Q4 2023 Lumos Pharma Inc Earnings Call - Q&A
Good afternoon, and welcome to Lamar's, Parma 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 Mueller, Vice President of Investor Relations. Please go ahead.
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.
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 any forward looking statements.
The 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 break Hawkins, CEO and chairman, John Mchugh, our President and Chief Scientific Officer, Dr. Joanna.
Do you Wanna, Chief Medical Officer, and Lori <unk>, 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.
Thank you Lisa and good afternoon, everyone.
After the market closed today, we issued a press release announcing our results for 2023.
Pleased to report a 2023 with a successful year for Luna led by our announcement of positive topline results from our Barbara <unk> 10 and <unk>.
212 trials of Lum 201 in pediatric growth hormone deficiency.
On today's call will briefly summarize our trial results and other progress made in 2023 King recap highlights for recent weeks.
Our plans for 2024.
It wont be happy to answer your questions.
Before I begin I'd like to take a moment to congratulate Dr percent, Duke protect Rihanna on his recent promotion to the role of Chief Medical Officer from Numis.
Dr. Dukes exceptional credentials are widely acknowledge given her to pronounce expertise <unk> disorders and his current leadership at the human growth Foundation, among various other distinguished roles and his team career.
Since joining us in 2022, Dr. Deep it played a pivotal role in steering our all grow trials to fruition provided invaluable guidance.
<unk> has an 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 them through a one P. J D.
We read them.
Emission.
Already garnered remarkable interest from pediatric endocrinologists globally. Thanks to this advocacy efforts.
Furthermore, Dr do could be an integral part of shaping the trial design for a pivotal one 201 trial in P. Ghd Etfs on which we're going to delve into the later during this call.
Dr <unk>, who joined the call today for our Q&A session.
Please join me in congratulating him on his desk.
Well deserved promotion.
Let's now revisit the key highlights of the results we disclosed last November.
As many of you are aware <unk> 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 injectable for patients with ph D.
Results from Barbara <unk> demonstrated that $1 six makes perfect dose of <unk> hundred one achieved annualized height velocity, where hps of $8 two centimeters a year or six months and eight centimeters a year at 12 months.
Mining consistently with historical growth rates for the modern <unk> population.
The difference in HV at six and 12 months between the optimal $1 six <unk> 201 dose and the recombinant growth hormone comparator arm fell within the non inferiority margin of less than two centimeters a year.
And that's a criterion recently used for FDA approvals.
Although our growth to 10, but 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 III trial to show non inferiority against the daily injectable growth hormone comparator arm.
As a preliminary 24 maximum 201 data we.
We presented in November.
Gathered from a subset of the Org, we're up to 10th.
Patient or participants who met the protocol requirements for an extension beyond 12 months combined with all of our $2 12 subjects.
These data revealed a sustained effect showing a minimal decrease of approximately 6% and annualized height velocity from year, one to year two as opposed to the significant decline of approximately 20% reported for daily recombinant growth hormone and modern <unk> patients.
<unk> successfully achieved its pre specified primary endpoint validating our predictive enrichment marker or pen test. While also meeting the secondary end point by demonstrating 100% reproducibility Perm positive classification.
Additional data from the oil of about 212 trial demonstrated that only 20% growth hormone concentration was needed to achieve comparable annualized height velocity.
That suggests that <unk> hundred one is more efficient than <unk> square format to promote growth.
These data confirm the importance of <unk> hundred one's unique mechanism of action to restore the natural fit physiology of wholesale gross growth hormone secretion.
Our data from both trials provided a clear safety profile for investigational am 201.
Following the release of topline 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 second quarter of this year.
Most likely at a major medical conference.
Data set will include 12 months HV data on all enrolled patients from the oil growth to 10 trial.
Additional 24 month data beyond the data set we announced in November will also be available.
We expect to present these data and additional analysis at several meetings throughout 2024 to capitalize on the growing interest and excitement in the pediatric endocrine community about <unk> 201 is potentially the first oral therapeutic in this population.
Following the announcement of our topline results, we've been diligently conducting additional analyses in preparation for in the phase II meeting with the FDA and is scheduled for the second quarter.
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 them 201 induces annualized height velocity consistent with historical levels for moderate PGA ASP on recombinant growth hormone.
That also demonstrate that oral <unk> hundred one produces a robust and enduring response at one six mix per kg dose, making use of our predictive enrichment marker or <unk> strategy for selecting suitable moderate <unk> patients.
Thereby further derisking, our phase III program.
As a result, we approached this end of phase II meeting with a high level of confidence.
How long before we announced topline results from our all of our trials. We've been actively involved in advanced planning for our pivotal phase III trial.
Following our phase II 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 <unk> hundred one in the <unk> indication.
With that I'll now turn it over to Laurent <unk>, our CFO for a brief overview of our financial results.
Thanks, Brett Le Monde pharma ended the year December 31, 2023, with cash cash equivalents and short term investments totaling $36 1 million compared to $67 4 million on December 31st 2022.
Cash on hand as of December 31st 2023 is expected to support operations through the third quarter of 2024 inclusive of our phase III preparations and other operational activities.
<unk> of our phase III clinical trial by end of 'twenty 'twenty four is subject to securing financing in the near term.
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.
<unk> 9 million in contract manufacturing expenses.
2 million in consulting expenses, and <unk> 2 million and other expenses, partially offset by a $4 million decrease in personnel related expenses general and.
Administrative expenses were $16 6 million, an increase of $1 9 million for the year ended December 31, 2023 compared to the same period in 2022, primarily due to increases of $1 5 million in personnel and related expenses <unk>.
$4 million in royalty expenses paid to the public health agency of Canada, and $1 million in travel expenses, partially offset by $1 million decrease in other expenses and.
The net loss for the year ended December 31, 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 million 102585 shares outstanding and with that I will turn it back to Rick for his closing remarks.
Thanks, Lori and to conclude we are absolutely thrilled by the progress of our program to date.
And by the excitement among the investment community regarding the potential for the first oral therapy for ph D.
We've done a podium presenting data on numerous endocrine conferences over the past two years.
I've seen the overwhelming acceptance of any excitement for oral lum 201.
We're confident in the distinct advantages offered by them 201 for patients with moderate Phd and enthusiastic about advancing to the next developmental stage. After this asset.
We firmly believe that the data set we are submitting to the FDA for upcoming end of phase II meeting.
<unk> provides ample support for our pivotal trial <unk> 301 at one 6% mix per kg dose affirming its potential as a viable alternative to injectable products for appropriately selected moderate <unk> patients and.
And we eagerly anticipate sharing further insights from our oil 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 customers.
Thank you, ladies and gentlemen to ask a question you wanted to first star one on your telephone and wait planning to be announced to withdraw your question simply press Star One again, please standby, while we compile the Q&A roster.
No first question coming from the line of Charles Duncan with Cantor. Your line is open.
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 two meeting with agency is that actually schedule 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.
Straightforward share shortly.
Shortly after the meeting.
I'm going to ask a good question Charles and thank you for it.
Ill turn that question over to John Mchugh.
Yeah. Thanks, Chuck Charles I appreciate the question so.
Before we start our phase III study, we really would like.
To use this end of phase two meeting to take everything we've learned from phase two data that we brought out.
We put that into our phase III protocol, and we want to walk away from that meeting with agreement on all 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 the path to move forward. So it is it will be a very.
It'll be a good step forward for us when we get through that and we have agreement on the phase III protocol. So we expect to communicate that information the outcomes to that.
When we hear back from them.
The FTAA.
The meeting minutes right. So.
We've said that that timeframe is in Q2 of this year.
Okay.
Charles I might add is that there are a lot of historical precedence here.
We're we feel confident about this meeting really an outstanding briefing book.
I think that historically these studies have been.
12 months non inferiority trials.
Against an active comparator.
Given the non inferiority margin historically has been one eight to two centimeters.
And.
We also expect to have a randomization to the $1 six makes perfect. Good day.
Two to one randomization to growth hormone.
I think most of the states have been about 200.
Subjects.
We expect.
Similar design.
And also of course.
<unk>.
We validated our perm strategy I.
I think we Derisked our program considered pretty considerably given.
Given the fact that we can select patients we believe will be our drug will be effective in and that is some moderate growth hormone deficient patients.
So.
We're pretty confident about our meeting with our upcoming meeting with the FDA.
That'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.
Design and conduct a smaller sample size or conduct one about that size with perhaps greater confidence in the signal to noise.
John do you want to go there.
Yes.
So I think the.
How we view the penthouses thats 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 its going to reduce that size that will make it more efficient trial that's right.
Okay last question in terms of.
Business development activity at one time, you had talked about possibly.
Ex U S. Partnering I guess Im wondering if you have any further thoughts on that and then hop back in the queue. Thanks.
Sure.
Okay.
Yes, thanks for that question Charles.
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.
And companies that have.
<unk>.
Completed an outreach to us or vice versa.
And we continue with those discussions and at the appropriate time.
I think that we will apartment with with.
Anyone who's got to got it.
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 thats one of the goals that we would have for ourselves.
Okay. Thanks for taking the question sorry.
Okay.
Thank you.
And our next question coming from the line of Leland.
Chris <unk> with Oppenheimer. Your line is open.
Thank you Rick and team for the update.
Just a question from from me with respect to additional indication potential.
For 201, as we think about <unk>.
Use of growth hormone products today in.
Indications such as Turners and other such disorders, just wondering if you could comment on any plans down the road.
To explore 200 ones.
Potential too to be approved for those additional label indications. Thank you.
Yes, and John I'm going to let you start with that answer and thank you for the question Leland.
Okay.
So we spent a lot of time Leland thinking through.
Sure.
The best opportunities to advance loomed Jordan.
P Ghd ghd as a great opportunity I think for us to.
To show the effect of the molecule.
We're storing natural pulse utility and we can take that same mechanism that philosophy and apply it to many of the other 11 indications that are approved for.
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 to make it make some good decisions shortly.
And Leland also.
Recall that we've got a pilot pilot program underway and broader cardio metabolic opportunity of non alcoholic fatty liver disease that MGH now that's an investigator initiated trial.
But this investigator did demonstrate with.
Recombinant growth hormone trial in this indication some pretty significant reductions in liver fat in this population. So we're pretty excited too.
To work with this investigator I think it's too premature right now to talk about anything beyond that but we're always looking for ways to increase shareholder value with the additional indications.
Got it real quick so at this point we don't.
We don't have a view on when she may have data on her her study of 201 correct.
No.
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.
Okay, we'll stay tuned and look forward to hearing updates following your peer activity. Thank you.
Okay.
Thank you.
And our next question coming from the lineup.
And <unk> with H C. Wainwright your line is now open.
Hi, Thanks for taking my questions.
So how should we be thinking about the phase III start in the fourth quarter.
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.
Okay.
Question Ed.
Will you answer that for us.
I think youre on mute, yes, sorry about that yeah that gives us very good Christian right as of now we do diligence like as John mentioned, we already have a plan for the phase III plan, we get ready to really discuss with the FDA. When we have done in the meantime, we.
Do a prep work, what we need to get done in regards to have a timeline that will complete the postal strike. So we think that in by the end of this year, we'd be able to be able to screen this object and get.
That timeline and really like what we proposed in the past and feel very optimistic because as Rick mentioned earlier that we have overwhelming requests from the kols around the world because they learned about phase two result, and they do believe that this can be a great potential.
That new drug therapy for that patient in the first oral therapy. So we do believe that a lot of investigator actually some of those participating in our phase II trial, and some news to loom tool one however.
Our request participation we do believe the timeline one we get these survey up and running and we have a final site.
Site identify I think that the rolling can go very quickly and as we proposed in the plan in the past.
Got it.
Okay.
And as.
Obviously, we have to we have to finalize the phase III protocol, we're obviously working with our vendor partners very actively.
We've been active in on the podium at all of these.
Meetings around the world and we've contacted in drug.
Contact with was really experienced investigators as a result.
There is 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 III 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 this study before the end of the year.
Okay. Thanks, Rick for taking my question.
Sure.
Thank you.
Next question coming from the line of Catherine Morgan with Jones Research. Your line is open.
Yes.
Oh, Hi, good afternoon guys.
Just one question can you remind us of what percent of the.
Oil growth patients enrolled were boys girls.
And there are difference between how these two populations might be managed by pediatric endocrinologists.
Set there.
<unk> could potentially encourage patients who might not choose to have a daily injection try and oil growth hormone therapy instead.
Duke why don't you answer that question.
I appreciate the question Catherine Yeah. Thank you Katherine that's a very good question I'll, let tries very kind of a unique way as you know when you look at most of the phase III trial, especially long acting incentives Novo Pfizer majority of those subjected to enrolling that trial and male predominance right.
Steady is pretty much that mail, a little bit more than female that's not that huge difference, which is very very interesting I think when we go back to the first global phase III trial, you can see that the number of countries and they increased the number of sites and increase because the timeline, we want to enroll we have a go.
And that we want to complete enrollment within 15 to 18 months, which is again this goal, it's actually gets a bit.
Better than the goal that those previous phase III trial have been running part of it is that you know that's dealing COVID-19 and now we don't have COVID-19 and not to mention that we do not expect a lot of competitor to run the phase III trial rates and in regards to the oral I think debt to to enroll these subjects.
I heard loud and clear when they start to phase <unk> tried to increase enrollment and then Tommy banner, we realize that a lot of subject really interested to participate in these oral loom till then try it again I don't think that we expect any stat.
Issue off get the patient enrolled.
Basically most of them would like to be able to be on these truck and especially during this clinical trial because it would take years for us to really get the drop through I hope that answers all the questions yet.
Yeah.
Yes, that's very helpful and actually just one more on the <unk>.
Phase two meeting.
You've heard over and over from Kols that go well beyond 12 months is really the most important to them.
Are these data is going to be a part of your discussion with FDA and how important is.
Is that to the phase Iia trial design.
Boy that is a very good question Catherine.
Ask Jon to answer it.
Okay.
Yes. Thank you Catharine so I think the data that we've shown.
About the small subset of kids that we have at 24 months on growth has been very telling.
The restoration of normal growth, our impulse utility normal IGF, one levels and then you know.
More natural growth that results from that has led to very consistent and durable growth comparing year to year, what really is only a small tiny bit 6% of our HB.
Compared to a much larger drop off that you see with daily injectable growth hormone.
That is a really important piece of data that.
Certainly we will chat with the FDA about.
We do anticipate much as Rick said earlier, a standard phase III trial design of 12 months, but because.
We have a number of subjects already that have gotten past that 12.
12 month timeframe, we're moving subjects.
From our 24 month long.
Our 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 III.
Get to an NDA. So I think that data will be very helpful. In addition to what we see in the phase III 12 month study that Rick described.
So it is it is very important and it will be a very nice package of durable data that we can bring.
Okay.
That's fair.
Very helpful. Thanks, Thanks, a lot guys.
Thank you.
And I'm showing no further questions in queue at this time, ladies and gentlemen, this concludes <unk> pharma fourth quarter 2023 earnings call.
Thank you and have a good day.
Yeah.
[music].
Okay.
Okay.
[music].
Yeah.
<unk>.
[music].
Okay.
[music].
So.
Dan.
[music].