique mechanism of action?
spk16: And, John, why don't you start with that?
spk13: So the only growth number deficiency population that Loom 201 has been tested in right now is in the PGHD population, right? So that's the population where we have a correlation of their PEM tests back to growth, right, the outcome. So each of the other indications that growth number is approved in has, you know, unique facets to the etiology of the disease, right? Sometimes there are things There is some growth number deficiency and there is some downstream modulation of receptor activity or IGF-1 activity. So that's really where we've been spending our time is looking through all those facets and trying to understand really the overall potential of LUM201 in that area and also thinking through how would pulsatility affect activity. each of those individual disease states versus, you know, the bolus, right? So we have good ideas of how that might have an impact in PGHD, and we want to understand that for some of the other indications. And then, you know, once we get past that, we do try to assess how frank is the growth hormone deficiency itself in each one of these indications, and that really can be an indicator of where our mechanism of action of stimulating the release of endogenous growth hormone is going to play in each one of these indications. And that's really what we're building that data set through, you know, mining information after a history date about each one of these diseases right now to put together our prioritized list of where we're going to go forward.
spk19: Thank you very much.
spk07: Thank you, Catherine.
spk24: Thank you. I'm showing no further questions in the queue at this time. I'll hand the call back to Mr. Hawkins for closing remarks.
spk17: Well, we thank you for joining us today, and we look forward to keeping everyone apprised of our program over the course of the year. Thank you very much.
spk24: Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.
Disclaimer