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spk03: Good afternoon and welcome to Loomis Pharma's 2022 Financial Results Conference Call.
spk06: Currently, all participants are in a listen-only mode.
spk03: We will conduct a question and answer session. We'll follow at that time. As a reminder, this conference call is being recorded. We'll now turn the call over to Lisa Miller, Senior Director of Investor Relations. Ma'am, please go ahead.
spk07: Good afternoon, and welcome to Loomis Pharma's 2022 Financial Results Conference Call. Currently, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. As a reminder, this conference call is being recorded. I will now turn the call over to – oh, sorry, so much – Thank you, Operator. Before we proceed with the call, I would like to remind everyone that certain statements made during this call are forward-looking statements under U.S. federal securities laws. These statements are subject to risks and uncertainties that could cause actual results to differ materially from historical experience or present expectations. Additional information concerning factors that could cause actual results to differ is contained in our periodic reports filed with SEC. The forward-looking statements made during this call speak only as of the date hereof, and the company undertakes no obligation to update or revise the forward-looking statements. Information presented on this call is contained in the press release we issued this afternoon, which may be accessed from the investors' page of the company's website and in our Form 10-K when filed. Speaking on today's call will be Rick Hawkins, CEO and Chairman, and Lori Lawley, our CFO. John McHugh, our President and Chief Scientific Officer, and David B. Karpf, our Chief Medical Officer, will join for questions and answer sessions after the call. I will now turn the call over to Rick.
spk10: Thank you, Lisa, and good afternoon, everyone. After the market closed today, we issued a press release announcing our 2022 four-year financial results and clinical updates. On today's call, we'll keep our prepared remarks brief so we can maximize time available for Q&A. I touched on the highlights in the year and in recent weeks before turning it to Lori for a review of our financial results. Then John McHugh and David Karpf will join us to answer your questions. So let's begin. As we reported this afternoon, 2022 was a year of significant progress in advancing our oral therapeutic candidate, LUM201, in idiopathic pediatric growth hormone deficiency, or PGHD. We are pleased to announce patient enrollment has been completed in both our Oral Growth 210 and Oral Growth 212 trials. Given the obstacles that we faced with the impact from COVID, the Ukraine-Russia conflict, and a fire at our single Oral Growth 212 site, we are elated to be able to announce that the last subjects in both trials have been enrolled, and we now expect to report primary outcome data on 82 subjects in the ORAC Growth 210 trial and 22 subjects in the PKPD ORAC Growth 212 trial. This accomplishment is a real tribute to the clinical team, and we expect to announce top-line results from both trials in the fourth quarter of 2023. Now, as most of you know, we announced the results of an interim analysis from these trials in November, and I'll just briefly recap the data and main conclusions now. Results from these interim data on approximately 10 subjects per arm at six months on treatment of the oral growth 210 trials showed annualized height velocity for 1.6 mg per kg of LUM201 cohort of 8.6 centimeters a year. meeting expectations and prior guidance based on comparisons to several large historical databases, which showed annualized high velocity of 8.3 to 8.6 centimeters a year for idiopathic or moderate PGHD subjects treated with growth hormone. Importantly, a durable response to LUM201 was observed at 9 and 12 months. No treatment-related severe adverse events were observed. There were no trial dropouts due to adverse events, and no adverse safety signals across the entire dose range evaluated. And as we stated previously, we believe these data support the selection of a dose of 1.6 mg per kg per day for a pivotal phase III PGHT trial, planning for which is now underway. As we mentioned previously, the imbalance in baseline characteristics that contributed to the faster growth seen in the growth hormone control arm should be minimized with full enrollment of approximately 20 subjects per arm. The interim data from our ORGO 212 trial were supportive of the ORGO 210 trial data. And recall that the subjects enrolled in these trials are an enriched population identified through our predictive enrichment marker or PEM strategy for selecting idiopathic or moderate PGHC patients with the potential to respond to LUM201. Each patient is given a single dose of 0.8 mg per kg of LUM201 during the screening process to determine if they meet the PEM cutoff prior to enrolling in the org growth trials. The primary endpoint for our growth 210 trial, now anticipated for the fourth quarter of 2023, is to confirm our PEM strategy and finalize the optimal dose selection for a pivotal phase three trial. Also in November, we hosted a key opinion leader webinar featuring Dr. Andrew Dauber of Children's National Hospital and Dr. Fernando Casorla of the University of Chile, both distinguished thought leaders in the field of pediatric endocrinology for discussion of the interim data. These clinicians shared their insights about oral LUM201 and its potential to treat children with idiopathic PGHD. They highlighted that the interim clinical data support the potential for LUM201 as a welcome oral alternative to current therapies that require frequent injections. A replay of this event is available on our website for those who wish to review the interim results in greater detail. In our press release this afternoon, we announced the acceptance of two abstracts for presentation at the 2023 International Meeting of Pediatric Endocrinology, or MPAE, which will be held in Buenos Aires, Argentina, March 4th through the 7th, 2023. Additional data from the interim analysis of our oral growth trials will be presented in an oral and a poster presentation during the conference. And we'll announce the details of the data update once presentations have been made. Turning to other developments, our previously announced clinical collaboration with Dr. Laura Dichtel in Massachusetts General Hospital to explore the potential of LUM201 in non-alcoholic fatty liver disease, or NAFLD, is continuing. This investigator-initiated pilot study was recently supported by data presented by Dr. Dick Tal at the Endo2022 medical conference, where she presented positive results from a trial evaluating injectable growth hormone in NAFLD, which supported the assessment of oral to LUM201 in the same indication. Enrollment in the Massachusetts General Pilot Study of LUM201 in NAPL is ongoing. And now in today's release, we also announced an important development involving our intellectual property position for LUM201. In November 2022, we filed a patent application titled Compactable Oral Formulations of Ibutamorin, which contains claims directed to certain improved LUM201 drug product formulations we intend to utilize in our Phase III trial and ultimately commercialize. The application is currently pending and is granted would provide composition of matter protection through November 2042 for the commercialized version of Loom 201. Now, before I turn it over to Laurie for a review of our financial results, I want to provide an update on our evaluation of next indications for Loom 201 beyond PGHD. We've done substantial work internally and have consulted external advisors and market participants to assess the potential LUM201 and other indications, and geographic regions. With narrative focus for the next indications include idiopathic short stature, or ISS, with a focus on the Asian markets. Growth hormone use in these markets have been growing by double digits in the last five years, approaching $2 billion in total, with ISS being a major driver of this growth. Our narrow target indications, include Plotter-Willi Syndrome, where we see an attractive global opportunity for Loom 201. And while we plan for the next steps for these opportunities, we remain committed to ensuring prudent use of our cash is focused on advancing our core program in PGHD. So with that, I'll turn it over to Lori for a review of our financial results. Lori?
spk05: Thank you, Rick. Lumos Pharma ended the year on December 31, 2022, with cash, cash equivalents, and short-term investments totaling $67.4 million, compared to $94.8 million on December 31, 2021. Total cash spent during 2022 was $27.4 million, which is below the guidance given for the year of $8.5 to $9.5 million cash earned per quarter. primarily due to lower-than-anticipated clinical trial costs incurred as a result of closing certain sites in Ukraine and Russia, lower-than-anticipated travel costs due to the continued impact of COVID in the first half of 2022, and a continued focus on discipline spend management. The company expects an average cash use of approximately $9.5 to $10.5 million per quarter through 2023 as we ramp up our efforts to prepare for a Phase III clinical trial. Cash and short-term investments on hand as of December 31, 2022, is now expected to support operations into the third quarter of 2024, extended from Q2 of 2024. Research and development expenses were $17.9 million, an increase of $1.6 million for the year ended December 31, 2022, compared to the same period in 2021, primarily due to increases of $1.1 million in clinical trial and contract manufacturing expenses 0.5Million in consulting expenses and 0.3Million in personnel related expenses. Offset by decreases of 0.2Million in stock compensation expense and 0.1Million in operating expenses for supplies, depreciation and rent. General and administrative expenses were 15.7Million. An increase of 0.4Million for the year ended December 31st, 2022 as compared to the same period in 2021. primarily due to increases of $0.9 million in royalty expenses, $0.4 million in travel expense, and $0.3 million in other expenses, offset by decreases of $0.4 million in personnel-related expenses, $0.4 million in stock compensation expense, $0.3 million in consulting expenses, and $0.1 million in operating expenses for supplies, depreciation, and rents. The net loss for the year ended December 31, 2022, with $31.1 million compared to a net loss of $32.4 million for the same period in 2021. We ended P4 2022 with 8,267,968 shares outstanding. Additional information may be found in a quarterly press release filed this afternoon. And with that, I will turn the call back to Rick to conclude for us.
spk10: Thank you, Lori. And to recap, our Phase II clinical trials evaluating oral LUM201 and PGHD are now fully enrolled. and we're in a position to report top-line data from both studies in the fourth quarter of 2023. Our confidence in these trials is based on the results of the interim data reported last November and corroborated by expert opinion leaders in growth-related disorders. Additional data from our oral growth trials will be presented later this week at the MPA conference, and we anticipate additional data presentations at other major medical conferences over the course of 2023. We continue to support the exploration of Loom 201 in the treatment of NAFLD through a pilot investigator-initiated trial, and we have narrowed our focus for future indications for Loom 201 to two compelling opportunities and attractive markets. In addition, by prioritizing our PJC program and being conservative with our cash usage, we're extending our current cash runway guidance into the third quarter of 2024. We also submitted a patent application for a novel drug product formulation of LUM201, which, if approved, will extend composition of matter protection for the commercialized version of LUM201 through November of 2042. So 2022 was a productive year for LUMOS, and we're positioned for 2023 to be even better. We believe we're poised to demonstrate that early administered LUM201 has the potential to disrupt the worldwide growth hormone market, has been dominated for almost 40 years now by injectable products. We're excited to continue to advance our programs and look forward to disclosing pipeline data in the fourth quarter of 2023. Thank you all very much. So, operator, we're ready to take questions.
spk03: Thank you. If you have a question at this time, please press star 1-1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1-1 again. One moment while we compile the Q&A roster. Our first question comes from the line of Charles Duncan with Cantor. Your line is open. Please go ahead.
spk08: Hi. This is for Charles. Hi, Rick and team. Congratulations on the enrollment completion for 210 and 212 studies. So I have a question regarding patient screen failure rate. Now that you've completed enrollment in 210 and 212, can you give us a sense of what the screen failure was and how does that compare to what you expected? And yeah, we'll start off there. Thanks.
spk10: Yeah, you know, I think our failure rate is quite low because obviously we use our predictive enrichment marker or PIM strategy to prescreen these patients, and our investigators are really well-versed in the patient population we're looking for, and that is the moderate idiopathic patients. And, David, do you want to add anything to that?
spk09: Yeah, it was the screen rate. Yes. Yes, thank you for the question. Rick is exactly right. The screen failure rate has been less than we had predicted in the protocol, and it's largely driven by a reduction in the screen failure because of not passing the PEM test, because the investigators are very used to diagnosing idiopathic GHD. And so, because they're screening that population, we've had a marked reduction in the anticipated screen failure rate for PEM failure. And the rest of the reasons for screen failure have been as predicted. So, overall, the screen failure rate is lower than we had predicted. Okay.
spk08: And then, in terms of the parameters that define the PEMs, do you believe they're going to remain the same for Phase III, or is there a possibility of an adjustment?
spk09: Sure. The parameters are well-defined, and those will not change, which is having a baseline IGF-1 level In absolute terms, it's higher than 30 nanograms per mil and showing at least a five nanogram per mil growth hormone level after receiving the low dose of LUM201. What will change in phase three is likely how we do the PEM test, which will be simplified. So it will not be like a stem test. It will be a single dose of LUM201 and a single blood test one hour later for the dynamic part of the PEM test. So that will be simplified, but the parameters will remain the same.
spk08: Okay.
spk09: Does that answer your question?
spk08: It does indeed. Thank you. So I, you know, I know you'll be presenting the data at conferences in the near term, but can you give me a sense of, you know, how the data presented last quarter, you know, has been received by KOLs and more broadly by the endocrinology community? And how does that sort of translate to enabling a phase three study? Do you get a sense that there's an appreciation for the PEMS approach? and allowing for more, let's say, precision medicine approach?
spk09: David, do you want to answer that question? That's a very good question. The short answer is yes to that. What I would say is that the investigators and the key opinion leaders are all very excited about the interim data, because to them, it really proves that LUM201 does what it's supposed to do. So, we have very, very engaged investigators. They like the fact that it It's natural, and it enhances the normal palpations of growth hormone as opposed to exogenous hormone. And so they remain very excited, very committed, and are looking forward to working with us in phase three.
spk10: Okay. I want to add, too, that our clinical and scientific advisory board, a group of really esteemed pediatric endocrinologists, are also well-versed in the results, and those results were well-accepted, let's put it that way.
spk08: Hi, good to hear. You know, look forward to the upcoming presentations. So, if you don't mind, just squeeze one last question in. So, can you just sort of help me understand the rationale for expanding into idiopathic short stature? You know, why would 201's mechanism of action, you know, have an effect in that patient population?
spk10: David, why don't you talk about the mechanisms of action and ISS?
spk09: Sure. By increasing baseline growth hormone AUC, it is very likely that ISS, patients with ISS will accelerate their high velocity. It may be similar to with growth hormone where you don't get quite the same high velocity as you do in more severe GHD kids. But it should, I see every reason to expect it should be comparable to the effects of growth hormone.
spk12: I think if I may add one thing to that, many of those kids, almost all those kids would be expected to pass our PEM test, right? And they all have the capacity to release growth hormone endogenously upon stimulation. And that's a key part of why we're thinking about that population.
spk09: Right. I mean, the Merck data showed in normal adults, you can substantially increased from baseline. Glyphamone and IGF-1. So there's every reason to expect that would apply to kids with ISS.
spk08: Okay. All right. Thank you very much. Thank you for taking our questions. Thank you.
spk03: Thank you. And one moment for our next question. Our next question comes from the line of Catherine Novick with Jones Trading. Your line is open. Please go ahead.
spk02: Hi, good afternoon, guys. Congrats on completing enrollment. I want to talk a little bit about, you know, loom 201 compared with the efficacy of loom 201 versus recombinant growth hormone. I know that you're not necessarily looking at efficacy as a primary in the phase two, but, you know, I'm curious about, what's clinically meaningful to prescribers? You know, hypothetically, let's say you end up with loom 201 having 8.5 centimeters AHV and G8 showing 10, as maybe expected. How important is that difference in the first year versus other things like safety, cost, convenience, durability, when it comes to how prescribers are looking at these different regimens?
spk10: Well, thank you for that question, Catherine. John, I'm going to let you start with the answer, and David maybe finish, and I'll probably chime in too. So go ahead, John.
spk12: Sure. So thanks, Catherine. And, you know, I think first we've given you kind of the general feedback from the KOLs and the investigators on our trial about their, you know, kind of how they're feeling about Loom 201 and thinking about moving forward. And that's really because the efficacy of the drug is actually running at a level of growth that we had anticipated and we had foretold based on historical data. And we've also, at our interim, talked about the safety profile of this drug being quite good. And I think those two key things, meeting the expectations on the efficacy of our drug and no safety concerns, in this population, I think are key contributors to the excitement that people have. They also have an understanding of what the expectation of growth hormone growth in this kind of less severe, moderate idiopathic growth hormone group of kids is. And, you know, most of them acknowledge that, you know, we have a couple of kids who are growing outside of that range. So I think the key thing really is just to focus on the efficacy of a drug Because as you said, phase two is not powered to show non-inferiority anyway. And so I think focusing on the efficacy of the drug and moving forward with that as we plan phase three is kind of the key part that we'd like to spend our time on. David, do you have anything to add?
spk09: Sure. I would just add that to our expert clinical advisory board, pediatric oncologists, their expectation in this population is not 10 centimeters a year with clostromon. It's actually much lower than that, which is why I think they were so encouraged by our results. I share their view, having run the high trial with scitrofa. I mean, it could be barely above 10 centimeters in a more severe population in that trial. So I really expect that growth hormone in this population in phase three will be below 10 centimeters, just as a point.
spk10: Yeah, and just one other point is that, you know, in a true Phase III non-inferiority trial, the last two companies to negotiate that difference with the FDA is 1.8 to 2 centimeters. And obviously, We get a product approved, and we're as an oral once a day. I think that it will be a significant and meaningful place as a treatment in the market as a first oral product.
spk02: Got it. That kind of leads into my second question. You may have answered it a little bit, but I want to talk about how important compliance is when it comes to the efficacy in daily recombinant growth hormone. You know, when we look at what you've cited from the Genesis database, for example, we see AHB that's significantly below control arms in recent phase three studies for growth hormones.
spk00: Does this, you know, this is due to higher compliance or.
spk02: Is there some things like baseline patient characteristics? So, this age more severe growth hormone deficiency that are contributing to that difference.
spk10: If I could yeah, go ahead David. Do you want to.
spk09: Yeah, sure. The Genesis database and the KIG database that we referenced for this, first of all, compliance in the first year of treatment is actually pretty good. And we're looking at the first 12 months of response in those large databases. So, I think that the lower AHV is more because of the population, either PEM positive in the Genesis database or the less severe in the KIG database, which more closely matches the population we'll be studying. then it has anything to do with compliance, quite honestly.
spk10: Okay. And John, anything else you want to add? Is that good? No, that's fine. I agree. Yeah. Okay. Catherine?
spk02: Okay. That was very helpful. Thanks so much for taking my questions.
spk10: No, thank you, Catherine.
spk03: And one moment for our next question. Our next question comes from the line of Yun Yang with Jefferies. Your line is open. Please go ahead.
spk04: Thank you. So assuming good data in the fourth quarter this year, if you embark on a Phase III trial sometime next year, how do you see enrollment taking? How long do you think enrollment would take? When do you think that you would be in a position to actually report the data?
spk10: Well, let me start, David, please. So, first of all, I mean, we're really pleased with the enrollment in this trial in spite of these really difficult times during the, you know, the pandemic and losing all of our sites in Russia and Ukraine. In spite of that, we've done really well. And I tell you that these investigators are all experienced. They've all done many clinical trials, and we certainly have their attention because this is the first oral product they've worked with in their careers. I think there's another set of investigators that we know about who only participate in phase three studies, and they are high enrollers. So I think we'll do well with that. Um, now we haven't really guided the market in terms of when we're going to actually start the phase 3 study. We, of course, would have to have an interface to meeting with the, with the FDA, have them agree on our phase 3 plan and move ahead. But we're doing everything that we can currently. to not only recruit these investigators, and that actually is going to start at the IMPE meeting this weekend in Buenos Aires, but have everything that we can possibly do in advance completed in order to start as quickly as we can. But we haven't given guidance beyond that.
spk04: I see. So when do you think you are going to be in a position to have a Phase II meeting with the FDA? Is the first quarter next year the reason? Is it reasonable?
spk10: John, I'm going to let you answer that question.
spk12: Again, we haven't guided on that publicly, but obviously we have to get through the data, sort it out, put in a, you know, a package and wait for the FDA to grant and schedule that meeting. So, you know, I think there are a lot of factors that lay in there, but we haven't projected that out yet to the public.
spk03: Thank you. Thank you, and one moment for our next question. Our next question comes from the line of Ed White with HC Wainwright. Your line is open. Please go ahead.
spk11: Good afternoon. Thanks for taking my question. So just saying on the potential phase three, I know that you don't really know much about it right now, what you're going to do. But I'm just wondering if you could tell us what you're expecting for perhaps The duration of the study, will this be following the patients for one year? Will it be six months? How are you thinking about that? And also, I assume that you're budgeting out for this, so just any cost expectations, you know, regarding perhaps the size of the trial. And also, the last question will be, since you lost Russia and Ukraine, where do you expect to enroll the most of the patients?
spk10: Okay, well, maybe I'll start and then David, if you'll chime in here. So, you know, we projected, I mean, the regulatory pathway for growth hormone and growth hormone products is really clear. Where we think we're going to be is this will be approximately 160 to 200 patients, same patient population we've been studying in our current study. but we'll randomize 2 to 1 to 1.6 versus the standard dose of growth hormone. All patients will be on drug for a year. We really haven't talked about our budget for that study to date, so I can't really give you any guidance on that. But I think I answered your question maybe a little bit earlier, Ed. And that is the loss of the Russian and Ukraine sites. Yes, that makes a difference. But there's a whole new set of investigators that all of us know who've been in this field for a long time, who have been prolific phase three investigators, actually, many of whom don't even participate in phase two studies. And, you know, we're going to meet, we've met with those folks, we will meet with them at MPEG this coming week, and we'll continue to talk to them and bring them on board. So, most of those sites are in the U.S., but there are other sites around the world that are prolific, you know, investigators in Phase 3 programs.
spk09: And if I could just add, I would say that I've been very pleased to see that the Polish investigators have enrolled more than the Russian-Ukrainian investigators did in the HITE program. So that's kind of a wash there. But also, I enrolled 164 subjects in the HITE trial in 12 months. And that was without the benefit that we'll be having of having high enrolling sites in places like China, which have been really tremendously rapid enrolling growth hormone deficiency trials. So I think that having high enrolling sites in the U.S., including, as Rick mentioned, some really key high enrollers who only participate in phase three, and having similar folks in the U.S., in Poland, in Europe, in Australia, in New Zealand, China, Japan, and Korea, I think that we are very bullish on the enrollment for the phase three trial.
spk12: Okay, thanks, David.
spk11: And perhaps just looking at the other indications you mentioned today, idiopathic short stature and Prader-Willi syndrome, for ISS, would you be looking for an Asian partner and would you be, how are you thinking about the timing to the start of a trial? Would this be a 2023 event or a 2024 event?
spk10: You know, Ed, we haven't guided on the start of such a study. I think it's pretty obvious that there would be a number of partners who would be interested in talking to us. Other than that, I mean, you know, it's pretty obvious that ISS is of high importance and expanding importance around the world, especially in Asia. John, do you have anything to add to that?
spk00: Yep. I think that's a great answer.
spk11: Okay. Thanks, Rick.
spk03: Thank you. And one moment for our next question. Our next question comes from the line of Yasmin Rahimi with Piper Sandler. Your line is open. Please go ahead.
spk13: Hi, guys. This is Lauren. I'm for Yaz. First of all, congrats on completing enrollment. We just want to ask, so could you help us understand, now that enrollment is complete, where you guys ended up in regards to baseline characteristics like age, MPH, height, starting height? And are you confident that having outliers has been minimized? Just some clarification on that. Thank you.
spk10: Good question, Lauren. And John, will you start with that answer?
spk12: Sure. So, you know, we announced our last patients enrolled last night. So we're working on finishing, you know, the baseline characteristics analysis for that data, and we would anticipate releasing that bigger data set at an upcoming medical conference. So I'll just reiterate that in, you know, at our interim analysis, we also released 75% enrolled baseline characteristic data, and we saw a nice shift between 50% and 75%. And the expectations, particularly for age, are that those are going to continue to come closer together. Randomization will be more effective as we go from 75% to 100%, but we're in the process of working through that data now.
spk13: Great. Thank you.
spk03: Thank you.
spk10: Thank you, Lauren.
spk03: And showing no further questions, this does conclude today's question and answer session. Ladies and gentlemen, this also does conclude today's conference call. You may now disconnect. Everyone have a great day.
spk05: The conference will begin shortly. To raise and lower your hand during Q&A.
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