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.
spk00: Good afternoon, ladies and gentlemen, and welcome to Lumos Pharma's first quarter 2021 financial results conference call. At this time, all participants are in a listen-only mode. Later, we will conduct a question and answer session, and instructions will follow at that time. As a reminder, this conference call is being recorded. I will now turn the call over to Lisa Miller, Senior Director of Investor Relations.
spk02: 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 earnings release we issued this afternoon and in our Form 8K, which may be accessed from the Investor Relations page of the company's website. Joining me on today's call are Rick Hawkins, CEO, President and Chairman, John McHugh, Chief Operating Officer and Chief Scientific Officer, Carl Langren, Chief Financial Officer, and Lori Lawley, Senior Vice President, Finance and Corporate Controller. Rick Hawkins will provide a corporate update. John McKee will review the company's lead therapeutic candidate and our clinical trials. And Lori Lawley will wrap up the call with a review of the first quarter 2021 financial results and an update of cash guidance. Following our prepared remarks, we will open the call to questions. I will now turn the call over to Rick.
spk05: Thank you, Lisa, and good afternoon, and thank you for joining us on today's call. After the market closed today, we issued a press release detailing our first quarter 2021 financial results and highlighting our recent clinical and corporate activity. I'm pleased to report that the quarter was a productive one, during which we saw a presentation and publication of new data and analyses supporting the differentiated mechanism of action of our novel oral therapeutic candidate, LUM201, for the treatment of pediatric growth hormone deficiency in patients identified by our predictive enrichment markers. We also continue to advance our clinical programs for LUM201, adding clinical sites for our Phase IIb Oral Growth 210 trial and moving towards initiation of our Oral Growth 212 trial during the second quarter. Finally, we completed the monetization of our priority review voucher, receiving in January the final $26 million tranche of the $60 million due to the company from this sale. With these resources in hand, we believe we are well positioned to advance our Loom 201 programs and explore business development options to enhance our pipeline and deliver value to our investors. And before turning the call over to John McHugh for a deeper dive on progress in our clinical programs, I just want to highlight a few recent events. Last week, we held a key opinion leader, a forum featuring presentations by Drs. Bradley Miller of the University of Minnesota and Fernando Casorla of the University of Chile. This was a well-attended and informative event where Dr. Miller and Dr. Casorla provided an overview of the current treatment landscape in PGAC and outlined the unmet medical needs in this space. As part of his presentation, Dr. Casorla also presented newly released PKPD data from a subgroup of the MERC 020 trial evaluating LUM201 in naive-to-treatment PGAC patients. These data were additive to the peer-reviewed data recently published in the Journal of Endocrine Society, demonstrating once again the potential of our predictive enrichment markers, or PEMs, of baseline IGF-1 levels and peak-stimulated growth hormone levels after a single dose of LUM201 to identify patients likely to respond to LUM201 therapy. Additional data were presented last month at the Endocrine Society 2021 annual meeting known as ENDO, further demonstrating LUM201's unique potential to elicit therapeutic-level response in PGHD patients and in differentiating this molecule from standard growth hormones, Cretagox. These results add to the previously mentioned data, further supporting the approach we are using in our clinical trials. John McHugh, our COO and Chief Scientific Officer, will have more to say about these data in a moment. So as I mentioned, our Phase IIb Oral Growth 210 trial in PGSD continues to enroll patients. Additional clinical sites have opened for enrollment with over 50% of our target number of sites now activated. Our Oral Growth 212 trial, a PK-PD study in PGSD, is expected to be initiated this quarter and with data from that trial anticipated to confirm prior data demonstrating the unique pulsatile MOA of LUM201. We believe LUM201 has the potential to disrupt the injectable therapeutic market for PGHD. We also believe that LUM201 is essentially a pipeline and a product with the potential to target up to 10 other indications for which growth hormone has been approved. We're focusing first on PGHD, and once we gather data from our current program, we plan to evaluate LUM201 in a subset of these other indications. We're also pursuing business development opportunities to add other rare disease assets to expand our portfolio. We are excited about the programs we're making toward our clinical programs, and with our solid balance sheet strengthened by the receipt of the final tranche of the $26 million from our PRV sale, we are in a good position to advance our corporate strategy. So with that, I'm going to turn the call over to John to review our Orgo Hormone Secretagogue Loom 201 and recently published data.
spk04: John? Thank you, Rick, and good afternoon, everyone. As Rick mentioned, we had some very compelling data presented and published in recent weeks, some of which we highlighted on our last call but are worth touching on again. Before getting into the details, I just want to remind everyone of the key differentiating factor in the LUM201 mechanism of action. As you may recall, growth hormone deficiency can be defined by low to nearly absent secretion of growth hormone from the pituitary gland. Current therapies and those in development consist of the delivery by injection of a bolus of growth hormone or a long-acting derivative, respectively, to restore growth. LUM201, on the other hand, is not a growth hormone, but is instead a growth hormone secretagogue that acts within the body's natural endocrine pathways to stimulate the body's ability to release growth hormone at the same intervals and subject to the same endocrine feedback loops that occur naturally. Utilizing this endogenous release mechanism should enable the naturally occurring IGF-1 feedback loop to be preserved to help regulate the balance of growth hormone and IGF-1 levels in the body. Given this endogenous nature of LUM201, to benefit from LUM201 therapy, individuals must have a functioning, though diminished, HPGH axis. For this reason, our approach is to target the moderate PGHD population of patients who are able to produce some growth hormone naturally, but not in sufficient amounts to attain normal adult height. We believe these patients can be identified using our predictive enrichment markers, or PEMs, to select the moderate PGHD patient group likely to respond to LUM201 therapy. Our analysis of the large genesis data set published in the Journal of Endocrine Society suggests that approximately 60% of the total PGHD population would fall into this moderate category. The new data, highlighted by Dr. Kersorla during our Key Opinion Leader event last week, supported this thesis. demonstrating that the specific PEMs of a baseline IGF-1 level greater than 30 nanograms per mil and a peak growth hormone level of greater than or equal to 5 nanograms per mil after a single dose of LUM201 identify this moderate PGHD patient population likely to benefit from LUM201. In his KOL event presentation, Dr. Krasorla discussed newly released data from Merck's O2O study as part of his overview of LUM201's mechanism of action. The subgroup of the Merck O2O trial specifically examined the effect of LUM201 had on the pulsatile secretion of growth hormone over 24 hours in patients with PGHD after six months of treatment with LUM201 compared to each patient's baseline GH secretion. As has been previously observed in adults, LUM201 increased the pulsatile release of growth hormone for 24 hours in two PEM-positive patients. Importantly, following six months of treatment with LUM201, area under the curve analysis showed increases of growth hormone of less than twofold in these PEM-positive patients that resulted in substantial increases in height velocity. As expected, the one PEM-negative patient showed no increase in growth hormone AUC over the 24-hour monitoring period and no increases in height velocity after six months of treatment with LUM201, further supporting the ability of the selected predictive enrichment markers to identify both those likely and those unlikely to respond to LUM201. We believe these data are important as they support preclinical data which show that pulsatile delivery of growth hormone produces greater efficacy than continuous exposure to the same amount of growth hormone. Data presented at ENDO in March distinguished LUM201 from standard growth hormone secretogox, a poster entitled LUM201 Elicits Greater growth hormone response than standard growth hormone secretagogues in pediatric growth hormone deficiency supports other data suggesting that LUM201 is unique in its ability among the secretagogues traditionally used to diagnose growth hormone deficiency. The poster presented an analysis of data from a prior clinical study comparing the peak growth hormone response of LUM201 to that of standard growth hormone secretagogues in children naive to treatment and previously diagnosed with growth hormone deficiency. The objective of the analysis was to determine whether LUM201 stimulates growth hormone response uniquely compared to standard growth hormone secretogox. The analysis demonstrated that in children with growth hormone deficiency, the growth hormone response to a single oral dose of LUM201 greatly exceeds that observed with standard growth hormone stimulation agents. The difference in growth hormone responses increases with higher baseline concentrations of IGF-1 and higher growth hormone stimulation test results. as identified by our predictive enrichment markers. The synergistic actions of LUM201 on the physiological pathways regulating growth hormone release explained by growth hormone responses are greater in response to LUM201 compared to traditional tests used to diagnose PGHD and indicate that the greatest difference may be found in children with more moderate degrees of growth hormone deficiency. These results further support data analyses recently published in the Journal of the Endocrine Society demonstrating that LUM201 has the potential to elicit a therapeutic response in pediatric patients with moderate growth hormone deficiency or approximately 60% of the total PGHC population as identified by the specific PEMs and gives us greater confidence in the potential efficacy of LUM201 in this patient population. Collectively, these data support the importance of PEM selection and the mechanism of action of LUM201 to identify patients likely to respond to LUM201. Given its oral delivery and its mechanism of action that depends on the natural HPGH axis, we believe that LUM201 may offer a preferred treatment option for approximately 60 percent of children suffering from growth hormone deficiency. We believe these data strongly support our clinical development strategy in PGHD and illustrate why these are the PEMs we are using in our Phase IIb Oral Growth 210 trial. This trial is a global multi-site trial involving approximately 80 PGHD patients randomized into one of three dose levels of LUM201 or a comparator arm of standard of care daily injectable growth hormone therapy. Only those PGHD patients determined to be PEM positive as evaluated by the predictive enrichment markers I described earlier, will be randomized in the study. During this Phase IIb study, the repeatability of the PEM classification will be evaluated during screening for randomization. Dosing will be administered over six months with annualized height velocity as the key clinical outcome measure. The main objectives for this Phase IIb study are to prospectively confirm the utility of our predictive enrichment marker strategy in selecting likely LUM201 responders, to assure that the PEM classification is consistent and repeatable, and to determine the optimal dose for a Phase III registration trial. The three dose levels of 0.8, 1.6, and 3.2 mg per kg of LUM201 were chosen based on supporting data from the Merck study in PGHD patient mentioned earlier, as well as a prior PKPD study of LUM201 in healthy adults. A post-hoc analysis of the Merck study in PGHD showed no statistical difference in average height velocity for PEM-positive patients dosed with 0.8 mg per kg LUM201 versus those dosed with standard of care recombinant human growth hormone. The PKPD study in healthy adults showed that 0.8 mg per kg pediatric equivalent dose is only approximately one-third of the way up the pharmacodynamic growth hormone dose response curve. These data showed that administering increasing doses of LUM201 in healthy adults up to 100 mg the equivalent of 2.8 mg per kg in children, result in higher plasma concentrations of growth hormone. The three doses chosen for our Phase IIb trial cover that full pharmacodynamic range and suggest the potential for greater growth hormone secretion and potential efficacy in the PGHD patients in our study. The trial opened to enrollment last quarter, and we currently have over 50% of the sites activated, with additional sites expected to open soon as we progress toward our goal of 40 to 50 trial sites in total. These sites were selected based on their prior history of enrolling PGHD patients in clinical trials, which should enhance the enrollment process and increase our confidence in our anticipated mid-2022 data readout. We expect to initiate a second concurrent trial of LUM201 for patients with PGHD shortly. The ORA Growth 212 trial is intended to further illustrate the mechanism of action of LUM201 in amplifying the natural pulsatile secretion of growth hormone The Oral Growth 212 trial will focus on pharmacodynamic endpoints at two different doses, 1.6 and 3.2 mg per kg, in approximately 24 children with PGHD. The purpose of this study is to replicate in a larger cohort of PGHD patients the pulsatility data in adults and in the small subset of children for the Merck O2O trial reviewed by our KOL. We plan to conduct this trial at a single specialized pediatric center with the ability to perform the more frequent sample collection and monitoring required for these types of clinical trials. The study will assess growth hormone levels over 24 hours at baseline and after six months on therapy to confirm LUM201's amplification of pulsatile secretion. Once initiated, the study will run in parallel with the Phase IIb or Growth 210 trial with the goal of providing supportive data in future regulatory filings and ultimately in any commercial marketing. efforts. On our last call, we reported that a fire occurred at the San Borja Arrearon Hospital in Santiago, Chile, the planned clinical site for the Orr Growth 212 trial. Our investigator's clinic was not directly involved in the fire, and access to the hospital has been restored, and we anticipate initiating the Orr Growth 212 trial in the current quarter. As we have previously stated, This trial is not on the critical path for regulatory approval of Loom 201, and we do not believe the brief interruption caused by fire will delay our timelines. Finally, as Rick mentioned, we believe that Loom 201 may serve as a platform therapy potentially applicable to other indications for which recombinant human growth hormone is approved. Pending results from our concurrent oral growth trials just discussed, we plan to evaluate Loom 201 and these other indications. Beyond LUM201, we continue to pursue business development opportunities to license or acquire other rare disease assets in order to expand our pipeline and our ability to provide innovative therapy to those suffering from rare diseases. Before we discuss our financial results, I will turn the call over to Rick to make a few comments about our announced CFO succession.
spk05: Thank you, John. And before we proceed, I first want to say a few words about Carl's well-earned retirement. As we announced on April the 20th, after a distinguished 40-year career as a financial executive, Carl will be retiring effective on July 4th. We want to thank him for his service to Loomis Pharma, and we want to congratulate Lori Lawley, currently our Senior VP for Finance and Accounting, and Carl's very capable deputies. we will be succeeding Carl as a CFO upon his retirement. So I'll now return the call over to Lori Lawley to discuss financial results for the first quarter of 2021. Lori?
spk01: Thanks, Rick. I look forward to working with the team and engaging with investors in my new role as Chief Financial Officer. We ended the first quarter with cash and cash equivalents totaling $114.1 million compared to $98.7 million on December 31, 2020. As Rick mentioned earlier, our current cash position includes the receipt in January of the final $26 million tranche from the sale of our priority review voucher. We expect an average cash use of approximately $8 to $9 million per quarter through 2021 and expect our current cash on hand to support operations through ORA Growth 210 readout and completion of the ORA Growth 212 trial. Net loss for the first quarter was $8.6 million compared to a net income of $0.3 million for the same period in 2020. Now I would like to turn the call back over to Rick before we open up for questions.
spk05: Rick? As you can tell, we're excited about our advancement of our oral growth trials in PGA State and look forward to the initiation of our PKPD Oral Growth 212 trial. We're on solid financial footing to execute on our strategy and believe that our clinical trials will confirm the potential for Loom 201 to disrupt the PGAC market. We look forward to continuing to provide updates as we progress. And with that, we'll open the call for questions. Operator?
spk00: As a reminder, to ask a question, you will need to press star 1 on your telephone keypad. Again, that is star 1 on your telephone keypad. To withdraw your question, you may press the sound key. Please stand by while we compile the Q&A roster. Your first question comes from the line of Charles Duncan from Cantor Fitzgerald. Your line is now open.
spk07: Hi. Yeah, thanks for taking my question, juggling lots of calls, and thanks, John, for all the detailed information. I had a couple of quick questions. One is on the oral growth 210 trial. You said that about 50% of sites were activated Can you give us a sense of enrollment activity and perhaps screen to enrollment ratio? Any early perspectives on that?
spk05: Chas, we haven't given any guidance to the market in terms of that update. You know, these trials... You usually start at a certain pace, but as more patients are screened in some of the more active sites, and I say experienced sites, enrollment improves dramatically, and I think that we're in that stage now, but we haven't given any guidance as to the two questions you asked.
spk07: Okay, okay. And then with regard to the PEM, there's a lot of good information that you presented here recently, but I'm wondering if you've had any feedback from the FDA on this strategy and if you can provide any color on that with regard to their, I guess, comfort level with this forming the basis of even making an observation out of a phase 2B.
spk05: John, why don't you take that question?
spk04: Yes, so we've engaged with the FDA from the beginning about this idea because it is really the basis for how we think this product is going to move forward, right? Selecting the correct patients and giving them the correct dose is really how this molecule is going to show itself to be effective. So, yes, we have engaged, and I think we need to bring them the results from this trial where we're applying the PEMs prospectively. to go any further with the idea that we brought to them.
spk07: Okay, that's helpful. And then one last question, then I'll hop back in the queue. Regarding pulsatile growth hormone release and the PEM strategy, do you think that that could go beyond call it a risk mitigator with regard to clinical trial conduct, but actually result in better outcomes or more effective growth stimulation and perhaps even slower or shorter duration of dosing. Given the mechanism with regard to stimulating release versus, say, a bolus dose, do you think that the outcome measures could actually be better, and what's the basis of that?
spk04: Go ahead, John. So, you know, there are some preclinical experiments that say, you know, given the same amount of growth hormone in a pulsatile fashion versus a continuous fashion, dwarf rats grow better with pulsatile release or administration of growth hormone. So I think the potential is there for, you know, the importance of the peak to nadir pulsatility in the release of growth hormone to really show its effect in this study. It's never really been looked at before because there hasn't been a way to, you know, pulse 23 to 25 pulses of growth hormone across a day in an individual child and then look at their growth in six months. But I think this study will really get at that, right, in a group of patients that we think we've selected to be responsive. So we'll have a lot of information. And I think there's a very good potential to show that this unique mechanism of action will give us quite a bit of growth while, you know, maintaining growth hormone and IGF-1 levels within the physiological ranges.
spk07: Thanks for the added color, and thanks for providing all the information.
spk04: Thank you, Jess.
spk00: Your next question comes from the line of Alina Kaminski from Jones Trading. Your line is now open.
spk03: Hi, good afternoon. Thank you for taking my question and congrats on the quarter. So I guess, you know, you did previously mention and also in your remarks that you're looking to expand Boom 201 into additional indications. So I was hoping maybe you can help us understand what proportion of patients for each of the indications you're looking for should be PEM positive and respond to until one? Thank you.
spk05: Well, we continue to engage the KOL community and the experts in each one of these diagnoses, and we are in the process of determining just that type of question. John, do you want to add any additional color?
spk04: Yeah, so all the data we have now on kids with growth hormone deficiency are kids with pediatric growth hormone deficiency. So we've done some thinking and extrapolation, but the data that we have points to the PTHD population. And I think each one of the other indications has some unique facets to its etiology and where the defects are. Some are more a combination of several events, others are are related to growth hormone deficiency pretty directly. So it's going to depend on each of the different disease indications, and it will depend on us acquiring a little bit more knowledge about our molecule and how it affects growth in this 210 trial before we really can put our hand down and understand broadly for some of these secondary indications how many responsive patients there may be.
spk03: Got it. Just one follow-up on this. Do you think you might need to adjust your PEM classification based on each indication, based on, you know, the cutoff for that?
spk05: Yeah. John, go ahead.
spk04: You know, we'll have to see. But I think, you know, in the end, this set of criteria is useful for identifying patients with a growth hormone with a, you know, partially functioning PEM. access to produce growth hormone. And that's kind of the first step in selecting patients that are going to be responsive to our molecule, right? You know, some of the other indications, you do have to think about whether some of the deficits are downstream of growth hormone production, right? Like some of them, you might have a deficit in growth hormone receptor interactions, right? And so we have to put all these pieces together and think through what's the best approach, and what kind of dose range do we have access to in the information we get out of the growth hormone deficiency, the PTHD trial. So I think there's a lot of really interesting data that's out there for these patients being treated with just percominant human growth hormone, and we can build on what's known about the etiology and find the best fit for our molecule to go after these secondary indications.
spk03: Got it. Well, thank you so much, and again, congrats on this quarter, and I'll jump back in this year.
spk05: Thank you, Yaz.
spk00: Your next question comes from the line of Derek Archila from CSAIL. Your line is now open.
spk06: Hi, guys. Good afternoon. This is Jacques on for Derek. Thanks for fitting us in here and taking our questions. Zooming out, can you talk about how you view adherence for an oral secretagogue versus weekly growth hormone injections? You know, what are some of the pros and cons there?
spk05: Let me start with an answer. Jacques, you know, there is so much data out there that any injectable product, whether it be in PGAC or other indications, is problematic in a pediatric population in terms of adherence. I think the longer-acting products will improve that, but we've done some direct market research that shows that when asked by both caregivers and clinicians which one they would prefer, and that is a weekly injection or a once-a-day oral, as our product is, they overwhelmingly choose an oral product. How that translates to compliance in the real world is another question I think we have to study some more, but we believe that it's definitely going to be a preferable treatment, and we believe that there's enough attention by the parents that they're going to stay on their children and make sure that they take this once-a-day oral product as they should. John, do you have anything to add to that?
spk04: No, that's perfect.
spk06: Got it. That's helpful. And then if I may, on the BB front, can you speak to putative licensing opportunities and how competitive that is right now to find an asset? And what indications and modalities are you guys thinking about? Thanks.
spk05: Good question, Jacques. And I can tell you we have a robust process that has been underway for quite some time. That is led by Aaron Shewhart, our CBO, and Aaron has 25 or more years of experience as a business officer with both large companies and small companies in the rare disease space. In addition to that, I think we have collectively as a team – many decades of experience of operating in this rare disease space. And as a result, those contacts have been very productive in bringing forth a number of interesting opportunities that we are, once again, just actively, you know, reviewing and making sure that we make the right choice here.
spk06: Great. That's very helpful. Thanks and congrats on the quarter, guys.
spk05: Thank you, Jacques.
spk00: 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.
spk05: Okay. 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. Really appreciate your time today.
spk00: This concludes today's conference call. Thank you for participating. You may now all disconnect.
Disclaimer