Q3 2021 Lumos Pharma Inc Earnings Call
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Okay.
Good afternoon, and welcome to farmland take like a reserve.
This 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.
Our director of Investor Relations.
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.
<unk> 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 the poor thinking state.
Yeah.
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 investors page of the company's website.
Speaking on today's call will be Rick Hough, CEO and chairman.
And Laurie Lolli, Chief Financial Officer, following their prepared remarks, Rick and Lori will be joined by John <unk>.
Our president and Chief Scientific officer, as well as Dr. David B Karp I, Chief Medical Officer for the class action and answer session I will now turn the call over to Rick.
Thank you Lisa good afternoon, everyone and thank you for joining us on today's call after.
After the market closed today, we issued a press release announcing our financial results for the <unk> 2021 third quarter and detailing our progress advancing <unk> 201.
The treatment of pediatric growth hormone deficiency or P. G H D.
On today's call I will provide the overview of our lean to zero, one program and broader development strategy and give an update on our clinical trials and Lord Lloyd will review our financial results then we'll be happy to take your questions.
So I'm pleased to report that since we last spoke with you. We've made steady progress on our lunar 201 program. The majority of sites for oil growth to 10 trial are now open for enrollment and importantly, most of those that we opened recently or will open soon our historically high enrollment sites.
With screening and enrollment for oil growth to 10 progressing we continue to expect the primary outcome data readout for this trial in the second half of 2023.
Enrollment has picked up in our oral ROE to 12 trial, you'll recall that oil growth to 12 as a single site open label trial designed to provide pharmacokinetic pharmacodynamic data on them to zero one.
Primary endpoint at six months data with additional 12 month data to be captured.
Since the trial is open label and print interim analysis may be conducted at the company's discretion.
Yeah.
As a reminder, our or grow to 10 trial is a global clinical study evaluating oral loom to zero, one and approximately 80 patients diagnosed with P. G H D.
Primary clinical outcome is annualized height velocity and patient selected by our predictive enrichment marker or P. M strategy.
Secondary outcome measures include comparisons of annualized height velocity of lunar 201, and three doses and three dose levels 181.6, and three point to the mix per kg versus a control arm of patients treated with recombinant growth hormone at a daily dose of <unk> to four <unk> per kg per week.
Dose levels of 1201 were selected to span the entire dose response curve elucidated in a prior PK PD study.
The goals of our or our ROE to 10 trial are to identify the optimal dose of bloom tool one to be used in a phase III registration trial and validate the P M strategy.
During the last quarter, we made significant progress toward our goal of opening approximately 50 sites.
Currently we have over 40 sites open and screening patients.
Additional high volume sites expected to open surely include those in Russia, Ukraine, New Zealand and Israel and.
And based on the progress made in recent weeks, we feel confident that enrollment supports our six month data readout for the oral growth to 10 trial in the second half of 2023 with additional 12 month data to be collected.
Turning now to our oil growth to 12 study. This is our our single site open label trial evaluating the pharmacokinetic and Pharmacodynamic effects of little to zero, one and up to 24 P. J T patients at two dose levels 163.2 mix per kg per day.
This trial continues to enroll patients at an encouraging pace.
The objectives of the or a road to 12 trial is to confirm prior clinical data demonstrating the ample amplified pulsatile release of endogenous growth hormone unique to allude to zero, one and the potential for this mechanism of action to increase growth hormone secretion across the entire dose response curve.
And the majority of P ghd patients.
The primary endpoint at six months of P. J P D.
And high velocity data with additional 12 month data to be captured.
And given the open label design of this trial, we have the ability to perform an interim analysis analysis at our discretion.
Now in addition to advancing our clinical trials for <unk> 201, and P. G. H D. During the quarter, we continued to explore expansion opportunities for lunar 201 and into other therapeutic areas, where injectable recombinant human human growth hormone is a standard of care.
As we've said previously we believe that 1201 is a pipeline in a product and through its unique mechanism of action may have the potential to be efficacious in indications such as Turner syndrome, <unk> Willi syndrome, idiopathic short stature and children born small for gestational age.
We are actively reviewing potential clinical development plans for <unk>, two zero or one and several of these indications and are in advanced discussions with key opinion leaders and our clinical and scientific Advisory Board about the best next step for expanding our lunar 201 pipeline.
So we look forward to providing a more detailed update on these plans soon.
We also continue to be judicious in our pursuit of rare disease assets beyond move to zero, one with careful attention to shareholder value creation.
That said, our priority remains low to zero, one and its development.
So before I turn the call over to Laura I, just want to remind everyone of some context surrounding our lunar two zero on program and some of the reasons why we have confidence in our current clinical trials and our potential boom 2201.
Generally.
Now you may recall, unlike standard of care P. J steep therapies or the weekly injectables coming to market them to zero. One is an oral growth hormone secretagogue that acts within the body's natural integrin pathway.
The move to zero, one acts selectively on receptors in the pituitary and hypothalamus.
Stimulate the body's ability to release growth hormone at the same intervals and subject to the same in Oregon feedback loops that occur naturally.
This mechanism enables the naturally occurring IGF, one feedback loop to be preserved to help regulate the balance of growth hormone and IGF one levels in the body.
So less severe P ghd patients or those with an intact pituitary growth hormone axis, but who are secreting insufficient growth hormone to attain normal height or those who should respond best to move to zero one.
Evidence suggests that this group represents approximately 60% of the total P. J C population in our trials are designed using specific predictive enrichment markers or pms to identify this addressable population.
And with that I'll pass it over to Laurie.
For a review of our third quarter financial results Laurie.
Thanks, Rick and good afternoon, everyone.
Our third quarter research and development expenses were $4 $1 million compared to 2.1 million in the same period. In 2020. This increase of 2 million was primarily due to increases of $1 8 million in clinical trial and contract manufacturing expenses $400000 in personnel related expenses and 100000.
Stock compensation expense offset by a decrease of 300000 in supplies and other expenses.
General and administrative expenses in the third quarter F. 'twenty, 'twenty, one or a $3 4 million compared to $5 2 million in the third quarter of 2020. This decrease of $1 8 million was primarily due to decreases of $1 3 million in personnel related expenses and 500000 in legal consulting and other operating expenditures.
For the third quarter, we recorded a net loss of $7 5 million compared to net income of $1 8 million for the prior year quarter.
In Q3 of 2020, the company recorded a gain of $6 5 million related to the sale of our priority review voucher and a tax benefit of $2 4 million during Q3 of 2020, which offset the operating expenses in that quarter at $7 2 million.
We ended the third quarter F. 2021 with cash and cash equivalents totaling 1.7 million compared to $98 7 million on December 31st 2020, We currently expect our cash on hand as of the end of third quarter to support operations for the six month data Readouts from both our core growth to Ted and Oregon to 12 trials.
I'll now turn it back to Rick for closing remarks.
Thank you Lori and before taking your questions just to remind everyone that our immediate focus is on the execution of our current clinical plan for Loopnet zero, one and exploration of the indications we will pursue next to expand this platform.
While lifecycle management of lunar 201 is our priority, we will opportunistically consider other business development upper opportunities as they present.
And then will remain a highly selective and focus on long term value creation for our shareholders. So.
So we look forward to sharing more details with you soon.
So operator, we're now ready to take questions.
Thank you. So asking question you will need to press star one on your telephone could we try a question press the pound key.
To ask a question please press star one.
And our first question is from Charles Duncan of Cantor Fitzgerald. Your line is open.
Yes. Thank you good afternoon, Rick and team thanks for taking our questions and thanks for the quick update.
Wanted to ask you a little bit about the 210.
You mentioned high enrolling sites and I guess I'm wondering how you know what what our high enrolling site is did you do some feasibility study.
To be able to demonstrate that and then with regard to timing.
I know that you have or deploying the predictive enrichment marker paradigm in and that makes sense, but could you remind us what your screen two enrollment assumptions are and give us a sense of color to the timelines for six month read out in that study.
Yeah.
Thank you for the question Charles.
You know in terms of the high enrollment sites I think these investigators are incredibly well known and as you know our growth hormone commonly growth hormone has been around for 35 years and there've been a number of clinical trials have been conducted over the years I think are mainly looking at data from.
Those trials in terms of investing.
Investigators in there and their enrollment patterns, there they become pretty clear and as I said.
Some of those sites of those high enrolling sites are now.
Coming online so our expectation of having.
And increased enrollment is certainly there.
We havent released any information on screen failures.
Or I'm not sure it's as relevant in a trial like this especially when we're using a predictive enrichment marker or P. M.
But we haven't released any data there Charles.
Okay, well I guess I'm, assuming you're you mentioned.
Later on in your comments that.
Patients who may respond.
You know two to growth hormone, but in.
In adequately represent maybe 50% of the patient population so.
That's probably a good place to start but I guess I'm wondering if you think that patients who you know.
They present, two clinical trials may be enriched in in any way in terms of.
More of them presenting that may respond to P M or not.
You know John do you want to answer that question.
John I think you're on hold.
You're on mute.
Yeah.
Alright community here can you hear me now yeah, I think that's the right now.
So you know the data indicates that upwards of 60% of patients diagnosed with P. J H D should be responsive to our molecule just based on database analysis and kind of the general.
Division of growth from a deficiency spectrum.
So I think with that said.
We.
We've done a lot of work to educate all of our investigators on what type of patients are going to be our most responsive to our molecule and and they've actually done quite a good job at.
At finding patients who fit the needs just generally for a patient with.
What's called kind of idiopathic growth from a deficiency, so not organic not the most severely growth I'm a deficient kids.
And so I think you know the screening rates, we're seeing right now or don't align with screening studies.
Studies that we've done in database large databases of everyone's diagnosis of course have a deficiency. So what I think we are getting a fairly high return rate.
Because the clinicians on our sites are pretty cognizant of the kind of patient who's going to respond to or therapeutics.
Okay very helpful. Last question is back to two or two to 12.
212 study.
Rick mentioned conducting an interim analysis that would be conducted at the company's discretion and I guess I'm wondering what kind of factors you may consider in doing that interim analysis does it have something to do with the protocol specified or a certain number of patients.
We would like to hear updates so looking forward to that but just kind of wondering what would drive that decision.
Yeah go ahead John.
So I think what we'd like to see out of that interim analysis.
Essentially a large enough and at each of the two doses that were exploring so at 1.6 and $3 two mix per kg per day, but you know that we you know we have enough. We have a data set that I think is important to share.
You know we've released data on the 0.8 make per kicked dose in three patients two P. M positives at one P. M negative and that's great kind of a case report type information, but we'd like to have a larger cohort when we released the higher doses.
And you know we have a we have defined that in our protocol.
And as you know as soon as we reach that that.
Interim decision will will move forward with that.
Okay. Thanks, John Thanks for taking the questions right.
Thank you Troy.
Next question is from yes mean rahimi of Piper Sandler Your line is open.
Hi can you hear me.
Yes.
Oh cool.
This is jesse on for yes.
Congrats on the quarter and thank you for taking our questions from our call.
I wanted to kind of get into that pipeline expansion I know that during your prepared remarks you.
And talking about essentially Turner syndrome and.
Great.
And I just wanted to know.
How do you.
How can you please explain how long until one works and woodwork in these indications and which one.
But limo most prefer to go after.
So John once you go ahead with the mechanism of action in those patient population.
Yes, so I think the way we've looked at this is is there.
A group of patients who who.
Yeah.
Have an active access that can be stimulated with the growth of them, it's accretive goglet gloom to want to increase their growth hormone release, Brian and that in turn increases IGF one downstream modulators.
And for growth or <unk> deficiency, it's it's for pediatric growth hormone deficiency. It aligns perfectly with our mechanism. If we choose our patient populations correctly and we spent a lot of time, just aligning our thinking around many of the other indications right, where there are kind of a constellation of things that might affect you know for many of them.
Individual childs.
Perth patterns right.
Some of those other indications are a little bit more complicated and so what we're spending time doing right now is weighing the different contributions.
And looking at how successful doses of growth on our in stimulating growth now that all these approved indications are growth driven there are indications for adults, but that's just an example of how we are trying to make sure that our mechanism.
And the kind of patients who can respond to our molecules those who have an active axis that can be stimulated with growth from its creek out to increase their growth from a production right. So that's really the thinking we're doing as we go through the 11 approved indications for recombinant human growth hormone and try to prioritize them as we go forward.
Yeah.
And then when you say the update that you can can you expect them on the call or 2022 you know what.
No.
Absolutely.
Hum.
Oh, sorry, I'm like well, we'll have an update on which indication.
On the first two fault or would it be in 2022 or beyond.
We haven't talked about the time, we're going to time period, we're going to do but we were we're actually going through our discussions with with our kols.
And our clinical and scientific Advisory board at the moment and we're not quite there yet, but but I think so we will we'll be able to tell you which ones we're going to first.
Alright. Thank you so much cool that's another question.
And our next question is from Albemarle Perez of Roth Capital Partners. Your line is open.
Good afternoon Rick.
And I just had.
One question. Please I just wanted to verify that are there clinical trials Gov information that's correct right.
I see that there are 42 sites or now open.
40 out of 42.
He's actually recruiting and you I think you plan to add 50 altogether, just trying to see how close to the final number.
Sure.
Yes.
Sure that that the site keeps up with the total number.
But at least we have at least Theres 40 sites.
The open.
And recruiting patients and as we speak where we're active.
Activating more of these sites and as I mentioned earlier I think there are some traditional high enrollment sites that are now just coming online. So we're optimistic that that's going to improve our enrollment considerably.
In the very near future.
Thank you for confirming metric.
Thank you tomer.
And our next question is from Catherine Novack of Jones Research. Your line is open.
Hi, Thanks, so much for taking my question I, just wanted to ask kind of like a.
Bigger picture question about the.
Pediatric growth hormone market, you know with the approval of the Transcon G. H I guess, how do you see that shaping the market and especially what have you heard from kols about potential prescribing practices for a weekly.
[noise] hormone you know given that this product is sometimes associated with higher than normal levels of IGF one.
We haven't gotten a lot of feedback we're really focused on our own programs. However, the market.
Released price, where we're oh.
Hum.
More than pleased with you can imagine is a small molecule and a lower cost of manufacturing.
That leaves us a great deal of flexibility in pricing, especially at that level.
Well I think it's a little too early to tell what the response to the in the market is going to be and from the pediatric endocrinologists, but maybe I can I could ask Dr carve too.
Talk about that a little bit more.
Yeah.
Sure.
Again, as we've said.
Let me state.
Worked very closely on that.
Became psychopaths program.
It has been approved I have not even heard yet it's been actually launched.
I would imagine it will take some time to get onto formularies, which is the real the real driver for sales because.
It's really the outpatient is it's the cost of the patient that dictates.
Sales in the market.
And there is no question that.
The cost of goods for.
Number 201 will be.
Much less than the cost of good scores cut drove up which does provide quite good flexibility.
Yes.
Yeah.
Got it.
Yeah, I think maybe what I was trying to get at is comfort in general with with Oh weekly.
Have you heard anybody who might be a little bit hesitant to prescribe weekly G. H.
Or.
Or is IGF, one levels not a concern for some kols.
Yeah.
I mean, David if you want to address that at all you shipped sure.
I think that most of the key opinion leaders that I talked with.
I was.
And the development of dissenters.
Did not have a big concern about.
<unk> one levels because traditionally.
Yes.
Because kids with growth hormone deficiency or rarely diagnosed right when they develop it.
There's a lag time to diagnosis to treatment.
You don't want to target.
Kind of the average IGF one value in these kids makes you got five years to treat them to adult height. So you want it tend to be in the upper half of the normal range and there are data showing the target of positive two you get better growth in that you targeted.
It's the idea of when it sits at zero and I guess initially targeting.
When it's just until you can have half about half below so I don't think that it's a big concern and there's no question that on everything else being equal.
Kids and parents, who prefer one injection of weak compared to seven injections per week, but by the same token it's not at all.
Unlikely that a lot of people would prefer a once daily oral compound to even the once weekly injection.
And Catherine I think that as pediatric endocrinologists many of them, we'll wait and see I believe to see what the patient population of reports where their colleagues report overtime I think theres going to be some early adopters, but I also think there'll be some PDR pediatric endocrinologists, who.
We will not jump in right away.
<unk> because of the big excursions of of.
Of IGF one.
Levels that are sometimes up sometimes can be of concern to some some.
Commissions.
Yeah.
Got it.
Thanks very much.
Thank you Catherine.
Yeah.
Okay.
Thank you I am showing no further questions in the queue at this time. Thank you.
Are you for training and enjoy your afternoon.
Thank you very much.
Okay.
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Good afternoon, and welcome to Luna for my third quarter results Conference call.
All participants are in a listen only mode. Later, we will conduct a question and answer session and instructions will follow at that time as a reminder, this conference call is being recorded I will now turn the call over to Lisa Miller Senior director of Investor Relations.
Yeah.
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. 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 part they can statements.
Information presented on this call is contained in the press release, we issued this afternoon and in our form 8-K.
It'd be accessed from the investors page of the company's website.
Speaking on today's call will be recalled skin's, CEO, and chairman and Lori Lolly, Chief Financial Officer, following their prepared remarks, Rick and Lori will be joined by John Mccain.
Our president and Chief Scientific officer, as well as Dr. David Karp, Our Chief Medical Officer for the class action and answer session I will now turn the call over to Rick.
Thank you Lisa 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 financial results for the <unk> 2021 third quarter and detailing our progress advancing 201 for the treatment of pediatric growth hormone deficiency or P. G. H D R.
On today's call I'll provide the overview of our limb to zero, one program and broader development strategy and give an update on our clinical trials and Loren Lloyd will review our financial results then we'll be happy to take your questions.
So I'm pleased to report that since we last spoke with you we've made steady progress on a limb to zero one program.
You're already a site for oil growth to 10 trial are now open for enrollment and importantly, most of those that we opened recently or will open soon our historically high enrollment sites.
With screening and enrollment for our ROE to 10 progressing we continue to expect the primary outcome data readout for this trial in the second half of 2023.
Enrollment has picked up in our off road to 212 trial, you'll recall that Oregon to 12 as a single site open label trial designed to provide pharmacokinetic pharmacodynamic data on them to zero one.
Primary endpoint at six months data with additional 12 month data to be captured.
Since the trial is open label and then put in interim analysis may be conducted at the company's discretion.
Yeah.
As a reminder, our oil growth to 10 trial is a global clinical study evaluating oral loom to zero, one and approximately 80 patients diagnosed with P. Ghd.
Primary clinical outcome as annualized height velocity and patient selected by our predictive enrichment marker or <unk> strategy.
Secondary outcome measures include comparisons of annualized height velocity of them 201, and three doses and three dose levels eight 1.6 and $3. Two it makes per keg versus a control arm of patients treated with recombinant growth hormone at a daily dose of point to for mix per kg per week.
Dose levels of 1201 were selected to span the entire dose response curve elucidated in a prior PK PD study.
The goals of our oil growth to 10 trial are to identify the optimal dose of bloom to or want to be used in a phase III registration trial and validate the PGM strategy.
During the last quarter, we made significant progress toward our goal of opening approximately 50 sites.
Currently we have over 40 sites open and screening patients.
Additional high volume sites expected to open shortly include those in Russia, Ukraine, New Zealand and Israel and.
And based on the progress made in recent weeks, we feel confident that enrollment supports our six month data readout for the oral growth to 10 trial in the second half of 2023 with additional 12 month data to be collected.
Yeah.
Turning now to our oil growth to 12 study. This is our single site open label trial evaluating the pharmacokinetics and Pharmacodynamic effects of little to zero, one and up to 24 P. J P patients at two dose levels, one six and $3 two makes per kg per day. This trial continues to enroll patients at an encourage.
Gene pace.
The objectives of the order of 212 trial is to confirm prior clinical data demonstrating the Apple amplified pulsatile release of endogenous growth hormone unique to lunar 201, and the potential for this mechanism of action to increase growth hormone secretion across the entire dose response curve.
And the majority of P ghd patients.
The primary endpoint at six months of PK, PD and high velocity data with additional 12 month data can be captured and.
And given the open label design of this trial, we have the ability to perform an interim analysis analysis at our discretion.
Now in addition to advancing our clinical trials for them to zero, one and P. G. H D. During the quarter, we continued to explore expansion opportunities for lunar 201.
And to other therapeutic areas, where injectable recombinant human growth hormone is a standard of care.
As we've said previously we believe that the enthusiasm of one is a pipeline in a product and through its unique mechanism of action may have the potential to be efficacious in indications such as Turner syndrome, <unk> Willi syndrome, idiopathic short stature and children born small for gestational age.
We are actively reviewing potential clinical development plans for Luna to zero or one and several of these indications and are in advanced discussions with key opinion leaders and our clinical and scientific Advisory Board about the best next step for expanding our lunar 201 pipeline.
So we look forward to providing a more detailed update on these plans.
We also continue to be judicious in our pursuit of rare disease assets beyond move to zero, one with careful attention to shareholder value creation.
That said, our priority remains to zero, one and its development.
So before I turn the call over to Laurie I, just want to remind everyone of some context surrounding our lunar two zero on program and some of the reasons why we have confidence in our current clinical trials and the potential of them 2201 generally.
Now you may recall, unlike standard of care P. J steep therapies or the weekly injectables coming to market them to zero. One is an oral growth hormone secretagogue that acts within the body's natural endocrine pathway and lunar 201 add selectively on receptors in the pituitary and hypothalamus.
To stimulate the body's ability to release growth hormone at the same intervals and subject to the same interconnect feedback loops that occur naturally.
This mechanism enables the naturally occurring IGF, one feedback loop to be preserved to help regulate the balance of growth hormone and IGF one levels in the body.
So less severe P ghd patients or those with an intact pituitary growth hormone axis, but who are secreting insufficient growth hormone to attain normal height or those who should respond best to move to zero one.
Evidence suggests that this group represents approximately 60% of the total P. J C population in our trials are designed using specific predictive enrichment markers or pms to identify this addressable population.
And with that I'll pass it over to Laurie.
For a review of our third quarter financial results Laurie.
Thanks, Rick and good afternoon, everyone.
Our third quarter research and development expenses were $4 $1 million compared to $2 1 million in the same period. In 2020. This increase of $2 million was primarily due to increases of $1 8 million in clinical trial and contract manufacturing expenses $400000 in personnel related expenses and 100000.
Stock compensation expense offset by a decrease of 300000 in supplies and other expenses.
General and administrative expenses in the third quarter of 2021, we're a $3 4 million compared to $5 2 million in the third quarter of 2020. This decrease of $1 8 million was primarily due to decreases of $1 3 million in personnel related expenses and 500000 in legal consulting and other operating expenditures.
For the third quarter, we recorded a net loss of $7 5 million compared to net income of $1 8 million for the prior year quarter.
In Q3 of 2020, the company recorded a gain of $6 5 million related to the sale of our priority review voucher and a tax benefit of $2 4 million during Q3 of 2020, which offset the operating expenses in that quarter of $7 2 million.
We ended the third quarter of 2021 with cash and cash equivalents totaling 1.7 million compared to $98 7 million on December 31st 2020, We currently expect our cash on hand as of the end of third quarter to support operations for the six month data Readouts from both our core growth to Ted and Oregon to 12 trials.
I'll now turn it back to Rick for closing remarks.
Thank you Lori and before taking your questions just to remind everyone that our immediate focus is on execution of our current clinical plan for Lindsay zero, one and exploration of the indications we will pursue next to expand this platform.
While lifecycle management of lunar 201 is our priority, we will opportunistically consider other business development opportunities as they present.
And then will remain a highly selective and focus on long term value creation for our shareholders.
So we look forward to sharing more details with you soon.
So operator, we're now ready to take questions.
Thank you and to ask any question you will need to press star one on your telephone to withdraw your question press the pound key again to ask a question. Please press star one.
And our first question is from Charles Duncan of Cantor Fitzgerald. Your line is open.
Yes. Thank you good afternoon, Rick and team thanks for taking our questions and thanks for the quick update.
Wanted to ask you a little bit about the 210 stuff.
Study, you mentioned high enrolling sites and I guess I'm wondering how you know what what our high enrolling site is did you do some feasibility study.
To be able to demonstrate that and then with regard to timing.
I know that you have or deploying their predictive enrichment marker.
Diamond and that makes sense, but could you remind us what your screen two enrollment assumptions are and give us a sense of color to the timelines for six month read out in that study.
Okay.
Thank you for the question Charles.
You know in terms of the high enrollment sites I think these investigators are incredibly well known and.
And as you know our growth hormone are predominantly.
Homeowners has been around for 35 years and there have been a number of clinical trials have been conducted over the years.
Mainly looking at data from from those trials in terms of the.
Investigators in there and their enrollment patterns.
They become pretty clear and as I said.
Some of those sites are those high enrolling sites are now.
Coming online so our expectation of having an.
And increased enrollment is certainly there.
We havent released any information on screen failures.
Or I'm not sure it's as relevant in a trial like this especially when we're using a predictive enrichment marker or a P. M.
But we haven't released any data there Charles.
Okay, well I guess I'm, assuming you're you mentioned.
You know later on in your comments that you know.
Patients who may respond to.
Two to growth hormone, but yeah in adequately represent maybe 50% of the patient population. So that's probably a good place to start but I guess I'm wondering if you think that patients who.
They present, two clinical trials may be enriched in in any way in terms of.
More of them presenting that may respond to a P M or not.
Yeah, John do you want to answer that question.
John I think you're on hold.
You're on mute.
Alright.
Can you hear me now yeah, I think that's there right now.
So you know the data indicates that upwards of 60% of patients diagnosed with P. T. H D should be responsive to our molecule just based on database analysis and kind of the general.
Division of growth from a deficiency spectrum.
So I.
I think with that said.
No we.
We've done a lot of work to.
All of our investigators on what type of patients are going to be most responsive to our molecule and they've actually done quite a good job at that.
That finding patients who fit the needs just generally for a patient with.
What's called kind of idiopathic growth from a deficiency, so not organic not the most severely gross number deficient kids.
And so I think you know the the screening rates, we're seeing right now or don't align with screening studies.
Studies that we've done in database large databases of everyone's diagnosis of course have a deficiency. So I think we're getting a fairly high return rate.
Because the clinicians on our sites are pretty cognizant of the kind of patient who's going to respond to or therapeutics.
Okay very helpful. Last question is back to two or two to 12.
212 study.
Rick mentioned conducting an interim analysis that would be conducted at the company's discretion and I guess I'm wondering what kind of factors you may consider in doing that the interim analysis does it have something to do with the protocol specified or a certain number of patients.
We would like to hear updates so looking forward to that but just kind of wondering what would drive that decision.
Yeah go ahead John.
Okay.
So I think what we'd like to see head of that interim analysis.
Essentially a large enough and at each of the two doses that were exploring so one six and $3 two mix per kg per day, but you know that we you know we have enough. We have a data set that I think is important to share.
You know we've released data on the 0.8 make per kicked dose in three patients two P. M positives at one P M negative and that's a great kind of.
This report type information, but we'd like to have a larger cohort when we released the higher doses.
And you know we have a we have defined that in our protocol.
And as you know as soon as we reach that.
Interim decision will will move forward with that.
Okay. Thanks, John Thanks for taking the questions right.
Thank you Charlie.
Next question is from yes mean rahimi of Piper Sandler Your line is open.
Hi team can you hear me.
Yes.
Okay. Thank you. This is Jesse entre, yes, congrats on the quarter and thank you for taking our questions from our call.
I wanted to kind of get into that plant and expansion and I know that during your prepared remarks you.
I'm talking about essentially Turner syndrome and prudently.
I just wanted to know how how do you.
How can you please explain how long until one works and woodwork in these indications and which one.
But.
Linda most prefer to go after.
So John once you go ahead with the mechanism of action in those patient population.
Yeah, So I think I'm the way we've looked at this is is there a group of patients who who.
Have an active access that can be stimulated with a growth from its accretive doglike look to want to increase their growth hormone release, right and that in turn increase the side, Jeff one downstream modulators.
And you know for growth or <unk> deficiency, it's it's for pediatric growth hormone deficiency. It aligns perfectly with our mechanism. If we choose our patient populations correctly and we spent a lot of time, just aligning our thinking around many of the other indications right, where there are kind of a constellation of things that might affect you know for many of them individually.
Old childs.
Perth patterns right. So some of those other indications are a little bit more complicated and so what we're spending time doing right now as you know weighing the different contributions.
And looking at how successful doses of growth somewhat arent stimulating growth now, but all of these approved indications are growth driven there are indications for adults, but that's just an example of how we are trying to make sure that our mechanism.
And the kind of patients who can respond to our molecules those who have an active axis and can be stimulated with growth from its creek out to increase their growth from a production right. So that's really the thinking we're doing as we go through the 11 approved indications for our recombinant human growth hormone and try to prioritize them as we go forward.
Thank you.
And then when you say the update there and can we expect them on a call or 2022.
You, let us know.
Absolutely.
Hum.
Oh, I'm, sorry, and I like where we have an update on which indication hum on the person or whether it be in 2022 or beyond.
We haven't talked about the time, we're going to time period, we're going to do but we were we're actually going through our discussions with the with our Kols.
And our clinical and scientific Advisory board at the moment and we're not quite there yet, but but I think soon we'll be able to tell tell you, which ones we're going to first.
Alright. Thank you so much cool that's my question.
And our next question is from Albemarle Payrolls of Roth Capital Partners. Your line is open.
Good afternoon Rick.
And I just had one question please.
Just wanted to verify that are the clinical trials. Both golf information is correct I see that there are 42 sites or now open.
And 40 out of 42.
<unk> is actually recruiting and I think you plan to have 50 altogether, just trying to see how close to the final number you are.
Yes, I'm not sure that that the site keeps up with the total number.
But at least we have at least that there's 40 sites.
Open.
And our recruiting patients and <unk>.
As we speak where we're active.
Activating more of these sites and as I mentioned earlier I think there are some traditional high enrollment sites that are now just coming online. So we're optimistic that that's going to improve our enrollment considerably.
In the very near future.
Thank you for confirming that Rick.
Thank you Oliver.
And our next question is from Catherine Novack of Jones Research. Your line is open.
Hi, Thanks, so much for taking my question I, just wanted to ask kind of like a.
Bigger picture question about the pediatric growth hormone market.
The approval of Genesis Transcon G. H I guess, how do you see that shaping the market and especially what have you heard from kols about potential prescribing practices for our weekly.
Growth hormone you know given that this product is sometimes associated with higher than normal levels of IGF one.
Yeah.
Well you know we haven't gotten a lot of feedback we're really focused on our own programs. However, the market.
Released price where we're.
Hum.
More than pleased with you can imagine is a small molecule and a lower cost of manufacturing.
That leaves us a great deal of flexibility in pricing, especially at that level.
We've got that gets a little too early to tell what the response to the.
And the market is going to be.
From the pediatric endocrinologists, but maybe I can I could ask Dr. <unk> to talk about that a little bit more.
Yeah.
Sure.
<unk>.
Again as Rick said.
Let me say I worked very closely on.
Became Tyco club program.
It has been approved I have not even heard of yet it's been actually launched.
I would imagine it will take some time to get onto formularies, which is the real the real driver for sales because.
It's really the outpatient is it's the cost of the patient that dictates.
Sales in our markets.
And there is no question that.
The cost of goods for.
201 will be.
Much less than the cost of goods for Sky drove up which does provide quite good flexibility.
Yeah.
Yeah.
Got it.
Yeah, I think maybe what I was trying to get at is comfort in general with with Oh weekly.
Have you heard anybody who might be a little bit hesitant to prescribe weekly G. H.
Or.
Whereas IGF one levels not a concern for some kols.
Yeah.
Yeah.
I mean, David if you want to address that at all sure.
I think that most of the key opinion leaders that I talked with.
I was.
And the development of dissenters.
Did not have a big concern of ours.
<unk> one levels because traditionally.
Because kids with Muslim inefficiency or rarely diagnosed right when they develop it.
There's a lag time to diagnosis to treatment.
You don't want to target.
Kind of the average IGF one value in these kids makes you got five years to treat them to adult height. So you wanted tend to be in the upper half of the normal range and there are data showing the target of positive two you get better growth in that you targeted.
If I could when it's just a zero and by definition if you're targeting.
When it's just too you could have half about that below so I don't think that it's a big concern and there's no question that on everything else being equal.
Kids and parents, who prefer one injection of weak compared to seven injections per week, but by the same token it's not at all.
Unlikely that a lot of people would prefer a once daily oral compound to even a once weekly injection.
And Catherine I think that as pediatric endocrinologists are many of them, we'll wait and see I believe to see what the patient population of reports where their colleagues report over time I think there's going to be some early adopters, but I also think there'll be some PDR pediatric endocrinologists, who.
We will not jump in right away.
<unk> because of the big excursions of.
Of IGF one.
Levels that are sometimes up sometimes can be a.
A concern to some.
Commissions.
Okay.
Got it.
Thanks, Thanks very much.
Thank you Catherine.
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 very much.