Q4 2023 Lumos Pharma Inc Earnings Call

Participants

Lisa Miller; Senior Director of IR; Lumos Pharma Inc

Rick Hawkins; CEO & Chairman; Lumos Pharma Inc

Lori Lawley; Chief Financial Officer; Lumos Pharma Inc

Charles Duncan; Analyst; Cantor Fitzgerald

John McKew; President and Chief Scientific Officer; Lumos Pharma Inc

Pisit Pitukcheewanont; Chief Medical Officer; Lumos Pharma Inc

Leland Gershell; Analyst; Oppenheimer & Co. Inc

Edward White; Analyst; H.C. Wainwright & Co, LLC

Catherine Novack; Analyst; JonesTrading Institutional Services, LLC

Presentation

Operator

Good afternoon, and welcome to Lumos Pharma 2023 fourth quarter and year end conference call. Currently, all participants are in a listen-only mode. Later we will conduct a question and answer session and instructions will follow at that time. As a reminder, this conference call is being recorded. I will now turn the call over to Lisa Miller, Vice President of Investor Relations. Please go ahead.

Lisa Miller

Thank you, operator. Before we proceed with this call, I'd like to remind everyone that certain statements made during this call are forward-looking statements under U.S. federal securities laws. These statements are subject to risks and uncertainties that could cause actual results to differ materially from historical experience or present expectations. Additional information concerning factors that could cause actual results to differ is contained in our periodic reports filed with the SEC. 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 information presented on this call is contained in the press release we issued this afternoon and in our Form 10 K, which may be accessed from the Investors page of the company's website. Speaking on today's call will be Rick Hopkins, CEO and Chairman, John McHugh, our President and Chief Scientific Officer, Dr. Tuan, Chief Medical Officer, and Lori Lilly, our Chief Financial Officer. Following our prepared remarks, the management team will be available for a question and answer session. I will now turn the call over to Rick.

Rick Hawkins

Thank you, Lisa, and good afternoon, everyone. After the market closed today, we issued a press release announcing our results for 2023. I'm pleased to report that 2023 was a successful year for Lumos, led by our announcement of positive top line results from our oral growth to 10 and will grow to 12 trials alone to a one in pediatric growth hormone deficiency.
On today's call, I will briefly summarize our trial results and other progress made in 2023. Can recap highlights for recent weeks in May, our plans for 2024, and we'll be happy to answer your questions.
Before I begin, I'd like to take a moment to congratulate Dr. percent Duke Project ONE and on his recent promotion to the role of Chief Medical Officer for Lumos. Dr. Duke's exceptional credentials are widely acknowledged, given his renowned expertise in growth disorders and his current leadership at the human growth foundation among various other distinguished roles and his team career since joining us in 2022, doctor, do it played a pivotal role in steering our overall trials to fruition, providing invaluable guidance, leveraging has influential network for enrollment and significantly contributing to analysis of trial data as our new CMO, Dr. group will continue to advocate for exploration of withdrawal one within the PTSD clinical community, a mission that has already garnered remarkable interest from pediatric endocrinologist globally, thanks to this advocacy efforts. Furthermore, Dr. Duke will be an integral part of shaping the trial design for a pivotal into one trial in PGHD details of which we're going to delve into later during this call.
Dr. Dupont, we joined the call today for our Q&A session. So please join me in congratulating him on his well-deserved promotion.
Let's now revisit the key highlights of the results we disclosed last November. As many of you are aware of them to a one has successfully met all primary and secondary endpoints in both worldwide trials, demonstrating clear proof of concept for its efficacy as an oral alternative to daily and weekly injectables for patients with PGHD results from our grow to 10 demonstrated that to 1.6 megs per kick dose of to tool Once achieved, annualized height velocities were a series of 8.2 centimeters a year, six months and 8 centimeters a year at 12 months, aligning consistently with historical growth rates for the moderate PGHD population. The difference in HV. six and 12 months between the optimal 1.6 megs per kg room to a one dose. And the recombinant growth hormone comparator arm fell within the noninferiority margin of less than two centimeters a year, and that's a criterion recently used for FDA approval. Although our growth to 10 was not specifically designed to establish noninferiority, we are pleased to note that the growth outcomes observed in this study are in line with the non-inferiority thresholds seen in recent successful FDA applications. These findings enable us to design a successful Phase three trial to show non-inferiority against the daily injectable growth hormone comparator arm.
Now the preliminary 24 months ago to a one data we presented in November, we've gathered from a subset of the world growth to tend to a patient or participants who met the protocol requirements for an extension beyond 12 months, combined with or over 12 subjects. The data revealed a sustained effect showing a minimal decrease of approximately 6% in annualized height velocity from year one to year two as opposed to a significant decline of approximately 20% reported for daily common and growth hormone in moderate PGHD patients. We'll go to Kent successfully achieved its prespecified primary endpoint, validating our predictive enrichment marker or pen test, while also meeting the secondary endpoint by demonstrating a 100% reproducibility of PAM positive classification. Additional data from the ORBIT two trial demonstrated that only 20% growth hormone concentration was needed to achieve comparable annualized height velocity that suggests that move to a one is more efficient than exogenous growth hormone to promote growth. These data confirm the importance of Bloom tool one's unique mechanism of action to restore the natural foods, the physiology of pulsatile gross growth hormone secretion. Our data from both trials provided a clear safety profile for investigational tool one following the release of top line results in November, ongoing data collection and analysis have continued. We anticipate presenting comprehensive 12-month annualized height velocity data from the oral growth to 10 trial in the second quarter of this year, most likely at a major medical conference. This dataset will include 12 month HV. data on all enrolled patients from zero to 10 trial. Additional 24 month data beyond the dataset we announced in November will also be available. We expect to present these data in additional analysis at several meetings throughout 2024 to capitalize on the growing interest and excitement in the pediatric endocrine community about moving to a one as potentially the first oral therapeutic in this population. Following the announcement of our top line results, we've been diligent conducting additional analyses in preparation for an end-of-Phase two meeting with the FDA scheduled for the second quarter. Our data package for this meeting is comprehensive, featuring a larger dataset compared to other growth hormone counterparts at this developmental St. These data show them to one induces annualized height velocity is consistent with historical levels for moderate PGHD on recombinant growth hormone data also demonstrate that oral Bloom to a one produces a robust and enduring response at 1.6 mix per kick dose, making use of our predictive enrichment marker or payment strategy for selecting suitable moderate PGHD patients, thereby further derisking our Phase three program. As a result, we have posted in the Phase two meeting with a high level of confidence and long before we announced top line results from our oral drug trials. We've been actively involved in advanced planning for a pivotal Phase three trial following our Phase two meeting with the FDA we will finalize the ultimate design of the trial, which we anticipate initiating in the fourth quarter of 2024. We believe that this trial's results will support a new drug application for limb tool, one in the PJSD. indication with data maternity or Wally, our CFO, for a brief overview of our financial results.

Lori Lawley

Thanks, Lumos Pharma ended the year December 31st, 2023, with cash, cash equivalents and short-term investments totaling $36.1 million compared to $67.4 million on December 31st, 2022 due cash on hand as of December 31st, 2023, is expected to support operations through the third quarter of 2024, inclusive of our Phase three preparations and other operational activities, initiation of our Phase three clinical trial by end of 2024, subject to securing financing in the near term for research and development expenses were $22.1 million, an increase of $4.2 million for the year ended December 31st, 2023 compared to the same period in 2022 primarily due to increases of $3.3 million in clinical trial expenses, $0.9 million in contract manufacturing expenses, $0.2 million in consulting expenses and $0.2 million in other expenses, partially offset by a $0.4 million decrease in personnel-related expenses.
General and administrative expenses were $16.6 million, an increase of $0.9 million for the year ended December 31st, 2023 compared to the same period in 2022, primarily due to increases of $0.5 million in personnel-related expenses, $0.4 million in royalty expenses paid to the Public Health Agency of Canada and $0.1 million in travel expenses, partially offset by a $0.1 million decrease in other expenses. Net loss for the year ended December 31st, 2023 was $34 million compared to a net loss of $31.1 million for the same period in 2022. We ended Q4 2023 with 8,102,555 shares outstanding. And with that, I will turn it back to Rick for his closing remarks.

Rick Hawkins

Very short and to conclude, we're absolutely thrilled by the progress of our program, David and by the excitement among the investment community.
Regarding the potential for the first oral therapy for PGHD, we've been on the podium presenting data on numerous endocrine conferences over the past two years. Have seen the overwhelming acceptance of and excitement for our oral loom tool, one for confident in the distinct advantages offered by them to a one for patients with moderate PGHD and enthusiastic about advancing to the next developmental stage for this asset. We firmly believe that the dataset we are submitting to the FDA for upcoming into Phase two meeting provides ample support for a pivotal trial linked to a one at the 1.6 megs per kg dose, affirming its potential as a viable alternative to injectable products for appropriately selected moderate PGHD patients, and we eagerly anticipate sharing further insights from our growth trials throughout this year and keeping you abreast of our progress.
So thank you for your attention, everyone. And operator, we can now open this up for questions.

Question and Answer Session

Operator

Team. Ladies and gentlemen, to ask a question, you will need to press star one one on your telephone and wait planning to be announced to withdraw your question, simply press star one. Once again, please stand by while we compile the Q&A roster. Yes.
And our first question coming from the line of Charles Duncan, Cantor you lend itself.

Charles Duncan

Yes. Good afternoon, Rick and team. Congrats on the progress last year. Looking forward to hearing more updates here in the near term.
Thanks for taking our question. I had a key question about the end-of-Phase two meeting with agency. Is it actually scheduled and can you provide us some insight on what the key question is, is it is it really something that needs to be answered? Or do you feel like this is more a point of execution in terms of going forward? And then when would you anticipate being able to share the outcome, would it be post the meeting minutes or do you think it will be pretty straightforward and you'll share with us shortly after the meeting?

Rick Hawkins

I'm going to ask a good question, Charles, and thank you for. I'm going to turn that question over to John McCue.

John McKew

Yes. Thanks, Chuck. And Charles. I appreciate the question. So before we started our Phase three study, we really would like we use this end of Phase two meeting to take everything we've learned from Phase two data that we've read out and we've put that into our Phase three protocol. And we want to walk away from that meeting with agreement on the specifics of that protocol. So I think it's a very important time for us to talk through any questions the agency has on the data as well as come to agreement on how on the path to move forward. So it is will be a very it will be a good step forward for us when we get through that. And we have agreement on the Phase three protocol. So we expect to communicate that information the outcome so that when when we hear back from them and the FDA probably in the meeting minutes, right? So we said that kind of timeframe was in Q2 of this year.

Rick Hawkins

Okay. And can you tell them I might add is that there are a lot of historical precedence here.
We're we feel confident about this meeting in a really an outstanding briefing book. I think that historically to these studies have been 12 months non-inferiority trials against an active comparator on the non-inferiority margin historically has been 1.8 to 2 centimeters. And um, we also expect to have a randomization to the 1.6 mix per take a day or two to one randomization to growth hormone. I think most of these states have been about 200 subjects, and we expect that a similar design.
And also, of course, we um, we validated our PEM strategy. I think we de-risk our program consisted of pretty considerably given the fact that we can select patients, we believe will be our drug will be effective in that as some moderate growth hormone deficient patients. So we're pretty confident about our meeting with the our community meeting with the FDA.

Charles Duncan

It's helpful. If I could just ask a follow-on question to that. Those two observations. Rick, would you anticipate the pen strategy to help you inside and conduct a smaller sample size or conduct one about that size, would you know, perhaps greater confidence in that symptom tonight?

Rick Hawkins

John, do you want to go there.

John McKew

Yes. So I think the how we view the Pentacel that's going to kind of raise our efficiency rate, we're going to be able to bring in subjects that are likely responders to our molecule. So I don't think it's going to reduce that size that will make it a more efficient trial at site.

Charles Duncan

Okay. First question in terms of business development activity at one time. You talked about possibly ex U.S. partnering. I guess I'm wondering if you have any further thoughts on that. And then I'll hop back in the queue.
Thanks.

Rick Hawkins

Yes, thanks for that question, Charles. And assure I think that you can imagine as the first oral product in this very large global market, there are certain ex U.S. regional markets that in companies that have a job, we completed an outreach to us or vice versa, and we continue with those discussions and at the appropriate time, I think that we will partner with anyone who's going to go to. We believe it's going to be a good partner in those markets. Obviously, this will allow us to bring in some non-dilutive money. And I think that's one of the goals that we would have for ourselves.

Charles Duncan

Okay. Thanks for taking the questions.

Operator

Yes, thank you. And our next question coming from the line of fill-in Gershell with Oppenheimer. Yolanda Shelton.

Leland Gershell

Thank you, Rick and team for the update from just a question from from me. With respect to additional indication potential for two oh one as we think about use of growth hormone products today and indications such as Turners and other such disorders. Just wondering if you could comment on any plans down the road to explore to a one's potential to have to be approved for those additional label indications.
Thank you.

Rick Hawkins

And John, I'm going to let you start with that as an answer and thank you for the question. Li-lan.

John McKew

We spent a lot of time lead and thinking through them the best opportunities to advance lumateperone and pass PGHDPJC. is a great opportunity, I think for us to show the effect of the molecule on restoring natural pulsatility and we can take that same mechanism and philosophy. It applies to many of the other 11 indications that are approved for home for injectable growth hormone. And so I think we were getting to the point where we have the data set where we can start to to think about how to move forward. But we have not yet made any decisions on the exact next steps that we're going to take. But we have I think the data and I've done the background research on each one of those indications to make it make some good decisions shortly.

Rick Hawkins

And we will also recover some recall, we've got a politic pilot program underway in a broader cardio-metabolic opportunity of nonalcoholic fatty liver disease at MGH now as an investigator initiated trial. But this investigator did demonstrate with a in a recombinant growth hormone trial in this indication, some pretty significant reductions in liver fat in this population. So we're pretty excited to have and to work with this investigator. I think it's a little premature right now to talk about anything beyond that, but we're always looking for ways to increase shareholder value with additional indications.

Leland Gershell

Got it, Rick, Rick. So at this point, we don't we don't have a view on when she may have data on her her study of Tier one, correct.

Rick Hawkins

I know I know she doesn't have the trial fully enrolled yet, and it's a six month trial. So we haven't really talked about that yet in the marketplace.

Leland Gershell

Okay. So stay tuned and look forward to hearing updates following your afternoon and thank you.

Operator

Yes. Thank you to our next question coming from the line of Ed White, H.C. Wainwright. Your line is open.

Edward White

Hi. Thanks for taking my questions. So how should we be thinking about the Phase three start in the fourth quarter, or is that just when you will be in opening sites?
Or do you intend or do you think that you can actually treat patients in during the fourth quarter.

Rick Hawkins

Okay. Thank you for that question. And do will you answer that for us? I think you're on mute.

Pisit Pitukcheewanont

Thank you. Yes, sorry, about that, I think gives us very good crushing rate. As of now, we do diligence, like John mentioned, right, we already have a plan for the Phase three plan. We get ready to really discuss with the FDA when we have planned. In the meantime, we do a prep work what we need to get done in regards to have a time line that would be proposed rates. So we think that by the end of this year, we'd be able to be able to screen the subject and get, you know, the time line enrolled like what we proposed in the past. And I feel very optimistic because, as Rick mentioned earlier, that we have overwhelming requests from the KOLs around the world because they learn about our Phase two results. And they do believe that this can be a great potentially the new drug therapy for their patient, the first oral therapy. So we do believe that you know, a lot investigator and actually some of those participated in our Phase two trial and some news to loom tool one. However, you know, with their request of participation, we do believe the time line one we get these are we up and running and we have a final, you know, site identify I think that the rules are going to go very quickly.
And as we proposed in the plan in the past.

Rick Hawkins

And as you know, obviously we have to we have to finalize the Phase three protocol. We're obviously working with our vendor partners very actively. It's good to be on.
We've been active in all of the podium at all these of endocrine meetings around the world, and we've contacted and direct contact with was really experienced investigators. As a result. There's a there's a lot more work that has to be done in terms of qualifying and activating the sites. And of course, we have our Phase three drug product manufactured and ready to go. So we will have a ready go. And so all the preparation that needs to be done, we're doing right now and we hope to have that deal plenty of patients screened in the study for the end of the year.

Edward White

Okay. Thanks, Rick, for taking my question.

Rick Hawkins

Sure.

Operator

And our next question coming from the line of Catherine Novack with Jones Research. Your line is open.

Catherine Novack

Oh, hi, good afternoon, guys. Just one question. Can you remind us of what percent of the OraGrowtH patients enrolled were boys versus girls. And there's a difference between how these two populations might be managed by pediatric endocrinologists in a sense that there and could potentially encourage patients who might not choose to have a daily injection trial and oral growth hormone therapy instead.

Rick Hawkins

And Duke once you answer that question. Thanks. Appreciate the question, Katherine.

Pisit Pitukcheewanont

Yes, thank you, Catherine. Decimate. Good questions. All applies very, you know, kind of unique, Ray, as you know, when you look at most of Phase three trial, especially long acting Ascendis, Novo Pfizer, majority of those subjects enrolled in a trial of male predominant right in our study is pretty much that, you know, mail a little bit more than female. That's not that choose different, which is very, very interesting. I think when we go back to the first global Phase three trial, you can see that, you know, the number of countries going to increase the number of cycles increase because you know the time why we want to enroll. We have a goal that, you know, we want to complete enrollment within 15 to 18 months, which is again, this goal, it's actually that genome better than the goal that those previous Phase three trial have been running. Part of it is that, you know, that's during COVID. And now we don't have COVID and not to mention that we do not expect a lot of competitor to run the Phase three trial.
Right. And in good regards to do you know, the oral, I think deck too, to enroll this subject, I heard loud and clear when they start to face true tried to increase enrollment in a timely manner. We realize that a lot of subject really interested to participate in these oral argument or one trial?
Again, you know, I don't think that we expect any, but this issue of you know, give the patient, an ROE, especially most of them would like to be able to be on this drug and especially during the clinical trial because it would take years for us to really get the drug approved. I hope that that answers the question.

Catherine Novack

Yes. Yes, that's very helpful. And actually, just one more on the end-of-Phase two meeting we've heard over and over from KOLs that growth beyond 12 months is really the most important aspect to them.
And are these data going to be a part of your discussion with FDA and how important is that to the for the Phase three trial design for that?

Rick Hawkins

That is a very good question. Katherine, among ask John to answer.

John McKew

Yes. Thank you, Katherine. So I think the data that we've shown about the small subset of kids that we have on 24 months on growth has been very telling the restoration of normal growth hormone pulsatility, normal IGF-1 levels and FeNO more natural growth. That results from that has led to very consistent and durable growth comparing year to year one, right, we lose only a small tiny bit 6% of our HV. compared to a much larger drop-off that you see with daily injectable growth hormone. So that is a really important piece of data that come. We certainly will chat with the FDA about. And we do anticipate much, as Rick said earlier, standard Phase three trial design of 12 months. But because we have a number of subjects already that have gone past but 12 month timeframe. We're moving subjects come from our 24-month loan, our growth to 10 study into a long-term safety extension. We will have quite a lot of longer-term data by the time we get to Phase three and get to an NDA. So I think that data will be very helpful in addition to what we see in the Phase three 12 one study that Rick described. So it is very important and it will be a very nice package of dermal data that Brian.

Catherine Novack

Great that that's very helpful. Thanks. Thanks a lot, guys.

Operator

Thanks, Ken.
And I'm showing no further questions in queue at this time.
Ladies and gentlemen, this concludes the Lumos Pharma Fourth Quarter 2023 earnings call. Thank you and Advocat.