Lumos Pharma Q4 2023 Earnings Call Transcript

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March 7, 2024

There are 9 speakers on the call.

Operator

Good afternoon, and welcome to Lummis Parmont 2023 4th 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.

Operator

Please go ahead.

Speaker 1

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.

Speaker 1

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 the forward looking statements. Information presented on this call is contained in the press release we issued this afternoon and in our Form 10 ks, which may be accessed from the Investors page of the company's website. Speaking on today's call will be Rick Hawkins, CEO and Chairman John McHugh, our President and Chief Scientific Officer Doctor. Duke Patibk Tawana, Chief Medical Officer and Lori Lalley, our Chief Financial Officer.

Speaker 1

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.

Speaker 2

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 GROW-two ten and oral GROW-two twelve trials, ILUM-two zero one and pediatric growth hormone deficiency. On today's call, we'll briefly summarize our trial results and other progress made in 2023, recap highlights for recent weeks and lay out plans for 2024. And we'll be happy to answer your questions.

Speaker 2

Before I begin, I'd like to take a moment to congratulate Doctor. Prasit Dukhacheprawanen on his recent promotion to the role of Chief Medical Officer for Lumos. Doctor. 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 in his esteemed career. Since joining us in 2022, Doctor.

Speaker 2

Duke had played a pivotal role in steering our oral growth trials to fruition, providing invaluable guidance, leveraging his influential network for enrollment and significantly contributing to analysis of trial data. As our new CMO, Doctor. Duke will continue to advocate for exploration of BLUM-two zero one within the PJC clinical community, a mission that has already garnered remarkable interest from pediatric endocrinologists globally, thanks to his advocacy efforts. Furthermore, Doctor. Duke will be an integral part of shaping the trial design for a pivotal LUM-two zero one trial in PGHD, details of which we're going to delve into later during this call.

Speaker 2

Doctor. Duke will be joining the call today for our Q and 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, LUNTL-1 has successfully met all primary and secondary endpoints in both oral growth trials, demonstrating clear proof of concept for its efficacy as an oral alternative to daily and weekly injectables for patients with PGHD.

Speaker 2

Results from ORG0210 demonstrated that the 1.6 mgs per kid dose of LUM201 achieved annualized high velocities or AHVs of 8.2 centimeters a year at 6 months and 8 centimeters a year at 12 months, aligning consistently with historical growth rates for the moderate PGHD population. The difference in AHV at 6 12 months between the optimal 1.6 mgkgLUM-two zero one dose and the recombinant growth hormone comparator arm fell within the non inferiority margin of less than 2 centimeters a year and that's a criterion recently used for FDA approvals. Although oral GROW2-ten was not specifically designed to establish non inferiority, 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 3 trial to show non inferiority against the daily injectable growth hormone comparator arm. Now the preliminary 24 month LIV-two zero one data we presented in November gathered from a subset of the Orgov-two ten patient or participants who met the protocol requirements for an extension beyond 12 months combined with Orbro-two twelve subjects.

Speaker 2

These data revealed a sustained effect showing a minimal decrease of approximately 6% in annualized height velocity from year 1 to year 2 as opposed to the significant decline of approximately 20% reported for daily recombinant growth hormone in moderate PTHD patients. Now ORGrowth-two ten successfully achieved its pre specified primary endpoint, validating our predictive enrichment marker or PEM test, while also meeting the secondary endpoint by demonstrating 100% reproducibility of PEM positive classification. Additional data from the ORILOGO-two twelve trial demonstrated that only 20% growth hormone concentration was needed to achieve comparable annualized site velocity. That suggests that BLUM-two zero one is more efficient than exogenous growth hormone to promote growth. These data confirm the importance of BLUM-two zero one's unique mechanism of action to restore the natural physiology of pulsatile growth hormone secretion.

Speaker 2

Our data from both trials provided a clear safety profile for investigational LUM-two zero one. Now 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 210 trial in the Q2 of this year, most likely at a major medical conference. This data set will include 12 month AHV data on all enrolled patients from the ORILOGO-two ten trial. Additional 24 month data beyond the data set we announced in November will also be available.

Speaker 2

We expect to present these data at additional analysis at several meetings throughout 2024 to capitalize on the growing interest and excitement in the pediatric endocrine community about LUM-two zero one as potentially the first oral therapeutic in this population. And following the announcement of our top line results, we've been diligently conducting additional analyses in preparation for end of Phase 2 meeting with the FDA and that's scheduled for the Q2. Our data package for this meeting is comprehensive, featuring a larger data set compared to other growth hormone counterparts at this developmental stage. These data show LUM201 induces annualized high velocities consistent with historical levels for moderate PGS D on recombinant growth hormone. That also demonstrate that oral LUM-two zero one produces a robust and enduring response at the 1.6 mg per kg dose, making use of our predictive enrichment marker or PEM strategy for selecting suitable moderate PGSD patients, thereby further derisking our Phase 3 program.

Speaker 2

As a result, we approached this end of Phase 2 meeting with a high level of confidence. Now long before we announced top line results from our oral growth trials, we've been actively involved in advanced planning for pivotal Phase 3 trial. Following our Phase 2 meeting with the FDA, we will finalize the ultimate design of the trial, which we anticipate initiating in the Q4 of 2024. We believe that this trial's results will support a new drug application for LIM-two zero one in the PJC indication. With that, I'm going to turn it over to Lori Lawley, our CFO for a brief overview of our financial results.

Speaker 3

Thanks Rick. Lumos Pharma ended the year of December 31, 2023 with cash, cash equivalents and short term investments totaling $36,100,000 compared to $67,400,000 on December 31, 2022. Cash on hand as of December 31, 2023 is expected to support operations through the Q3 of 2024, inclusive of our Phase 3 preparations and other operational activities. Initiation of our Phase 3 clinical trial by end of 2024 is subject to securing financing in the near term. Research and development expenses were $22,100,000 an increase of 4 2023 compared to the same period in 2022, primarily due to increases of $3,300,000 in clinical trial expenses, dollars 900,000 in contract manufacturing expenses, dollars 200,000 in consulting expenses and $200,000 in other expenses, partially offset by a $400,000 decrease in personnel related expenses.

Speaker 3

General and administrative expenses were $16,600,000 an increase of $900,000 for the year ended December 31, 2023 compared to the same period in 2022, primarily due to increases of $500,000 in personnel related expenses, dollars 400,000 in royalty expenses paid to the Public Health Agency of Canada and $100,000 in travel expenses, partially offset by a $100,000 decrease in other expenses. The net loss for the year ended December 31, 2023 was $34,000,000 compared to a net loss of $31,100,000 for the same period in 2022. We ended Q4 20 23 with 8,102,555 shares outstanding. And with that, I will turn it back to Rick for his closing remarks.

Speaker 2

Thanks, Laurie. And to conclude, we're absolutely thrilled by the progress of our program to date and by the excitement among the immigrant community regarding the potential for the 1st oral therapy for PGHD. We've been on a podium presenting data on numerous endocrine conferences over the past 2 years and have seen the overwhelming acceptance of and the excitement for oral LUM-two zero one. We're confident in the distinct advantages offered by LUM-two zero one for patients with moderate PTSD and enthusiastic about advancing to the next developmental stage for this asset. We firmly believe that the data set we are submitting to the FDA for upcoming end of Phase 2 meeting provides ample support for pivotal trial LUNTL-one at the 1.6 mgkg dose, affirming its potential as a viable alternative to injectable products for appropriately selected moderate PJC patients.

Speaker 2

And we eagerly anticipate sharing further insights from our oral 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.

Operator

Thank you. First question coming from the line of Charles Duncan with Cantor. Your line is open.

Speaker 4

Hey, 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 a question about the end of Phase 2 meeting with agency.

Speaker 4

Is that actually scheduled? And can you provide us some insight on what the key question is? 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 it shortly after the meeting?

Speaker 2

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

Speaker 5

Yes. Thanks, Charles. I appreciate the question. So before we start our Phase 3 study, we really would like to use this end of Phase 2 meeting to take everything we've learned from Phase 2 data that we've read out. We put that into our Phase 3 protocol and we want to walk away from that meeting with agreement on all the specifics of that protocol.

Speaker 5

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 the path to move forward. So it is it 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 3 protocol. So we expect to communicate that information, the outcomes to that when we hear back from the FDA probably in the meeting minutes, right? So we've said that that timeframe is in Q2 of this year.

Speaker 4

Okay.

Speaker 2

And Charles, I might add is that there are a lot of historical precedence here. We feel confident about this meeting and really an outstanding briefing book. I think that historically these studies have been 12 month non inferiority trials against an active comparator. The non inferiority margin historically has been 1.8 to 2 centimeters. And we also expect to have a randomization to the 1.6 mgs per kg a day, 2:one randomization to growth hormone.

Speaker 2

I think most of these studies have been about 200 subjects. We expect a similar design. And also, of course, we validated our PEM strategy. I think we derisk our program pretty considerably, given the fact that we can select patients we believe will be our drug will be effective in and that is the moderate growth hormone deficient patient. So we're pretty confident about our meeting with the FDA.

Speaker 4

That's helpful. If I could just ask a follow on question to that, those two observations. Rick, would you anticipate the PEM strategy to help you design and conduct a smaller sample size or conduct one about that size with perhaps greater confidence in the signal to noise?

Speaker 2

John, do you want to go there? Yes.

Speaker 5

So I think the how we view the pen test is it's going to kind of raise our efficiency, right? 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. It will make it a more efficient trial.

Speaker 4

Okay. Last question in terms of business development activity. At one time, you had talked about possibly ex U. S. Partnering.

Speaker 4

I guess I'm wondering if you have any further thoughts on that? And then I'll hop back in the queue.

Speaker 2

Thanks. Thanks for that question, Charles. And sure, 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 and companies that have completed an outreach to us or vice versa.

Speaker 2

And we continue with those discussions and at the appropriate time, I think that we will partner with anyone who's going to we believe is 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.

Speaker 4

Okay. Thanks for taking the questions, Whit.

Operator

Thank you. And our next question coming from the line of Leland Grochsel with Oppenheimer. Your line is open.

Speaker 6

Thank you, Rick and team for the update. Just a question from me with respect to additional indication potential for 201 as we think about use of growth hormone products today and indications such as Turner's and other such disorders. Just wondering if you could comment on any plans down the road to explore 201's potential to be approved for those additional label indications? Thank you.

Speaker 2

Yes. And John, I'm going to let you start with that as an answer. And thank you for the question, Leland.

Speaker 5

So we spent a lot

Speaker 4

of time, Leland, thinking through

Speaker 5

the best opportunities to advance LUM-two zero one and pass PGHD. PGHD 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 and apply to many of the other 11 indications that are approved for injectable growth hormone. So I think we were getting to the point where we have the data set where we can start to think about how to move forward, but we have not yet made any decisions on the exact next step that we're going to take. But we have, I think, the data and have done the background research on each one of those indications to make some good decisions shortly.

Speaker 2

And Leland also recall that we've got a pilot program underway in a broader cardiometabolic opportunity of nonalcoholic fatty liver disease at MGH. Now it's an investigator initiated trial, but this investigator did demonstrate with a recombinant growth hormone trial in this indication some pretty significant reductions in liver fat in this population. So we're pretty excited 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.

Speaker 6

Got it. Great. So, at this point, we don't have the view on when she may have data on her study of 201, correct?

Speaker 2

No. She doesn't have the trial fully enrolled yet and it's a 6 month trial. So we haven't really talked about that yet in the marketplace.

Speaker 6

Okay. Well, stay tuned and look forward to hearing updates following your FDA meeting. Thank you. Thanks.

Operator

Thank you. And our next question coming from the line of Ed White with H. C. Wainwright. Your line is open.

Speaker 2

Hi. Thanks for taking my questions. So how should we be thinking about the Phase 3 start in the Q4? Is that just when you'll be in opening sites or do you intend or do you think that you can actually treat patients during the Q4? Okay.

Speaker 2

Thank you for that question, Ed. And Duke, will you answer that for us? I think you're on mute.

Speaker 7

Thank you. Yes, sorry about that. Thank you. That was a very good question, right. As of now, we do diligent, like as John mentioned, right.

Speaker 7

We already have a plan for the Phase 3 plan. We get ready to really discuss with the FDA when we have plan. In the meantime, we do a prep work what we need to get done in regards to have a timeline that will be proposed, right? So we think that by the end of this year, we'd be able to screen the subject and get the timeline 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 KOL around the world because they learn about our Phase 2 results and they do believe that this can be a great potentially the new drug therapy for their patient, the first oral therapy.

Speaker 7

So we do believe that a lot investigator actually some of those participate in our Phase II trial and some news to LUM-two zero one. However, with the request of participation, we do believe the timeline when we get this survey up and running and we have a final site identified, I think that the rolling can go very quickly and as we proposed in the plan in the past.

Speaker 2

Okay. And as you obviously we have to finalize the Phase 3 protocol. We're obviously working with our vendor partners very actively. It's good to be we've been active in all on the podium at all these endocrine meetings around the world. And we've contacted and had direct contact with really experienced investigators as a result.

Speaker 2

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 3 drug product manufactured and ready to go. So we will have it ready to go. And so all the preparation that needs to be done, we're doing right now. And we hope to have plenty of patients screened in the study before the end of the year.

Speaker 2

Okay. Thanks Rick for taking my question. Sure.

Operator

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

Speaker 8

Hi. Good afternoon, guys. Just one question. Can you remind us of what percent of the oral growth patients enrolled were boys versus girls? And there is difference between how these two populations might be managed by pediatric endocrinologists, a sense that there could potentially encourage patients who might not choose to have a daily injection, try an oral growth hormone therapy instead?

Speaker 2

Duke, why don't you answer that question? Thanks. Appreciate the question, Catherine.

Speaker 7

Yes. Thank you, Catherine. That's a very good question. Our trial is very kind of like unique, right? As you know, when you look at most of Phase 3 trial, especially long acting, as in this novel or Pfizer, majority of those subject that enroll in a trial are male predominant, right?

Speaker 7

In our study, it's pretty much that male a little bit more than female, but not that huge difference, which is very, very interesting. I think when we go back to the 1st global Phase 3 trial, you can see that the number of countries going to increase, the number side going to increase because the time line we want to enroll, we have a goal that we want to complete enrollment within 15 to 18 months, which is again, this goal is actually is a bit better than the goal that those previous Phase III trials have been running. Part of it is that that's dealing 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 3 trial, right? And in regards to the oral, I think that to enroll these subjects, I heard loud and clear when I start the Phase II, try to increase enrollment in a timely manner. We realized that a lot of subject really interested to participate in these oral LUMCO1 trial.

Speaker 7

Again, I don't think that we expect any issue of get the patient enrolled, especially most of them would like to be able to be on this drug and especially during this clinical trial, because it would take years for us to really get the drug approved. I hope that that answers all the questions you had.

Speaker 8

Yes, that's very helpful. And actually just one more on the end of Phase 2 meeting. We've heard over and over from KOLs that growth beyond 12 months is really the most important aspect to them. Are these data going to be a part of your discussion with FDA? And how important is that to the Phase 3 trial design?

Speaker 2

That is a very good question, Catherine. And I'm going to ask John to answer.

Speaker 5

Yes. Thank you, Catherine. So I think the data that we've shown about the small subset of kids that we have on at 24 months on growth has been very telling. The restoration of normal growth hormone pulsatility, normal IGF-one levels and more natural growth that results from that has led to very consistent and durable growth comparing year 2 to year 1, 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.

Speaker 5

So that is a really important piece of data that we certainly will chat with the FDA about. We do anticipate much as Rick said earlier, a standard Phase 3 trial design of 12 months. But because we have a number of subjects already that have gone past that 12 month timeframe. We're moving subjects, from our 24 month long, Org Growth 210 study into a long term safety extension. We will have quite a lot of longer term data by the time we get to Phase 3 and get to an NDA.

Speaker 5

So I think that data will be very helpful in addition to what we see in the Phase 3 12 month study that Rick described. So it is very important and it will be a very nice package of durable data that we can bring.

Speaker 8

Great. That's very helpful. Thanks. Thanks a lot guys.

Operator

Thank you. And I'm showing no further questions in queue at this time. And ladies and gentlemen, this concludes the Lumos Pharma 4th quarter 2023 earnings call.

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Key Takeaways

  • The company reported positive top‐line results from its oral GROW-210 and GROW-212 Phase 2 trials, meeting all primary and secondary endpoints and demonstrating non‐inferior growth velocities versus injectable growth hormone.
  • An end‐of‐Phase 2 meeting with the FDA is scheduled for Q2 2024, with management expressing high confidence in securing agreement on the pivotal Phase 3 protocol, expected to start in Q4 2024.
  • As of December 31, 2023, the company had $36.1 million in cash, cash equivalents, and short-term investments, providing a runway only through Q3 2024 and necessitating near-term financing to initiate Phase 3 activities.
  • R&D expenses rose to $22.1 million and G&A expenses to $16.6 million in 2023, contributing to a net loss of $34.0 million versus $31.1 million in 2022.
  • Management highlighted strong interest from pediatric endocrinologists, plans to present additional 12- and 24-month data at major conferences in 2024, and ongoing discussions for potential ex-U.S. partnerships.
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Earnings Conference Call
Lumos Pharma Q4 2023
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