Q1 2021 Lumos Pharma Inc Earnings Call
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Good afternoon, ladies and gentlemen, and welcome to the mouse pharma.
Pharma is pretty is quite a tiny tiny one financial results conference call.
At this time all the participants are in a listen only mode and later, we will conduct a question and answer session and instructions will follow at that time.
As a reminder of this conference call is being recorded.
I will now turn the call over two of the sound Neely Senior director of Investor Relations.
Thank you 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 edition.
Information concerning factors that could cause actual results to differ is can say contained in our periodic reports filed with the SEC the for them.
We're looking statements made during this call speak only as of the date hereof and the company undertakes no obligation to update or revise these forward looking statements <unk>.
Information presented on this call is contained and the earnings release, we issued this afternoon and in our form 8-K, which may be accessed from the Investor Relations page of the company's website.
Joining me on today's call of Rick Hawkins, CEO, President and Chairman, John Mchugh, Chief Operating Officer, and Chief Scientific Officer, Karl language, and Chief Financial Officer, and Lori Lolli, Senior Vice President Finance and corporate controller, Rick Hawkins will provide the of corporate update John Mccabe will review the company's lead therapeutic candidate.
And our clinical trials and Lori Lolly will wrap up the call with a review of the first quarter 2021 financial results and an update of the cash guidance. Following our prepared remarks, we will open the call to questions I will now turn the call over to Rick.
Thank you Lisa and good afternoon, and thank you for joining us on today's call.
After the market closed today, we issued a press release detailing our first quarter 2021 financial results and highlighting our recent clinical and corporate activity.
I'm pleased to report that the quarter was the productive one during which we saw the presentation and publication of new data and analysis of supporting the differentiated mechanism of action of our novel oral therapeutic candidate <unk> two zero of one for the treatment of pediatric growth hormone deficiency and patients identified by <unk>.
Our predictive enrichment markers.
We also continue to advance our clinical programs for <unk> 201, adding clinical sites for our phase two the oil growth to 10 trial and moving towards the initiation of our ore of growth to 12 trials during the second quarter.
Finally, we completed the monetization of our priority review voucher, receiving and January the final of $26 million tranche of the $60 million due to.
Two the company from the sale.
With these resources on hand, we believe we are well positioned to advance our lunar 201 programs and explore business development options to enhance our pipeline and deliver value to our investors.
And before turning the call over to John the queue for a deeper dive on progress and our clinical programs I just want to highlight a few recent events.
Last week, we held a key opinion leader of Forum featuring presentations by Dr. Bradley Miller of the University of Minnesota, and Fernando Cazorla of the University of Chile.
This was a well attended and informative event, where Dr. Miller and Dr. Cazorla provided an overview of the current treatment landscape and P GAC and outlined the unmet medical needs and this space.
As part of his presentation. Dr. Christoph also presented newly released the least PK PD data from a subgroup of the Merck <unk> trial, evaluating <unk> 201, and naive to treatment <unk> patients.
These data were additive to the peer reviewed data recently published in the journal of Endocrine Society.
Demonstrating once again the potential of our predictive enrichment markers for Pms of Bay.
The swine IGF, one levels and peak stimulated growth hormone levels. After a single dose of the 201 to identify patients likely to respond to lunar two zero on therapy.
Additional data were presented last month at the Endocrine Society 2021 annual meeting known as Endo further demonstrating the loom to zero once unique potential to elicit therapeutic level response and PTH the patients.
And then the differentiating this molecule from standard growth hormone secretagogue the.
These results add to the previously.
You mentioned data further supporting the approach we are using and our clinical trials.
Joe Mchugh of our COO and Chief Scientific Officer will have more to say about these data and a moment.
So as I mentioned, our phase <unk> trial and P. J C continues to enroll patients.
Additional clinical sites of opened for enrollment with over 50% of our target number of sites now activated.
Our oil growth to flow trial of PK PD study and <unk> is expected to be initiated this quarter with data from net trial anticipated to confirm prior data demonstrating the unique pulsatile MLA of lunar 201.
We believe <unk> 201 has the potential to disrupt the injectable therapeutic of market for PGA state.
We also believe the <unk> is essentially a pipeline and a product with the potential to target up to 10, other indications for which growth hormone has been approved.
We're focusing first on PGA ste and once the gathered data from our current program, we plan to evaluate moving to zero.
And a subset of these other indications.
We are also pursuing business development opportunities to add other rare disease assets to expand our portfolio.
And we're excited about the progress, we're making and our.
For our clinical programs and with our solid balance sheet strengthened by the receipt of the final tranche of the $26 million from our <unk> sales.
The position to advance our corporate strategy.
So with that I'm going to turn the call over to John to review of our or growth hormone Secretagogue link to zero, one and recently published data John.
And.
Thank you Rick and good afternoon, everyone as Rick mentioned, we had some very compelling data are presented and published in recent weeks some of which we highlighted on our last call, but are worth touching on again before getting into the details I just want to remind everyone of the key differentiating factor and the link to one mechanism of action.
As you may recall of growth hormone deficiency can be defined by low to nearly absent secretion of growth on one from the pituitary gland current therapies and those and development consists of the delivery by injection of a bolus of growth hormone or long acting derivative respectively to restore growth.
Link to a one on the other hand is not of growth hormone, but is instead of growth hormone secretagogue that ex within the body's natural endocrine pathways to stimulate the body's ability to release growth hormone at the same intervals and subject to the same and a good feedback loops that occurred naturally.
Utilizing this endogenous release mechanism should enable the naturally occurring IGF one feedback loop.
To be preserved to help regulate the balance of growth hormone and IGF, one levels and the body.
Given this endogenous nature of lunar two of one to benefit from moving to a one therapy individuals' must have a functioning though diminished.
<unk> access for this reason our approach is the target the moderate PTH D population of patients who are able to produce some growth from her and naturally, but not and sufficient amounts to attain normal adult height.
We believe these patients can be identified using our predictive enrichment markers for pbms to select the moderate PTH the patient group likely to respond to link to on therapy.
Our analysis of the large Genesis dataset published and the journal of Endocrine side Endocrine Society suggests that approximately 60 per cent of the total P. Ghd population would fall into this moderate category.
The new data highlighted by Doctor Cazorla during our key opinion leader event last week supported the thesis demonstrating that the the specific PFS of of baseline IGF, one level of greater than 30, nanograms per mil and of peak growth on the level of greater than or equal to five nanograms per mil. After a single dose of <unk> hundred one identify.
Of this modern PTH D patient population and likely to benefit from lunar two of one.
And its kols event presentation, Dr. Chris Orla discussed newly released data from Merck's O to O study as part of his overview of Alimta of one's mechanism of action. The subgroup of the Merck O to O trials, specifically exam and the effect of link to a one had.
On the pulsatile secretion of growth hormone over 24 hours and patients with ph D. After six months of treatment with the two of one compare to each patient's baseline GH secretion.
As has been previously observed in adults link to one increase the pulsatile release of growth hormone for 24 hours and two P M positive patients and.
Importantly, following six months of of treatment with link to one area under the curve analysis showed increases of growth hormone of less than two fold and these PM positive patients that resulted in substantial increases in height velocity.
And as expected the one P. M negative patients showed no increase and growth hormone AUC over the 24 hour monitoring period of no increases of height velocity. After six months of treatment with <unk> hundred one further supporting the ability of the selected predictive enrichment markers to identify both of those likely and those unlikely to respond to loom tool.
The one.
We believe these data are important as they support preclinical data, which showed that pulsatile delivery of growth on real produces greater efficacy and continuous exposure to the same amount of growth hormone.
Data presented at Endo and March distinguished Loom to of one from standard growth hormone Secretagogue ex a poster entitled <unk> 201 elicit greater.
Growth hormone responsive and standard growth hormone secretagogue and pediatric growth hormone deficiency supports other data, suggesting that link to one is unique and its ability of among the secretagogue is traditionally used to diagnose growth from a deficiency.
And the poster presented an analysis of data from our prior clinical study comparing the peak growth hormone responsive loop two of one to that of standard growth of them, it's accretive Alex and children naive to treatment and previously diagnosed with growth from the deficiency. The objective of the analysis with the determined whether lube tier one stimulates growth hormone response uniquely compared to standard.
Both of them, it's accretive dogs.
The analysis demonstrated that in children with growth on the deficiency of the growth of and response to a single oral dose of <unk> hundred one greatly exceeds that of observed with standard growth hormone stimulation agents, the difference and growth of them and responses increases with higher baseline concentrations of IGF, one and higher growth hormone stimulation test results as identified by our per.
Predictive enrichment markers.
The synergistic actions of link to one of the physiological pathways regulating growth hormone release explained by growth on one responses are greater and response to <unk> compared to traditional tests used to diagnose Phd and indicate that the greatest difference may be found and children with more moderate degrees of growth from a deficiency.
These are for these results further support data analyses recently published in the journal of the Endocrine Society, demonstrating that link to one has the potential to elicit a therapeutic response and pediatric patients with moderate growth from a deficiency or approximately 60 per cent of the total P. J H D population as identified by the specific <unk>.
And gives us greater confidence and the potential efficacy of link to a one and this patient population.
Collectively these data support the importance of Tem selection and the mechanism of action of link to <unk> to identify patients likely to respond to link 201, given it sort of delivery and its mechanism of action that depends on the natural H P. G. H axis, we believe that link to one may offer of preferred treatment option for them.
Approximately 60 per cent of children suffering from growth hormone deficiency.
We believe these data strongly support our clinical development strategy and ph D and illustrate why these are the <unk>, we are using and our phase two b or growth to 10 trial. This trial is the global multi site trial involving approximately 80 P. Ghd patients randomized into one of three dose levels of lunar two of one or a comparator.
The arm of standard of care daily injectable growth hormone therapy.
Only those P ghd patients determined to be P. M positive as evaluated by the predictive enrichment markers I described earlier will be randomized and the study during this phase <unk> study of the repeatability of the P. M classification will be evaluated during screening for randomization.
<unk> will be administered over six months with annualized height velocity as the key clinical outcome measure the main objectives for the phase <unk> study are to prospectively confirm the utility of our predictive enrichment marker strategy and selecting likely lunar two of one responders to assure that the PM classification, as consistent and repeatable and determined.
The optimal dose for phase III registration trial.
The three dose levels of 0.816 and $3 two mix per kg of lunar 201 were chosen based on supporting data from the Merck study and PTH. The patient mentioned earlier as well as of prior PK PD study of <unk> hundred one and healthy adults of Poe.
The stock analysis of the Merck study and P. G. H T showed no statistical difference and average height velocity for P. M positive patients dosed with zero point of makes per kick loomed, two of one versus versus those dose with standard of care for <unk>.
<unk> human growth hormone the PK PD study in healthy adult showed the zero point of big kick pediatric of equivalent dose is.
Only approximately one third of the way up the Pharmacodynamic growth hormone dose response curve.
These data showed that administering increasing doses of link to one and healthy adults up to a 100 migs.
The equivalent of two point of it makes for King and children.
The result, and higher plasma concentrations of growth hormone the <unk>.
Three doses chosen for our phase two b trial cover that full pharmacodynamic range and suggest the potential for greater growth from its accretion and potential efficacy and the ph D patients and our study.
The trial opened to enrollment last quarter and we currently have over 50% of the sites activated with additional sites expected to open soon as we progress toward our goal of 40 to 50 trial sites. In total. These sites were selected based on their prior history of enrolling P. T. H D patients on clinical trials, which should enhance the enrollment process.
And increase our confidence and our anticipated mid 2022 data readout.
We expect to initiate a second concurrent trial of Bloom to of one for patients with ph D. Shortly the order growth to 12 trial is intended to further illustrate the mechanism of action of loop to of one and amplifying the natural pulsatile secretion of growth hormone.
The order growth to 12 trial will focus on Pharmacodynamic endpoints at two different doses, one six and $3 two mixed per kit and approximately 24 children with P. Ghd.
The purpose of the study is to replicate and a larger cohort of P. Ghd patients the pulsatile the data in adults and and the small subset of children for the Merck O to O trial were viewed by our K O L.
We plan to conduct this trial at a single specialized pediatric center with the ability to perform the more frequent sample collection and monitoring required for these types of clinical trials.
The study will assess growth on rent levels over 24 hours at baseline and after six months on therapy to confirm linked to of one's amplification of pulsatile accretion.
And once initiated the study will run in parallel with the phase two b or growth to 10 trial with the goal of providing support of data and future regulatory filings and ultimately and any commercial marketing efforts.
On our last call, we reported that of fire occurred at the sand Borgia ARIA or on the hospital and Santiago, Chile. The planned clinical site for the oil growth to 12 trial, our investigators clinic was not directly involved and the fire and.
And access to the hospital has been restored and we anticipate initiating the order growth to 12 trial and the current quarter.
As we have previously stated this trial is not on the critical path for regulatory approval of them two of one and we do not believe the brief interruption caused by fire will delay our timelines.
Finally, as Rick mentioned, we believe that the them to of one may serve as a platform therapy potentially applicable to other indications for which we're calling on human growth hormone and its approved pending results from our concurrent or growth trials. Just discussed we plan to evaluate link to one and these other indications.
The link to a one and we continue to pursue business development opportunities to license or acquire other rare disease assets in order to expand our pipeline and our ability to provide innovative therapy to those suffering from rare diseases, but.
Before we discuss our financial results I will turn the call over to Rick to make a few comments about our announced CFO succession.
Thank you John and before we proceed of first I wanted to say a few words about cross well earned retirement.
As we announced on April the 20th factor Distinguished 40 year career as a financial executive Karl will be retiring effective on July 4th.
And we want to thank him for his service of them of pharma and we want to congratulate congratulate Lorie law currently our senior VP for finance and accounting and Carl's very capable deputy will be ex exceeding Carl as the CFO upon his retirement.
So I'll now return the call over to Lori Huawei to discuss financial results for the first quarter of 2021 Laurie.
Thanks, Rick and Luke.
For it to working with the team and engaging with investors and my new role as Chief Financial Officer. We ended the first quarter with cash and cash equivalents totaling $114 1 million compared to $98 7 million on December 31st 2020, as Rick mentioned earlier, our current cash position includes the receipt in January of the final 26 million.
Tranche from the sale of our priority review voucher, we expect and average cash use and approximately $8 million to $9 million per quarter through 2020, one and expect our current cash on hand at the port operations through or growth to 10 readout and completion of the oil growth of 212 trial net.
Loss for the first quarter was $8 6 million compared to the net income of <unk> 3 million for the same period and 2020 now.
Now I would like to turn the call back over to Rick before we open up for question Greg.
As you can tell we're excited about our advancement of our ongoing trials and PGA scale and look forward to the initiation of our of our PK PD or growth of $2 12 trial.
We are on solid financial footing to execute on our strategy and believe that our clinical trials will confirm the potential from them 200 want to disrupt the PGA seed market we.
We look forward to continuing to provide updates as we progress and wood.
That will open the call for questions operator.
As a reminder to ask a question you will need to pause for one on the telephone keypad again that inspire one on your telephone keypad domains on your question cash flow.
The standby, while we compile the Q&A roster.
Yeah and France.
Sure and comes from the line of Charles Duncan from Cantor Fitzgerald. Your line is now open.
Hi.
Thanks for taking my question and juggling and lots of calls and thanks, John for all of the detailed information and I had a couple of quick questions.
One is on the overall gross to net.
50% of sites were activated can you give us.
A sense of the enrollment.
Activity and and then perhaps screen to enrollment ratio any any early perspectives on that.
Chaz, we haven't given any guidance for the market and in terms of debt update.
These trials.
Usually start of certain pace, but as the more patients are screened and some of the more active sites and I would say experienced sites and.
Enrolment of improved dramatically and I think debt.
We're in that stage now, but we haven't given any guidance.
The two questions you ask.
Okay, Okay and.
And then with regard to the Pea and there isn't a lot of.
The good information that you presented here recently and I'm wondering if you've had any feedback from the FTE on the strategy.
And if you can provide any color on that with regard to their I guess comfort level with this forming the basis of the then.
Given the Nike and aster and out of the face and B.
And John why don't you take that question.
Yes, so we've engaged with the FDA from from the beginning about this idea of because it is really the basis for how we think this product is going to move forward rate selecting the correct patients and giving them the.
The correct dose is really how this molecule is kind of show itself to be effective. So yes, we have engaged and I think we.
We need to bring them the results from this trial, where we're applying the pms prospectively.
To go to go any further with the with the idea that we brought to them.
Okay. That's helpful. And then one last question and then I'll hop back in the queue and recurring.
And pulsatile growth hormone release, and the PGM strategy.
Thanks, Ed and could go beyond call it of risk navigator with regard to clinical trial.
But as a result in better outcomes are more effective.
Gross stimulation and per.
Perhaps even.
Slow or shorter duration of dosing.
And then given the mechanism with regard to stimulating release versus a bolus dose do you think that the outcome measures could actually do better and that's the basis of that.
Go ahead John.
So there are some preclinical experiments, let's say given the same amount of growth hormone.
Pulsatile of fashion versus the continuous <unk>.
<unk> door for ads grow better with pulsatile release of our administration of growth hormone.
So I think of the potential is there for the.
The importance of the peak to nadir.
Pulsatile 80, and the release of growth on that to really show its effect in the study it's never really been looked at before because there hasn't been a way to pulse 23 to 25 pulses of growth hormone across the day in and individual child, and then look at their growth and six months, but I think this the study will really get.
At that rate and a group of patients that we think we have selected to be responsive. So we'll have a lot of information.
And I think that's a very good potential to show that this unique mechanism of action will give us quite of bit of growth.
While maintaining growth of on and IGF one levels within the physiological ranges.
Thanks for the added color and thanks for.
Providing all of the information.
Thank you Jess.
Your next question comes from the line of alumina.
And I'll come and SKU from Jones trading your line is now open.
Hi, good afternoon, and thank you for taking my question and congrats on the quarter.
So I guess you the previously mentioned and nothing of congratulate Mark that you are looking to expand boom Hill, one and two.
The additional indication.
So I was hoping maybe you can help us understand what the portion of patients for each of the indication of what you're looking for my.
How should be P M positive and response to the until one.
Thank you.
Yes.
Well, we continue to engage the KOL community and the experts and each one of these diagnoses.
And we are and the process of determining just that type of question Jon do you want to add any additional color.
Yeah. So the the data all of the data we have now.
On kids of North of a deficiency of our kids with pediatric growth hormone deficiency alright. So.
We've done some thinking and extrapolation, but were really the data that we have points to the PTH D population and I think each one of the the other indications has some unique facets to its etiology and where the defects are some are more.
The combination of several events others are are are related to growth of inefficiency pretty directly so it's going to depend on each of the different disease indications and it'll depend on us acquiring a little bit more knowledge about our molecule and how it affects our growth and the 210 trial before we really of.
And can put our put our hand on it and understand broadly for some of the secondary indications how many response of patients there maybe.
Got it just just one.
And one follow up on debt do you think you might need to.
And you adjust your P. M classification based on each indication based on the debt.
And the Caddo for that.
Yes.
For the John go ahead.
Okay.
We'll have to see but I think you know and the and this set of criteria.
As you saw for identifying patients with with the growth hormone with us with the partially functioning axis to produce growth hormone and thats kind of the first step in selecting patients that are going to be responsive to our molecule right.
Some of the other indications.
You do have to think about whether some of the the.
Deficits are downstream of growth hormone production rate like some of them you might have a deficit of growth hormone receptor interactions right.
And so we have to put all of these pieces together and.
And think through what's the best approach and what kind of dose range do we have access to and our and the information we get out of the growth from a deficiency of the PTH day trial, So I think.
There's a lot of really interesting data that's out there for these patients being treated with just for covenant human growth hormone or we can we can build on what's known about the ideology and think and.
Find the best fit for for our molecule to go after the secondary indications.
Got it well. Thank you so much and again congrats on the quarter and al.
Back in the queue.
Thank you yes.
Your next question comes from the line of Gerrick, our Chiller promise Stifel. Your line is now open.
Hey, guys.
Good afternoon. This is <unk> on for Derik, Thanks for fitting of thin here and taking our questions.
The zooming out can you talk about how you view adherence for an oral secretagogue versus weekly growth hormone injections.
Some of the pros and cons there.
Yeah.
Let me start with an answer.
<unk>.
And there isn't so much data out there that any injectable product.
And would it be and P J C or other indications.
And that's problematic and and a pediatric population in terms of adherence.
I think of the longer acting products will improve that but.
And we've done some direct market research that shows that debt.
When asked both caregivers.
And clinicians, which when they would prefer and that is.
A weekly injection oral once a day oral is our product is.
The overwhelmingly choose and oral product.
How that translates to.
Of compliance and.
The real world.
And as another question I think we have to the study some more.
But we believe that it's definitely going to be a preferable treatment and we believe that there is a nerf attention by the by the parents.
This is that theyre going to stay on their children and to make sure of that they take debt. This once a day oral product as they should.
John do you have anything to add to that.
No that's perfect. Thank you.
Got it that's helpful. And then if I may on the BD front can you speak too punitive licensing licensing opportunities and the outcome.
And how competitive that is right now to find and asset and.
And what indications and modalities are you guys thinking about.
Thanks.
Good question, Jack and I can tell you we have a robust process that has been underway.
For quite some time that is led by Aaron shoe heart, our CPO and Aaron has 25 or more years of experience as the.
The business officer with both the large companies and small companies and the.
Our rare disease space and.
In addition of that I think we have a <unk>.
Collectively as a team.
And many decades of experience of operating and this rare disease space and as a result, those contacts have been very productive and bringing forth a number of interesting opportunities that we are once again just actively.
Reviewing and making sure that we make the right choice here.
Great.
Very helpful. Thanks, and congrats on the quarter guys.
Thank you John.
Thank you I'm showing no further questions in the queue at this time on hand.
On the call back to Mr. Hawkins for closing remarks.
Okay.
We thank you for joining us today, and we look forward to keeping everyone apprised of our program over the course of the year really appreciate your time today.
This concludes today's conference call. Thank you for participating you may now all disconnect.
Yes.
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