Q4 2021 Lumos Pharma Inc Earnings Call
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Yeah.
Good afternoon, and welcome to the most for most of the fourth quarter of 'twenty 'twenty. One results conference call. Currently all participants are in a listen only mode. Later, we will conduct a question and answer session and instructions will follow at that time as a reminder, this conference call is being recorded.
I will now turn the call over to Lisa Mueller Senior director of Investor Relations Ma'am. Please go ahead.
Thank you operator before we proceed with the call I would like to remind everyone that certain statements made during this call are forward looking statements under U S. Federal Securities laws. These statements are subject to risks and uncertainties that could cause actual results to differ materially from historical experience or present expectations.
Information concerning factors that could cause actual results to differ is contained in our periodic filings.
<unk> 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 8-K, which may be accessed from the inverse.
<unk> page at the company's website joy.
Joining today's call will be Rick Hawkins, CEO , and chairman, John Mchugh, President and Chief Scientific Officer Dr.
Dr. David Karp, our Chief Medical Officer, and Lori Lolli, Chief Financial Officer.
Following prepared remarks, we will open the call to questions I will now turn the call over to Rick.
Thank you Lisa and good afternoon, everyone and thank you for joining us on today's call.
After the market closed today, we issued a press release announcing our new plan for an interim analysis of two of our ongoing trials and our financial results for the full year 2021.
On today's call I will provide a clinical update before turning it over to Lori who will review our financial results.
And John and David who will join us for Q&A session.
I'll begin with the exciting news, we announced today for off road to 10 to 12 trials. We're very pleased to report the screening and enrollment trends in both trials are sufficient for us to announce interim analyses of clinical and safety data.
The results of which we expect to report by year end 2022.
Early look at the data, we will evaluate the safety and annualized height velocity on three dose levels of <unk> 201 against the standard dose of recombinant human growth hormone and 40 patients.
Six months on therapy.
We look forward to providing this preliminary assessment of 120 ones potential to treat idiopathic PGA see patients who would otherwise face years, a burdensome injections as their only course of treatment.
Now as a reminder, our order growth to 10 trial as our global clinical study evaluating oral <unk> zero, one and approximately 80 patients diagnosed with idiopathic PGA ste.
Primary clinical outcome as annualized height velocity and patients selected by our predictive enrichment marker or <unk> strategy.
The key clinical endpoint is a comparison of the annualized height velocity of lunar 201 at three dose levels.
One six and 3.2 milligrams per kilogram versus a control arm of patients treated with injectable recombinant growth hormone daily dose 0.24 milligrams per kilogram per week.
Dose levels of the Bloom to zero, one were selected to span the entire dose response curve elucidated prior PK PD study.
The goals of our Oregon to 10 trial are to identify the optimal dose of bloom to zero <unk> to be used in a phase III registration trial as well as validate the PGM strategy.
The Oregon to 10 trial is nearing the 50% enrollment milestone and essentially all of our trial sites, excluding Ukraine and Russia are open for enrollment.
Given the recent events in Ukraine, and Russia, we have suspended all clinical activity of 12 locations in Russia, and unable to enroll patients in Ukraine due to the ongoing conflict.
We are therefore now targeting approximately 40 sites for oral growth 210 trial.
The enrollment and screening at these approximately 40 sites have maintained an encouraging trajectory and therefore, we continue to anticipate primary outcome data on 80 patients from Org ROE to 10 in the second half of 2023.
The ongoing conflict may however, adversely impact our business in the future and it remains too early to evaluate all the potential effects of this crisis.
Before moving on I'd like to express our concerns and our support for our research colleagues and the people of Ukraine as well as our sincere hope for their safety and rapid restoration of peace.
Yeah.
So enrollment continues to meet our expectations and our Org wrote 212 trial, our single site open label trial evaluating the pharmacokinetic and Pharmacodynamic effects of loom to zero, one and up to 24 P. Ghd patients at two dose levels 1.6, and $3 two milligrams per cut.
Per kilogram per day.
We're pleased to report that the minimum subject enrollment we set for the interim analyses will soon be met with 10 subjects and we anticipate announcing the results of this interim analysis evaluating safety and height velocity data by the end of 2022.
You'll recall that the objective of our 212 trial is the confirmed prior clinical data demonstrating the amplified pulsatile release of endogenous growth hormone unique to loom to zero, one and the potential for this mechanism of action to increase growth from its accretion across the entire dose response curve.
And the majority of P ghd patients.
The primary endpoint is six months of PK, PD and height velocity data with a total of 12 months of height velocity data to be captured in this trial.
Turning now to our Org 213 trial or the exploratory switch study we have initiated an open label multi center phase II study evaluating the growth effects and safety of <unk> 201, following 12 months of daily recombinant growth hormone and up to 20 <unk>.
Patients who have completed the world growth to 10 trial.
Subjects will be administered loom to zero, one and dose level of 3.2 milligrams per kilogram per day for up to 12 months.
Primary outcome for the 2014 trial is annualized height velocity and secondary outcomes include safety and other PD measures.
In addition to advancing our clinical trials for <unk> 201, and <unk>, we continue to explore expansion opportunities for lunar 201 into other therapeutic areas, where injectable recombinant human growth hormone is the standard of care.
We believe that loom to zero, one as a pipeline in a product and through its unique mechanism of action may have the potential to treat many of the indications comprising a $3.4 billion growth hormone market such as probably.
Okay.
I'll pass.
Over to Laurie for a review of our full year 2021 financial results Laurie.
Thank you Rick and good afternoon, everyone. We ended our fiscal year end December 31st 'twenty, 'twenty, one with cash and cash equivalents totaling $94 8 million compared to our prior year cash balance of $98 7 million. We expect an average cash use of approximately eight five to $9 5 million per quarter through 2020.
Cash on hand as of year end 2021 is expected to support our operations through the primary outcome data readout for both work with to turn and Ora to 12 trial right.
Research and development expenses were $16 2 million for the full year ending December 30, <unk> 'twenty 'twenty. One this is an increase of $7 million over the R&D expenses of $9 2 million for the full year ending December 30, <unk> 'twenty 'twenty. This.
This increase was primarily due to increases of $5 5 million in clinical trial and contract manufacturing expenses and 2 million in personnel related expenses.
Our G&A expenses were $15 $3 million for the full year ending December 31st 'twenty 'twenty. One. This is a decrease of $1 9 million over the G&A expenses compared to the same period in 2020. The decrease is primarily due to decreases in legal and consulting expenses, which were higher in 'twenty 'twenty do.
Due to merger related expenses.
Net loss for year ended December 31st 'twenty, 'twenty, one with $38 4 million compared to a net loss of $5 7 million for the same period in 'twenty 'twenty.
I will now turn it back to Rick for closing remarks.
Thank you Laurie and closing our fourth quarter and the start of 2022 have been productive for Lou most and we are cautiously optimistic for the year ahead enrollment.
Enrollment in our oral growth trials is progressing despite numerous challenges over the course of the prior year and were now able to provide an early look at loom to zero one efficacy in patients with idiopathic PGE HD with interim data for both our 210 and 212 trials by the end of 2022 .
Based upon prior trials of growth hormone and PGA D. We believe these data from both or growth to Tien and Oregon, <unk> trials should be adequate to provide an initial indication of lunar two zero one's impact on height velocity compared to growth hormone.
Our position is strong and sufficient to carry us through the primary outcome data readouts for both of these trials. So we look forward to updating you on our progress as the year unfolds.
And operator, we're now ready to take questions.
Thank you as a reminder to ask a question you will need to press Star and then the number one on your telephone keypad again, just press Star and then the number one on your telephone keypad and do we draw. Your question just press the pound key.
Standby, while we compile the Q&A roster.
Your first question comes from the line of Charles Duncan with Cantor Fitzgerald, Sir you May proceed with your question.
Thank you.
Afternoon, Rick and team congrats congratulations on your.
Your progress and thanks for the update on the clinical trials.
So had a couple of questions.
Specifically with regard to the <unk> homes, I guess I'm wondering if those were discussed with the FDA or if that wasn't necessary since here that youre going to be looking on a blinded basis.
Secondly, I'm wondering if you could provide us some additional color on the predictive it predictive enrichment markers in terms of screen failure rate.
Okay, I'm going to let the first question John Mchugh, if you'd answer that I'd appreciate it.
Sure happy to Rick.
Nice to hear your voice again Charles.
So the 212 study as you know as we settle along is an open label study and we knew we were going to look at that data is as time went on for the 210 study.
We've simply reached.
A time, where it's appropriate.
Number of patients per cohort that we feel very comfortable taking a peek at that data.
Seeing how they play out when we compared to that same group of patients and there were common human growth hormone arm.
Okay.
Will there be any kind of.
Stopping rules say for futility or some certain amount of efficacy that you'd like to see that continue or are you just taking a look and we'll be continuing to enroll I guess you will have already enrolled quite a few more patients at that time anyway right.
Yeah that is that is correct, yeah, and we we.
Obviously the data are what the data are will respond to them when the when we get to see them.
But are you know our plan is to fully enroll this trial and look at the dataset.
When it rolls out, but I, you know I will say that the cohort size of it.
For 210 of about 10 patients for each cohort and then the top two doses will have an additional four of five patients from the <unk> hundred 12 study that we could combine it it's a fairly realistic sized dataset.
For us to take a look at it really just get a an initial look at how the efficacy is progressing with am 201.
And David would you address that projected enrichment marker in the screen failure question, Charles head I'd be happy to.
Charles Nice hearing you again.
I'm glad you asked about that because we've worked very hard to educate the investigators on the optimal patients screened for the study and they are really coming through as a result.
We have a far far lower screen failure rate based upon the end screening that we need to.
Just a 40% and Rudy.
Can't give you a precise number but it's at least a fourfold lower screen failure rate based upon the time.
So that's going very very well if I could just add a little bit extra to your first question.
Hmm.
But are you supposed to ask would be that the interim analysis in the 210 said he actually is in the current protocol that was it.
To mitigate the FDA.
We were only announcing it now because we because of the enrollment we have confidence that we will.
Had the interim analysis by the end of the year.
Okay. That's helpful added color I appreciate that David and then one quick follow up and then I'll hop back in the queue and that is relative to the switch study I guess I'm not sure I fully understand it maybe stay something when you were talking about it but.
In terms of the patients that will switch those are patients who are on the 210 study and on the on the control arm and then they will switch over to.
To the.
The agent or how will it exactly work, yet and then I'll come you're working with the highest dose.
That's right Charles but David If you go ahead and answer the rest of that question go ahead sure yet it's precisely right Charles so the.
The eventual 20 subjects, who will be in the 210 steady on.
Nordic dropping becoming human growth hormone will.
We will have the opportunity to transfer into this.
Early switch study so they can get 12 months treating with loomed tool one.
And we chose that dose because at the present time seen good tolerability across all the doses and knowing the dose response curve.
The prior PK PD study, we thought it made sense to put them on the top dose.
So.
That's why the design is what it is.
Okay.
Okay. Thank you for taking my questions.
Yes.
Thank you. Your next question comes from the line of Yes mean Rahimi with Piper Sandler Ma'am. Please proceed with your question.
Tim can you guys hear me.
Yes, we can great.
Great. This is Jesse on for yes, I had a question about the <unk>.
Term analysis for 210.
So what are you guys expecting to see in terms of height velocity on safety and Tom.
And I guess same question goes for four to 12 as well.
Thanks Damon.
Inventory.
Endpoint.
John Why don't you go ahead and answer sorry with that question Okay.
Absolutely so.
I see.
Every almost every phase II study that's been run in this space with either growth I'm under long acting says generally run a comparator arm of recombinant human growth alongside their exploratory cohorts.
And we've done the same and we've set up.
We've subset the population by using our enrichment marker to enrich in patients that can be responsive and and really the key thing here is now that we have a clinical study that's looking only at patients that we selected with our markers. It was really just to compare how our arms are doing compared to the concurrent recovery human growth.
One arm.
So that's really going to be a key piece of data that comes out of this.
You know and will work well that's one of the key things that we'll be looking at right. We do have three doses of our molecule.
We'd be very excited to see a dose response, we would predict there'd be a dose response somewhere across those three doses and we'd like to see see that as well, but really the key is comparing and trying to get.
Tried to identify the dose that gives us a comparable growth to the recovery of human growth hormone arm that's run concurrently.
Great. Thank you.
The same same reason for that for the 212 or what.
So.
The trial.
Yeah, So for 212.
There's a lot of data that we're generating.
The initial data that we're going to have the opportunity to look at it is really just annualized height velocity and since these kids are essentially the same patient population as the 210. It does enable us to two as a post hoc kind of mix, but the two sets of kits together and increase the b N of the top two doses.
So I think you know.
Longer term, we have a lot of pharmacodynamic data, that's going to come out of that trial takes a little bit longer to pull that out and analyze it then it does annualized height velocity. So right now we're just focusing on the annualized height velocity for the 212.
Great. Thank you.
Yeah.
Thank you.
Thank you. Your next question comes from the line of Ed White with H C. Wainwright Sir Please go ahead.
Good afternoon, Thanks for taking my questions.
So I think most of them have been asked already.
Maybe just on the number of sites open.
With the <unk>.
War in the Ukraine, and Russia, you had mentioned that you were now planning.
For targeting 40 sites and I think it is.
In November you had mentioned that you had more than 40 sites open.
Where are you now after removing the sites for Russia and the Ukraine.
Ed Thanks for that good question and I think it's a question that everyone in our industry, who has Ah study sites in that part of the world are facing.
We had 95 five in Russia for in Ukraine, as we told you that everything is on pause.
No patients had been randomized from those two sites.
So even without.
Those locations.
Locations are patients coming from from those locations.
We're really encouraged about our enrollment.
We're also.
Adding a few sites here and there.
But.
Things are going well as we speak and we think that that will we certainly believe that we will reach the goal that we just mentioned in our.
Our prior discussion.
Before the end of 2023, and but things are going well in spite of that.
Yeah.
Okay. Thanks, Rick and I guess, just the last question.
That we all ask each quarter. It's just you always mentioned.
Other indications and I was just wondering if there if you're making any progress in any other indications.
Or.
Has there been any change to the strategy or anything that any one indication that you're prioritizing now over the others. Thank you.
Yeah, John you want to answer that question.
Sure as we said you know we've we've dug in and looked at a lot of.
These different secondary indications thought about how our unique mechanism of action of limb to a one would fit into those settings and we've always wanted to get a sense of what the dose responsiveness of Bloom two O. One would be in the P. G. H D population first and that really does help us.
Kind of do the final refinement of where where we plan to go for the secondary indication. So we're getting to a point our I think.
Once we have that data, we'll be able to.
Finalize our discussions and our decisions on those secondary indications.
Yes.
So Chad.
<unk>.
Just thinking ahead since you're planning on announcing interim data in the second by year end.
Is that when we should expect to hear more.
Well, yeah, I mean, we were expecting by the very end of the year, we do want to look at that data and that tried to apply it across the secondary indications we've done much of the footwork, but it really.
When the data comes in we'll be able to make some decisions about how it applies to other indications but.
So I won't I won't promise right, then, but probably shortly thereafter.
Okay. Thanks for taking my questions.
Thank you and our next question.
Thank you. Your next question comes from the line of gasoline Nowak with children's Research Ma'am. Please go ahead.
Hi.
Hi, Catherine.
Thanks, so much for taking my question.
I have.
Question on the interim analysis for 212. In addition to the annualized height velocity velocity I guess I'm kind of curious about whats key to understanding the 24 hour Pulsatilla D and these.
Positive enrichment Margaret.
Patients.
Yeah.
Can you remind me how many 12 hour pulsatile the assessments, we're gonna get during the 12 month dosing period, and what what do we expect to see in terms of change from baseline as far as how that look.
John I think youre closer to the data from the prior study but also.
Possible about the details of the blood draws.
Once you start in and David you can add.
Great.
Nice to chat with you again, Katherine I think you know the data. We've shared previously is on the 0.8 Meg per kg per day.
Cohort from the O to O study.
And we've shown kind of two P M positive patients and how they how.
How their pulse fertility changed generally looking at it in the context of just a visual representation, but also in terms of AUC.
And that's kind of the baseline data that got us excited about repeating the study on a larger scale.
In that study, we're not doing any 0.8 Meg per kg per day kids, just the top two doses 1.6, and three point too.
And at baseline and at six months on treatment, we will do the same pulse utility assessment. It would be every 10 minutes sampling for 12 hours after they take their morning dose of <unk>.
Two one so we'll get a very.
A very nice assessment.
Of how their pulse utility changes with respect to their baseline and I think that will be a very interesting piece of data at those top two doses gives us an opportunity to potentially look at dose responsiveness on the PD marker and it also gives us a little bit of a sense of trying to understand you know when we module.
Blake.
Growth hormone levels in it in a pulsatile fashion by amplifying each of those individual peaks what impact does it have really on the 12 or 20 for growth hormone levels and how does that relate to growth at six months.
Those P. D analyses are pretty complex and theyre going to take a little bit more time than just calculating the annualized height velocity. So so that full PD analysis, which I think ties very nicely and with the mechanism of action of Alimta or one.
We will take a little bit longer than the annualized height velocity that we expect to be able to show you folks at the end of the year.
Does that help her.
Yeah that's helpful.
I think it's just an interesting.
Measurement, so kind of curious to get get more information about how to how to be preparing to set the.
When they come out.
Hmm.
My second question was the bound.
2014 study.
This already factored into your cash runway and you still expect to be.
Cash efficiency until the after the oral growth Q.
<unk> six month data.
And then another question with you now.
I wasn't sure how many patient FDA wanted 12 months safety data on so you know can at 213 study would be used to accelerate the 12 month safety data requirement that was previously announced.
Let's answer the financial question first and Laura go ahead.
Hi, Catherine.
The cash at the end of 2020 , one as we said is sufficient to get us through both the Readouts for 210 and 12 on the primary data, which is the six month treatment and then also continue to support operations. During those times does that answer your question.
Yes that answers that question. Thanks.
And then John the 12 month safety.
So I think overall, what we've tried to do with our phase two program has built a really comprehensive and deepen dataset right. So we have the 210 study where we're going to have all of those patients extended for 12 months, who have taken 12 additional to 12 months for.
For those patients who are on live to a one and then this new study basically takes.
The patients who were on or the subjects, who are on the we're kind of human growth hormone are many who.
Entered this trial because they wanted they thought the odds are good they would get an oral at not a daily injectable, but they they drew the short straw and took an injectable every day, we did want to make sure that they had an opportunity to take an oral growth hormone secretagogue for a year and see and see how they responded to that I think all of them.
The safety data collecting 12 months of data and our 210 study.
And our 212 study. In addition, the switch study is really going to build a very nice dataset I think both in terms of efficacy.
This little switch study will give us some interesting data.
About kids, who could switch from common human growth hormone onto loomed tool one it's not large enough to really be.
Very robust, but I think with 20 patients will get a very good insight into how house, which patients would respond and they're all P. A positive kids because they were P. A positive to get into the 210 study. So I think I just look at it in terms of how robust the data set is going to be when we finish our phase two studies and I think it's going to be pretty impressive.
Okay. Thanks, Thanks for that alright.
Alright, congrats on the quarter.
And thanks, Catherine I appreciate it very much.
Thank you. Your next question comes from the line of Adam or P. Ross with Ross Capital Partners. Sir. Please go ahead.
Yes, hi.
But I'd like to clarify Rick.
I think Charles alluded to the interim analysis being blinded I wasn't sure. If that is the case if you could please clarify that.
Yeah. Good question Elmer and John go ahead, if you will.
So I think we have obviously, one arm getting a daily injectable and the other arms getting an oral right. So I think there this isn't double blinded by any sense. So I think.
So I think we have the opportunity to look at this data and analyze it obviously we are.
You know, we're going to blind the folks, making medical decisions to the data right.
And so in what way.
Compromised continued enrollment.
Widely successful.
Okay.
I didn't I didn't understand that in what.
In what way.
If you announce that they're on.
World.
201 is a critical and they had the highest dose to.
Daily growth hormone injections.
Is there a risk that patients.
I would want to.
Actually what I wouldn't want to continue to enroll or be treated.
Maybe a very hypothetical.
Yes, David once you answer that question as a clinician store.
<unk>.
The eight based on when the interim analysis data will be announced I don't know that we would have liked.
Any more enrollments during the fall quite honestly.
But so that would be a theoretical.
A concern, but I think it will not have any impact.
Okay. Okay. Thank you. Thank you for clarifying that.
And just to make sure that I also understood correctly. So there hasn't been any patients enrolled from the Russian and Ukrainian sites.
There is not no patients randomized to either of those two items.
These two countries.
Okay. Okay.
And that was all thank you so much and we'll see you next week at our conference.
Looking forward to it in person.
Yes. Thank you.
<unk>.
Thank you and your last.
Question would be a follow up from Sir Charles Duncan with Cantor Fitzgerald, Sir. Please go ahead.
Uh huh.
Oh, I'm, sorry about that I was on mute thanks for taking the follow up.
Appreciate L a mere pointing out the blinded thing.
But I do wonder whether or not there.
There will be any opportunity to make this an adaptive design trial. So you you feel like at that time, you'll have there.
Trial completely enrolled and then and therefore patients randomized to the dose and no opportunity to make it adaptive.
John Why don't you answer <unk> question.
So our current predictions are that we're not going to be quite fully enrolled by the time, we read that out but the vast majority of folks will be enrolled in the study. So I think there's there's not that much opportunity to to make a difference with an adaptive design.
Okay.
And then kind of as a follow up I'm just I'm just wondering about guidance for that.
I can't half of the year full data on that.
Let's say that you know sometime in December you're fully.
Enrolls you do the interim would seem to me that certainly by June you may have the data and I understand it takes a while to work through the data set et cetera, but is that what's really governing.
Timelines, maybe a little conservative with them.
Data analysis.
Chad Thanks for asking that excellent question I mean, I think you know us by now we're trying to be conservative in our approach.
Two two to the market and making sure that we follow through and we do exactly as we would tell you where we're going to do.
You know there I think these are still uncertain times.
We certainly hope that enrollment is going to continue.
At the pace that we're currently seeing.
You know we were counting on enrolled.
Enrollment from from Ukraine, and Russia.
In other parts of the world are going to have to to pick up.
So I think it's a little too early to.
<unk>.
That will happen before the end of 2023.
But.
You know as usual.
We'll be one of the first to know.
I'm, just hoping to St.
Same time, everyone else knows but.
Okay.
Okay.
Yeah. So let me let me ask you about 2014.
<unk> talked about there being 20 patients that you intend to enroll in that study and it seems like a really interesting study so I'm kind of wondering.
While you wouldn't enroll all 40 patients who are on the control arm from 210.
David wanted to answer that question.
Sure the.
The pulte.
<unk> study is enrolling 80 subjects 20 subjects in each group. So the 20 subjects is the intended enrolment of the control arm.
Charles Thanks for clear got it okay. Thanks for clarifying.
To add on that.
Okay.
Bill Lennie on randomized.
Human lipoma.
But you do have an O L E that you intend to M. A.
Switch people, our patients over and give us an update on that later on this year.
Yes.
Yes, we will.
Okay. Thanks for taking my follow up.
Okay.
Thank you I'm showing no further questions in the queue at this time, thank you for joining us and enjoy your afternoon.
Thank you Rachel you May now disconnect you're welcome have a good day.
Alright Bye bye.
Yeah.
Battery.
Hum.
Yes.
Yes.
Okay.
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Okay.
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