Q1 2022 Lumos Pharma Inc Earnings Call
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[music].
Good afternoon, and welcome to aluminum farmers first quarter 2022 results and clinical update 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 would now like to turn the call over to Lisa Miller Senior director of Investor Relations. 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.
Additional information concerning factors that could cause actual results to differ is contained in our periodic reports filed with the SEC. The forward looking statements made during this call speak only as of the date hereof and the company undertakes no obligation to update or revise the forward looking statements.
Formation 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 Companys website.
Speaking on today's call will be Rick Hawkins, CEO , and chairman, John Mchugh, Our President and Chief Scientific Officer, Dr. David Karp, Our Chief Medical Officer, and Lori <unk>, Our Chief Financial Officer, 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 financial results for the 2022 first quarter and detailing our progress advancing our clinical programs for the treatment.
<unk> with idiopathic or moderate pediatric growth hormone deficiency, where pega see using our daily oral therapeutic bloom to zero one.
On today's call I'll provide a brief update on our 201 clinical trials and other developments before turning it over to John who will discuss our new collaboration with Massachusetts General Hospital for an investigator initiated phase II pilot trial.
And David will discuss an update to our clinical team and finally, Lori will review our financial results.
First I want to say that during these turbulent times, both in our industry and in the markets in general Muumuus pharma is fortunate to be in good position to advance our trials through key data milestones. Our company is on solid financial footing and continues to have sufficient cash on hand to get us.
Through not only the interim readout by the end of this year, but also the primary outcome readout of our oil growth 210 trial.
<unk> the second half of 2023, and our Oregon with 212 trial as well.
Now I am pleased to report.
2022 has begun on a positive note.
Encouraging enrollment trends, we highlighted on our last call for oil growth to turn and Ora growth to 12 trials have continued.
<unk> has now exceeded 50% randomization milestone.
Enrollment trends in both trials are sufficient for us to reiterate our commitment to conducting an interim analysis of clinical efficacy and safety data and the results of which we will announce by the end of this year.
Our interim analysis will provide an early look at the safety and annualized height velocity data at three dose levels of <unk> 201 against the standard dose of recombinant human growth hormone and 40 subjects after at least six months on therapy.
As a reminder, our <unk> trial is a global clinical study evaluating oral <unk> in approximately 80 subjects diagnosed with idiopathic or moderate Phd.
The primary clinical outcome as annualized height velocity in these subjects collected by our predictive enrichment marker or <unk> strategy.
Secondary outcome measures include comparison of annualized height velocity of boom to zero, one at three dose levels.
816, and $3 two mix per gig versus a control arm of patients treated with recombinant growth hormone daily dose of <unk> four megs per kit a week.
Dose levels of <unk>, one were selected to span the entire dose response curve elucidated in a prior PK PD study.
The goals of oil growth to 10 are to identify the optimal dose of lunar 201 to be used in our phase III registration trial as well as validate the PGM strategy.
Also remember that on our last call, we announced the initiation of our oil growth to 13 trial or the switch study.
This is an open label multi center phase II study evaluating the growth effects and safety of <unk> 201, following 12 months of daily injectable growth hormone and up to 20, <unk> patients who have completed the oil growth to 10 trial.
Subjects will be administered move to zero wanted to dose three to mix per gig a day for up to 12 months prior.
Primary outcome for the Oregon, $2 13 trial is annualized height velocity and secondary outcomes include safety and PK PD measures.
Now that we're about 212 trial is our single site open label trial evaluating the pharmacokinetic and pharmacodynamic effects of whom to zero, one and up to 24 PGA ski subjects at two dose levels, one six and $3 two mix per kg today.
The objective of our 212 trial is to confirm prior clinical data demonstrating the amplified pulsatile release of endogenous growth hormone unique to lunar 201, and the potential for this mechanism of action to increase growth on this accretion across the entire dose response curve and the majority of <unk> patients.
Primary endpoint of six months of PK, PD and high velocity data with additional 12 month data to be collected.
We continue to anticipate primary outcome data from the oral growth to 10 to 12 trials in the second half of 2023.
And we're also pleased to announce that the U S food and drug administration is now permitting treatment with world.
<unk> zero, one beyond 12 months following a review of preliminary unblinded safety and efficacy data from our oil growth trials.
As you'll recall in July 2021, we announced that the FDA had restricted treatment with lunar two zero to one to no more than 12 months and based on the agency's review of preliminary safety and efficacy data from both or growth trials, we have in progress.
<unk> is listed as partial hold and will now from a treatment with whom to zero one beyond 12 months.
As a result, we are extending the oral grow 210 trial to up to 24 months on treatment to allow subjects to continue to zero one therapy uninterrupted.
Additionally, we plan to extend or grow 212 trial.
These extensions will not change the anticipated timing of primary outcome data readout to occur after six months on treatment again primary data readout for our <unk> trial is anticipated for the second half of 2023.
In addition to the progress we.
Been making in our prospective trials, we're pleased to see the publication of a retrospective analysis by Dr. George Bright and Michael Foreigner in a journal hormone research in pediatrics.
This peer reviewed manuscript titled Loom to zero, one elicit greater growth hormone response than standard growth hormone secretagogue in pediatric growth hormone deficiency.
Presents an analysis of data from our prior clinical study comparing the peak growth hormone response of lunar 201 to that of standard diagnostic growth hormone secretagogue children previously diagnosed with growth hormone deficiency, who are naive to treatment.
The analysis demonstrates that in children with growth hormone deficiency.
Both hormone response to a single oral dose of <unk> eight mixture tigger boom to zero, one and that is our lowest dose administered in our work both trials greatly exceeds that observed standard growth hormone stimulation agents.
Also this study were originally presented at the 2021 Endo annual meeting and further support prior data, suggesting the loom to zero, one therapeutic potential when administered to pediatric subjects with idiopathic growth hormone deficiency.
I'll now turn the call over to John Mchugh, President and CFO to discuss our new collaboration with Massachusetts General Hospital John .
Thank you Rick last week, we announced a new collaboration with Dr. Laura <unk>, and Massachusetts General Hospital to explore the potential of <unk> hundred one in patients with non alcoholic fatty liver disease or an apple.
This investigator initiated phase II pilot trial is a single site six month open label study of daily oral dual <unk> hundred one in adults with <unk>.
Trial will evaluate a dose of 25 milligrams per day of <unk> hundred one in 10 adult subjects with novel, then relative IGF one deficiency.
The primary endpoints will be to determine the reduction in liver lipid content inflammation and potentially fibrosis. In these subjects administered daily oral loop 201 compared to historical placebo treated controls studied under identical procedures.
While we remain focused on our core loop to have one program in PTH D. We are pleased to support mass General's exploration of Bloom 200 one's potential in this indication the.
The condition estimated to be prevalent and approximately 25% of adults worldwide.
Apple can often advance to the to the more serious liver diseases, such as non alcoholic stay out of hepatitis Nash with fibrosis and potentially liver cancer.
<unk> associated liver failures, one of the leading causes of liver transplants in the United States.
In addition to advancing our clinical trials for <unk> hundred one in PTH D. We continue to explore expansion opportunities for limb tier one into other rare endocrine disorders, where injectable recombinant growth hormone is the standard of care.
Believe that <unk> is a pipeline in a product and through its unique mechanism of action may have the potential to treat many of the indications comprising.
The $3 4 billion dollar growth hormone market.
Indications such as <unk> Willi syndrome, idiopathic short stature children, born small for gestational age and Turner syndrome.
With that I will pass it over to David for an exciting announcement regarding an addition to our clinical team David.
Maybe you're on mute David.
Okay. Thank you John .
It is my great pleasure to announce that Doctor is it Q1 also known as Dr. Duke has just joined Louis Oklahoma as Vice President of Global clinical development and Medical Affairs.
Dr. Duke is steamed pediatric endocrinologist and noted career in academia and in both the nonprofits incorporate realms.
He is a professor of clinical Pediatrics children's hospital of Los Angeles School of Medicine of the University of Southern California, where he has worked since 1998.
Importantly, since 2011 and until very recently, Dr. Duke has also served as president of the human growth Foundation.
Where he has been instrumental in raising the organization's profile and involvement in the <unk>.
In the endocrine community and spearheading a supportive research education and patient advocacy related to growth disorders.
Dr. Duke has also served by multiple advisory and executive boards for noted pharmaceutical and rare disease companies.
He has presented numerous times at national and International medical conferences.
And appeared on local and National News platforms is an expert on pediatric endocrine disorders.
During his career. He has received numerous research brands and has authored over 70 publications.
Patrick do completed medical school and residency program at the Chiengmai University.
A residency program in pediatrics at the University of Tennessee, Memphis, and its ownership in pediatrics endocrinology and metabolism at the University of Tennessee, both in Knoxville and.
Most recently, Dr. Duke served as Vice President of Medical Affairs, and Vice President Global Medical Ambassador and medical Education Edison just pharma.
Had the pleasure of working with them on the development of Sky Trophy. The once weekly injectable for pediatric growth hormone deficiency that is FDA approved this past August .
And with pharma and I are thrilled to have him join our team as we advance our clinical programs evaluating an oral therapies loom to zero one for the same indication.
We believe Dr. Luke will be instrumental in helping the most execute our current trials.
Also help us with the advanced preparation for our phase III program.
With that I'll pass it over to Laurie for a review of our first quarter financial results Laurie.
Thanks, David and good afternoon, everyone.
Quarter ended on March 31st 2022, we ended with cash and cash equivalents totaling $86 8 million compared to $94 8 million on December 30, <unk> 2021.
Continue to expect average cash yield of approximately $8 $5 million to $95 million per quarter through the remainder of 2020 to cash on hand as of the end of our first quarter is expected to support our operations through the primary outcome data readouts from our <unk> trial anticipated in the second half of 2023.
Hi.
<unk> 212 trial.
Research and development expenses were $4 2 million for the first quarter, a decrease compared to $4 7 million for the same period in 2021.
Primarily due to a decrease of <unk> 8 million in personnel and stock compensation expense offset by an increase of $5 million in clinical trial and manufacturing expenses.
The general and administrative expenses were $3 6 million for the first quarter compared to 4 million for the same period in 2021, primarily due to a decrease of $1 5 million in legal consulting and stock compensation expenses offset by an increase of <unk> three.
$3 million in licensing and other expenses.
The net loss for the first quarter was $7 7 million compared to a net loss of $8 6 million for the same period in 2021 and with that I'll turn it back to Rick for closing remarks.
Thank you Laurie and so our first quarter was a productive one for momo's enrollment trends for our oil growth trials are positive and we will be able to announce the results of our interim analysis by the end of this year.
After a review of unblinded preliminary data the FDA now permits treatment with Luna to zero on beyond 12 months. So the extended treatment for both trials beyond that timeframe.
Primary outcome data for these phase II trials will be captured at six months on therapy.
The extension of treatment duration will allow subjects to continue uninterrupted on therapy and will create a more robust data package for regulatory and commercial purposes.
The therapeutic potential of enthusiasm one was highlighted in a recent peer reviewed publication and we have a new collaboration with mass General hospital to explore the potential of <unk> to 201, and Apple and we continued to prudently explore other opportunities to expand this platform.
And finally as you know the financial markets continue to be to be volatile and many of our biotech peers do not have sufficient funds to advance our clinical programs.
In contrast, numerous farmers cash position is strong sufficient to carry us beyond our primary outcome data readout in the second half of 2023.
So we look forward to reporting interim data by the end of this year and updating you on our continuing progress.
Operator, we're now ready to take questions.
Thank you.
The lines are now open for questions. If you would like to ask a question. Please press star one on your Touchtone telephone. If your question has been answered or you wish to remove yourself in the queue. Please press the pound key.
Please stand by while we compile the Q&A roster.
I show. Our first question comes from the line of Charles Duncan from Cantor Fitzgerald. Please go ahead.
Hi, Bobby on the line for <unk>.
Thank you for taking our questions and congrats on the enrollment progress. So I guess my first question is what what could you provide some color on the.
Safety and tolerance, tolerability learnings or the FDA that had been previously learned.
From the Merck trial, and then I have a couple of follow ups.
John That's a regulatory question. So why don't you go ahead.
Youre on mute genre, yes sure thing.
So the.
As you know this molecule has been in over a 1000 adult some of them for up to two years and up to 200 kits for.
At least six months as well and so it had a very clean safety and tolerance tolerability package already going into this trial that we're running right now and we've seen nothing to contradict that clean safety data.
I think John that.
The FDA was more concerned about.
Feed patients on drug for longer than.
12 months based on on efficacy is that right. Yes. So I think the question was mostly on safety and Tolerability is that correct, but I'm happy to answer other aspects of that question. If you have them.
Absolutely that's very helpful. Thank you.
Yes.
Couple of follow ups with regards to that whole program. So I guess could you discuss mechanistic rationale for it so look for us until one in Nashville.
Regards to the study design and our patients.
Patient is going to be a lot on concomitant medications. Thank you.
Okay go ahead John .
So there is.
There's kind of a long history first kind of just of the actions of growth hormone.
Promoting lipolysis.
The actions of growth hormone itself and secretive Alex like two to one being having immune.
Modulator effects and anti inflammatory properties and then there are there is quite a bit of clinical data already and molecules with similar mechanism mechanisms of action, So tests umbrella, which is a.
Peptide analog of growth hormone releasing hormone.
Is approved for patients with.
With HIV lipodystrophy, they published data on HIV patients with fatty liver and the impact that a daily growth hormone releasing hormone injection.
On those patients and they have recently started a larger phase III trial more broadly in the <unk> space.
There is also.
Our clinical trial that you can find in clinical trials dot Gov that was run by Dr. Laura <unk>, who has actually used growth hormone as the daily injectable in the Snapple population.
I think theres a lot of mechanistic data there is a lot of preclinical data and there is also quite a bit of clinical data to support. This all the molecules that have been used to date are daily Injectables that I think we have probably a better mechanism of action when it comes to comparing against tests umbrella.
Because we modulate boats matter stab and growth from releasing hormone and where an oral compared to those daily Injectables, which I think will have a hard time finding a foothold in this in this market.
In a market like Nashville.
Okay. Thank you and then I guess with regards to concomitant medications for patients in the study.
Yes.
Yes.
Yes, there'll be able to.
To use concomitant medications as well obviously there are some.
Hum.
There are some that.
We watch out for obviously as with any medication, but I think there is there should be a broad range of.
Absolutely.
No problem concomitant medications.
John .
We cannot take another agent to get live events, such as Tvs et cetera.
Yes, yes.
I don't know if thats the concomitant medication you were talking about were not.
These patients are on.
They are not currently being treated for fatty liver, if thats or diabetes, if thats what youre asking.
Yeah that was exactly yeah.
Yes, thank you for the color and congrats on the quarter. Thank you for taking our questions.
Thank you.
Our next question comes from the line of gasoline Rahimi from Piper Sandler. Please go ahead.
Yes, hi team. Thank you so much for taking the question.
I think that would be really helpful for us and our clients who are listening.
You could walk through.
Sure.
What range.
Height velocity do you expect to see.
Interim laid out across both of the study.
If you could also shed a little bit light onto that H.
50% of the patients that will be reported out.
That could also be really helpful for us so.
I think it would be really helpful. If you could help us understand what sort of expectation going into the data.
That would be great. Thank you.
David Once you take the clinical question sure.
Sure.
Thank you very much.
Yes so.
It's important to understand that.
About three or four decades of work with growth hormone showing that the magnitude of the response to become Nicholas Mone depends upon the severity of disease. So more severely more deficient patients respond much better growth hormone and less severe less so all the data with the population that we are steady.
Which could be broadly characterized as idiopathic ghd.
Three different sources show that in contrast to the values that you might see numerous severest patient setting.
And the Transcon bolt on program you might see.
511 centimeters.
Annualized growth.
You actually see a mean of about $8 three centimeters.
And that's with growth hormone, so thats really the target that we are expecting to see it.
The standard dose of ammonia in the population we're studying in the 210 study and we are hopeful that with the dose range that we are setting up until the one that we will find its piste when dose that looks to be comparable to that.
Okay, and I think that there is a question.
Age of the patient.
Ed.
Sure.
In answer to that question, except to say to give the range in the protocol.
We have a pretty broad range.
Glen.
10 to 11, depending upon gender.
Probably the average age I would guess would be somewhere right around.
Seven.
Okay.
And then maybe just a quick follow up.
<unk> are you planning to.
Put the two data sets across dose study combined or with the data include.
Two separate analyses as well as our combined.
Yes.
Hi.
Great.
This agreement with FCA will be presenting the data at several different ways, we'll be reporting the data separately from each trial and will also be performing a combined analysis.
By the sub debts from the 212 study will augment and add to the mid in the high dose arms in the 210 study.
Okay.
Okay. Thanks International I'll jump back into the queue.
Alright, Thanks, Jeff.
Thank you.
Thank you I show. Our next question comes from the line of Ed White from H C. Wainwright. Please go right.
Good afternoon, Thanks for taking my questions.
With regard to the.
FDA, giving you the ability to follow the patients for longer than 12 months.
I believe you opened the 212 trial.
In late June last year, but the 210 trial was opened.
You announced that on the third quarter 'twenty results call I think it was November of 2020.
So I'm just curious as to how many patients you have that are bumping up against that 12 months.
Timeframe.
And.
Where some patients.
<unk> to 12 months.
Can you address them now by putting them back on drug.
David do you want to answer that.
Sure.
As it turns out.
Initial patients randomized into the trial randomized to the combatant arm. So they are being covered by the $2 13 study.
We have not lost any patients who would be 12 months to date and we are anticipating that we will be able to capture all of them.
Yeah.
In the revised 24 months vertical.
Okay, great. Thank you.
And.
Just a question on.
You have this investigator sponsored trial now.
And another indication I was just wondering if and when you are planning on entering.
Another indication of company sponsored.
Trial.
And what your thoughts are as we've talked about it in the past you have many different opportunities. So I'm just wondering if you would have narrowed the field yet.
Yes.
John Why don't you go ahead.
So Ed we are we.
We are still working to continue to narrow down to one indication that we felt quite comfortable with the two announces our next indication for not in a place yet where it made that decision, but where we are getting close.
Okay. Thanks, Scott those are all the questions I had.
Okay.
Thank you I show. Our next question comes from the line of Catherine Novak from Johnson Research. Please go ahead.
Hi, Thanks, so much for taking my question.
I'm kind of curious you talked a little bit about <unk>.
Internal.
Scotland efficacy or.
Hi, Ken.
And.
Given that the study is open label.
I anticipate the interim data in <unk> patients.
Okay.
Moving into pivotal studies or can you talk about sort of wanted to see with the drug before making that decision.
David is a clinical question. So go ahead.
Sure.
So.
It's a little bit premature to know until we execute the data it is entirely possible based upon the sample size that.
At the interim look at the end of this year will be sufficient for us to narrow down the optimal dose.
<unk> said that initiation of phase III depends on a number of factors.
Including.
<unk> efficacy and safety from Fe from Phase II, as well as having adequate drug product availability.
So I think that.
Or if you're looking at this interim results and if the data are robust that would allow us to choose.
She was an optimal dose at an earlier time point and the full readouts.
Hi, debate, III, which could jumpstart a lot of the planning for phase III. This is contacting some regulatory agency that have much longer lead time for example, the FDA does so I think it could be very very helpful. If the data are positive robust.
But it may not necessarily in band the initiation of phase III per se, but it could certainly jumpstart a lot of it to frontload a lot of the activities.
Definitely.
While.
Okay.
That's kind of helpful to know.
And then I guess on that same vein could you talk a little bit about the enrollment dynamics that you've been seen an issue.
Seen a reversal of some of the headwinds.
We had.
In the fall and winter months.
Yes.
It is.
I wouldn't recommend any company start.
<unk> site global trial in middle of a pandemic.
But we responded really well and I think thats because of the.
Experience and quality of our clinical development team.
But I also think that there is.
A number of investigators.
Out there not only with who have a great deal of experience but.
And working with the recombinant growth hormone, but this is the first chance that they had to work with.
Once a day oral product. So I think they are pretty sound excited for themselves and for their patient population.
So theres a lot of interest there is no question about it.
We're cautiously optimistic about where we're headed next.
I think we announced in the past that.
Last a number of sites.
Russia, and Ukraine, although we've added additional sites beyond that plus we're coming online in other parts of the world.
That haven't entered any patient so yet, but now are fully cleared and.
Screening patients so cautiously optimistic going forward.
Okay.
Okay.
Great alright, thanks, so much for answering my questions.
Sure.
Thank you Catherine.
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.
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Good afternoon, and welcome to Lou most farmers the first quarter of 2022 results and clinical update 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 would now like to turn the call over to Lisa Miller Senior director of Investor Relations. 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.
Additional information concerning factors that could cause actual results to differ is contained in our periodic reports filed with the SEC.
The forward looking statements made during this call speak only as of the date hereof and the company undertakes no obligation to update or revise the forward looking statements information presented on this call is contained in the press release, we issued this afternoon and in our form 8-K, which may be accessed from the investors page of the company's website.
On today's call will be Rick Hawkins, CEO , and chairman, John Mchugh, Our President and Chief Scientific Officer, Dr. David Karp, Our Chief Medical Officer, and Lori <unk>, Our Chief Financial Officer, 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.
Now after the market closed today, we issued a press release announcing our financial results for the 2022 first quarter and detailing our progress advancing our clinical programs for the treatment of patients with idiopathic or moderate pediatric growth hormone deficiency or <unk> using our daily oral therapeutic.
<unk> 201.
On today's call I'll provide a brief update on our 201 clinical trials and other developments before turning it over to John who will discuss our new collaboration with Massachusetts General Hospital for an investigator initiated phase II pilot trial.
And David will discuss an update to our clinical team and finally, Lori will review our financial results.
First I wanted to say that during these turbulent times, both in our industry and in the markets in general numerous pharma is fortunate to be in good position to advance our trials through key data milestones. Our company is on solid financial footing and continues to have sufficient cash on hand to get us through.
Not only in the interim readout by the end of this year, but also the primary outcome readout of our oil growth to 10 trial expected in the second half of 2023, and our Oregon at 212 trial as well.
Now I am pleased to report that 2022 has begun on a positive note.
Encouraging encouraging enrollment trends, we highlighted on our last call for oil growth to turn in oil growth to 12 trials have continued.
And the <unk> 210 trial has now exceeded 50% randomization milestone.
Enrollment trends in both trials are sufficient for us to reiterate our commitment to conducting an interim analysis of clinical efficacy and safety data and the results of which we will announce by the end of this year.
Our interim analysis will provide an early look at the safety and annualized height velocity data at three dose levels of 1201 against the standard dose of recombinant human growth hormone and 40 subjects after at least six months on therapy.
As a reminder, for all go to 210 trial as our global clinical study evaluating oral <unk> 201, and approximately 80 subjects diagnosed with idiopathic moderate Phd.
Primary clinical outcome as annualized height velocity in these subjects collected by our predictive enrichment marker or <unk> strategy.
Secondary outcome measures include comparison of annualized height velocity of boom to zero, one at three dose levels.
816, and $3 two to make strategic versus a control arm of patients treated with recombinant growth hormone daily dose of <unk> four make strategic a week.
Dose levels alumina is one were selected to span the entire dose response curve elucidated in a prior PK PD study that.
The goals of oil growth to 10 are to identify the optimal dose alone 201 to be used in our phase III registration trial as well as validate the PGM strategy.
You'll also remember that on our last call, we announced the initiation of our oil growth to 13 trial or the switch study.
This is an open label multi center phase II study evaluating the growth effects and the safety of <unk> 201, following 12 months of daily injectable growth hormone and up to 20, <unk> patients who have completed the oil growth to 10 trial.
Subjects will be administered move to zero and wanted to dose three to mix per kg a day for up to 12 months.
I'm Mary outcome for the oral route 213 trial is annualized height velocity and secondary outcomes include safety and PK PD measures.
Now the order of 212 trial is our single site open label trial evaluating the pharmacokinetic and pharmacodynamic effects of whom to zero, one and up to 24 Phd subjects at two dose levels, one six and $3 two makes perfect. Good day.
The objective of our 212 trial is to confirm prior clinical data demonstrating the amplified pulsatile release of endogenous growth hormone.
<unk> 201, and the potential for this mechanism of action to increase growth how much accretion across the entire dose response curve and the majority of PTSD patients pre.
Primary endpoint of six months of PK, PD and high velocity data with additional 12 months.
Data to be collected.
We continue to anticipate primary outcome data from the oral growth to 10 to 12 trials in the second half of 2023.
And we're also pleased to announce that the U S food and drug administration is now permitting treatment with world.
201 beyond 12 months following a review of preliminary unblinded safety and efficacy data from our oil growth trials.
As you'll recall in July 2021, we announced that the FDA had restricted treatment with lunar two zero to one to no more than 12 months and based on the agency's review of preliminary safety and efficacy data from both oil growth trials, we have in progress.
<unk> is listed as partial hold and will now from a treatment with Luna to zero, one beyond 12 months.
As a result, we are extending the oral grow 210 trial to up to 24 months on treatment to allow subjects to continue to zero one therapy uninterrupted.
Additionally, we plan to extend or grow 212 trial.
These extensions will not change the anticipated timing of primary outcome data readout to occur after six months on treatment again primary data readout for our <unk> trial is anticipated for the second half of 2023.
In addition to the progress.
Been making in our prospective trials, we're pleased to see the publication of a retrospective analysis by Dr. George Bright and micro foreigner in a journal hormone research in Pediatrics. This peer reviewed manuscript titled Loom to zero, one elicit greater growth hormone response than standard growth hormone secretagogue.
In pediatric growth hormone deficiency.
Presents an analysis of data from our prior clinical study comparing the peak growth hormone response of loan to zero one to that of standard diagnostic growth hormone Secretagogue and children previously diagnosed with growth hormone deficiency, who are naive to treatment.
The analysis demonstrates that in children with growth hormone deficiency or <unk>.
Growth hormone response to a single oral dose of <unk> eight mixture tigger boom to zero, one and that is our lowest dose administered in our workload trials greatly exceeds that observed with standard growth hormone stimulation agents.
Results of this study were originally presented at the 2021 Endo annual meeting and further support prior data, suggesting that loom to zero, one therapeutic potential when administered to pediatric subjects with idiopathic growth hormone deficiency.
I'll now turn the call over to John Mchugh, President and CFO to discuss our new collaboration with Massachusetts General Hospital John .
Thank you Rick last week, we announced a new collaboration with Dr. Laura <unk>, and Massachusetts General Hospital to explore the potential of <unk> hundred one in patients with non alcoholic fatty liver disease or <unk>.
This investigator initiated phase II pilot trial is a single site six month open label study of daily oral dose <unk> in adults with <unk>.
The trial will evaluate a dose of 25 milligrams per day of linked to a one in 10 adult subjects with novel then relative IGF one deficiency.
The primary endpoints will be to determine the reduction in liver lipid content inflammation and potentially fibrosis. In these subjects administered daily oral loop 201 compared to historical placebo treated controls studied under identical procedures.
While we remain focused on our core loop to have one program in PTH D. We are pleased to support mass General's exploration of Bloom 200 one's potential in this indication.
The condition estimated to be prevalent and approximately 25% of adults worldwide.
Now I will turn off in advance to the to the more serious liver diseases, such as non alcoholic stay out of hepatitis Nash with fibrosis and potentially liver cancer.
Also NASA associated liver failure is one of the leading causes of liver transplants in the United States.
In addition to advancing our clinical trials for <unk> hundred one and PGA HD we can.
Continue to explore expansion opportunities for Alimta, one into other rare endocrine disorders, where injectable recombinant growth hormone is the standard of care. We believe that <unk> is a pipeline in a product and through its unique mechanism of action may have the potential to treat many of the indications comprising.
The $3 4 billion dollar growth hormone market.
Indications such as <unk> Willi syndrome, idiopathic short stature children, born small for gestational age and Turner syndrome.
With that I will pass it over to David for an exciting announcement regarding an addition to our clinical team David.
Maybe you're on mute David.
Yeah. Thank you John .
It is my great pleasure to announce the Doctor is it Q1 also known as Dr. Duke has just joined Loomis pharma as Vice President of Global clinical development and medical Affairs.
That could Duke isn't a sting TV, Patrick Endocrinologist noted career in academia and in both the nonprofit incorporate realms.
He is a brook that sort of clinical pediatrics children's hospital of Los Angeles School of Medicine of the University of Southern California.
Worst since 1998.
Importantly, since 2011 until very recently, Dr. Duke has also served as president of the human growth Foundation.
Where he has been instrumental in raising the organization's profile and involvement in the <unk>.
In the endocrine community and spearheading a supportive research education and patient advocacy related to growth disorders.
Dr. Duke has also served multiple advisory and executive boards for noted pharmaceutical and rare disease companies.
He has presented numerous times at national and International medical conferences.
Appeared on local and National news platforms is an expert on pediatric endocrine disorders.
During his career. He has received numerous research brands and has authored over 70 publications.
Dr Do completed medical school and residency program at the Chiengmai University.
A residency program in pediatrics at the University of Tennessee, Memphis, and its ownership in pediatrics endocrinology and metabolism at the University of Tennessee.
Accel and.
Most recently, Dr. Duke served as Vice President of Medical Affairs, and Vice President Global Medical Embassador and medical education at ascend is pharma.
I had the pleasure of working with them on the development of Sky Trophy. The once weekly injectable for pediatric growth hormone deficiency to this FDA approved this past August .
And with pharma and I are thrilled to have him join our team as we advance our clinical programs.
<unk> and oral therapies loom to zero one for the same indication.
We believe Dr. Luke will be instrumental in helping the most execute our current trials.
It also help us with the advanced preparation for our phase III program.
With that I'll pass it over to Laurie for a review of our first quarter financial results Laurie.
Thanks, David and good afternoon, everyone for the quarter ended on March 31st 2022, we ended with cash and cash equivalents totaling $86 $8 million compared to $94 8 million on December 30, <unk> 2021.
We continue to expect average cash yields of approximately $8 $5 million to $95 million per quarter through the remainder of 2020 to cash on hand as at the end of our first quarter is expected to support our operations as a primary outcome data readouts from our <unk> trial anticipated in the second half of 2002.
23 and <unk>.
Trial <unk>.
<unk> and development expenses were $4 2 million for the first quarter, a decrease compared to $4 7 million for the same period in 2021.
Primarily due to a decrease of <unk> $8 million in personnel and stock compensation expense offset by an increase of $5 million in clinical trial and manufacturing expenses.
The general and administrative expenses were $3 $6 million for the first quarter compared to 4 million for the same period in 2021, primarily due to a decrease of $1 5 million in legal consulting and stock compensation expenses offset by an increase of one point.
$3 million in licensing and other expenses.
The net loss for the first quarter was $7 7 million compared to a net loss of $8 6 million for the same period in 2021 and with that I'll turn it back to Rick for closing remarks.
Thank you Laurie and so our first quarter was a productive one for numerous enrollment trends for our oral drug trials are positive and we will be able to announce the results of our interim analysis by the end of this year.
And Thats a review of unblinded preliminary data the FDA now permits treatment with Luna to zero on beyond 12 months. So the extended treatment for both trials beyond that timeframe.
Primary outcome data for these phase II trials will be captured at six months on therapy.
The extension of treatment duration will allow subjects to continue uninterrupted on therapy and will create a more robust data package for regulatory and commercial purposes.
The therapeutic potential of linked to zero one was highlighted in a recent peer reviewed publication and we have a new collaboration with mass General hospital to explore the potential of lunar two.
201, and Apple and we continue to prudently explore other opportunities to expand this platform.
And finally as you know the financial markets continued to be volatile.
And many of our biotech peers do not have sufficient funds to advance our clinical programs.
In contrast, numerous farmers cash position is strong sufficient to carry us beyond our primary outcome data readout in the second half of 2023.
Look forward to reporting interim data by the end of this year and updating you on our continuing progress.
Operator, we're now ready to take questions.
Thank you.
Is it now open for questions. If you would like to ask a question. Please press star one on your Touchtone telephone. If your question has been answered or your wisdom of yourself in the queue. Please press the pound key.
These standby, while we compile the Q&A roster.
I show. Our first question comes from the line of Charles Duncan from Cantor Fitzgerald. Please go ahead.
Hi, Bobby on the line first of all thank you for taking our questions and congrats on the enrollment and Thats why it progress. So I guess my first question is what what could you provide some color on the.
Safety and tolerance and Tolerability learnings or the FDA that had been previously.
From the Merck trials, and then I have a couple of follow ups.
John That's a regulatory question. So why don't you go ahead.
Youre on mute genre, yes sure thing.
So the.
As you know this this molecule has been in over 1000 adults some of them for up to two years and up to 200 kits for.
At least six months as well and so it had a very clean safety and tolerance tolerability package already going into this trial that we're running right now and we've seen nothing to contradict that clean safety data.
I think John that.
The FDA was more concerned about.
Keep patients on drug for longer than.
12 months based on an efficacy is that right. Yes. So so I think the question was mostly on safety and Tolerability is that correct, but im happy to answer other aspects of that question. If you have them.
Absolutely that's very helpful. Thank you.
And then just had a.
Couple of follow up so with regards to the Napa program. So I guess could you discuss the mechanistic rationale for.
He is until one in Nashville.
With regards to the study design and our patients.
Are patients going to be.
Prominent medication. Thank you.
Okay go ahead John .
So there is a.
There's there's kind of a long history first kind of just of the actions of growth hormone be promoting lipolysis.
The actions of growth hormone itself.
So cretic, Alex like two to one being having immune.
Modulator effects and anti inflammatory properties and then there are there is quite a bit of clinical data already and molecules with similar mechanism mechanisms of action, So tests umbrella, which is a.
Peptide analog of growth hormone releasing hormone.
Is approved for patients with.
With HIV lipodystrophy, they've published data on HIV.
<unk> with fatty liver and the impact that a daily growth hormone releasing hormone injection.
On those patients and they have recently started a larger phase III trial more broadly in the <unk> space.
There is also.
Our clinical trial that you can find in clinical trials dot Gov that was run by Dr. Laura <unk>, who has actually used growth hormone is a daily injectable and the snapple population. So I think theres a lot of mechanistic data. There's a lot of preclinical data and there is also quite a bit of clinical data to support.
This all the molecules that have been used to date are daily Injectables that I think we have a probably a better mechanism of action when it comes to comparing against Tessa Marella, because we modulate both somatostatin and growth from releasing hormone.
And we're an oral compared to those daily Injectables, which I think will have a hard time, finding a foothold in this in this market.
In a market like Nashville.
Okay. Thank you and then I guess with regards to concomitant medication to all patients in the study.
Yes.
Yes.
Yes, yes, there'll be able to.
To use concomitant medications as well obviously there are some.
There are some that.
We watch out for obviously with any medication, but I think there is there.
It would be a broad range of.
Absolutely.
No problem concomitant medications.
John .
We cannot take another agent to just live with that.
Tvd's et cetera.
Yes, yes, I mean, that's the whole I don't know if thats. The concomitant medication you were talking about were not.
These patients are on.
They are not currently being treated for fatty liver, if that's or diabetes, if thats what youre asking.
Okay.
Yeah that was exactly yeah yeah.
Yes, thank you for the color and congrats on the quarter. Thank you for taking our questions.
Thank you.
Our next question comes from the line of Jasmine Rahimi from Piper Sandler. Please go ahead.
Yes, hi team. Thank you so much for taking the questions.
I think it would be really helpful for us and our clients who are listening in.
You could walk us through.
What range.
Height velocity do you expect to see.
Interim laid out across both of the study.
If you could also shed a little bit light onto that H.
The 50% of the patients that will be reported out.
That could also be really helpful for us so.
I think it would be really helpful. If you could help us understand what sort of expectation going into the data.
That would be great. Thank you.
David why don't you take that clinical question.
Sure.
Thank you very much.
Yes so.
It's important to understand that.
About three or four decades work with political mode, showing that the magnitude of the response to becoming a growth mode. It depends upon the severity of the disease. So more severely more deficient patients respond much better growth hormone and less severe less so all the data with the population that we are steady.
Which could be broadly characterized as idiopathic ghd.
Three different sources show that in contrast to the values that you might see numerous severest patient setting.
Transcon bolt on program, we might see 10 511 centimeters.
Annualized growth.
You actually see a mean of about $8 three centimeters.
And that's with growth hormone, so thats really the target that we are expecting to see.
The standard goes through red promoting the population we're studying in the 210 study and we are hopeful that with the dose range that we are setting up until one that we will find at least one dose that looks to be comparable to that.
Okay, and I think that there is a question.
Age of the patient.
Okay.
Sure.
In answer to that question, except to say to give the range in the protocol.
We have a pretty broad range.
Glen.
211, depending upon gender.
Probably the average age I would guess would be somewhere right around.
Seven.
Okay.
And then maybe just a quick follow up and in that regard are you planning to.
Put the two data sets across both studies combined or with the data include.
Two separate analysis as well as our combined.
Yes.
Hi.
Okay.
This agreement with FCA will be presenting the data at several different ways, we'll be reporting the data separately from each trial and will also be performing a combined analysis.
By the subjects from the 212 study will augment and add to the made in the high dose arms in the 210 study.
Okay.
Okay. Thank international I'll jump back into the queue.
Alright, thanks, guys.
Thank you.
Thank you I show. Our next question comes from the line of Ed White from H C. Wainwright. Please go right.
Good afternoon, and thanks for taking my questions.
With regard to the.
FDA, giving you the ability to follow the patients for longer than 12 months.
I believe you opened the 212 trial.
In late June last year, but the 210 trial was opened.
You announced that on the third quarter 'twenty results call I think it was November of 2020.
So I'm just curious as to how many patients you have that are bumping up against that 12 months.
Timeframe.
And.
Where some patients.
The 12 months and can you address them now by putting them back on drug.
David do you want to answer that.
Sure.
As it turns out.
Initial patients randomized into the trial randomized to recombinant arm. So they are being covered by the <unk> study.
We have not lost any patients who would be 12 months to date and we are anticipating that we will be able to capture all of them got it.
In the revised 24 months vertical.
Okay.
Okay, great. Thank you.
<unk>.
Just a question on.
You have this is an investigator sponsored trial now.
And another indication that I was just wondering if and when you are planning on entering.
Another indication of our company sponsored.
Trial.
And what your thoughts are as we've talked about it in the past you have many different opportunities. So I'm just wondering if you would have narrowed the field yet.
Yeah.
John Why don't you go ahead.
So Ed we are we are still working to continue to narrow down to one indication that we feel quite comfortable with the two analysis. Our next indication we're not in a place yet where it made that decision, but where we are getting close.
Okay. Thanks, Scott those are all the questions I had.
Okay.
Thank you.
Our next question comes from the line of Catherine Novack from Johnson Research. Please go ahead.
Hi, Thanks, so much for taking my question.
I'm kind of curious you talked a little bit about your potential internal.
Scotland ethical fee or.
For the higher dose 10.
And.
Given that the study is open label.
I anticipate the interim data in <unk> patients.
Okay.
Going into pivotal studies or can you talk about sort of wanted to see with the drug before making that decision.
David is a clinical question. So go ahead.
Sure.
So.
Yes.
So a little bit premature to know until we execute the data it is entirely possible based upon the sample size that.
At the interim look at the end of this year will be sufficient for us to narrow down the alcohol dose, having said that the initiation of phase III depends on a number of factors.
<unk>.
Efficacy and safety from Fe from Phase II, as well as having adequate drug product availability.
So I think that.
Or if you're looking at this interim results and.
The data are robust that would allow us to choose.
Choose an optimal dose an earlier time point than the full readouts.
Hi.
Hi.
Could jumpstart a lot of the planning for phase III. This is contacting some regulatory agency that have much longer lead time for example, the FDA does so I think it could be very very helpful. If the data are positive robust.
It may not necessarily advance the initiation of phase III per se, but it could certainly jumpstart a lot of it is correct load a lot of the activities.
For <unk>.
II trial.
Okay.
That's kind of helpful to know.
And then I guess on that same vein could you talk a little bit about the enrollment dynamics that you've been seeing and if you've.
<unk> seen a reversal of some of the headwinds.
We had.
In the fall and winter months.
Yes.
It is.
Wouldn't recommend any company start.
<unk> site global trial in the middle of a pandemic.
But we responded really well and I think thats because of the.
Experience and quality of our clinical development team.
<unk>.
But I also think that there is.
A number of investigators.
Out there not only with a great deal of.
Experience, but.
And working with the recombinant growth hormone, but this is the first chance that they had to work with.
Once a day oral product. So I think they are pretty sound excited for themselves and for their patient population.
And so theres a lot of interest there is no question about it.
We're cautiously optimistic about where we're headed next.
I think we announced in the past that we love.
The number of sites.
Russia and Ukraine.
Though we've added additional sites beyond that plus we're coming online in other parts of the world.
That haven't entered any patient so yet, but now are fully cleared and.
Screening patients.
So cautiously optimistic going forward.
Okay.
That sounds great alright, thanks, so much for answering my questions.
Sure.
Thank you Catherine.
Thank you I'm showing no further questions in the queue at this time, thank you for joining us and enjoy your afternoon.